Wave Life Sciences announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative trial designs (CID). The selection was based on the design of Wave’s Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for people with Duchenne who are amenable to exon 51 skipping. This marks the first time that the FDA has selected clinical protocols for its CID pilot program that was announced in August 2018.
Drug development and designing clinical trials in rare diseases is different than that of large, common diseases. There are not as many patients available to participate in clinical trials, and thus rare diseases are not as well studied. Not unlike the principles behind platform trial design that PPMD continues to explore, the FDA, in its commitment to helping drug development for rare diseases, launched a program to provide enhanced regulatory interactions and guidance for companies who are developing a clinical trial that uses innovative trial designs (such as incorporating adaptive trial design and/or using historical control data to enrich placebo arm data) and is aimed at treating a serious disease with no treatment. We are delighted to learn that Wave Life Sciences, as they continue developing an exon skipping platform of drugs using a novel backbone, will be one of the first participants in this FDA program. Because the program is designed to benefit all in rare disease drug development, much of the learnings will be public and transparent, allowing others to learn quickly.
Read the announcement from Wave:
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilot Program
Suvodirsen (WVE-210201) Phase 2/3 trial chosen in inaugural round of pilot program
FDA pilot program is a 21st Century Cures Act initiative
CAMBRIDGE, Mass., Jan. 03, 2019 (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 planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative trial designs (CID). The selection was based on the design of Wave’s Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with DMD who are amenable to exon 51 skipping. This marks the first time that the FDA has selected clinical protocols for its CID pilot program that was announced in August 2018.
In evaluating submissions for the CID pilot program, the FDA considered two key criteria: the innovative features of the trial design and the therapeutic need (i.e., therapies being developed for use in disease areas where there are limited or no treatment options). Wave’s application for the CID pilot program includes a plan to leverage DMD historical control data to augment the placebo arm of the suvodirsen Phase 2/3 clinical trial, among other innovative design elements. Through this pilot program, Wave intends to reduce the number of patients required to deliver conclusive clinical efficacy results, thereby minimizing the number of patients required in the placebo treatment arm and potentially accelerating study completion. As a participant in the pilot program, the company will also have additional opportunities to meet with FDA staff to discuss the design elements of the trial, including the use of Bayesian methods to adapt the trial and allow for more efficient and productive clinical determinations. Details of Wave’s Phase 2/3 trial design will be presented at upcoming scientific meetings.
“In designing our clinical trials, we are constantly looking to maximize the probability of a definitive result, incorporate the feedback of patients and their families, and reduce the burden on those who are already bravely enduring the challenges associated with serious, genetically-defined diseases. The FDA’s recognition of our plan reflects the thoughtful and collaborative way in which we approach clinical development,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We look forward to further discussions with the FDA in the coming months and sharing learnings from our trial design with others in the rare disease drug development community to drive greater efficiency and productivity in future clinical studies.”
Wave anticipates initiating the global, placebo-controlled Phase 2/3 efficacy and safety clinical trial of suvodirsen in DMD patients amenable to exon 51 skipping in 2019. The trial is designed to measure clinical efficacy and dystrophin expression, and Wave intends to use the results of this trial to seek regulatory approvals globally. Currently, suvodirsen is being studied in an ongoing open-label extension (OLE) study and Wave expects to deliver an interim analysis of dystrophin expression from this study in the second half of 2019.
The FDA CID pilot program is an initiative under the 21st Century Cures Act, with an objective to modernize clinical trial design, help streamline and advance drug development and inform easier regulatory decision-making. In order to qualify for the CID pilot program, companies must intend to provide substantial evidence of efficacy through a complex, novel design that incorporates innovative trial design elements such as seamless trial designs, modeling and simulations to assess trial operating characteristics, the use of biomarker enriched populations, complex adaptive designs, Bayesian models and other benefit-risk determinations, among others. For more information, visit https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm617212.htm.