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Progeria Fact Sheet

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Progeria Fact Sheet Progeria Fact Sheet Eiger BioPharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing first-in-class, well-characterized drugs for patients with life-threatening rare and ultra-rare diseases with high About Eiger unmet medical needs. Eiger has a late-stage pipeline that includes three breakthrough therapy designated programs including its lead clinical program in hepatitis delta virus (HDV) infection. What is Progeria Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria), is an ultra-rare, multisystemic disease that accelerates mortality in young patients due to accumulation of cellular progerin.1 Progeria manifests most visibly with rapid aging.2 Children and young adults with Progeria die of the same cardiovascular disease that affects millions of normally aging adults (arteriosclerosis). Separate and distinct from Progeria, Progeroid Laminopathies are genetic conditions that do not produce progerin, but progerin-like proteins, that result in accelerated aging.3 There are 126 children and young adults identified with Progeria and 55 identified with Progeroid Laminopathies worldwide by The Progeria Research Foundation as of October 2020.4 In the U.S., there are 14 children and young adults with Progeria and 6 with Progeroid Laminopathies.4 See summary of Important Safety Information below and attached full Prescribing Information At-A-Glance 400 children and young adults worldwide estimated to be living with Progeria4 53 countries identified that have children and young adults living with Progeria4 14.5 YEARS Average life expectancy of a patient with Progeria without treatment with Zokinvy5 Zein, Egypt, age 3. Photo courtesy of The Progeria Research Foundation with permission 96 children and young adults with Progeria and Progeroid Laminopathies from more than 30 countries have participated in clinical trials that administered Zokinvy6 Symptoms Disease manifestation results in severe failure to thrive, along with: Accelerated Atherosclerosis with Cardiovascular Decline2,3 Debilitating Strokes2 Scleroderma-like Skin2 Global Lipodystrophy2 Joint Contractures2 Skeletal Dysplasia2 Total Alopecia2 See summary of Important Safety Information below and attached full Prescribing Information Zokinvy™ (lonafarnib) Offers New Hope Zokinvy is a farnesyltransferase inhibitor indicated in adult and pediatric patients 12 months of age and older to reduce the risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and for the treatment of processing-deficient Progeroid Laminopathies.6 Zokinvy is a disease-modifying agent that has demonstrated a statistically significant survival benefit in children and young adults with Progeria.6 Until Zokinvy, there was no approved treatment for Progeria.5 Zokinvy was granted Orphan Drug Designation for Progeria and processing-deficient Progeroid Laminopathies by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) and Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the FDA.7,8 On November 20, 2020, Zokinvy received approval from the U.S. FDA as the first-ever treatment for Progeria and processing-deficient Progeroid Laminopathies. Clinical Snapshot In clinical studies, Zokinvy demonstrated survival benefit in children and young adults with Progeria.6 60% Many children and young adults 10+ YEARS with Progeria have received Reduced incidence therapy for >10 years.4 of mortality.6 2.5 Increase in average Dosed in more than 90 children and 6 90 6 YEARS survival time. young adults in clinical studies. The most commonly reported adverse reactions were gastrointestinal.6 See information below. See summary of Important Safety Information below and attached full Prescribing Information Indication & Usage ZOKINVY is indicated in patients 12 months of age and older with a body surface area of 0.39 m2 and above: • To reduce risk of mortality in Hutchinson-Gilford Progeria Syndrome • For treatment of processing-deficient Progeroid Laminopathies with either: - Heterozygous LMNA mutation with progerin-like protein accumulation - Homozygous or compound heterozygous ZMPSTE24 mutations Limitations of Use • Not indicated for other Progeroid Syndromes or processing-proficient Progeroid Laminopathies. Based upon its mechanism of action, ZOKINVY would not be expected to be effective in these populations. Contraindications • Strong or moderate CYP3A inhibitors or inducers • Midazolam • Lovastatin, simvastatin, and atorvastatin Important Safety Information • The most common adverse reactions are vomiting (90%), diarrhea (81%), infection (78%), nausea (56%), decreased appetite (53%), fatigue (51%), upper respiratory tract infection (51%), abdominal pain (48%), musculoskeletal pain (48%), electrolyte abnormalities (43%), headache (37%), decreased weight (37%), increased aspartate aminotransferase (35%), myelosuppression (35%), cough (33%), decreased blood bicarbonate (33%), hypertension (29%), and increased alanine aminotransferase (27%). • Gastrointestinal adverse reactions were the most frequently reported adverse reactions: The majority were mild or moderate in nature. • Over the course of the trials, 18 (29%) patients had hypertension based on systolic blood pressure or diastolic blood pressure measurements above the 95th percentile on 3 or more occasions. • As nephrotoxicity was seen in animal studies, renal function should be monitored at regular intervals during ZOKINVY therapy. • Increased liver enzymes were generally mild to moderate in nature, asymptomatic with no evidence of impaired liver function. Please see full Prescribing Information at www.Zokinvy.com References 1. Eriksson M., Brown W.T., Gordon L., et. al. (2003). Recurrent de novo point mutations in lamin a cause hutchinson-gilford progeria syndrome. Nature. 423:293-297 2. Merideth M.A., Gordon L., Clauss S., et. al. (2008). Phenotype and course of hutchinson-gilford progeria syndrome. New England Journal of Medicine. 358:592-604 3. Gerhard-Herman M., Smoot L.B., Wake N., et. al. (2012). Mechanisms of premature vascular aging in children with hutchinson-gilford progeria syndrome. Hypertension. 59:92-97 4. Progeria Research Foundation International Progeria Registry. https://www.progeriaresearch.org/international-progeria-registry/. Accessed October 27, 2020 5. Gordon L.B., Shappell H., Massaro J., et. al. (2018). Association of lonafarnib treatment vs no treatment with mortality rate in patients with hutchinson-gilford progeria syndrome. JAMA : The Journal of the American Medical Association. 319:1687-1695 6. Zokinvy (lonafarnib) [package insert]. Approved: [2020, November 20] 7. Eiger Biopharmaceuticals. (2018, October 22). Eiger BioPharmaceuticals Receives FDA Rare Pediatric Disease (RPD) Designation for Lonafarnib for the Treatment of Progeria and Progeroid Laminopathies and Plans NDA Filing in 2019. [Press Release]. Retrieved from [https://www.prnewswire.com/news-releases/eiger-biopharmaceuticals-receives-fda-rare-pediatric-disease-rpd-designation-for- lonafarnib-for-the-treatment-of-progeria-andprogeroid-laminopathies-and-plans-nda-filing-in-2019-300734993.html] 8. Eiger Biopharmaceuticals. (2020, March 23). Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib for Treatment of Progeria and Progeroid Laminopathies. [Press Release]. Retrieved from [https://www.prnewswire.com/news- releases/eigerbiopharmaceuticals-completes-submission-of-new-drug-application-to-fda-for-lonafarnib-for-treatment-of-progeria-and- progeroidlaminopathies-301027939.html] HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use ADVERSE REACTIONS ZOKINVY safely and effectively. See full prescribing information The most common adverse reactions (incidence ≥25%) are vomiting, for ZOKINVY. diarrhea, infection, nausea, decreased appetite, fatigue, upper respiratory tract infection, abdominal pain, musculoskeletal pain, electrolyte TM abnormalities, decreased weight, headache, myelosuppression, increased ™ ZOKINVY (lonafarnib) capsules, for oral use aspartate aminotransferase, decreased blood bicarbonate, cough, Initial U.S. Approval: 2020 hypertension, and increased alanine aminotransferase (6.1) To report SUSPECTED ADVERSE REACTIONS, INDICATIONS AND USAGE contact Eiger BioPharmaceuticals, Inc. at 833-267-0545 ZOKINVY is a farnesyltransferase inhibitor indicated in patients 12 months or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. of age and older with a body surface area of 0.39 m2 and above (1): • To reduce risk of mortality in Hutchinson-Gilford Progeria Syndrome DRUG INTERACTIONS • For treatment of processing-deficient Progeroid Laminopathies with • Reduce to or continue at 115 mg/m2 twice daily with concomitant use either: of weak CYP3A inhibitors (2.3, 7) ○ Heterozygous LMNA mutation with progerin-like protein • See Full Prescribing Information for additional information regarding accumulation drug interactions (2.4, 4, 5.1, 7) ○ Homozygous or compound heterozygous ZMPSTE24 mutations See 17 for PATIENT COUNSELING INFORMATION and FDA-approved Limitations of Use: patient labeling Not indicated for other Progeroid Syndromes or processing-proficient Revised: 11/2020 Progeroid Laminopathies. Based upon its mechanism of action, ZOKINVY would not be expected to be effective in these populations (1) DOSAGE AND ADMINISTRATION FULL PRESCRIBING INFORMATION: CONTENTS* 2 • Start at 115 mg/m twice daily with morning and evening meals (2.1) 1 INDICATIONS
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