Plans in place for Phase 3 testing of ALS therapy NP001 next year
Neuvivo, FDA agree to use vital capacity as primary efficacy measure
Neuvivo and the U.S. Food and Drug Administration (FDA) have agreed on a regulatory pathway toward the potential approval of NP001 for amyotrophic lateral sclerosis (ALS), with Phase 3 clinical testing planned for 2026.
The details were agreed upon in a Type C meeting and the pivotal clinical trial will use a measure of lung function called vital capacity as the primary efficacy measure. While no other information is available yet, patients who want to be considered for it can email [email protected].
“I appreciate the collaborative meeting with the FDA. If approved, NP001 will be the first ALS treatment using vital capacity as the primary outcome of a double-blinded, placebo-controlled, multinational Phase III pivotal trial” Ari Azhir, PhD, the founder and CEO of Neuvivo, said in a company press release. “It is our goal to start the Phase 3 trial in 2026. I commit to keeping the ALS community updated on our progress.”
Muscle weakness and other symptoms of ALS occur due to the gradual loss of the nerve cells called motor neurons that are responsible for voluntary movement. It’s long been known that overactive immune cells such as macrophages may cause inflammation in the brain and spinal cord, and damage motor neurons.
Testing NP001 in ALS patients
NP001 is a small molecule that’s designed to rebalance the immune system by reprogramming inflammatory macrophages from a toxic to a noninflammatory state. This could help keep excessive inflammation under control, easing symptoms and slowing their progress.
In two clinical trials — a Phase 2a (NCT01281631) and a Phase 2b (NCT02794857) — NP001 was administered through monthly injections into a vein. Results of the Phase 2b trial showed it didn’t significantly slow functional decline over a placebo.
A combined analysis of those given NP001 for six months in the two studies showed the therapy did extended survival over a placebo by 4.8 months at the 2 mg/kg dose. The benefit was even more pronounced in patients ages 65 or younger. Also, NP001 extended survival by a median of 22 months in patients whose symptoms didn’t worsen during the six months of treatment, compared with those on the placebo. These patients also had significantly slower decline of lung function with preserved vital capacity.
“Post hoc analyses of our Phase 2 data indicate a strong correlation between NP001 treatment with preservation of vital capacity (greater than 15%) and overall survival (up to 25 months) compared to placebo,” said Michael McGrath, MD, PhD, co-founder and chief scientific officer of Neuvivo.
Joining the company’s board of directors to help advance NP001 is John Curnutte, MD, PhD. “I look forward to working with him and the rest of the board to bring this important therapeutic option to ALS patients,” Azhir said.
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