- AEF0217 is being developed as the first treatment for
cognitive deficits caused by a hyperactivity of the CB1 receptor,
the first indication being those linked to Down syndrome (Trisomy
21), which is currently a major unmet medical need.
- The single dose and multiple ascending dose trial of AEF0217
has demonstrated the safety, tolerability and good bioavailability
of AEF0217 in healthy volunteers.
- On the basis of these positive results, the AEMPS, the
Spanish agency for medicines and medical devices, has authorized a
phase 1/2 clinical trial in adults with Trisomy 21. This study will
test the safety, tolerability and plasma exposure and may provide
preliminary indications of AEF0217’s activity.
- Enrollment in the phase 1/2 clinical trial is expected to
start before the end of the year.
Regulatory News:
Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS, PEA-PME
eligible) (Paris: AELIS), a clinical stage biopharmaceutical
company specializing in the development of treatments for brain
diseases, announces today positive results from its phase 1
clinical trials in healthy volunteers with its drug candidate
AEF0217. AEF0217 is being developed for the treatment of cognitive
deficits caused by a hyperactivity of the CB1 receptor, and as a
first indication those associated with Trisomy 21 (Down syndrome).
Acute and chronic administration of AEF0217, up to 30 times the
anticipated therapeutic range, is well tolerated and presents
favorable safety and plasma exposure profiles, which allow the next
stage of clinical development.
AEF0217 is the second drug candidate of the new class of drugs
developed by Aelis Farma: the Signaling Specific inhibitors of the
CB1 receptor (CB1-SSi). The CB1 is the main receptor of the brain’s
endocannabinoid system that is responsible for regulating several
physiological and cognitive processes. Recent research has
suggested that cognitive disorders linked to Trisomy 21 involve a
hyperactivity of the CB1 receptor. AEF0217 is positioned as the
first treatment for cognitive deficits caused by hyperactivity of
the CB1 receptor. AEF0217 aims to capitalize on the distinctive
characteristics of CB1-SSi which allow to inhibit a hyperactivity
of the CB1 receptor without affecting normal physiological
functions and without causing significant side effects, two
criteria that are particularly important for vulnerable populations
such as those with Down syndrome.
Pier Vincenzo Piazza, CEO of Aelis Farma, commented: “The
very positive results of the phase 1 program achieved with AEF0217
constitute a significant step in the development of this drug
candidate to treat cognitive deficits and in particular those
linked to Trisomy 21, also known as Down syndrome. They also
confirm the strong potential of the therapeutic class developed by
Aelis Farma, the CB1-SSi, showing again an excellent safety profile
as well as favorable pharmacokinetic characteristics. AEF0217 can
now move forward and enter a phase 1/2 trial in people with Trisomy
21. This significant progress enables us to swiftly move towards
the confirmation of AEF0217’s therapeutic potential to improve the
cognitive deficits of people with Trisomy 21, for which there are
no effective therapeutic solutions today.”
The phase 1 clinical program of AEF0217 combines three clinical
trials authorized by the Spanish health authority, the AEMPS, in
September 2021: a single ascending doses trial, a multiple (7 days)
ascending doses trial, and a pharmacokinetic trial analyzing the
impact of food consumption on drug absorption. This overall
clinical program was designed to assess the safety, tolerability
and absorption of AEF0217, in the range between 0.2 mg and 6 mg, in
comparison with a placebo, in 68 healthy volunteers, aged between
18 and 55 years old.
The results obtained have shown that all the doses of AEF0217
were well tolerated by healthy volunteers with no serious adverse
reaction. Only 3 adverse events related to AEF0217, manifesting as
low intensity diarrhea, were observed during the full program. In
addition, no relevant clinical changes in routine biological tests,
electrocardiograms or vital signs were observed. Similarly,
psychometric tests assessing main psychological dimensions, such as
depression, anxiety, psychosis and suicidal tendencies did not show
significant difference between AEF0217 and placebo. The
pharmacokinetic profile of AEF0117 has also proven favorable and,
as expected, is characterized by very good plasma absorption,
linearity between doses and a long half-life.
“These positive results for AEF0217 are all the more encouraging
as the safety of the compound is a particularly important criterion
for the treatment of the Down syndrome population and for
acceptance by the families of a treatment for cognitive deficits
for these vulnerable people. We are delighted to be part of this
revolutionary and exciting project to assess this drug candidate,
which represents real hope for many people affected by Down
syndrome and their families,” added Prof. Rafael de la Torre
Fornell, principal investigator of the clinical trials and
coordinator of the ICOD project.
