- Positive results of in vivo toxicology studies in which
AEF0117 shows a favorable therapeutic index >13,000 time the
active dose:
- Chronic oral toxicity studies (6 months in rats and 9 months
in dog) allowing for unrestricted chronic treatment in
humans.
- Reproductive toxicology studies showing that AEF0117 does
not modify embryonic and embryofetal development.
- Juvenile tox studies permitting the administration of
AEF0117 in adolescent, an important target population in cannabis
use disorder (CUD).
- Positive results of phototoxicity studies showing that
AEF0117 does not sensibilize to sunlight.
- Finalization of the drug substance and drug product
late-stage developments allowing its administration in Phase 3
clinical trials.
- Completion of environmental risks assessment indicating that
AEF0117 does not pose any risk for the environment.
- These non-clinical developments confirm the positive
feedback from an independent safety committee (DSMB) on the first
115 patients of the phase 2b of AEF0117 in CUD (end of recruitment
Q4 2023) that has confirmed the good tolerability of AEF0117 in
people with CUD.
Regulatory News:
Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS),
a clinical-stage biopharmaceutical company specializing in the
development of treatments for brain disorders, today announces that
it has successfully completed all the regulatory non-clinical
studies necessary to enter in phase 3 clinical trials AEF0117, the
Company’s most advanced signaling specific inhibitor of the CB1
receptor of the endocannabinoid system. AEF0117 is currently tested
in a large phase 2b study in people with cannabis use disorder
(CUD). CUD is the current definition of cannabis abuse and
dependence in the DSM5, the reference diagnostic manual for
psychiatric diseases. Recruitment is on track and results are
expected in Q2 2024. Thanks to the successful completion of the
non-clinical development ahead of schedule, AEF0117 will be
prepared to enter pivotal phase 3 clinical trials for CUD upon
successful completion of the present phase 2b clinical study and
regulatory clearance of the phase 3 protocol.
Pier Vincenzo Piazza, CEO of Aelis Farma, said: “We are
very pleased with the successful end of this campaign of
non-clinical studies which shows three important things. First,
AEF0117 confirms its safety profile with a safe dose 13,000 times
higher than the active dose, which means that no side effects of
AEF0117 have been identified in animals. Second, AEF0117 will be
ready to enter phase 3 clinical studies after the completion of the
current phase 2b in cannabis use disorder, optimizing the pathway
to market. Third, Aelis Farma delivers on its promises, in this
case ahead of time, highlighting our ability to perform complex
clinical and non-clinical programs in parallel. I would like to
take this occasion to thank the Aelis team for this great
achievement”.
The favorable safety profile of CB1-SSi resolves a bottleneck
in CB1 pharmacology and opens the way to several markets with
strong medical needs
CB1-inhibitors, a main objective for drug development upended
by safety concerns
Developing CB1 antagonists has been a major goal for the
pharmaceutical industry with most of the main players engaged in
this endeavor. Indeed, due to its important and diverse
physiological roles, CB1 receptors could be a gateway to addressing
several peripheral and central nervous system diseases with
significant medical needs. For example, for the periphery:
metabolic, fibrotic and skin diseases and for the brain: addiction,
cognitive impairments, psychosis and autism spectrum disorders.
Unfortunately, the first generation of CB1 antagonists had
unacceptable behavioral side effects and poor therapeutic index
often inferior to 10 which led either to their withdrawal from the
market (Sanofi, rimonabant) or to the suspension of their
developments (e.g. Pfizer, Merck).
To circumvent this problem, some companies have developed
periphery restricted CB1 antagonists, i.e. drugs that work like
rimonabant but do not access the brain, such as the compound
developed by Inversago, recently acquired by Novo Nordisk. While
these drugs are likely safer than the previous generation of
CB1-antagonists, they only target peripheral diseases, and cannot
be used for brain disorders.
