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ALETH / LIFESCI-BRIEF / 2026-08-21 / MODERNA ADDS $50BN ON LANDMARK CANCER DATA

Moderna adds $50bn on landmark cancer data

Also this week: Trump nominated White House aide Heidi Overton to run the FDA, AstraZeneca won two late-stage lung cancer trials and stopped another, and argenx delivered a phase 3 win in myositis.

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

The week in five lines:

Browse by day:

Weekend catch-up

Redx Pharma agreed a reverse merger with Nasdaq-listed Skye Bioscience to create Fibrx Therapeutics, backed by $125m of concurrent financing.

The deal finally gives Redx the US public-market listing it has pursued since its failed 2023 reverse with Jounce and 2024 AIM delisting. Fibrx will be built around Redx's fibrosis pipeline and led by its management. The $125m is expected to fund it into 2029 and through phase 2 topline data for lead asset RXC008 in H2 2028.

The FDA approved Lantheus’ tau PET imaging agent for Alzheimer’s disease.

Tauklarify (florquinitau F 18, previously MK-6240) is cleared to image tau neurofibrillary tangle pathology in adults with cognitive impairment being evaluated for Alzheimer’s disease, complementing amyloid PET. The approval comes as Lantheus is being acquired by radiopharma rival Curium in a deal worth up to $8bn.

Biogen’s antibody felzartamab won its first approval anywhere, in China.

The anti-CD38 antibody was approved for multiple myeloma, its first authorisation anywhere. More important for Biogen, felzartamab is also in global phase 3 trials in immune-mediated diseases, where the company sees the larger opportunity.

Monday 17 August

AstraZeneca won two late-stage lung cancer trials and stopped another.

In DESTINY-Lung04, Enhertu, partnered with Daiichi Sankyo, improved progression-free survival (PFS) over chemotherapy plus Keytruda as first-line treatment of HER2-mutant advanced non-small cell lung cancer. In SAFFRON, Tagrisso plus Orpathys, partnered with HUTCHMED, improved PFS and overall survival in EGFR-mutated disease with high MET overexpression after progression on Tagrisso.

The data extend AstraZeneca’s position in molecularly defined lung cancer, although they came on the same day it discontinued a separate Phase 3 trial of volrustomig.

argenx’s Vyvgart Hytrulo met its phase 3 endpoint in myositis.

In the ALKIVIA trial, Vyvgart Hytrulo (efgartigimod) delivered a 15.4-point greater improvement in mean Total Improvement Score at week 52 than placebo (p=0.0011) across the combined immune-mediated necrotising myopathy and dermatomyositis population, with benefit from week 4. It is the first phase 3 trial to show a benefit in immune-mediated necrotising myopathy, which has no approved therapy.

EyePoint’s Duravyu missed its primary endpoint in wet AMD.

In LUGANO, the first of two phase 3 trials, Duravyu (vorolanib) failed to meet its prespecified non-inferiority endpoint against Eylea on mean change in best corrected visual acuity. EyePoint said the result was affected by nine patients, about 4% of the cohort, with vision loss it considered unrelated to wet AMD. An ad hoc analysis excluding those patients met the non-inferiority threshold. The company still plans to file in H1 2027, subject to results from the identical LUCIA trial due in Q4 2026.

Sandoz agreed to pay up to $322m for three Henlius biosimilars.

Shanghai Henlius granted Sandoz exclusive registration and commercialisation rights outside China to as many as 10 proposed antibody and antibody-drug conjugate biosimilars, with proposed cetuximab, evolocumab and belimumab copies first. Sandoz pays up to $322m in total, of which up to $100.5m is invoiced in 2026.

Slate agreed a merger with Fulcrum Therapeutics and $245m in commitments.

The all-stock merger would take migraine developer Slate onto Nasdaq, alongside a committed $245m private placement led by Frazier Life Sciences. The combined company will retain the Slate name. Lead asset SLTE-1009 is a subcutaneous anti-PACAP/VIP antibody, and the financing is expected to fund operations into 2029.

Tuesday 18 August

Amylyx’s GLP-1 antagonist avexitide cut hypoglycaemic events by more than half in phase 3.

In the LUCIDITY trial in 78 patients with post-bariatric hypoglycaemia after gastric bypass, avexitide reduced the composite rate of Level 2 and Level 3 hypoglycaemic events by 55% versus placebo (p=0.000003) and met every secondary endpoint. Amylyx plans to file by year-end for a condition with no approved treatment.

LEO Pharma agreed to acquire worldwide rights to dersimelagon from Tanabe Pharma for up to $435m.

Dersimelagon is an oral MC1R agonist for erythropoietic protoporphyria and X-linked protoporphyria. It has completed phase 3, is filed and awaits an FDA decision. The deal gives LEO a late-stage rare-disease asset close to potential launch in dermatology.

BioMarin agreed to buy Alesta Therapeutics for $275m upfront.

