Industry Partner News: Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)

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December 6, 2018

Dear Duchenne Community,

We would like to share an exciting update about our lead Duchenne muscular dystrophy (DMD) clinical program. This morning, Wave Life Sciences announced positive safety and tolerability results from the WVE-210201 Phase 1 clinical trial in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

The data from this trial support our moving forward with a Phase 2/3 clinical trial of WVE-210201, which we intend to initiate in 2019, and propel us towards achieving our collective goal of inducing meaningful, natural dystrophin expression in boys with DMD.

For more information, we invite you to read our announcement here.

Importantly, thank you to all of the boys and their families who are participating in this Phase 1 trial and its open-label extension, as well as the advocacy organizations that have provided invaluable guidance and collaboration along the way. Your contributions and partnership are deeply appreciated.

At Wave Life Sciences, we are focused on delivering transformational therapies for patients with serious, genetically-defined diseases. Today’s announcement has taken us one step closer to achieving this objective. We look forward to continuing our engagement with you as we work together to make a difference for people impacted by Duchenne.

Sincerely,

Paul Bolno, MD, MBA
President and Chief Executive Officer

Wendy Erler, MBA
Vice President, Patient Advocacy and Market Insights
Wave Life Sciences

Industry Partner News: Santhera Enters into Agreement to Acquire Option from Idorsia for Exclusive Sub-License of First-in-class Dissociative Steroid Vamorolone

Santhera will hold a webcast tomorrow, November 21, 2018 at 13:00 CET, 12:00 GMT, 07:00 EST. Details at the end of statement.

Santhera Enters into Agreement to Acquire Option from Idorsia for Exclusive Sub-License of First-in-class Dissociative Steroid Vamorolone

• Vamorolone in clinical development for Duchenne muscular dystrophy (DMD) by ReveraGen BioPharma Inc. – pivotal VISION-DMD Phase IIb study ongoing

• Vamorolone has the potential to become standard of care in young patients with DMD

• Positions Santhera as a leading company in the DMD space with two late-stage assets addressing the medical need of DMD patients at all disease stages

• Idorsia to become the largest shareholder in Santhera with a 13.3% equity stake

Pratteln and Allschwil, Switzerland, November 20, 2018 – Santhera Pharmaceuticals (SIX: SANN) and Idorsia Ltd (SIX: IDIA) have entered into an agreement under which Santhera will acquire the option to exclusively in-license, by way of sub-license, the first-in-class dissociative steroid vamorolone in all indications and all countries worldwide except Japan and South Korea. Initial clinical data suggest that vamorolone has the anti-inflammatory efficacy of steroids with reduced steroid-associated safety concerns, which would represent a significant improvement over current standard of care glucocorticoid therapy in patients with Duchenne muscular dystrophy (DMD), vamorolone’s lead indication.

Thomas Meier, PhD, Chief Executive Officer of Santhera, said: “Vamorolone is a highly promising drug candidate for the treatment of patients with DMD and a perfect strategic fit alongside idebenone. Our late-stage DMD drug portfolio covers a broad DMD patient spectrum, irrespective of genetic background, disease stage or age. This agreement underscores our strategy of in-licensing high-quality, late-stage rare disease assets, which leverage our existing capabilities and expertise. We are also delighted to welcome Idorsia as our largest shareholder and partner and look forward to working with ReveraGen in the development of vamorolone, which has the potential to replace standard glucocorticoids as treatment for DMD.”

Vamorolone – first-in-class dissociative steroid Vamorolone is a first-in-class drug candidate that binds to the same receptors as glucocorticoids 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 glucocorticoids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose glucocorticoids have severe systemic side effects, which limit long-term usage.

Vamorolone was discovered by US-based ReveraGen BioPharma Inc. and has been developed with participation in funding and design of studies by 12 international non-profit foundations, the US National Institutes of Health, the US Department of Defense and the European Commission’s Horizon 2020 program. Actelion had acquired an option to license the product in 2016. This option was subsequently transferred to Idorsia following the acquisition of Actelion by Johnson & Johnson in 2017.

Eric Hoffman, PhD, Chief Executive Officer of ReveraGen, commented: “Our hope for vamorolone is that it can replace existing glucocorticoids in DMD therapy. Early clinical development of vamorolone in patients with DMD, using an innovative approach with an array of pre-selected biomarkers in multiple contexts of use, suggests that vamorolone preserves anti-inflammatory efficacy while decreasing steroid- associated safety concerns. I am delighted to work with Santhera to advance this exciting therapeutic candidate for patients with DMD.”

Vamorolone in DMD Following single and multiple ascending dose clinical pharmacology studies (VBP15-001) in healthy volunteers [1] vamorolone completed a Phase IIa study (VBP15-002) in 48 boys with DMD aged 4 to <7 years. Vamorolone was reported to be safe and well tolerated up to 6.0mg/kg/day, around 10 times the standard glucocorticoid dose [2]. A 6-month extension study (VBP15-003) also demonstrated dose- dependent improvement in timed function tests which was comparable to standard glucocorticoid treatment.

The ongoing Phase IIb VISION-DMD study (VBP15-004) builds on the available promising preliminary safety and efficacy data from Phase IIa and is designed to bridge exploratory biomarker data to clinical outcomes. This pivotal study will enroll approximately 120 boys aged 4 to <7 with DMD that have not yet been treated with glucocorticoids, randomized to one of four groups: low dose vamorolone (2 mg/kg/day), high dose vamorolone (6 mg/kg/day), prednisone (0.75 mg/kg/day), or placebo. After the initial 24-week treatment period, the prednisone and placebo groups will cross-over to low dose or high dose vamorolone. The second treatment period then has all patients treated for an additional 20 weeks with vamorolone. Clinical outcomes for efficacy include timed function tests and measures of muscle strength and endurance. Clinical outcomes for safety include monitoring of bone changes, weight changes, cataracts, and biomarkers of metabolic disturbances.

The study is being conducted at approximately 30 sites across North America, Europe, Israel and Australia. Enrolment, which began in August 2018, is expected to take about 12 months, with a total study duration of about 24 months. If successful, the data filing with health authorities in the US is anticipated by the end of 2020 and in the EU in 2021. Vamorolone has received Orphan Drug Designation in the US and in Europe and fast-track status in the US.

Santhera management anticipates peak sales potential for vamorolone for the DMD indication of USD 500 million.

The Agreement Under the terms of the agreement, Idorsia will grant Santhera the option to obtain an exclusive sub- license for vamorolone in all indications and all territories except Japan and South Korea. Idorsia will receive as consideration for entering into the agreement 1,000,000 (one million) new registered shares from Santhera’s existing authorized share capital and an upfront cash component of USD 20 million, of which USD 15 million is intended to compensate Idorsia for its investment into the Phase IIb VISION-DMD study currently conducted by ReveraGen. While the cash component of the consideration is subject to financing, the share component of the consideration is unconditional and, like the cash component, not redeemable under any circumstances. As a consequence of the transaction, Idorsia will become the largest shareholder in Santhera with a 13.3% equity position. The shares to be issued to Idorsia will be subject to a lock-up undertaking expiring if and when vamorolone receives marketing authorization in DMD in the United States. Santhera may exercise the option upon receipt of data from the Phase IIb VISION-DMD study (VBP15-004) and following a one-time consideration to Idorsia of USD 30 million.

