Pharma and biotech news, week of Aug 24 – Aug 30, 2026

The U.S. FDA has approved Jazz Pharmaceuticals' Ziihera (zanidatamab-hrii) regimens with and without Tevimbra (tislelizumab-jsgr) plus chemotherapy for first-line HER2-positive advanced gastroesophageal adenocarcinoma. Supported by Phase 3 trial results, the approval triggers a $250 million milestone payment to Zymeworks from Jazz. Zymeworks remains eligible for additional milestone payments and royalties while advancing its pipeline.AI summary

Ionis Pharmaceuticals partner GSK announced that Hibsago (bepirovirsen) has been approved in Japan. It is approved as the first and only functional cure for chronic hepatitis B.AI summary

Roivant announced that the U.S. FDA has approved LISRAYA (brepocitinib) 30 mg for the treatment of adults with dermatomyositis. Approval was based on data from the landmark Phase 3 VALOR trial, demonstrating improvements in disease activity and steroid-sparing benefits. The once-daily oral pill is available immediately in the U.S., and Roivant will host an investor call tomorrow.AI summary

The U.S. FDA has approved Revolution Medicines' oral tablet RASONQUE (daraxonrasib) for adults with metastatic pancreatic adenocarcinoma who received prior systemic therapy or cannot receive multiagent systemic therapy. Approval was based on Phase 3 RASolute 302 trial data showing RASONQUE reduced the risk of death by 60% compared to chemotherapy. The once-daily drug is now available in the U.S.AI summary

The FDA has approved Johnson & Johnson's IMAAVY (nipocalimab-aahu) as the first therapy specifically for warm autoimmune hemolytic anemia (wAIHA) in patients 12 and older previously or currently treated with corticosteroids. In the Phase 2/3 ENERGY trial, approximately three times as many patients on the approved dose achieved a durable hemoglobin response by 24 weeks compared to placebo. Johnson & Johnson offers patient support through its IMAAVY withMe program.AI summary

The FDA approved a new drug application from Takeda Pharmaceuticals America, Inc. for Mimrylo (rusfertide) for injection. The approval provides for the treatment of erythrocytosis in adults with polycythemia vera. PDUFA goal date: 2026-08-31.AI summary

The FDA has approved a supplemental biologics license application for PharmaEssentia Corporation's Besremi (ropeginterferon alfa-2b-njft) injection. The approved supplement provides for a new indication for treating adults with essential thrombocythemia. PDUFA goal date: 2026-08-30.AI summary

The FDA approved the supplemental Biologics License Application for Pfizer and BioNTech's 2026-2027 COVID-19 vaccine formula targeting the XFG variant (COMIRNATY XFG) for adults ages 65 and older and for individuals ages 5 through 64 with at least one high-risk underlying condition. The approval is based on cumulative evidence, including manufacturing and non-clinical data showing strong immune responses against currently circulating variants. Shipping will begin immediately, with availability in pharmacies, hospitals, and clinics across the U.S. in the coming days.AI summary

The FDA approved Gilead Sciences, Inc.'s new drug application for the Bixlenvo Sunlenca Starter Pack. The co-packaged product provides for initiating Bixlenvo to treat HIV-1 infection in virologically suppressed adults replacing their current stable antiretroviral regimen. PDUFA goal date: 2026-08-27.AI summary

The FDA approved Gilead Sciences, Inc.'s new drug application for Bixlenvo (bictegravir and lenacapavir) tablets. The approved product is a fixed-dose combination complete regimen to treat HIV-1 in virologically suppressed adults replacing their current stable antiretroviral regimen.AI summary

BioCryst Pharmaceuticals announced that Japan's Ministry of Health, Labor and Welfare has approved once-daily ORLADEYO for prophylactic therapy in pediatric hereditary angioedema patients aged 2 to <12 years. The approval is based on positive interim results from the APeX-P trial. BioCryst plans to launch the drug in Japan following the completion of the National Health Insurance System reimbursement pricing process.AI summary

The FDA has extended the PDUFA target action date for Capricor Therapeutics' Biologics License Application for Deramiocel from August 22, 2026, to November 22, 2026. The three-month extension follows Capricor's submission of a major BLA amendment containing 24-month open-label extension data from the Phase 3 HOPE-3 study. Capricor submitted the new data to support a refined proposed indication focused on upper limb function.AI summary

Japan's Ministry of Health, Labour and Welfare granted marketing authorization to Rhythm Pharmaceuticals' IMCIVREE (setmelanotide) for acquired hypothalamic obesity. The approval was supported by positive Phase 3 TRANSCEND trial data showing a 18.8% placebo-adjusted BMI reduction. Rhythm expects to launch the drug in Japan before the end of 2026.AI summary

