Clinical Trial News

A novel gene therapy for hemophilia A, SPK-8011, demonstrated sustained FVIII expression, reducing bleeding episodes significantly. The phase 1/2 trial, led by CHOP, showed stable coagulation factor VIII levels in patients, with a 91.5% reduction in bleeding. Funded by Spark Therapeutics, the study supports liver-directed AAV gene therapy for long-term hemophilia A treatment.

Rafael Pharmaceuticals' CPI-613 (devimistat) received EMA orphan drug designation for treating refractory/relapsed Burkitt’s lymphoma, a rare NHL variant. Devimistat targets tumor cell survival mechanisms, marking its third EMA ODD. It also holds seven FDA ODDs, offering hope for patients with limited treatment options.

WHO-led trials on remdesivir, hydroxychloroquine, lopinavir, and interferon for Covid-19 showed no significant reduction in mortality, ventilation initiation, or hospital stay duration among 11,330 hospitalized patients across 30 countries.

FDA approved Novartis’ Scemblix for treating chronic myeloid leukemia (CML) in adults with specific conditions, including resistance or intolerance to TKIs. Scemblix, a STAMP inhibitor, targets the ABL myristoyl pocket, showing promise in clinical trials with fewer adverse reactions compared to Bosulif. It's now available in the U.S. for qualified patients.

Cambridge Cognition has been awarded a significant contract to provide digital cognitive assessments and study management services for a pivotal schizophrenia trial, marking a continued partnership with an existing client and a step forward in the company's expansion into new therapeutic areas.

Half-dose glucarpidase (mean 25 U/kg) effectively reduced toxic MTX plasma concentrations by ≥97.7% in patients with HDMTX-associated acute kidney injury, making MTX levels manageable with intensified folinic acid rescue. This suggests lower glucarpidase doses may be sufficient, offering significant cost savings.

Orphagen Pharmaceuticals received a $2.0 million SBIR grant from the NCI for preclinical development of OR-449, a first-in-class therapy targeting adrenocortical carcinoma (ACC). OR-449 inhibits SF-1, crucial for ACC pathogenesis. Orphagen's CEO highlighted the team's expertise in discovering OR-449, aiming to advance ACC treatment.

HepaRegeniX begins Phase 1 trial for HRX-0215, a MKK4 inhibitor aimed at treating liver diseases by enhancing liver regeneration. The trial involves 48 healthy males to evaluate safety and pharmacokinetics. Preclinical studies showed HRX-0215's efficacy in liver disease models, marking a significant step towards new liver disease treatments.

Preliminary results from a phase I trial indicate that 225Ac-J591 may be effective in treating metastatic castration-resistant prostate cancer, with a significant percentage of patients experiencing a decline in PSA levels.

Sequana Medical has received FDA approval to expand patient enrolment in its North American pivotal study, POSEIDON, for the alfapump treatment of recurrent or refractory ascites due to liver cirrhosis. The expansion aims to compensate for higher attrition rates, with the goal of implanting the alfapump in 50 patients. The study is on track for primary endpoint read-out in Q4 2022 and FDA regulatory submission by mid-2023.