Clinical Trials

Date: 2017-05-05

Type of information: Presentation of results at a congress


Announcement: presentation of results at the American Association of Clinical Endocrinology

Company: Novelion Therapeutics

Product: metreleptin (analog of the human hormone leptin)

Action mechanism: peptide. Metreleptin is a recombinant analogue of human peptidic hormone leptin.

Disease: generalized lipodystrophy

Therapeutic area: Hepatic diseases - Liver diseases


Trial details:

Latest news:

  • • On May 5, 2017, Novelion Therapeutics announced the presentation of data by academic researchers at the American Association of Clinical Endocrinology taking place in Austin.
  • The study titled "Impact of Metreleptin on Hepatomegaly in Patients with Generalized Lipodystrophy" suggests treatment with metreleptin may reduce liver volume in patients with enlarged liver (hepatomegaly) resulting from complications of generalized lipodystrophy. The study also demonstrated improvements from baseline of key metabolic parameters, including hemoglobin A1c, liver enzymes and triglycerides.
  • The post-hoc analysis of an open-label, prospective study in patients with generalized lipodystrophy assessed both the impact on liver volume and key metabolic parameters when patients were treated with metreleptin as leptin replacement therapy. Of the 34 evaluable patients, 22 had baseline liver volumetric measurements using MRI; all 22 patients had an enlarged liver. Normal liver volume was defined as less than 25 mL/kg of body weight. The post-hoc analysis completed by Elif Oral , M.D., Associate Professor of Medicine, Michigan Medicine, reviewed patients who participated in a prospective, open-label study conducted at National Institutes of Health between 2000-2008, with study drug provided by Novelion Therapeutics' subsidiary. For patients assessed within one year of initiating treatment with metreleptin (N=21), liver volume decreased an average of 25 percent. The mean treatment duration was 10 months. Among these same patients who had additional follow ups after one year (N=14) and had a mean duration of metreleptin treatment of 46 months, liver volume decreased by 35 percent. The most commonly reported adverse events occurring in more than 10 percent of patients were abdominal pain, hypoglycemia, ear infection, fatigue, proteinuria, back pain, diarrhea, headache, menorrhagia, nausea, ovarian cyst, upper respiratory tract infection and weight decline. Patients treated with metreleptin also experienced clinically meaningful reductions from baseline in HbA1c, liver enzymes (both AST and ALT) and triglycerides. Novelion believes that metreleptin may demonstrate clinical results in a wide range of low leptin-mediated rare and metabolic diseases. John Orloff, MD, executive vice president and head of research and development for Novelion, stated, "Analysis like this, combined with 14 years of additional patient data, provides evidence supporting the implications of low leptin levels on various physiologic functions. We are currently evaluating conducting clinical trials to investigate the use of metreleptin in multiple other low leptin-mediated conditions."

Is general: Yes