A federal jury in the Northern District of California found on Thursday, September 24, 2026 that Eli Lilly breached the implied covenant of good faith and fair dealing in its license agreement with Nektar Therapeutics for rezpegaldesleukin (rezpeg), an autoimmune disease drug, and awarded Nektar $90 million in damages plus interest to be set by the court. Nektar filed the suit in August 2023, and the trial began on September 8. The verdict remains subject to post-trial proceedings, and any judgment may be appealed.
Roche said on September 23, 2026 that a prespecified interim analysis of the Phase 3 IMAgINATION trial found sefaxersen produced a statistically significant reduction in 24-hour urine protein-to-creatinine ratio versus placebo at 37 weeks in adults with primary IgA nephropathy at high risk of progression. The trial randomized 459 people 1:1 to sefaxersen, a once-monthly subcutaneous antisense drug that reduces liver production of complement factor B, or to placebo for 105 weeks. Roche did not disclose the size of the proteinuria reduction; the trial remains blinded to week 105 to measure change in eGFR, and safety was consistent with prior data. Roche licensed sefaxersen from Ionis.
Amgen announced on September 22, 2026 that OASIZ 301, a randomized, double-blind, placebo-controlled Phase 3 trial in about 621 adults with Sjögren's disease and moderate-to-severe systemic activity (ESSDAI of 5 or higher), met its primary endpoint of change from baseline in ESSDAI at week 48. Amgen described the improvement as statistically significant and seen as early as week 4, but did not release effect sizes. The company describes dazodalibep as a potential first-in-class CD40L antagonist fusion protein. The most common adverse events were nasopharyngitis, urinary tract infection, hypertension, and infusion-related reactions; a second Phase 3 trial, OASIZ 303, is expected to complete in the fourth quarter of 2026.
Immunovant, a majority-owned Roivant subsidiary, reported on September 23, 2026 that a 57-patient randomized, double-blind, placebo-controlled proof-of-concept trial of IMVT-1402 (imeroprubart) in cutaneous lupus erythematosus did not reach statistical significance on percent change in CLASI-A score at week 12. The company reported numerical trends favoring the drug and an association between deeper IgG reductions and clinical response, but said the results did not meet its internal bar and it will stop development in the indication. Trials of the FcRn inhibitor continue in Graves' disease, difficult-to-treat rheumatoid arthritis, myasthenia gravis, CIDP, and Sjögren's disease.
Celldex Therapeutics reported on Tuesday, September 22, 2026 that both Phase 3 trials of barzolvolimab, a humanized monoclonal antibody that binds the KIT receptor on mast cells, met their primary endpoint of mean change in weekly urticaria activity score (UAS7) at week 12 in adults with chronic spontaneous urticaria who remained symptomatic on H1 antihistamines. EMBARQ-CSU1 randomized 963 patients and EMBARQ-CSU2 randomized 976, for 1,939 in total. UAS7 fell by 19.7 to 20.5 points on the two barzolvolimab regimens (150 mg every 4 weeks or 300 mg every 8 weeks) versus 10.7 and 11.4 points on placebo, and complete response (UAS7 of 0) rates were 42.1% to 45.7% versus 9.3% and 12.6% on placebo. All key secondary endpoints were met, benefit was reported in omalizumab-refractory patients, and the drug was well tolerated through 24 weeks. Treatment continues to 52 weeks, and Celldex plans to file a Biologics License Application in 2027.
NewAmsterdam Pharma and Menarini Group announced on Monday, September 21, 2026 that the European Commission approved Ubeslo (obicetrapib 10 mg) and Evlarco (obicetrapib 10 mg plus ezetimibe 10 mg) for adults with primary hypercholesterolemia, including heterozygous familial and non-familial forms, or mixed dyslipidemia. The companies called it the first regulatory approval of obicetrapib, an oral, low-dose CETP inhibitor, anywhere in the world. The approval cites the Phase 3 BROADWAY, BROOKLYN, and TANDEM trials, which showed LDL-C reductions of up to 40% with obicetrapib alone and about 50% with the ezetimibe combination versus placebo, with tolerability comparable to placebo. Menarini holds exclusive European commercialization rights and pays NewAmsterdam tiered double-digit royalties, with up to €833 million more in milestones.
