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ALETH / LIFESCI-BRIEF / 2026-09-25 / FDA PANEL BACKS GRAIL’S GALLERI CANCER TEST

FDA panel backs Grail’s Galleri cancer test

Also this week: Telix agreed to buy ITM for $1.65bn, the White House announced MFN pricing for all 50 state Medicaid programmes, and nine pharma chairs urged action on Europe’s research decline.

The Aleth Briefs link to the sources behind the stories and show how the week unfolded.

The week in five lines:

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Weekend catch-up

All 50 US state Medicaid programmes will get most-favoured-nation drug prices, the White House said on Friday.

Under the GENEROUS model, participating manufacturers pay rebates on covered brand-name drugs so that Medicaid’s final price does not exceed the MFN price. The Council of Economic Advisers puts the saving at $36.6bn to the federal government and $27.6bn to states over a decade.

Public Citizen published heavily redacted versions of Pfizer’s and Lilly’s MFN agreements, obtained through freedom-of-information requests.

According to its analysis, Pfizer’s agreement provides for sharing with the US government some additional revenue from higher drug prices abroad. Lilly’s allows a country’s price to be excluded from the MFN calculation if it stops supplying the drug there. Its Mounjaro and Zepbound products are excluded from the GENEROUS Medicaid pricing programme and covered by a separate arrangement.

US Treasury rules could preserve most drug licensing deals with China, Reuters reported.

Restrictions would focus on pathogens and biotechnology that could be weaponised, according to sources briefed on the process. The rules have not been finalised. The Wall Street Journal also reported that drugmakers were lobbying the administration to protect their ability to license Chinese drugs.

The FDA approved Lilly’s Inluriyo with Verzenio for ESR1-mutated advanced breast cancer.

The approval covers adults with ER-positive, HER2-negative disease that progressed after at least one line of endocrine therapy. In EMBER-3’s ESR1-mutated subgroup, the oral SERD Inluriyo (imlunestrant) plus Verzenio (abemaciclib) doubled median progression-free survival to 11.1 months, versus 5.5 months with Inluriyo alone.

A White House plan to give political appointees veto power over NIH grants stalled after bipartisan opposition.

The proposed executive order would add political review after scientific assessment of grant applications. Senate Appropriations Committee chair Susan Collins wrote to budget director Russ Vought and NIH director Jay Bhattacharya on Tuesday urging them not to proceed. By Wednesday, the plan was “looking dead”, STAT reported.

The FDA approved IntraBio’s Aqneursa for ataxia-telangiectasia.

Aqneursa (levacetylleucine) was cleared for ataxia in patients weighing ≥15 kg, on a crossover trial in 73 patients aged 4 to 50. IntraBio says it is the first drug approved for the disease anywhere. It was already approved in Niemann-Pick disease type C.

Monday 21 September

Telix agreed to buy isotope maker ITM for $1.65bn upfront.

The deal includes $1.25bn in Telix shares and $302m in net debt, with up to $700m tied to approvals and sales of ITM-11, ITM’s lutetium-177 treatment for neuroendocrine tumours. ITM had $273m in sales in 2025. The acquisition strengthens isotope supply for Telix’s pipeline and adds ITM’s development assets. ITM plans to resubmit ITM-11 after an FDA rejection in August over manufacturing and third-party facility issues. According to Telix, the FDA identified no safety or efficacy deficiencies.

Beacon’s eye gene therapy met its primary endpoint in a pivotal phase 2/3 trial.

Laru-zova met the FDA-endorsed primary endpoint in VISTA, an 85-patient trial in males with X-linked retinitis pigmentosa (XLRP), an inherited disease causing progressive vision loss. At 12 months, 31.0% of the high-dose group and 24.1% of the low-dose group gained at least 15 letters on a low-light visual acuity test, compared with none in the untreated group. Beacon plans to begin a rolling US approval application later this year. UK investor Syncona owns 38.4% of Beacon. Its stake was valued at £183.4m at 30 June, before these results.

Novo’s shares fell 8% as its new strategy failed to reassure investors.

