Drug Development

Karyopharm sets a course for the FDA with positive PhIIb multiple myeloma data

Investigators for Karyopharm Therapeutics $KPTI rolled out a positive set of data for their lead cancer drug selinexor bright and early Monday, touting a set of responses among patients who had run through a string of other treatments and mapping a clinical path that could point straight to the FDA’s door in 2018.

The drug was being tested among multiple myeloma patients who had proved resistant to a long slate of drugs, an indication of just how crowded MM has grown in recent years. Roughly one in 5 of the 78 evaluable patients demonstrated an objective response characterized as a partial response or a “very good” partial response.

The biotech says that bottom line from a single-arm study compares well with the historical data collected for Darzalex and isatuximab. And that was good enough to drive a quick spike in the share price, with the company’s stock shooting up 17% on the news.

The Newton, MA-based biotech now plans to add 120 refractory patients to this study, if it’s positive hustle the data to the FDA in search of an accelerated approval, with top-line data due in 2018. Kayopharm also plans to launch a Phase III combo study that will add Velcade and dexamethasone to their drug and test it among patients who had become resistant to three other drugs.

The biotech also said it had no unexpected safety issues to discuss today. Almost exactly a year ago investigators spooked investors with word that the drug was linked with serious cases of sepsis, forcing a change in dosing.

Lead investigator Keith Stewart had this to say:

“The STORM data are compelling because they demonstrate that oral selinexor achieves a 20.8% response rate in the quad-refractory group, similar to recently reported intravenous anti-CD38 therapy results in the same patient population. Selinexor also achieves an equally notable 20.0% response rate in the penta-refractory group, with the significant advantage of oral administration.  We are currently unaware of any other therapy, oral or intravenous, reporting such activity in these difficult-to-treat patients who have exhausted all available therapies.”

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