Alnylam Pharmaceuticals Inc. has launched a Phase 1 trial for a drug candidate targeting a rare disease known as ATTR. Although it is an early-stage trial, investors and analysts are taking notice because it involves a potential breakthrough technology, RNA interference, and there are currently just a handful of RNAi human trials ongoing. RNAi, researchers say, has the potential to revolutionize drug discovery by contributing to our understanding of how to turn genes off and on.
The Cambridge-based biotech company’s Phase 1 trial is being conducted in Portugal, Sweden and the U.K., and it is designed to enroll approximately 28 ATTR patients. The first patient has already received the experimental intravenous medication, called ALN-TTR01. The primary objective is to evaluate the safety of the drug target.
“We are very excited about the potential for our ALN-TTR program to make a significant impact in the treatment of this disease,” said Dr. Akshay Vaishnaw, senior vice president of clinical research at Alnylam. “Our pre-clinical data in this program are very encouraging and point to the potential for significant clinical impact.”
ATTR is caused by mutations in a gene that is primarily expressed in the liver. The mutation can cause serious harm to the peripheral nervous system, heart and the gastrointestinal tract and can be fatal. The drug target is aimed at silencing this gene. Previous studies in primates and mice have yielded promising results, researchers said. There are currently no approved drugs for this disease and the only available treatment is a liver transplant.
Simos Simeonidis, biotechnology analyst at New York investment bank Rodman and Renshaw, in a research note issued Wednesday, noted the importance of the trial launch. He is predicting a one-year target share price of $37 for the company.
Alnylam (Nasdaq:ALNY) stock was trading at $15.30 midday on Wednesday, up from $15.06 at the previous close.