Acoramidis, an experimental medicine developed by BridgeBio Pharma, has shown promising results in a Phase 3 trial, offering new hope to patients suffering from transthyretin amyloidosis cardiomyopathy (ATTR-CM), a deadly heart condition. The trial results have positioned BridgeBio to seek U.S. approval for this potential breakthrough treatment.
ATTR-CM is a debilitating heart disease caused by the toxic buildup of a protein called transthyretin (TTR), which corrodes heart tissue. Historically, this condition had been difficult to detect and treat effectively, leading to poor patient outcomes. However, with advances in medical care and the availability of treatments like Pfizer's Vyndamax, patients now have access to improved care and therapies that can help manage the disease.
Acoramidis is a next-generation, orally-administered, highly potent small molecule designed to stabilize TTR and prevent its misfolding. In preclinical testing, acoramidis demonstrated a higher efficacy in stabilizing TTR than Vyndamax, raising expectations for its potential to have a stronger effect on the disease.
The ATTRibute-CM trial, a closely watched Phase 3 study, evaluated the efficacy and safety of acoramidis in patients with ATTR-CM. The results were highly promising, showing that more study participants treated with acoramidis instead of a placebo were alive and out of the hospital after two-and-a-half years.
81% of patients who received acoramidis were alive at 30 months, compared to 74% in the placebo group. This represents a reduction in the risk of death by about 6% in absolute terms and 25% comparatively. Acoramidis also demonstrated a significant effect on hospitalization from heart problems, reducing the relative risk of such events by 50% compared to the placebo group. The treatment also showed consistent improvements in patients' quality of life and heart health, as measured by several markers of mortality, morbidity, function, and quality of life.
The positive results of the ATTRibute-CM trial mark a significant rebound for BridgeBio, as the drug had faced a setback in a previous trial. In 2021, acoramidis appeared to improve heart function based on surrogate measures, but the study missed one of its main goals when patients who received a placebo performed better on a walking test than expected. However, BridgeBio attributed this outcome to the better overall care received by patients with ATTR-CM due to early detection and awareness of available treatments like Vyndamax.
BridgeBio plans to submit an approval application to the U.S. Food and Drug Administration (FDA) later this year, hoping to bring acoramidis to patients as soon as possible. If approved, acoramidis could represent a significant advancement in the treatment of ATTR-CM and offer a new therapeutic option for patients with this life-threatening heart condition.
As the world awaits regulatory approvals, the success of this innovative medicine marks a significant advancement in the field of cardiovascular medicine and a beacon of hope for patients facing the challenges of ATTR-CM.
Sources: BridgeBio Pharma, BioPharma Dive, ClinicalTrials.gov, BioPharma Dive (2022)