BOULDER, CO, USA I July 12, 2022 I Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today announced the initiation of the CANYON Phase 2 clinical trial evaluating EDG-5506 in individuals with BMD. EDG-5506 is an orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as BMD and Duchenne muscular dystrophy (DMD). There are currently no approved treatments for BMD.
CANYON will assess the effect of EDG-5506 over a 12-month period on safety, pharmacokinetics (PK), biomarkers such as creatine kinase (CK), and functional measures in individuals with BMD aged 12 years and above. This placebo-controlled trial is anticipated to recruit approximately 66 individuals at up to 14 sites in the United States, United Kingdom and Netherlands. To learn more about this trial (NCT05291091), go to clinicaltrials.gov.
“We are delighted to advance EDG-5506 into Phase 2, based on the safety and positive biomarker data observed in our Phase 1 and ARCH studies,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “In the CANYON trial, we aim to understand the effect of EDG-5506 on circulating biomarkers, muscle MRI, and functional measures, all of which could provide indications of a treatment effect.”
“I look forward to working with fellow investigators and the BMD community on this Phase 2 trial of EDG-5506,” said Brenda L. Wong, M.D., Clinical Director, Duchenne Muscular Dystrophy Center, UMass Memorial Medical Center and Professor of Pediatrics and Neurology, University of Massachusetts Medical School. “Individuals with BMD and their families have been waiting for a while for clinical trials and it is indeed an exciting time to be a part of this trial. BMD is a serious, progressive, debilitating neuromuscular disorder, which causes great hardship to individuals with BMD and their families and caregivers.”
About Becker Muscular Dystrophy
BMD is a serious, progressively debilitating, and potentially fatal inherited X-linked neuromuscular disorder. BMD results from mutation of the dystrophin gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing muscle fiber (myofiber) degeneration that eventually leads to fibrosis, progressive loss of skeletal muscle function, and that can lead to severe disability and early death. BMD typically presents with juvenile onset of muscle wasting and progressive symmetrical, proximal muscle weakness, calf hypertrophy, activity-induced muscle cramping and elevated creatine kinase activity. While the course of BMD is variable, it is unidirectional in terms of the inevitable progressive limb weakness resulting in severe disability. BMD is also associated with early mortality from cardiac disease. The incidence of BMD is approximately 1 in every 18,450 live male births. It is estimated that there are between 4,000 to 5,000 individuals with BMD in the U.S., with similar numbers of individuals living with BMD in Europe. Despite the seriousness of the disease, for many with BMD, the disease remains one of considerable unmet medical need as there are no approved therapies in the U.S.
About EDG-5506 for DMD and BMD
EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.
The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center trial is assessing the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). We anticipate initiation of a Phase 2 trial in individuals with DMD in the second half of 2022.
About Edgewise Therapeutics
Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare neuromuscular and cardiac disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle-focused drug discovery platform. Edgewise’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, Twitter and Facebook.
SOURCE: Edgewise Therapeutics
Post Views: 165
BOULDER, CO, USA I July 12, 2022 I Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today announced the initiation of the CANYON Phase 2 clinical trial evaluating EDG-5506 in individuals with BMD. EDG-5506 is an orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as BMD and Duchenne muscular dystrophy (DMD). There are currently no approved treatments for BMD.
CANYON will assess the effect of EDG-5506 over a 12-month period on safety, pharmacokinetics (PK), biomarkers such as creatine kinase (CK), and functional measures in individuals with BMD aged 12 years and above. This placebo-controlled trial is anticipated to recruit approximately 66 individuals at up to 14 sites in the United States, United Kingdom and Netherlands. To learn more about this trial (NCT05291091), go to clinicaltrials.gov.
“We are delighted to advance EDG-5506 into Phase 2, based on the safety and positive biomarker data observed in our Phase 1 and ARCH studies,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “In the CANYON trial, we aim to understand the effect of EDG-5506 on circulating biomarkers, muscle MRI, and functional measures, all of which could provide indications of a treatment effect.”
“I look forward to working with fellow investigators and the BMD community on this Phase 2 trial of EDG-5506,” said Brenda L. Wong, M.D., Clinical Director, Duchenne Muscular Dystrophy Center, UMass Memorial Medical Center and Professor of Pediatrics and Neurology, University of Massachusetts Medical School. “Individuals with BMD and their families have been waiting for a while for clinical trials and it is indeed an exciting time to be a part of this trial. BMD is a serious, progressive, debilitating neuromuscular disorder, which causes great hardship to individuals with BMD and their families and caregivers.”
About Becker Muscular Dystrophy
BMD is a serious, progressively debilitating, and potentially fatal inherited X-linked neuromuscular disorder. BMD results from mutation of the dystrophin gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing muscle fiber (myofiber) degeneration that eventually leads to fibrosis, progressive loss of skeletal muscle function, and that can lead to severe disability and early death. BMD typically presents with juvenile onset of muscle wasting and progressive symmetrical, proximal muscle weakness, calf hypertrophy, activity-induced muscle cramping and elevated creatine kinase activity. While the course of BMD is variable, it is unidirectional in terms of the inevitable progressive limb weakness resulting in severe disability. BMD is also associated with early mortality from cardiac disease. The incidence of BMD is approximately 1 in every 18,450 live male births. It is estimated that there are between 4,000 to 5,000 individuals with BMD in the U.S., with similar numbers of individuals living with BMD in Europe. Despite the seriousness of the disease, for many with BMD, the disease remains one of considerable unmet medical need as there are no approved therapies in the U.S.
About EDG-5506 for DMD and BMD
EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.
The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center trial is assessing the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). We anticipate initiation of a Phase 2 trial in individuals with DMD in the second half of 2022.
About Edgewise Therapeutics
Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare neuromuscular and cardiac disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle-focused drug discovery platform. Edgewise’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, Twitter and Facebook.
SOURCE: Edgewise Therapeutics
Post Views: 165