Chiesi Global Rare Diseases Showcases Ongoing Commitment to Multiple Rare Diseases Communities at ENDO 2024 Meeting Debut

- Results presented from a large, pooled database from three Phase 3 clinical trials evaluating MYCAPSSA (octreotide) for the treatment of people living with acromegaly -

- Additional presentations provide insights into the validation of the Lipodystrophy Severity Scoring tool and the Chiesi Management of Acromegaly (MACRO) registry -

BOSTON, June 14, 2024 - Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, announced updates from their rare endocrine disease portfolio with two late-breaking poster presentations and one oral presentation during the Endocrine Society’s ENDO 2024 meeting that was held June 1-4, 2024, in Boston, Massachusetts. Presentations included a post hoc analysis of three Phase 3 clinical trials (CH-ACM-01, OPTIMAL, MPOWERED) studying MYCAPSSA (octreotide) as well as real-world MYCAPSSA use data from the Chiesi Management of Acromegaly (MACRO) registry and presentation of the Lipodystrophy Severity Scoring tool designed to assess the disease burden of lipodystrophy.

“We’re thrilled to support this community of people living with burdensome rare endocrine diseases through the innovation of new resources and validated medicines to aid in disease management,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases. “As we approach the four-year anniversary of MYCAPSSA being on the market later this month, our commitment remains steadfast to commercializing solutions for the acromegaly and lipodystrophy patient communities.”

Oral Presentation
Title: Biochemical Response, Symptom Control, and Safety of Oral Octreotide Capsules Treatment in Acromegaly: A Post Hoc Analysis of a Large Pooled Database from Three Phase 3 Clinical Trials²

Key Highlights:

• MYCAPSSA demonstrated a 72% response rate in patients entering the studies with IGF-I values of ≤ 1x the upper limit of normal. A patient’s prior dose level of injectable medication did not significantly impact response rates.
• In this post hoc analysis, symptom improvement was notable with significant reductions in joint pain (36% to 22%) and swelling (15% to 10%) in patients responding to MYCAPSSA. Median decrease of one symptom per patient and a mean decrease of 0.8 in Acromegaly Index of Severity (AIS) further underscored symptom improvement.
• Safety analysis revealed gastrointestinal issues as the most common adverse events with a median duration and time to onset of roughly two weeks for the first occurrence and a low rate of discontinuation.
  
  

Late-Breaker Poster Presentations
Title: Real-world Biochemical and Symptoms Outcomes With Oral Octreotide: Management of Acromegaly (MACRO) Registry Experience³

Key Highlights:

• Of the 230 patients participating in Chiesi Management of Acromegaly (MACRO) registry, 32 (14%) received MYCAPSSA at baseline, with an additional 26 (11%) initiating post-baseline. Common reasons for initiation included patient preference and lack of symptom or biochemical control.
• The mean time on OOC was 19.7 months, with the majority receiving treatment for at least 6 months. Most patients remained well-controlled biochemically and symptomatically, with 87% biochemically controlled and 71% symptomatically controlled at the last follow-up.
• The discontinuation rate was 33% and in cases attributed to a lack of biochemical or symptom control, dose titration to 80mg/day was not implemented in many cases.
• Overall, OOC demonstrated efficacy in maintaining or achieving biochemical control with the majority of newly initiated patients reporting well-controlled symptoms.

Title: Lipodystrophy Severity Score: Validation of a Tool to Assess Disease Burden in Lipodystrophy⁴

Key Highlights:

• The lipodystrophy severity score (LDS) is a validated tool that captures severity in patients with diverse manifestations of lipodystrophy. The LDS allows comparisons among patient groups and changes within patients over time.
• The LDS is a tool designed to quantify the diverse complications of lipodystrophy, a group of rare, potentially life-threatening disorders that affect how the body accumulates and stores fat.
• The LDSs showed high reliability and strong correlation with the Clinical Global Impression (CGI) of severity, effectively capturing the severity of lipodystrophy and changes over time. This tool is intended to compare patient groups and monitor individual progress with future analysis involving in real-world data sets.
• Development into an online platform enhances functionality – a versatile and valuable resource for the lipodystrophy community.
• Future work will assess the LDS using real-world datasets.
  
