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ALETH / LIFESCI-BRIEF / 2026-09-11 / FDA OVERRIDES PANEL TO CLEAR AZ’S ETCAMAH

FDA overrides panel to clear AZ’s Etcamah

Also this week: two Novartis drugs failed phase 3 and an investor called for board changes, Amgen’s Imdelltra extended survival in small cell lung cancer, AstraZeneca’s tozorakimab received priority review & Bayer’s Hyrnuo was approved as first treatment.

The Aleth Briefs trace each story to its original source and show how the week unfolded.

The week in five lines:

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Story of the week

Two Novartis drugs failed phase 3 in four days, and a top-20 investor called for board changes.

On Friday evening Novartis said the Lp(a)HORIZON trial of 8,323 patients with high lipoprotein(a) and established cardiovascular disease had missed its primary endpoint of fewer cardiovascular deaths, heart attacks, strokes and emergency heart procedures. Pelacarsen, an antisense drug licensed from Ionis, lowered Lp(a) but did not reduce cardiovascular events.

Four days later del-desiran failed the phase 3 HARBOR trial in myotonic dystrophy type 1. It did no better than placebo on the time patients take to open their hand after gripping. It was the lead drug in Novartis’s purchase of Avidity Biosciences. Novartis saw some activity on secondary measures, will discuss the data with regulators, and kept its forecast of 5-6% annual sales growth to 2030.

On Thursday David Samra of Artisan Partners, a top-20 shareholder, told Reuters that chairman Giovanni Caforio “needs to make changes at the board level”, including to its dealmaking team. Novartis said it takes a disciplined approach to capital allocation.

Weekend catch-up

The FDA approved AstraZeneca’s Etcamah despite a negative panel vote.

On Friday the FDA granted accelerated approval to Etcamah (camizestrant) with a CDK4/6 inhibitor in HR-positive, HER2-negative advanced breast cancer. Patients switch to it once a blood test detects an ESR1 mutation during first-line therapy, before scans show progression. The agency called it the first cancer approval guided by a resistance mutation in circulating tumour DNA.

Median progression-free survival was 16 months against 9.2 months for patients who stayed on an aromatase inhibitor. The FDA’s oncology advisers had voted 6 to 3 against it in April. AstraZeneca pegged peak sales at >$5bn, Fierce Pharma reported.

The FDA approved Guardant’s blood test as the companion diagnostic for Etcamah.

Guardant360 CDx became the first FDA-approved liquid biopsy for repeat monitoring in breast cancer, with testing every three months during therapy to catch emerging ESR1 mutations. It was approved on the same day as the drug.

ADARx Pharmaceuticals filed for a Nasdaq IPO.

The RNAi developer submitted its S-1 on Friday to list under the ticker ADRX. AbbVie paid it $335m upfront for an option deal in 2025. Its most advanced drug, onvuzosiran, targets prekallikrein to prevent hereditary angioedema attacks, with STOP-HAE trial data due by the end of 2027.

Spire Healthcare agreed a £1.03bn takeover by Toscafund, Three Hills and Ares.

On Saturday the UK private hospital operator backed a 250p-a-share cash offer, a 66% premium to the share price before the approach became public in May. Chair-designate Debbie White cited material cost pressure from higher national insurance contributions and the minimum wage.

Monday 7 September

Wegovy brought two in five children below the obesity threshold.

In the phase 3 STEP Young trial of 165 children aged six to under 12, 40.4% of those on Wegovy (semaglutide) plus diet and exercise were no longer classed as obese at 68 weeks, against none on placebo. The figure assumes every child kept taking the drug.

Novo Nordisk stopped two more phase 3 trials of ziltivekimab.

Novo told investigators on Friday it was ending the HERMES and ATHENA heart failure trials, which enrolled 4,900 and 673 patients, after its data monitoring committee said they were unlikely to succeed where July’s ZEUS trial failed. That leaves ARTEMIS, in patients after a heart attack, which reads out in H1 2027.

Tuesday 8 September

Amgen’s Imdelltra extended survival earlier in small cell lung cancer.

In the phase 3 DeLLphi-305 trial, Imdelltra (tarlatamab) plus Imfinzi (durvalumab) extended overall survival compared with Imfinzi alone at an interim analysis. Patients had extensive-stage disease that had not progressed after first-line chemotherapy and Imfinzi. The combination also improved progression-free survival and response rate.

Imdelltra is already approved after progression on platinum chemotherapy.

AstraZeneca’s tozorakimab received FDA priority review in COPD.

The FDA accepted the IL-33 antibody’s filing under priority review as AstraZeneca presented the phase 3 OBERON and TITANIA trials at the European Respiratory Society (ERS) congress and in the New England Journal of Medicine. Given every four weeks on top of inhaled therapy, tozorakimab cut moderate and severe exacerbations by 29% and 34% in former smokers, and by 30% and 29% overall.

R&D head Sharon Barr said the results “set a new standard for COPD treatment outcomes in a broad population of patients”.

Sanofi presented phase 2b asthma data for lunsekimig at ERS.

In the AIRCULES trial, the TSLP/IL-13 bispecific nanobody cut the yearly rate of asthma attacks by about 39% overall and 55% at the top dose, investigator Njira Lugogo told HCPLive. The benefit was larger in more severe patients, which will shape phase 3.

