Metreleptin

-- Presentations highlight new clinical, real-world, and patient-centered insights on metreleptin across lipodystrophy populations --BOSTON, June 18, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced multiple presentations at the Endocrine Society’s Annual Meeting (ENDO), held June 13 – 16, 2026, in Chicago, Illinois. The five clinical abstracts focus on the metreleptin clinical program in partial lipodystrophy, the safety/tolerability profile of metreleptin in generalized lipodystrophy, the combination of metreleptin with GLP-1 receptor agonists in adults with lipodystrophy, and the impact of mental health challenges and care gaps in lipodystrophy. Collectively, these data expand the clinical understanding of metreleptin, the overall disease burden that people living with lipodystrophy face, and reflect Chiesi’s ongoing commitment to advancing care for people living with lipodystrophy. “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. “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. By combining scientific rigor with patient-centered insight, we aim to help inform more comprehensive and effective models of care.” Data from a survey of patients and caregivers 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, highlighting a critical gap. Proactive screening, especially at diagnosis, can help providers move beyond metabolic control to wholistic patient care. “For people living with lipodystrophy, the burden of disease can extend beyond what can be measured clinically,” said Stuart Siedman, Vice President, Patient Advocacy, Chiesi Global Rare Diseases. “By advancing research that captures both the biological and lived experience, we aim to help ensure care reflects the full reality of patients’ needs.” 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. Details of Chiesi-sponsored presentations are as follows: Title: A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily Metreleptin Treatment in Patients with Generalized LipodystrophyFormat: Poster PresentationPresenter: Dr. Elif A. OralDate: Saturday, June 13 Title: Characterizing Mental Health Challenges And Care Gaps In Patients With Lipodystrophy And Their Care PartnersFormat: Poster PresentationPresenter: Kate StrattonDate: Saturday, June 13 Title: A 12-Month Randomized, Multicenter, Double-Blind, Placebo-Controlled Phase 3 Study to Evaluate the Safety and Efficacy of Metreleptin in Subjects with Partial Lipodystrophy (METRE-PL Study)Format: Poster PresentationPresenter: Dr. Elif A. OralDate: Sunday, June 14 Title: Effectiveness and Safety of Metreleptin Therapy in Patients with Partial Lipodystrophy: A Systemic ReviewFormat: Poster Presentation Presenter: Dr. Baris AkinciDate: Sunday, June 14 Title: Real-World Prescribing Patterns of Combined Metreleptin and GLP-1 Receptor Agonist Therapy in Adults with LipodystrophyFormat: Poster PresentationPresenter: Dr. Lindsay T. FourmanDate: Monday, June 15 IMPORTANT SAFETY INFORMATION INDICATION: 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. LIMITATIONS OF USE: 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. Myalept is not indicated for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without concurrent evidence of generalized lipodystrophy. WARNING: RISK OF ANTI-METRELEPTIN ANTIBODIES WITH NEUTRALIZING ACTIVITY AND RISK OF LYMPHOMA Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with Myalept. The consequences of these neutralizing antibodies are not well characterized but could include inhibition of endogenous leptin action and/or loss of Myalept efficacy. Severe infection and/or worsening metabolic control have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients who develop severe infections or show signs suspicious for loss of Myalept efficacy during treatment. Contact Chiesi Farmaceutici S.p.A. at 1-866-216-1526 for neutralizing antibody testing of clinical samples. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with Myalept. Carefully consider the benefits and risks of treatment with Myalept in patients with significant hematologic abnormalities and/or acquired generalized lipodystrophy. Because of these risks associated with the development of anti-metreleptin antibodies that neutralize endogenous leptin and/or Myalept and the risk for lymphoma, Myalept is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myalept REMS PROGRAM. CONTRAINDICATIONS: Myalept is contraindicated in general obesity not associated with congenital leptin deficiency. Myalept has not been shown to be effective in treating general obesity. The development of anti-metreleptin neutralizing antibodies have been reported in obese patients treated with Myalept. Myalept is contraindicated in patients with prior severe hypersensitivity reactions to metreleptin or to any of its components. WARNINGS AND PRECAUTIONS: A dose adjustment, including possible large reductions, of insulin or insulin secretagogue may be necessary in some patients to minimize risk of hypoglycemia. Closely monitor blood glucose in patients on concomitant insulin, especially those on high doses, or insulin secretagogue. Cases of progression of autoimmune hepatitis and membranoproliferative glomerulonephritis (associated with massive proteinuria and renal failure) were observed in some patients with acquired generalized lipodystrophy treated with Myalept. A causal relationship between Myalept and the development and/or progression of autoimmune disease has not been established. Carefully consider the benefits and risks of Myalept treatment in patients with autoimmune disease. Hypersensitivity reactions (eg, anaphylaxis, urticaria or generalized rash) have been reported. Patient should promptly seek medical advice about discontinuation of Myalept if a hypersensitivity reaction occurs. Myalept contains benzyl alcohol when reconstituted with Bacteriostatic Water for Injection. The preservative benzyl alcohol has been associated with serious adverse events and death in pediatric patients, particularly in neonates and premature infants. Preservative-free Water for Injection is recommended for use in neonates and infants. ADVERSE REACTIONS: Most common adverse reactions (≥10%) in clinical trials were headache, hypoglycemia, decreased weight, and abdominal pain. Please see Full Prescribing Information, including