Although the drug didn’t hit its pre-defined goals in a mid-stage clinical trial, the company said Tuesday, an analysis of the results indicated that the drug stopped the disease, also known as amyotrophic lateral sclerosis, from progressing.
The news is significant for patients with the rare and fatal muscle-crippling disease because there is only one drug approved for ALS and it extends patients’ lives only by weeks.
It also is a victory for the small, privately held Palo Alto company, which is developing the drug in a disease that is poorly understood and where much-larger Biogen Idec (NASDAQ: BIIB) also has an ALS program in Phase III. It also could set up the company to be sold to a larger biotech or pharmaceutical company or strike a collaboration deal.
Yet, perhaps most of all, the decision to move forward with the drug, called NP-001, demonstrates the power of patients to fight for a drug they believe will alter the course of a disease that can kill patients within three to five years of diagnosis. A core group of ALS patients and their caregivers fought to fill Neuraltus’ Phase II trial.