U.S. Stem Cell Responds to FDA’s Regenerative Medicine Guidelines

Sunrise, FL Nov. 21, 2017 – U.S. Stem Cell, Inc. (OTC: USRM), a leader in the development of proprietary, physician-based stem cell therapies and novel regenerative medicine solutions, today responded to the recently announced Food and Drug Administration’s comprehensive policy framework for the development and oversight of regenerative medicine products, including stem cell treatments.

The framework is described in a suite of four guidance documents that build upon the FDA’s existing, risk-based regulatory approach, in order to more clearly describe what products are regulated as drugs, devices, and/or biological products. Two of the documents were issued as drafts, and the other two were issued as final.

This new framework also includes more regulatory guidance for off-the-shelf products that, due to their construct, require more complex, lengthy and expensive entries to market. This is partially because they can involve using third-party stem cells that have been modified, including genetically, which can require more intense scrutiny by the FDA. USRM believes that’s it’s in-clinic stem cell procedures should not be subject to the same regulatory process as off-the-shelf drug products.

Likewise, due to the high ramp-up costs with an off-the-shelf gene or cell therapy product, patients can be up against prices of $475,000 per treatment.  USRMs proprietary in-clinic protocols using the patients own tissues/cells are not off-the-shelf gene or cell therapies and can be safely performed in-clinic instead of a hospital, by physicians that are USRM-trained and certified in autologous stem cell therapy practices — procedures that have been and continue to be well documented in the scientific literature with very few complications reported.  The costs to patients typically range from $5000-$12,000.

“As U.S. Stem Cell continues to offer products, education and in-clinic therapeutic services, we are continuing our ongoing communication with the FDA in order to determine more specifically how we best fit into the regulatory model for stem cell therapies,” said Mike Tomas, President & CEO of U.S. Stem Cell, Inc. “Our goal is to provide patients in need with regenerative medicine using the cells inside their own body, and we are hopeful that the FDA is willing to continue to work with us to help define fair regulation for the public regarding these therapies.”

According to the FDA, this modern framework is intended to balance the agency’s commitment to safety with mechanisms to drive further advances in regenerative medicine, so that innovators can bring new, effective therapies to patients as quickly and as safely as possible.

“We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; new genes can be introduced into the body to combat disease; and adult stem cells can generate replacements for cells that are lost to injury or disease. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine,” said FDA Commissioner Scott Gottlieb, MD, who is quoted in the guidelines.

“Just as Dr. Gottlieb has described it, the emerging field of stem cell therapy is dynamic and complex,” said Dr. Kristin Comella, Chief Science Officer of USRM. “That’s one of the reasons why the FDA is taking great consideration, to ensure a comprehensive framework that will simultaneously increase access to these remarkable innovations, while at the same time ensuring public safety. It is also one of the reasons why we here at USRM are so excited to pioneer the holistic approach of using one’s own stem cells in clinic — the sophistication of using autologous stem cell therapies, combined with simplicity of application and use, gives clinics we operate and physicians we train incredible scalability with powerful results. As we continue to grow and expand our business, we are confident that our place in this world of emerging science will be reflected in the FDA’s ongoing regulatory decisions. We look forward to being a part of this dialogue as this historic science unfolds.”

In addition to the final documents, the two draft guidances also provide important information to help spur development and access to innovative regenerative therapies. The first draft guidance, which builds off the regenerative medicine provisions in the 21st Century Cures Act, addresses how the FDA intends to simplify and streamline its application of the regulatory requirements for devices used in the recovery, isolation, and delivery of regenerative medicine advanced therapies (RMATs), including combination products.

The second draft guidance describes the expedited programs that may be available to sponsors of regenerative medicine therapies, including the new Regenerative Medicine Advanced Therapy (RMAT) designation created by the 21st Century Cures Act, Priority Review, and Accelerated Approval. In addition, the guidance describes the regenerative medicine therapies that may be eligible for RMAT designation – including tissue engineering products, human cell and tissue products, and combination products using any such therapies or products, as well as gene therapies that lead to a durable modification of cells or tissues (including genetically modified cells). USRM intends to take full advantage of this RMAT opportunity and join strategic partners to apply for consideration of its MyoCell product for heart failure patients.

Based on these draft guidance documents, the FDA will continue to require a robust clinical trial program for these products which typically costs hundreds of millions of dollars and years of studies. USRM has spent more than $100 million over two decades on both preclinical (animal) and clinical (human) trials using its MyoCell™ product. The trials published to date show culture-expanded autologous stem cells directly injected into the heart can provide benefit to congestive heart failure patients. USRM is actively seeking strategic partners to pursue commercialization of the MyoCell product and finalize RMAT designation based on the draft guidelines published.

At U.S. Stem Cell and our clinical affiliates, we have demonstrated a strong safety profile with our in-clinic physician medical procedures which is completely in line with the risk-based approach that the FDA intends to enforce. We believe that patients have a right to utilize the cells in their own body to help promote natural healing and we have demonstrated the safety of these therapies in our recent publication in a peer reviewed journal. The study describes the use of stromal vascular fraction (SVF) in the clinic for degenerative diseases in orthopedics, neurological and systemic conditions in 676 patients.

U.S. Stem Cell, Inc., is an emerging leader in the regenerative medicine / cellular therapy industry and has, to management’s knowledge, completed more clinical treatments than any other stem cell company in the world in the past 20 years. We look forward to pioneering this space and working closely with regulatory bodies to ensure safety.

 

About U.S. Stem Cell, Inc.

U.S. Stem Cell, Inc. is an emerging enterprise in the regenerative medicine / cellular therapy industry. We are focused on the discovery, development and commercialization of cell-based therapies that prevent or treat disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function. We believe that regenerative medicine / cellular therapeutics will play a large role in positively changing the natural history of diseases, ultimately, we contend, lessening patient burdens, as well as reducing the associated economic impact disease imposes upon modern society.

Our business, which includes three operating divisions (U.S. Stem Cell Training, Vetbiologics and U.S. Stem Cell Clinic) includes the development of proprietary cell therapy products, as well as revenue generating physician and patient-based regenerative medicine / cell therapy training services, cell collection and cell storage services, the sale of cell collection and treatment kits for humans and animals, and the operation of a cell therapy clinic. Management maintains that revenues and their associated cash in-flows generated from our businesses will, over time, provide funds to support our clinical development activities as they do today for our general business operations. We believe the combination of our own therapeutics pipeline combined with our revenue generating capabilities provides the Company with a unique opportunity for growth and a pathway to profitability.

Forward-Looking Statements: Except for historical matters contained herein, statements made in this press release are forward-looking statements. Without limiting the generality of the foregoing, words such as “may”, “will”, “to”, “plan”, “expect”, “believe”, “anticipate”, “intend”, “could”, “would”, “estimate”, or “continue”, or the negative other variations thereof or comparable terminology are intended to identify forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Also, forward-looking statements represent our management’s beliefs and assumptions only as of the date hereof. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

The Company is subject to the risks and uncertainties described in its filings with the Securities and Exchange Commission, including the section entitled “Risk Factors” in its Annual Report on Form 10-K for the year ended December 31, 2016, and its Quarterly Reports on Form 10-Q.