The Pharma Closeout | Daily Pharmaceutical and Biotech News in 15 Minutes

Vera reports a 76% reduction in composite kidney-disease progression risk and near-stable two-year kidney function for TRUTAKNA (atacicept-vymj) in sponsor topline data, addressing the clinical-benefit question behind its accelerated approval in IgA nephropathy. Curium's BEXLUTRY (lutetium Lu 177 dotatate) brings an adult neuroendocrine-tumor competitor to Lutathera, with delivery and access questions still open. GSK buys a preclinical myeloma development bet, while Lilly backs nervous-system genetic-medicine delivery. We also examine Connect's mixed asthma data, portfolio changes, longer-term lung-cancer evidence and the FDA's trial-startup pilot. Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Enhertu delayed lung cancer progression in a Phase 3 trial, but immature survival results and fatal lung toxicity complicate the first-line case. Definium reports a second Phase 3 anxiety-study win for DT120, while new ivonescimab results quantify a survival benefit previously announced. We also cover Sanofi's proposed Cheplapharm transaction, Cellectis's gene-editing pivot, Corbus's oral obesity candidate and regulatory updates from Amgen and Telix. This is the September 14 edition, released September 15. Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

AstraZeneca's broader-use trial of Etcamah (camizestrant)missed its primary goal in advanced breast cancer, while the existing approval stands. Scholar Rock's ISEMBYLD (apitegromab) adds a muscle-directed option inspinal muscular atrophy, while Roivant's mosliciguat links vascular improvement with better walking distance in pulmonary hypertension associated with interstitial lung disease. Novartis faces pipeline and acquisition scrutiny asBiohaven pauses new BHV-7000 enrollment and Karyopharm confronts near-term financing pressure. Encoded's ETX101 results frame its Dravet syndromefinancing, and BioNTech's lung-cancer presentations lead the week ahead. Alex Mercer and Maya Patel host. Get the daily rundown inyour inbox — subscribe at https://thepharmacloseout.com.

FDA extended its review of Exelixis's zanzalintinib-Tecentriq combination for metastatic colorectal cancer to March 3, 2027, after classifying updated data as a major amendment. Karyopharm missed a $15.8 million loan payment and secured a creditor standstill through October 15 tied partly to its selinexor-ruxolitinib myelofibrosis filing. Roche's Enspryng gained Priority Review in MOGAD; COSCIENS bought oral-film manufacturer Nualtis, and Frazier raised more than $1.1 billion for its Public Fund. Plus, Telix's Pixclara FDA decision remained unverified at our reporting cutoff. Alex Mercer and Maya Patel host the Daily Roundup for September 11. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Biohaven's FDA partial hold pauses new epilepsy-trialenrollment; the company still expects RISE-3 results this year. AbbVie reports LUNA menstrual-migraine results for oral Qulipta (atogepant), while Shionogi misses a stroke endpoint and Disc Medicine reports fewer blood-removalprocedures in early polycythemia vera data. Novartis faces shareholder pressure, Solstice raises $225 million, and MannKind partners with Rose Pharma on inhaled weight management. NICE questions the value of Gilead's lenacapavir for HIV prevention, and new consensus guidance addresses type 1 diabetes screening. Alex Mercer and Maya Patel host the Daily Roundup forSeptember 10. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Bayer's Hyrnuo (sevabertinib) gains first-line accelerated approval in HER2-mutant lung cancer, joining an established rival. Permanent FDA center leaders follow repeated turnover, while Structure's experimental obesity pill aleniglipron adds 72-week follow-up without a concurrent placebo. Kura, EMD Serono and Encoded back new development efforts; Tyra reports early bladder cancer responses, Evommune changes its eczema program, and Beeline advances lupus studies. BioMarin's Voxzogo (vosoritide) filing targets hypochondroplasia, and Nanoscope reports application acceptance for severe retinitis pigmentosa. Alex Mercer and Maya Patel host the Daily Roundup for September 9. Get the daily rundown in your inbox — subscribe at thepharmacloseout.com .

Novartis's del-desiran missed HARBOR's primary endpoint in myotonic dystrophy, adding pressure to a growth plan already facing setbacks. Roivant and Pulmovant's mosliciguat data advance Monday's PHocus preview, while AstraZeneca, Pharvaris, Amgen, Inhibrx and Bristol Myers add clinical updates across lung disease, hereditary angioedema and cancer. BrainChild Bio's financing, the proposed Sernova-Seraxis merger and Intellia's lonvo-z Priority Review round out the slate. Two resolved Rasonque access cases show why Revolution Medicines' pancreatic cancer launch requires more than approval: patients still need to navigate authorization, support and dispensing. Alex Mercer and Maya Patel host the Daily Roundup for September 8. Get the daily rundown in your inbox — subscribe at thepharmacloseout.com .

