Capricor Therapeutics Inc. received a complete response letter (CRL) from the U.S. FDA on the BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy patients. The company’s CEO said the letter was unexpected.
Nuclidium AG is poised to circumvent the complex production and supply issues that have held back access to radiopharmaceuticals after closing a CHF79 million (US$99.3 million) series B, to advance the clinical development of its copper isotopes and extend the global manufacturing network.
Ultragenyx Pharmaceutical Inc. and Mereo Biopharma Group plc, along with investors, had hoped for an early halt to the phase III study of setrusumab in treating brittle bones. They didn’t get it, as the clinical trial’s data monitoring committee wants the study to roll on through its final analysis.
China has proved to be a fertile ground for innovation as evidenced by some big deals in the antibody-drug conjugate (ADC) space, and the number of candidates entering clinical trials in China or being advanced in the U.S. by Chinese companies.
Rhythm Pharmaceuticals Inc. will be sitting down with regulators in the U.S. and Europe to discuss phase III plans in the wake of positive phase II data from the study testing bivamelagon (formerly LB5-4640), an oral melanocortin-4 receptor agonist, in patients with acquired hypothalamic obesity.
Representatives of patients’ groups, industry bodies and venture philanthropy funders are calling for a renewal of the U.K. Rare Diseases Framework, to put fresh momentum behind translational research and clinical trials, streamline regulatory oversight and improve access to therapies.
In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III Reach-DMD trial, a randomized, placebo-controlled, double-blind and open-label, extension study of pizuglanstat in patients with DMD showed no significant difference in the mean change from baseline to 52 weeks in the primary endpoint of time to rise from the floor in the ambulatory cohort of the study.
Tickling Wall Street’s already strong interest in the mechanism of action was Aurinia Pharmaceuticals Inc., which June 30 made public positive results from the phase I single ascending-dose study with aritinercept (AUR-200), the company’s dual inhibitor of B cell-activating factor (BAFF) and a proliferation-inducing ligand (APRIL).
A mixed bag of top-line phase II data prompted stellar stock results for Prokidney Corp. and its chronic kidney disease (CKD) and diabetes therapy, rilparencel. The results came from two arms of the study, each with its own treatment regime. The market heartily took to the results as the company’s stock (NASDAQ:PROK) closed 515% upward at $3.73 per share after starting the day under $1 each.
Apogee Therapeutics Inc. plans to enter a crowded atopic dermatitis market with established drugs, such as Dupixent (dupilumab, Regeneron Pharmaceuticals Inc. and Sanofi SA) and Ebglyss (lebrikizumab-lbkz, Eli Lilly and Co.), which are sold by much larger competitors. But the company thinks it can compete with its anti-IL-13 antibody, APG-777, thanks to the drug’s 77 day half-life that may allow it to be dosed only two to four times a year in the maintenance phase, compared to 13 to 26 injections per year for the established competitors.
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