The race for the new generation of hepatitis C treatments continues as Bristol-Myers Squibb (NYSE: BMY) takes first place in seeking approval for a truly all-oral treatment independent of interferon and ribavirin.
Gilead Sciences (NASDAQ: GILD)'s sofosbuvir and Johnson & Johnson (NYSE: JNJ)'s simprevir both made significant progress toward FDA approval by the end of this year. The oral agents have unfortunately only undergone studies in conjunction with the injectable interferon, which is associated with both its cumbersome method of delivery and unpleasant flu-like side effects. Other agents have undergone studies in combination with ribavirin, which is associated with side effects of anemia and rash. The ultimate goal is to develop a stand-alone oral agent independent of both interferon and ribavirin, achieve both ease of delivery and improved compliance and more benign side effects.
Gilead, J&J, Bristol-Myers, Merck (NYSE: MRK), AbbVie, and until recently, Vertex, were all vying to be the first in that space. However, Gilead has been the perceptual the leader of the pack due to sofosbuvir's unanimous recommendation by FDA panel last week and impressive study results; J&J's simeprevir is less far-reaching and will target a much smaller market and genotype of hepatitis C.
Bristol-Myers, however, has won the race in Japan. The company announced over the weekend its submission to Japan's Pharmaceutical and Medical Devices Agency for approval of its purely oral and interferon-free hepatitis C combination of daclatasvir with asunaprevir.
This announcement followed phase 3 data of the two drugs in 222 Japanese patients showing an 84.7% overall cure rate over 24 weeks. Japan is not a bad market to be the first for approval. According to the WHO, as compared to the 150,000 new cases annually reported in the US and Western Europe, Japan has an incidence of approximately 350,000 new cases each year. Around 1.2 million people in Japan have hepatitis C, and more than 90% of hepatocellular cancer in Japan results from hepatitis C infection, making treatment essential in this population.
The race for a cure is far from over. As Gilead and J&J expect New Drug Applications this year, Bristol-Myers plans to begin phase 3 trials of a combination of the existing daclatasvir and asunaprevir with a third agent, BMS791325, into a three-drug single pill taken over just a 12-week course. Gilead is working on its own combination therapy, and AbbVie has long had a late-stage agent in the making .
Although late to the game, Merck, in collaboration with Bristol-Myers, is making headwind in phase 2 trials as well for a new combination therapy. Responsible for current hepatitis C therapy Victrelis, albeit with struggling sales down to $502 million in 2012, Merck earned breakthrough designation for its new combination of MK-5172 and MK-8742. The first breakthrough designation drug was approved last week, giving more credibility to the new FDA program to expedite review and approval of certain drugs. Merck recently announced phase 2 data of a study showing cure rates of higher than 90% when used both with and without ribavirin. The therapy is intended to also be an all-oral agent used independent of interferon and ribavirin.
There clearly continues to be significant movements and improvements from several main players in big pharma, and this will continue throughout the year until an agent is approved. Analysts project a $20 billion market for the new therapy, and that number continues to grow as more patients are diagnosed each year and as physicians continue to hold off initiating patients on old therapies in anticipation of newer better agents. As these therapies throttle down the pipeline, the winner will be not only the markets, but also the patients.