NTI 15.5% 4.9¢ neurotech international limited

It is a possibility, but Acadia also have the rights for...

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    It is a possibility, but Acadia also have the rights for NNZ-2591 in Fragile X and Rett Syndrome. This is on their pipeline as "ACP-2591"

    By all accounts it is a better compound but with a similar mechanism of action. The improvements are bioavailability (ability to penetrate the blood/brain barrier), lower manufacturing costs, and less side effects including no serious GI issues reported like with Trofinetide.

    It seems very likely they will develop 2591 to overlap Daybue patent expiry or when there is competition on the horizon, ie "Daybue 2.0".

    That being said a combination with Daybue or NNZ-2591 may be beneficial so would be worth considering. I would imagine NTI-164 could possible further reduce neuroinflammation whilst Daybue/NNZ-2591 are working to restore microglia function and regulate IGF-1 levels. Interestingly enough NNZ-2591 has been shown to be state dependant so it increases IGF-1 or decreases it - depending on the syndrome.

    https://hotcopper.com.au/data/attachments/6340/6340856-e8d7cdb72a02180a1c6c437ed1b12a1f.jpg
    https://hotcopper.com.au/data/attachments/6340/6340860-cc1590d730564405d1dc62cabf3d8794.jpg




    There has been more research released on the "real world" experience with Daybue outside of clinical trials. It is an interesting read because it shows how it was important to increase dosage steadily whereas the clinical trials were full dose which lead to higher side effects.


    "The patient received trofinetide in November 2021 as a participant of the DAFFODIL study at 3 years and 9 months of age. The patient had no relevant comorbidities and received physical, speech and occupational therapies. She also had aqua therapy and hippo therapy depending on the time of the year. Her Clinical Global Impression–Severity (CGI-S) score was 4, indicating that she was moderately ill. The initial dose of trofinetide was 10 ml (2 g) twice a day (BID) and was increased to 30 ml (6 g) BID by December 2021. Improved alertness and faster response times were reported within 2 months of trofinetide treatment. By May 2023, at the end of her trial participation, the patient experienced improved verbal language skills, acquiring up to 44 words that also included a handful of short phrases (i.e., “I'm done,” “I'm happy”, “already ate” and “I did it”). In addition to her verbal improvement, she also had improvement in using her augmentative communication device with longer phrases and faster production of communication. Furthermore, the patient’s gait and gross motor skills improved, and she is now able to walk faster and climb stairs and playground equipment. Finally, the patient also had mild improvement in fine motor skills. She was able to bring loaded utensils to her mouth and started using adaptive crayons. Her occupational therapist also documented improvement on timed therapy tasks. Diarrhea with trofinetide treatment was intermittent and often happened with antibiotic treatment for infections or while traveling. The caregivers reported that the incidence of diarrhea was manageable, and it did not lead to treatment discontinuation or dose reductions during the trial. This patient had no changes in appetite or significant vomiting. At the end of the trial, her CGI-I score was 1, indicating that her symptoms of RTT very much improved."

    Full article: Improvement in symptoms of Rett syndrome with trofinetide beyond clinical trial efficacy assessments: case reports (tandfonline.com)
 
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