Sanofi, France’s largest drugmaker, said its experimental medicine Lemtrada led to an improvement in disability scores in patients with multiple sclerosis compared to an older treatment.
Lemtrada, also known as alemtuzumab, slowed the progression of disability versus Merck KGaA’s Rebif in a late-stage trial, and scores improved in some patients who were given Lemtrada in the two-year study, suggesting a reversal of disability, Paris-based Sanofi said today in an e-mailed statement.
At two years, 29 percent of patients treated with Lemtrada had experienced a six-month reduction in disability, compared with 13 percent of those treated with Rebif, said Sanofi, which presents full data from the trial at the 2012 American Academy of Neurology meeting in New Orleans today.
The disability data “is big news,” Michael Panzara, Genzyme’s therapeutic area head for multiple sclerosis, immune diseases and neurology, said in a telephone interview today. “Alemtuzumab versus Rebif improves function; that’s unique.”
Chief Executive Officer Chris Viehbacher has been building up Sanofi’s multiple sclerosis business since last year’s $20.1 billion purchase of U.S. biotechnology company Genzyme Corp., from which it gained Lemtrada. Sanofi needs new drugs such as Lemtrada to help offset revenue losses deriving from generic competition to best-sellers such as the Lovenox blood-thinner.
Among patients treated with Lemtrada, 65 percent didn’t experience relapses at two years, compared with 47 percent for those who were given Rebif in the 840-patient study, dubbed CARE-MS II, Sanofi said today.
The medicine’s prospects were at the heart of the Genzyme takeover talks last year. As part of the deal, Sanofi set up a so-called contingent value right tied to Lemtrada’s future sales. The CVR, which is listed on the Nasdaq Stock Market, was designed to protect the French drugmaker against the potential failure of the treatment and reward Genzyme investors if it succeeds.
Stockholders got one CVR for each Genzyme share they owned. Investors stood to receive payments of as much as $14 per CVR if Lemtrada wins U.S. approval and reaches sales goals, and if the company met production goals for existing Genzyme drugs. Sanofi said on July 28 that it missed the first production goal.
Today’s data shows Lemtrada “becomes a very real treatment option at all stages of the disease,” Panzara said. “It sets it apart in terms of efficacy from anything else out there.”
In spite of its effectiveness, analysts such as Vincent Meunier of Exane BNP Paribas have said Lemtrada may be too toxic to become a blockbuster.
“The drug’s sales potential will depend on its safety profile,” Pierre Corby, an analyst at Aurel BGC in Paris, said in a telephone interview today, before the data was published. Corby, who has a buy recommendation on Sanofi shares, is forecasting peak annual sales of 667 million euros for Lemtrada by 2016.
Sanofi didn’t provide additional information on Lemtrada’s safety risks today. It reiterated that 16 percent of patients developed an autoimmune thyroid-related side effect during the trial. That compares with 5 percent for Rebif, Sanofi said. It also said 0.9 percent of patients suffered from immune thrombocytopenia, a reduction of platelets in blood cells. These cases were detected early and treated with conventional therapies, Sanofi said.
Lemtrada’s safety profile “is well-characterized,” Panzara said. “Because it’s known, it can be managed and we anticipate it will be managed in the marketplace based on what we’ve learned.”
The most common adverse events associated with alemtuzumab, the chemical name for Lemtrada, were reactions linked to infusion of the drug, such as headaches, rashes, fever, nausea, itching and fatigue, Sanofi said on Nov. 14.
Full data from the CARE-MS II trial is likely to “reinforce the high efficacy/high adverse event profile of Lemtrada,” Ravi Mehrotra and other analysts at Credit Suisse Group AG wrote in an April 12 note to clients.
Sanofi is seeking ways to determine which patients are more susceptible to suffering from thyroid side effects, Panzara said.
“It’s a very active area of research right now,” he said. “We’re looking for various predictors for thyroid, for any of the other adverse events also. We will not have any of that sorted out in time for our planned submission” but “the more we can characterize that predictive risk, the greater potential” for Lemtrada, Panzara said.
Sanofi is “on track” to file for U.S. and European approval of Lemtrada in relapsing multiple sclerosis this quarter, Panzara said, declining to give more details. Genzyme is developing Lemtrada together with Bayer AG.