Chiasma Developing Oral Alternative to Injectable Somatostatin Analogs; Phase 3 Trial Under Way
HOUSTON, June 26, 2012 /PRNewswire-USNewswire/ -- Chiasma, a privately held biopharma company, today presented data at the annual meeting of The Endocrine Society (ENDO 2012) that show Octreolin, an investigational new oral drug being developed for use in acromegaly, reduced pituitary growth hormone secretion in healthy volunteers as effectively as shown in published studies of injectable octreotide, with similar safety, pharmacokinetic and pharmacodynamic profiles.
Octreolin is an oral formulation of injectable octreotide, a somatostatin analog with proven efficacy and safety that is commercially available only by injection. While acromegaly is a treatable disease, management with injectable somatostatin analogs can result in injection-site pain and sub-optimal quality-of-life issues for patients during lifelong treatment.
"We are pleased to report these results indicating that our investigational oral product Octreolin could potentially be effective as a twice-daily medication to suppress growth hormone for treatment of acromegaly," said Chiasma Chief Medical Officer, Sam Teichman, MD. "We hope to confirm these findings in patients with acromegaly in our ongoing phase 3 trial, the results of which are expected in mid-2013."
Highlights from the data presented at ENDO include:
- Octreolin Pharmacodynamics: An Oral Octreotide Formulation to Treat Acromegaly Suppresses Growth Hormone in Healthy Volunteers
Oral presentation, Abstract OR49-4, June 26 - A phase 1 study of healthy subjects assessed the ability of oral Octreolin to suppress growth hormone–a key indicator of therapeutic response for acromegaly patients. Study participants received an oral dose of Octreolin followed by growth hormone releasing hormone to stimulate growth hormone secretion. A single oral dose of Octreolin suppressed not only the stimulated growth hormone secretion but also the basal level of growth hormone in every volunteer. Octreolin was well-tolerated among healthy subjects, with no serious adverse events reported.
- Pharmacokinetic Modeling of Oral Octreotide (Octreolin) in Healthy Volunteers and Dosing Regimen Optimization for Acromegaly Patients
Oral presentation, Abstract OR29-6, June 25 - Phase 1 study results comparing oral Octreolin with injectable octreotide in healthy volunteers show that Octreolin achieved octreotide plasma levels known to be therapeutic in acromegaly patients. Results from this study indicate that oral Octreolin could potentially be effective as a twice-daily treatment to suppress growth hormone for treatment of acromegaly. Octreolin was well-tolerated among healthy study volunteers, with no serious adverse events reported.
- A Novel Formulation of Octreotide Enables Its Enteral Absorption and Growth Hormone Inhibition in Animals
Poster presentation, Poster MON-688, June 25 - Data from this poster presentation demonstrate that the Chiasma proprietary technology, Transient Permeability Enhancer (TPE), may facilitate oral administration of octreotide by enabling the drug to permeate the gastrointestinal wall and reach the bloodstream in its active form in a safe manner, as was demonstrated in toxicology studies. This action of the TPE was shown to be transient and reversible.
"These data presented at ENDO 2012 and accepted for publication in the Journal of Clinical Endocrinology and Metabolism, along with our preclinical and toxicology results, enabled Chiasma to launch a global phase 3 trial this past March," said Roni Mamluk, PhD, Chiasma's Chief Operating Officer. "This program represents the first initiative to bring patients a possible oral alternative to injectable somatostatin analogs, which could provide them with freedom from injections by a healthcare provider."
Acromegaly is a rare lifelong condition caused by excessive production of growth hormone, usually occurring as a result of a non-cancerous tumor on the pituitary gland. Left untreated, acromegaly is associated with severe co-morbidities including cardiovascular disease, diabetes, hypertension, sleep apnea, arthritis, osteoporosis and increased mortality. However, with proper attention and care, acromegaly is a highly treatable disease. There are an estimated 20,000 diagnosed people with acromegaly living in the U.S. today, of which half are on drug therapy.[i],[ii],[iii]
Chiasma aims to transform injectable-only drugs into oral products, providing patients with pain-free medications that are self-administered. The Company's lead product under investigation, Octreolin, is an oral form of octreotide. Octreotide is a somatostatin analog with more than 25 years of proven efficacy and safety. As a treatment for acromegaly, Octreolin is in a phase 3 pivotal trial with results expected in mid-2013. Pending positive phase 3 results, Chiasma intends to file a New Drug Application with the U.S. Food and Drug Administration (FDA) using the agency's 505(b)(2) regulatory pathway, which is permitted when a drug product in development represents a modification of an FDA-approved product. Chiasma has already received an Orphan Designation for Octreolin from the U.S. FDA. In Europe, pending positive phase 3 results, Chiasma will apply for Orphan Drug Designation and intends to file a Marketing Authorization Application using the Hybrid application.
Chiasma is also developing an oral small molecule intended to replace a drug that is currently available by intravenous injection to treat a complication of chronic kidney disease (CKD); it is planned to enter clinical trials later in 2012. The company is evaluating additional proteins, peptides and small molecules that can be applied to its Transient Permeability Enhancer (TPE) technology designed to enable oral delivery of drugs that previously were available by injection only.
Chiasma is a Delaware corporation with a 100 percent owned Israeli subsidiary. It is backed by MPM Capital, ARCH Venture Partners, F-2 and F-3 Ventures and 7 Med Health Ventures.
Additional information can be found at: www.ChiasmaPharma.com.
[i] Fernandez A, et al. Clin Endocrinology. 2010;72(3):377-382.
[ii] Schneider HJ, et al. Clin Endocrinology. 2008;69(3):432-435.
[iii] "Acromegaly." NIDDK, NIH Publication No. 08-3924. Available online: http://endocrine.niddk.nih.gov/pubs/acro/acro.aspx. Accessed 5/1/2012.