San Diego, CA, USA, August 1st, 2018 – Steminent Biotherapeutics Inc. (“Steminent” or “Company”) a private, clinical-stage cell-therapy company with offices in Taiwan, San Diego and Shanghai, is pleased to announce that the Clinical Trial Notification (“CTN”), submitted by their Japan partner; ReproCELL, to the Japan Pharmaceuticals and Medical Device Agency (“PMDA”) for a Stemchymal® SCA Phase II clinical trial for Polyglutamine Spinocerebellar Ataxia (“PolyQ SCA”) has been approved. This is another key milestone for Steminent’s international Stemchymal® SCA Phase II clinical development program with trials now moving ahead in Taiwan, US, and Japan. [Read more…]
On November 11, 2016, ReproCELL and Steminent Biotherapeutics entered into a partnership to “develop and commercialize Steminent’s allogeneic stem cell therapy product, Stemchymal, in Japan.”
ReproCELL and Steminent Partnership
In this article:
- ReproCELL in Japan
- Repro Therapeutics Advancement
- Financial Aspects of the Agreement Between ReproCELL and Steminent for Development of Stemchymal
ReproCELL in Japan
Based on the terms of the agreement between ReproCELL and Steminent:
- ReproCELL will get exclusive rights for the development and commercialization of Stemchymal for treating Spinocerebellar ataxia (“SCA”) in Japan
- ReproCELL will have right of first negotiation for the development and commercialization of Stemchymal for other disease indications
Japan and Regenerative Medicine
The timing of this partnership is significant because the “Act on the Safety on Regenerative Medicine” and revisions to the “Pharmaceutical and Medical device Act” took effect in Japan in 2014. There are subtle, but important differences between these two acts and how they support regenerative medicine development in Japan.
The aim of the Act on the Safety of Regenerative Medicine is to accelerate the clinical application and commercialization of regenerative products, including cell and gene therapies, as well as tissue engineered products. It covers clinical research and medical practice using processed cells and specifies the procedures required to deliver these cells to humans.
In contrast, the PMD Act introduces a regulatory framework for regenerative medicine products. It allows for conditional and time-limited marketing approval to be given to a regenerative medicine product after exploratory clinical trials have demonstrated probable benefit and proven safety.
Time-Limited Conditional Approval
Under these new laws, once a company has demonstrated safety and basic efficiency data in humans and the cell product can be manufactured to the standards described within the Pharmaceutical and Medical Devices (PMD) Act, the cell therapy can be awarded conditional approval for up to 7 years. This allows for the commercial use of the cell product with data reporting requirements and potential for national insurance coverage.
Japan also accelerated its position as a hub for regenerative medicine research with support from Prime Minister Shinzo Abe, who identified regenerative medicine and cellular therapy as key to Japan’s strategy to drive economic growth. The Prime Minister has encouraged a growing range of collaborations between private industry and academic partners through an innovative legal framework approved last fall.
These regulatory changes have the potential to speed up the commercialization of cell therapies within Japan by allowing “time-limited conditional approval.” Assuming safety and probable efficacy have been demonstrated during early-stage clinical trials, companies are permitted to sell cell therapy products within Japan while continuing late-stage clinical trials.
Repro Therapeutics Advancement
According to REPROCELL’s recent press release, this is the first time that REPROCELL will compete within the regenerative medicine market, within Stemchymal representing its first pharmaceutical product.
If all goes as planned, ReproCELL and Steminent state that they will:
- Initiate clinical trials for Stemchymal in 2017
- Secure “time-limited conditional approval” in 2020
- Receive marketing approval in Japan by 2023
Financial Aspects of the Agreement Between ReproCELL and Steminent for Development of Stemchymal
Under the financial terms of the agreement:
- ReproCELL will invest US$1M in Steminent as an allocation of new shares to a 3rd party
- REPROCELL will pay development milestones which will total approximately US$4M
- REPROCELL may make royalty payments during the commercialization of Stemchymal
To learn more, click here to view the full announcement issued by ReproCELL and Steminent.
In your opinion, how has Japan’s attitude towards regenerative medicine affected stem cell research? Share your thoughts in the comments section below.
