Press Releases

20.07.2022

EffRx Pharmaceuticals announces the launch of Alkindi^® as replacement therapy for pediatric adrenal insufficiency in Switzerland

FREIENBACH, Switzerland – EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announces the launch of Alkindi® (hydrocortisone capsules for oral use) in Switzerland as a replacement therapy for adrenal insufficiency in infants, children and adolescents (from birth to <18 years old).

Alkindi® is the first approved pediatric-specific hydrocortisone formulation for the replacement therapy for adrenal insufficiency (AI) in pediatric patients. Alkindi® is available in color-coded capsules with four different dose strengths (0.5 mg, 1 mg, 2 mg, 5 mg) and allows for individualized, accurate and flexible dosing – without the need for magisterial formulations or tablet fragmentation.

Pediatric AI, including the related genetic condition congenital adrenal hyperplasia (CAH), is a condition characterized by deficiency in cortisol. Untreated, the disease is associated with significant morbidity and increased mortality. In Switzerland, there are approximately 200 patients suffering from pediatric AI. This condition in children is currently treated by compounding hydrocortisone or crushing/splitting tablets of adult preparations with the potential risks of lack of efficacy and/or increased side effects.

Dr. Lorraine Zakin, Senior Director Medical Affairs at EffRx commented: “I am very pleased that this medicine, specifically studied in children and designed to treat children with adrenal insufficiency, is available in Switzerland. Alkindi® addresses the shortfall in pediatric-specific medicines.”

Alkindi® advances the therapeutic options to help young patients suffering from pediatric AI and CAH and provides the first regulated, consistent preparation of hydrocortisone to ensure efficacy, safety and ease of use for children suffering from AI and their caregivers.

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About Pediatric Adrenal Insufficiency (AI) and congenital adrenal hyperplasia (CAH)

Pediatric AI and the related genetic condition CAH are characterized by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue, risk of adrenal crisis and death in case of inadequate cortisol replacement.

About Alkindi®

Alkindi^® is the first preparation of hydrocortisone specifically designed for use in children suffering from pediatric AI. Alkindi® is a patented, oral, immediate-release pediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate, yet flexible and personalized dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from pediatric AI and the related condition CAH.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late-stage development and commercialization of medicines for niche and orphan indications. The business model is centered around providing superior clinical value propositions for physicians, payers and patients.

EffRx pro-actively seeks in-licensing opportunities for Switzerland and Europe in niche therapeutic areas, with a primary interest for rare diseases. EffRx’s go-to-market competence is proven by the development, launch and expansion of Binosto® in highly competitive European markets as well as US, selected MEA and Asian countries.

12.05.2022

World Orphan Drug Alliance – combining forces to reach patients with rare diseases across the globe

World Orphan Drug Alliance (WODA) is a new global alliance of full-service regional distributors for orphan drugs.
WODA covers 68 countries on 4 continents.
There are more than 64 million potential patients with rare diseases in the WODA regions.

Zurich, Ljubljana, Moscow, Sao Paulo, Dubai, May 12, 2022 – EffRx Pharmaceuticals, Medis, Orpharm, OrphanDC, and Vector Pharma, announced today the newly formed World Orphan Drug Alliance (WODA). WODA aims to provide comprehensive support to pharmaceutical and biotech companies with rare diseases, oncology and highly specialized therapeutics portfolio, starting from named patient programs through to full commercialization.

By bringing together experts for rare diseases, WODA has capabilities to provide all the services necessary for orphan medicines to reach patients in need, replacing local branches of global distributors or multinational companies.

“Under WODA alliance we are offering future partners the opportunity to cover our entire territory or cherry pick the territories they require, while using local teams in each territory with the local knowledge and skill set in the therapeutic area we cover,” commented Vector Pharma Managing Director Samer Semaan. “Due to our flexible setup, we have the ability to adjust to partners’ needs instantaneously and provide a tailored solution.”

In total, WODA covers 68 countries in the following regions: Turkey, Middle East and North Africa, Russia and CIS region, Central and Eastern Europe, Latin America and Switzerland.

“WODA focuses on the markets, that are complex and often overlooked by big pharmaceutical and biotech companies. However, WODA territories provide both, a pool of patients in need and potential funding opportunities for rare and specialized diseases. There are potentially more than 64 million rare disease patients in the WODA regions,” adds Martina Perharič, Medis CEO.

About Founding Partners:

EffRx Pharmaceuticals is a Switzerland based company focused on the late-stage development and commercialization of prescription medications for niche and orphan indications.
Medis based in Slovenia is the commercialization partner of choice for innovative pharmaceutical and biotech companies seeking strong business growth in Central and Eastern Europe.
Orpharm is a Moscow based full-service distributor covering Russia and the Commonwealth of Independent States (CIS).
OrphanDC based in Sao Paolo, Brazil acts as a partner for biotech companies in Latin America. They focus on supporting their clients from the clinical development stage throughout the product lifecycle.
Vector Pharma is a Dubai based full-service distributor covering Middle East, North Africa and Turkey.

More about WODA: www.woda-alliance.com

26.04.2022

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Efmody^® in Switzerland

FREIENBACH, Switzerland –-BUSINESS WIRE–- EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced it has recently entered into an exclusive license agreement with Diurnal Group plc, for the registration and commercialization of Efmody^® as treatment for congenital adrenal hyperplasia (CAH) in Switzerland. Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Efmody^® in Switzerland.

Efmody^® is a modified-release preparation of hydrocortisone that has been specifically designed for the treatment of patients with CAH, a rare condition caused by a genetic deficiency of adrenal enzymes. According to our estimates, there are approximately 450 patients in Switzerland suffering from CAH.

EffRx intends to submit a Market Authorisation Application (MAA) to Swissmedic as treatment for adolescent and adult patients (12 years and older) with the rare condition congenital adrenal hyperplasia (CAH) in Switzerland during the second half of 2022. The MAA submission to Swissmedic for Efmody^® will be based on the European regulatory dossier and published clinical trial data, with EffRx expecting potential market launch in Switzerland in 2024.

“We are excited to sign this additional agreement with Diurnal, enabling us to build on the momentum we have achieved with Alkindi^® in Switzerland following approval from Swissmedic in November 2021. We believe the unique release profile of Efmody^®, that mimics the body’s natural cortisol circadian rhythm, could have a genuine impact on CAH patients’ symptoms. We are aligned with Diurnal’s strategy to address the unmet medical need in patients suffering from diseases of cortisol deficiency and look forward to working with the Diurnal team to bring Efmody^® to patients suffering from CAH in Switzerland”, Lorenzo Bosisio, Chief Executive Officer of EffRx, commented.

In May 2021, Efmody^® was granted marketing authorisation in the European Union and was subsequently launched in Germany, Austria and the UK in September 2021. “We are pleased to deepen our relationship with EffRx to include the distribution and marketing of Efmody^® in Switzerland. We have been impressed by the progress EffRx has made with the regulatory approval and reimbursement of Alkindi^® and look forward to continuing to work with them as they prepare to submit an MAA to Swissmedic for Efmody^®”, Richard Bungay, Interim Chief Executive Officer of Diurnal, commented.

Media contact: Ms. Pamela Saredi, psaredi@effrx.com

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About congenital adrenal hyperplasia

Congenital adrenal hyperplasia (CAH) is an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase. This enzyme is required to produce the adrenal steroid hormone, cortisol. The block in the cortisol production pathway causes the over-production of male steroid hormones (androgens), which are precursors to cortisol. The condition is congenital (inherited at birth) and affects both sexes. The cortisol deficiency and over-production of male sex hormones can lead to increased mortality, infertility and issues during sexual development including ambiguous genitalia, premature (precocious) sexual development and short stature. Sufferers, even if treated, remain at risk of death through an adrenal crisis.

Current therapy for CAH uses a variety of generic glucocorticoid (steroid) preparations including hydrocortisone, dexamethasone, prednisolone and prednisone in the US, with no standard treatment regimen. Approximately two-thirds of CAH patients are estimated to have poor disease control, leading to elevated androgen levels. The condition is estimated to affect a total of approximately 16,000 patients in the US, with over 400,000 in the rest of the world.

About Efmody^® (hydrocortisone modified-release hard capsules)

Efmody^® is a preparation of hydrocortisone that has been specifically designed to mimic the circadian rhythm of cortisol when given in a twice-a-day “toothbrush” regimen (administered last thing at night before sleep and first thing in the morning on waking) to control androgen excess and chronic fatigue in patients with diseases of cortisol deficiency. The first indication for Efmody^® is congenital adrenal hyperplasia (CAH) in adults and adolescents (children older than 12 years of age). Efmody^® has been extensively studied in 239 human subjects including 138 CAH patients who have taken part in clinical trials in Europe and the US.

The MHRA and European Commission marketing authorisation approval of Efmody^® was based on a Phase 3 study conducted in a total of 122 patients enrolled across 11 clinical sites, including sites in Great Britain, the largest ever interventional clinical trial completed in CAH. The Phase 3 data was supported by detailed analysis of data from an open-label safety extension study for patients completing treatment in the Phase 3 study, which is assessing the impact of treatment with Efmody^® over an extended period, with a number of patients on this trial having been treated for over five years. Summary of Product Characteristics (SmPC) for UK (Northern Ireland) can be found here.

About EffRx Pharmaceuticals

EffRx pro-actively seeks in-licensing opportunities for Europe in niche therapeutic areas, with a primary interest in rare diseases, where EffRx has received an orphan drug designation (ODD) from the FDA. EffRx’s go-to-market competence is proven by the development, launch and lucrative expansion of Binosto® in a highly competitive European market. Our lead commercialized product, Binosto^® for the treatment of osteoporosis, is marketed in the US as well as selected European and Asian countries.

