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

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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

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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.

Industry Partner News: Capricor Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Duchenne Muscular Dystrophy Therapy

Capricor Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted CAP-1002, its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, the Regenerative Medicine Advanced Therapy (RMAT) designation.

“The RMAT designation is recognition by the FDA of the potential of CAP-1002 and the importance of bringing this therapy to market to serve the unmet needs of boys and young men who have lost the ability to walk because of Duchenne muscular dystrophy,” said Linda Marbán, Ph.D., Capricor president and chief executive officer. “CAP-1002 is one of the few therapies currently in development to help non-ambulant patients with Duchenne muscular dystrophy, and it’s important that we move forward into the next phase of clinical development to potentially help them maintain what function they have in their arms and hands.”

For the full announcement and more information, please click here. 

Industry Partner News: Santhera Launches U.S. Expanded Access Program with Idebenone for Patients with Duchenne Muscular Dystrophy (DMD)


Santhera Pharmaceuticals announces the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for patients with DMD.

Through the BreatheDMD program, eligible patients in the U.S. with DMD who are 10 years and older and in respiratory function decline, can obtain access to investigational idebenone, at no cost, through a growing network of research centers across the U.S. 

“Several patient advocacy organizations in the U.S., including Parent Project Muscular Dystrophy (PPMD), Jett Foundation, CureDuchenne and the Muscular Dystrophy Association (MDA) have consistently mentioned that patients need access to additional treatment options, especially older patients in respiratory decline, and that these patients cannot afford to continue to wait. To help address this important unmet need, Santhera is proud to be launching this EAP, allowing eligible patients to obtain access to investigational idebenone,” said Thomas Meier, PhD, CEO of Santhera.

For Santhera's full announcement, please click here. For more about BreatheDMD, visit

Industry Partner News: Summit Announces that Ezutromid Significantly Reduced Muscle Damage in DMD Patients in 24-week Interim Data from Summit's PhaseOut DMD Clinical Trial


This morning, Summit Therapeutics plc announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. This data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies. Summit plans to have a webinar with the community – please stay tuned for details.


1.       What does this data mean? This is an early look at data from PhaseOut DMD. In these data, we see that ezutromid treatment led to a significant reduction in muscle damage and increased production of utrophin in muscle fibers. This fits with our expectation that utrophin modulation maintains utrophin production in mature muscle fibers, enabling utrophin to replace the need for dystrophin in Duchenne muscular dystrophy muscles. We don’t yet know if ezutromid will be able to produce long term functional or other clinical benefits, but this is a very exciting step for ezutromid and utrophin modulation. Importantly, ezutromid has been well tolerated to date in all patients in PhaseOut DMD. We look forward to the results from the full 48 weeks of the trial expected in the third quarter of 2018.

2.       Will there be another clinical trial? When will that start/where will it take place? We expect to have to conduct another clinical trial aimed at getting regulatory approval for ezutromid to be marketed in the US and Europe. With these positive interim data in hand, we are actively planning for the next trial and expect to provide a timeline for the start of that trial once we have the 48-week data. It is expected to be a global trial, and the participating countries and sites will be announced closer to the initiation of the trial.

3.       When will ezutromid be available on the market? We are awaiting the 48-week data from PhaseOut DMD prior to finalizing our plans for the next trial. Once we have these plans in place, we will be able to better predict when we could potentially file for regulatory approvals. These filings would need to be approved by the applicable regulatory authorities before ezutromid would be available on the market.

4.       Can I get access to ezutromid before it’s approved? Running clinical trials that can support regulatory approvals is the best way to ultimately ensure wide access for patients to ezutromid. All of our efforts are therefore focused on conducting rigorous clinical trials to establish the safety and potential clinical benefits that ezutromid may have. At this point in the development, we cannot support any use of ezutromid outside of our clinical trials and any associated extension phases.

For more information, please see the full Summit press release by clicking here.

Industry Partner News: Santhera Anticipates Receiving a Negative CHMP Opinion on Appeal for Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy

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Santhera Pharmaceuticals anticipates Receiving a Negative CHMP Opinion on Appeal for Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy.

Liestal, Switzerland, January 24, 2018 – Santhera Pharmaceuticals (SIX: SANN) announces that on January 23 it had an oral explanation at the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in relation to the re-examination of its Marketing Authorization Application (MAA) for Raxone® (idebenone) in Duchenne muscular dystrophy (DMD). The company now anticipates that the CHMP will maintain its original position to issue a negative opinion on Santhera’s MAA filed as Type II variation of its existing marketing authorization for Leber’s hereditary optic neuropathy (LHON).

Santhera will provide further update on Friday, January 26, as originally announced.

For more information, please see their full release by clicking here.

Industry Partner News: Capricor Announces Licensing of Additional Patent Applications from Cedars-Sinai Medical Center for Cellular and Exosome-based Technologies


Capricor Therapeutics announced January 16th, 2018 that it has added seven new patent applications to its existing Exclusive License Agreements with Cedars-Sinai Medical Center for technologies related to cardiosphere-derived cells (CDCs) and CDC-derived extracellular vesicles, including exosomes. 

These new patent applications will pave the way for Capricor to develop new therapeutic platforms for Duchenne muscular dystrophy and other disorders. 

Read their full release by clicking here.

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Industry Partner News: Santhera Announces New Awareness Campaign

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Santhera Pharmaceuticals announces the launch of a disease awareness campaign for the Duchenne muscular dystrophy (DMD) community in the U.S. with a focus on respiratory care.

The "Take a Breath DMD" campaign will underscore the importance of respiratory care for people living with Duchenne muscular dystrophy. The mission of this educational campaign is to help people living with DMD and their families receive information to help manage respiratory complications, including information about breathing, coughing and pulmonary care.

For more information, please see their full release by clicking here.

Industry Partner News: Summit Completes Initial 24 Weeks of Dosing of Ezutromid in Patients with DMD in PhaseOut DMD Clinical Trial


Summit announces the completion of the initial 24-weeks of dosing of ezutromid in patients with DMD in its Phase 2 clinical trial called PhaseOut DMD. With this milestone, Summit continues to expect to report results from this initial 24-week dosing period in the first quarter of 2018. The 24-week results are expected to include data from muscle biopsies, MRI and functional tests, as well as safety data.

For more information, please see the Summit press release by clicking here.