ALETH / LIFESCI-BRIEF / 2026-09-04 / NOVARTIS HALTS CAR-T TRIALS AFTER THREE DEATHSThe Aleth Briefs link to the sources behind the stories and show how the week unfolded.
The week in five lines:
Novartis halted autoimmune CAR-T trials after three patient deaths.
The White House signed MFN pricing deals with nine more drugmakers.
Lilly agreed to buy Merida Biosciences for up to $2.9bn.
Ionis won the first FDA approval for Alexander disease.
Ivonescimab beat Keytruda on overall survival in a China phase 3 trial.
Browse by day:
Weekend catch-up: Wainua · Myqorzo · Mounjaro · Mimrylo · BioNTech
Monday: Jazz Ziihera · Lilly-Merida · Repatha · US MFN · SEC-FDA · CAR-T deaths
Tuesday: UK spinout · Vifor · MHRA · ARPA-H · GSK · Roche-Simcere · Remibrutinib
Wednesday: Novartis-Alteogen · uniQure · FDA sites · Ultragenyx · Ivonescimab
Thursday: GSK-HUTCHMED · AbbVie · eGenesis · Ionis
Weekend catch-up
Full data showed why AstraZeneca and Ionis’ Wainua failed in ATTR cardiomyopathy.
In CARDIO-TTRansform, presented at the European Society of Cardiology congress on Friday, Wainua (eplontersen) did not reduce cardiovascular death and recurrent cardiovascular events in 1,432 patients with transthyretin amyloid cardiomyopathy (rate ratio 0.89, p=0.277). The 57% already receiving a stabiliser showed no added primary-endpoint benefit, while patients not receiving one had fewer events.
Eplontersen trial did not meet its primary endpoint in ATTR cardiomyopathy · ESC
Cytokinetics’ Myqorzo met both primary endpoints in non-obstructive hypertrophic cardiomyopathy.
In the phase 3 ACACIA-HCM trial, Myqorzo (aficamten) improved symptom scores and peak exercise capacity at week 36 against placebo. Cytokinetics plans a supplemental FDA filing in Q4 2026.
Positive ACACIA-HCM results presented at ESC · Cytokinetics
The FDA approved Lilly’s Mounjaro to reduce cardiovascular risk in adults with type 2 diabetes.
The label covers heart attack, stroke and cardiovascular death in adults at high cardiovascular risk. In SURPASS-CVOT, Mounjaro (tirzepatide) was non-inferior to Trulicity (dulaglutide) for these events, with a hazard ratio of 0.92. Superiority was not established.
FDA approves Mounjaro to reduce CV risk in adults with type 2 diabetes · Eli Lilly
The FDA approved Takeda’s Mimrylo, the first hepcidin mimetic for rare blood disorder polycythaemia vera.
In the phase 3 VERIFY trial, 76.9% of patients on Mimrylo (rusfertide) needed no phlebotomy over 32 weeks, against 32.9% on placebo.
FDA approves first drug of its kind for polycythaemia vera · FDA
FDA approves MIMRYLO for polycythaemia vera · Takeda
BioNTech and Genentech ended a phase 2 trial of their personalised colorectal cancer vaccine.
In resected stage II-III colorectal cancer positive for circulating tumour DNA, autogene cevumeran was being tested as monotherapy. An independent monitoring board found a numerical imbalance in overall survival and judged further continuation unlikely to change the efficacy outcome. It reported no new safety signal. A phase 2 trial in pancreatic cancer, pairing the vaccine with checkpoint inhibition and chemotherapy, continues. BioNTech shares fell 8.4%, wiping $2.6bn off its market value, reflecting concerns about personalised mRNA cancer vaccines as monotherapy.
Update on phase 2 of autogene cevumeran in resected colorectal cancer · BioNTech
Monday 31 August
Jazz’s Ziihera produced further positive overall survival data in HER2-positive gastro-oesophageal cancer.
At the second interim analysis of phase 3 HERIZON-GEA-01, Ziihera (zanidatamab) plus chemotherapy showed a further improvement in overall survival versus Herceptin (trastuzumab) plus chemotherapy in first-line disease. The FDA had approved two Ziihera-based first-line regimens just six days earlier, on 25 August.
Ziihera demonstrates significant OS benefit vs trastuzumab · Jazz Pharmaceuticals
FDA approves Ziihera for HER2-positive gastro-oesophageal cancer · FDA
Lilly agreed to buy Merida Biosciences for up to $2.9bn.
Merida engineers biologics that selectively degrade the autoantibodies driving disease rather than suppressing the immune system. Its lead drug, MER511, is in phase 1 for Graves’ disease and thyroid eye disease. The all-cash price includes contingent milestones, and the deal is expected to close in Q4 2026.
Lilly to acquire Merida Biosciences for autoimmune and allergic diseases · Eli Lilly
A prespecified analysis found Amgen’s Repatha cut mortality by 20% in high-risk patients without a prior heart attack or stroke.
