Back in July Alnylam ($ALNY) caught the biotech industry's attention after it wowed investors with the efficacy readout it reaped from a Phase I study of its drug for transthyretin-mediated vases of amyloidosis--or ATTR--a deadly and rare genetic disease that wreaks havoc on the nervous system and heart. Today the RNAi company revealed that the rare-disease specialists at Genzyme--the biologics arm of pharma giant Sanofi ($SNY)--had stepped in to license Asian rights to its program for ATTR, putting down $22.5 million as an upfront along with an unspecified lineup of milestones.
An initial target of their collaboration will be a program for familial amyloidotic polyneuropathy, a particularly deadly form of ATTR, along with other types of the disease. And Alnylam stands to gain a rich royalty stream ranging from the mid-teens to the mid-20s while stepping ahead with its plans to ramp up clinical development on 5 RNA programs.
"Our ALN-TTR program holds promise as a breakthrough therapy for the treatment of ATTR, a debilitating orphan disease. As the lead program in our 'Alnylam 5x15' product strategy, we also view this program as a key part of building Alnylam for the future," said John Maraganore, Ph.D., the CEO of Alnylam. "In this important collaboration, Genzyme will advance our ALN-TTR program with their proven capabilities in the Japanese and broader Asian market, while we maintain our plans to develop and commercialize this potential breakthrough medicine in the U.S., Europe, and rest of world. In addition, a key part of the value proposition in this alliance for Alnylam is the potential for significant royalty payments on sales of products."
By knocking down the TTR protein with its RNA technology, Alnylam logically believes it can treat the disease. And back in January the biotech reported that in 17 healthy Phase I subjects recruited for the study, the drug effectively slashed TTR by 94%, indicating that it could be a winner in the patient population--especially as they believe that a 50% reduction could be enough to stabilize or improve patients. The news triggered a jaw-dropping 50% spike in the company's share price, underscoring an enthusiastic response from analysts who follow the company.
It's rare for any Phase I study to generate that kind of market response, but with extremely rare diseases positive, early-stage efficacy data among small patient groups can whip up investors in ways usually not seen until late-stage data rolls in. Strategically, that R&D advantage has allowed Alnylam's stock to boom shortly after pharma's cool attitude to RNA forced a restructuring. More deals like this could significantly reignite enthusiasm for RNA among other potential partners as well.
- here's the press release