PharmAla Files IP Protection For Novel MDXX Molecule: Viability As Autism Spectrum Treatment

Canadian biotech company PharmAla Biotech Holdings PMBHF has filed a patent covering composition of matter of Novel Chemical Entity (NCE) PharmAla-1. 

PharmAla-1 was discovered through computational in-silico modeling exercises via an innovation grant-funded partnership with University of Windsor. It’s the company’s first molecule produced through this means. 

“Traditionally, you make drugs and test them. In silico-modeling basically means that you model the drugs on a computer before you make them,” CEO Nick Kadysh explains. “You have an idea of their receptor binding profiles and characteristics before you even get to animal research -so it's a fantastic way to narrow down potential drug candidates, saving time and money.” 

So following synthesis, PharmAla-1 underwent a year of preclinical (animal) proof-of-concept research at the University of Arkansas for Medical Sciences in Prof. William Fantegrossi’s lab.

Bridging Entactogens And Classical Psychedelics 

PharmAla-1’s therapeutic potential would combine “a number of highly desirable drug characteristics” including a potency that would make patients need a reduced amount to profit from its effects.

“All drugs have a toxicology profile of some kind: cytotoxicity (cell damage), genotoxicity (gene damage), cardiotoxicity (heart damage), neurotoxicity (brain/CNS damage). In the case of MDMA, its major toxicological endpoints are cardiotox and, to a lesser extent, neurotox,” says Kadysh. “We believe Pharmala-1 will have an improved toxicology profile for both of those endpoints.” 

However, what the team is most excited and interested about is what they call "table stakes" in drug development. In this case, it’s Pharmala-1's receptor binding as an indication of its efficacy. 

“We see a molecule we think will bridge the divide between entactogens like MDMA and classical psychedelics like psilocybin. Stronger binding to the serotonin receptors that you want, like 5-HT2A, and the ability to cross the cell membrane and bind to serotonin receptors inside the cell, triggering neuronal growth and neuroplasticity,” Kadysh told Benzinga. “That's a very potent combination in our opinion.”

For research VP Dr. Harpreet Kaur, PharmAla-1 brings together PharmAla’s drug discovery ethos “in an exciting way,” focusing both on “regulatory excellence” and “cutting-edge science.” 

In addition to holding favorable safety pharmacology profiles compared to traditional MDXX compounds, as a non-controlled drug it would allow for faster regulatory development.

PharmAla’s novel drug development pipeline as a whole has advanced throughout 2023, with the ALA family soon moving into clinical development.

To the question whether PharmAla’s upcoming pipeline will include non-MDXX class molecules, Kadysh replied that the company is “highly focused” on this class of molecules in terms of NCE development. “We believe in doing a few things, but being the absolute best in the world at them.”

An Alternative To ASD Treatment: New Published Review

PharmAla’s latest review article, "Balancing therapeutic efficacy and safety of MDMA and novel MDXX analogues as novel treatments for Autism Spectrum Disorder (ASD)," points at the potential of these substances to elicit pro-social effects and alleviate the common social anxiety and avoidance seen in the existing millions of ASD cases. 

The article explains these entactogens’ pharmacology, their drug-binding sites, metabolic enzymes and chemical structure-activity relationships.

According to author Dr. Fantegrossi, the therapeutic potential of MDMA and MDXX analogues “was recognised very early on but was masked by abuse-related and toxic effects.” 

A better understanding of the complex pharmacology of these substances has allowed “significant advancements” in drug design and formulations to mitigate their adverse effects while preserving clinically useful ones, and the review describes some of this work in the context of ASD treatment to “hopefully lay the groundwork for further research in this area.”

Kadysh told Benzinga the article’s purposes include supporting PharmAla’s drug development efforts; allowing researchers to see the data; finding prospect partners for its upcoming research; and establishing its “expertise and bona fides” in the space.

PharmAla aims to take lead candidate and MDMA analog ALA-002 to the clinic next together with research partner -and MDMA client- the University of Sydney.

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Photo: Benzinga edit with photo by Irina Anosova and ANDREI ASKIRKA on Shutterstock.