Replies to post #139216 on Biotech Values

[DewDiligence]

John LaMattina weighs in on the PCSK9 pricing dilemma:

http://www.forbes.com/sites/johnlamattina/2012/03/26/new-cholesterol-lowering-drugs-will-test-the-future-healthcare-system

We are now in a world where generic statins exist. Over the coming months, the cost of therapy for even high doses of atorvastatin (the active ingredient of Lipitor) might be as low as $1 per day. Antibodies, on the other hand, are expensive with the majority costing tens of thousands of dollars per year. Insurance companies and other payers will undoubtedly look at this situation not necessarily with regard to the degree of LDL lowering provided by these compounds but rather with regard to cost. Why reimburse the costs for a PCSK-9 antibody at $20,000 per year when a generic statin at $400 per year is almost as effective? What degree of extra lipid-lowering will justify a 50-fold increase in cost per patient?

…My guess would be that these agents will be reserved for people at high risk and for whom statins are not very effective or for whom statins are not tolerated. Given that these drugs will have a high price tag, they should be a commercial success. But the initial use will be limited.

[pcrutch]

Regeneron/Sanofi REGN727 Phase III program to include CV outcomes trial, therapeutic self-administration — PI
2012-03-27 BioPharm Insight

Regeneron (NASDAQ:REGN) and Sanofi's (NYSE:SNY) Phase III program for LDL cholesterol-lowering therapy, REGN727, will consist of approximately half a dozen trials, according to Phase II principal investigator, Dr James McKinney, CEO of National Clinical Research.

The "rigorous" Phase III program will include assessment of REGN727 in heterozygous familial hypercholesterolemia, patients who don't get to LDL goal on standard therapy and a cardiovascular outcomes study in very high risk patients, McKinney said.

Regeneron and Sanofi have provided guidance for 2Q12 initiation of the Phase III program. Michael Aberman, vice president, strategy and investor relations at Regeneron indicated more details regarding the program will be announced 2Q12.

REGN727 is a subcutaneous, fully-human antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9).

The outcomes trial is likely to be a "typical" study in about 2,000 to 5,000 patients and would be three to five years in duration, McKinney said. He noted it is likely to assess high risk acute coronary syndrome (ACS) patients or those with recent myocardial infarction, unstable angina, and diabetic patients who have had a cardiovascular event.

Phase II assessment, presented 26 March as a late-breaking trial at this year's American College of Cardiology meeting, revealed REGN727 lowered LDL by 40% to 72% in patients given 50-150mg doses every two weeks and by 43% and 48% for the 200mg and 300mg doses, administered every four weeks.

Several lipid experts, including Dr Martin Landray, reader in epidemiology and Honorary Consultant Physician at Oxford University, were encouraged by the Phase II results. Landray questioned, however, if patient compliance can be maintained as patients in Phase II had to be injected with therapy every two or four weeks at clinical centers.

McKinney indicated, though, the agent would be self-administered in Phase III assessment, allaying any compliance concerns. He noted the company would ultimately want an injection syringe where patients would be able to dial in the dose.

Landray noted REGN727 would not be a replacement to statin therapy, mainly due to cost considerations. The agent would have the largest role as an additive therapy, though its major impact will be determined by whether the cholesterol effects will translate to benefit on cardiovascular events, Landray said.

Dr Paul Thompson, director of cardiology, Hartford Hospital, Connecticut, added REGN727 should be assessed as a monotherapy in statin intolerant patients. While the FDA would be more inclined to first approve the agent on top of statin therapy, REGN727 may inevitably be given as a montherapy in the future, McKinney said.

Despite an incidence of leukocytoclastic vasculitis in the Phase II trial—a rare condition which can be associated with immune-based disease such as rheumatoid arthritis, ulcerative colitis, and Crohn's disease, Phase III trials will not exclude such patients, McKinney said. He indicated leukocytoclastic vasculitis will be monitored very carefully. Since REGN727 is a fully human monoclonal antibody, it is not expected to produce antigens, he added.