Nivalis fails phase 2 cystic fibrosis test, but vows to continue on

The biotech saw its shares hit hard last night on the news.

Nivalis saw more than half its value wiped out after hours last night in reaction to the news that its cystic fibrosis (CF) candidate did not hit its targets, leaving its future hanging in the balance.

In a brief update, the co said the 138 patient-strong midstage trial of its experimental CF med cavosonstat did not hit its primary endpoint in patients with the rare disease who had two copies of the F508del-CFTR mutation and were being treated with Vertex’ combo CF med Orkambi (ivacaftor/Kalydeco and lumacaftor).

This was because it could not show any benefit in absolute change in percent predicted FEV1 (forced expiratory volume in 1 second, a key outcome in CF) the trial’s primary endpoint, or in sweat chloride reduction at 12 weeks.

“While we are disappointed in the outcome of this trial, we plan to continue to investigate the therapeutic potential of cavosonstat and our S-nitrosoglutathione reductase (GSNOR) inhibitor portfolio to determine next steps,” said Jon Congleton, president and CEO of Nivalis.

The study was set up with two doses of cavosonstat (200mg and 400mg) given twice a day in specific CF patients who were homozygous for the F508del-CFTR mutation and being treated with Orkambi.

There were 138 patients in total treated with cavosonstat (200 mg) with Orkambi (n = 44), cavosonstat (400 mg) with Orkambi (n = 48) and a dummy treatment with Orkambi (n = 46) for 16 weeks.

The trial included a 4-week withdrawal and follow-up period once patients had completed 12-weeks of dosing.

Dave Rodman, chief medical officer and EVP of discovery at Nivalis, added: “Although we did not meet the primary endpoint, these data help inform the overall body of CF research, and we remain dedicated to completing our current clinical CF research program.”

The Boulder, Colorado-based biotech saw its shares obliterated on the news, down from $6.25 per share to just $2.94 after evening trading hours last night.

The biotech only got its $77 million IPO off last year based predominately from the early work on cavosonstat.