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Worldwide rights of Vamorolone for DMD

Santhera exercises option to obtain worldwide rights of Vamorolone for Duchenne muscular dystrophy (DMD) and All Other Indications

La Force is happy to share this press release provided by Santhera Pharmaceuticals > Pratteln, Switzerland, and Rockville, MD, USA, September 2, 2020

 

Santhera Pharmaceuticals announces that it has signed agreements with Idorsia and ReveraGen BioPharma Inc., making Santhera a direct license holder of vamorolone. Under the agreements, Santhera has obtained an exclusive license from ReveraGen, the originator of vamorolone, for all indications worldwide. The agreements create additional value for Santhera through the transfer of rights for the previously excluded markets Japan and South Korea, the right to grant sublicenses and a share in the expected Priority Review Voucher. Vamorolone, a first-in-class anti-inflammatory drug candidate with a novel mode of action, is currently being investigated in the pivotal Phase 2b VISION-DMD study in patients with Duchenne muscular dystrophy (DMD) by originator ReveraGen and completion of study enrollment is expected shortly.

 

Quickly

  • The license gives Santhera worldwide rights to vamorolone, now also including the major markets Japan and South Korea, and paves the way for partnering in additional indications
  • Agreements with Idorsia and ReveraGen give Santhera immediate control over vamorolone and defer milestone-related payments
  • The transaction establishes Santhera as a leading company in rare neuromuscular diseases with two late-stage assets addressing the medical need of DMD patients from early to late disease stages

 

Dario Eklund, Chief Executive Officer of Santhera –  “We are excited about the license transfer of vamorolone to Santhera. Our decision to exercise the option now has been driven by a combination of factors including the availability of encouraging clinical efficacy and safety data with vamorolone, enhanced deal terms and the ability to gain full control over the asset. We look forward to contributing our significant expertise to advancing vamorolone in DMD and exploring additional business development opportunities. We believe that having two promising, complementary, late-stage assets for DMD in our pipeline will enable increased access to potentially transformative treatments for a wider patient population. We are grateful to Idorsia, our anchor shareholder, for enabling early access to the license, highlighting its confidence in Santhera as the best-suited company to bring vamorolone to patients.”

 

Eric Hoffman, Ph.D., Vice President of Research of ReveraGen BioPharma –  “We are delighted about the revised contractual arrangement and being able to work directly with Santhera as the licensee for vamorolone. Santhera’s experience in both development of DMD drug candidates and the commercialization of a rare disease product positions it well to bring vamorolone to patients. Our work to date clearly shows that vamorolone not only holds the potential to become a new standard of care for patients with DMD but also could benefit patients in a number of other inflammatory diseases.”

 

With Puldysa® and vamorolone, Santhera is building a complementary DMD product portfolio.

Vamorolone is in development for young DMD patients requiring an anti-inflammatory, muscle strengthening treatment before the onset of respiratory function decline. Based on the cumulative knowledge obtained from extensive non-clinical studies and Phase 1 and Phase 2a clinical studies with vamorolone, ReveraGen is currently conducting the pivotal Phase 2b VISION-DMD trial and anticipates full study enrollment shortly. Subject to positive results of the first 6-month treatment period, now expected in the second quarter of 2021 due to delays caused by the Covid-19 pandemic, this would pave the way for regulatory submission to the US FDA in the fourth quarter of 2021.

Puldysa (idebenone) for patients with DMD in respiratory function decline who are not taking glucocorticoids is currently under regulatory review in Europe for which Santhera anticipates a CHMP opinion in the fourth quarter of 2020. The Company expects the combination of vamorolone and Puldysa to address the medical needs of DMD patients, from early to late disease stages, irrespective of age, underlying dystrophin mutation or ambulatory status. 

Vamorolone and Puldysa have been granted Orphan Drug status in the US and Europe, Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status by the UK MHRA. In the UK, Puldysa is available to patients through the Early Access to Medicines Scheme (EAMS).

 

About Vamorolone

Vamorolone is a first-in-class anti-inflammatory drug candidate with a novel mode of action Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and, therefore, could emerge as a valuable alternative to corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a clear unmet medical need in this patient group as high dose corticosteroids have significant systemic side effects that detract from the patient’s quality of life. Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with participation in funding and design of studies by several international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program.

 

About Santhera

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with a high unmet medical need. Santhera is building Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone; a first-in-class dissociative steroid presently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. Santhera out-licensed ex-North American rights to its first approved product; for further information, please visit www.santhera.com.

 

About ReveraGen BioPharma

ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Muscular Dystrophy Association USAParent Project Muscular DystrophyFoundation to Eradicate DuchenneSave Our SonsJoiningJackAction DuchenneCureDuchenne, Ryan’s Quest, Alex’s WishDuchenneUK, Pietro’s Fight, Michael’s Cause, and Duchenne Research Fund. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).