Aelis Farma’s CB1-SSi allow to address safely the entire
spectrum of CB1-related diseases as they reproduce a natural
mechanism used by the brain to control the hyperactivity of the
CB1
CB1-SSi are not antagonists, which block all the activity of the
receptor, but they are able to selectively inhibit only some
functional outputs (signalings) of the CB1. Their name, Signaling
Specific inhibitors (SSi), stems from this characteristic. In
particular, CB1-SSi seem able to selectively inhibit the signaling
of the receptor linked to pathological states while preserving
normal physiological activities. This characteristic confers them
their very favorable safety profile. A striking aspect of their
action is that they do not modify behavior per se, in animals or in
humans, but are able to potently reverse the hyperactivation of the
CB1 induced by cannabis (AEF0117, Nature Medicine1) or restore
certain impaired cognitive functions (AEF0217).
CB1-SSi can have this disease specific effect because instead of
having an artificial mechanism of action like antagonists, they
copy a natural inhibitory mechanism that the brain uses to control
a hyperactivity of the CB1. This mechanism, discovered by the CEO
of Aelis Farma, Dr. Pier Vincenzo Piazza, has been fine-tuned by
evolution and is mediated by the hormone pregnenolone. Pregnenolone
can inhibit CB1 activity in this signaling specific manner
countering the overactive receptor while preserving its normal
basal functioning (Science paper2). Unfortunately, pregnenolone
cannot be used as a therapeutic drug. It has very low oral
absorption, a very short half-life because is rapidly transformed
in several active steroids (progesterone, testosterone etc..) that
can potentially induce serious side effects. CB1-SSi are new
molecular entities that reproduce the signaling inhibition of the
CB1 by pregnenolone, but are well absorbed, stable and not
transformed in steroids.
Aelis Farma is developing a differentiated pipeline of
CB1-SSi which will allow to access a large spectrum of
CB1-dependent diseases.
Aelis Farma pipeline overview
AEF0117 is the first CB1-SSi developed by Aelis Farma, designed
to counteract the effects of THC (Δ9- tetrahydrocannabinol), the
active ingredient of cannabis. The company has a second CB1-SSi at
the clinical stage, AEF0217, designed to treat cognitive disorders
by opposing the effects of an excess of endocannabinoids production
by the brain. Both compounds have a favorable safety profile and no
effect on normal behavior per se. Aelis Farma, using its
proprietary screening platform, has also identified new families of
CB1 compounds, chemically and functionally differentiated from the
first two, but sharing the core mechanism of action.
The development of AEF0117, for the treatment of cannabis use
disorder is progressing according to plan.
AEF0117 is currently in a phase 2b study conducted in 11
clinical centers in the US and coordinated by Pr. Frances Levin
(Columbia University, New York). The study, that aims to enroll up
to 330 patients, progresses according to plan with recruitment
expected to be finalized by Q4 2023. The first results should be
available in Q2 2024.
To ensure a smooth transition of AEF0117 to the phase 3 study
after a positive phase 2b, Aelis Farma initiated, in parallel with
the phase 2b clinical studies, a series of toxicology studies
required by the regulatory authorities. These confirmed the very
favorable therapeutic index >13,000 time the active dose.
Importantly, chronic oral toxicity studies (6 months in rats and 9
months in dog) allow AEF0117 to be used as unrestricted chronic
treatment in humans and juvenile toxicity studies enable its
administration in adolescents, an important target population in
CUD. Furthermore, the reproductive toxicology studies show that
AEF0117 does not modify embryonic and embryofetal development.
Finally, Aelis Farma has successfully completed environmental risk
assessments, indicating that AEF0117 poses no risk to the
environment, as well as phototoxicity study, showing that AEF0117
does not sensitize to sunlight. The favorable safety profile
observed in these non-clinical studies confirm the positive
feedback from an independent safety committee (DSMB) on the first
115 patients of the phase 2b that has confirmed the good
tolerability of AEF0117 in cannabis addicts.
