ALETH / LIFESCI-BRIEF / 2026-03-06 / FDA AND UNIQURE CLASH OVER HUNTINGTON’SThe Aleth Briefs link to the sources behind the stories and show how the week unfolded.
The week in five lines:
The FDA turned down uniQure’s Huntington’s data, then called them distorted.
Moderna agreed a patent deal worth $950m upfront and up to $2.25bn in all.
Roche’s fenebrutinib met its goal in a third MS trial, with eight deaths on the drug.
Oxford Nanopore replaced its co-founder Gordon Sanghera as chief executive.
Zealand Pharma reported 10.7% weight loss for petrelintide.
Browse by day:
Weekend catch-up: Flu-Covid jab · Avidity · Padcev · Ascendis · Welireg
Monday: Roche MS · UK Oxford Nanopore · UK Smith & Nephew · Intellia
Tuesday: Kyowa Kirin · FDA letters · US envoy on drug prices · Moderna
Wednesday: Bayer · UK trials funding · Dravet
Thursday: J&J myeloma · uniQure · Roche-Zealand · MiniMed IPO
Friday: China obesity approval
Weekend catch-up
The EMA’s human medicines committee recommended Moderna’s mCombriax, the first combined flu and Covid-19 vaccine, for people aged 50 and over.
At its February meeting the Committee for Medicinal Products for Human Use (CHMP) also backed Novartis’s Rhapsido (remibrutinib) in chronic spontaneous urticaria and, conditionally, Ipsen’s Ojemda (tovorafenib) in paediatric low-grade glioma. It recommended against Acadia’s Daybue (trofinetide) in Rett syndrome and Vanda’s Fanapt (iloperidone) in schizophrenia.
Meeting highlights from the CHMP, 23-26 February 2026 · EMA
CHMP backs mCombriax combined flu and Covid vaccine · Moderna
Novartis completed its $12bn acquisition of Avidity Biosciences, spining-out Atrium Therapeutics, which launched with $270m.
Avidity shareholders received $72 a share in cash. Atrium starts with two preclinical heart-disease RNA candidates, ATR 1072 for PRKAG2 syndrome and ATR 1086 for PLN cardiomyopathy, and is led by former Avidity senior executive Kathleen Gallagher.
Novartis successfully completes acquisition of Avidity Biosciences · Novartis
Atrium launches with about $270m for RNA heart drugs · Atrium Therapeutics
Padcev plus Keytruda improved overall survival in muscle-invasive bladder cancer in the phase 3 KEYNOTE-B15 trial.
Given before and after surgery, Padcev (enfortumab vedotin) plus Keytruda (pembrolizumab) cut the risk of death by 35% and the risk of recurrence, progression or death by 47% against cisplatin-based chemotherapy before surgery. The data were presented at the ASCO Genitourinary Cancers Symposium. Merck ran the study in collaboration with Pfizer and Astellas.
Padcev plus Keytruda cuts recurrence or death risk by nearly 50% · Astellas
The FDA granted accelerated approval to Ascendis Pharma’s once-weekly achondroplasia treatment Yuviwel (navepegritide) on Friday 27 February.
The approval covers children aged 2 and older with open growth plates and brings a priority review voucher. Yuviwel now competes with BioMarin’s daily Voxzogo (vosoritide), which sold $927m in 2025.
Novel drug approvals for 2026 · FDA
FDA approves once-weekly Yuviwel in achondroplasia · Ascendis Pharma
Merck’s Welireg (belzutifan) cut the risk of progression or relapse in two phase 3 kidney cancer trials presented at the ASCO Genitourinary Cancers Symposium.
Added to Lenvima (lenvatinib) after immunotherapy, Welireg cut the risk of progression or death by 30% against Cabometyx (cabozantinib) in LITESPARK-011, with overall survival showing only a trend so far. Added to Keytruda after surgery, it cut the risk of recurrence or death by 28% in LITESPARK-022. The FDA will decide on the LITESPARK-011 filings by 4 October.
Welireg plus Lenvima cut progression or death risk by 30% · Eisai
Adjuvant Keytruda plus Welireg cut the risk of recurrence or death by 28% · Merck
Monday 2 March
Roche’s fenebrutinib met its goal in a third phase 3 multiple sclerosis trial, but eight patients died on the drug across two studies.
