Alnylam’s “Incredibly Deep” Pipeline Continues to Impress As Commercial Stage Looms, Says Analyst

February 27, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor

Alnylam Pharmaceuticals is continuing to impress Wall Street with its “knockout” drug platform and solid performance, but it could still face risks as it bring products to the commercial stage, said Ritu Baral, an analyst with Cowen and Co. in a note to investors Friday.

“We think Alnylam is the undisputed leader in the field of RNAi therapeutics,” wrote Baral in an overall anaylsis of the company.

“The company has shrewdly built a strong IP position around both construct and delivery technology, and taken a modular, liver-targeted approach to drug development,” she said. “We view Alnylam's current clinical programs, particularly FAP and FAC, as extremely promising with a high probability of success. The company has established a strong development and commercialization partnership with Genzyme, the commercial force in orphan drug, for rare genetic diseases.”

Alnylam is currently pursuing RNAi based therapeutics for genetically defined targets of rare, cardiometabolic, and liver-based diseases. Their in-house technology is focused on suppressing endogenous protein using short, double stranded RNA to mediate the sequence-specific degradation of mRNA.

“This technology allows Alnylam's drugs to have mechanisms involving previously undruggable targets,” said Baral. The company’s most “clinically advanced” compounds, Patisiran and Revusiran, are in Phase III clinical trials.

“Alnylam also has an incredibly deep pipeline of four other compounds in the clinic, and one poised to enter the clinic shortly,” said Baral. “The company has a broad development and commercialization partnership with Genzyme for genetic rare diseases as well as a partnership with The Medicines Company for ALN-PCS, an RNAi PCSK9 knock-down therapy.”

Still, having promising drugs at the trial stage is a whole different ballgame than bringing them successfully to market, and analysts are keeping a close eye on how to company executes its commercial strategy for its pipeline in coming quarters.

“Clinical track record no guarantee of commercial success in a few years however. We note however, that while we expect ALNY to continue to successfully develop drugs in the clinic for the next few years, the company will face a whole new type of execution risk as lead drugs reach commercial stage,” wrote Baral. “Alnylam has carefully prepared for this by carefully building an experienced commercial force, many of which came from Orphan market leader Genzyme. While we believe ALNY will prove to be successful, we note this layer of risk to the forward story.”