Main Reading Room
(Library 1 ―  MRR)

Novartis receives FDA approval for Rhapsido® (remibrutinib), the only oral, targeted BTKi treatment for chronic spontaneous urticaria (CSU) < September 30, 2025

• Rhapsido® (remibrutinib) received FDA approval as an oral treatment for adult patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. Rhapsido is a pill taken twice daily and does not require injections or lab monitoring. It is the first FDA-approved Bruton’s tyrosine kinase inhibitor (BTKi) for CSU. Rhapsido helps to inhibit the release of histamine and other proinflammatory mediators by targeting BTK, offering a unique approach to CSU treatment.  CSU is a mast cell-driven condition thought to be caused by immune dysregulation. In people with CSU, the immune system can become activated through allergic (IgE) or autoimmune (IgG) pathways. 

U.S. FDA approves Inluriyo (imlunestrant) for adults with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer < Lilly, SERD, September 25, 2025

• FDA has approved Inluriyo (imlunestrant, 200 mg tablets), an oral estrogen receptor antagonist, for the treatment of adults with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2–), ESR1-mutated advanced or metastatic breast cancer (MBC) whose disease progressed after at least one line of endocrine therapy (ET). In the Phase 3 EMBER-3 trial, Inluriyo reduced the risk of progression or death by 38% versus ET. Among patients with ESR1-mutated MBC, Inluriyo significantly improved progression-free survival (PFS) versus fulvestrant or exemestane, with a median PFS of 5.5 months vs 3.8 months (HR=0.62 [95% CI: 0.46-0.82]); p-value=0.0008.

Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol < REGN, September 26, 2025

• FDA has approved Evkeeza® (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH). Evkeeza was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH based on a placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower LDL-C by about 50% compared to placebo in this high unmet need population. It was then approved for children aged 5 to 11 in 2023. 

Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol < REGN, September 26, 2025

• FDA has approved Evkeeza® (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH). Evkeeza was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH based on a placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower LDL-C by about 50% compared to placebo in this high unmet need population. It was then approved for children aged 5 to 11 in 2023. 

Koselugo recommended for approval in the EU by CHMP for plexiform neurofibromas in adults with neurofibromatosis type 1 < AZN, MEK, 22 September 2025

• Koselugo (selumetinib), an oral, selective MEK inhibitor, has been recommended for approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from KOMET, the largest and only placebo-controlled global Phase III trial in this patient population, which were presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet.

Tezspire recommended for approval in the EU by CHMP for chronic rhinosinusitis with nasal polyps < AZN, TSLP, 22 September 2025

• AstraZeneca and Amgen’s Tezspire (tezepelumab) has been recommended for approval in the European Union (EU) for the treatment of adult patients with chronic rhinosinusitis with nasal polyps (CRSwNP). CHMP based its positive opinion on results from the WAYPOINT Phase III trial, which were presented at the 2025 American Academy of Allergy Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress and simultaneously published in The New England Journal of Medicine. In the WAYPOINT trial, Tezspire demonstrated a statistically significant and clinically meaningful reduction in nasal polyp severity, as measured by the co-primary endpoints; Nasal Polyp Score (NPS) by -2.08 (95% CI: -2.40, -1.76; p<0.001) and nasal congestion (measured by participant-reported Nasal Congestion Score [NCS]) by -1.04 (95% CI: -1.21, -0.87; p<0.001) at week 52 compared to placebo.

TREMFYA® (guselkumab) achieves U.S. approval for subcutaneous induction in adults with ulcerative colitis, now the first and only IL-23 inhibitor with a fully subcutaneous regimen < JNJ, September 19, 2025

• The U.S. FDA has approved a subcutaneous (SC) induction regimen of TREMFYA® (guselkumab) for the treatment of adults with moderately to severely active ulcerative colitis (UC). TREMFYA® is the first and only approved fully-human, dual-acting monoclonal antibody that blocks IL-23 while also binding to CD64, a receptor on cells that produce IL-23. IL-23 is a cytokine secreted by activated monocyte/macrophages and dendritic cells that is known to be a driver of immune-mediated diseases including UC. Findings are based on in vitro studies. Historically, IL-23 inhibitors have required IV infusions at the start of therapy.

FDA Approves Merck’s KEYTRUDA QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph) Injection for Subcutaneous Use in Adults Across Most Solid Tumor Indications for KEYTRUDA® (pembrolizumab) < s.c. admin., September 19, 2025

•  KEYTRUDA QLEX is the first and only subcutaneously administered immune checkpoint inhibitor that can be given by a health care provider in as little as one minute. FDA has approved KEYTRUDA QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph) injection for subcutaneous administration in adults across most solid tumor indications for KEYTRUDA® (pembrolizumab). Berahyaluronidase alfa is a variant of human hyaluronidase developed and manufactured by Alteogen Inc. KEYTRUDA QLEX must be administered by a health care provider (HCP). 

