By Natalie Huet
PARIS (Reuters) - A new cholesterol-lowering drug from Sanofi and Regeneron has the potential to become a $3-billion-dollar-plus blockbuster, according to analysts awaiting imminent late-stage trial results.
The drug, dubbed alirocumab, is part of a group of biotech medicines known as PCSK9 inhibitors that offer a new way of fighting the build-up of artery-clogging fatty deposits that put millions of patients worldwide at risk of heart attacks.
PCSK9 inhibitors are eyed as potentially the biggest advance in the fight against heart diseases since statin drugs such as Lipitor and Zocor, now widely available as cheap generics, were launched more than two decades ago.
The drugs are self-injectable, man-made antibodies targeting a protein that prevents the body from eliminating "bad" LDL cholesterol from the bloodstream. They work differently from statins - pills that inhibit the liver's production of LDL cholesterol in the first place.
"Physician feedback suggests high awareness amongst cardiologists and planned prescribing habits support multi-billion dollar potential for the class," Deutsche Bank analysts wrote in a note on Wednesday, forecasting peak annual sales of alirocumab of more than $3 billion.
Independent research firm BioMedTracker sees sales of alirocumab reaching $3.7 billion by 2023.
Sanofi and U.S. biotech firm Regeneron are among leaders in the race to bring a drug from this new class to market, neck-and-neck with Amgen. Pfizer, Eli Lilly and Roche have rival products at earlier stages of clinical development.
If positive, results from a first 100-patient trial - due any day now - will boost Sanofi's confidence in its drug, which may reach the market by 2015. But its ultimate success depends on longer-term studies involving thousands of patients, some of which will only produce results in around five years.
In the first Phase III study, alirocumab, which has cut levels of LDL cholesterol by up to 72 percent in Phase II trials, is being tested against Merck's Zetia, which can lower LDL cholesterol by 19-24 percent. Deutsche analysts expect it to easily achieve its goal in the trial.
Alirocumab is likely to used in patients with genetically-high cholesterol levels, high-risk patients who do not hit their cholesterol goals on statins alone, and patients intolerant to statins, whose side effects can include muscle pain.
Doctors at the European Society of Cardiology congress in Amsterdam earlier this month said PCSK9 drugs could be a valuable new weapon against heart disease.
But many were wary of predicting widespread use before seeing final-stage clinical data, following past disappoints with other heart drugs, including pills raising "good" HDL cholesterol.
The requirement for the new medicines to be injected and their expected high cost are also seen as potential deterrents, with pricing expected to be especially an issue in Europe, where healthcare spending is pressured by deficit-slashing policies.
For alirocumab, analysts expect a price of around $15 per day, comparable to the cost of injectable diabetes drugs known as GLP-1s, such as Novo Nordisk's Victoza.
At an investor conference earlier this month, Sanofi's CEO Chris Viehbacher said the French drugmaker could look at nearly doubling its stake in Regeneron, voicing confidence in the drug's success.
"When you look at the number of patients who are willing to inject themselves daily for diabetes, and you know a lot of those patients are also going to be in your patient population for PCSK9, I actually think that the injectable part is not going to be as big a barrier as people think," Viehbacher said.
"I think it is going to be a paradigm shift for healthcare and a potentially huge opportunity for us."
(Additional reporting by Ben Hirschler in London; Editing by David Cowell)