Financing and Revenue - The company has not generated significant revenues and anticipates requiring approximately $20 million in financing to commence a Phase 2 clinical trial for BRTX-100, with an additional $45 million needed for further clinical trials and general operations [35]. - The company has outstanding debt of approximately $5,161,916 as of December 31, 2018, which needs to be addressed alongside funding for clinical trials [35]. - The company has incurred $1,513,150 and $2,152,433 in research and development expenses for the years ended December 31, 2018 and 2017, respectively [95]. - The company has entered into multiple research agreements, including a $250,000 initial payment from Pfizer and up to an additional $525,000 during a two-year term [73]. - The company has not generated significant revenues yet, making it difficult to predict the regulatory status of its products [110]. Clinical Trials and Product Development - BRTX-100 is an autologous mesenchymal stem cell product derived from the patient's own bone marrow, designed to alleviate chronic lower back pain caused by degenerative disc disease [40]. - The Phase 2 clinical trial for BRTX-100 is planned to involve 72 patients, randomized 2:1, with a primary efficacy endpoint at 12 months and safety follow-up at 24 months [58]. - The company has received FDA authorization to commence the Phase 2 clinical trial for BRTX-100, expected to start in Q3 2019, contingent on funding [41]. - The animal study for BRTX-100 showed a statistically significant increase in disc height and improvement in disc histology compared to the control group at day 120 [56]. - The company is exploring potential licensing agreements for BRTX-100 to reduce the need for substantial capital to complete clinical trials and commercialization [59]. - The company is also pursuing a pre-clinical research initiative, the ThermoStem Program, utilizing brown adipose-derived stem cells for metabolic diseases [64]. - The company is developing a bioengineered implantable brown adipose tissue construct to target obesity and metabolic disorders using BADSCs [68]. - Pre-clinical animal models showed significant reductions in weight and blood glucose levels, with differentiated BADSCs supported by a biological scaffold [69]. - The next generation BAT is expected to have a higher purity of BADSC and a greater percentage of functional brown adipocytes, enhancing therapeutic effects [69]. - The company is focusing on the development of treatment protocols utilizing allogeneic cells from genetically similar donors [68]. - The company anticipates beginning preclinical animal studies for its generation 2 encapsulated brown adipose tissue construct by the second quarter of 2019 [79]. - The company anticipates that its cell product candidates will be marketed to healthcare professionals, hospitals, and research institutions upon regulatory approval [104]. Regulatory Compliance and Challenges - The FDA requires a series of clinical trials (Phase 1, 2, and 3) to establish the safety and efficacy of drugs and biologics before they can be marketed [117][121][123]. - If the FDA does not regulate the company's product candidates solely under HCT/P regulations, they may be classified as drugs or biological products, requiring significant resources for compliance [129]. - The company must comply with extensive FDA regulations regarding the manufacturing, testing, and marketing of its products, which can impose costly procedures [125]. - The regulatory process for obtaining FDA approval can take many years and is unpredictable, depending on the product and FDA requirements [129]. - The FDA has broad regulatory authority over drugs and biologics marketed in the U.S., including oversight of clinical trials and manufacturing practices [116]. - The company may face enforcement actions from the FDA if it fails to comply with regulatory requirements, which could adversely affect its operations [114]. - The FDA can impose various enforcement actions for non-compliance, including fines, product seizures, and potential criminal prosecutions, which could materially adversely affect the company [132]. - The FDA's Fast Track program allows expedited review for products intended to treat serious conditions, potentially improving the development timeline [133]. - Products may qualify for Breakthrough Therapy designation if they show substantial improvement over existing therapies, leading to intensive guidance from the FDA [134]. - Priority review can reduce the FDA's review time to six months for products that significantly improve treatment safety or effectiveness [136]. - Accelerated approval may be granted based on surrogate endpoints, requiring post-marketing studies to confirm clinical benefits [137]. - The FDA's premarket clearance process can take three to twelve months for 510(k) submissions and one to four years for PMA approvals, with no guarantee of approval [144]. - Compliance with Current Good Manufacturing Practices (cGMP) is essential for products regulated as drugs, biological products, or devices [146]. - The company must comply with the Health Insurance Portability and Accountability Act (HIPAA) and other data privacy regulations, with potential penalties for non-compliance [154]. - Violations of healthcare laws may result in civil and criminal penalties, operational restructuring, and exclusion from federal healthcare programs [159]. Company Operations and Structure - The company has established a laboratory with a clean room facility for the production of cell products, including BRTX-100, for clinical trials and research purposes [43]. - The company has established a laboratory in Melville, New York, for research and potential production of cell-based product candidates [83]. - The company currently has nine employees, with eight being full-time, indicating a small workforce [164]. - There are no definitive plans for establishing stem cell therapy clinics in any country, but the company intends to explore opportunities as they arise [162]. - The company faces uncertainty regarding compliance with foreign government regulations, which may be more stringent than FDA regulations in the U.S. [160]. - The complexity of new and emerging cell therapy regulations in various countries creates greater uncertainty for the international regulatory process [160]. - The company has not thoroughly explored applicable laws and regulations for potential expansion into foreign jurisdictions [160]. - The principal executive offices are located at 40 Marcus Drive, Melville, New York [163]. - The company is classified as a smaller reporting company and is not required to provide supplementary financial information [407].
BioRestorative Therapies(BRTX) - 2018 Q4 - Annual Report