Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced
that the first patient has been dosed in the Phase 1/2 GALILEO-1
clinical trial evaluating FLT201, its adeno-associated virus (AAV)
gene therapy candidate, in Gaucher disease type 1. Gaucher disease
is a debilitating genetic disorder in which a deficiency of the
GCase enzyme leads to a buildup of fatty substances in the organs,
causing symptoms including enlarged spleen and liver, low blood
counts, bone pain and reduced lung function.
“We believe that FLT201 has life-changing potential for people
with the most common type of Gaucher disease,” said Pamela Foulds,
M.D., Chief Medical Officer at Freeline. “Existing therapies do not
sufficiently penetrate deeper tissues, and enzyme levels wane in
between infusions, giving harmful substrates the opportunity to
build back up in all disease-affected organs. FLT201 is a highly
differentiated gene therapy candidate for Gaucher disease type 1
that delivers a longer-acting, rationally engineered enzyme.
Preclinically, FLT201 has been shown to produce sustained levels of
enzyme and penetrate deeper tissues, including bone and lung,
giving us reason to believe it has the opportunity to stop disease
progression and improve outcomes with a one-time treatment.”
“The dosing of the first patient in the GALILEO-1 trial of
FLT201 marks an important step forward for the Gaucher community,”
said Dr. Ozlem Goker-Alpan, founder and CEO of the Lysosomal and
Rare Disorder Research and Treatment Center (LDRTC) and an
investigator in GALILEO-1. “There is a clear need for better
treatment options. Existing therapies come with a heavy lifelong
treatment burden, and many patients continue to experience serious
symptoms. FLT201 represents a promising approach for addressing the
underlying cause of Gaucher disease with a one-time gene therapy,
potentially providing a more effective and less burdensome option
for patients. I look forward to further evaluating FLT201 in this
clinical trial.”
GALILEO-1 is a first-in-human,
international, multicenter Phase 1/2 dose-escalation study. The
trial will assess safety, tolerability and efficacy of a single
intravenous dose of FLT201 in up to four cohorts of escalating
doses, with the aim of identifying a dose for further development
in a Phase 3 clinical trial. Visit clinicaltrials.gov to learn more
about GALILEO-1.
Freeline expects to report initial data, with a focus on safety
and enzyme activity, from the first cohort of GALILEO-1 in the
third quarter of this year.
Extending Innovation into GBA1-linked Parkinson’s
Disease Freeline unveiled its research program in
GBA1-linked Parkinson’s disease. The program builds on its work
with Gaucher disease, leveraging its rationally engineered
longer-acting GCase variant to extend the impact of its innovation
to develop a gene therapy candidate for a subset of Parkinson’s
disease patients with mutations in the GBA1 gene, which encodes for
GCase. As in Gaucher disease, the GBA1 mutations lead to a
deficiency of GCase and the accumulation of pathological
substrates. GBA1 mutations increase the risk of developing
Parkinson’s disease by up to 30-fold and are associated with
earlier onset of disease, more severe symptoms and increased
likelihood of progression to dementia.
“Our GBA1-linked Parkinson’s disease program is a natural
extension of our work in Gaucher disease and an opportunity to
extend the therapeutic potential of our longer-acting GCase variant
into a genetically defined patient population with a serious unmet
need,” said Michael Parini, Chief Executive Officer at Freeline.
“There are no approved disease modifying therapies for Parkinson’s
disease, and symptomatic treatments become less effective as the
disease progresses. Preclinically, our GCase variant has
demonstrated at least 20-fold greater activity levels compared to
wildtype enzyme in various cell lines, including brain epithelial
and neuroblastoma cells, and our goal is to leverage our
longer-acting GCase variant to create a life-changing gene therapy
for this patient population.”
About
Gaucher DiseaseGaucher disease is caused by a mutation in
the GBA1 gene that results in abnormally low levels of
glucocerebrosidase (GCase), an enzyme needed to metabolize a
certain type of lipid. As a result, harmful substrates
glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up
in cells that then accumulate in various organs, causing
inflammation and dysfunction. Gaucher disease is hereditary and
presents in various subtypes. Freeline is currently focused on
Gaucher disease type 1, the most common form of the disease, which
affects the health of the spleen, liver, bone and lungs. Despite
treatment with existing therapies, many people with Gaucher disease
continue to experience symptoms and disease progression. Gaucher
disease affects approximately 18,000 people in the United States,
United Kingdom, France, Germany, Spain, Italy and Israel.
About GBA1-linked Parkinson’s
DiseaseParkinson’s disease (PD) is a progressive
neurodegenerative disorder that results in tremors, muscle
rigidity, difficulty walking, anxiety, depression and cognitive
impairments. Approximately 5-15% of PD patients have mutations in
the GBA1 gene, which encodes for the glucocerebrosidase (GCase)
enzyme. The most common genetic risk factor for PD, GBA1 mutations
increase the risk of developing PD by 5- to 30-fold. GBA1 mutations
are also associated with earlier onset and more severe disease.
There are no approved disease-modifying therapies for PD, and
current treatments, which focus on managing symptoms, become less
effective over time. Freeline estimates GBA1-linked PD affects
approximately 190,000 patients in the United States, United
Kingdom, France, Germany, Spain and Italy.
About FLT201 FLT201
is an adeno-associated virus (AAV) gene therapy candidate that is
currently being investigated in the GALILEO-1 Phase 1/2 clinical
trial in adults with Gaucher disease type 1. FLT201 is designed to
generate durable increases in glucocerebrosidase (GCase) and reduce
the accumulation of harmful substrates, with the aim of providing a
one-time treatment that can stop disease progression, improve
outcomes and free people from lifelong treatment. FLT201 uses
Freeline’s proprietary AAVS3 capsid to introduce a novel transgene
into liver cells to produce a rationally engineered GCase variant.
