LIMB-GIRDLE MUSCULAR DYSTROPHY COALITION TO HOST EXTERNALLY-LED DRUG DEVELOPMENT MEETING
Falls Church, Virginia (July 5, 2022) — On September 23, 2022 at 10am EDT, a coalition of limb-girdle muscular dystrophy (LGMD) nonprofit organizations will lead an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD). This online event is open to the public with registration required. The meeting will be summarized in a Voice of the Patient report which, along with recorded footage of the meeting, will be publicly available.
This EL-PFDD will focus on six subtypes of LGMD: LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. The goal of this meeting is to provide the U.S. Food and Drug Administration (FDA), clinicians, medical product developers, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.
The meeting agenda will include speakers and panelists who are living with LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9, as well as caregivers of people living with these six subtypes. The morning sessions will focus on symptoms that matter most to them, and the toll these take on daily living. The afternoon session will include discussions on experiences with current treatments and attitudes towards future treatments, including tolerance to potential risks.
For the first time, representatives from Coalition to Cure Calpain 3, CureLGMD2I, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation, have joined together to organize this event. Jointly referred to as the LGMD Coalition, these nonprofit organizations are encouraging members of the global patient community to make their voices heard. Individuals living with the subtypes of LGMD listed above, as well as their caregivers, are invited to participate in the meeting by partaking in live polling and by calling and writing in with comments. Academic investigators, clinicians, regulators, and industry representatives are encouraged to watch the meeting and incorporate patient/caregiver perspectives when developing and reviewing new therapies to best meet the needs and expectations of our community. Pre-registration is available at LGMDPFDD.com.
The LGMD Coalition is grateful for support from our sponsors, AskBio, Edgewise Therapeutics, ML BioSolutions, Sarepta Therapeutics, and Vita Therapeutics. We also acknowledge our Advocacy Partners, the Muscular Dystrophy Association and the LGMD Awareness Foundation.
LGMD is a term used for a group of rare neuromuscular diseases which are inherited and cause muscle weakness and wasting. The muscles most affected are those closest to the body (proximal), specifically the muscles of the shoulders, upper arms, pelvic area, and thighs. More than 30 subtypes of LGMD exist. Together, LGMDs have an estimated prevalence of about 2 in every 100,000 individuals. There is currently no cure for any subtype of LGMD.
About the LGMD Coalition
The LGMD Coalition is a group of six 501(c)3 nonprofit organizations — Coalition to Cure Calpain 3, CureLGMD2I, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation – that are collaborating to host an LGMD EL-PFDD focused on subtypes LGMD2A/R1, LGMD2C/R5, LGMD2D/R3, LGMD2E/R4, LGMD2F/R6, and LGMD2I/R9. Learn more at LGMDPFDD.com.
At the end of November, the Kurt+Peter Foundation met with both Myonexus and Sarepta. Myonexus is projecting a 2020 start for a Phase 1/2 clinical trial for LGMD2C, with the qualification that the schedule remains fluid because of manufacturing capacity. We will continue to update the LGMD2C community as we learn more about Myonexus’s plans for the clinical trial.
We have discussed proposals from Dr. McNally at Northwestern University for the Kurt+Peter Foundation to fund(i) further work on the LGMD2C exon skipping program and (ii) a study of the impact of an alternative steroid dose regimen on LGMD2C patients. Kurt and Peter have started as the first two patients in the alternative steroid regimen study.
The Kurt+Peter Foundation discussed with Northwestern University and the University of Chicago avenues to further the LGMD2C exon skipping program.
The Kurt+Peter Foundation participated in a Myonexus Therapeutics Patient Advisory Board telephone conference with senior management of Myonexus Therapeutics and learned about Myonexus’s plans for engagement with the LGMD community.
Exciting news! Sarepta Therapeutics, Inc. announced that it has entered into a partnership with Myonexus Therapeutics that will allow Myonexus Therapeutics to fund further development, including manufacturing for various forms of Limb-girdle muscular dystrophies (LGMDs), including LGMD2C. As described here, Sarepta will make an upfront payment of $60 million and additional development-related milestone payments. More exciting news! Elizabeth McNally and Eugene Wyatt at Northwestern University published this paper describing how exon skipping works in LGMD2C. The publication of this peer-reviewed article helps further the exon skipping program. The research was exclusively funded by the Kurt+Peter Foundation.
The Kurt+Peter Foundation continued its dialogue with Myonexus Therapeutics regarding manufacturing for a gene therapy clinical trial.
On the occasion of the annual JP Morgan biotechnology conference in San Francisco, the Kurt+Peter Foundation met with the leadership of Myonexus Therapeutics to further discuss how the foundation could support manufacturing for a gene therapy trial for LGMD2C.
Thanks to the generosity of our friends and family, the Kurt+Peter Foundation held a very successful fundraiser. Thank you to everyone that contributed to the success of a terrific event! The San Francisco Business Times published a nice article about the Kurt+Peter Foundation here.
To build on its grant funding preclinical work for gene therapy for LGMD2C, and recognizing that manufacturing for a clinical trial for LGMD2C was a significant expense and potential source of delay, the Kurt+Peter Foundation began discussions with Myonexus Therapeutics [link] to see if the foundation could help fund a deposit with the contract manufacturer that would guarantee a position in line for manufacturing for a gene therapy clinical trial.
The U.S. Food and Drug Administration grants orphan drug status for the exon skipping technique developed by Dr. Elizabeth McNally, currently the Director of the Center for Genetic Medicine at, Northwestern University Feinberg School of Medicine.
Myonexus Therapeutics launches to develop and commercialize the technology developed at Nationwide Children’s Hospital for gene therapy for five different forms of limb girdle muscular dystrophy, including LGMD2C.
OptionB.Org, a foundation dedicated to helping individuals build resilience in the face of adversity, published the story of Kurt, Peter, Charlotte, and Scott, and the efforts of the Kurt+Peter Foundation.
The Kurt+Peter Foundation made a $126,000 grant to Nationwide Children’s Hospital to fund pre-clinical work necessary to pursue gene therapy for LGMD2C.
The Kurt+Peter Foundation made a $35,000 grant to Northwestern University to further the pre-clinical research into exon skipping being conducted by Dr. Elizabeth McNally.