Agios signs $17.5M siRNA deal with Alnylam

Aug. 3, 2023

Agios Pharmaceuticals has inked a license agreement with Alnylam Pharmaceuticals under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with a rare blood cancer.

In the exclusive worldwide agreement signed by the two Cambridge, Massachusetts-based companies, Alnylam will receive $17.5 million upfront and is eligible for up $130 million in potential future milestone payments and royalties.

The siRNA development candidate aims to treat patients with polycythemia vera (PV), a rare hematologic disease that currently has no disease-modifying treatments. The asset targets knockdown of TMPRSS6, a key driver of red blood cell production. The preclincial treatment has demonstrated low off-target activity, a favorable safety profile in rats, and a 90% knockdown of TMPRSS6 mRNA over 3 months in non-human primates, supporting the potential for an infrequent dosing regimen.

Agios has one approved drug on the market — a PK activator branded Pyrukynd, which was approved last year as the first and only disease-modifying therapy for pyruvate kinase deficiency, a rare, debilitating hemolytic anemia.

Alnylam also picked up an FDA approval last year for its drug, Amvuttra, for the treatment of the polyneuropathy of transthyretin-mediated (hATTR) amyloidosis — a rare, rapidly progressive and fatal disease that presents debilitating polyneuropathy episodes.

It's been a good summer for Alnylam. The Agios deal comes with the ink barely dry on Alnylam's potential $2.8 billion deal with Roche, signed last week. The two entered into a strategic agreement to develop and commercialize zilebesiran, Alnylam’s investigational RNAi therapeutic for the treatment of hypertension, which is currently in phase 2 development.