10 September 2024

Argent BioPharma Ltd.

(Argent BioPharma or the Company)

CannEpil®

CannEpil®, one of Argent BioPharma's flagship products, is an enhanced iteration of a compounded isolated cannabinoid formulation. It leverages well-known historical data to become one of the first compounded prescription investigational drugs used for seizure management over the past years. The active ingredients in this formulation include two isolated cannabinoids, each dissolved in a liquid solution.

While the 'Food and Drug Administration' in the USA (FDA) has already approved the use of cannabidiol alone for treating seizures in a few rare conditions, CannEpil® as a compounded formulation may offer additional advantages. Different combinations have shown a synergistic effect in animal models of seizures, enabling improved control with significantly lower doses.

However, different cannabinoid combinations have not yet been thoroughly studied for the treatment of seizure disorders in well-controlledclinical trials. This is due to several factors, with the primary challenge being the lack of rigorous standardization.

This is where our research and development efforts come into play: We adhere to strict standard procedures for sourcing the raw materials, produce them in a 'Good Manufacturing Practice' (GMP)-certified facility by the national Competent  Health Authorities using compliant processes, and thus have a rigorously manufactured, stable, effective and quality product branded as CannEpil® at our disposal.

Early clinical data has been gathered from the use of CannEpil® in the treatment of epilepsy through Australia's and Ireland's Special Access Scheme programs. Two key findings have emerged from this initial experience:

·    CannEpil® was prescribed at 28-35% lower doses compared to the CBD-only drug it was compared with, demonstrating its potent efficacy.

·    The retention rate, or continued prescription use, for CannEpil® was observed to be higher than that of the CBD-only counterpart, potentially indicating an improved safety profile alongside enhanced efficacy.

We are currently addressing the preclinical and clinical gaps in knowledge and data as part of the pathway to registering CannEpil® and obtaining a marketing authorization. These activities include completing the missing toxicology data through pre-clinical studies in the upcoming quarters. This will be followed by initial clinical trials to establish the pharmacokinetics of CannEpil® in humans and early-phase efficacy trials.

A few haphazard comments may be added in favor of CannEpil®:

Clinical

·    Combined cannabinoid treatments have been occasionally observed to rescue seizure control after the failure of pure CBD therapies. This opens the possibility that CannEpil® might be effective in patients with treatment-resistant epilepsy who have failed treatment with CBD-alone or other approved, conventional treatments.

·    Isolated reports indicate that treatment with cannabinoid combinations is associated with significantly less liver toxicity as compared to the most common pure-CBD only preparation.

·    The absence of any sugar in CannEpil® renders it as an acceptable add-on treatment in patients maintaining a ketogenic diet; and

·    A randomized controlled trial on 31 healthy participants was completed, and the effect of acute dosing with CannEpil® on driving skills was assessed. The results are compatible with a good safety profile.

Markets

·    Ireland: In 2019, CannEpil® became eligible for patients in Ireland and fully covered under Ireland's Primary Care Reimbursement Service as part of the country's Medicinal Cannabis Access Program (MCAP). In 2021, the Irish Health Service Executive (HSE) extended its support by including CannEpil® in its reimbursement scheme under the Special Access Program. This inclusion allows patients to access CannEpil® without bearing the full financial burden, significantly enhancing its accessibility for those in need;

·    UK: in early 2023, CannEpil® was introduced into the UK and made available through the "Named Patient Request" system, a specialized mechanism that allows healthcare professionals on the General Medical Council (GMC) Specialist Register to prescribe it for patients with severe, treatment-resistant epilepsy. The approval for CannEpil®'s importation and prescription in the UK has been granted by the Medicines and Healthcare products Regulatory Agency (MHRA);

·    The UK National Health Service's Refractory Epilepsy Specialist Clinical Advisory Service (RESCAS) affords support by ensuring access to innovative treatments, such as CannEpil®, that can provide hope and improve outcomes for patients with challenging medical conditions;

·    The value of this avenue is illustrated by the successful treatment of "patient M" who gained access to CannEpil® thanks to support from the 'I AM Billy Foundation'. The real-world data gathered in this case highlights the transformative potential of CannEpil® in managing refractory epilepsy, with a remarkable reduction in seizures and improved quality of life observed after 1.5 years of treatment. Beyond seizure control, patient M has experienced enhancements in cognitive function, motor skills (fine and gross), speech, and sleep patterns;

·    Argent BioPharma is assessing CannEpil® clinical development program in the UK, with ongoing real-world data collection to gather further data on its safety and efficacy; and

·    USA: In 2024, the company began a pre-IND process with the FDA, leading to getting assigned with  an Investigational New Drug (IND) number. This move positions CannEpil® for potential entry into the US market, contingent upon successful clinical outcomes.

In 2024, as part of a strategic restructuring into Argent BioPharma, the company temporarily paused its clinical trials to reassess and refine the regulatory pathway for CannEpil®. This restructuring aims to incorporate additional intellectual property (IP) protections and potential commercial safeguards, ensuring the drug's long-term viability and market competitiveness.

Argent BioPharma's current efforts include submitting an Orphan Drug Designation (ODD) application with the FDA, with another application in the conceptualization phase. Additionally, Argent BioPharma's medical team is exploring a unique indication associated with a novel mechanism of action, which is believed to hold significant potential for an IP claim.

To summarize, by leveraging insights from the Pre-IND dialogues and emerging data, particularly from UK case studies, Argent BioPharma aims to establish a new clinical development pathway for CannEpil®.  These efforts are designed to position Argent BioPharma favorably within the regulatory landscape, creating an enhanced route to market that could potentially benefit millions of epilepsy patients worldwide.

-Ends-

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About Argent BioPharma

Argent BioPharma Limited (the "Company") (ASX: RGT; LSE: RGT; OTCQB: RGTLF) an innovative multidisciplinary drug development Company within the biopharmaceutical sector.  The Company focuses on multidisciplinary methods with Nanotechnology, developing multi-target therapies for comprehensive disease management, especially concerning the central nervous system ("CNS") and Immunology treatments.

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