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

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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

Glyn Edwards, CEO of Summit


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


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

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.


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 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

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

-- First quarter 2017 EXONDYS 51® (eteplirsen) total net revenues of $64.6 million --

-- Sarepta signs exclusive partnership and buy-out option with Myonexus Therapeutics; pipeline expands from 16 to 21 programs --

-- Company announces date of first R&D day, at which clinical data from gene therapy micro-dystrophin program will be announced --

-- Company receives negative trend vote following its CHMP oral explanation; will request re-examination and Scientific Advisory Group to be convened --

CAMBRIDGE, Mass., May 03, 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, today reported financial results for the three months ended March 31, 2018.

“In the first quarter, we continued our successful launch of EXONDYS 51 and advanced our pipeline to bring life-enhancing therapies to those suffering from rare disease around the world,” said Doug Ingram, Sarepta’s president and chief executive officer. “We accelerated our gene therapy and RNA platform, and in that regard are excited to announce that our first R&D day will take place on June 19 to showcase the breadth, depth and progress of our pipeline. Significantly, at this event we will report preliminary safety and gene expression data from at least two patients from our micro-dystrophin gene therapy trial underway with Nationwide Children’s Hospital.”

Mr. Ingram continued, “Aligned with our stated goal of leveraging our expertise beyond DMD, we announced today a collaboration with Myonexus Therapeutics for the development of five potentially transformative gene therapies to treat a debilitating set of diseases, all under the umbrella of Limb-girdle muscular dystrophy. Through this collaboration, we have expanded our pipeline to 21 therapies in development. Our confidence in the Myonexus collaboration comes from the similarities between the Myonexus and Sarepta approaches to gene therapy.  Both are seeking to treat rare neuromuscular disease through the AAVrh.74 vector; and both rely upon the unparalleled expertise of Dr. Louise Rodino-Klapac in developing and executing gene therapy constructs. This partnership with Myonexus enables us to expand our efforts beyond DMD while maintaining our unwavering commitment to those suffering from DMD.” 

Mr. Ingram concluded, “Unfortunately, in addition to our successes in the first quarter, we also have had a delay in our effort to bring eteplirsen to patients in Europe who could potentially benefit from it. I could not be prouder of our Sarepta team and the team of experts who spoke on behalf of eteplirsen at the CHMP oral explanation last week. The rigorous work that was done to prepare for the hearing only strengthened our resolve that eteplirsen should urgently be made available to those waiting in Europe. Unfortunately, the CHMP’s trend vote was negative. Based on discussions with CHMP representatives, it is our understanding that the CHMP did not conclude that eteplirsen is ineffective for exon 51 amenable patients, but rather that Sarepta has not yet met the regulatory threshold for conditional approval, in part due to the use of external controls as comparators in the studies. Sarepta plans to file for re-examination and will request that a Scientific Advisory Group (SAG), which is made up of DMD and neuromuscular specialists, be convened to provide expert guidance and insight into, among other things, the validity of the external controls used and the importance of slowing pulmonary decline in patients with DMD.”

Financial Results

For the first quarter of 2018, on a GAAP basis, Sarepta reported a net loss of $35.4 million, or $0.55 per basic and diluted share, compared to net income of $84.1 million reported for the same period of 2017, or $1.53 per basic share and $1.50 per diluted share. On a non-GAAP basis, the net loss for the first quarter of 2018 was $17.9 million, or $0.28 per share, compared to a net loss of $31.4 million for the same period of 2017, or $0.57 per share.

Net Revenues

For the three months ended March 31, 2018, the Company recorded net product revenues of $64.6 million, compared to net revenues of $16.3 millionfor first quarter of 2017. The increase primarily reflects increasing demand for EXONDYS 51 in the U.S.

Cost and Operating Expenses

Cost of sales (excluding amortization of in-licensed rights)

For the three months ended March 31, 2018, cost of sales (excluding amortization of in-licensed rights) was $5.6 million, compared to $0.2 millionfor the same period of 2017. The increase primarily reflects royalty payments to BioMarin Pharmaceuticals (BioMarin) as a result of the execution of the settlement and license agreements with BioMarin in July 2017 as well as higher inventory costs related to increasing demand for EXONDYS 51 during 2018. Prior to the approval of EXONDYS 51, the Company expensed related manufacturing and material costs as research and development expenses.

