I met a PhD who does Alzheimer's research for a firm that consults for a few big pharmas, and her area includes amyloid beta oligomers. Most folks here know so little about the disease and pharma and assume that there are prejudicial camps in some kind of war of ideas. In reality, people like my acquaintance are scientists and they understand the complexity of AD. She's spent years learning what role AB plaques play in it. She explains incredibly complicated cellullar cascades, what is known about them, what is not known, the many theories, the failures, the progress. She explains that AB plaque removal is not a cure, even though this is her research area. The difference between serious scientists doing important work and unserious lay people speculating on a pet stock and seeing conspiracies.
Through me, she's familiar with the S1 receptor and Anavex, and thinks our approach might be therapeutic. She thinks other approaches might be as well, and that combo therapies will ultimately prove necessary. She explains that progress was made with the plaque and tangle MABs, and is optimistic that more progress lies ahead, in her area, our area, and others. Speaking with her, you understand how Anavex can be a worthwhile risk as a small investment. Other biotechs, too. She does not invest in any.
Her initial impression from our CTAD materials was that it wasn't "something a reputable biotech or pharma would release at a conference". I told her about our upcoming paper and EMA application and she said a paper is much needed, but with regulators she cautioned that our trial was still "really small" for AD.
I think Missling knows we have an uphill challenge in AD. He's laid out our regulatory approach, appealing to our: being a safe cheap pill, meeting the newly lowered endpoint guidelines, and having (albeit unorthodox) biomarker support. If there are no bad surprises in our paper, which will give us a clearer view into our little trial's outcomes, I think we have a chance at some contingent approvals. My biggest fear: what Missling called "ITT" is really an mITT that gives regulators pause, given our already small n. Fingers crossed there was no 'spin'.
