Since the publication of our Executive Informational Overview® (EIO) on Amarantus BioScience Holdings, Inc. (AMBS-OTC) in early September 2014, the company has advanced development of each of its three lead programs. Amarantus published data from a 72-patient study of the LymPro Test® demonstrating the test’s accuracy at identifying patients with moderate-to-severe Alzheimer's Disease versus healthy controls, completed enrollment of an expanded 140-patient study of LymPro for which results are expected by mid-December 2014, submitted its Phase 2b trial design for Eltoprazine in Parkinson's Disease Levodopa-Induced Dyskinesia to the FDA for review and written feedback, and submitted an Orphan Drug application to the FDA for MANF in retinitis pigmentosa, a degenerative eye disease. By the end of 2014, Amarantus expects to be able to launch the LymPro Test® for research markets (Investigational Use Only), and believes it can use the data from the expanded 140-patient LymPro study in support of a broader product launch in 2015.
The development-stage biopharmaceutical company also announced a major milestone this week regarding a potential new product acquisition. Amarantus has entered into an exclusive option agreement to acquire a novel regenerative medicine technology from Lonza Walkersville, Inc., a subsidiary of global life science company Lonza Group Ltd. If exercised by the end of 2014, Amarantus would obtain an Engineered Skin Substitute (ESS) that has been shown in preclinical studies to create a functional skin barrier. It is an autologous (made from a patient’s own tissue) skin replacement product intended to treat severe burn cases where over 50% of a patient’s body has been burned.
Prior to its November 2014 announcement of the potential ESS acquisition, Amarantus conducted 18 months of due diligence. The ESS product candidate has an IND on file with the FDA and holds Orphan Drug status, with early development work conducted under a U.S. defense grant. Amarantus aims to move ESS into a Phase 1/2a clinical trial in the second quarter 2015. As part of the option agreement, Amarantus has also agreed to engage Lonza via a long-term manufacturing services agreement (MSA) to produce ESS for human clinical trials and subsequent commercial distribution.
Amarantus has forecasted a U.S. market opportunity for an improved method to heal severe burns at approximately $500 million per year, based on a patient population of between 500 and 2,000 individuals a year who require costly treatments for severe burns. Without complications, the company has stated that the average cost of treating a severe burn patient is $1.6 million. With complications, this cost can increase to over $10 million as a result of repeated attempts to rehabilitate and reconstruct patients while dealing with infections, delayed wound healing, scarring, disfigurement, fragile skin, and psychological impacts. Globally, this market may exceed $1 billion annually (Source: Amarantus’s November 17, 2014, press release).
As an autologous skin substitute made from a patient’s own tissue, ESS may be less likely to be rejected by the immune system than alternative tissue sources, such as porcine or cadaver grafts. It is also believed that this skin substitute, made of both epidermal and dermal components, can be produced in a large enough size to close severe wounds and burns on a patient’s body. As a result, ESS may have the potential to reduce or even eliminate burn complications as a result of providing better wound coverage and replacing both the dermal and epidermal skin layers.