Duchenne and Becker Muscular Dystrophy

– Company to host an Industry Forum to discuss its approach to protecting and preserving dystrophic muscle featuring key opinion leaders – BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on EDG-5506, an investigational therapy designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies, at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. The conference will take place at the Hilton Orlando in Orlando, FL from March 3-6, 2024. Details of the Edgewise presentations at MDA: Podium Presentation in Clinical Trial Updates Session Title: Functional and Muscle Damage Biomarker Changes Following Treatment with EDG-5506, a Fast Myosin Modulator, in Adults with Becker Muscular Dystrophy Dr. Phan’s presentation will include 12-month data from the ARCH trial of EDG-5506. Presenter: Han C. Phan, M.D., Head of Research & Principal Investigator, Pediatric Neurologist, Rare Disease Research, LLC Date: Wednesday, March 6, 2024, 11:15 – 11:30 am ET Scientific Posters Title: Comparison of Short- and Long-Term Proteomic Response to the Fast Skeletal Myosin Inhibitor, EDG-5506, in Becker Muscular Dystrophy (BMD) (Poster # M145) Title: North Star (NSAA and NSAD) Functional Assessments in Individuals with Becker Muscular Dystrophy (poster # M147) A companion poster presentation will be available for Dr. Phan’s presentation (poster # M146). Posters will be showcased during the Networking & Poster Reception in the Exhibit Hall on Monday, March 4, 2024, from 6 – 8 PM ET. The full MDA 2024 Conference program is available here: . The Edgewise presentation and posters will be available on the Edgewise website when they are presented. Edgewise Industry Forum with Key Opinion Leaders On March 4, 2024, at 12 pm ET, Edgewise is sponsoring an Industry Forum, “Protecting and preserving dystrophic muscle: The balance between exercise and contraction-induced muscle injury.” The forum will feature presentations by leading neuromuscular disease experts, John Vissing, M.D., Ph.D., Professor of Neurology, Director Neuromuscular Clinic and Research Unit, Department of Neurology, University of Copenhagen, and Tanja Taivassalo, Ph.D., Associate Professor, Department of Physiology and Aging, University of Florida, as well as Edgewise Chief Medical Officer, Joanne Donovan, M.D., Ph.D. Only registered conference attendees can register for the symposium. About EDG-5506 for Duchenne and Becker Muscular Dystrophies EDG-5506 is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Duchenne and Becker. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle damage that leads to functional impairment, EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders. Its unique mechanism of action provides the potential to establish EDG-5506 as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. The Company is advancing EDG-5506 through the clinic including completing enrollment of a Phase 2 trial cohort, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker, which has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON, which is currently enrolling. In Duchenne, the Company is advancing its Phase 2 clinical trials, LYNX, assessing safety, pharmacokinetics and biomarkers of muscle damage, and FOX, which includes children and adolescents previously treated with gene therapy. For more information on Edgewise’s clinical trials . About Edgewise Therapeutics Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in muscle physiology is driving a new generation of first-in-class therapeutics. EDG-5506 is an orally administered skeletal myosin inhibitor in clinical trials in patients with Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel cardiac sarcomere modulator for the treatment of HCM and other disorders of cardiac diastolic dysfunction. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: or follow us on LinkedIn, X (formerly Twitter), Facebook, Instagram and Threads.

