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.
The reason why these results are important is because Hemophilia A and B patients replenish themselves with factor replacement therapies. That is noted in the quote below by John Pasi M.D., Ph.D:
"Current hemophilia management is based on factor replacement therapies that require frequent intravenous infusions to maintain adequate factor trough levels. Significant unmet need remains for additional therapeutic agents"
As noted above new treatment options are desperately needed for this market segment. Especially, with patients having to receive treatment so often. On the other hand, Alnylam and Sanofi drug Fitusiran only requires monthly subcutaneous dosing. Another treatment option such as this would be more welcomed than frequent factor replacement therapies. Alnylam stated that it has just began a phase 3 program, known as ATLAS, just last week in treating patients with Fitusiran that have Hemophilia A and B. This late-stage study will evaluate safety and efficacy of Fitusiran in this patient population. Results from this study are expected to be readout by mid to late 2019. If the results of the ATLAS study are successful, then both companies will file an NDA for FDA approval.
According to the 10-Q SEC filing, Alnylam has cash and cash equivalents of $812.2 million as of March 31, 207. A lot of the cash comes from the research collaboration between Anlylam and Sanofi that was established in 2014. In addition, Alnylam also has a collaboration with The Medicines Company (MDCO) for a drug named inclisiran. With these collaborations along with the cash on hand there is no significant risk of any near-term dilution. Alnylam even mentions of partnering many of its other programs as well.
There are quite a few risks associated with investment in Alnylam. The first of which is that the positive results in the phase 2 OLE extension study are early in nature. That means that there were only 33 patients recruited in the study. The drug must be tested in a larger patient population, which is why Alnylam and Sanofi must confirm efficacy of Fitusiran in the phase 3 ATLAS study. The financial situation of Alnylam rests on the collaboration of Sanofi and The Medicines Company. Which means if these partnerships fall apart for any reason, Alnylam might forced to sell shares of it stock to raise cash.
Alanylam and Sanofi have achieved good results in the phase 2 extension study. More testing will be needed to confirm the findings, but the initial results are encouraging for these patients. Hemophilia patients need a new treatment option that doesn't require frequent infusion periods. If Fitusiran makes it to market patients will only need to take monthly subcutaneous doses to treat their disease. That would be a more friendlier treatment option for these patients, and it would be an improvement in quality of life.
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