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Diamyd Medical Warrants Expire as Phase 3 Trial Readout Nears

  • Diamyd Medical's warrants of series TO 3 expired on September 25, 2024, concluding the exercise period that began on September 2, 2024.
  • Several board members and senior executives, including CEO Ulf Hannelius, intended to exercise their warrants, totaling approximately SEK 5.1 million.
  • The company anticipates an earlier readout from the DIAGNODE-3 Phase 3 trial around March 2026, focusing on Type 1 Diabetes precision medicine.
  • Diamyd Medical received SEK 17.5 million from collaborations and the sale of Companion Medical shares, supporting accelerated approval efforts.

Shield Therapeutics' Accrufer Demonstrates Efficacy in Pediatric Phase III Trial for Iron Deficiency

  • Shield Therapeutics announced positive results from its Phase III clinical trial evaluating Accrufer in pediatric patients with iron deficiency.
  • The study met its primary endpoint, demonstrating a statistically significant increase in hemoglobin levels compared to baseline.
  • Accrufer was well-tolerated in the pediatric population, with a safety profile consistent with previous studies.
  • These findings support the potential of Accrufer as a novel treatment option for iron deficiency in children.

Epigenetic Drug Development Sees Record $342M Series A Funding Surge in Early 2024

  • Series A venture financing for epigenetic drug development has reached $342M in early 2024, marking a dramatic 375% increase from 2023 and setting a five-year record.
  • Avenzo Therapeutics secured the largest Series A round of $150M to advance CDK2 inhibitor ARTS-021, currently in Phase I/II trials for solid tumors and metastatic breast cancer.
  • The surge in investment follows FDA's approval of Vertex's Casgevy, highlighting growing confidence in epigenetic approaches as safer alternatives to traditional gene editing.

Capricor Therapeutics Advances Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy

  • Capricor Therapeutics plans to file a Biologics License Application (BLA) with the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.
  • The BLA will be supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 OLE trials, compared with natural history data.
  • Capricor has initiated its rolling submission process with the FDA for deramiocel, with completion expected by the end of 2024.
  • Deramiocel has shown immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure in clinical studies.

Open-label Extension of the HOPE-2 Trial

NCT04428476Active, Not RecruitingPhase 2
Capricor Inc.
Posted 8/5/2020

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT03406780CompletedPhase 2
Capricor Inc.
Posted 4/4/2018

A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy

NCT05126758Active, Not RecruitingPhase 3
Capricor Inc.
Posted 6/22/2022

Panbela Therapeutics Initiates Phase I Trial of CPP-1X-S in STK11 Mutant NSCLC

  • Panbela Therapeutics has dosed the first patient in a Phase I trial evaluating CPP-1X-S (eflornithine sachets) in STK11 mutant non-small cell lung cancer (NSCLC).
  • The Phase I dose escalation study aims to determine the maximum tolerated dose of eflornithine in combination with Keytruda, an immune checkpoint inhibitor.
  • Initial Phase I data is expected by mid-2025, with a Phase II efficacy trial planned to commence in 2024 to assess the combination's clinical potential.
  • The trial will explore polyamine modulation via CPP-1X-S to potentially restimulate the immune system in these historically hard-to-treat tumors.

Targeting ODC as an Immunotherapeutic Target in STK11 (LKB1) Pathway-Deficient NSCLC (DFMO)

NCT06219174RecruitingPhase 1
H. Lee Moffitt Cancer Center and Research Institute
Posted 8/7/2024

pGTI Applied in STAR-JIA Trial to Assess Steroid Toxicity in Children with Juvenile Idiopathic Arthritis

  • The STAR-JIA trial will use Steritas' Pediatric Glucocorticoid Toxicity Index (pGTI) to compare intravenous versus oral glucocorticoids in children with Juvenile Idiopathic Arthritis (JIA).
  • The randomized controlled trial aims to determine the most effective route of steroid administration for treating inflammation in JIA while assessing toxicity.
  • The pGTI offers a systematic approach to measuring and monitoring steroid toxicity in children aged 2-18, providing weighted scores for researchers and clinicians.
  • This initiative marks the first use of the pGTI in clinical practice, potentially enabling early detection of steroid toxicity and improving patient outcomes.

