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Clinical Trial News

Mixed Results for Adjuvant Immunotherapy in High-Risk Cutaneous Squamous Cell Carcinoma

Oncology
  • The phase 3 KEYNOTE-630 trial found that adjuvant pembrolizumab did not significantly improve progression-free survival in patients with high-risk cutaneous squamous cell carcinoma following surgery and radiation.
  • Despite missing its primary endpoint, pembrolizumab demonstrated a 50% reduction in cancer recurrence rates, with locoregional recurrence occurring in 13.8% versus 25.3% of placebo patients.
  • The competing phase 3 C-POST trial showed cemiplimab reduced the risk of recurrence or death by 68% compared to placebo, with FDA approval expected for this indication.
  • Treatment paradigms are shifting toward neoadjuvant immunotherapy approaches, with upcoming trials exploring response-adapted surgery and radiation strategies.

Pembrolizumab (MK-3475) Versus Placebo Following Surgery and Radiation in Participants With Locally Advanced Cutaneous Squamous Cell Carcinoma (MK-3475-630/KEYNOTE-630)

NCT03833167Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 4/1/2019

Study of Adjuvant Cemiplimab Versus Placebo After Surgery and Radiation Therapy in Patients With High Risk Cutaneous Squamous Cell Carcinoma

NCT03969004Active, Not RecruitingPhase 3
Regeneron Pharmaceuticals
Posted 6/4/2019

Dabrafenib and Trametinib Followed by Ipilimumab and Nivolumab or Ipilimumab and Nivolumab Followed by Dabrafenib and Trametinib in Treating Patients With Stage III-IV BRAFV600 Melanoma

NCT02224781Active, Not RecruitingPhase 3
National Cancer Institute (NCI)
Posted 9/8/2015

Study of REGN2810 in Patients With Advanced Cutaneous Squamous Cell Carcinoma

NCT02760498CompletedPhase 2
Regeneron Pharmaceuticals
Posted 4/7/2016

A Study to Compare the Administration of Encorafenib + Binimetinib + Nivolumab Versus Ipilimumab + Nivolumab in BRAF-V600 Mutant Melanoma With Brain Metastases

NCT04511013RecruitingPhase 2
SWOG Cancer Research Network
Posted 1/6/2021

Study of REGN2810 (Anti-PD-1) in Patients With Advanced Malignancies

NCT02383212CompletedPhase 1
Regeneron Pharmaceuticals
Posted 2/2/2015

deepeye Medical Receives CE Mark for AI-Powered Therapy Planning Assistant for AMD Treatment

AI
  • German healthtech company deepeye Medical has received CE mark approval for its predictive AI therapy planning assistant deepeye TPS, designed to enhance intravitreal injection therapy planning for neovascular AMD patients.
  • The AI system has been trained on thousands of patient cases from over 200 retina centers and clinically validated through collaboration projects with major pharmaceutical companies including Novartis, Bayer, and Roche.
  • The assistant analyzes OCT scans to provide disease activity assessment, 12-month therapy prognosis, and treatment recommendations, with the goal of reducing patient dropout and improving therapy adherence.
  • Clinical validation data shows the system helped triple visual acuity gains and double therapy adherence compared to other real-world studies when conducting double readings of OCT imaging.

Sandoz Launches First Interchangeable Denosumab Biosimilars in US, Expanding Access for Osteoporosis and Cancer Patients

Monoclonal AntibodyDrug Approval
  • Sandoz has launched WYOST® and Jubbonti®, the first and only FDA-approved interchangeable denosumab biosimilars in the United States, providing cost-effective alternatives to reference medicines XGEVA® and Prolia®.
  • The biosimilars target over 10 million US adults with osteoporosis and approximately 330,000 patients living with bone metastases from cancer.
  • Both products are human monoclonal antibodies that bind to RANKL protein and are approved for all indications of their respective reference medicines with identical dosing regimens.
  • The launch represents a significant milestone in biosimilar accessibility, potentially reducing treatment costs for patients with bone-related conditions and cancer complications.

