Drugs / NNZ-2591
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT04379869 | May 2020 → Feb 2021 | — | Neuren Pharmaceuticals Limited | Completed | No outcome recorded |
| Phase 24 trials · 2 met primary | ||||||
| Phase 2 | NCT05879614 | Sep 2023 → Jun 2025 | Prader-Willi syndrome | Neuren Pharmaceuticals Limited | Withdrawn | No outcome recorded Stop: Business |
| Phase 2 | NCT05025332 | Oct 2022 → Feb 2024 | Pitt-Hopkins syndrome | Neuren Pharmaceuticals Limited | Completed | Met primary |
| Phase 2 | NCT05025241 | Aug 2022 → Nov 2023 | Phelan-McDermid syndrome | Neuren Pharmaceuticals Limited | Completed | Met primary |
| Phase 2 | NCT05011851 | Jul 2022 → May 2024 | Angelman syndrome | Neuren Pharmaceuticals Limited | Completed | No outcome recorded |
| Phase 32 trials | ||||||
| Phase 3 | NCT07593391 | Jun 2026 → Oct 2028 expected | Phelan-McDermid syndrome | Neuren Pharmaceuticals Limited | Recruiting | No outcome recorded |
| Phase 3 | NCT07281079 | Nov 2025 → Oct 2027 expected | Phelan-McDermid syndrome | Neuren Pharmaceuticals Limited | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 44
| Date | Issuer | Release |
|---|---|---|
| 2026-02-04 | Neuren Pharmaceuticals Limited | Regulatory Priority Review Voucher program reauthorized by US Congress neurenpharma.com ↗
Neuren (NEU) – ASX Announcement 5 February 2026 |
| 2026-01-06 | Neuren Pharmaceuticals Limited | Results NNZ-2591 PMS Phase 2 trial published in Neurology Genetics neurenpharma.com ↗
We are pleased that Neurology® Genetics has published our Phase 2 trial results in PMS, which provided the strong scientific basis to further investigate NNZ-2591 as a potential treatment for PMS in our ongoing Koala Phase 3 study. |
| 2025-12-21 | Neuren Pharmaceuticals Limited | Regulatory Health Canada approves Clinical Trial Application for Koala neurenpharma.com ↗
Health Canada has approved Neuren’s Clinical Trial Application (CTA) for the Koala Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome |
| 2025-12-08 | Neuren Pharmaceuticals Limited | Regulatory One-month administrative delay to FDA meeting for HIE neurenpharma.com ↗ |
| 2025-11-09 | Neuren Pharmaceuticals Limited | Results Investor Presentation, 10 November 2025 neurenpharma.com ↗
16/18 subjects showed improvement |
| 2025-10-19 | Neuren Pharmaceuticals Limited | Regulatory Fast Track granted by FDA for NNZ-2591 in Phelan-McDermid neurenpharma.com ↗ |
| 2025-02-23 | Neuren Pharmaceuticals Limited | Regulatory Neuren receives Rare Pediatric Disease Designation from FDA neurenpharma.com ↗ |
| 2025-02-19 | Neuren Pharmaceuticals Limited | Regulatory Fast Track granted by FDA for NNZ-2591 in Pitt Hopkins neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for NNZ-2591 for the treatment of Pitt Hopkins syndrome (PTHS). |
| 2024-08-09 | Neuren Pharmaceuticals Limited | Results NNZ-2591 Angelman top-line results presentation neurenpharma.com ↗
NNZ-2591 Angelman syndrome Phase 2 trial top-line results |
| 2024-08-09 | Neuren Pharmaceuticals Limited | Results Phase 2 trial shows significant improvements in Angelman neurenpharma.com ↗
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). |
| 2024-07-18 | Neuren Pharmaceuticals Limited | Regulatory Neuren receives Rare Pediatric Disease Designation from FDA neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has received Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for NNZ-2591 in Phelan-McDermid syndrome. |
| 2024-05-27 | Neuren Pharmaceuticals Limited | Results Investor presentation, 27 May 2024 neurenpharma.com ↗
NNZ-2591 Pitt Hopkins syndrome Phase 2 trial top-line results |
| 2024-05-26 | Neuren Pharmaceuticals Limited | Results Phase 2 trial shows significant improvements in Pitt Hopkins neurenpharma.com ↗
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Pitt Hopkins syndrome (PTHS). |
| 2024-01-31 | Neuren Pharmaceuticals Limited | Results Q4 2023 quarterly activity report neurenpharma.com ↗ |
| 2023-12-17 | Neuren Pharmaceuticals Limited | Results Investor presentation, 18 December 2023 neurenpharma.com ↗
