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Altus Pharmaceuticals Announces Positive Results from Phase II Study in Growth Hormone Deficient Patients with Once-Per-Week Growth Hormone.


CAMBRIDGE, Mass. -- Altus Pharmaceuticals Inc. (Nasdaq: ALTU), a biopharmaceutical company focused on the development and commercialization of oral and injectable protein therapeutics for patients with gastrointestinal and metabolic diseases, today announced results from a multi-center Phase II clinical trial Noun 1. phase II clinical trial - a clinical trial on more persons than in phase I; intended to evaluate the efficacy of a treatment for the condition it is intended to treat; possible side effects are monitored
phase II
 in growth hormone growth hormone or somatotropin (sōmăt'ətrō`pən), glycoprotein hormone released by the anterior pituitary gland that is necessary for normal skeletal growth in humans (see protein).  deficient adults for ALTU-238, a long-acting crystalline formulation of recombinant human growth hormone human growth hormone (HGH): see growth hormone.  (hGH, somatropin). ALTU-238 is being developed as a subcutaneously administered, once-per-week formulation of human growth hormone that employs Altus' proprietary protein crystallization Crystallization

The formation of a solid from a solution, melt, vapor, or a different solid phase. Crystallization from solution is an important industrial operation because of the large number of materials marketed as crystalline particles.
 and formulation technology.

The Phase II clinical trial was conducted using two dose levels of ALTU-238 in adults with growth hormone deficiency growth hormone deficiency Hypopituitarism Endocrinology A condition which affects 1:4000 children; ♂:♀, 3-4:1 Etiology 70% of GHD is idiopathic and attributed to a prenatal insult, possibly due to hypothalamic dysfunction, given that GHD children . The primary objectives of the trial were to determine the safety and tolerability of ALTU-238, as well as to evaluate its pharmacokinetic and pharmacodynamic profile. ALTU-238 was administered once-per-week, using a fine 30-gauge needle, over a three-week period. The results of this trial confirmed results of earlier Phase I studies of ALTU-238 in which the pharmacokinetic and pharmacodynamic profile observed was supportive of a once-per-week injection of ALTU-238. The study identified doses of ALTU-238 that maintained IGF-1 levels within the normal range for age and gender over the course of the study. Once-per-week dosing of ALTU-238 appeared to result in a consistent, linear dose response of hGH and IGF-1 levels in the blood. Altus anticipates that these positive results will support dose selection for ALTU-238 in upcoming Phase III trials in adults and pediatric patients which we anticipate will start later this year.

ALTU-238 was shown to be well-tolerated by patients enrolled in the study with generally mild injection site reactions being the most commonly reported event. No serious adverse events or antibody formations were reported. The Company expects to present the full results of the study at an upcoming medical meeting later in the year.

Sheldon Berkle, President and Chief Executive Officer of Altus Pharmaceuticals said, "We are greatly encouraged by these study results and the potential to provide patients with a safe, effective long-acting treatment option, with fewer injections than current products which require daily injections. ALTU-238 could represent a significant milestone in the treatment of growth hormone disorders, offering greater patient convenience, comfort and improved compliance. We are looking to gain agreement with the regulatory authorities on the upcoming Phase III programs for both adult and pediatric patients with a goal of quickly bringing this product to patients."

"We believe that these positive Phase II data indicate that our proprietary protein crystallization technology can effectively deliver long-acting growth hormone therapy to growth hormone deficient patients through a once-weekly injection using a fine-gauge needle," said Dr. Alexey Margolin, Chief Scientific Officer at Altus Pharmaceuticals. "In a number of clinical trials - including a large multi-center Phase II study with our orally administered enzyme replacement therapy Enzyme replacement therapy is a medical treatment replacing an enzyme in patients in whom that particular enzyme is deficient or absent. Usually this is done by giving the patient an intravenous (IV) infusion containing the enzyme.  for pancreatic insufficiency pancreatic insufficiency A relative or absolute lack of pancreatic enzymes, a finding typical of cystic fibrosis. See Cystic fibrosis.  and now our Phase I and II growth hormone studies - our proprietary crystallization and formulation technology has repeatedly demonstrated the potential to be a robust platform for development of both oral and injectable biopharmaceuticals."