Following the exercise of the worldwide vamorolone license option by Idorsia and exercise of the vamorolone sub-license option for all territories worldwide except Japan and South Korea by Santhera, Santhera will pay to Idorsia regulatory and commercial milestone payments of up to USD 80 million in the DMD indication and four one-time sales milestone payments of up to USD 130 million in aggregate. Regulatory milestone payments by Santhera to Idorsia for three additional indications amount to up to USD 205 million in aggregate. Upon commercialization of vamorolone, Santhera has committed to pay tiered royalties ranging from a single-digit percentage to low double-digit percentage on the annual net sales of vamorolone to Idorsia.

Jean-Paul Clozel, MD, Chief Executive Officer of Idorsia, concluded: “With four compounds in late-stage clinical development and more innovative compounds coming through the pipeline, Idorsia’s newly established commercial function has many assets to focus on. We have decided to hand the option to license vamorolone to Santhera because they are ideally placed to maximize the potential of this asset. If successful, Santhera’s network and expertise in the field of DMD will allow patients to benefit from this potential new treatment approach as soon as possible. In addition, with this agreement we become Santhera’s largest shareholder, so we remain highly motivated and committed to make vamorolone a success.”

Centerview Partners acted as exclusive strategic and financial advisor to Santhera for this transaction.

Santhera Webcast & Conference Call
Santhera will hold an audio webcast / conference call tomorrow, November 21, 2018 at 13:00 CET, 12:00 GMT, 07:00 EST to discuss the agreement on vamorolone. Participants are invited to join either the audio webcast or telephone conference 10-15 minutes before the start: Webcast: click this link to access the webcast Telephone conference: dial one of the following numbers (no code required): Europe: +41 58 310 50 00 UK: +44 207 107 0613 USA: +1 631 570 5613

For the full Press Release please click here. For further information, please visit santhera.com.

Industry Partner News: Solid Biosciences Reports Third Quarter 2018 Financial Results And Provides Business Update

Two Additional Patients Dosed With SGT-001 Gene Transfer in IGNITE DMD Clinical Trial

Company Plans to Report Preliminary Results from IGNITE DMD in the First Quarter of 2019

CAMBRIDGE, Mass., Nov. 13, 2018 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ: SLDB) today reported financial results for the third quarter ended September 30, 2018 and provided a business update.

“We are pleased to have made significant progress toward our goal of bringing meaningful treatments to patients with Duchenne muscular dystrophy,” said Ilan Ganot, Chief Executive Officer, Co-Founder and President of Solid Biosciences. “Most notably, we resumed dosing in the Phase I/II IGNITE DMD clinical trial for our SGT-001 microdystrophin gene therapy program. This progress was complemented by continued work on our innovative and scalable manufacturing process, which enabled us to move forward with the study as planned and without delay. We believe that the unique attributes included in SGT-001 could translate to significant benefit for patients and now look forward to providing preliminary biopsy data from IGNITE DMD in the first quarter of 2019. We also remain on track to provide data from our previously communicated interim analysis in the second half of 2019.”

“We are pleased with the progress we have made on IGNITE DMD, dosing two additional patients with SGT-001 since the study resumed,” said Jorge Quiroz, M.D., Chief Medical Officer of Solid Biosciences. “Six patients have now been randomized in IGNITE DMD, three to the active treatment group, all of whom are doing well, and three to the delayed treatment control group. Continuing to enroll IGNITE DMD is a top priority, and we look forward to understanding the potential of SGT-001 in the clinic. As always, we remain grateful to the patients and families participating in IGNITE DMD and the team at the University of Florida.”

Recent Developments

  • Solid is continuing to enroll IGNITE DMD, the Company’s randomized, controlled Phase I/II clinical trial to assess the safety and efficacy of SGT-001 (AAV-mediated microdystrophin gene transfer) for the treatment of Duchenne muscular dystrophy (DMD). In total, six patients have been randomized in IGNITE DMD, three to the active treatment group and three to the delayed treatment control group. This number includes two additional patients who have been dosed since the study resumed in June. All three patients who have received SGT-001 are currently doing well.

    There were no serious adverse events observed in the second or third patients dosed. Laboratory findings, including a transient decline in platelet count that has fully resolved, were quickly identified and managed per the study protocol. Solid continues to enroll patients in IGNITE DMD and plans to communicate preliminary results, including microdystrophin expression data, in the first quarter of 2019.          

  • In September, Solid announced that the Company appointed clinical and gene therapy expert Sukumar Nagendran, M.D., to its Board of Directors. Dr. Nagendran brings to Solid more than 30 years of experience in key functional areas, including gene therapy development, clinical strategy, medical affairs and diagnostics. Most recently, Dr. Nagendran was Chief Medical Officer & Senior Vice President of AveXis Inc., where he was responsible for overseeing and driving all clinical development and medical affairs strategy, notably for the company’s late-stage AAV-mediated gene therapy program for Spinal Muscular Atrophy (SMA). 
     

  • In October, Solid received Fast Track Designation for SGT-001 from the U.S. Food and Drug Administration (FDA). The Fast Track program is designed to expedite the development and review of drugs to treat serious or life-threatening conditions and fill an unmet medical need.

Financial Highlights 
Solid Biosciences reported a net loss of $19.0 million for the third quarter of 2018 as compared to $13.5 million for the third quarter of 2017. The net loss was due to research and development expenses, as well as investments in the Company’s infrastructure.

Research and development expenses for the third quarter of 2018 were $14.7 million as compared to $10.6 million for the prior year period. This increase was primarily due to personnel and facility related expenses and costs related to clinical development and manufacturing activities for SGT-001, as well as our other product candidates. These increases were offset by a reduction in preclinical costs associated with SGT-001.

General and administrative expenses were $4.5 million for the third quarter of 2018 as compared to $3.1 million for the prior year period. This increase was primarily due to personnel and facility related costs, as well as other corporate expenses.  

Solid ended the third quarter of 2018 with $145.4 million in cash, cash equivalents and available-for-sale securities as compared to $69.1 million as of December 31, 2017. The increase was primarily the result of the completion of the Company’s initial public offering on January 30, 2018.

About SGT-001
Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of Duchenne muscular dystrophy (DMD), mutations in the dystrophin gene that result in the absence or near-absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin transgene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). SGT-001 utilizes AAV9, which has an affinity for muscle and is currently being evaluated in multiple clinical programs in other indications. Data from Solid’s preclinical program suggest that SGT-001 has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

About Solid Biosciences
Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices. For more information, please visit www.solidbio.com.

