Alnylam Reports Successful Results in Highly Anticipated Heart Drug Study

An experimental medication has demonstrated potential as an aid for certain people with a potentially fatal heart ailment, suggesting a decrease in hospitalizations and improved survival, according to an observed clinical trial. This could result in approval from regulatory authorities and assist its developer, Alnylam Pharmaceuticals, in overcoming a previous major setback.

Information shared indicated a lower incidence of death from any cause or recurrent cardiovascular events in participants with transthyretin amyloidosis cardiomyopathy who had been administered Alnylam’s vutrisiran compared to those given a placebo. Moreover, among new patients not receiving Pfizer’s drug tafamidis at the beginning of the test, vutrisiran reduced the risk by 33 percent compared to placebo.

These outcomes were covered under the primary objectives of the study after Alnylam made modifications to the statistical details of the trial in February. Similar to the case of vutrisiran, marketed as Amvuttra for another form of the disease, there were significant enhancements in the secondary endpoints relating to heart health. In addition, even performing an analysis in which survival alone was considered, a 36% reduction in the mortality rate was identified for the overall analyzed population during 42 months, and 35% for those not receiving tafamidis.

Alnylam stressed that the improvement with vutrisiran was similar across subgroups of patients enrolled in the trial, irrespective of the primary CGI severity. The comparative analysis of the results showed that the incidence of adverse events, serious adverse events, and side effects requiring study termination was similar in the drug and placebo groups. Notably, none of the adverse events in the vutrisiran group were three percent worse than those in the placebo group.

However, some aspects of the drug and its benefits remain undisclosed as the company withheld full details of clinical trials, raising certain questions about the drug. The complete result data will be available for analysis during the European Society of Cardiology meeting at the end of August.

Alnylam looks forward to filing these outcomes with the relevant authorities and using a priority review voucher to expedite the evaluation process. Stifel analyst Paul Matteis opined that the data could pave the way for vutrisiran to become a blockbuster drug for TTR-cardiomyopathy. In the wake of this announcement, Alnylam’s shares soared by as much as one-third, recovering levels last seen toward the end of 2022.
The potential success in treating transthyretin amyloidosis cardiomyopathy might lead to a financial turnaround. Though Alnylam only has sales rights to vutrisiran and Onpattro (the latter targeting the less common polyneuropathy type of the disease), the cardiomyopathy type affects over 250,000 people worldwide. The polyneuropathy form affects 30,000-50,000 individuals in the US and Europe, where diabetic neuropathy is estimated to affect 50,000-100,000 people.

Tafamidis, the only currently approved treatment, generated $3.3 billion

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