I was honored to have been invited to represent the Fragile X community and the NFXF at the recent meeting sponsored by Neuren Pharmaceuticals, in which they shared some very encouraging news regarding their Phase 2 clinical trial.

From my perspective, it’s encouraging to know that there’s a pharmaceutical company still working in FX, the drug is successfully progressing through the typical stages required for FDA approval and we are all hopeful that the news remains positive.

For more details, please read the press release issued by Neuren. The announcement is lengthy because it’s designed for multiple audiences (including shareholders), but the first page does a good job summarizing the results.

Best regards,

Tony Ferlenda

Neuren Pharmaceuticals today announced top-line results from its Phase 2 clinical trial in Fragile X syndrome. The trial has successfully established proof of concept and provides a strong rationale for Neuren to move forward with developing trofinetide for FXS. In this initial small trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement, observed in both clinician and caregiver assessments.

After only 28 days of treatment, improvements were seen across core symptoms of Fragile X syndrome, including higher sensory tolerance, reduced anxiety, better self-regulation and more social engagement. Beneficial effects of trofinetide have now been observed in two different neurodevelopmental disorders, Fragile X syndrome and Rett syndrome. This is consistent with known actions of trofinetide, expected to normalize a number of biological processes in the brain that are impacted by each syndrome.

Based on these results and feedback from clinical experts in Fragile X syndrome, Neuren is strongly encouraged to advance to the next step in clinical development. This will likely involve a study in 1 of 6 younger children with Fragile X syndrome and may examine a longer treatment duration with higher doses. This next study will also refine the outcome measures that may be used in a Phase 3 study. Neuren intends to discuss the trial results and drug development plan with the US Food and Drug Administration (FDA) in early 2016.

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