Biopharma · FDA Approvals

Castle Biosciences: ACG 2026 data show TissueCypher identifies elevated BE progression risk beyond pathology

New data from three studies (two case reports, one multi-center cohort) presented at ACG 2026 demonstrate that TissueCypher provides objective molecular risk stratification (e.g., 5-year progression probabilities of 62% and 47% in high-risk cases) that changes management in 96% of patients compared to histopathology or PIB score alone, supporting earlier intervention or adjusted surveillance intervals for Barrett's Esophagus patients.

JNJ TREMFYA (guselkumab) shows consistent perianal fistula remission in Crohn's subgroups at ACG 2026

New prespecified analyses from the Phase 3 FUZION study demonstrated consistent combined fistula remission rates with TREMFYA versus placebo across difficult-to-treat subgroups including prior biologic exposure, proctitis, and higher luminal disease activity. These data, presented at ACG 2026, build on the primary Week 24 results where TREMFYA achieved up to 28% remission versus 10% for placebo.

Savara to Present Molgramostim IMPALA-2 and IMPALA Data at CHEST 2026

Savara will present new analysis from the Phase 2/3 IMPALA trial and encore long-term efficacy and safety data from the Phase 3 IMPALA-2 open-label period of molgramostim in aPAP at CHEST 2026 (October 18-21). The company will also host a Learning Theatre on aPAP with a key opinion leader and patients.

Bausch Health/Salix to Present Cirrhosis Clinical Pathway Pilot Study at ACG 2026

A six-month pilot study of a standardized cirrhosis clinical pathway in five community gastroenterology practices showed modest improvements in adherence to five guideline-based performance indicators. The results will be presented as a poster at the American College of Gastroenterology Annual Scientific Meeting taking place October 9-14, 2026.

REGENXBIO to Host Investor Event on Sura-vec in Wet AMD on Oct 20, 2026

REGENXBIO will host an investor event on October 20, 2026 featuring clinical program overview, KOL perspectives on ATMOSPHERE and ASCENT pivotal trials, and commercial readiness for sura-vec in wet AMD. This is ahead of anticipated topline pivotal data readout in 4Q 2026.

Racura Oncology clears RC220 dose escalation to 100 mg/m2 in HARNESS-1 Phase 1 trial

The Safety Review Committee cleared escalation of RC220 from 50 mg/m2 to the next planned dose level of 100 mg/m2 in combination with osimertinib after no dose-limiting toxicities were observed in Cohort 1. Pre-screening is underway to enroll the single patient in Cohort 2 at the higher dose. This is a routine Phase 1 dose-escalation step with no new efficacy data reported.

Stoke Therapeutics Presents STK-002 ADOA Data at AAO 2026

Stoke presented preclinical, natural history (FALCON), and early clinical data on STK-002 at the AAO 2026 meeting. The ongoing Phase 1 OSPREY trial has completed dosing in the first two cohorts, with the third cohort expected to complete by end-2026 and initial safety/efficacy data from later cohorts guided for H1 2027.

BMY Announces Positive Phase 3 ACTION-1 Topline for RYZ101 in GEP-NETs

The Phase 3 ACTION-1 trial of RYZ101 (225Ac-DOTATATE) versus investigator's choice of standard of care met its primary endpoint of statistically significant and clinically meaningful improvement in progression-free survival by blinded independent central review in patients with SSTR+ GEP-NETs who progressed after Lu177-based therapy. Safety was consistent with the known profile. This is the first Phase 3 data for an alpha-isotope (225Ac) targeted radiopharmaceutical therapy; RayzeBio plans to engage FDA on next steps toward submission.

BMY Admilparant Phase 3 ALOFT-IPF Trial Meets Primary Endpoint in IPF

The Phase 3 ALOFT-IPF trial met its primary endpoint of statistically significant improvement in absolute change in FVC from baseline to Week 52 at the 60 mg twice-daily dose versus placebo; the 120 mg dose slowed FVC decline but did not reach statistical significance. Admilparant showed a favorable safety profile with low adverse-event discontinuations comparable to placebo. BMS will evaluate full data, engage regulators for planned submissions, and present detailed results at a future medical meeting; ALOFT-PPF topline remains expected in early 2027.

