Acromegaly is a rare endocrine condition caused by hypersecretion of human growth hormone (GH) that results in elevated circulating levels of GH and insulin-like growth factor-1 (IGF-1). Opko Biologics Ltd. has presented data on the development and characterization of OPKO-8801001, a long-acting human GH receptor (GHR) antagonist with prolonged systemic exposure and pharmacological activity designed for subcutaneous administration.
Researchers at Theramir Ltd. presented preclinical proof-of-concept data for a new extracellular vesicle (EV) platform engineered to deliver miR-885-5p (EVmiR885-5p), targeting leukocyte-specific protein-1 (LCP-1)-mediated cytoskeletal remodeling in triple-negative breast cancer (TNBC) models.
The alternative pathway (AP) of the complement system is crucial for innate immunity and its dysregulation may lead to several diseases, including paroxysmal nocturnal hemoglobinuria (PNH). Alnylam Pharmaceuticals Inc. and Regeneron Pharmaceuticals Inc. recently presented data on ALN-CFB, a siRNA targeting hepatic complement factor B (CFB) that inhibits the production of CFB in the liver.
Light chain multiple myeloma (LCMM) is a cancer driven by malignant plasma cells that produce excessive pathogenic free light chains (FLCs) that may cause kidney dysfunction and form amyloid deposits in key organs, thus leading to poor outcomes. Ab Studio Inc.’s CATA-001 is a bispecific antibody targeting both CD38 and aggregated light chains (ALs) designed to deplete CD38+ plasma cells and clear both circulating and tissue-deposited pathogenic FLC aggregates for the treatment of LCMM and AL amyloidosis.
Ethyreal Bio Inc. has developed ETHY-001, a half-life-extended monoclonal antibody targeting thyroid-stimulating hormone receptor (TSHR) designed to prevent the production of pathogenic autoantibodies (TSAbs) to treat Graves’ hyperthyroidism and thyroid eye disease (TED).
At the recent Endocrine Society meeting in Chicago, Kalohexis Inc. presented preclinical efficacy data on 710GO, an oral dual MC3R/MC4R agonist, in obese nonhuman primates (NHPs).
Researchers from Rivus Pharmaceuticals Inc. presented preclinical efficacy data on RV‑202, a novel oral mitochondrial uncoupler with activity attributed to adenine nucleotide translocase (ANT) activation, in models of obesity.
Advanced penile cancer (PeCa) is a rare cancer affecting the genitourinary tract with a 5-year survival rate of about 10% when metastatic. First-line therapies achieve objective response rates of 40%-50%, while immunotherapy has not been established.
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