Corbus Pharmaceuticals has bagged $25 million from the Cystic Fibrosis Foundation, which will carry its cystic fibrosis drug, lenabasum, through a 415-patient phase 2b trial.
The development award comes in a new venture philanthropy model for the Foundation—in addition to receiving a royalty stream, “it is the first time the CF Foundation is going to be an equity partner in a company developing a drug for CF […] and that is very innovative for them,” said Corbus CEO Yuval Cohen.
The trial will begin dosing shortly and is expected to wrap up in 2020, Cohen said. It will be the first cystic fibrosis study that uses pulmonary exacerbations as its primary endpoint, rather than alongside the co-primary endpoint of FEV1, or forced expiratory volume.
Unlike other CF meds, lenabasum—formerly called anabasum—doesn’t aim to hydrate patients’ mucus or target the genetic mutation behind the disease, which are captured with FEV1, Cohen said. Instead, it targets the underlying inflammation of the disease.
“The outcome of that for these patients is they have these very serious, very damaging and increasingly, more frequent pulmonary exacerbations as they grow older,” he said. These may lead to irreversible lung damage and death.
And what sets Corbus’ drug aside, Cohen said, is that it targets all CF patients, no matter which CFTR mutation they have, what pathogen they may have, or whether they are already on a stable background medication, such as Kalydeco or Orkambi.
Lenabasum hit its primary objective in an 85-patient phase 2 study last year, showing an “acceptable safety and tolerability profile at all doses,” as well as a dose-dependent reduction in a number of acute pulmonary exacerbations.
The new trial will enroll about 415 patients with CF who are at least 12 years of age and at risk for pulmonary exacerbations. The double-blinded, randomized, placebo-controlled study will take place at about 100 sites in North America, Europe, Israel and Australia.
“With about 415 patients treated for 6-months, the next Phase 2 study is large enough and long enough to potentially provide statistically significant data regarding benefit of lenabasum in pulmonary exacerbations in CF,” said Barbara White, M.D., Corbus chief medical officer, in a statement.