March 23, 2017
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON – Biogen ’s new hemophilia drug spinoff company Bioverativ officially launched in January and the new entity is already a threat to a potential hemophilia treatment developed by neighboring biotech Alnylam Pharmaceuticals , according to at least one analyst.
Alnylam is developing fitusiran for the treatment of hemophilia A and hemophilia B. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT). It is designed to prevent bleeding in patients with hemophilia by lowering levels of AT with the goal of promoting sufficient thrombin generation and fibrin clot formation to restore hemostasis, according to Alnylam. The RNAi therapy is currently in clinical development. Interim data presented in December 2016 from a Phase II trial was positive. Once per month injections of the drug “achieved consistent lowering of AT and increases in thrombin generation, resulting in a median estimated annualized bleeding rate (ABR) of 1.0 in patients with hemophilia A or B without inhibitors.” If the drug continues to be successful in clinical trials, it is expected to generate more than $1 billion when it launches, but that won’t be until 2019 or 2020. Phase III trials are expected to begin later this year.
But Alnylam is already feeling the heat from Bioverativ, which launched with two approved hemophilia drugs, Eloctate for Hemophilia A and Alprolix for Hemophilia B. In 2016, the two drugs produced about $850 million in sales. In addition to its approved drugs, Bioverativ is developing other candidates to target the bleeding disorder. Part of that investigation includes gene therapy programs and the use of Xten technology, a non-factor bi-specific antibody program to treat patients with hemophilia A. In 2014, Biogen entered into a research deal with the San Raffaele-Telethon Institute for gene therapy to develop a durable treatment for hemophilia. Bioverativ will also explore treatments for sickle cell disease.
The successful launch of the Biogen spinout came around the same time Credit Suisse analyst Alethia Young shifted her financial forecast for Alnylam, according to Investor’s Business Daily. Young predicts a 20 percent probability of success for Alnylam’s hemophilia candidate fitusiran to meet its goals. If it does meet those clinical goals, Young pegs revenue projections at $989 million, which is up from an earlier prediction of $834 million. But, she also predicts Alnylam investors will see a share loss of 13 cents, which is down from her previous prediction of an 18 cent per share gain, IBD said. Shares of Alnylam are currently trading at $52.69 as of 10:20 a.m. Shares plunged earlier this week from $59.71 as the market reacted to proposed health care legislation being debated in Congress.
In contrast, Bioverativ saw gains of about 4.8 percent on that day. However, the stock is currently down, trading at $50.90 as of 10:24 a.m. Since Bioverativ launched, its share price is up from its opening price of $44.44 on Feb. 2, its first day of trading.
Bioverativ stock received a consensus rating of “buy” from eight brokerage houses, according to Chaffey Breeze. Alnylam’s stock was lowered to a rating of “hold” by Credit Suisse on March 1.
