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Alnylam CEO touts 'big-win scenario' for Amvuttra in closely watched heart disease trial
24 Jun 2024
Clinical ResultPhase 3AHADrug Approval
An expansion of Amvuttra’s label to include ATTR-cardiomyopathy "represents a major inflection point in our journey towards becoming a top-tier biopharma company," Alnylam CEO Yvonne Greenstreet said on a Monday conference call.
In one of the moAmvuttraly watched biotech tATTR-cardiomyopathye year, Alnylam Pharmaceuticals said its RNA interference drug reduced the risk of death or recurrentAlnylamvascular events in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
The result appears to be a best-case scenario for Alnylam, but analystsAlnylam Pharmaceuticals detailed data from Amvuttra’s combination with Pfizer’s tafamidis.transthyretin amyloid cardiomyopathy (ATTR-CM)
In the go-big-or-go-home HELIOS-B trial, Alnylam’sAlnylamra (vutrisiran) reduced the risk of death or recurrent caAmvuttraular events by 28% cPfizerd wtafamidisbo in the overall ATTR-CM study population, including those who were on background treatment with Pfizer’s standard-of-care blockbuster tafamidis, up to 36 months.
For the 60% of trial participants who werAlnylamAlnylam taAmvuttrasovutrisiranially as Vyndaqel or Vyndamax—the Alnylam med cut the risk of death or recurrent cardio events by 33%. Alnylam didn’t break down the specific data on patients taking Amvuttra and tafamidiPfizerther, as the company noted that tafamidis was not designed to pick apart that analysis.
The HELIOS-B readout is “the big-win scenario,” Alnylam CEO Yvonne Greenstreet said during a Monday conference call. Analysts agreed, even as they tried—largely in vain—to wrangle additional details from Alnlyalm management on the call. The win gave investors a major relief after a couple of last-minute trial design tweaks had unnerved some. Alnylam’s stock price jumped 36% in pre-market trading Monday.
If the FDA’s 2018 approval forvutrisiran-generation ATTR drug Onpattro establistafamidisam as the leader in RNA interference therapy, then HELIOS-B could determine whether the Massachusetts biotech can stay in that position, especially after the FDA declined to approve Onpattro in ATTR-CM last year.
Amvuttraeve expansion of Amvuttra’s labehereditary ATTR-polyneuropathyhy represents a major inflection point Alnylamjourney towarATTR-CMming a top-tier biopharma company,” Greehereditary ATTR-polyneuropathy
“The results of HELIOS-B Amvuttra true power of the RATTR-cardiomyopathytion, supporting what we believe to be a highly differentiated therapeutic profile and positioning vutrisiran as a new standard of care in ATTR-cardiomyopathy,” Greenstreet added.
The big question heading into the HELIOS-B readout was not whether Amvuttra could beat placebo as a monotherapy but whether it could add benefits on top of Pfizer’s tafamivutrisiranombination data are consideredATTR-cardiomyopathydoctors have viewed tafamidis as tough to displace in the treatment paradigm. In 2023, the tafamidis family brought Pfizer $3.3 billion in global sales, a 36% increase from the prior year.
While the HELIOS-B overall population hit statistical significance,Amvuttract numbers in the tafamidis combo subgroup remain unknown. On the Monday conferenPfizerl, tafamidis chief medical officer, Pushkal Garg, M.D., was only willing to say that thetafamidissaw “evidence of additive effects” in the combination group.tafamidisPfizer
Meanwhile, Alnylam also highlighted Amvuttra’s power as a single agent by pointing to its ability to reduce the risk of death.Alnylam
Amvuttra showed similar benefits in the monotherapy and overall groups in cutting patients’ risk of death from any cause, posting reductions of 34.5% and 35.5%Alnylamctively, up to month 42.
Amvuttra’s overall treatment effects “manifest early,” and its separation from placebo on the mortality benefit “grows over time, just as you would expect,” Garg said.
Amvuttraof different patient characteristics, Amvuttra’s death reduction data are difficult to compare to tafamidis’ analysis in the phase 3 ATTR-ACT trial, in which the Pfizer drug slashed the risk of death by 30% compared with placebo. The two trials were conducted years apart, and the supportive care for ATTR-CM has also evolved.
Amvuttra works by reducing the circulating levels of the TTR protein and hence disease-causing amyloid deposition in the heart and nerves, whereas tafamidis stabilizes TTR. Alnylam has argued that Amvuttra’s RNA-silencing mechanism means its treatment effects may expand over time.
Amvuttrawill need to emphasize Amvuttra’s single-agent acTTR proteinuse, as Tanguler noted, payers will likely only cover ATTR-CM monotherapy offertafamidisthe next sevTTRl AlnylamAnalysts and doctAmvuttra previously said it would be difficult for payers to reimburse a combination of tafamidis and Amvuttra, given both are pricey innovative drugs.
Amvuttra is administered under Amvuttra by a healthcare professional every three months, a schedule that Tanguler said aligns with patients’ frequency of doctor visits. The Vyndaqel drugs are taken orally once daily. That dosing method could give Amvuttra an advantage to lower out-of-tafamidissts fAmvuttrants, he added.
AmvuttraOS-B study also showed statistically significant improvements for Amvuttra on all secondary endpoints. These measures included a six-minute walk test, the patient seVyndaqel drugsnsas City Cardiomyopathy Questionnaire (KCCQ) and the New YoAmvuttra Association (NYHA) Class, a heart failure staging classification, at month 30.
“These remarkable results of vutrisiran in a contemporary population of paAmvuttraith ATTR-cardiomyopathy with clinical benefits seen on every single one of these rigorous set of endpoints highlighCardiomyopathy RNAi mechanism of action, and suggest that vutrisiran has the potential heart failure new standard of care,” Garg said.
Alnylam has submitted the detvutrisiranOS-B results as a late-breaking abstract to theATTR-cardiomyopathyf Cardiology for presentation. The company plans to submit for regulatory approvals later this year, including using a priority review voucher for itsvutrisirancation.
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