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Cushing's Syndrome Investor and Analyst Day April 5, 2018 Welcome and Overview Matthew Pauls President & Chief Executive Officer 2 Forward-Looking Statements This document contains forward‐looking statements relating to the Company’s strategy, objectives, business development plans and financial position. All statements other than statements of historical facts included in this document, including, without limitation, statements regarding the Company’s future financial position, strategy, anticipated investments, costs and results, status and results of clinical trials, size of patient population, plans, outcomes of product development efforts, and objectives of management for future operations, may be deemed to be forward‐looking statements. You can identify forward-looking statements by words such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of those terms, and similar expressions that convey uncertainty or future events or outcomes. These forward‐looking statements involve known and unknown risks, uncertainties, and other factors that may cause the Company’s actual results, performance, or achievements or industry results to be materially different from those contemplated, projected, forecasted, estimated or budgeted, whether expressed or implied, by these forward‐looking statements. Given these risks and uncertainties, investors should not place undue reliance on forward‐looking statements as a prediction of actual results. A discussion of certain of these risks may be found in the filings the Company makes with the U.S. Securities and Exchange Commission. None of these forward‐looking statements constitutes a guarantee of the future occurrence of such events or of actual results. These statements are based on data, assumptions, and estimates that the Company believes are reasonable. The forward‐looking statements contained in this document are made only as of the date hereof. Except as otherwise required by law, the Company expressly disclaims any obligation or undertaking to release publicly any updates of any forward-looking statements contained in this document to reflect any change in its actual results, assumptions, expectations or any change in events, factors, conditions, or circumstances on which any forward‐looking statement contained in this document is based. 3 Agenda • Welcome & Overview » Matthew Pauls, President & Chief Executive Officer • KEVEYIS » Stephen J. Moloney, MD, Vice President, Global Medical Affairs » Amit Sachdev, MD, Assistant Professor and Director at the Division of Neuromuscular Medicine, Michigan State University » Dave Bonnell, Senior Vice President, Sales and Marketing • KEVEYIS Q&A • Macrilen » Matthew Pauls » Beverly M. K. Biller, MD, Faculty Member at Massachusetts General Hospital Neuroendocrine Unit, Professor of Medicine at Harvard Medical School » Scott Wilhoit, Senior Vice President, Global Market Access and Patient Services • Macrilen Q&A • RECORLEV » Fred Cohen, MD, Chief Medical Officer » Richard Auchus, MD, PhD, Professor of Internal Medicine, Division of Metabolism, Endocrinology & Diabetes, University of Michigan » Matthew Pauls • RECORLEV Q&A • Closing Remarks » Matthew Pauls 4 5 Building Therapeutically Aligned Rare Disease Franchises Rare Rare Rare Disease Neuromuscular Endocrine Franchise #3 Adult Growth Hormone Deficiency Primary Periodic Paralysis RECORLEVTM Business (levoketoconazole) development Cushing’s Syndrome opportunities Veldoreotide Acromegaly 5 Rare Disease Commercial and Late-Stage Portfolio RECORLEV™ Veldoreotide (levoketoconazole) modified-release FDA-APPROVED FDA-APPROVED PHASE 3 PRECLINICAL The 1st and only The 1st and only An investigational Potential FDA-approved drug FDA-approved next-generation next-generation for ultra-rare oral drug for cortisol inhibitor somatostatin Primary Periodic assessing for Cushing’s analog for Paralysis* Adult Growth Syndrome Acromegaly (PPP) Hormone Deficiency (AGHD) ACQUIRED JANUARY 2018 ORPHAN ORPHAN ORPHAN ORPHAN *FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis 6 Medical Affairs Stephen J. Moloney, MD 7 Strongbridge Speakers & Scientific Advisors in PPP Oregon Health and Science University University of SUNY Rochester Buffalo Michigan State University of California University of Utah University San Francisco Brigham and The Ohio Women’s Hospital University of Kansas State University Medical Center University of California Loma Linda University of Cincinnati Los Angeles University Gardener Neuroscience Institute University of Southern California UT Southwestern Medical Center UT Health University of Miami San Antonio 8 Uncovering Periodic Paralysis: No-Cost Genetic Testing • No-cost gene panel sequencing* for