Anavex 2021 Third Quarter CC
- 509 patient AD 2b/3 top-line data expected in 2H 2022
- Restoration of complete housekeeping function in the body
- Anavex is continuing to pioneer approach of big data to leverage holistic genomic sequencing and biomarker. This increases chances of success in our trials.
- AVATAR enrollment was exceeded and expected top-line in 2H 2021.
- Enough cash for beyond 2025 (157 mil)
- Q: what do you think of the RS-001 Rett trial patient size, what about AVATAR size?
- A: U.S. study was a small trial with low dose, but we garbed large effect sizes. In AVATAR, dosed are higher with larger patient population. We are expecting a good dose response curve and expect good data from AVATAR.
- Q: is the pediatric (EXCELLENCE) study being slowed down?
- A: we are still enrolling and can’t say for sure at this time how much impact new COVID restrictions will have in the long term. As of now, projections for trial completion are correct.
- Q: any further information IRT enrollment on AD phase 2b/3 into OLE?
- A: very impressed by very high rollover rate. Over 95% in fact. We have been informed that patients are reaching the end of the OLE, and are requesting additional time towards further extension.
- Q: is AVATAR study large enough? Any additional information?
- A: in dialogue with agency, have multiple FDA indications (orphan and fast track), also have voucher. Will update regulatory information when possible. Keeping exact high dose undisclosed at this time to prevent possible unblinking due to tight knit patient population.
- Q: baseline MMSE for AD 2b/3?
- A: patients above 20 MMSE did much better in the AD 2a. These patients are able to comply with trial regimen better, and efficacy is higher. Early Alzheimer’s is the highest unmet need for cognition today, and this just so happens to address that patient group.
- Q: preclinical information on blarcamesine and its protective role? Which indication may be addressed best for protection?
- A: in preclinical animal models, blarcamesine was able to completely prevent cognitive impairment as compared to untreated rodents. Blarcamesine is upstream and can prevent the cellular stress and therefore these negative effects. Earlier phase patients would likely respond better towards preventative medicine if caught early. Potential to treat even before symptoms are present, much like a mini/aspirin for cardiovascular problems. We will one day be able to tackle that and we are planning such a study.
- Q: Rett trials and applicability to Fragile X?
- A: ADAMS is a secondary score in Rett and is normally primary in Fragile X trials. It was overwhelmingly positive in the RS-001 Rett trial. Peer review paper expected soon. This is a great indicator towards success in the Fragile X trial - Fragile X being the largest need in autism spectrum disorder. Symptoms are very similar between the two disorders.
- Q: phase 3 prevention study status?
- A: need to discuss further with regulatory agency. No details at this time. Seems to elude to the trial including broad cognitive indications such as Alzheimer’s, Parkinson’s, and other CND disorders in a single trial. Need to discuss with agency before providing more detail.
- Q: any more about post-PDD trial?
- A: the trial itself has finished but analysis is ongoing. Working analysis other than primary and secondary endpoints. Intel on RNA and whole genome which is being put together right now and will create an ultimate package for regulators which will totally fuel our 2b/3 PDD and PD trials.
- Q: is the MJFF imaging study going well, any other details?
- A: starting it this year, will detail blarcamesine in the brain of PD patients.
- Q: any information on the undisclosed indication? Any info on the 3-71 trial?
- A: several animal models have been positive towards this undisclosed indication. Ultra rare disease. Before we move forward we want to make sure we are choosing the right indication as there are multiple available to choose from. 3-71 is in phase 1 now and data is expected this year. First trial will be FTD or a different indication. The 3-71 is a safety trial. (Missling leaves it open to suggestion that there could be more than just safety data. Perhaps he is referring to genome data). Anavex has been efficacious and has dose response curve in all trials. All trials have also had very strong predictive biomarker response.
Overall a very positive conference call. To me the most important points are related to the ADAMS test for Rett/Fragile X indications. In regards to effect size, ADAMS scored highest on the RS-001 trial with 1.31 very large effect size. This seems to indicate Fragile X will be a home run. Also, Missling confirmed that for now (pending regulatory guidance) the preventative trial is being planned to address CNS-wide indications (Alzheimer’s, PDD, etc.).
