Clinical Trials

Regulatory Science

Attralus wins FDA orphan drug status for AL amyloidosis candidate AT-02

Zamubafusp alfa now holds four orphan designations globally as Attralus advances the pan-amyloid removal antibody through a Phase 2 trial.
Zamubafusp alfa now holds four orphan designations globally as Attralus advances the pan-amyloid removal antibody through a Phase 2 trial.
Genomics

Biogen's salanersen wins FDA Breakthrough Therapy tag for SMA

The designation is backed by Phase 1b data showing motor function gains in children who had a suboptimal response to prior gene therapy.
The designation is backed by Phase 1b data showing motor function gains in children who had a suboptimal response to prior gene therapy.
Pharmaceuticals
Regulatory Science

GH Research publishes Phase 2a PPD data for inhaled GH001

All ten patients in GH Research's open-label trial achieved remission within two hours of a single inhaled dose of mebufotenin, the company reports.
All ten patients in GH Research's open-label trial achieved remission within two hours of a single inhaled dose of mebufotenin, the company reports.
Regulatory Science

Gilead and Lakefront close Ouro Medicines deal for $1.675bn

The acquisition adds gamgertamig, a BCMAxCD3 T cell engager, to Gilead's inflammation portfolio; registrational studies are expected as early as 2027.
The acquisition adds gamgertamig, a BCMAxCD3 T cell engager, to Gilead's inflammation portfolio; registrational studies are expected as early as 2027.
Regulatory Science

Novartis ALIGN data show Vanrafia slows IgAN kidney decline by 34%

Final 2.5-year Phase III results, published in The Lancet, back a traditional approval filing for atrasentan in IgA nephropathy later this year.
Final 2.5-year Phase III results, published in The Lancet, back a traditional approval filing for atrasentan in IgA nephropathy later this year.
Biotech

Ona Therapeutics raises $86.6m Series B to bring ADCs into clinic

The Barcelona biotech closed an oversubscribed round to advance its lead ADC, ONA-255, toward clinical proof of concept in breast cancer.
The Barcelona biotech closed an oversubscribed round to advance its lead ADC, ONA-255, toward clinical proof of concept in breast cancer.
Diagnostics

Spectral Medical PMX shows sustained mortality benefit at 12 months

Tigris Phase 3 data show PMX reduced 12-month mortality to 52.8% versus 66.7% for standard care, with a 95.9% Bayesian probability
Tigris Phase 3 data show PMX reduced 12-month mortality to 52.8% versus 66.7% for standard care, with a 95.9% Bayesian probability
Regulatory Science

Iovance wins TGA approval for lifileucel in advanced melanoma

Amtagvi becomes the first T cell therapy approved for a solid tumour in Australia, the country with the world's highest melanoma incidence.
Amtagvi becomes the first T cell therapy approved for a solid tumour in Australia, the country with the world's highest melanoma incidence.
Regulatory Science

Novartis Cosentyx Phase III PMR data published in NEJM

Phase III REPLENISH trial showed secukinumab achieved sustained remission in twice as many PMR patients as placebo at 52 weeks, with significant steroid sparing.
Phase III REPLENISH trial showed secukinumab achieved sustained remission in twice as many PMR patients as placebo at 52 weeks, with significant steroid sparing.
Regulatory Science

Abivax Phase 3 data show obefazimod hits all UC endpoints

Both doses of Abivax's oral miR-124 enhancer achieved placebo-adjusted remission rates above 39% at 44 weeks, with an NDA filing planned for
Both doses of Abivax's oral miR-124 enhancer achieved placebo-adjusted remission rates above 39% at 44 weeks, with an NDA filing planned for
Genomics

Artios doses first patient in Phase 2 POLKA trial of ART6043

Artios Pharma has started its randomised Phase 2 POLKA study of Pol-theta inhibitor ART6043 plus olaparib in gBRCA-mutated HER2-negative breast cancer.
Artios Pharma has started its randomised Phase 2 POLKA study of Pol-theta inhibitor ART6043 plus olaparib in gBRCA-mutated HER2-negative breast cancer.
Diagnostics

BeyondSpring plinabulin triple shows durable NSCLC benefit

Phase 2 data presented at ASCO 2026 show a 58% two-year survival rate in metastatic NSCLC patients with acquired resistance to checkpoint inhibitors.
Phase 2 data presented at ASCO 2026 show a 58% two-year survival rate in metastatic NSCLC patients with acquired resistance to checkpoint inhibitors.
Regulatory Science

Candel prostate therapy shows 30% DFS gain in Lancet Oncology

Candel Therapeutics' aglatimagene cut disease recurrence by 30% versus placebo in a 745-patient Phase 3 trial, with a BLA filing planned for Q4 2026.
Candel Therapeutics' aglatimagene cut disease recurrence by 30% versus placebo in a 745-patient Phase 3 trial, with a BLA filing planned for Q4 2026.
Diagnostics
Genomics

Cardiff Oncology: onvansertib hits 72% ORR in RAS-mutated mCRC

Phase 2 CRDF-004 data presented at ASCO 2026 support a registrational Phase 3 trial after FDA End-of-Phase 2 alignment.
Phase 2 CRDF-004 data presented at ASCO 2026 support a registrational Phase 3 trial after FDA End-of-Phase 2 alignment.
Diagnostics

CG Oncology: cretostimogene combo shows high CR in NMIBC

Phase 2 Cohort CX data show 92.3% complete response and no Grade 3 adverse events for cretostimogene plus gemcitabine in high-risk bladder cancer.
Phase 2 Cohort CX data show 92.3% complete response and no Grade 3 adverse events for cretostimogene plus gemcitabine in high-risk bladder cancer.
Diagnostics
Regulatory Science

Disc Medicine RALLY-MF data show broad anemia responses in MF

Updated Phase 2 data for DISC-0974 show durable anemia responses across all patient subgroups, with an FDA end-of-phase meeting expected by year
Updated Phase 2 data for DISC-0974 show durable anemia responses across all patient subgroups, with an FDA end-of-phase meeting expected by year
Biotech

JJP Biologics: nebaprubart shows early efficacy in rare LAD trial

Interim Phase 1b data show rapid blister reduction and dapsone tapering in Linear IgA Disease, supporting a rheumatoid arthritis trial in Q3 2026.
Interim Phase 1b data show rapid blister reduction and dapsone tapering in Linear IgA Disease, supporting a rheumatoid arthritis trial in Q3 2026.
Regulatory Science

J&J nipocalimab meets Phasea 2 primary endpoint in lupus study

Phase 2 JASMINE data show nipocalimab reduced SLE disease activity through 52 weeks, with stronger responses in autoantibody-positive patients.
Phase 2 JASMINE data show nipocalimab reduced SLE disease activity through 52 weeks, with stronger responses in autoantibody-positive patients.
Genomics

Ziftomenib triplet shows 87% ORR in venetoclax-naive R/R AML

Blood publication of KOMET-007 data shows deep, durable responses for ziftomenib plus venetoclax/azacitidine in NPM1-mutated relapsed/refractory AML.
Blood publication of KOMET-007 data shows deep, durable responses for ziftomenib plus venetoclax/azacitidine in NPM1-mutated relapsed/refractory AML.
Cell & Gene Therapy
Biotech

Legend Biotech reports 100% ORR in Phase 1 in vivo CAR-T NHL trial

LB2501, a single-infusion in vivo CAR-T candidate, achieved 100% objective response in six relapsed/refractory NHL patients with no dose-limiting toxicities.
LB2501, a single-infusion in vivo CAR-T candidate, achieved 100% objective response in six relapsed/refractory NHL patients with no dose-limiting toxicities.
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