PTC Therapeutics said on Monday, September 21, 2026 that it completed its acquisition of ST-920 (isaralgagene civaparvovec) from Sangamo Therapeutics. ST-920 is a one-time AAV gene therapy for Fabry disease that is designed to enable long-term production of the missing alpha-galactosidase A enzyme in place of chronic enzyme replacement therapy. Under terms announced in August, PTC won the asset in Sangamo's Chapter 11 auction for $111 million upfront plus up to $100 million in approval milestones; Eli Lilly separately bought Sangamo's zinc finger programs for $50 million. ST-920 holds FDA Orphan Drug, Fast Track, and RMAT designations and EMA PRIME eligibility, and PTC expects to complete a rolling Biologics License Application for accelerated approval in the fourth quarter of 2026.
Insmed announced on Monday, September 21, 2026 that the FDA granted Priority Review to its supplemental New Drug Application for ARIKAYCE (amikacin liposome inhalation suspension) to treat Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial regimen, with a PDUFA target action date of January 28, 2027. The filing rests on the Phase 3b ENCORE study, which enrolled 425 patients, 82.4% of them with a first MAC infection, and met its primary endpoint and its multiplicity-controlled secondary culture-conversion endpoints. ARIKAYCE's PULMOVANCE liposomal technology delivers amikacin to lung macrophages while limiting systemic exposure. The drug received FDA accelerated approval in 2018 for refractory MAC lung disease and was the first product approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs.
Cue Biopharma announced on Sunday, September 20, 2026 topline results from a Phase 2 trial in China of CUE-221, a humanized anti-IgE IgG1 monoclonal antibody that the company says also reduces IgE synthesis, in 145 patients with moderate-to-severe chronic spontaneous urticaria. Patients were randomized to one of three CUE-221 doses, placebo, or omalizumab 300 mg every four weeks. At week 12, complete hive resolution (HSS7 of 0) was reached by 54%, 53%, and 43% of patients on the 4, 2, and 1 mg/kg doses, versus 11% on placebo and 41% on omalizumab. There were no treatment-related serious adverse events and no hypersensitivity reactions. Cue, which licensed CUE-221 from Ascendant Health, plans a Phase 2b/3 study in chronic spontaneous urticaria and a Phase 2 study in food allergy.
PureTech Health announced on Monday, September 21, 2026 a successful end-of-Phase 1 meeting with the FDA and Fast Track designation for LYT-200 in combination with a hypomethylating agent (HMA) for relapsed/refractory high-risk myelodysplastic syndromes (HR-MDS). LYT-200 is a fully human monoclonal antibody against galectin-9, developed by PureTech's founded entity Gallop Oncology. In the Phase 1b study, 11 efficacy-evaluable patients who received 12 mg/kg LYT-200 with an HMA had an overall response rate of 45.5% and a complete response rate of 27.3%, with no dose-limiting toxicities. The planned Phase 2 STRIDE-MDS trial will randomize about 125 patients 2:2:1 to 12 mg/kg or 7.5 mg/kg LYT-200 with an HMA, or placebo with an HMA; LYT-200 also holds Fast Track designation in acute myeloid leukemia.
Beacon Therapeutics reported on Monday, September 21, 2026 that the Phase 2/3 VISTA trial of laruparetigene zovaparvovec (laru-zova), an AAV-based gene therapy that delivers full-length RPGR, met its primary endpoint in X-linked retinitis pigmentosa (XLRP). At month 12, 31.0% of high-dose and 24.1% of low-dose patients had at least a 15-letter improvement in low-luminance visual acuity, versus no patients in the untreated control group, in a trial of 85 males aged 12 to 48. Beacon says no other late-stage XLRP trial has met its primary endpoint, and it reported a favorable safety profile with mostly mild-to-moderate ocular adverse events. The company plans a rolling BLA submission later this year and will present more data at the AAO annual meeting on October 10, 2026; it cites XLRP prevalence of about 1 in 25,000 males. Syncona is among Beacon's investors.
Novo Nordisk announced on Thursday, September 17, 2026 that the EMA's CHMP recommended marketing authorization for FREHEMGO (denecimig) to treat hemophilia A, with or without inhibitors, in adults and children. Denecimig is a factor VIIIa-mimetic bispecific antibody that bridges factor IXa and factor X to mimic the cofactor function of activated factor VIII. Novo says it is the first FVIIIa mimetic to offer once-monthly, once-every-two-weeks, and once-weekly prophylaxis in a single-use pre-filled pen. The application was based on the FRONTIER2, FRONTIER3, and FRONTIER4 trials, and FRONTIER5 showed no new safety signals in patients switching from emicizumab. Pending a European Commission decision, Novo expects launches in the first European countries in Q4 2026 and across the EU starting early 2027.