 

More links

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FDA Acceptance of Casimersen

Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45

Original press release, CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE)

Quickly

  • FDA grants Priority Review Status and sets regulatory action date for February 25, 2021
  • FDA has indicated it does not currently plan to hold an advisory committee meeting to discuss the application
  • Received FDA’s conditional approval of AMONDYS 45™ as a brand name for casimersen
  • Casimersen has been studied for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne

About the FDA Acceptance of Casimersen

Sarepta Therapeutics, Inc. announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date of February 25, 2021. The FDA has indicated it does not currently plan to hold an advisory committee to discuss the application. In addition, the Company has received conditional approval of AMONDYS 45 as the brand name for casimersen. Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the dystrophin gene.

The Company submitted its NDA filing in June 2020 and requested priority review, which the FDA granted. The NDA included data from the casimersen arm of the ESSENCE study (also known as Study 4045-301), a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of casimersen in patients amenable to skipping exons 45. An interim analysis from ESSENCE demonstrated a statistically significant increase in dystrophin production as measured by western blot in patients who received casimersen compared to baseline and placebo. The study is ongoing and remains blinded to collect additional efficacy and safety data.

Doug Ingram, president and chief executive officer, Sarepta Therapeutics – “The FDA’s acceptance of our NDA for casimersen is an important step toward our goal of rapidly bringing therapies to patients living with Duchenne muscular dystrophy. If it is approved, casimersen, our third exon-skipping medicine in our PMO RNA-based platform, will offer treatment to the 8% of Duchenne patients who are amenable to exon 45 skipping.”

About Casimersen

Casimersen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the Duchenne gene. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for the production of an internally truncated dystrophin protein. Read more here.

About the ESSENCE Study

The ESSENCE study is a double-blind, placebo-controlled, multi-center Phase 3 study evaluating the efficacy and safety of casimersen (SRP-4045) and golodirsen (SRP-4053, approved as VYONDYS 53™). Eligible patients with out-of-frame deletion mutations amenable to exon 45 or 53 skippings are randomized to receive once-weekly intravenous (IV) infusions of 30 mg/kg of SRP-4045 or 30 mg/kg of SRP-4053, respectively (combined-active group) or placebo for up to 96 weeks. This is followed by an open-label extension period in which all patients will receive open-label active treatment for 48 weeks, up to week 144 of the study.

Clinical efficacy is being assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT). All patients undergo a muscle biopsy at baseline and will undergo a second muscle biopsy either at week 48 or week 96.

Safety is being assessed through the collection of adverse events, laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.

About Sarepta Therapeutics

The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow them on TwitterLinkedInInstagram and Facebook.

More about DMD

DMD is caused by genetic mutations that prevent dystrophin production. Patients with DMD experience progressive and irreversible muscle loss with symptoms appearing as early as two years of age. 

Duchenne muscular dystrophy occurs primarily in males. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with DMD may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. Read more here.

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Viltolarsen, exon 53 skipping therapy, approved

N.S. Pharma’s VILTEPSO™ (viltolarsen) injection now FDA-Approved in the U.S. for the treatment of Duchenne muscular dystrophy in patients amenable to exon 53 skipping therapy.

 

  • Patients taking VILTEPSO showed an increase in dystrophin expression to an average of 5.9% of normal after 20-24 weeks of treatment.
  • Overall, in a pivotal study of VILTEPSO, 100% of patients showed an increase in dystrophin levels after treatment, and 88% of patients showed dystrophin levels of 3% of average or higher.

Press release > PARAMUS, NJ: August 12, 2020 – N.S. Pharma, Inc.

 

N.S. Pharma announced that the U.S. Food & Drug Administration (FDA) has approved VILTEPSO™ (viltolarsen) injection for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy. VILTEPSO received an Accelerated Approval by the FDA based on an increase in dystrophin, an essential protein for supporting muscle health. Since a lack of dystrophin is the underlying cause of DMD, increasing dystrophin as much and as early as possible is a key goal in the treatment of DMD. VILTEPSO is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in children as young as four years old. The continued approval of VILTEPSO may be contingent on confirmation of a clinical benefit in the Phase 3 confirmatory trial.

 

More about the study

The VILTEPSO New Drug Application (NDA) submission included results from a Phase 2, a two-period study in patients aged four to less than ten years of age conducted in North America (Study 1, N=16) and a multicenter, open-label study in boys five to less than 18 years of age conducted in Japan (Study 2, N=16).

In Study 1, of those patients who received the recommended dose of 80 mg/kg/wk (N=8), 100% of patients (8/8) showed an increase in dystrophin levels after treatment with VILTEPSO and 88% of patients (7/8) showed dystrophin levels of 3% or greater than normal. Overall, after 20-24 weeks of treatment, a mean increase in dystrophin expression to nearly 6% of normal was observed with VILTEPSO (80 mg/kg/wk) versus 0.6% at baseline. The most common side effects of VILTEPSO included upper respiratory tract infection, injection site reaction, cough and fever.

