Vutrisiran Sodium

BridgeBio Pharma (Nasdaq: BBIO) reported 54-month open-label extension data for acoramidis (Attruby) in ATTR-CM associated with a 44.7% reduction in all-cause mortality and 49.3% reduction in cardiovascular mortality versus patients who had received placebo before crossing over to active treatment, with both results reaching p<0.0001. The ATTRibute-CM open-label extension is an active, unblinded follow-on study enrolling adults with transthyretin amyloid cardiomyopathy who completed the parent randomized controlled trial, comparing outcomes in patients who received continuous acoramidis against those who transitioned from placebo. At Month 54, the mortality separation between the continuous-treatment and placebo-crossover groups was statistically robust on both endpoints. Acoramidis also attenuated the rise in NT-proBNP — a marker of cardiac wall stress and a surrogate for disease progression — to a degree the company characterized as exceeding what has been observed with other disease-modifying treatments in ATTR-CM. Heart failure-related quality of life, measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary score, was stabilized and maintained through Month 54 in patients who received early and continuous treatment. A secondary analysis showed that an early increase in serum transthyretin at Day 28 was associated with slower KCCQ-OS decline at Month 30, suggesting the pharmacodynamic response predicts longer-term functional benefit. While continuous acoramidis treatment was associated with statistically significant reductions in all-cause and cardiovascular mortality versus patients who crossed over from placebo, because the extension was open-label and non-randomized, the findings should be interpreted as supportive long-term durability data rather than evidence of superiority versus other approved ATTR-CM therapies. The safety profile remained consistent with the parent trial. Diarrhea occurred in 11.6% of acoramidis-treated patients versus 7.6% on placebo, and upper abdominal pain in 5.5% versus 1.4%; both were predominantly mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between groups (9.3% versus 8.5%). Developed by BridgeBio Pharma and licensed from Stanford University, acoramidis is a second-generation oral transthyretin (TTR) stabilizer approved in late 2024 (USA) and early 2025 (EU, Japan) for transthyretin amyloid cardiomyopathy (ATTR-CM). Designed to mimic a protective genetic variant, it offers near-complete stabilization (around 90%) of the TTR protein, a figure that distinguishes it mechanistically from tafamidis (Vyndaqel/Vyndamax), the incumbent standard of care, which achieves approximately 60% stabilization in vitro. Whether that pharmacological difference translates into a clinically meaningful outcome advantage over tafamidis has not been established in a direct head-to-head trial; cross-trial comparisons are limited by differences in patient populations, trial design, and follow-up duration. A comparative imaging study of tafamidis versus acoramidis is currently recruiting, and a Phase IV switch study is listed as not yet recruiting. The competitive landscape in ATTR-CM has shifted since acoramidis received FDA approval in November 2024. Alnylam’s vutrisiran (Amvuttra), an RNA interference therapy that silences hepatic TTR production rather than stabilizing the protein, received FDA approval for ATTR-CM in March 2025, introducing a mechanistically distinct option with quarterly subcutaneous dosing, following a Phase III trial in ATTR-CM. The entry of an RNAi agent into the same patient population creates a two-axis competitive dynamic: acoramidis competes with tafamidis on potency within the oral stabilizer class, and with vutrisiran on route of administration and mechanism. A real-world survey presented alongside the OLE data at the American College of Cardiology meeting found that physicians were unsatisfied with treatment in more than half of patients currently prescribed therapy, and that more than one-third of patients received no treatment at all — with oral therapy the most preferred format among patients. The OLE data were presented as a late-breaking oral at the ACC Annual Scientific Sessions and simultaneously published in JAMA Cardiology, lending the readout additional visibility in the cardiology community. The open-label, non-randomized design of the extension study introduces limitations that are standard for this type of analysis: the comparator group is patients who started treatment later rather than a concurrent control arm, and unblinding may influence both patient behavior and clinical management decisions. The patient population size for the OLE was not disclosed in the press release. BridgeBio stated that additional Attruby data are planned for future medical meetings. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

The fund tops Blackstone\'s own BLXS V, which raised $4.6 billion in 2020.\n One of the world’s leading investment firms has broken its own record with the closing of its $6.3 billion Blackstone Life Sciences VI (BLXS VI) fund, which is 40% larger than its predecessor.According to a March 30 release, the oversubscribed fund closed at its hard cap and is the largest private fund ever dedicated to life sciences. Since its launch in 2018, Blackstone Life Sciences has grown to manage $15 billion in assets.Blackstone portfolio companies have produced 34 regulatory approvals for medicines and devices, the firm said. These include Novartis’ cholesterol treatment Leqvio, Alnylam’s transthyretin-mediated (ATTR) amyloidosis drug Amvuttra and Pharmacyclics’ chronic lymphocytic leukemia therapy Imbruvica.In a statement, Nicholas Galakatos, Ph.D., global head of Blackstone Life Sciences, said the fund’s record “highlights how we work successfully with industry trailblazers to help bring their most important products to patients around the world.” Blackstone reported that BLXS investments have achieved an 86% approval success rate for phase 3 assets. Over the past 12 months, the platform has made $2 billion in new investments, including $400 million to support Teva’s Sanofi-partnered IBD drug duvakitug and $700 million for Merck’s antibody-drug conjugate sacituzumab tirumotecan.In July 2020, Blackstone reached its fundraising target for BLXS V, raising $4.6 billion to support late-stage product development and growth investments in emerging companies.