About Diurnal Group plc

Diurnal Group plc is a European, UK-headquartered, specialty pharmaceutical company dedicated to developing hormone therapeutics to aid lifelong treatment for rare and chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency, hypogonadism and hypothyroidism. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

For further information about Diurnal, please visit www.diurnal.com

16.11.2021

EffRx Obtains Swiss Marketing Authorization for Alkindi^® for Paediatric Adrenocortical Insufficiency

FREIENBACH, Switzerland –-BUSINESS WIRE— EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Alkindi^® for the treatment of paediatric adrenocortical insufficiency (AI).

Alkindi^®, developed by Diurnal Group plc, is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric adrenocortical insufficiency (AI). Alkindi^® is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from paediatric AI and the related condition congenital adrenal hyperplasia (CAH).

Alkindi^® is approved and marketed in the European Union and is the first preparation of hydrocortisone specifically designed for use in children suffering from AI. On September 29^th, 2020 the US Food and Drug Administration (FDA) has also approved Alkindi^® for AI. This new treatment approach is expected to be available on the Swiss market by H1 2022. In Switzerland there are approximately 200 patients suffering from paediatric AI.

The marketing authorisation approval in Switzerland is based upon a pivotal open-label Phase III clinical trial conducted in 24 children, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported.

“We are delighted to have received Swissmedic approval for Alkindi^®. This approval is a major breakthrough for paediatric patients with adrenal insufficiency, with Alkindi^® being the first licensed treatment in Switzerland specifically designed for use in infants, children and adolescents (from birth to less than 18 years old)” commented Lorraine Zakin, Senior Director Medical Affairs at EffRx Pharmaceuticals.

Paediatric AI and the related genetic condition CAH is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue and patients are at risk of adrenal crisis and death if they do not have adequate cortisol replacement. AI is either primary or secondary, with primary AI resulting from diseases intrinsic to the adrenal gland and secondary AI resulting from pituitary diseases where there is a failure of stimulation of the adrenal by the pituitary of the signalling hormone ACTH (adrenocorticotropic hormone).

About EffRx Pharmaceuticals SA

EffRx pro-actively seeks in-licensing opportunities for Europe in niche therapeutic areas, with a primary interest for rare diseases, where one of its pipeline assets has received an orphan drug designation (ODD) from the FDA in US.

About Diurnal Group plc

Founded in 2004, Diurnal is a UK-headquartered, European specialty pharma company developing high-quality products for the global market for the life-long treatment of chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency and hypogonadism. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

Media contact: Ms. Pamela Saredi, psaredi@effrx.com

04.11.2021

EffRx Obtains Swiss Marketing Authorization for Bronchitol^® in Cystic Fibrosis

FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced that Swissmedic has approved Bronchitol^® (inhaled mannitol) for the treatment of cystic fibrosis (CF) in adults and in children aged 6 years and above as add‐on to other medicines.

Bronchitol^® is a precision spray‐dried form of mannitol that is delivered to the lungs by a specially designed, portable inhaler. Bronchitol^® improves lung function and helps to clear mucus from the lungs in patients suffering from cystic fibrosis. Patients are required to pass a tolerance test prior to being prescribed Bronchitol^® to ensure that they are not hyperresponsive to mannitol.

Bronchitol^® is currently marketed in Europe, Russia, Australia and the United States and was developed by Pharmaxis Ltd, a listed pharmaceutical research company in Australia. The clinical program supporting the registration of Bronchitol^® consisted of three large-scale global clinical trials and enrolled a total of 1,065 subjects. Bronchitol^® use led to a sustained improvement in FEV1 (Forced Expiratory Volume) versus control. The statistically significant improvement in FEV1 was observed over the 26-week treatment period in those patients receiving Bronchitol^® when compared to patients in the control group. The most common (≥1/100, <1/10) adverse reactions include cough, hemoptysis, oropharyngeal pain, vomiting, wheezing and headache.

“We’re excited to have achieved this important milestone and that patients living with cystic fibrosis will be able to benefit from this treatment in Switzerland. Bronchitol^® offers a portable and discreet option for CF management, with no routine cleaning or maintenance of the inhaler device required,” said Lorraine Zakin, Senior Director Medical Affairs at EffRx Pharmaceuticals.

Cystic fibrosis (CF) is an inherited, life-limiting disease that affects the body’s exocrine glands, which produce mucus, saliva, sweat and tears. In the lungs of a CF patient, the thick mucus and the thinning of the airway surface liquid make it nearly impossible for the cilia to clear bacteria from the airway. According to the Swiss Society for Cystic Fibrosis (CFCH), around 320,000 Swiss people are carriers of an altered gene that can cause CF (approximately one in 25 people)[1].

EffRx expects the availability of Bronchitol^® in Switzerland in the second half of 2022.

[1] Was ist Cystische Fibrose | Cystische Fibrose Schweiz

About EffRx Pharmaceuticals

EffRx pro-actively seeks in-licensing opportunities for Switzerland and Europe in niche therapeutic areas, with a primary interest for rare diseases, where EffRx had received an orphan drug designation (ODD) from the FDA.

About Pharmaxis

Pharmaxis Ltd is an Australian pharmaceutical research company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue. Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia.

22.06.2021

A Prospective Study in More Than 1,000 Osteoporosis Patients Concluded That Binosto^® (Buffered Soluble Alendronate) May Increase Patient Satisfaction, Long-Term Adherence and Therefore Efficacy

FREIENBACH, Switzerland–(BUSINESS WIRE)–

Corporate IR Press Release

In a newly published international multicenter study¹ in over 1,000 osteoporosis patients treated with Binosto^® and followed for 12 ± 3 months,

Binosto was well tolerated
Treatment persistence was high
Mean overall compliance was high too
Patient satisfaction remained at approximately 90%

EffRx Pharmaceuticals SA, a commercial-stage pharmaceutical company that commercializes niche and orphan medicines in Switzerland and Europe, announces the full paper publication of the GastroPASS Study Results of Binosto^® (buffered soluble alendronate) in JBMR^® Plus, a premier peer-reviewed medical journal. Prof. Salvatore Minisola will present the study results as an oral communication (OC 19) at the WCO-IOF-ESCEO² virtual congress on August 28, 2021 at 11:40 am.

GastroPASS was a prospective, observational, multicenter, multinational, single-arm, post-authorization safety study. The study enrolled 1,084 postmenopausal women with osteoporosis, newly prescribed Binosto^® and naïve to bisphosphonate therapy. At baseline, 31% of enrolled patients reported a medical history of gastrointestinal (GI) tract conditions that did not exclude study participation. Patients were followed for 12 ± 3 months. The primary objective was to evaluate the incidence of upper GI adverse events and medication errors during treatment. Secondary aims were to evaluate persistence, discontinuation and reasons for discontinuation.

The cumulative incidence of all upper GI AEs related to Binosto^® observed during the duration of the study was low at 9.6%. The study authors stated that “the incidence of upper GI AEs in this “real-world” study were found to be lower than that observed in alendronate-treated groups from randomized controlled trials with a duration of 12 months.” Also, the incidence rate of individual gastric AEs related to Binosto was low (ranging from 0 to 2.1 per 1,000 patients/month) and decreased over the three follow-up periods of the study. No events for gastric ulcer, gastric perforation, gastric hemorrhage, and gastric stenosis were observed.

Treatment persistence reported in this study was high at 79.7% at 12 ± 3 months. These results are well in line with the recently published findings by Giusti et al.³ Mean overall compliance was high, too, at 92.8 using the Morisky-Green questionnaire and 94.8 based on the number of tablets missed.

Patient satisfaction and preference were also high, at approximately 90%, both in terms of how easy it was to take Binosto^® and how much easier it was to take Binosto compared with other medications.

“Prof Minisola et al have conducted an interesting study. Results are interesting and needed, considering that alendronate is still a very important medication. (…) Oral bisphosphonates are considered the first line treatment in patients after fragility fractures. The present data are encouraging in terms of low prevalence of GI side effects. (…) To prescribe medications with low rate of side effects is important also during the pandemic, given the restrictions and limited access to osteoporosis clinics.”, commented one of the independent reviewers of JBMR Plus.

Overall, post-menopausal women with osteoporosis treated with Binosto^® (buffered soluble alendronate) in a real-world setting experienced few upper GI AEs. In addition, they had a low discontinuation and high compliance compared to other formulations, suggesting that buffered soluble alendronate may increase patient satisfaction and therefore long-term adherence and efficacy.

“Based on my experience as the PI and first author of the pivotal trial on alendronate, a first-line treatment in osteoporosis, I believe that this study was needed and the results are relevant to provide good evidence for this alternative formulation of alendronate. The low incidence of upper GI AEs, the low discontinuation rate and the high compliance observed in the study reinforce previous clinical data regarding Binosto. These results offer confidence that Binosto could lead to increased patient satisfaction and therefore better long-term adherence and efficacy than pill forms.” commented Prof. Dennis M Black, study co-author.

¹The full paper publication can be found at: JBMR^® Plus

² World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases

³Giusti A et al. A novel effervescent formulation of oral weekly alendronate (70 mg) improves persistence compared to alendronate tablets in post-menopausal women with osteoporosis. Aging Clin Exp Res 2021; https://doi.org/10.1007/s40520-020-01777-9.

About JBMR^®Plus

JBMR^® Plus is the premier open access journal of the American Society for Bone and Mineral Research (ASBMR). A companion to internationally recognized title Journal of Bone and Mineral Research, JBMR® Plus aims to improve global musculoskeletal health by publishing innovative research covering endocrinology, geriatrics, orthopedics and rheumatology.

About EffRx Pharmaceuticals

EffRx’s go-to-market competence is proven by the development, launch and successful expansion of Binosto^® in highly competitive markets.

EffRx pro-actively seeks in-licensing opportunities for Europe in niche therapeutic areas, with a primary interest for rare diseases, where EffRx has received an orphan drug designation (ODD) from the FDA for a pipeline asset.