At its capital markets day in London, Novo targeted over five blockbuster launches by 2030 and over DKK150bn in pipeline sales in 2035. Chief executive Mike Doustdar acknowledged that management had to convince investors there was a bright future for Novo. Novo’s shares fell nearly 11% over the week to Thursday’s close, wiping approximately $20bn off its market value. Positive phase 3 results for CagriSema, its combination of cagrilintide and semaglutide, were not enough to lift sentiment.

AI drug developer Iambic Therapeutics filed for a Nasdaq IPO and announced an AbbVie discovery partnership.

Its lead candidate, IAM1363, is an oral, brain-penetrant HER2 inhibitor in phase 1/1b, with a registrational trial potentially starting in 2027. The multi-year AbbVie collaboration will apply Iambic’s AI platform to small-molecule discovery in immunology, neuroscience and oncology. Iambic will receive an upfront payment and could earn milestones and royalties; amounts were not disclosed.

Tuesday 22 September

Celldex’s barzolvolimab met the primary endpoint in two phase 3 trials in chronic spontaneous urticaria.

The anti-KIT antibody met all key secondary endpoints in EMBARQ-CSU1 and CSU2, which enrolled 1,939 patients whose symptoms were inadequately controlled by antihistamines. Across both trials and doses, 42–46% had no itch or hives at week 12, versus 9–13% on placebo. Benefits were also seen in patients who had not responded to omalizumab. Celldex plans to file for FDA approval in 2027.

Roche’s enicepatide produced 15.5% weight loss in a phase 2 diabetes trial.

Enicepatide (formerly CT-388), its once-weekly GLP-1/GIP receptor agonist, produced 15.5% weight loss and reduced HbA1c by 2.65 percentage points at the highest dose in a 447-patient diabetes trial. The 15.5% weight loss at 48 weeks puts Roche’s candidate in the same range as Lilly’s tirzepatide results in patients with diabetes, achieved over 72 weeks. Weight loss had not plateaued, although the separate trials do not establish comparative superiority. Phase 3 obesity trials are already underway.

Amgen’s dazodalibep met its phase 3 primary endpoint in Sjögren’s disease.

The CD40L-blocking fusion protein reduced systemic disease activity versus placebo at 48 weeks in OASIZ 301, which enrolled c.620 patients. Sjögren’s is an autoimmune disease that can cause debilitating dryness, fatigue, pain and organ damage, with no FDA-approved systemic treatment. Amgen has yet to release detailed results. A second phase 3 study, focused on patients with substantial symptoms but lower systemic disease activity, is expected to complete in Q4.

Otsuka and Ionis reported the first positive phase 3 trial in FUS-ALS.

Ulefnersen, an RNA-targeted drug designed to reduce production of the FUS protein, met the primary endpoint in FUSION. It improved a combined measure of survival, rescue treatment and function versus placebo over 505 days (p=0.0005). FUS-ALS is a rare fast-progressing genetic form of motor neurone disease with no approved treatment targeting its underlying cause. The partners plan to discuss expedited regulatory submission routes with the FDA.

Nine European pharma chairs called for action to reverse Europe’s declining share of drug research.

The chairs of companies including AstraZeneca, GSK, Novartis and Roche urged European governments to increase investment in medicines, accelerate clinical trials and speed up access to new treatments. Their joint letter said Europe’s share of global pharmaceutical R&D had fallen from 43% in 1990 to 31%, while its share of commercial clinical trials had halved over the past decade to 9%.

Wednesday 23 September

Roche’s sefaxersen met its phase 3 primary endpoint in kidney disease.

The monthly injectable RNA-targeted therapy, licensed from Ionis, significantly reduced protein in the urine versus placebo at 37 weeks in IgA nephropathy, a progressive autoimmune kidney disease. Sefaxersen reduces production of complement factor B, which contributes to kidney damage.

London’s Basecamp Research raised a $140m Series C to develop bio AI models.

S32 led, with backing from NVIDIA and Menlo Ventures. Basecamp collects genomic data through biodiversity partnerships in >30 countries to train its EDEN models. The funding will support further model development and the company’s ambition to translate AI-designed DNA sequences into therapies. Its therapeutic work is preclinical.

Enveda raised $311m to advance medicines derived from natural compounds.

