Catalyst Pharmaceuticals To Present At MDA Clinical & Scientific Conference Details Of Registry For Study Of Long-Term Safety And Quality Of Life In Duchenne Muscular Dystrophy Patients Treated With AGAMREE

Author: Benzinga Newsdesk | February 27, 2024 09:17am

Catalyst Pharmaceuticals, Inc. ("Catalyst") (NASDAQ:CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases, today announced an upcoming poster about the establishment of a registry to study long-term safety and quality of life (QoL) in patients with Duchenne muscular dystrophy (DMD) who are treated with AGAMREE® (vamorolone) at the upcoming 2024 MDA Clinical & Scientific Conference taking place March 3-6 in Orlando.

 

The poster will detail the establishment of a multicenter, observational, prospective, longitudinal registry at approximately 25 U.S. sites. Investigators will assess the progress of 250 male patients who are at least 2 years old at week 12 and then every 12 months for up to five years. The first interim analysis is expected in May 2025.

Poster Information:

Title: An Imminent Registry to Study Long-term Safety and Quality of Life in Patients with Duchenne Muscular Dystrophy treated with Vamorolone (AGAMREE®)

Poster Number: M174

"This registry will provide real-world data regarding long-term use of the novel corticosteroid, generating additional data to the current clinical results and underscoring the potential of AGAMREE® to reshape the DMD treatment paradigm with a focus on improving the quality of life for patients," said Gary Ingenito, MD, Ph.D., Chief Medical and Regulatory Officer of Catalyst. "We encourage physicians treating patients with DMD to consider enrolling eligible patients in this registry."

Learn more about #MDAConference here: https://www.mdaconference.org/

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 with dissociative properties in maintaining efficacy that we hope 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 vamorolone 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, vamorolone 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, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

AGAMREE was granted U.S. FDA approval on October 26, 2023, and was granted Orphan Drug and Rare Pediatric Disease designation status for DMD in the U.S., making it eligible for seven years of orphan drug exclusivity upon approval. AGAMREE also has issued and pending patents that could provide protection until 2040. In Europe, it has received Promising Innovative Medicine (PIM) status from the UK MHRA for DMD.

References:

[1] Dang UJ et al. (2024) Neurology 024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.

[2] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.

[3] Liu X et al. (2020). Proc Natl Acad Sci USA 117:24285-24293

[4] Heier CR et al (2019). Life Science Alliance DOI: 10.26508

[5] Ward et al., WMS 2022, FP.27 - Poster 71. Link.

[6] Hasham et al., MDA 2022 Poster presentation. Link.

[7] Applicable drug labeling: Summary of Product Characteristics (SmPC). English.

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