Market Mover | shares of Neuren Pharmaceuticals jump 11% on positive Phase 2 trial results
May 27, 2024 - Today, shares of $Neuren Pharmaceuticals Ltd(NEU.AU$ jumped 11.05% to reach A$23.00 after the company announced that the Phase 2 trial shows significant improvements in PittHopkins.
NNZ-2591 is a therapy designed by Neuren Pharmaceuticals to improve the lives of individuals affected by Pitt Hopkins syndrome, a neurodevelopmental disability. The Phase 2 trial results show that the improvements were seen in clinically important aspects of Pitt Hopkins syndrome, including communication, social interaction, cognition and motor abilities.
About Pitt Hopkins syndrome
Pitt Hopkins syndrome (PTHS) is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. The incidence of PTHs has been estimated at between 1 in 34,000 and 1 in 41,000 people. Characteristics of PTHs are a range of developmental delay with moderate-to-severe intellectual disability and behavioral differences, hyperventilation and/or breath holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features. Some individuals with PTHS are diagnosed with autism. Further information about PTHS is available at: Home - Pitt Hopkins Research Foundation
Pitt Hopkins syndrome (PTHS) is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. The incidence of PTHs has been estimated at between 1 in 34,000 and 1 in 41,000 people. Characteristics of PTHs are a range of developmental delay with moderate-to-severe intellectual disability and behavioral differences, hyperventilation and/or breath holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features. Some individuals with PTHS are diagnosed with autism. Further information about PTHS is available at: Home - Pitt Hopkins Research Foundation
About Neuren
Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug" designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases. DAYBUE" (trofinetide) is approved by the US food and Drug Administration (FDA) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc, for the development and commercialisation of trofinetide.
Neuren's second drug candidate, NNZ-2591, is in Phase 2 development for multiple neurodevelopmental disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid syndrome and Pitt Hopkins syndrome.
Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. Recognising the urgent unmet need, all programs have been granted “orphan drug" designation in the United States. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases. DAYBUE" (trofinetide) is approved by the US food and Drug Administration (FDA) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc, for the development and commercialisation of trofinetide.
Neuren's second drug candidate, NNZ-2591, is in Phase 2 development for multiple neurodevelopmental disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid syndrome and Pitt Hopkins syndrome.
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