The Medicines Company ($MDCO) will have to continue on with a study for its investigational heart drug after an independent body said early data were not strong enough to stop the test early.
The Milano-Pilot proof-of-concept study was looking at its new anticholesterol candidate MDCO-216 to help reduce atherosclerotic plaque burden and related CV events in 40 patients with coronary artery disease.
As preplanned, the New Jersey biotech allowed an Independent Data Monitoring Committee to look at its data and see whether it hit key efficacy targets, which would allow the trial to stop early. But it said this week that the data were not strong enough to stop, so the test must go on.
In a statement, the company said: “A planned, interim analysis of the first 40 randomized patients completing treatment in the Milano-Pilot study of MDCO-216 has been reviewed by the Independent Data Monitoring Committee (IDM Committee). Based on the protocol and pre-defined criteria in its Charter, the IDM Committee has reported the 40-patient data to the Company in summary form and recommended that the Milano-Pilot study continue. The pre-defined, upper statistical boundary for efficacy, which permits early termination of the study by the company, was not met and, given the limited number of patients included in the first interim analysis, the summary data received by the company from the IDM Committee are, at this time, inconclusive.”
Though far from terminal, the fact that it didn’t meet its efficacy criteria to stop early has spooked some investors, with shares in the $2.86 billion market cap biotech trading down by more than 6% after hours when the news broke.
Clive Meanwell, CEO of The Medicines Company, said: “We had hoped that the 40-patient data from the Milano-Pilot study would meet the pre-defined efficacy threshold for early termination, but they did not and, so far, the data are inconclusive. The study is continuing and we look forward to receiving full access to the final data.”
The Medicines Company said it has now completed enrollment of the planned 120 patients in the test and expects that results from Milano-Pilot will be presented by its principal investigator, eminent cardiologist Dr. Steven Nissen of the Cleveland Clinic, in mid-November.
MDCO-216 mimics pre-beta HDL and induces cholesterol efflux, which is the first step in the reverse cholesterol transport, a process of removal of deposited cholesterol from vessel walls, and therefore has a potential to reduce plaque burden in patients with coronary artery disease.
The company added that the Phase II trial for its other candidate, PCSK9si, is still ongoing and completed enrollment ahead of schedule. The biotech hopes this drug will beat out other PCSK9 rivals from Sanofi ($SNY) and Regeneron ($REGN), as well as Amgen ($AMGN).
The Medicines Company has picked up a number of FDA approvals over the past few years, including for the antibiotic Orbactiv (oritavancin) in 2014, Raplixa (fibrin sealant [human]), a spray-on sealant designed to stem bleeding during surgery; Ionsys (fentanyl iontophoretic transdermal system), a once-abandoned drug-device combo that delivers fentanyl under the skin to treat postoperative pain; and Kengreal (cangrelor), a long-delayed blood thinner used after surgery.