Novo is ending the HERMES and ATHENA ziltivekimab trials after a monitoring committee judged them unlikely to succeed, leaving ARTEMIS as its remaining Phase 3 cardiovascular path. We examine ADARx's IPO filing, the Aptar-Aceso ACT-101 collaboration in cystic fibrosis, and STEP Young semaglutide results in children ages six to under twelve. SynAct's resomelagon reanalysis asks what another rheumatoid arthritis trial must prove after two primary-endpoint misses. This late Labor Day edition reflects information available through Monday and previews Roivant and Pulmovant's PHocus mosliciguat data in pulmonary hypertension associated with interstitial lung disease. Alex Mercer and Maya Patel host the Daily Roundup for September 7. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Bristol Myers Squibb told regulators in early June. The rest of us found out this week, after an analyst note. Three months sat between those two facts, and that gap is the story of the week. Also this week, a cardiovascular outcomes trial in more than eight thousand patients missed its endpoint and put a whole drug class in question. A mid-stage lupus readout took fifty five percent off a company in a day. And the FDA approved a cancer drug over its own advisers, while memos surfaced on a second approval it had granted over its own reviewers. What do you do with a week like that? Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Novartis's pelacarsen missed its cardiovascular endpoint despite lowering Lp(a)—what should Amgen and Lilly rethink? FDA accelerated approval brings AstraZeneca's Etcamah (camizestrant) to ESR1-mutated, HR-positive, HER2-negative advanced breast cancer before progression, with confirmatory benefit still required. Following Monday's CAR-T coverage, new reporting traces Bristol Myers Squibb's notifications to early June, months before public confirmation. Also: Revolution Medicines' Rasonque (daraxonrasib) data in previously treated RAS-mutant lung cancer, and Superluminal's $60 million financing for rare genetic and hypothalamic obesity. Alex Mercer and Maya Patel host the Daily Roundup for September 4. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

AbbVie's BCMA x CD3 bispecific etentamig met both primary endpoints in the Phase 3 CERVINO myeloma study — a 74.0% response rate versus 45.7% for standard therapy and a 60% reduction in the risk of progression or death — with a safety profile AbbVie is positioning for outpatient and community care. GSK is paying HUTCHMED $110 million upfront — up to $1.295 billion in total — for HMPL-A830, a dual KRAS/EGFR antibody-targeted conjugate whose global Phase 1 hasn't started yet. Also: the FDA approved Ionis' Zanvastro (zilganersen), the first treatment for Alexander disease, two weeks ahead of its PDUFA date. Plus Gan & Lee licenses bofanglutide (GZR18) to Menarini across 39 European countries (EUR 62M upfront, up to EUR 726M total), and Cipla partners with Qilu on QL2107, a Keytruda (pembrolizumab) biosimilar for the US. Alex Mercer and Maya Patel host the Daily Roundup for September 3. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

uniQure filed AMT-130 for FDA and UK approval on three-year Phase 1/2 data with a propensity-matched external control, and a potential accelerated-approval action date in late April or early May 2027. The same day, Ultragenyx's Phase 3 Aspire study of apazunersen in Angelman syndrome missed both its primary and key secondary endpoints, and Akeso reported that ivonescimab met overall survival against pembrolizumab in first-line PD-L1-positive lung cancer. Also: Teva's anti-IL-15 antibody hits in a Phase 2a celiac study, Alteogen options its subcutaneous platform to Novartis for up to $3.223 billion, nine more manufacturers join the White House MFN pricing deals, and newly released memos show a CBER office director overruled her review team to approve Replimune's melanoma drug. Alex Mercer and Maya Patel host the Daily Roundup for September 2. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Alumis shares fell 55% after envudeucitinib missed every endpoint in a mid-stage systemic lupus study - and the CEO still sees a path to Phase 3 built on a prespecified interferon-high subgroup. We unpack what that pivot costs and why the whole TYK2 class is now a show-me story outside psoriasis. Also: Roche pays Simcere Zaiming $75 million upfront in a deal worth up to $1.53 billion for trispecific CD79a/CD19/CD3 antibody SIM0660, and Novartis's Rhapsido (remibrutinib) clears two Phase 3 multiple sclerosis trials with no Hy's Law liver cases - the signal that sank Sanofi's tolebrutinib. Plus Kazia Therapeutics reports paxalisib cutting tumor burden 52% in immunotherapy-resistant MSS/pMMR colorectal cancer models ahead of a five-arm Phase 2. Alex Mercer and Maya Patel host the Daily Roundup for September 1. Get the daily rundown in your inbox - subscribe at https://thepharmacloseout.com.

Two of the biggest names in cell therapy just hit the brakes on the most ambitious idea in immunology - Novartis and Bristol Myers Squibb have paused their autoimmune CAR-T trials after serious immune reactions, with trade press reporting three deaths in CAR-T trials. Also today: nine more drugmakers sign on to most-favored-nation pricing, and the FDA hands down two blood-disorder approvals in one week - Takeda's Mimrylo and PharmaEssentia's Besremi - that just turned the myeloproliferative space into a live competitive collision. Plus: Eli Lilly buys Merida Biosciences, and Jazz's Ziihera scores a second survival win. Alex Mercer and Maya Patel host the Daily Roundup for August 31. Get the daily rundown in your inbox - subscribe at https://thepharmacloseout.com.

