Alnylam’s franchise-extension plan bears fruit

Alnylam already has one marketed amyloidosis therapy in the form of Onpattro. It could soon have another, more convenient option, following today’s hit in the phase III Helios A study of vutrisiran. Alnylam is not saying much for now about Helios A, in the polyneuropathy subtype, except that this met its primary endpoint, mNIS+7 at nine months versus historical placebo data, as well as hitting both secondary measures. Vutrisiran also showed an improvement on a cardiac biomarker, NT-proBNP; more details will be needed to gauge the project’s chances in the cardiomyopathy amyloidosis subtype, for which Onpattro is not approved but Pfizer’s Vyndaqel and Vyndamax are. Full data from Helios A are expected at the American Academy of Neurology meeting in April; 18-month results, due later this year, should also include more details on cardiac endpoints. Alnylam’s study of vutrisiran in cardiomyopathy, Helios B, is not set to complete until 2024, but the group hopes that the Helios A data will be enough to help it penetrate the mixed amyloidosis phenotype “above and beyond” what Onpattro has managed. Alnlyam, which is also working on a twice-yearly formulation of vutrisiran, saw its stock open up 8% this morning.

Top five amyloidosis therapies in 2026
Project Company Status Delivery 2026e sales ($m)
Vyndaqel/Vyndamax Pfizer Marketed* Oral once daily 3,973
AG10 (acoramidis) Bridgebio Ph3  Oral twice daily 1,820
Vutrisiran  Alnylam Ph3  SC every 3mths 1,290
Onpattro Alnylam Marketed** IV every 3wks 1,015
Tegsedi Ionis Marketed** SC once weekly 617
*Approved in US for cardiomyopathy subtype only; **Approved in US for polyneuropathy subtype only. Source: EvaluatePharma.

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