Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Founded in 1996, the Company is publicly traded on the NASDAQ Global Market under the ticker symbol SBBP.
The primary focus of Strongbridge Biopharma is to build and advance a portfolio of vertical, therapeutically-aligned rare disease franchises. Strongbridge's rare endocrine franchise includes RECORLEV® (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies for the treatment of endogenous Cushing's syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both levoketoconazole, the active ingredient of RECORLEV, and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The Company’s rare neuromuscular franchise includes KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis. KEVEYIS has orphan drug exclusivity in the United States. Significant unmet needs remain in the diagnosis and treatment of these rare diseases.
The Company will continue to identify and evaluate the acquisition of products or product candidates that would be complementary to its existing rare endocrine and neuromuscular franchises or that would form the basis for new rare disease franchises.
Strongbridge Biopharma’s Product Portfolio
RECORLEV® (levoketoconazole) is an investigational cortisol synthesis inhibitor in development for the treatment of patients with endogenous Cushing’s syndrome, a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. Levoketoconazole is the pure 2S,4R enantiomer of ketoconazole, a steroidogenesis inhibitor. RECORLEV has demonstrated in two successful Phase 3 studies to significantly suppress serum cortisol and has the potential to be a next-generation cortisol inhibitor.
The Phase 3 program for RECORLEV includes SONICS, LOGICS and OPTICS: three multinational studies designed to evaluate the safety and efficacy of RECORLEV when used to treat endogenous Cushing’s syndrome. The SONICS study met its primary and secondary endpoints, demonstrating a statistically significant normalization rate of urinary free cortisol at six months. The LOGICS study, which met its primary endpoint, is a double-blind, placebo-controlled randomized-withdrawal study of RECORLEV that is designed to supplement the long-term efficacy and safety information supplied by SONICS. The ongoing long-term open-label OPTICS study will gather further useful information related to the long-term use of RECORLEV.
Levoketoconazole has received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing's syndrome.
Veldoreotide extended release, a pre-clinical next-generation somatostatin analog, is being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Veldoreotide may provide the acromegaly community with a new treatment option offering a unique clinical profile and delivery system. Strongbridge has completed the screening of potential long-acting release (LAR) technologies for veldoreotide, and selected a proprietary formulation based upon PLGA microspheres for further development. PLGA is a well-known polymer, which has been widely applied in LAR formulations due to its biocompatibility, biodegradability, and favorable release kinetics. Veldoreotide has received orphan drug designation from the FDA and the European Medicines Agency.
The safety and efficacy of RECORLEV and veldoreotide have not been established.
KEVEYIS is the first and only FDA-approved drug for the treatment of hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, a spectrum of rare and chronic genetic, neuromuscular disorders with autosomal dominant inheritance that cause recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis.1 As they age, some patients may experience permanent muscle weakness2. KEVEYIS was approved by the FDA on August 7, 2015 and has received orphan drug exclusivity in the United States through August 7, 2022. Primary periodic paralysis is very rare, affecting approximately 4,000 to 5,000 diagnosed individuals in the U.S.3 The most common forms of the disease are hyperkalemic and hypokalemic Primary Periodic Paralysis, and time between symptom onset and diagnosis can take more than 20 years.2,4
- Grieg SL. Dichlorphenamide: a review in primary periodic paralyses. Drugs. 2016;76:501- 507.
- Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613.
- Data on file. Feasterville-Trevose, PA: Strongbridge Biopharma; 2017.
- Cavel-Greant D, Lehmann-Horn F, Jurkat-Rott K. The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients. Acta Myol. 2012;31:126-133.