Nine promising small molecule drug discovery companies to look out for in 2025

 


Ascentage Pharma is discovering and developing small molecule drugs for the treatment of cancer that block or degrade protein targets implicated in cancer. It has active clinical programs targeting all three known classes of key apoptosis regulators that restore programmed cell death, a natural process where cells self-destruct in a controlled manner to maintain tissue homeostasis and eliminate damaged or potentially harmful cells. If this process is dysfunctional, however, it can lead to various diseases, especially cancer. 

The company’s most advanced candidate is olverembatinib, which is cleared in China for certain patients with chronic myeloid leukemia, and is in phase 3 trials in multiple countries for the same indication, as well as for two other forms of cancer. 

The company’s lead candidate is a PARG inhibitor called ETX-19477, which is in a phase 1 trial for solid tumors. PARG – which stands for poly(ADP-ribose) glycohydrolase – is an enzyme that plays an essential role in DNA repair. While the better-known poly(ADP-ribose) polymerases (PARPs) detect DNA damage and repair it by adding poly(ADP-ribose) chains to various proteins involved in the repair process, PARG is responsible for removing these chains once the job is done. By inhibiting PARG, the normal cycle of poly(ADP-ribose) metabolism is disrupted, in turn hampering the cells’ ability to repair DNA damage efficiently. Because cancer cells are particularly vulnerable to this disruption, PARG inhibitors can selectively target cancer cells while sparing normal, healthy cells that have more robust repair mechanisms.

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