- Clinical trial data demonstrated Zokinvy treatment extended
life by an average of 4.3 years in children and young adults with
Hutchinson-Gilford progeria
- Eiger to receive $500,000
approval milestone payment from AnGes
- Zokinvy approved in the U.S. (2020), 30 European countries
(2022), and now Japan (2024)
PALO
ALTO, Calif., Jan. 18,
2024 /PRNewswire/ -- Eiger BioPharmaceuticals, Inc.
(Nasdaq:EIGR), a commercial-stage biopharmaceutical company focused
on the development of innovative therapies for rare metabolic
diseases, today announced that it and its partner AnGes, Inc.
received marketing approval from the Ministry of Health, Labour and
Welfare for Zokinvy (lonafarnib), a treatment for
Hutchinson-Gilford progeria syndrome (HGPS) and processing
deficient progeroid laminopathy (PDPL).
"We and our partner, AnGes, are pleased that Zokinvy is now
approved in Japan for patients
living with progeria, an ultra-rare and fatal pediatric disease
that can result in premature death," said David Apelian, MD, PhD, MBA, CEO of Eiger. "We
would like to thank the Progeria Research Foundation for their
continued support of the regulatory submission as well as the
patients and their families."
Collectively known as progeria, HGPS and PL are devastating
ultra-rare and fatal pediatric diseases that cause dramatically
accelerated aging and premature death. The main cause of death is
heart attack or stroke due to severe hardening of the
arteries.3,4
The approval was based on the positive results of two pivotal
clinical trials demonstrating that Zokinvy, an oral
disease-modifying agent which targets the cause of progeria,
lowered the risk of death in children by 72% and extended life by
an average of 4.3 years (p<0.0001) in children and young adults
with HGPS.2
About Progeria
Collectively known as progeria,
Hutchinson-Gilford progeria syndrome and progeroid laminopathies
are ultra-rare, fatal, genetic premature aging diseases that
accelerate mortality in young patients.
HGPS is caused by a point mutation in the LMNA gene, yielding
the farnesylated aberrant protein, progerin. Progeroid
laminopathies are genetic conditions of accelerated aging caused by
a constellation of mutations in the LMNA and/or ZMPSTE24 genes
yielding farnesylated proteins that are distinct from
progerin.4,5
Without Zokinvy therapy, children with HGPS commonly die of the
same heart disease that affects millions of normally aging adults
(arteriosclerosis), by an average age of 14.5 years. Disease
manifestations include severe failure to thrive, scleroderma–like
skin, global lipodystrophy, alopecia, joint contractures, skeletal
dysplasia, global accelerated atherosclerosis with cardiovascular
decline, and debilitating strokes.3
About Zokinvy® (lonafarnib)
Zokinvy is a
first-in-class disease-modifying agent that blocks the accumulation
of defective progerin and progerin-like proteins which leads to
cellular instability and premature aging in children and young
adults with progeria. Zokinvy has demonstrated a statistically
significant survival benefit in children and young adults with
HGPS.1,4
The most commonly reported adverse reactions were
gastrointestinal (vomiting, diarrhea, nausea), and most were mild
or moderate (Grade 1 or 2) in severity. Many progeria patients have
received continuous Zokinvy therapy for more than 10
years.1,2
Zokinvy is FDA approved for the treatment of patients 12 months
of age and older with a genetically confirmed diagnosis of
Hutchinson-Gilford progeria syndrome or a processing-deficient
progeroid laminopathy associated with either a heterozygous LMNA
mutation with progerin-like protein accumulation or a homozygous or
compound heterozygous ZMPSTE24 mutation.
For Important Safety Information and prescribing information for
Zokinvy in the U.S., please visit www.zokinvy.com
Eiger and AnGes entered into an exclusive distribution agreement
for the treatment of HGPS and PDPL indications, Zokinvy
(Lonafarnib), in Japan on
May 10, 2022. In March 2023, the Ministry of Health, Labour and
Welfare designated Zokinvy as an orphan drug.
About Eiger
Eiger is a commercial-stage biopharmaceutical company focused on
the development of innovative therapies for rare metabolic
diseases. Eiger's lead product candidate, avexitide, is a well
characterized, first-in-class GLP-1 antagonist for the treatment of
post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism
(HI). Avexitide is the only drug in development for PBH with
Breakthrough Therapy designation from the FDA.
For additional information about Eiger and its clinical
programs, please visit www.eigerbio.com.
Note Regarding Forward-Looking Statements
This press
release contains forward-looking statements within the meaning of
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. All statements other than statements of
historical facts, including statements regarding our future
financial condition, timing for and outcomes of clinical results,
prospective products, preclinical and clinical pipelines,
regulatory objectives, business strategy and plans and objectives
for future operations, are forward-looking statements.
Forward-looking statements are our current statements regarding our
intentions, beliefs, projections, outlook, analyses or current
expectations concerning, among other things, the timing of our
ongoing and planned clinical development; our capability to provide
sufficient quantities of any of our products or product candidates
for studies or to meet anticipated full-scale commercial demands;
our ability to identify, pursue and enter into partnering
opportunities for our virology assets; the sufficiency of our cash,
cash equivalents and investments to fund our operations into the
fourth quarter of 2024, including the scope and impact of any
savings from our workforce reduction and cash conservation efforts;
the revenue potential of avexitide in post-bariatric hypoglycemia
and congenital hyperinsulinism; our ability to finance,
independently or through collaborations, the continued advancement
of our development pipeline; and the potential for success of any
of our products or product candidates. Various important factors
could cause actual results or events to differ materially from the
forward-looking statements that Eiger makes, including additional
applicable risks and uncertainties described in the "Risk Factors"
section in Eiger's Quarterly Report on Form 10-Q for the quarter
ended September 30, 2023 and Eiger's
subsequent filings with the SEC. The forward-looking statements
contained in this press release are based on information currently
available to Eiger and speak only as of the date on which they are
made. Eiger does not undertake and specifically disclaims any
obligation to update any forward-looking statements, whether as a
result of any new information, future events, changed circumstances
or otherwise.
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
swheeler@wheelhouselsa.com
Media:
Aljanae Reynolds
Wheelhouse Life Science Advisors
areynolds@wheelhouselsa.com
References:
1. Data on file, Eiger
BioPharmaceuticals.
2. Summary of Product Characteristics, July
2022.
3. Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford Progeria
Syndrome. 2003 Dec 12
[Updated 2019 Jan 17]. In: Adam MP, Ardinger HH, Pagon RA, et
al., editors. GeneReviews® [Internet]. Seattle (WA):
University of Washington, Seattle;
1993-2020.
4. Gordon LB, Shappell H, Massaro J, et al. Association of
lonafarnib treatment vs no treatment with mortality rate in
patients with Hutchinson-Gilford progeria syndrome.
JAMA. 2018;319(16):1687-1695. doi:10.1001/jama.2018.3264.
5. Marcelot A, Worman HJ, and Zinn-Justin
S. Protein structural and mechanistic basis of
progeroid laminopathies. FEBS Journal. 2021:288:2757-2772.
Doi:10.111/febs.15526.
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