Convergent Therapeutics Advances Prostate Cancer Therapy with Phase II Trial and $40M Funding

• Convergent Therapeutics initiates Phase II CONVERGE-01 trial of CONV01-α for metastatic castration-resistant prostate cancer (mCRPC). • The trial will assess the safety and efficacy of CONV01-α, an Ac-225 rosopatamab tetraxetan, in PSMA PET-positive mCRPC patients. • Convergent secures a $40 million Series A extension from Novo Holdings to support its radiopharmaceutical pipeline development. • Richard Messmann, MD, a veteran in oncology drug development, joins Convergent as Chief Medical Officer.

Study Evaluating Dosimetry, Randomized Dose Optimization, Dose Escalation and Efficacy of Ac-225 Rosopatamab Tetraxetan in Participants With PSMA PET-Positive Castration-Resistant Prostate Cancer (CRPC)

NCT06549465RecruitingPhase 2
Convergent Therapeutics
Posted 8/6/2024

Barinthus Bio Initiates Phase 1 Trial of VTP-1000 for Celiac Disease

  • Barinthus Bio has commenced a Phase 1 clinical trial for VTP-1000, an investigational immunotherapy for celiac disease, a condition affecting approximately one in 100 people globally.
  • The AVALON trial (NCT06310291) is a randomized, placebo-controlled study designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of VTP-1000 in adults with celiac disease.
  • VTP-1000 utilizes the SNAP-TI platform to deliver gluten-derived peptide antigens and rapamycin, aiming to induce immune tolerance to gluten and reduce inflammation in the small intestine.
  • The trial includes a controlled gluten challenge to evaluate VTP-1000's impact on patients' exposure to gluten, addressing a significant unmet need as there are currently no approved treatments for celiac disease.

VTP-1000 in Adults with Celiac Disease

NCT06310291RecruitingEarly Phase 1
Barinthus Biotherapeutics
Posted 8/1/2024

Sanofi Presents New Data on Dupixent and Immunology Pipeline at EADV 2024

  • Sanofi presented 21 abstracts for Dupixent, highlighting its impact on atopic dermatitis, prurigo nodularis and chronic spontaneous urticaria, showcasing disease remission and long-term efficacy data.
  • Data from an open-label extension study showed Dupixent's potential for clinical remission in children aged 6-17 with moderate-to-severe atopic dermatitis, even after stopping treatment.
  • Sanofi shared data from its immunology pipeline, including amlitelimab, an OX40-ligand monoclonal antibody, demonstrating safety and efficacy in moderate-to-severe atopic dermatitis with potential for quarterly dosing.
  • Rilzabrutinib, a novel oral BTK inhibitor, showed positive impacts on symptoms of atopic dermatitis and moderate-to-severe chronic spontaneous urticaria in poster presentations.

Nemolizumab Shows Long-Term Efficacy in Atopic Dermatitis and Prurigo Nodularis

  • Interim analysis of the ARCADIA long-term extension study shows continuous nemolizumab treatment leads to increasing improvements in atopic dermatitis symptoms over 56 weeks.
  • Biomarker data from ARCADIA trials demonstrates nemolizumab's impact on reducing itch, skin lesions, and inflammation associated with atopic dermatitis.
  • OLYMPIA DURABILITY study indicates continued nemolizumab use beyond 52 weeks significantly reduces relapse rates in prurigo nodularis patients compared to withdrawal.

A Study to Evaluate the Durability of Response and Safety of Nemolizumab for 24 Weeks in Participants With Prurigo Nodularis

NCT05052983CompletedPhase 3
Galderma R&D
Posted 1/24/2022

Long-term Safety and Efficacy of Nemolizumab With Moderate-to-severe Atopic Dermatitis

NCT03989206Active, Not RecruitingPhase 3
Galderma R&D
Posted 12/30/2019

A Long-term Study of Nemolizumab (CD14152) in Participants With Prurigo Nodularis (PN)

NCT04204616Active, Not RecruitingPhase 3
Galderma R&D
Posted 1/11/2021

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