  

MYCAPSSA IMPORTANT SAFETY INFORMATION

INDICATION AND USAGE
MYCAPSSA (octreotide) delayed-release capsules, for oral use, is a somatostatin analog indicated for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

CONTRAINDICATIONS
Hypersensitivity to octreotide or any of the components of MYCAPSSA. Anaphylactoid reactions, including anaphylactic shock, have been reported in patients receiving octreotide.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
MYCAPSSA can cause problems with the gallbladder. Monitor patients periodically. Discontinue if complications of cholelithiasis are suspected. Blood sugar, thyroid levels, and vitamin B12 levels should be monitored and treated accordingly.

Bradycardia, arrhythmia, or conduction abnormalities may occur. Treatment with drugs that have bradycardia effects may need to be adjusted.

ADVERSE REACTIONS
The most common adverse reactions (incidence >10%) are nausea, diarrhea, headache, arthralgia, asthenia, hyperhidrosis, peripheral swelling, blood glucose increased, vomiting, abdominal discomfort, dyspepsia, sinusitis, and osteoarthritis.

DRUG INTERACTIONS
The following drugs require monitoring and possible dose adjustment when used with MYCAPSSA: cyclosporine, insulin, antidiabetic drugs, calcium channel blockers, beta blockers, lisinopril, digoxin, bromocriptine, and drugs mainly metabolized by CYP3A4. Counsel women taking an oral contraceptive to use an alternative non-hormonal method of contraception or a backup method when taking MYCAPSSA. Patients taking proton pump inhibitors, H2-receptor antagonists, or antacids concomitantly with MYCAPSSA may require increased dosages of MYCAPSSA.

PREGNANCY
Advise premenopausal females of the potential for an unintended pregnancy.

Please contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.

About Acromegaly
Acromegaly typically develops when a benign tumor of the pituitary gland produces too much growth hormone, ultimately leading to significant health problems. Common features of acromegaly are facial changes, intense headaches, joint pain, impaired vision, and enlargement of the hands, feet, tongue, and internal organs. Serious health conditions associated with the progression of acromegaly include type 2 diabetes, hypertension, respiratory disorders, and cardiac and cerebrovascular disease. Acromegaly affects over 25,000 people in the United States alone with roughly 8,000 being treated with injectable somatostatin receptor ligands (iSRLs). A significant percentage of these people experience burdens associated with receiving iSRLs, including prolonged injection-related pain and poorly controlled symptoms.

About Lipodystrophy
Lipodystrophy syndromes are a heterogeneous group of rare, potentially life-threatening disorders that affect how the body accumulates and stores fat. These syndromes are categorized into two main forms: generalized lipodystrophy, characterized by the near-complete absence or progressive loss of fat (adipose) tissue, and partial lipodystrophy, where the tissue loss is more limited, typically impacting areas like limbs or upper body. Lipodystrophy is also categorized by etiology with inherited and acquired forms¹.

About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the healthcare system.

For more information visit www.chiesirarediseases.com.

About Chiesi Group
Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.

By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.

With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.

For further information please visit www.chiesi.com

1. Garg A. Acquired and inherited lipodystrophies. N Engl J Med. 2004 Mar 18;350(12):1220-34. Doi: 10.1056/NEJMra025261. PMID: 15028826.
2. Fleseriu M, et al. Biochemical Response, Symptom Control, and Safety of Oral Octreotide Capsules Treatment in Acromegaly: A Post Hoc Analysis of a Large Pooled Database From Three Phase 3 Clinical Trials. Presented at: Endocrine Society’s ENDO 2024 Meeting; June 1-4, 2024; Boston, Massachusetts.
3. Yuen K, et al. Real-world Biochemical and Symptoms Outcomes With Oral Octreotide: Management of Acromegaly (MACRO) Registry Experience. Presented at: Endocrine Society’s ENDO 2024 meeting; June 1-4, 2024; Boston, Massachusetts.
4. Brown R, et al. Lipodystrophy Severity Score: Validation of a Tool to Assess Disease Burden in Lipodystrophy. Presented at: Endocrine Society’s ENDO 2024 meeting; June 1-4, 2024; Boston, Massachusetts.
   
   

Chiesi Group Media Contact
Chiara Travagin
Rare Communication Manager
Tel: +39 348 8818985
Email c.travagin@chiesi.com

Sky Striar
LifeSci Communications
Tel: 617-797-6672
Email sstriar@lifescicomms.com

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