With an eye to keeping its lead on Amgen ($AMGN), Sanofi ($SNY) and Regeneron ($REGN) today noted that their rival PCSK9 inhibitor triggered a significant drop in LDL levels among patients with a high level of the "bad" kind of cholesterol. Responding to the promising Phase II data, Sanofi R&D chief Elias Zerhouni vowed to pivot into a late-stage program in the second quarter.
The key numbers for Sanofi were recorded in two groups among the 183 patients recruited for the study. In one group patients taking SAR236553/REGN727 in a subcutaneous injection twice a month registered a 72% mean drop in LDL compared to a 5% drop in the placebo arm. A separate cohort taking the twice-monthly treatment along with 80 mg of Lipitor saw a 73% reduction compared to an 18% drop for the Lipitor-only arm.
"I've not seen anything like this in all my days of research," says James McKenney, CEO of National Clinical Research and the lead investigator, who has been studying drugs for three decades. "This is so impressive."
The mid-stage data reflected an improvement over early-phase results, adds McKenney. In an interview with FierceBiotech he added that the longer you give the drug to a patient, the more efficacious it becomes. And one of the central objectives of this study was to zero in on the best dosage and frequency of treatment.
"No one else has finished the Phase II," he added. "We're the first ones out."
"There are a lot of implications for people who receive gold standard statins and either don't respond in a way that gives them a vigorous reduction or can't tolerate high doses, experiencing muscle pain and weakness," added McKenny. "That's the group that is an obvious low-hanging fruit that needs additional help. And this product based on what we found can give them a remarkable and dramatic additional lowering" of LDL. Later, he added, investigators will be able to assess the potential health benefits for the broader population.
Just how big that market might be triggered some chatter on Twitter over the weekend, with at least one analyst raising concerns that only a small subpopulation of patients could benefit from LDL drugs like the ones Amgen and Sanofi/Regeneron are investigating. But in a recent note, Jefferies analyst Biren Amin reportedly noted estimates that 77 million U.S. adults have high levels of LDL, with an estimated 3.4 million high-risk patients that could benefit most from a treatment like this.
According to their release, serious adverse events occurred in one patient receiving placebo and three patients in the active treatment arms, including a patient on active treatment who experienced a skin rash diagnosed as leukocytoclastic vasculitis.
"Based on this finding and the results of our Phase II trials, Sanofi and Regeneron plan to initiate the SAR236553/REGN727 Phase III program in the second quarter," says Zerhouni.
- here's the press release