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Targeted Therapies Transform Biliary Tract Cancer Treatment Landscape with FGFR2 Inhibitors Leading Clinical Advances

Targeted TherapyPrecision MedicineOncology
  • Biliary tract cancer treatment has evolved from traditional cytotoxic chemotherapy to include targeted therapies based on specific molecular alterations, with FGFR2 inhibitors showing particularly promising results.
  • Pemigatinib and futibatinib, selective FGFR inhibitors, have demonstrated significant clinical efficacy with response rates of 35% and 42% respectively in patients with FGFR2 fusions.
  • Comprehensive next-generation sequencing has become the standard approach for identifying actionable molecular alterations, with IDH1/2 mutations, FGFR2 fusions, and ERBB2 alterations being the most clinically significant biomarkers.
  • The rarity of biliary tract cancers presents unique challenges in treatment access, making clinical trials essential for patients with molecular alterations that have FDA-approved therapies in other disease states.

Real-World Data Confirm Ivosidenib Efficacy in IDH1-Mutated Cholangiocarcinoma

Targeted TherapyReal World EvidenceOncology
  • The phase 3b ProvIDHe study demonstrated that ivosidenib achieved a median progression-free survival of 4.7 months and overall survival of 15.5 months in patients with IDH1-mutated cholangiocarcinoma.
  • Real-world data from 262 patients across 80 sites in 15 countries confirmed the efficacy profile established in the pivotal ClarIDHy trial that led to FDA approval.
  • The study enrolled heavily pretreated patients with a median of 2 prior lines of therapy, showing a disease control rate of 51.5% and objective response rate of 5.7%.

Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy)

NCT02989857CompletedPhase 3
Institut de Recherches Internationales Servier
Posted 2/20/2017

An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma

NCT05876754RecruitingPhase 3
Servier Affaires Médicales
Posted 5/3/2023

Onco-Innovations Appoints Dr. Islam Mohamed as Scientific Advisory Board Chair to Advance Oncology Pipeline

Oncology
  • Onco-Innovations Limited has appointed Dr. Islam Mohamed, an experienced radiation oncologist and clinical researcher, as Chair of its Scientific and Clinical Advisory Board to strengthen its clinical leadership capabilities.
  • Dr. Mohamed brings over two decades of experience in oncology innovation, having led numerous clinical trials in breast, lung, and oligometastatic cancers while serving on research ethics boards and national clinical trial committees.
  • The appointment reflects the company's commitment to advancing its oncology pipeline and translational research initiatives, with Dr. Mohamed's expertise in stereotactic radiation delivery and biotech investment expected to guide strategic R&D decisions.
  • Onco-Innovations holds an exclusive worldwide license to patented technology targeting solid tumors and aims to accelerate innovative oncology solutions through expert-led research.

Targeted Protein Degradation Emerges as Multi-Billion Dollar Therapeutic Frontier with First PROTAC Approval Expected in 2026

Drug ApprovalOncology
  • Targeted protein degradation (TPD) has evolved from niche science to mainstream drug development, with over 40 PROTAC candidates in clinical testing and the first potential market approval expected for Arvinas/Pfizer's ARV-471 by June 2026.
  • Major pharmaceutical companies have committed over $10 billion in partnerships since 2024, with deals including AbbVie's $1.64 billion agreement with Neomorph and LEO Pharma's $1.7 billion alliance with Gilead Sciences.
  • The Asia-Pacific region, particularly China and South Korea, has emerged as a leading hub for TPD research, with multiple companies advancing candidates through late-stage clinical trials.
  • The global TPD market is projected to surge from $1 billion currently to $6.94 billion by 2035, driven by advances in computational tools and AI-enabled drug design.

BrECADD Chemotherapy Preserves Fertility Better Than Standard Treatment in Advanced Hodgkin's Lymphoma

Oncology
  • The new BrECADD chemotherapy regimen preserves fertility significantly better than the previous standard eBEACOPP treatment in advanced Hodgkin's lymphoma patients without compromising cure rates.
  • Three years post-treatment, 95% of women and 86% of men in the BrECADD group had normal hormone levels compared to 73% and 40% respectively in the eBEACOPP group.
  • The HD21 phase III trial involving over 1,500 participants across 233 centers in nine countries demonstrates BrECADD's superior fertility preservation, particularly benefiting male patients' chances of biological paternity.
  • BrECADD has become the new standard first-line treatment for patients wishing to have children at University Hospital Cologne and in current Onkopedia guidelines.