About ALTU-238

ALTU-238 is a long-acting subcutaneous formulation of recombinant human growth hormone that employs Altus' proprietary protein crystallization and formulation technology. Altus' technology preserves the structure of the human growth hormone molecule without the need for polymers or encapsulation and enables ease of administration through a fine gauge needle. Recombinant human growth hormone is approved for treating multiple growth disorders in children, adolescents and adults. Global sales of recombinant human growth hormone were approximately $2.4 billion in 2005.

About Growth Hormone Deficiency and Growth Failure

Growth hormone disorders occur when the production of growth hormone, secreted via the hypothalamic-pituitary axis hypothalamic-pituitary axis Endocrinology Any feedback system that coordinates the activity of major peptide hormones; the hypothalamus synthesizes releasing hormones, which act on the pituitary, which evokes end-organ responses

 (GHRH-GH-IGF1), is disrupted. Growth hormone plays a critical role in stimulating bone growth and development and it is involved in the production of muscle and in the breakdown of fats. In children, when there is a complete absence of growth hormone, severe growth retardation results. When there is a partial deficiency of growth hormone in children, short stature results. Recombinant human growth hormone (rhGH) products are currently approved to treat multiple growth disorders, including pediatric pediatric /pe·di·at·ric/ (pe?de-at´rik) pertaining to the health of children.

pe·di·at·ric
adj.
Of or relating to pediatrics.
 and adult growth hormone deficiency, Turner Syndrome Turner syndrome

Chromosomal disorder (from the presence of only one sex chromosome, X, in all or some of the body's cells) that causes abnormal sexual development in females.
, chronic renal insufficiency, idiopathic short stature Idiopathic short stature (ISS) refers to extreme short stature that does not have a diagnostic explanation (idiopathic designates a condition that is unexplained or not understood) after an ordinary growth evaluation. , Prader-Willi Syndrome, short children born small for gestational age small for gestational age Intrauterine growth retardation Neonatology adjective Referring to an infant whose gestational age and weight gain are < expected for age. See Low birthweight. , short bowel syndrome Short bowel syndrome
A condition in which the bowel is not as long as normal, either because of surgery or because of a congenital defect. Because the bowel has less surface area to absorb nutrients, it can result in malabsorption syndrome.
 and muscle wasting in patients with HIV HIV (Human Immunodeficiency Virus), either of two closely related retroviruses that invade T-helper lymphocytes and are responsible for AIDS. There are two types of HIV: HIV-1 and HIV-2. HIV-1 is responsible for the vast majority of AIDS in the United States. .

About Altus Pharmaceuticals Inc.

Altus Pharmaceuticals, headquartered in Cambridge, MA, is a biopharmaceutical company focused on the development and commercialization of oral and injectable protein therapeutics for patients with gastrointestinal and metabolic disorders. The Company's website is http://www.altus.com.

Certain statements in this news release concerning Altus' business are considered "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees and  of 1995. These statements include, but are not limited to, the anticipated timing for enrollment in the Phase III clinical trial Noun 1. phase III clinical trial - a large clinical trial of a treatment or drug that in phase I and phase II has been shown to be efficacious with tolerable side effects; after successful conclusion of these clinical trials it will receive formal approval from the  of ALTU-238, and the ability of Altus' proprietary protein crystallization technology to effectively deliver growth hormone therapy through a once-weekly injection using a fine gauge needle. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Altus might make or by known or unknown risks and uncertainties, including, but not limited to uncertainties as to the future success of ongoing and planned clinical trials; and the unproven safety and efficacy of products under development. Consequently, no forward-looking statement can be guaranteed, and actual results may vary materially. Additional information concerning factors that could cause actual results to materially differ from those in the forward-looking statements is contained in Altus' reports to the Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2005. However, Altus undertakes no obligation to publicly update forward-looking statements, whether because of new information, future events or otherwise.
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Date:Jun 23, 2006
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