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Clinical Trial News

Q3 2025 Biopharma Catalyst Monitor Highlights 18 Key FDA Decisions and Clinical Trial Milestones

Drug ApprovalRare Disease
  • A new quarterly report identifies 18 significant biopharma catalyst events expected in Q3 2025, including FDA approval decisions for multiple companies across diverse therapeutic areas.
  • Key regulatory milestones include potential approvals for Ascendis Pharma's Skytrofa for adult growth hormone deficiency, PTC Therapeutics' sepiapterin for phenylketonuria, and Lenz Therapeutics' presbyopia treatment LNZ100.
  • The report, based on key opinion leader interviews, also covers ongoing clinical trials for Eli Lilly's orforglipron for obesity and aTyr Pharma's efzofitimod for pulmonary sarcoidosis.
  • Industry experts will assess the commercial potential of these treatments and their impact on existing therapeutic landscapes across multiple disease areas.

SurgiVance Secures $2.2M NIH Grant to Advance Direct-to-Digital Pathology Platform

AIPrecision Medicine
  • SurgiVance Inc. received a $2,245,000 National Cancer Institute Small Business Innovation Research Direct-to-Phase II grant to commercialize its "pathology lab-in-a-box" technology.
  • The platform enables direct-to-digital, in-office pathology using advanced imaging hardware and AI processing on whole tissue without traditional glass microscope slides.
  • Clinical studies will be conducted at NYU Langone Health, The Rockefeller University, Northwell Health, and Oregon Health & Science University targeting cancer at cellular and molecular levels.
  • The technology promises to deliver histopathologic diagnosis in minutes, potentially transforming cancer management and precision medicine through 3D molecular imaging biomarkers.

Answer ALS Launches AI-Powered Drug Discovery Initiative to Accelerate ALS Treatment Development

AIDrug Discovery
  • Answer ALS has launched LADDIA, a groundbreaking AI-powered drug discovery collaboration with GATC Health, Pennington Biomedical Research Center, and Tulane University to accelerate ALS treatment development.
  • The initiative leverages the world's largest open-access ALS dataset through the Neuromine Data Portal and GATC Health's proprietary AI platform to identify druggable therapeutic targets.
  • Currently, there are no known viable treatments for ALS, making this AI-driven approach critical for identifying new therapeutic pathways for the neurodegenerative disease.
  • The collaboration will roll out in two phases, focusing first on building infrastructure and then activating collaborative projects to generate high-impact scientific outputs.

Sutro Biopharma Partners with FDA to Establish Regulatory Standards for Antibody Drug Conjugates

Oncology
  • Sutro Biopharma has entered into a research collaboration with the FDA to develop reference materials that will improve regulatory standards and enhance analytical methods for antibody drug conjugate development.
  • The partnership will utilize Sutro's cell-free XpressCF® technology to precisely engineer ADCs with predefined attributes, combined with FDA's analytical capabilities to fully characterize these materials.
  • The collaboration involves Sutro and the FDA's Office of Pharmaceutical Quality jointly leading study design and selection of target antigens, payload-linkers, and drug conjugation sites representative of both approved and developmental ADCs.
  • Results from this collaboration are expected to enhance the FDA's capacity for analytical characterization of ADCs and improve quality assessments across the industry.

FDA Requests Additional Manufacturing Data for Telix's Kidney Cancer Diagnostic Drug Zircaix

Oncology
  • The FDA has requested additional manufacturing data from Telix Pharmaceuticals for its kidney cancer diagnostic drug Zircaix, citing deficiencies in proving commercial manufacturing comparability to clinical trial processes.
  • Zircaix would be the first PET scan-based drug specifically designed for kidney cancer detection in the United States, targeting clear cell renal cell carcinoma which affects over 430,000 people worldwide.
  • Telix shares plunged 18.8% following the announcement, marking the company's worst trading day on record as investors reacted to potential regulatory delays.
  • The company believes the FDA's concerns are readily addressable and plans to begin submission remediation immediately to advance the approval process.

Iovance Biotherapeutics Shares Surge 14% on Amtagvi FDA Approval and New CEO Appointment

Drug ApprovalLeadership Change
  • Iovance Biotherapeutics shares surged 14.46% in pre-market trading following FDA approval of its cancer treatment Amtagvi and positive clinical trial results.
  • The company appointed Corleen Roche as new CEO, bringing extensive biotech financial management and commercial launch experience to enhance strategic direction.
  • Despite reducing guidance, Iovance projects $275 million in product revenue for 2025, primarily driven by Amtagvi sales.
  • Recent financial results showed $49.32 million in revenue with a 21.7% gross margin, though operational expenses exceeded $121 million.

PhoreMost Unveils GlueSEEKER Platform to Accelerate Rational Molecular Glue Drug Discovery

Drug Discovery
  • PhoreMost published validation data for its GlueSEEKER platform, a high-throughput technology designed to systematically engineer molecular glue degraders rather than relying on traditional serendipitous discovery methods.
  • The platform integrates high-throughput biological data with computational drug discovery tools to provide blueprints for small molecule development across a wide range of E3 ligases and therapeutic areas including oncology and inflammation.
  • FDA-approved molecular glues have already treated millions of patients and generated billions in revenue, positioning GlueSEEKER to potentially expand this therapeutic modality through rational design approaches.
  • The study includes an end-to-end case study demonstrating the platform's capabilities and validates its potential to accelerate drug development programs while expanding the druggable proteome.

