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Clinical Trial News

iOnctura Secures €80 Million Series B to Advance PI3Kδ Cancer Therapy Through Phase II Trials

Orphan Drug
  • iOnctura raised €80 million in Series B financing led by Syncona to advance its pipeline of oral cancer treatments targeting neglected and hard-to-treat cancers.
  • Lead asset roginolisib, the first allosteric modulator of PI3Kδ, demonstrated promising efficacy in uveal melanoma with 70% overall survival rate and minimal toxicity in Phase I trials.
  • The funding will accelerate Phase II trials for roginolisib in uveal melanoma and expand development into non-small cell lung cancer and primary myelofibrosis starting late 2024.
  • Second asset cambritaxestat represents the only autotaxin inhibitor in clinical development for highly fibrotic tumors like metastatic pancreatic cancer.

A Study to Assess a PI3Kδ Inhibitor (IOA-244) in Patients with Metastatic Cancers

NCT04328844Active, Not RecruitingPhase 1
iOnctura
Posted 2/25/2020

Dose Escalation Phase 1 Study of IOA-289 in combination with gemcitabine/nab-paclitaxel

2024-515674-27-00Not Yet RecruitingPhase 1
iOnctura SA

Sarepta's Elevidys Receives Full FDA Approval for Expanded Use in Duchenne Muscular Dystrophy

  • The FDA granted full approval for Sarepta's Elevidys gene therapy for Duchenne muscular dystrophy (DMD) patients aged 4 and older with confirmed DMD gene mutations.
  • Accelerated approval was also granted for non-ambulatory patients, contingent on Phase III study results, marking a significant label expansion for Elevidys.
  • This decision follows Pfizer's recent DMD gene therapy failure, highlighting the critical unmet need and limited treatment options for DMD patients.
  • Analysts predict substantial market potential for Elevidys, estimating peak revenues of up to $4 billion, driven by rapid adoption and treatment of a significant portion of the DMD population.

FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy

NCT05135091RecruitingPhase 1
Neurona Therapeutics
Posted 6/16/2022

A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE)

NCT06422923RecruitingPhase 1
Neurona Therapeutics
Posted 11/14/2024

Ruxolitinib Maintains Clinical Benefit in Myelofibrosis Patients Despite Treatment-Related Anemia

Real World Evidence
  • Post-hoc analysis of the phase 3b JUMP trial demonstrates that ruxolitinib maintains clinical benefit in myelofibrosis patients who develop new or worsening anemia during treatment.
  • The study found no significant difference in overall survival between patients with and without new or worsening anemia, with median OS of 58.3 months versus not evaluable in baseline anemic patients.
  • Results from the largest ruxolitinib trial in myelofibrosis support continued use of the drug regardless of baseline anemia status or development of treatment-related anemia.
  • Findings validate previous COMFORT trial results and provide real-world evidence for ruxolitinib's sustained efficacy across diverse patient populations including those with lower platelet counts.

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

NCT00952289CompletedPhase 3
Incyte Corporation
Posted 8/1/2009

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

Pfizer Explores Brentuximab Vedotin for Systemic Sclerosis Treatment in Development Pipeline

  • Brentuximab vedotin (Adcetris), currently approved for various lymphomas, is being investigated as a potential treatment for diffuse cutaneous systemic sclerosis, expanding its therapeutic applications.
  • The drug, an anti-neoplastic agent administered intravenously, has established efficacy in treating multiple CD30-positive lymphomas and has received multiple regulatory approvals for various indications.
  • Pfizer's development program represents a novel approach to treating systemic sclerosis, a rare autoimmune condition with limited treatment options currently available.

AI-Powered Blood Test Shows Promise for Early Parkinson's Detection with 100% Accuracy

  • Researchers from UCL and UMC Goettingen have developed an AI-powered blood test that demonstrated 100% accuracy in identifying Parkinson's disease through eight protein biomarkers.
  • The innovative test successfully predicted Parkinson's disease in 79% of individuals with idiopathic REM sleep behavior disorder up to seven years before symptom onset.
  • This breakthrough could revolutionize Parkinson's diagnosis, offering a less invasive alternative to current methods and potentially enabling earlier therapeutic intervention.

Novel Agents Identified for Osteosarcoma Treatment in Children and Young Adults

  • The Children's Oncology Group has prioritized several novel agents for osteosarcoma treatment, addressing the lack of progress in outcomes for decades.
  • Multitargeted tyrosine kinase inhibitors are identified for frontline evaluation and will be incorporated into an upcoming phase III study.
  • Immunotherapies targeting B7-H3 and CD47-SIRPα inhibitors, telaglenastat, and epigenetic modifiers are also under consideration for clinical trials.
  • The New Agents for Osteosarcoma Task Force will continue to reassess and evaluate novel agents as new data become available.

Kymera's KT-333 Demonstrates Antitumor Activity in Hematological Cancers in Phase 1 Trial

  • Kymera Therapeutics' KT-333, a STAT3 degrader, shows antitumor activity in relapsed/refractory hematological cancers at well-tolerated doses.
  • Complete responses were observed in Hodgkin's lymphoma patients, with some proceeding to stem cell transplants post-treatment.
  • Partial responses were seen in cutaneous T-cell lymphoma patients, and a complete response was achieved in a STAT3-mutated NK-cell lymphoma patient.
  • KT-333 induced an IFN-γ stimulated gene signature, suggesting potential for combination with anti-PD1 drugs in solid tumors.

CLL Patients Prioritize PFS Benefits in Treatment Choices, Survey Reveals

  • A comprehensive survey of 229 CLL patients found that increasing progression-free survival from 70% to 90% at 2 years was their highest treatment priority, especially when confirmed by MRD testing.
  • Patients demonstrated strong preferences for daily oral medications over monthly IV infusions, and fixed-duration treatments over continuous therapy until disease progression.
  • The study revealed patients were willing to accept certain treatment risks, including tumor lysis syndrome, in exchange for improved survival outcomes and more convenient administration methods.

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