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-
Authorised for release by the board of directors, for further
information please contact:
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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