HD21 for Advanced Stages

NCT02661503Active, Not RecruitingPhase 3
University of Cologne
Posted 7/1/2016

BBOT Completes $382 Million SPAC Merger to Advance Next-Generation RAS-Targeted Cancer Therapeutics

Oncology
  • BridgeBio Oncology Therapeutics (BBOT) completed its business combination with Helix Acquisition Corp. II on August 11, 2025, raising approximately $382 million in gross proceeds to advance RAS-pathway cancer therapeutics.
  • The company secured $261 million through a PIPE investment led by Cormorant Asset Management, with only 39% shareholder redemption rate marking the second lowest for biotech de-SPAC transactions since 2022.
  • BBOT's pipeline includes three clinical-stage programs targeting KRAS mutations and PI3Kα malignancies, including BBO-8520 for KRASG12C tumors, BBO-10203 as a novel RAS-PI3Kα interaction blocker, and BBO-11818 as a panKRAS inhibitor.
  • The combined company expects to have approximately $490 million in cash and equivalents to accelerate development of therapeutics targeting the two most prevalent oncogenes in human tumors.

BBO-11818 in Adult Subjects With KRAS Mutant Cancer

NCT06917079RecruitingPhase 1
TheRas, Inc., d/b/a BBOT (BridgeBio Oncology Therapeutics)
Posted 3/31/2025

Dose Escalation and Dose Expansion Study of BBO-10203 in Subjects with Advanced Solid Tumors

2024-519445-29-00RecruitingPhase 1
Theras Inc.
Posted 10/29/2024

Open-label Study of BBO-8520 in Adult Subjects With KRASG12C Non-small Cell Lung Cancer

NCT06343402RecruitingPhase 1
TheRas, Inc., d/b/a BBOT (BridgeBio Oncology Therapeutics)
Posted 5/22/2024

Compass Therapeutics Receives FDA Fast Track Designation for CTX-009 Bispecific Antibody

Oncology
  • Compass Therapeutics received FDA Fast Track Designation for CTX-009, its bispecific DLL4/VEGF-A antibody, when used in combination with other treatments.
  • The designation indicates FDA recognition of CTX-009's potential to address unmet medical needs and commitment to supporting its development.
  • CMPX shares surged 4.01% on the news, marking the third consecutive day of gains with a cumulative increase of 7.95% over three days.
  • The Fast Track Designation is expected to expedite the development and approval process while potentially attracting increased investor attention and collaboration opportunities.

Genmab Advances Two Phase 3 and Phase 2 Trials Testing Acasunlimab Combinations in Lung Cancer and Melanoma

Oncology
  • Genmab is conducting a Phase 3 trial evaluating acasunlimab combined with pembrolizumab versus docetaxel in PD-L1 positive metastatic non-small cell lung cancer patients who progressed after prior PD-1/PD-L1 inhibitor and platinum-containing chemotherapy.
  • The company is simultaneously running a Phase 2 study testing acasunlimab as monotherapy and in combination with pembrolizumab for advanced melanoma patients who progressed after checkpoint inhibitor treatment.
  • Both trials represent significant opportunities to establish new treatment options for challenging cancer types with limited therapeutic alternatives, potentially impacting Genmab's market position and stock performance.

PDS Biotech's Novel IL-12 ADC Advances to Phase 2 Stage 2 in Metastatic Colorectal Cancer

Oncology
  • PDS Biotech's PDS01ADC met RECIST v1.1 criteria for expansion to Stage 2 of a Phase 2 trial in metastatic colorectal cancer, achieving at least 6 objective responses in 9 patients.
  • The novel antibody-drug conjugate combines IL-12 with NHS76 antibody to target tumor necrosis regions while minimizing systemic IL-12 exposure and associated toxicities.
  • The NCI-led study will expand enrollment to 22 patients total, with completion of recruitment anticipated by Q4 2025 for this deadly cancer affecting over 150,000 Americans annually.

PDS01ADC in Combination With Hepatic Artery Infusion Pump (HAIP) and Systemic Therapy for Subjects With Metastatic Colorectal Cancer, Intrahepatic Cholangiocarcinoma, or Metastatic Adrenocortical Carcinoma

NCT05286814RecruitingPhase 2
National Cancer Institute (NCI)
Posted 10/24/2022

Formycon Completes Enrollment for Keytruda Biosimilar Study, Skips Phase III Trial with FDA Approval

FDA ApprovalOncology
  • Formycon AG successfully completed patient enrollment for its Dahlia pharmacokinetic study with 96 participants, comparing biosimilar candidate FYB206 to Keytruda in melanoma patients.
  • The FDA approved a streamlined clinical development program allowing Formycon to skip the Phase III trial, accelerating development and reducing costs significantly.
  • Results from the primary endpoint are expected in Q1 2026, with potential market entry after Keytruda's exclusivity expires in 2029 for the US and 2030 for the EU.
  • The development targets the world's best-selling drug with $29.5 billion in 2024 sales, potentially reaching over $50 billion by 2032.

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