Pharma and biotech news, week of Aug 31 – Sep 6, 2026

The US FDA has granted accelerated approval to AstraZeneca's Etcamah (camizestrant) in combination with a CDK4/6 inhibitor for adults with HR-positive, HER2-negative advanced breast cancer with an emergent ESR1 mutation. Approval was based on the SERENA-6 Phase III trial, where the combination reduced the risk of disease progression or death by 56%. The trial will continue evaluating overall survival as a key secondary endpoint.AI summary

China's National Medical Products Administration has approved the subcutaneous autoinjector formulation of LEQEMBI (lecanemab) as an initiation treatment for early Alzheimer's disease. The approval provides a once-weekly at-home administration option based on Phase 3 Clarity AD study data. Eisai plans to launch the subcutaneous formulation in China during its fiscal year 2026, ending March 31, 2027.AI summary

The European Commission has approved AstraZeneca and Daiichi Sankyo’s Enhertu in combination with pertuzumab for the first-line treatment of adult patients with unresectable or metastatic HER2-positive breast cancer. In the Phase 3 DESTINY-Breast09 trial, the combination reduced the risk of disease progression or death by 44% compared to THP, extending median progression-free survival to 40.7 months. The approval triggers a $100 million milestone payment from AstraZeneca to Daiichi Sankyo.AI summary

The U.S. FDA has approved Takeda's MIMRYLO (rusfertide) to treat erythrocytosis in adults with polycythemia vera. Approval was based on data from the Phase 3 VERIFY trial, where 76.9% of patients achieved a clinical response during Weeks 20-32. Takeda is now working with international regulators to bring MIMRYLO to patients worldwide.AI summary

The FDA has approved Ionis Pharmaceuticals' ZANVASTRO (zilganersen) as the first disease-modifying treatment for Alexander disease in pediatric and adult patients. Approval was based on pivotal study data showing statistically significant stabilization of gait speed at Week 61. ZANVASTRO will be available in the U.S. in the coming weeks.AI summary

China's National Medical Products Administration has approved Milestone Pharmaceuticals' etripamil nasal spray for the treatment of paroxysmal supraventricular tachycardia in adults. The decision triggers a $2.0 million milestone payment to Milestone from Everest Medicines. Everest will lead commercialization efforts in Greater China.AI summary

PDUFA goal date 2026-09-03. Outcome: withdrawn.

Scholar Rock announced that the FDA has granted Fast Track and Orphan Drug designations to apitegromab for facioscapulohumeral muscular dystrophy (FSHD). Additionally, participant dosing is now underway in the Phase 2 FORGE clinical trial. The study will enroll approximately 60 adult participants to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of apitegromab.AI summary

aTyr Pharma provided an update regarding the timing of a regulatory response from the FDA. Based on FDA feedback, the company anticipates receiving a response by mid-September 2026 for a protocol submitted in June 2026. The submission is for a planned global Phase 3 trial of efzofitimod in patients with pulmonary sarcoidosis.AI summary

Canada’s Drug Agency has issued a final positive recommendation supporting public reimbursement of LEQEMBI (lecanemab) for eligible early Alzheimer’s disease patients. The recommendation follows a CDA-AMC reconsideration. Next steps include negotiations via the pan-Canadian Pharmaceutical Alliance, followed by provincial and territorial public drug plan reimbursement decisions.AI summary

Ionis Pharmaceuticals and Novartis announced topline results from the Phase 3 Lp(a)HORIZON study of pelacarsen. The trial did not meet its primary endpoint of reducing cardiovascular event risks compared to placebo, though substantially lower Lp(a) levels were achieved. The full study data will be presented at an upcoming medical congress.AI summary

Novartis announced that its Phase III Lp(a)HORIZON trial evaluating pelacarsen in patients with elevated Lp(a) and established cardiovascular disease did not meet its primary endpoint of reducing cardiovascular events compared to placebo. Although lower Lp(a) levels were achieved, this did not translate to reduced cardiovascular risk in the overall study population. The trial data will be presented at an upcoming medical congress.AI summary