 

Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago – “For decades, neurologists who treat DMD have hoped for the discovery of therapies capable of significantly improving dystrophin production, and the magnitude of dystrophin increases observed with VILTEPSO are impressive. The approval of VILTEPSO is an exciting development for DMD patients amenable to exon 53 skipping therapy and may rapidly become a foundational treatment for these patients.”

 

  • In clinical studies, no patients experienced kidney toxicity during treatment with VILTEPSO. However, kidney toxicity from drugs like VILTEPSO may be possible. Your doctor may monitor the health of your kidneys before starting and during treatment with VILTEPSO.
  • The most common side effects of VILTEPSO included upper respiratory tract infection, injection site reaction, cough and fever.
  • For additional safety information, please see the full Prescribing Information.

N.S. Pharma continues to study the safety and efficacy of VILTEPSO in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling. The purpose of this Phase 3 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.

 

About VILTEPSO™ (viltolarsen) injection

Before its approval in the U.S., VILTEPSO was granted Priority Review as well as Rare Pediatric Disease, Orphan Drug and Fast Track Designations. In March 2020, VILTEPSO was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Before its approval in Japan, VILTEPSO was granted the SAKIGAKE designation, Orphan Drug designation, and designation of Conditional Early Approval System.

VILTEPSO is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under Accelerated Approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VILTEPSO. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. It is an infusion that doctors administer into the bloodstream.

 

How Viltolarsen works

Viltolarsen contains an artificial piece of mRNA that masks exon 53, causing cells to “skip” this exon when they are making mature mRNA. This skip restores the so-called “reading frame” of the mRNA molecule. In other words, it ensures that the remaining exons fit together again, allowing a cell’s protein-making machinery to synthesize a shorter but working dystrophin protein.

Because viltolarsen is specific to exon 53, the treatment is effective only in those DMD patients who have a mutation that is amenable to exon 53 skipping. Thanks to Muscular Dystrophy News Today for this description.

 

N.S. Pharma

NS Pharma, Inc. is a wholly-owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit www.nspharma.com. N.S. Pharma is a registered trademark of the Nippon Shinyaku group of companies.

N.S. Pharma will provide families, physicians and healthcare professionals dedicated and individualized resources every step of the way through the N.S. Support program. N.S. Pharma will be hosting a series of webinars on the comprehensive care coordination available through N.S. Support. Follow them on LinkedIn and Twitter for information and registration for upcoming webinars.

 

Tsugio Tanaka, President, NS Pharma, Inc. – “On behalf of NS Pharma and Nippon Shinyaku, I would like to express our deepest gratitude to the families and physicians who participated in our clinical trials and made today’s approval possible. We are proud to now offer an important new treatment option to help address the significant unmet needs caused by this devastating disease.”

 

More about DMD

DMD is caused by genetic mutations that prevent dystrophin production. Patients with DMD experience progressive and irreversible muscle loss with symptoms appearing as early as two years of age. 

Duchenne muscular dystrophy occurs primarily in males. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with DMD may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications. Read more here.

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Translarna: Update for Non-Ambulatory Patients with DMD

PTC Therapeutics Announces that The Committee for Medicinal Products for Human Use (CHMP) Recommendation of Translarna ™ (ataluren) Label Update for Non-Ambulatory Patients with Duchenne Muscular Dystrophy

Original press release > SOUTH PLAINFIELD, N.J., June 29, 2020,/PRNewswire/ — PTC Therapeutics, Inc.

 

PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended by a majority of votes to remove the statement “efficacy has not been demonstrated in non-ambulatory patients” from the Summary of product characteristics (SmPC) for Translarna™ (ataluren). This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation. The change also should support reimbursement agencies granting continued access to Translarna for patients who become non-ambulatory during their treatment. The CHMP’s positive opinion is subject to final approval by the European Commission, which is typically granted in a two-month time frame.

 

Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics – “We are excited to see that the CHMP adopted the positive opinion for this label modification allowing patients who become non-ambulatory to continue to use Translarna. All nonsense mutation Duchenne patients should be able to benefit from continued Translarna use, ensuring they have the best chance of preserving muscle function for as long as possible.”

