NNZ-2591 FOR MULTIPLE NEURODEVELOPMENTAL DISORDERS Neuren is developing NNZ-2591 for multiple serious neurodevelopmental disorders with different genetic origins that emerge in early childhood and have no or limited approved treatment options. The estimated number of potential patients being targeted across these disorders is more than five times larger than Rett syndrome. Neuren’s programs for Phelan-McDermid syndrome (PMS), Pitt Hopkins syndrome (PTHS), Angelman syndrome (AS) and Prader-Willi syndrome (PWS) have all been granted Orphan Drug designation by the FDA and are being developed under Investigational New Drug (IND) applications. In designing and executing the NNZ-2591 development program, Neuren has been able to leverage the extensive and highly relevant experience the Neuren team has gained from the trofinetide Rett syndrome program across manufacturing, non-clinical, clinical and regulatory. The FDA has also granted Rare Pediatric disease Designation for NNZ-2591 in each of PMS, PTHS and AS. With this designation in place, Neuren may be awarded a PRV if the Rare Pediatric Disease PRV program is reauthorized by the US Congress and NNZ-2591 receives marketing authorisation for any of these indications by the FDA. The Rare Pediatric Disease PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor. As noted above, Neuren’s partner Acadia received a PRV on marketing authorization of DAYBUE in Rett syndrome and sold the PRV for US$150 million. OPERATING REVIEW CONTINUED Successful Phase 2 clinical trials across three syndromes In May 2024, Neuren announced positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with PTHS. After treatment for 13 weeks, 9 out of 11 children showed improvement assessed by clinicians and significant improvement was observed by both clinicians and caregivers in clinically important aspects of PTHS, including communication, social interaction, cognition and motor abilities. NNZ-2591 was well tolerated and demonstrated a good safety profile. Neuren recently announced that the FDA has granted Fast Track designation for NNZ-2591 for the treatment of PTHS. Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious conditions. In August 2024, Neuren announced positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with AS. After treatment for 13 weeks, 11 out of 13 children showed improvement assessed by clinicians, with improvements seen in clinically important aspects of AS. In the 3-12 years age group all 8 children showed improvement. The positive results of NNZ-2591 in PTHS and AS followed the announcement of positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with PMS. Phase 2 trial results validating multi-indication platform 1 # ' )Ҋ -($ .4) -*( җ Ҙ ۙрчѶ рт 2 &. $// *+&$). .4) -*( җ Ҙ ۙррѶ рт 2 &. )" '( ) .4) -*( җ Ҙ ۙртѶ рт 2 &. ! /4 о /*' - $'$/4 ! ) 2 '' /*' - / Ѷ 2$/# )* ( )$)"!0' /- ) . $) ' *- /*-4 1 '0 . *- */# - . ! /4 + - ( / -. 0-$)" /- /( )/ Efficacy All 3-12 years '$)$ $ ) '* ' (+- ..$*) *! (+-*1 ( )/ ( ) . *- җ ڿ *! + /$ )/. $(+-*1 Ҙ сѵу җчш ڿ Ҙ сѵх җчс ڿ Ҙ тѵп җчф ڿ Ҙ 2.8 (100%) - "$1 - (+- ..$*) *! # )" ( ) . *- җ ڿ *! + /$ )/. $(+-*1 Ҙ сѵц җчт ڿ Ҙ тѵп җцт ڿ Ҙ тѵс җхц ڿ Ҙ 2.6 (100%) ҿ + /$ )/. 2$/# '$)$ ' '* ' (+- ..$*) *! 1 -$/4 $(+-*1 ( )/ җ ڿ *! + /$ )/.Ҙ ц җтш ڿ Ҙ х җфф ڿ Ҙ у җтр ڿ Ҙ *).$./ )/ $(+-*1 ( )/ $) '$)$ ''4 $(+*-/ )/ .+ /. *((0)$ /$*)Ѷ # 1$*-Ѷ *")$/$*)Ѷ .* $ ' *((0)$ /$*)Ѷ .* $ 'Ѷ *")$/$*)Ѷ (*/*- *((0)$ /$*)Ѷ # 1$*-Ѷ *")$/$*)Ѷ (*/*- Phase 2 trial results validating multi-indication platform Neuren Pharmaceuticals Limited Annual Report 2024 9
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