Novartis offloads a late-stage PI3Kẟ rare disease program in a discount deal
Looking to build on their franchise HAE drug Ruconest, Dutch biotech Pharming has snapped up a late-stage rare disease drug from Novartis for a modest upfront of only $20 million.
The pharma giant is selling global rights to CDZ173, a small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor in development for Activated Phosphoinositide 3-kinase Delta Syndrome —or APDS. And Novartis will complete the Phase II/III registration study as well as an ongoing open-label expansion study for Pharming.
In this disease, a mutation in the PIK3CD gene spurs over-activation of PI3Kẟ, leading to a dysfunctional immune system, lesions as well as cancer. GSK has one in Phase II for APDS, dubbed GSK2269557, though it recently culled a COPD trial using that drug as the company sliced back on the respiratory pipeline.
Pharming sees the drug as a nice addition to their HAE therapy, approved 5 years ago, as APDS is generally handled by the same set of physicians, easing commercialization work.
It’s a relatively small amount to pay for a late-stage drug, indicating that the commercial upside here may not be huge. PI3Kẟ inhibition certainly isn’t new, with Gilead out with Zydelig and others like duvelisib joining the mix for oncology, with limited commercial success. Novartis was unusual in advancing the drug for non-cancer uses.
Social image: Novartis, AP Images