Just some thoughts about today's financial report, conference call & items of interest. The conference call notes at the bottom do answer some of my initial questions.
"Planned initiation of a pivotal Phase 2/3 study in Fragile X Syndrome, the most frequent genetic cause of autism spectrum disorder."
According to globenewswire, FXS has a market size of about 33.8 million in the 7 major markets, with the U.S. having the largest portion. Market size is expected to accelerate dramatically through 2030.
I think it is awesome we are going straight to a phase 2/3 trial.
I think it is very interesting that Anavex went out of the way to add "the most frequent genetic cause of autism spectrum disorder". It seems to me that they are going to try to use the FXS trial as a catch-all for autism spectrum disorders which would cover a wide net of other sister disorders. This falls in line with comments from Dr. Randi Jenssen Hagerman during the Ladenburg Thalmann webinar and her continued mention from her upcoming 12 January Fragile X Spectrum webinar. This is very exciting, who knows how the FDA will play.
"Planned initiation of a Phase 2/3 clinical trial for the treatment of a new, rare-disease indication."
What a surprise! I have seen some mention of Batten Disease as a possibility and that does seem plausible after some posts I reviewed by our number 1 investigator (we all know who I'm talking about). Preclinical testing for Anavex compounds began in early 2020 and were conducted by Sanford (Kevin Francis). Dr. Francis said, “Maybe we could also use this as kind of a model project that we can apply to other rare diseases as well.” This is particularly interesting as Anavex has clearly made a push to initiate a vast number of rare disease trials, many being in later stages.
According to globenewswire, Batten Disease has a market size of about 40 million.
A question I have is "why is this undisclosed", is there potential this will be developed via a partnership? Or are they still nailing down a partial funding plan from an organization like they did with MJFF and RSAA. BDRSA and BBDF both seem likely candidates if Batten Disease is the correct assumption. I must admit, for personal reasons I wish they were doing this towards Angelman Syndrome, but that seems unlikely given the pipeline chart. I think Angelman could be prescribed off-label based on Rett outcomes anyways, they are very similar.
"Planned initiation of ANAVEX®2-73 imaging-focused Parkinson’s disease clinical study."
According to the pipeline chart (has been updated today), Anavex will be conducting two PD trials, one PD, and one PDD. The PD is brand new and likely the trial being partially funded by Shake It Up. To my understanding, an imaging focus will deviate from Anavex's current data collection schemas. An imaging focus will allow Anavex to visually show regulators a marked improvement in Parkinsons disease, which will increase the likelihood of approval. This is extremely interesting and comments towards this would be greatly appreciated.
Shares outstanding increased ~9 million from 48.9 to 58.2 million.
CONFERENCE CALL SPECIFIC NOTES:
• Based on U.S. Rett trial, plans to meet with FDA for accelerated pathway
• Pipeline expansion for 2-73: initiation of pivotal 2/3 for Fragile X and new 2/3 trial for undisclosed disorder
• First half 2021 for 3-71 phase 1 data
• November's PDD data was overwhelmingly positive.
• Patent application was approved and lasts until at least 2037. Covers range of diseases like Rett, Angelman, MS, and more.
• COVID-19 didn't slow down Anavex significantly.
• 80% Enrolled in AD trial.
• 47.6 million cash as of today, expected to last next 2 years. Raised around 20 million in 4th quarter. That's excellent as it should have reduced dilutive burden considering higher share price.
• What made AVATAR go pivotal? Anavex swapped the secondary efficacy endpoints to primary endpoints (RSBQ/CGI). AVATAR is on higher dose, so Missling believes the efficacy will be proven nicely. In regards to sample size, Missling does not believe they need to change the size. But they may increase the number of enrollment sites. Receiving further guidance from FDA in regards to the changing on sites. "Very strong effect size" currently. Missling wants to make this the final pivotal study for Rett.
• When will Rett data and PDD data be fully analyzed and released? Ongoing as Anavex is really building out the genome of the patients. Expect more details of PDD and Rett to come out in "early 2021".
• What is the undisclosed trial, is it a part of the new patent diseases? They didn't want to mention it because Anavex is in discussions with FDA about new preclinical data, and these discussions are strategic in nature (this trial vs. other potential, trial design). "Rare disease, maybe even ultra rare disease". This screams Batten Disease mentioned earlier, as Batten Disease is actually more rare than Rett Syndrome.
• When will first AD data be available? After last patient is enrolled add 48 weeks. Expect data sometime after that. Early 2022.
• Extra money post September, where did it come from? Utilized purchase agreement to take advantage of larger share price. Won't need to use again in large quantities. Could this be because of expected sales of Rett?
• Imaging focused PD study, can you explain? Want to explore the physical aspect more heavily, hence the PD-only branch.
• Why is Anavex having issues recruiting in Australian AD study? Started in Australia but it is an international study. Want to ensure the 'international' nature of the trial is captured as there are many "advantages" to having an international study (likely multi-approval/virtual world-wide approval).
• Interim AD data? Option is there, but Missling thinks data can be misleading as seen in other companies. They MAY still exercise this option.
• AVATAR 2/3 trial adult vs. child? Adult population has not received much attention, most trials have been towards pediatric due to higher chances of effect in younger brains. Have seen very strong response in adults - highly favorable. Missling would like to capture entire market. EXCELLENCE study is currently enrolling.
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