NRG Therapeutics doses first participants in landmark ALS & Parkinson’s trial

The mPTP plays a key role in mitochondrial dysfunction, inflammation, and neuronal death in neurodegenerative diseases

Cellenkos scores FDA orphan drug designation for CK0804 in rare blood cancer

Receiving Orphan Drug Designation is an important milestone in the clinical development of CK0804 for myelofibrosis

EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard plasma-derived therapy in adults with AATD

Sanofi flags delay in FDA review of Tolebrutinib for progressive multiple sclerosis

GSK’s experimental lung cancer drug gains FDA orphan drug status

GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to advance its development and commercialization

Teva files FDA application for once-monthly schizophrenia injection

The investigational therapy uses SteadyTeq, a proprietary copolymer technology from Medincell designed to release olanzapine steadily over time

AL-S Pharma shows promising Phase 2 results for its therapy in ALS treatment

Adverse events were similar to placebo, and no AP-101-induced antibody responses were detected

Pfizer’s therapy scores major win in Phase 3 trial for Hemophilia patients with inhibitors

HYMPAVZI’s safety profile was generally favorable

Takeda seals $1.2 billion oncology deal with Innovent Biologics

Takeda will pay Innovent US$1.2 billion upfront, including a US$100 million equity investment in Innovent

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes