Chiesi Global Rare Diseases Highlights Advancements in Lipodystrophy Research at ENDO 2026
Key Insights
Chiesi Global Rare Diseases (search) presented five clinical abstracts at ENDO 2026 covering metreleptin research in both generalized and partial lipodystrophy (search), including a Phase 3 and Phase 4 study.
A survey of patients and caregivers revealed that more than half lacked mental health support despite significant psychological burden, highlighting a critical care gap in lipodystrophy (search) management.
Real-world data on combined metreleptin and GLP-1 receptor agonist (search) therapy were presented, expanding the clinical understanding of treatment approaches in rare endocrine disorders.
BOSTON, June 18, 2026 — Chiesi Global Rare Diseases (search), a business unit of the Chiesi Group, announced the presentation of five clinical abstracts at the Endocrine Society's Annual Meeting (ENDO 2026), held June 13–16 in Chicago, Illinois. The data span the metreleptin clinical program in partial lipodystrophy (search), the safety and tolerability profile of metreleptin in generalized lipodystrophy, real-world prescribing patterns of combined metreleptin and GLP-1 receptor agonist (search) therapy, and the mental health burden faced by patients and caregivers.
"Continued research across both clinical and real-world settings is essential to advancing our understanding of treatment approaches in rare endocrine and metabolic disorders," said Rachele Berria, MD, PhD, Senior Vice President, Head of Global Medical Affairs, Chiesi Global Rare Diseases (search). "We are proud to share these findings at ENDO 2026, which reflect our commitment to generating meaningful data while integrating the experiences of people living with lipodystrophy (search)."
Phase 3 and Phase 4 Metreleptin Data
Among the presentations, Dr. Elif A. Oral presented results from a 12-month randomized, multicenter, double-blind, placebo-controlled Phase 3 study evaluating the safety and efficacy of metreleptin in subjects with partial lipodystrophy (search) (the METRE-PL Study). A separate 36-month, multicenter, open-label Phase 4 study assessed the immunogenicity of daily metreleptin treatment in patients with generalized lipodystrophy, also presented by Dr. Oral.
Dr. Baris Akinci contributed a systematic review examining the effectiveness and safety of metreleptin therapy in patients with partial lipodystrophy (search), while Dr. Lindsay T. Fourman presented real-world prescribing patterns of combined metreleptin and GLP-1 receptor agonist (search) therapy in adults with lipodystrophy.
Mental Health Burden and Care Gaps
Kate Stratton presented findings from a survey of patients and caregivers that characterized mental health challenges and care gaps in lipodystrophy (search). The data highlighted that the mental health burden in lipodystrophy is multifaceted, driven by factors such as altered body image and diagnostic uncertainty. Despite this, more than half of the respondents lacked mental health support, underscoring a critical gap in comprehensive care.
"For people living with lipodystrophy (search), the burden of disease can extend beyond what can be measured clinically," said Stuart Siedman, Vice President, Patient Advocacy, Chiesi Global Rare Diseases (search). "By advancing research that captures both the biological and lived experience, we aim to help ensure care reflects the full reality of patients' needs."
About Lipodystrophy (search) and Myalept
Lipodystrophy (search) 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 generalized lipodystrophy, characterized by near-complete absence or progressive loss of adipose tissue, and partial lipodystrophy, where tissue loss is more limited, typically impacting limbs or the upper body. Lipodystrophy is further categorized by etiology into inherited and acquired forms.
Myalept (metreleptin) for injection is a leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (search). The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established. Myalept is not indicated for use in patients with HIV-related lipodystrophy or in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy.
Myalept carries a Boxed Warning regarding the risk of anti-metreleptin antibodies with neutralizing activity and the risk of lymphoma. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy (search), both treated and not treated with Myalept. Due to these risks, Myalept is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). The most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain.
ENDO 2026 marks an important moment of scientific advancement for the endocrine community and reflects Chiesi's ongoing commitment to advancing research in rare endocrine and metabolic diseases through research, collaboration, and holistic care.