In the phase 3 VESALIUS-CV trial in more than 12,000 patients, Repatha (evolocumab) added to lipid-lowering treatment reduced the risk of death by 20%. The benefit appeared after about 1.5 years.
Repatha reduces risk of death in patients at risk of 1st heart attack/stroke · Amgen
Trump signed MFN pricing deals with nine more drugmakers.
The White House named Alcon, Astellas, BeOne Medicines, BridgeBio, CSL, Kyowa Kirin, Sun Pharma, Teva and UCB. Each agreed to give state Medicaid programmes most-favoured-nation prices and guarantee MFN prices on new medicines, taking the total to 26 companies, covering 89% of the branded US drug market. Incyte separately disclosed an agreement with the CMS under which Medicaid gets Jakafi (ruxolitinib) at prices aligned with selected international markets. Incyte expects to escape the proposed GUARD and GLOBE pricing models.
Trump announces deals with nine additional pharmas · The White House
Incyte Corporation Form 8-K, 31 August 2026 · SEC
The SEC and FDA agreed to share information on drug-related market disclosures.
Their memorandum of understanding sets protocols for exchanging information relevant to both agencies. SEC chairman Paul Atkins said FDA-related company disclosures “have a significant impact on our markets”.
SEC & FDA announce MOU to bolster cooperation and ensure market integrity · SEC
Novartis halted autoimmune CAR-T trials after three patient deaths.
Novartis suspended trials of its CAR-T therapy rap-cel in lupus, myasthenia gravis, multiple sclerosis and other autoimmune conditions on 24 August after three cases of immune effector cell-associated haemophagocytic syndrome resulted in “fatal outcomes”. Its cancer trials continue. Bristol Myers Squibb also voluntarily paused enrolment in autoimmune trials of its CAR-T therapy zola-cel, “out of an abundance of caution”, after detecting “transient and reversible inflammatory events”. Both companies confirmed the suspensions directly to journalists on 31 August.
Novartis, BMS pause autoimmune CAR-T trials on safety concerns · BioPharma Dive
Novartis halts CAR-T trials after three deaths; BMS pauses studies · Fierce Biotech
Tuesday 1 September
UK ministers are considering an exit tax on university spinouts that move abroad, The Times reported.
The proposed levy would deter companies built with taxpayer money from taking intellectual property and jobs overseas. Minister Chris McDonald separately said British discoveries are developed abroad because start-ups cannot find enough investment or lab space at home.
Ministers plot exit tax for university spinouts moving abroad · The Times
UK science breakthroughs are being exploited overseas, minister says · The Times
The UK’s MHRA suspended Avacopan Vifor for new patients after doubts over its pivotal trial data.
The Medicines and Healthcare products Regulatory Agency found the benefit-risk balance of Avacopan Vifor, previously Tavneos, no longer positive after information raised concerns about the integrity and reliability of the study behind its approval. Existing patients get a six-month managed withdrawal, and the MHRA intends to revoke the licence on 1 March 2027.
MHRA concludes Avacopan Vifor review reassessment of benefit-risk · GOV.UK
The UK government proposed powers for the MHRA to license medical devices directly.
One of three amendments tabled to the Health Bill would create the legal basis for a future regime under which devices sold in Great Britain through a domestic route could require an MHRA licence instead of third-party conformity assessment. Any regime would follow consultation, and UKCA marking remains in place in the meantime.
MHRA regulatory reform amendments tabled in Government’s Health Bill · GOV.UK
ARPA-H committed up to $224m to AI for rare diseases and on-demand RNA medicine manufacturing.
The US agency announced awards under two programmes. RAPID will invest up to $98.5m over 4.5 years in AI tools and data resources for earlier rare disease diagnosis and drug development. GIVE will provide up to $125m to develop automated, distributed manufacturing systems capable of producing individualised RNA medicines on demand, with integrated quality control.
Awards to advance AI for rare disease diagnosis, discovery and treatment · ARPA-H
Teams selected to enable made-to-order genetic medicine manufacturing · ARPA-H
GSK plans to start a phase 3 trial of its mRNA flu vaccine this month.
Phase 2 data presented at the OPTIONS XIII flu conference showed higher immune responses against all tested strains than licensed standard- and high-dose vaccines. It will be the first phase 3 trial of an mRNA flu vaccine targeting both haemagglutinin and neuraminidase.
mRNA seasonal flu vaccine advances to phase III after positive phase II data · GSK
Roche licensed Simcere Zaiming’s trispecific T-cell engager for $75m upfront and up to $1.5bn.
SIM0660 is a preclinical CD79a/CD19/CD3 T-cell engager designed for B-cell malignancies and autoimmune diseases. Roche gets exclusive global development, manufacturing and commercial rights, with Simcere also eligible for tiered royalties.
