At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
A paper published March 22 in the journal Chest highlighted research that found patients with interstitial lung disease (ILD) who had poor quality of life specifically related to cough were more likely to be hospitalized for respiratory issues, need a lung transplant and die. The findings in 1,447 patients matter because they back previous investigations and provide a way of predicting the advance of ILD, of which idiopathic pulmonary fibrosis (IPF) is a prominent type. A handful of companies are working on drugs to tackle cough in IPF, often an early symptom of the disease.
Regenerative medicine company Orthocell Ltd. reported final data from its nerve reconstruction study that showed patients continued to improve between 12- and 24-months post-treatment with regenerative nerve repair device Remplir.
Penumbra Inc. presented new data at the LINC Symposium in Leipzig, Germany, evaluating its Indigo aspiration system for submassive pulmonary embolism (PE). The subgroup analysis evaluated performance outcomes of patients with main pulmonary artery (PA) emboli vs. discrete unilateral or bilateral PA emboli without main PA involvement. Data from the study suggests the system can be used effectively for both proximal and distal aspiration thrombectomy in the pulmonary arteries.
Hua Medicine Ltd. presented findings from two phase III trials of its first-in-class dual-acting glucokinase activator dorzagliatin that show the drug significantly improved early phase insulin secretion and glucose sensitivity in patients with type 2 diabetes, thereby restoring glucose homeostasis to treat the underlying cause of diabetes.
Pointing to an anomalously high placebo response rate at eastern European trial sites in its Forward phase III trial testing fostamatinib in warm autoimmune hemolytic anemia (wAIHA), Rigel Pharmaceuticals Inc. executives remained confident there could be a path forward for the SYK inhibitor in treating the rare blood disorder. The disappointing top-line data, however, sent company shares (NASDAQ:RIGL) falling more than 60% June 8 to close at 70 cents, a penny above its same day 52-week low.
Based on a preliminary data analysis of its second and newest COVID-19 booster candidate, Moderna Inc. said it plans to ask the U.S. FDA to approve mRNA-1273.214 ahead of potential shipping in late summer 2022. The vaccine contains mRNA-1273 (Spikevax) and a vaccine candidate targeting omicron. New phase II/III results show the candidate hitting all the study’s primary endpoints when compared to Moderna’s original vaccine, mRNA-1273.
Sophia Genetics SA shared preliminary findings from its multimodal Deep-Lung-IV clinical study at the 2022 American Society of Clinical Oncology annual meeting. The study is utilizing Sophia’s cloud-based, artificial intelligence diagnostic platform to identify multimodal predictive signatures of response to immunotherapy for patients with advanced lung cancer.
Several companies showed promising results for automated insulin delivery (AID) systems at the American Diabetes Association (ADA) annual meeting in New Orleans. Studies of the systems, also known as artificial pancreas systems, indicate that integration with continuous glucose monitoring (CGM) systems enables substantially improved glycemic control, with more time in range (TIR) and less hypoglycemia than seen with multiple daily injection (MDI) therapy or insulin-pump therapy.
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