Industry Partner News: Sarepta Therapeutics Announces Third Quarter 2018 Financial Results and Recent Corporate Developments

Sarepta Therapeutics reports financial results for the third quarter of 2018.

“We are pleased to report another positive quarter, delivering strong EXONDYS 51 sales and tracking to achieve our full-year sales objectives while both advancing our RNA pipeline and making substantial progress in the creation of an enduring gene therapy engine,” stated Doug Ingram, Sarepta’s president and chief executive officer. “We continued this quarter to advance our RNA pipeline, PMOs and nextgeneration PPMO platform, with urgency. Further, the strides we have taken in service of our gene therapy engine, including unprecedented resultsin our micro-dystrophin program, rightsto what are now 14 gene therapy programs, the addition of manufacturing partners, and the continued hiring of gene therapy talent, speak to our vision. Others may be content with steady progress. We see a revolution and it is our intention to lead that revolution to the benefit of countless genetic disease patients awaiting lifeenhancing therapies.”

Read the full press release here.

Industry Partner News: Jerry Mendell, M.D., Presented Positive Updated Results at World Muscle Society from Four Children Dosed in Sarepta's Gene Therapy Micro-dystrophin Trial for DMD

 
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Dear Duchenne Community,

At the World Muscle Society in Argentina today, Dr. Jerry Mendell of Nationwide Children’s Hospital shared additional data relating to our micro-dystrophin gene therapy program. In particular, as a follow up to his presentation of the first three patients at our R&D Day in June of this year, Dr. Mendell today shared micro-dystrophin results for the fourth patient and provided positive functional signals for all patients.

It is important to remember that while we are very encouraged by all of the results we have to date, these are early days relating to our first four patients. It is important that we quickly commence a controlled trial to confirm these results. Fueled by our preliminary data, we are moving with a sense of urgency to move to a study that, if successful, could bring this therapy to those patients who can benefit from it.

As mentioned in June, the next micro-dystrophin trial will take place in the United States, will be carried out by Nationwide Children’s Hospital, and will be limited in size. Sarepta is mapping ways to expand the gene therapy clinical program to a broader population with considerations of study inclusion and geography. We will update the community about the plans as we finalize them.

Given the volume of inquiries received about this investigational effort, we know that there is a high level of anticipation within the worldwide Duchenne community surrounding gene therapy. Physicians and advocacy groups alike have expressed hope that families will stay focused on current treatment plans and investigational options selected in consultation with their healthcare team.

Let us once again be reminded that as encouraged as we are, these are preliminary results and we must continue to follow our initial patients and commence a controlled trial. But also know this, we are investing our energy, resources and creativity to moving the development forward as fast as is possible, planning meetings with the FDA and other agencies around the world to take their input, building a compelling access and reimbursement package, and establishing sufficient manufacturing capacity to fully serve the community if our program is successful.

I will provide additional updates as the program moves forward.

Sincerely,

Douglas S. Ingram
President and Chief Executive Officer
Sarepta Therapeutics, Inc

Industry Partner News: Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy

 
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Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne

CAMBRIDGE, Mass., September 25, 2018 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy (DMD). Edasalonexent inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD. The PolarisDMD trial will evaluate the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent. Top-line results from the Phase 3 PolarisDMD trial are expected in the second quarter of 2020.

 Click to read the Catabasis Quarterly for September 2018.

Click to read the Catabasis Quarterly for September 2018.

PolarisDMD clinical trial sites across the United States will open in the coming days for enrollment of the Phase 3 PolarisDMD trial, with enrollment expected to run through this year and into next year. Additional sites in Australia, Canada, Europe and Israel are also expected to open early next year. In total, the PolarisDMD trial is expected to include approximately 40 clinical trial sites globally. The trial design was informed by discussions with the U.S. Food and Drug Administration (FDA) as well as input from treating physicians, patient organizations and families of boys affected by Duchenne.

“We are very excited to initiate our Phase 3 PolarisDMD trial as we believe edasalonexent has tremendous potential to become the new standard of care for all affected by Duchenne, regardless of mutation type and from the time of diagnosis throughout their lifespan,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “Edasalonexent inhibits NF-kB, which plays a fundamental role in skeletal and cardiac muscle disease in Duchenne. In the MoveDMD trial, edasalonexent slowed disease progression and preserved muscle function.”

The Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen may be eligible to enroll. The primary efficacy endpoint will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints include the age-appropriate timed function tests time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also included. Two boys will receive 100 mg/kg/day of edasalonexent for each boy that receives placebo and after 12 months, all boys are expected to receive edasalonexent in an open-label extension.

“We are so glad to bring edasalonexent into Phase 3 for boys affected by Duchenne that could benefit from this potential therapy,” said Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and a Principal Investigator for the Phase 2 and Phase 3 trials with edasalonexent. “With the stabilization of muscle function and excellent safety profile seen with edasalonexent treatment in the MoveDMD trial, patients and families are eager for a treatment for Duchenne that has demonstrated slowed disease progression in the clinic.”

“PPMD has conducted preference studies of those impacted by Duchenne to understand what is most important for novel therapies. Families responded that their highest priority is to stop or even slow disease progression and maintain muscle function,” Pat Furlong, Founding President and Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD). “We are enthusiastic to see edasalonexent, with these potential effects, entering the Phase 3 PolarisDMD trial as an oral therapy that could treat all of those affected by Duchenne.”

Edasalonexent has been shown to preserve muscle function and substantially slow DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to stand, 4-stair climb and 10-meter walk/run) compared to control in the MoveDMD® Phase 2 trial and open-label extension. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been well tolerated through more than 45 patient-years of treatment with no safety signals observed.

More information about the Phase 3 PolarisDMD clinical trial will be available on www.clinicaltrials.gov in the coming days or contact the Catabasis clinical team via email at DMDtrials@catabasis.com.

About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential new standard of care for all patients affected by DMD, regardless of their underlying mutation. Edasalonexent inhibits NF-kB, which is the key link between loss of dystrophin and disease progression. NF-kB has a fundamental role in skeletal and cardiac muscle disease in DMD. Catabasis is currently enrolling the single global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent continues to be dosed in an open-label extension of the MoveDMD Phase 2 clinical trial. 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
At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. The global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and our Phase 3 PolarisDMD trial, please visit www.catabasis.com or www.twitter.com/catabasispharma.

Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company’s global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward- looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward- looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release.

###

Investor and Media Contact
Andrea Matthews
Catabasis Pharmaceuticals, Inc.
T: (617) 349-1971
amatthews@catabasis.com



Industry Partner News: Sarepta Announces Clinical Hold Lifted for its DMD Micro-dystrophin Gene Therapy Program

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CAMBRIDGE, Mass., September 24, 2018 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that the Food and Drug Administration (FDA) has lifted the clinical hold for the Company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical hold due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot. In response, and in collaboration with Nationwide Children’s Hospital, an action plan was developed and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.

