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NHS Becomes First Health System Globally to Approve 'Trojan Horse' Cancer Therapy for Multiple Myeloma

Drug ApprovalTargeted TherapyOncology
  • The NHS in England has become the first health system worldwide to approve belantamab mafodotin (Blenrep), a novel antibody-drug conjugate therapy for multiple myeloma patients.
  • Clinical trials demonstrated the treatment delayed disease progression by an average of three years compared to just over one year with standard daratumumab therapy.
  • Approximately 1,500 patients annually with relapsed or refractory multiple myeloma will be eligible for this targeted therapy through the NHS Cancer Drugs Fund.
  • The "Trojan horse" mechanism allows the drug to infiltrate cancer cells and release lethal molecules from within, representing a significant advancement in myeloma treatment.

Tazemetostat-Pinometostat Combination Shows Promise for B-Cell Lymphoma Treatment Resistance

Targeted Therapy
  • Researchers at The Institute of Cancer Research, London, have demonstrated that combining tazemetostat with DOT1L inhibitor pinometostat can overcome treatment resistance in B-cell lymphoma patients.
  • The drug combination significantly shrunk tumors in preclinical models, with treated tumors shrinking while control tumors tripled in size after 15 days.
  • The combination therapy could potentially benefit thousands of patients with diffuse large B-cell lymphoma, the most common type affecting around 5,000 UK patients annually.
  • Clinical trials are anticipated to test this dual-targeting approach that combines EZH2 and DOT1L inhibition to overcome cancer's drug resistance mechanisms.

Johnson & Johnson's Bleximenib Shows Promising Results in Phase 1b AML Trial with 82% Response Rate

Targeted Therapy
  • Johnson & Johnson's investigational menin inhibitor bleximenib demonstrated an 82% overall response rate in relapsed/refractory AML patients when combined with venetoclax and azacitidine in Phase 1b trials.
  • The combination therapy showed a favorable safety profile with only 4% of patients experiencing differentiation syndrome and no cardiac safety signals at the recommended Phase 2 dose.
  • Bleximenib targets AML patients with KMT2A gene rearrangements or NPM1 mutations, representing genetically defined subpopulations with particularly poor prognoses and limited treatment options.

A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies

NCT05453903RecruitingPhase 1
Janssen Research & Development, LLC
Posted 10/4/2022

Toripalimab Emerges as Preferred PD-1 Inhibitor for Metastatic Nasopharyngeal Carcinoma Despite Access Challenges

CAR-TTargeted Therapy
  • Toripalimab has received NCCN category 1 recommendation for nasopharyngeal carcinoma based on positive phase 3 data in both first-line and second-line settings, unlike pembrolizumab which showed negative results in the KEYNOTE-122 trial.
  • Clinical practitioners report easier insurance coverage for pembrolizumab compared to toripalimab, leading many to use pembrolizumab off-label in combination with chemotherapy despite inferior clinical evidence.
  • PD-1 inhibitors combined with chemotherapy represent the established standard of care for systemic recurrent nasopharyngeal carcinoma, with EBV levels serving as useful biomarkers for monitoring treatment efficacy.
  • Future treatment approaches may focus on novel combinations including CAR-T cell therapy, tumor-infiltrating lymphocytes, and targeted therapies like EGFR inhibitors to improve outcomes while reducing toxicity.

PD-1 Blockade Combined With De-intensified Chemoradiotherapy Sparing Concurrent Cisplatin in Nasopharyngeal Carcinoma

NCT04907370Active, Not RecruitingPhase 3
Sun Yat-sen University
Posted 8/1/2021

Study of Pembrolizumab (MK-3475) in Platinum Pre-treated Recurrent/Metastatic Nasopharyngeal Cancer (MK-3475-122/KEYNOTE-122)

NCT02611960CompletedPhase 3
Merck Sharp & Dohme LLC
Posted 4/18/2016

The Efficacy and Safety Study of TORIPALIMAB INJECTION Combined With Chemotherapy for Nasophapyngeal Cancer

NCT03581786CompletedPhase 3
Shanghai Junshi Bioscience Co., Ltd.
Posted 10/18/2018

Monopar Therapeutics Launches FDA-Authorized Expanded Access Program for uPAR-Targeted Radiopharmaceuticals in Advanced Cancers

Targeted Therapy
  • Monopar Therapeutics and EDNOC have received FDA authorization for an expanded access program providing MNPR-101-Zr imaging agent and MNPR-101-Lu therapeutic agent to patients with advanced solid tumors.
  • The program targets aggressive cancers including triple-negative breast, pancreatic, and colorectal cancers through selective targeting of the urokinase plasminogen activator receptor (uPAR).
  • EDNOC in Houston, Texas serves as the treatment center and is among the first private outpatient facilities designated as a Radiopharmaceutical Therapy Center of Excellence by SNMMI.
  • The expanded access program follows the initiation of Phase 1 clinical trials in Australia and represents continued progress in Monopar's radiopharmaceutical pipeline development.