 

Translarna is the only treatment for the underlying cause of Duchenne caused by a nonsense mutation and works by restoring dystrophin production. The EMA approves it for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged two years and older. Real-world evidence and clinical trials have demonstrated the potential of Translarna to slow disease progression and improve outcomes before and beyond the loss of ambulation:

  • In a long-term, open-label extension study (Study 019), Translarna preserved lung function in non-ambulatory patients for an additional four years compared with patients treated with standard of care from a long-term natural history study (the Cooperative International Neuromuscular Research Group (CINRG) natural history database of Duchenne patients).
  • Data from the STRIDE Registry, the first international drug registry for Duchenne patients receiving Translarna, demonstrated that boys treated with Translarna and standard of care (SoC) preserved the ability to walk for years longer than those on SoC alone, as well as experienced a slower decline in lung function.
  • Children treated with Translarna in a real-world setting as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort in the CINRG natural history study, with a median age at loss of ambulation of 14.5 years and 11 years, respectively (72% relative risk reduction).
  • There was a trend toward a delay in the age at the decline in pulmonary function in STRIDE patients compared with CINRG patients, as measured by predicted FVC < 50% and FVC < 1 L.4
  • These data suggest that treatment with ataluren, in addition to SoC, may delay loss of ambulation, as well as pulmonary functional decline, in patients with nmDMD.
  •  View the original content here.

About Translarna (ataluren)

Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna, the tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older. Ataluren is an investigational new drug in the United States.

 

Ataluren in Canada

At this moment, PTC Therapeutics has not applied for marketing approval with Health Canada.

 

About PTC Therapeutics

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and their mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit them at www.ptcbio.com. Read more here > La Force DMD Blog / PTC Therapeutics.

 

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Sarepta is seeking the approval of casimersen for DMD patients

Sarepta Therapeutics Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045) for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45

  • Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne
  • Casimersen is the third exon-skipping medicine using the Company’s proprietary PMO RNA-based platform. 

 

CAMBRIDGE, Mass., June 26, 2020 (GLOBE NEWSWIRE) >Original press release here

Sarepta Therapeutics, Inc. has completed the submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is engineered to treat patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene.

The completion of the rolling submission includes data from the casimersen arm of the ESSENCE study (also known as study 4045-301), a global, randomized, double-blind, placebo-controlled Phase 3 study evaluating efficacy and safety in patients amenable to skipping exons 45 and 53. An interim analysis from ESSENCE demonstrated a statistically significant increase in dystrophin production as measured by western blot* in patients who received casimersen compared to baseline and placebo. The study is ongoing and remains blinded to collect additional efficacy and safety data. If the casimersen NDA is accepted and granted accelerated approval, the completed ESSENCE study will serve as a post-marketing confirmatory study.

 

Doug Ingram, president and chief executive officer, Sarepta Therapeutics. – “The completion of our casimersen submission is an important milestone in our journey to advance treatments for the greatest possible number of people living with Duchenne muscular dystrophy. If approved, casimersen will be our third approved therapy for sub-populations of Duchenne. Together with our other approved therapies, we have the potential to treat nearly 30% of Duchenne patients in the United States. Our proprietary PMO platform is an important focus of our pipeline, and we owe our clinical progress to the patients and families participating in our studies.”

 

About Casimersen

Casimersen uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 45 of the DMD gene. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA* processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow the production of an internally truncated dystrophin protein.

What about Canada?

At this moment, casimersen is not available in Canada. We hope that shortly, Sarepta Therapeutics will file a request for marketing approval with Health Canada.

For a better understanding

  • * The western blot is a widely used analytical technique in molecular biology, immunogenetics and other molecular biology disciplines to detect specific proteins in a sample of tissue homogenate or extract.
  • * Ribonucleic acid (RNA) is a polymeric molecule essential in various biological roles in coding, decoding, regulation and expression of genes. RNA and DNA are nucleic acids and, along with lipids, proteins and carbohydrates, constitute the four major macromolecules essential for all known forms of life. Like DNA, RNA is assembled as a chain of nucleotides, but unlike DNA, it is more often found in nature as a single-strand folded onto itself, rather than a paired double-strand.
  • Read more here
  • Grounded in the DMD community

About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is sporadic that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

About Sarepta Therapeutics

The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow them on TwitterLinkedInInstagram and Facebook.

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SIDEROS, the largest ongoing trial in Duchenne

Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa® (Idebenone) in Duchenne Muscular Dystrophy (DMD)

Pratteln, Switzerland, May 20, 2020

Original press release

Santhera Pharmaceuticals announces the full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The sample-size and variability re-assessment performed according to study protocol demonstrated that with the currently enrolled patients the study has a very high power (>99%). Given the strong powering of SIDEROS, the Company is now assessing the potential of conducting an interim analysis to test for overwhelming efficacy with a view of completing the trial early.

With patient recruitment into the 18-month international SIDEROS trial in its final stages, Santhera performed the planned sample size and variability re-assessment in accordance with the study protocol to confirm adequate study power. This blinded analysis showed that variability is lower than anticipated per protocol and, with the current number of enrolled patients, the SIDEROS study has a very high power of over 99% to detect a treatment difference. On this basis, Santhera has taken the decision to complete enrollment into the SIDEROS trial. At present, approximately half of the recruited patients in SIDEROS have completed 18 months of treatment and about two-thirds of patients have completed 12 months of treatment.

