ALS treatment tazbentetol gets FDA fast track status
Designation follows positive data from clinical trial
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- Tazbentetol, an experimental oral therapy for ALS, received FDA fast-track designation.
- The treatment promotes synapse formation to restore nerve cell communication.
- A Phase 1/2a trial showed tazbentetol was safe and slowed ALS progression in 82% of patients.
The U.S. Food and Drug Administration (FDA) has granted fast-track designation to tazbentetol, an experimental oral therapy being developed by Spinogenix for amyotrophic lateral sclerosis (ALS).
Fast-track status is intended to speed the development and review of treatments for serious conditions with unmet medical needs. It will allow Spinogenix to have more frequent interactions with the FDA during development and may make tazbentetol eligible for accelerated approval and priority review if certain requirements are met.
Tazbentetol, formerly known as SPG302, holds orphan drug status for ALS in the U.S. and Europe. This designation provides incentives to encourage the development of therapies for rare diseases.
“Receiving Fast Track Designation is another significant milestone in our journey to develop a new therapeutic to combat ALS,” Stella Sarraf, PhD, Spinogenix’s founder and CEO, said in a company press release. “This designation will allow us to work more closely with the FDA in planning our next trial to help accelerate its development to address this critical unmet need.”
ALS is marked by the progressive loss of motor neurons, the specialized nerve cells that control voluntary movement. Exactly what causes it remains poorly understood, but the loss of synapses — the connections that allow nerve cells to send signals to one another — may play a role. Synapses are essential for brain processes such as cognition, movement, and sensation, but these structures start to deteriorate in the early stages of ALS.
Treatment targets synapses to aid nerve cell communication
Tazbentetol is designed to promote the formation of new synapses, with the goal of helping preserve or restore nerve cell communication. This may help slow disease progression and ease ALS symptoms.
The FDA’s move follows promising results from a Phase 1/2a trial (NCT05882695) in Australia.
The first two parts of the study involved healthy volunteers and showed an acceptable safety profile. The Phase 2a portion then tested a 300 mg dose of tazbentetol in 23 people with ALS. Participants received the experimental therapy or a placebo as a once-daily pill for 28 days, after which all received tazbentetol for about five additional months in an open-label extension.
The study met its main goal of demonstrating that tazbentetol had a favorable safety profile in people with ALS, with no treatment-related serious side effects reported over six months.
Some 82% of participants on tazbentetol had a stable or reduced rate of decline at the end of treatment, as assessed with the ALS Functional Rating Scale-Revised (ALSFRS-R).
Compared with matched historical controls from the PRO-ACT database, the rate of decline in the ALSFRS-R was 76% slower in people receiving the experimental therapy.
These effects were accompanied by improvements in ALS-associated patterns in electroencephalograms (EEGs), which measure the brain’s electrical activity using electrodes placed on the scalp.
Phase 2 clinical trials are also testing tazbentetol for Alzheimer’s disease and schizophrenia.
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