Financing and Grants - The company completed a $23,000,000 public offering of securities, issuing 2,300,000 shares of common stock and warrants for 2,645,000 shares[25]. - The company received a National Institutes of Health STTR Phase 1 grant for $256,000 to evaluate therapeutic effects on hypoxic cultured bone marrow derived mesenchymal stem cells[22]. - The company anticipates requiring approximately $35,000,000 in additional financing to complete the Phase 3 clinical trial for BRTX-100[43]. Clinical Trials and Research - The company has initiated a Phase 2 clinical trial for BRTX-100, which is aimed at treating chronic lower back pain from degenerative disc disease[32]. - The Phase 2 clinical trial for BRTX-100 involves 99 patients randomized 2:1, with a primary efficacy endpoint at 12 months, focusing on improvement in function and reduction in pain[68]. - The FDA authorized the Phase 2 clinical trial in February 2017, and the company has commenced the trial with a CRO agreement and expanded laboratory capabilities[49][66]. - A National Institutes of Health STTR Phase 1 grant of $256,000 was awarded to evaluate the therapeutic effects of BRTX-100 after encapsulation with a PEG-peptide hydrogel[57]. - The company has established a laboratory with clean room facilities for the production of cell products, including BRTX-100, for clinical trials and research purposes[52]. - The company plans to initiate additional preclinical animal studies in 2022 to optimize delivery and explore additional indications[103]. - The company is pursuing its Disc/Spine Program with the investigational therapeutic product BRTX-100, which has received FDA authorization to commence a Phase 2 clinical trial[210]. Product Development and Technology - The company has obtained five United States patents and nine foreign patents related to the ThermoStem Program, along with three United States patent applications and eight foreign patent applications[41]. - The company has licensed a curved needle device for delivering therapeutic products, pending FDA approval[35]. - BRTX-100 is an autologous hypoxic cultured mesenchymal stem cell product derived from a patient's own bone marrow, designed to enhance viability and therapeutic potential with an expected cell count of approximately 40 million per dose[54][55]. - The anticipated cost of a single treatment using BRTX-100 is expected to be less expensive than common surgical procedures, which can range from $20,000 to $150,000[46]. - The company is focused on developing therapies for disc/spine disease and metabolic disorders using adult stem cells[32]. - The ThermoStem Program is focused on utilizing brown adipose-derived stem cells for metabolic diseases, with initial success in animal models[78][81]. - The company is developing a bioengineered implantable brown adipose tissue construct to target obesity and metabolic disorders using BADSCs[83]. - The next generation BAT is expected to have a higher purity of BADSC and a greater percentage of functional brown adipocytes, enhancing therapeutic effects compared to the first generation product[85]. - The company has developed a promising encapsulation technology for therapeutic delivery, which may prevent immune response and increase safety[85]. Regulatory and Compliance - The FDA issued guidance in July 2020 regarding HCT/P Regulations, with enforcement discretion until May 31, 2021, after which non-compliant products may face immediate action[138]. - If regulated under HCT/P provisions, the company must meet several requirements, including registration, donor eligibility, and adverse event reporting[139]. - Non-compliance with FDA regulations could lead to enforcement actions such as fines, recalls, or criminal prosecution, which could materially affect the company[142]. - If products are regulated as drugs or biologics, significant resources will be required for compliance, and the approval process can take many years[158]. - The FDA may expedite review processes for NDAs and BLAs through programs like Fast Track, Breakthrough Therapy, and Priority Review[161][163]. - The FDA's accelerated approval pathway allows products to be approved based on clinical trials showing effects on surrogate endpoints, which may predict clinical benefits[165]. - The company intends to comply with all applicable foreign governmental requirements for products developed outside the U.S.[143]. - The FDA has broad regulatory authority over drugs and biologics, impacting research, clinical testing, and marketing[144]. Financial Performance and Operations - The company incurred $729,058 and $876,829 in research and development expenses for the years ended December 31, 2021 and 2020, respectively, indicating a decrease of approximately 17%[119]. - The company has historically generated a modest amount of revenue, primarily incurring losses from research and development and marketing expenses[213]. - As of December 31, 2021, the accumulated deficit of the company was $134,146,129[213]. - The company currently has seven full-time employees, indicating a small workforce[191]. - The principal executive offices occupy 6,800 square feet under a lease expiring in December 2024, with annual base rental costs ranging from $153,748 to $173,060[194]. - The company did not receive any proceeds from the issuance of unregistered securities during the three months ended December 31, 2021[203]. Intellectual Property and Competition - The company has filed twelve patent applications related to the Disc/Spine Program and has been issued a patent for a curved needle therapeutic delivery device[111]. - The company has multiple active patent applications in the U.S. related to its Disc/Spine and Metabolic programs, including U.S. Patent No. 11,278,573 B2 for methods to facilitate repair of avascular tissue[114]. - The company has secured registrations for trademarks including BRTX-100 and THERMOSTEM, and has an allowed application for the trademark BRTX[117]. - The company believes that its product BRTX-100 has competitive advantages over Mesoblast's adult stem cell biologic due to the use of autologous cells, which results in a lower risk of rejection and a greater safety profile[130]. - The company faces competition from various pharmaceutical and biotechnology companies, many of which have greater resources[125]. - The company is focused on developing and protecting its proprietary technology through a combination of patents, trade secrets, and other legal protections[118]. Strategic Partnerships and Collaborations - The company is engaged in a Research and Development Agreement with Rohto Pharmaceutical Co., Ltd., which has now expired upon completion of the services[115]. - The company has established a Scientific Advisory Board to provide guidance on scientific matters, including a Disc Advisory Committee focused on the Disc/Spine Program[120]. - The collaboration with the University of Utah has resulted in the acquisition of rights to two provisional patent applications related to human brown fat cell lines[86]. - The company is exploring potential sublicensing of BRTX-100 technology to strategic partners to facilitate FDA approval and commercialization[51][70]. Market Potential - The total annual healthcare costs related to pain in the U.S. are estimated at $600 billion, highlighting the potential market for BRTX-100[77]. - The company intends to market its cell product candidates to healthcare professionals, hospitals, and research institutions upon regulatory approval, targeting physicians skilled in spinal injections[131]. - The company plans to explore opportunities for establishing stem cell therapy clinics internationally as they arise[189].
BioRestorative Therapies(BRTX) - 2021 Q4 - Annual Report