hypokalemic and hyperkalemic periodic paralysis • Panel includes comprehensive analysis of the following three most commonly associated genes: SCN4A, CACNA1S, and KCNJ2 • If medically appropriate, re-requisition to a more expansive neuromuscular panel within 90 days of original test report • Family variant testing is available to any first-degree relative of a patient newly diagnosed through the program *Eligibility: Physician-ordered for patients 18+ years of age with episodic muscle weakness/paralysis attacks provoked by at least one of the common triggers for hyperkalemic or hypokalemic Primary Periodic Paralysis 9 The First US Patient Registry Focused on PPP • Collaborative efforts are ongoing with physician experts and advocacy groups to create a patient-focused registry • Areas of focus: » Disease burden » Long-term outcomes of PPP » Impact of treatment interventions • Guide education and research focus in PPP Registries that track patients over time lead to better understanding of the disease, its subtypes, and the impact of interventions. 10 Key Publication Activities • Current activities: » AAN posters » PK study publication » Review article – Diagnosis and Treatment of PPP (Statland et al.) • Ongoing activities: » Analysis of clinical data – Long-term efficacy, characterization and management of adverse events, quality of life exploration » Diagnosis and genetic testing review article 11 Amit Sachdev, MD Assistant Professor of Neurology and Director of the Division of Neuromuscular Medicine, Department of Neurology and Ophthalmology, Michigan State University • Board-certified in neurology, neuromuscular medicine, and electrodiagnostic medicine • Education » MD, Michigan State University » MS, Biomedical Engineering, Wayne State University » BS, Biomedical Engineering, University of Michigan • Postdoctoral Training » Neurology training (Thomas Jefferson University) » Fellowship in neuromuscular medicine (University of Michigan) • Expertise and research interests are focused on neuromuscular medicine The following slide presentation does not reflect the opinions of Michigan State University. 12 Clinical Experience Amit Sachdev, MD The following slide presentation does not reflect the opinions of Michigan State University. 13 Primary Periodic Paralysis (PPP): A Rare, Complex, and Physically Disabling Condition • A spectrum of rare and chronic neuromuscular disorders with autosomal dominant inheritance1 • Recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis2-5 ~4K-5K • Paralytic attacks are acute episodes that can Diagnosed individuals 6 be incapacitating in the U.S.7 • Attacks may last from one hour to several days depending on the type of muscle channel involved2 • As they age, patients may experience permanent muscle weakness, further impacting their quality of life over time4 1. Greig SL. Drugs. 2016;76:501-507. 2. Charles G. J Neurol. 2013;260:2606-2613. 3. Cannon SC. Compr Physiol. 2015;5:761-790. 4. Cavel-Greant D, et al. Acta Myol. 2012;31:126-133. 5. Arya SN. Journal, Indian Academy of Clinical Medicine. 2002;3:374-382. 6. Sansone V, et al. Cochrane Database Syst Rev. 2008 Jan 23;(1):CD005045. 7. Data on file. Available from Strongbridge Biopharma plc. 14 Neuromuscular Disorders That Present With Transient Episodes: The Most Challenging to Diagnose Epilepsy Neurologists commonly consider a range of brain- Migraine centered diagnoses when patients present with an acute Stroke attack of weakness or paralysis: Conversion Disorder 15 Neuromuscular Disorders That Present With Transient Episodes: The Most Challenging to Diagnose Epilepsy ALS Neuromuscular medicine encompasses the Migraine Limb Girdle neurologic system from the neck down: Stroke Myasthenia Gravis PPP NeuromuscularConversion Disorder medicine officially became a board- certified subspecialty in 2012. Conversion Disorder 16 KEVEYIS Clinical Experience—Actual Patient Journey Susan L (41-year-old female)—A Patient with HyperPPP Medical History » Episodes of fluctuating weakness Family History Triggers dating back to her teens » One brother who has » Inactivity and » Predominantly shoulder and hip girdle unexplained episodes cold exposure weakness and overwhelming sense of of stiffness dating back predominantly Presenting Sign fatigue to childhood » Presents to » Attacks occurred for hours and were » Brother does not have ER unable to worse in the morning, worsened a formal diagnosis lift arms above throughout adulthood head x 1 2 3 4 17 KEVEYIS Clinical Experience—Actual Patient Journey (cont.) Susan L – HyperPPP Diagnosis Differential Diagnosis Uncovering Initial Treatment » Outpatient sleep study Periodic Paralysis » Susan became disabled and
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