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Genor Biopharma Secures NMPA Approval for Lerociclib CDK4/6 Inhibitor in Advanced Breast Cancer

Drug ApprovalTargeted TherapyOncology
  • Genor Biopharma Holdings Ltd. received China National Medical Products Administration (NMPA) approval for Lerociclib (GB491), a novel CDK4/6 inhibitor for advanced breast cancer treatment.
  • The oral bioavailable drug targets hormone receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer in adult patients.
  • Lerociclib will be used in combination with aromatase inhibitors as initial therapy or with fulvestrant following endocrine therapy progression.
  • The approval represents a significant milestone resulting from successful collaboration between Genor Biopharma and G1 Therapeutics Inc.

Pharmacists Lead Implementation of Advanced Cell and Gene Therapies Across Major Health Systems

Drug Approval
  • Pharmacists are uniquely positioned to lead the implementation of advanced cell and gene therapies, serving as both clinical and operational experts in health systems managing these complex treatments.
  • Major health systems are already delivering widespread advanced therapeutics, with Mayo Clinic offering 9 active therapies, UC San Diego Health providing 12, and Children's Hospital of Philadelphia making 12 available to eligible patients.
  • Significant challenges include high costs, manufacturing variability, patient hesitancy toward gene manipulation, and the need for new governance structures and educational frameworks.
  • The first CRISPR-based medicine, exagamglogene autotemcel (Casgevy), was approved in 2023 for treating sickle cell disease and transfusion-dependent β-thalassemia, marking a milestone in gene therapy applications.

Eton Pharmaceuticals Receives FDA Approval for KHINDIVI, First Hydrocortisone Oral Solution for Pediatric Adrenal Insufficiency

Drug ApprovalRare Disease
  • Eton Pharmaceuticals announced FDA approval of KHINDIVI (hydrocortisone) oral solution, marking the first and only FDA-approved liquid hydrocortisone formulation for pediatric patients aged 5 and older with adrenocortical insufficiency.
  • The ready-to-use 1mg/ml oral solution eliminates the need to split or crush tablets, offering precise dosing for children who have difficulty swallowing pills or require gastric tube administration.
  • Commercial launch is expected the week of June 2nd, with Eton projecting combined peak sales of KHINDIVI and ALKINDI SPRINKLE to exceed $50 million annually.
  • The approval addresses a decades-long unmet need in pediatric endocrinology, providing accurate dosing capabilities critical for managing the more than 5,000 U.S. patients aged 5-17 with adrenal insufficiency.

FDA Approves First-in-Class TRPM8 Receptor Agonist Tryptyr for Dry Eye Disease Treatment

Drug Approval
  • The FDA approved Tryptyr (acoltremon ophthalmic solution) 0.003%, a first-in-class TRPM8 receptor agonist that stimulates corneal sensory nerves to rapidly increase natural tear production in dry eye disease patients.
  • In pivotal Phase 3 trials COMET-2 and COMET-3 involving over 930 patients, up to four times more Tryptyr patients experienced at least a 10mm increase in natural tear production at Day 14 compared to vehicle (42.6% vs 8.2% and 53.2% vs 14.4%, respectively).
  • The approval addresses a significant unmet need, as approximately 38 million individuals in the U.S. suffer from dry eye disease, yet only 13% of surveyed patients feel their condition is well-managed with current treatments.
  • Alcon expects to launch Tryptyr in the U.S. during the third quarter of 2025, marking the company's first prescription pharmaceutical approval since becoming an independent publicly traded eye care company.

Study Evaluating the Safety and Efficacy of AR-15512 (COMET-3)

NCT05360966CompletedPhase 3
Aerie Pharmaceuticals
Posted 7/18/2022

Study Evaluating the Safety and Efficacy of AR-15512

NCT05285644CompletedPhase 3
Aerie Pharmaceuticals
Posted 5/9/2022

GSK's Tebipenem HBr Phase III Trial Stopped Early for Efficacy in Complicated UTI Treatment

Drug Approval
  • GSK and Spero Therapeutics announced the pivotal Phase III PIVOT-PO trial for tebipenem HBr was stopped early for efficacy following a recommendation from an Independent Data Monitoring Committee based on data from 1,690 patients.
  • The trial met its primary endpoint of non-inferiority compared to intravenous imipenem-cilastatin in hospitalized adult patients with complicated urinary tract infections, including pyelonephritis.
  • If approved, tebipenem HBr would be the first oral carbapenem antibiotic for US patients with complicated UTIs, addressing a significant unmet medical need in a market with 2.9 million annual cases.
  • GSK plans to include the data in a US FDA filing in the second half of 2025, marking the company's second anti-infective program stopped early for efficacy in Phase III.

