Orgenesis leading the charge at an exciting time for cell 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. is focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform. Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues. 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. In February 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 in a bid to maximize value for shareholders and accelerate the rollout of our other divisions, with a focus on expanding the point-of-care business. The successful sale 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 Orgenesis's evolving point-of-care cell therapy business and develop advanced therapy medicinal products.  How is it doing: Orgenesis has been making good progress in 2021. On July 6, the company announced multiple milestones in the development of Ranpirnase for multiple indications, including anogenital warts, adenoviral conjunctivitis and even coronavirus (COVID-19).  Orgenesis said it has completed its pre-Investigational New Drug (IND) consultation with the US Food and Drug Administration (FDA), discussing RanTop (Ranpirnase topical gel) for the treatment of anogenital warts (AGW). The company is on track to start Phase 2 trials in this indication after completing FDA pre-IND requests.  A pair of Phase 1/2A trials were previously completed for the treatment of AGW caused by human papillomavirus, which demonstrated tolerability and signs of preliminary clinical efficacy, the company added.  Additionally, Orgenesis said its licensing partner, Okogen Inc, had recently presented positive interim Phase 2 clinical trial results of OKG-0301 (Ranpirnase) in acute adenoviral conjunctivitis. The trial, called RUBY, was presented at the Association for Research in Vision and Ophthalmology (ARVO) 2021 virtual meeting. Okogen said that the results support its plans to progress to a Phase 3 trial in combination with an ocular decongestant, having presented its plans to the FDA. Okogen has an exclusive, worldwide license to Ranpirnase for use in ophthalmic (eye) indications, and as part of the deal, Orgenesis is eligible to receive development and commercialization milestones and a single-digit royalty on any future net sales. On the coronavirus front, Orgenesis said it and partner Leidos Inc are preparing additional materials in advance of an IND submission for a clinical trial of Ranpirnase to treat patients infected by SARS-CoV-2, the virus that causes COVID-19.  The companies are also planning to test Ranpirnase against other potential target infections high on the US National Institute of Allergy and Infectious Diseases (NIAID) emerging infectious diseases/pathogens list, including Ebola and eastern equine encephalitis virus. Furthermore, Orgenesis said that feasibility studies to combine Ranpirnase with its own Bioxome technology are underway. Bioxomes are synthetic extracellular vesicles that the company believes can provide the same therapeutic benefit that administering entire cells does, without the associated risks and difficulties.  This reformulation of Ranpirnase could enhance the delivery of the antiviral activity of Ranpirnase without an increase in risk to the patient, and the company has started its pilot program to manufacture and develop a recombinant version of Ranpirnase.  Ranpirnase is currently derived from oocytes of the Northern leopard frog, and a recombinant Ranpirnase would improve manufacturing scalability for future potential clinical and commercial needs, the company said. Orgenesis acquired Ranpirnase in April 2020, when it bought Tamir Biotechnology Inc.  A month earlier, Orgenesis had announced that enrollment had commenced for a clinical trial using the company’s Tissue Genesis Icellator in rotator cuff repair surgeries at the Hospital for Special Surgery (HSS) in New York. Nine of an eventual 56 total patients have been enrolled so far, the company said. The Phase 2 trial, supported by the National Stem Cell Foundation and an Orthopedic Research and Education Foundation grant, will evaluate the safety and efficacy of autologous stromal and vascular fraction cells (SVF) that are injected after surgical rotator cuff repair. The Tissue Genesis Icellator will be utilized for the cell recovery process, the company said.  Orgenesis acquired the Icellator technology in October to supplement its offering of POCare technology systems. It functions as an automated, functionally closed system for the recovery of SVF from autologous adipose (read: fat) tissue. It is designed for use at the point of care, the company said. Also in May, Orgenesis revealed that it was poised to carry out the first-in-human trial of its Tissue Genesis Icellator2 to treat Acute Respiratory Distress Syndrome (ARDS) caused by COVID-19 after receiving the go-ahead from the US Food and Drug Administration (FDA).  The company described being granted the Investigational Device Exemption (IDE) approval for the pilot trial as a 'significant milestone' for the company. The FDA approval covers a study of 21 patients at one clinical site in the US. Among collaboration news, in March, Orgenesis unveiled a tie-up with Rome's Bambino Gesù children's hospital in Rome, Italy to establish a point-of-care cell therapy centre there. Work will focus on supporting clinical trials related to therapies developed by both the hospital and Orgenesis, joint research, development of new approaches in advanced cell and gene therapies, the company said. Following the implementation of the center, Orgenesis aims to then deploy its Orgenesis Mobile Processing Units and Labs (OMPULs) onsite, which serve as multi-purpose, mobile, autonomous good manufacturing practice (GMP) facilities.  In the same month, Orgenesis also revealed a collaboration with MIDA Biotech BV to establish point-of-care centers at hospitals and other medical institutions across western Europe. The comp[any said it and MIDA plan to deploy Orgenesis Mobile Processing Units and Labs (OMPULs) at leading hospitals for the onsite development of promising cell and gene therapies and immunotherapies from MIDA. The OMPULs are multi-purpose, mobile, autonomous good manufacturing practice (GMP) facilities used to develop, optimize, and manufacture cell and gene therapies at the point of care.  And also in March, Orgenesis said it had entered the second phase of its collaboration with Hospital Infantil Universitario Niño Jesús in Madrid, Spain, focused on the hospital’s proprietary solid tumor treatment, Celyvir. The collaboration is focused on an exclusive license agreement to further develop and commercialize Celyvir.  The agreement follows an initial collaboration between the parties announced in May 2020 to establish a point-of-care center leveraging the Orgenesis POCare platform, announced in May 2020.  On the financial front, in May Orgenesis reported a four-fold increase in revenue in its first quarter to end-March, as the company continues its journey to build a long-term profitable business with its Point of Care (POCare) strategy.  Revenue in the three months came in at $9.4 million, up from $1.8 million in the first quarter of 2020.  The POCare Network continues to grow and now includes facilities in various countries across North America, Europe, Asia, and the Middle East. The latest reported revenues reflect just the first phase of the company's joint venture (JV) partnerships. Inflection points: More news on the development of Ranpirnase Clinical trials news for Tissue Genesis Icellator2 Continued growth of POCare Network Further collaboration deals What the boss says: In the company's most recent announcement, Orgenesis CEO Vered Caplan said: “We believe that the outcomes of the FDA pre-IND consultation for anogenital warts and Okogen’s positive Phase 2 data in acute adenoviral conjunctivitis provide validation of Orgenesis’ acquisition of the Tamir Biotechnology assets last year. We believe the Ranpirnase platform presents a significant opportunity for Orgenesis and we look forward to continued progress of our development of the technology in various therapeutic areas.” Contact the author at jon.hopkins@proactiveinvestors.com