CHMP recommends EU approval of Roche’s subcutaneous formulation of Lunsumio for people with relapsed or refractory follicular lymphoma < CD20, September 19, 2025

• Subcutaneous Lunsumio has potential to substantially reduce treatment administration time with an approximately one minute injection, compared with 2-4 hours IV infusion. If approved, Lunsumio would be the first treatment available for people with follicular lymphoma after two or more lines of systemic therapy, which is both fixed-duration and subcutaneously administered.  The CHMP opinion is based on results from a primary analysis of the phase II GO29781 study. Data show subcutaneous Lunsumio had pharmacokinetic non-inferiority compared to intravenous (IV) administration, with no unexpected safety signals. Overall, the rate and severity of cytokine release syndrome was low (29.8%); events were low grade (Grade 1-2, 27.7%; Grade 3, 2.1%), occurred during cycle 1 and all fully resolved in a median of two days (range 1-15 days).

Incyte Announces Additional FDA Approval of Opzelura® (Ruxolitinib) Cream in Children Ages 2-11 with Atopic Dermatitis < JAK, September 18, 2025

• FDA has approved Opzelura® (ruxolitinib) cream 1.5%, a topical Janus kinase (JAK) inhibitor, for the short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised children two years of age and older whose disease is not well controlled with topical prescription therapies, or when those therapies are not recommended. Opzelura is the first topical JAK inhibitor approved in the U.S. for pediatric atopic dermatitis (AD)

AbbVie Submits Biologics License Application (BLA) to U.S. FDA for Pivekimab sunirine (PVEK) - an Investigational Antibody-Drug Conjugate (ADC) to Treat Rare Cancer with Limited Treatment Options < September 30, 2025

• ... for treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). PVEK is a CD123-targeting Antibody-Drug Conjugate (ADC) in clinical development for hematological malignancies (blood cancers), including BPDCN and acute myeloid leukemia (AML). The submission is based on data from the Phase 1/2 CADENZA trial, a global study evaluating the safety and efficacy of PVEK in BPDCN. BPDCN is a rare and aggressive blood cancer that has features of both leukemia and lymphoma. Patients typically present with skin lesions and the disease often spreads to the bone marrow, central nervous system and the lymph nodes. 

AbbVie Submits New Drug Application to U.S. FDA for Tavapadon for the Treatment of Parkinson's Disease < Dopamine, September 26, 2025

• AbbVie has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tavapadon, a novel selective dopamine D1/D5 receptor partial agonist that was studied as a once daily oral treatment for Parkinson's disease. The submission is based on results from the TEMPO clinical development program that evaluated the efficacy, safety and tolerability of tavapadon across a broad Parkinson's disease population. This includes two Phase 3 trials (TEMPO-1 and TEMPO-2) in early Parkinson's disease, and one Phase 3 trial (TEMPO-3) with tavapadon as adjunctive to levodopa in patients experiencing motor fluctuations.

Enhertu plus pertuzumab (Perjeta) granted Priority Review in the US as 1st-line treatment for patients with HER2-positive metastatic breast cancer < AZN, Daiichi, 24 September 2025

• Based on DESTINY-Breast09 Phase III trial results which showed Enhertu plus pertuzumab reduced the risk of disease progression or death by 44% vs. THP with a median progression-free survival of greater than three years. If approved, AstraZeneca and Daiichi Sankyo’s Enhertu-based treatment approach would move into the 1st-line HER2-positive metastatic setting with potential to become a new standard of care. HER2-positive metastatic breast cancer is an aggressive disease driven by overexpression or amplification of HER2 that affects 15% to 20% of patients with metastatic breast cancer.

Biogen Provides Regulatory Update on High Dose Regimen of Nusinersen < CRL, SMA, September 23, 2025

• FDA issued a Complete Response Letter (CRL) for the Company’s supplemental New Drug Application (sNDA) for the high dose regimen of nusinersen for the treatment of spinal muscular atrophy (SMA). The FDA letter requested an update to the technical information be included in the Chemistry Manufacturing and Controls (CMC) module of the sNDA. The letter did not cite any deficiencies in the clinical data of the high dose regimen. The FDA provided options for resolution, and Biogen is planning to resubmit the application promptly based upon readily available information.

Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1 < September 23, 2025

• Myotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines. SAR446268 employs a vectorized RNA interference (RNAi) approach to silence DMPK expression through a single administration. By reducing DMPK transcripts, the gene therapy aims to eliminate the abnormal and toxic RNA foci responsible for splicing defects in muscle tissue, thereby restoring normal splicing and improving muscular function. 

Anti-MTBR (microtubule binding region) Tau Antibody Etalanetug Granted FDA Fast Track Designation < Eisai, Alzheimer's, September 17, 2025

• Eisai announced that etalanetug (development code: E2814), an investigational anti-MTBR (microtubule binding region) tau antibody, was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). Etalanetug is an anti-tau antibody that targets specific tau species containing MTBR, tau seeds that spread tau pathology to different brain regions. Etalanetug was discovered as part of the research collaboration between Eisai and University College London. 