A Study of Oral Tebipenem Pivoxil Hydrobromide (TBP-PI-HBr) Compared to Intravenous Imipenem-cilastatin in Participants With Complicated Urinary Tract Infection (cUTI) or Acute Pyelonephritis (AP)

NCT06059846CompletedPhase 3
Spero Therapeutics
Posted 12/21/2023

ImmunityBio Partners with Saudi Arabia to Bring FDA-Approved Cancer BioShield Platform to Middle East

Drug Approval
  • ImmunityBio signed a strategic MOU with Saudi Arabia's Ministry of Investment, KFSHRC, and KAIMRC to introduce the FDA-approved Cancer BioShield platform to the Middle East region.
  • The BioShield platform, powered by Anktiva (nogapendekin alfa inbakicept), represents the world's first FDA-approved IL-15 superagonist that activates natural killer cells and T cells rather than suppressing them like conventional treatments.
  • This collaboration aims to establish a regional center of excellence for immune-restorative therapies, addressing lymphopenia as the root cause of cancer mortality rather than treating cancer as the primary disease.
  • The partnership will enable clinical trials, technology transfer, and training of healthcare professionals in advanced cell therapy protocols across the MENA region.

FDA Expands MenQuadfi Meningococcal Vaccine Approval to Include Infants as Young as 6 Weeks

Drug Approval
  • The FDA approved an expanded indication for Sanofi's MenQuadfi meningococcal vaccine to include children aged 6 weeks to 23 months, making it the only MenACWY vaccine with no upper age limit.
  • Clinical trials involving 4,321 infants demonstrated non-inferior immune responses compared to licensed comparator vaccines, with no unexpected safety concerns in the youngest age groups.
  • The approval addresses a significant increase in meningococcal disease cases in the United States, with infants having the highest risk of invasive meningococcal disease that can be fatal within 24 hours.
  • The vaccine protects against Neisseria meningitidis serogroups A, C, W, and Y, with the majority of current US cases being serotype Y which is covered by the vaccine.

DLL3-Targeted Therapies Show Promise for Refractory Small Cell Lung Cancer Despite Clinical Challenges

CAR-TDrug Approval
  • Delta-like ligand 3 (DLL3) emerges as a critical therapeutic target in small cell lung cancer, with expression in 70-80% of SCLC cases and minimal presence in normal tissues.
  • Tarlatamab, the first FDA-approved DLL3-targeting bispecific T-cell engager, demonstrates clinical efficacy in extensive-stage SCLC patients who progressed after chemotherapy.
  • Multiple DLL3-targeted approaches including antibody-drug conjugates, CAR-T cells, and trispecific antibodies are advancing through clinical trials despite early setbacks with rovalpituzumab tesirine.
  • Clinical challenges persist including cytokine release syndrome, tumor heterogeneity, and the need for biomarker-guided patient selection strategies.

A Trial of SHR-4849 in Advanced Solid Tumors

NCT06443489RecruitingPhase 1
Suzhou Suncadia Biopharmaceuticals Co., Ltd.
Posted 6/26/2024

AMG 757 and AMG 404 in Subjects With Small Cell Lung Cancer (SCLC)

NCT04885998CompletedPhase 1
Amgen
Posted 9/27/2021

A Phase 2 Study of Tarlatamab in Patients With Small Cell Lung Cancer (SCLC)

NCT05060016Active, Not RecruitingPhase 2
Amgen
Posted 12/1/2021

Rovalpituzumab Tesirine (SC16LD6.5) in Recurrent Small Cell Lung Cancer

NCT01901653CompletedPhase 1
Stemcentrx
Posted 7/1/2013

Study Evaluating Tarlatamab in Chinese Participants With Advanced Small Cell Lung Cancer After Two or More Prior Lines of Treatment

NCT06502977Active, Not RecruitingPhase 2
Amgen
Posted 8/27/2024

A Phase 1 Study Evaluating the Safety, Tolerability and Efficacy of AMG 119 in Subjects With RR SCLC

NCT03392064SuspendedPhase 1
Amgen
Posted 9/10/2018

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Anti-tumor Activity of RO7616789 in Advanced Small Cell Lung Cancer and Other Neuroendocrine Carcinomas

NCT05619744CompletedPhase 1
Hoffmann-La Roche
Posted 1/23/2023

Durvalumab ± Tremelimumab in Combination With Platinum Based Chemotherapy in Untreated Extensive-Stage Small Cell Lung Cancer (CASPIAN)

NCT03043872Active, Not RecruitingPhase 3
AstraZeneca
Posted 3/27/2017

A Phase 1 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Tarlatamab in Subjects With Small Cell Lung Cancer (DeLLphi-300)

2023-506541-39-00Active, Not RecruitingPhase 1
Amgen Inc.
Posted 12/26/2017

Study of DLL3-CAR-NK Cells in the Treatment of Extensive Stage Small Cell Lung Cancer

NCT05507593Unknown StatusPhase 1
Tianjin Medical University Cancer Institute and Hospital
Posted 9/1/2022