Eyam Health and MMV Partner to Develop $1 Malaria Prevention Shot Using AI-Designed Monoclonal Antibodies

Monoclonal Antibody
  • Eyam Health and Medicines for Malaria Venture announce a strategic partnership to develop next-generation monoclonal antibody-based malaria therapies costing less than $1 per dose.
  • The collaboration leverages Eyam's Jennerator AI platform to design biologics and the Gemini delivery system that can carry multiple antibodies without cold storage requirements.
  • A single long-acting injection could protect over 50 million young children and pregnant women for an entire 4-5 month rainy season during peak malaria transmission.
  • The nucleic acid-based Gemini platform eliminates expensive lipid nanoparticles and can be readily adapted for local African manufacturing to address pandemic preparedness.

FDA Issues Complete Response Letter for Replimune's RP1 Melanoma Therapy, Citing Trial Design Concerns

  • The FDA issued a Complete Response Letter rejecting Replimune's biologics license application for RP1 plus nivolumab in advanced melanoma, stating the IGNYTE trial does not provide substantial evidence of effectiveness.
  • The agency cited concerns about patient population heterogeneity in the IGNYTE study and raised design issues with the confirmatory phase 3 trial IGNYTE-3, though no safety concerns were identified.
  • Replimune expressed surprise at the decision, noting that issues raised in the CRL were not flagged during mid- and late-cycle reviews, and plans to request an urgent Type A meeting with the FDA within 30 days.
  • The rejection comes despite the therapy receiving Priority Review status and Breakthrough Therapy designation earlier this year, highlighting the challenges in cancer drug development for patients with limited treatment options.

A Phase 1/2a Study of VS-7375 in Patients With KRAS G12D-Mutated Solid Tumors

NCT07020221RecruitingPhase 1
Verastem, Inc.
Posted 6/24/2025

A Phase III Study Evaluating Glofitamab in Combination With Gemcitabine + Oxaliplatin vs Rituximab in Combination With Gemcitabine + Oxaliplatin in Participants With Relapsed/Refractory Diffuse Large B-Cell Lymphoma

NCT04408638Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 2/23/2021

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

A Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Efficacy of TT125-802 in Subjects With Advanced Solid Tumors

NCT06403436RecruitingPhase 1
TOLREMO therapeutics AG
Posted 11/7/2023

Beamion LUNG-1: A Study to Test Different Doses of Zongertinib in People With Different Types of Advanced Cancer (Solid Tumours With Changes in the HER2 Gene)

NCT04886804RecruitingPhase 1
Boehringer Ingelheim
Posted 7/2/2021

ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

NCT03295396TerminatedPhase 2
Chimerix
Posted 10/30/2017

A Study of ZW171 in Participants With Advanced or Metastatic Mesothelin-expressing Cancers

NCT06523803Active, Not RecruitingPhase 1
Zymeworks BC Inc.
Posted 9/30/2024

Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline High Grade Gliomas

NCT03134131No Longer Available
Chimerix

A Study of Osimertinib With or Without Chemotherapy as 1st Line Treatment in Patients With Mutated Epidermal Growth Factor Receptor Non-Small Cell Lung Cancer (FLAURA2)

NCT04035486Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/2/2019

Oral ONC201 in Adult Recurrent Glioblastoma

NCT02525692TerminatedPhase 2
Chimerix
Posted 1/1/2016

VO and Nivolumab vs Physician's Choice in Advanced Melanoma That Progressed on Anti-PD-1 & Anti-CTLA-4 Drugs (IGNYTE-3)

NCT06264180RecruitingPhase 3
Replimune Inc.
Posted 7/11/2024

ONC201 in Pediatric H3 K27M Gliomas

NCT03416530TerminatedPhase 1
Chimerix
Posted 1/25/2018

Safety, Efficacy, and Pharmacokinetic Behavior of Leuprolide Mesylate (LMIS 25 mg) in Subjects With Prostate Cancer

NCT03261999CompletedPhase 3
Foresee Pharmaceuticals Co., Ltd.
Posted 9/26/2017

A Study of GFH375 in Patients With Advanced Solid Tumors With KRAS G12D Mutations

NCT06500676RecruitingPhase 1
Genfleet Therapeutics (Shanghai) Inc.
Posted 7/9/2024

Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas

NCT05392374No Longer Available
Chimerix

Sanofi Acquires Vicebio for $1.15 Billion to Expand Respiratory Vaccine Pipeline with Molecular Clamp Technology

  • Sanofi announced plans to acquire London-based biotechnology company Vicebio for $1.15 billion in cash, with potential milestone payments up to $450 million based on development achievements.
  • The acquisition brings Vicebio's proprietary Molecular Clamp technology that stabilizes viral proteins and enables development of fully liquid combination vaccines stored at standard refrigeration temperatures.
  • Vicebio's pipeline includes VXB-241, a bivalent vaccine candidate targeting RSV and hMPV currently in phase 1 trials, and VXB-251, a preclinical trivalent vaccine candidate.
  • The transaction is expected to close in Q4 2025 and will expand Sanofi's respiratory vaccines capabilities beyond its existing flu and RSV prevention portfolio.

A Study of RSV-HMPV Bivalent Vaccine VXB-241 in Older Adults

NCT06556147Active, Not RecruitingPhase 1
Vicebio Australia Proprietary Limited
Posted 8/13/2024

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