APN-1607 Gets Fast Track Designation, ALS Agent BIIB105 Terminated, ION582 Safe in Angelman Syndrome

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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 granted fast track designation to Aprinoia’s APN-1607, a first-in-class radioactive diagnostic PET imaging tracer of tau aggregates, for patients with suspected progressive supranuclear palsy (PSP). APN-1607 is currently being tested in a global phase 3 trial to evaluate its performance as a biomarker for the early diagnosis of PSP. Currently, there are no FDA-approved diagnostic markers for PSP or any other rare tau-related disorders such as frontotemporal dementia. APN-1607, a radioactive fluorinated molecule developed to visualize and quantify 3R and 4R tau aggregates by PET imaging, has already previously received orphan drug designation and has been clinically utilized in over 3000 patients through investigator-initiated and sponsor trials. With the fast track designation, the belief is that this will help facilitate the development and expedite the review of product candidates like APN-1607 to help address unmet medical need.

Ionis and Biogen have announced topline results from the phase 1/2 ALSpire trial (NCT04494256) showing that BIIB105, an investigational agent in development for amyotrophic lateral sclerosis (ALS), did not result in the desired reduction in neurofilament light (NfL) and impact on clinical outcomes. Despite reaching target engagement in reducing expression of ataxin-2 (ATXN2) protein, the companies have decided to terminate development of the therapy, which acts as an antisense oligonucleotide (ASO). The trial, a randomized, placebo-controlled, dose-escalating study, included 99 participants with ALS who were randomly assigned patients 3:1 or 2:1 to either BIIB105 or placebo for 3 to 6 months. During the 6-month period, treatment with the agent resulted in statistically significant reductions in expression of ATXN2 and in cerebrospinal fluid (CSF); however, it failed to impact outcomes of function, breathing, and strength.

Newly announced topline data from the phase 1/2 HALOS trial (NCT05127226) showed that treatment with investigational ION582 (Ionis Pharmaceuticals) was safe and well tolerated in patients with Angelman syndrome, with robust improvements in outcomes of cognition, motor function, and communication. The company is expecting to report additional data from the study at the upcoming Angelman Syndrome Foundation meeting in July. Topline results were available for all patients at 4 months and 6 months, and were consistent with preliminary findings reported at the 2023 Foundation for Angelman Syndrome Therapeutics (FAST) meeting. All told, the therapy was considered well tolerated at all doses, with adverse events (AEs) that were consistent with patient medical histories, Angelman syndrome diagnosis, or related to intrathecal administration.

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