Azafaros announces positive topline Phase 2 study data with nizubaglustat in GM2 gangliosidosis and Niemann-Pick disease type C
16 July 2024 - 9:00AM
Business Wire
- Study designed to support safety profile showed nizubaglustat
was safe and well tolerated
- Results support continued development of nizubaglustat for GM2
gangliosidosis and Niemann-Pick disease type C with Phase 3
trial[s] planned for 2025
- Data to be presented at the SSIEM Annual Symposium 2024 to be
held 3-6 September in Porto, Portugal
Azafaros B.V. today announced positive topline data from its
RAINBOW study, a Phase 2 clinical trial investigating nizubaglustat
in patients with a genetic diagnosis of either GM2 gangliosidosis
or Niemann-Pick disease type C (NPC).
About the Trial
The trial was conducted across three sites in Brazil, involving
13 patients older than 12 years of age, with the aim of determining
the safety, pharmacodynamics, and pharmacokinetics of two different
doses of nizubaglustat. The results will guide the identification
of the target dose for Azafaros’ planned Phase 3 pivotal
studies.
With the 12-week, double-blind and placebo-controlled part of
the study complete, patients have now entered the extension phase,
where all participants receive the study drug.
The data from the RAINBOW study will be presented at the SSIEM
Annual Symposium 2024, which will take place on 3 - 6 September in
Porto, Portugal.
Stefano Portolano, CEO at Azafaros, said: “The results of the
Phase 2 RAINBOW study mark a pivotal moment in the development of
nizubaglustat. The dual mode of action sets nizubaglustat apart as
a leap forward from other agents. We are excited by the results of
the study and more than confident that nizubaglustat has the
potential to become a convenient, life-changing therapy for
patients affected by GM1 and GM2 gangliosidoses and NPC. Azafaros
is thankful to the patients and their families for their important
contribution to the RAINBOW study. Azafaros is now poised to embark
on a global Phase 3 trial next year.”
Lead RAINBOW investigator, Roberto Giugliani, added: “We are
pleased to report the completion of this important step in the
development of a new therapy for GM2 gangliosidosis and
Niemann-Pick disease type C, which delivers data to determine the
best dosing of nizubaglustat and allows the planning of the next
phase.”
In a statement, a representative from the Associação
Niemann-Pick & Batten Brasil (ANPB), a patient organisation in
Brazil, said there is an urgent need for new treatments for
NPC.
"Current therapeutic options are limited and do not offer a
definitive cure. Clinical research plays a crucial role in this
process, allowing the development and evaluation of new therapies
that can improve patients' quality of life and slow the progression
of the disease. Therefore, we are excited about developing
innovative and convenient therapies.”
About nizubaglustat
Nizubaglustat is a small molecule, orally available and brain
penetrant azasugar with a unique dual mode of action, developed as
a potential treatment for rare lysosomal storage disorders with
neurological involvement, including GM1 and GM2 gangliosidoses and
Niemann-Pick disease type C (NPC).
Nizubaglustat has received the following designations and
support:
United States Food and Drug Administration (FDA)
Rare Pediatric Disease Designations (RPDD) for the treatment of
GM1 and GM2 gangliosidoses and NPC.
Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff
and Tay-Sachs Diseases) and NPC.
IND Clearance and Fast Track Designation for GM1/GM2
gangliosidoses and NPC
European Medicines Agency (EMA)
Orphan Medicinal Product Designation (OMPD) for the treatment of
GM2 gangliosidosis.
UK Medicines and Healthcare Products Regulatory Agency
(MHRA)
Innovation Passport for the treatment of GM1 and GM2
gangliosidoses.
About GM1 and GM2 Gangliosidoses
GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and
Sandhoff diseases) are lysosomal storage disorders caused by the
accumulation of GM1 or GM2 gangliosides respectively, in the
central nervous system (CNS), resulting in progressive and severe
neurological impairment and early death. These diseases mostly
affect infants and children, and no disease-modifying treatments
are currently available.
About Niemann-Pick Disease Type C (NPC)
Niemann-Pick disease type C (NPC) is a progressive,
life-limiting neurological lysosomal storage disorder caused by
mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal
trafficking, leading to the accumulation of various lipids,
including gangliosides in the CNS. The onset of disease can happen
throughout the lifespan of an affected individual, from prenatal
life through adulthood.
About Azafaros
Azafaros is a clinical-stage company founded in 2018 by
scientists with a deep understanding of rare genetic disease
mechanisms, using discoveries made by scientists at Leiden
University and Amsterdam UMC. Azafaros is led by a team of highly
experienced industry experts and aims to build a pipeline of
disease-modifying therapeutics to offer new treatment options to
patients and their families. The Azafaros team is dedicated to
rapidly bring new drugs to the rare disease patients who need them.
The company is supported by a syndicate of leading Dutch and Swiss
investors including Forbion, BioGeneration Ventures (BGV),
BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders
Capital.
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