pp-060 Tablets

Indication: Autism spectrum disorder (ASD), including pediatric patients with social-communication deficits and neurobehavioral symptoms; additional evaluation in neurological conditions characterized by lysosomal dysfunction, mitochondrial stress, and dopaminergic pathway abnormalities. Clinical stage: Phase 2 clinical development in pediatric ASD, supported by completed Phase 1 PK/PD modeling and randomized placebo-controlled studies. Modality: Small molecule (essential amino acid formulation) Mechanism / Target: Pharmaceutical-grade L-serine supporting neuronal metabolism; modulates mitochondrial function, reduces oxidative and lysosomal stress, restores cathepsin activity, and improves dopaminergic neuronal signaling—mechanisms implicated in ASD pathophysiology. Route / form: Oral administration (dose-titrated formulations used in clinical studies) Differentiation: First-in-class neurometabolic therapy targeting cellular stress pathways in ASD; demonstrated improvements in social communication in clinical trials and robust neuroprotective effects in multiple preclinical models.