Emily Schoenthaler

Today’s BioPharm Brief leads with a late-stage clinical setback for Ultragenyx, a major gene therapy filing from uniQure, and a billion-dollar oncology licensing deal between HUTCHMED and GSK.

Sebastian Andersen, CEO and founder of ClarityNorth Partners, discusses H1 2026 biopharma M&A, oncology deal activity, Lilly’s strategy, capital deployment, and how transaction structure can help companies manage risk.

Sebastian Andersen, CEO and founder of ClarityNorth Partners, discusses 2026 biopharma dealmaking, including M&A trends, oncology deal activity, Lilly’s acquisition strategy, and how transaction structure can help companies manage risk.

TScan Therapeutics is reorganizing to prioritize in vivo-engineered TCR-T therapy for solid tumors, pausing its phase 3 trial and autoimmune program and cutting about 75% of its workforce, even as new phase 1 data showed complete donor chimerism in all tracked heme malignancy patients.

uniQure submitted a Biologics License Application to the FDA and a Marketing Authorisation Application to the UK's MHRA for ifezuntirgene inilparvovec (AMT-130), an AAV5 gene therapy for Huntington's disease, based on three-year Phase I/II data — a potential first disease-modifying treatment for a condition with no approved therapies that slow progression.

Ultragenyx published 96-week phase 3 data for GENGLYCOS, its AAV8 gene therapy for glycogen storage disease type Ia, in The Journal of Inherited Metabolic Disease, showing sustained cornstarch-intake reductions and continued glycemic control roughly six weeks after the therapy's FDA accelerated approval.

ArsenalBio is halting all development of its ex vivo CAR T programs for solid tumors and laying off the majority of its workforce as it pivots the company entirely toward in vivo CAR T therapy, joining a fast-growing field that already includes Johnson & Johnson, Eli Lilly, and several other developers.

Today’s BioPharm Brief covers a new first-line HER2 breast cancer approval in Europe, a $1.5 billion Roche deal, and new survival data for Ziihera in gastroesophageal cancer.

BeOne Medicines and Jazz Pharmaceuticals announced positive topline overall survival results from the second interim analysis of the Phase 3 HERIZON-GEA-01 trial, reinforcing the benefit of Ziihera-containing regimens just days after their FDA approval in first-line HER2-positive gastroesophageal adenocarcinoma.

GSK will begin a phase 3 trial in September 2026 for its investigational mRNA seasonal flu vaccine after phase II data showed stronger immune responses than licensed comparators. The candidate is designed to target both hemagglutinin and neuraminidase, a dual-antigen approach not yet tested in a late-stage mRNA flu trial.

Today’s BioPharm Brief looks at a new treatment for essential thrombocythemia, a halted cancer vaccine trial, and updated COVID-19 vaccine approvals ahead of the 2026-2027 season.

Novavax's partners Sanofi and Takeda have received regulatory approvals for an XFG variant-adapted formulation of Nuvaxovid, the company's protein-based, non-mRNA COVID-19 vaccine, across the US, EU, and Japan for the 2026-2027 vaccination season, positioning Novavax to continue earning royalties without maintaining its own commercial infrastructure.

BioNTech has terminated the Phase 2 BNT122-01 trial of autogene cevumeran as an adjuvant monotherapy in ctDNA-positive resected colorectal cancer, following a DSMB recommendation citing a numerical imbalance in overall survival, while the drug's separate pancreatic cancer trial continues unaffected.

Today’s BioPharm Brief looks at a new cardiovascular indication for Mounjaro, a billion-dollar ADC deal, and a potential new biosimilar competitor for Kadcyla.

Eron Kelly, CEO of ConcertAI, and Shaalan Beg, MD, chief medical officer, oncology, ConcertAI, discuss how artificial intelligence (AI) can accelerate clinical trial design, patient identification, and site selection while maintaining trust in clinical data.

