Alnylam Pharmaceuticals (ALNY) delivered a bullish update on its transthyretin-mediated amyloidosis (TTR) franchise at the Wells Fargo 21st Annual Healthcare Conference on September 8, 2026, disclosing that quarterly revenue for the franchise had crossed $1 billion just roughly 18 months after its cardiomyopathy launch in March 2025.
John Kennedy, head of commercial at Alnylam, called the milestone "very, very strong." He highlighted the company's extensive distribution network, noting that approximately 90% of U.S. patients can access AMVUTTRA (vutrisiran) within 10 miles of home, and most patients face zero out-of-pocket cost. The company maintains a broad network of buy-and-bill alternate sites of care to support access.
Alnylam's total revenue surged 95% over the trailing twelve months to $4.8 billion, with a gross profit margin nearing 80%. The company's market capitalization stood at $35.6 billion, and its shares traded at $266.11, up 0.61%, before settling at an after-hours price of $263.16, down 1.11%. The stock's P/E ratio was 47.
On market dynamics, Kennedy noted the cardiomyopathy segment typically sees roughly 80% of treatment starts as first-line and 20% as second-line. About half of category growth is originating from community cardiologists. Among physicians with prior experience prescribing the therapy, AMVUTTRA captured north of 50% of new starts.
Pushkal Garg, chief of research and development, contrasted Alnylam's RNAi silencing platform against alternative mechanisms. He pointed to the CARDIO-TTRansform trial data, noting that nucresiran achieved approximately 70% mean knockdown, while vutrisiran reached 81% to 87% depending on whether mean or median figures were used — a "pretty sizable difference" in knockdown levels, he said.
Real-world evidence presented at the conference underscored the competitive advantage of silencing therapies over stabilizers such as tafamidis. An analysis of more than 800 patients showed roughly 50% of those treated with stabilizers experienced cardiac worsening within about one year on average. Daily oral treatment adherence also deteriorated sharply: approximately 30% of patients stopped taking their medication after about a year, and another 30% discontinued in the following year.
HELIOS-B trial data demonstrated about a 40% reduction in all-cause mortality over four years, and a post-hoc safety analysis suggested a halving of gastrointestinal issues or manifestations.
Mivelsiran (ALN-TTRca02), an investigational RNAi therapeutic, has shown a median knockdown of 95%, described as the deepest ever studied. The TRITON-CM trial for mivelsiran was expanded from 1,250 to 1,750 patients.
Pipeline developments included ALN-HuntTTR, targeting Huntington's disease, with Phase 1 data expected in late October at the European Huntington's Disease Network conference. The Phase 1 target knockdown is at least 50%. ALN-6400 is expected to release Phase 1 safety and knockdown data later in 2024, followed by initial efficacy data in hereditary hemorrhagic telangiectasia (HHT) and von Willebrand disease data in 2025. HHT affects roughly 70,000 U.S. patients, about half of whom become anemic and require transfusions.
Pfizer's tafamidis is expected to lose exclusivity in late 2031, extending the current period of competitive isolation for Alnylam's TTR franchise.
