Neuraltus Pharma eyes PhIII after missing endpoints in trial for ALS drug
Despite falling short of goals in a Phase II trial, Neuraltus Pharmaceuticals saw enough evidence of efficacy in Lou Gehrig's disease patients on its experimental drug to push forward plans for a late-state study. Its decision to start the Phase III program next year underscores the critical need for new therapies against the muscle-disabling disorder.
The Palo Alto, CA-based biotech says that it didn't hit statistical significance in the pre-defined endpoints of the mid-stage study of 136 patients with Lou Gehrig's or amyotrophic lateral sclerosis (ALS). However, a post hoc analysis of the trial showed that 27% of patients on the higher of two doses of the company's candidate, NP001, had no disease progression for 6 months. When the company analyzed the data from the study with historic placebo responses, the efficacy in patients on that dose in the trial reached statistic significance.
"ALS is a rare neurodegenerative disease that typically leads to respiratory failure and death within five years of diagnosis. The results from this study with NP001 are most encouraging, as halting or slowing the rate of disease progression, in a subset of patients, as this study suggests, would translate into a clear clinical benefit for these patients," Dr. Robert Miller, the principal investigator of the study from California Pacific Medical Center, said in the company's release.
In patients with ALS, even therapies with unproven or modest benefits are in demand. Earlier this year, in fact, do-it-yourselfers rallied to pursue further study of a version of NP001 even as doctors disapproved of the maneuver to sidestep sanctioned development. Short on existing options, patients are faced with a desperate search for new therapies. Biogen Idec ($BIIB) has an ALS drug in late-stage development, providing one potential new option for patients with the disease.
Still, Neuraltus wants to roll the dice that NP001 will be a winner in Phase III based on a mixed bag of data. Even when presented with an opportunity to address a major gap in treatment, it's a risky proposition to enter late-stage development on the strength of a Phase II study with some failures. And the company, which aims to begin the Phase III program in the second half of 2013, acknowledges that post hoc analyses can offer biased results.