In 2013, a number of new drugs got approvals from the FDA. This provided a boost to the biotech industry as a whole. Now, this speculative growth market is so sensitive that even positive results from clinical trials can take a company’s stock to the moon. This is what happened with Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) last week.
What a week
Last week, when Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) released its early-stage clinical data of ALN-TTRsc, a developmental therapy to cure a rare genetic disorder, the company’s stock shot up almost 32%. This increase in stock price was induced by the remarkable signs of improvement seen in the therapy’s clinical data. This particular program is developing an internally administered RNAi therapeutic for TTR mediated amyloidosis.
Wondering what RNAi and amyloidosis are?
RNA is a cutting-edge technology for preventing specific genes in the human body from producing harmful proteins. Amyloidosis is a condition in which soluble proteins become insoluble and are deposited in extra-cellular spaces of different tissues, disrupting the normal function of the organs. Transthyretin(TTR) is a harmful protein which causes amyloidosis. Hence, a simple explanation to this study being conducted by Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is that it is developing a therapy to deactivate the genes which produce harmful amounts of TTR protein, so that amyloidosis can be cured.
Positive Phase I trials
Like I mentioned, last week, the company released its Phase I study results of the program in which the ALN-TTRsc compound reduced the levels of TTR by 80% in the healthy volunteers. The company also announced that its experimental batch of the drug was well received by the clinical subjects. This proved to be a critical point for investors who were cautious about the drug’s safety in the past.
After this data came out, Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)’s stock jumped from $37 to $51, eventually coming down to $44.95 in recent days. This huge change, just after Phase I clinical trials, leads me to think that the stock still has room for growth. This is because as the ALN-TTRsc Phase I trial made the stock jump so high, imagine how high it would go once the drug gets its FDA approval. Moreover, future growth of the company can also be determined by its pipeline.
Currently, the company has one Phase II and two Phase I drugs. The rest of its projects are either in discovery or developmental phases. The company also has two partner programs. One of them is a collaboration with Kyowa Hakko Kirin for the development and commercialization of RNAi products for the treatment of RSV in Asia. The other one is ALN-VSP, which is in collaboration with Ascletis, a privately held company, for the treatment of liver cancer.