Editor’s Note – This post was originally published on December 26, 2016 and has been updated for quality and relevancy.
Since the discovery of induced pluripotent stem cells (iPSCs) in 2006, a large and diverse market for iPSC research tools has emerged.
Included below is a ten-year history of iPSC grants (source: RePORT.NIH.gov), clinical trials (source: ClinicalTrials.gov), and scientific publications (source: PubMed.gov).
Each graph below suggests that the iPSC market is expanding. [Read more…]
One of the induced pluripotent stem cells advantages is that they are non-controversial, while being capable of turning into every cell type within the human body.
Stem cell research and experimentation have been in process for well over five decades, as stem cells have the unique ability to divide and replicate repeatedly. In addition, their “unspecialized” nature allows them to differentiate into a wide variety of specialized cell types. Traditionally, scientists have worked with both embryonic and adult stem cells. While the appeal of embryonic stem cells (ESCs) has been their ability to differentiate into any type of cell, there has been significant ethical, moral, and spiritual controversy surrounding their use. On the other hand, adult stem cells are uncontroversial, but they can have a limited differentiation capacity, creating narrowed options for use.
Therefore, induced pluripotent stem cells are advantaged in that they display a promising combination of adult and embryonic stem cell characteristics.
A Look Into Induced Pluripotent Stem Cells Advantages
In this article:
- Discovery of Induced Pluripotent Stem Cells (iPSCs)
- Induced Pluripotent Stem Cells Issues
- Accelerating Progress with Induced Pluripotent Stem Cells (iPSCs)
- BioInformant’s “Induced Pluripotent Stem Cell Industry Report”
- Key Report Findings
Discovery of Induced Pluripotent Stem Cells (iPSCs)
A groundbreaking experimentation in 2006 led to the introduction of induced pluripotent stem cells (iPSCs). These were adult cells that were isolated and then transformed into embryonic-like stem cells through the manipulation of gene expression, as well as other methods. Research and experimentation using mouse cells at Kyoto University in Japan were the first instances in which there was a successful generation of the iPSC. In 2007, a series of follow-up experiments were done at Kyoto University in which human adult cells were transformed into iPSC cells.
Induced Pluripotent Stem Cells Issues
While there has been continued excitement at the prospect of what such artificially re-manufactured cells could contribute to medical advances, there have also been issues along the way. By 2010, there were a number of private companies that were ready to capitalize on the breakthrough technology that iPSCs represent. One such company, Advanced Cell Technology (now Ocata Therapeutics) discovered several problematic issues while conducting experiments for the purpose of applying for FDA approval to use iPSCs in therapeutic applications. Concerns such as premature cell death, mutation into cancer cells, and low proliferation rates were some of the problems that surfaced.
Nonetheless, the progress with iPSCs continued to accelerate within both private industry and academia, and recent years have been a time of massive change for the industry.
Accelerating Progress with Induced Pluripotent Stem Cells (iPSCs)
Cellular Dynamics International as The Global Leader for iPSCs
In a landmark event, Cellular Dynamics International (CDI) went public in July 2013 with an offering that raised $43 million dollars for the company, securing the company’s position as the global leader in producing high-quality human iPSCs and differentiated cells in industrial quantities. Also in 2013, Cellular Dynamics International and the Coriell Institute for Medical Research announced receiving multi-million-dollar grants from the California Institute for Regenerative Medicine (CIRM) for the creation of iPSC lines from 3,000 healthy and diseased donors, a result that will create the world’s largest human iPSC bank.
The First Human Transplant for iPSCs
Furthermore, 2013 was the first year in which clinical research involving transplant of iPSCs into humans was initiated, with Masayo Takahashi of the RIKEN Center in Japan investigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration. While the trial was initiated in 2013 and production of iPSCs from patients began at that time, it was not until August of 2014 that the first patient, a Japanese woman, was implanted with retinal tissue generated using iPSCs derived from her own skin cells. A team of three eye specialists, led by Yasuo Kurimoto of the Kobe City Medical Center General Hospital, implanted a 1.3 by 3.0mm sheet of iPSC-derived retinal pigment epithelium cells into the patient’s retina. Unfortunately, this trial has since been paused due to potential safety concerns, and its continued progress is “on hold” until these safety issues are resolved.
