CytRx Announces Orphazyme’s U.S. Expansion Ahead of Potential Regulatory Approval of Arimoclomol for Niemann-Pick Disease T...
December 07 2020 - 10:50AM
Business Wire
Highlights Orphazyme’s Establishment of U.S.
Headquarters in Chicago and Recruitment of More Than 30 U.S.
Employees
Comments That Orphazyme’s Investments in the
U.S. are Intended to Support Regulatory Efforts and the Prospective
Commercialization of Arimoclomol for Niemann-Pick Disease Type C
and Possibly Other Rare Diseases in the Future
CytRx Corporation (OTCQB: CYTR) (“CytRx” or the “Company”), a
specialized biopharmaceutical firm focused on research and
development for the oncology and neurodegenerative disease
categories, today highlighted that Orphazyme A/S (NASDAQ: ORPH)
(“Orphazyme”) has announced the expansion of its U.S. presence and
workforce ahead of potential Food and Drug Administration (“FDA”)
approval of arimoclomol for the treatment of Niemann-Pick disease
Type C (“NPC”). CytRx has an agreement with Orphazyme that can
yield potential milestone payments and future royalties paid on
sales of arimoclomol.
In addition to establishing its U.S. headquarters in Chicago,
Orphazyme has already recruited more than 30 U.S. employees.
Orphazyme has also named three U.S. leaders to its global executive
team:
- Molly Painter, U.S. President, is heading the launch and
commercial operations in the market.
- Terri Stevens, Chief Business Officer, is responsible for
global strategy, corporate development, and business development
and licensing.
- Molly Carey Poarch, Global and U.S. Head of Corporate
Communications.
Orphazyme’s U.S. team, which is focused on regulatory review
efforts and a first potential commercial launch in 2021, includes
legal, commercial, finance, advocacy relations, and regulatory and
medical affairs functions.
Illinois State Senator Mattie Hunter issued the following
statement last week:
“I am excited to welcome Orphazyme, a fresh and innovative
biopharmaceutical to Chicago’s robust group of health care
companies. I believe their methodology will make leeway in the
fight against rare neurodegenerative diseases while bringing job
growth to the city.”
Orphazyme’s Chief Executive Officer delivered the following
statement last week:
“We have established a U.S. headquarters in Chicago to allow us
to more closely engage with our partners and community members as
we work together to pursue innovation for debilitating
neurodegenerative diseases based on our pioneering science. Our
U.S. growth will be fueled by the team of industry experts we have
been assembling. We are excited to add three key U.S.-based leaders
to our corporate executive team as we expand to support our
ambition of serving rare disease communities around the world.”
Steven A. Kriegsman, Chairman and Chief Executive Officer of
CytRx, commented:
“We are very pleased to see Orphazyme establish a strong U.S.
footprint by investing in headquarters in Chicago and retaining
more than 30 employees ahead of potential commercialization of
arimoclomol for NPC upon prospective FDA approval in the first
quarter of 2021. In our view, Orphazyme is taking the right steps
to begin future distribution and deepen engagement with U.S.
providers, patients, regulators, and the clinical testing community
in 2021. It is also noteworthy that during the year 2021, Orphazyme
may receive a response to its submission for regulatory approval in
Europe for arimoclomol to treat NPC. Also, on or before June 30,
2021, Orphazyme is expected to report Phase 3 data on arimoclomol
for sporadic Inclusion Body Myositis and Amyotrophic Lateral
Sclerosis. We remain optimistic about Orphazyme’s continued
progress.”
We will continue to provide updates that are relevant to our
agreement with Orphazyme.
About CytRx Corporation
CytRx Corporation (OTCQB: CYTR) is a biopharmaceutical company
with expertise in discovering and developing new therapeutics
principally to treat patients with cancer and neurodegenerative
diseases. CytRx's drug candidate, arimoclomol, was sold to
Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA.CO) in exchange
for milestone payments and royalties. Orphazyme is developing
arimoclomol in four indications including Amyotrophic Lateral
Sclerosis (“ALS”), Niemann-Pick disease Type C (“NPC”), Gaucher
disease and sporadic Inclusion Body Myositis (“sIBM”). Learn more
at www.cytrx.com.