Celcuity Inc. has submitted a supplemental New Drug Application to the U.S. FDA for REVTORPYK (gedatolisib) to treat HR+/HER2- locally advanced or metastatic breast cancer with a PIK3CA mutation. The filing is supported by Phase 3 VIKTORIA-1 trial data, where REVTORPYK regimens demonstrated a 49% to 50% reduction in the risk of disease progression or death compared to alpelisib plus fulvestrant. Celcuity aims to work with the FDA to potentially expand the drug's label to include PIK3CA-mutated patients.AI summary

ADARx Pharmaceuticals has received FDA Fast Track designation for onvuzosiran, an investigational siRNA therapeutic candidate for the prevention of hereditary angioedema (HAE) attacks. The treatment is designed to inhibit prekallikrein generation at the mRNA level. Onvuzosiran is currently being evaluated in the Phase 3 STOP-HAE clinical trial.AI summary

The US FDA has accepted a supplemental Biologics License Application for Jemperli (dostarlimab) for priority review to treat patients with previously untreated stage II and III dMMR/MSI-H locally advanced rectal cancer. The submission is supported by Phase 2 AZUR-1 trial data demonstrating a sustained clinical complete response for 12 months. The FDA assigned a PDUFA action date of February 2027.AI summary

The FDA cleared Adicet Bio's Investigational New Drug application for ADI-212 to treat patients with metastatic castration-resistant prostate cancer. ADI-212 is an allogeneic gamma delta cell therapy targeting prostate-specific membrane antigen. Adicet plans to begin Phase 1 trial enrollment in the fourth quarter of 2026.AI summary

The U.S. FDA has placed a clinical hold on REGENXBIO's investigational gene therapy RGX-121 after asymptomatic spine MRI findings were discovered in five participants in the CAMPSIITE study. Consequently, REGENXBIO does not expect to resubmit the RGX-121 Biologics License Application in the near term. The company and partner NS Pharma are evaluating additional imaging and follow-up data to determine next steps.AI summary

Arrowhead Pharmaceuticals presented results from two Phase 3 studies, SHASTA-3 and SHASTA-4, showing plozasiran reduced median triglyceride levels by 79% and 81% at Month 12 in severe hypertriglyceridemia patients. A pooled analysis also demonstrated a 78% reduction in acute pancreatitis events compared to placebo. Arrowhead plans to submit a supplemental New Drug Application to the FDA by year-end 2026.AI summary

Cytokinetics announced positive primary results from the pivotal Phase 3 ACACIA-HCM trial of aficamten in patients with symptomatic non-obstructive hypertrophic cardiomyopathy. Treatment demonstrated statistically significant improvements in both dual primary endpoints, KCCQ-CSS and pVO2, at 36 weeks. Based on these findings, Cytokinetics plans to submit a supplemental New Drug Application to the FDA in the fourth quarter of 2026.AI summary

Amgen and AstraZeneca announced positive top-line results from the Phase 3 CROSSING trial evaluating TEZSPIRE in patients with eosinophilic esophagitis. TEZSPIRE demonstrated statistically significant and clinically meaningful improvements across co-primary endpoints, histologic remission and dysphagia frequency and severity, at week 24, which were sustained through week 52. Full results will be shared with regulatory authorities and presented at an upcoming medical meeting.AI summary

Summit Therapeutics announced positive topline results from Akeso's Phase III HARMONi-GI1 trial evaluating ivonescimab plus chemotherapy in first-line advanced biliary tract cancer. The ivonescimab regimen achieved statistically significant superiority in overall survival compared to durvalumab plus chemotherapy, while also meeting key secondary endpoints of progression-free survival and objective response rate. Detailed safety and efficacy data will be presented at an upcoming medical congress and published in a peer-reviewed journal.AI summary

Priovant Therapeutics announced the publication of skin-specific outcomes from the Phase 3 VALOR trial evaluating brepocitinib in dermatomyositis in JAMA Dermatology. Brepocitinib 30 mg demonstrated rapid, durable, and clinically meaningful improvements across skin disease activity, itch, and skin-related quality of life compared to placebo through Week 52. These published results reinforce the drug's potential as a treatment for cutaneous manifestations of dermatomyositis.AI summary

Arrowhead Pharmaceuticals announced it will present new clinical data at the European Society of Cardiology Congress. The presentations include 12-month Phase 3 results for plozasiran in severe hypertriglyceridemia and Phase 3 trial data for zodasiran in Chinese patients with homozygous familial hypercholesterolemia. Arrowhead will host an investor webcast on August 31, 2026, to review the data.AI summary

BioNTech will terminate the Phase 2 BNT122-01 trial of autogene cevumeran in ctDNA-positive, resected Stage II (high risk) or Stage III colorectal cancer after a new recommendation from the independent DSMB. The DSMB identified a numerical overall survival imbalance between arms and said further continuation was unlikely to change the efficacy outcome; no new safety signals were identified. BioNTech will analyze the trial data, and the IMcode003 pancreatic trial continues as planned.AI summary