Owing to the decision to complete enrollment of this advanced study, its very high power as well as the urgent unmet medical need, Santhera is assessing the potential of conducting an interim analysis to test for overwhelming efficacy with a view of completing the trial early. Such an interim analysis would be performed by the independent Data and Safety Monitoring Board (DSMB) to preserve the integrity of the study. If overwhelming efficacy is not established in the interim analysis, the study could continue as planned with the currently enrolled patients and the corresponding high power. However, if overwhelming efficacy is demonstrated, it would be considered unethical to continue with the blinded study and the Company would decide to end the study later this year. This would result in the acceleration of corresponding regulatory filings by approximately one year both in Europe and the US.

 

Gunnar Buyse, MD, Ph.D., Professor of Child Neurology at the University Hospitals Leuven (Belgium), SIDEROS Principal Investigator and Lead Investigator for Europe – “Santhera is the only company that has dedicated its clinical development program towards finding a treatment to preserve respiratory function in DMD. The large Phase 3 SIDEROS study was designed to confirm the efficacy of idebenone in patients with respiratory function decline who are concurrently taking glucocorticoids. We are truly excited that SIDEROS has completed recruitment and is on track to generate a comprehensive dataset in an area of such high unmet need.”

 

Oscar Henry Mayer, MD, Medical Director of the Pulmonary Function Testing Laboratory at the Children’s Hospital of Philadelphia and Lead Investigator for the US – “There are currently no approved treatments to slow the rate of respiratory function decline leading to respiratory failure, which remains a leading cause of premature death in young men at the advanced stages of DMD. By slowing the rate of respiratory function decline, we open the possibility of delaying the time to chronic respiratory failure and the need to assisted ventilation and reducing the risk of other life-threatening respiratory complications.”

 

Sjöblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera – “We are delighted to have reached such an important milestone and wish to express our sincere thanks to patients and families, caregivers, physicians and study personnel for their support and commitment.”

 

About SIDEROS

SIDEROS, the largest currently ongoing clinical trial in DMD, is a double-blind randomized placebo-controlled Phase 3 study evaluating the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD. Patients on any stable glucocorticoid treatment scheme and irrespective of the underlying dystrophin mutation or ambulatory status were randomized to receive oral idebenone (900 mg/day three times a day) or placebo for 18 months. The primary endpoint of the trial estimates the treatment difference in FVC%p (forced vital capacity % predicted). Patients completing the trial are offered the opportunity to enroll in an open-label extension study where all patients receive idebenone. The study is currently conducted in 62 sites in the United States, Europe and Israel. Further information is available at ClinicalTrials.gov NCT#02814019.

About idebenone

Idebenone is a synthetic molecular formula similar to coenzyme Q10. Chemically, it is an organic compound of the family of quinones that can slow the loss of respiratory function.

Mitochondria are specialized structures in the human body that serve as batteries, powering various functions of the cell and the organism as a whole.

Mitochondria produce the energy necessary for the cell functioning through a process called “cellular respiration” which requires oxygen and provides energy. During cellular respiration, some toxic forms of oxygen (called oxygen free radicals) can be produced. These free radicals must be neutralized by other substances to avoid cellular damage.

Idebenone is expected to act as a neutralizer of these toxic forms of oxygen. Thus, idebenone is expected to have an antioxidant effect, and consequently prevent cellular damage.

Idebenone is optimized to dissolve in water and lipids and able to cross the mitochondrial membrane.

Idebenone is a medicine that is under investigation for the treatment of DMD. It has not yet been approved by the U.S. FDA, and the safety and efficacy continue to be evaluated in clinical trials.

More about idebenone

About Santhera Pharmaceuticals

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with a high unmet medical need. Santhera is building Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class anti-inflammatory drug candidate with a novel mode of action, currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. The clinical-stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone® (idebenone), for the treatment of Leber’s hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit www.santhera.com. Raxone® and Puldysa® are trademarks of Santhera Pharmaceuticals.

Sources

EU Globe News Wire

Santhera’s SYROS Study Shows Long-term Efficacy with Idebenone in Slowing Respiratory Function Loss in Patients with Duchenne Muscular Dystrophy

www.siderosdmd.com

Raxone-guides-spring-2019

www.takeabreathdmd.com

www.breatheduchenne.com

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Latest edition of the Catabasis Connection newsletter

La Force is happy to share the latest edition of the Catabasis Connection newsletter with updates on edasalonexent and information that Catabasis shared at the MDA Virtual Poster Session.

Earlier this month, Catabasis shared data from three scientific posters during the Muscular Dystrophy Association (MDA) Virtual Poster Session. Learn more about edasalonexent and its potential to provide positive effects in Duchenne here.

