Having been lauded by clinicians at the main multiple sclerosis conference in Amsterdam last week, investors are now flocking to Biogen Idec after the firm released yet more positive data on its pill for the disease, BG-12.
The US biotech major has presented top-line data from CONFIRM, the second of two Phase III trials looking at BG-12 (dimethyl fumarate) in people with relapsing-remitting RRMS. The results from the 1,430-patient study show that when given twice a day, 240mg of the drug cut the annualised relapse rate (ARR) by 44% at two years versus placebo, and by 51% taken three times a day.
The CONFIRM study’s reference comparator, Teva's blockbuster subcutaneous daily injection Copaxone (glatiramer acetate), reduced the ARR by 29% compared with placebo at two years.
The CONFIRM data comes hot on the heels of detailed results from the other Phase III trial, DEFINE, which were presented at the ECTRIMS/ACTRIMS meeting in Amsterdam. In that 1,237-patient study, BG-12 significantly reduced the risk of relapse at two years by 49% and 50% at the twice- and three-times daily doses, respectively, versus placebo.
Filings 1H 2012
Doug Williams, Biogen's R&D chief, said "we now have strong positive results for BG-12 in two robust pivotal clinical trials with more than 2,600 patients”. He added that "these strong efficacy and safety results…combined with BG-12’s oral route of administration, position it as a potentially important MS therapy".
Dr Williams went on to say "we are working aggressively to prepare our regulatory submissions with the goal of making BG-12 available to MS patients as quickly as possible", probably in the first half of 2012.
If all goes well on the regulatory front, BG-12 would compete with Novartis' Gilenya (fingolimod), the only MS pill currently on the market and which garnered sales of $153 million in the third quarter.
There was a lot of excitement at the Amsterdam conference over BG-12 and how it will make inroads on Gilenya's headstart in the MS oral market. Speaking to PharmaTimes World News at the meeting, Gilmore O'Neill, who leads the MS clinical development group at Biogen, modestly noted that while there is much talk about daily tablets, and the data for BG-12 is striking, the disease is different for everybody, and for some patients, "once-a-month might be better".
The aim is to offer "a larger choice of treatment with very targeted therapies", he said. The Biogen MS armoury is already considerable with two blockbusters - Avonex (interferon beta-1a), which is injected once a week, and Tysabri (natalizumab), administered as an intravenous infusion once every four weeks. In Europe, it also recently launched Fampyra, a prolonged-release tablet formulation of fampridine to improve walking in adults with MS licensed from Acorda Therapeutics.
Biogen MS pipeline packed with promise
Dr O'Neill also highlighted the promise being shown by PEG-Avonex and Abbott-partnered daclizumab, both of which are expected to report Phase III data in 2013. The company is also extremely excited about the potential for Anti-Lingo, which is in Phase I and has shown promise in actually repairing the myelin sheath, the protective coating around nerve fibres damaged by MS.
However it is BG-12 that has sent investors and analysts into a frenzy. Following the CONFIRM data, Biogen shares ended the day up 9.4% to $116.92 and analysts at Oppenheimer raised their price target from $109.00 to $127.00. Piper Jaffray upped its target to $135 from $129.
Research notes were flying all over the place. Mark Schoenebaum at ISI Group said the data generally confirm BG-12's efficacy "and clearly show that it's likely a more effective drug than Teva's Copaxone". Leerink Swann analyst Joshua Schimmer, wrote of the drug's "pristine safety profile", saying it is now "one move closer to checkmating the MS market".