Research and development

Research and development expenses were $46.2 million for the first quarter of 2018, compared to $29.1 million for the same period of 2017, an increase of $17.1 million. The increase in research and development expenses primarily reflects the following:

  • $4.4 million increase in clinical and manufacturing expenses primarily due to increased patient enrollment in the Company’s ongoing clinical trials in golodirsen and casimersen, as well as a ramp-up of manufacturing activities for the Company’s PPMO platform. These increases were partially offset by a ramp-down of clinical trials in eteplirsen primarily because the PROMOVI trial has been fully enrolled;
  • $3.2 million increase in collaboration cost sharing with Summit on its utrophin platform;
  • $2.7 million increase in compensation and other personnel expenses primarily due to a net increase in headcount;
  • $2.4 million increase in professional services primarily due to an expansion of the Company’s research and development pipeline; and
  • $1.6 million increase in preclinical expenses primarily due to the continuing ramp-up of toxicology studies in the Company’s PPMO platform as well as golodirsen and casimersen.

Non-GAAP research and development expenses were $43.3 million for the first quarter of 2018, compared to $26.7 million for the same period of 2017, an increase of $16.6 million.

Selling, general and administration

Selling general and administrative expenses were $43.3 million for the first quarter of 2018, compared to $26.2 million for the same period of 2017, an increase of $17.1 million. The increase in selling, general and administrative expenses primarily reflects the following:

  • $6.4 million increase in professional services primarily due to continuing global expansion as well as preparation for a potential product launch in the EU should the Company’s Marketing Authorization Application be approved by the European Medicines Agency;
  • $5.3 million increase in compensation and other personnel expenses primarily due to a net increase in headcount; and
  • $4.6 million increase in stock-based compensation primarily due to the impact of revising the forfeiture rate assumption for officers and Board of Directors as well as an increase in stock price.

Non-GAAP selling, general and administrative expenses were $33.7 million for the first quarter of 2018, compared to $21.1 million for the same period of 2017, an increase of $12.6 million.

Amortization of in-licensed rights

Amortization of in-licensed rights was $0.2 million during the first quarter of 2018, compared to less than $0.1 million for the same period of 2017. The increase was primarily due to the BioMarin transactions that occurred in July 2017.

Other (loss) Income

Gain from sale of Priority Review Voucher

In connection with the completion of the sale of the Priority Review Voucher (PRV) in March 2017, the Company recorded a gain of $125.0 from sale of the PRV in the first quarter of 2017. There was no similar activity in the first quarter of 2018.

Interest (expense) income and other, net

For the three months ended March 31, 2018 and 2017, the Company recorded $4.5 million interest expense and other, net and $0.3 interest income and other, net, respectively.  The period over period unfavorable change primarily reflects the interest expense accrued on the Company’s debt facilities partially offset by interest income from higher balances of cash, cash equivalents and investments.

Cash, Cash Equivalents, Restricted Cash and Investments

The Company had $1.0 billion in cash, cash equivalents, restricted cash and investments as of March 31, 2018 compared to $1.1 billion as of December 31, 2017. The decrease is primarily driven by the use of cash to fund the Company’s ongoing operations during the first quarter of 2018.

Use of Non-GAAP Measures

In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements. The non-GAAP loss is defined by the Company as GAAP net loss excluding interest expense/(income), income tax expense/(benefit), depreciation and amortization expense, stock-based compensation expense, restructuring expense and other items. Non-GAAP research and development expenses are defined by the Company as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense, restructuring expense and other items. Non-GAAP selling, general and administrative expenses are defined by the Company as GAAP selling, general and administrative expenses excluding depreciation and amortization expense, stock-based compensation expense, restructuring expense and other items.

1. Interest, tax, depreciation and amortization

Interest income and expense amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of the Company’s operations.  Tax amounts can vary substantially from period to period due to tax adjustments that are not directly related to underlying operating performance. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to the Company’s operating performance. Amortization expense associated with in-licensed rights as well as patent costs are amortized over a period of several years after acquisition or patent application or renewal and generally cannot be changed or influenced by management.

