MC2 Therapeutics A/S has discovered that carbamylation of proteins, peptides and amino acids in the skin, caused by isocyanate, may be the root cause of several urea-associated skin diseases, such as chronic kidney disease-associated pruritus (CKD-aP, or uremic pruritus) and genital lichen sclerosus.
Mas-related G-protein coupled receptor X2 (MRGPRX2) is expressed particularly on mast cells and its activation leads to the mast cell degranulation with release of inflammatory mediators.
Blueprint Medicines has identified new cAMP-dependent protein kinase catalytic subunit alpha (PRKACA) inhibitors reported to be useful for the treatment of Carney complex (NAME syndrome, LAMB syndrome), McCune-Albright syndrome, polycystic kidney disease and cancer.
As the PDUFA date looms for Bristol Myers Squibb Co. with its candidate, deucravacitinib, for psoriasis, others – notably Dice Therapeutics Inc. – strive for new solutions to the skin disease, which has remained problematic for many patients despite approvals of multiple drugs in various classes.
Shares of Pharvaris NV (NASDAQ:PHVS) fell 34% Aug. 22 to close at $12.15 after the company reported the U.S. FDA placed a clinical hold on PHA-121, its oral bradykinin B2-receptor antagonist for hereditary angioedema (HAE), citing a review of nonclinical data. PHA-121, which goes after the same target as well-established injectable HAE drug Firazyr (icatibant, Takeda Pharmaceutical Co. Ltd.), is the active ingredient in Pharvaris’ two lead programs: PHVS-416, a softgel capsule formulation, and PHVS-719, an extended-release tablet formulation.
TFChem SAS has described new cyclic glycoaminoacid derivatives reported to be useful for the treatment of dry skin, atopic dermatitis, aging, inflammatory disorders, fibrosis and psoriasis, among other disorders.
Allorion Therapeutics has divulged imidazolopyridazines or pyrazolopyrimidines acting as nonreceptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of psoriasis, psoriatic arthritis, ulcerative colitis, Crohn's disease and systemic lupus erythematosus.
Lynk Pharmaceuticals' LNK-01004 has been cleared by China's National Medical Products Administration (NMPA) for clinical trials in the treatment of atopic dermatitis.
Genentech Inc. is paying Kiniksa Pharmaceuticals Ltd. $100 million in up-front and near-term payments for the development and commercialization rights to vixarelimab, a fully human monoclonal antibody. Kiniksa also could receive up to approximately $600 million in certain clinical, regulatory and sales-based milestones, as well as royalties on annual net sales.
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