Summit Therapeutics noted that partner Akeso reported positive overall survival results from the Phase III HARMONi-2 study evaluating ivonescimab monotherapy against pembrolizumab monotherapy. Ivonescimab demonstrated a statistically significant overall survival improvement in patients with PD-L1-positive locally advanced or metastatic non-small cell lung cancer. Results from this preplanned analysis will be presented at an upcoming medical conference.AI summary

Telix has completed patient enrollment of 350 patients in the Phase 3 BiPASS study of Illuccix and Gozellix for prostate cancer imaging in the pre-biopsy setting. Telix has also aligned with the FDA on a New Drug Application pathway for BiPASS that, if approved, would support reimbursement as a new product and broaden patient access. BiPASS is designed to evaluate the diagnostic performance of 68Ga-PSMA-11 PET combined with MRI for detection of prostate cancer prior to biopsy.AI summary

Novartis announced positive topline results from its Phase III REMODEL-1 and REMODEL-2 trials evaluating oral remibrutinib in relapsing multiple sclerosis. The studies met their primary endpoint, showing significant reductions in annualized relapse rate compared to teriflunomide with a favorable safety profile. Novartis plans to present late-breaking data at MSToronto2026 and seek global regulatory approvals.AI summary

Orca Bio announced that three clinical data and study updates will be presented at the 14th Annual Meeting of the Society of Hematologic Oncology. The presentations feature infection data comparing TREGZI to conventional alloHSCT from the Phase 3 Precision-T trial, preliminary profile data for Orca-Q, and the design of the Serene-T trial. Presentation copies will be available on the company's website.AI summary

Elevar Therapeutics presented a post hoc analysis from the Phase 3 CARES-310 study evaluating camrelizumab plus rivoceranib in unresectable hepatocellular carcinoma. The combination demonstrated improved progression-free survival and overall survival compared to sorafenib across age subgroups. Elevar is addressing manufacturing deficiencies noted in a recent FDA Complete Response Letter to prepare for regulatory resubmission.AI summary

AstraZeneca and HUTCHMED announced positive high-level results from the SANOVO Phase III trial in China. The combination of Tagrisso and Orpathys demonstrated a statistically significant and highly clinically meaningful improvement in progression-free survival compared to Tagrisso alone in 1st-line MET-overexpressing EGFR-mutated non-small cell lung cancer. The data will be presented at an upcoming medical meeting and shared with regulatory authorities.AI summary

Jazz Pharmaceuticals announced second interim overall survival results from the Phase 3 HERIZON-GEA-01 trial evaluating Ziihera (zanidatamab-hrii) plus chemotherapy, with and without Tevimbra (tislelizumab-jsgr), in first-line HER2-positive advanced gastroesophageal adenocarcinoma. The Ziihera combinations demonstrated statistically significant and clinically meaningful overall survival improvements versus trastuzumab plus chemotherapy. Jazz plans to present the data at a medical meeting in the fourth quarter of 2026 and submit them to global regulatory authorities.AI summary

Amgen presented new data from a pre-specified secondary analysis of the Phase 3 VESALIUS-CV trial evaluating Repatha in high-risk adults without a prior heart attack or stroke. Repatha reduced the risk of all-cause death by 20% when added to statins or other LDL-C-lowering treatments. Amgen also presented real-world analyses highlighting persistent gaps in lipid management.AI summary

Roivant and Pulmovant announced that topline results from the Phase 2 PHocus study of mosliciguat in patients with PH-ILD will be presented at the ERS Congress 2026 on September 8, 2026. Marc Humbert will present the data during the conference. Roivant will also host an investor call and webcast the same morning.AI summary

FORE Biotherapeutics announced a positive outcome from the first planned interim efficacy analysis of its Phase 2 FORTE basket study evaluating plixorafenib monotherapy in advanced solid tumors with BRAF fusions. The Independent Data Monitoring Committee recommended the study continue as planned. Fore expects to report topline results from this basket in the second half of 2027.AI summary

