Summary of Answers to Statistical and Regulatory Questions at GtreeBNT Shareholder Meeting
March 18, 2021*
1. Regarding topline data of ARISE-3 trial announced on March 18th
- The topline data we received from a statistical analysis service company was around 15~20% of total data to be analyzed.
- We’ll have a discussion with the statistical analysis service company and CRO using remaining prespecified parameters and post-hoc analysis to be available. All data analysis of ARISE-3 will be finished around the end of April.
- Based on all the data of ARISE-3, one of future development scenarios would be to explore the prospects of a pre-BLA meeting as announced on the news release dated March 18th.
2. The ground of the prospects of a pre-BLA meeting
① Dry Eye: Developing Drugs for Treatment – Guidance for Industry (draft) announced by the FDA in December 2020
(Source: FDA Dry Eye: Developing Drugs for Treatment Guidance for Industry, December 2020)
② The case of NDA approval from the FDA of Novartis’ Xiidra as the treatment of dry eye
- Novartis submitted NDA with three clinical trials (Phase 2, two Phase 3 trials (OPUS-1 and -2), but FDA declined to approve it and requested an additional clinical study (2015).
- Resubmission of NDA by including 4th clinical trial results (OPUS-3) (2016.1)
- The FDA approved Xiidra based on evidence from four clinical trials (2016.7)
- When Xiidra was approved, there was no FDA guidance to develop drugs for treatment of dry eye. FDA announced the draft guidance in December 2020 very first.
③ We have secured data shown statistical significance in secondary prespecified efficacy parameters in ARISE-1 and ARISE-2.
3. A rough expected timeline to submit a meeting request of pre-BLA
- To be finished all data analysis of ARISE-3 (End of April)
- Advisory and strategic establishment of the prospect of pre-BLA meeting with regulatory experts (End of July)
- Preparation and submission of Pre-BLA meeting request (End of August)
* Information provided by GtreeBNT to RegeneRx without editing