Clinical Trial News
Orphagen Pharmaceuticals Receives $2 Million NCI Grant for Novel ACC Therapy
• Orphagen Pharmaceuticals secured a $2 million SBIR Phase II grant from the National Cancer Institute to advance OR-449, a first-in-class drug for adrenocortical carcinoma (ACC).
• OR-449 is a selective inhibitor of steroidogenic factor-1 (SF-1), a key transcription factor crucial for adrenal gland development and implicated in ACC pathogenesis.
• The funding supports preclinical development of OR-449, representing a potential new treatment option for ACC, a rare cancer with limited therapeutic solutions and poor prognosis.
COVID-19 Pandemic Catalyzes Major Transformation in Global Regulatory Processes
• The COVID-19 pandemic has dramatically accelerated regulatory processes, enabling vaccine development and deployment within 12 months through unprecedented collaboration between regulatory bodies and pharmaceutical companies.
• Real-world data (RWD) and digital technologies are reshaping clinical research, with new platforms emerging for real-time data sharing and streamlined approval processes across global regulatory authorities.
• Regulatory reliance models and integrated pathways for medical devices and diagnostics are showing promise in reducing approval delays and advancing global health equity.
HepaRegeniX Initiates Phase 1 Trial of MKK4 Inhibitor HRX-0215 for Liver Diseases
• HepaRegeniX has commenced a Phase 1 clinical trial for HRX-0215, a first-in-class MKK4 inhibitor, to treat acute and chronic liver diseases.
• The Phase 1 trial is a single-center, double-blind, randomized, placebo-controlled study involving 48 healthy male volunteers.
• The trial aims to evaluate the safety, tolerability, and pharmacokinetics of HRX-0215 in single and multiple ascending dose regimens.
• HRX-0215 has demonstrated compelling efficacy in preclinical models of both acute and chronic liver disease, warranting its clinical investigation.
Preliminary Data on 225Ac-J591 Shows Promise in Treating mCRPC
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.
Highlighted Clinical Trials:
Weill Medical College of Cornell University
Posted 10/10/2017
Landmark TOSCA Registry Reveals New Insights into Tuberous Sclerosis Complex Management
• The multinational TOSCA registry, encompassing 2,221 TSC patients from 31 countries, has provided crucial data on disease manifestations and treatment patterns in Tuberous Sclerosis Complex.
• Subependymal giant-cell astrocytomas (SEGAs) were found in 25% of patients, with mTOR inhibitors emerging as a primary treatment option alongside surgery for tumor management.
• The study revealed significant associations between TSC2 mutations and more severe disease manifestations, while also highlighting the substantial impact on patients' quality of life and the need for improved transition from pediatric to adult care.
FDA Approves Expansion of Patient Enrolment in Sequana Medical's POSEIDON Study for Alfapump Treatment
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.
Highlighted Clinical Trials:
Sequana Medical N.V.
Posted 9/16/2019
Acalabrutinib, Venetoclax, and Obinutuzumab Show Promise in Frontline CLL Treatment
• Acalabrutinib, venetoclax, and obinutuzumab demonstrate a high level of activity and a favorable tolerability profile in previously untreated chronic lymphocytic leukemia (CLL).
• The triplet therapy allows for a shorter, more convenient 4-week venetoclax dose ramp-up, compared to the traditional 5-week ramp-up, through initial tumor debulking.
• The combination represents a promising chemotherapy-free, time-limited option for CLL patients, including those with high-risk disease, warranting further investigation.
• A phase 3 registrational trial (ACE-CL-311) is underway to assess the potential of acalabrutinib, venetoclax, and obinutuzumab as a new standard of care.
Bedinvetmab Shows Strong Safety Profile in Canine Laboratory Studies
• Bedinvetmab, a canine monoclonal antibody targeting nerve growth factor (NGF), demonstrated a favorable safety profile in laboratory Beagle dogs over a six-month period.
• The studies found no treatment-related adverse changes in clinical evaluations, neurological or ophthalmic examinations, joint health, or immune function at 1 mg/kg SC monthly.
• Concurrent administration of bedinvetmab with carprofen, a nonsteroidal anti-inflammatory drug (NSAID), showed no negative impact on safety parameters.
• The research supports bedinvetmab's potential as a safe analgesic therapeutic by inhibiting NGF interaction with its receptors without significant adverse effects.
4th RNA-Targeted Drug Discovery Summit to Showcase Latest Advances in Small Molecule Therapeutics
• Leading pharmaceutical companies including Novartis, AstraZeneca, and Roche will present breakthrough developments in RNA-targeted small molecule drug discovery at the December summit.
• The summit will address critical challenges in RNA drug development, focusing on improving specificity, selectivity, and drug-like properties of small molecule therapeutics.
• Expert speakers from academia and industry will share insights on RNA structural analysis, ligand interactions, and novel chemical approaches to accelerate clinical development.
Myocardial Infarction: Promising Therapies Advance Through Clinical Trials
• Several companies are actively developing innovative therapies for myocardial infarction, with Idorsia Pharmaceuticals leading the way with its Phase III drug, selatogrel.
• Faraday Pharmaceuticals' FDY-5301, an elemental reducing agent, is under Phase 3 evaluation with an SPA agreement from the FDA for treating acute ST-segment elevation myocardial infarction (STEMI).
• Novel approaches such as stem cell therapies and anti-inflammatory agents like Dapansutrile are being explored to improve outcomes and reduce cardiac injury post-myocardial infarction.