Clinical Trial News

Sanofi and SK bioscience expand collaboration to develop next-gen pneumococcal conjugate vaccines (PCVs), initiating phase 3 for PCV21, the first 21-valent PCV candidate in infants & toddlers. The partnership aims to address unmet needs in invasive pneumococcal disease, leveraging both companies' expertise and capabilities.

FDA approves Alhemo®, a subcutaneous injection for hemophilia A or B with inhibitors, reducing bleeds by 86% in prophylaxis users.

HKBU's aptamer for treating X-linked hypophosphatemia (XLH) received Orphan Drug and Rare Pediatric Disease Designations from the U.S. FDA. The aptamer, Apc001, targets sclerostin's loop3 domain to promote bone formation without cardiovascular risks, and is set for clinical trials in China and the U.S.

In 2024, the FDA approved over 130 drugs, including 40 novel ones. Madrigal's Rezdiffra is the first treatment for MASH, X4's Xolremdi for WHIM syndrome, Day One's Ojemda for pediatric low-grade glioma, Geron's Rytelo for MDS, and ImmunityBio's Anktiva for NMIBC. These approvals mark significant milestones for these companies and address high unmet medical needs.

2024 saw highs and lows in neuroscience drug development, including FDA approval of BMS's Cobenfy for schizophrenia and Eli Lilly's donanemab for Alzheimer's, alongside failures like Sage Therapeutics' dalzanemdor in Parkinson's, Alzheimer's, and Huntington's. Despite setbacks, progress continues in treating intractable neurological diseases.

The RH5.1/Matrix-M malaria vaccine, tested in a phase 2b trial on 361 children, shows safety, effectiveness, and high immunogenicity, potentially becoming the first blood-stage vaccine, complementing existing pre-erythrocytic vaccines.

Zepbound, a weight loss drug by Eli Lilly & Co., is the first prescription medication approved by the FDA to treat obstructive sleep apnea (OSA) in adults with obesity. Studies show Zepbound, used with a reduced-calorie diet and exercise, significantly reduces OSA symptoms. The American Academy of Sleep Medicine supports its use but notes it's not for everyone, emphasizing sustained weight loss is crucial for its effectiveness.

Dextromethorphan, an FDA-approved drug commonly used in cough medicine, shows potential in treating lung fibrosis by impeding collagen that forms scars. A study published in Science Translational Medicine by EMBL Heidelberg scientists revealed its efficacy in reducing lung fibrosis in mouse models and human lung tissue. The research aims to identify the drug's targets in cells to develop improved variants.

Pfizer announced FDA approval of BRAFTOVI (encorafenib) in combination with cetuximab and mFOLFOX6 for metastatic colorectal cancer (mCRC) with BRAF V600E mutation. The approval is based on the phase 3 BREAKWATER trial, showing significant improvement in response rate and durability of response. The regimen offers new hope for patients with limited treatment options.

Aurobindo Pharma shares rose 2% to Rs 1,265 on BSE after CuraTeQ Biologics received UK MHRA approval for Bevgolva, a biosimilar of bevacizumab for treating multiple cancers. The drug will be available in 4 mL and 16 mL vials for intravenous use, also indicated for non-squamous non-small cell lung cancer, advanced renal cell carcinoma, cervical cancer, and ovarian-related cancers.