Developed as an autologous TCR-T cell therapy, LioCyx-M004 leverages mRNA technology to encode T-cell receptors that specifically recognize HBV antigens. Preclinical and early studies demonstrated its ability to reduce viral antigen load while maintaining a favorable safety profile. The therapy represents the first TCR-T candidate to enter clinical development for Chronic Hepatitis B, a condition that affects more than 290 million people globally.
“Our ability to successfully redirect our lead candidate from oncology to virology highlights the versatility of our platform,” added Dr. Tina Wang, Chief Medical Officer of Lion TCR. “With this IND clearance, we are one step closer to exploring LioCyx-M004’s potential as a functional cure for CHB.”
The company is also advancing its AI-powered TCR discovery platform and an in vivo TCR-T therapy program, validated by leading U.S. research institutes, with plans to expand into additional solid tumor indications such as lung, breast, and gastrointestinal cancers.
Corstasis Therapeutics Wins FDA Approval for ENBUMYST™ Nasal Spray to Treat Edema in CHF, Liver, and Kidney Disease
Corstasis Therapeutics Inc. announced that the FDA has approved ENBUMYST (bumetanide nasal spray) for the treatment of edema associated with congestive heart failure (CHF), hepatic disease, and renal disease, including nephrotic syndrome in adults. The therapy is positioned as the first self-administered outpatient alternative, bridging the gap between oral loop diuretics and intravenous (IV) therapy.
“The FDA approval of ENBUMYST represents a meaningful advancement in the treatment of edema for patients and providers,” said Ben Esque, Chief Executive Officer of Corstasis Therapeutics. “This new option can help improve access to care while addressing critical limitations of current therapies.”
In clinical studies, ENBUMYST demonstrated rapid absorption and a predictable diuretic effect, showing comparable efficacy to IV bumetanide injection. Traditional oral loop diuretics are often limited by poor gastrointestinal absorption, while IV therapies require hospital-based care. By offering a convenient nasal spray, ENBUMYST has the potential to reduce costly hospitalizations and readmissions, which remain a leading driver of healthcare spending in CHF and chronic kidney disease.
“ENBUMYST was designed in direct collaboration with cardiologists to address practical challenges in outpatient care,” added Dr. Brian Kolski, Chief Medical Director of Corstasis. “Its ability to be administered outside of the hospital could change the standard of care for managing fluid overload.” Corstasis expects to launch ENBUMYST in the U.S. in Q4 2025, with a commercial focus on cardiologists, nephrologists, hepatologists, and outpatient heart failure clinics.
Amneal Gains FDA Approval for Sodium Oxybate Oral Solution in Narcolepsy Patients with Cataplexy and Excessive Daytime Sleepiness
Amneal Pharmaceuticals, Inc. (“Amneal” or the “Company”) announced that the FDA has approved its sodium oxybate oral solution 500 mg/mL Abbreviated New Drug Application (ANDA), referencing Jazz Pharmaceuticals’ XYREM. The approval follows Amneal’s prior distribution of an authorized generic of sodium oxybate in limited quantities.
“This approval expands Amneal’s Affordable Medicines portfolio into a critical therapy area for patients living with narcolepsy, a rare neurological condition affecting approximately 150,000 individuals in the U.S.,” said Tony Rosa, Senior Vice President, Retail Affordable Medicines. “By offering sodium oxybate, Amneal is providing patients, providers, and payers with a more affordable alternative in a category historically limited to a single manufacturer.”
Sodium oxybate is a central nervous system depressant indicated for the treatment of cataplexy and excessive daytime sleepiness (EDS) in patients with narcolepsy. It is considered a standard-of-care therapy due to its ability to consolidate nighttime sleep and significantly reduce cataplexy episodes. Common adverse reactions in adults include nausea, dizziness, vomiting, somnolence, enuresis, and tremor, while pediatric patients may experience nausea, enuresis, vomiting, headache, decreased appetite, weight loss, dizziness, and sleepwalking.
The FDA approval positions Amneal to broaden access to this critical therapy, offering a safe and effective treatment option for patients with narcolepsy while supporting affordability and patient choice in a previously limited market.
Greenwich LifeSciences’ GLSI-100 Granted FDA Fast Track Designation for HER2-positive Breast Cancer
Greenwich LifeSciences, Inc., a clinical-stage biopharmaceutical company, announced that the FDA has granted Fast Track designation for GLSI-100 in patients with HLA-A*02 genotype and HER2-positive breast cancer following standard HER2/neu-targeted therapy. The designation may allow for more frequent interactions with the FDA and the potential use of a rolling Biologic License Application (BLA) review process.
Dr. Jaye Thompson, VP Clinical and Regulatory Affairs, commented, “Greenwich is pleased that the FDA sees the potential of GLSI-100 to change important clinical outcomes in this population of breast cancer patients. We continue to work earnestly to collect data to support a BLA filing demonstrating this benefit.”
GLSI-100 is being evaluated in the company’s Phase III clinical trial, FLAMINGO-01, which is focused on preventing breast cancer recurrence. The Fast Track designation recognizes the therapy’s potential to improve invasive breast cancer-free survival and address an unmet medical need.
CEO Snehal Patel added, “We are excited to have received Fast Track designation. The FDA review included an assessment of GLSI-100’s potential to prevent metastatic breast cancer recurrence in the patient population we are studying. We plan to continue discussions with the FDA and explore additional ways to make GLSI-100 available to larger populations.”
Akeso’s Ligufalimab Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia
Akeso Inc. (9926.HK) announced that its next-generation humanized IgG4 monoclonal antibody targeting CD47, ligufalimab (AK117), has been granted Orphan Drug Designation (ODD) by the FDA for the treatment of acute myeloid leukemia (AML). The designation provides comprehensive FDA guidance, tax incentives, and up to seven years of market exclusivity upon approval.
Ligufalimab is currently under international clinical development for both hematologic malignancies and solid tumors. Patient enrollment has completed in a randomized, double-blind, multicenter phase II study evaluating ligufalimab combined with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS). Additionally, ligufalimab is the first CD47 monoclonal antibody to enter registrational phase III trials in solid tumors, including PD-L1-positive head and neck squamous cell carcinoma (HNSCC) and pancreatic cancer.
AML is a heterogeneous hematologic malignancy and the most common acute leukemia in adults. Existing treatments for patients ineligible for intensive chemotherapy are limited, with more than half relapsing within 6–9 months despite therapies such as venetoclax in combination with azacitidine. Ligufalimab’s mechanism blocks CD47 on tumor cells, enhancing macrophage-mediated phagocytosis and inhibiting tumor growth while preventing red blood cell agglutination.
Preclinical and clinical studies show that ligufalimab, alone or combined with azacitidine or venetoclax, enhances “eat me” signals, promoting efficient immune response. In first-line AML patients, the phase II study demonstrated favorable safety and promising efficacy, with complete remission (CR) rates reaching 50% and composite CR (cCR) rates of 55% at the target dose of 45 mg/kg administered biweekly.