Raludotatug Deruxtecan Granted Breakthrough Therapy Designation by U.S. FDA for Patients with CDH6 Expressing Platinum-Resistant Ovarian, Primary Peritoneal, or Fallopian Tube Cancers Previously Treated with Bevacizumab < MRK, Daiichi, September 15, 2025

•  Raludotatug deruxtecan (R-DXd) has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with platinum-resistant epithelial ovarian, primary peritoneal or fallopian tube cancers expressing CDH6 who have received prior treatment with bevacizumab. Raludotatug deruxtecan is a specifically engineered, potential first-in-class CDH6 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed by Daiichi Sankyo and Merck (NYSE: MRK), known as MSD outside of the United States and Canada.

Sanofi’s SAR402663 earns fast track designation in the US for neovascular age-related macular degeneration (nAMD)< Gene, September 11, 2025

• FDA has granted fast track designation to SAR402663, an investigational one-time intravitreal gene therapy for the treatment of neovascular age-related macular degeneration (AMD). SAR402663 delivers genetic material encoding soluble FLT01 designed to inhibit vascular endothelial growth factor (VEGF). Sanofi is currently evaluating SAR402663 in a phase 1/2 (clinical study identifier: NCT06660667), for the treatment of patients with neovascular AMD. 

Lilly's olomorasib receives U.S. FDA's Breakthrough Therapy designation for the treatment of certain newly diagnosed metastatic KRAS G12C-mutant lung cancers < September 4, 2025

• FDA has granted Breakthrough Therapy designation to olomorasib, in combination with anti-PD-1 therapy KEYTRUDA (pembrolizumab), for the first-line treatment of patients with unresectable advanced or metastatic non-small cell lung cancer (NSCLC) with a KRAS G12C mutation and PD-L1 expression ≥ 50%, as determined by FDA approved tests. Olomorasib is a potent and highly selective second-generation inhibitor of KRAS G12C with preliminary evidence of central nervous system (CNS) activity.   

Eisai Initiated Rolling Supplemental Biologics License Application to the U.S. FDA for LEQEMBI® IQLIK™ (lecanemab-irmb) as a Subcutaneous Starting Dose for the Treatment of Early Alzheimer’s Disease Under Fast Track Status < Amyloid beta, September 3, 2025

• Eisai has initiated the rolling submission of the Supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for lecanemab-irmb (U.S. brand name: LEQEMBI®) subcutaneous autoinjector (SC-AI), LEQEMBI IQLIK, as a weekly starting dose after the FDA granted Fast Track Status. LEQEMBI is indicated for the treatment of Alzheimer’s disease (AD) in patients with Mild Cognitive Impairment (MCI) or mild dementia stage of disease (collectively referred to as early AD).  The sBLA is based on evaluation of subcutaneous (SC) lecanemab administration across a range of doses and as part of sub-studies within the Phase 3 Clarity AD open-label extension (OLE), following the 18-month core study in individuals with early AD.

WINREVAIR (sotatercept-csrk) Reduced the Risk of Clinical Worsening Events by 76% Compared to Placebo in Patients Recently Diagnosed With PAH on Background Therapy in Phase 3 HYPERION Trial < MRK, Activin, September 30, 2025

• Results showed the benefits of early initiation of WINREVAIR within the first year after PAH diagnosis. MRK announced positive results from the Phase 3 HYPERION trial evaluating WINREVAIR™ (sotatercept-csrk) versus placebo (both in combination with background therapy) in recently diagnosed adults with pulmonary arterial hypertension (PAH, WHO* Group 1) functional class (FC) II or III at intermediate or high risk of disease progression. In the study, WINREVAIR reduced the risk of clinical worsening events by 76% (hazard ratio [HR] 0.24 [95% confidence interval [CI], 0.14 to 0.41]; p<0.0001) as measured by a composite endpoint of all-cause death, the need for non-planned PAH-related hospitalization ≥24 hours, atrial septostomy, lung transplantation or PAH deterioration. 

Bristol Myers Squibb Announces Phase 3 EXCALIBER-RRMM Study Evaluating Iberdomide in Combination with Standard Therapies Demonstrated a Significant Improvement in Minimal Residual Disease Negativity Rates in Relapsed or Refractory Multiple Myeloma <  September 23, 2025

• The Phase 3 EXCALIBER-RRMM study evaluating iberdomide, an investigational cereblon E3 ligase modulator (CELMoD™), combined with standard therapies (daratumumab + dexamethasone) in patients with relapsed or refractory multiple myeloma (RRMM) demonstrated a statistically significant improvement in minimal residual disease (MRD) negativity rates, compared with the control arm, in a planned interim analysis of the MRD endpoint. In accordance with the trial design and based on the recommendation from the Data Monitoring Committee, the trial will continue without changes to evaluate the other dual-primary endpoint of progression-free survival (PFS), and the key secondary endpoint of overall survival and safety. 

