The trial should include around 45 participants
with Down syndrome (trisomy 21) and should be completed in the
second quarter of 2023
Regulatory News:
Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS, PEA-PME
eligible), a clinical-stage biopharmaceutical company specializing
in the development of treatments for brain diseases, announces
today the recruitment of the first patient for its phase 1/2 trial
of AEF0217 in participants with Down syndrome.
AEF0217 is Aelis Farma’s second drug candidate. It belongs to a
new class of drugs discovered by the company, signaling-specific
inhibitors of the CB1 receptor of the endocannabinoid system
(CB1-SSi), a receptor implicated in many brain disorders. AEF0217
is being developed as the first treatment of cognitive impairments
caused by a hyperactivity of the CB1 receptor and, as a first
indication, of those associated with Down syndrome (trisomy 21).
AEF0217, like other CB1-SSis, is able to inhibit only certain
components of CB1’s activity, which seems to enable this compound
to counteract a pathological hyperactivity of the receptor without
altering its normal physiological functions and, as a consequence,
without causing major side effects.
Aelis Farma recently published the positive results of the phase
1 clinical trials analyzing the safety and pharmacokinetics of
AEF0217 in healthy volunteers. These studies have confirmed the
particularly favorable safety profile of CB1-SSi, a particularly
important characteristic for fragile populations such as those with
Down syndrome. The results of these trials showed that all doses of
AEF0217 were well tolerated without any serious adverse effects and
that AEF0217 has a favorable pharmacokinetic profile. These
positive results allow now to administer AEF0217 to people with
Down syndrome.
The phase 1/2 clinical trial with AEF0217 is a monocentric,
double-blind study comparing one dose of AEF0217 to placebo, after
28 days of treatment once a day in approximately 45 participants
with Down syndrome. The main objective of the trial is to assess
the safety and absorption of AEF0217 and it could also provide the
first indications of activity as a treatment for the cognitive
impairments of Down syndrome.
The first patient has been recruited by the teams of Prof.
Rafael de la Torre Fornell at the Hospital del Mar Medical Research
Institute (IMIM) in Barcelona (Spain). Depending on the rate of
recruitment, the study should be completed in the second quarter of
2023.
Prof. Rafael de la Torre Fornell, the trial’s principal
investigator, explains: “Administering AEF0217 for the first
time to a person with Down syndrome is a key event for me and my
teams. It is a crucial step towards developing a treatment that can
improve the cognitive abilities of these charming and unique people
that desperately need it”.
Pier Vincenzo Piazza, CEO of Aelis Farma, concludes: “I
would like to congratulate the teams of Prof. Rafael de la Torre
Fornell and of Aelis Farma for this major step forward in the
development of AEF0217, a drug candidate based on our innovative
approach that allows to selectively inhibit only part of the
activity of the CB1 receptor. If the results are favorable, we will
be significantly closer to having a promising therapy that could
considerably improve the quality of life of people with Down
syndrome, and also potentially pave the way for the treatment of
other cognitive impairments”.
About the AEF0217 clinical program for the treatment of
cognitive disorders in Down syndrome: the European ICOD
project.
The phase 1/2 trial of AEF0217 is part of the European H2020
ICOD project (Improving COgnition in Down syndrome, Grant Agreement
ID 899986), and is being run in collaboration with the Hospital del
Mar Medical Research Institute (IMIM) in Barcelona (Spain) and
Prof. Rafael de la Torre Fornell, project coordinator and principal
investigator of the trial. In February 2021, the ICOD project
received funding of €6 million from the European Commission to
finance the clinical development of AEF0217 for the treatment of
cognitive impairments of 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. By reproducing this natural
mechanism, CB1-SSi appear to be able to selectively inhibit the
disease-related activity of the CB1 receptor, without disrupting
its normal physiological activity. 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 has entered a phase 2b clinical trial
in Q2 2022, which will include 330 patients in 9 clinical centers
in the United States. 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
carry any 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 (trisomy 21), has successfully
completed safety and pharmacokinetic trials (phase 1 clinical
program) in healthy volunteers and started in December 2022 a phase
1/2 trial in people with Down syndrome. This new study will assess
the safety and the pharmacokinetics of AEF0217 and could also
provide the first indications of its activity as a treatment of
cognitive disorders. 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
animals. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221215005975/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
Aelis Farma (EU:AELIS)
Historical Stock Chart
Von Feb 2023 bis Mär 2023
Aelis Farma (EU:AELIS)
Historical Stock Chart
Von Mär 2022 bis Mär 2023
