A phase 2 trial of Reata Pharmaceuticals’ omaveloxolone in patients with Friedreich’s ataxia (FA) has met its primary endpoint. The positive result, which surprised analysts, tees Reata up to file for FDA approval of the activator of Nrf2 and expand development to other neurodegenerative diseases.
Analysts at Jefferies said most people assumed the pivotal study would fail, reflecting the mixed data generated earlier in development. However, omaveloxolone performed better in the second part of the phase 2 trial, recording a 2.4 placebo-adjusted improvement on a disease scale at 48 weeks. The result was strong enough for the study to hit its primary endpoint.
The data may also be strong enough to get omaveloxolone to market. Based on a conversation with a key opinion leader (KOL) earlier in the year, the Jefferies analysts think a two-point improvement may be enough to get Reata approved.
Omaveloxolone cleared that threshold in the phase 2 trial, and the placebo-adjusted improvement was still rising at the 48-week cutoff. That could be important for the prospects of omaveloxolone.
“A KOL we spoke w/ in May emphasized the importance of durability and how efficacy out to 48 wks would be meaningful to the FA community,” analysts at Jefferies wrote in a note to investors.
Reata now plans to file for approval in the U.S. and international markets, giving it a shot at making omaveloxolone the first therapy cleared for use in FA. Currently, people with the rare neuromuscular disorder are typically wheelchair dependent within 15 years of diagnosis and unlikely to survive past their mid-30s.
With an estimated 5,000 people suffering from FA in the U.S., Reata has chance to establish the drug as a commercially important product if it gets it to market with a label that supports its use.
However, the readout also features details to support a more skeptical reading of the situation. The primary endpoint data are based an 82-subject analysis population that stripped out 21 patients with a musculoskeletal foot deformity. Reata also recorded mixed results on secondary endpoints, linking the drug to statistically significant improvements in Patient Global Impression of Change in the full trial population but not the primary analysis population.