Positive phase III results show Roche’s giredestrant significantly improved progression-free survival in ER-positive advanced breast cancer < NVS, SERD, 22 September 2025

• Roche announced positive results from the phase III evERA study evaluating investigational giredestrant in combination with everolimus in people with oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer previously treated with cyclin-dependent kinase (CDK) 4/6 inhibitor and endocrine therapy. The study met both co-primary endpoints, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) in both the intention-to-treat and ESR1-mutated populations, compared with standard-of-care endocrine therapy plus everolimus. Overall survival (OS) data were immature, but a clear positive trend was observed. Follow-up continues to the next OS analysis. The giredestrant combination was well tolerated and adverse events were consistent with the known safety profiles of the individual study treatments, and no new safety signals were observed. This is the first positive head-to-head phase III trial investigating an all-oral selective oestrogen receptor degrader-containing regimen versus a standard of care combination.

First Parkinson’s disease patient treated in BlueRock’s pivotal Phase III trial of investigational cell therapy bemdaneprocel < Bayer, September 22, 2025

• exPDite-2 is the first Phase III pivotal clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease. In a Phase I study with 12 participants, bemdaneprocel was well tolerated, with no serious adverse events related to drug product at 24 months post-surgery. In addition, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery.

Saphnelo self-administration TULIP-SC Phase III trial meets primary endpoint in patients with systemic lupus erythematosus based on an interim analysis < AZN, IFN-R, 17 September 2025

• Subcutaneous administration of first-in-class biologic Saphnelo demonstrates statistically significant and clinically meaningful reduction in disease activity. Positive high-level results from a pre-specified interim analysis of the Phase III TULIP-SC trial in patients with systemic lupus erythematosus (SLE) showed that the subcutaneous (SC) administration of AstraZeneca’s Saphnelo (anifrolumab) demonstrated a statistically significant and clinically meaningful reduction in disease activity compared to placebo.1 The  safety profile observed in the interim analysis was consistent with the known clinical profile of Saphnelo administered as an intravenous (IV) infusion.

Incyte Announces New 24-Week Phase 3 Data from the STOP-HS Clinical Trial Program of Povorcitinib in Hidradenitis Suppurativa at EADV 2025 < JAK, September 17, 2025

• Across both STOP-HS1 and STOP-HS2, povorcitinib treatment resulted in continued clinically meaningful and statistically significant improvements for patients with active moderate to severe hidradenitis suppurativa (HS) through Week 24. INCY announced new 24-week interim data evaluating the safety and efficacy of povorcitinib, an oral small-molecule highly-selective JAK1 inhibitor, from the pivotal Phase 3 STOP-HS clinical trial program in adult patients (≥18 years) with moderate to severe hidradenitis suppurativa (HS). These data will support the planned regulatory submissions for povorcitinib in HS in Europe and the United States in 2025 and early 2026, respectively. 

Icotrokinra shows superiority to deucravacitinib in first reported head-to-head trials reinforcing promise of novel targeted oral peptide for treatment of plaque psoriasis < JNJ, IL-23, p.o. admin. , September 17, 2025

• Icotrokinra demonstrated superior skin clearance at Weeks 16 and 24 compared to deucravacitinib and similar adverse event rates to placebo in Phase 3 ICONIC-ADVANCE studies. Icotrokinra also showed sustained skin clearance and favorable safety profile in both adults and adolescents at Week 52 in Phase 3 ICONIC-LEAD study. JNJ announced new data from the Phase 3 ICONIC-ADVANCE 1 and 2 studies which assessed the superiority of icotrokinra, a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor, compared to deucravacitinib in patients with moderate-to-severe plaque psoriasis. 

Lilly's oral GLP-1, orforglipron, demonstrated meaningful weight loss and cardiometabolic improvements in complete ATTAIN-1 results published in The New England Journal of Medicine < September 16, 2025

• LLY announced detailed results from the Phase 3 ATTAIN-1 trial, evaluating the safety and efficacy of orforglipron, an investigational oral glucagon-like peptide-1 (GLP-1) receptor agonist, in adults with obesity, or overweight with a weight-related medical problem and without diabetes. At 72 weeks, all three doses (6 mg, 12 mg and 36 mg) of orforglipron met the primary endpoint of superior body weight reduction compared to placebo. In addition, all three doses delivered clinically meaningful results compared to placebo across the key secondary endpoints of body weight reduction (≥10%, ≥15% and ≥20%), and waist circumference reduction. 