A Phase 3 Study to Compare Between CT-P41 and US-licensed Prolia in Postmenopausal Women With Osteoporosis

NCT04757376CompletedPhase 3
Celltrion
Posted 6/17/2021

Sanofi Acquires Blueprint Medicines for $9.1 Billion to Strengthen Rare Disease and Immunology Portfolio

Rare DiseaseOrphan Drug
  • Sanofi will acquire Blueprint Medicines Corporation for $129.00 per share in cash, representing an equity value of approximately $9.1 billion, with additional contingent value rights potentially worth $9.5 billion total.
  • The acquisition adds Ayvakit/Ayvakyt (avapritinib), the only approved medicine for advanced and indolent systemic mastocytosis, which achieved net revenues of $479 million in 2024 with over 60% year-on-year growth.
  • Blueprint's pipeline includes elenestinib, a next-generation systemic mastocytosis treatment in phase 2/3 trials, and BLU-808, a wild-type KIT inhibitor with potential for broad immunological applications.
  • The transaction is expected to close in Q3 2025 and will be immediately accretive to gross margin and accretive to business operating income and EPS after 2026.

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

NCT04910685RecruitingPhase 2
Blueprint Medicines Corporation
Posted 11/30/2021

FDA Denies Approval for Elamipretide Despite Dramatic Heart Function Improvements in Barth Syndrome Patients

Rare DiseaseDrug ApprovalOrphan Drug
  • The FDA denied approval for elamipretide, a drug treating Barth syndrome, despite an advisory committee's 10-6 recommendation for approval and documented heart function improvements from 20% to normal levels.
  • One-year-old Jaylen Karle experienced heart failure from birth due to Barth syndrome, but his heart function normalized within a month of starting elamipretide treatment in early 2024.
  • Barth syndrome affects approximately 150 people in the U.S., and families fear losing access to the experimental drug if FDA approval doesn't come within six months.
  • The FDA provided a pathway for accelerated approval, but advocates call the denial "unconscionable" given the drug's life-saving potential for this rare genetic disorder.

InnoCare Presents Promising Clinical Data for Multiple Oncology Pipeline Candidates at Major Medical Conferences

Monoclonal Antibody
  • InnoCare presented clinical data for multiple oncology pipeline candidates at the 2025 ASCO Annual Meeting and EHA 2025 Congress, demonstrating significant therapeutic potential across various cancer types.
  • The BCL2 inhibitor mesutoclax (ICP-248) showed 100% overall response rates in both BTK-naïve chronic lymphocytic leukemia and mantle cell lymphoma patients, with combination therapy achieving 97.6% response rates in treatment-naïve patients.
  • The pan-TRK inhibitor zurletrectinib (ICP-723) demonstrated 100% confirmed response rates in pediatric patients with NTRK fusion-positive solid tumors and 83.7% response rates in adult patients.
  • The anti-CCR8 antibody ICP-B05 achieved 33.3% partial response rates in relapsed/refractory cutaneous T-cell lymphoma patients, with an 82.5% six-month progression-free survival rate.

FDA Tightens Nitrosamine Testing Requirements with New Guidelines Taking Effect This August

  • The FDA's new nitrosamine guidelines, effective August 2024, expand testing requirements to cover more than 250 compounds with detection limits as low as one part per billion.
  • Since 2018, the FDA has issued over 500 drug recalls due to unacceptable nitrosamine levels, making contamination control a critical industry priority.
  • Noncompliance with the new guidelines triggers immediate FDA Class II recalls and blocks new drug approvals, requiring companies to implement robust testing programs.
  • The enhanced requirements necessitate advanced analytical equipment and specialized expertise, with SK Pharmteco reporting an eight-fold increase in nitrosamine testing services since 2020.

J INTS BIO's Fourth-Generation EGFR-TKI JIN-A02 Shows Promising Anti-Tumor Activity in Resistant NSCLC Patients

  • J INTS BIO presented interim Phase 1/2 clinical trial results for JIN-A02, a fourth-generation EGFR-TKI designed to overcome resistance mutations that develop after third-generation EGFR-TKI treatment failure.
  • Confirmed partial responses were observed across multiple dose groups, with the 50mg cohort achieving a 77.3% tumor size reduction sustained over six consecutive treatment cycles.
  • The drug demonstrated a favorable safety profile with no dose-limiting toxicities up to 300mg and showed therapeutic activity against brain metastases.
  • J INTS BIO received government recognition through the 2025 Baby Unicorn Fostering Project and plans to initiate Phase 2 trials before year-end following FDA discussions.

