Date: 2015-11-03
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the Society for Endocrinology BES 2015 conference being held in Edinburgh, UK from November 2-4, 2015
Company: Strongbridge Biopharma (Ireland) previously known as Cortendo (Sweden)
Product: atesidorsen (COR-004 -ATL1103 - 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor)
Action
mechanism: antisense drug. COR-004, previously known as ATL1103 is a second-generation antisense drug designed to block growth hormone receptor (GHr) expression thereby reducing levels of the hormone insulin-like growth factor-1 (IGF-1) in the blood and is a potential treatment for diseases associated with excessive growth hormone and IGF-1 action. These diseases include acromegaly, an abnormal growth disorder of organs, face, hands and feet, diabetic retinopathy, a common disease of the eye and a major cause of blindness, diabetic nephropathy, a common disease of the kidney and major cause of kidney failure, and some forms of cancer. In a Phase I study in healthy subjects, the antisense drug demonstrated a preliminary indication of drug activity, including suppression of IGF-1 and the target GHr (via circulating growth hormone binding protein) levels. In a Phase II trial in acromegalic patients, ATL1103 met its primary efficacy endpoint by showing a statistically significant average reduction in sIGF-1 levels from baseline (P<0.0001) at week 14 (one week past the last dose) at the twice weekly 200 mg dose tested. Antisense is currently undertaking a higher dose study (2 x 300 mg/week) in acromegaly patients. Under its technology collaboration with Isis, Antisense Therapeutics’ will pay Isis a percentage (single digit) of the licensing revenue it earns from the product.
Disease: acromegaly
Therapeutic area: Rare diseases - Hormonal diseases - Endocrine diseases
Country:
Trial details:
Latest
news: * On November 3, 2015, Strongbridge Biopharma announced that results from a secondary efficacy analysis of the completed Phase 2 trial for COR-004 in adult patients with acromegaly will be presented at the Society for Endocrinology BES 2015 conference being held in Edinburgh, UK from November 2-4, 2015. The Phase 2 clinical trial was a randomized, open-label, parallel group clinical trial of the safety, tolerability, pharmacokinetics and efficacy of two subcutaneous administration regimens of COR-004 in 26 adult patients with acromegaly treated over 13 weeks. The trial met its key efficacy endpoint demonstrating a statistically significant average reduction in the serum insulin-like growth factor 1 (IGF-1) levels from baseline in the higher dose group. COR-004 was safe and well tolerated. The main adverse event was mild to moderate injection site reactions in the majority of patients. As one of the secondary endpoints, the level of growth hormone (GH)-binding protein (GHBP) as a marker of growth hormone receptor (GHR) expression was evaluated. With the larger dose, a continuous decrease in GHBP was found that reached statistical significance at the end of the study, coinciding with a significant reduction in IGF-1. These data provide further evidence for the efficacy of COR-004 and its ability to inhibit growth hormone receptor (GHR) expression. Data from future clinical trials will help to evaluate the relationship between COR-004 dose, GH levels, GHBP levels and the change in IGF-1 with treatment. Presentation title: In patients with acromegaly antisense oligomer therapy directed at the GH receptor is associated with reduction in circulating GHBP levels (Abstract Number: P308 (p155) Earlier this year, Strongbridge Biopharma plc entered into an exclusive licensing agreement for commercialization rights to Antisense Therapeutics' ATL1103, now known as COR-004, for endocrinology applications.