FENhance 1 cut the yearly relapse rate by 51% against Aubagio (teriflunomide), after a 59% cut in FENhance 2. Across the two trials one patient died on teriflunomide and eight, from various causes, on fenebrutinib. Roche says it is still analysing the deaths.
Each arm of FENhance 1 had one case meeting Hy’s Law, the liver injury pattern regulators watch for. Both were without symptoms and resolved. Roche will submit all three studies, including FENtrepid in primary progressive disease, to regulators.
Fenebrutinib positive in third phase III study, FENhance 1 · Roche
Oxford Nanopore replaced co-founder Gordon Sanghera as chief executive and reported 2025 revenue up 22%.
Revenue was £224m, up 24% at constant currency, and the adjusted EBITDA loss narrowed to £86.7m. Guidance for 2026 is constant-currency growth of 21-25%, a gross margin of about 62% and adjusted operating cost growth of 0-5%.
Van Parys, formerly of Danaher’s Radiometer and Cytiva/GE Healthcare, started as CEO on 2 March. Sanghera, who has led the company since 2005, left the board and stays as an adviser into early 2027. Adjusted EBITDA break-even is still targeted for FY27.
Annual results for the year ended 31 December 2025 · Oxford Nanopore
Smith & Nephew said it expects 2026 trading profit to grow about 8% organically after completing its 12-point plan.
It expects underlying revenue growth of about 6% and free cash flow of about $800m. Growth and savings are offsetting tariffs, an inventory revaluation, US reimbursement changes for skin substitutes and China’s bulk-buying rules for ENT products. It has also completed the Integrity Orthopaedics acquisition.
Smith+Nephew fourth quarter and full year 2025 results · Smith & Nephew
United Therapeutics’ oral ralinepag cut the risk of clinical worsening in pulmonary arterial hypertension by 55% in phase 3.
80% of patients were already taking two other PAH drugs. Six-minute walk distance also improved. United Therapeutics expects to submit a new drug application by the second half of 2026.
Ralinepag cut clinical worsening risk by 55% in PAH · United Therapeutics
The FDA lifted the clinical hold on Intellia’s phase 3 MAGNITUDE trial of the CRISPR therapy nexiguran ziclumeran in ATTR cardiomyopathy.
The hold, imposed on 29 October 2025 after a patient had severe rises in liver enzymes and bilirubin, now comes with mitigation measures: tighter liver monitoring, steroid guidance and exclusion of patients with certain liver abnormalities. The hold on MAGNITUDE-2 was lifted in January.
FDA lifts hold on MAGNITUDE trial in ATTR-CM · Intellia Therapeutics
Tuesday 3 March
Kyowa Kirin and Amgen stopped all trials of the anti-OX40 antibody rocatinlimab after further cases of Kaposi’s sarcoma.
A safety review found a new confirmed case and a suspected one, on top of an earlier confirmed case. The companies say the drug’s mechanism may be the cause. They concluded the risks may outweigh the benefits in atopic dermatitis, asthma and prurigo nodularis. The studies end once participants finish safety follow-up.
Kyowa Kirin announces discontinuation of rocatinlimab clinical trials · Kyowa Kirin
The FDA published its rejection letters to AstraZeneca for Saphnelo and GSK for Exdensur, both citing problems with the data.
The AstraZeneca letter cites “critical data quality issues that affect key analyses, including the primary endpoint” in the lupus application, after the company submitted a corrected dataset late in the review. The GSK letter says the data do not give adequate evidence that Exdensur works in chronic rhinosinusitis with nasal polyps.
Complete response letter to AstraZeneca, BLA 761451 (anifrolumab) · FDA
FDA letters reveal Saphnelo data errors, Exdensur doubts · Fierce Pharma
Donald Trump’s ambassador to the UK rebuked the head of the UK’s medicines watchdog over drug pricing, the Financial Times reported.
Trump’s ambassador rebukes head of UK medicines watchdog · Financial Times
Moderna agreed to pay $950m upfront, and up to $1.3bn more, to settle Arbutus and Genevant’s patent claims over its mRNA vaccines.
The $950m is a lump sum due by 8 July. The other $1.3bn depends on Moderna’s appeal over whether section 1498, a federal contracting law, shields vaccine doses sold to the US government. It is paid in full if the appeal fails or is dropped, and in part if it partly succeeds. Moderna owes no royalties and gets a non-exclusive licence to Genevant’s lipid nanoparticle technology for infectious disease vaccines.
