REDONDO BEACH, CA / ACCESSWIRE / November 19, 2015 / The regenerative medicine industry, considered by many to be the next pillar of healthcare, is projected to grow from $2.6 billion to $6.5 billion from 2013 to 2019, according to Transparency Market Research, representing an attractive 12.8% compound annual growth rate. By stimulating the body's own repair mechanisms, these technologies promise to functionally heal previously irreparable tissues or organs and are currently in late stage trials for a growing number of conditions.
Cesca Therapeutics Inc.'s (the "Company") (NASDAQ: KOOL) core platform, SurgWerks(TM) aims to apply regenerative medicine principles to the treatment of ischemic vascular diseases, which are characterized by inadequate blood flow to an organ or part of the body. But unlike many regenerative medicine companies, Cesca's point-of-care ("POC") approach is the commercialization of an "all-in-one" clinical platform, including an innovative portable surgical workstation containing all essential equipment plus an indication specific kit with single-use disposable devices - a classic "razor-razor blade" business model applied to cell therapy.
From start to finish the procedure takes just 90 to 120 minutes; each patient's own cells are collected, processed, analyzed for QC (with companion diagnostics), and injected into the target tissue in the same procedure using a proprietary system of devices. The analytics component assures each patient, being biologically unique, receives a minimum viable cell dose, a feature missing from other autologous cell therapy platforms. Each patient simply makes a single visit to an operating room or endovascular catheterization lab for treatment, making this process one of the most efficient approaches to cellular medicine; in the words of Leonardo Da Vinci "simplicity is the ultimate sophistication."
Innovative Solution to an Unmet Need
Though the Company has a well-developed pipeline of SurgWerks applications, Cesca's most advanced clinical program targets the $2 billion critical limb ischemia ("CLI") market characterized by severe obstruction of the arteries that reduces blood flow to the extremities. CLI is a late phase of Peripheral Artery Disease ("PAD"), which is often associated with diabetes, and progresses to varying degrees of amputation due to non-healing and infected ulcers - all a result from lack of sufficient blood circulation. While medications and surgical options are available to many advanced cases of the disease, according to the think tank Sage Group, an unfortunate 70,000 or more patients are forced to undergo major amputations each year in the U.S. alone, a considerable market that is in need of a solution. The U.S. FDA has approved Cesca to initiate a pivotal clinical trial to study its unique SurgWerks-CLI(TM) platform in this no-option population. SurgWerks-CLI and the VXP System is comprised of a fully-integrated, highly-specialized protocol, single-use disposable kit, and ancillary equipment platform that enables a rapid, door-to-door, 90+ minute intraoperative treatment with the patient's own bone marrow derived cocktail, a combination of stem cells, growth factors and plasma. Once these multipotent cells and growth factors are transplanted, they may promote angiogenesis and alleviate amputation in high-risk CLI patients.
Distinctive Path to Commercialization
Cesca Therapeutics unique path differs in several important ways from its competitors. The SurgWerks System is regulated as a medical device in the United States for which Cesca, upon successful completion of the pivotal trial, will seek FDA approval under a premarket approval ("PMA") to treat CLI. This approach is in stark contrast to the rather lengthy process typical of a biologics license application ("BLA") and new drug application ("NDA") regulatory pathways that non-POC approaches must follow. These latter regulatory requirements are required by essentially all of Cesca's cell therapy peers including Athersys Inc. (NASDAQ: ATHX), and Caladrius Biosciences, Inc. (NASDAQ: CLBS) to mention a few. The PMA pathway potentially represents a timesavings of years over BLAs & NDAs, and thus provides a recognizable advantage to Cesca over its competition.
Furthermore, Cesca's autologous approach (e.g. leveraging the patient's own stem cells) differs from allogeneic programs (use of unrelated cells) in that the safety risks associated with cellular manipulation/expansion, immune response and complications of cell cryopreservation, storage and transport are not a factor. These (and many other) allogeneic challenges continue to confront "off-the-shelf" allogeneic cell therapy models that have yet to be proven efficacious in ischemic diseases. We will continue to watch allogeneic products like Pluristem's (NASDAQ: PSTI) PLX-PAD for advancements in ischemic applications, however, it appears that autologous cell products and not allogeneic are leading the commercial race in this area of development, particularly CLI. Currently, Cesca has very few direct competitors, the most advanced of which is Zimmer Biomet Holdings, Inc. (NYSE: ZBH), which employs a point-of-care medical device and autologous bone marrow treatment protocol; this similarity between Cesca Therapeutics and a surgical device leader like Zimmer Biomet lends a great deal of support to Cesca's approach.
The CLI space with cell therapy solutions has thinned out over the past several years, a typical phenomenon seen as trials progress towards commercialization leaving the front-runners with the lions-share of the market opportunity. Vericel Corp. (NASDAQ: VCEL) - formerly Aastrom Biosciences, for example, appears to have shelved their CLI program leaving Cesca Therapeutics and Zimmer Biomet in good market position. While both companies share the autologous POC approach, Cesca delivers an intra-operative "smart" solution that includes all of the hardware, software, and disposable components. This solution facilitates better control of the complete process, provides real-time companion diagnostics for a consistent therapeutic dose, and closely follows the FDA's current guidance pertaining to commercialization of cellular therapies.
Critical Limb Ischemia Stem Cell Therapy ("CLIRST III") - CLI Pivotal Trial
In June, the Company received FDA IDE approval of a pivotal clinical trial for treatment of CLI in late-stage, no-option patients. The CLIRST III trial will enroll over 200 patients from sites throughout the U.S. and India, with a primary endpoint of major amputation-free survival (see www.clinicaltrials.gov, NCT02538978). The clinical trial, based on prior successful safety and early efficacy feasibility studies, according to the Company's prior public statements is expected to begin enrollment next year.
Diversified Pipeline
Cesca Therapeutics has built up a diversified clinical platform that includes indications well-beyond CLI, including cardiac ischemia. The Company's active cardiovascular programs target an aggregate $2.7 billion market opportunity. Aside from the cardiovascular market, the Company's most advanced orthopedic programs, non-union fractures and avascular necrosis, target an aggregate $6.1 billion market opportunity, while its bone marrow transplant program targets a $100 million opportunity, for a total potential market of $9 billion dollars.
By leveraging its India-based clinical research organization embedded within the Fortis network of hospitals, the Company has built a highly cost-effective approach to feasibility studies and early-stage clinical trials. The Company has also amassed data from over 500 treated patients around the world with several more applications in the pipeline to protect this intellectual property and build significant shareholder value over the long-term.
Looking Ahead
Cesca Therapeutics trades with a market capitalization of just $12 million, despite having FDA approval for a pivotal Phase III CLI clinical trial, and eight other "early but promising shots at the goal" in the pipeline. With its low-cost infrastructure and unique medical device approach, the Company differs greatly from many other regenerative medicine companies in the space - ones that are focused on higher risk allogeneic models or autologous approaches which haven't yet integrated the most advanced medical devices critical to point-of-care cell therapy.
For more information, visit the company's website at www.cescatherapeutics.com.
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SOURCE: Emerging Growth LLC
