- REGENERATE-PD, which is now enrolling, will study AB-1005, an
adeno-associated virus 2 (AAV2) glial cell line-derived
neurotrophic factor (GDNF) gene therapy for the treatment of
moderate-stage Parkinson’s disease
- Follows January announcement that Phase 1b trial of AB-1005 met
its primary endpoint, and presentation of 18-month results at AAN
in April 2024
- Enrolment will initially begin in the United States, with
clinical trials sites in the UK and Europe opening late
Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy
company wholly owned and independently operated as a subsidiary of
Bayer AG, today announced initiation of recruitment to
REGENERATE-PD, a Phase 2 clinical study assessing efficacy and
safety of AB-1005, an investigational adeno-associated virus 2
(AAV2) glial cell line-derived neurotrophic factor (GDNF) gene
therapy for the treatment of moderate-stage Parkinson’s
disease.
“By enhancing levels of a naturally occurring growth factor,
glial cell line-derived neurotrophic factor (GDNF) gene therapy is
intended to promote the survival and functioning of vulnerable
brain cells that degenerate in Parkinson’s disease”, said Alan
Whone, MD, PhD, Consultant Senior Lecturer in Movement Disorder,
Bristol Medical School, and Honorary Consultant Neurologist at
North Bristol Trust, UK. “The advancement of AB-1005 is a
significant milestone in the development of a gene therapy for
Parkinson’s disease and has the potential to bring an effective
treatment one step closer to patients”. Dr Whone will act as
European Lead Principal Investigator (PI) on REGENERATE-PD once the
program becomes active in the UK.
According to the Parkinson’s Foundation, more than 10 million
people worldwide suffer from Parkinson’s disease.1
“Following the encouraging results from the Phase 1b study and
the presentation of 18-month data at the American Association of
Neurology (AAN) meeting in April, we are excited to be progressing
AB-1005 to this larger, Phase 2 study,” said Krystof Bankiewicz,
MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “This
latest advancement highlights our confidence in the potential of
AB-1005 to provide a transformative impact for patients with
Parkinson’s disease.”
In January, AskBio announced that the Phase 1b trial of AB-1005
met its primary endpoint, which was to evaluate the safety of a
one-time bilateral delivery of AB-1005 directly to the putamen. The
investigational gene therapy for the treatment of Parkinson’s
disease was well tolerated with no serious adverse events that were
considered related to AB-1005 in all 11 patients at 18 months.2
AskBio is also exploring GDNF therapy beyond Parkinson’s disease
and is currently enrolling patients in the US with the parkinsonian
subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to
assess the preliminary safety, tolerability, and efficacy of GDNF
therapy for this rapidly progressing condition.3
AB-1005 is an investigational gene therapy that has not been
approved by any regulatory authority, and its efficacy and safety
have not been established or fully evaluated.
About Parkinson’s disease
Parkinson’s disease is a progressive neurodegenerative disorder
caused by the death of nerve cells in the brain, leading to
decreased dopamine levels.4 At diagnosis, it is estimated that
patients have already lost 50-80% of their dopaminergic
neurons.5 The loss of these neurons leads to a progressive
loss of motor function and symptoms such as tremors, muscle
rigidity, and slowness of movement.6 Even with medication, the
symptoms of Parkinson’s disease can fluctuate during the course of
the day. According to the Parkinson’s Foundation, more than 10
million people worldwide suffer from Parkinson’s disease, with
approximately one million living in the United States.1 There
is no cure, and the effectiveness of current treatments frequently
decreases over time.7
About REGENERATE-PD
REGENERATE-PD is a Phase 2, randomized, double-blind,
surgery-controlled study of the efficacy and safety of
intraputaminal AAV2-GDNF in the treatment of adults (45-75 years)
with moderate stage Parkinson's disease. Subjects will
receive either bilateral image-guided infusion of AAV2-GDNF into
the putamen, single dose (active treatment arm), or bilateral
partial burr/twist holes (control arm). The trial will include an
estimated 87 subjects with study sites located in the United
States, UK and Europe. For more information about the REGENERATE-PD
clinical trial, visit clinicaltrials.gov (NCT06285643), or visit
www.askbio.com.
About AB-1005
AB-1005 is an investigational gene therapy based on
adeno-associated viral vector serotype 2 (AAV2) containing the
human glial cell line-derived neurotrophic factor (GDNF) transgene,
which allows for stable and continuous expression of GDNF in
localized regions of the brain after direct neurosurgical injection
with MRI-monitored convection enhanced delivery8,9 GDNF is a
homodimer that is a distantly related member of the transforming
growth factor-β superfamily. In midbrain neuronal cell cultures,
recombinant human GDNF promoted the survival and morphological
differentiation of dopaminergic neurons and increased their
high-affinity dopamine uptake. GDNF has long been evaluated as a
potential treatment for diseases, such as Parkinson’s, marked by
progressive degeneration of midbrain dopaminergic neurons.10
About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and
independently operated subsidiary of Bayer AG, is a fully
integrated gene therapy company dedicated to developing life-saving
medicines and changing lives. The company maintains a portfolio of
clinical programs across a range of neuromuscular, central nervous
system, cardiovascular, and metabolic disease indications with a
clinical-stage pipeline that includes therapeutics for congestive
heart failure, Huntington’s disease, limb-girdle muscular
dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe
disease. AskBio’s gene therapy platform includes Pro10™, an
industry-leading proprietary cell line manufacturing process, and
an extensive capsid and promoter library. With global headquarters
in Research Triangle Park, North Carolina, and European
headquarters in Edinburgh, Scotland, the company has generated
hundreds of proprietary capsids and promoters, several of which
have entered pre-clinical and clinical testing. An early innovator
in the gene therapy field, with over 900 employees in five
countries, the company holds more than 800 patents and patent
applications in areas such as AAV production and chimeric capsids.
