Gene Therapy AMT-130 in Huntington Disease, NDA Submitted for Troriluzole in Spinocerebellar Ataxia, FDA Clears Alzheimer Assays

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Neurology News Network for the week ending July 1, 2023. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

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

Recently, uniQure, the drug makers of AMT-130, a gene therapy in development for Huntington disease (HD) announced positive findings from a phase 1/2 trial, with patients showing preserved function and reductions in relevant biomarkers over a 24-month period. Based on these data, the company anticipates initiating a third cohort to evaluate the impact of low and high dose AMT-130 when used in combination with perioperative immunosuppression. The company also noted that it expects to present new clinical data from the phase 1/2 studies of AMT-130, including addition follow-up data from the treated patients in the US trial and the 12-month follow-up data from the low-dose patients in the European trial, to come in the fourth quarter of 2023. Patient enrollment in the high-dose cohort of the European clinical trial is expected to be complete by the third quarter of this year after it was briefly paused in November 2022.

According to a recent announcement, Biohaven Pharmaceuticals submitted a new drug application (NDA) for its investigational agent troriluzole, a third-generation prodrug and new chemical entity that modulates glutamate, for the treatment of spinocerebellar ataxia type 3 (SCA3). The NDA was supported by data from the phase 3 Study BHV4157-206 with confirmatory evidence from the 3-year, long-term open-label extension (OLE) phase of that study, and an additional study (BHV4157-201) using a matching adjusted indirect comparison analysis. In study BHV4157-206, the agent failed to reach statistical significance on its primary end point; however, there were numerical treatment benefits observed in a subgroup of those with SCA3, the most common form of SCA. In total, SCA3 represented 41% of study participants.

According to an announcement, the FDA has granted 510(k) clearance to Roche for its Alzheimer disease (AD) cerebrospinal fluid Elecsys assays—including beta-amyloid1-42 CSF II (Abeta42) and total tau (tTau) assay. Used for individuals ages 55 and older being evaluated for AD and other causes of cognitive impairment, the new Elecsys tTau/Abeta42 ratio will be available in the final quarter of this year. As changes in beta-amyloid and total-tau levels occur at early stages of the disease, the assays are designed to detect AD pathology in earlier stages of the disease. The ratio of these biomarkers is consistent with a negative amyloid PET scan if the result is less than or equal to the cutoff, and with a positive amyloid PET scan if the result is above the ratio cutoff.

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