About Binosto^®

Binosto^® (Buffered Soluble Alendronate) is a first-line treatment of postmenopausal osteoporosis patients that is associated with preferable pharmacologic properties translating into clinical benefit for patients. Alendronate as recommended by guidelines is a first line treatment for postmenopausal women with osteoporosis. Alendronate 70 mg tablet once weekly is the most commonly used bisphosphonate but adherence is problematic, with >50% discontinuing treatment within the first year. To address this, Binosto^®, a novel buffered soluble alendronate 70 mg effervescent tablet formulation was specifically developed to improve GI tolerability and adherence to treatment.

21.10.2020

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Alkindi^®and Submits Market Authorization Application in Switzerland

FREIENBACH, Switzerland (BUSINESS WIRE)-– EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced it has recently entered into an exclusive license agreement with Diurnal Group plc, a specialty pharmaceutical company targeting patient needs in chronic endocrine diseases, for the registration and commercialization of Alkindi^® for pediatric adrenocortical insufficiency (AI) in Switzerland. Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Alkindi^® in Switzerland.

Alkindi^® is the first preparation of hydrocortisone specifically designed for use in children suffering from paediatric adrenocortical insufficiency (AI). Alkindi^® is a patented, oral, immediate-release paediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate age-appropriate dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from pediatric AI and the related condition congenital adrenal hyperplasia (CAH).

Alkindi^® is already approved and marketed in the European Union and is the first preparation of hydrocortisone specifically designed for use in children suffering from AI. On September 29^th, 2020 the US Food and Drug Administration (FDA) has also approved Alkindi® for AI.

EffRx has recently submitted to Swissmedic (Switzerland) a Market Authorization Application for the registration of Alkindi®. Pending successful regulatory registration, this new treatment approach is expected to be available on the Swiss market by 2022. In Switzerland there are approximately 200 patients suffering from pediatric AI.

“We are extremely pleased to announce this partnership with Diurnal which demonstrates EffRx capability to expand its portfolio with promising niche and orphan medicines”, commented Lorenzo Bosisio, CEO of EffRx Pharmaceuticals. “We look forward to bringing this novel therapeutic approach to Switzerland. We are confident that Alkindi® provides a tangible advancement for young patients suffering from AI and their carers.”

Martin Whitaker, CEO of Diurnal, commented: “This partnership with EffRx further validates the quality of our products and broadens the future availability of Alkindi®. EffRx is well-placed to register and market our product Alkindi® in Switzerland. We have made strong progress with the sales of Alkindi® across Europe since its approval and subsequent launch in 2018, and we are confident this agreement will enable further growth.”

Pediatric AI, including the genetic condition CAH is a condition characterised by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue and patients are at risk of adrenal crisis and death if they do not have adequate cortisol replacement. AI is either primary or secondary, with primary AI resulting from diseases intrinsic to the adrenal gland and secondary AI resulting from pituitary diseases where there is a failure of stimulation of the adrenal by the pituitary of the signalling hormone ACTH (adrenocorticotropic hormone).

About EffRx Pharmaceuticals

EffRx’s go-to-market competence is proven by the development, launch and lucrative expansion of Binosto® in a highly competitive European market. Our lead commercialized product, Binosto^® for the treatment of osteoporosis, is marketed in the US as well as selected European and Asian countries.

About Diurnal Group plc

Founded in 2004, Diurnal is a UK-headquartered, European specialty pharma company developing high quality products for the global market for the life-long treatment of chronic endocrine conditions, including congenital adrenal hyperplasia, adrenal insufficiency and hypogonadism. Its expertise and innovative research activities focus on circadian-based endocrinology to yield novel product candidates in the rare and chronic endocrine disease arena.

For further information about Diurnal, please visit www.diurnal.co.uk

Media contact: Ms. Pamela Saredi, psaredi@effrx.com

13.6.2019

EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Commercialization of Bronchitol^®in Switzerland

FREIENBACH, Switzerland–(BUSINESS WIRE)– EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announced it has entered into an exclusive license agreement with Pharmaxis Ltd, a listed pharmaceutical research company in Australia, for the registration and commercialization of Bronchitol^® (mannitol) for cystic fibrosis in Switzerland.

Under the terms of the agreement, EffRx will take responsibility for registering, obtaining pricing and reimbursement as well as commercializing Bronchitol^® in Switzerland. Bronchitol^® works by rehydrating the airway/lung surface and promoting a productive cough, hence improving mucus clearance and lung function. The product is inhaled as dry powder twice daily using a small handheld device and it is expected to be available on the Swiss market by 2021.

Cystic fibrosis is a hereditary rare disorder affecting the exocrine glands. It causes the production of abnormally thick mucus, leading to disease manifestations primarily in the lungs, but also in pancreas, intestines, liver and kidneys. Cystic fibrosis patients often suffer from frequent respiratory tract infections. According to the Swiss Society for Cystic Fibrosis (CFCH), there are about 1,000 people affected by cystic fibrosis in Switzerland. While there is no cure for the disease, thanks to medical advances, a majority of cystic fibrosis sufferers today reach adulthood.

“We are extremely pleased to announce this exclusive licensing agreement for Bronchitol^®. We look forward to making this novel therapeutic option accessible to Swiss patients suffering from cystic fibrosis”, commented Lorenzo Bosisio, CEO of EffRx Pharmaceuticals. “We are encouraged by the clinical data and experience with Bronchitol^® which show that this convenient treatment is well-tolerated and can lead to improvements in lung function.”

Gary Phillips, CEO of Pharmaxis said, “Our team is delighted to have entered this collaboration with EffRx. We are keen to leverage the go-to-market expertise of EffRx to bring Bronchitol^® to the Swiss market and are pleased to secure in EffRx an experienced commercial partner.”

This licensing agreement underpins EffRx strategy to expand its portfolio through licensing collaborations in Europe and Switzerland with innovative biopharmaceutical companies developing promising niche and orphan medicines.

Bronchitol^® has been the subject of three large-scale, global clinical trials conducted by Pharmaxis. It is approved and marketed in Europe, Russia, Australia and several other countries. In the United States, Bronchitol^® has recently received a positive recommendation from a Committee advising the US Food and Drug Administration (FDA) on the use of Bronchitol^® for adult cystic fibrosis patients; with the FDA expected to advise its decision within the next few weeks.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late-stage development and commercialization of prescription medications for niche and orphan indications. The business model is centered around providing superior clinical and commercial value propositions for physicians, payers and patients. EffRx aims to be the preferred accelerator for late clinical and regulatory development, approval, valorization of niche and orphan medicines in Switzerland and Europe.

EffRx’s go-to-market competence is proven by the development, launch and lucrative expansion of Binosto® in a highly competitive European market. Our lead commercialized product, Binosto^® for the treatment of osteoporosis, is licensed in 50+ countries and marketed in the US, Europe, selected MENA and Asian countries.

About Pharmaxis

Pharmaxis (ACN 082 811 630) is an Australian pharmaceutical research company focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval. Its product Bronchitol^® for cystic fibrosis is marketed in Europe, Russia and Australia. Its product Aridol^® for the assessment of asthma is sold in the United States, Europe, Australia and Asia. The company’s development pipeline is centered on its expertise in amine oxidase chemistry and includes a series of Lysyl Oxidase Inhibitors under clinical development targeting fibrotic diseases of the heart, kidney, liver and lung. Pharmaxis is listed on the Australian Securities Exchange (symbol PXS). The company’s head office, manufacturing and research facilities are located in Sydney, Australia. For more information about Pharmaxis, please see www.pharmaxis.com.au

Media Contact: Pamela Saredi, psaredi@effrx.com

2.4.2019

EffRx to Showcase Binosto^® at the WCO-IOF-ESCEO 2019 in Paris

FREIENBACH, Switzerland–(BUSINESS WIRE)– EffRx Pharmaceuticals SA is proud to announce that it will participate in the 19th Edition of the World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases, taking place April 4-7, 2019 in Paris. The WCO-IOF-ESCEO Congress is the world’s leading clinical conference on bone, joint and muscle health and therefore the ideal venue for EffRx to showcase its flagship product Binosto^®, the only buffered soluble alendronate for the treatment of postmenopausal osteoporosis.

EffRx Pharmaceuticals SA is also pleased to inform that new safety data on Binosto^® will be presented at the WCO-IOF-ESCEO Congress. The data will be presented as a poster titled “Upper Gastrointestinal Safety with the Buffered Solution of Alendronate 70 mg: 6 Years of Post-Marketing Experience” authored by P. Fardellone, B. Boëzennec and B. Cortet on Saturday, 6^th of April 2019, from 14:00 to 15:00 (poster #579).

The authors of the new Binosto^® safety data conclude that Binosto^® is associated with a lower frequency of upper gastrointestinal adverse reactions than reported for alendronate tablets and that it is a well-tolerated oral bisphosphonate for the management of osteoporosis.

“These updated data reinforce the safety profile of Binosto^® after 6 years of post-marketing experience,” says Dr Lorraine Zakin, Medical Affairs Director at EffRx Pharmaceuticals.

Medical professionals from around the world will be able to learn more about Binosto^® and its exciting properties at the Binosto^® booths 20 and 21 in the exhibition area of the Palais des Congrès de Paris.

EffRx looks forward to welcoming you to the Binosto^® booth at the WCO-IOF-ESCEO Congress in Paris!

About EffRx Pharmaceuticals SA

EffRx Pharmaceuticals SA is a commercial-stage functionally integrated pharmaceutical company, based in Switzerland, that develops and commercializes niche & orphan medicines in Europe. EffRx aims to be the preferred accelerator for late clinical & regulatory development, approval, valorization of niche & orphan medicines in Europe and Switzerland.

About Binosto^®/Steovess^®

Our lead commercialized product, Binosto^® is indicated for the treatment of osteoporosis and marketed in US, Europe and Asia. Binosto^® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of postmenopausal osteoporosis. It reduces the risk of hip, spine, and non-spine fractures.¹ The buffered solution with high acid-neutralizing capacity minimizes the risk of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate.^2,3 Binosto^® is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto^® has the potential to improve adherence to therapy⁴, thereby decreasing the risk of disabling fractures.