Learn more at www.askbio.com or follow us on LinkedIn.
About Bayer
Bayer is a global enterprise with core competencies in the life
science fields of health care and nutrition. In line with its
mission, “Health for all, Hunger for none,” the company’s products
and services are designed to help people and the planet thrive by
supporting efforts to master the major challenges presented by a
growing and aging global population. Bayer is committed to driving
sustainable development and generating a positive impact with its
businesses. At the same time, the Group aims to increase its
earning power and create value through innovation and growth. The
Bayer brand stands for trust, reliability and quality throughout
the world. In fiscal 2023, the Group employed around 100,000 people
and had sales of 47.6 billion euros. R&D expenses before
special items amounted to 5.8 billion euros. For more information,
go to www.bayer.com.
AskBio Forward-Looking Statements
This press release contains “forward-looking statements.” Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Words such as “believes,” “anticipates,” “plans,” “expects,”
“will,” “intends,” “potential,” “possible,” and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include, without limitation, statements
regarding AskBio’s clinical trials. These forward-looking
statements involve risks and uncertainties, many of which are
beyond AskBio’s control. Known risks include, among others: AskBio
may not be able to execute on its business plans and goals,
including meeting its expected or planned clinical and regulatory
milestones and timelines, its reliance on third-parties, clinical
development plans, manufacturing processes and plans, and bringing
its product candidates to market, due to a variety of reasons,
including possible limitations of company financial and other
resources, manufacturing limitations that may not be anticipated or
resolved in a timely manner, potential disagreements or other
issues with our third-party collaborators and partners, and
regulatory, court or agency feedback or decisions, such as feedback
and decisions from the United States Food and Drug Administration
or the United States Patent and Trademark Office. Any of the
foregoing risks could materially and adversely affect AskBio’s
business and results of operations. You should not place undue
reliance on the forward-looking statements contained in this press
release. AskBio does not undertake any obligation to publicly
update its forward-looking statements based on events or
circumstances after the date hereof.
Find more information about AskBio
at www.askbio.com
References
[1] Parkinson’s Foundation. Who has Parkinson’s? Available
at: https://www.parkinson.org/understanding-parkinsons/statistics.
Accessed: June 2024.
[2] Asklepios BioPharmaceutical, Inc. (January 2024). AskBio
Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s
disease meets primary endpoint [Press Release].
https://www.askbio.com/askbio-phase-ib-trial-of-ab-1005-gene-therapy-in-patients-with-parkinsons-disease-meets-primary-endpoint
[3] ClinicalTrials.gov. GDNF Gene Therapy for Multiple System
Atrophy. Available at:
https://clinicaltrials.gov/study/NCT04680065. Accessed: June
2024.
[4] Michael J. Fox Foundation. Parkinson’s 101 – What is
Parkinson’s disease? Available
at: https://www.michaeljfox.org/parkinsons-101.
Accessed: June 2024.
[5] DeMaagd G, Philip A. Parkinson’s Disease and Its Management:
Part 1: Disease Entity, Risk Factors, Pathophysiology, Clinical
Presentation, and Diagnosis. P T. 2015 Aug;40(8):504-32. PMID:
26236139; PMCID: PMC4517533.
[6] Parkinson’s Foundation. Motor-fluctuations. Available
at: https://www.parkinson.org/library/fact-sheets/motor-fluctuations.
Accessed: June 2024.
[7] Mayo Clinic. Parkinson’s disease. Available
at: https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062.
Accessed: June 2024.
[8] Heiss JD, Lungu C, Hammoud DA, Herscovitch P, Ehrlich DJ,
Argersinger DP, Sinharay S, Scott G, Wu T, Federoff HJ, Zaghloul
KA, Hallett M, Lonser RR, Bankiewicz KS. Trial of magnetic
resonance-guided putaminal gene therapy for advanced Parkinson’s
disease. Mov Disord. 2019 Jul;34(7):1073-1078.
[9] Kells AP, Eberling J, Su X, Pivirotto P, Bringas J, Hadaczek
P, Narrow WC, Bowers WJ, Federoff HJ, Forsayeth J, Bankiewicz KS.
Regeneration of the MPTP-lesioned dopaminergic system after
convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul
14;30(28):9567-77.
[10] Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F. GDNF: a
glial cell line-derived neurotrophic factor for midbrain
dopaminergic neurons. Science. 1993;260(5111):1130-1132.
Andrew Thomas
Asklepios BioPharmaceutical, Inc. (AskBio)
+44 (7803) 585254
athomas-c@askbio.com