EAP Program Authorized for ALS Agent SPG302, PTC518 Meets Primary End Point, Capricor Completes Mid-Cycle Review for Deramiocel

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Welcome to this special edition of Neurology News Network. I'm Marco Meglio.

According to a new announcement, the FDA has authorized Spinogenix’s expanded access program (EAP) for its investigational amyotrophic lateral sclerosis (ALS) agent SPG302 in the United States for patients living with the disease that meet the program’s eligibility criteria. The company also noted that it recently completed a phase 1/2 trial (NCT05882695) in Australia, and patients were offered continuation of the treatment in the open label extension trial. In the study, investigators randomly assigned healthy volunteers aged between 18 and 55 years to either SPG302 or a placebo. The first part of the trial featured only single ascending doses of SPG302 and the second tested multiple ascending doses of SPG302 for 5 days.

According to a new announcement from PTC Therapeutics, investigational PTC518 met its primary end point in the phase 2 PIVOT-HD study (NCT05358717), with results showing a statistically significant change in blood Huntingtin (HTT) protein levels over a 12-month period and promising 24-month data as well. Based on the totality of the evidence, PTC plans to work with the FDA on an accelerated approval path for the agent as a potential treatment for Huntington disease (HD). After 12 months of treatment with the small molecule splicing modifier, investigators observed a 23% reduction in blood HTT levels for the 5 mg dose level for both Stage 2 and 3 patients, as well as a 39% and 36% attenuation at the 10 mg dose level for Stage 2 and 3 patients, respectively. More notably, there was a dose-dependent trend of benefit among Stage 2 patients in terms of clinical scales like Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) subscale. This was not the same for Stage 3 patients, as results indicated the 5 mg group was more effective, suggesting that Stage 3 patients may have a different treatment effect than Stage 2.

According to a new announcement, Capricor Therapeutics has completed a mid-cycle review meeting with the FDA for its biologics license application (BLA) seeking full approval for deramiocel, an investigational cell therapy for patients with Duchenne muscular dystrophy (DMD) cardiomyopathy. During the meeting, the agency stated that no significant deficiencies have been identified by the review committee and that the package is on track for a PDUFA action date set for August 31, 2025. The company also noted that the agency confirmed its intent to hold an advisory committee meeting, although an official date has not yet been scheduled. The BLA submission is supported by Capricor’s cardiac data from its phase 2 HOPE-2 (NCT03406780) and HOPE-2 open label extension (OLE) trials, which used patient level data from an FDA-funded and published dataset on the natural history of DMD-cardiomyopathy, as well as data on potential biomarkers of disease progression. Capricor noted that efficacy from the ongoing phase 3 HOPE-3 study (NCT05126758) is not part of this BLA package submission.

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