Disclaimer

Please refer to the official Binosto^®/Steovess^® prescribing information and approved indications, contraindications, and warnings in your country.

References:

Black DM et al. Fracture Risk Reduction with Alendronate in Women with Osteoporosis: The Fracture Intervention Trial. J Clin Endocrinol Metab 2000;85:4118-4124.
Binosto Summary of Product Characteristics. 2017.
Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. Int J Pharm 2012;432:57-62.
Giusti A et al. Persistence with the buffered solution of alendronate 70 mg: prospective observational study. Osteoporos Int 2018;29(S1):S431.

Media Contact: Pamela Saredi, psaredi@effrx.com

14.11.2018

EffRx Announces Expansion of the Executive Team

FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA, a commercial-stage pharmaceutical company focused on niche therapeutics in Europe today announced the recent expansion of the Executive Team with Lorraine Zakin, MD, as Director, Medical Affairs, Peter Lichtlen, MD, PhD as Chief Medical Advisor, Christiane Sievert as Director, Regulatory Affairs and Victor Papavasileiou as Vice President, Business Development.

Lorenzo Bosisio, CEO of EffRx, commented, “We are thrilled to have attracted such talent and experience to drive the further growth of our company. Over the last few years, I feel proud to have been part of the transformation of EffRx into an integrated specialty pharmaceutical company with strong competence in accessing and harnessing the European market. With the addition of this experienced team we are advancing on our path to become an accelerator for late-stage development, valorization and commercialization of niche & orphan medicines in Europe.”

Lorraine Zakin, MD, Director Medical Affairs, is an experienced Medical Affairs and Clinical Development expert. She began by specializing in general surgery and had 7 years of international hospital practice before entering the pharmaceutical industry. During her decade-long tenure in the biopharmaceutical industry, Dr. Zakin has led Medical Affairs and Clinical Development initiatives at regional and global levels across several therapeutic areas, including cardiology, oncology and women’s health.

Peter Lichtlen, MD, PhD, Chief Medical Advisor, has 20 years of biotech and pharma experience both with listed and private companies. Acting as Chief Medical Officer and Head of clinical R&D in his prior career Dr. Lichtlen has led global clinical development programs across different therapeutic areas and was responsible for international medical affairs and pharmacovigilance teams. Dr. Lichtlen has an extensive industry network and concurrently serves in board-, advisory- and leadership-roles in several EU- and US-based innovation companies.

Christiane Sievert, Director Regulatory Affairs, brings over 30 years of experience in Regulatory Affairs ranging from early to last stage development projects in orphan, specialty as well as generic indications for the EU, US and ROW. Ms. Sievert has proven, in-depth knowledge in Regulatory Affairs across international markets, first-hand experience in interacting with health authorities, as well as expertise in setting up, leading & optimizing Regulatory Affairs processes and infrastructures.

Victor Papavasileiou has more than 10 years of Business Development experience in the global pharmaceutical and biotech industry. With a background in science and engineering he moved on to Business Development and strategy, where he has worked for both start-ups and mature companies. He brings extensive experience in leading all aspects of the transactional phase. In his career he has successfully contributed to the build-up of profitable product portfolios in several therapeutic areas through M&A and strategic licensing transactions.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late stage development and commercialization of prescription medications for niche and orphan indications. The business model is centered around providing superior clinical and commercial value propositions for physicians, payers and patients. Our vision is to be the preferred and dedicated partner in Europe for the late clinical development, approval and valorization of emerging niche biopharmaceuticals.

25.10.2018

Osteoporosis Patients in Southeast Asia Get Access to Binosto^®

EffRx launches Binosto^® – buffered soluble alendronate – in Malaysia and Singapore

FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA today announced that physicians in Malaysia and Singapore treating patients with postmenopausal osteoporosis have a new therapeutic option: buffered soluble alendronate (Binosto^®). This novel therapeutic option, which is already available in fifteen countries worldwide, provides gold-standard fracture risk reduction and offers better upper gastrointestinal safety profile than reported for alendronate tablets.^1-3

In both Malaysia and Singapore, Binosto^® is marketed by Zuellig Pharma. For EffRx Pharmaceuticals SA, who has developed Binosto^®, the launch in Southeast Asia marks another important milestone. “With the addition of Malaysia and Singapore, our geographic footprint grows significantly,” says EffRx Commercial Director Michael Schmid. “We are happy to have selected a strong and renowned local partner. While growth of Binosto^® in our existing markets remains high, geographic expansion through strong local partnerships continues to be a key priority for Binosto^® to bring the its promise to patients worldwide.”

The launch of Binosto^® in Malaysia and Singapore will be followed in 2019 by launches in other Southeast Asian markets. For Zuellig Pharma, who holds the marketing rights in these countries, Binosto^® presents a very exciting opportunity to add a unique flavor to the management of osteoporosis. “With its innovative properties and compelling efficacy and safety evidence, it can be part of many physicians’ toolkit for thousands of osteoporotic patients across Asia”, says Frederick Fong, Director Marketing Excellence (Asia) at Zuellig Pharma.

Data presented at this year’s WCO-IOF-ESCEO Congress and published in Osteoporosis International suggest that Binosto^® is associated with higher persistence, i.e. better treatment adherence, than alendronate tablets.⁴ The same data also showed significantly fewer patients discontinuing Binosto^® due to gastrointestinal adverse events – confirming the favorable GI safety profile of Binosto^®.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company, which focuses on developing and commercializing prescription medications for specialty and orphan indications. EffRx actively seeks collaboration opportunities with U.S. bio-pharmaceutical companies, which are looking to commercialize their products in Europe. EffRx can act as accelerator of EU go-to-market strategies and be a spring board for an initial international set-up in Switzerland.

About Zuellig Pharma

Zuellig Pharma is one of the largest healthcare services group in Asia. We provide world-class distribution, digital and commercial services to support the growing healthcare needs in this region. The company was started almost a hundred years ago and has grown to become a US$10 billion business covering 13 markets in Asia with over 10,000 employees. Our purpose is to make healthcare more accessible. Our people serve over 320,000 medical facilities in Asia and we work with over 1000 clients including the top 10 pharmaceutical companies in the world. More recently, we launched our Zuellig Health Solutions Innovation center to develop new services and address some pressing healthcare needs in Asia. Since then, our teams have been focused on creating data, digital and disease management solutions, supporting patients with chronic conditions and helping payers manage healthcare costs. We are also making headway in using big data to inform our decisions and those of our clients, on how to optimize for greater operational accuracy and efficiency.

About Binosto^®

Binosto^® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of postmenopausal osteoporosis. It reduces the risk of hip, spine, and non-spine fractures.¹ The buffered solution with high acid-neutralizing capacity minimizes the risk of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate.^2,5 Binosto^® is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto^® has the potential to improve adherence to therapy⁴, thereby decreasing the risk of disabling fractures.

Disclaimer

Please refer to the official Binosto® prescribing information and approved indications, contraindications, and warnings in your country.

References

Black DM et al. Fracture Risk Reduction with Alendronate in Women with Osteoporosis: The Fracture Intervention Trial. J Clin Endocrinol Metab 2000;85:4118–4124.
Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. Int J Pharm 2012;432:57–62.
Hruska J et al. Upper Gastrointestinal Safety with the Buffered Solution of Alendronate 70mg: Postmarketing Experience. J Bone Miner Res 2017;32 (Suppl 1):S269. Available at http://www.asbmr.org/education/AbstractDetail?aid=e14d6b16-9338-4e96-b070-d744f2d208da. Accessed October 16, 2018.
Giusti A et al. Persistence with the buffered solution of alendronate 70 mg: prospective observational study. Osteoporos Int 2018;29(S1):P853.
Binosto 70mg SPC. www.mhra.gov.uk; accessed 16.10.2018.

26.09.2018

Binosto® Persistence and Efficacy Safety Data to be Presented at the ASBMR 2018 Congress in Montréal, Canada

FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA is proud to announce that recent data from a standardized clinical database suggest an improved safety and tolerability profile of Binosto^® (alendronate effervescent tablets) over conventional alendronate tablets in postmenopausal women with osteoporosis, translating into higher treatment persistence of patients. Binosto was also found to be as effective as traditional alendronate tablets based on assessment of bone mineral density scores and bone turnover markers.

The data will be presented as posters at the ASBMR 2018, the American Society for Bone and Mineral Research, held from the 28th of September until 01th October – 2018 at the Palais des Congrès de Montréal, in Montréal QC, Canada.

The poster entitled, “Persistence with Buffered Solution of Alendronate 70mg: Prospective Observational Study,” authored by Giusti et al. presentation number SAT-0322, will be presented at the Congress in the ASBMR Discovery Hall – Room 220 B-E on Saturday, 29th of September from 12:30-2:30 pm.

The second Binosto^® safety data poster, “Effect of Buffered Solution of Alendronate 70mg on Bone Mineral Density and Bone ALP: Prospective Observational Study,” authored by Giusti et al. presentation number SUN-0697, will be presented by Prof. Dennis Black a day later at the Congress in the ASBMR Discovery Hall – Room 220 B-E on Sunday, 30th of September from 12:30-2:30 pm.

Binosto^®, the world’s first and only buffered soluble alendronate for the treatment of osteoporosis, was developed with the aim to capitalize on the excellent efficacy profile of alendronate sodium while improving gastrointestinal tolerability. Upper gastrointestinal side effects are known to be problematic with tablet forms of alendronate sodium as well as of other bisphosphonates.

“Poor adherence and persistence with oral bisphosphonates can result in patients being at increased risk of osteoporotic fractures,” states Lorraine Zakin, MD, Director Medical Affairs at EffRx Pharmaceuticals. She further states, “the data presented therefore strongly suggest that alendronate effervescent tablets have substantial potential by increasing treatment persistence to improve the long-term outcome of patients with osteoporosis.”

