- The Medicines Company on Tuesday eagerly highlighted positive topline data supporting the efficacy of its experimental PCSK9 synthesis inhibitor, announcing an interim analysis of a Phase 2 study showed "significant and durable" reductions in LDL-C, or "bad" cholesterol," through 90 days.
- While specific numbers were not disclosed, company CEO Clive Meanwell said the data confirmed the drug's "highly competitive profile." Detailed results will be presented at the American Heart Association (AHA) meeting in November.
- Amgen and the Sanofi/Regeneron team are already on the market with their respective PCSK9 monoclonal antibodies Repatha and Praluent. Unlike those treatments, however, The Medicines Company's drug acts by inhibiting PCSK9 synthesis in the liver rather than binding to the PCSK9 protein in the blood.
The initial topline results released by The Medicines Company come from all 501 patients in the Phase 2 study through 90 days of follow up.
In addition to demonstrating significant LDL-C reductions, treatment with PCSK9si was well-tolerated. Patients did not show drug-related elevation of liver enzymes, neuropathy or change in renal function.
Despite the sparse details, analysts from Jefferies and Evercore ISI were encouraged by the seemingly strong efficacy and safety profile.
Company executives also touted the potential for more convenient dosing.
"These results validate a triannual, and potentially biannual, low volume subcutaneous dose regimen, which we believe represents a compelling, highly-differentiated approach for treating patients with hypercholesterolemia," said David Kallend, global medical director at The Medicines Company.
Amgen's Repatha is administered either every two weeks or as a single monthly injection, while Sanofi and Regeneron's Praluent is taken once every two weeks.
The detailed data, which will be presented at the upcoming AHA meeting, will include day 180 follow-up on the first 200 patients, which should give further clarity on dosing options. The company also indicated full day 180 follow-up on all 501 patients would be disclosed before year end.
The Medicines Company stock rose by around 4% in early Tuesday trading on the news. While the results augur well for the drug, both Repatha and Praluent have struggled to gain traction on the market as payers have balked at high prices.