Very interesting and clever choice that neatly sidesteps some of the potential drawbacks to the current monogenic neurodevelopmental disorder indications.
HIE qualifies as an orphan disease but is relatively high prevalence. For example, if 2 to 3 newborns each year in the US suffer from HIE, this suggests 7,000 - 11,000 new patients each year in the US. If also indicated as a chronic therapy for existing HIE patients, the numbers become very large.
Whilst classified as a neurodevelopmental disorder, HIE is not gene-specific. Hence, one avoids the inevitable competition that comes in this space from gene-targeted antisense and gene therapies.
Also, as HIE is a non-gene-specific neurodevelopmental disorder, one doesn’t rely upon quality/expensive genetic testing for diagnosis. This reduces the challenge of patient identification for trial recruitment, potential market and reimbursement.
Choice of HIE as a clinical program adds substantially to the interest and value of NNZ-2591 as a therapeutic asset, by highlighting the drug’s potential beyond monogenic paediatric neurodevelopmental disorders to much broader indications triggered by injury to the brain that are of interest to big pharma. These include conditions such as concussion, CTE, TBI and stroke.
NEU Price at posting:
$12.19 Sentiment: Hold Disclosure: Held
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