PPMD’s Duchenne Drug Development Roundtable

PPMD’s Drug Development Roundtable (DDDR) is a group of over forty industry partners, engaged in developing therapeutics for Duchenne, that come together in the pre-competitive space to address some of the most urgent issues we face finding a cure for Duchenne. The goal is to accelerate the development of meaningful treatments for Duchenne muscular dystrophy through open discussion and collaboration. Membership is open to all biopharmaceutical sponsors within the Duchenne space; an annual fee structure to support DDDR meetings and activities is required of participants.

The DDDR Steering Committee

DDDR Steering Committee members are selected by PPMD Executive Leadership, are of a voluntary capacity, and represent the following categories all relevant to Duchenne: an small industry sponsor organization, a mid-size industry sponsor organization, a regulatory policy expert, a representative from the Duchenne patient community, and representatives from different research modalities.

  • Cliff Bechtold
  • Paul Bolno, MD, President & CEO, Wave Life Sciences
  • Joanne Donovan, MD, PhD, Chief Medical Officer, Catabasis
  • Cartier Esham, PhD, Executive Vice President of Emerging Companies, Biotechnology Innovation Organization (BIO)
  • Kim McCleary, Managing Director, FasterCures
  • Thomas Meier, PhD, CEO, Santhera Pharmaceuticals
  • Stu Peltz, PhD, CEO, PTC Therapeutics
  • Jeff Watkins (Patient Community Representative)

And from PPMD

  • Abby Bronson, MBA, SVP Research Strategy, PPMD
  • Annie Kennedy, SVP Legislation & Public Policy, PPMD

Building on Momentum

The key focus of 2017 was how we apply the ‘regulatory learnings’ of 2015 and 2016 back into our robust Duchenne pipeline to ensure as many effective therapies yield from our current pipeline as possible. To achieve this, we convened a ‘small meeting series’ during the spring comprised of three one-day meetings. Each meeting included leaders and innovators from outside the Duchenne space to present on opportunities to enhance our existing trial designs, tools, and infrastructure.

The meeting series topics include:

Clinical Trial Readiness – Patients, Families, and Treating Physicians

Optimizing Clinical Trial Design

Clinical Trial Readiness – Sites & Investigators

Looking Ahead

The three meetings yielded two distinct efforts that are each being moved forward by PPMD team members, and workgroups comprised of representatives from industry, clinicians, and the Duchenne community.

Clinical Trial Harmonization

We are working to establish a code of conduct for clinical trials in Duchenne. The code of conduct to will include ‘best practices in clinical trials in the Duchenne space’ and will be drafted by the DDDR working group and then refined by the full consortium throughout 2018.

Developing a  Master Protocol for Duchenne

The Master Protocol effort is one that is facilitating the exploration of a platform trial concept for Duchenne. Currently, each trial in the Duchenne space is designed and executed as an individual exercise, yet many steps that need to be completed are done for each trial, often at the same sites who run multiple trials at once. Each protocol is slightly different, yet most are using the same screening processes and outcome measures. A single protocol that harmonizes and standardizes some of these aspects of trials could enhance efficiency greatly.  The Master Protocol design team, a group of 10+ individuals representing industry, patients, and a biostatistics expert, is working to create a protocol that has the capacity to test multiple therapeutics at one time, eventually testing combinations of these therapies as well. This protocol will be presented and discussed at a Public Workshop in the fourth quarter of 2018.

Both the Clinical Trial Harmonization and Master Protocol exploration efforts are aimed at introducing efficiencies into our trial pipeline and ensuring that at all within our Duchenne community benefit from experimental therapies in development. Public report outs from these meetings are forthcoming and will continue outline progress and next steps in each area.

Annual DDDR Meeting – Convening Our Community

Each June, we convene our Annual Duchenne Drug Development Roundtable meeting which brings together all Duchenne industry sponsors, as well as global Duchenne advocacy partners. The meeting allows for a robust exchange of information about challenges before us, as well as novel opportunities for coming together to work through them.

We are confident the efforts of the DDDR will result in more efficiency, less redundancy and better science, leading to faster therapeutic options for our community.

Learn More

For more information please contact PPMD’s SVP of Development, Kaylan Moitoso.