Curated news and analysis on clinical trials, drug approvals, and medical research.
Researchers have identified KHDC1L as a circulating biomarker for DUX4 activity in facioscapulohumeral muscular dystrophy (FSHD). The discovery could enable non-invasive blood-based monitoring of disease progression instead of muscle biopsies. The finding emerged from collaboration between academic researchers and Avidity Biosciences scientists.
AIxCrypto Holdings reported its first full-year results after transforming from a biotech firm to an AI and blockchain infrastructure company. The company completed a $41.0 million PIPE financing, rebranded, and began Nasdaq trading in November 2025. Financial results show approximately $31 million in total assets, $19 million in cash, and platform metrics of over 5.8 million registered wallets.
Oral semaglutide demonstrated significant improvements in multiple cardiometabolic risk factors in adults with type 2 diabetes at high cardiovascular risk. The SOUL trial analysis showed reductions in systolic blood pressure, cholesterol, triglycerides, HbA1c, and body weight compared to placebo. These benefits were observed as early as 13 weeks and contributed to a 14% reduction in major adverse cardiovascular events.
Research shows chloramine disinfection successfully stopped a Legionnaires' disease outbreak in Minnesota, while clinical data reveals Legionella pneumonia has 11.9% 30-day mortality and disproportionately affects immunocompromised patients. The studies highlight both prevention strategies and diagnostic challenges.
Iterion Therapeutics has dosed the first patient in a phase 1/2 trial of its Wnt/β-catenin inhibitor tegavivint for metastatic colorectal cancer. The expansion builds on promising monotherapy activity observed in advanced hepatocellular carcinoma, targeting a pathway activated in over 90% of colorectal cancer patients.
NIH has obligated only 15% of its $38 billion budget halfway through the fiscal year, causing universities to cut PhD admissions and implement hiring freezes. Early-career scientists face declining grant success rates despite increased applications. Congress recently reversed proposed 40% NIH budget cuts that had threatened research facility construction.
A phase 1 trial shows denileukin diftitox administered before CAR-T therapy has a favorable safety profile and encouraging efficacy in high-risk DLBCL patients, with an 86% overall response rate and 77% one-year progression-free survival. The treatment demonstrated consistent regulatory T-cell depletion, supporting its immunomodulatory mechanism. Larger controlled studies are needed to confirm these preliminary findings.
PDS Biotechnology will report 2025 financial results and provide clinical updates on March 30, 2026. The company faces critical regulatory decisions for its Phase 3 VERSATILE-003 trial in HPV16-positive head and neck cancer and is expanding its immunotherapy platform to prostate cancer and other solid tumors.
China's newly revised Implementation Regulations of the Drug Administration Law will take effect on May 15, 2026, representing the first comprehensive revision in over two decades. The regulations strengthen the marketing authorization holder system, improve drug development processes, and introduce new provisions for clinical trials, data protection, and market exclusivity for pediatric and rare disease drugs.
Clinical trials show psychedelic substances like psilocybin, LSD and MDMA combined with therapy can effectively treat depression, anxiety and addiction. Several substances are in Phase III trials, with esketamine already approved for treatment-resistant depression. Research suggests these treatments may work with fewer doses than traditional medications.
Two research teams have developed catalytic methods for synthesizing chiral sulfur compounds with antiviral potential. One approach uses an organocatalytic strategy to create vinyl sulfinamides that bind to SARS-CoV-2 and HIV-1 proteins, while another employs copper catalysis for high-yield production of chiral sulfilimines. Both methods address critical gaps in accessing valuable chemical space for drug discovery.
Diamyd Medical anticipates March 2026 interim results from its Phase 3 diabetes trial that could support a BLA pathway. Capricor Therapeutics awaits an August 2026 FDA decision on its Duchenne muscular dystrophy treatment BLA. Bicara Therapeutics prepares for Phase 3 study initiation of its oncology candidate toward future BLA submission.
Italian pharmaceutical company Recordati reported 2025 revenues of €2.6 billion and adjusted net profit of €651.1 million, with rare disease therapies driving 29.7% growth. The company projects 2026 core earnings of €995 million to €1.03 billion, anticipating continued momentum in its specialized rare disease treatments.
NASA successfully completed a wet dress rehearsal for the Artemis II moon mission, fueling the SLS rocket and testing launch procedures. The test involved loading over 700,000 gallons of propellant and conducting terminal count runs, with the four-person crew observing from launch control. Preparations continue for a potential March launch window.
Researchers have identified a new genetic disease characterized by premature aging and cognitive deficits, tracing it to a mutation in the IVNS1ABP gene. Using cellular reprogramming, they discovered the mutation causes cellular senescence and DNA damage during cell division through altered actin dynamics. The findings highlight the potential of patient-derived stem cell models to study rare diseases and identify potential treatment approaches.
The myelofibrosis therapeutic pipeline now includes over 40 candidates from more than 35 companies, with recent developments including Orphan Drug Designation for CK0804 and multiple Phase III trial initiations. Key clinical milestones include completed enrollment in Karyopharm's SENTRY trial and upcoming Phase I data from Incyte's combination therapy studies.
Interim results from the ABC phase 1/2b trial show a calcineurin inhibitor-free GVHD prevention regimen combining posttransplant cyclophosphamide, bortezomib, and abatacept achieved low GVHD rates and favorable survival outcomes. The approach enables earlier introduction of posttransplant maintenance therapies while reducing toxicity associated with conventional immunosuppressants.
ImmunityBio reported a fivefold revenue increase to $38.29 million, driven by a 750% surge in unit sales of its cancer drug Anktiva. The company is expanding internationally through partnerships in the Middle East and preparing additional FDA submissions for Anktiva's broader use.
New research challenges the theory that ketamine's psychedelic effects drive its therapeutic benefits for alcohol use disorder. A study of 96 participants found no link between ketamine's psychoactive experiences and improved abstinence rates. The findings suggest other mechanisms, such as brain network alterations, may explain ketamine's effectiveness in treating addiction.
NICE has upheld appeals to review its guidance on Alzheimer's drugs lecanemab and donanemab, sending the decision back to committee. The manufacturers argued NICE failed to account for the treatments' wider impact on unpaid carers. This comes amid broader challenges in neurodegenerative disease research following recent high-profile clinical trial failures.
