Kye Pharmaceuticals Secures Exclusive License, Supply and Commercialization Agreement with Catalyst Pharmaceuticals for AGAMREE®, a novel treatment for Duchenne muscular dystrophy, in Canada

Duchenne muscular dystrophy is the most common form of muscular dystrophy.The Canadian Neuromuscular Disease Registry estimates there are more than 800 boys and young men in Canada currently living with Duchenne muscular dystrophy and coping with its debilitating symptoms.1

MISSISSAUGA, ON, July 24, 2024 /CNW/ – Kye Pharmaceuticals, Inc. (“Kye”), a commercial-stage biopharmaceutical company focused on identifying, licensing, and commercializing novel medicines for patients in Canada, today announced that it has entered into a License, Supply and Commercialization Agreement with Catalyst Pharmaceuticals Inc. (“Catalyst”), securing exclusive Canadian commercial rights to AGAMREE® (vamorolone), an novel corticosteroid for the treatment of Duchenne Muscular Dystrophy (“DMD”). Under the terms of the agreement, Catalyst will supply the product to Kye, who will assume full responsibility for obtaining regulatory approval for AGAMREE for the treatment of DMD from Health Canada and all future aspects of commercialization of the product within Canada. Kye currently markets FIRDAPSE® in Canada, Catalyst’s flagship product for the treatment of Lambert Eaton myasthenic syndrome.

“Our expanded partnership with Catalyst highlights the intrinsic value of our collaboration in developing innovative treatments for rare diseases, including DMD, and exemplifies our shared dedication to improving health outcomes across the U.S. and Canada” stated John McKendry, CEO and President of Kye Pharmaceuticals. “Together, we are committed to delivering novel therapies and bridging clinically significant treatment gaps.”

“We are pleased to enter into the licensing agreement with Kye Pharmaceuticals for AGAMREE, marking a pivotal milestone in our strategic initiative to expand the product’s footprint in North America. This collaboration leverages our combined expertise, fortifies our alliance by licensing our second therapeutic rare disease product for Canada, and demonstrates our sustained commitment to patient care,” stated Richard J. Daly, CEO and President of Catalyst. “Building upon the U.S. approval of AGAMREE, we are committed to helping facilitate access to this novel alternative corticosteroid treatment to DMD patients and their healthcare providers in Canada. We look forward to working closely with our partner, who will spearhead the regulatory approval process for AGAMREE, with application submission to Health Canada anticipated by early 2025.”

Under the terms of the agreement, Kye will have the exclusive Canadian rights to commercialize AGAMREE® (vamorolone) oral suspension and will be responsible for funding all regulatory, marketing, and commercialization activities in Canada. Catalyst Pharmaceuticals, Inc., will be responsible for clinical and commercial supply and provide support to Kye Pharmaceuticals in its efforts to obtain regulatory approval for the product from Health Canada.

About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects males, with symptoms typically appearing in early childhood, around ages 3 to 5. DMD is caused by mutations in the gene that encodes dystrophin, a protein that plays a crucial role in maintaining the structure and function of muscle fibers. Without dystrophin, muscle cells become fragile and easily damaged, leading to progressive muscle degeneration. Symptoms of DMD usually begin with difficulty in walking, frequent falls, and muscle weakness, particularly in the legs and pelvis. As the disease progresses, individuals may experience difficulty standing, climbing stairs, and eventually, complete loss of mobility. Other complications can include respiratory and cardiac issues due to muscle weakness.

About AGAMREE® (vamorolone)
AGAMREE’s unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid designed to achieve dissociative properties while maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

About Kye Pharmaceuticals Inc.
Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye’s portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada. For more information, please visit www.kyepharma.com.

About Catalyst Pharmaceuticals, Inc.
With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare and difficult-to-treat diseases. Catalyst’s flagship U.S. commercial product is FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome (“LEMS”) for adults and for children ages six to seventeen. On July 31, 2020, Health Canada approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS. On July 18, 2023, Catalyst acquired an exclusive license for North America for AGAMREE® (vamorolone) oral suspension 40 mg/mL, a novel corticosteroid treatment for Duchenne Muscular Dystrophy. AGAMREE previously received FDA Orphan Drug and Fast Track designations and was approved by the FDA for commercialization in the U.S. on October 26, 2023. AGAMREE became commercially available by prescription in the U.S. on March 13, 2024.

For more information about Catalyst Pharmaceuticals, Inc., please visit the Company’s website at www.catalystpharma.com.

SOURCE Kye Pharmaceuticals Inc.