Clinical Trials

Regulatory Science

Ascendis Pharma TransCon PTH sustains efficacy at 3.5 years in Phase 3

Week 182 PaTHway Trial data show palopegteriparatide maintained biochemical normalisation and quality-of-life gains across multi-organ systems in adults with
Week 182 PaTHway Trial data show palopegteriparatide maintained biochemical normalisation and quality-of-life gains across multi-organ systems in adults with
Genomics

Rhythm reports positive Phase 2 setmelanotide data in PWS

Six-month interim data from 17 patients showed BMI reductions, fat mass loss and improved hyperphagia scores, reinforcing the case for Phase 3.
Six-month interim data from 17 patients showed BMI reductions, fat mass loss and improved hyperphagia scores, reinforcing the case for Phase 3.
Biotech

Shattuck Labs prices $75m public offering to fund DR3 programme

Shattuck Labs priced a $75 million public offering of shares and pre-funded warrants, with proceeds set to advance its DR3 antibody pipeline.
Shattuck Labs priced a $75 million public offering of shares and pre-funded warrants, with proceeds set to advance its DR3 antibody pipeline.
Cell & Gene Therapy

SL Bio completes SPAC merger, lists on Nasdaq as SL Science

The Taiwan-based cell therapy company closes its Horizon Space SPAC combination, debuting on Nasdaq under ticker SLBT with a $5.6bn implied valuation.
The Taiwan-based cell therapy company closes its Horizon Space SPAC combination, debuting on Nasdaq under ticker SLBT with a $5.6bn implied valuation.

Alpha Tau cleared to complete GBM trial after 100% local disease control

FDA has authorised Alpha Tau to enrol the final seven patients in its REGAIN recurrent glioblastoma trial following encouraging interim data from the first three
FDA has authorised Alpha Tau to enrol the final seven patients in its REGAIN recurrent glioblastoma trial following encouraging interim data from the first three
Regulatory Science

Belite Bio completes NDA submission for tinlarebant in Stargardt disease

Belite Bio has filed its full NDA for tinlarebant, a once-daily oral therapy that would be the first approved treatment for STGD1.
Belite Bio has filed its full NDA for tinlarebant, a once-daily oral therapy that would be the first approved treatment for STGD1.
Genomics

Cogent Biosciences presents APEX pivotal data for bezuclastinib in AdvSM

Bezuclastinib hit a 65% objective response rate in advanced systemic mastocytosis; Cogent plans to file its NDA in June 2026.
Bezuclastinib hit a 65% objective response rate in advanced systemic mastocytosis; Cogent plans to file its NDA in June 2026.
Genomics

Disc Medicine reports durable anemia responses in RALLY-MF Phase 2

DISC-0974 hit major response rates of 50–64% across transfusion subgroups in myelofibrosis, with End of Phase 2 FDA talks targeted for late 2026.
DISC-0974 hit major response rates of 50–64% across transfusion subgroups in myelofibrosis, with End of Phase 2 FDA talks targeted for late 2026.

Imviva Biotech CTD402 shows 80–86% response in R/R T-ALL at EHA2026

Allogeneic anti-CD7 CAR-T candidate CTD402 posted strong remission rates in adult and paediatric T-cell leukaemia patients across two EHA2026 poster presentations.
Allogeneic anti-CD7 CAR-T candidate CTD402 posted strong remission rates in adult and paediatric T-cell leukaemia patients across two EHA2026 poster presentations.
Genomics

Kyntra Bio roxadustat shows TI benefit in LR-MDS regardless of RS status

Post-hoc MATTERHORN data presented at EHA 2026 show roxadustat achieved transfusion independence in both RS-positive and RS-negative lower-risk MDS patients.
Post-hoc MATTERHORN data presented at EHA 2026 show roxadustat achieved transfusion independence in both RS-positive and RS-negative lower-risk MDS patients.

Lyell Immunopharma reports ronde-cel safety data at EHA 2026

Updated Phase 1/2 data in 108 LBCL patients show no Grade 3+ CRS and a 97% manufacturing success rate, supporting outpatient use.
Updated Phase 1/2 data in 108 LBCL patients show no Grade 3+ CRS and a 97% manufacturing success rate, supporting outpatient use.
Regulatory Science

Novartis Rhapsido meets Phase 3 endpoints across three CIndU subtypes

RemIND trial data presented at EAACI 2026 show remibrutinib doubled complete response rates versus placebo in symptomatic dermographism, cold urticaria and cholinergic
RemIND trial data presented at EAACI 2026 show remibrutinib doubled complete response rates versus placebo in symptomatic dermographism, cold urticaria and cholinergic
Regulatory Science

Sanofi wins FDA approval for Tzield in stage 3 type 1 diabetes

The accelerated approval covers children aged eight to 17 recently diagnosed with stage 3 T1D, making Tzield the first disease-modifying therapy in this setting.
The accelerated approval covers children aged eight to 17 recently diagnosed with stage 3 T1D, making Tzield the first disease-modifying therapy in this setting.
Regulatory Science

Rafael Holdings completes Phase 3 NPC trial, targets NDA in H2 2026

Rafael Holdings hit last-patient-last-visit in its pivotal TransportNPC trial and expects to file an NDA for Trappsol Cyclo in the second half
Rafael Holdings hit last-patient-last-visit in its pivotal TransportNPC trial and expects to file an NDA for Trappsol Cyclo in the second half
Diagnostics

Roche gains FDA nod for first PTEN companion diagnostic

The VENTANA PTEN (SP218) RxDx Assay is the first IHC test approved to identify PTEN-deficient prostate cancer patients eligible for capivasertib.
The VENTANA PTEN (SP218) RxDx Assay is the first IHC test approved to identify PTEN-deficient prostate cancer patients eligible for capivasertib.
Diagnostics

Syndax revumenib shows broad activity across leukaemia studies at EHA

Five datasets presented at EHA 2026 show revumenib achieving high remission and MRD-negativity rates across multiple acute leukaemia subtypes and treatment settings.
Five datasets presented at EHA 2026 show revumenib achieving high remission and MRD-negativity rates across multiple acute leukaemia subtypes and treatment settings.
Regulatory Science

Ascendis Pharma reports five-year Phase 2 data for TransCon PTH

Palopegteriparatide sustained multi-organ benefits over five years in hypoparathyroidism, with 82% of patients meeting the multi-component response endpoint.
Palopegteriparatide sustained multi-organ benefits over five years in hypoparathyroidism, with 82% of patients meeting the multi-component response endpoint.
Diagnostics

Caribou Biosciences CB-011 shows 83% CR rate in r/r myeloma at EHA

Updated CaMMouflage phase 1 data show deep, durable responses from a single dose in heavily pretreated, BCMA-naïve multiple myeloma patients.
Updated CaMMouflage phase 1 data show deep, durable responses from a single dose in heavily pretreated, BCMA-naïve multiple myeloma patients.
Cell & Gene Therapy

Climb Bio posts Phase 1b ITP data for budoprutug at EHA 2026

Early data from 15 heavily pretreated patients show robust B-cell depletion and durable platelet responses, with no serious adverse events reported.
Early data from 15 heavily pretreated patients show robust B-cell depletion and durable platelet responses, with no serious adverse events reported.
Diagnostics

Roche's Tecentriq wins FDA Priority Review in stage III colon cancer

The FDA accepted Roche's sBLA for Tecentriq plus chemotherapy in dMMR/MSI-H stage III colon cancer, with a decision expected by 9 October
The FDA accepted Roche's sBLA for Tecentriq plus chemotherapy in dMMR/MSI-H stage III colon cancer, with a decision expected by 9 October
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