Alnylam Pharmaceuticals (NASDAQ:ALNY) Chief Financial Officer Jeff Poulton said the company remains confident in its TTR revenue guidance despite a slower first-quarter sequential growth rate, pointing to access, international launches and prescriber expansion as key drivers for the rest of the year.
Speaking at a Bank of America healthcare conference in Las Vegas with Tazeen Ahmad, senior biotech analyst at Bank of America, Poulton described Alnylam as a 25-year-old company built around RNA interference, or RNAi, technology. He said the company has moved from being loss-making to profitable and is now focused on investing in a broader pipeline while maintaining its leadership in TTR.
“We wanna be more than just a TTR company,” Poulton said, while adding that the TTR franchise remains “incredibly important” to Alnylam.
AMVUTTRA Launch Remains Central to Growth Outlook
Ahmad noted that Alnylam has guided to total TTR revenue of $4.4 billion to $4.7 billion, which Poulton clarified is “almost all AMVUTTRA.” Poulton said Alnylam reported $910 million in TTR revenue in the first quarter, representing more than 150% year-over-year growth, though sequential growth was lower than in the fourth quarter.
Poulton said the company had previously warned investors that first-quarter growth would be below the $134 million sequential increase reported in the fourth quarter. Outside the United States, Alnylam expected TTR revenue to decline by about $25 million from the fourth quarter because of pricing changes in Germany tied to the cardiomyopathy, or CM, launch. Instead, international revenue declined by $7 million, as stronger performance in Japan’s CM launch and the polyneuropathy, or PN, business offset some of the German pricing impact.
In the United States, TTR revenue grew by $59 million in the first quarter, down from $111 million of growth in the fourth quarter. Poulton cited insurance reauthorizations early in the year and the timing of product ordering and revenue recognition as factors. He said Alnylam’s closed distribution network includes one major distribution partner handling about 80% of volume, with weekly Monday orders, Tuesday shipments and Wednesday revenue recognition. The fourth quarter had 14 Wednesdays, compared with 12 in the first quarter.
To reach the midpoint of guidance, Poulton said Alnylam must average roughly $150 million of sequential quarterly growth for the rest of the year. He said the company expects international markets to contribute more meaningfully as the German pricing headwind does not repeat, and he expects a step-up in U.S. growth.
Access and Prescriber Expansion in Focus
Poulton said Alnylam has built what he described as a strong foundation for AMVUTTRA access. He said more than 90% of patients can access AMVUTTRA as a first-line treatment, and most patients have no out-of-pocket copay. He also said Alnylam has established AMVUTTRA on formularies at major health systems and created a network of about 2,000 third-party infusion clinics, putting most patients within 10 miles of a clinic.
He said the company is now focused on increasing demand by expanding the number of physicians prescribing AMVUTTRA.
“When you get a physician to try AMVUTTRA for the first time, it leads them to prescribing more AMVUTTRA,” Poulton said. He added that Alnylam plans to report on the expansion of its prescriber base quarterly.
Asked about second-quarter trends, Poulton declined to give specifics but said Alnylam remains confident in the guidance it reiterated. He said second-quarter performance will be important because the company needs to show an inflection from the first quarter.
Competition and Combination Therapy
Poulton discussed upcoming data from Ionis Pharmaceuticals and AstraZeneca’s silencer program, saying Alnylam expects the study to be successful. He noted that the study is much larger than Alnylam’s HELIOS-B trial and includes a large group of patients on background tafamidis therapy.
If Ionis and AstraZeneca show a statistically significant result in patients receiving a silencer plus tafamidis, Poulton said Alnylam believes that would likely be viewed as a class benefit. He said AMVUTTRA already has combination data in its label from a prespecified subgroup in HELIOS-B.
However, Poulton said payer policies currently restrict combination use in much of the commercial and Medicare Advantage markets. He said a broader increase in combination therapy is more likely when tafamidis goes generic, which he said now appears to be in the middle of 2031 based on Pfizer’s announced settlements.
Nucresiran Positioned as Next-Generation TTR Product
Poulton highlighted nucresiran, Alnylam’s third-generation TTR product, which he said is being studied in PN and CM. He said phase 1 data showed about 95% TTR knockdown, compared with AMVUTTRA in the mid-80% range, with less variability. He also said nucresiran is designed for twice-yearly subcutaneous administration, compared with AMVUTTRA’s quarterly dosing and WAINUA’s monthly dosing.
Poulton said nucresiran could be economically meaningful because it does not carry the same royalty burden as AMVUTTRA, which he said is close to 30% on a weighted average basis. He said Alnylam has guided to operating margins around 30% through 2030, with gross margin weighed down by AMVUTTRA royalties, and sees a path to mid-40% operating margins after 2030 if nucresiran succeeds.
Alnylam recently increased the size of its nucresiran CM outcomes study from about 1,250 patients to 1,750 patients. Poulton said enrollment was progressing quickly, and the decision was primarily intended to manage timeline risk in an event-driven study.
Pipeline Readouts and Balance Sheet
Poulton said Alnylam expects three notable data readouts in the second half of the year:
- A phase 1 update from its Huntington’s disease program with Regeneron, focused on safety and knockdown.
- A phase 2 proof-of-concept readout from its plasminogen-targeting bleeding disorder program in HHT, measuring nosebleeds.
- Early phase 1 data from its obesity program targeting ACVR1C in adipose tissue.
He said HHT has an estimated U.S. prevalence of about 70,000 people and currently has no on-label treatments. Alnylam also plans an R&D webinar on the bleeding disorder program in the second quarter and expects to start a phase 2 study in a second indication this year.
On the balance sheet, Poulton said Alnylam ended the year with around $3 billion in cash and expects to begin adding cash now that it is profitable. He said the company’s priorities are supporting the TTR launch, continuing internal innovation and adding external innovation, particularly in delivery technologies as Alnylam works toward reaching 10 tissues by 2030.
About Alnylam Pharmaceuticals (NASDAQ:ALNY)
Alnylam Pharmaceuticals, Inc (NASDAQ: ALNY) is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.
Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing.
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