Clinical Trials

Biologics
Pharmaceuticals

Biogen publishes Phase 3 EMPAVELI paediatric data in CJASN

Adolescents with C3G or IC-MPGN achieved a 75% relative reduction in proteinuria on pegcetacoplan versus placebo in the VALIANT subgroup analysis.
Adolescents with C3G or IC-MPGN achieved a 75% relative reduction in proteinuria on pegcetacoplan versus placebo in the VALIANT subgroup analysis.
Biologics
Cell & Gene Therapy

SNIPR enrolls first Phase 2 patient in CRISPR phage trial

SNIPR has dosed its first Phase 2 patient with SNIPR001, expanding the E. coli bloodstream infection trial to 66 patients across ten sites including MSK
SNIPR has dosed its first Phase 2 patient with SNIPR001, expanding the E. coli bloodstream infection trial to 66 patients across ten sites including MSK
Diagnostics
Pharmaceuticals

Sol-Gel hits LPLV milestone in Phase 3 Gorlin syndrome trial

Sol-Gel Technologies has completed patient follow-up in its Phase 3 patidegib gel study, with top-line results expected in late November 2026.
Sol-Gel Technologies has completed patient follow-up in its Phase 3 patidegib gel study, with top-line results expected in late November 2026.
Cell & Gene Therapy
Pharmaceuticals
Regulatory Science
Biologics

Ultragenyx wins FDA approval for FAYUVI in Sanfilippo syndrome Type A

FAYUVI becomes the first approved treatment for MPS IIIA, backed by eight years of follow-up data showing significant cognitive benefit over natural history.
FAYUVI becomes the first approved treatment for MPS IIIA, backed by eight years of follow-up data showing significant cognitive benefit over natural history.
Diagnostics
Digital Health

uMETHOD Health publishes real-world study on AI dementia care plans

A retrospective study of 345 paired tests found 83.5% of patients on RestoreU showed stable or improved cognition over 14 months.
A retrospective study of 345 paired tests found 83.5% of patients on RestoreU showed stable or improved cognition over 14 months.
Medical Devices

Wandercraft launches Eve self-balancing exoskeleton for home use

Wandercraft's FDA-cleared Eve exoskeleton offers hands-free upright mobility to wheelchair users with spinal cord injuries at any neurological level.
Wandercraft's FDA-cleared Eve exoskeleton offers hands-free upright mobility to wheelchair users with spinal cord injuries at any neurological level.
Pharmaceuticals
Biologics

Avacta reports clinical proof of mechanism for AVA6103 PDC in FOCUS-01

Avacta's next-generation pre|CISION peptide-drug conjugate showed a clean safety profile and matched preclinical PK predictions across three dose levels in solid
Avacta's next-generation pre|CISION peptide-drug conjugate showed a clean safety profile and matched preclinical PK predictions across three dose levels in solid
Medical Devices
Digital Health

Flow Neuroscience launches FDA-cleared at-home tDCS device FL-100

Flow Neuroscience's FL-100 headset is now available by US prescription, becoming the first FDA-approved at-home tDCS treatment for moderate to severe
Flow Neuroscience's FL-100 headset is now available by US prescription, becoming the first FDA-approved at-home tDCS treatment for moderate to severe
Pharmaceuticals

Remedy Plan advances RPT1G to Cohort 2 in AML/MDS Phase 1 trial

The Gaithersburg biotech also secured a $30m Series A extension and won FDA IND acceptance for RPT1G in solid tumours.
The Gaithersburg biotech also secured a $30m Series A extension and won FDA IND acceptance for RPT1G in solid tumours.
Biologics
Pharmaceuticals

Roche's Lunsumio plus lenalidomide meets Phase III PFS endpoint in FL

Roche's CELESTIMO study showed mosunetuzumab plus lenalidomide significantly improved progression-free survival in relapsed or refractory follicular lymphoma.
Roche's CELESTIMO study showed mosunetuzumab plus lenalidomide significantly improved progression-free survival in relapsed or refractory follicular lymphoma.
Digital Health
Medical Devices

