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
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
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
About
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
Forward-Looking Statements
This press release contains forward-looking statements, as that term is defined in the Private Securities Litigation Reform Act of 1995. These include statements regarding Catalyst's expectations, beliefs, plans or objectives regarding the intended use of net proceeds therefrom. Forward-looking statements involve known and unknown risks and uncertainties, which may cause Catalyst's actual results in future periods to differ materially from forecasted results. A number of factors, including (i) whether DMD patient will agree to enroll in the registry, and (ii) those factors described in Catalyst's Annual Report on Form 10-K for the fiscal year 2022 and its other filings with the
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