A Phase 1/2 Study to Evaluate the Safety, Tolerability and PK of JIN-A02 in Patients With EGFR Mutant Advanced NSCLC

NCT05394831RecruitingPhase 1
J Ints Bio
Posted 7/30/2023

Imugene Activates First Australian Site for Phase II Colorectal Cancer Vaccine Trial

CAR-T
  • Imugene has activated its first Australian site for the Phase II Neo-POLEM trial, expanding recruitment for its PD1-Vaxx immunotherapy in both Australia and the United Kingdom.
  • The trial targets patients with mismatch repair-deficient or microsatellite instability-high colorectal cancer, a genetic subtype representing about 15% of colorectal cancer cases.
  • PD1-Vaxx is a neoadjuvant B-cell vaccine designed to trigger immune responses against PD-1, administered before surgery to evaluate major pathological response and tumor shrinkage.
  • Colorectal cancer affects more than 1.2 million new patients annually worldwide, with approximately 80% presenting with localized disease yet maintaining a 50% global mortality rate.

Researchers Unveil New Strategies to Harness Tumor Microenvironment for Enhanced Cancer Immunotherapy

Drug Approval
  • Researchers at the 2025 ASCO Annual Meeting revealed how the tumor microenvironment's complex balance of pro-tumor and anti-tumor immune forces can be manipulated to improve immunotherapy responses.
  • A clinical study combining the JAK inhibitor itacitinib with pembrolizumab achieved a 67% overall response rate in non-small cell lung cancer patients by resetting chronic interferon signaling.
  • The dual blockade of PD-1 and LAG3 with relatlimab plus nivolumab produces "marathon runner" T-cells with enhanced durability compared to PD-1 inhibition alone.

Phase 1b Study of Dato-DXd in Combination With Immunotherapy With or Without Carboplatin in Advanced or Metastatic Non-Small Cell Lung Cancer

NCT04612751Active, Not RecruitingPhase 1
AstraZeneca
Posted 2/2/2021

Study of E7046 in Subjects With Selected Advanced Malignancies

NCT02540291TerminatedPhase 1
Eisai Inc.
Posted 7/30/2015

A Phase 3 Study of Pembrolizumab + Epacadostat or Placebo in Subjects With Unresectable or Metastatic Melanoma (Keynote-252 / ECHO-301)

NCT02752074CompletedPhase 3
Incyte Corporation
Posted 6/21/2016

HS-20089 in Patients With Ovarian Cancer and Endometrial Cancer

NCT06014190RecruitingPhase 2
Hansoh BioMedical R&D Company
Posted 12/18/2023

A Study to Evaluate the Efficacy and Safety of Simtuzumab Combined With Gemcitabine for Metastatic Pancreatic Adenocarcinoma

NCT01472198CompletedPhase 2
Gilead Sciences
Posted 11/1/2011

A Study To Investigate The Safety And Toleration Of A Single Dose Of PF-04418948 In Healthy Volunteers

NCT01002963CompletedPhase 1
Pfizer
Posted 9/1/2009

Study of Mogamulizumab + MEDI4736 (Durvalumab) and Mogamulizumab + Tremelimumab in Subjects w/ Advanced Solid Tumors

NCT02301130CompletedPhase 1
Kyowa Kirin, Inc.
Posted 11/26/2014

Basket Study to Evaluate the Therapeutic Activity of Simlukafusp Alfa as a Combination Therapy in Participants With Advanced and/or Metastatic Solid Tumors

NCT03386721TerminatedPhase 2
Hoffmann-La Roche
Posted 2/19/2018

A Study of Safety, Pharmacokinetics, Pharmacodynamics of JNJ-61610588 in Participants With Advanced Cancer

NCT02671955TerminatedPhase 1
Janssen Research & Development, LLC
Posted 1/1/2016

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