A Study of Carboplatin Plus Etoposide With or Without Atezolizumab in Participants With Untreated Extensive-Stage (ES) Small Cell Lung Cancer (SCLC)

NCT02763579CompletedPhase 3
Hoffmann-La Roche
Posted 6/7/2016

A Study in Participants With Advanced Cancers Associated With Expression of DLL3 (MK-6070-001/HPN328-4001)

NCT04471727RecruitingPhase 1
Harpoon Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Posted 12/14/2020

A Study of Rovalpituzumab Tesirine as Maintenance Therapy Following First- Line Platinum-Based Chemotherapy in Participants With Extensive Stage Small Cell Lung Cancer (MERU)

NCT03033511TerminatedPhase 3
AbbVie
Posted 2/7/2017

First-Line Tarlatamab in Combination With Carboplatin, Etoposide, and PD-L1 Inhibitor in Subjects With Extensive Stage Small Cell Lung Cancer (ES-SCLC)

NCT05361395RecruitingPhase 1
Amgen
Posted 8/24/2022

A Study of Hospital-at-Home for People Receiving Tarlatamab

NCT06957314RecruitingNot Applicable
Memorial Sloan Kettering Cancer Center
Posted 4/23/2025

Study Comparing Rovalpituzumab Tesirine Versus Topotecan in Subjects With Advanced or Metastatic Small Cell Lung Cancer With High Levels of Delta-like Protein 3 (DLL3) and Who Have First Disease Progression During or Following Front-line Platinum-based Chemotherapy (TAHOE)

NCT03061812CompletedPhase 3
AbbVie
Posted 4/11/2017

PET/CT Imaging of Small Cell Lung Cancer Using 89Zr-DFO-SC16.56

NCT04199741RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 12/11/2019

Study of Rovalpituzumab Tesirine (SC16LD6.5) for Third-Line and Later Treatment of Subjects With Relapsed or Refractory Delta-Like Protein 3-Expressing Small Cell Lung Cancer

NCT02674568CompletedPhase 2
AbbVie
Posted 1/25/2016

A Study to Test Different Doses of BI 764532 in Patients With Small Cell Lung Cancer and Other Neuroendocrine Tumours That Are Positive for DLL3

NCT04429087RecruitingPhase 1
Boehringer Ingelheim
Posted 7/29/2020

Study Comparing Tarlatamab With Standard of Care Chemotherapy in Relapsed Small Cell Lung Cancer

NCT05740566Active, Not RecruitingPhase 3
Amgen
Posted 5/31/2023

Study Evaluating Tarlatamab After Chemoradiotherapy in Limited-Stage Small-Cell Lung Cancer (LS-SCLC)

NCT06117774RecruitingPhase 3
Amgen
Posted 2/20/2024

World ADC San Diego Conference Highlights Growing Industry Focus on Antibody-Drug Conjugates

OncologyDrug Approval
  • The 15th World ADC San Diego conference (November 4-7, 2024) represents the industry's largest and most comprehensive ADC-focused forum, featuring 120+ speakers across six development tracks.
  • Recent ADC breakthroughs include Enhertu's tumor-agnostic accelerated approval and practice-changing combination results from PADCEV, driving unprecedented industry attention.
  • Major pharmaceutical companies including Pfizer, AbbVie, J&J, Merck, and others have engaged in billion-dollar ADC partnerships and acquisitions, positioning ADCs at the forefront of oncology R&D.
  • The conference addresses end-to-end ADC development challenges from discovery to manufacturing, reflecting the field's evolution toward front-line cancer treatments.

Eisai Launches DAYVIGO, First Orexin Receptor Antagonist for Insomnia Treatment in China

Drug Approval
  • Eisai Co., Ltd. has launched DAYVIGO (lemborexant), the first orexin receptor antagonist approved for insomnia treatment in China, targeting approximately 172.5 million Chinese adults suffering from the condition.
  • The dual orexin receptor antagonist works through a novel mechanism by regulating sleep-wake rhythm rather than acting directly on sleepiness like conventional benzodiazepine treatments.
  • DAYVIGO received Chinese regulatory approval on May 27, 2025, based on global Phase 3 studies involving approximately 2,000 patients and a China-specific Phase 3 trial.
  • The drug is now approved in more than 25 countries worldwide and offers a new treatment option for the 15.0% of Chinese adults affected by insomnia.

A Study of Lemborexant in Chinese Participants With Insomnia Disorder

NCT04549168CompletedPhase 3
Eisai Co., Ltd.
Posted 11/6/2020

Study of the Efficacy and Safety of Lemborexant in Subjects 55 Years and Older With Insomnia Disorder (SUNRISE 1)

NCT02783729CompletedPhase 3
Eisai Inc.
Posted 5/31/2016

Long-term Study of Lemborexant in Insomnia Disorder (SUNRISE 2)

NCT02952820CompletedPhase 3
Eisai Inc.
Posted 11/15/2016

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