Editor’s Briefing: September 23, 2026

In Short. Wednesday’s tape is kidney and autoimmunity: Roche’s Ionis-partnered sefaxersen hits an IgA nephropathy phase 3 interim that tees a fight with Novartis’s Fabhalta, Amgen’s Horizon fusion protein clears a first Sjögren’s phase 3, Celldex’s hive antibody wins two pivotal trials while shares sink on anaphylaxis, and Vertex stretches its APOL1 kidney bet as Viking posts obesity maintenance data and Washington installs an autism-research chair tied to leucovorin.

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Roche tees an IgAN fight with Novartis

Roche reported a phase 3 interim win for sefaxersen (formerly IONIS-FB-LRx), the Ionis-partnered antisense oligonucleotide that dials down complement factor B, in primary IgA nephropathy (IgAN). At a prespecified Week 37 look in the Imagination study, monthly subcutaneous injections reduced proteinuria versus placebo on 24-hour urine protein-to-creatinine ratio (UPCR), the primary endpoint. Safety and tolerability matched earlier trials. The study stays blinded for kidney-function change at Week 105. Roche plans regulator talks on next steps. Jay Garg, its global nephrology head, said in March that Week 37 proteinuria data would “hopefully lead to accelerated approval.” Commercial math diverges. Investor-relations head Bruno Eschli said street models then put 2030 sales near €400 million ($457 million). Roche has floated peak sales of €1 billion to €2 billion. Those forecasts predate the readout. The commercial foil is Novartis’s Fabhalta, also a factor B inhibitor, which won accelerated IgAN approval in 2024 and full authorization this year as an oral twice-daily pill. Sefaxersen is once-monthly under the skin. Phase 2 showed no cholesterol rise of the kind Fabhalta can cause. Roche paid Ionis $55 million upfront for the IgAN license in 2022, after an earlier option. (Fierce Biotech)

Amgen’s Horizon Sjögren’s bet clears first phase 3

Amgen said dazodalibep, a CD40-ligand fusion protein inherited with Horizon Therapeutics, met its primary endpoint in OASIZ 301, a 651-patient phase 3 in moderate-to-severe Sjögren’s disease. After 48 weeks, treated patients improved more than placebo on the EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI). Common side effects were mild-to-moderate nasal inflammation, urinary tract infection, hypertension, and infusion reactions. Dive notes Amgen gave no numeric delta, only “statistically significant and clinically meaningful,” with benefit from week 4 and no imbalance in clots or opportunistic infections. A second pivotal, OASIZ 303 (high symptom burden, milder systemic activity), is still out. William Blair expects FDA to want two positives. There is still no FDA-approved disease-modifying Sjögren’s therapy. Novartis’s ianalumab notched twin phase 3 wins last year. Sanofi and others have exited. (Fierce Biotech; BioPharma Dive)

Celldex’s hive antibody wins, then the market prices anaphylaxis

Celldex’s barzolvolimab, a KIT-blocking monoclonal aimed at mast cells, met primary and secondary endpoints in Embarq CSU1 and CSU2, twin phase 3 trials in treatment-refractory chronic spontaneous urticaria (CSU, the itchy hives condition). Across 1,939 patients, both dose regimens improved weekly urticaria activity score (UAS7) at week 12 versus placebo, with more complete responses at weeks 12 and 24. In the Xolair-resistant subgroup on the 150 mg every-four-weeks arm, complete response at week 12 reached 55.3% and 41.7% versus 9.3% and 15.1% on placebo across the two studies. Celldex plans an FDA filing in 2027. The catch is safety optics. Two patients had anaphylaxis after dosing. Overall discontinuation was about 16%, mostly consent withdrawals. BioPharma Dive says shares fell about 13% early Tuesday as investors weighed those cases against efficacy Cantor called among the best pivotal CSU sets yet. Celldex called incidence “extremely low” across roughly 2,400 treated patients. Stifel still sees meaningful sales even with a possible boxed warning. (Fierce Biotech; BioPharma Dive)

