If at first you don't succeed in Phase III, try, try again. Only enroll lots more patients and improve the trial design. That was The Medicines Company's ($MDCO) strategy when it opted to pay for a third pivotal study of the blood thinner cangrelor.
Over the weekend the drug developer detailed the positive data investigators accumulated from the 11,000 patients enrolled to test the anticlotting drug against now-generic Plavix. The key score: 4.7% of the patients taking cangrelor either died, had a heart attack, experienced a repeat procedure or had a blood clot develop on their stent 48 hours after surgery (stent thrombosis). That compared favorably to the 5.9% rate for the Plavix group, representing a 22% reduction in risk. And specifically for the threat of stent thrombosis at 48 hours, a secondary endpoint, the reduction in risk was 38%.
That's significant because oral Plavix, which is provided to patients ahead of surgery, has long been considered a risky therapy. Because it's associated with heavy risks, often requiring close patient supervision, a full slate of developers has set to do better. In cangrelor's case, investigators believe that the fast-acting IV therapy, with a faster shut-down mechanism, would make it safer for patients. The biotech had earlier announced top-line results from the study.
Now that it finally has the pivotal data it needs, the San Francisco-based Medicines Company says it will file for regulatory approval in the U.S. and Europe in the next quarter.
The successful Phase III represented a bold, high-stakes gamble on the developer's part. After reportedly sinking $100 million into the first two failed studies, The Medicines Company believed that after tinkering with the trial design and expanding its reach with more patients, it was reasonable to believe that it could produce pay-dirt data at a point most developers would have given up.
"A lot of people thought we were taking a risk in time and money," co-lead investigator Deepak Bhatt told The Wall Street Journal. "We felt the first two trials provided a strong signal of benefit."
Jefferies' Biren Amin has estimated peak U.S. sales at about $370 million, while RBC's Adnan Butt estimated peak sales at $400 million. But there's a catch. Analysts also believe that this drug will have to be carefully priced to make it a preferred therapy. If the biotech company can wrestle a regulatory approval, market hurdles may prove even more daunting than the need for three late-stage studies.