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Drug Licensed for Precision Autism Spectrum Therapies

Brain activity graphic

(Gordon Johnson, Pixabay)

10 Oct. 2022. A company making precision therapies for neurological disorders is acquiring rights to a compound first designed as a treatment for cancer, inflammation, and oxidative stress. The agreement with Stalicla SA in Geneva, Switzerland is expected to bring Evgen Pharma in Alderley, U.K. as much as $161 million if all terms of the deal are fulfilled.

Evgen Pharma is developing medications based on sulforaphane, a compound derived from plant-based chemicals called polyphenols associated with prevention of oxidative stress, inflammation, cancer, cardiovascular diseases, diabetes, and neurodegenerative diseases. On its own, says Evgen, sulforaphane is highly unstable, with research conducted only on extracts of the compound frozen at -20 degrees C. The company says its lead product code-named SFX-01 is a formulation of sulforaphane and alpha-cyclodextrin, made from carbohydrate molecules shown to be odorless, tasteless, and stable up to 200 degrees C.

Evgen Pharma produces SFX-01 in oral tablet form, and is testing it in clinical trials as a treatment for metastatic breast cancer. The company is also evaluating forms of SFX-01 in preclinical tests with glioblastoma, a brain cancer, and blood-related cancers. Evgen says it’s also studying SFX-01 with nuclear factor erythroid-2-related factor 2, or Nrf2, a transcribed protein associated with expression of anti-oxidant enzymes. Among the targets for this pathway are conditions causing inflammation and fibrosis, but also neurological diseases.

Stalicla is a five year-old developer of precision therapies for neurodevelopmental conditions, starting with autism spectrum disorder. The company says it seeks to address specific biomarkers revealed through genomic and related analysis, instead of basing therapies on observed behaviors, which can lead to erroneous conclusions. Stalicla cites research showing neurodevelopmental disorders can be traced to genetic sources, with many of the conditions overlapping and some patients experiencing multiple neurological diseases at once, sharing common symptoms.

Discover treatments addressing precise genomic conditions

Stalicla says its technology, called databased endophenotyping patient identification or DEPI, discovers treatments addressing precise genomic conditions and biologic indicators, rather than behavioral observations. The company says DEPI applies systems biology and machine learning algorithms from multiple genomic databases to identify sub-groups of patients exhibiting specific genomic and related “omic” factors, who may benefit from drug candidates addressing those variables. Stalicla is testing in a clinical trial a therapy package code-named STP1 that targets an identified and validated sub-type of autism spectrum disorder that the company says comprises about 20 percent of the total autism spectrum disorder population.

Evgen Pharma is licensing worldwide rights for SFX-01 to Stalicla for development of therapies for neurodevelopmental disorders and schizophrenia, beginning with a second sub-type of autism spectrum disorder. Stalicla says its DEPI technology identified SFX-01 as a treatment candidate for this autism spectrum disorder sub-type.

Under the agreement, Stalicla is responsible for clinical development of SFX-01 for autism spectrum disorder or ASD, while the two companies plan to collaborate on regulatory approvals. Stalicla is providing to Evgen an initial payment of $0.5 million, with Evgen eligible for further milestone payments totaling $160.5 million, as well as royalties on product sales.

“There are no current approved treatments for ASD,” says Stalicla founder and CEO Lynn Durham in a company statement released through Cision. “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.”

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