Novelion Therapeutics Inc. (NASDAQ:NVLN) (TSX:NVLN), a
biopharmaceutical company dedicated to developing new standards of
care for individuals living with rare diseases, today announced the
presentation of data by academic researchers at the American
Association of Clinical Endocrinology taking place in Austin, TX.
The study titled “Impact of Metreleptin on
Hepatomegaly in Patients with Generalized Lipodystrophy” suggests
treatment with metreleptin may reduce liver volume in patients with
enlarged liver (hepatomegaly) resulting from complications of
generalized lipodystrophy (GL). The study also demonstrated
improvements from baseline of key metabolic parameters, including
hemoglobin A1c (HbA1c), liver enzymes and triglycerides.
The post-hoc analysis of an open-label,
prospective study in patients with GL assessed both the impact on
liver volume and key metabolic parameters when patients were
treated with metreleptin as leptin replacement therapy. Of the 34
evaluable patients, 22 had baseline liver volumetric measurements
using MRI; all 22 patients had an enlarged liver. Normal liver
volume was defined as less than 25 mL/kg of body weight.
The post-hoc analysis completed by Elif Oral,
M.D., Associate Professor of Medicine, Michigan Medicine, reviewed
patients who participated in a prospective, open-label study
conducted at National Institutes of Health between 2000-2008, with
study drug provided by Novelion Therapeutics’ subsidiary.
For patients assessed within one year of
initiating treatment with metreleptin (N=21), liver volume
decreased an average of 25 percent. The mean treatment duration was
10 months. Among these same patients who had additional follow ups
after one year (N=14) and had a mean duration of metreleptin
treatment of 46 months, liver volume decreased by 35 percent.
The most commonly reported adverse events
occurring in more than 10 percent of patients were abdominal pain,
hypoglycemia, ear infection, fatigue, proteinuria, back pain,
diarrhea, headache, menorrhagia, nausea, ovarian cyst, upper
respiratory tract infection and weight decline.
“GL patients lack fat cells and the absence of
adipocytes can lead to the accumulation of fat in the liver that
can potentially progress to various forms of liver disease,” said
Dr. Oral. “We were pleased to see a continued treatment
effect in patients who had a longer duration of therapy.”
Patients treated with metreleptin also
experienced clinically meaningful reductions from baseline in
HbA1c, liver enzymes (both AST and ALT) and triglycerides, a type
of fat in the blood.
Novelion believes that metreleptin may
demonstrate clinical results in a wide range of low leptin-mediated
rare and metabolic diseases. John Orloff, MD,
executive vice president and head of research and development
for Novelion, stated, “Analysis like this, combined with 14 years
of additional patient data, provides evidence supporting the
implications of low leptin levels on various physiologic
functions. We are currently evaluating conducting clinical trials
to investigate the use of metreleptin in multiple
other low leptin-mediated conditions.”
About Lipodystrophy
Lipodystrophy syndromes (LD) are ultra-rare
disorders characterized by the irreversible loss of adipose tissue.
In patients with lipodystrophy syndromes, levels of leptin are
often very low. Leptin is a naturally occurring hormone produced in
adipose tissue and is an important regulator of energy
homoeostasis, fat and glucose metabolism, reproductive capacity,
and other diverse physiological functions.
With GL, the loss of fat affects the whole body.
With partial lipodystrophy, the loss of fat typically occurs in the
arms, legs, head and trunk regions, while accumulation of fat may
occur in other areas of the body, including the neck, face and
intra-abdominal regions. Metreleptin is approved in the U.S. to
treat GL and is not approved to treat partial lipodystrophy.
U.S. INDICATIONS AND IMPORTANT SAFETY
INFORMATION
MYALEPT® (metreleptin) for injection is a
leptin analog indicated as an adjunct to diet as replacement
therapy to treat the complications of leptin deficiency in patients
with congenital or acquired generalized lipodystrophy. LIMITATIONS
OF USE: The safety and effectiveness of MYALEPT for the
treatment of complications of partial lipodystrophy or for the
treatment of liver disease, including nonalcoholic steatohepatitis
(NASH), have not been established. MYALEPT is not indicated for use
in patients with HIV-related lipodystrophy. MYALEPT is not
indicated for patients with metabolic disease, including diabetes
mellitus and hypertriglyceridemia, without concurrent evidence of
generalized lipodystrophy.
