RECORDATI: TO BE HIGHLIGHTED AT AMERICAN ASSOCIATION OF CLINICAL ENDOCRINOLOGY (AACE) ANNUAL MEETING--THE EXPANDED INDICATION FOR ISTURISA® (OSILODROSTAT) FOR THE TREATMENT OF ENDOGENOUS HYPERCORTISOLEMIA IN ADULT PATIENTS WITH CUSHING'S SYNDROME FOR WHOM SURGERY IS NOT AN OPTION OR HAS NOT BEEN CURATIVE

RECORDATI: TO BE HIGHLIGHTED AT AMERICAN ASSOCIATION OF CLINICAL ENDOCRINOLOGY (AACE) ANNUAL MEETING--THE EXPANDED INDICATION FOR ISTURISA® (OSILODROSTAT) FOR THE TREATMENT OF ENDOGENOUS HYPERCORTISOLEMIA IN ADULT PATIENTS WITH CUSHING'S SYNDROME FOR WHOM SURGERY IS NOT AN OPTION OR HAS NOT BEEN CURATIVE

• ISTURISA^® (osilodrostat) is a cortisol synthesis inhibitor that blocks the enzyme 11beta-hydroxylase to help normalize hypercortisolemia in Cushing's syndrome, a rare endocrine condition that can have significant impact on patients and their families
• The expanded indication is supported by an extensive clinical development program

MILAN and BRIDGEWATER, N.J., May 15, 2025 /PRNewswire/ -- Today, Recordati Rare Diseases Inc. announced that further insights into ISTURISA^® as well as its expanded indication will be presented at this year's gathering of the AACE from May 15 through May 17, 2025, in Orlando, FL. This includes commercial and medical information booths for more information on the expanded indication, a LINC 6 study poster presentation on ISTURISA^®, and 2 product theaters, one involving cortisol in Cushing's syndrome and the other, unrelated to ISTURISA^®, highlighting the diagnosis and treatment of acromegaly.

Mario Maldonado, MD, Head of Development and Global Endocrinology at Recordati Rare Diseases commented, "The Recordati team is excited to speak at AACE 2025 about the label expansion of ISTURISA^® to endogenous hypercortisolemia in adult patients with Cushing's syndrome for whom surgery is not an option or has not been curative. Aside from that big news, I take great pride in presenting the LINC 6 poster. Given the nature of endogenous Cushing's syndrome, which often requires ongoing management, the LINC 6 study provides crucial data on the long-term safety and efficacy of ISTURISA^®. This 2-year real-world interim analysis underscores our commitment to advancing treatment options for those affected by this challenging condition."

Key data presentations at AACE 2025:

• Visit Booth #109 to learn about the expanded indication for ISTURISA^® and what it can mean for the treatment of Cushing's syndrome
  
  
• LINC 6 Poster (Session #792): Assessing Long-Term Safety and Efficacy of Osilodrostat in Prior- and New-Use Patients With Endogenous Cushing's Syndrome Enrolled in the Non-Interventional, Multinational LINC 6 Study: 2-Year Real-World Interim Analysis
  • Presented by Mario Maldonado, MD, Head of Development and Global Endocrinology at Recordati Rare Diseases on May 15, from 1:30 PM to 1:45 PM (EDT)
    
    
• Product Theater 1: Is Cortisol the Culprit? An interactive discussion panel to help recognize the signs and symptoms of endogenous hypercortisolemia in patients with Cushing's syndrome
  • Friday, May 16, from 12:15 PM to 1:00 PM (EDT) at the Lunch Product Theater, Learning Zone Theater 2, Orlando Ballroom

RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.p.A.

Registered Office
Via Matteo Civitali, 1 20148 Milano, Italy
Tel. +39 02 487871
Fax +39 02 40073747
www.recordati.com

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Milano R.E.A. No. 401832

Company subject to the Management and Coordination Activity of Rossini Luxembourg S.àr.l

• • The panel will include Anthony Heaney, MD, PhD (Professor in Residence of Medicine at the David Geffen School of Medicine, UCLA), Lauren F. Gratian, MD (Wilmington Health), and Divya Yogi-Morren, MD (Medical Director of the Pituitary Center, Cleveland Clinic)

• Product Theater 2: Breaking Barriers in Acromegaly: A Personalized Approach to Identifying, Diagnosing, and Optimizing Treatment
  • Friday, May 16, from 2:15 PM to 3:00 PM (EDT) at the ET Product Theater, Learning Zone Theater 2, Orlando Ballroom
  • The panel will include Wenyu Huang, MD, PhD, (Associate Professor at the Division of Endocrinology, Northwestern University Feinberg School of Medicine) and Michael H. Shanik, MD, FACP, FACE (Managing Partner at Endocrine Associates of Long Island, Smithtown, NY; Associate Professor at Stony Brook University Hospital, Stony Brook, NY)

In April, Recordati announced that the U.S. Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for ISTURISA^® (osilodrostat) for the treatment of endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is not an option or has not been curative. This is an expansion of the previous indication for the treatment of patients with Cushing's disease, which is a sub-type of Cushing's syndrome. The ISTURISA^® indication expansion was supported by the ISTURISA^® extensive clinical development program, which includes over 350 patients.

