CureDuchenne – Clinical Trials – FDA

Following the long awaited news of Sarepta’s phase IIB results for eteplirsen and continued Phase III progress of GSK/Prosensa’s drisapersen, as well as the recent developments with PTC Therapeutics and Summit PLC among others, CureDuchenne took the initiative to reach out for patients and advocacy groups alike to begin a dialogue with the FDA and the broader community on issues related to future clinical trial design, expedited approval strategies, clinical endpoints and strategies for multiple exon skipping. It is hoped that these efforts will benefit those product opportunities currently in clinical development as well as future development programs.

It is encouraging to see so many product opportunities making their way into human clinical trials, with the most advanced getting closer to regulatory approval. However it is important to remember that the overall process is incredibly risky and while we remain very hopeful that the first drug will be approved soon, the drug development and approval process still suffers from high attrition rates and has the ability to disappoint at any moment.  It’s important to also remember that a lot of requirements must be met for an approved product to reach those who need it – and not all of the hurdles are regulatory in nature.  A product can be approved as safe and efficacious but could still present challenges to the patient and caregiver for example: issues such as production on a commercial scale, product availability, cost to patient and potential reimbursement issues can be stumbling blocks to patients getting access to the drug.



Initiative What We Want To Accomplish? CureDuchenne’s Involvement
FDA Communication
  1. Work in partnership with the FDA, reinforcing the urgency in getting drugs to treat DMD approved as quickly as possible.
  2. In parallel, work with the agency to help define an understanding of the Risk/Benefit profile that is acceptable to Duchenne families and patients.
CureDuchenne has met with FDA representatives and is in the process of expanding discussions around Risk/Benefit in DMD.
Longitudinal Study of Duchenne and Collection of Natural  History Data This process will help us understand the natural progression of the disease, as well as its different stages, and defines reliability and validity of outcome measures. All of this contributes to best clinical trial design for Duchenne with appropriate outcome measures CureDuchenne has supported this effort for three years. Most recently the 3rd outcomes measure workshop took place in conjunction with the CINRG Annual Meeting, and the NIDRR Annual Meeting, where all of these issues were discussed.
Best Practices in Clinical Study Design for Rare Diseases This meeting is designed to provide a forum for stakeholders (academics, industry, FDA, NIH, EMA) to discuss different aspects of clinical trial design in rare disease, including DMD. The meeting will have four roundtable sessions; statistical challenges, therapeutic development challenges, clinical development tool challenges and regulatory challenges.  The meeting hopes to provide valuable scientific information on specific aspects of rare disease drug development that may facilitate product development in rare disease. CureDuchenne has been asked to attend the meeting and participate as a panelist in the Therapeutic Development Challenges Roundtable. The meeting will take place in April and is not a public meeting.
Meeting on Clinical Study Endpoints in Non-Ambulatory DMD TRND, part of the NIH, has two Duchenne projects and is interested in exploring alternate end points to the 6MWT, and those appropriate for non-ambulatory boys. They attended the CINRG/NIDRR meeting and currently are developing their agenda with input from all stakeholders. CureDuchenne is involved in defining the agenda to ensure that it is synergistic with the meeting in April (above). This meeting will be held in May.
Upper Limb Scale Development A group of global Physical Therapists are working on an upper limb scale that could be used as an outcome measure in clinical studies. Recent results were presented at the CINRG/NIDRR meeting. Efforts are ongoing to refine this. CureDuchenne has supported these efforts through the Outcome Workshops initiative started in 2010.
Sarepta – Exon 51 Development Sarepta has completed a phase llb trial and will seek accelerated approval for its exon 51 skipping drug. CureDuchenne, in collaboration with CNMC and Foundation to Eradicate Duchenne, provided the funding which enabled Sarepta (AVI) to move into clinical trials.
Sarepta  – Exon 45 and 53 Development Through a development agreement with Sarepta, Carolinas Medical Center, and CNMC, a drug targeting exon 45 is being tested in preparation for filing an IND. Additionally, a drug targeting exon 53 is being prepared. Abby Bronson, partially supported by CureDuchenne, is the project manager for this effort and is ensuring it moves as quickly as possible.
Prosensa – Exon 51 Glaxo Smith Kline, which has licensed Prosensa’s exon 51 skipping drug, is completing its phase lll pivotal trial and is expected to apply for an NDA in 2014. CureDuchenne provided the early seed money to Prosensa to develop their exon skipping drugs.  Our funding was crucial to enable the company to attract major investors which have contributed to their drug development programs.
Prosensa – Exon 44 Prosensa is conducting a Phase IIa clinical trial and has obtained an orphan drug designation in the EU and the US. CureDuchenne provided the early seed money to Prosensa to develop their exon skipping drugs.
PTC Therapeutics Develop and market a drug that will replace dystrophin in Duchenne patients that have a stopcodon mutations. CureDuchenne provided significant funding for the initiation of this research.
Summit PLC Develop and market a drug which will upregulate utrophin in Duchenne patients. This could help all patients, regardless of mutation. CureDuchenne, along with several collaborators, funded the phase l trial.
Halo Therapeutics Develop and market a drug which will reduce fibrosis in Duchenne patients, a very important target which could work in a complementary way with dystrophin/utrophin restoring drugs. CureDuchenne, along with several collaborators, funded an IND enabling studies and first clinical study.

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