“Thanks to the diligent and rapid work of my Sarepta colleagues and Nationwide Children’s Hospital in compiling and submitting a complete response and the expeditious evaluation by the FDA in reviewing the response and removing this clinical hold, we have been able to address the clinical hold in record time and without delay to this profoundly important clinical program,” stated Doug Ingram, Sarepta’s president and chief executive officer. “Our focus now is on meeting with the Division to take guidance and gain alignment around what we hope to be our registration trial for our micro-dystrophin program and achieving our goal of commencing that trial by year-end 2018.”

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.

Industry Partner News: Community Letter from Sarepta Regarding CHMP's Negative Opinion for Eteplirsen

Please read the following community letter from Douglas S. Ingram, President and Chief Executive Officer of Sarepta Therapeutics, Inc., regarding the Committee for Human Medicinal Products (CHMP) in Europe’s negative opinion for eteplirsen.

21 September 2018

Dear European Duchenne Community,

This morning Sarepta announced that following its request for re-examination, the Committee for Human Medicinal Products (CHMP) in Europe issued a negative opinion for eteplirsen. While we recognized the probability of success was low, we committed our full resources and energy to this re-examination because we believe that the European Duchenne community deserves access to eteplirsen.

We are grateful for your insights, contributions and thoughtful engagement during this process. We also appreciated the open additional dialogue and advice we received from the CHMP, who signaled an openness to considering a regulatory pathway that could balance the needs of patients and their families with the requirements for additional supportive data. For eteplirsen, our next step is to initiate a request for follow up scientific advice, which we plan to do in 2019.

Given our discussions and commitment to the Duchenne community, we will continue to engage the European Medicines Agency (EMA) and explore the most appropriate path forward for eteplirsen and our other investigational medicines. We will seek scientific advice for our clinical programs as we continue to advance our pipeline of Duchenne therapies, including next-generation PPMO RNA therapy and the micro-dystrophin gene therapy candidate.

Despite today’s news, we continue to believe that there is a path forward in Europe to bring new therapies that can improve the lives of individuals living with Duchenne. Sarepta will continue to sponsor clinical trials in Europe. The ESSENCE trial is still enrolling in Europe as a method to understand the safety and efficacy of golodirsen and casimersen. There is also an eteplirsen study focused on individuals with Duchenne who are 6 months-48 months old in process. We will soon be initiating our high dose eteplirsen study, as well as trials for our next generation PPMO candidates in Europe.

We look forward to continued engagement as we advance these programs together.

Sincerely,

Douglas S. Ingram
President and Chief Executive Officer
Sarepta Therapeutics, Inc

 
 

Industry Partner News: Community Letter From Genetech/Roche Regarding RG6026

Please read the following Community Letter pertaining to Roche/Genentech's RG6206 program. 

13 September 2018

Dear Duchenne community,

By now, many have heard about Pfizer’s decision to stop their clinical studies of domagrozumab in Duchenne Muscular Dystrophy. We have received questions as to whether Pfizer’s decision has an impact on our clinical development program of RG6206 in Duchenne.

RG6206 is an investigational anti-myostatin adnectin that is designed to block the activity of myostatin and is given subcutaneously (by injection under the skin). The safety and efficacy of RG6206 in ambulatory boys (6-11 years old) is being evaluated in the Phase 2/3 SPITFIRE clinical study (www.clinicaltrials.gov reference NCT03039686, Roche study WN40227). Enrollment in the SPITFIRE clinical study is ongoing globally, and there are no planned changes to the study or its timelines as a result of Pfizer’s recent decision.

When evaluating investigational therapies, individual clinical studies are designed to address questions of safety and/or efficacy in a way that is specific and unique to the investigational molecule being studied. This means that studies with different study designs cannot be easily or directly compared. As such, it is not possible to draw comparisons or conclusions about RG6206 based on studies of other molecules with different study designs.

We are very grateful and appreciate the continuous partnership of the Duchenne community and would like to extend our thanks to all study participants and their families.

If you have questions:

  • Regarding your ongoing participation in the SPITFIRE study, or if you are in the screening phase, please contact your study physician.

  • If you are not in the SPITFIRE study, but have questions about RG6206, please contact me at elena.zhuravleva.ez1@roche.com.

Warm regards,

Elena Zhuravleva, MD, PhD on behalf of the Roche Duchenne team
Patient Partnership Director, Rare Disease, Roche, Switzerland

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Industry Partner News: Pfizer Announces Decision to Discontinue Domagrozumab Clinical Studies

Today, Pfizer announced the disappointing news that it is discontinuing its clinical studies of Domagrozumab. Please read their letter and press release below for more information.

Dear Jett Foundation,

It is with profound disappointment that we announce our decision to discontinue the
clinical studies evaluating domagrozumab for the treatment of Duchenne muscular
dystrophy (DMD). The two ongoing clinical trials include; a Phase 2 safety and efficacy
study (B5161002) and an open-label extension study (B5161004). The decision comes
following completion of the primary analysis. The primary analysis was based on a
statistical test which compared the difference in the time to climb 4 standard stairs
(known as the 4 Stair Climb) after 1 year of treatment with domagrozumab as compared
to placebo treated subjects. This analysis showed no statistical difference between these
two groups at the one year period. Evaluation of the totality of evidence, including
secondary endpoints, also did not support a significant treatment effect. It is important
to note that the studies were not discontinued for safety reasons.

The discontinued Phase 2 double-blind, placebo-controlled, multicenter, clinical trial
investigated the efficacy and safety of domagrozumab, administered in monthly IV doses,
in 121 boys aged 6 to 15 with DMD, regardless of underlying mutation. It was designed as
a two-year, placebo-controlled study (with the above noted primary analysis performed
after one year); all subjects used background corticosteroid management. The primary
efficacy endpoint was mean change from baseline in 4 Stair Climb (in seconds). Secondary
endpoints included evaluation of the change from baseline of participants on other
functional tests, including forced vital capacity, Northstar Ambulatory Assessment, the
six-minute walk test, muscle strength, range of motion, and Performance of Upper Limb
(PUL). For more information, please visit www.clinicaltrials.gov (NCT02310763).

We expect that this news will deeply disappoint the many families living with DMD who
directly participated in the study, as well as all of the families living with DMD hoping for
therapies that will alter the trajectory of this devastating disease. In recognition of the
continued burden of unmet needs in DMD, Pfizer continues to advance research and
clinical development efforts focused on DMD, including our investigational mini-
dystrophin gene therapy program. In addition, we are involved in efforts focused on
optimizing drug development for DMD. We intend to share data from the phase 2 clinical
study of domagrozumab with the DMD community in order to contribute to the body of
scientific knowledge which may enable the development of new therapies for patients.

