News & Analysis

Curated news and analysis on clinical trials, drug approvals, and medical research.

Researchers Develop New Methods for Chiral Sulfur Compounds with Antiviral Potential

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.

Biotech Firms Advance BLA Pathways with Key Clinical Milestones

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.

Recordati Reports Strong 2025 Results, Projects 2026 Growth Driven by Rare Diseases

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 Completes Key Fuel Test for Artemis II Moon Mission

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.

Scientists Discover New Genetic Disease Causing Premature Aging and Cognitive Deficits

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.

Myelofibrosis Pipeline Expands with Over 40 Candidates from 35+ Companies

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.

Novel GVHD Prevention Regimen Shows Strong Results Without Standard Immunosuppressants

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.

Ketamine's Therapeutic Benefits Not Linked to Psychedelic Effects in Alcohol Use Disorder

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 to Review Alzheimer's Drug Guidance After Appeals Upheld

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.

DOPA Decarboxylase Biomarker Improves Diagnosis of Parkinson's and Lewy Body Dementia

Researchers have identified DOPA decarboxylase as a quantitative biomarker in cerebrospinal fluid that improves diagnosis of Parkinson's disease and Lewy body dementia. The protein levels are up to 2.5 times higher in affected patients and can differentiate these conditions from Alzheimer's disease. The discovery provides clinicians with an objective diagnostic tool for conditions that are frequently misdiagnosed due to overlapping symptoms.

FDA Approves Subcutaneous Amivantamab for EGFR-Mutated NSCLC

The FDA approved subcutaneous amivantamab for EGFR-mutated NSCLC in December 2025 based on phase 3 PALOMA-3 trial data showing noninferior efficacy with reduced infusion reactions and 5-minute administration time. The formulation decreases infusion-related reactions from 66% to 13% and improves patient convenience and quality of life. Recent MARIPOSA trial updates show amivantamab plus lazertinib extends median overall survival by at least 12 months versus osimertinib monotherapy.

Cell Therapy Market Growth Accelerates with CAR-T Dominance and New Research Milestones

The cell therapy manufacturing market is projected to reach $14.01 billion by 2035, with CAR-T therapies dominating at 65% market share. Recent FDA approvals for new CAR-T indications and Japanese regulatory acceleration highlight growing clinical adoption, while research advances include new anti-aging protein platforms and CRISPR-based treatments.

Egetis Therapeutics Reports Progress on Emcitate for MCT8 Deficiency

Egetis Therapeutics completed its rolling New Drug Application for Emcitate® (tiratricol) for MCT8 deficiency in the USA on January 29, 2026. The drug received European Commission approval in February 2025 and launched in Germany in May 2025. The company also provided updates on Japanese regulatory progress and the status of its Aladote® candidate for paracetamol overdose.

Elicio Therapeutics Grants Inducement Stock Options to New Employees

Elicio Therapeutics granted inducement stock options to new employees, with one grant of 1,600 options at $12.16 per share and another of 30,300 options at $8.59 per share. The clinical-stage biotech company is developing novel immunotherapies targeting mKRAS-positive cancers, with its ELI-002 program showing promising Phase 1 results including 16.3-month median recurrence-free survival.

Women With Cancer Show Improved Survival But Higher Risk of Severe Side Effects

A meta-analysis of 20,806 cancer patients shows women have significantly improved survival rates but higher risk of severe treatment side effects compared to men. Separate research indicates nutrition counseling could support fertility in female cancer survivors, though implementation faces barriers. Specialized cancer nurses play a crucial role in providing stability and support throughout cancer treatment.

Ocular Therapeutix's Axpaxli Outperforms Eylea in Wet AMD Trial

Ocular Therapeutix's experimental drug Axpaxli showed superior vision maintenance compared to Regeneron's Eylea in a late-stage wet AMD trial. Meanwhile, Ocugen reported 12-month data showing its gene therapy OCU410 reduced geographic atrophy lesions by 31%, though less than earlier interim results. Both companies are advancing toward regulatory submissions and further clinical development.

Ocugen Reports Phase 2 Data for Geographic Atrophy Gene Therapy OCU410

Ocugen reported Phase 2 data for its geographic atrophy gene therapy OCU410 showing 31% lesion growth reduction versus control. The company plans Phase 3 initiation in Q3 2026 and maintains its goal of three BLA filings in three years. Shares fell 11.48% following the data release which trailed earlier interim results.

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