Pfizer and Sangamo re-open PhIII of hemophilia A gene therapy; AEON headed to PhIII

A year after voluntarily pausing the Phase III study of their hemophilia A gene therapy program, and four months after FDA lifted the clinical hold, Pfizer and Sangamo Therapeutics say they will resume dosing next month.

The move pushes back the original data readout by two years. At the time of the first patient dosing in October 2020, a Pfizer spokesperson had told Endpoints News that a pivotal readout would come in early 2022. Following the hurdles caused by the clinical hold, Pfizer said Friday morning the new anticipated timeline is the first half of 2024.

With the AFFINE study now recruiting patients again, Pfizer and Sangamo plan to have all sites active before year’s end. Pfizer had paused the study after patients experienced abnormally high levels of an essential blood clotting factor known as factor VIII. The trial looks to enroll more than 60 men aged 18-64 years with moderate to severe forms of the hereditary bleeding disorder.

Sangamo and Pfizer are testing if a single injection of the gene therapy, dubbed giroctocogene fitelparvovec, improves the annualized bleeding rate through 15 months of treatment, comparing that metric to prior factor VIII prophylaxis replacement therapy.

The duo follows behind BioMarin, which received the first gene therapy conditional nod for hemophilia A last month in the EU. The company plans to resubmit its application to the FDA by the end of this month.

While not a gene therapy, Sanofi’s factor VIII replacement drug, efanesoctocog alfa, gained priority review from the FDA last month.

AEON’s neck muscle drug headed to Phase III 

With statistical significance in a Phase II now in hand, AEON Biopharma’s ABP-450 injection is likely to enter a Phase III next year for cervical dystonia in adults.

Marc Forth

The neurologic condition causes the head to involuntarily twist and tilt, affecting neck muscle contraction. Two of the three doses, which includes the botulinum toxin, were shown to have statistical significance at week four in reducing signs and symptoms of CD based on a scale known as the Toronto Western Spasmodic Torticollis Rating. The highest dose “narrowly missed statistical significance,” the company said Friday morning. The p-values came in at 0.007 for 150 units of the injection, 0.0406 for 250 units and 0.0864 for 350 units.

The primary safety goal was met in the placebo-controlled 57-patient study, the company said. Across all three doses, the adverse event rates were either similar to or lower than other botulinum toxin products used for CD. Adverse events related to the treatment were mild or moderate, according to AEON.

The Irvine, CA biotech is also testing the injection in patients with migraines. Phase II data for the migraine prevention study will come out next year, president and CEO Marc Forth said in a statement.

The private company picked up another $30 million from existing investors last month, via a convertible note, to fuel the late-stage pipeline.

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