FDA Aligns on Pathway for CNM-Au8, AMT-130 Eyes Accelerated Approval, Tavapadon Meets Primary and Secondary End Points

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

A month after Clene’s recent meeting with the FDA and presentation of new data, the agency has provided written guidance to the company regarding a potential accelerated approval pathway for its investigational product CNM-Au8 in amyotrophic lateral sclerosis (ALS). Clene announced its intention to follow the FDA’s recommendations for CNM-Au8, expressing confidence in its ability to address the agency’s requests. Earlier this year, the company was initially advised that the data presented in its briefing package for the ALS agent was insufficient to support a new drug application (NDA) submission under the accelerated approval pathway. With the FDA’s recommendation, Clene will need to investigate whether additional data from the ongoing compassionate use expanded access programs (EAPs) can be leveraged to substantiate the effect of CNM-Au8 on neurofilament light chain (NfL) decline.

Following uniQure’s recent regenerative medicine advanced therapy (RMAT) Type B meeting with the FDA, the agency has reached agreement with the company on key elements of an accelerated approval pathway for its investigational gene therapy AMT-130 for patients living with Huntington disease (HD). The FDA concurred that data from the ongoing phase 1/2 clinical trials (NCT0543017; NCT04120493) of AMT-130, utilizing a natural history external control as a comparator, could serve as the primary basis for a biologics license application submission under the accelerated approval pathway. Thereby, eliminating the requirement for an additional presubmission study. Furthermore, the agency agreed that the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) could be utilized as an intermediate clinical end point, with reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid (CSF) to provide supportive evidence of therapeutic efficacy for the accelerated approval submission.

According to a new announcement, topline results from AbbVie’s pivotal phase 3 TEMPO-2 trial assessing investigational tavapadon as a flexible-dose monotherapy revealed that the treatment met its primary and secondary end point among patients with early Parkinson disease (PD). The company noted full results from the trial will be submitted for presentation at a future medical meeting and that it is on track to submit the new drug application to the FDA in 2025. Among 304 participants in TEMPO-2, those treated with tavapadon as a monotherapy experienced a statistically significant reduction from baseline compared with placebo in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III combined score at week 26 (placebo: -1.2; tavapadon: -10.3; P-value <.0001 versus placebo). In addition, results demonstrated statistically significant and clinically meaningful improvement in motor aspects of experiences of daily living (MDS-UPDRS Part II) in the tavapadon group compared with placebo at 26 weeks.

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