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 and Fragile X syndrome.
Lead Product(s): Cyclo-Glycyl-proline Analog
Therapeutic Area: Genetic Disease Product Name: NNZ-2591
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Acadia Pharmaceuticals
Deal Size: $463.0 million Upfront Cash: $100.0 million
Deal Type: Licensing Agreement July 13, 2023
Details:
Scenesse® (afamelanotide) belongs to the family of melanocortins, hormones, which activates human pigmentation, reduce oxidative damage, inflammation and swelling, and optimise the response of skin cells to UV-induced damage. It is being investigated for XP disorder.
Lead Product(s): Afamelanotide
Therapeutic Area: Genetic Disease Product Name: Scenesse
Highest Development Status: Phase II Product Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 16, 2023
Details:
Three of the four subjects included in Paradigm's open-label phase 2 trial of pentosan polysulfate sodium (PPS) in MPS-I have completed the 48-week study, with a 6-month treatment extension available.
Lead Product(s): Pentosan Polysulfate Sodium
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 12, 2022
Details:
ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4. Antisense inhibition of VLA-4 expression has demonstrated activity in animal models of inflammatory disease. ATL1102 has also shown to be very effective in reducing inflammatory brain lesions in patients with MS.
Lead Product(s): ATL1102
Therapeutic Area: Genetic Disease Product Name: ATL1102
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 09, 2022
Details:
The poster presentation includes the recently reported data from the Phase II clinical trial of the Company’s immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD) where ATL1102 met the primary endpoint.
Lead Product(s): ATL1102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 11, 2020
Details:
Primary endpoint met with confirmation of drug’s safety and tolerability. Strong effects on secondary endpoints on activity markers and disease progression along with improvement or stabilisation across different measures of motor function & strength were noted.
Lead Product(s): ATL1102
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 21, 2020