CAPVAXIVE® (Pneumococcal 21-valent Conjugate Vaccine) Demonstrates Positive Immune Responses in Children and Adolescents at Increased Risk of Pneumococcal Disease < MRK, September 11, 2025

•  MRK announced positive results from the Phase 3 STRIDE-13 trial evaluating CAPVAXIVE® (Pneumococcal 21-valent Conjugate Vaccine) at the 6th European Society of Clinical Microbiology and Infectious Diseases (ESCMID) Conference on Vaccines, taking place in Lisbon, Portugal. The trial evaluated the safety, tolerability and immunogenicity of CAPVAXIVE compared to PPSV23 (pneumococcal 23-valent polysaccharide vaccine) in children and adolescents aged 2 to <18 years who have completed a primary pediatric pneumococcal vaccination regimen and have one or more chronic medical conditions that put them at an increased risk of pneumococcal disease.

Lilly's Jaypirca (pirtobrutinib), the first and only approved non-covalent (reversible) BTK inhibitor, significantly improved progression-free survival in patients with treatment-naïve CLL/SLL < September 8, 2025

• Results from BRUIN CLL-313 and BRUIN CLL-314 will form the basis for seeking label expansions in earlier lines of therapy, with global regulatory submissions beginning later this year. LLY announced positive topline results from the Phase 3 BRUIN CLL-313 clinical trial of Jaypirca (pirtobrutinib), a non-covalent (reversible) Bruton tyrosine kinase (BTK) inhibitor, versus chemoimmunotherapy (bendamustine plus rituximab), in treatment-naïve patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) without 17p deletions. 

Pfizer and BioNTech Announce Topline Data Demonstrating Robust Immune Response With Their LP.8.1-Adapted COVID-19 Vaccine 2025-2026 Formula < September 08, 2025

• Phase 3 clinical trial cohort of adults 65+ and 18-64 with at least one underlying risk condition shows at least a 4-fold increase in LP.8.1-neutralizing antibody titers after receiving the LP.8.1-adapted COVID-19 vaccine 2025-2026 Formula. The preliminary data show a robust increase in neutralizing antibodies targeting the LP.8.1 sublineage of SARS-CoV-2 following vaccination. These clinical findings reinforce pre-clinical data that supported the recent U.S. Food and Drug Administration (FDA) approval of the LP.8.1-adapted COVID-19 vaccine, which demonstrated improved immune responses against multiple circulating SARS-CoV-2 sublineages.

Amgen and Kyowa Kirin announced preliminary top-line results from the ASCEND study evaluating rocatinlimab, an investigational T-cell rebalancing therapy targeting the OX40 receptor, in adults and adolescents with moderate to severe atopic dermatitis (AD) < OX40, September 8, 2025

• The ongoing ASCEND study, which includes approximately 2,600 patients, is designed to evaluate the long-term safety and efficacy of rocatinlimab (150 mg and 300 mg) administered every four or eight weeks in individuals who completed a previous ROCKET program trial (IGNITE, HORIZON, SHUTTLE, ASTRO, ORBIT or VOYAGER). This analysis focused on adults who completed the first 24 weeks of therapy in a previous ROCKET trial and continued in ASCEND for an additional 32 weeks.

Data published in The New England Journal of Medicine demonstrate RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE® (lazertinib) is re-setting survival expectations in first-line EGFR-mutated lung cancer < JNJ, MET, EGFR, September 7, 2025

• The New England Journal of Medicine (NEJM) published results from the Phase 3 MARIPOSA study, which showed RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE® (lazertinib) demonstrated a statistically significant and clinically meaningful overall survival (OS) improvement for patients with previously untreated (first-line) locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or L858R substitution mutations.1 The article is available with free access here for a limited time. At a median follow-up of 37.8 months, RYBREVANT® plus LAZCLUZE® showed a statistically significant reduction in the risk of death compared to osimertinib (hazard ratio [HR], 0.75; 95 percent confidence interval [CI], 0.61-0.92, P=0.005). The median OS for the combination has not yet been reached (95 percent CI, 42.9-not estimable). Median OS for the combination is projected to exceed over four years (absolute increase of over one year) compared to the median of three years observed with osimertinib.

Ifinatamab Deruxtecan Demonstrated Clinically Meaningful Response Rates in Patients with Extensive-Stage Small Cell Lung Cancer in IDeate-Lung01 Phase 2 Trial < MRK, Daiichi, B7-H3, September 7, 2025

• Results from the IDeate-Lung01 Phase 2 trial showed that ifinatamab deruxtecan (I-DXd) demonstrated clinically meaningful response rates in patients with previously treated extensive-stage small cell lung cancer (ES-SCLC). Response was assessed in patients who received ifinatamab deruxtecan (12 mg/kg) in both the dose optimization and single-arm expansion parts of the trial. A confirmed objective response rate (ORR) of 48.2% (95% confidence interval [CI]: 39.6–56.9) was observed with ifinatamab deruxtecan in 137 patients with previously treated ES-SCLC as assessed by blinded independent central review (BICR). Three complete responses (CRs), 63 partial responses (PRs) and 54 cases of stable disease (SD) were seen. A median duration of response (DOR) of 5.3 months (95% CI: 4.0–6.5) and a disease control rate (DCR) of 87.6% (95% CI: 80.9–92.6) were observed. Median progression-free survival (PFS) was 4.9 months (95% CI: 4.2–5.5) and median overall survival (OS) was 10.3 months (95% CI: 9.1–13.3).  

