A new phase 2, interventional, open-label, single-group assignment study of 5 years of adjuvant imatinib treatment in patients with gastrointestinal stromal tumors (GISTs) is expected to open on July 1, 2022. The aim is to assess the safety and efficacy of 5 years of adjuvant imatinib treatment in patients with large tumors (10 cm or…
Boehringer Ingelheim has reported encouraging 12-week phase 2 trial data on their investigational BI 1015550 therapy, a novel phosphodiesterase 4B inhibitor for patients with idiopathic pulmonary fibrosis (IPF). The results, reported via news release, were presented at the American Thoracic Society (ATS) International Conference breaking news session in San Francisco, California on May 16, 2022.…
Nabiximols therapy reduced baseline spasticity symptoms in patients with multiple sclerosis (MS), a new study published in the European Journal of Neurology found. One-third of the studied patients maintained the treatment at 18 months. “This study aimed to assess the relationship between changes in spasticity severity (measured on the 0-10 [numerical rating scale] and the…
Inhibrx Incorporated announced favorable initial results of its phase 1 clinical trial on the safety, pharmacokinetics, and pharmacodynamics of the optimized recombinant human alpha-1 antitrypsin (AAT)-Fc protein therapy INBRX-101 for patients with alpha-1 antitrypsin deficiency (AATD). The results, announced via news release, showed favorable safety and tolerability, and no drug-related severe or serious adverse events.…
Researchers reported a rare case of calcitonin-negative medullary thyroid carcinoma (MTC) with brain metastasis in a 64-year-old man and found that although serum calcitonin and carcinoembryonic antigen (CEA) concentrations typically serve as biomarkers for MTC diagnosis, both were normal in this case. “We describe a case of metastatic MTC that, despite tumor positivity for calcitonin,…
Researchers identified associations between the bone morphogenetic protein 6 (BMP6), Klotho (KL), and annexin A2 (ANXA2) polymorphisms and avascular necrosis (AVN) in patients with sickle cell disease (SCD), as published in Revista Paulista de Pediatria. The review was based on the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines and covered 12 studies between…
Researchers showed that sotatercept, an activin receptor type IIA-Fc (ActRIIA-Fc) fusion protein used in patients with pulmonary arterial hypertension (PAH), normalizes macrophage infiltration and reverses inflammation and proliferative gene expression in PAH rats. They noted that vasodilators such as the phosphodiesterase type 5 inhibitor sildenafil did not have the same beneficial effects on diseased rat…
Cogent Biosciences announced an upcoming presentation of initial data from the phase 2 APEX trial on bezuclastinib for patients with advanced systemic mastocytosis (SM). The presentation, announced via news release, will occur at the European Hematology Association (EHA) Annual Congress on June 10, 2022. “There is a significant unmet need for novel, safe, and effective treatment…
Albireo Pharma announced in a news release that their phase 3 ASSERT study of odevixibat (Bylvay™) in patients with Alagille syndrome (ALGS) is fully enrolled and initial results are expected by the end of the year. They also announced that the commercial launch of odevixibat in the United States, Germany, and the United Kingdom is…
Amicus Therapeutics announced Prescription Drug User Fee Act action dates of May 29, 2022, for the New Drug Application and July 29, 2022, for the Biologic License Application regarding the 2-component cipaglucosidase alfa/miglustat (AT-GAA) therapy for Pompe disease (PD). The company noted that the US Food and Drug Administration and European Union regulatory reviews of…
