Clinical Trial News
NIDA Funds Small Businesses to Develop Decentralized Clinical Trial Tools for Substance Use Disorders
- The National Institute on Drug Abuse (NIDA) is offering Small Business Innovation Research (SBIR) grants to develop tools for decentralized clinical trials (DCTs) for substance use disorders (SUD).
- The goal is to increase participation, diversity, and retention in SUD clinical trials by facilitating remote data collection and telehealth visits.
- NIDA intends to commit approximately $3M in FY 2025 to fund 5-10 awards, supporting projects developing wearable devices, software solutions, and systems for low-bandwidth communication.
- The initiative aims to address limitations of traditional in-person clinical trials, such as travel burdens, stigma, and lack of access in rural areas, by leveraging technology for remote monitoring and data management.
FDA Approves Rezdiffra (Resmetirom) as First Treatment for NASH with Liver Fibrosis
- The FDA has granted accelerated approval to Rezdiffra (resmetirom) for adults with noncirrhotic NASH and moderate to advanced liver fibrosis.
- Rezdiffra, a THR-β agonist, is the first FDA-approved medication for NASH, also known as MASH, marking a significant milestone.
- Approval was based on Phase 3 MAESTRO-NASH trial data, which demonstrated improved liver fibrosis and NASH resolution compared to placebo.
- Madrigal plans to launch Rezdiffra in the U.S. in April, with a patient support program to aid access and affordability.
Dimerix's DMX-200 Shows Promise in Phase 3 Trial for Rare Kidney Disease FSGS
- Dimerix Limited's DMX-200 demonstrates successful interim results in a Phase 3 trial for Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease.
- The trial included 72 patients and showed potential in treating FSGS, which currently has no approved drugs globally, marking a significant advancement.
- Dimerix plans to expand the trial to include more countries, such as China, and broaden the patient base to include children over 12 years old.
- The treatment, co-invented by researchers at the Harry Perkins Institute, could prevent kidney damage and the need for dialysis or transplantation in FSGS patients.
Korean Drugmakers Lead in Biosimilar Market and New Denosumab, Eculizumab Deals
Korean drugmakers dominate the biosimilar market with over 50% share in top-selling biosimilars, alongside new partnerships for denosumab and eculizumab biosimilars. Celltrion's denosumab biosimilar shows promise for affordable osteoporosis treatment, and Sandoz's biosimilar denosumab demonstrates equivalence to the originator.
I-Mab Advances Immunotherapy Pipeline with Uliledlimab, Givastomig, and Ragistomig
- I-Mab is preparing to file an IND for uliledlimab in combination with chemotherapy and checkpoint inhibitors for newly diagnosed NSCLC patients in 1H 2024.
- The first patient has been dosed in a triplet combination, dose escalation study of givastomig for gastric cancer and esophageal adenocarcinoma in 1Q 2024.
- Strategic divestiture of assets and business operations in China is expected to close by the end of March 2024, impacting the company's financial condition.
- I-Mab reported RMB2.3 billion (US$321.8 million) in cash, cash equivalents, and short-term investments as of December 31, 2023.
Protein Discovery Offers New Hope for Treating Aggressive Cancer
A groundbreaking study led by WEHI and the Peter MacCallum Cancer Centre has identified two proteins critical for the development of B-cell acute lymphoblastic leukaemia, offering new treatment possibilities. By targeting these proteins, researchers were able to kill leukaemia cells and stop their growth in lab models, paving the way for future clinical trials.
CAP-1002 Shows Promise in Preserving Limb and Heart Function in DMD Patients
- CAP-1002, an investigational cell therapy, continues to demonstrate slowed declines in upper limb and heart function in boys and young men with Duchenne muscular dystrophy (DMD).
- Two-year results from the HOPE-2 open label extension (OLE) study indicate a 64% slowing of disease progression with CAP-1002 treatment.
- Data showed stabilization or improvement of cardiac function in 67% of patients, suggesting a potential disease-modifying effect of CAP-1002.
- A pivotal Phase 3 trial (HOPE-3) is underway, with data readout expected this year to support a potential FDA regulatory application.
Highlighted Clinical Trials:
Capricor Inc.
Posted 4/4/2018
Capricor Inc.
Posted 6/22/2022
Gilgamesh Pharmaceuticals Receives $14 Million NIDA Grant for Cardiac-Safe Ibogaine Analog to Treat Opioid Use Disorder
- Gilgamesh Pharmaceuticals secured a $14 million grant from the National Institute on Drug Abuse (NIDA) to advance GM-3009, a novel ibogaine analog, for opioid use disorder (OUD).
- GM-3009 aims to provide a safer alternative to ibogaine by mitigating cardiovascular risks while maintaining or enhancing its therapeutic efficacy for OUD.
- The funding will support IND-enabling toxicology studies, GMP manufacturing, and Phase 1/1b clinical trials to assess the safety and efficacy of GM-3009.
- GM-3009 represents a potential breakthrough in OUD treatment, offering a rapid-acting, effective, and durable solution to combat the opioid epidemic.
LYT-200 Receives FDA Orphan Drug Designation for Acute Myeloid Leukemia Treatment
The FDA has granted orphan drug designation to LYT-200, a fully human IgG4 monoclonal antibody targeting galectin-9, for the treatment of acute myeloid leukemia (AML). LYT-200 is currently undergoing phase 1 trials for AML, solid tumors, and myelodysplastic syndrome (MDS), showing promising safety and tolerability profiles.
Highlighted Clinical Trials:
PureTech
Posted 12/15/2020
Biotech Stock Roundup: Moderna's Earnings, Iovance's Approval, and RAPT's Clinical Hold
- Moderna's Q4 earnings surpassed expectations, driven by strong Spikevax sales, and the company anticipates initial regulatory approvals for its RSV vaccine in the first half of 2024.
- Iovance Biotherapeutics received accelerated FDA approval for Amtagvi (lifileucel) for advanced melanoma patients previously treated with a PD-1 blocking antibody.
- RAPT Therapeutics' stock plummeted after the FDA placed a clinical hold on its zelnecirnon trials for atopic dermatitis and asthma due to a liver failure event.
- Sarepta Therapeutics' application to expand the label for its Duchenne muscular dystrophy drug, Elevidys, has been accepted for Priority Review by the FDA.