Vertex widens the APOL1 kidney map

Vertex posted phase 2b Amplified data for inaxaplin, its oral APOL1 channel inhibitor, in populations outside the severe-proteinuria Amplitude pivotal. In 22 people with APOL1-mediated kidney disease (AMKD) and modest proteinuria, urine albumin-to-creatinine ratio (UACR) fell a mean 42.7% at week 13. In 17 with AMKD plus Type 2 diabetes, UACR fell 17.3%. UPCR moves were similar (−44.7% and −25.4%). Vertex will discuss the data with regulators alongside Amplitude, now fully enrolled, with a preplanned interim expected early next year that could support an accelerated filing. Investor-relations commentary put Amplitude’s addressable pool near 150,000 in the U.S. and Europe, with an Amplified-style label adding about 100,000. Guggenheim recently put success odds at 65% in primary AMKD and 45% in the diabetes arm. Maze’s rival MZE829 remains the cross-trial foil. (Fierce Biotech)

Viking’s maintenance dosing, and a rare ALS hit

Viking said participants on dual GLP-1/GIP agonist VK2735 kept most of their weight loss after switching from weekly induction to less frequent shots. In a 180-person phase 2, 21 weeks of weekly dosing produced 16% to 19% mean weight loss by dose. After transition to every-other-week or monthly maintenance for 12 weeks, average retention ran 82% to 97% by regimen (about 90% for the broader biweekly group, 85% monthly). Shares jumped as much as 36% on the open. Separately, Otsuka and Ionis said ulefnersen hit its primary endpoint in Fusion, a phase 3 in FUS-mutation ALS (about 0.6% of ALS, often fast in the young). Among 73 patients in the primary analysis, the RNA drug beat placebo at week 72 on a composite of function and survival. Secondaries on a neurodegeneration biomarker and time to death, ventilation, rescue, or progression withdrawal also favored drug. Otsuka took worldwide rights for $10 million upfront in 2024 and says it will move urgently with regulators. Guggenheim sees small Ionis royalties ($14 million by 2030). (BioPharma Dive; Fierce Biotech)

Capital and geography footnotes

Novartis licensed an undisclosed preclinical radioligand from Suzhou’s BoomRay for up to $900 million in upfront, milestones, and royalties, a day after Telix’s ITM deal reset the radiopharma chessboard (noted yesterday, not re-led). BMS filed a New Jersey WARN for another 265 Princeton-tied jobs between December 2026 and May 2027, pushing Garden State cuts announced this year past 700 as it chases $2 billion in annual savings by end-2027. Separately, board chairs of AstraZeneca, Boehringer, Chiesi, Ipsen, GSK, Novo, Novartis, Roche, and Sanofi wrote EU leaders that Europe’s share of global pharma R&D has fallen to 31% from 43% in 1990, and that commercial clinical-trial share has halved in a decade to 9%, urging conditions that keep next-generation investment from draining to the U.S. and China. (Fierce Biotech; Fierce Pharma; Fierce Pharma)

Policy sidebar: autism chair and leucovorin

STAT’s ungated Morning Rounds reports that HHS secretary Robert F. Kennedy Jr. installed pediatric neurologist John Gaitanis as chair of the Interagency Autism Coordinating Committee, which helps steer roughly $2 billion in federal autism research allocation. Gaitanis recently began work at NIH’s child-health institute and co-founded Meadow Biosciences, a telehealth firm focused on getting leucovorin (folinic acid) to autistic children. President Trump promoted the drug at a news conference last year. STAT notes the clinical evidence that it improves speech remains sparse. Prior chair Sylvia Fogel stays on the council. (STAT News)

What to watch

pdufa.bio still lists Merck Winrevair (sotatercept) HYPERION (recently diagnosed PAH label update) as Awaiting after the September 21 goal date (days-to-decision −2), with no public decision logged yet. Ionis Zilganersen (ION373) for Alexander disease flipped to Decided on the September 22 goal date in the same API, but the feed carries no decision_source or approval-versus-CRL note yet. Could not confirm the letter from open copy overnight. Next dated PDUFA: Mirum/Incyte zilurgisertib (FOP) September 26. October: Roche Tecentriq adjuvant MSI-H/stage III colon (October 9), Merck/Daiichi I-DXd ES-SCLC (October 10), Roche Enspryng TED (October 15), Viatris/Opus MR-141 phentolamine ophthalmic (October 17). BioProcess International’s Boston conference continues through September 25. Overnight BPI hard-news remained thin beyond the same Sanofi Toronto Fluzone High-Dose facility and Merck KGaA Bengaluru hub notes. (pdufa.bio; BioProcess International)