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Alnylam And Sanofi Produce Monster Alternative In Hemophilia Market


  • Fitusiran continues to perform well in patients with Hemophilia A or B
  • New treatment options are needed in the Hemophilia patient population
  • Financials are in good order

Today, Alnylam (NASDAQ:ALNY) and Sanofi (NASDAQ:SNY) announced positive results for a phase 2 study treating patients with Hemophilia A or B with or without inhibitors. Results from this trial were presented today at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress in Berlin, Germany. The Fitusiran drug is an investigational RNAi therapeutic that targets antithrombin for the treatment of hemophilia A and B. It is designed to lower levels of antithrombin to help prevent bleeding.

Phase 2 Extension Study

The phase 2 OLE extension study recruited up to a total of 33 patients. At least 27 with Hemophilia A, and 6 with Hemophilia B. The Fitusiran treatment was administered on a low volume -- less than 1 mL -- monthly subcutaneous doses of 50 mg to 13 patients and 80 mg to 20 patients respectively. Patients were treated for up to 20 months in this study. Those who were treated with Fitusiran achieved around 80% in lowering of antithrombin -- AT. That resulted in an increase in thrombin generation which is good for Hemophilia patients. The study even saw patients improve in the annualized bleeding rate -- ABR. A median ABR of 1 was observed in all 33 patients. The ABR is scored within an interquartile range of 0 to 3, with the lower number being better. There was a median ABR rate of 0 for a subset of patients, 14 of whom took inhibitors along with treatment. Phase 1 data from a group of healthy volunteers along with Hemophilia A and B patients without inhibitors was published onlinetoday in "The New England Journal of Medicine". The actual results of the print won't appear until the print issue to be brought out in September 7, 2017. This publication notes safety and initial efficacy of the Fitusiran treatment in this patient population.

This article was written by

Terry Chrisomalis profile picture
Actionable ideas on small-large cap biotech stocks through deep analysis.

I am the Founder of Biotech Analysis Central, A subscription service on Seeking Alpha's Marketplace. If you want to learn more about biotech investing or you want to check out my biotech analysis you can do so with a free 2-week trial to my service. Just hit the "Learn More" button on the bottom of the Marketplace Research Tab. I have a Bachelors of Applied Science Degree In Technology Management, Industrial and Business Services Management from St. Petersburg College Florida. I have been investing in biotech stocks for many years, and I prefer to invest as a long term investor. With that In mind I seek stocks that have long term value! I primarily Like to Invest In biotechnology stocks and I accept the risks. I Write for the Healthcare Sector and Stock market in general. I contribute to Seeking Alpha.

You can follow me on stocktwits.com under the name BiopharmaPro where I currently have (62.5K) followers. Join me in my quest to find the best biotechnology stocks that deliver results to help patients with new treatment options.

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