Ascent Pharmaceuticals APG-115 for the treatment of stage IIB-IV melanoma was approved as an orphan drug by the FDA

On July 21, Ascent Pharmaceuticals announced that the company’s original innovative drug MDM2-p53 inhibitor APG-115 has been granted Orphan Drug Designation (ODD) by the U.

It is reported that this is the fifth orphan drug designation granted by APG-115 following gastric cancer, acute myeloid leukemia, soft tissue sarcoma, and retinoblastoma

Since 2011, immunotherapy represented by Immune Checkpoint Inhibitors (ICI) has brought unprecedented breakthroughs in the clinical treatment of melanoma

In tumor therapy, p53 gene is one of the most widely studied tumor suppressor genes.

Structure of APG-115

APG-115 is an oral, highly selective inhibitor of MDM2-p53 under development by Ascent Pharmaceuticals.

The data shows that in the cohort of APG-115 combined with pembrolizumab in the treatment of PD-1/PD-L1 inhibitor-resistant melanoma, one patient achieved complete remission (CR), and the objective response rate (ORR) of this cohort It reached 24.

In addition to APG-115, global MDM2 inhibitors are also Roche's pyrrolidine derivatives RG7388, RG7112, Rain Therapeutics' RAIN-32, and Amgen's AMG232

Reference materials:

[1]https://mp.

[2]https://mp.