Leading Duchenne Organization Expands Certification of Clinics that Provide Optimal Care for People with Duchenne Muscular Dystrophy with Addition of Clinic in Eastern Pennsylvania WASHINGTON, Feb. 21, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced the expansion of their renowned Certified Duchenne Care Center (CDCC) Program with the certification of the clinic at Penn State Health Children's Hospital in Hershey, Pennsylvania. This designation marks a significant step for the CDCC Program and continues the growth and expansion to bring comprehensive care to all people living with Duchenne and Becker muscular dystrophy. Under the guidance of Dr. Ashutosh Kumar, neurologist and clinic director, Penn State Health Children's Hospital is a growing clinic that currently serves approximately 50 individuals living with Duchenne and Becker muscular dystrophy. "The Penn State Health Pediatric Neuromuscular Multidisciplinary Care Team has worked tirelessly to provide a family and patient-centered approach to the care and support of children with Duchenne and Becker muscular dystrophy," said Dr. Ashutosh Kumar, pediatric neurologist and assistant professor of pediatrics and neurology at Penn State Health Children's Hospital. "We believe strongly in the mission and goals of PPMD and are extremely proud to achieve this prestigious care distinction. This achievement underscores the organization's dedication to promoting excellence in Duchenne care. Penn State Health is committed to collaborating with PPMD to help obtain early diagnosis, maximize functional abilities, improve patient quality of life, and further research and treatment efforts for this complex and challenging disease." As part of the rigorous CDCC application process, Dr. Mena Scavina, PPMD's Certified Duchenne Care Advisor, conducted a comprehensive site visit to the clinic in late 2023 and praises the team's dedicated efforts in delivering an all-encompassing and supportive clinic experience for patients and their families. Dr. Scavina noted, "The commitment of the team at Penn State Children's to care for individuals with dystrophinopathies is commendable. Their emphasis on providing a comprehensive and resourceful clinic experience for families, active engagement in clinical trials, and enthusiasm about working with colleagues in the CDCC network, showcases their dedication to advancing care for their patients." PPMD's Certified Duchenne Care Center Program supports standardized, comprehensive care and services for all people living with Duchenne. Certification means centers maintain the highest standards in clinical and sub-specialty services, rapidly apply new evidence-based knowledge, minimize heterogeneity in clinical research outcomes, and comply with standards in clinical care that were established by the CDC's Care Considerations. As part of its ongoing mission to end Duchenne, PPMD continues to insist that all people with Duchenne receive comprehensive care. Rachel Schrader MS, APRN, CPNP-PC, PPMD's Vice President of Clinical Care and Education, says the certification of Penn State Health Children's Hospital is an exciting addition to the CDCC network and its ultimate goal of bringing optimal Duchenne care to all people, which will now expand to the eastern region of Pennsylvania and surrounding areas. "It is evident that the neuromuscular team at Penn State Health Children's Hospital goes above and beyond for their patients and families affected by Duchenne and Becker. We are thrilled to add their clinic to the growing CDCC network and look forward to expanding access to comprehensive and coordinated dystrophinopathy care." To learn more about PPMD's Certified Duchenne Care Center Program, visit PPMD's website. Click here to learn more about the history of PPMD's Certified Duchenne Care Center Program and to access PPMD's first published article on the program. ABOUT PARENT PROJECT MUSCULAR DYSTROPHY: Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne. We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won seven FDA approvals. Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube. ABOUT PENN STATE HEALTH CHILDREN'S HOSPITAL: Penn State Health Children's Hospital is the only children's hospital between Pittsburgh and Philadelphia fully equipped to treat the most severely ill children of central Pennsylvania, with both the highest-level neonatal intensive care unit and a Level I pediatric trauma center. Children's Hospital physicians and nurses provide comprehensive support and specialized care to infants, children and adolescents every day. Located on the campus of Penn State Health Milton S. Hershey Medical Center in Hershey, Pa., the Children's Hospital focuses on providing first-rate health care to all children, from those with complex heart disease to childhood cancers, allowing patients to receive the full spectrum of highest-quality care close to their homes. SOURCE Parent Project Muscular Dystrophy (PPMD)

The fast track designation is set to expedite the development and review of EDG-5506. Credit: Gorodenkoff / Shutterstock.com. The US Food and Drug Administration (FDA) has granted fast track designation to Edgewise Therapeutics’ investigational drug, EDG-5506, for Duchenne muscular dystrophy. The designation expedites the development and review of EDG-5506, an oral small molecule for the prevention of contraction-induced muscle damage in dystrophinopathies such as Duchenne and Becker muscular dystrophy. The FDA had previously recognised the potential of the therapy by granting it orphan drug and rare paediatric disease designations for Duchenne. It had also received fast track status for the treatment of Becker muscular dystrophy. EDG-5506 is being evaluated in ongoing placebo-controlled clinical trials including the Phase II LYNX trial in children with Duchenne aged four to nine years and the Phase II FOX study in children and adolescents who previously received gene therapy. The three-month controlled, dose-ranging findings from the LYNX trial are anticipated in the second quarter of 2024 and will inform the Phase III dose. See Also: Otsuka wins FDA breakthrough therapy designation for IgAN drug QBiotics wins FDA orphan status for rainforest-derived cancer drug LYNX is analysing the safety, pharmacokinetics and biomarkers of muscle damage. In addition to the LYNX and FOX trials, the company is completing subject enrolment for the CANYON cohort of its Phase II trial in adult males with Becker muscular dystrophy. This cohort will assess the safety and impact on function and muscle damage biomarkers in adult males with Becker. The GRAND CANYON cohort, an extension of this trial, is enrolling an additional 120 adult participants. Edgewise president and CEO Kevin Koch stated: “Every day is important for individuals living with Duchenne and their families, and we are pleased that the FDA granted fast track designation. “Our roster of regulatory designations granted to EDG-5506 highlights the urgent and critical need for new and better therapeutic options for people living with these rare and life-threatening muscle disorders.”