The American Society for Bone and Mineral Research (ASBMR) is a professional, scientific and medical society established to bring together clinical and experimental scientists who are involved in the study of bone and mineral metabolism. The conference attracts nearly 4’000 expert attendees from around the globe.

About EffRx Pharmaceuticals

About Binosto^®

Binosto^® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of osteoporosis with proven antifracture efficacy. The buffered solution minimizes the possibility of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate and thus reducing the risk of upper gastrointestinal toxicity^1,2. It is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto^® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures.

Disclaimer

Please refer to the official Binosto^® prescribing information and approved indications, contraindications, and warnings in your country.

References:

Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. International Journal of Pharmaceutics 2012;432:57-62.
Binosto 70mg SPC. mhra.gov.uk; accessed 25.09.2018

11.04.2018

EffRx to Showcase Binosto^® at WCO-IOF-ESCEO 2018 in Krakow, Poland

FREIENBACH, Switzerland–(BUSINESS WIRE)–EffRx Pharmaceuticals SA is proud to announce that it will participate in the World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases in Krakow, Poland, from 19 to 22 April 2018. The WCO-IOF-ESCEO Congress is the world’s leading clinical conference on bone, joint and muscle health and therefore the ideal venue for EffRx to showcase its flagship product Binosto^®, the only buffered soluble alendronate for the treatment of postmenopausal osteoporosis.

EffRx Pharmaceuticals SA is thrilled to inform that data on Binosto^® persistence and efficacy will be presented at the WCO-IOF-ESCEO Congress. The data will be presented in the poster area on Saturday, 21 April 2018, from 14:00 to 15:00 (posters #853 and #857).

The authors of the Binosto^® persistence and efficacy studies concluded that Binosto^® is associated with higher persistence than alendronate tablets and as efficacious as alendronate tablets on surrogate endpoints for anti-fracture efficacy.

“Last year we presented post-marketing safety data which supported our hypothesis that the unique Binosto^® properties have a positive impact on reducing the incidence of upper gastrointestinal side effects¹,” says Dr. Josef Hruska, Executive Medical Director at EffRx Pharmaceuticals. “This year we are strengthening the available evidence with very exciting, new Binosto^® data, indicating that patient persistence is significantly higher than with alendronate tablets. Furthermore, and as expected, there was no difference in efficacy between Binosto^® and alendronate tablets.”

Medical professionals from around the world will be able to learn more about Binosto^® and its exciting properties at the Binosto^® booth in the exhibition area of the ICE Krakow Congress Center (booth #21).

Please join EffRx at the WCO-IOF-ESCEO Congress in Krakow!

About EffRx Pharmaceuticals SA

EffRx Pharmaceuticals SA is a Swiss-based commercial-stage pharmaceutical company developing and commercializing prescription medications for specialty indications. Our lead commercialized product, Binosto^® is indicated for the treatment of osteoporosis and marketed in US, Europe and Asia. We strive to address true unmet medical needs and provide improved outcomes to patients with musculoskeletal, genitourinary and rare disorders.

About Binosto^®/Steovess^®

Binosto^® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of osteoporosis with proven anti-fracture efficacy. The buffered solution minimizes the possibility of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate and thus reducing the risk of upper gastrointestinal toxicity.^2,3 In the EU, Binosto^® is approved for the treatment of postmenopausal osteoporosis. It is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto^® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures.

Disclaimer

Please refer to the official Binosto^®/Steovess^® prescribing information and approved indications, contraindications, and warnings in your country.

References:

Hruska J et al. Upper Gastrointestinal Safety with the Buffered Solution of Alendronate 70mg: Postmarketing Experience. J Bone Miner Res 2017;32(S1):S269. Available at http://www.asbmr.org/education/AbstractDetail?aid=e14d6b16-9338-4e96-b070-d744f2d208da. Accessed March 22, 2018.
Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. International Journal of Pharmaceutics 2012;432:57-62.
Binosto® 70mg SPC. mhra.gov.uk; accessed 16.4.2018

Media Contact: Pamela Saredi, psaredi@effrx.com

19.09.2017

Binosto Data Presented at the ASBMR Congress in Denver

EffRx Pharmaceuticals SA is proud to inform that the latest safety data on Binosto^® were presented as a poster on Sunday, 10^th of September 2017 at the American Society for Bone and Mineral Research (ASBMR) 2017 Annual Meeting in Denver, Colorado, USA attended by more than 3,000 scientists from 70 countries.

The authors of the ASBMR publication concluded that Binosto is associated with a lower frequency of upper gastrointestinal side effects than reported for tablet forms of alendronate sodium.

“This is an important milestone for EffRx as the post-marketing safety data support our hypothesis that the unique Binosto properties have a positive impact on incidence of upper gastrointestinal side effects,” says Dr. Josef Hruska, the first author and Executive Medical Director at EffRx Pharmaceuticals. He further states, “The reduction of upper gastrointestinal side effects will potentially lead to increased adherence which in turn results in realizing full antifracture efficacy.”

The Abstract will be published by the Journal of Bone Mineral Research.

About EffRx Pharmaceuticals SA

EffRx Pharmaceuticals SA is a Swiss-based commercial-stage pharmaceutical company developing and commercializing prescription medications for specialty indications. Our lead commercialized product, Binosto^® (buffered soluble alendronate 70 mg) is indicated for the treatment of osteoporosis and marketed in US, Europe and Asia. We strive to address true unmet medical needs and provide improved outcomes to patients with musculoskeletal, genitourinary and endocrine disorders.

About Binosto^®

Binosto^® (alendronate 70 mg effervescent tablets) is the first and only buffered solution for the treatment of osteoporosis with proven antifracture efficacy. The buffered solution minimizes the possibility of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate and thus reducing the risk of upper gastrointestinal toxicity.^1,2 In the EU, Binosto^® is approved for the treatment of postmenopausal osteoporosis. It is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the esophagus. Binosto^® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures.

Disclaimer

Please refer to the official Binosto^® prescribing information and approved indications, contraindications, and warnings in your country.

References:

Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. International Journal of Pharmaceutics 2012;432:57-62.
Binosto 70mg SPC. www.mhra.gov.uk; accessed 19.9.2017

Media Contact: Pamela Saredi, psaredi@effrx.com

08.06.2017

Osteoporosis patients in Germany and Russia get access to Binosto^®

EffRx launches Binosto^® – the first and only buffered solution for the treatment of osteoporosis – in two more markets.

Freienbach, Switzerland, 8. June 2017 – EffRx Pharmaceuticals SA today announced that physicians in Germany and Russia treating patients with postmenopausal osteoporosis have a new therapy option: buffered soluble alendronate (Binosto^®). This novel formulation, which is already available in eleven countries worldwide, provides gold-standard fracture risk reduction and offers the potential for enhanced gastric tolerability.¹

In Germany, Binosto^® is marketed by Recordati Pharma GmbH and in Russia by Polypharm. For EffRx Pharmaceuticals SA, who has developed Binosto^®, the launch in Germany and Russia marks another important milestone. “With the addition of these two major markets, our geographic footprint grows significantly,” says EffRx Commercial Director Michael Schmid. “We are happy to have selected two strong and renowned local partners. While the growth of Binosto^® in our existing markets remains high, geographic expansion through strong local partnerships continues to be a key priority for EffRx to bring the promise of Binosto^® to patients worldwide.”

Data presented at the recent WCO-IOF-ESCEO Congress and published in Osteoporosis International suggest that Binosto^® is associated with a lower frequency of upper gastrointestinal adverse reactions than reported for conventional alendronate tablets.² “These data support the hypothesis that Binosto with its unique properties does enhance gastrointestinal tolerability,” says Dr. Josef Hruska, Medical Director. He adds, “This in turn may lead to improved compliance and persistence which are known to be problematic with conventional bisphosphonate tablets.”

Disclaimer

Please refer to the official Binosto^® prescribing information and approved indications, contraindications, and warnings in your country. Please click here for the approved SmPC in Europe.

References:

Hodges LA et al. Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate. International Journal of Pharmaceutics 2012;432:57-62.
Hruska J et al. Upper gastrointestinal safety with the buffered solution of alendronate 70 mg: post-marketing experience. Osteoporos Int 2017;28(S1):P589. http://www.wco-iof-esceo.org/sites/wco17/pdf/WCO17-AbstractBook.pdf.
Media Contact: Pamela Saredi, psaredi@effrx.com

01.06.2017

Paolo Agnoluzzi Appointed CFO of EffRx Pharmaceuticals

Freienbach, Switzerland, June 1^st, 2017 – EffRx Pharmaceuticals SA announced today the appointment of Mr. Paolo Agnoluzzi as Chief Financial Officer effective June 1st, 2017. Mr. Agnoluzzi joined EffRx in August 2016 as Interim Chief Financial Officer and led the re-organization of the Finance Department. Among other achievements he executed a number of debt and equity transactions, including a capital increase.

Mr. Agnoluzzi brings over 30 years of international financial experience, predominantly in biopharmaceutical companies. Most recently, he served as Finance Director for VistaJet Group. His responsibilities included acting as deputy CFO, leading the financial planning and analysis group, heading the treasury function and directing special projects within Finance. Previously, Mr. Agnoluzzi held the position of Director Finance Europe and Commercial Head DACH Region at Endocyte Europe GmbH. Prior to Endocyte, he spent more than 20 years at Amgen Europe/International driving its early European set-up of Amgen and serving various senior financial positions, including among others, Head of EU Finance Oncology, Bone & New Products and Director Finance & Administration.