GSK presented positive phase II trial data for its investigational mRNA seasonal influenza vaccine candidate, FLUm3HA.b-3NA. The candidate demonstrated higher immune responses against all tested strains compared to standard and high-dose licensed vaccines, with acceptable safety. Based on these results, GSK plans to initiate a phase III efficacy trial in September 2026.AI summary

Alumis announced topline results from its Phase 2b LUMUS trial of envudeucitinib in systemic lupus erythematosus, which failed to meet its primary and secondary endpoints in the overall trial population. However, robust responses were observed in a prespecified subgroup of patients with a high interferon gene signature. Alumis plans to engage regulators to discuss Phase 3 development for envudeucitinib in SLE.AI summary

Stoke Therapeutics and Biogen presented long-term Phase 1/2a open-label extension data for zorevunersen in Dravet syndrome at the 16th European Epilepsy Congress. Results showed four-year reductions in seizures and improvements in cognition and behavior, while remaining generally well tolerated. A Phase 3 EMPEROR study data readout is anticipated in Q3 2027.AI summary

The New England Journal of Medicine published Phase 1/2 clinical data for Revolution Medicines' RASONQUE (daraxonrasib) in patients with previously treated advanced RAS mutant non-small cell lung cancer. In a subgroup receiving 160 mg to 220 mg doses, RASONQUE achieved a confirmed objective response rate of 42% and median progression-free survival of 8.3 months. These results support the ongoing global Phase 3 RASolve 301 trial.AI summary

Opus Genetics announced plans to present topline clinical data from Cohort 1 of its Phase 1/2 study of OPGx-BEST1. The data will be shared during an investor conference call and webcast on September 9, 2026. Full clinical datasets and additional analyses will also be presented at several upcoming medical conferences.AI summary

Stoke Therapeutics and Biogen announced data presentations at the 16th European Epilepsy Congress showing 4-year results from Phase 1/2a open-label extension studies of zorevunersen in Dravet syndrome. Results demonstrated sustained improvements in cognition and behavior, durable seizure reductions, and a generally well-tolerated safety profile. Results from the global, pivotal Phase 3 EMPEROR study are anticipated in the third quarter of 2027.AI summary

Kymera Therapeutics announced it will present posters featuring previously reported Phase 1b data for its oral STAT6 degrader, KT-621, at upcoming ERS and EADV congresses. KT-621 is currently in parallel Phase 2b testing for atopic dermatitis and asthma. Topline data for the BROADEN2 trial in atopic dermatitis are expected by year-end 2026, with Phase 3 trials planned for mid-2027.AI summary

Adicet Bio has completed the last patient visit for a planned Phase 1 analysis of prula-cel in systemic lupus erythematosus. Topline safety and efficacy results from 22 patients are expected in September 2026. Additionally, the FDA cleared an IND application for ADI-212 in metastatic castration-resistant prostate cancer, with a Phase 1 study launching in late 2026.AI summary

Kyverna reaffirmed the safety profile of its fully human CD19 CAR T-cell therapy, miv-cel, across autoimmune indications following third-party reports. Data from over 100 treated patients showed a consistent profile with no high-grade CRS, ICANS, or reported cases of IEC-HS. Kyverna expects to complete its rolling BLA submission for SPS in the fourth quarter and complete Phase 3 gMG trial enrollment by mid-2027.AI summary

AbbVie completed its acquisition of Apogee Therapeutics, with Apogee shareholders receiving $135.11 per share in cash, for a total equity value of approximately $10.9 billion. Apogee's lead asset is zumilokibart, a late-stage, half-life extended monoclonal antibody targeting IL-13, in development for atopic dermatitis. AbbVie expects the acquisition to negatively impact adjusted diluted EPS by $0.14 in 2026 and approximately $0.46 in 2027, with accretion beginning in 2032.AI summary

Vertex Pharmaceuticals has completed its acquisition of Crinetics Pharmaceuticals for a total equity value of approximately $10.0 billion, or about $8.8 billion net of estimated cash acquired. The acquisition expands Vertex's pipeline into rare endocrine diseases, adding drugs PALSONIFY and atumelnant. Vertex also announced leadership additions, including integration responsibilities and new executive appointments.AI summary