Merck’s Investigational Oral PCSK9 Inhibitor Enlicitide Decanoate Met All Primary and Key Secondary Endpoints in Adults with Hypercholesterolemia in Pivotal CORALreef Lipids Study < September 2, 2025

• The CORALreef Lipids trial successfully met all primary and key secondary endpoints. Treatment with enlicitide resulted in statistically significant and clinically meaningful reduction in low-density lipoprotein cholesterol (LDL-C) compared to placebo at Week 24. Statistically and clinically significant reductions were also seen for enlicitide versus placebo across all key secondary endpoints including in non-high-density lipoprotein cholesterol (non-HDL-C), apolipoprotein B (ApoB), and lipoprotein(a) [Lp(a)]. CORALreef Lipids represents the largest completed Phase 3 study evaluating enlicitide in a broad range of participants with elevated LDL-C and a history of or increased risk for major atherosclerotic cardiovascular disease events despite treatment with at least a moderate or high intensity statin (or with documented statin intolerance). 

Enhertu demonstrated highly statistically significant and clinically meaningful improvement in invasive disease-free survival vs. T-DM1 in DESTINY-Breast05 Phase III trial in patients with high-risk early breast cancer following neoadjuvant therapy < AZN, Daiichi, HER2, 29 September 2025

• Second positive Phase III trial of AstraZeneca and Daiichi Sankyo’s Enhertu in HER2-positive early breast cancer reinforces its potential to become a foundational treatment option in curative-intent setting. Positive high-level results from a planned interim analysis of the DESTINY-Breast05 Phase III trial showed Enhertu (trastuzumab deruxtecan) demonstrated a highly statistically significant and clinically meaningful improvement in invasive disease-free survival (IDFS) versus trastuzumab emtansine (T-DM1) in patients with HER2-positive early breast cancer with residual invasive disease in the breast or axillary lymph nodes after neoadjuvant treatment and a high risk of disease recurrence. 

Johnson & Johnson’s investigational seltorexant shows numerically higher response in patients with depression with insomnia symptoms, with fewer side effects compared to quetiapine XR < Orexin, September 22, 2025

• Seltorexant is an investigational, first-in-class selective antagonist of the human orexin-2 receptor being studied for the adjunctive treatment of MDD with insomnia symptoms. The Phase 3 randomized, double-blind, parallel-group study showed a numerically greater response rate at 26 weeks with seltorexant than quetiapine XR (57.4 percent versus 53.4 percent), a commonly prescribed adjunctive therapy for MDD patients experiencing insomnia symptoms, though this difference did not reach statistical significance for the primary endpoint.1 Seltorexant and quetiapine XR showed large and clinically meaningful improvements in depressive symptoms (-23.0 and -22.7, respectively), as measured by change in Montgomery-Asberg Depression Rating Scale (MADRS) total score from baseline.

UCB announces successful first-in-patient trial for galvokimig in moderate-to-severe atopic dermatitis at EADV < IL-13, IL-17, MsAb, September 18, 2025

• UCB announced new 12-week efficacy and 18-week safety data from the Phase 1/2a first-in-patient trial for galvokimig, a novel multi-specific antibody-based therapeutic currently under clinical investigation for adults living with moderate-to severe atopic dermatitis (AD). Galvokimig demonstrated clinically meaningful improvements in stringent efficacy measures for AD, a common, chronic, inflammatory skin disease affecting 2–10% of adults worldwide. Galvokimig is a multi-specific antibody-based therapeutic that is designed to inhibit IL-13, IL-17A and IL-17F, with an extended half-life through albumin binding. 

Update on the RESOLUTE Phase III trial for Fasenra in chronic obstructive pulmonary disease < Fail, AZN, IL-5, 17 September 2025

• The RESOLUTE Phase III trial of AstraZeneca’s Fasenra (benralizumab), despite showing numerical improvement, did not achieve statistical significance in the primary endpoint in patients with chronic obstructive pulmonary disease (COPD). Fasenra is currently approved as an add-on maintenance treatment for severe eosinophilic asthma (SEA) in more than 80 countries, including the US, Japan, EU and China. It is also approved for SEA in children and adolescents ages six and above in the US and Japan. Fasenra is also approved in more than 60 countries for the adult treatment of eosinophilic granulomatosis with polyangiitis (EGPA), and is under regulatory review for the treatment of hypereosinophilic syndrome (HES).

