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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 commercial portfolio within its rare neuromuscular and rare endocrine franchises includes KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, and MACRILEN (macimorelin), the first and only FDA-approved oral drug indicated for the diagnosis of Adult Growth Hormone Deficiency. The Company’s rare endocrine franchise also includes a clinical-stage pipeline of therapies:
RECORLEV (levoketoconazole), a cortisol synthesis inhibitor currently in Phase 3 development for the treatment of endogenous Cushing's syndrome, and veldoreotide, a next-generation somatostatin analog being investigated for the treatment of acromegaly. Both RECORLEV and veldoreotide have received orphan designation from the U.S. Food and Drug Administration and the European Medicines Agency. 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 neuromuscular and endocrine franchises or that would form the basis for new rare disease franchises.

Strongbridge Biopharma’s Product Portfolio

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 orphan designation status 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.4-5.

MACRILEN (macimorelin), an oral growth hormone secretagogue receptor agonist, is the first and only FDA-approved oral drug indicated for the diagnosis of Adult Growth Hormone Deficiency.6 MACRILEN was approved by the FDA on December 20, 2017, has been granted orphan drug designation in the U.S., and has patents with expiration dates through late 2027. Adult growth hormone deficiency (AGHD) affects more than 50,000 adults in the U.S. Growth hormone is a protein synthesized, stored, and secreted by the pituitary gland. Adults need growth hormone to maintain healthy tissue and organ function. Adult growth hormone deficiency is typically caused by injury/insult to the pituitary and leads to metabolic, cardiovascular, musculoskeletal, and mental health issues. It is treatable with recombinant exogenous growth hormone.7-9

RECORLEV, a cortisol synthesis inhibitor, is being evaluated in a global Phase 3 clinical program for the treatment of endogenous Cushing’s syndrome. SONICS, the first global Phase 3 study is fully enrolled and supported by LOGICS, a second global Phase 3 study designed to supplement the long-term efficacy and safety data from the SONICS study in a randomized, double-blind, placebo-controlled study. Endogenous Cushing’s syndrome is a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. People with uncontrolled disease are seriously ill and have a 2- to 4-fold higher mortality rate than age- and gender-matched controls, mainly due to metabolic and cardiovascular complications. RECORLEV has been granted orphan drug designation by the FDA and the European Medicines Agency (EMA). The Company is developing RECORLEV, 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 for ketoconazole.

Veldoreotide is a novel, investigational somatostatin analogue (SSA) with proven and differentiated pharmacology in pre-clinical models and patients with acromegaly. 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 been granted orphan drug designation by the FDA and the EMA.

The safety and efficacy of RECORLEV and veldoreotide have not been established.

 

Important Safety Information for KEVEYIS

What should you tell your healthcare provider before taking KEVEYIS?

Tell your doctor if you are allergic to dichlorphenamide or other sulfa drugs; if you take high doses of aspirin, or if you have lung or liver disease; if you are pregnant, plan to become pregnant, or are breastfeeding or plan to breastfeed.

 

What should you know about KEVEYIS?

  • Severe allergic and other reactions have happened with sulfonamides (drugs such as KEVEYIS) and have sometimes been fatal. Stop taking KEVEYIS at the first sign of skin rash, swelling, difficulty breathing, or any other unexpected side effect or reaction, and call 911 right away.

  • Tell your healthcare provider if you take aspirin or if another healthcare provider instructs you to begin taking aspirin. High doses of aspirin should not be taken with KEVEYIS.

  • KEVEYIS can cause your body to lose potassium, which can lead to heart problems. Your healthcare provider will measure the potassium levels in your blood before you start treatment and at certain times during treatment.

  • Tell your healthcare provider about all other prescription and over-the-counter medicines you take, including supplements, as some medicines can interact with KEVEYIS.

  • While taking KEVEYIS, your body may produce too much acid or may not be able to remove acid from the body. Your healthcare provider may run tests on a regular basis to check for signs of acid buildup.

  • KEVEYIS may increase your risk of falling. Tell your healthcare provider right away if you experience a fall while taking KEVEYIS.

  • The risks of falls and acid buildup are greater in elderly patients.

  • It is not known whether KEVEYIS is safe or effective for people younger than 18 years of age.

 

What are the most common side effects with KEVEYIS?

The most common side effects are a feeling of numbness, tingling or burning (“pins and needles”) in the toes, feet, hands or fingers; trouble with memory or thinking; feeling confused; and unpleasant taste in the mouth.

 

These are not all of the possible side effects of KEVEYIS. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see full Prescribing Information.

 

Important Safety Information for MACRILEN

What is MACRILEN?

MACRILEN (pronounced ma-kri-len) (macimorelin) is a prescription oral solution that is used to test for adult growth hormone deficiency (AGHD).

 

What should you know about MACRILEN?

  • Taking MACRILEN with certain other medications may cause irregular changes to your heart rhythm. Before taking MACRILEN, tell your healthcare provider about all your medications, as you may need to temporarily stop taking some medications before you take MACRILEN.

  • Some medications may cause a false positive result when taken with MACRILEN. Before taking MACRILEN, tell your healthcare provider about all the medications you take, including growth hormone.

  • Tell your healthcare provider if you were recently diagnosed with hypothalamic disease, as this can cause a false negative result with MACRILEN.

  • You will need to fast (go without food) for at least 8 hours before taking MACRILEN.

 

What are the most common side effects with MACRILEN?

The most common side effects were changed sense of taste, dizziness, headache, fatigue, nausea, hunger, diarrhea, upper respiratory tract infection, feeling hot, excessive sweating, sore nose and throat, and decreased heart rate.

 

These are not all of the possible side effects of MACRILEN. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see MACRILEN Important Safety Information and Full Prescribing Information.

  1. Grieg SL. Dichlorphenamide: a review in primary periodic paralyses. Drugs. 2016;76:501- 507.
  2. 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.
  3. Data on file. Feasterville-Trevose, PA: Strongbridge Biopharma; 2017.
  4. 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.
  5. 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.
  6. MACRILEN Prescribing Information. 2018.
  7. Monson JP, Brooke AM, Akker S. Adult Growth Hormone Deficiency. [Updated 2015 May 19]. In: De Groot LJ, Chrousos G, Dungan K, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000-.
  8. 1. Devesa, Jesús, Cristina Almengló, and Pablo Devesa. “Multiple Effects of Growth Hormone in the Body: Is It Really the Hormone for Growth?” Clinical Medicine Insights. Endocrinology and Diabetes 9 (2016): 47–71. PMC. Web. 16 Jan. 2018.
  9. Gupta, Vishal. “Adult Growth Hormone Deficiency.” Indian Journal of Endocrinology and Metabolism 15.Suppl3 (2011): S197–S202. PMC. Web. 16 Jan. 2018.

 







© 2018 Strongbridge Biopharma plc
STRONGBRIDGE BIOPHARMA® is a registered trademark of the Strongbridge Biopharma plc. companies, which include Strongbridge Ireland Limited and Strongbridge U.S. Inc.
KEVEYIS® is a registered trademark licensed exclusively in the U.S. to Strongbridge Biopharma plc.
MACRILEN is a trademark of Aeterna Zentaris GmbH, licensed exclusively in the U.S. and Canada to Strongbridge Ireland Limited.
MLR-0010 V13 01/2018

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info@strongbridgebio.com

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