In preclinical studies, the GCase variant has demonstrated a
20-fold increase in half-life at lysosomal pH conditions compared
to wildtype human GCase. Preclinically, FLT201 has shown robust
GCase expression, as well as significant GCase uptake and substrate
reduction in key tissues.
About Freeline
TherapeuticsFreeline is a clinical-stage biotechnology
company focused on developing transformative gene therapies for
chronic debilitating diseases. Freeline uses its proprietary,
rationally designed AAV vector and capsid (AAVS3), along with novel
promoters and transgenes, to deliver a functional copy of a
therapeutic gene into human liver cells, thereby expressing a
persistent functional level of the missing or dysfunctional protein
into a patient’s bloodstream. The company is currently advancing
FLT201, a highly differentiated gene therapy candidate that
delivers a novel transgene, in a Phase 1/2 clinical trial in people
with Gaucher disease type 1. Freeline has additional programs in
research, including one focused on GBA1-linked Parkinson’s disease
that leverages the same novel transgene as FLT201. Freeline is
headquartered in the UK and has operations in the United States.
For more information, visit www.freeline.life or connect with
Freeline on LinkedIn and Twitter.
Forward-Looking
StatementsThis press release contains statements that
constitute “forward-looking statements” as that term is defined in
the United States Private Securities Litigation Reform Act of 1995,
including statements that express the opinions, expectations,
beliefs, plans, objectives, assumptions or projections of Freeline
Therapeutics Holdings plc (the “Company”) regarding future events
or future results, in contrast with statements that reflect
historical facts. All statements, other than historical facts,
including statements regarding FLT201’s potential to be a first-
and best-in-class gene therapy, stop disease progression and
improve outcomes with a one-time treatment, that the Company
expects to report initial data from the first cohort of the
GALILEO-1 Phase 1/2 clinical trial of FLT201 in the third quarter
of this year, and regarding the potential to extend the Company’s
GCase variant into GBA1-linked Parkinson’s disease and create a
life-changing gene therapy for this patient population are
forward-looking statements. In some cases, you can identify such
forward-looking statements by terminology such as “anticipate,”
“intend,” “believe,” “estimate,” “plan,” “seek,” “project,”
“expect,” “may,” “will,” “would,” “could” or “should,” the negative
of these terms or similar expressions. Forward-looking statements
are based on management’s current beliefs and assumptions and on
information currently available to the Company, and you should not
place undue reliance on such statements. Forward-looking statements
are subject to many risks and uncertainties, including the
Company’s recurring losses from operations; the uncertainties
inherent in research and development of the Company’s product
candidates, including statements regarding the timing of
initiation, enrollment, continuation, completion and the outcome of
clinical studies or trials and related preparatory work and
regulatory review, regulatory submission dates, regulatory approval
dates and/or launch dates, as well as risks associated with
preclinical and clinical data, including the possibility of
unfavorable new preclinical, clinical or safety data and further
analyses of existing preclinical, clinical or safety data; the
Company’s ability to design and implement successful clinical
trials for its product candidates; whether the Company’s cash
resources will be sufficient to fund the Company’s foreseeable and
unforeseeable operating expenses and capital expenditure
requirements for the Company’s expected timeline in light of
management’s substantial doubt regarding the Company’s ability to
continue as a going concern for at least 12 months from the
issuance date of its most recent quarterly financial statements;
the Company’s failure to demonstrate the safety and efficacy of its
product candidates; the fact that results obtained in earlier stage
clinical testing may not be indicative of results in future
clinical trials; the Company’s ability to enroll patients in
clinical trials for its product candidates; the possibility that
one or more of the Company’s product candidates may cause serious
adverse, undesirable or unacceptable side effects or have other
properties that could delay or prevent their regulatory approval or
limit their commercial potential; the Company’s ability to obtain
and maintain regulatory approval of its product candidates; the
Company’s limited manufacturing history, which could result in
delays in the development, regulatory approval or commercialization
of its product candidates; and the Company’s ability to identify or
discover additional product candidates, or failure to capitalize on
programs or product candidates. Such risks and uncertainties may
cause the statements to be inaccurate and readers are cautioned not
to place undue reliance on such statements. The Company cannot
guarantee that any forward-looking statement will be realized.
Should known or unknown risks or uncertainties materialize or
should underlying assumptions prove inaccurate, actual results
could vary materially from past results and those anticipated,
estimated, or projected. Investors are cautioned not to put undue
reliance on forward-looking statements. A further list and
description of risks, uncertainties, and other matters can be found
in the Company’s Annual Report on Form 20-F for the fiscal year
ended December 31, 2022, and in subsequent reports on Form 6-K, in
each case including in the sections thereof captioned “Cautionary
Statement Regarding Forward-Looking Statements” and “Item 3.D. Risk
factors.” Many of these risks are outside of the Company’s control
and could cause its actual results to differ materially from those
it thought would occur. The forward-looking statements included in
this press release are made only as of the date hereof. The Company
does not undertake, and specifically declines, any obligation to
update any such statements or to publicly announce the results of
any revisions to any such statements to reflect future events or
developments, except as required by law. For further information,
please reference the Company’s reports and documents filed with the
U.S. Securities and Exchange Commission (the “SEC”). You may review
these documents by visiting EDGAR on the SEC website at
www.sec.gov.
Media and Investor
Contact:Naomi Aokinaomi.aoki@freeline.lifeSenior Vice
President, Head of Investor Relations & Corporate
Communications+ 1 617 283 4298
Freeline Therapeutics (NASDAQ:FRLN)
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Freeline Therapeutics (NASDAQ:FRLN)
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