In this newsletter you can read:

  • Boys with Duchenne demonstrate capsule swallowing abilities
  • Phase 3 PolarisDMD trial enrolled expected patient population
  • Age-normative growth and normal adrenal function observed with edasalonexent

About Edasalonexent (CAT-1004)

Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. Our ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results, please visit www.catabasis.com.

About Catabasis

The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families. There lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and the Phase 3 trial, please visit www.catabasis.com.

About La Force DMD

The Force’s mission is to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate in the funding of promising research projects. Where access to treatments for rare diseases is concerned, it is essential that our community be strong: each member must be an active spokesperson who helps raise awareness for DMD among the general public, as well as for the challenges associated with access to treatment.

 

Edasalonexent is an investigational drug that is not yet approved in any territory.

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Translarna™ Preserves the ability to walk for longer in children with DMD

PTC Therapeutics Announces First Publication of Real-World Data Showing Translarna™ (ataluren) Significantly Preserves Ability to Walk for Longer in Children with Duchenne Muscular Dystrophy

 

  • STRIDE registry analysis shows Translarna preserved ambulation and physical function by years compared with those in CINRG Duchenne Natural History Study, with no new safety signals
  • The trend toward delayed worsening of pulmonary function compared with natural history study
  • Read the full press release here

 

PTC Therapeutics, Inc. announced real-world data showing that boys with nonsense mutation Duchenne muscular dystrophy treated with Translarna™ (ataluren) and standard of care (SoC), preserved the ability to walk for years longer than those on SoC alone. Pulmonary function was also preserved in those treated with Translarna. The analysis, presented in the publication of an interim analysis of preliminary real-world data, compared children treated with Translarna in a real-world setting from the STRIDE Registry with a matched cohort in a long-term natural history study, CINRG. In addition, no new safety signals were observed in the patients treated with Translarna, consistent with what has been shown in previous clinical trials. The interim data have been published in the Journal for Comparative Effectiveness Research. The final data from the STRIDE registry is expected in 2025.

 

Dr. Andrés Nascimento, Pediatric Neurology, Neuromuscular Diseases Unit, SJD Children’s Hospital, Barcelona, Spain – “Duchenne muscular dystrophy is a devastating disease that causes irreversible muscle wasting and progressively robs young people of their ability to walk, move, and breathe naturally without a ventilator, and it reduces their autonomy in daily life tasks. In a real-world setting, children and adolescents treated with Translarna experience a delay in the disease progression, are able to maintain more mobility and have a higher level of physical autonomy concerning the course of the natural history of the disease. This is not only clinically relevant but especially important for the quality of life of patients and their families.”

 

Children treated with Translarna in a real-world setting as part of the STRIDE registry were able to walk independently for an additional 3.5 years compared with a propensity-score matched cohort in the CINRG natural history study, with a median age at loss of ambulation of 14.5 years and 11 years.

Additional analyses from the registry demonstrated that Translarna sustained the ability of boys with Duchenne to complete everyday tasks by years compared with the natural history cohort.  In timed function tests, Translarna sustained their ability to stand up from lying down, in under 5 and 10 seconds, for three years longer than in boys treated with SoC alone. Boys treated with Translarna were also still able to climb four stairs in under 5 and 10 seconds for 1.5 and 3.6 years longer, respectively, than boys on SoC alone.

Also, the analysis showed a trend toward delayed worsening of pulmonary function in routine clinical practice for patients treated with Translarna, compared to the matched patients in CINRG.  After the loss of ambulation and loss of the use of the arms, the respiratory muscles of people with Duchenne start to progressively deteriorate, leading to the risk of life-threatening respiratory complications and the need for ventilation support. 

 

Dr. Claudio Santos, SVP, Global Medical Affairs, PTC Therapeutics – “The data from the STRIDE registry are consistently confirming the benefits seen in Translarna clinical trials and the difference it is making to patients and their families – more years of being independent and physically able without reliance on a wheelchair or ventilator.”

 

About Translarna (ataluren)

Translarna (ataluren), discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged two years and older. Ataluren is an investigational new drug in the United States. In the video, Dr. Ellen Welch answers our questions about nonsense mutation and ataluren > Here

 

Ataluren in Canada

At this moment, PTC Therapeutics has not filed an application for marketing approval with Health Canada, but it has started a conversation with the legislator.

 

About the STRIDE Registry

The STRIDE (Strategic Targeting of Registries and International Database of Excellence) Registry is an ongoing, multicenter, observational study of the safety and effectiveness of Translarna in routine care.  It is the first patient data repository to provide real-world experience regarding the long-term use of Translarna in routine clinical practice.

Effectiveness information may include neuromuscular function measures, cardiac function, pulmonary function, and quality of life measures. Assessments of musculoskeletal health, rehabilitation, orthopedic and gastrointestinal management, as well as other measures of psychosocial management, will be collected to allow for comparison of patient health-management activities in routine clinical care to those of published treatment guidelines.

