Amphista Therapeutics Ltd. has obtained IND clearance from the FDA for AMX-883, an orally bioavailable degrader of BRD9, for the treatment of acute myeloid leukemia (AML). A phase I trial in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome is expected to begin in the second half of this year.

Previous work found that certain short RNAs can induce cell death in a RISC-dependent fashion by targeting several networks of survival genes simultaneously, therefore triggering multiple cell death pathways. This form of cell death was named death induced by survival gene elimination, or DISE, an effect that depends on a toxic 6-mer seed.

About 90% of brain metastases are often limited therapeutically speaking due to the impermeable blood-brain barrier (BBB). Nanocarry Therapeutics Ltd. has presented AxS007, a novel insulin-mediated nanocarrier that delivers multiple copies of trastuzumab and pertuzumab across the BBB, using native insulin as a brain transporter and increasing brain exposure.

The University of Texas MD Anderson Cancer Center reported findings from studies of CBT-001-2334, a radionuclide peptide targeting carbonic anhydrase IX (CAIX/CA9) designed for diagnostic gallium labeling and downstream therapeutic isotope pairing. It features a DOTA chelator for use in theranostic applications through chelating either diagnostic or therapeutic radionuclides. Because it has limited expression in normal tissue, CAIX is an attractive target in clear cell renal cell carcinoma (ccRCC).

Researchers from Pfizer reported preclinical efficacy of PF-08052667, an antibody-drug conjugate (ADC) targeting integrin β6 (ITGB6), in non-muscle-invasive bladder cancer (NMIBC) models.