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Clinical Trial News

FDA Approves First Non-Surgical Treatment for Non-Muscle Invasive Bladder Cancer

Drug ApprovalOncology
  • The FDA approved UroGen Pharma's Zusduri as the first drug to treat non-muscle invasive bladder cancer that hasn't spread beyond the inner layers of the organ.
  • In a late-stage trial with 223 patients, 78% showed complete response with all signs of cancer disappearing after treatment.
  • The gel-based formulation offers a non-surgical alternative to traditional procedures, administered as a simple drug instillation in a doctor's office.
  • The approval addresses a significant unmet need for approximately 82,000 Americans affected by this type of bladder cancer annually, with 59,000 experiencing recurrence.

Major US Pharmaceutical Wholesalers Expand Market Control Through Strategic Practice Management Acquisitions

  • The three largest US pharmaceutical wholesalers—Cardinal Health, Cencora, and McKesson—are leveraging vertical integration to expand their influence beyond traditional drug distribution into the buy-and-bill market.
  • These companies have strengthened their positions by acquiring stakes in practice management firms through private equity roll-up activities, enhancing their control over provider-administered drug channels.
  • The strategic acquisitions are creating new implications for biosimilar market access and adoption within the buy-and-bill segment.
  • This vertical integration approach is fundamentally reshaping traditional pharmaceutical distribution business models and creating new opportunities within the healthcare sector.

BBOT's BBO-10203 Shows Broad Anti-Tumor Activity in Science Publication, Advances to Phase 1 Trial

Oncology
  • BBOT published preclinical data in Science demonstrating that BBO-10203, a first-in-class oral inhibitor, effectively blocks RAS-PI3Kα interaction without inducing hyperglycemia across multiple tumor types.
  • The compound showed significant tumor growth inhibition in preclinical models and enhanced efficacy when combined with targeted therapies including CDK4/6 inhibitors and HER2 inhibitors.
  • BBO-10203 is currently being evaluated in the Phase 1 BREAKER-101 study for patients with HER2+ breast cancer, HR+/HER2- breast cancer, KRAS mutant colorectal cancer, and KRAS mutant non-small cell lung cancer.

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

Autonomize AI Secures $28 Million Series A to Scale AI-Driven Healthcare Operations Platform

AI
  • Autonomize AI raised $28 million in Series A funding led by Valtruis, The Cigna Group Ventures, and Tau Ventures to advance its Agentic AI Orchestration platform for healthcare operations.
  • The company's AI agents have already demonstrated significant impact, saving 36,000 clinical hours monthly and creating over 100,000 automated care plans while accelerating prior authorization decisions by up to 50%.
  • The platform addresses critical healthcare challenges by reducing administrative burden on clinicians through purpose-built AI agents that handle complex workflows like care management and utilization management.
  • Founded in 2022, Autonomize AI has gained traction across multiple healthcare segments, serving Fortune 100 healthcare enterprises and top pharmaceutical companies with measurable operational improvements.

European Commission Grants Orphan Drug Designation to Florbetaben (18F) for ATTR Amyloidosis Diagnosis

Rare Disease
  • Life Molecular Imaging's florbetaben (18F) has received orphan drug designation from the European Commission for diagnosing transthyretin (ATTR) amyloidosis, expanding its diagnostic applications beyond Alzheimer's disease.
  • The radiotracer, originally approved for detecting brain amyloid plaques, is being evaluated in a Phase 3 trial to validate its efficacy in diagnosing cardiac amyloidosis using PET imaging.
  • ATTR amyloidosis remains below the orphan disease threshold despite rising incidence rates, with fewer than five cases per ten thousand inhabitants in the general population.
  • The designation supports development of improved diagnostic tools for a rare disease where early detection is crucial for accessing life-saving therapies.

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088RecruitingPhase 3
Life Molecular Imaging GmbH
Posted 1/13/2023

Parallel Bio Raises $21 Million Series A to Revolutionize Drug Discovery with Human Organoid Platform

AIDrug Discovery
  • Parallel Bio secured $21 million in Series A funding led by AIX Ventures to advance its human-first drug discovery platform that uses organoids and AI to replicate the human immune system.
  • The company has partnered with eight pharmaceutical companies, including three Fortune 500 firms, to test over 50 drugs and immunotherapies using its organoid-based platform.
  • Centivax completed the first preclinical study on the platform for its universal flu vaccine candidate, demonstrating broad immune responses in human organoids derived from flu-exposed patients.
  • The platform aims to address the 95% failure rate of drugs in human trials despite success in animal studies by starting with true-to-life human models from the beginning.

Specialised Therapeutics Expands Incyte Partnership to Bring Two Novel Cancer Therapies to Asia-Pacific

Monoclonal AntibodyRare DiseaseOncology
  • Specialised Therapeutics has expanded its partnership with Incyte to include axatilimab and retifanlimab for distribution in Australia, New Zealand, and Singapore, with potential expansion to other Asia-Pacific countries.
  • Axatilimab, a first-in-class CSF-1R-blocking antibody approved by the FDA in August 2024, treats chronic graft-versus-host disease after failure of at least two prior systemic therapies.
  • Retifanlimab, a PD-1 inhibitor, is approved for treating squamous cell carcinoma of the anal canal and Merkel cell carcinoma, with Australia having the highest global incidence of the latter condition.
  • Both therapies are expected to undergo regulatory and reimbursement approval submissions in the region during 2025.

Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

NCT05067127CompletedPhase 3
Apellis Pharmaceuticals, Inc.
Posted 11/12/2021

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

NCT06585774RecruitingPhase 3
Incyte Corporation
Posted 1/21/2025

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564RecruitingPhase 2
Incyte Corporation
Posted 10/11/2024

MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06132256RecruitingPhase 2
Syndax Pharmaceuticals
Posted 12/11/2023

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

NCT04710576Active, Not RecruitingPhase 2
Syndax Pharmaceuticals
Posted 3/4/2021

Apex Labs Expands Phase 2b Psilocybin PTSD Trial to Israel with Ministry of Health Approval

  • Apex Labs received Israeli Ministry of Health approval to add two clinical trial sites for its SUMMIT-90 phase 2b study evaluating psilocybin for PTSD treatment.
  • The 160-patient double-blind, placebo-controlled trial will test APEX-90, a psilocybin macrodose capsule administered with psychotherapy for severe depression in PTSD patients.
  • Israel faces a severe mental health crisis with 44% of adults reporting depression and 42% experiencing PTSD, significantly higher than US and Canadian rates.
  • The expansion includes Tel Aviv University's Institute for Psychedelic Research and Be'er Yaakov Mental Hospital's Center for Psychedelic Studies as new trial sites.

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

Federal Circuit Upholds Invalidation of Agilent's CRISPR Guide RNA Patents in Synthego Victory

Gene Editing
  • The U.S. Court of Appeals for the Federal Circuit affirmed PTAB decisions invalidating Agilent Technologies' patents on chemically modified CRISPR guide RNAs, following challenges by Synthego Corp.
  • The court clarified that anticipation under patent law requires only one operable embodiment, not the full scope enablement mandated for patent validity under Section 112.
  • The ruling removes patent barriers for chemically modified guide RNAs featuring 2'-O-methyl and phosphonoacetate modifications, potentially accelerating CRISPR therapeutic development.
  • Synthego's CEO emphasized the decision keeps the path clear for broader innovation in the CRISPR therapeutics market and supports advancement of curative cell and gene therapies.

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