Catalyst Pharmaceuticals Announces Publication of Santhera Pharmaceutical’s VISION-DMD Vamorolone (AGAMREE) Study Results in the Peer-Reviewed Journal Neurology

Santhera's Study Reports the Results of the 48-Week Treatment with Vamorolone in Patients with Duchenne Muscular Dystrophy (DMD) in the VISION-DMD Study, Supporting the Long-Term Efficacy and Safety Profile of Vamorolone

CORAL GABLES, Fla., Feb. 21, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst” or “Company”) (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 that the peer-reviewed journal Neurology has published Santhera Pharmaceutical's ("Santhera") study titled “Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy” presenting the findings from Santhera's VISION-DMD study conducted by Santhera [1]. Catalyst holds the exclusive rights to commercialize AGAMREE (vamorolone) in North America.

Santhera reports that the VISION-DMD study was a randomized, double-blind, placebo-controlled, and prednisone-controlled clinical trial of 2 doses of vamorolone in participants with Duchenne muscular dystrophy (“DMD”) in the ages four years to younger than seven years at baseline that was conducted at 33 sites in 11 countries from 2018 to 2021. Based on the published study, Santhera has reported that the results of the VISION-DMD study support the long-term efficacy and safety profile of vamorolone and conclude that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [2]. As cited in Santhera's recent press release, the Neurology publication of their study states:

“Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD)[2]. This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).

A total of 121 participants with DMD were randomized. Vamorolone at a dose of 6 mg/kg/d showed maintenance of improvement for all motor outcomes to week 48 (e.g., for primary outcome, time to stand from supine [TTSTAND] velocity, week 24 least squares mean [LSM] [SE] 0.052 [0.0130] rises/s vs week 48 LSM [SE] 0.0446 [0.0138]). After 48 weeks, vamorolone at a dose of 2 mg/kg/d showed similar improvements as 6 mg/kg/d for North Star Ambulatory Assessment (NSAA) (vamorolone 6 mg/kg/d– vamorolone 2 mg/kg/d LSM [SE] 0.49 [1.14]; 95% CI −1.80 to 2.78, p = 0.67), but less improvement for other motor outcomes. The placebo to vamorolone 6 mg/kg/d group showed rapid improvements after 20 weeks of treatment approaching benefit seen with 48-week 6 mg/kg/d of vamorolone treatment for TTSTAND, time to run/walk 10 m, and NSAA. There was significant improvement in linear growth after crossover in the prednisone to vamorolone 6 mg/kg/d group, and rapid reversal of prednisone-induced decline in bone turnover biomarkers in both crossover groups. There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups.