Acoramidis

Open-label extension data from ATTRibute-CM study shows sustained benefit of acoramidis on cardiovascular outcomes, including a statistically significant reduction in ACM within 36 months.

Intellia Therapeutics reports early-stage data indicating its gene editing treatment, NTLA-2001, may stabilize or improve outcomes for ATTR-CM patients, showing a median improvement in the six-minute walk test and 92% maintaining or improving NYHA class. The treatment uses CRISPR to reduce TTR production, with a 90% mean reduction observed. Intellia is now in Phase III trials for NTLA-2001, with potential revenue of $1.2bn by 2030.

BridgeBio Pharma (BBIO) reported Q3 2024 revenue of $2.7M and a net loss of $162.0M, with a cash position of $405.7M. Acoramidis, their lead drug for ATTR-CM, showed positive Phase 3 results, with an FDA PDUFA date set for November 29, 2024. Upon approval, BBIO expects a $500M milestone payment and $105M in additional regulatory milestones for European and Japanese territories. Three Phase 3 readouts are expected in 2025, with notable pipeline progress including completed screening for CALIBRATE and enrollment for FORTIFY trials, and Breakthrough Therapy Designation for infigratinib in achondroplasia.

Acoramidis (Attruby; BridgeBio) approved by FDA for transthyretin amyloid cardiomyopathy (ATTR-CM), based on ATTRibute-CM trial results. BridgeBio committed to providing the drug free for life to trial participants, while the drug's $244,000/year price draws scrutiny. Acoramidis mimics a natural TTR gene mutation, targeting ATTR-CM's root cause, and BridgeBio plans to seek approval in Europe, Japan, and Brazil. Additionally, gene-editing therapy nexiguran ziclumeran (nex-z; Intellia Therapeutics) shows promise in early trials for ATTR-CM.

FDA approved Attruby (acoramidis) for transthyretin amyloidosis, a deadly heart condition, marking a significant step in treatment. Priced at $244,000 annually, it competes in a lucrative market against drugs like Pfizer's tafamidis. Despite investor skepticism, BridgeBio Pharma aims for a 30% market share, highlighting Attruby's potential advantages in survival and hospitalization rates.

October saw FDA approvals for Pfizer's Hympavzi, Astellas' Vyloy, and Iterum's Orlynvah. November awaits decisions on Journey Medical's DFD-29, Merus' Zenocutuzumab, Astellas' IZERVAY, PTC Therapeutics' Upstaza, Autolus' Obe-cel, Applied Therapeutics' Govorestat, BridgeBio's Acoramidis, and Jazz Pharmaceuticals' Zanidatamab.

AstraZeneca and Ionis' Wainzua, a treatment for ATTR polyneuropathy, is set for EU approval after EMA's CHMP positive opinion. Wainzua, already approved in the US and Canada, reduces TTR levels by 82% and slows neurological impairment progression. It is also being developed for ATTR cardiomyopathy and is seen as a key drug for AZ's CVRM unit. Wainzua competes with Alnylam's Onpattro and Amvuttra, and Pfizer's Vyndamax/Vyndaqel.

Alnylam CEO discusses vutrisiran's potential in ATTR-CM, while Merck's RSV prophylactic shows competitive efficacy. Biotech investors focus on autoimmune diseases, and Genentech advocates for a comprehensive approach to drug costs.

Bio-pharma advances in endocrinology, dermatology, cardiovascular, oncology, rare diseases, pulmonology, gastroenterology, and neurology highlight promising therapies like Eli Lilly’s orforglipron, Amgen’s MariTide, Novo Nordisk’s oral semaglutide, Johnson & Johnson’s JNJ-2113, Sanofi’s amlitelimab, Ionis’s olezarsen, BridgeBio’s acoramidis, NewAmsterdam Pharma’s obicetrapib, Jazz Pharmaceuticals’ zanidatamab, Syndax’s revumenib, Merus’ zenocutuzumab, Johnson & Johnson’s nipocalimab, Novo Nordisk’s Mim8, Solid Biosciences’ SGT-003, Merck’s sotatercept, Johnson & Johnson’s Tremfya, and Crossject’s Zepizure, with analyses including clinical data, revenue forecasts, and expected launch dates.