2. Stock-based compensation expenses

Stock-based compensation expenses represent non-cash charges related to equity awards granted by Sarepta. Although these are recurring charges to operations, management believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within management's control. Therefore, management believes that excluding these charges facilitates comparisons of the Company’s operational performance in different periods.

3. Restructuring expenses

The Company believes that adjusting for these items more closely represents the Company’s ongoing operating performance and financial results.

4. Other items

The Company evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relates to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue on a regular basis. These other items include the aforementioned gain from the sale of the Company’s PRV.

The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income adjustments, non-GAAP income tax expense, non-GAAP net loss, and non-GAAP basic and diluted net loss per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table "Reconciliation of GAAP to Non-GAAP Net Loss.”

First Quarter and Recent Corporate Developments

  • Golodirsen (SRP-4053): Based on Sarepta’s Type C meeting with the FDA’s Division of Neurology Products to solicit the Division's guidance on the development pathway for golodirsen, the Company remains on track to complete a rolling NDA submission by year-end 2018, seeking accelerated approval based on an increase in dystrophin protein as a surrogate endpoint.
  • Myonexus Therapeutics Partnership: Sarepta and Myonexus Therapeutics entered into a partnership to advance multiple gene therapies for various forms of Limb-girdle muscular dystrophies (LGMDs). The lead program, MYO-101, has generated encouraging pre-clinical safety and efficacy data utilizing the AAVrh.74 vector system, the same vector used in the micro-dystrophin gene therapy program Sarepta is developing with Nationwide Children’s Hospital. A Phase 1/2a study of MYO-101 is scheduled to begin in mid-2018. The companies plan to report on 60-day biopsy data in late-2018 or early 2019. Additionally, Myonexus is advancing MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 for LGMD2L. Under the terms of the agreement, Sarepta will make an upfront payment of $60 million and additional development-related milestone payments to purchase an exclusive option to acquire Myonexus at a pre-negotiated, fixed price with sales-related contingent payments. If all development-related milestone payments are met, Sarepta will make payments of up to $45 million over an approximately two-year evaluation period. Sarepta has the option to purchase Myonexus at any time, including upon review of proof-of-concept data.
  • Sarepta R&D Day (Tuesday, June 19, 2018): Sarepta management, along with several key-opinion leaders, will provide an in-depth look into the Company’s pipeline programs across several modalities, included RNA-targeted therapies, gene therapy and gene editing. Of particular note, we look forward to presenting our micro-dystrophin expression data from at least two patients enrolled in the Phase 1/2a gene therapy clinical trial underway with Drs. Jerry Mendell and Louise Rodino-Klapac of Nationwide Children’s Hospital. To date, the Company has enrolled four patients in this study and no significant adverse events have been reported. In addition, Dr. Rodino-Klapac, who is also chief scientific officer and co-founder of Myonexus, will present data from Myonexus’ entire LGMD program. For all to access, Sarepta’s R&D day will be webcast live under the investor relations section of the Company’s website at: and will be archived there following the event for 90 days.

Conference Call

The Company will be hosting a conference call at 4:30 p.m. Eastern Time, to discuss these financial results and provide a corporate update. 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 2798939. Please specify to the operator that you would like to join the "Sarepta First Quarter 2018 Earnings Call”. The conference call will be webcast live under the investor relations section of Sarepta's website at 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 EXONDYS 51

EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Important Safety Information About EXONDYS 51

Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the full Prescribing Information.

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 DMD drug candidates. For more information, please visit

Industry Partner News: Capricor Launches HOPE-2 Clinical Trial of CAP-1002 in Duchenne Muscular Dystrophy


UC Davis Medical Center First Site in Nation To Enroll and Treat Participants

LOS ANGELES – April 30, 2018 – Capricor Therapeutics (NASDAQ: CAPR) today announced it has initiated the HOPE-2 clinical trial at UC Davis Medical Center. The trial will test the safety and efficacy of Capricor’s novel cellular therapy, CAP-1002, in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure.