The FDA approved Eli Lilly's Inluriyo (imlunestrant) in combination with Verzenio (abemaciclib) on Friday, September 18, 2026 for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer whose disease progressed after at least one line of endocrine therapy. The FDA also approved the Guardant360 CDx assay as the companion diagnostic for detecting ESR1 mutations. The approval rests on the EMBER-3 trial, which enrolled 874 adults; in the 159-patient ESR1-mutated subgroup, median progression-free survival was 11.1 months with the combination versus 5.5 months with imlunestrant alone (hazard ratio 0.53). Objective response rates were 35% and 15%, respectively.
Novartis announced on Friday, September 18, 2026 that the CHMP adopted a positive opinion for Cosentyx (secukinumab) to treat polymyalgia rheumatica (PMR) in adults who have had an inadequate response to steroids or who relapse during steroid taper. Novartis says Cosentyx would be the first IL-17A inhibitor licensed in Europe for PMR. The opinion is based on the global Phase III REPLENISH trial, run in 27 countries, in which all primary and secondary endpoints were met across both the 300 mg and 150 mg arms; Cosentyx doubled sustained remission rates and delivered steroid-sparing effects versus placebo, with no new safety signals. The data were published in the New England Journal of Medicine, and the European Commission is expected to decide within about two months.
Merck announced on Friday, September 18, 2026 that the CHMP recommended approval of Keytruda (pembrolizumab) with Padcev (enfortumab vedotin) as neoadjuvant treatment, continued after radical cystectomy as adjuvant treatment, for adults with resectable muscle-invasive bladder cancer; the recommendation also covers subcutaneous Keytruda. In the Phase 3 KEYNOTE-B15 (EV-304) trial, the regimen reduced the risk of an event-free survival event by 47% and the risk of death by 35% versus gemcitabine and cisplatin chemotherapy plus surgery, and the pathologic complete response rate was 55.8% versus 32.5%. Merck said it would be the first and only PD-1 inhibitor plus antibody-drug conjugate regimen for muscle-invasive bladder cancer regardless of cisplatin eligibility in the EU. A European Commission decision is expected by the fourth quarter of 2026, and the FDA approved the combination for this use in July 2026. Merck's partners on the regimen are Astellas and Pfizer.
mAbxience, majority-owned by Fresenius with partial ownership by Insud Pharma, and Sandoz announced on Friday, September 18, 2026 a licensing, development, manufacturing, and commercialization agreement for a biosimilar candidate of emicizumab for hemophilia A. mAbxience will develop and manufacture the product at its GMP-approved facilities in Spain and Argentina, and Sandoz will hold exclusive global commercialization rights outside Argentina, Uruguay, and Paraguay. The companies estimate the global reference market at about $5.7 billion. Financial terms were not disclosed.
Xenon Pharmaceuticals announced on Thursday, September 17, 2026 that it submitted a New Drug Application to the FDA for azetukalner, a Kv7 potassium channel opener, in focal seizures, supported by the Phase 2b X-TOLE and Phase 3 X-TOLE2 studies. The company also paused enrollment of new patients in its major depressive disorder and bipolar depression studies after an analysis of neuropsychiatric adverse events; the pause was put in place with its Data Safety Monitoring Board, is expected to be temporary, and does not affect enrolled participants. Xenon said the events were consistent with azetukalner's known safety and tolerability profile. X-NOVA2, its depression study, had enrolled about 80% of its initial 450-patient target, and topline data are now expected in the first quarter of 2027.
Several analysts raised their Ultragenyx price targets on Friday, September 18, 2026, a day after the FDA's standard full approval of FAYUVI for the neurologic manifestations of Sanfilippo syndrome type A (MPS IIIA) in children. Cantor Fitzgerald moved to $39 from $33 with an Overweight rating, raised its probability of success to 100% from 90%, and valued the MPS IIIA opportunity at about $12 to $13 per share; Canaccord Genuity moved to $39 from $37 (Buy), Morgan Stanley to $20 from $18 (Equal Weight), and Citi to $32. Cantor also estimated that the priority review voucher Ultragenyx received with the approval, together with another voucher the company already holds, could bring in about $400 million if sold. Ultragenyx expects FAYUVI to be available to ship to qualified treatment centers within 30 to 60 days.