Amarantus BioScience Holdings, Inc. is a clinical-stage diagnostics and therapeutics companyfocused on diseases associated with neurodegeneration and protein misfolding-related apoptosis. The Company is building a portfolio across multiple indications and sectors in order to mitigate risk while still leaving significant potential upside in any one or all of its product candidates.
The Company’s lead and most advanced program is a blood diagnostic test for Alzheimer’s disease (AD), called the Lymphocyte Proliferation Test (the LymPro Test®). Being able to differentiate AD early on in individuals with dementia—specifically AD versus those with another type of dementia, for example frontotemporal dementia (FTD), Parkinson’s disease (PD) dementia, vascular dementia, etc.—is a critical component in the diagnostic process. Amarantus believes that it can add significant value in this area, initially and specifically, within the context of patient selection for clinical trials. To date, data has been published in peer-reviewed publications on the LymPro Test®, demonstrating an overall 95% accuracy rating in 160 patients.
The Company is currently looking at a retrospective, prognostic patient record clinical study, where data that was produced in 2005 is now being examined. Patients are being evaluated at the present time in order to study how they have progressed over a nine-year period—data the Company believes could prove to be valuable in positioning the LymPro Test® going forward.
Amarantus anticipates being able to bring the LymPro Test® to market as a Laboratory Developed Test (LDT) under the Clinical Laboratory Improvement Amendments (CLIA) in the U.S. within the fourth quarter of 2014. The assay could have considerable utility in AD clinical trials due to a well-documented history of patient recruitment errors stemming from inaccurate AD diagnoses using earlier methods. The ultimate goal is to gain approval as a companion diagnostic paired with a therapeutic in treating AD. On September 2, 2014, the Company announced the initiation of CLIA-enabling studies at ICON Central Laboratories, a CLIA-certified laboratory that will serve as the facility that carries out the LymPro assay for CLIA launch. As well, the LymPro Test® is in preclinical development for traumatic brain injury (TBI)/chronic traumatic encephalopathy (CTE).
The Company continues to make progress regarding the reimbursement strategy for broad-scale use, which could bring greater leverage for future potential partnership discussions. Amarantus believes that it has identified Current Procedural Terminology (CPT) codes relating to cell cycle dysfunction and AD that could facilitate reimbursement under CLIA or commercial sale—potentially providing a major competitive advantage.
Roughly 5.4 million U.S. individuals suffer from AD and over 500,000 are newly diagnosed each year (one in eight older Americans), with the disease being the third leading cause of death in the U.S. Costs for AD are likely to exceed $1.4 trillion by 2050. Between 2000 and 2010, deaths from AD increased 66% while deaths from other major diseases decreased, with it being the only cause of death among the top 10 in the U.S. that cannot be prevented, cured, or slowed. Amarantus believes the market opportunity for an effective blood diagnostic for AD, such as its LymPro Test®, could surpass $500 million per year in the U.S.
Eltoprazine, a Phase 2b-ready 5-HT1a/1b partial agonist, was in-licensed from PGI Drug Discovery LLC (formerly PsychoGenics Inc.) in January 2014. The lead indications under development for this compound are Parkinson’s disease Levadopa-Induced Dyskinesia (PD-LID), a condition linked to the PD medication levadopa, as well as for Adult Attention Deficit Hyperactivity Disorder (ADHD). To date, over 700 patients have been dosed with Eltoprazine at varying doses as high as 30mg, noting that the active dose in both PD-LID and Adult ADHD is 5mg. Primary and secondary endpoints have been met in Phase 2 trials for both PD-LID and Adult ADHD. Amarantus is now sourcing contract manufacturers for clinical-grade material and establishing study designs with the European Medicines Agency (EMEA) and with the U.S. FDA for the initiation of Amarantus’ next clinical studies, for which Phase 2b is expected to begin in 2014.
In 2012, the Michael J. Fox Foundation (MJFF) funded human clinical proof-of-concept studies with Eltoprazine. As well, the National Institutes of Mental Health (NIMH) in Cognitive Impairment Associated with Schizophrenia (CIAS) funded work on Eltoprazine. Statistically significant effects were seen in clinical data from Adult ADHD patients.
Mesencephalic-Astrocyte-derived Neurotrophic Factor (MANF), a preclinical program, is a therapeutic protein being developed for Retinitis Pigmentosa (RP) and other orphan diseases, as well as PD, Alzheimer’s disease, Wolfram’s Syndrome, diabetes, and ischemic heart disease. Amarantus is currently working toward filing an Investigational New Drug (IND) Application with the FDA for MANF. Functional experiments are ongoing at the University of Miami Bascom Palmer Eye Institute, which has been U.S. News & World Report’s top-ranking hospital in ophthalmology for over a decade.
Amarantus is preparing to engage with the FDA’s Office for Orphan Products Development to initially develop the compound as a disease-modifying treatment for RP—an inherited, degenerative eye disease that causes severe vision impairment and often blindness by age 40. The Company has made a strategic decision to focus development in orphan indications and is leveraging data assembled internally by a number of contract research organizations (CROs), and by academic collaborators, such as the Buck Institute for Research on Aging, the University of Massachusetts Medical School, and the University of Miami.
During 2014, the Company considerably expanded its management team and Board of Advisors. In February 2014, the Company appointed Mr. Kerry Segal as head of business development and Ms. Tiffini Clark as head of regulatory affairs. In March 2014, Dr. Louis Kirby, a board-certified neurologist and a specialist in developing drugs, medical devices, and laboratory developed diagnostics, joined the Board of Advisors, assisting Amarantus’ LymPro Test® efforts. In April 2014, Amarantus hired Dr. Charlotte Keywood as chief medical officer (CMO) of the therapeutics division, bringing her experience in developing drugs for PD-LID, as well as brought on Mr. Robert Farrell as chief financial officer (CFO), prior CFO of Titan Pharmaceuticals Inc., Sanovas Inc., One World Health, and Fresenius USA.
For the next 12 months, Amarantus intends to focus primarily on the following goals: (1) the commercialization of the LymPro Test®; (2) further clinical development of Eltoprazine; and (3) preclinical development of MANF. The Company has worked to improve its cash position, access to capital, and balance sheet with a goal of listing to a national exchange. At June 30, 2014, the Company had cash and cash equivalents of approximately $1.4 million and access to an additional $19.6 million of equity capital available under a financing facility with Lincoln Park Capital Fund, LLC.