Three hundred eighty-one events versus three hundred ninety-two — AstraZeneca and Ionis's Wainua (eplontersen) missed in ATTR-CM, and the Munich post-mortem points at background therapy, not the molecule. With roughly four in five patients on a stabilizer, the trial answered an add-on question it was never built to ask — while at the same congress Alnylam put up a prespecified HELIOS-B subgroup of vutrisiran by baseline tafamidis use (259 of 654 randomized and treated patients), published in the Journal of the American College of Cardiology. Also this week: FDA approved Eli Lilly's Mounjaro (tirzepatide) for cardiovascular risk reduction in type 2 diabetes on a head-to-head trial against Trulicity (dulaglutide); BioNTech and Genentech terminated a Phase 2 of autogene cevumeran in colorectal cancer on an overall survival imbalance; Novartis pushed pacibekitug toward an outcomes trial in chronic kidney disease; and Teva bid for BioXcel's dexmedetomidine film out of bankruptcy. Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Cytokinetics' aficamten hit both primary endpoints in non-obstructive hypertrophic cardiomyopathy — a 3.0-point symptom score gain — and the stock still fell about 6%. We break down what that effect size means for the Q4 sNDA and the pricing fight ahead, plus Moderna's upsized $2.6 billion convertible for oncology, BioNTech and Genentech terminating a Phase 2 of autogene cevumeran after a survival imbalance, and Roche's $45 million upfront for DualityBio's DUPAC ADC payload platform. Also: FDA clears Lisraya (brepocitinib) in dermatomyositis at $35,000 a month, Wainua (eplontersen) misses in TTR cardiomyopathy and amplifies pressure on Alnylam, and Teva's stalking-horse bid for BioXcel's dexmedetomidine sublingual film as BioXcel shares collapse 66%. Alex Mercer and Maya Patel host the Daily Roundup for August 28. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Roivant and Priovant won FDA approval for LISRAYA (brepocitinib) in dermatomyositis — 55% of patients achieved both symptom improvement and minimal steroid use at 52 weeks. But the label carries a full JAK boxed warning, which makes the steroid-sparing case the entire commercial argument. Also: Spyre Therapeutics' anti-TL1A antibody missed in rheumatoid arthritis, erasing more than $1 billion in market value; Amgen and AstraZeneca's Tezspire (tezepelumab) hit all endpoints in Phase 3 eosinophilic esophagitis, setting up a fight with Dupixent; CStone's CS2009 trispecific posted deep responses in first-line lung cancer; and Verrica and Corvus each advanced their programs. Alex Mercer and Maya Patel host the Daily Roundup for August 27. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

Revolution Medicines filed on July 22nd and had FDA approval just over a month later — a five-week review for a pancreatic cancer drug that nearly doubled median survival. Rasonque (daraxonrasib) showed 13.2 months versus 6.7 on chemotherapy in Phase 3 and cleared under the Commissioner's National Priority Voucher program, with RBC modeling roughly $11.5 billion in peak sales for a company that has never commercialized a product. Also in this episode: Spyre Therapeutics shares fell 13% after anti-TL1A antibody SPY072 missed in rheumatoid arthritis, narrowing the mechanism back toward inflammatory bowel disease where Merck, Roche and Sanofi are already in Phase 3. And Biohaven sold global rights to focal epilepsy drug opakalim to SK Biopharmaceuticals for up to $795 million ahead of a year-end pivotal readout. Alex Mercer and Maya Patel host the Daily Roundup for August 26. Get the daily rundown in your inbox — subscribe at https://thepharmacloseout.com.

The FDA cleared Jazz Pharmaceuticals' Ziihera (zanidatamab) in first-line HER2-positive gastroesophageal adenocarcinoma with median overall survival of 26.4 months — but the second regimen on that same label has never shown a significant survival benefit, and the analysis that settles it is due by the end of September. Alex and Maya dig into the label breadth the FDA granted, the boxed warning for diarrhea, and the $250 million milestone Zymeworks just banked on approval. Plus: McKesson buys Precision Medicine Group for $2.25 billion, pushing a drug distributor deep into trial design and commercialization. And Sentivera — the ARCH and Population Health startup founded after Metsera — licenses a preclinical Type 2 inflammation asset from Haisco Pharmaceutical in a deal worth up to $1.5 billion. Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at thepharmacloseout.com.

Regenxbio shares plunged 26% to $7.96 after the FDA placed a second clinical hold on RGX-121 (clemidsogene lanparvovec) — but the real trigger was the company abandoning its Hunter syndrome BLA resubmission. Spine MRI findings in five CAMPSIITE participants dosed three to six years ago surfaced through expanded monitoring Regenxbio built itself. Also: Roche's Genentech pays Hanmi $190 million up front, up to $2.3 billion total, for UCN2 analog HM17321 in muscle-sparing obesity; courts reject Merck and AstraZeneca challenges to Medicare price negotiation; the FDA action date lands for Biogen and Eisai's Leqembi Iqlik (lecanemab-irmb) subcutaneous starting dose; and Capricor's deramiocel Duchenne decision moves to November 22 behind a refined upper-limb indication. Alex Mercer and Maya Patel host. Get the daily rundown in your inbox — subscribe at thepharmacloseout.com.