CDI’s cGMP HLA Superdonor Stem Cell Lines
By February 2015, Cellular Dynamics International had announced continued innovation with iPSCs, stating that it would be manufacturing cGMP HLA “superdonor” stem cell lines that will support cellular therapy applications through genetic matching. Currently, CDI has two HLA superdonor cell lines that provide a partial HLA match to approximately 19% of the population within the U.S., and it aims to expand its master stem cell bank by collecting more donor cell lines that will cover 95% of the U.S. population. The HLA superdonor cell lines were manufactured using blood samples and used to produce iPSC lines, giving the cells the capacity to differentiate into nearly any cell within the human body.
Announcement For the Opening of iPSC Therapy Center
Also in February 2015, Kyoto University Hospital in Kobe, Japan, announced it would be opening an iPSC therapy center by 2019, for purposes of conducting clinical studies on iPSC therapies. The announcement further positioned Japan as the leading nation committed to bringing iPSC therapies to clinics. Officials for Kyoto Hospital said it will open a 30-bed ward to test the efficacy and safety of the therapies on volunteer patients, with the hospital aiming to initiate construction at the site in February of 2016 and complete construction by September 2019.
FUJIFILM Holdings Corporation’s Acquisition of CDI
By March 2015, FUJIFILM Holdings Corporation had acquired Cellular Dynamics International (CDI) for $307 million, making CDI a holding company underneath its multinational Japanese parent company. FUJIFILM Holdings is a multinational company headquartered in Tokyo that has historically been known as a photography and imaging company. However, it recently re-positioned itself to become a major stakeholder in the regenerative medicine industry. In addition to acquiring CDI, FUJIFILM has been gradually increasing its reach within the regenerative medicine field, with another notable acquisition being the majority position that it took in Japan Tissue Engineering Co., Ltd., in December 2014.
The Introduction of ORIG3N in the iPSC Market
Finally, a small American biotechnology company, ORIG3N, is now bringing iPSC banking directly to the public through its creation of the world’s largest blood cell repository for iPSC production. Founded in April 2014 and funded by a February 2015 investment of $3.1 million from an investor syndicate led by Hatteras Venture Partners, ORIG3N has been doing this through an innovative program called “LifeCapsule,” in which individuals can store their own iPSCs for future regenerative medicine needs.
In summary, major advances in iPSC clinical research applications, production and differentiation technologies, and biobanking have been seen in recent years. It is clear that iPSCs are a vital research trend within the scientific community and that the cells represent a promising tool for use in the reversal and repair of many previously incurable diseases.
Market Metrics and End-User Survey Results from iPSC Researchers
Since their discovery in 2006, a large and thriving research products market has grown into existence for iPSCs. Indeed, the number of iPSC research products sold worldwide has been growing at a double-digit rate, and 22% of all stem cell researchers now self-report as having used induced pluripotent stem cells within a research project. It is clear that iPSCs are a vital research trend within the scientific community.
BioInformant’s “Global Induced Pluripotent Stem Cell Industry Report”
To assess opportunities and threats within the rapidly changing iPSC marketplace, BioInformant recently released a global strategic report: the “Complete 2015-16 Induced Pluripotent Stem Cell Industry Report.”
A distinctive feature of this report is an end-user survey of 274 researchers (131 U.S./143 International) that identify as having induced pluripotent stem cells as their core research focus. These survey findings reveal iPSC researcher needs, technical preferences, key factors influencing buying decisions, and more. Therefore, they can be used to make effective product development decisions, create targeted marketing messages, and produce higher prospect-to-client conversion rates.