In terms of next steps:

  • All clinical sites participating in the domagrozumab DMD studies have been provided with instructions for actions to take with enrolled participants. 

  • Patients and caregivers who have questions regarding their study participation should speak with their study physician for more information. 

  • Pfizer will share additional information about these results with the community when we are able.

We recognize the commitment that the DMD community has provided to these studies
and to our research and development efforts. It is with heartfelt gratitude that we
extend our sincerest ‘thank you’ to all the boys and families who expressed interest in the
study, underwent screening for the study, and to those ultimately participating in the
study. We are also grateful to the advocacy associations and advocates who provided the
tools and support needed for families to engage in clinical research and who continue to
care for the Duchenne community, and to the clinical research sites and staff who
collaborated with us on this important research.

Kind regards,
Michael Binks, MD
Shannon Marraffino
Katherine Beaverson
Rare Disease Research Unit
Pfizer Inc.

PRESS RELEASE

Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne Muscular Dystrophy

NEW YORK, NY, August 30, 2018 - Pfizer Inc. (NYSE: PFE) announced today that it is
terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study
(B5161002) and an open-label extension study (B5161004). The Phase 2 study (B5161002), did
not meet its primary efficacy endpoint, which was to demonstrate a difference in the mean
change from baseline in 4 Stair Climb (in seconds) following one year of treatment with
domagrozumab as compared to placebo in patients with DMD. Further evaluation of the totality of evidence including secondary endpoints did not support a significant treatment effect. The
decision comes after a thorough review of data available at the time of the primary analysis,
which evaluated all study participants after one year of treatment, as well as those participants
who were in the trial beyond one year. The studies were not terminated for safety reasons. Pfizer
will continue to review the data to better understand any insights they may provide, and will share results with the scientific and patient community.

“We are disappointed by these results and while we are not progressing with the studies, the
data will contribute to a greater understanding of this disease and we will evaluate the total
data set to see if there is a place for this medicine in muscular diseases,” said Seng Cheng,
PhD, Senior Vice President and Chief Scientific Officer, Pfizer Rare Disease Research Unit.
“We are extremely grateful to all those involved with this trial, especially the boys who
participated, and their families.”

Pfizer is continuing research in DMD and rare neuromuscular diseases, with the goal of bringing
therapies to patients with unmet needs. The company’s continued partnership with advocacy
associations and the community is critical to finding innovative therapies for these diseases.
Pfizer has one ongoing clinical trial in DMD with a gene therapy, PF-06939926, which is an
investigational, recombinant AAV9 capsid carrying a truncated or shortened version of the human
dystrophin gene (mini-dystrophin) under the control of a human muscle specific promotor. 1

About the Domagrozumab Clinical Studies 1
The Phase 2 double-blind, placebo-controlled, multicenter clinical trial investigated the efficacy
and safety of domagrozumab, administered in monthly IV doses, in 121 boys aged 6 to 15 with
DMD, regardless of underlying mutation. It was designed as a two-year, placebo-controlled study
(with the primary analysis after one year); all subjects used background corticosteroid therapy.
The open-label extension study was designed to evaluate long-term safety and efficacy of
domagrozumab.

About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare, serious, debilitating childhood genetic disease
characterized by progressive muscle degeneration and weakness and significantly shortened life
expectancy. It is the most common form of muscular dystrophy worldwide and primarily affects
boys, with incidence of 1 in every 3500 to 5000 live male births each year. 2 , 3 Children with DMD typically present with signs of weakness, including late walking, trouble getting up, and difficulty running or climbing stairs, usually manifesting in early childhood between the ages of 1 and 4 years. 4 The progressive muscle degeneration leads to a loss of the ability to walk in the early teenage years, on average. Weakness of respiratory muscles ultimately leads to use of mechanical ventilatory support, and weakness in cardiac muscle involvement results in cardiomyopathy.


Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses and impacts millions of patients
worldwide, 5 representing an opportunity to apply our knowledge and expertise to help make a
significant impact on addressing unmet medical needs. 1 The Pfizer focus on rare disease builds
on more than two decades of experience, a dedicated research unit focusing on rare disease,
and a global portfolio of multiple medicines within a number of disease areas of focus, including
hematology, neuromuscular, and inherited metabolic disorders. Error! Bookmark not defined.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases
work with insights from innovative strategic collaborations with academic researchers, patients,
and other companies to deliver transformative treatments and solutions. We innovate every day
leveraging our global footprint to accelerate the development and delivery of groundbreaking
medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients,
engage communities in our clinical development programs, and support programs that heighten
disease awareness.

Working together for a healthier world ®
At Pfizer, we apply science and our global resources to bring therapies to people that extend and
significantly improve their lives. We strive to set the standard for quality, safety and value in the
discovery, development and manufacture of health care products. Our global portfolio includes
medicines and vaccines as well as many of the world&#39;s best-known consumer health care
products. Every day, Pfizer colleagues work across developed and emerging markets to advance
wellness, prevention, treatments and cures that challenge the most feared diseases of our time.
Consistent with our responsibility as one of the world&#39;s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com . In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and
@Pfizer_News , LinkedIn , YouTube and like us on Facebook at Facebook.com/Pfizer .

Industry Partner News: Wave Life Sciences Receives US Orphan Drug and Rare Pediatric Disease Designations for WVE-210201

CAMBRIDGE, Mass., Aug. 16, 2018 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (NASDAQ:WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne muscular dystrophy (DMD). The European Commission previously granted orphan drug designation for WVE-210201 in July 2018.  

"Our team is motivated by a sense of urgency and compassion for the patients, families and caregivers affected by Duchenne muscular dystrophy and other serious, life-threatening conditions with high areas of unmet need," said Michael Panzara, MD, MPH, Neurology Franchise Lead of Wave Life Sciences. "We are very pleased to receive these two important designations from the FDA and believe they further reinforce the potential of WVE-210201 to help boys suffering from DMD."

The Orphan Drug Act provides for economic incentives to encourage the development of drugs intended to treat, diagnose or prevent rare diseases and conditions affecting fewer than 200,000 people in the United States. In determining orphan drug designation, the FDA's Office of Orphan Products Development evaluates preclinical and clinical data to identify products as promising for rare disease. If market approval is granted by the FDA for WVE-210201 for the treatment of DMD, orphan drug designation would entitle Wave to seven years of market exclusivity in the United States. Additional incentives may include tax credits related to clinical trial expenses, exemption from prescription drug user fees and FDA assistance in clinical trial design.

Rare pediatric disease designation by the FDA is granted in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States in which the serious or life-threatening manifestations are primarily in individuals 18 years of age and younger. The designation provides regulatory incentives for companies to develop and market therapies that treat these conditions. The sponsor of a drug for a rare pediatric disease may be eligible for a priority review voucher upon approval of the drug that can be used to obtain a priority review of a subsequent marketing application.

