"I am very excited about the future of rhAC. Similar to other enzyme replacement therapies, rhAC has the potential to be a transformative therapy for patients afflicted with Farber disease, a population with clear unmet medical needs," said Dr. Edward Schuchman, Genetic Disease Foundation Francis Crick Professor, Vice Chair for Research, Department of Genetics & Genomic Sciences, at the Icahn School of Medicine at Mount Sinai in New York City. Professor Schuchman will continue to play an integral role in the development of rhAC.
Enzyvant expects to rapidly progress the development of rhAC by:
- Working toward regulatory filings to enable initial clinical trials
- Developing a framework for the systematic collection of natural history data on all patients diagnosed to date
- Seeking collaboration with all physicians and institutions who are caring for Farber patients, or who have diagnosed patients in the past
- Accelerating efforts to educate the medical community about Farber disease, and to provide easily accessible diagnostic testing and guidance for physicians
- Progressing its strategic plan to connect and support patients from around the world, and help them to engage with the larger rare disease patient community
Vivek Ramaswamy, Chief Executive Officer of Roivant Sciences, Inc., commented: "At Enzyvant, we have formed an outstanding team of experts in the field of Farber disease, drawing from the pioneering research performed at Mount Sinai and the patient-focused initiatives at Plexcera. Enzyvant plans to rapidly advance rhAC in an efficient manner centered on serving the needs of patients with Farber disease and their families."
About Farber disease
Farber disease is an ultra-rare lysosomal storage disease caused by a mutation in both alleles of the ASAH1 gene, resulting in the deficiency of the lysosomal enzyme acid ceramidase. This leads to the accumulation of the pro-inflammatory sphingolipid ceramide, and a macrophage driven inflammatory process causing the development of the typical clinical symptoms. Like many other lysosomal storage diseases, Farber disease has a broad phenotypic spectrum, and is likely underdiagnosed.
Farber patients typically present with the cardinal symptoms of:
- Joint contractures or arthritis
- Subcutaneous nodules
- Weak or hoarse voice
It may take years for all three cardinal symptoms to appear together, and they may vary greatly in severity. Patients may also present with systemic inflammation (fever), severe pain, peripheral osteolysis, failure to thrive, and developmental delay.
In addition to Farber disease, a mutation in the ASAH1 gene and the resulting acid ceramidase deficiency may also lead to a severe neurological disorder called spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME) that causes muscle weakness and wasting (atrophy) and a combination of seizures and uncontrollable muscle jerks (myoclonic epilepsy). It is anticipated that rhAC may have a therapeutic role in these patients as well and Enzyvant looks forward to exploring this possibility, taking advantage of ongoing work by a network of academic and clinical research collaborators established by Plexcera and Dr. Schuchman.
If you know of a child or adult with any of the symptoms of Farber disease or SMA-PME, or are a physician interested in collaboration, please get in touch with the Enzyvant team at email@example.com
Recombinant human acid ceramidase (rhAC) is an enzyme replacement therapy (ERT) for Farber disease first developed by Dr. Edward Schuchman at the Icahn School of Medicine at Mount Sinai. Initially licensed by Plexcera, rhAC showed positive results in various preclinical studies, and the IND-enabling work will now be continued at Enzyvant.
Enzyvant Sciences is a new biopharmaceutical company formed by Roivant Sciences and Plexcera Therapeutics focused on the treatment of patients with Farber disease. Enzyvant is completing the pre-clinical studies for rhAC to enable a clinical trial in patients with Farber disease. Enzyvant will benefit from both the leadership and drug development expertise at Roivant and the medical and scientific expertise from Plexcera. Enzyvant Sciences Ltd. is a trading name of Roivant Ultra Orphan Ltd., a Bermuda registered company.
About Roivant Sciences
Roivant Sciences is a unique biopharmaceutical company with world-class drug development experts working across multiple clinical and functional areas to complete the development of promising late-stage drug candidates with the potential to significantly improve the lives of patients and their families. Roivant delivers R&D solutions to its industry partners by helping them unlock value from their pipelines and to realize the full potential of their research by completing the product development cycle.
Roivant's pipeline spans multiple therapeutic areas through strategic alliances, collaborations and partnerships with academic institutions and pharmaceutical companies, including Takeda Pharmaceuticals, Eisai, Vertex Pharmaceuticals, GlaxoSmithKline, Arena Pharmaceuticals, Arbutus Biopharma, Duke University and Cincinnati Children's Hospital Medical Center.
Additional information about Roivant Sciences is available through its website, www.roivant.com.
About Plexcera Therapeutics
Plexcera Therapeutics LLC was formed in 2013 to develop rhAC therapy for Farber disease based on technology developed in the laboratory of Dr. Edward Schuchman and licensed from the Icahn School of Medicine at Mount Sinai. QOL Medical LLC, a specialty biopharmaceutical company with two FDA approved therapies for rare gastrointestinal diseases (www.qolmed.com), provided the Series A funding for Plexcera and is a minority co-investor with Roivant in Enzyvant.
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/roivant-sciences-and-plexcera-therapeutics-announce-the-formation-of-enzyvant-sciences-to-focus-on-the-treatment-of-farber-disease-300297825.html
SOURCE Roivant Sciences Ltd.