Exclusive license agreement with Roche for SIM0660 global development · Simcere
Novartis’ remibrutinib beat teriflunomide in two phase 3 multiple sclerosis trials with no liver safety signal.
In REMODEL-1 and -2, the oral Bruton’s tyrosine kinase inhibitor reduced annualised relapse rates and brain lesions against teriflunomide in relapsing multiple sclerosis. Novartis plans regulatory submissions worldwide.
Remibrutinib reduces relapse rates, & shows favourable safety in phase III · Novartis
Wednesday 2 September
Novartis signed an option and licence deal with Alteogen worth up to $3.2bn.
The agreement gives Novartis options to use Alteogen’s ALT-B4 enzyme to convert several intravenous biologics into subcutaneous injections. It is Alteogen’s fourth licensing deal this year, reinforcing the Korean company’s position as a competitor to Halozyme in subcutaneous drug delivery. The maximum value includes contingent option payments, milestones and royalties, with no upfront payment disclosed.
Option & license agreement with Novartis for Hybrozyme SC products · Alteogen
uniQure filed its Huntington’s disease gene therapy with the FDA and the MHRA.
It seeks US accelerated approval of AMT-130 (ifezuntirgene inilparvovec) on three-year phase 1/2 data, and UK marketing authorisation. In March the FDA had queried whether the externally controlled data were sufficient for approval, before agreeing in July that submission under the accelerated approval pathway was reasonable.
uniQure submits BLA for AMT-130 in Huntington’s disease · uniQure
FDA leaders set out tougher oversight of foreign clinical trial sites.
The heads of the agency’s drug, biologics, device and oncology centres wrote that the FDA can refuse data it cannot validate. It is adding resources for foreign inspections, becoming more systematic about telling sponsors and the public when sites are unavailable for inspection, and training reviewers to spot problem sites.
Good Clinical Practices Are Not Optional · FDA
Ultragenyx shares fell 44% after its Angelman syndrome drug failed in phase 3.
The decline wiped approximately $1.15bn off the company’s market value, reducing it from $2.6bn to $1.5bn. In Aspire, apazunersen missed its primary endpoint on cognition and its key secondary endpoint. Ultragenyx will reassess the programme and plans “significant expense reductions”.
Ultragenyx announces phase 3 Aspire results in Angelman syndrome · Ultragenyx
Ivonescimab beat Keytruda on overall survival in a China phase 3 trial.
Akeso and its partner Summit said a prespecified interim analysis of HARMONi-2 showed a statistically significant and clinically meaningful overall survival improvement for ivonescimab over Keytruda (pembrolizumab) in first-line PD-L1-positive non-small cell lung cancer. Detailed overall survival data were not disclosed and will be presented at a medical meeting. HARMONi-2 had previously met its progression-free survival primary endpoint. Shares in US partner Summit Therapeutics rose 17.3%, adding $2bn to take its market value to $13.7bn.
Ivonescimab meets OS secondary endpoint in phase III HARMONi-2 · Akeso
Ivonescimab shows OS benefit vs Keytruda in HARMONi-2 · Summit Therapeutics
Thursday 3 September
GSK licensed HUTCHMED’s KRAS-EGFR conjugate for $110m upfront.
The deal for HMPL-A830, worth up to $1.3bn, covers the world excluding China, Hong Kong, Macau and Taiwan. The drug delivers a KRAS inhibitor payload through an anti-EGFR antibody, with clinical development expected to begin in H2 2026.
Licensing agreement with GSK for KRAS-EGFR Ab conjugate therapy · HUTCHMED
AbbVie’s etentamig met both primary endpoints in phase 3 multiple myeloma.
At the first planned interim analysis of CERVINO in triple-class exposed relapsed or refractory myeloma, the BCMAxCD3 bispecific produced a 74% response rate against 45.7% with standard therapies and cut the risk of progression or death by 60%.
Positive topline results from phase 3 CERVINO trial of etentamig · AbbVie
Three patients have lived more than eight months without dialysis on eGenesis engineered pig kidneys.
Five patients at Mass General Brigham have received EGEN-2784 since March 2024. Two later moved to human donor kidneys, the first such transitions reported, and the phase 1/2/3 RESTORE trial is due to start in Q1 2027.
Clinical update from study of EGEN-2784 in patients with kidney failure · eGenesis
The FDA approved Ionis’ Zanvastro, the first drug for Alexander disease.
Zanvastro (zilganersen) is an antisense drug injected into the spinal canal every three months for children and adults with the rare, progressive neurological disorder. In a randomised study of 49 patients, treated patients aged five and over with measurable walking difficulty at baseline walked faster at 61 weeks than untreated ones.
FDA approves first drug to treat Alexander disease · FDA
Zanvastro FDA approved as 1st disease-modifying drug for Alexander disease · Ionis
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