Watchers of the percolating myasthenia gravis space are waiting eagerly for data from Dianthus Therapeutics Inc.’s phase II Magic study testing DNTH-103, an active C1s inhibitor, compared to placebo in patients with anti-AChR-positive generalized disease.
Days after Pfizer Inc. pulled the plug on its oral GLP-1 candidate danuglipron, Eli Lilly and Co. aired positive top-line data from the phase III trial called Achieve-1 testing orforglipron vs. placebo in adults with type 2 diabetes and inadequate glycemic control with diet and exercise alone.
More than a year after Ironwood Pharmaceuticals Inc.’s phase III hitch with apraglutide in short bowel syndrome, the other shoe fell from regulators as did the Boston-based firm’s shares (NASDAQ:IRWD), which ended April 14 at 64 cents, down 29 cents, or 31.5%.
With a promising IL-36 inhibitor for atopic dermatitis (AD, or eczema) at the phase II stage, Turn Therapeutics Inc. has gained $75 million in post-public commitments and meanwhile is pursuing a grassroots strategy to keep the coffers in balance.
The sparsity of mid-to-late stage prospects in atopic dermatitis (AD, or eczema) – which has proved an especially challenging indication – plus some newsmaking fizzles in the space have caused developers to probe new targets with particular intensity. Most popular approaches thus far involve IL-4, IL-13, thymic stromal lymphopoietin and JAK. Developers have stumbled for varying reasons such as high placebo response rates, safety or lack of clinical proof of concept. Among the potential AD rescuers is Nektar Therapeutics Inc. with rezpegaldesleukin (rezpeg), which takes aim at IL-2.
As developers continue to search for better amyotrophic lateral sclerosis (ALS) therapies, Neurosense Therapeutics Ltd. turned up some hopeful findings from its phase IIb Paradigm trial with PrimeC. The drug, a combination therapy (ciprofloxacin and celecoxib) designed to target multiple ALS pathways, is having salutary effects on microRNA modulation (miRNA), Neurosense said, with the study showing a “profound and consistent” downregulation of 161 mature miRNAs across all time points in the double-blind period of the experiment.
Cartesian Therapeutics Inc. followed up December’s phase IIb data with more good news regarding Descartes-08, offering 12-month efficacy and safety results that whetted Wall Street’s appetite for the phase III Aurora effort ahead. Milos Miljkovic, chief medical officer, told BioWorld that minimum symptom expression – among the areas where Descartes-08 shone, providing relief for “months and months after the last dose of treatment” – is especially important to patients.
Rhythm Pharmaceuticals Inc.’s sales with Imcivree (setmelanotide) in weight management could exceed $1 billion by 2028 if hypothalamic obesity (HO) is added to the label of the drug, Oppenheimer analyst Leland Gershell said in a report. Boston-based Rhythm rolled out positive top-line data from the pivotal phase III study called Transcend with the melanocortin-4 receptor agonist in HO. The global trial met its primary endpoint with a statistically significant and highly clinically meaningful reduction in body mass index in adult and pediatric HO patients vs. placebo.
Last October, having chalked another trial failure with E-selectin antagonist uproleselan, Glycomimetics Inc. made known its acquisition plan for privately held, solid tumor-focused Crescent Biopharma Inc. – backed by $200 million from a syndicate of investors who liked the odds of success with CR-001, a preclinical VEGFxPD-1 bispecific antibody.
Aldeyra Therapeutics Inc. is hoping that research already underway will satisfy the U.S. FDA, which delivered to the company another complete response letter (CRL) related to the NDA for reproxalap in dry eye disease (DED).
