Every year, the promise of safer and more effective drugs coming to market gives an aging population hope. According to the Phrma organization's website, roughly $49 billion was spent on researching some 2,900 compounds last year alone. The truly amazing part is that we've only touched the tip of the iceberg on what diseases are left to be researched and/or cured. We still have a long way to go with regards to cancer research, and many rare diseases still have largely unmet needs.
Each week for the next five weeks, I'm going to look at an upcoming drug that has every indication of being not only a revenue blockbuster, but a true life changer. Keep in mind that none of the five drug hopefuls I'll be highlighting are approved by the Food and Drug Administration, so there are plenty of obstacles left to be encountered, including safety data, which could still stand in the way of an ultimate approval.
The first game-changing drug hopeful: BG-12 from Biogen Idec (BIIB 2.93%).
Biogen's BG-12 is a pill targeted at treating early stage multiple sclerosis. Currently, there are only two MS treatments given in pill form, including the recently approved Aubagio from Sanofi's (SNY 0.71%) Genzyme unit, which demonstrated a 30% lower rate of relapse than those patients taking the placebo, and Novartis' (NVS 1.00%) Gilenya.
The typical treatment plan for an MS patient involves receiving interferons. If the disease progresses, then either Elan's (ELN +0.00%) Tysabri or Novartis' Gilenya, is administered as a second-line treatment. Within the U.S., however, Gilenya is also approved as a first-line MS treatment. Unfortunately, both Tysabri and Gilenya come with nasty side effects and the need for extra monitoring. In April, for instance, the once-daily pill Gilenya was forced to add additional heart risk warnings to its label following an FDA review.
BG-12 isn't targeted at treating advanced stages of MS, but in two late-stage clinical trials (Define and Confirm), it showed remarkable efficacy in treating MS and potentially even staving off its progression. The combined results of both studies showed an almost 50% reduction in relapse rates versus the placebo for those taking the pill twice a day. Furthermore, the number of new or newly enlarging brain lesions was reduced by 71% to 99% depending on the study and the type of lesion in question. Results from the Define study also note a 38% reduction in progression of the disease leading to disability.
If these results aren't remarkable, then perhaps the fact that these results were achieved with considerably better tolerability than the other two oral treatments will raise your eyebrows. With side effects ranging from diarrhea to vomiting (at the most severe) -- and diminishing for many patients after a few weeks -- this is a marked improvement over the patients' existing choices.
Biogen isn't stopping there with BG-12 and is considering combining the drug with other therapies. It currently has a partnership with Abbott Labs (ABT 1.86%) and is in the middle of a late-stage MS trial for daclizumab, a subcutaneous injection for relapsing and remitting MS . More directly related to BG-12, Biogen is exploring its combination potential in a phase 2 trial (known as Explore) with interferons and Teva Pharmaceutical's (TEVA 3.71%) MS blockbuster, Copaxone.
The market potential for the drug, according to RBC Capital analyst Michael Yee, is for $3 billion in peak sales. The human potential for the drug, assuming it does reduce relapses by 50% and lessen the progression of the diseases in a significant number of patients, is priceless. Assuming approval, BG-12 looks like a drug that'll change people's lives for the better.
Be sure to check back next week when I unveil the next pipeline drug capable of changing your life for the better.
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