Disease

Genomics

Agios RISE UP Phase 3 data show mitapivat cuts transfusion burden in SCD

New analyses from Agios's EHA 2026 plenary presentation show mitapivat reduced transfusion burden by 41% and improved pain and fatigue in sickle cell responders.
New analyses from Agios's EHA 2026 plenary presentation show mitapivat reduced transfusion burden by 41% and improved pain and fatigue in sickle cell responders.
Synthetic Biology

Genmab epcoritamab shows high response rates in elderly DLBCL patients

Phase 2 data presented at EHA 2026 show epcoritamab monotherapy and a combination regimen achieved strong complete response rates in first-line elderly DLBCL patients.
Phase 2 data presented at EHA 2026 show epcoritamab monotherapy and a combination regimen achieved strong complete response rates in first-line elderly DLBCL patients.

Intellia Phase 3 HAELO data show 87% HAE attack reduction

Phase 3 results for lonvo-z, published simultaneously in the NEJM, showed 62% of patients attack-free over six months with a single dose.
Phase 3 results for lonvo-z, published simultaneously in the NEJM, showed 62% of patients attack-free over six months with a single dose.
Regulatory Science

MBX Biosciences: one-year canvuparatide data in hypoparathyroidism

One-year open-label extension data show a 57% responder rate for canvuparatide, with Phase 3 on track to start in Q3 2026.
One-year open-label extension data show a 57% responder rate for canvuparatide, with Phase 3 on track to start in Q3 2026.
Pharmaceuticals

J&J talquetamab plus daratumumab cuts RRMM death risk by up to 53%

Phase 3 MonumenTAL-3 data show talquetamab combinations beat standard care on PFS and OS in earlier-line relapsed myeloma.
Phase 3 MonumenTAL-3 data show talquetamab combinations beat standard care on PFS and OS in earlier-line relapsed myeloma.
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.
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.
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

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.
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