Lilly's oral GLP-1, orforglipron, superior to oral semaglutide in head-to-head trial < Obesity, September 17, 2025

• ... positive topline results from ACHIEVE-3, an open-label randomized Phase 3 clinical trial evaluating the safety and efficacy of orforglipron compared to oral semaglutide, administered according to approved label instructions, in 1,698 adults with type 2 diabetes inadequately controlled with metformin. The 52-week trial compared orforglipron (12 mg and 36 mg) to oral semaglutide (7 mg and 14 mg) across four active treatment arms to assess glycemic control and weight loss. At 52 weeks, orforglipron met the primary and all key secondary endpoints across each dose comparison vs. oral semaglutide, delivering greater improvements in A1C and weight.

Sanofi’s brivekimig achieved positive results in hidradenitis suppurativa in phase 2a study < OX40, September 17, 2025

• New data from the HS-OBTAIN phase 2a study (clinical study identifier: NCT05849922) show that treatment with brivekimig led to clinically meaningful improvements in the primary endpoint of Hidradenitis Suppurativa Clinical Response (HiSCR50) in patients naïve to biologics with moderate-to-severe hidradenitis suppurativa (HS). The HS-OBTAIN phase 2a study is a randomized, double-blind, placebo-controlled, proof-of-concept study assessing the efficacy and safety of brivekimig in adults with moderate-to-severe HS. The primary analysis population included biologic-naïve HS patients who were randomized 2:1 to receive brivekimig 150 mg or placebo subcutaneously every two weeks. 

AskBio Announces Completion of Enrollment in Phase 1 Clinical Trial of AB-1005 Gene Therapy for Multiple System Atrophy-Parkinsonian Type (MSA-P) < Bayer, SEPTEMBER 17, 2025

• REGENERATE MSA-101 is a Phase 1 clinical trial of AB-1005 investigational gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P). MSA-P can initially be difficult to distinguish from Parkinson’s disease and is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting, among other symptoms. Individuals experience increasing difficulty with movement and autonomic dysfunction. This is a result of progressive loss of nerve cells in the brain and spinal cord. Affecting approximately 400,000 people worldwide, MSA is a rare disease that in most cases seems to occur for unknown reasons. Symptoms tend to develop in people over 50 years of age, followed by rapid progression within 5–10 years.

Libtayo® (cemiplimab) Plus Chemotherapy Results at Five Years Reinforce Significant and Durable Improvements in Survival Outcomes for Advanced Non-small Cell Lung Cancer < REGN, PD-1, September 9, 2025

• REGN announced five-year follow-up results on overall survival (OS) from the Phase 3 EMPOWER-Lung 3 trial, which evaluated Libtayo® (cemiplimab) plus platinum-based chemotherapy versus chemotherapy alone as a first-line treatment for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with no EGFR, ALK or ROS1 aberrations. The late-breaking data will be presented in a mini oral session at the IASLC 2025 World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer.

First Disclosure of Global Interim Phase 2 Data for BioNTech and Bristol Myers Squibb PD-L1xVEGF-A Bispecific Antibody Pumitamig (BNT327/BMS986545) in Patients with Extensive-Stage Small Cell Lung Cancer Shows Encouraging Antitumor Activity < September 08, 2025

• PD-L1xVEGF-A bispecific antibody pumitamig (BNT327/BMS986545) plus chemotherapy continues to show encouraging antitumor activity in patients with extensive-stage small cell lung cancer (”ES-SCLC”), expanding evidence for its potential to set a new standard of care in first-line ES-SCLC and beyond. Global interim Phase 2 data showed a 76.3% confirmed objective response rate (cORR), 100% disease control rate (DCR), a median progression free survival (PFS) of 6.8 months and a manageable safety profile. Data confirm dose selection for the ongoing global pivotal Phase 3 ROSETTA LUNG-01 trial

Eisai Presents Clinical Study Results of Novel Orexin Receptor Agonist E2086 for Narcolepsy at World Sleep 2025 < September 8, 2025

• The results from a Phase Ib clinical trial of in-house developed novel selective orexin 2 receptor agonist E2086 have been presented at the World Sleep 2025 congress, held in Singapore from September 5 to 10. These data demonstrate that once-daily dosing of E2086 has the potential to improve daytime wakefulness in individuals with narcolepsy type 1 (NT1). This randomized, double-blind, single-dose, multiple crossover, Phase Ib clinical trial was conducted in the United States and Canada to evaluate the efficacy, safety, and tolerability of E2086 in people meeting criteria for NT1, compared with placebo and an existing treatment (modafinil). Efficacy was assessed using the objective Maintenance of Wakefulness Test (MWT) to evaluate excessive daytime sleepiness (EDS), and subjective sleepiness was measured using the Modified Karolinska Sleepiness Scale (Modified KSS)*2 at the end of each MWT session.