NNZ-2591 Phelan-McDermid syndrome Phase 2 trial top-line results |
| 2023-12-17 | Neuren Pharmaceuticals Limited | Results P2 trial shows significant improvements in Phelan-McDermid neurenpharma.com ↗
Melbourne, Australia: Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome (PMS). |
| 2023-01-22 | Neuren Pharmaceuticals Limited | Regulatory Prader-Willi syndrome IND for NNZ-2591 approved by FDA neurenpharma.com ↗ |
| 2022-12-22 | Neuren Pharmaceuticals Limited | Regulatory Neuren submits IND & first patients complete Phase 2 trials neurenpharma.com ↗
The new IND application was submitted to the US Food and Drug Administration (FDA) on 22 December for approval to proceed with a Phase 2 trial in Prader-Willi syndrome. |
| 2022-03-24 | Neuren Pharmaceuticals Limited | Regulatory FDA approval for Pitt Hopkins syndrome IND and Phase 2 trial neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has received approval from the US Food and Drug Administration (FDA) to proceed with the Phase 2 trial of NNZ-2591 in Pitt Hopkins syndrome (PTHS). |
| 2022-03-22 | Neuren Pharmaceuticals Limited | Regulatory FDA approval for Phelan-McDermid IND and Phase 2 trial neurenpharma.com ↗ |
| 2022-03-10 | Neuren Pharmaceuticals Limited | Regulatory Ethics approval for Angelman Phase 2 trial in Australia neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has received approval from the Human Research Ethics Committee for its Phase 2 clinical trial of NNZ-2591 in Angelman syndrome (AS). |
| 2022-02-24 | Neuren Pharmaceuticals Limited | Regulatory FDA approval for Angelman IND and Phase 2 trial neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has received approval from the US Food and Drug Administration (FDA) to proceed with the Phase 2 trial of NNZ-2591 in Angelman syndrome (AS). |
| 2021-10-21 | Neuren Pharmaceuticals Limited | Regulatory Neuren receives feedback on IND for Phelan-McDermid syndrome neurenpharma.com ↗
Consistent with the feedback on the similar application for Angelman syndrome, which was reported by Neuren on 1 October, the IND has been placed on hold, with a formal hold letter to be issued by the FDA within the next 30 days. |
| 2021-10-01 | Neuren Pharmaceuticals Limited | Regulatory Feedback on Angelman IND and submission of Pitt Hopkins IND neurenpharma.com ↗
The IND has been placed on Clinical Hold until the changes to the protocol are agreed with the FDA. |
| 2021-09-20 | Neuren Pharmaceuticals Limited | Regulatory IND application submitted for NNZ-2591 in Phelan-McDermid neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for NNZ-2591 to treat Phelan-McDermid syndrome (PMS). |
| 2021-09-03 | Neuren Pharmaceuticals Limited | Regulatory FDA grants Orphan Drug designation for Prader-Willi syndrome neurenpharma.com ↗
Neuren (NEU) – ASX announcement 3 September 2021 FDA grants Orphan Drug Designation for NNZ-2591 to treat Prader-Willi syndrome |
| 2021-08-31 | Neuren Pharmaceuticals Limited | Regulatory Angelman IND and Ethics applications submitted for NNZ-2591 neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for NNZ-2591 to treat Angelman syndrome. |
| 2021-02-14 | Neuren Pharmaceuticals Limited | Results Successful Phase 1 trial for Neuren's NNZ-2591 neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today reported that in its Phase 1 trial twice daily oral dosing of NNZ-2591 for seven days was safe and well tolerated in healthy volunteers. |
| 2021-01-10 | Neuren Pharmaceuticals Limited | Regulatory European Commission grants 3 NNZ-2591 Orphan designations neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) received notice that the European Commission has adopted the European Medicines Agency (EMA) recommendations to grant Orphan designation for NNZ-2591 in Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome. |
| 2020-12-06 | Neuren Pharmaceuticals Limited | Regulatory Positive opinion for all 3 Orphan applications in Europe neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) has received notice from the European Medicines Agency (EMA) of positive opinions for all three Orphan designation applications that were submitted for NNZ-2591 in Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome. |
