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Pushing the frontier of precision medicine into the NDD space

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Rebekah Jordan sat down with Lynn Durham, CEO & founder of STALICLA, to discuss STALICLA’s recent partnership with Evgen Pharma that aims bring a therapeutic option to currently underserved patients with neurodevelopmental disorders (NDDs).

Key insights:

Clinical psychiatric and neurodevelopmental diagnoses encompass a broad diversity of underlying biology. This contributes to drug development failure by diluting true responders in a population of patients with heterogeneous biology.

Furthermore, a major weakness in the drug development process for Neurodevelopmental Disorders (NDDs) and neuropsychiatric disorders is that behaviour is a poor biomarker.

STALICLA developed its proprietary DEPI platform – Databased Endophenotyping Patient Identification - as a first precision medicine platform for patients diagnosed with NDDs. By aggregating molecular data and human genetic information, DEPI discovers “biologically-based endophenotypes within classical neuropsychiatric diagnostic groups.” 

As it stands, there are no approved medicines for the core symptoms of autism. In light of this, Evgen Pharma recently partnered with STALICLA for the worldwide in-licensing of Evgen’s SFX-01 – a potential treatment for autism spectrum disorder (ASD).

With the terms of the licensing, STALICLA is able to target of patient groups most likely to benefit from SFX‐01, not only de‐risking the clinical development but potentially bringing a therapeutic option to those individuals who are currently underserved.

How DEPI platform works

The ML engine converges molecular data with human genetic information and non-behavioural clinical signs and symptoms, to define biologically related subgroups and create testable clinical hypotheses related to that biology. DEPI, then matches these patient subgroups with an NDD-targeted drug (or drug combination) by integrating and analysing large omics data sets, including genomics, proteomics, pharmacogenomics and clinical data.

Durham said: “The DEPI platform has been validated in clinical settings. In prospectively designed trials, the platform shows high specificity, sensitivity, and positive predictive value in calling “high” responder patients to previously failed drug candidates. Additionally, DEPI has identified two distinct subgroups of patients with Autism Spectrum Disorder, ASD-Phenotype1 and ASD-Phenotype2, together with tailored treatments for each.” 

Both projects are expected to progress into clinical Phase 2 in 2023.

Durham commented: “DEPI has already reached validation through the clinical identification of biologically defined subgroups of patients with ASD, and their respective candidate treatments. SFX-01 will strengthen STALICLA’s pipeline to advance precision medicine for a new subgroup of patient with Autism Spectrum Disorder.”

Acquiring SFX-01 for ASD treatment

“SFX‐01 is a patented composition of synthetic sulforaphane and alpha‐cyclodextrin and is the only grade of sulforaphane suitable for clinical research and eventual approval as a medicine.”

Although previous research has shown the link of clinical efficacy of sulforaphane in improving symptoms of ASD, patient heterogeneity still stands as a barrier in identifying the patients that will most likely respond to this type of treatment.

Its capabilities in identifying ASD patients who are most likely to respond to certain compounds allowed STALICLA to identify SFX-01 as the best treatment candidate for its second subgroup of patients, ASD-Phenotype 2.

Durham added: “SFX-01 in-licensing follows the completion, in early 2022, of Phase 1b for STP1, STALICLA's lead candidate tailored to ASD-Phenotype1, a first subgroup of patients identified by DEPI.

“Bringing precision treatments to patients such as SFX-01 could radically alter this landscape thanks to an effective oral option for a subgroup of ASD patients. This agreement with Evgen to advance SFX-01 expands STALICLA's option to improve the quality of life of a biologically defined subgroup of patients with ASD.”

Under the agreement terms, STALICLA will be responsible for the clinical development in the NDD field, and there will be cooperation between both companies to adhere to the regulations and secure approval of the treatment.

Pushing the frontier in NDD

With the positive results from SFX-01 and a first indication for a biologically-characterised subgroup of patients with autism spectrum disorder, STALICLA aims to push the frontier of precision medicine further into the NDD space.

Beyond this, DEPI platform could be applied in the neurodegenerative disease space “provided similar, systematic, system-biology and biosampling driven data collection effort in neurodegenerative populations.”

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