Tuesday June 5, 7:15 am Eastern Time
Press Release
SOURCE: Human Genome Sciences, Inc.
Human Genome Sciences' Albutropin IND Cleared by
FDA
New and Improved Growth Hormone Drug Candidate for Treatment of
Adults
ROCKVILLE, Md., June 5 /PRNewswire/ -- Human Genome Sciences, Inc. (Nasdaq:
HGSI - news) today announced that its investigational new drug application, or IND, for
Albutropin(TM), a long-acting recombinant growth hormone, has been cleared by the US
Food & Drug Administration (FDA). Human Genome Sciences is now able to proceed with
its planned Phase 1 human clinical trial for Albutropin(TM) in growth hormone deficient
adults. HGS also is planning a future trial of Albutropin for treatment of growth hormone
deficiency in children.
HGS will now proceed to initiate a Phase 1 trial of the product in adult patients. This Phase 1
trial will be a multi-center, open-label, escalating dose study to determine the safety and
pharmacology of single and multiple doses of Albutropin in adult patients suffering from
growth hormone deficiency.
David C. Stump, M.D., Senior Vice President, Drug Development, said, ``Growth hormone
deficiency in adults is a serious problem that, if left untreated, may lead to reduced lean body
mass, decreased bone density and abnormal blood lipids with increased cardiovascular risk.
Yet, only a small percentage of growth hormone deficient adults currently receive therapy.
Patient unwillingness to accept a regimen of daily subcutaneous injections is one key factor.
A new, long-acting therapy such as Albutropin that may offer a safe, well-tolerated and
convenient treatment option should address one of the major barriers to treatment in this
population.''
William A. Haseltine, Ph.D., Chairman and Chief Executive Officer, said, ``The Albutropin
IND clearance by FDA demonstrates the value of our albumin fusion technology. We expect
that the drugs we create with this technology will be new, improved therapeutic proteins that
offer incremental therapeutic and convenience benefits versus currently available therapies.
This technology also offers Human Genome Sciences the opportunity to improve the pharmaceutical characteristics of human
proteins that we discover ourselves.''
Dr. Haseltine continued, ``Albutropin is the second drug to enter clinical trials as a result of our albumin fusion technology. The
first is Albuferon(TM) (albumin interferon alpha) which is currently undergoing Phase 1 human clinical trials for treatment of
Hepatitis C infection. The other three HGS drugs in on going clinical trials are: repifermin for venous ulcers, mucositis and
ulcerative colitis; mirostipen for the chemoprotection of hematopoietic stem cells; and, B Lymphocyte Stimulator, or
BLyS(TM), for treatment of common variable immunodeficiency disease. We are very proud to have brought five drugs to the
clinic so rapidly and are pleased with the contributions that albumin fusion-based products are making to our overall pipeline.''
Albutropin is a protein created by fusing the gene of a human protein, human growth hormone, to the gene of another human
protein, albumin. Based on preclinical studies, Albutropin may offer sustained therapeutic activity, which is accomplished by
using recombinant human albumin as a carrier molecule. Preclinical data suggest that Albutropin is eliminated from the body 50
times more slowly than regular growth hormone and is detectable in the blood for at least a week after dosing. Therefore,
Albutropin may offer patients a more convenient administration schedule when compared to the existing short-acting therapies.
Growth hormone deficiency affects approximately 70,000 adults and between 20,000 and 27,000 children in the US. Growth
hormone deficiency in adults encompasses two groups of patients: patients with idiopathic childhood onset deficiency and
patients with adult onset deficiency due to organic causes such as pituitary disease or tumor. The results of a recent study
conducted by the Pituitary Network Association suggest that up to 6% of the adult population worldwide may have a
non-cancerous pituitary tumor, which is clinically active and causes significant health problems.
Individuals interested in Albutropin are encouraged to contact Human Genome Sciences at 301-610-5790, extension 3550, or
via the Internet at hgsi.com .
Human Genome Sciences is a company with the mission to treat and cure disease by bringing new gene-based drugs to
patients.
HGS, Human Genome Sciences, Albutropin, Albuferon and BLyS are registered trademarks of Human Genome Sciences, Inc.
For additional information on Human Genome Sciences, Inc., visit the company's web site at hgsi.com . Copies of
HGS press releases are also available by fax 24 hours a day at no charge by calling 800-758-5804, ext. 121115.
This announcement contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements are based
on Human Genome Sciences' current intent, belief and expectations. These statements are not guarantees of future performance
and are subject to certain risks and uncertainties that are difficult to predict. Actual results may differ materially from these
forward-looking statements because of the company's unproven business model, dependence on new technologies, uncertainty
as to clinical trial results, ability to develop and commercialize products, dependence on collaborators for services and revenue,
substantial indebtedness, intense competition, uncertainty of patent and intellectual property protection, dependence on key
management, uncertainty of regulation of products, dependence on key suppliers and other risks that may be described in the
company's filings with the Securities and Exchange Commission. Existing and prospective investors are cautioned not to place
undue reliance on these forward-looking statements, which speak only as of today's date. Human Genome Sciences undertakes
no obligation to update or revise the information contained in this announcement whether as a result of new information, future
events or circumstances or otherwise.
Backgrounder:
Human Genome Sciences' Albutropin IND Cleared By FDA
New and Improved Growth Hormone Drug Candidate for Treatment of Adults
Human Genome Sciences, Inc. (Nasdaq: HGSI - news) today announced that the Food & Drug Administration (FDA) has
cleared its investigational new drug application, or IND, for Albutropin(TM). Human Genome Sciences is now able to proceed
with the Phase 1 clinical trial for Albutropin in growth hormone deficient adults proposed in the IND.