STRIDE is a collaborative partnership between TREAT-NMD and PTC Therapeutics, led by a Steering Committee comprised of leading experts in Duchenne, patient advocates from around the world and PTC representatives.

The Registry also fulfils a post-marketing commitment to the Pharmacovigilance Risk Assessment Committee of the European Medicines Agency.

 

About TREAT-NMD

TREAT-NMD is a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since its launch, in January 2007 the network’s focus has been on the development of tools that industry, clinicians and scientists need to bring novel therapeutic approaches through preclinical development and into the clinic, and on establishing best-practice care for neuromuscular patients worldwide. The network has developed from its European roots to become a global organization that brings together leading specialists, patient groups and industry representatives to ensure preparedness for the trials and therapies of the future while promoting best practice today.

Further information about TREAT-NMD can be found here > TREAT-NMD

 

About CINRG

The Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study was a prospective, longitudinal study of more than 400 patients with Duchenne muscular dystrophy (DMD) who were followed up between 2006 and 2016 at 20 worldwide centers as part of the academic clinical trial network, CINRG.

 

More links

  • Read the full press release here
  • Pioneer in DMD Therapy here
  • Learn more about nonsense mutation here
  • Learn more about ataluren (Translarna™) at ptcbio.com

SOURCE PTC Therapeutics, Inc.

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FDA grants accelerated approval to Vyondys 53

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53

Source: Sarepta Therapeutics, Inc., Dec 12, 2019 – Read the original news here– 

The U.S. Food and Drug Administration granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. It is estimated that about 8% of patients with DMD have this mutation.

 

Billy Dunn, M.D., acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research – “The FDA recognizes the urgent need for new medical treatments for serious neurological disorders and we have a long-standing commitment to working with researchers, drug companies and patients to facilitate the development and approval of treatments for rare diseases. With today’s accelerated approval, patients with Duchenne muscular dystrophy who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping will now have available the first treatment targeted specifically for this disease subtype. Use of the accelerated approval pathway will make Vyondys 53 available to patients based on initial data and we look forward to learning more about the drug’s clinical benefit from the ongoing confirmatory clinical trial.”

 

Doug Ingram, president and chief executive officer, Sarepta – “Today is monumental for Sarepta and, more importantly, for the DMD community. VYONDYS 53, our second approved exon-skipping RNA therapy for DMD, may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutations.  Along with EXONDYS 51® (eteplirsen), we now offer treatment options for approximately 20% of those with DMD in the U.S.”

 

Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD link here) – “With the approval of VYONDYS 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease. For 25 years, PPMD has been working with researchers, clinicians, industry, and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne.”

 

More about Vyondys 53 (golodirsen)

Like Exondys 51, golodirsen, which Sarepta hopes to sell under the name Vyondys 53, is designed to treat a group of Duchenne patients with a particular type of mutation. Exondys 51 works for about 13% of DMD patients—those whose disease is amenable to exon 51 skipping. If approved, golodirsen would offer treatment to patients with a mutation in exon 53—about 8% of the DMD population.

VYONDYS 53 is priced at parity to EXONDYS 51, the price of which has not increased since its launch in 2016. Patients and physicians can access more information at www.SareptAssist.com or by calling 1-888-727-3782.

Important Safety Information for VYONDYS 53

Approval of Vyondys 53

Vyondys 53 was approved under the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally offer a meaningful advantage over existing treatments. Approval under this pathway can be based on adequate and well-controlled studies showing the drug affects a surrogate endpoint that is reasonably likely to predict clinical benefit to patients. This pathway provides earlier patient access to promising new drugs while the company conducts clinical trials to verify the predicted clinical benefit.

The accelerated approval of Vyondys 53 is based on the surrogate endpoint of an increase in dystrophin production in the skeletal muscle observed in some patients treated with the drug. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. A clinical benefit of the drug, including improved motor function, has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease and the lack of available therapy. Read more here.

The Status

  • VYONDYS 53 is approved under accelerated review based on an increase in dystrophin production in skeletal muscle of patients amenable to exon 53 skipping. Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.
  • VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.
  • The commercial distribution of VYONDYS 53 in the U.S. will commence immediately
  • Information for patients and clinicians is available at www.SareptAssist.com

What is exon skipping?

Mutations in the dystrophin gene are one cause of DMD. Most commonly, one or more exons (a portion of a gene) are missing, and the remaining exons don’t fit together correctly. (Think of a zipper that doesn’t work properly, because teeth are missing.)

Because of this error, cells cannot make the dystrophin protein that muscles need to work properly. Without it, muscle cells become damaged and, over time, are replaced with scar tissue and fat.