| 2020-11-18 | Neuren Pharmaceuticals Limited | Regulatory Approval received for final stage of NNZ-2591 clinical trial neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that it has received Ethics Committee approval to move forward with the final stage of its first clinical trial of NNZ-2591. |
| 2020-09-28 | Neuren Pharmaceuticals Limited | Regulatory Neuren requests Orphan designations for NNZ-2591 in Europe neurenpharma.com ↗ |
| 2020-03-16 | Neuren Pharmaceuticals Limited | Regulatory Neuren to capture substantially greater value after Phase 3 neurenpharma.com ↗
FDA granted Orphan Drug designation in each of Phelan-McDermid, Angelman and Pitt Hopkins syndromes |
| 2020-03-09 | Neuren Pharmaceuticals Limited | Results Additional information neurenpharma.com ↗ |
| 2020-03-05 | Neuren Pharmaceuticals Limited | Results Compelling results for 2591 dose ranging study in PMS model neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today reported compelling results from a dose ranging study of NNZ-2591 in a model of Phelan-McDermid syndrome (PMS), which will be used to inform dose selection for Neuren’s planned clinical trials in patients. |
| 2019-10-15 | Neuren Pharmaceuticals Limited | Regulatory FDA grants Orphan for both Phelan-McDermid and Pitt Hopkins neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Neuren’s NNZ-2591 for the treatment of each of Phelan-McDermid syndrome and Pitt Hopkins syndrome. |
| 2019-10-11 | Neuren Pharmaceuticals Limited | Regulatory FDA grants Orphan Drug Designation for NNZ-2591 in Angelman neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to Neuren’s NNZ-2591 for the treatment of Angelman syndrome. |
| 2019-08-15 | Neuren Pharmaceuticals Limited | Regulatory New patent granted for Neuren's NNZ-2591 in Japan neurenpharma.com ↗ |
| 2019-07-28 | Neuren Pharmaceuticals Limited | Regulatory Neuren submits 3 NNZ-2591 Orphan Drug applications to FDA neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) announced today that it has submitted 3 applications for Orphan Drug designation to the US Food and Drug Administration. |
| 2019-05-16 | Neuren Pharmaceuticals Limited | Results NNZ-2591 positive effects in Pitt Hopkins syndrome model neurenpharma.com ↗ |
| 2019-05-16 | Neuren Pharmaceuticals Limited | Results NNZ-2591 positive effects in Angelman syndrome model neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that treatment with its drug candidate NNZ-2591 has normalized all deficits in a pre-clinical model of Angelman syndrome (AS). |
| 2019-05-16 | Neuren Pharmaceuticals Limited | Results Validation for NNZ-2591 neurodevelopmental disorder pipeline neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) announced earlier today that NNZ-2591 has demonstrated positive results in pre-clinical models of two additional neurodevelopmental disorders – Angelman syndrome and Pitt Hopkins syndrome. |
| 2019-02-17 | Neuren Pharmaceuticals Limited | Results NNZ-2591 positive effects in Phelan-McDermid syndrome model neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that its drug candidate NNZ-2591 has demonstrated positive effects in a pre-clinical model of Phelan-McDermid syndrome (PMS). |
| 2015-11-29 | Neuren Pharmaceuticals Limited | Regulatory Grant of composition patent in Europe for Neuren's NNZ-2591 neurenpharma.com ↗
Neuren Pharmaceuticals (ASX: NEU) today announced that the European Patent Office has confirmed the grant of a new patent covering the composition of Neuren’s drug candidate NNZ-2591. |
All press releases naming this drug 62 releases
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | NNZ-2591 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07281079 ↗ |
| Known as | Cyclo(-l-glycyl-l-2-allylproline) | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802144 ↗ |
| Modality | Small molecule | “NNZ-2591, a synthetic analog of the insulin-like growth factor 1 metabolite cyclic glycine-proline” PMID 41450730 ↗ Dec 2025 |
| Route | Oral | “oral NNZ-2591” NCT04379869 ↗ |