Alnylam says it wants to enter larger disease areas using partnership-driven approach

Alnylam's Laurence Reid

RNAi specialist Alnylam ($ALNY) is focusing on rare diseases at the moment--with the exception of hepatitis B--but Chief Business Officer Laurence Reid told investors at the Leerink Partners Rare Disease Roundtable that the company is also looking to enter bigger markets via partnerships.

He said the mounting human pharmacology and safety data about its ESC-GalNAc-siRNA conjugates drug delivery platform is "absolutely opening up opportunities for Alnylam to participate in chronic diseases and diseases outside the rare disease space. We think those will continue to be driven by targets in the liver."

Alnylam must balance its desire to enter new and larger markets with its traditional rare disease focus as it seeks to be the first to take an RNAi therapy to market.

"We're constantly thinking about how we should allocate capital between those two broad buckets," Reid said. "I think in simple terms, where there are rare disease opportunities with great genetically driven targets in the liver, we're going to keep exploiting those and working on those with our friends at Genzyme. And then where we see opportunities beyond hepatitis B outside the liver, we're very interested in those. In simple terms I think we'll take more of a partnership-driven approach to moving into those bigger disease areas."

"We still have a number of what we think are exciting opportunities that we haven't yet shared with you. There are more opportunities to address rare diseases in the liver," he added.

Reid provided many details and insights into the company's 12-candidate pipeline, including three that are in the clinical testing phase.

Investors are anxiously awaiting the results of the company's programs to develop therapies for the genetic disease TTR-mediated amyloidosis. The first readout of the primary endpoint of the company's Phase III study of the candidate patisiran will occur during the Oct. 12-14 American Neurological Association meeting in Baltimore, Reid said.

But there are several caveats. The patients are in an open-label extension study of patisiran with the same primary endpoint as the Phase III study, and the data will only be released for 20 patients.

"In terms of the clinical efficacy, it's too early for us to be able to make any definitive deductions, which is frustrating for all concerned, but it is what it is, and it's the clinical and statistical reality of the size of the study and its open-label nature," he said, adding that the Phase III readout will "eventually tell us the answer to that (efficacy) question."

Patisiran is in Phase III, and Reid said Alnylam hopes to move its other candidate for TTR-mediated amyloidosis, ALN-TTRsc, into Phase III by the end the year. ALN-TTRsc treats a different form of the disease (Familial Amyloidotic Cardiomyopathy, or FAC) that affects at least 40,000 people (larger than the market size of patisiran, estimated to be 10,000). Reid explained that Alnylam is also running a trial of 1,000 patients to study the association between FAC and the heart disorder cardiac amyloidosis.

Regarding other Alnylam development programs, Reid said the company aims to share more human data about Phase I hemophilia candidate, ALN-AT3, at the Dec. 6-9 meeting of the American Society of Hematology in San Francisco. "We should have the full study results available sometime in the middle of next year," he said.

Finally, he said that Alnylam believes the paroxysmal nocturnal hemoglobinuria (PNH) patient population is the optimal market to gain approval for its developmental stage candidate, ALN-CC5, for complement-mediated diseases.

- listen to the presentation (reg. req.)