About Orphazyme
Orphazyme is a biopharmaceutical company focused on bringing
novel treatments to patients living with life threatening or
debilitating rare diseases. Their research focuses on developing
therapies for diseases caused by misfolding of proteins including
lysosomal storage diseases. Arimoclomol, the company’s lead
candidate, is in clinical development for four orphan diseases:
Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion
Body Myositis, and Amyotrophic Lateral Sclerosis. Orphazyme is
headquartered in Denmark and has operations in the U.S. and
Switzerland. Orphazyme shares are listed on Nasdaq (ORPHA). For
more information, please visit www.orphazyme.com.
About NPC
Niemann-Pick disease Type C (NPC) is a rare, genetic and
progressive disease that impairs the ability of the body to move
cholesterol and other fatty substances (lipids) inside the cells.
The result is an accumulation of lipids within the body’s tissue,
including the brain tissue, causing damage to the affected 2 areas.
The symptoms upon onset of NPC vary from fatality during the first
months after birth to a progressive disorder not diagnosed until
adulthood. The disease affects neurologic and psychiatric functions
as well as various internal organs. Systemic symptoms of NPC are
more common in infancy or childhood and the rate of progression is
usually much slower in individuals with onset of symptoms during
adulthood. NPC is usually fatal and the majority of individuals
with the disease die before the age of 20. NPC has been granted
Orphan Drug Designation (EU and U.S.) for the treatment of NPC. It
is estimated the incidence of NPC to be one in 100,000 live births
and the number of NPC patients in the United States and in Europe
to be approximately 1,800 individuals. There are no approved
treatments for NPC in the U.S. and only one approved product in
Europe called miglustat.
About Arimoclomol
Arimoclomol is an investigational drug candidate that amplifies
the production of heat-shock proteins (HSPs). HSPs can rescue
defective misfolded proteins, clear protein aggregates, and improve
the function of lysosomes. Arimoclomol is administered orally,
crosses the blood brain barrier, and has been studied in seven
Phase 1, four Phase 2 and one pivotal Phase 2/3 clinical trial.
Arimoclomol is in clinical development at Orphazyme for the
treatment of NPC, Gaucher disease, sIBM and ALS. Arimoclomol has
received orphan drug designation for NPC, sIBM and ALS in the US
and EU, as well as fast-track designation from the US Food and Drug
Administration (FDA) for NPC, sIBM and ALS. In addition,
arimoclomol has received breakthrough therapy designation and
rare-pediatric disease designation from the FDA for NPC.
Forward-Looking Statements
This press release contains forward-looking statements,
including statements relating to the potential receipt of EMA and
FDA approval of arimoclomol, the Company’s potential receipt of
future milestone and royalty payments from Orphazyme and the
achievement of long-term value for the Company’s stockholders. Such
statements involve risks and uncertainties that could cause actual
events or results to differ materially from the events or results
described in the forward-looking statements, including risks and
uncertainties relating to the ability of Orphazyme to obtain
regulatory approval for, manufacture and commercialize its products
and therapies that use arimoclomol; the results of clinical trials
involving arimoclomol; the amount, if any, of future milestone and
royalty payments that we may receive from Orphazyme; and other
risks and uncertainties described in the most recent annual and
quarterly reports filed by the Company with the SEC and current
reports filed since the date of the Company’s most recent annual
report. All forward-looking statements are based upon information
available to the Company on the date the statements are first
published. The Company undertakes no obligation to publicly update
or revise any forward-looking statements, whether as a result of
new information, future events or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20201207005141/en/
For Investors: Greg Marose / Charlotte Kiaie
cytrx@profileadvisors.com
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