Alnylam presents positive late-stage results for RNAi therapeutic in ATTR with cardiomyopathy

26 Jun 2024
pmlive.com
Clinical ResultPhase 3Priority Review
Alnylam presents positive late-stage results for RNAi therapeutic in ATTR with cardiomyopathy
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Source: PMLiVE
Alnylam Pharmaceuticals’ RNA interference (RNAi) therapeutic has shown promise in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
The phase 3 HELIOS-B study has been evaluating the efficacy and safety of Amvuttra (vutrisiran) 25mg versus placebo in patients with ATTR-CM, both receiving and not receiving tafamidis at baseline.
ATTR amyloidosis is a rapidly progressive and debilitating disease characterised by misfolded transthyretin (TTR) proteins that accumulate amyloid deposits in multiple parts of the body, including the nerves, heart and gastrointestinal tract.
The two forms of ATTR include hereditary ATTR (hATTR), caused by a TTR gene variant, which affects approximately 50,000 people globally, and wild-type ATTR, which occurs without the TTR gene variant and is estimated to affect up to 300,000 people worldwide.
The HELIOS-B study met both its primary and secondary endpoints across all key subgroups, including baseline tafamidis use, ATTR disease type and measures of disease severity.
Amvuttra works to reduce serum vitamin A levels in the blood, decreasing the build up of TTR in the body.
Amvuttra demonstrated a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular in the overall population and the monotherapy population by month 36 and demonstrated statistically significant improvements across all secondary endpoints in both the overall and monotherapy populations, including on key measures of disease progression.
In addition, the investigational RNAi therapeutic demonstrated encouraging safety and tolerability consistent with its established profile.
Pushkal Garg, chief medical officer, Alnylam, commented: “I’m thrilled by this overwhelmingly positive data from the HELIOS-B study, which suggests that [Amvuttra] has the potential to address the needs of patients with ATTR-CM.
“The results showed that [Amvuttra] improved cardiovascular outcomes, including survival, function and quality of life, in all patient groups with ATTR-CM [and] we are moving with urgency to file this compelling data with regulators to bring this medicine to patients around the world.”
Alnylam intends to proceed with global regulatory submissions for Amvuttra in ATTR-CM which are set to begin later this year, including a supplemental New Drug Application with the US Food and Drug Administration using a priority review voucher.
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