“Mr. Agnoluzzi joins at an exciting and important stage in EffRx’s journey towards integrated multi-product pharmaceutical company. Paolo’s significant breadth and depth of financial and accounting expertise will be a major asset to expand the business of our company, building on Binosto® success,” said Mr. Lorenzo Bosisio, CEO of EffRx. “I want to express my genuine appreciation for his hard work, dedication and leadership over the past year in championing EffRx transformation. I wish him all the best for this well-earned promotion.”

Mr. Agnoluzzi commented, “I believe the potential for growth and value creation is tremendous, given the smart and passionate people in the Company and its attractive international footprint. I am confident that we can develop and execute plans to drive consistent sales growth. I am very excited.”

11.10.2016

Lorenzo Bosisio Appointed CEO of EffRx Pharmaceuticals

Freienbach, Switzerland, October 11^th, 2016 – EffRx Pharmaceuticals SA announced that the Company has made some changes and additions to its management team.

Lorenzo Bosisio has been appointed as Chief Executive Officer effective July 1st, 2016. Prior to his promotion to CEO, Mr. Bosisio served as Chief Commercial Officer since 2013. During his tenure in EffRx, he has built the commercial infrastructure from the ground, successfully launched the Company’s lead product, Binosto^®, enabling the transformation of EffRx from a drug development company to an integrated biopharmaceutical company with an international footprint.

Mr. Bosisio brings significant strategic and operational leadership experience from senior positions at Amgen, Alexion, PwC, and Tissot Medical. He has an excellent track record in building new international operations and brands as well as driving transformational growth in biopharmaceutical companies.

“I am highly satisfied with this appointment. Mr. Bosisio has exactly the right cross-functional experience and the right mindset to lead the expansion of our core franchise and to generate new business development opportunities,” said Mr. Jonas Ekblom, Chairman of EffRx.

Mr. Bosisio commented, “It has been a challenging and rewarding journey to bring EffRx to what it is today. I look forward to seeing further success and to leading EffRx through the next stage of its development”.

The company also announced that Mr. Paolo Agnoluzzi will serve as interim CFO, Dr. Josef Hruska has taken on the role as Medical / Medical Affairs Director and that Mr. Michael Schmid has joined as International Marketing Director.

20.05.2016

Internis launches GI friendly alendronate formulation

PharmaTimes, May 20, 2016 — Internis Pharma Binosto is now widely available across the UK, offering patients with osteoporosis the chance to access the first buffered formulation of alendronate designed to minimise gastrointestinal side effects commonly linked with traditional versions of the drug.

The National Institute for Health and Clinical Excellence recommends alendronate as an option for the prevention of first fractures in a number of groups who have a high risk of fracture, as long-term treatment with the drug is known to improve bone mineral density (from 2.4 percent to 8.8 percent) and cut the risk of fracture (from 12 percent to 49 percent).

However, the side effects of the drug has led to poor compliance rates, with nine out of ten patients who are taking traditional form of alendronate having to drop therapy as a result. Eighty percent of patients come off treatment in the first year and over one in three fail to pick up a second indication, largely because of the gastrointestinal effects associated with bisphosphonates like alendronate.

Osteoporosis is estimated to affect more than three million people in the UK, most of whom are only aware they have it when they break a bone. Addressing poor adherence to treatment has been a key goal for some time, as it is estimated to result in a 37 percent higher risk of hospitalisation and a 17 percent higher risk of fracture, the firm notes.

Binosto’s novel, buffered alendronate formulation dissolves in water, helping to overcome the difficulties in swallowing pills that some patients experience and, crucially, as it increases pH levels in the stomach, thus minimising the gastrointestinal side effects commonly linked to these medicines, it has the potential to boost adherence.

The cost is £22.80 for four once-weekly tablets. This is a higher purchase price than a generic alendronate tablet but is lower than alternative treatments for patients who fail on alendronate (such as denosumab, etc). A spokesperson for the firm told PharmaTimes that Binosto should “save considerably on downstream costs”, given its potential to improve adherence and reduce the incidence of osteoporotic fracture, which costs the NHS around £5 million per day or £2 billion per year.

“I believe that, over time, data will show that Binosto’s novel alendronate formulation helps to improve the longterm tolerability of alendronate and helps to ensure that more women with osteoporosis are properly treated. This could result in fewer fractures, improved patient quality of life and lower overall costs,” said Dr Andrew Pearson, consultant radiologist, NHS Borders, commenting on its potential.

This impact will be more significant as our ageing population grows therefore, it is essential that patients have access to effective treatments that they are able to take over many years,” added Internis’ chief operating officer Paul Tredwell.

www.pharmatimes.com/news/internis_launches_gi_friendly_alendronate_formulation_1025980

20.05.2016

Binosto now available for the effective treatment of postmenopausal osteoporosis

BioPortfolio, May 20, 2016 – The first buffered alendronate designed to minimise gastrointestinal side effects commonly associated with traditional alendronate treatment

Huddersfield, 19 May, 2016, Internis Pharmaceuticals Ltd: Binosto, the first buffered alendronate (alendronate acid 70mg); is now available on prescription as an effervescent tablet. Binosto has been specifically designed to overcome the serious difficulties patients have with gastrointestinal side effects of traditional alendronates and to offer patients an effective alternative, which helps them to remain compliant with their treatment. Binosto dissolves to create a pleasant tasting strawberry-flavoured liquid formulation, taken just once a week, to treat osteoporosis in post-menopausal women.

Referred to as the ‘silent disease’, osteoporosis is estimated to affect more than three million people in the UK, most of whom are only aware they have it when they break a bone. Currently, the National Institute for Health and Clinical Excellence (NICE) recommends alendronate as an option for the prevention of first fractures in a number of groups who have a high risk of fracture. Long-term treatment with alendronate is effective at improving bone mineral density (from 2.4% to 8.8%) and reducing the risk of fracture (from 12% to 49%).

Unfortunately, compliance with alendronate tablets is poor; nine out of ten patients who are taking traditional alendronate are unable to continue taking the tablets due to side effects. Of more concern, poor adherence has been estimated to result in a 37% higher risk of hospitalisation and a 17% higher risk of fracture. Modelling predicts that if adherence to alendronates can be improved this will lead to fewer fractures for patients.

Supplied by Internis, a leading UK bone health company, Binosto’s novel, buffered alendronate formulation completely dissolves in just 120ml of plain water and helps to overcome the difficulties in swallowing pills that some patients experience. Importantly, Binosto’s buffered formulation has been shown to increase the pH of the patient’s stomach, minimising the gastrointestinal side effects that are common with these medicines. Internis believes that patients will find Binosto easier to take than alendronate tablets and therefore be likely to adhere to their medication for longer. This, in turn, could help to reduce the number of fractures seen in patients who cannot tolerate traditional alendronate tablets. Internis will be closely monitoring prescription data to confirm that patients adhere to Binosto for longer.

Dr Andrew Pearson, Consultant Radiologist, NHS Borders, commented:

Chief Operating Officer of Internis, Paul Tredwell commented: “Following recent research on over 1.6 million prescriptions, we know that patients don’t comply with their traditional bisphosphonate tablets, with 80% of patients coming off treatment in the first year and over 1 in 3 patients not picking up a second prescription. This compliance issue is, in part, due to the gastrointestinal side effects, which are well known to be associated with bisphosphonates like alendronate. With the launch of Binosto and its novel, buffered formulation, Internis aims to increase this compliance, which, in the long term, will result in fewer fractures and a reduction in associated costs to the NHS. This impact will be more significant as our ageing population grows therefore, it is essential that patients have access to effective treatments that they are able to take over many years.

19.05.2016

Adherence hopes for effervescent osteoporosis drug

Pharmaceutical Field, Amy Schofield, May 19, 2016 — Binosto, the first buffered alendronate (alendronate acid 70mg) is now available on prescription as an effervescent tablet.

Binosto has been specifically designed to overcome the serious difficulties patients have with gastrointestinal side effects of traditional alendronates. Supplied by UK bone health company Internis, Binosto offers patients an alternative which helps them to remain compliant with their treatment.

Binosto is taken once a week, dissolved in water, to treat osteoporosis in post-menopausal women and helps to overcome the difficulties in swallowing pills that some patients experience. The National Institute for Health and Clinical Excellence (NICE) currently recommends alendronate as an option for the prevention of first fractures in a number of groups who have a high risk of fracture.

Osteoporosis is estimated to affect more than three million people in the UK, many of whom are not aware that they have the condition until they break a bone.

Long-term treatment with alendronate is effective at improving bone mineral density (from 2.4% to 8.8%) and reducing the risk of fracture (from 12% to 49%). Unfortunately, compliance with alendronate tablets is poor. Modelling predicts that if adherence to alendronates can be improved this will lead to fewer fractures for patients.

Binosto’s buffered formulation increases the pH of the patient’s stomach, minimising the gastrointestinal side effects that are common with these medicines.

Chief Operating Officer of Internis, Paul Tredwell, commented: “Following recent research on over 1.6 million prescriptions, we know that patients don’t comply with their traditional bisphosphonate tablets, with 80% of patients coming off treatment in the first year and over 1 in 3 patients not picking up a second prescription. With the launch of Binosto and its novel, buffered formulation, Internis aims to increase this compliance, which, in the long term, will result in fewer fractures and a reduction in associated costs to the NHS.”

Link:: www.pharmafield.co.uk/Pf-Fox-News/General/2016/05/Adherence-hopes-for-effervescent-osteoporosis-drug

07.01.2016

First-in-Class Osteoporosis Treatment Binosto Launches in South Korea

Freienbach, January 7, 2016 — EffRx Pharmaceuticals SA announced today that its Korean partner has received regulatory clearance from the Korean Ministry of Food and Drug Safety (MFDS) to market Binosto® buffered alendronate for the treatment of osteoporosis in Korea.

Binosto® – the first and only buffered solution for the treatment of osteoporosis – delivers gold standard fracture-risk reduction and offers the potential to improve adherence to therapy. Buffering minimizes the possibility of exposing the esophagus (in case of reflux) and the stomach to acidified alendronate1.

