News

In the phase 3 SHINE-2 trial, half of pediatric patients who responded to mirikizumab induction were in clinical remission at week 52. Lilly plans to file for a pediatric UC label expansion.

Guselkumab (Tremfya) sustained perianal fistula healing through 1 year in Crohn disease, with remission climbing to 35% in one FUZION dosing arm.

Roche will take ex-China development of Hansoh’s two preclinical ADCs and multiple programs against Roche-proposed targets once Hansoh completes global phase 1a trials. The deal includes $400 million upfront and more than $15 billion in potential milestones.

Cemiplimab's proposed EU regimen could cut Libtayo infusion visits from every 3 weeks to every 6 weeks, trimming infusion time by up to half.

Opus Genetics reports sustained vision gains with gene therapy, the FDA approves Roche’s Enspryng for at-home treatment of thyroid eye disease, and Estuary Biotherapeutics launches with $103 million to develop gene therapies for polycystic kidney disease.

When large pharmaceutical companies deprioritize drug candidates, smaller developers may license or acquire them, but due diligence, deal structure, and trial planning determine whether a program can move forward.

Interim phase 1/2 data presented at Retina in Rio 2026 showed sustained visual improvements with Opus Genetics’ AAV8 gene therapy OPGx-LCA5 and improved retinal sensitivity with OPGx-BEST1 in inherited retinal diseases.

The FDA approved Roche's satralizumab (Enspryng), an IL-6 receptor-targeting monoclonal antibody, for adults with thyroid eye disease based on the Phase 3 SatraGO-1 and SatraGO-2 trials, giving patients the first at-home subcutaneous treatment option.

EST-201 and EST-101 target the genetic root cause of ADPKD, a disease affecting more than 8 million people worldwide, with trials starting 2027-2028.

FDA expands Anktiva’s bladder cancer indication, Alvotech and Advanz submit a proposed Eylea 8 mg biosimilar in Europe, and Lilly reports new remission data for Omvoh in inflammatory bowel disease.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.

The FDA expanded ImmunityBio’s Anktiva plus BCG to include patients with BCG-unresponsive non-muscle invasive bladder cancer with papillary-only tumors, extrapolating efficacy from the QUILT-3.032 carcinoma in situ cohort.

The FDA expanded Fast Track status for invikafusp alfa, Marengo Therapeutics’ TCR Vβ-targeting antibody-IL-2 fusion protein, from TMB-H colorectal cancer to TMB-H solid tumors regardless of tissue of origin.

Nathan Edwards of Arya Consulting moderates a discussion with Elisabeth Gardiner of Tevard Biosciences and Donny Xu of the RTW Institute about rare disease treatment development and navigating the FDA’s Rare Pediatric Disease Priority Review Voucher Program.

Mirikizumab (Omvoh) achieved comprehensive disease control in 27.7% of UC patients and deep remission in 20.4% with Crohn disease at 1 year.

The FDA launches a new rare disease development initiative, BioNTech’s BNT113 misses a progression-free survival endpoint, and Viking Therapeutics begins Phase III trials of its oral obesity candidate.

As ArsenalBio, TScan, Bristol Myers Squibb, and others abandon ex vivo programs for in vivo engineering, early clinical data and platform differences point to both the promise and the unresolved safety, manufacturing, and regulatory questions of generating CAR-T cells inside the body.

FDA’s rare disease framework effort will revisit evidence expectations and manufacturing review, with public input starting Oct. 28.

BNT113 plus pembrolizumab failed to significantly extend progression-free survival in a 285-patient HPV16-positive head and neck cancer trial.

Viking Therapeutics has started two Phase 3 trials of once-daily oral VK2735, a dual GLP-1/GIP receptor agonist, in adults with obesity, with and without type 2 diabetes.

Gilead will develop up to three PRO-XTEN T-cell engagers discovered by Vir Biotechnology for solid tumors in a deal worth up to $1.74 billion.

The FDA has accepted Pierre Fabre Pharmaceuticals’ resubmitted BLA for tabelecleucel, an allogeneic T-cell immunotherapy for EBV+ post-transplant lymphoproliferative disease. The FDA’s target action date is March 31, 2027.

FDA reviews a proposed Keytruda biosimilar, Roche expands its obesity pipeline with an RNAi deal, and guselkumab shows promise for fistula remission in Crohn disease.

Dr. Paz Vellanki discusses Bayesian methods, challenges in developing rare disease endpoints, and why sponsors should engage FDA early in clinical development.

Johnson & Johnson's guselkumab achieved up to 29% fistula remission in hard-to-treat Crohn disease subgroups, including prior biologic failures.

Rona's ALK7 RNAi candidate, licensed to Roche for up to $1.06 billion in milestones, aims to cut visceral fat while sparing lean mass.

FDA has accepted for review the biologics license application for FYB206, a proposed pembrolizumab biosimilar developed without a comparative phase 3 efficacy trial. A decision could come in the third quarter of 2027.

In an eight-week obese non-human primate study, lean mass made up 6% of the weight lost with iBio’s Activin E antibody IBIO-610 plus semaglutide, compared with 22% with semaglutide alone. iBio plans to begin first-in-human studies in the first half of 2027.