Disc Medicine Inc. CEO John Quisel said that top-line phase II findings from the study called Aurora with bitopertin in erythropoietic protoporphyria are “hard for us to interpret. This package of data is something that we’re going to have to sort through,” and the Watertown, Mass.-based firm expects to talk with the U.S. FDA about next steps in the second half of this year.
In a potential watershed moment for South Korea’s Hanmi Group, a hair-splitting vote at the 51st shareholder meeting favored the two sons of the late founder and Chairman Lim Sung-ki, effectively halting a merger between Hanmi and OCI Holdings Co. Ltd.
Novo Nordisk A/S is the latest drug company to be challenged by U.S. Sen. Bernie Sanders (I-Vt.), who has made tilting at prescription drug prices one of the hallmarks of his tenure as chair of the Senate Health, Education, Labor and Pensions (HELP) Committee.
Hoth Therapeutics Inc.’s subsidiary, Merveille.ai, has used advanced artificial intelligence to identify a promising new therapeutic candidate for obesity.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Cinfina Pharma Inc. has obtained FDA clearance of its IND application for CIN-110, a PYY3-36 analogue, allowing it to initiate a first in-human study in otherwise healthy subjects with obesity. CIN-110 is a stable and long-acting analogue of PYY3-36 being developed both as a monotherapy and for co-administration for obesity.
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
The fast-shifting obesity space gained more clinical results as Viking Therapeutics Inc. shared data from its phase I, multiple ascending-dose trial with oral VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
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