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FDA leadership shuffle at vaccines and biologics
The FDA named Katherine Szarama as acting director of the Center for Biologics Evaluation and Research (CBER), according to an HHS official cited by STAT. CBER regulates vaccines, gene therapies,...
Oncology regulatory decisions on camizestrant
The FDA’s oncology advisory ecosystem moved against AstraZeneca’s oral SERD camizestrant in two separate settings, extending uncertainty over the “new paradigm” that would broaden how and when the...
Confirmatory trial failure threatens accelerated approval for Krazati
Bristol Myers Squibb’s Krazati (fruquintinib) lost support as its confirmatory trial in second-line colorectal cancer failed to replicate required benefit, putting the company’s...
CAR T and cell therapy platform: memory-enriched CAR T shows remission signal
A first-in-human study reported that stem cell memory CAR T cells (T_SCM) achieved complete remissions at low doses without chemotherapy preconditioning, according to the research description. The...
Gene therapy regulatory path: UniQure seeks UK approval for Huntington’s AMT-130
UniQure said it plans to submit a marketing application for its Huntington’s gene therapy AMT-130 to UK regulators in the third quarter, after a positive meeting with drug regulators there. The...
AAV gene therapy for HIV: long-term SHIV suppression signal
Researchers at Oregon Health & Science University reported long-term suppression of SHIV replication in macaques using an AAV gene therapy encoding a CCR5-blocking antibody derived from...
M&A: Chiesi buys KalVista to expand hereditary angioedema portfolio
Chiesi Group agreed to acquire KalVista Pharmaceuticals in a deal valued at about $1.9 billion to add Ekterly (sebetralstat), the first oral, on-demand hereditary angioedema (HAE) therapy for...
IPO: Avalyn Pharma raises for inhaled pulmonary fibrosis programs
Avalyn Pharma priced a $300 million IPO to fund late-stage studies of inhaled therapies for pulmonary fibrosis, including a reformulated inhaled form of pirfenidone for progressive pulmonary...
FDA real-time trial pilot direction
The FDA is moving toward real-time clinical trial data review, using new technologies to reduce reporting lag for regulators. The agency plans to start with oncology studies led by Amgen and...
Company deal: Guardant and Nuvalent expand companion diagnostic collaboration
Guardant Health and Nuvalent entered a multi-year strategic collaboration to develop and potentially commercialize oncology companion diagnostics. The companies plan to use Guardant’s tissue and...
Huntington’s gene therapy regulatory push
UniQure said it will seek UK approval for its Huntington’s disease gene therapy AMT-130 after meeting with UK regulators and planning a marketing application later this year. The submission will...
Big Pharma M&A in rare disease immunology
Chiesi Group is buying KalVista Pharmaceuticals in a roughly $1.9 billion deal to expand its rare disease portfolio with Ekterly (sebetralstat), an oral on-demand therapy for hereditary angioedema...
Biotech capital markets: Avalyn IPO
Avalyn Pharma launched a $300 million initial public offering, planning to trade on Nasdaq to fund late-stage work on reformulated inhaled respiratory drugs. The IPO is positioned to support...
Oncology biotech partnering and precision diagnostics scale-up
CareDx agreed to acquire Naveris in a deal worth up to $260 million, giving it access to NavDx, a liquid biopsy test for HPV-related cancers. The acquisition adds tumor-naïve minimal residual...
FDA seeks real-time clinical trial data
The FDA announced a pilot effort to review clinical trial data in real time, aiming to reduce reporting lag and improve reviewer access as data accrues. The agency said the program will build on...
Neonatal outcome evidence: HIE and beyond
French researchers published population-based 3-year outcomes data for children who experienced hypoxic-ischemic encephalopathy (HIE) as neonates, adding long-horizon evidence on incidence and...
mRNA vaccine design principle: cell-type detargeting
Researchers at the Icahn School of Medicine at Mount Sinai reported that detargeting mRNA expression away from hepatocytes can strengthen T-cell immunity in preclinical lymphoma models. Published...
Clinical trial disruption: Huntington’s rival PTC’s interim readout
PTC Therapeutics released top-line results from the 24-month interim analysis of Pivot-HD, reporting dose-dependent “favorable” effects on disease progression for stage 2 Huntington’s disease...
Public health trial interruption: Newron clinical hold
The FDA placed a clinical hold on enrollment in Newron Pharmaceuticals’ U.S. Phase 3 schizophrenia trial ENIGMA-TRS 2 after a patient death occurred outside the country. Newron said the sudden...
CAR-T manufacturing expansion: South Korea’s first homegrown CAR T
South Korea’s MFDS approved Curocell’s anbalcabtagene-autoleucel (Limcato) as the first homegrown CAR T-cell therapy for patients with advanced diffuse large B-cell lymphoma. The approval marks a...