Alnylam cuts 2026 TTR revenue outlook, advances Alzheimer's and rare disease pipeline

Alnylam Pharmaceuticals reported second quarter 2026 results on July 30, 2026, alongside a revision to its full-year TTR revenue guidance. The company narrowed its 2026 total TTR net product revenue guidance from a prior range of $4,400 million to $4,700 million down to $4,200 million to $4,500 million.¹ The company attributed the change to a moderation in second line demand growth for AMVUTTRA in early 2026, which it now views as a normalized level following an early launch period that had benefited from a backlog of patients who had been waiting for a new treatment while progressing on stabilizers.¹

On the pipeline side, Alnylam started two new mid-stage studies during the quarter. It began a Phase 2 trial of ALN-6400, targeting plasminogen, in adult and adolescent female patients with von Willebrand disease who also have heavy menstrual bleeding.¹ It also launched a Phase 1 trial of ALN-6222, aimed at inhibin E (INHBE), in adults with obesity.¹ Separately, the company started a Phase 2 trial of mivelsiran in patients with Down syndrome-associated Alzheimer's disease.¹

On the regulatory front, partner Regeneron disclosed that the FDA and European Medicines Agency have accepted applications for cemdisiran in generalized myasthenia gravis, with the FDA granting Priority Review and setting a target action date in November 2026 using a Priority Review Voucher, while a European Commission decision is expected in the second half of 2027.¹

Alnylam also expanded its AI partnerships, entering a collaboration with Inceptive Nucleics intended to speed RNAi drug discovery by combining Alnylam's proprietary data with Inceptive's AI models.¹

On the balance sheet, cash, cash equivalents and marketable securities totaled $3.3 billion as of June 30, 2026, up from $2.9 billion at the end of 2025, driven mainly by cash generated from operations.¹