Alnylam stock jumped Friday after the drugmaker won a second Food and Drug Administration approval for its drug, Amvuttra.
Amvuttra was already approved to treat patients with transthyretin amyloid polyneuropathy, a rare disease in which abnormal protein builds up on the nerves, causing systemic damage. Now, the FDA has signed off on it in patients with cardiomyopathy, a significantly bigger market.
Alnylam Pharmaceuticals will take on Pfizer's Vyndaqel and BridgeBio Pharma's new Attruby. But, while Vyndaqel and Attruby are daily pills, Amvuttra is a shot given every six months.
"With its impressive efficacy and safety profile, along with its differentiated treatment regimen of subcutaneous (under-the-skin dosing, we expect strong uptake of Amvuttra in ATTR-CM," William Blair analyst Myles Minter said in a report.
Alnylam stock jumped 11.8% to 283.34. Shares bounded off their 50-day line and are working their way toward a buy point at 304.39 out of a consolidation, according to MarketSurge.
First Silencer To Hit Cardiomyopathy
Amvuttra is the first in its class to enter the ATTR cardiomyopathy market. Vyndaqel and Attruby work by stabilizing the errant amyloid protein. Amvuttra, instead, works by silencing the gene that makes amyloid.
Analysts largely expected Amvuttra's approval, though had split view on the price. Alnylam said it would retain the $477,000 per year Amvuttra price for the cardiomyopathy launch. That's nearly double the $268,000 and $245,000 annual price tag for Vyndaqel and Attruby, respectively, Needham analyst Joseph Stringer said in a report.
He noted Alnylam plans to reduce Amvuttra's net pricing over time "as patient uptake increases in the relatively larger ATTR-CM indication (compared to ATTR-PN)." The company reiterated its call for $1.6 billion to $1.725 billion in sales this year from Amvuttra.
Stringer kept his buy rating and 320 price target on Alnylam stock.
Vyndaqel Launches Remains Impressive
Stringer says the market is big enough for all three players.
Pfizer's Vyndaqel earned $5.5 billion in sales last year and only captured about 20% of the market. In 2030, analysts expect Vyndaqel to do $1.9 billion in sales — falling after a patent cliff — with Attruby bringing in $1.7 billion and Amvuttra in the lead at $5.1 billion.
Further, diagnosis rates are on the rise and Amvuttra appears favorable for younger and healthier cardiomyopathy patients, William Blair's Minter said. He rates Alnylam stock an outperform.
This "positions Amvuttra well for first-line usage in the milder and younger disease population that is reflective of the current disease population," he said.
But Leerink Partners analyst Mani Foroohar says Alnylam could experience "significant" headwinds thanks to its huge pricing premium over Vyndaqel and Attruby.
"While management prudently managed expectations — reiterating that ATTR-CM launch will be a 2H25 story — elevated long-term Street projections for market share and crowded long positioning in ALNY skew risk to the downside for the next 12-18 months," he said in a report.
He has a market perform rating and 206 price target on Alnylam stock.
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