Up to 84 boys and young men with Duchenne muscular dystrophy will be enrolled in HOPE-2, a Phase 2, randomized, double-blind, placebo-controlled trial that will test CAP-1002 in participants with advanced stages of Duchenne muscular dystrophy. The medical center in Sacramento is the first site in the nation to begin enrolling and treating participants. Approximately 12-15 investigative sites are expected to participate in the trial.

“We are very pleased to begin this important clinical trial of CAP-1002,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and Rehabilitation. “The HOPE-2 trial will study whether CAP-1002 can maintain or improve cardiac and skeletal muscle function. Because many of the participants are non-ambulatory, the study will focus primarily on the impact on arm mobility.”

Linda Marbán, Ph.D., Capricor president and chief executive officer, said CAP-1002 is one of the very few clinical initiatives to focus on helping boys and young men whose ability to walk has been seriously impaired by the loss of muscle function that occurs as Duchenne muscular dystrophy progresses.

“We are thrilled to begin enrolling participants in HOPE-2 because we have seen the potential for improvements in muscle function in both pre-clinical studies and in our earlier HOPE-Duchenne trial,” she said. “We have also been granted the RMAT and orphan disease designations by the U.S. Food and Drug Administration (FDA). These designations will enable us to work closely with the FDA in finalizing the regulatory approval pathway for CAP-1002 and to receive expedited FDA reviews. We are hopeful that HOPE-2 may potentially be a registration trial.”

Dr. Marbán said CAP-1002 could be an important tool in the toolbox to treat Duchenne muscular dystrophy.

“While gene and other therapies have the potential to restore dystrophin expression and sustain muscle function, there will still be significant inflammation and fibrosis which can offset the restorative effects,” she said. “CAP-1002 may work synergistically with the emerging disease-modifying therapies to control those additional pathological aspects of Duchenne muscular dystrophy because CAP-1002’s primary mechanism of action is immunomodulatory, meaning it can help balance inflammation in this chronic inflammatory disease.”

Capricor’s previous clinical trial, the HOPE-Duchenne trial, evaluated the safety and efficacy of a single dose of CAP-1002 in boys and young men with heart disease related to Duchenne muscular dystrophy. It found CAP-1002 was generally safe, well tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function.

Participants in the HOPE-2 trial will be randomized to receive either placebo or CAP-1002, delivered intravenously every three months for a total of four administrations. Participants will be followed for a one-year period following randomization. An open label extension is planned if trial evidence suggests an appropriate risk/benefit profile of CAP-1002. For more information, please

For more information, please visit Capricor's website. 

Industry Partner News: Summit Therapeutics Completes 48 Weeks of Dosing in PhaseOut DMD

Summit logo.png

Summit Completes Dosing of Ezutromid in PhaseOut DMD Clinical Trial
Top-line Data On-track to be Reported During Q3 2018

Oxford, UK, and Cambridge, MA, US, 19 April 2018 – Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM) announces the completion of ezutromid dosing in patients with Duchenne muscular dystrophy (‘DMD’) for the full 48-week PhaseOut DMD clinical trial. Top-line data from the full trial continue to be expected in the third quarter of 2018.

“We believe the early improvements seen in muscle health in the interim data from PhaseOut DMD indicate ezutromid is reducing DMD disease severity. In the full trial results, we aim to see continued utrophin modulation and sustained changes in magnetic resonance parameters,” said Dr David Roblin, Chief Medical Officer and President of R&D of Summit. “These results, if positive, could form the basis of a regulatory filing of ezutromid, bringing this universal treatment to patients more rapidly.” 

PhaseOut DMD is a Phase 2 open-label, multi-centre trial of the Company’s utrophin modulator, ezutromid, in patients with DMD. Previously announced 24-week interim data from PhaseOut DMD showed evidence of activity across three different measures. Specifically, ezutromid:

  • Maintained the production of utrophin, a naturally occurring protein that can potentially substitute for dystrophin, as measured by muscle biopsy;
  • Significantly and meaningfully reduced muscle damage, as measured by muscle biopsy; and
  • Significantly reduced muscle inflammation, as measured by magnetic resonance.

About Utrophin Modulation in DMD

DMD is the most common and severe form of muscular dystrophy, impacting 50,000 patients in the developed world alone. DMD is caused by the lack of dystrophin, a protein that maintains healthy muscle function. The absence of dystrophin results in a catastrophic cycle of muscle damage and repair that leads to progressive loss of functional ability and ultimately in premature death.

