Clinigen Group plc (AIM: CLIN, ‘Clinigen’), the global pharmaceutical and services company, has partnered with Eiger BioPharmaceuticals, Inc. (NASDAQ: EIGR, ‘Eiger’) to launch a worldwide lonafarnib Managed Access Program for patients with Progeria and Progeroid Laminopathies.
Progeria, also known as Hutchinson‐Gilford Progeria Syndrome, is a rare and fatal genetic condition of accelerated aging in children that researchers believe is caused by a genetic mutation that results in an overabundance of the farnesylated aberrant protein named progerin. Signs of Progeria are typically observed within the first two years of life and include growth failure, loss of body fat and hair, aged‐looking skin, stiffness of joints, hip dislocation, generalised atherosclerosis, cardiovascular (heart) disease and stroke.
Lonafarnib is a first‐in‐class, orally active inhibitor of farnesyltransferase, an enzyme involved in the modification of proteins through a process called prenylation. Lonafarnib has been granted Orphan Drug designation for Progeria by FDA and EMA and Breakthrough and Rare Pediatric Disease designation by FDA. Eiger BioPharmaceuticals is currently preparing an NDA with plans to file in 2019.
Healthcare professionals can obtain details about the lonafarnib Managed Access Program by calling the customer service team at +44 1932 824 123 (RoW) or +44 1932 824 100 (UK) or emailing email@example.com; or calling The Progeria Research Foundation at 978‐535‐2594 or emailing firstname.lastname@example.org.
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Clinigen Group plc
Tel: +44 (0) 1283 495010
Shaun Chilton, Group Chief Executive Officer
James Winterman, Senior Vice President, Unlicensed Medicines
Tel: +44 (0) 20 7457 2020
Melanie Toyne‐Sewell / Alex Shaw / Deborah Bell
Notes to Editors
Progeria, also known as Hutchinson‐Gilford Progeria Syndrome (HGPS), is a rare and fatal genetic condition of accelerated aging in children. Progeria is caused by a point mutation in the LMNA gene, encoding the lamin A protein, yielding the farnesylated aberrant protein, progerin. Lamin A protein is part of the structural scaffolding that holds the nucleus together. Researchers now believe that progerin may make the nucleus unstable, and that cellular instability may lead to the process of premature aging in Progeria. Children with Progeria die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), but at an average age of 14.5 years. Disease manifestations include severe failure to thrive, scleroderma‐like skin, global lipodystrophy, alopecia, joint contractures, skeletal dysplasia, global accelerated atherosclerosis with cardiovascular decline, and debilitating strokes. It is estimated that 400 children worldwide have Progeria.
About Progeroid Laminopathies
Progeroid Laminopathies are genetic conditions of accelerated aging caused by a constellation of mutations in the lamin A and/or Zmpste24 genes yielding farnesylated proteins that are distinct from progerin. While non‐ progerin producing, these genetic mutations result in disease manifestations with phenotypes that have overlap with, but are distinct, from Progeria. Collectively, worldwide prevalence of Progeroid Laminopathies is likely greater than Progeria.
Lonafarnib is a well‐characterized, late‐stage, orally active inhibitor of farnesyltransferase, an enzyme involved in modification of proteins through a process called prenylation. Progerin is a farnesylated protein that researchers believe cannot be cleaved, resulting in tight association with the nuclear envelope, which is believed to lead to changes in nuclear envelope morphology and subsequent cellular damage. Lonafarnib blocks the farnesylation of progerin and has been dosed in over 80 children with Progeria at Boston Children’s Hospital in Phase 1/2 and Phase 2 studies funded by The Progeria Research Foundation (www.progeriaresearch.org).
Lonafarnib has been granted Orphan Drug designation for Progeria by the FDA and EMA and Breakthrough and Rare Pediatric Disease designation by the FDA. Lonafarnib is not approved for any indication, and is licensed by Eiger from Merck Sharp & Dohme Corp.
About Clinigen Group
Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing ethical access to medicines. Its mission is to deliver the right medicine to the right patient at the right time through three areas of global medicine supply; clinical trial, unlicensed and licensed medicines. The Group has sites in North America, Europe, Africa and Asia Pacific. In October 2018, the Group acquired CSM, a specialist provider of packaging, labelling, warehousing and distribution services, with sites in the US and Europe, and iQone, a specialist pharmaceutical company in Switzerland. For more information, please visit the website at www.clinigengroup.com.
For more information, please visit www.clinigengroup.com
About Eiger BioPharmaceuticals, Inc.
Eiger is a late‐stage biopharmaceutical company focused on the accelerated development and commercialization of a pipeline of targeted, first‐in‐class therapies for rare and ultra‐rare diseases. The company’s lead program is in Phase 3, developing lonafarnib, a first‐in‐class prenylation inhibitor for the treatment of Hepatitis Delta Virus (HDV) infection. The company is also preparing an NDA with plans to file in 2019 for lonafarnib in the treatment of Hutchinson‐Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies. For additional information about Eiger, please visit www.eigerbio.com.
About The Progeria Research Foundation
The Progeria Research Foundation (PRF) was established in 1999 by Leslie Gordon, MD, PhD and Scott Berns, MD, MPH, parents of a child with Progeria. In 2003 the PRF Genetics Consortium, led by Francis Collins, MD, PhD, isolated the Progeria gene. PRF has been the driving force behind studies to evaluate lonafarnib as a potential treatment for Progeria. PRF has developed programs and services to aid those affected by Progeria and the scientists who conduct Progeria research. Today, PRF is the only non‐profit organization solely dedicated to finding treatments and a cure for Progeria. For more information, please go to www.progeriaresearch.org.