June must be the month for cardiovascular pipeline news with surprisingly positive upside.
Most recently, all-but-forgotten CETP inhibitor anacetrapib hit its primary endpoint of reducing major coronary events, while earlier in the month the monoclonal antibody ACZ885 (already marketed as Ilaris for various autoimmune disorders) met its endpoint of reducing major cardiovascular events like heart attack and stroke.
Although in each case, the results came in Phase III CV outcomes studies, questions remain on both agents.
Merck said pipeline drug anacetrapib reduced the risk of heart problems and stroke compared to placebo in at-risk patients who were already taking atorvastatin. The news was particularly surprising as most analysts and investors had written off the CETP class of cholesterol-lowering drugs after three high-profile failures from Pfizer, Roche, and most recently Eli Lilly.
CETP inhibitors work by raising HDL, or “good” cholesterol, and reducing LDL, or “bad” cholesterol. All 30,000 patients in the Phase-III trial received a common statin to lower LDL levels and then either received anacetrapib or a placebo. Merck will provide the full results of the trial at the European Society of Cardiology Meeting in August.
Still, one analyst expressed caution and drew attention to the careful wording in Merck’s announcement. Credit Suisse analyst Vamil Divan wrote in an investor note Tuesday, “the company also mentions they would like to discuss the full results with external experts before deciding on whether to file the drug for approval.”
Divan noted that one of the concerns around anacetrapib has been its long-term accumulation in body fat, writing that the drug is detectable in some patients “as much as four years after the last treatment dose.” That issue, he said, and its effect on an FDA filing is likely what Merck will discuss with outside experts.
Evercore ISI analyst Umer Raffat posited Tuesday that if approved Merck could find a niche in patients who need additional cholesterol-lowering beyond what statins can provide and yet who also don’t need the dramatic lipid lowering effects of PCSK9 inhibitors, like Amgen’s Repatha.
“There is a need for control beyond statins,” he wrote, “if you need modestly more benefit, you could consider [Merck’s] Vytorin and now anacetrapib. If you need much more LDL benefit, you could consider PCSK9s.”
For Novartis, new data for ACZ885 was heralded “as very much unexpected,” from Sanford Bernstein analyst Tim Anderson in an investor note. The Swiss drugmaker revealed last week that its Phase-III trial, dubbed CANTOS, hits its primary goal of reducing the risk of heart problems by targeting inflammation.
If filed and approved, the drug’s profile makes it a likely candidate for significant adoption for two reasons: it has a unique mechanism of action and it’s already been on the market and tested in the real-world since 2009 as juvenile arthritis drug, Ilaris.
Owing to that profile, Anderson wrote that it could be added to the current standard of care in patients following a heart attack, and would “not be threatened by generic alternatives in the same or similar drug classes.” He noted that Novartis had previously estimated the patient population for the drug would be roughly 4 million patients. That population with a price point of $15,000 per year, Anderson posits, would represent a market opportunity of $60 billion, “an almost unfathomable number,” he pointed out, adding that the “the bottom line is that this could end up becoming a multi-billion dollar product.”