Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Chiesi Farmaceutici SpA scored U.S. FDA clearance of the enzyme replacement therapy (ERT) Lamzede (velmanase alfa-tycv) for non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. An ultra-rare, progressive lysosomal storage disorder, AM is caused by deficiency in the enzyme alpha-mannosidase. Lamzede is the first ERT to win approval in the indication, characterized by an inability to properly break down certain groups of complex sugars in the body’s cells.
The steadily percolating psychedelic drug space stands poised to generate a near-term stream of potentially encouraging developments in a range of mental health disorders, including depression, anxiety, post-traumatic stress disorder and more.
Corbus Pharmaceuticals Holdings Inc. signed a back end-loaded licensing deal worth up to $692.5 million with CSPC Megalith Biopharmaceutical Co. Ltd, a subsidiary of CSPC Pharmaceutical Group Ltd., for the latter’s Nectin-4-targeting antibody-drug conjugate CRB-701 (SYS-6002).
Corbus Pharmaceuticals Holdings Inc. signed a back end-loaded licensing deal worth up to $692.5 million with CSPC Megalith Biopharmaceutical Co. Ltd, a subsidiary of CSPC Pharmaceutical Group Ltd., for the latter’s Nectin-4-targeting antibody-drug conjugate CRB-701 (SYS-6002).
Leveraging ferroptosis, or iron-mediated cell death, Hillstream Biopharma Inc. has turned up intriguing early stage data in non-small-cell lung cancer (NSCLC) when the company’s candidate called HSB-1216 was paired with the anti-PD-1 antibody Keytruda (pembrolizumab, Merck & Co. Inc.).
With the U.S. FDA go-ahead Feb. 2 for GSK plc’s oral daprodustat for anemia in patients with chronic kidney disease (CKD), the picture brightened for would-be competitors in the hypoxia-inducible factor prolyl hydroxylase (HIF-PHI) inhibitor space, including high-profile Akebia Therapeutics Inc., which has appealed last spring’s the complete response letter from gatekeepers with regard to vadadustat.
Spinal muscular atrophy (SMA) has been surfacing more regularly in scientific journals lately, as drug developers – such names as Biohaven Inc., Roche Holding AG and Scholar Rock Inc. – continue to search for improved therapies directed at the condition, one that takes in a group of hereditary, motor neuron-destroying diseases.
Evelo Biosciences Inc.’s late-stage efforts with EDP-1815 are forging ahead despite unfavorable data in atopic dermatitis (AD), and talks so far with the U.S. FDA have proven encouraging, the company said. Cambridge, Mass.-based Evelo made public findings from the first three cohorts of the phase II trial with EDP-1815 in AD, where the compound fell short of the primary endpoint: proportion of patients who achieve an outcome of at least a 50% improvement from baseline in Eczema Area and Severity Index (EASI) score compared to placebo at week 16. Evelo blamed an unusually high placebo response rate.
