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Clinical Trial News

Akadeum Life Sciences Secures $20M+ to Advance Cell Separation Technology for Gene and Cell Therapy Manufacturing

  • Akadeum Life Sciences closed a $20 million+ financing round led by Michigan Capital Network to scale commercial operations and support customers entering clinical trials.
  • The company recently launched a GMP-compliant product suite designed for clinical trial use and demonstrated integration capabilities with existing cell therapy manufacturing tools.
  • Akadeum's buoyancy-based cell separation platform delivers higher cell yields, improved viability, and easier workflows compared to conventional methods according to industry partners.
  • The technology has shown therapeutic relevance in preclinical cancer treatment models and is gaining adoption among leading biopharma companies including Catalent, Charles River Laboratories, ElevateBio, and Lonza.

Transpire Bio Licenses PDE4 Inhibitor ITG-1052 for Idiopathic Pulmonary Fibrosis Treatment

  • Transpire Bio has secured exclusive licensing rights to ITG-1052 (lenamilast), an investigational PDE4 inhibitor from Suzhou Intragrand Pharma for development as an inhaled therapeutic.
  • The agreement grants Transpire Bio rights to develop, manufacture, and commercialize ITG-1052 in all territories except China, targeting idiopathic pulmonary fibrosis and other respiratory indications.
  • ITG-1052 represents a new chemical entity addition to Transpire Bio's pipeline of inhaled therapeutics, leveraging the company's expertise in inhalation drug delivery platforms.
  • The licensing deal aims to address unmet medical needs in idiopathic pulmonary fibrosis, a progressive respiratory disease characterized by irreversible lung function decline.

Phase 3 Trial Shows Promise for Adjuvant BRAF/MEK Inhibitor Combination in High-Risk Melanoma

Targeted TherapyOncology
  • The phase 3 EORTC-2139-MG/Columbus-AD trial demonstrated that adjuvant encorafenib plus binimetinib improved 12-month recurrence-free survival to 86% versus 70% with placebo in patients with stage IIB/C BRAF V600-mutant melanoma.
  • The combination therapy was generally well tolerated with a manageable safety profile, though 33% of patients discontinued treatment due to adverse events.
  • This represents the first randomized adjuvant trial of BRAF-directed therapy in stage IIB/IIC melanoma, potentially offering patients an alternative to immunotherapy in the adjuvant setting.

Adjuvant Encorafenib and Binimetinib in High-risk Stage II Melanoma With a BRAF Mutation.

NCT05270044Active, Not RecruitingPhase 3
Pierre Fabre Medicament
Posted 5/2/2022

Study Comparing Combination of LGX818 Plus MEK162 Versus Vemurafenib and LGX818 Monotherapy in BRAF Mutant Melanoma

NCT01909453CompletedPhase 3
Pfizer
Posted 9/12/2013

CAR-T Immunotherapy Shows Unprecedented Five-Year Survival in Advanced Multiple Myeloma Patients

CAR-T
  • A third of 97 patients with advanced multiple myeloma achieved complete remission lasting five years or more after receiving a single infusion of CAR-T immunotherapy developed by Legend Biotech.
  • The patients had exhausted all treatment options and faced hospice care before receiving the experimental therapy in what researchers called a last-ditch effort.
  • Oncologists are using the term "potential cure" for the first time in multiple myeloma treatment, marking an unprecedented breakthrough in a disease previously considered incurable.
  • The study results were presented at the American Society of Clinical Oncology annual conference and published in The Journal of Clinical Oncology.

Computer Modeling Identifies Two Broad-Spectrum Antiviral Compounds Against RNA Viruses

  • An interdisciplinary research team involving the German Center for Infection Research has identified two antiviral drug candidates, phenformin and atpenin A5, effective against a wide range of RNA viruses through computer-aided modeling combined with laboratory validation.
  • Phenformin, a previously used diabetes medication, significantly reduced SARS-CoV-2 viral load in infected hamsters and inhibited dengue virus multiplication in cell cultures, offering potential for rapid clinical translation due to its established human safety profile.
  • The study demonstrates a novel computational approach that identifies metabolic processes essential for viral replication but not cellular survival, accelerating antiviral drug development for future pandemic preparedness.

Daewoong Pharmaceutical Advances Phase 2 Trial of Novel IPF Drug Bersiporocin with Diverse Patient Population

Orphan Drug
  • Daewoong Pharmaceutical presented interim Phase 2 results for Bersiporocin (DWN12088), a first-in-class oral antifibrotic drug targeting idiopathic pulmonary fibrosis, at the 2025 American Thoracic Society International Conference.
  • The ongoing randomized, double-blind, placebo-controlled study is enrolling 102 patients across 30 sites in the U.S. and South Korea, with over 50% of participants being Asian, enabling assessment across ethnic subgroups.
  • Bersiporocin selectively inhibits Prolyl-tRNA Synthetase (PRS) to interrupt collagen biosynthesis and fibrotic cascade progression, representing a novel mechanism of action in IPF treatment.
  • The drug has received Orphan Drug Designation from both FDA and EMA, plus Fast Track designation from FDA, with 80% of target enrollment completed as of April 2025.

