NMOSDRight now it is very hard for the market to value the NMOSD program. It's a preclinical program and preclinical assets are very uncertain with potential very risky timelines and cash burns. The biotech market also hasn't been favorable for early-stage assets with many trading below cash. That can change with a potential fall in interest rates in the US which appears before us. There is also no regulatory path forward to clinical trials for the asset. A recent company filing said a regulatory meeting has been scheduled in the near term. What if US interest rates are cut and a clear regulatory path forward for the asset is established? A regulatory filing also says: "
Our pre-clinical plans include expanding the already available proof-of-concept data for the treatment of NMOSD in both in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate; and manufacturing process development for the selected candidate." With the merger completed, an update could be interesting as these have been the next stated objectives for some time as shown in the July excerpt below from last year.
From AEZS's October 25, 2023 presentation: Beyond proof in animal models AEZS has also experimented with human blood and can see the mode of action(stimulation of and recruitment of regulatory t-cells; suppression of cytotoxic t-cells) in healthy doners and those with the disease(NSMOD, Parkinson's). This human data is supportive of potential partnering. Massachusetts General Hospital, the original and largest teaching hospital of Harvard, has been involved with the past NSMOD projects and is also experimenting with their specific models. They expected to report their findings in the very near future according to an older presentation and I don't know if that data was released. -------- AEZS has said that its most exciting pipeline program is probably AIM Biologicals. The lead product candidate(NMOSD) alone targets a US$2.5 billion market. What could this program be worth if successfully derisked to the point of human clinical trials and a clear regulatory path forward? A diagnostic deal could also provide upfront cash to support thee program. AEZS has spent the last several years building the preclinical proof for this program and we could soon find out its Phase I value. It is already believed to have an excellent tolerability profile based on the science. That substantially leaves efficacy risk as CSCI targets a large market. Does it work? It is based on a 'program' already approved by nature, in a sense. The immune system of expecting mothers doesn't reject a fetus despite 50% of the fetus being foreign protein from the father. The expecting mother is said to have feto-maternal tolerance. AEZS believes it can make the human immune system desensitized to highly specific antigens. Even auto-immune diseases can subside during pregnancy. AEZS has tested its potential breakthough approach in multiple in animal and other models across more than one disease. It appears to work across these different models and indications. The last update in July 2023: "Recent Highlights
- Demonstrated positive pre-clinical proof-of-concept in various in-vitro and in-vivo models.
- In-depth profiling of a limited set of candidates for potential use in clinical development.
- Confirmed AIM Biologicals’ mechanism of action and efficacy in ex-vivo studies in human blood samples from both NMOSD and PD patients.
Next Steps
- Evaluation of manufacturing strategy and initiation of production of selected development candidates.
- Ongoing compilation of a comprehensive pre-clinical data package for scientific advice meetings with regulatory authorities, expected to take place in the near future."
They believe they know how the immune system does it from testing in human blood of subjects; including those with the disease. They have developed a rigorous proof for this program from tolerability to expected efficacy across multiple models and for more than one disease. “Our continued progress advancing our development and regulatory strategies are evolving and designed to provide us with a pathway towards our first-in-human clinical studies with our lead indication, neuromyelitis optica spectrum disorder (NMOSD)." August 2023
Will AEZS soon announce that it has nailed down a clinical trial candidate for its lead AIM Biologicals program and is ready to initiate human clinical trials. What will the clinical trial program look like and how soon to key human data establishing proof of principle in humans. They have already tested the blood of human patients and believe they know the mechanism-of-action and expect it to be well tolerated based on the science. What could this lead program targeting a US$2.5 billion market be worth in Phase I with a rigorous proof? Importantly this is a potential platform technology that could be used to treat other diseases beyond the initial US$2.5 billion opportunity. Other approaches target immune suppression which can make the patient susceptable to infectious diseases or cancer. The treatments are given over a lifetime.