Utrophin and dystrophin play a similar role in maintaining muscle function, but do so at different times. Utrophin plays this role when new muscle fibres are being formed, or when damaged fibres are being repaired, but then switches off to make way for dystrophin to perform this role in mature muscle fibres. Since patients with DMD are not able to produce dystrophin, a cycle of muscle damage and repair occurs, which eventually leads to muscle fibre failure. Utrophin modulation aims to address the root cause of DMD by maintaining the production of utrophin to substitute for the missing dystrophin. The presence of utrophin in mature muscle fibres could break the cycle of damage and repair and ultimately slow, or even stop, disease progression. Importantly, this approach has the potential to treat all patients with DMD regardless of their underlying dystrophin gene mutation.

The Company’s lead utrophin modulator, ezutromid, is an orally administered, small molecule drug. DMD is an orphan disease, and the US Food and Drug Administration (‘FDA’) and the European Medicines Agency have granted orphan drug status to ezutromid. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval. In addition, ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the FDA. 

About PhaseOut DMD

PhaseOut DMD is an open-label, multi-centre trial that has enrolled 40 patients in the US and UK, aged from their fifth to their tenth birthdays. PhaseOut DMD is 48 weeks in length after which patients have the option of enrolling into an extension phase and continuing to be dosed with ezutromid. The primary endpoint is the change from baseline in magnetic resonance parameters related to the leg muscles. Biopsy measures evaluating utrophin and muscle damage are included as secondary endpoints. Exploratory endpoints include the six-minute walk distance, the North Star Ambulatory Assessment and patient reported outcomes.

About Summit Therapeutics

Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at and Summit can be followed on Twitter (@summitplc).

Industry Partner News: Letter to the Duchenne Community from Solid Biosciences: Update on IGNITE DMD Clinical Trial


CAMBRIDGE, MASS, April 18, 2018

Dear Duchenne Community Members,

As valued partners in the fight against Duchenne muscular dystrophy (DMD), we wanted to share with you an update on our ongoing interactions with the U.S. Food and Drug Administration (FDA) on IGNITE DMD, our Phase I/II clinical trial to assess the safety and efficacy of SGT-001 in patients with DMD. We also shared this update in a press release that can be found here.

As you know, we recently announced that the FDA put IGNITE DMD on full clinical hold due to a serious adverse event (SAE) experienced by the first patient dosed with SGT-001. We reported the event to the FDA and have recently received the formal clinical hold letter, which outlined the agency’s additional information requests. We are working tirelessly to develop a comprehensive plan to address these questions and are committed to maintaining dialogue with the FDA with the hope of resuming IGNITE DMD as soon as possible. Most importantly, we’re pleased to say the treating physician has reported the patient is doing well.

As part of our update, we are also happy to share that we have been able to successfully resolve the previously-announced partial clinical hold on the planned high dose of SGT-001 in IGNITE DMD, which was due to manufacturing-related questions from the FDA. While encouraging, IGNITE DMD remains on hold until we address the full clinical hold as mentioned above.

Solid is deeply committed to advancing our mission to solve DMD. Our goal is and always has been to bring forward only the programs that we believe have life-changing potential and develop a portfolio of treatments to benefit all patients, regardless of their underlying genetic mutation, age or stage of disease. We knew this wasn’t going to be easy, but our mission and the possibility of helping those affected by this cruel disease guide every decision we make and project we take on. 

We appreciate the Duchenne community’s continued support. We will provide additional updates in the future.

The Solid Team

Industry Partner News: Catabasis Pharmaceuticals Aligns Resources to Focus on Lead Program Edasalonexent for the Treatment of Duchenne Muscular Dystrophy


CAMBRIDGE, Mass., April 17, 2018Catabasis Pharmaceuticals, Inc. , a
clinical-stage biopharmaceutical company, today announced a restructuring of the organization to focus resources on the Company’s late-stage lead program, edasalonexent for the treatment of Duchenne muscular dystrophy (DMD). Catabasis is prioritizing this program to deliver against its goal of bringing a life changing therapy to those affected by Duchenne.

