Agios looks beyond PK activators with $17.5M deal for Alnylam's siRNA blood disease program

Agios Pharmaceuticals is basking in the “tremendous progress” of its PK activator pipeline, but that hasn’t stopped the biotech from looking for ways to freshen up its hematology portfolio courtesy of a preclinical siRNA program from Alnylam.

Agios is paying $17.5 million upfront for the program with up to $130 million in development and regulatory milestones potentially to follow. In return, Agios is taking on sole responsibility for ushering the TMPRSS6 program through the clinic and, hopefully, to market, with Alnylam providing manufacturing support for phase 1 only.

The program is being developed for patients with polycythemia vera, a disease characterized by excessive production of red blood cells that can ultimately prove fatal. Despite affecting around 100,000 patients in the U.S., according to Agios, there are no disease-modifying treatments available.

The aim of the siRNA program is to target knockdown of TMPRSS6, which is a key driver of red blood cell production. So far, the program has demonstrated a 90% knockdown of TMPRSS6 mRNA over three months in nonhuman primates, which Agios said supports “the potential for an infrequent dosing regimen.”

“This program is highly aligned with our core scientific expertise, clinical development and commercial capabilities in rare hematology as well as our business development strategy to expand beyond our industry-leading pipeline of PK activators,” Agios CEO Brian Goff said in the release. “We look forward to initiating IND-enabling studies this year with the aim of delivering a transformative treatment option for this patient community with profound unmet need.”

Agios describes itself as the “pioneering leader in PK activation.” In the company’s second-quarter earnings release, which coincided with the licensing announcement, Goff pointed to positive phase 2 data from the biotech’s approved PTK inhibitor Pyrukynd in sickle cell disease last month.

But rather than double down on the “tremendous progress executing across our industry-leading pipeline of PK activators,” Goff used the earnings release to announce that the company is “further expanding our portfolio beyond PK activation through focused business development.”

The latest licensing deal also marks a good summer for Alnylam, which only last week pocketed $310 million from Roche in exchange for most of the rights to the midstage hypertension med zilebesiran.