Ann: Positive preliminary results from ATL1102 DMD Phase II trial, page-116

ANP currently market cap: A$32m/USD$22m.

Exondys 51/Eteplirsen, trial 201 was only 12 patients.

ANP trial, 9 patients.

Eteplirsen can’t cure, just control, selling for USD$300,000 per year. It's a bit crazy price.

Global 44,000 patients, potential market size USD$13.2B.
No wonder Sarepta ran from US$60M to US$9B.

Good luck to ANP holders.


2.Eteplirsen new drug declaration data

This new drug application includes data from three clinical trials. Trial 201 was a single-center, double-blind, placebo-controlled trial. Twelve DMD patients were assigned to the Eteplirsen 30 mg/kg/week group, the 50 mg/kg/week group, and the placebo group in a 1:1:1 ratio.

Run 202 is a continuation test of 201. A placebo group of 201 patients began receiving Eteplirsen treatment in this 202 trial.

Trial 301 was an open-label test of external control, and all patients received Eteplirsen 30 mg/kg/week. The original application data only included 201/202 data, and later in June this year, it was requested to submit data for 13 patients in 301.

The primary endpoint of the trial included some indicators of action such as walking speed. The dystrophin protein content in the muscle serves as a surrogate endpoint. Western blot and immunohistochemistry (IHC) are two laboratory techniques for measuring dystrophin protein content.