A stellar launch of rare heart disease med Amvuttra has boosted Alnylam’s market cap above $50 billion.
With Alnylam’s second-quarter revenue of $774 million beating Wall Street’s expectations by 18.8%, the RNA interference biotech’s stock price surged more than 15% Thursday. The company’s market cap stood at $51.1 billion at the Nasdaq closing bell on July 31.
The significant beat was driven by Amvuttra. In its first full quarter into a closely watched transthyretin amyloidosis cardiomyopathy (ATTR-CM) launch, Amvuttra more than doubled sales year over year to reach $492 million, coming in well above consensus expectations of $351 million.
“The launch is off to a very strong start, and, whilst it’s still early days, we’re encouraged by the pace, and we’re deeply focused on laying the groundwork for long-term leadership in TTR,” Alnylam CEO Yvonne Greenstreet said on the company’s second-quarter earnings call Thursday.
In what Alnylam Chief Commercial Officer Tolga Tanguler called a “clear signal of early momentum,” about 1,400 ATTR-CM patients have started treatment with Amvuttra since its FDA nod late March, translating to roughly $150 million in revenue, he told investors on the call. The RNA interference drug is also approved in ATTR-polyneuropathy (ATTR-PN).
Utilization of Amvuttra among ATTR-CM patients has been “broad and balanced” between new patients and those who’ve progressed on a TTR stabilizer such as Pfizer’s Vyndamax (tafamidis) as well as across both academic and community settings, Tanguler said. Greenstreet noted that the initial uptake has been faster in the stabilizer-progressing patients because doctors are looking for new treatments for them, but the drug is also achieving a “really healthy share” of first-line patients.
Nearly all of the approximately 170 priority health systems targeted by Alnylam have put Amvuttra on formulary, Tanguler said. Those networks cover about 80% of the ATTR-CM patient volume.
Within just three months, the provider account setup has reached a level that Alnylam had originally planned to achieve in one year, the commercial chief said.
The “large majority” of patients across all payer types—Medicare or commercial—have access to Amvuttra as a first-line treatment, according to Tanguler. That means Alnylam is only seeing a low, single-digit percentage of reimbursement policies that require patients try a stabilizer first. And the company finds these policies “relatively easy to manage” because there’s no limit on how long a patient must stay on another therapy, plus it’s up to the physician to determine whether the patient is progressing, Tanguler added.
A ”very small portion” of patients are also getting Amvuttra in combination with a stabilizer, Tanguler said, a situation that Alnylam expects to be more popular once Pfizer’s tafamidis franchise goes generic.
Buoyed by the stellar performance, Alnylam significantly dialed up its full-year TTR sales guidance by about 34% at the midpoint to a new range of $2.175 billion to $2.275 billion.
Still, some analysts suggested that Alnylam may be conservative in its estimate, especially considering an uptick is expected from ex-U.S. markets such as Japan and Europe in the second half of the year.
“[W]e think it’s likely Amvuttra will still beat its newly guided numbers this year as mgt continues executing on this high-profile launch,” Citi analysts wrote in a Thursday note.
Based on the impressive performance seen in the quarter, William Blair analysts have raised their near-term and peak sales estimates for Amvuttra, now projecting the drug to reach $9 billion sales in 2035 across ATTR-CM and -PN. For reference, Pfizer’s tafamidis franchise, first approved by the FDA in 2019, is currently tracking at about $6 billion in annual sales.
“Alnylam is firing on all cylinders,” Greenstreet said, “and we’re swiftly establishing ourselves as a top-tier biotech company.”