Amarantus Bioscience Holdings Inc (EM)【AMBS】株式ニュース

how's that "MANF March" doing Gerald...?

Amarantus Bioscience – Chairman’s Blog

http://www.thechairmansblog.com/amarantus-bioscience/blogs/

you know the one - AMBS CEO Blog #29

29.) MANF Begins its March Towards the Clinic
May 14th, 2015 - by Joseph Rubinfeld

Throughout my 50+ years in the biopharmaceutical industry, I have seen many programs with great promise, but very few programs that possess the fundamentally groundbreaking scientific potential to modify disease biology inherent in MANF. In the early days of Amgen, I remember EPO was Number 9 on the priority list for product development. I had to lobby extremely hard to get it moved up the ranks to Number 5 (largely due to the concept of using it in an orphan indication, replacement therapy in kidney dialysis patients). Despite the strong science behind EPO, most people in Amgen’s leadership wanted to focus on some other perceived near term potential opportunities like interlukins, inteferon, chicken growth hormone, malaria, hybridomas, etc. Without this lobbying, I am not certain we would have seen the Amgen we now know today.

I saw a similar situation with Amarantus in 2012. Towards the end of that year when I first began investing in Amarantus, and initially joined the Company as an advisor, I believed that the vast potential of the MANF program could be harnessed into a major medical breakthrough by expanding the focus of preclinical studies beyond Parkinson’s disease, and into a broader area of orphan drugs. In 2013, the Company had its first breakthrough in this area, through some groundbreaking work from the University of Miami’s Bascom Palmer Eye Institute. By demonstrating that MANF had tremendous potential to protect rods and cones in degenerative models of retinitis pigmentosa (RP), we had identified an orphan indication for which no competition existed in the market that would allow the Company to conduct some additional proof of concept work to validate prior to moving MANF into the clinic. Shortly thereafter, we generated data supporting the use of MANF in a broad range of ocular conditions, including orphan conditions such as retinal artery occlusion, as well as large indications such as glaucoma. Taken together, MANF’s potential to treat vision loss associated with a range of ophthalmological conditions alone makes it a compelling product opportunity worthy of further development.

However, we now know full well ophthalmology is just the beginning of the MANF opportunity. Either by internal development, collaboration with academics, or evaluation of the scientific literature, we now know that the MANF program’s potential expands far beyond ophthalmology, and into the areas neurology, otology, cardiology and metabolism (diabetes), among others. This vast potential for MANF is what makes today’s announcement personally so exciting for me, as the initiation of IND-enabling studies (which start with initiation of clinical grade manufacturing) has been the rate-limiting step for true product development value creation for MANF not only for the ophthalmology programs, but also the rest of the MANF pipeline. We have spent the last 12 months evaluating clinical grade material suppliers in parallel with generating the scientific and regulatory framework to support a robust IND-enabling program that will take MANF into the clinic where we can get a first glimpse of MANF’s human efficacy profile. We believe Catalent is the best clinical-grade supplier to support our development, and are extremely pleased that the process is now moving forward. Before this major step forward MANF was a wish, now it is on the path to becoming a reality.

Going forward, we will be formalizing agreements with key constituents including key opinion leaders, patient advocacy groups and regulatory experts in order to drive this program into the clinic and begin to treat patients suffering from the many afflictions MANF may impact. This is where true value will be generated and we are extremely pleased that the process has now begun.

As a Board member, I am very pleased to be helping guide the Company’s strategy forward as we have created a truly de-risked product development pipeline that can create value in many different areas, all the while providing MANF with the necessary infrastructure to be diligently moved forward without the need to rush its development and compromise its true value.

Thank you for your continued interest and support, we look forward to sharing more updates as MANF progresses towards the clinic.

Best,
Joseph Rubinfeld, PhD
Independent Director
Amarantus BioScience Holdings