Consumer Important Safety Information

WHAT is ISTURISA?

ISTURISA (osilodrostat) is a prescription medicine used to treat elevated levels of cortisol in the blood (endogenous hypercortisolemia) in adults with Cushing's syndrome:

• who cannot have surgery, or
  
  
• who have had surgery which did not cure their Cushing's syndrome It is not known if ISTURISA is safe and effective in children.

IMPORTANT SAFETY INFORMATION

Before starting ISTURISA tell your healthcare provider about all your medical conditions, including if you:

• have or had heart problems, such as an irregular heartbeat, including a condition called prolonged QT syndrome (QT internal prolongation). Your healthcare provider will check the electrical signal of your heart (called an electrocardiogram) before you start taking ISTURISA, 1 week after starting ISTURISA, and as needed after that.
  
  
• have a history of low levels of potassium or magnesium in your blood.
  
  
• have liver problems.
  
  
• are or plan to become pregnant. It is not known if ISTURISA will harm your unborn baby. There are risks to the mother and unborn baby associated with active Cushing's syndrome during pregnancy.
  
  
• are breastfeeding or plan to breastfeed. It is not known if ISTURISA passes into your breast milk. You should not breastfeed if you take ISTURISA and for 1 week after stopping treatment.

Tell your healthcare provider about all the medicines you take, including any prescription and over- the-counter medicines, vitamins, or herbal supplements.

Especially tell your healthcare provider if you take medicines used to treat certain heart problems. Ask your healthcare provider if you are not sure whether your medicine is used to treat heart problems.

ISTURISA can cause serious side effects including:

• Low cortisol levels in your blood (hypocortisolism). Tell your healthcare provider right away if you experience more than one of the following symptoms, as these may be symptoms of very low cortisol level, known as adrenal insufficiency: nausea, vomiting, tiredness (fatigue), low blood pressure, problems with body salt (electrolyte) levels in your blood, stomach (abdominal) pain, loss of appetite, dizziness, low blood sugar.
  
  If you get symptoms of hypocortisolism while taking ISTURISA, your healthcare provider may change your dose or ask you to stop taking it.

• Heart problem or a heart rhythm problem, such as an irregular heartbeat which could be a sign of a heart problem called QT prolongation. Call your healthcare provider right away if you have irregular heartbeats.
  
  
• Increase in other adrenal hormone levels. Your other adrenal hormones may increase when you take ISTURISA. Your healthcare provider may monitor you for the symptoms associated with these hormonal changes while you are taking ISTURISA:
  • Low potassium (hypokalemia).
    
    
  • High blood pressure (hypertension).
    
    
  • Swelling (edema) in the legs, ankles or other signs of fluid retention.
    
    
  • Excessive facial or body hair growth (hirsutism).
    
    
  • Acne (in women).

Call your healthcare provider if you have any of these side effects.

The most common side effects of ISTURISA include very low cortisol levels (adrenal insufficiency), tiredness (fatigue), nausea, headache, and swelling of the legs, ankles or other signs of fluid retention (edema).

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

Please see full Prescribing Information

About Endogenous Hypercortisolemia in Cushing's Syndrome

Hypercortisolemia, which is marked by elevated levels of cortisol, is the underlying cause of endogenous Cushing's syndrome, a rare and serious disease of excess cortisol for any reason (pituitary and nonpituitary). Cushing's disease (a sub-type of Cushing's syndrome) is cortisol elevated on the basis of pituitary overstimulation (ACTH, adrenocorticotropic hormone) of the adrenal glands. Elevated cortisol can lead to a wide range of associated conditions and complications, such as weight gain, high blood glucose, high blood pressure, osteoporosis, thin and fragile skin that bruises easily, muscle weakness, depression, anxiety, and irritability. If endogenous hypercortisolemia in Cushing's syndrome is left untreated, it is associated with severe complications and diseases, including diabetes, osteoporosis, cardiovascular issues, and even increased risk of infection due to the suppression of the immune system.

About ISTURISA^®
ISTURISA^® is a cortisol synthesis inhibitor that works by preventing 11beta-hydroxylase, an enzyme responsible for the final step of cortisol biosynthesis in the adrenal gland, from being created. ISTURISA^® is also approved for the treatment of patients with endogenous Cushing's syndrome in multiple countries outside the U.S. including the European Union (January 2020) and China (September 2024). ISTURISA^® received orphan drug designation from the FDA and the European Medicines Agency for the treatment of endogenous Cushing's syndrome.

Recordati is an international pharmaceutical group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care, and rare diseases. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,450 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose of "unlocking the full potential of life" aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest.

Investor Relations

Eugenia Litz
+44 7824 394 750
investorelations@recordati.it

Gianluca Saletta
+39 348 979 4876
investorelations@recordati.it

Media Relations

ICR Healthcare US:
Alexis Feinberg
+1 203 939 2225
recordatiuspr@westwicke.com

UK, Europe & Rest of World: Jessica Hodgson
+44 7561 424 788
recordati@consilium-comms.com

This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from those forecast for a variety of reasons, most of which are beyond the Recordati group's control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising.