The FDA has approved Lilly's tirzepatide (Mounjaro) to reduce major adverse cardiovascular events in adults with type 2 diabetes at high risk, based on the head-to-head SURPASS-CVOT trial showing non-inferiority to dulaglutide (Trulicity), making it the first GIP/GLP-1 receptor agonist with this indication.

Today’s BioPharm Brief covers positive Phase 3 data for Akeso’s ivonescimab in biliary tract cancer, FDA Fast Track designation for CytomX’s Varseta-M in colorectal cancer, and positive Phase 3 results for Tezspire in eosinophilic esophagitis.

Celularity has entered a US manufacturing collaboration with MuseCell Innovations to produce Dezawa MuseCells and related exosome and secretome products at Celularity's Florham Park, New Jersey facility, a relationship that could generate more than $300 million in aggregate purchases over five years.

Akeso's PD-1/VEGF bispecific antibody ivonescimab met its primary endpoint of overall survival against durvalumab plus chemotherapy in the phase 3 HARMONi-GI1 trial for first-line advanced biliary tract cancer, marking the first phase 3 readout for ivonescimab outside non-small cell lung cancer.

Today’s BioPharm Brief covers FDA approval of daraxonrasib for metastatic pancreatic cancer, the first patient dosed in HaemaLogiX’s KMCAR CAR-T trial, and a new collaboration to advance cell-free synthetic DNA for viral vector manufacturing.

FDA's 2025–2026 guidance changes would substantially streamline biosimilar development, including by reducing comparative efficacy and switching-study requirements. Here's what's confirmed, what industry has criticized about the existing pathway, and what's still unresolved.

HaemaLogiX has dosed the first patient in its Phase 1 KOALA trial of KMCAR T-cell therapy, a novel CAR-T designed to selectively target kappa-restricted multiple myeloma cells while sparing healthy immune cells, with the treatment reported as well tolerated and a second patient enrolling at a higher dose.

4basebio and Genezen have entered an expanded, non-exclusive collaboration giving drug developers access to 4basebio's hpDNA cell-free synthetic DNA technology within Genezen's viral vector development and manufacturing services, aiming to streamline gene and cell therapy programs from early development through commercial manufacturing.

A retrospective cohort study published in Diabetes, Obesity and Metabolism found that sustained Zepbound (tirzepatide) use in adults over 55 with obesity was associated with lower monthly healthcare costs and fewer hospital admissions and emergency department visits compared with untreated matched controls.

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. It’s a busy day for FDA action, with two approvals and a new designation spanning cancer, autoimmune disease, and rare disease. Here’s what we have today.

The FDA has approved two zanidatamab-hrii-containing regimens for first-line treatment of HER2-positive advanced gastroesophageal adenocarcinoma, based on Phase 3 HERIZON-GEA-01 data showing a median overall survival of more than two years, marking the second FDA approval for the bispecific antibody in under two years.

The FDA has granted Rare Pediatric Disease Designation to Mahzi Therapeutics' MZ-1866, an investigational AAV9-TCF4 gene replacement therapy for Pitt Hopkins syndrome, as the Phase 1/2 UNITE study surpasses 50% enrollment.

The FDA has approved IMAAVY (nipocalimab-aahu), an FcRn-blocking antibody from Johnson & Johnson, as the first therapy specifically approved for warm autoimmune hemolytic anemia (wAIHA), based on Phase 2/3 ENERGY trial data showing durable hemoglobin response and reduced fatigue versus placebo.

Today’s BioPharm Brief covers FDA priority review for GSK’s Jemperli in locally advanced rectal cancer, Genentech’s $2.3 billion deal for Hanmi’s investigational obesity drug, and an extended FDA review of Capricor’s deramiocel for Duchenne muscular dystrophy.

The FDA has extended the PDUFA target action date for Capricor Therapeutics' deramiocel BLA from August 22 to November 22, 2026, after the company submitted 24-month HOPE-3 open-label extension data supporting a refined indication focused on upper limb function.