PPMD and Santhera Partner Up on Duchenne Risks Study

Parent Project Muscular Dystrophy (PPMD) has partnered with Swiss pharmaceutical company Santhera Pharmaceuticals to conduct a collaborative study on the benefits and risks of Duchenne muscular dystrophy therapies. The researchers, which will use data from Santhera’s phase 3 clinical trial of idebenone, will be focused on determining both patients’ and caregivers’ preferences about DMD therapeutics.

The company and the organization have joined forces, as the former is currently developing innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, while the latter is focused on advocating and supporting other projects that contribute to treating and curing Duchenne, which is the most common fatal genetic condition that affects about 1 in every 3,500 to 5,000 male births.

“Since entering the Duchenne space, Santhera has proven their dedication to this community and to finding therapies to treat this disease,” explained PPMD’s Founding President and CEO, Pat Furlong. “They are committed to provide thorough and reliable data and information in the submission to the FDA when they reach that stage. We are happy to collaborate with Santhera’s team to ensure the patient’s voice is included in the benefit/risk equation. The FDA has told us they want to know what the patients want and thanks to this study we will be able to tell them.”

Earlier this year, PPMD convened a large coalition including more than 80 DMD stakeholders as a way of creating a draft of the first patient advocacy-initiated guidance for a rare disease, to be submitted to the U.S. Food and Drug Administration (FDA). The main purpose of the coalition is to accelerate the development and revision of potential new treatments for Duchenne. Among the recommendations, the creation of partnerships between patient groups and industry members was the onus for the PPMD/Santhera collaboration. Their partnership will seek to gain more insight into understanding the benefits and risks related to the disease, and increasing awareness of new drug applications within the DMD community.

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“Following the successful outcome of our Phase 3 trial, we are excited about this collaboration with PPMD to determine patients’ and caregivers’ views of the benefit/risk balance for Catena/Raxone,” said the CEO of Santhera, Thomas Meier. “PPMD has become a powerful and reliable partner of industry in facilitating the development of promising treatment options. With this study of the benefit/risk preferences of the disease community, which is the first of its kind, we will proactively meet regulators’ expectations in the content of our NDA filing.”

The PPMD is dedicated to advocacy and awareness activities for DMD, which most recently included naming the Kennedy Krieger Institute in Baltimore, Maryland a Certified Duchenne Care Center, in recognition of its dedication to the improvement of healthcare services for Duchenne patients. In November, the organization also hosted the second annual “Strength, Science, & Stories of Inspiration” event, in partnership with the Massachusetts Institute of Technology (MIT) and the Myotonic Dystrophy Foundation (MDF).