References:

1. Isturisa. Package insert. Recordati Rare Diseases Inc; 2025.

2. Pivonello R, Isidori AM, De Martino MC, Newell-Price J, Biller BMK, Colao A. Complications of Cushing's syndrome: state of the art. Lancet Diabetes Endocrinol. 2016;4(7):611-629. doi:10.1016/S2213-8587(16)00086-3

3. Bertagna X, Pivonello R, Fleseriu M, et al. LCI699, a potent 11β-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing's disease: results from a multicenter, proof-of-concept study. J Clin Endocrinol Metab. 2014;99(4):1375-1383. doi:10.1210/ jc.2013-2117

4. Fleseriu M, Pivonello R, Young J, et al. Osilodrostat, a potent oral 11β -hydroxylase inhibitor: 22-week, prospective, Phase II study in Cushing's disease. Pituitary. 2016;19(2):138-148. doi:10.1007/s11102-015-0692-z

5. Fleseriu M, Biller BMK, Bertherat J, et al. Long-term efficacy and safety of osilodrostat in Cushing's disease: final results from a Phase II study with an optional extension phase (LINC 2). Pituitary. 2022;25(6):959-970. doi:10.1007/s11102-022-01280-6

6. Pivonello R, Fleseriu M, Newell-Price J, et al. Efficacy and safety of osilodrostat in patients with Cushing's disease (LINC 3): a multicentre phase III study with a double-blind, randomised withdrawal phase. Lancet Diabetes Endocrinol. 2020;8(9):748-761. doi:10.1016/S2213-8587(20)30240-0

7. Fleseriu M, Newell-Price J, Pivonello R, et al. Long-term outcomes of osilodrostat in Cushing's disease: LINC 3 study extension. Eur J Endocrinol. 2022;187(4):531-541. doi:10.1530/EJE-22-0317

8. Gadelha M, Bex M, Feelders RA, et al. Randomized trial of osilodrostat for the treatment of Cushing disease. J Clin Endocrinol Metab. 2022;107(7):e2882-e2895. doi:10.1210/clinem/dgac178

9. Gadelha M, Gatto F, Wildemberg LE, Fleseriu M. Cushing's syndrome. Lancet. 2023;402(10418):2237-2252. doi:10.1016/S0140-6736(23)01961-X

10. Tabarin A, Bertherat J, Decoudier B, et al. Safety and effectiveness of osilodrostat in patients with non-pituitary Cushing's syndrome: Results from the retrospective observational LINC 7 study. Poster presented at The Endocrine Society Annual Meeting (ENDO); June 1-4, 2024. Boston, MA.

11. Tanaka T, Satoh F, Ujihara M, et al. A multicenter, phase 2 study to evaluate the efficacy and safety of osilodrostat, a new 11β -hydroxylase inhibitor, in Japanese patients with endogenous Cushing's syndrome other than Cushing's disease. Endocr J. 2020;67(8):841-852. doi:10.1507/endocrj.EJ19-0617

12. Reincke M, Fleseriu M. Cushing syndrome: a review. JAMA. 2023;330(2):170-181. doi:10.1001/jama.2023.11305

13. Fleseriu M, Auchus R, Bancos I, et al. Consensus on diagnosis and management of Cushing's disease: a guideline update. Lancet Diabetes Endocrinol. 2021;9(12):847-875. doi:10.1016/S2213-8587(21)00235-7

14. Nieman LK, Biller BMK, Findling JW, et al. Treatment of Cushing's syndrome: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab. 2015;100(8):2807-2831. doi:10.1210/jc.2015-1818

15. Nieman LK. Cushing's syndrome: update on signs, symptoms and biochemical screening. Eur J Endocrinol. 2015;173(4):M33-M38. doi:10.1530/EJE-15-0464

16. Pivonello R, De Martino MC, De Leo M, et al. Cushing's disease: the burden of illness. Endocrine. 2017;56(1):10-18. doi:10.1007/s12020-016-0984-8

17. Nieman LK, Biller BMK, Findling JW, et al. The diagnosis of Cushing's syndrome: an Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab. 2008;93(5):1526-1540. doi:10.1210/ jc.2008-0125

18. Hu A. Monthly report: new drug approvals in China: September 2024. BaiPharm. Published October 14, 2024. Accessed March 28, 2025. https://baipharm.chemlinked.com/news/monthly-report-new-drug-approvals-in-china- september-2024

19. FDA approves new treatment for adults with Cushing's disease. News release. US Food and Drug Administration. March 6, 2020. Accessed March 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-adults-cushings-disease

20. European Medicines Agency. Public summary of opinion on orphan designation—Osilodrostat for the treatment of Cushing's syndrome. Accessed March 28, 2025. https://www.ema.europa.eu/en/documents/orphan-designation/eu3141345-public-summary-opinion-orphan-designation-osilodrostat-treatment-cushings-syndrome_en.pdf

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SOURCE Recordati