Strongbridge Biopharma (SBBP)

[Immie]

Some info:

http://www.strongbridgebio.com/

Strongbridge Biopharma is a biopharmaceutical company focused on the development, in-licensing, acquisition and eventual commercialization of complementary product candidates across multiple franchises that target rare diseases.

Strongbridge Biopharma’s primary focus has been to build its franchise around rare endocrine disorders, which includes product candidates for the treatment of endogenous Cushing’s syndrome and acromegaly, two rare diseases with a high unmet need for innovative treatment options. Given the well-identified and concentrated prescriber base addressing its target markets, the Company intends to use a small, focused sales force to market its products, if approved, in the United States, European Union and other key global markets. The Company intends to identify and in-license or acquire products or product candidates that would be complementary to its existing rare endocrine franchise or that would form the basis for new rare disease franchises.

Strongbridge Biopharma’s rare disease franchise includes the following product candidates:

COR-003 (levoketoconazole), a cortisol synthesis inhibitor, is being evaluated in a Phase 3 pivotal trial for the treatment of endogenous Cushing’s syndrome. Endogenous Cushing’s syndrome is a rare endocrine disorder characterized by sustained elevated cortisol levels that most commonly result from a benign tumor of the pituitary gland. COR-003 has been granted orphan drug designation by the U.S. Food and Drug Administration, or the FDA, and the European Medicines Agency, or the EMA. The Company is developing COR-003, a single enantiomer of ketoconazole, as a new chemical entity, or NCE, under the FDA 505(b)(2) regulatory pathway, and intends to reference the FDA’s prior conclusions of safety and effectiveness for ketoconazole.

COR-004, a second-generation antisense oligonucleotide, is in Phase 2 clinical development for the treatment of acromegaly. Acromegaly is a rare endocrine disorder that most commonly results from a benign tumor of the pituitary gland, leading to excess production of growth hormone, or GH, and insulin-like growth factor 1, or IGF-1, a key regulator of growth and metabolism. COR-004 has a novel mechanism of action targeting human GH receptor messenger RNA, or GHR mRNA, a molecule that is necessary for the synthesis of GHR protein. Currently, somatostatin analogs, or SSAs, are the most commonly used drug therapy for the treatment of patients with acromegaly. Up to one-half of treated patients do not adequately respond to SSAs and need alternative or adjunctive drug therapies. The novel mechanism of action of COR-004 may result in a differentiated safety and efficacy profile as compared to pegvisomant, the most common drug therapy used as an alternative to or in combination with SSAs.

COR-005, a novel somatostatin analog (SSA), is in Phase 2 clinical development for the treatment of acromegaly. Based on the differentiated activation pattern of COR -005 to somatostatin receptor subtypes, or SSTRs, and preclinical and clinical data COR -005 may offer an improved efficacy and safety profile relative to existing drug therapies for acromegaly. COR -005 has been granted orphan drug designation by the FDA and the EMA.