 The Oral Human Growth Hormone Program in Collaboration with Novartis
In September 2004, Emisphere entered into a licensing agreement with Novartis AG to develop an oral formulation of recombinant human growth hormone (rhGH), a hormone which stimulates muscle and bone growth in children and maintains muscle and bone quality in adults. The companies formed the agreement following successful completion of pre-clinical feasibility studies for rhGH with Emisphere's eligen® technology.
A significant body of preclinical data demonstrated bioactivity of oral rhGH using the eligen® technology. These include an increased weight gain in hypophysectomized rats (pituitary gland surgically removed) after a 10-day repeated daily dose of oral rhGH
study.
In a proof-of-concept study conducted in healthy volunteers, after orally dosing rhGH in conjunction with an EMISPHERE® delivery agent, hGH levels demonstrated a quick onset of action, consistent with the desired physiological relevant levels.
In
September 2006, Novartis Pharma AG notified
Emisphere of new Phase I data indicating that
recombinant human growth hormone (rhGH) can be
absorbed when given to growth hormone-deficient
(GHD) patients in a prototype oral formulation using
Emisphere's eligen®
delivery technology.
In a small proof of concept study, 8 GHD patients
were given oral rhGH. Novartis investigated the
pharmacokinetics as well as the pharmacodynamic
properties of the orally-delivered rhGH. The study
showed that growth hormone peaks were recorded in
all patients at some time points, although with
considerable variability and minor endogenous growth
hormone interference. An increase in IGF-I was seen
in some patients, leading to a statistically
significant increase in mean serum IGF-I at day 7
compared with end of wash-out.
Market Opportunity / Expansion
There are an estimated 150,000 children in the U.S. alone who suffer from hGH deficiency. The weekly dosage of hGH given to a child could vary between 3.5 mg to 10 mg, depending on the age and weight. The cost of therapy is typically $10,000 - $ 30,000 per child per year. As a result, sales of growth hormone from the 5 major marketers are significant. The total annual sales of hGH products from the five top marketing companies in 2004 was over $2 billion.
AIDS wasting is a metabolic disorder that causes the body to consume vital muscle and organ tissue (lean body mass) for energy instead of primarily relying on the body's stored fat. AIDS patient typically experience a loss of 5-10% or more of lean body mass, which consists of muscle tissue, body organs and blood cells. The administration of hGH to these patients improves appetite and increases weight and lean body mass.
There is also evidence indicating that hGH can be used in several new indications - Increase cardiac function; Increase lipolysis; Improve fluid balance; Reverse osteoporosis; Improve exercise capacity; Improve lung function; Improve immunity; Regrow vital organs; Increase sense of well-being; Restore REM sleep. These are yet to be evaluated by the FDA.
About Human Growth Hormone
Human Growth Hormone (hGH) is a protein hormone secreted from the anterior pituitary gland that stimulates tissue growth and is responsible for linear, skeletal, organ and cell growth. In addition, hGH has salutary effects on metabolism of proteins, lipids, carbohydrates and minerals and stimulates the synthesis of chondroitin sulfate and collagen. The various functions of hGH make it a critical hormone for tissue repair, muscle growth, wound healing, bone strength and energy metabolism. Endogenous hGH secretion is pulsatile, with the pulse amplitude is greatest at night..
Clinical Indications
Traditional indications for hGH supplementation or replacement include: pediatric and adult patients who lack adequate endogenous hGH secretion, patients with chronic renal insufficiency, and patients with Turner Syndrome. More recent indications for hGH therapy include children born short for gestational age, those with short stature of unknown origin and patients with AIDS related wasting. About 10-15% of children born small for gestational age (SGA) fail to show catch-up growth in height above the third percentile during the first years of life and have an increased risk of being short in adult life. hGH treatment in children born SGA results in a normalization of height during childhood and normalization of bone maturation.
In July 2003, the FDA approved hGH for the long-term treatment of children with idiopathic (of unknown origin) short stature, called non-growth hormone-deficient short stature. Currently all hGH indications require subcutaneous injections of the hormone either daily or several times per week.
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