"These designations from U.S. regulators represent the significant progress being made by Wave Life Sciences and our community is grateful to Wave for their ongoing commitment to the Duchenne muscular dystrophy community," said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy. "We look forward to their exon 51 skipping program advancing and Wave's other initiatives in DMD."

WVE-210201 is currently being studied in an ongoing global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in DMD patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients, including ambulatory and non-ambulatory patients, between the ages of 5 and 18 years of age. As patients complete the Phase 1 trial, they have the option to enroll in an ongoing open label extension study in which they receive continued treatment with WVE-210201.

About Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic neuromuscular disorder caused predominantly by out-of-frame deletions in the dystrophin gene, resulting in absent or defective dystrophin protein. Dystrophin protein is needed for normal muscle maintenance and operation. Because of the genetic mutations in DMD, the body cannot produce functional dystrophin, which results in progressive and irreversible loss of muscle function, including the heart and lungs. Globally, DMD affects approximately one in 5,000 newborn boys.

About WVE-210201

WVE-210201 is an investigational stereopure oligonucleotide that has been shown to induce skipping of exon 51 of dystrophin pre-mRNA in preclinical studies and is intended for the treatment of Duchenne muscular dystrophy (DMD). Approximately 13% of DMD patients have genetic mutations that are amenable to treatment with exon 51 skipping therapy. Exon-skipping technology has the potential to induce cellular machinery to ‘skip over' a targeted exon and restore the reading frame, resulting in the production of internally truncated, but functional, dystrophin protein. Wave preclinical in vitro experiments using gymnotic delivery (free uptake) of WVE-210201 in DMD patient-derived myoblasts demonstrated efficient exon 51 skipping and dystrophin protein restoration. Preclinical Western blot studies of WVE-210201 demonstrated 52% dystrophin protein restoration as compared with normal skeletal muscle tissue lysates.

About Wave Life Sciences

Wave Life Sciences is a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases. Its chemistry platform enables the creation of highly specific, well characterized oligonucleotides designed to deliver superior efficacy and safety across multiple therapeutic modalities. The company's pipeline is initially focused on neurological disorders and extends across several other therapeutic areas. For more information, please visit www.wavelifesciences.com.

Industry Partner News: Sarepta Therapeutics Announces that Phase 1/2a Duchenne Muscular Dystrophy (DMD) MicroDystrophin Gene Therapy Trial Placed on Clinical Hold

-- Out-of-specification lot resulted from the presence of trace levels of DNA fragment in research-grade . . raw material plasmid sourced from third-party manufacturer --

-- Fragment fully characterized; preliminary testing and analysis indicates no safety signals --

-- Subject to FDA review of a corrective action plan, which will include the use of GMP-s plasmid for all . . . future production lots --

-- Clinical timeline to commence dosing of patients in pivotal trial by year-end 2018 remains on track --

-- Sarepta to host conference call on Wednesday, July 25, 2018 at 5:00 p.m. ET --

CAMBRIDGE, Mass., July 25, 2018 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, has been notified by the Research Institute at Nationwide Children’s Hospital (the Research Institute) that they have received a letter from the Food and Drug Administration (FDA) on July 24, 2018, stating that their Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial has been placed on clinical hold due to the presence of a trace amount of DNA fragment in research-grade third-party supplied plasmid. Preliminary in-vivo testing performed by the Research Institute indicates that the trace fragment does not result in protein expression and is quickly cleared.

The Research Institute, working with Sarepta, has developed their action plan with immediate plans to submit for review by the FDA, which will include the use of GMP-s plasmid for the program. Subject to the FDA’s acceptance of the action plan, Sarepta does not anticipate any material delay in dosing patients as originally planned by year-end 2018.

“Patient safety is our top priority at Sarepta as we know it is for Nationwide Children’s Research Institute,” stated Doug Ingram, Sarepta’s president and chief executive officer. “We intend to rapidly respond to the FDA’s clinical hold letter, including a commitment to the Agency to only use GMP-s plasmid. Independently, we will also request a meeting with the Agency to discuss the micro-dystrophin program with the goal of commencing a pivotal trial by year-end 2018.”

Sarepta will host a conference call today, Wednesday, July 25, 2018 at 5:00 p.m. ET. The conference call may be accessed by dialing 844-534-7313 for domestic callers and +1-574-990-1451 for international callers. The passcode for the call is 8689739. Please specify to the operator that you would like to join the "Sarepta Conference Call." The conference call will be webcast live under the investor relations section of Sarepta's website at www.sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit www.sarepta.com.

For more information, visit sareptatherapeutics.com 

Industry Partner News: PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two Years with Nonsense Mutation Duchenne Muscular Dystrophy

SOUTH PLAINFIELD, N.J., July 9, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the presentation of data from the Translarna (ataluren) Phase II Study 030 demonstrating that the safety and pharmacokinetic profile of Translarna in children from two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD) was consistent with that for older children.1  Importantly, the data also showed that treatment with Translarna resulted in improvements in timed function tests and the North Star Ambulatory Assessment from baseline at weeks 28 and 52, with mean changes showing as much as a 25 percent improvement after one year.1 The data at 28 weeks formed the basis of the recent positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to expand the current indication of Translarna to include nmDMD ambulatory children from two to five years of age.2  The data was presented at the International Congress on Neuromuscular Diseases in Vienna.

Translarna is the only approved treatment to address the underlying cause of nmDMD, a rare, genetic, muscle-wasting disease,1 and is currently licensed in Europe for ambulatory patients aged five years and older.3

"We are excited to demonstrate that Translarna showed an improvement over one year of treatment in patients with nonsense mutation Duchenne as young as two years of age," stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "Irreversible muscle damage starts before the age of five. Early intervention is critical to maintain muscle function and delay disease progression."

An interim analysis of Study 030 demonstrated that at week 28, the safety and pharmacokinetic profile for Translarna in children aged two to five years is consistent with that for older children.1 Clinical benefits were also observed at 28 weeks with Translarna, with decreases versus baseline in the time to run/walk 10 meters, climb four stairs, and stand from lying face up (supine).1 The most common adverse events included pyrexia, ear infection, and nasopharyngitis.1

Study 030 evaluated changes in timed function tests (TFTs) and the 3-part, 8-part and full (16) items North Star Ambulatory Assessment (NSAA) scales, adopted for children under five years of age (N=12).1 Results summarized in the table below.