Spyre Therapeutics announced topline results from the rheumatoid arthritis sub-study of its Phase 2 SKYWAY trial evaluating SPY072. Both doses demonstrated statistically significant benefits compared to placebo on primary or secondary endpoints and were well tolerated. However, the magnitude of effect did not meet targets to prioritize monotherapy development in rheumatoid arthritis, though data for psoriatic arthritis and axial spondyloarthritis remain on track for Q4 2026.AI summary

Alkermes announced the publication of phase 2 Vibrance-1 study data for alixorexton in adults with narcolepsy type 1 in The Lancet Neurology. Once-daily alixorexton demonstrated statistically significant improvements in excessive daytime sleepiness and cataplexy, and was generally well tolerated. Alkermes has initiated the global phase 3 Brilliance Studies evaluating alixorexton in adults with narcolepsy type 1 and type 2.AI summary

Precision BioSciences has dosed the first patient in the Phase 1/2 FUNCTION-DMD clinical trial evaluating PBGENE-DMD. The in vivo gene editing candidate is designed for patients with Duchenne muscular dystrophy who have mutations between exons 45 and 55. Precision BioSciences expects to report initial safety data from the trial by year-end 2026.AI summary

TRexBio announced positive unblinded topline results from the Phase 1a portion of its Phase 1a/b trial of TRB-061 in healthy participants. The candidate was generally well tolerated, showed dose-proportional pharmacokinetics, and selectively activated and expanded immunoregulatory Tregs. These results support dose selection for the ongoing Phase 1b trial in patients with moderate-to-severe atopic dermatitis, with topline data expected in mid-2027.AI summary

Ascendis Pharma announced an oral presentation of Week 104 data from the pivotal ApproaCH trial of once-weekly TransCon CNP in children with achondroplasia at ISDS 2026. The long-term data demonstrated durable improvements in height and physical functioning with a safety profile similar to placebo. Ascendis also anticipates a European regulatory decision for YUVIWEL in the fourth quarter of 2026.AI summary

PTC Therapeutics announced that multiple Sephience (sepiapterin) clinical and real-world data presentations will be featured at the 2026 SSIEM Annual Symposium. New analyses from the AMPLIPHY study showed a 100% greater reduction in blood Phe for participants switching from sapropterin to Sephience. The company will present 17 abstracts and presentations at the event.AI summary

AstraZeneca's subsidiary, AstraZeneca Finance LLC, priced four tranches of Eurobonds totaling €2.55 billion. The offering includes fixed rate notes maturing between 2030 and 2038 with coupons ranging from 3.402% to 4.169%. The transaction is expected to close on September 1, 2026, with proceeds intended for general corporate purposes.AI summary

Jazz Pharmaceuticals announced the pricing of an upsized private offering of $1.1 billion aggregate principal amount of 1.875% exchangeable senior notes due 2032. Concurrently, the company agreed to repurchase approximately $225.0 million of its ordinary shares using cash on hand. The offering is expected to close on August 31, 2026, with net proceeds of approximately $1,079.0 million intended for general corporate purposes.AI summary

Jazz Pharmaceuticals announced that its subsidiary intends to offer $1.0 billion of exchangeable senior notes due 2032 in a private offering, with an option for initial purchasers to buy up to an additional $150.0 million in notes. Concurrently, Jazz expects to repurchase up to $225.0 million of its ordinary shares. The company plans to use the net proceeds from the offering for general corporate purposes.AI summary

BioXcel Therapeutics entered into an asset sale agreement with Teva Pharmaceuticals for substantially all of its assets. Teva will act as the stalking horse bidder in a court-supervised Chapter 11 auction process. BioXcel secured a commitment for $19 million in debtor-in-possession financing to support ongoing operations during the sale.AI summary

argenx has completed its acquisition of Forte Biosciences, Inc., through a cash tender offer of $77.00 per share. The transaction adds FB102, a first-in-class anti-CD122 antibody, to argenx's immunology pipeline. Following the merger, Forte became a wholly owned subsidiary of argenx and its stock will no longer trade on Nasdaq.AI summary

Elanco Animal Health completed a 25,000-square-foot expansion of its Elwood, Kansas manufacturing facility to boost its monoclonal antibody production. The $120 million investment increases site production capacity fourfold and supports products like Befrena and Trutect. An additional $30 million investment is planned through 2028 for warehousing, bringing the total investment to $150 million.AI summary

Biohub researchers developed an AI-based system to model malignant cell states and predict targeted combination therapies to prevent cancer drug resistance. In mouse models of diffuse midline glioma, combining the targeted drugs avapritinib and ruxolitinib produced synergistic effects and doubled survival compared to monotherapy. Biohub is now working with partners to launch a clinical trial for the combination regimen.AI summary