Binosto is marketed in Korea by Ahn-Gook Pharmaceutical who has a strong local sales presence and expertise. Commercial launch activity in Korea is anticipated early January 2016. Jin Auh, Ahn-Gook President & CEO, said that the Korean market is worth 115M USD, with alendronate accounting for more than 90%. “We expect Binosto® to overcome certain limitations of oral bisphosphonates and to provide improved adherence to therapy for patients suffering from osteoporosis.”

Lorenzo Bosisio, Chief Commercial Officer of EffRx Pharmaceuticals, comments “we are pleased to receive the Korean regulatory approval for Binosto, expanding physician and patient access to our product. Korea is a sizeable and important pharmaceutical market. We expect continued momentum as we launch in new territories around the world.”

Binosto® is currently available by prescription in the United States, Italy, Spain, Portugal, and the UK. The initial uptake in Europe with almost half a million monthly prescriptions in the first year has been very promising. Binosto® is licensed in over 50 countries and in the process of gaining regulatory clearance in several territories, hence further expanding its global footprint.

About EffRx Pharmaceuticals
EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that develops and commercializes novel therapeutic entities for orphan and specialty indications by applying its proprietary technology platform. EffRx developments focus at improving the quality of life of patients with unmet medical needs.

About Ahn-Gook
Ahn-Gook Pharmaceutical Co., Ltd. is a Korean-based pharmaceutical company. The Company’s products include respiratory agents, gastrointestinal agents, anti-inflammatories, analgesics, ophthalmics and circulatory system agents. It also provides antibiotics, antifungal drugs, antihyperlipidemics, antiallergic drugs, anti-diabetic drugs, urinary system drugs and skin disease remedies. Anh-Gook also markets OTC and ethical drugs.

About Binosto
Binosto® – the first and only buffered solution for the treatment of osteoporosis – delivers gold standard fracture-risk reduction and offers the potential to improve adherence to therapy. Buffering minimizes the possibility of exposing the oesophagus (in case of reflux) and the stomach to acidified alendronate1. Binosto® is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the oesophagus. Binosto® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures.

Disclaimer
Please refer to the official Binosto® Prescribing Information and approved indications, contraindications, and warnings in your country.

1Approved SmPC in Europe: www.mhra.gov.uk/home/groups/spcpil/documents/spcpil/con1447998343651.pdf

Contact
Pamela Saredi
Communications Manager
+41 44 503 78 64
psaredi@effrx.com

17.12.2015

Successful Label Variations Granted to Binosto in Europe

Freienbach, December 17, 2015 — EffRx Pharmaceuticals SA today announced that the MHRA (UK Medicines & Healthcare products Regulatory Agency) has adopted two positive variations for the labelling of Binosto® – buffered effervescent alendronate for oral solution. These variations will be implemented through the Decentralized Procedure (DCP) in the other European territories where Binosto® is approved.

The label now indicates that the specific Binosto® pharmaceutical formulation “minimises the possibility of exposing the oesophagus (in case of reflux) and the stomach to acidified alendronate.”

The Binosto® label also includes study data published in the International Journal of Pharmaceutics, “Modulation of gastric pH by a buffered soluble effervescent formulation: A possible means of improving gastric tolerability of alendronate.” The updated label also highlights that Binosto® is a buffered formulation for oral solution and provides the pH of this novel pharmaceutical form.

For anyone with osteoporosis, the best protection against suffering from a life-threatening fracture is taking medications as prescribed. However, studies show that many patients are not adherent to therapy or have trouble in swallowing pills, and stop taking their medication. The updated label supports the rationale that Binosto® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures. The new label will also help to differentiate the hybrid approved Binosto® from generic alendronate formulation in the market.

Aileen Dowling, Head of Regulatory Affairs at EffRx, comments that “this label update reinforces the hybrid nature of the product, a different pharmaceutical form, being easier to swallow and incorporating a buffer system that can address oesophageal tolerability of alendronate tablets.“

Binosto® is currently available by prescription in Europe in the following countries: UK, Italy, Spain, and Portugal. The initial uptake in Europe with almost half a million monthly prescriptions in the first year has been very promising. Binosto® is licensed in over 50 countries and in the process of gaining regulatory clearance in several territories, hence further expanding its global footprint.
About EffRx Pharmaceuticals

EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that develops and commercializes novel therapeutic entities for orphan and specialty indications by applying its proprietary technology platform. EffRx developments focus at improving the quality of life of patients with unmet medical needs.

About Binosto®
Binosto® – the first and only buffered solution for the treatment of osteoporosis – delivers gold standard fracture-risk reduction and offers the potential to improve adherence to therapy. Buffering minimizes the possibility of exposing the oesophagus (in case of reflux) and the stomach to acidified alendronate1. Binosto® is taken once a week as an easy-to-swallow, buffered solution with no risk of a tablet getting lodged in the oesophagus. Binosto® has the potential to improve long-term patient adherence to therapy, thereby decreasing the risk of disabling fractures.

Disclaimer
Please refer to the official Binosto® Prescribing Information and approved indications, contraindications, and warnings in your country.

1Approved SmPC in Europe: www.mhra.gov.uk/home/groups/spcpil/documents/spcpil/con1447998343651.pdf

Contact
Pamela Saredi
Communications Manager
+41 44 503 78 64
psaredi@effrx.com

New Partnership in Russia and CIS Further Expands Footprint for EffRx Proprietary Technologies

Freienbach, Switzerland, January 13, 2015 – EffRx Pharmaceuticals SA, a specialty pharmaceutical company focused on the development of novel therapeutic entities for a variety of orphan and specialty indications, today announced the signing of an agreement with Andrus Ltd. to develop products for commercialization in Russia and CIS countries.

“We are excited to further expand the footprint of our proprietary technologies with the development of novel therapeutic entities for these fast-growing emerging markets” said Christer Rosén, EffRx Chief Executive Officer. “Although our core focus within EffRx is to develop our own un-encumbered orphan products we will still utilize this business segment to generate a steady increasing revenue stream.”

Under the terms of the agreement EffRx will develop novel, tailored therapeutic entities (6 products are presently identified) for commercialization by Andrus in Russia and CIS countries. “We are excited to expand our partnership with Andrus beyond our earlier agreement with Binosto®, our first approved product for osteoporosis, and to bring new products that will address an unmet medical need in these countries” said Christer Rosén.

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About EffRx Pharmaceuticals
EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that develops novel therapeutic entities including orphan indications by applying its proprietary technology platform to well characterized molecules. EffRx developments focus at improving the quality of life of patients with unmet medical needs.

About Andrus Ltd.
Andrus Ltd. is dedicated to creating a novel Russian biopharmaceutical platform by bridging the gap between novel biopharmaceutical technologies and the Russian Healthcare market. Through partnering with leading development companies and leveraging Andrus’ domestic operations, the Company is able to build a healthcare platform based on best in class science and drug development.

03.06.2014

EffRx Signs Binosto® Distribution Agreements in Russia and Africa

New Partnerships Significantly Expand Global Footprint for Proprietary Buffered Effervescent Osteoporosis Therapy

Freienbach, Switzerland — EffRx Pharmaceuticals SA, a specialty pharmaceutical company, today announced the signing of two new distribution agreements that will bring its osteoporosis treatment, Binosto®, to strategic markets in Russia, CIS countries and Africa.

“We are excited to expand the global footprint of Binosto® with the addition of these fast-growing emerging markets”

EffRx has partnered with Andrus, Ltd. to distribute Binosto® in Russia, Armenia, Azerbaijan, Belarus, Georgia, Kazakhstan, Kyrgyzstan, Moldova, Tajikistan, Turkmenistan, Ukraine and Uzbekistan. Through another partnership, with Adcock Ingram Heathcare (Pty) Ltd., Binosto® will be available in South Africa, Namibia, Botswana, Swaziland, Lesotho, Zimbabwe, Kenya and Ghana.

EffRx’s first commercial product, Binosto®, is a buffered, effervescent formulation of alendronate, the most broadly prescribed treatment for osteoporosis. The product was approved by the U.S. Food & Drug Administration in 2012. With these agreements, Binosto® will be commercially available in 53 countries worldwide.

“We are excited to expand the global footprint of Binosto® with the addition of these fast-growing emerging markets,” said Lorenzo Bosisio, EffRx Chief Commercial Officer. “Binosto® is our first approved product and provides an important proof of concept for our development pipeline of novel therapeutic entities, and we look forward to making it available to an increasing population with these two agreements.”

“The continued worldwide adoption of Binosto® is the first of our current out-licensing initiatives and is expected to provide a steady income stream in the future. We continue to focus on the two other, potentially larger, elements of our threefold growth strategy – products in the orphan drug segment, including the ongoing development of our PCOS product, and lifecycle management,” said Christer Rosén, EffRx Chairman and Chief Executive Officer.

04.02.2014

EffRx signs distribution agreements for Binosto in Italy, Spain and Portugal

Freienbach, February 4^th 2014 – EffRx Pharmaceuticals S.A. today announced that it has signed exclusive distribution agreements with three leading local pharmaceutical companies in Italy, Spain and Portugal for Binosto® – EffRx’s innovative osteoporosis medication. Under the terms of the agreement, EffRx grants exclusive marketing authorization and distribution rights to Abiogen Pharma SpA. for Italy, Lacer S.A. for Spain, and Laboratorios Atral S.A. for Portugal.

Binosto® is the first and only buffered solution for the treatment of osteoporosis, delivering fracture-risk reduction at the hip & spine. Binosto is administered as an effervescent tablet for oral solution and offers convenience for the patient. Binosto is currently marketed in the United States, and approved in Europe and Australia; approvals in other territories are pending. Binosto® was originally developed by EffRx through an agreement with Merck & Co, Inc. granting EffRx worldwide rights to all Merck effervescent and related patents protecting alendronate.