Boxed Warning. About LipodystrophyLipodystrophy 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 forms.1 About Chiesi GroupChiesi is a research-oriented international biopharmaceutical 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, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is 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 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development center in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. For more information visit www.chiesi.com About Chiesi Global Rare DiseasesChiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have 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 health care system. For more information visit www.chiesirarediseases.com. Follow @ChiesiGlobalRareDiseases on LinkedIn, Facebook, Instagram, X and YouTube. Chiesi Global Rare Diseases Media ContactSky StriarLifeSci CommunicationsEmail: sstriar@lifescicomms.com References 1) Garg A. Acquired and inherited lipodystrophies. N Engl J Med. 2004 Mar 18;350(12):1220-34. Doi: 10.1056/NEJMra025261. PMID: 15028826. PP-RA-02187 V1.0

SAN DIEGO--( BUSINESS WIRE )-- Entos ® Pharmaceuticals (Entos) , a clinical-stage genetic medicines company committed to using its Fusogenix™ PLV™ technology to develop cures and improve the lives of patients and their families, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M USD grant. The funds will support the completion of IND-enabling activities for the development of ENTLEP001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy (CGL). CGL is a rare genetic disorder caused by mutations in the human leptin gene. People with CGL are leptin-deficient and cannot make adipose tissue. The disorder is associated with severe, hard-to-treat diabetes, hypertriglyceridemia, and potential complications affecting the liver and heart. ENTLEP001 is a systemically administered investigational gene therapy using the Fusogenix PLV platform to express unmodified human leptin in the body. The treatment is designed to be durable, redosable, and provide biologically relevant levels of native human leptin expression. Preclinical studies with ENTLEP001 using a leptin-deficient mouse model resulted in clinically relevant leptin expression, disease-modifying activity including normalization of glucose and insulin levels, and decreased weight gain and food intake. The Fusogenix PLV platform is a fusion-powered delivery system for genetic medicines that combines the best aspects of viral and non-viral approaches. Using a completely new mechanism for intracellular delivery of RNA, DNA and gene editing therapies, Fusogenix PLV is suitable for a wide range of best-in-class genetic medicines. Details regarding the platform’s safety, efficacy, and biodistribution in various animal models were published in Cell in September 2024. "Despite advancements in CGL treatment with FDA-approved metreleptin, there are still significant unmet needs," said Steve Chen, MD, CMO of Entos Pharmaceuticals. "Metreleptin requires daily subcutaneous injections and in some patients is associated with significant injection site adverse events. Although rare, there are reported cases of neutralizing anti-leptin antibody in patients receiving metreleptin. In contrast, ENTLEP001 is a genetic medicine that expresses native human leptin and may require dosing only once or twice a year, eliminating injection site reactions, simplifying patient compliance, and improving outcomes.” "This award from CIRM is an important validation for our lead program in CGL as we work to advance our second product into the clinic using the Fusogenix PLV platform,” said John Lewis, CEO of Entos Pharmaceuticals. “We deeply appreciate their support and recognition of the crucial need to address rare genetic disorders like congenital generalized lipodystrophy and the potential for novel genetic medicine approaches like the Fusogenix PLV to create life-changing treatments for patients and their families." About Congenital Generalized Lipodystrophy (CGL) Congenital generalized lipodystrophy (CGL), also known as Berardinelli-Seip syndrome, is a rare genetic disorder characterized by the near total loss of adipose tissue associated with extreme muscularity that is often present at birth or soon thereafter. CGL is associated with metabolic complications related to insulin resistance such as severe diabetes and hypertriglyceridemia. Diabetes associated with CGL is often very difficult to treat. Additional complications affecting the liver and heart can also occur. The symptoms and severity of CGL can vary greatly from one person to another. CGL is caused by mutations in the human leptin gene LEP . About Entos Pharmaceuticals Inc. A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and re-dosable nucleic acid delivery technologies. Since its inception in 2016, Entos® has been dedicated to advancing next-generation genetic medicines using our proprietary Fusogenix™ PLV™ drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California and London, United Kingdom, respectively. For more information, visit www.entospharma.com , or follow Entos on LinkedIn . About California Institute for Regenerative Medicine (CIRM) At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to www.cirm.ca.gov . Forward Looking Statements This press release contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements contained in this press release include statements regarding Entos and its belief as to the mode of action and potential and benefits of the Fusogenix proteolipid vehicle platform and other statements related to anticipated developments in the Company’s business and technologies. In any forward-looking statement in which Entos expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation, or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause the Company’s actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of Fusogenix as a nucleic acid delivery vehicle, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results, and financial condition. Investors should consult with the U.S. Securities Commission for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements except as required by applicable laws. Entos ® word mark and design logo, Fusogenix™ and PLV™ are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.