Anti-metreleptin antibodies with
neutralizing activity have been identified in patients treated with
MYALEPT. T-cell lymphoma has been reported in patients
with acquired generalized lipodystrophy, both treated and not
treated with MYALEPT. For more detailed information, please see
additional Important Safety Information and
the Prescribing Information, including boxed warning, for
MYALEPT.
MYALEPT is available only through a restricted program called
the MYALEPT REMS PROGRAM.
About University of Michigan,
Metabolism, Endocrinology and Diabetes Division
The University of Michigan, Metabolism,
Endocrinology and Diabetes Division and Brehm Center for Diabetes
collectively house a major referral for the study of lipodystrophy
syndromes. For more information, contact Adam Neidert at (734)
615-0539.
About Novelion Therapeutics
Novelion Therapeutics is a biopharmaceutical company dedicated to
developing new standards of care for individuals living with rare
diseases. The company seeks to advance its portfolio of rare
disease therapies by investing in science and clinical development.
Novelion has a diversified commercial portfolio through its
indirect subsidiary, Aegerion Pharmaceuticals, Inc. (Aegerion), and
is also developing zuretinol acetate for the potential treatment of
inherited retinal disease caused by underlying mutations in RPE65
or LRAT genes.
Forward-Looking Statements
Certain statements in this press release
constitute "forward-looking statements" of Novelion within the
meaning of applicable laws and regulations and constitute
"forward-looking information" within the meaning of applicable
Canadian securities laws, including the statement regarding
expectations as to label expansion for metreleptin. Forward-looking
statements are based on estimates and assumptions made by Novelion
in light of current conditions and expected future developments, as
well as other factors that Novelion believes are appropriate in the
circumstances, including but not limited to, our financial position
and execution of our business strategy, receipt of regulatory
approvals, indication prevalence, and product competition, market
acceptance, sales, pricing, reimbursement and side effects. These
forward-looking statements are neither promises nor guarantees of
future performance, and are subject to a variety of risks and
uncertainties, many of which are beyond our control, which could
cause actual results to differ materially from those contemplated
in these forward-looking statements. Many such risks, uncertainties
and other factors are taken into account as part of our assumptions
underlying these forward-looking statements and include, among
others, the following: the risks that metreleptin may not obtain
regulatory approval for additional indications; that, if approved,
metreleptin may not obtain reimbursement approvals on a timely
basis or at all or at the levels necessary to support
commercialization; that the prevalence estimates for a desired
indication may be inaccurate; the risk that orphan drug, data and
marketing exclusivity or the patent protection for metreleptin may
not be sufficient or may be invalidated; and the other risks common
to seeking regulatory approval for, developing and commercializing
orphan drugs. For additional disclosure regarding these and other
risks we face, see the disclosure contained in the "Risk Factors"
section of our Annual Report on Form 10-K filed on March 30, 2017,
and our other public filings with the SEC, available on the SEC's
website at www.sec.gov. Except as required by law, we undertake no
obligation to update or revise the information contained in this
press release, whether as a result of new information, future
events or circumstances or otherwise.
Investors and others should note that we
communicate with our investors and the public using our company
website www.novelion.com, including, but not limited to, company
disclosures, investor presentations and FAQs, SEC filings, press
releases, public conference calls transcripts and webcast
transcripts. The information that we post on these websites could
be deemed to be material information. As a result, we encourage
investors, the media and others interested to review the
information that we post there on a regular basis. The contents of
our website shall not be deemed incorporated by reference in any
filing under the Securities Act of 1933, as amended.
CONTACT:
Amanda Murphy, Director, Investor Relations & Corporate Communications
Novelion Therapeutics
857-242-5024
amanda.murphy@novelion.com
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