STALICLA SA, today announced first patient enrolled in its Phase 1b clinical trial with STP1, a first in class precision medicine for patients within the autism spectrum disorder (ASD) Phenotype 1 subgroup (ASD-Phen1), a novel biological subgroup identified by STALICLA's proprietary discovery platform. STP1 is a novel combination of compounds: a pan-PDE inhibitor with anti-inflammatory properties and a modulator of NKCC1, specifically targeting the biological dysregulations linked to ASD-Phen1.
Autism spectrum disorder (ASD) is a group of neurodevelopmental disorders (NDDs) currently diagnosed based on core behavioral features, including persistent deficits in social communication and social interaction, restricted and repetitive patterns of behavior, interests, or activities. Significant impairment in daily functioning should also be present. Globally, ASD is highly prevalent and affects approximately 10 million patients in Europe and the United States and more throughout the world. There are no biological criteria for this diagnosis. ASD is characterized by high levels of clinical and genetic heterogeneity, supporting the existence of ASD biological subtypes requiring differential pharmacological treatment strategies.
By combining real-world patient data and its proprietary systems biology and AI-powered drug discovery platform called DEPI (Databased Endophenotyping Patient Identification), STALICLA is uncovering novel patterns of non-behavioral and biologically defined parameters amongst patients with ASD. STALICLA has already identified ASD Phenotype 1, a first subpopulation of patients with idiopathic ASD that can be identified by distinct multi-omics biomarkers. ASD Phenotype 1 is characterized by dysregulation of key intracellular pathways, transcription factors and immune factors. The most recent biomarker data related to ASD Phenotype 1 will be presented by STALICLA ahead of publication at the BRAIN Foundation Synchrony 2020 conference on December 12, 2020 (online).
"STALICLA is the first company developing precision medicine treatments for a biologically defined subgroup of patients with ASD. A diagnosis of autism must be integrated with other layers of data to capture biologically specific mechanisms, enabling the identification of precision drug candidates to improve on the outcome of clinical trials. STALICLA has built its DEPI drug discovery platform to achieve just this. By defining novel ASD phenotypes based on clinical signs and symptoms and not only on behaviors, STALICLA is creating a precision medicine approach to discovering and developing novel treatments for patients with ASD," said Lynn Durham, M.Sc. Chief Executive Officer and founder of STALICLA.
"This Phase 1b study for STP1 marks the first time that a precision therapeutic will be investigated in patients with a non-monogenetic cause of ASD. While the trial progresses, STALICLA will continue to drive forward its proprietary and partnered programs to define additional ASD Phenotypes and advance a robust pipeline of therapeutic candidates for ASD and other NDDs," said, Delphine Charvin, PhD, STALICLA's Chief Scientific Officer.
"Despite the high prevalence of neurodevelopmental disorders in the U.S. and Europe, most NDDs are diagnosed and treated based on behavioral-based classification systems alone," said Study Investigator, Craig Erickson, MD, Cincinnati Children's Hospital Medical Center. "I look forward to investigating the potential of STP1 in the first clinically validated subpopulation of patients with idiopathic ASD."
The double-blind, placebo-controlled, first-in-human study is being conducted at Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio. The primary objective of the trial is to evaluate the safety and tolerability of a two-week oral treatment with STP1 in adult patients who match the ASD-Phen1 clinical criteria. Two ascending dose cohorts of approximately 12 patients each will be enrolled. The study will also assess the pharmacokinetic and pharmacodynamic effects of STP1 and includes exploratory efficacy surrogate endpoints.
About ASD Phenotype 1
ASD Phenotype 1 (ASD-Phen1) is a subgroup of patients with autism spectrum disorder which has been identified by STALICLA's platform and includes as many as 20% of the total ASD population with a non-monogenetic cause. ASD-Phen1 was first validated by an observational clinical trial conducted at the Greenwood Genetic Center (South Carolina, U.S.). ASD-Phen1 criteria were applied to a cohort of 313 patients with ASD. The study led to the identification of patients who matched the required clinical criteria and represented the first clinical validation of the ASD-Phen1 subgroup. Further biological characterization of ASD-Phen1 revealed a specific biological signature, or "fingerprint," of the patient subgroup that was consistent with the signature proposed by STALICLA's DEPI drug discovery platform.
STALICLA's investigational new drug, named STP1, is a first-in-class precision medicine being developed as part of the company's proprietary pipeline for the treatment of ASD for patients within the Phenotype 1 subgroup (ASD-Phen1). STP1 is a novel combination of a pan-PDE inhibitor with anti-inflammatory properties and a modulator of NKCC1. STP1 was identified as a potential match for ASD Phenotype 1 using the DEPI platform, addressing ASD-Phen1 biological dysfunctions and limiting the emergence of compensatory disease pathways. In addition, STP1 has shown a good safety profile in nonclinical toxicology studies. Taken individually, both compounds have also demonstrated good clinical safety and tolerability profiles over a broad range of doses and non-ASD indications.
STALICLA is reframing neurodevelopmental disorders on the basis of novel insights into their biological underpinnings. By combining real-world patient data and a proprietary systems biology and AI-powered drug discovery platform called DEPI, STALICLA is uncovering novel patient subgroups, enabling targeted clinical development and fueling a rich pipeline of precision therapies. STALICLA has formed a global network of partnerships to bring the first precision medicine treatments to patients with NDDs as efficiently as possible with an initial focus on autism spectrum disorder (ASD). https://stalicla.com/
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