Attralus Receives European Medicines Agency Committee for Orphan Medicinal Products (COMP) Positive Opinions for AT-02 for the Treatment of Both ATTR and AL Amyloidosis
2024年7月29日 - 8:00PM
Attralus, Inc., a clinical stage biopharmaceutical company
developing transformative medicines to improve the lives of
patients with systemic amyloidosis, announced today that the
European Medicines Agency’s (EMA) Committee for Orphan Medicinal
Products (COMP) has adopted positive opinions for orphan medicinal
product designations for AT-02 for the treatment of the two most
common forms of systemic amyloidosis, transthyretin-associated
amyloidosis (ATTR) and immunoglobulin light-chain-associated (AL)
amyloidosis.
AT-02, the company’s lead pan-amyloid removal
therapeutic candidate, is currently being evaluated in a three-part
Phase 1 clinical trial and a Phase 2 open label extension trial in
ATTR and AL amyloidosis patients to support its use as an
immunotherapy in patients with systemic amyloidosis. These
designations granted for each of the two indications support the
unique properties of AT-02 to potentially treat ATTR and AL
amyloidosis, as well as other types of systemic amyloidosis. AT-02
is the first and only therapeutic to receive positive opinions for
orphan medicinal product designation for both ATTR and AL
amyloidosis from the COMP.
“We are pleased to receive positive opinions on
our requests for orphan medicinal product designations from the
COMP for AT-02 in ATTR and AL, further supporting our efforts to
develop a potentially transformative therapy for systemic
amyloidosis,” said Gregory Bell, M.D., Chief Medical Officer at
Attralus. “Current approved therapies for systemic amyloidosis
target precursor protein production, reducing the formation of new
amyloid, but there is a significant unmet need for new therapies
that can remove the existing toxic amyloid fibrils that cause organ
damage and mortality.”
In the European Union (EU), orphan medicinal
product designation is granted to drugs and biologics intended for
treating, diagnosing, or preventing rare, life-threatening or
chronically debilitating conditions. To qualify, the condition’s
prevalence in the EU must be no more than five in 10,000 people. If
satisfactory diagnosis, prevention, or treatment methods already
exist for the disease, orphan designation may only be granted when
the drug or biologic has the potential to provide significant
benefit over existing therapies. Following the adoption of a
positive opinion by the COMP, the legally binding final decision on
the granting of orphan designation is issued by the European
Commission. Following Commission Decision, orphan designation
allows companies certain benefits, including a special category of
scientific advice, reduced regulatory fees, research grants and up
to ten years of market exclusivity in the EU if a product is
approved. When combined with the EU Small or Medium-Sized
Enterprise (SME) status granted to Attralus earlier this year,
orphan-designated programs receive additional administrative and
procedural assistance from the Agency's SME office and even greater
fee reductions.
About AT-02, Pan-Amyloid Removal
TherapeuticAT-02 is the company’s lead pan-amyloid removal
(PAR) therapeutic candidate for systemic amyloidosis. AT-02 is a
humanized IgG1 monoclonal antibody genetically fused with the
company’s proprietary pan-amyloid binding peptide, enabling binding
to multiple types of amyloid deposits. The Fc region of the
antibody stimulates the immune system to remove amyloid deposits
that are bound by AT-02. AT-02 uses a similar pan-amyloid peptide
to 124I-evuzamitide, the company’s diagnostic agent, which has been
shown in multiple clinical trials to selectively bind to amyloid
deposits in the heart, liver, kidney, and other organs in multiple
types of amyloidosis. As a result, the company expects AT-02 to
bind specifically to amyloid in systemic amyloidosis patients.
Preclinical data have shown the ability of AT-02 to bind to
multiple amyloid types in major organs induce macrophage mediated
amyloid phagocytosis and amyloid removal. AT-02 is currently being
evaluated in a 3-part Phase 1 trial and a Phase 2 open label
extension trial in ATTR and AL amyloidosis patients.
About Systemic
Amyloidosis Systemic amyloidosis encompasses a
diverse group of rare diseases that occur due to accumulation of
toxic amyloid deposits in tissues and organs, a consequence of
aberrant protein misfolding events. These diseases are progressive,
debilitating and often fatal. Systemic amyloidosis is significantly
underdiagnosed due to low awareness, lack of specific symptoms, and
no current disease-specific diagnostics. There are approximately 17
different types of systemic amyloidosis, in the United States, the
European Union and Japan. The two most common forms of systemic
amyloidosis are transthyretin-associated amyloidosis (ATTR) and
immunoglobulin light-chain-associated (AL) amyloidosis. While
currently approved treatments slow disease progression by targeting
precursor proteins, there is a significant unmet need for new
therapies that can remove toxic amyloid deposits across all amyloid
types and improve organ function and patient quality of life.
About
Attralus Attralus is a clinical stage
biopharmaceutical company focused on creating transformative
medicines to improve the lives of patients with systemic
amyloidosis. The company’s proprietary pan-amyloid removal (PAR)
therapeutics are designed to directly bind to and remove toxic
amyloid in organs and tissues. By targeting the disease-causing
pathology in systemic amyloidosis diseases, PAR therapeutics have
the potential to treat and reverse disease in patients with all
types and stages of systemic amyloidosis. Attralus was founded by
scientific experts in the field of amyloidosis and the company is
headquartered in Burlingame, CA.
Forward-Looking
Statements
This press release contains forward-looking
statements, including statements related to the efficacy, continued
development, and potential of AT-01. Words such as “developing,”
“potential,” “shown” and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements are based upon Attralus’ current expectations.
Forward-looking statements involve risks and uncertainties.
Attralus’ actual results and the timing of events could differ
materially from those anticipated in such forward-looking
statements as a result of these risks and uncertainties. Attralus
expressly disclaims any obligation or undertaking to release
publicly any updates or revisions to any forward-looking statements
contained herein to reflect any change in Attralus’ expectations
with regard thereto or any change in events, conditions, or
circumstances on which any such statements are based.
Contact:Krishna Gorti, M.D.
FRCSCorporate Developmentkgorti@attralus.com