Key Report Findings
Furthermore, this 2015 report contains 175 pages of detailed industry analysis, including:
- Charts, Timelines, and Forecasts for the iPSC Market
- Trend Rate Data for iPSC Grants, Scientific Publications, Clinical Trials, and Patents
- Market Size Determinations, with 5-Year Forecasts
- Competitive Environment Analysis, including Market Share by Competitor
- Preferred Species for iPSC Research
- Breakdown of Market Share by iPSC Product Category
- Consumer Behavioral Patterns
- Preferred Providers of iPSC Products
- Crucial Trends and Unmet Market Needs
- “Tested Sentences” for Selling to iPSC Scientists
- Breakdown of the Marketing Methods Used by Industry Participants
- End-User Survey of iPSC Researchers (131 U.S. / 143 International)
In summary, the “Global Complete 2015-16 Induced Pluripotent Stem Cell Industry Report” is a must-read industry report for executives and investors to optimally position themselves to sell iPSC products. To profit from this lucrative and rapidly expanding market, you need to understand your key strengths relative to the competition, intelligently position your products to fill gaps in the marketplace, and take advantage of crucial iPSC trends.
Remember, all BioInformant products come with a “Greater than 100% Money Back Guarantee.” If the strategies and tools aren’t helpful to you, then we will cheerfully refund your money and you can keep the product.
Claim this report now to profit from this expanding market – or your competition will.
What other induced pluripotent stem cells advantages should be added to this article? Share them in the comments section.
ReproCELL Enters iPSC Marketplace by 2009
In 2006, Shinya Yamanaka’s lab at Kyoto University in Japan created the first induced pluripotent stem cells (iPSCs) from mouse cells. By 2007, a series of follow-up experiments had been competed at Kyoto University in which human adult cells were transformed into iPSCs. Nearly simultaneously, a research group led by James Thomson at the University of Wisconsin-Madison accomplished the same feat of deriving iPSC lines from human somatic cells.
Just a couple years later in 2009, a forward-thinking company, ReproCELL, entered the iPSC marketplace. At that time, the scientific community was just beginning to explore the cell type, including how to derive, maintain, and differentiate iPSCs.
Established in 2003 as a venture company originating from the University of Tokyo and Kyoto University, ReproCELL was the first company to make induced pluripotent stem cell (iPSC) products commercially available with the launch of its human iPSC-derived cardiomyocytes, which it called “ReproCario.” Other stem cell derived cardiomyocytes are now available commercially from Cellular Dynamics International, GE Healthcare, Cellectis, and others.
ReproCELL’s Commercialization of Induced Pluripotent Stem Cell (iPSC) Products
ReproCELL’s innovation in the area of iPSC commercialization has been driven in part by joint research relationships it established in 2003 with Tokyo University and in 2004 with Kyoto University, the eventual site of iPSC discovery in 2006. Since 2009, ReproCELL has expanded its line of iPSC reagents and iPSC-derived cell lines to include heart, liver, and nerve cells.
The company primarily sells these products as research tools, although they also have the potential for use in toxicology and drug discovery applications. Currently, ReproCELL offers the following iPSC products:
- Research reagents optimized for human iPSC culture
- Human iPSC-derived cardiomyocytes, which launched in April of 2009 (the first iPSC product to be sold commercially)
- Human iPSC-derived neurons, launched in October of 2010
- Human iPSC-derived hepatocytes, launched in May of 2012
- Disease model cell generation using human iPS cell technologies
ReproCELL Expands Through Strategic Acquisitions
To date, ReproCELL has furthered its dominance in the area of iPSC products through a series of strategic acquisitions, including acquisition of Reinnervate, BioServe Biotechnologies, and Stemgent, and all occurring in 2014. Over time, ReproCELL has acquired a growing selection of smaller companies, making strategic acquisitions one of its primary techniques for marketplace dominance and company growth.
Most recently, on November 24, 2015, ReproCELL announced that it would be expanding its drug discovery services through its acquisition of the firm Biopta. Based in Glasgow, UK, and Maryland, USA, the Biopta Group describes itself as “providing contract research services to the pharmaceutical industry since 2002 and has established itself as the world leader in the use of fresh functional human tissues to better predict drug activity prior to clinical trials.”
Click here to read the full press release about the event, titled “ReproCELL Acquires Biopta to Expand its Drug Discovery Services.”
To learn more about emerging trends and opportunities within stem cell markets, view our global strategic reports for the stem cell and cord blood industry.