
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Neuren Phase 2 trial shows significant improvements in Pitt Hopkins syndrome
Details : Neuren lead product candidate NNZ-2591 is being evaluated in the mid-stage clinical trial studies for the treatment of patients suffering from Pitt Hopkins syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 27, 2024
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Acadia Pharmaceuticals
Deal Size : $463.0 million
Deal Type : Licensing Agreement
Details : Under the agreement, Acadia will acquire ex-North American rights to the drug as well as global rights in Rett syndrome and Fragile X syndrome to Neuren’s development candidate NNZ-2591, an investigational synthetic analogue of cGP, for Rett syndrome a...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : $100.0 million
July 13, 2023
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Acadia Pharmaceuticals
Deal Size : $463.0 million
Deal Type : Licensing Agreement

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Prader-Willi Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 30, 2023
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Phelan-McDermid Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
August 27, 2021
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)
Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Pitt-Hopkins syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
August 27, 2021
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Angelman Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
August 18, 2021
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Positive FDA Meetings Enable Neuren to Proceed with INDs for Three Phase 2 Trials
Details : NNZ-2591 has ideal attributes – a unique mechanism of action, consistent and compelling results in the model of each disorder, a clearly identified optimum dose and proprietary manufacturing with high purity and yield.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
June 01, 2021
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Undisclosed
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
To Evaluate the Safety, Tolerability and Pharmacokinetics of Oral NNZ-2591 in Healthy Volunteers
Details : NNZ-2591 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of undefined medical condition.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 08, 2020
Lead Product(s) : Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-
Therapeutic Area : Undisclosed
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II
Sponsor : International Rett Syndrome Foundation
Deal Size : Inapplicable
Deal Type : Inapplicable
A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
Details : NNZ-2566 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Rett Syndrome.
Product Name : Undisclosed
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
March 22, 2016
Lead Product(s) : Trofinetide
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II
Sponsor : International Rett Syndrome Foundation
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Trofinetide
Therapeutic Area : Trauma (Emergency, Injury, Surgery)
Study Phase : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Safety and Efficacy Study of NNZ-2566 in Patients With Mild Traumatic Brain Injury (mTBI)
Details : NNZ-2566 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Brain Concussion.
Product Name : Undisclosed
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
March 31, 2014
Lead Product(s) : Trofinetide
Therapeutic Area : Trauma (Emergency, Injury, Surgery)
Highest Development Status : Phase II
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
