- The Medicines Company said Monday the first in a trio of Phase 3 studies testing inclisiran succeeded, a result that reinforces the company's plans to submit the cholesterol drug for regulatory approval by year's end.
- Specific data results won't be released until Sept. 2 at the European Society of Cardiology's annual meeting. But the Parsippany, New Jersey-based biotech described the results as showing efficacy consistent with prior studies of the PCSK9 drug and a safety profile "at least as favorable" as a Phase 2 study and its open-label expansion.
- Inclisiran is an oligonucleotide, designed to block the expression of genes that encode for PCSK9, a protein that checks the liver's ability to absorb LDL cholesterol from the blood. If approved, the drug would compete with two other drugs that target PCSK9: Amgen's Repatha and Sanofi and Regeneron's Praluent.
Statins are still a fundamental component of cholesterol management, but a slate of new drugs that complement statins have shown the ability to further lower LDL cholesterol.
In Medicines Co.'s study, dubbed ORION-11, roughly 1,600 patients were randomized to receive either inclisiran or placebo. Trial participants had to have atherosclerotic cardiovascular disease or equivalent risk with high cholesterol levels, despite maximum statin therapy.
Evercore ISI analyst Umer Raffat called ORION-11's topline success a "major milestone," saying inclisiran could eventually be seen as the first "vaccine" in treating high cholesterol. The drug is taken twice a year.
A positive outcome also addresses lingering safety questions for inclisiran, Raffat said.
How inclisiran stacks up versus its potential PCSK9 rivals will depend greatly on the specific results from ORION-11 as well as from Medicines Co.'s two other Phase 3 trials, called ORION-9 and ORION-10.
Among a similar patient population in previous studies, Repatha (evolocumab) lowered LDL by an average of 57% more than placebo after 72 weeks, while Praluent (alirocumab) led to a 55% drop after 48 weeks.
Even with that clinical benefit, Repatha and Praluent have largely disappointed commercially in their first few years on the market. Last October, the makers of both drugs slashed list prices by more than half in competing efforts to win placement on insurer formularies.
Medicines Co. in-licensed inclisiran from Alnylam Pharmaceuticals in 2013, paying $25 million upfront and lining up $180 million in potential milestones.
Earlier this year, Alnylam said it had earned $30 million of those milestones, with $25 million more coming if inclisiran is approved in the U.S. The Cambridge, Massachusetts-based biotech will also receive royalties on worldwide sales in the low- to high-teens.
Medicines Co. will host a Sept. 2 investor call at 8 a.m. to discuss the study results. The biotech plans to release results from ORION-9 and ORION-10 later this quarter.
The company anticipates those results will set up U.S. and European regulatory submissions in the fourth quarter of 2019 and the first quarter of 2020, respectively.
Shares in Medicines Co. rose by about 10% by noon Monday.