“This decision best positions us to achieve success with our most advanced program to help Duchenne patients and to support the long-term growth of Catabasis. However, on a personal
level, this decision was difficult and I want to thank the talented and dedicated colleagues who
are affected for their hard work and commitment to our mission,” said Jill C. Milne, Ph.D., Chief
Executive Officer of Catabasis. “Based on disease-slowing data from our MoveDMD trial, we
believe edasalonexent can make a significant difference in the lives of boys affected by Duchenne. These important corporate changes will allow us to focus our resources on continuing
to advance edasalonexent and improving the lives of boys affected by this devastating disease.”

For more on this decision, please see the full press release here.

Industry Partner News: Pfizer’s Letter to the Duchenne Community Regarding Initiation of Gene Therapy Trial


April 12, 2018

Pfizer is pleased to provide the following update regarding the Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD). The first boy received an infusion of the mini-dystrophin gene on March 22nd, administered under the supervision of the principal investigator at the study site.  This first participant continues to be monitored.

The study will enroll approximately 12 ambulatory boys aged 5 to 12 years with DMD.  In addition to evaluating safety and tolerability, the study will evaluate measurements of dystrophin expression and distribution, as well as assessments of muscle strength, quality, and function. As part of the screening process, potential candidates invited by the study principal investigator will be tested to confirm a negative result for antibodies against the adeno-associated virus, serotype 9 (AAV9) capsid and for a T-cell (immune) response to dystrophin. Screening and enrollment of patients is expected to continue at up to four clinical research sites in the United States.   Early data from this trial are expected in the first half of 2019, once the first patient completes one full year post-treatment. More information about the trial and participating sites may be found at (NCT03362502).

We recognize the commitment that the DMD community has shown for this study and for Pfizer’s research and development efforts related thereto. We further recognize the potential impact innovative therapies, like gene therapy, may have in transforming the lives of individuals and families affected by DMD and we share with you that hope and vision. It is with heartfelt gratitude that we extend our sincerest ‘thank you’ to all the boys and families who continue to express interest in and who participate in clinical research.  We are furthermore indebted to the advocacy associations and advocates who provide the tools and support needed for families to engage in clinical research, who continue to care for the Duchenne community and who lend their expertise in the research and development process.

Additional public-facing information about this milestone achievement can be found at:

Kind regards,
Katherine Beaverson
Patient Advocacy Lead

Industry Partner News: Letter to the Duchenne Community from Roche

Dear Duchenne community members,

We are happy to let you know that recruitment of participants into our Phase 2/3 clinical study (study WN40227) with the investigational molecule, RG6206, has now restarted.

RG6206 is an anti-myostatin adnectin which blocks the activity of myostatin and is given by subcutaneous injection (injection under the skin). Clinical study WN40227 is assessing the safety and efficacy of RG6206 in ambulatory boys aged 6-11 years with Duchenne ( reference NCT030393686).

Study WN40227 is open to new participants in the US and additional study sites in Europe, Australia, Argentina, Japan, the US, and Canada will be opened in the upcoming months.
Please visit and for more information about the
WN40227 study, the countries and sites that are part of the study. You can also read more about our Duchenne programme at

If you have questions about the WN40227 study or about RG6206, please contact me at

Best regards,
Sangeeta Jethwa, MD, on behalf of the Roche Duchenne team
Head, Patient Partnership, Rare Disease, Roche, Switzerland

Letter to the Duchenne community from Solid Biosciences about the status of the IGNITE DMD Clinical Trial

Dear Duchenne community members,

Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment and, as of the writing of this letter, is home with his family with no symptoms. Details about the event can be found in the press release we issued today, which is available here. The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.

Since our inception, we have been focused on working to bring meaningful treatments to patients with Duchenne muscular dystrophy (DMD). This cause is deeply personal to our company, which was founded and is led by individuals touched by DMD, and we are committed to identifying and bringing medicines to patients safely and responsibly. These principles underlie everything we do as an organization, and we take them seriously.

We greatly appreciate the trust placed in us by patients and families and will honor that through our dedication and work to end this disease. We appreciate your patience while we work to resolve this situation.

