NoNO Inc announced results from the phase 3 ESCAPE-NA1 study of nerinetide for the treatment of acute ischemic stroke.

Nerinetide is an eicosapeptide that inhibits post-synaptic density protein 95 (PSD-95) signaling. It is believed to act as a neuroprotectant that promotes cell survival and disrupts toxic cell signals following damage to neurons.

The phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluated the efficacy and safety of nerinetide in 1105 patients with acute ischemic stroke who underwent endovascular thrombectomy and received alteplase when indicated (N=1105). Patients were randomized 1:1 to receive nerinetide 2.6mg/kg intravenously or placebo. 

The primary end point was the proportion of patients who achieved a favorable functional outcome 90 days after randomization, defined as a modified Rankin Scale score of 0-2, across all trial participants (those not receiving alteplase and those who received alteplase before nerinetide). Key secondary end points included functional independence, mortality rate and infarction volumes.

Results showed that the primary end point of the study did not reach statistical significance; 61.4% of patients (n=337/549) in the nerinetide treatment arm and 59.2% (n=329/556) in the placebo arm reached functional independence (adjusted risk ratio 1.04; 95% CI, 0.96-1.14; P =.335). 

In patients who did not receive alteplase, nerinetide provided a statistically significant improvement in functional independence vs placebo (59.4% [n=130/219] vs 49.8% [n=113/227]; P =.028), respectively. Additionally, nerinetide showed a placebo-adjusted reduction in mortality and infarction volumes of 22% (P =.048) and 40% (P =.034), respectively. The safety profile of nerinetide was comparable to placebo. 

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“Compared to placebo, almost 20% more patients who received nerinetide along with endovascular treatment, but did not receive alteplase, recovered from a devastating stroke – a difference between paralysis and walking out of the hospital,” said Michael Hill, MD, one of the ESCAPE-NA1 global coordinating investigators. “In the patients who received both drugs, the alteplase negated the benefits of the nerinetide.”

The Company is currently evaluating nerinetide for acute cerebral ischemia in the pivotal phase 3 FRONTIER trial.

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