Tagrisso plus chemotherapy demonstrated a median overall survival of nearly four years, the longest benefit ever reported in a global Phase III trial in EGFR-mutated advanced lung cancer < AZN, EGFR, 7 September 2025

• Positive results from the final overall survival (OS) analysis of the FLAURA2 Phase III trial showed AstraZeneca’s Tagrisso (osimertinib) with the addition of pemetrexed and platinum-based chemotherapy demonstrated a statistically significant and clinically meaningful improvement in the key secondary endpoint of OS compared to Tagrisso monotherapy in the 1st-line treatment of patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC). In the final OS analysis, Tagrisso plus chemotherapy demonstrated a median OS of nearly four years (47.5 months) compared to approximately three years (37.6 months) for Tagrisso monotherapy. At 57% data maturity, results showed Tagrisso plus chemotherapy reduced the risk of death by 23% compared to Tagrisso monotherapy (based on a hazard ratio [HR] of 0.77; 95% confidence interval [CI] 0.61-0.96; p=0.0202). 

AbbVie Announces Updated Results From Phase 2 EPCORE® NHL-6 Study Evaluating the Potential for Outpatient Monitoring of Epcoritamab in Patients With Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) < September 03, 2025

• ABBV announced updated results from the Phase 2 EPCORE® NHL-6 trial (NCT05451810) evaluating the feasibility of dosing and monitoring patients in the outpatient setting for the first full dose of epcoritamab monotherapy, a T-cell engaging bispecific antibody administered subcutaneously, in adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least one prior line of systemic therapy.  Results from the study demonstrated that the incidence and severity of cytokine release syndrome (CRS) and immune cell-associated neurotoxicity syndrome (ICANS) following treatment with epcoritamab were consistent with previous epcoritamab studies in R/R DLBCL. 

Sanofi’s amlitelimab met all primary and key secondary endpoints in the COAST 1 phase 3 study in adults and adolescents with atopic dermatitis <  OX40, September 4, 2025

• Positive results from the global COAST 1 phase 3 study (clinical study identifier: NCT06130566) showed that amlitelimab, a fully human non-T cell depleting monoclonal antibody that targets OX40-ligand (OX40L), dosed either every four weeks (Q4W) or every 12 weeks (Q12W), met all primary and key secondary endpoints, demonstrating statistically significant and clinically meaningful skin clearance and disease severity compared to placebo at Week 24 in patients aged 12 years and older with moderate-to-severe atopic dermatitis (AD). Amlitelimab was well-tolerated, with no new safety concerns identified in this study.

Eisai to Present Latest Data on Lemborexant and Novel Orexin Receptor Agonist E2086 at The World Sleep 2025 < Narcolepsy, September 4, 2025

• Eisai will deliver a total of 11 presentations, including clinical data on the selective orexin 2 receptor agonist (OX2R) E2086 and the latest findings on its in-house discovered orexin receptor antagonist lemborexant (product name: DAYVIGO®) at the World Sleep Congress (World Sleep 2025) to be held in Singapore from September 5 to 10. Key presentations include results from the Phase Ib clinical study exploring the efficacy (Proof of Mechanism) of E2086 in people diagnosed with narcolepsy type 1 (NT1) and the Phase IV clinical study (SELENADE Study) investigating the effect of lemborexant in people whose insomnia was comorbid with depressive episodes of major depressive disorder and bipolar disorder (Poster 3-133). 

Pfizer to Acquire Metsera and its Next-Generation Obesity Portfolio < M&A, September 22, 2025

• Pfizer will acquire Metsera, a clinical-stage biopharmaceutical company accelerating the next generation of medicines for obesity and cardiometabolic diseases. Metsera has a portfolio of promising therapeutic candidates and combinations with four programs in clinical development and several next-generation programs with IND-enabling studies ongoing. This includes MET-097i, a weekly and monthly injectable GLP-1 receptor agonist (RA), both in Phase 2 development; MET-233i, a monthly amylin analog candidate being evaluated as monotherapy and in combination with MET-097i in Phase 1 development; two oral GLP-1 RA candidates expected to begin clinical trials imminently; and additional preclinical nutrient-stimulated hormone therapeutics. 

Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease in Italy < September 18, 2025

• CASGEVY is the first, and only, gene editing therapy approved for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD) in Europe; reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). In Italy, there are approximately 5,000 people 12 years and older living with TDT and around 2,300 with SCD. Italy joins a number of countries that have signed reimbursement agreements for CASGEVY including Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia and the United Arab Emirates.

Roche enters into a definitive merger agreement to acquire 89bio, and its phase 3 FGF21 analog for the therapy of moderate to severe MASH < FGF, M&A, September 18, 2025

• Roche has entered into a definitive merger agreement to acquire 89bio, Inc. (Nasdaq: ETNB), a publicly listed clinical-stage biopharmaceutical company pioneering the development of innovative therapies for the treatment of liver and cardiometabolic diseases. 89bio’s pegozafermin is a FGF21 analog currently in late-stage development for MASH in moderate and severe fibrotic patients (F2 and F3 stages) as well as cirrhotic patients (F4 stage). 89bio’s pegozafermin is a glycoPEGylated analog of fibroblast growth factor 21 (FGF21) specifically designed to address critical unmet needs in MASH.