Their separate US case against Pfizer and BioNTech continues.
Form 8-K on the Arbutus and Genevant settlement · Moderna
$2.25bn global settlement with Moderna · Arbutus Biopharma
Wednesday 4 March
Bayer guided to stable currency-adjusted sales and earnings in 2026 after a €3.62bn net loss driven by litigation charges for its Roundup class settlement.
Group sales were €45.6bn and EBITDA before special items €9.67bn, down 4.5%. Free cash flow fell to €2.08bn and will be hit by settlement payments in 2026. The proposed dividend is €0.11 a share.
Bayer projects solid 2026, focused on strategic priorities · Bayer
The UK government is giving £48m to 130 NHS trusts and primary care organisations in England to run commercial clinical trials.
The National Institute for Health and Care Research (NIHR) money goes to 51 NHS trusts and 79 primary care organisations. It pays for equipment, refurbishments and modular buildings and includes money drugmakers pay under the VPAG medicines pricing scheme. Primary care organisations were eligible for the first time.
NIHR capital investment for research, written statement · UK Parliament
Stoke Therapeutics’ zorevunersen cut monthly convulsive seizures in Dravet syndrome by a median 59% to 91% in open-label studies.
The studies, published in the New England Journal of Medicine, enrolled 81 patients aged 2 to 18. The falls were measured over the first 20 months of the extension studies, in patients who started on 70 mg and then took up to 45 mg. Most adverse events were mild or moderate, but two patients died from sudden unexpected death in epilepsy and one from malnutrition.
Zorevunersen in Dravet syndrome · New England Journal of Medicine
Thursday 5 March
The FDA approved J&J’s Tecvayli plus Darzalex Faspro in multiple myeloma after at least one prior line of therapy.
Patients must already have had a proteasome inhibitor and an immunomodulatory drug. The approval rests on the phase 3 MajesTEC-3 trial, in which three-year survival was 83.3% with the combination against 65.0% on standard regimens. The FDA selected the application for its Commissioner’s National Priority Voucher pilot.
FDA approves Tecvayli plus Darzalex Faspro in myeloma · Johnson & Johnson
The FDA rejected uniQure’s Huntington’s data as the basis for a filing, and an official accused it of distorting them.
On Monday uniQure said the FDA will not accept its phase 1/2 data on AMT-130, compared against an external control, as the main evidence for approval. The agency strongly recommended a randomised, double-blind trial against sham surgery. uniQure’s shares fell by more than 40%.
On Thursday a senior FDA official, speaking anonymously on a call with reporters, said the agency never agreed to accept the external comparison and accused uniQure of using “distorted” data to mask a failed therapy. The sham arm would involve anaesthesia and “one to three nicks in the scalp”, not a partial burr hole.
uniQure says its data warrant more dialogue on the FDA’s promised regulatory flexibility, and that it is “confident in the strength of the data” it has submitted.
uniQure provides regulatory update on AMT-130 · uniQure
uniQure shares crash 40% as FDA rejects early approval path · Fierce Biotech
Senior FDA official refutes uniQure’s claims · Fierce Biotech
Roche and Zealand Pharma’s petrelintide delivered up to 10.7% mean weight loss at week 42 in a phase 2 obesity trial.
Placebo patients lost 1.7% in ZUPREME-1, which enrolled 493 people. At the most effective dose there were no cases of vomiting, and discontinuations for adverse events were 4.8% against 4.9% on placebo.
Roche announces positive Phase II results for petrelintide · Roche
MiniMed, Medtronic’s diabetes business, priced its IPO of 28m shares at $20, raising $560m before the over-allotment option.
The shares are expected to begin trading on Nasdaq under MMED on 6 March, with Medtronic retaining about 90% after the offering.
MiniMed announces pricing of initial public offering · Medtronic
Friday 6 March
China approved Sciwind Biosciences’ ecnoglutide for chronic weight management, and Pfizer holds exclusive rights to sell it in mainland China.
The approval covers Chinese adults with overweight or obesity. At week 48, Sciwind reported 15.1% placebo-adjusted weight loss with the highest 2.4 mg dose, and 92.8% of participants lost at least 5% of their baseline weight.
China approves ecnoglutide for weight management · Sciwind Biosciences
Sciwind partners with Pfizer China on ecnoglutide · Sciwind Biosciences
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