AstraZeneca PLC announced that its subsidiary, AstraZeneca Finance LLC, has admitted four series of debt securities to trading on the London Stock Exchange's Main Market. The total bond offering equals €2.55 billion across notes maturing between 2030 and 2038. The debt securities were admitted to trading on 1 September 2026.AI summary

Moderna announced a proposed private placement of $2.0 billion aggregate principal amount of Convertible Senior Notes due 2032. Initial purchasers will also have an option to buy up to an additional $300.0 million in notes. Net proceeds will fund capped call transactions and general corporate purposes, including oncology business growth and debt repayment.AI summary

AstraZeneca has completed an exclusive global license agreement with Dizal Pharmaceutical to develop and commercialise Zegfrovy worldwide. AstraZeneca will make an upfront payment of $600m to Dizal, with up to $900m in potential milestones plus tiered royalties. AstraZeneca plans to launch Zegfrovy in the US during the fourth quarter of 2026.AI summary

Intellia Therapeutics entered into a $400 million non-dilutive senior secured term loan facility with healthcare investment firm OrbiMed. The transaction includes an initial term loan of $75 million funded at closing, with up to $325 million available through milestone-tied tranches and mutual agreement. The financing supports Intellia as it advances toward a planned U.S. approval and commercial launch of lonvoguran ziclumeran for hereditary angioedema.AI summary

Biotechnology company. Total offering $65M; first sale 2026-08-10. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $150M; first sale 2026-08-20. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $15M; first sale 2026-08-17. Form D is filed up to 15 days after the first sale.

Evotec SE and Plectonic Biotech have entered into a research collaboration to explore a novel immunotherapy approach for solid tumors. The partnership combines Plectonic's programmable LOGIBODY technology with Evotec's proprietary BiTco platform. Under the terms of the agreement, the companies will generate preclinical proof-of-concept data to evaluate the combined T-cell-engaging approach.AI summary

Ferring Pharmaceuticals and RedHill Biopharma have entered into a strategic partnership regarding Rebyota and Clenpiq. Under the agreement, RedHill acquires exclusive global commercial rights to Rebyota and exclusive U.S. commercial rights to Clenpiq. Ferring will remain the Marketing Authorisation Holder for both products and continue to manufacture and supply them to RedHill.AI summary

Ascendis Pharma has entered into a binding term sheet for a global settlement and license agreement with BioMarin Pharmaceutical regarding YUVIWEL (navepegritide). Under the agreement, BioMarin grants Ascendis a worldwide license to commercialize navepegritide-related products and both companies will dismiss all proceedings against each other. Ascendis will pay BioMarin net sales royalties of 20% in the U.S. and 18% in the European Union, South Korea, and Brazil through May 20, 2030.AI summary

Vaxcyte, Inc. has appointed Luis Jodar, Ph.D. as Chief Medical Officer and David McAvoy, J.D. as Chief Legal Officer. The company narrowed its timeline for disclosing topline safety, tolerability and immunogenicity data from the Phase 3 OPUS-1 trial of VAX-31. Vaxcyte now expects to report the trial's topline results by the end of October 2026.AI summary

Dyne Therapeutics announced it will present preclinical data for DYNE-253, DYNE-245, DYNE-244, and DYNE-255 at the 27th Annual NMSG Scientific Meeting. The data support the ongoing development of these four investigational therapies for Duchenne muscular dystrophy. All four programs are currently advancing into IND-enabling studies.AI summary

Attovia Therapeutics reported second quarter 2026 financial results and provided pipeline updates. The company completed an upsized initial public offering generating $332.4 million in gross proceeds, extending its cash runway into 2030. Complete Phase 1b data for ATTO-1310 are expected in Q4 2026, with Phase 2 studies planned to initiate in 1H 2027.AI summary

BridgeBio Pharma has entered into a voluntary agreement with the U.S. government to lower drug costs and expand access to its medicines for American patients. Under the agreement, BridgeBio will expand state Medicaid access to its marketed drug Attruby via the GENEROUS Model while continuing to offer it via Medicare Part D. The company does not expect to be subject to future pricing mandates, and specific financial terms remain confidential.AI summary