Acoramidis

BridgeBio's acoramidis, a transthyretin stabilizer, may soon compete with Pfizer's tafamidis for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis, with an FDA action date of Nov. 29, could see approval, potentially challenging Pfizer's established position in the $5.2 billion ATTR-CM market. BridgeBio's Phase III trial showed acoramidis improved survival rates and reduced hospitalizations, though it faces challenges like twice-daily dosing and lack of mortality benefit data compared to tafamidis. Emerging RNA therapies, like Alnylam's Amvuttra and Intellia's nex-z, also show promise in treating ATTR-CM.

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.

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.

Bayer submitted an MAA to EMA for elinzanetant, seeking approval for treating moderate to severe VMS associated with menopause or adjuvant endocrine therapy. The submission is based on OASIS phase III studies showing elinzanetant's efficacy and safety. The FDA also accepted Bayer's NDA for elinzanetant, and Bayer continues global submissions. Bayer aims to strengthen its pharmaceutical portfolio through new drug approvals.

FDA reviews DFD-29 for rosacea, eladocagene exuparvovec for AADC deficiency, inavolisib for PIK3CA-mutated HR-positive breast cancer, acoramidis for transthyretin amyloid cardiomyopathy, and zanidatamab for HER2-amplified biliary tract cancer, with PDUFA dates in November 2024.