EffRx expects the launch in these markets beginning in the second quarter 2014. Italy, Spain and Portugal are 3 major markets for osteoporosis treatment in Europe. EffRx is confident that the promise of Binosto® will also become true in other European countries with the appointment of distribution partners that have a sizeable footprint as well as in-depth understanding of their local markets.

Christer Rosén, Chairman and CEO of EffRx Pharmaceuticals said, “We are thrilled to announce the partnerships with Abiogen, Lacer, and Atral. These partners, with their well-established sales forces, are well positioned to drive Binosto® to a market leadership position.”

EffRx is committed to making Binosto® available to osteoporosis patients around the world and is currently discussing the commercialization of Binosto® with pharmaceutical companies in other key Regions and countries.

About EffRx Pharmaceuticals
EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that exploits its proprietary technology platform to create novel therapeutic entities.

EffRx strives to make existing good medicines better and thereby improve the quality of life of patients. EffRx targets improvements to leading medications, focused on tolerability, absorption, compliance and convenience, thus creating best in class products.

EffRx has established its proprietary pipeline in metabolic bone disease and pediatric medications and is presently aggressively expanding its product portfolio.

Enquiries
Lorenzo Bosisio
lbosisio@effrx.com
+41 44 503 7867

03.06.2013

EffRx signs distribution agreement with Ahn-Gook Pharma for Binosto for Korea

Freienbach, 03 June 2013 – EffRx Pharmaceuticals S.A. today announces the signing of an exclusive distribution agreement with Ahn-Gook Pharmaceutical Co. Ltd for Binosto – EffRx’s innovative osteoporosis medication – for the Korean market.

Under the terms of the agreement, EffRx grants exclusive rights to Ahn-Gook to register, market and distribute Binosto® in South Korea. This agreement will leverage Ahn-Gook’s strong local presence and expertise in Korea.

Binosto® is the first and only buffered solution for the treatment of osteoporosis, delivering fracture-risk reduction at the hip & spine and offering the potential to improve adherence to therapy. Binosto® is taken with a small amount of water and lowers gastric acidity in the first 30 minutes after administration, a time when the patient must not eat or drink anything other than plain water.

Christer Rosen, EffRx Chairman and CEO said at the signing ceremony “We are pleased to initiate a partnership with Ahn-Gook who has a dynamic and outstanding sales force, and we are convinced that Ahn-Gook becomes a new strong player in the Korean osteoporosis market.

Jin Auh, Ahn-Gook President & CEO said that the Korean market is worth 140M USD, and bisphosphonates account for more than 90%. “We highly expect Binosto® to overcome limitations of current bisphosphonates”

Binosto is expected to be registered in Korea in 2014 and launched within the same year.

Enquiries
Lorenzo Bosisio
+41 44 503 7867

About Binosto®
Binosto® (buffered alendronate sodium 70mg) is an effervescent tablet for oral solution and has been approved in the United States, Europe and Australia for the treatment of osteoporosis. It has been marketed since October 2012 in the United States by Mission Pharmacal, and is expected to be launched in other territories in 2014.

Binosto has been developed with EffRx innovative patented effervescent technology. EffRx is operating under exclusive worldwide rights to all effervescent and related patents of alendronate licensed from Merck & Co, Inc.

About EffRx
EffRx Pharmaceuticals SA is an innovative specialty pharmaceutical company that exploits its proprietary technology platform to improve the efficacy, tolerability and convenience of medicines, thereby generating better products and improving quality of life for patients. The primary focus is on metabolic bone disease, pediatric medications and supporting larger companies in Life Cycle Management.

About Ahn-Gook
Ahn-Gook Pharmaceutical Co., Ltd. is a Korea-based pharmaceutical company. The Company’s products include respiratory agents, gastrointestinal agents, anti-inflammatories, analgesics, ophthalmics and circulatory system agents. It also provides antibiotics, antifungal drugs, antihyperlipidemics, antiallergic drugs, anti-diabetic drugs, urinary system drugs and skin disease remedies. Anh-Gook also markets OTC and ethical drugs.

Disclaimer
Please refer to the official Binosto® Prescribing Information and approved indications, contraindications, and warnings in your country.

04.02.2013

Agreement with Hikma Pharmaceuticals

EffRx signs a distribution agreement with Hikma Pharmaceuticals for Binosto® in MENA

Freienbach, 4 February 2013 – EffRx Pharmaceuticals SA today announces the signing of a distribution agreement with Hikma Pharmaceuticals for Binosto®, EffRx’s innovative osteoporosis medication, for the Middle East and North Africa (“MENA”) region. Binosto® is the first and only buffered solution for the treatment of osteoporosis, delivering fracture-risk reduction and offering the potential to improve adherence to therapy.

Under the terms of the agreement, EffRx grants the exclusive rights to Hikma to register, market and distribute Binosto® in 19 countries across MENA. The agreement will leverage Hikma’s strong local presence and regulatory expertise in MENA, with over 1,600 sales and marketing representatives across the region.

Binosto® was developed by EffRx through an agreement with Merck & Co, Inc. granting EffRx the worldwide rights to all effervescent and related patents of alendronate. Patents have been granted to EffRx providing exclusivity for Binosto® through February 2023. Additional patents are pending. Binosto (buffered alendronate sodium 70mg) is an effervescent tablet for oral solution and has recently been approved in the United States, Europe and Australia[1] for the treatment of osteoporosis in postmenopausal women. In the United States it is also approved for the treatment of men with osteoporosis to increase bone mass. Approvals in other territories are pending.

Mazen Darwazah, Hikma’s Vice Chairman and CEO of MENA said, “We are pleased to be licensing this excellent product from EffRx. The agreement supports our strategy to continue working with global partners to strengthen our product portfolio in growing therapeutic categories, including women’s health. Hikma is committed to bringing a broad range of high quality, pharmaceutical products to patients in MENA.”

Christer Rosén, Chairman and CEO of EffRx Pharmaceuticals said, “We are thrilled to announce this partnership with Hikma, a company who we believe to be an ideal partner for EffRx in MENA. We believe Hikma’s well-established sales force across the region can drive Binosto® to a market leadership position and we are extremely proud to have Hikma as a partner.”

EffRx is currently discussing the commercialization of Binosto with pharmaceutical companies in other key Regions. Lorenzo Bosisio, VP Business Development said, “This agreement is an important milestone for EffRx. It comes just after the recently announced reacquisition of rights to Binosto from Takeda. We are very excited about the strong interest for Binosto from partners in the remaining territories, such as Europe, Asia, and Latin America. We are committed to fulfil the promise of Binosto for patients around the world.”

20.07.2022

EffRx Pharmaceuticals announces the launch of Alkindi® as replacement therapy for pediatric adrenal insufficiency in Switzerland

12.05.2022

World Orphan Drug Alliance – combining forces to reach patients with rare diseases across the globe

26.04.2022

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Efmody® in Switzerland

16.11.2021

EffRx Obtains Swiss Marketing Authorization for Alkindi® for Paediatric Adrenocortical Insufficiency

04.11.2021

EffRx Obtains Swiss Marketing Authorization for Bronchitol® in Cystic Fibrosis

22.06.2021

A Prospective Study in More Than 1,000 Osteoporosis Patients Concluded That Binosto® (Buffered Soluble Alendronate) May Increase Patient Satisfaction, Long-Term Adherence and Therefore Efficacy

21.10.2020

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Alkindi® and Submits Market Authorization Application in Switzerland

13.6.2019

EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Commercialization of Bronchitol® in Switzerland

2.4.2019

EffRx to Showcase Binosto® at the WCO-IOF-ESCEO 2019 in Paris

14.11.2018

EffRx Announces Expansion of the Executive Team

25.10.2018

Osteoporosis Patients in Southeast Asia Get Access to Binosto®

26.09.2018

Binosto® Persistence and Efficacy Safety Data to be Presented at the ASBMR 2018 Congress in Montréal, Canada

11.04.2018

EffRx to Showcase Binosto® at WCO-IOF-ESCEO 2018 in Krakow, Poland

19.09.2017

Binosto Data Presented at the ASBMR Congress in Denver

08.06.2017

Osteoporosis patients in Germany and Russia get access to Binosto®

EffRx Signs Binosto® Distribution Agreements in Russia and Africa

New Partnerships Significantly Expand Global Footprint for Proprietary Buffered Effervescent Osteoporosis Therapy

04.02.2014

EffRx signs distribution agreements for Binosto in Italy, Spain and Portugal

03.06.2013

EffRx signs distribution agreement with Ahn-Gook Pharma for Binosto for Korea

04.02.2013

Agreement with Hikma Pharmaceuticals

EffRx signs a distribution agreement with Hikma Pharmaceuticals for Binosto® in MENA

EffRx Pharmaceuticals announces the launch of Alkindi^® as replacement therapy for pediatric adrenal insufficiency in Switzerland

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Efmody^® in Switzerland

EffRx Obtains Swiss Marketing Authorization for Alkindi^® for Paediatric Adrenocortical Insufficiency

EffRx Obtains Swiss Marketing Authorization for Bronchitol^® in Cystic Fibrosis

A Prospective Study in More Than 1,000 Osteoporosis Patients Concluded That Binosto^® (Buffered Soluble Alendronate) May Increase Patient Satisfaction, Long-Term Adherence and Therefore Efficacy

EffRx Pharmaceuticals Signs Exclusive License Agreement with Diurnal for the Registration and Commercialization of Alkindi^®and Submits Market Authorization Application in Switzerland

EffRx Pharmaceuticals Signs Exclusive License Agreement with Pharmaxis for the Registration and Commercialization of Bronchitol^®in Switzerland

EffRx to Showcase Binosto^® at the WCO-IOF-ESCEO 2019 in Paris

Osteoporosis Patients in Southeast Asia Get Access to Binosto^®

EffRx to Showcase Binosto^® at WCO-IOF-ESCEO 2018 in Krakow, Poland

Osteoporosis patients in Germany and Russia get access to Binosto^®