Looking ahead to a prospective FDA approval of its experimental multiple sclerosis drug Lemtrada and a new product launch into a competitive and fast-changing market, Genzyme executives today rolled out positive data from a one-year extension study of its pivotal Phase III trial that gives the company some boasting rights for a drug that demonstrated a durable response in most patients.
The bottom line of the extension data backs Genzyme's claim that the majority of the patients in the pivotal two-year trial continued to report that the disease was largely held in check for the following 12 months. Roughly 90% of the late-stage volunteers signed up for the extension study, with the understanding that they could receive additional treatment if they experienced a single relapse or the enlargement of at least two brain or spinal lesions. In the treatment naïve arm of the study, 65% of the patients receiving Lemtrada were relapse-free compared to 55% of the patients in another arm who had previously relapsed while using another treatment.
"In this 1-year extension study 80% of patients did not require an additional course of treatment, over 70% were stable or had improved disability scores," says Mike Panzara, therapeutic area head, multiple sclerosis and neurology. "This sends the message we are not going to be doing things in a me-too way. We're doing things that add value and move the field forward in treating the disease."
Genzyme is building on the successful showdown of Lemtrada vs. Rebif, a standard therapy for MS. Last November investigators reported that in a head-to-head study with Rebif (interferon beta-1a), Lemtrada was better at reducing the annualized rate of relapses, according to the trial results published in The Lancet. Also, the "accumulation of disability" was slower in the Lemtrada arm while an improvement in disability scores "suggested" a reversal of disability in some patients. Lemtrada is the commercial name for alemtuzumab, which had also been approved as Campath to treat leukemia. That cheaper drug, though, was shelved to make way for what is expected to be a far more expensive MS therapy.
If Lemtrada gets a green light from the FDA, though, it will be entering a roiled marketplace. Biogen Idec ($BIIB) is likely nearing an OK for the oral MS drug Tecfidera (BG-12), which has been overshadowing Genzyme's new drugs--oral Aubagio and the infusion therapy Lemtrada. Aubagio, approved last September, has been prescribed by 80% of all MS specialists, says Bill Sibold, senior vice president, head of multiple sclerosis for the big Sanofi ($SNY) subsidiary. Most of those new patients are being switched from other treatments, with half coming from Avonex (Biogen Idec) and Copaxone, a mainstay at Teva Pharmaceutical Industries ($TEVA).
"I don't see any effect of Tecfidera on Lemtrada," says Sibold. "I consider them completely different animals." Lemtrada has had a "profound impact on disability, where patients not only slowed (the disease), some had improvements. That's something we haven't seen."
A thumb's up from the FDA in the second half of 2013 would come later than Genzyme had initially expected. Its first application to the agency was handed back last year for a rewrite, shoving the Sanofi drug further back in the queue. The FDA recently accepted the revised NDA for review.
- here's the press release