It is thanks to these positive data that the AEMPS has
authorized the advancement of AEF0217 to a first clinical trial in
participants with Down syndrome. The main objective of this phase
1/2 study will be to demonstrate the safety, tolerability and the
pharmacokinetic profile of AEF0217 in people with Down syndrome.
The trial could also provide initial evidence of the activity of
this drug candidate. The first enrollments are expected before the
end of 2022 and the first clinical results should be available in
mid-2023.
***
About the clinical program of AEF0217 for the treatment of
cognitive disorders in Trisomy 21: Europe’s ICOD project.
The phase 1 program of AEF0217 is part of the European H2020
ICOD project (Improving COgnition in Down syndrome, Grant N°
899986), and is being performed in collaboration with the Hospital
del Mar Medical Research Institute (IMIM) in Barcelona (Spain) and
Prof. Rafael de la Torre Fornell, coordinator of the project and
principal investigator of the trial. In February 2021, the ICOD
project received funding of €6 million from the European Commission
in order to finance the clinical development of AEF0217 for the
treatment of cognitive deficits linked to Down syndrome.
About AELIS FARMA
Founded 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). These new molecular entities hold great potential in the
treatment of many brain diseases. CB1-SSi were developed by Aelis
Farma on the basis of the discovery of a new natural defense
mechanism of the brain made by the team of Dr. Pier Vincenzo
Piazza, CEO of the Company, when he was Director of the Magendie
Neurocenter of Inserm in Bordeaux. For these discoveries, Dr.
Piazza was awarded the Inserm Grand Prix and the Grand Prix of
Neurology of the French Academy of Sciences, which are among the
most prestigious French awards in medicine and neurology.
Aelis Farma is developing two first-in-class drug candidates
that are at the clinical stage, AEF0117 and AEF0217, and has a
portfolio of innovative CB1-SSi for the treatment of other diseases
associated with a dysregulation of the activity of the CB1
receptor.
AEF0117, which targets disorders due to excessive cannabis use
(addiction and psychosis), has shown indications of efficacy in a
phase 2a clinical trial and entered a phase 2b clinical trial in
the United States in Q2 2022. Aelis Farma has an exclusive
option-license agreement with Indivior PLC, a leading
pharmaceutical company in the treatment of addiction, for the
development and commercialization of AEF0117 for disorders due to
excessive cannabis use. As part of this agreement, Aelis Farma has
already received $30 million (option payment). If Indivior
exercises the license option at the end of the phase 2b, Aelis
Farma will receive a $100 million license fee (potentially in 2024)
and Indivior will take responsibility for additional development
costs. The agreement also includes 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%.
AEF0217, which targets various cognitive disorders including
those associated with Down syndrome, has successfully completed
safety and pharmacokinetic trials (phase 1 clinical program) in
healthy volunteers and will enter phase 1/2 trials before the end
of 2022. This new trial will assess the safety and the
pharmacokinetics of AEF0217 in people with Down syndrome and could
also provide the first indications of activity. The results are
expected in Q2 2023. AEF0217 has undergone an extensive preclinical
proof-of-concept program using highly innovative tests to assess
cognitive functions. In this context, AEF0217 demonstrated its
ability to completely reverse the cognitive impairments observed in
several animal models of cognitive disorders, such as Down syndrome
and Fragile X syndrome, as well as in certain cognitive deficits
associated with aging.
Based in Bordeaux, within the Magendie Neurocenter, Aelis Farma
has a team of 23 highly qualified employees and has benefited from
investments from the Nouvelle-Aquitaine Region, Inserm Transfert
Initiative, Bpifrance, regional funds ACI, NACO and Aqui-invest and
from IRDI Capital Investissement.
For more information: www.aelisfarma.com
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 detailed in Chapter 3 "Risk
Factors" of Aelis Farma's registration document approved by the
Autorité des marchés financiers on 14 January 2022 under number
I.22-003.
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20221115006181/en/
AELIS FARMA Pier Vincenzo Piazza CEO
contact@aelisfarma.com
NewCap Dusan Oresansky/Hugo Willefert 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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