Highlight

1. Effective PI3K/mTOR dual inhibitor.

1) Blockage of PI3K/mTOR pathway decreases cell proliferation, playing an anti-tumor and anti-fibrosis role. Asset is able to inhibit four different subtypes of PI3K(α, β, γ, δ) kinase as well as mTOR kinase, so that the down-stream proteins function is affected by vary degrees.

2) Asset showed stronger PI3K (α, β, γ, δ) and mTOR inhibitory activity than competitor, and showed PI3K phosphorylated inhibitory activity in vitro.

2. Huge market for IPF.

1) The total IPF patient population worldwide was 1.27 M in 2019, and Five-year survival rates from initial diagnosis of an Acute IPF is 18.4%, which is less than that of most cancers.

2) Two marked drugs for IPF are limited effect with sever AEs and still unable to stop IPF progression. No drug has been recommended as Class I strategy for IPF in China.

3) Asset phase 1 trial for IPF has been completed in US.

3. Excellent for IPF treatment and prevention.

1) Asset inhibited HFL1 cell proliferation in a nanomolar lever, much more effective than Pirfenidone and Nintedanib (micromolar level) for IPF in vitro.

2) Asset showed better anti-fibrosis effect at 3 mg/kg/d dose than Pirfenidone (100 mg/kg/d) and Nintedanib (100 mg/kg/d) in vivo.

3) For preventive treatment, asset showed showed better anti-fibrosis effect at 0.75 mg/kg/d dose than Pirfenidone (100 mg/kg/d) and Nintedanib (100 mg/kg/d) in vivo.

4. High potential for anti-solid tumor.

1) Only one PI3K/mTOR dual inhibitor: paxalisib is under phase 3 trial.

2) Asset is in phase 1 clinical trial for solid tumor.

3) The potential of asset to treat patients with PI3KCA-mut or PTEN-mut tumor is testing.

5. Effective for a variety of solid tumors.

1) Asset strongly inhibited proliferation of tumor cells from multiply tissue in a nanomolar lever.

2) Asset showed better suppression of tumor growth than BKM120 at a 10-time less concentration (3 mg/kg/d) in HCC1954 (breast), PC3 (prostate) and NCI-H1975 (NSCLC) subcutaneous tumor model.

6. Good safety and phase1 trial in China and US.

1) hERG IC50/Free Cmax >50, 1mg/kg and all genotoxicity tests are negative in preclinical study.

2) Oral administration for patients with advanced solid tumor (breast, colon, and neuroendocrine) phase 1 trial in China has been completed.

3) No dose-related AEs and no SAE were found in US phase 1 trial for IPF.

Research progress：

1. IPF andsolid tumor indications are in phase 1 trials;

2. For IPF, asset show better effective than Pirfenidone and Nintedanibat a less dose;

3. For solid tumor, asset show better effective than BKM120.