The 2nd European Conference on Phelan-McDermid
Syndrome was held in Madrid from June 9 to 11, 2023
Regulatory News:
Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS), a
clinical-stage biopharmaceutical company specialized in the
development of treatments for brain diseases, today announced the
presentation of new preclinical data on its drug candidate AEF0217
at the 2nd European Conference on Phelan-McDermid Syndrome, which
was held from June 9 to 11, 2023 at CEU San Pablo University in
Madrid1.
Phelan-McDermid Syndrome (PMS), which is caused by the deletion
of chromosome 22q13 including the SHANK3 gene or by a sequence
variation in this gene, is one of the most frequently observed
genetic mutations in autism. It is an orphan disease for which
there is currently no treatment. In affected people, these
mutations can lead to development delays in multiple areas, in
particular delayed speech, intellectual disability and often autism
spectrum disorder.
Dr. Pier Vincenzo Piazza, CEO of Aelis Farma, alongside Flavio
Tomasi, PhD student in Dr. Catalina Betancur’s INSERM/CNRS
laboratory at the Sorbonne university, gave an oral communication
entitled: “Inhibition of the cannabinoid CB1 receptor rescues
deficits in a mouse model of Phelan-McDermid syndrome” at the 2nd
European Conference on Phelan-McDermid Syndrome, organized by the
Spanish Phelan-McDermid Syndrome Association.
The presented data was obtained within the context of a
collaboration between several laboratories coordinated by Dr.
Betancur. The results showed the ability of AEF0217 to
statistically significantly reverse behavioral, cognitive and motor
deficits observed in a genetic mouse model of Phelan-McDermid
Syndrome. In these mice, ARF0217 also reversed a neurological
alteration (cortical hyperactivity), considered as a
neurobiological marker of autism. Based on these promising results
Aelis will now analyze the feasibility to develop of AEF0217 in
this indication and more generally in autism spectrum disorder.
Pier Vincenzo Piazza, CEO of Aelis Farma, said: “The
preclinical data obtained by Dr. Betancur’s group have generated
considerable excitement in the scientific community present at the
meeting and encourage us to evaluate, in addition to the ongoing
program in trisomy 21, the feasibility of developing AEF0217 in
this new indication. More generally, the data suggest that AEF0217
could also help patients with autism spectrum disorder, which would
significantly expand the fields of application of our second drug
candidate.”
Dr. Catalina Betancur of the Neuroscience Paris Seine
laboratory (INSERM, CNRS, Sorbonne University, Paris), who
coordinated the studies presented, commented: “We are delighted
to have had the opportunity to present the results of the effects
of AEF0217 on a mouse model of Phelan-McDermid Syndrome at this
conference, which brings together the most prominent international
experts in this rare disease for which there is no effective
treatment. Our preclinical data have been very favorably received
by scientists and families attending the conference and encourage
us to push forward the evaluation of AEF0217 in autism spectrum
disorder. We are delighted to be participating in the development
of a potential new treatment for a population of patients in dire
need.”
AEF0217 is the second drug candidate developed by Aelis Farma.
It belongs to a new generation of drugs discovered by the Company,
the Signaling-Specific inhibitors of the CB1 receptor of the
endocannabinoid system (CB1-SSi). The CB1 is one of the most
expressed neurotransmitter receptors in the brain implicated in
many diseases. AEF0217 is currently being developed for the
treatment of cognitive disorders in people with Down syndrome
(trisomy 21) and evaluated in a phase 1/2 study in this
population.
About Phelan-McDermid Syndrome2
Phelan-McDermid Syndrome (PMS) is one of the most frequent
genetic causes of autism spectrum disorder. It is an orphan disease
resulting from the loss of genetic material at the terminal end of
chromosome 22 (22q13 deletion) or from a mutation in the SHANK3
gene. The genetic characteristic that affected people have in
common is the absence or the mutation of the SHANK3 gene. The
absence or the mutation of a copy of this gene results in a general
developmental delay, and in particular in a delayed or absent
speech, intellectual disability, autism spectrum disorder and motor
skill deficits. Some affected people may also suffer from epilepsy.
This mutation generally occurs spontaneously and it is not
hereditary.
About the European Conference on Phelan-McDermid
Syndrome
This conference, organized by the Spanish Phelan-McDermid
Syndrome Association, aims to inform professionals and families
about the latest advances in the scientific research and care of
Phelan-McDermid Syndrome (PMS). It brings together international
experts in various disciplines and provides the families of people
with this syndrome an opportunity to discuss the difficulties and
challenges they face.
***
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 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 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 people
with Down syndrome in Spain. The Company also has a portfolio of
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
Aelis Farma is one of the partners of this
conference.
2
Sources: Phelan-McDermid Syndrome
Associations in Spain and France
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230611306798/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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