To fix the broken genetic machinery, scientists are developing drugs that skip over parts that contain missing or defective exons. In this way, the machinery can produce a less imperfect dystrophin protein, which may improve muscle function in children with exon mutations. > Pipeline exon-skipping

What about Canada?

At this moment, Vyondys 53 is not available in Canada. We hope that shortly, Sarepta Therapeutics will file a request for marketing approval with Health Canada.

More interesting links

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Positive data with vamorolone in DMD

Santhera Announces Presentation by ReveraGen of Positive 18-Month Data with Vamorolone in Duchenne Muscular Dystrophy

 

La Force is happy to share this press release provided by Santhera Pharmaceuticals, October 7, 2019, > PRESS RELEASE <

 

Santhera Pharmaceuticals announces the presentation of data showing continued improvement of muscle function and improved tolerability compared with corticosteroids of 18-month vamorolone treatment in Duchenne muscular dystrophy (DMD). These top-line data were presented on October 5 by Eric Hoffman, Ph.D., CEO of ReveraGen at the World Muscle Society (WMS) congress.

In a late-breaking presentation at the WMS international conference in Copenhagen, Denmark, ReveraGen presented motor function and tolerability data from 23 DMD patients treated with 2.0 or 6.0 mg/kg/day with vamorolone for at least 18 months in the ongoing VBP15-LTE study.

 

Eric Hoffman, Ph.D., Chief Executive Officer of ReveraGen – “These data demonstrate that vamorolone treatment results in persistently improved motor function in DMD patients, similar to that of corticosteroids. Importantly, however, vamorolone treatment over a period of 18 months showed better tolerability with less corticosteroid-specific side effects, including no stunting of the growth of DMD children.”

 

Vamorolone is a first-in-class steroidal anti-inflammatory investigational drug in development as a treatment for DMD to substitute standard corticosteroids (prednisone, deflazacort). This trial is an extension study of the VBP15-003 trial in which 48 DMD patients treated for 6 months over a broad dose range (0.25 to 6.0 mg/kg/day) showed dose-related improvements in multiple gross motor outcomes. Upon exiting this 6-month trial, patients and their physicians preferred to continue vamorolone treatment. 45 boys transitioned to the 2-year long-term extension study VBP15-LTE and all doses were increased to 2.0 or 6.0 mg/kg/day of vamorolone.

At the WMS conference, Dr. Hoffman presented data from 23 patients treated with 2.0 or 6.0 mg/kg/day vamorolone for at least 18 months. Vamorolone treatment consistently and significantly improved standardized motor function outcomes measured as velocity to stand from supine, to run/walk 10 meters and to climb 4 stairs from baseline to month 18. Motor function outcomes for vamorolone treated patients also were consistently better than outcomes for age-matched, steroid naïve patients from an external natural history study (velocity to stand from supine: p=0.085; run/walk 10 meters: p=0.005; climb 4 stairs: p=0.036; all in favour of vamorolone treatment).

Vamorolone compared to standard corticosteroids

Motor function outcomes of vamorolone-treated DMD boys were compared to age-matched prednisone-treated patients from an external control group. Both vamorolone and prednisone treated groups showed similar improvements in these gross motor outcomes, demonstrating that vamorolone exerts therapeutic efficacy similar to standard corticosteroids. Importantly, vamorolone-treated boys showed normal growth rates, and less physician-reported weight gain and Cushingoid features compared to published studies of prednisone and deflazacort. These findings confirm earlier data that indicate a better tolerability profile of vamorolone compared to standard corticosteroids.

Together with previously reported molecular and clinical data, the new 18-month data suggest that dissociative steroidal drug vamorolone maintains efficacy and decreases adverse effects typically reported for corticosteroids in the treatment of DMD. Vamorolone has been granted Orphan Drug status in the US and Europe and has received Fast Track and Rare Pediatric Disease designations by the US FDA.

About Vamorolone – first-in-class dissociative steroid

Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is a significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from treatment compliance and patient quality of life.

The currently ongoing 48-week Phase IIb VISION-DMD study is designed as a pivotal trial to demonstrate efficacy and safety of vamorolone compared with prednisone and placebo in 120 boys aged 4 to <7 with DMD that have not yet been treated with corticosteroids. Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with participation in funding and design of studies by several international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program. In November 2018, Santhera acquired from Idorsia the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide (except Japan and South Korea).

Clinical trial in Canada

About Santhera

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with a high unmet medical need. Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class dissociative steroid currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. Santhera out-licensed ex-North American rights to its first approved product, For further information, please visit www.santhera.com. Raxone® and Puldysa® are trademarks of Santhera Pharmaceuticals

About ReveraGen BioPharma

ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan’s Quest, Alex’s Wish, DuchenneUK, Pietro’s Fight, Michael’s Cause, and Duchenne Research Fund. ReveraGen has also received generous support from the US Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).

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