10th Annual SVB Leerink Global Healthcare Conference:
- All compounds protected worldwide until at least 2030-2039 without patent extensions
- 2:45min: Rett syndrome annual sales are projected to be enough to support development of a full fledged pharmaceutical company (Missilng wants to go alone)
- 4:14min: not the end of the story, these are not all the indications we can treat. Plans to move into Fragile X (5x larger market than Rett Syndrome).
- 4:40min: PDD is a phase 2/3 pivotal study, and POTENTIALLY PD will be pivotal as well (that's new info)
- U.S. Rett Syndrome phase 2 study and full PDD data due late 1st quarter or early 2nd quarter
- 8:00min: Missling explains the MOA. SIGMA1R activation is downstream beneficial with inflammation and tau, but also upstream at the nucleus. It prevents cellular stress before and after RNA interference/disturbance. It does this through chromatin remodeling by preventing toxic RNA expressing into toxic proteins. This explains broad therapeutic approach.
- 9:35min: SIGMAR1R activation is critical, and is going to be further confirmed in a soon-to-be published peer-review journal (PDD)
- 10:37min: Missling mentions Anavex 2-73s benefit in autophagy - the only way cells regenerate themselves
- Very low dose in adult Rett study had very pleasing outcome - this is great because it is harder to find signal in adults
- ALL RNA and DNA are analyzed from ALL patients in ALL studies - allows for faster and more robust future studies
- FDA insists on cognitive and daily living for AD studies. Swapped from MMSE to ADAS-COG in the 2/3.
- SIGMA1 Gene and COMT gene exclusion = much stronger signal. All patients vs placebo, and all WT vs. non-WT will be shown. Expect heavy correlation.
- PDD is a tough indication, three failures recently across the industry. PDD has multiple pathologies which makes it so hard to treat. Our data is intriguing because we had a strong signal across the patients. All domains will be readout in the upcoming data release. Dose-dependent improvement in PDD, 30mg stopped decline, 50 mg improved patient. The 50mg patients "came out of the study with less dementia". 70% of this data has direct correlation with ADAS-COG (AD primary endpoint). REM was also improved and so was digital vigilance and episodic memory. No safety issues.
- Anavex is targeting largest markets. Please note the $277B on the slide is ECONOMIC BURDEN, this is NOT market size. Market size is smaller than economic burden.
- Q. What is the bar for good data in the pediatric study and also for the second adult study? Rett study data to date has been clinically meaningful in the low dose adult study, data in higher dose study will likely be greater which is incredible considering they already have clinically meaningful result (effect size was 1.1-1.2 which is very high).
- Q. Acadia drug, what thoughts do you have on that product? Their published data shows mild effect in the higher dose (in pediatric). Our data shows vastly superior data so far even in low dose. Safety is somewhat lesser as well.
- Q. SIGMA1 gene variant, can you talk about that more? The studies we have done have ALL showed patients responding to the drug, but a few patients have a gene that results in lesser efficacy. This gene is found in 10-20% of the population. These patients do still respond however, and may just need more drug or longer dosing period, or both.
- Q. Biogen drug, what do you think of their data/approval chances? Approval of Adu would be positive for the industry because it shows that a signal clinical study is sufficient for approval. However, if the drug is not approved we will still continue as planned.
AI