AEF0117 development is supported by Indivior, a leading
pharmaceutical company in the treatment of addiction
For the development and commercialization of AEF0117 for
disorders due to excessive cannabis use, Aelis Farma has an
exclusive option-license agreement with Indivior PLC, a leading
pharmaceutical company in the treatment of addiction. As part of
this agreement, Aelis Farma has received already $30 million
(option payment). If Indivior exercises the license option at the
end of the phase 2b (H2 2024), Aelis Farma will receive a $100
million license fee and up to $340 million in additional payments
contingent on the achievement of development, regulatory and
commercial milestones as well as, royalties on net sales of AEF0117
ranging between 12% and 20%. Following the exercise of the option,
all development, registration, and commercialization costs of
AEF0117 will be borne by Indivior.
***
About AELIS FARMA
Founded in Bordeaux in 2013, Aelis Farma is a biopharmaceutical
company that is developing a new class of drugs, the
Signaling-Specific inhibitors of the CB1 receptor of the
endocannabinoid system (CB1-SSi). CB1-SSi have been developed by
Aelis Farma based on the discovery of a natural brain defense
mechanism made by the team led by Dr. Pier Vincenzo Piazza, the
Company’s CEO, when he was director of Neurocentre Magendie of the
INSERM in Bordeaux. By mimicking this natural mechanism, CB1-SSi
appear to selectively inhibit the disease-related activity of the
CB1 receptor without disrupting its normal physiological activity.
CB1-SSi have consequently the potential to provide new treatments
for several brain diseases. Aelis Farma is currently developing two
first-in-class clinical-stage drug candidates: AEF0117 for the
treatment of cannabis use disorder (CUD), currently being tested in
a phase 2b study in the United States; and AEF0217 for cognitive
disorders, including those of Down Syndrome (Trisomy 21), currently
in a phase 1/2 study in Spain in people with Down syndrome. The
Company also has a portfolio of new innovative CB1-SSi for the
treatment of other disorders associated with a dysregulation of the
activity of the CB1 receptor. Aelis Farma draws on the talents of
more than 20 highly qualified employees. For more information,
visit www.aelisfarma.com and follow us on LinkedIn
and Twitter.
ISIN: FR0014007ZB4 Ticker: AELIS B Compartment
of Euronext Paris
Disclaimer
Forward-looking statements
Some information contained in this press release are
forward-looking statements, not historical data. These
forward-looking statements are based on current beliefs,
expectations, and assumptions, including, but not limited to,
assumptions about Aelis Farma's current and future strategy and the
environment in which Aelis Farma operates. They involve known and
unknown risks, uncertainties, and other factors, which may cause
actual results, performance, or achievements, or industry results
or other events, to differ materially from those described or
implied by such forward-looking statements. These risks and
uncertainties include those set out and described in detail in
Chapter 3 "Risk Factors" of Aelis Farma's Universal Registration
Document approved by the Autorité des Marchés Financiers on April
26, 2023, under number R.23-018.
These forward-looking statements are made only as of the date of
this press release and Aelis Farma expressly disclaims any
obligation or undertaking to release any updates or corrections to
the forward-looking statements included in this press release to
reflect any change in expectations or events, conditions, or
circumstances on which any such forward-looking statement is based.
Forward-looking information and statements are not guarantees of
future performance and are subject to various risks and
uncertainties, many of which are difficult to predict and generally
beyond Aelis Farma's control. Actual results could differ
materially from those described in, or implied or projected by,
forward-looking information and statements.
1 Signaling-specific inhibition of the CB1 receptor for cannabis
use disorder: phase 1 and phase 2a randomized trials; Nature
Medicine; 29; pages 1487–1499 (2023); June 8, 2023 2 Pregnenolone
Can Protect the Brain from Cannabis Intoxication; Science; Vol 343,
Issue 6166; pp. 94-98; January 3, 2014
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version on businesswire.com: https://www.businesswire.com/news/home/20231107000699/en/
AELIS FARMA Pier Vincenzo Piazza CEO
contact@aelisfarma.com
NewCap Dusan Oresansky/Aurélie Manavarere Investor
Relations aelis@newcap.eu +33 1 44 71 94 92
NewCap Arthur Rouillé Media Relations aelis@newcap.fr +33
1 44 71 00 15
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