Vertex Pharmaceuticals Inc. has obtained IND clearance from the FDA for VX-407, an investigational first-in-class small-molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.
Maze Therapeutics Inc. has divulged apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of chronic kidney disease, focal segmental glomerulosclerosis, diabetic, hypertensive, HIV-associated nephropathy, lupus nephritis, pre-eclampsia and sepsis.
With a second batch of phase Ib data from the trial testing RGLS-8429 in hand from Regulus Therapeutics Inc., Wall Street is looking forward to results from the third cohort in midyear and weighing prospects with the compound in autosomal dominant polycystic kidney disease. Screening of the fourth cohort will start during the second quarter. Meanwhile, the company has begun to mull a pivotal phase II trial that would launch in the middle of next year, and may help with accelerated approval by the U.S. FDA.
Dimerix Ltd. announced a AU$20 million ($US13.22 million) capital raise following the news that its lead candidate, DMX-200, was successful in a prespecified interim analysis of the efficacy endpoint in its pivotal phase III trial in focal segmental glomerulosclerosis, a rare kidney disease.
Mironid Ltd. has synthesized phosphodiesterase PDE4 activators reported to be useful for the treatment cancer, hyperparathyroidism, autosomal dominant polycystic kidney disease and autosomal recessive polycystic kidney disease.
Paraneoplastic syndromes are a group of disorders associated with tumors that are prevalent in patients with lymphatic, lung, ovarian or breast cancers.
Fibrocor Therapeutics Inc. has entered into a research and development collaboration with the Mcquade Center for Strategic Research and Development LLC (MSRD).
Scientists at Mount Sinai School of Medicine and U.S. Department of Veterans Affairs have synthesized mothers against decapentaplegic homolog 3 (SMAD3) inhibitors reported to be useful for the treatment of renal, hepatic and pulmonary fibrosis.
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