Moderna Agrees to Placebo-Controlled Trial for New COVID-19 Vaccine Following FDA Approval

Drug Approval
  • Moderna has agreed to conduct a placebo-controlled trial of its newly approved second-generation COVID-19 vaccine mNexspike, as announced by HHS Secretary Robert F. Kennedy Jr.
  • The FDA required this future placebo-controlled study as a condition for approving the new vaccine, specifically targeting adults ages 50 to 64 without high-risk conditions.
  • The new vaccine received limited approval for adults 65 and older and anyone 12 and over with at least one risk factor for severe disease, marking a narrower approval than previous COVID-19 vaccines.
  • This requirement represents an unusual regulatory approach that has raised ethical concerns about exposing participants to preventable illness by administering placebo instead of existing vaccines.

Circulating Tumor DNA Emerges as Powerful Prognostic Biomarker in Locally Advanced Cervical Cancer

  • An exploratory analysis from the phase 3 CALLA trial demonstrated that undetectable circulating tumor DNA (ctDNA) at cycle 6 was associated with a 95% reduction in risk of disease progression or death in patients with locally advanced cervical cancer.
  • Patients with low baseline ctDNA levels showed improved survival outcomes compared to those with high ctDNA levels, regardless of whether they received durvalumab plus chemoradiotherapy or chemoradiotherapy alone.
  • The ultrasensitive tumor-informed assay showed high sensitivity for ctDNA detection, with positivity rates decreasing from 99% at baseline to 23-36% by cycle 6.
  • ctDNA detection at cycle 3 served as a negative prognostic factor, with 68% of ctDNA-positive patients experiencing disease progression and a median lead time of 164 days between detection and progression.

Study of Durvalumab With Chemoradiotherapy for Women With Locally Advanced Cervical Cancer (CALLA)

NCT03830866CompletedPhase 3
AstraZeneca
Posted 2/15/2019

ASCO 2025 Showcases Advances in Myelofibrosis Treatment with Novel JAK Inhibitors and Personalized Approaches

  • The 2025 ASCO Annual Meeting highlighted significant progress in myelofibrosis treatment, featuring novel JAK inhibitors like momelotinib and pacritinib that offer alternatives to standard ruxolitinib therapy.
  • Momelotinib demonstrated superior anemia management in the MOMENTUM trial with 25% symptom response versus 9% with danazol, particularly benefiting patients with ruxolitinib-induced anemia.
  • Pacritinib showed consistent efficacy regardless of baseline cytopenias, making it valuable for patients with low platelet counts below 50,000/μL who have limited treatment options.
  • Pegylated interferons continue to play an important role with 70-80% response rates and superior molecular responses, especially beneficial for younger patients with potential disease-modifying activity.

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions

NCT04717414Active, Not RecruitingNot Applicable
Celgene
Posted 2/25/2021

Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia

NCT02055781TerminatedPhase 3
CTI BioPharma
Posted 2/1/2014

Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

NCT01773187TerminatedPhase 3
CTI BioPharma
Posted 1/1/2013

Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis

NCT01969838CompletedPhase 3
Sierra Oncology LLC - a GSK company
Posted 12/6/2013

COntrolled MyeloFibrosis Study With ORal JAK Inhibitor Treatment: The COMFORT-I Trial

NCT00952289CompletedPhase 3
Incyte Corporation
Posted 8/1/2009

Pegylated Interferon Alpha-2b Versus Hydroxyurea in Polycythemia Vera

NCT01949805CompletedPhase 3
AOP Orphan Pharmaceuticals AG
Posted 9/1/2013

A Study of Momelotinib Versus Danazol in Symptomatic and Anemic Myelofibrosis Participants (MOMENTUM)

NCT04173494CompletedPhase 3
Sierra Oncology LLC - a GSK company
Posted 2/7/2020

INC424 for Patients With Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis.

NCT01493414CompletedPhase 3
Novartis Pharmaceuticals
Posted 8/16/2011

Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II Trial

NCT00934544CompletedPhase 3
Novartis Pharmaceuticals
Posted 7/1/2009

Efficacy of Momelotinib Versus Best Available Therapy in Anemic or Thrombocytopenic Subjects With Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF

NCT02101268CompletedPhase 3
Sierra Oncology LLC - a GSK company
Posted 6/19/2014

Essential Thrombocythemia Market Set for Significant Growth as Novel Therapies Enter Pipeline

Orphan Drug
  • Essential thrombocythemia affects approximately 167,000 individuals in the US as of 2024, with 75% of cases linked to JAK2 gene mutations and only one approved therapy currently available in Europe.
  • The market is projected to grow from $417 million in 2024 at a significant compound annual growth rate through 2034, driven by emerging therapies with novel mechanisms of action.
  • Several promising pipeline candidates are advancing through clinical trials, including Bomedemstat from Merck, ropeginterferon alfa-2b from PharmaEssentia, and Pelabresib targeting BET proteins.
  • Current treatment approaches rely on risk stratification, with low-risk patients receiving aspirin and high-risk patients managed with cytoreductive therapies like hydroxyurea.

A Phase 2 Study of CPI-0610 with and Without Ruxolitinib in Patients with Myelofibrosis

NCT02158858CompletedPhase 1
Constellation Pharmaceuticals
Posted 7/16/2014

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