The invention concerns a novel oral small molecule HDAC6 inhibitor, MPT0G211, which has been shown as a potential therapeutic for cancer and neurodegenerative diseases. MPT0G211 showed superior selectivity and sensitivity than competitors Ricolinostat and Citarinostat. In multiple cancer xenograft models, MPT0G211 exerted significant anticancer activity both in single or in combination with cytotoxic and target therapies without toxicity. It especially synergized the effect of bortezomib with 97% Tumor Growth Inhibition in multiple myeloma. MPT0G211 also ameliorated cognitive impairment, showing better spatial memory than vehicle group in two AD animal models. In 3xTg-AD mice model, MPT0G211 both inhibited amyloid accumulation and Tau phosphorylation, two key pathological factors in AD. The accelerator SV Advantage has granted $1.5 M USD conditional funding offer for this project. It will help on fundraising plans to explore the oversea market.

Three HDAC6 inhibitors are in clinical trials for cancer treatment (Ricolinostat, Citarinostat, and KA-2507). MPT0G211 has superior selectivity and sensitivity than competitors, and well tolerated without cardiac toxicity. Current two marketed AD drugs only relieve symptoms with side effects; while MPT0G211 can block pathological factors (-amyloid and p-Tau). These are great advantages over other HDAC6 inhibitors and AD drugs on the market.

MPT0G211 will finish IND enabling studies around Q3 2021 and enter Phase I clinical trials around Q4 2021. MPT0G211 is currently testing for the therapeutic potential in neuropathic pain, and is planning for PD and ALS. MPT0G211 shows better selectivity and sensitivity, as well as block pathological factors in AD. These are great advantages of MPT0G211 on the market. The market potential of MM and AD will be $12.0 billion USD by 2024 and $12.6 billion USD by 2023. The accelerator Silicon Valley Advantage has granted $1.5 million USD conditional funding offer for this project. It will help on building business strategy and fundraising plans to explore the oversea market.