NightWare wearable cuts nightmare distress in first responder study

A peer-reviewed pilot in Frontiers in Sleep found NightWare halved nightmare distress in San Francisco police and fire personnel with PTSD over 60 days.
A peer-reviewed pilot in Frontiers in Sleep found NightWare halved nightmare distress in San Francisco police and fire personnel with PTSD over 60 days.
Cell & Gene Therapy
Biologics

Tempest Therapeutics options Senlang lentiviral in vivo CAR-T platform

Tempest secures an exclusive option on Senlang's CD7-targeted lentiviral vector platform, adding a Phase 1 multiple myeloma programme to its in vivo CAR
Tempest secures an exclusive option on Senlang's CD7-targeted lentiviral vector platform, adding a Phase 1 multiple myeloma programme to its in vivo CAR
Genomics
Diagnostics

Bionano OGM detects hidden genomic risk in MDS and AML studies

Three peer-reviewed studies across the US, Finland, and Spain show optical genome mapping uncovers clinically actionable variants missed by standard karyotyping.
Three peer-reviewed studies across the US, Finland, and Spain show optical genome mapping uncovers clinically actionable variants missed by standard karyotyping.
Pharmaceuticals
Diagnostics

BioVie's bezisterim hits subgroup endpoints in Phase 2 Long COVID trial

BioVie reported statistically significant improvements in Long COVID patients with high symptom burden, with Phase 3 now planned for bezisterim.
BioVie reported statistically significant improvements in Long COVID patients with high symptom burden, with Phase 3 now planned for bezisterim.
Pharmaceuticals
Diagnostics

BlossomHill's BH-30643 hits 45% response rate in C797S NSCLC

Updated SOLARA Phase 1/2 data show an 88% disease control rate in a resistance population with no approved targeted therapies.
Updated SOLARA Phase 1/2 data show an 88% disease control rate in a resistance population with no approved targeted therapies.
Biologics
Pharmaceuticals

Connect Biopharma's rademikibart cuts asthma treatment failure by 66%

Phase 2 data show rademikibart reduced treatment failure and improved lung function in acute asthma, with COPD readout and FDA engagement planned.
Phase 2 data show rademikibart reduced treatment failure and improved lung function in acute asthma, with COPD readout and FDA engagement planned.
Pharmaceuticals
Diagnostics

Dogwood Therapeutics completes enrolment in Halneuron CINP Phase 2b

The Atlanta-based biotech enrolled 239 patients at the top of its target range, with topline data expected in November 2026.
The Atlanta-based biotech enrolled 239 patients at the top of its target range, with topline data expected in November 2026.
Pharmaceuticals
Regulatory Science

Elevar Therapeutics files EMA application for lirafugratinib in CCA

Elevar has submitted a marketing authorisation application to the EMA for its selective FGFR2 inhibitor, backed by a 46.5% response rate in second-line
Elevar has submitted a marketing authorisation application to the EMA for its selective FGFR2 inhibitor, backed by a 46.5% response rate in second-line
Pharmaceuticals
Biologics

ENA Respiratory begins Part B dosing in INNA-051 Phase IIa study

The Melbourne biotech has started dosing up to 900 participants in Part B of its community study of the virus-agnostic nasal spray INNA-051.
The Melbourne biotech has started dosing up to 900 participants in Part B of its community study of the virus-agnostic nasal spray INNA-051.
Pharmaceuticals

Kestrel Therapeutics wins FDA Fast Track for pan-KRAS inhibitor KST-6051

Kestrel's oral pan-KRAS inhibitor enters Phase 1 with Fast Track status, targeting KRAS-mutant solid tumours across PDAC, CRC and NSCLC.
Kestrel's oral pan-KRAS inhibitor enters Phase 1 with Fast Track status, targeting KRAS-mutant solid tumours across PDAC, CRC and NSCLC.
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