The drug: MK-3475
The disease: Cancer
The developer: Merck
Peak sales potential: Maybe $500 million for melanoma, with the blockbuster money coming if a whole lineup of cancers is added. Bernstein estimates $3 billion by 2020.
The scoop: Merck ($MRK) needs this one badly, and to his credit new R&D chief Roger Perlmutter understands that perfectly. Under growing pressure from Wall Street, which has come to expect nothing but disappointment, delay and failure from Merck over the past few years, the pharma giant is circling its best research wagons around this PD-1 immunotherapy drug.
Believing it had something special, Merck R&D leader Alise Reicin was put in charge of a very unusual Phase I study which enrolled 1000 patients. Phase I studies typically number enrollees in the dozens, at most. It went straight for proof of concept, turning heads at ASCO last summer with data demonstrating an interim overall response rate at 38%, according to investigators, who reported interim data on 135 patients with advanced melanoma. The highest response rate was an impressive 52% in the arm receiving 10 mg/kg every two weeks, the highest dose in the study. Ten percent of the patients in that arm achieved a complete response, with response duration ranging from 28 days to 8 months. Four out of 5 patients who responded stayed on treatment. And after a median follow-up time of 11 months, the median rate of response has not yet been reached in the study.
Armed with the solid backing of Perlmutter and a breakthrough drug designation from the FDA, the R&D team is racing ahead on this one, with a clear understanding that mid-stage data could serve as a potential foundation for a marketing application. Phase Ib studies are giving way to Phase II/III studies in melanoma and non-small cell lung cancer.
Jewel in the crown. Top program. Breakthrough drug. Whatever you want to call it, MK-3475 (which they briefly called lambrolizumab) has all the earmarks of a big winner. Barring a blowup in the clinic, this program has the potential to prove that the company still knows how to do serious drug research effectively.
The stakes are incredibly high, with no room for failure or even temporary delays.
Editor's Corner: ASCO confab highlights immunotherapies, 'breakthrough' drugs and small victories
Cancer immunotherapies turn a corner in race to megablockbuster market
Perlmutter prunes R&D at Merck amid calls for change
Merck's 'breakthrough' PD-1 cancer drug in showdown with Bristol-Myers combo