AbbVie presents new Phase 3 AFFIRM and long-term FORTIFY data for SKYRIZI in Crohn's disease at ACG 2026

AbbVie presented new Phase 3 AFFIRM results for subcutaneous risankizumab showing higher clinical remission and endoscopic response rates versus placebo in advanced therapy-naïve Crohn's disease patients. Additional open-label extension data from FORTIFY and SEQUENCE demonstrated sustained endoscopic healing up to 144-148 weeks, including in fistulizing disease and across disease locations. Real-world evidence for RINVOQ in advanced Crohn's disease was also highlighted.

Celltrion to Present ZYMFENTRA Analyses at ACG 2026

Celltrion will present new post-hoc and late-breaking analyses of ZYMFENTRA (infliximab-dyyb) at the ACG 2026 meeting on Oct 13-14. The data compare SC vs IV infliximab in ileum-dominant Crohn's disease and evaluate outcomes after IV-to-SC switch regardless of prior ADA status in CD and UC.

Mirum to Report Phase 3 EXPAND Topline Results for LIVMARLI (maralixibat)

Mirum will host an investor call on October 12, 2026 to share topline results from the Phase 3 EXPAND study of LIVMARLI (maralixibat) in ultra-rare cholestatic liver diseases. This is the first disclosure of the primary efficacy and safety data from the randomized, placebo-controlled trial evaluating cholestatic pruritus in patients 6 months and older, including those with biliary atresia.

Merck Presents BRUNELLO Phase 2b/3 One-Year Remigromig Results at AAO 2026

Merck presented one-year topline data from the pivotal Phase 2b/3 BRUNELLO trial of remigromig (MK-3000) in DME at the AAO 2026 meeting. Both doses met the primary endpoint of non-inferiority to ranibizumab on mean BCVA change at one year but did not show superiority on key secondary endpoints; higher rates of PDR and treatment discontinuations were observed with remigromig. The results support continued evaluation in the ongoing BAROLO Phase 3 study and discussions with regulators.

BioMarin presents new VOXZOGO data at ASBMR 2026

BioMarin will present 13 studies at the ASBMR 2026 Annual Meeting, including new post-hoc imaging results on VOXZOGO's effect on foramen magnum and craniocervical junction growth in children (including infants) with achondroplasia, plus data on bone strength, hypochondroplasia Phase 3 results, and investigational BMN 333. The company recently submitted an sNDA for VOXZOGO in hypochondroplasia with a potential 2027 launch if approved.

Johnson & Johnson presents 2-year ICOTYDE (icotrokinra) Phase 3 data at Fall Clinical Dermatology 2026

Johnson & Johnson presented new two-year Phase 3 ICONIC-LEAD results for ICOTYDE (icotrokinra) at the Fall Clinical Dermatology conference. The data show sustained high rates of skin clearance (PASI 90 ≥72%, IGA 0/1 ≥70%) and symptom improvement through Week 112 with a consistent safety profile in adults and adolescents with plaque psoriasis.

Lilly presents Taltz + Zepbound biomarker data from TOGETHER-PsO at Fall Clinical 2026

Exploratory biomarker data from the Phase 3b TOGETHER-PsO trial were presented at the 2026 Fall Clinical Dermatology Conference. Concomitant Taltz and Zepbound produced broader changes in psoriasis, immune, and metabolic biomarkers versus Taltz alone as early as Week 12.

Vertex Presents ALYFTREK Pancreatic Function Recovery Data at NACFC

Vertex presented new interim data from the open-label extension study of ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) in children 2-5 years old with cystic fibrosis. The data show recovery of exocrine pancreatic function in some children, enabling discontinuation of pancreatic enzyme replacement therapy (PERT), which was previously considered irreversible. Additional real-world and clinical evidence on CFTR modulators was also presented at the North American Cystic Fibrosis Conference.

relacorilant · NCT03604198 registry update

countries: {'total_sites': 44, 'us_sites': 25} → {'total_sites': 48, 'us_sites': 25}

gedatolisib · NCT06757634 registry update

countries: {'total_sites': 203, 'us_sites': 22} → {'total_sites': 239, 'us_sites': 30}

atirmociclib · NCT03424005 registry update

countries: {'total_sites': 53, 'us_sites': 24} → {'total_sites': 55, 'us_sites': 26}