About Study 0301

Study 030 was an open-label, Phase 2 study designed to evaluate the safety and pharmacokinetics (PK) of ataluren (10, 10, and 20 mg/kg) in patients aged ≥2 to <5 years with nmDMD. The study includes a 4-week treatment period, a 48-week extension period, and a 4-week follow-up period. Secondary objectives in Study 030 evaluated changes in timed function tests (TFTs) and the total, 3-part,8-part and full (16) items North Star Ambulatory Assessment (NSAA) scales, adapted for children <5 years of age. All patients were male (N=14) with genotypic confirmation of nmDMD. Two patients were excluded from the current analysis: one patient did not have reported functional assessment at Week 28; and one patient did not have baseline measurement all for post-line evaluations, resulting in N=12. Seven out of the fourteen patients in the safety population (50%) reported ≥1 treatment-emergent adverse event (TEAE) during the extension phase, all of which were deemed unrelated to the study drug; there were no serious TEAEs or discontinuations due to a TEAE. Pyrexia, ear infection, and nasopharyngitis were the most common TEAEs, each occurring in 2 patients (14.3%).

About 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, tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States. The development of ataluren has been supported by grants from the Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

About Duchenne Muscular Dystrophy

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information on the signs and symptoms of Duchenne can be found at: www.duchenneandyou.com

About PTC Therapeutics, Inc.

PTC is a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint. This success is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

Industry Partner News: Catabasis announces plans for edasalonexent Phase 3 POLARIS DMD trial and newsletter

-- Global Phase 3 POLARIS DMD Trial Expected to Initiate in the Second Half of 2018 and Enroll Approximately 125 Patients --

-- Phase 2 MoveDMD® Trial and Open-Label Extension Showed Substantially Slowed Duchenne Disease Progression in Patients Treated with Edasalonexent --

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 9, 2018-- Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced plans for the Phase 3 POLARIS DMD trial with edasalonexent in patients with Duchenne muscular dystrophy (DMD). Catabasis plans to initiate the global POLARIS DMD trial in the second half of 2018 with top-line results expected in the second quarter of 2020.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180709005089/en/

The POLARIS DMD trial will evaluate the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent. The trial design was informed by discussions with the U.S. Food and Drug Administration (FDA) as well as input from treating physicians and families of boys affected by Duchenne.

The randomized, double-blind, placebo-controlled POLARIS DMD trial has many key elements in common with the Phase 2 MoveDMD® trial, including the patient population and functional endpoints. Catabasis anticipates enrolling approximately 125 patients between the ages of 4 and 7 regardless of mutation type who have not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen may be eligible to enroll. The primary efficacy endpoint will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints are planned to include the age-appropriate timed function tests time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also planned to be included. Two boys will receive edasalonexent for every boy that receives placebo and after 12 months, all boys are expected to receive edasalonexent in an open-label extension.

“We have designed a robust study with POLARIS DMD to evaluate edasalonexent as a potential new treatment for Duchenne. We have benefited from input from many people that are part of the Duchenne community and we are well underway with our preparations to begin the trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We are very excited to advance edasalonexent through this potentially last phase of clinical development with the hope of providing a new treatment option to all boys affected by this disease. We believe that edasalonexent has great potential as a therapy to be taken on its own as well as in combination with other treatments.”

Edasalonexent is a potential oral foundational therapy that is being developed for all patients affected by DMD. Edasalonexent is being developed for use as monotherapy and in possible combination with dystrophin upregulation therapies. Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD Phase 2 trial and open-label extension. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been safe and well tolerated through more than 45 patient-years of treatment.

About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in DMD and drives inflammation, fibrosis and muscle degeneration and suppresses muscle regeneration. Edasalonexent continues to be dosed in an open-label extension of the MoveDMD Phase 2 clinical trial, and Catabasis is preparing to initiate a single global Phase 3 trial in the second half of 2018 to evaluate the efficacy and safety of edasalonexent for registration purposes. 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 reported to-date, please visit www.catabasis.com.

About Catabasis
At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. Edasalonexent was designed using our SMART (Safely Metabolized And Rationally Targeted) Linker drug discovery platform that enables us to engineer molecules that simultaneously modulate multiple targets in a disease. For more information on edasalonexent or our drug discovery platform, please visit www.catabasis.com.

Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company’s plans to commence a single global Phase 3 trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; the Company’s ability to obtain financing on acceptable terms and in a timely manner to fund the Company’s planned Phase 3 trial of edasalonexent in DMD for registration purposes; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2018, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release.

Industry Partner News: Summit Announces PhaseOut DMD Did Not Meet Primary Endpoint

Screen Shot 2018-06-27 at 10.08.02 AM.png
Summit logo.png

To the DMD community,

After reviewing the top-line results of PhaseOut DMD, we have made the difficult decision to discontinue the development of ezutromid.

We recognize that this decision will be disappointing for the Duchenne community, but the data from PhaseOut DMD were clear that ezutromid, while well-tolerated, did not provide a benefit to patients with DMD.

These results are not what we had hoped for, and certainly not what we had expected based on the encouraging interim results from PhaseOut DMD. But, they provide a clear answer to the important scientific question of ezutromid’s effect in DMD. The results provided no evidence that ezutromid is having a meaningful effect on slowing DMD progression. We therefore cannot support further development of ezutromid. We are now working with our clinical trial investigators to bring the PhaseOut DMD clinical trial and associated extension phase to a close. Patients in PhaseOut DMD should contact their study doctors for more information.

We sincerely thank the patients, families and clinical trial sites who have been involved in all of our clinical trials of ezutromid. We plan to explore ways that the information gathered as part of PhaseOut DMD can be made available to support other research activities in DMD for the benefit of the entire community.

We are also grateful for the support that we’ve had from patient organizations worldwide in the development of ezutromid.

We believe that the future of Duchenne research is bright. There are numerous clinical trials and research studies taking place in this field, and there is hope on the horizon for all those living with DMD.

If you have further questions, please contact Michelle Avery, PhD, our Director of Patient Engagement, at michelle.avery@summitplc.com.

Sincerely,
Glyn Edwards, CEO of Summit

FAQs

1.       What do these data mean? We designed PhaseOut DMD to answer if ezutromid is safe and efficacious in patients with DMD. Unfortunately, the results are quite clear that ezutromid is not having a clinical benefit for patients with DMD. It does continue to be well-tolerated, but given the lack of evidence we’ve seen, we have decided to discontinue its development.

2.       Will patients in PhaseOut DMD be allowed to continue to receive ezutromid? We are working with our clinical trial investigators to bring the trial and extension phase to a close, including any patients who have been enrolled into the additional cohort of the trial that was opened in March 2018. We have taken this difficult decision as the data were clear that patients in PhaseOut DMD were not benefitting from ezutromid treatment.

3.       Are you continuing to work on future generation compounds? While we still believe utrophin modulation could have a place in the treatment of DMD and other dystrophinopathies, our focus will be on the development of our new mechanism antibiotic pipeline.

To read the full press release, please visit Summit's website here.