Bristol Myers Squibb’s GPRC5D CAR-T met its registrational endpoint in myeloma.

In the phase 2 QUINTESSENTIAL trial, arlo-cel met its goals on overall and complete response rates in heavily pretreated multiple myeloma, including after BCMA-targeted therapy. Bristol Myers Squibb called it the first pivotal trial in that population.

Pharvaris’s deucrictibant cut hereditary angioedema attacks by 83% in phase 3.

In CHAPTER-3, once-daily oral deucrictibant XR reduced monthly attacks by 83% against placebo (p<0.0001) across all three types of the disease and met every secondary endpoint. Pharvaris plans marketing applications from H1 2027.

Roivant’s inhaled mosliciguat met its phase 2 goal in lung disease-linked pulmonary hypertension.

In the 135-patient PHocus trial, once-daily inhaled mosliciguat cut pulmonary vascular resistance by 56.3% against placebo at week 16 (p<0.0001) and added 35.2 metres to six-minute walk distance. The phase 3 PHrontier trial is enrolling.

Sandoz plans to double sales with biosimilars.

At its Capital Markets Day Sandoz set a target of >100 biosimilars by 2040, up from 13 today, and aims to more than double net sales from 2025 to 2035. A $300m plant in Ljubljana, Slovenia, opens in 2029.

Boston Scientific said a cyberattack will probably make it miss its 2026 guidance.

In an SEC filing, the medtech company said the August attack, which disrupted manufacturing and order shipments, is likely to have a material impact on Q3 and full-year results. It is unlikely to meet its sales growth and adjusted EPS guidance, and will update its outlook on 28 October.

The ABPI said UK pharma investment has started to recover.

The trade body’s 2026 competitiveness report found the UK is recovering from years of decline, with £2bn of new investment committed since September 2025. Chief executive Richard Torbett warned that “this recovery is not yet secure”. The rebate drugmakers pay the NHS on newer medicines has fallen to 14.5% from 22.9% in 2025.

Wednesday 9 September

Encoded Therapeutics closed a $275m series F for its Dravet syndrome genetic medicine.

GV co-led the round. It funds the pivotal study of ETX101 in infants and young children with Dravet syndrome, an expansion study up to age 18 and commercial-scale in-house manufacturing.

Bayer’s Hyrnuo was approved in the US as first treatment for HER2-mutant lung cancer.

The FDA granted accelerated approval to Hyrnuo (sevabertinib), an oral tyrosine kinase inhibitor, as first treatment for advanced non-squamous non-small cell lung cancer with activating HER2 mutations, based on the phase 1/2 SOHO-01 trial. It was first approved for previously treated patients in November 2025.

Solstice Oncology launched with a $225m series A for a neoadjuvant CTLA-4 antibody.

RA Capital led the round. A phase 2 trial will test porustobart, a second-generation Fc-enhanced CTLA-4 antibody, with Keytruda (pembrolizumab) before surgery in stage II-III microsatellite-stable colon cancer. It opens in Q4 2026, with data due in H2 2027.

The FDA partially halted enrolment in Biohaven’s epilepsy trials.

Biohaven disclosed a partial clinical hold, issued on 4 September, that pauses new enrolment for BHV-7000 while it generates data on a metabolite seen in rodents. Dosing continues for >600 randomised patients, and pivotal data from the fully enrolled BHV7000-303 trial remain due in H2 2026. Biohaven agreed in August to license the drug to SK Biopharmaceuticals.

NICE provisionally rejected Gilead’s twice-yearly HIV prevention jab for the NHS.

In draft guidance, NICE accepted that Yeytuo (lenacapavir) cuts HIV risk more than oral PrEP and probably works as well as Apretude (cabotegravir). But at the current price it is not cost-effective for the NHS. Consultation runs to 30 September.

Tyra Biosciences identified a potential registrational dose for its oral bladder cancer drug.

In the phase 2 SURF302 trial in FGFR3-altered low-grade intermediate-risk non-muscle invasive bladder cancer, dabogratinib at 60 mg daily produced a 79% response rate and a 64% complete response rate in 14 patients. Tyra expects to use 60 mg in its planned registrational trial.

Thursday 10 September

A UK commission urged staged approvals and lifelong monitoring for AI medical devices.

The National Commission into the Regulation of AI in Healthcare, set up by the MHRA in 2025, recommended authorising new AI models in stages, like L-plates, before fuller approval. It wants AI devices monitored in real-world use throughout their life and stronger enforcement powers for the MHRA. The government and the MHRA will respond formally.

AbbVie’s Qulipta met its phase 3 goal in menstrual migraine.

In the LUNA study, Qulipta (atogepant) cut migraine days around the menstrual period against placebo, meeting the primary endpoint and all eight ranked secondary endpoints (p<0.0001). No treatment is approved for menstrual migraine.

Frazier Life Sciences raised >$1.1bn for its public biotech fund.

The fund, which backs listed small and mid-cap biotechs and crossover rounds, has now raised about $2.8bn since its 2021 launch.

Friday 11 September

Biotech’s 2026 IPO class is trading well above issue.

Of the 21 biotechs that listed on Nasdaq this year, 18 were above their offering price at 1 September and 13 were up >30%, BioCentury found. The median gain was 36% and the average 48%. The 21 listings compare with 14 in all of 2025.

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