BridgeBio Pharma Inc. (BBIO) presented encouraging data from its Phase 3 ATTRibute-CM and open-label extension study of acoramidis in ATTR-CM at the European Society of Cardiology (ESC) 2024. The data highlighted a significant increase in serum TTR levels in participants who switched to acoramidis from either placebo or tafamidis, suggesting the drug’s potential in managing this rare and serious heart condition.
Transthyretin amyloidosis (ATTR-CM) is a disease where the liver produces faulty transthyretin (TTR) proteins. These abnormal proteins form clumps (fibrils) that build up in the heart’s main pumping chamber, leading to various complications.
The data presented at ESC 2024 showed a mean increase of 3.0 mg/dL in serum TTR at Month 1 and 3.4 mg/dL at Month 6 of the open-label extension (OLE) in participants who transitioned from placebo and tafamidis to acoramidis. This significant increase in serum TTR levels is promising, as it demonstrates the potential of acoramidis to effectively manage ATTR-CM.
The FDA has accepted BridgeBio’s marketing application for acoramidis with a PDUFA action date of November 29th. Furthermore, BridgeBio has granted Bayer AG exclusive rights to commercialize acoramidis for ATTR-CM in Europe.
The positive data release has significantly boosted BBIO’s stock price, which jumped 12.50% to $27.69 at the last check on Friday. This strong market reaction signifies investor confidence in acoramidis’ potential as a treatment for ATTR-CM.
Overall, the data presented at ESC 2024 provides further evidence of acoramidis’ efficacy in managing ATTR-CM and strengthens its position as a potential therapeutic option for patients suffering from this debilitating condition.