AB Science announces that EMA granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia
April 23 2025 - 12:59PM
PRESS RELEASE
EUROPEAN MEDICINES AGENCY (EMA) HAS
GRANTED ORPHAN DRUG DESIGNATION IN THE EUROPEAN UNION FOR AB8939 IN
THE TREATMENT OF ACUTE MYELOID LEUKEMIA
Paris, April 23, 2025, 6.45pm CET
AB Science SA (Euronext -
FR0010557264 - AB) announces today that the European Medicines
Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted
orphan drug status to AB8939 for the treatment of acute myeloid
leukemia (AML).
AB8939 had already obtained orphan drug
designation from the US Food and Drug Administration (FDA) in
AML.
Granting of this orphan drug status in the EU is
a significant milestone because it means that the COMP considered
that AB8939 offers a significant benefit to people suffering from
this condition in addition to existing treatments.
Indeed, the criteria to obtain orphan drug
designation at EMA differ from those at FDA and are very stringent
for the following reasons :
-
There must be no satisfactory method of diagnosis, prevention or
treatment of the condition or, if such a method exists, the
medicinal product must offer a significant benefit to
patients.
-
Because the application is based on an assumption of significant
benefit, a comparison with authorized treatments is required.
-
Significant benefit means that a medicine produces a clinically
relevant advantage or makes a major contribution to patient care,
as compared with existing methods to treat the condition. Thus,
orphan designation is given to a product that will improve
patients’ current treatment, having considered what else is
available.
- To follow the spirit of the orphan
legislation, which makes it clear that an orphan application may be
made at any stage of the development, significant benefit will be
based on the available evidence at the stage of designation.
In support of the significant benefit of AB8939
in AML, AB Science has released preclinical data from mouse models
demonstrating a significant benefit of AB8939 treatment over
current therapies, such as cytarabine, azacitidine (Vidaza®) and
venetoclax (Venclexta®). This included:
-
Efficacy on resistant cells: AB8939 manages to have an effect on
the cancer cells (blasts) of AML patients, even when these cells
are resistant to other drugs such as vincristine or cytarabine. For
example, 45% of vincristine-resistant cells and 66% of
cytarabine-resistant cells still respond to AB8939, including in
severe cases with complex genetic mutations (MECOM, TP53).
-
Convincing results in xenograft models derived from refractory AML
patients: In these mouse models which mimick human AML, AB8939
reduces tumors and prolongs survival, even when cells are resistant
to cytarabine.
-
Additive effect with reference treatments: When used with other
treatments (cytarabine, Vidaza® or venetoclax), AB8939 further
improves results. For example, with venetoclax, it eliminates
cancer cells in the blood, spleen and bone marrow, without serious
side effects.
-
Furthermore, unlike venetoclax, AB8939 does not cause blood
toxicity (hematotoxicity) and appears to act synergistically with
other treatments, reinforcing its efficacy.
AB Science also presented preliminary efficacy
and safety data from phase 1 of AB8939 as a monotherapy, with a
3-day treatment cycle (stage 1 of phase 1) and a 14-day treatment
cycle (stage 2 of phase 1).
Professor Olivier Hermine, President of AB
Science's Scientific Committee, member of the French Academy of
Sciences and Head of the Hematology Department at Necker Hospital,
commented: "This designation testifies to the potential of AB8939
for the treatment of AML. Indeed, AB8939 has shown activity as a
monotherapy on Ara-C-resistant patient lines, including in
unfavorable genetic situations (MECOM, TP53 mutations) that have
resisted all treatments administered to date, as well as a
synergistic effect with the reference treatments Vidaza® and
Venclexta®. The ongoing Phase 1 trial will now evaluate the
combination of AB8939 with these reference treatments in refractory
patients".
About AB8939AB8939 is a new
synthetic molecule which jointly targets cancer cells, by
destabilizing the microtubules essential for cell division, and
cancer stem cells, by inhibiting enzymes (ALDH1A1 and ALDH2)
essential for maintaining their physiological state and survival.
The molecule
‘1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one’
is the chemical name of AB8939. The intellectual property of AB8939
is 100% owned by AB Science.
About the benefits of orphan drug
designationEuropean orphan drug designation is granted by
the European Commission to medicinal products intended for the
treatment of life-threatening or chronically debilitating
conditions that affect no more than 5 in 10,000 people in the
European Union (EU), or for which it must be unlikely that the
marketing of the medicinal product will generate sufficient revenue
to justify the investment required for its development. In
addition, there must be no satisfactory method of diagnosis,
prevention or treatment of the condition concerned, or, if such a
method exists, the drug must offer a significant benefit to
sufferers.An orphan drug designation in the EU confers a range of
benefits on sponsoring companies, including scientific advice on
all aspects of product development at a reduced fee, direct access
to the centralized marketing authorization procedure, and
eligibility for certain financial incentives made available by the
Community and Member States to support research and development of
orphan drugs. If the product is approved for marketing, the
designation also confers 10 years of marketing exclusivity from
product registration if the orphan drug designation still prevails
at the time of marketing authorization.In February 2018, the EMA
published a Q&A document to clear up common misunderstandings
about the meaning of orphan drug designation and other aspects
related to orphan drugs:
https://www.ema.europa.eu/en/documents/other/rare-diseases-orphan-medicines-getting-facts-straight_en.pdf
About AB ScienceFounded in
2001, AB Science is a pharmaceutical company specializing in the
research, development and commercialization of protein kinase
inhibitors (PKIs), a class of targeted proteins whose action are
key in signaling pathways within cells. Our programs target only
diseases with high unmet medical needs, often lethal with short
term survival or rare or refractory to previous line of treatment.
AB Science has developed a proprietary portfolio of molecules and
the Company’s lead compound, masitinib, has already been registered
for veterinary medicine and is developed in human medicine in
oncology, neurological diseases, inflammatory diseases and viral
diseases. The company is headquartered in Paris, France, and listed
on Euronext Paris (ticker: AB).
Further information is available on AB Science’s
website: www.ab-science.com.
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These forward-looking statements can often be
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While AB Science believes these forward-looking statements are
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forward-looking information and statements. These risks and
uncertainties include the uncertainties related to product
development of the Company which may not be successful or to the
marketing authorizations granted by competent authorities or, more
generally, any factors that may affect marketing capacity of the
products developed by AB Science, as well as those developed or
identified in the public documents published by AB Science. AB
Science disclaims any obligation or undertaking to update the
forward-looking information and statements, subject to the
applicable regulations, in particular articles 223-1 et seq. of the
AMF General Regulations.
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