FDA puts a clamp on Curis cancer drug program after patient dies of liver failure

A year after Lexington, MA-based Curis ($CRIS) agreed to pay Genentech $9.5 million upfront to license the early-stage cancer therapy recoded as CUDC-427 and borrowed $30 million to launch its own study, the FDA has clamped a partial clinical hold on the program following the death of one patient taking the drug. Investigators will now stop recruiting patients and scramble to come up with additional data and analysis on patients along with a proposed protocol amendment.

Curis's stock dropped 17% this morning on the news.

A few months ago researchers launched a Phase I study of the drug, aiming to find a good dose to take into a mid-stage trial. Then the liver enzymes in one patient with extensively metastasized breast cancer gyrated upward, causing the liver to fail and killing the patient. While biotech execs says they've seen similar liver toxicity triggered in other patients, this time the enzymes failed to return to normal after investigators stopped treatment and she died a month later.

No other patients are currently taking the drug--which spurred seriously elevated liver enzymes at least twice before while Genentech was studying the drug--but Curis says it plans to respond in a timely fashion with an eye to getting back to work. That may be easier said than done, though.

The oral drug, GDC-0917 while it was at Genentech, is designed to block inhibitors of apoptosis proteins, which help protect cancer cells from destruction. Genentech, one of the premier players in cancer drug research, had tested the drug in 42 patients over a 21-day cycle: two weeks of therapy followed by a week of rest. That trial was also aimed at finding a maximum tolerated dose and an ideal dose for Phase II. And Curis was planning to work on the drug as a single agent as well as a combo player with other therapeutics.

"Based on available data from treated patients, we believe that CUDC-427 could play an important role in oncology treatment, either as monotherapy or in combination with other anticancer agents," noted Curis President Ali Fattaey. "Accordingly, we will work diligently to address the FDA's request in an effort to resolve the partial clinical hold."

Curis picked up a $6 million milestone from Roche ($RHHBY) last summer after the European Commission approved Erivedge (vismodegib), along with a trickle of royalty revenues due under their licensing agreement. CUDC-427 is one of three early stage cancer drugs in Curis's pipeline.

- here's the press release

Suggested Articles

Solid Bio fixed the manufacturing issue that led to a partial clinical hold on its DMD trial, but a full hold remains.

Commissioner Scott Gottlieb has laid out the reasons why he thinks the FDA needs a double-digit budget bump.

GW's cannabis-based epilepsy drug scored a favorable review from FDA staff ahead of an expert panel meeting.