Catalio Capital Management led the Series E, joined by investors including T. Rowe Price, Citadel’s Surveyor Capital, ICONIQ and existing backer Baillie Gifford. The round takes total funding above $845m. Enveda uses AI to identify promising natural compounds and medicinal chemistry to develop them into oral drugs. Funding will advance three clinical programmes spanning eczema, metabolic disease and inflammatory bowel disease into mid- and later-phase trials, advance further candidates into the clinic and expand the discovery platform.

Anthropic introduced its biology lab and reported an AI-assisted finding.

The Claude developer’s new Bay Area laboratory pairs AI analysis of genomic data with experiments conducted by human scientists. Its first reported findings concern a previously uncharacterised system of DNA repeats and an accessory gene alongside a known reverse transcriptase. Initial experiments showed that the repeats produce short RNAs, but the system’s function remains unknown. Independent commentary highlighted Claude’s ability to pursue promising leads, while noting that the study does not establish an advantage over conventional bioinformatics methods.

An FDA panel backed Grail’s Galleri cancer test.

The advisory panel voted 7-2, with one abstention, that the blood test’s benefits outweigh its risks for screening adults aged 50 and older. Members unanimously supported its safety but split 6-4 on effectiveness. The panel questioned whether an “early detection” claim was supported, with some favourable votes conditional on revised labelling and further studies. Long-term benefits remain unestablished. The vote is non-binding, with an FDA decision expected in the coming months.

Thursday 24 September

Lilly signed a discovery deal with China’s InnoCare worth up to $3.35bn.

InnoCare will discover and advance compounds under an agreement offering up to $100m in upfront and near-term payments, plus approximately $3.25bn in potential development and commercial milestones. It could also receive tiered single-digit royalties on sales. The targets and therapeutic areas were not disclosed.

The FDA approved Lilly’s Onswik, a once-weekly basal insulin for type 2 diabetes.

Onswik (insulin efsitora alfa) reduces basal insulin injections from 365 to approximately 52 a year. Approval was supported by four phase 3 QWINT trials involving more than 3,400 adults, which showed comparable HbA1c reductions to daily basal insulins. Lilly expects US pharmacy availability in the coming months. It follows Novo’s Awiqli, approved in March as the first weekly insulin in the US.

Merck’s remigromig matched Lucentis in a pivotal diabetic macular oedema trial.

In the 984-patient BRUNELLO trial in diabetic macular oedema, both doses were non-inferior to Lucentis on improvement in vision at 52 weeks. The antibody activates the Wnt pathway, which helps maintain the blood-retinal barrier. However, proliferative diabetic retinopathy, vitreous haemorrhage and discontinuations due to adverse events were more frequent with remigromig. Merck is investigating these findings and will present detailed results in October. A second phase 2b/3 trial is ongoing.

Japan’s Kobayashi Pharma received a take-private approach from NSSK and CVC.

The Japanese consumer-health group confirmed a preliminary, non-binding approach from affiliates of private equity firms NSSK and CVC Capital Partners. Reporting on Thursday put a potential deal at more than ¥500bn ($3.2bn), with possible participation by the founding family. Kobayashi is still recovering from a 2024 scandal involving contaminated red yeast rice supplements linked to kidney damage, and faces pressure from activist shareholder Oasis Management. No deal has been agreed.

Novo licensed Nanexa’s technology to develop monthly and quarterly injections.

The agreement covers up to five peptide programmes in obesity, type 2 diabetes and other cardiometabolic diseases. Swedish biotech Nanexa’s PharmaShell platform coats drug particles with an ultrathin shell to control their release, potentially extending the interval between injections. Novo will lead development and commercialisation. Nanexa could receive up to €1.165bn plus royalties, including €615m in combined upfront, development and regulatory payments (upfront not disclosed).

ADARx priced an upsized $446.3m IPO at the top of its range.

The siRNA drug developer priced 26.25m shares at $17, increasing the offering from approximately 21.9m shares. A concurrent investment by partner AbbVie brings expected combined gross proceeds to $535.2m and would give AbbVie a 4.9% stake. ADARx develops RNA-targeted medicines designed to reduce production of disease-driving proteins. Nasdaq trading under ADRX was scheduled to begin on Friday.

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