Industry Partner News: Sarepta Announces that Jerry Mendell, M.D. Presented Positive Preliminary Results from the First Children Dosed in Gene Therapy Micro-dystrophin Trial to Treat DMD Patients

dinarudick.bostonglobe

Dear Duchenne Community,

Today Sarepta Therapeutics held its first ever Research and Development Day, an opportunity to gain insight into Sarepta’s pipeline, its collaborations, and its progress toward its mission of bringing a better life to those living with Duchenne muscular dystrophy, Limb-girdle muscular dystrophy and other rare diseases.

Multiple speakers shared information regarding their work in Duchenne muscular dystrophy today. From these presentations a notion of Sarepta’s full body of work is emerging. The good news is that we are making progress toward our goal of developing therapeutic options for 100% of the individuals with Duchenne. Speakers included (in order of appearance):

  • Doug Ingram, President & Chief Executive Officer, Sarepta Therapeutics
  • Gilmore O’Neill, M.B., M.M.SC., Chief Medical Officer, Sarepta Therapeutics
  • Kevin M. Flanigan, M.D., Director, Center for Gene Therapy, Nationwide Children’s Hospital
  • Serge Braun, Pharm.D., Ph.D., Chief Scientific Officer, AFM-Téléthon; Director Neuromuscular Strategy, Genethon; President, Genosafe SAS; Member, French National Academy of Pharmacy
  • Jerry R. Mendell, M.D., Curran-Peters Chair of Pediatric Research, Center for Gene Therapy, Nationwide Children’s Hospital
  • Louise Rodino-Klapac, Ph.D., Vice President, Gene Therapy, Sarepta Therapeutics; Chief Scientific Officer, Myonexus Therapeutics
  • Charles A. Gersbach, Ph.D., Associate Professor of Biomedical Engineering, Director, Center for Biomolecular and Tissue Engineering, Duke University
  • Gunnar J. Hanson, Ph.D., Senior Director, Research Chemistry, Sarepta Therapeutics
  • Marco Passini, Ph.D., Senior Director, Biology, Sarepta Therapeutics

Notably, Drs. Jerry Mendell and Louise Rodino-Klapac presented positive preliminary results from the first three children dosed in the Phase 1/2a gene therapy micro-dystrophin trial to treat patients with Duchenne muscular dystrophy. Dr. Mendell shared the following:

  • Biopsies performed at Day 90 showed robust micro-dystrophin expression in muscle measured by all methods and observed in all three patients
  • Significant decrease in levels of serum creatine kinase (CK), an enzyme biomarker strongly associated with muscle damage caused by Duchenne muscular dystrophy
  • No serious adverse events (SAEs) observed

We should remember that these are preliminary results involving three patients; much more work
remains to be done. Nevertheless, this data is encouraging and gives us confidence that we are on the right track. 

Regarding the micro-dystrophin gene therapy program, we want to pause and acknowledge every person who has helped to bring this work forward including Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, Pat Furlong, and Parent Project Muscular Dystrophy, who from the earliest days supported this work. We also want to thank the individuals with Duchenne and their families who participated in our clinical trials. Their courage and dedication benefits all living with Duchenne.

Great science is born from great questions, and we know that this community has enormous insight to share. If you have questions related to today’s news, please reach out to your advocacy partners. We will be offering a conference call with them in the near future to discuss community questions. Advocacy groups are instrumental in helping digest movement and change, and they can be a fantastic conduit for ongoing conversation with the community. If you would like to receive future updates from Sarepta, please register on www.duchenne.com/connect.

Thank you to the community for allowing us to engage in this fight with you. We are humbled and inspired by your spirit daily and will remain dedicated to finding meaningful solutions.

Sincerely,

Douglas S. Ingram
President & Chief Executive Officer
Sarepta Therapeutics, Inc.

To read Sarepta's full Press Release, please click here.

Industry Partner News: Activities to Resume Enrollment in Solid's IGNITE DMD Phase I/II Clinical Trial are Underway

CAMBRIDGE, Mass., June 18, 2018 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). In its letter, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. Solid has begun activities to resume the clinical trial and plans to reinitiate enrollment as quickly as possible.

“We believe SGT-001 has the potential to offer significant benefit to patients with DMD, regardless of their age or stage of disease,” said Ilan Ganot, Founder and Chief Executive Officer of Solid Biosciences. “We are pleased to have been able to provide the FDA with a comprehensive response resulting in the removal of the clinical hold so we can continue development of this important potential treatment.”

As previously disclosed, the FDA placed a clinical hold on IGNITE DMD following the Company’s report of a Serious Adverse Event (SAE) in the first patient dosed with SGT-001. The event was characterized by a decrease in platelet count followed by a reduction in red blood cell count, transient renal impairment and evidence of complement activation. There were no signs of bleeding or clotting abnormalities and no laboratory evidence of liver dysfunction. The patient received standard medical care, a modified steroid regimen and a limited course of eculizumab for the observed complement activation. He remained clinically stable and generally asymptomatic throughout the event, which fully resolved.

“Gene therapy has the potential to dramatically change the course of DMD and may offer long-term benefit for those who suffer from this devastating disease,” said Barry Byrne, M.D., Ph.D., Director, University of Florida Powell Gene Therapy Center and Professor, Pediatrics and Molecular Genetics & Microbiology at the University of Florida College of Medicine. "After a thorough analysis of the clinical and laboratory data for the patient, I am confident the event was easily monitored and medically manageable. Our patient quickly returned to his normal activities and planned study assessments. We look forward to continuing the IGNITE DMD study and providing additional children and adolescents with this promising investigational therapy.” 

In connection with the lifting of the clinical hold, Solid has made changes to the IGNITE DMD protocol, including the addition of IV glucocorticoids in the initial weeks post administration of SGT-001 and enhanced monitoring measures that include a panel for complement activation. The amended protocol also specifies that eculizumab will be available as a treatment option if complement activation is observed.

The Company plans to enroll and dose several children prior to dosing additional adolescents. In addition, Solid now has the choice to obtain the intermediate muscle biopsy at 45 days post administration of SGT-001 to collect additional information about the time course of microdystrophin expression.

As a result of the clinical hold, Solid now expects to report initial data from a pre-specified interim analysis of IGNITE DMD in the second half of 2019.

Conference Call
Solid's management team will host a conference call and webcast at 8:30 a.m. ET today to discuss the lifting of the clinical hold. The conference call can be accessed by dialing +1 866 763 0341 for domestic callers and +1 703 871 3818 for international callers. The passcode for the call is 8892798. A live webcast of the conference call can also be accessed through the "Investors" tab on the Solid Biosciences website at www.solidbio.com. A webcast replay will be available online after the call.

About SGT-001
Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of Duchenne muscular dystrophy (DMD), mutations in the dystrophin gene that result in the absence or near-absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin transgene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). SGT-001 utilizes AAV9, which has an affinity for muscle and is currently being evaluated in multiple clinical programs in other indications. Data from Solid’s preclinical program suggest that SGT-001 has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and the European Union.

About Solid Biosciences
Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Currently, Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices. For more information, please visit www.solidbio.com.