Industry Partner News: Sarepta Therapeutics Announces Plan to Submit a New Drug Application for Accelerated Approval of Golodirsen in Patients with Duchenne Amenable to Skipping Exon 53


CAMBRIDGE, Mass., - Sarepta Therapeutics announced on March 12, 2018 that it recently received final minutes from a February 2018 Type C meeting held with the Division of Neurology Products, United States Food and Drug Administration (the Division), to solicit the Division's guidance on the development pathway for Sarepta's therapeutic candidate, golodirsen, a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy (DMD) who have genetic mutations subject to skipping exon 53 of the DMD gene.

Please see the full release from Sarepta for more details. In the coming and weeks and months, we hope to learn more from Sarepta about what this guidance means for our patient community, especially those who may benefit from exon 53 skipping.

Industry Partner News: Update on Roche's RG62026 Phase 2/3 Study


Roche recently shared an update regarding their phase 2/3 clinical study (study 227) with RG6206, an
investigational anti-myostatin adnectin, in ambulatory boys with Duchenne muscular dystrophy ( reference

An unexpected technical issue in the drug supply process was detected, potentially impacting the availability of RG6206 for this ongoing study. Their priority is to ensure that boys in the study can continue study drug uninterrupted. To ensure they meet their commitments to new study participants, we have paused recruitment at all sites until we have resolved this situation.

For more information and resources, please see their most recent community update letter.

Industry Partner News: Summit Announces New Analysis Showing Ezutromid Significantly Reduced Muscle Inflammation in Phase 2 Clinical Trial in DMD

Summit logo.png

Summit Therapeutics announced additional positive data from PhaseOut DMD, showing a significant reduction in muscle inflammation after 24 weeks of ezutromid dosing. The findings in this 24-week interim data are consistent with the expected activity of ezutromid to stabilize muscle fiber membranes and thereby reduce muscle fiber damage and inflammation. A statistically significant and meaningful reduction in muscle fiber damage was observed in previously reported 24-week findings from patient biopsies in PhaseOut DMD, and now we’re reporting a decrease in muscle inflammation.

Learn more at

Industry Partner News: PTC Therapeutics Receives Formal Dispute Resolution Request Decision from the FDA's Office of New Drugs

The FDA’s Office of New Drugs has denied PTC Therapeutics' appeal of the Complete Response Letter in relation to the New Drug Application (NDA) for ataluren. The FDA has recommended a possible path forward for ataluren review in the US. This would involve PTC resubmitting an NDA for ataluren under the accelerated approval framework utilizing the current efficacy and safety data in conjunction with new data to be generated on dystrophin production in nonsense mutation Duchenne patients. PTC Therapeutics is in discussions with the FDA on the methods to collect the dystrophin data and expedite this potential path forward.

To read the full announcement, please click here.

FDA published “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry


Today, the Food and Drug Administration published “Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment” Guidance for Industry. Guidances such as these are reflective of the agency's current thinking on this topic. Jett Foundation hopes that this guidance will encourage further drug development in Duchenne, and provide a clearer path towards the FDA approval of safe and effective treatments that improve and extend the lives of children and young people living with Duchenne. 

Read more: Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry

Industry Partner News: Catabasis Reports Edasalonexent Preserved Muscle Function and Substantially Slowed Duchenne Muscular Dystrophy Disease Progression

Catabasis Pharmaceuticals, Inc. reported new positive efficacy and safety results showing preservation of muscle function and sustained disease-modifying effects in boys with Duchenne muscular dystrophy (DMD) in the MoveDMD trial open-label extension following 48 and 60 weeks of treatment with edasalonexent. Consistent improvements in all assessments of muscle function were observed after more than a year of oral 100 mg/kg/day edasalonexent treatment compared to the rates of change in the pre-specified control period for boys prior to receiving edasalonexent treatment. Additionally, supportive changes in non-effort based measures of muscle health were seen, with significant longer-term reductions in muscle enzymes and C-reactive protein (CRP), supporting the durability of edasalonexent treatment effects. Edasalonexent continued to be well tolerated with no safety signals observed in the trial. These data will be presented on Saturday, February 17, 2018, at 14:15 CET at the XVI International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy, and detailed at future scientific conferences.

Read more by clicking here.