PDE7

inhibitor for

Fragile X syndrome, Angelman

Syndrome and related rare

neurodevelopmental disorders

Why PDE7?

Cyclic AMP signalling pathway relevant to neuronal and synaptic function.

Data room contents

Mechanistic rationale · in vitro pharmacology · selectivity · PK /brain exposure · behavioural efficacy · tolerability · IP position · proposed development plan

Why partner now?

Confidential package available for licensing

PDE7

inhibitors do not cause the emetic effects (nausea and vomiting) that limit other similar drugs

Target

PDE7

Modality

Small molecule

Stage

Advanced preclinical

Status

Available

Lead indication
  • Fragile X syndrome
  • Angelman Syndrome
  • Neurofibromatosis (NF1)

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