Background
Growth hormone deficiency in a condition in which the body fails to secrete, or secretes inadequate levels of growth hormone
(somatropin). Secretion of growth hormone is low in infants, high in puberty, and lower in adulthood than puberty. A
progressive decline in growth hormone secretion occurs after 30, with secretion reduced further with advancing age and with
obesity. Growth hormone secretion is also gender dependent, with pre- menopausal women secreting more growth hormone
than men of the same age.
Symptoms/Effects
Common symptoms of growth hormone deficiency in adults include abnormal body composition, with reduced lean body mass
and increased abdominal adiposity, reduced strength and exercise capacity, and impaired psychological well-being,
characterized by reduced energy, depressed mood, emotional lability, impaired self-control, anxiety, and increased social
isolation.
Patients with growth hormone deficiency have a reduced life expectancy versus that of healthy controls. Longstanding growth
hormone deficiency in adulthood predisposes the patient to development of premature atherosclerosis.
Diagnosis and Treatment
The diagnosis of growth hormone deficiency in adults is based on insulin tolerance tests and arginine stimulation tests. Patients
with severe growth hormone deficiency, defined as those with a peak growth hormone response to hypoglycemia < 3 mcg/L,
are appropriate candidates for treatment. Patients with mild to moderate growth hormone deficiency, defined as those with a
peak growth hormone response to hypoglycemia of 3 to 5 mcg/L, are also appropriate candidates.
The goals of treatment with somatropin replacement therapy are to restore normal body function, including body composition,
energy, and metabolism and to improve overall quality of patient's life. Several pharmaceutical and biotechnology companies
market their short-acting versions of the recombinant somatropin for treatment of growth hormone deficiency in adults. The
various short-acting products all appear to be comparable in safety and efficacy.
Patient Population
Growth hormone deficiency affects approximately 70,000 adults and between 20,000 and 27,000 children in the US. Growth
hormone deficiency in adults encompasses two groups of patients: patients with idiopathic childhood onset deficiency and
patients with adult onset deficiency due to organic causes such as pituitary disease or tumor. The results of a recent study
conducted by the Pituitary Network Association suggest that up to 6% of the adult population worldwide may have a
non-cancerous pituitary tumor, which is clinically active and causes significant health problems.
Albutropin
Albutropin is a protein created by fusing the gene of a human protein, human growth hormone, to the gene of another human
protein, albumin. Based on preclinical studies, Albutropin may offer sustained therapeutic activity, which is accomplished by
using recombinant human albumin as a carrier molecule. Preclinical data suggest that Albutropin is eliminated from the body 50
times more slowly than regular growth hormone and is detectable in the blood for at least a week after dosing. Therefore,
Albutropin may offer patients a more convenient administration schedule when compared to the existing short-acting therapies.
Albumin Fusion Technology
Relying on Human Genome Sciences' core competency and expertise in molecular engineering and biopharmaceutical
production, we intend to create, develop and manufacture novel long-circulating versions of therapeutic proteins that can
maintain effective but non-toxic blood levels for extended periods of time.
The albumin fusion technology consists of fusing the gene for albumin with the gene for a therapeutic protein or peptide. This
fused gene then signals to produce the albumin fusion protein, which is manufactured using our proprietary expression systems.
Delivery of protein therapeutics in a manner that is both safe and cost- effective has been a major industry challenge. Most
biopharmaceuticals are injected to avoid rapid degradation in the digestive tract. However, most injected proteins are also
rapidly cleared from the blood. Therefore, relatively high doses and/or frequent injections are typically needed to maintain
therapeutic blood levels, resulting in potentially increased side effects and increased treatment costs as well as significant patient
inconvenience and non-compliance.
Our albumin fusion technology may allow re-engineering of existing recombinant protein therapeutics to address the above
shortcoming of existing therapies. In addition, new proteins discovered by Human Genome Sciences are expected to benefit
from the enhanced performance and cost-effectiveness provided by this technology.
About Human Genome Sciences, Inc.
Human Genome Sciences is a company with the mission to treat and cure disease by bringing new gene-based drugs to
patients.
HGS, Human Genome Sciences and Albutropin are registered trademarks of Human Genome Sciences, Inc. For additional
information on Human Genome Sciences, Inc., visit the company's web site at hgsi.com . Copies of HGS press
releases are also available by fax 24 hours a day at no charge by calling 800-758-5804, ext. 121115.
This announcement contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements are based
on Human Genome Sciences' current intent, belief and expectations. These statements are not guarantees of future performance
and are subject to certain risks and uncertainties that are difficult to predict. Actual results may differ materially from these
forward-looking statements because of the company's unproven business model, dependence on new technologies, uncertainty
as to clinical trial results, ability to develop and commercialize products, dependence on collaborators for services and revenue,
substantial indebtedness, intense competition, uncertainty of patent and intellectual property protection, dependence on key
management, uncertainty of regulation of products, dependence on key suppliers and other risks that may be described in the
company's filings with the Securities and Exchange Commission. Existing and prospective investors are cautioned not to place
undue reliance on these forward-looking statements, which speak only as of today's date. Human Genome Sciences undertakes
no obligation to update or revise the information contained in this announcement whether as a result of new information, future
events or circumstances or otherwise.
SOURCE: Human Genome Sciences, Inc. |