MNPR-101-PCTA-177Lu Expanded Access Program (EAP) for Patients With Solid Tumor Cancer

NCT06980519available
Monopar Therapeutics

MNPR-101-DFO*-89Zr Expanded Access Program (EAP) for Patients With Solid Tumor Cancer

NCT06980506available
Monopar Therapeutics

Bristol Myers Squibb Acquires Novel Prostate Cancer Radiopharmaceutical for $1.35 Billion

Targeted TherapyOncology
  • Bristol Myers Squibb agreed to pay $350 million upfront to Philochem for worldwide rights to OncoACP3, an experimental radiopharmaceutical targeting the ACP3 protein for prostate cancer diagnosis and treatment.
  • OncoACP3 targets ACP3, a biomarker highly expressed in prostate cancer that potentially exceeds PSMA expression levels, offering an alternative pathway to current PSMA-targeted therapies.
  • The deal includes up to $1 billion in additional milestone payments and royalties, positioning Bristol Myers to compete with Novartis in the rapidly expanding radiopharmaceutical market.
  • RayzeBio, Bristol Myers' radiopharmaceutical subsidiary acquired for $4.1 billion, will lead development of OncoACP3 through Phase 1 therapeutic testing.

Triplet Therapy Shows Superior Efficacy in BRAF-Mutant Melanoma Brain Metastases

Targeted Therapy
  • The triplet combination of encorafenib, binimetinib, and nivolumab achieved a median progression-free survival of 6.2 months compared to 1.5 months with nivolumab plus ipilimumab in BRAF V600-mutant melanoma brain metastases.
  • Intracranial response rates were significantly higher with the triplet regimen at 75% versus 13% with the standard immunotherapy doublet.
  • The phase 2 SWOG S2000 trial represents the first randomized study comparing treatment approaches in this challenging patient population with historically poor outcomes.

Anti-PD 1 Brain Collaboration for Patients With Melanoma Brain Metastases

NCT02374242Active, Not RecruitingPhase 2
Melanoma Institute Australia
Posted 11/4/2014

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

Day One Biopharmaceuticals Appoints Michael Vasconcelles as Head of R&D to Drive Pipeline Expansion

OncologyTargeted TherapyLeadership Change
  • Day One Biopharmaceuticals has appointed Michael Vasconcelles, M.D., as Head of Research and Development, bringing over 25 years of oncology research and development expertise to the company.
  • Dr. Vasconcelles will oversee the company's research, development, and medical affairs infrastructure while supporting OJEMDA's growth and expanding the pipeline of targeted therapies.
  • The appointment comes at a pivotal time for Day One as it transitions from focusing primarily on pediatric cancer to developing transformative medicines for patients of all ages with life-threatening diseases.
  • Dr. Vasconcelles previously served as Executive Vice President at ImmunoGen, where he led the transformation into a global biotech enterprise and optimized ELAHERE's commercialization strategy.

Nordic Capital Invests in Minerva Imaging to Build Global Radiopharmaceutical Platform

Targeted TherapyPrecision MedicineOncology
  • Nordic Capital has announced a strategic investment in Minerva Imaging, a Denmark-based contract research organization specializing in targeted radioligand therapies for cancer treatment.
  • The partnership aims to accelerate Minerva's international expansion and strengthen its integrated platform serving pharmaceutical and biotechnology companies in radiopharmaceutical development.
  • Minerva reported revenue of more than DKK 225 million ($35 million) in 2024 and employs over 150 professionals, including PhD-level scientists at its Copenhagen headquarters.
  • The global radiopharmaceuticals market is positioned for significant growth driven by increasing cancer incidence and growing demand for precision medicines.

Citius Oncology Partners with Cardinal Health for LYMPHIR Distribution Ahead of Commercial Launch

Targeted TherapyDrug ApprovalOrphan Drug
  • Citius Oncology has entered into a distribution services agreement with Cardinal Health to support the upcoming U.S. commercial launch of LYMPHIR for cutaneous T-cell lymphoma treatment.
  • LYMPHIR is an FDA-approved targeted immunotherapy indicated for adults with relapsed or refractory Stage I-III cutaneous T-cell lymphoma after at least one prior systemic therapy.
  • The partnership leverages Cardinal Health's proven specialty pharmaceutical distribution capabilities to ensure efficient and reliable access to LYMPHIR for healthcare providers and patients.
  • Management estimates the initial market for LYMPHIR currently exceeds $400 million and is underserved by existing therapies.

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