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Clinical Trial News

Hemogenyx's FLT3 Assay Ready for HEMO-CAR-T Phase I Trials in AML

  • Hemogenyx Pharmaceuticals has developed a clinical-grade assay to assess FLT3 protein expression in acute myeloid leukemia (AML) cells.
  • The FLT3 assay will be used to identify and recruit patients for the Phase I clinical trials of Hemogenyx's HEMO-CAR-T product candidate.
  • Phase I trials are set to commence at MD Anderson Cancer Center, with plans to expand to the University of Pennsylvania Medical Center.

FDA-Approved Oral JAK Inhibitors Show Promise in Treating Moderate-to-Severe Atopic Dermatitis

  • Oral JAK inhibitors abrocitinib and upadacitinib have received FDA approval for treating moderate-to-severe atopic dermatitis, offering a new therapeutic approach distinct from conventional treatments.
  • These medications demonstrate rapid itch relief and significant symptom improvement, making them an attractive option for patients who have not responded adequately to traditional therapies.
  • Clinical data supports the long-term safety and efficacy of JAK inhibitors in atopic dermatitis management, with flexible dosing options allowing for personalized treatment approaches.

PharmaEssentia Launches BESREMi® in Singapore for Polycythemia Vera Treatment

  • PharmaEssentia's BESREMi® (ropeginterferon alfa-2b) is now available in Singapore, marking the first Southeast Asian market to offer this treatment for Polycythemia Vera (PV).
  • BESREMi® is the first therapy approved by Singapore's Health Sciences Authority (HSA) for adults with PV, regardless of prior treatment history, offering a new option for patients.
  • Polycythemia Vera, a rare blood cancer affecting around 500 individuals in Singapore, is characterized by uncontrolled blood cell growth, and early detection is crucial to prevent complications.
  • PharmaEssentia emphasizes raising awareness for MPN Awareness Day on September 12, encouraging early detection and improved quality of life for individuals affected by PV.

Monalizumab Shows Promise in NeoCOAST-2 Trial for Early-Stage NSCLC

  • Interim results from the NeoCOAST-2 Phase 2 trial show monalizumab, combined with durvalumab and chemotherapy, demonstrates encouraging clinical outcomes in early-stage NSCLC.
  • The monalizumab arm achieved a pathological complete response rate of 26.7% and a major pathological response rate of 53.3% in resectable NSCLC patients.
  • Monalizumab, targeting the NKG2A receptor, aims to enhance anti-tumor responses mediated by NK and T cells, potentially improving outcomes in NSCLC treatment.
  • The manageable safety profile and maintained surgical rate in the monalizumab arm support its further investigation in combination strategies for NSCLC.

Neoadjuvant and Adjuvant Treatment in Resectable Non-small Cell Lung Cancer

NCT05061550RecruitingPhase 2
AstraZeneca
Posted 4/14/2022

PolTREG's PTG-007 Shows Long-Term Remission in Type 1 Diabetes Patients

  • PolTREG's PTG-007 cell therapy induced clinical remission in some patients with type 1 diabetes for up to 12 years, showcasing its long-term potential.
  • A subset of patients treated with PTG-007 remained insulin-independent for a period of 18 to 24 months, suggesting a significant therapeutic effect.
  • The company is preparing to initiate a pivotal Phase II/III trial for PTG-007 in type 1 diabetes and is actively seeking partnership funding to support this endeavor.
  • Data from the study, which monitored 54 patients over 7-12 years, were presented at the INNODIA EASD conference, highlighting the therapy's safety and efficacy.

Alzheimer's Disease: Late-Stage Pipeline Explores Novel Therapeutic Targets Beyond Amyloid and Tau

  • The Alzheimer's disease therapeutic pipeline is expanding beyond amyloid and tau targets, with 32 therapeutics in 48 Phase III trials exploring neuroprotection, neurotransmitters, and inflammation.
  • Recent approvals of anti-amyloid antibodies like Leqembi and Kisunla have shown modest clinical benefits but also carry risks, prompting the development of alternative approaches.
  • Several Phase III candidates, such as Athira's fosgonimeton, Anavex's ANAVEX 2-73, and Novo Nordisk's semaglutide, target different mechanisms like neurotrophic factors, neuronal homeostasis, and neuroinflammation.
  • Experts suggest that combination therapies and brain-healthy lifestyles may be essential for effectively managing the heterogeneous nature of Alzheimer's disease.

A Research Study Investigating Semaglutide in People With Early Alzheimer's Disease (EVOKE)

NCT04777396Active, Not RecruitingPhase 3
Novo Nordisk A/S
Posted 5/18/2021

MDA Launches Kickstart Program for Ultra-Rare Neuromuscular Disease Gene Therapy Development

  • The Muscular Dystrophy Association (MDA) has initiated the MDA Kickstart program to advance gene therapy development for ultra-rare neuromuscular diseases.
  • The first project focuses on gene therapy for Congenital Myasthenic Syndrome (CMS) caused by mutations in the CHAT gene, affecting around 200 individuals in the US.
  • MDA partners with UC Davis and Forge Biologics to accelerate preclinical development and manufacturing of AAV-based gene therapies for ultra-rare NMDs.
  • The program seeks FDA Orphan Drug and Rare Pediatric Disease designations, emphasizing the need for Rare Pediatric Disease Priority Review Voucher reauthorization.

Healios KK's MultiStem Therapy Advances to Global Phase 3 Trial for ARDS

  • Healios KK has secured FDA agreement to proceed with a pivotal global Phase 3 trial, REVIVE-ARDS, evaluating MultiStem therapy for Acute Respiratory Distress Syndrome (ARDS).
  • The REVIVE-ARDS trial will enroll up to 550 patients and use Ventilator Free Days as the primary endpoint, with interim analyses planned at 300 and 400 patient milestones.
  • Healios intends to pursue conditional and time-limited approval in Japan, leveraging prior Phase 2 results and the forthcoming confirmatory Phase 3 study.
  • MultiStem's efficacy and safety in treating ARDS will be rigorously assessed in this global trial, potentially addressing a critical unmet need in respiratory medicine.

Boryung Pharmaceutical's BR-6002 Advances in Cardiovascular Disease Treatment

  • Boryung Pharmaceutical is developing BR-6002, an orally administered drug, for cardiovascular diseases, as well as gastric and duodenal ulcers.
  • BR-6002's development leverages GlobalData's predictive analytics for assessing the likelihood of approval based on historical drug development data.
  • Boryung Pharmaceutical focuses on chronic diseases, including hypertension, dyslipidemia, diabetes, CNS disorders, and cancers, with BR-6002 potentially expanding their cardiovascular portfolio.

Pidnarulex Shows Promise in Advanced Solid Tumors with BRCA/PALB2 Mutations

  • Pidnarulex (CX-5461) demonstrates acceptable tolerability and preliminary efficacy in end-stage oncology patients with BRCA1/2 or PALB2 mutations, even after PARP inhibitor failure.
  • In a Phase Ib study, 40% of evaluable patients achieved clinical benefit with stable disease as the best response, including ovarian cancer patients with BRCA1/HRD mutations.
  • The trial aims to determine the Phase II dose and evaluate the safety and antitumor activity of Pidnarulex in patients with BRCA2/PALB2 deficiencies across various tumor types.
  • Senhwa Biosciences' Pidnarulex, a novel DDR drug, shows potential as a rescue medication for PARP inhibitor-resistant patients and may enhance immunotherapy efficacy.

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