The deal brings ALE1, an oral small molecule that could become the first oral therapy for hypophosphatasia, a rare bone disease. Alesta will spin out its other assets before closing, expected this quarter. ALE1 adds to BioMarin’s skeletal conditions portfolio.

A federal appeals court handed Teva a partial win against Medicare price negotiations.

The US Court of Appeals for the D.C. Circuit sent back to a lower court the question of whether CMS's “bona fide marketing” standard, which determines when a drug becomes eligible for negotiation, complies with the Inflation Reduction Act. The court did not rule that the standard itself was unlawful. Teva's broader constitutional challenges were unsuccessful.

Biokin’s iza-bren, partnered with BMS, met its phase 3 endpoint in lung cancer.

In the China BL-B01D1-301 trial, the EGFR×HER3 bispecific ADC met its primary progression-free survival endpoint at an interim analysis in EGFR-mutated non-small cell lung cancer after EGFR-TKI treatment, with a positive trend in overall survival. It is the fourth phase 3 study in which iza-bren has met its primary endpoint.

A third patient death was reported in China's investigator-initiated cell and gene therapy trials.

Endpoints reported a previously undisclosed death in a CAR-T study, following two recently reported deaths in experimental gene-therapy trials. The cases have increased scrutiny of transparency and oversight in China’s investigator-initiated trial (IIT) system.

Wednesday 19 August

Moderna’s mRNA cancer therapy met two phase 3 endpoints in melanoma.

In INTerpath-001, intismeran autogene, Moderna’s personalised mRNA neoantigen therapy, plus Merck’s Keytruda beat Keytruda alone on recurrence-free survival and distant metastasis-free survival at a prespecified interim analysis in patients with completely resected stage IIB-IV melanoma. The trial continues to evaluate other secondary endpoints, including overall survival.

It is the first positive phase 3 trial of an individualised neoantigen therapy and the first for an mRNA cancer therapy. The result validates a personalised treatment model in which tumour sequencing, computational neoantigen selection and patient-specific mRNA manufacturing are combined to generate anti-tumour T-cell responses in vivo. Moderna shares rose 177%, adding about $45bn to its market value, reflecting the broader potential of the platform beyond melanoma.

Regeneron won FDA approval for FOP drug Pasatru.

Pasatru, an antibody targeting Activin A, was approved to reduce new heterotopic ossification lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva, the ultra-rare disease in which soft tissue turns to bone. In the phase 3 trial, two doses reduced new lesions by 90% and 94% vs placebo at 56 weeks.

Ultragenyx won its first gene therapy approval, in a rare metabolic disease.

The FDA granted accelerated approval to Genglycos for patients eight and older with glycogen storage disease type Ia. Genglycos is designed to address enzyme deficiency and reduce dependence on cornstarch used to maintain blood glucose. It is Ultragenyx’s first marketed gene therapy, and came with a Priority Review Voucher.

Trump picked Heidi Overton to run the FDA.

The president nominated White House policy aide Heidi Overton, M.D., Ph.D., as FDA commissioner. She faces Senate confirmation and would take over an agency that has lost around one in six of its staff over the past year (>3,000), after months of leadership turnover and regulatory controversy. Reuters reported concern among drugmakers and FDA staff over her limited management experience.

The FDA cleared the first standalone robotic blood-draw device.

The FDA authorised Vitestro's Aletta through the De Novo pathway, the first standalone robotic device able to draw blood from a patient's arm without hands-on intervention. It uses near-infrared imaging and ultrasound to locate a vein and automates the draw. A phlebotomist supervises up to three devices, with sensors that stop or pause the procedure if the patient moves or another safety issue is detected. FDA said success rates were comparable with or better than trained phlebotomists.

A US appeals court sided with Parse Biosciences over 10x Genomics on patents.

The Federal Circuit upheld the Patent Trial and Appeal Board's invalidation of three 10x Genomics patents in its dispute with Qiagen-owned Parse Biosciences. The ruling removes three patents from 10x's enforcement arsenal in the single-cell sequencing market, where the two companies compete directly.

Aurinia settled with Teva over Lupkynis generic entry in December 2036.

The settlement allows Teva to launch its generic version of Lupkynis (voclosporin) in the US no earlier than 7 December 2036, subject to defined contingencies and FDA approval. Teva also acknowledged two Aurinia patents as valid, enforceable and infringed by its proposed product. Aurinia continues to litigate separate generic challenges from other companies, so the agreement does not resolve all generic risk.

Thursday 20 August

NICE backed Lilly’s weekly insulin Onswik for the NHS.

The cost watchdog recommended insulin efsitora alfa, also known as Onswik, for type 2 diabetes in final draft guidance. It controls blood glucose as well as widely used long-acting insulins while cutting injections by 85%, from daily to once a week. NICE said it could particularly benefit people who rely on a carer or nurse for injections. The recommendation remains subject to MHRA licensing before NHS use.

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