BridgeBio Pharma Inc (NASDAQ: BBIO) will feature updated information from its Phase II acoramidis for transthyretin amyloid cardiomyopathy (ATTR) at the Annual Scientific Session and Expo at Washington D.C. Moreover, the event will be between April 2 and April 4.
BridgeBio will also discuss the high unmet need for ATTR patients. The presentation will help bring awareness to the situation as the company finds ways to treat people living with the disease and improve their lives.
Acoramidis is still under investigation
BridgeBio is currently conducting a phase III study for acoramidis and expects to release topline data in 2023. Dr. Ahmad Masri will be the presenter for the current event.
Acoramidis, which is taken orally, is still under investigation. The drug works by stabilizing tetrameric transthyretin. Therefore it prevents the steps that occur with ATTR before they begin.
Scientists designed the drug to act similarly to T119M, a TTR gene variant. T119M is a rescue mutation as it minimizes or prevents ATTR in people with the pathogenic mutation that causes TTR. In addition, stabilizing TTR in ATTR patients could help scientists find the cause of the disease or preserve TTR.
ATTE affects the heart and peripheral nerves
ATTR, which is underdiagnosed, is a fatal disease with few therapies. The disorder is detrimental to the nervous and cardiac systems. ATTR is relatively rare and affects about 400,000 people worldwide.
ATTR occurs when TTR is unstable. The instability is often due to aging or inherited variants. When ATTR is dangerous, it accumulates in different organs in form of amyloid fibrils leading to ATTR.
The amyloid fibril gets mainly in the peripheral nerves and the heart. When it collects in the heart, it causes ATTR cardiomyopathy. If the deposits result in peripheral nerve, it leads to ATTR polyneuropathy.
Patients with the disease often experience a rapid decline in their lifestyles, life expectancy, and functional independence. It also places a burden on caregivers, who are sometimes family members, because of the patient’s quick decline. ATTR patients who cannot receive treatment often die within five years.
BridgeBio, pharmaceutical company, focuses on delivering, discovering, heating, and crafting ground-breaking therapies for cancers with known genetic causes and other genetic illnesses.