Tafamidis Meglumine

iStock, Deagreez In its first commercial quarter for ATTR-cardiomyopathy, Alnylam’s Amvuttra reached roughly 1,400 patients and made more than $490 million. In its first commercial quarter in the ATTR-CM market, Alnylam’s siRNA therapy Amvuttra carried the biotech’s revenues to a comfortable beat over consensus estimates and gave the company enough confidence to substantially raise its full-year 2025 guidance. Amvuttra made $491.9 million in the quarter, a 112% increase from the same period last year. Those sales are from both of its indications: polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTR), for which Amvuttra was initially approved in 2022 , and ATTR-cardiomyopathy (CM), its newest indication that was granted in March . Some 1,400 ATTR-CM patients were on Amvuttra at the end of its first quarter of availability for the condition, according to Alnylam’s second-quarter earnings report on Thursday. Writing to investors on Thursday, analysts at Stifel called Amvuttra’s Q2 performance “very impressive,” noting that its total revenues deliver a “significant beat versus Street expectations” of $342 million. Investors also seemed impressed by Alnylam’s execution of Amvuttra’s ATTR-CM launch. The company was up more than 15% at market close. “Even in the backdrop of high Street expectations, Amvuttra performance’s here clearly aligns with the bull case and makes us more optimistic on peak sales potential,” Stifel added. BMO Capital Markets wrote in their own note late Thursday that Amvuttra’s sales “reflect strong patient demand,” given a growing prescriber base, robust uptake without price adjustments and apparent penetration of the drug into first and second line settings.Given Amvuttra’s strong launch, Alnylam on Thursday lifted its full-year sales guidance from $2.05 billion to $2.25 billion, as previously forecasted, to $2.65 billion to $2.8 billion, a 27% increase. The company also now expects the growth of its net product sales to hit 59% to 68%, year-on-year at constant exchange rates, versus its prior guidance of 26% to 39%. The biotech made $773.7 million in Q2, representing a 17% year-on-year growth and a substantial beat over the consensus revenue forecast of $651 million. In the ATTR-CM market, Alnylam is facing off against Pfizer, which owns the transthyretin stabilizer tafamidis. That drug was approved in 2019 as Vyndaqel and Vyndamax and has used its first-to-market advantage well: In 2024, Pfizer logged $5.45 billion in sales for its tafamidis products, a hefty 60% year-on-year growth. Pfizer remains confident that it can protect its slice of the ATTR-CM market, with CEO Albert Bourla telling investors at a TD Cowen event in March that as long as treatment is going well, doctors “will never switch a patient to something different.” Pfizer “will maintain a lion’s market share” for new ATTR-CM prescriptions, Bourla added, noting the pharma’s extensive network and deep understanding of physician prescription patterns. Also competing with Alnylam is BrdigeBio, which owns Attruby, another transthyretin stabilizer. Since its approval in November 2024 , Attruby had delivered 1,028 unique prescriptions as of Feb. 17. As of April 25, more than 2,000 unique patients had been treated with Attruby, resulting in $36.7 million in sales during the drug’s first commercial quarter.

A recent label expansion is sending sales of Alnylam’s Amvuttra through the roof. The drug pulled in $492.0 million in revenue in the second quarter, up 114% from the $230.1 million it reported in the same period a year ago. It’s a significant increase in sales for the first full quarter after Amvuttra was approved in March to treat a rare heart disease called transthyretin amyloid cardiomyopathy, or ATTR-CM. Alnylam’s “TTR” revenues, which include Amvuttra and an older drug called Onpattro, increased 77% to $544.5 million, up from $307.3 million in last year’s second quarter. Sales in this business also jumped 51% from the first three months of the year. The increase in sales is driven by about 1,400 ATTR-CM patients taking the drug as of June 30. It also prompted Alnylam to raise its full-year guidance for product revenue. Alnylam lifted its projected range to $2.65 billion to $2.8 billion, up from $2.05 billion to $2.25 billion. Most of its product revenue is expected to come from Amvuttra, which now makes up about 73% of its sales. Alnylam said that total “TTR” revenues will now fall between $2.2 billion and $2.3 billion this year, up from an earlier projection of $1.6 billion to $1.7 billion. Onpattro recorded $53 million in second-quarter sales. Alnylam’s shares $ALNY were up about 17% at market open on Thursday. An approval in ATTR-CM was seen as a bellwether for Alnylam , with the disease’s market expected to surpass $10 billion by 2030. The company has never ended a year with a profit despite pioneering RNAi drug development and winning four new drug approvals since its founding in 2002. But Alnylam’s impressive second-quarter results are likely to give investors confidence that the company can keep its promises. CEO Yvonne Greenstreet said earlier this year that Amvuttra’s approval would represent the next phase of Alnylam’s growth as a company, and it would reach “sustainable non-GAAP profitability” by the end of the year. Alnylam’s chief commercial officer Tolga Tanguler told Endpoints News on Wednesday that the company is on track to reach that goal. “Let’s be honest: Even though Amvuttra is already a billion-dollar franchise, we haven’t really had a true blockbuster,” Tanguler said. “We’re still in the early innings, but we’re certainly showing the world that now we’re a fully-integrated biotech.” Tanguler said the swift uptake of Amvuttra in ATTR-CM is mostly to do with formulary placement, through which about 80% of patients get their prescriptions. Alnylam had originally thought getting Amvuttra through the formulary process would take until the end of 2025, but it has been “essentially accomplished” within the first three months of the drug’s label expansion. The “majority of payers” have also made Amvuttra eligible for ATTR-CM patients who have not received any prior treatment, Tanguler said, and that’s another driver of the strong sales numbers. Skeptics believed getting Amvuttra into the first-line setting would be a challenge, given the availability of oral stabilizers like Pfizer’s Vyndaqel/Vyndamax and BridgeBio’s Attruby that are cheaper and more convenient than Amvuttra injections. But Tanguler said patients are getting Amvuttra in the first-line setting and also switching from the stabilizers — the latter representing a potential threat to Pfizer and BridgeBio’s market share. Tanguler declined to specify how many patients taking Amvuttra received their first treatment or how many switched from stabilizers. “It’s not just because we’re keeping it close to our chest, but it’s difficult to get that very specifically, because it’s claims data,” Tanguler said. If Alnylam can keep up the momentum, it will have a lot of new money to spend on its pipeline over the next several years. The company unveiled new ambitions at its R&D day earlier this year, which included a potential successor to Amvuttra called nucresiran and multiple programs that hope to deliver its gene silencer tech to areas of the body beyond the liver. Much of the new Amvuttra revenue is expected to be funneled into that pipeline, Tanguler said. “Both our hypertension program zilebesiran as well as our nucresiran program are going to be large, significant investments, and we’re really excited about that,” he said. Editor’s note: The company’s stock reaction has been added to the story.