Orgenesis leads the charge at an exciting time for cell and gene therapy

Global biotech unlocking the potential of personalized therapies and closed processing systems helpful in manufacturing Maryland-based firm has a Cell and Gene Therapy (CGT) Biotech platform The platform consists of three core elements: POCare Therapeutics, POCare Technologies, and POCare Network What Orgenesis does: Orgenesis Inc (NASDAQ:ORGS) is a global biotech group focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform. The Germantown, Maryland-based company’s aim is to provide life-changing treatments at the point-of-care to patients at low cost. It's Cell and Gene Therapy Biotech platform has three key elements. The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies. Finally, the third component rests on a point-of care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which Orgenesis is able to in-license technologies or advanced therapy medicinal products (ATMPs) and co-develop them with its partners. On February 11, 2020, Orgenesis completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million. The successful sale has spotlighted Orgenesis boss Vered Caplan’s considerable leadership skills. She has since been named one of the top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of the world’s top drugmakers. Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of just $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million. Caplan has indicated that she plans to use the Masthercell sale proceeds to grow the group’s evolving point-of-care cell therapy business and develop advanced therapy medicinal products.  How is it doing: In August, Orgenesis posted second quarter results revealing surging revenue from its point-of-care cell and gene therapy (CGT) network. For the period ended June 30, 2020, Orgenesis posted revenue of $1.7 million, up 55%, compared to revenue of $1.1 million for the second quarter of 2019.  The company also maintained a solid balance sheet with over $97.5 million in cash and equivalents at the end of June, reflecting the successful sale of its Masthercell Global contract development manufacturing organization. Orgenesis is using the Masthercell sale proceeds to expand the company’s point-of-care cell therapy business. Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient, per year. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model. The biotech is focused on therapies which span a wide range of treatments, such as cell-based cancer immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration, as well as antiviral therapies. In August, Orgenesis signed an agreement with contract research firm Therapeutics Inc to develop the topical formation of its drug Ranpirnase for the treatment of external genital warts (EGW) caused by human papilloma virus (HPV). The company plans to seek guidance for an early 2021 Phase 2 study from the FDA. The study will build on data from a placebo-controlled phase 1/2 clinical study of 75 subjects with EGW. In that study, topical Ranpirnase applied twice daily for 8 weeks showed a significant reduction in the lesion area compared to placebo and was shown to be safe. Meanwhile, Orgenesis has advanced the development of a cell-based vaccine platform targeting coronavirus (COVID-19) and other viral diseases. It has launched BioShield to set up a first line of defense against COVID-19 through the discovery of neutralizing human antibodies to contain the spread of viral pathogen. Orgenesis has also signed a preliminary agreement with Leidos, a Fortune 500 science company, to develop, and potentially obtain FDA approval of Ranpirnase for the systemic treatment of COVID-19 patients. Ranpirnase has exhibited strong preclinical antiviral activity in viral diseases such as influenza, Ebola, SARS, and cytomegalovirus. As a part of its $12 million cash and stock deal to acquire Tamir Biotechnology, Orgenesis acquired the broad-spectrum Ranpirnase antiviral. Ranpirnase is a member of the superfamily of enzymes that cause the degradation of RNA and mediate biological activities, including cell death. Earlier in April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a new joint venture to produce certain stem cells. The two firms plan to leverage Orgenesis’s autologous CGT Biotech platform to advance clinical trials. Under the deal, RevaTis and Orgenesis will use the former’s patented technique to obtain muscle-derived mesenchymal stem cells (mdMSC) as a source of exosomes and various other cellular products. Orgenesis and RevaTis are hoping to build on RevaTis’s early success in animals to develop therapies and advance human trials using Orgenesis’s expertise and point-of-care platform, which include automated systems, 3D printing and bioreactor technologies. Meanwhile, Orgenesis and ExcellaBio have developed a breakthrough manufacturing process for so-called bioxomes, which are synthetically made exosomes or extracellular vesicles (EVs). The latter are what transfer DNA, RNA, and proteins to other cells, thereby altering the function of targeted cells. Until now, exosome/EV production has been based on conventional, complex and costly methods of ultrafiltration. However, the two companies have demonstrated the scale up of Bioxomes through a proprietary technique, while generating consistent and repeatable results, including uniform particles sizes. Orgenesis added the University of California, Davis to its point-of-care network. The first project with UC Davis Health is on developing and commercializing a lentiviral manufacturing system which will address a significant unmet need in the market for an efficient and scalable manufacturing process. Infection points: Massive upside potential as the sale of Masthercell Global equips Orgenesis with a war chest for acquisitions Acquisition of Tamir Biotechnology and its broad-spectrum antiviral platform ranpirnase, provides Orgegenesis with a significant opportunity Possible gains from leveraging its expertise in the field of cell and gene therapy, including anti-viral technologies relevant to the coronavirus Likely to gain traction from its joint ventures with several leading universities and healthcare institutions in Asia, North America and Europe Expanding cell therapy market pegged at more than $32 billion What the boss says: Orgenesis CEO Vered Caplan noted that the company has “dramatically transformed” since the beginning of 2020 helped by the Masthercell sale. “The sale was an important step in our strategy to leverage our unique capabilities that directly address the key challenges facing the cell and gene therapy industry,” Caplan said in a statement. “Our goal is to transform the delivery of cell and gene therapy through our point-of-care therapeutics, technologies and network, thereby lowering costs and unlocking the power of cell and gene through a more decentralized and integrated approach,” she added. Contact the author Uttara Choudhury at uttara@proactiveinvestors.com Follow her on Twitter: @UttaraProactive