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FDA Pre-IND consultation completed; submission of phase 2 trial of Ranpirnase for anogenital warts on schedule
Licensing partner, Okogen, Inc., presented positive interim phase 2 clinical trial results of OKG-0301 (Ranpirnase) in acute adenoviral conjunctivitis
GERMANTOWN, Md., July 06, 2021 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (“Orgenesis” or the “Company”), a global biotech company working to unlock the full potential of cell and gene therapies, today announces it has achieved multiple Ranpirnase development milestones and provides an update on its resultant plans.
Orgenesis has completed its pre-Investigational New Drug (“IND”) consultation with the U.S. Food and Drug Administration (“FDA”) regarding the development of 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 the FDA pre-IND requests. Two phase 1/2a trials were previously completed for the treatment of AGW caused by human papillomavirus. In both these trials, Ranpirnase 0.1% gel was applied twice daily to the affected areas for up to 8 weeks. The results demonstrated the product’s tolerability and signs of preliminary clinical efficacy.
Ophthalmic development by Okogen
Orgenesis’ licensing partner, Okogen, Inc. (“Okogen”) has recently presented positive interim phase 2 clinical trial results of OKG-0301 (Ranpirnase) in acute adenoviral conjunctivitis (the “RUBY Trial”). The RUBY trial was presented by Okogen at the Association for Research in Vision and Ophthalmology (ARVO) 2021 virtual meeting. It provides encouraging scientific validation for continued development of Ranpirnase in ophthalmic indications. Okogen stated that these results support its plans to progress to a phase 3 trial of OKG-0301 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 indications. Orgenesis is eligible to receive development and commercialization milestones and a single-digit royalty on net sales should Okogen successfully develop Ranpirnase in ophthalmic indications. Orgenesis is also a minority shareholder of Okogen.
SARS-CoV-2 collaboration with Leidos
Orgenesis and Leidos, Inc., as part of the previously announced collaboration, are preparing additional materials leading to an IND submission for a clinical trial of Ranpirnase for the systemic treatment of patients infected by coronavirus 2 (SARS-CoV-2), the virus that causes COVID-19. The companies will carefully evaluate the clinical need for this program prior to commencing this clinical trial, given the dynamic nature of the COVID-19 pandemic. Orgenesis and Leidos are also planning to test Ranpirnase against other potential target infections which are high on the US National Institute of Allergy and Infectious Diseases (NIAID) emerging infectious diseases/pathogens list, including Ebola and eastern equine encephalitis virus.
Feasibility studies to combine Ranpirnase with the Orgenesis Bioxome™ technology have started. Bioxomes are synthetic extracellular vesicles that we believe can provide the same therapeutic benefit of cells without the risks and difficulties of administering entire cells to patients. This reformulation of Ranpirnase may enhance delivery of the antiviral activity of Ranpirnase without an increase in risk to the patient. Orgenesis has also started its pilot program to manufacture and develop a recombinant Ranpirnase. Ranpirnase is currently derived from oocytes of the Northern leopard frog. A recombinant Ranpirnase would improve manufacturing scalability for future potential clinical and commercial needs.
“We congratulate Okogen on the RUBY trial results and their continued progress in the development of Ranpirnase for ophthalmic indications,” said Vered Caplan, CEO of Orgenesis. “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.”
Orgenesis acquired Ranpirnase from Tamir Biotechnology, Inc. (“Tamir”), as well as substantially all of Tamir’s assets, in April 2020. Ranpirnase catalyzes the degradation of RNA, and mediates several essential biological activities, including the regulation of cell proliferation, maturation, differentiation, and cell death. Therefore, it is a potential candidate for the development of therapeutics for life-threatening diseases, including viral and autoimmune diseases that require anti-proliferative and pro-apoptotic properties. Over 1,000 patients have been dosed with Ranpirnase in previous cancer/mesothelioma clinical trials. Ranpirnase has demonstrated a strong safety and tolerability profile. Ranpirnase has also demonstrated preclinical antiviral activity in serious viral diseases with unmet medical needs, such as cytomegalovirus (CMV), influenza, HIV, Ebola, and SARS. This is based on its unique mechanism of action, which was shown to eradicate the virus and modulate the immune system in a series of in vitro studies.
Okogen is a clinical-stage, specialty biotechnology company focused on developing therapeutics to help patients with ocular diseases. The company’s lead development candidate, OKG-0502, is a fixed dose combination of a Ranpirnase (OKG-0301) that functions intracellularly to inhibit viral replication and an ocular decongestant that addresses the associated signs and symptoms of adenoviral conjunctivitis. www.okogen.com
Orgenesis is a global biotech company working to unlock the full potential of cell and gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCare Platform is comprised of three enabling components: a pipeline of licensed POCare Therapeutics that are processed and produced in closed, automated POCare Technology systems across a collaborative POCare Network. Orgenesis identifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The POCare Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. www.orgenesis.com
Notice Regarding Forward-Looking Statements
This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to achieve and maintain overall profitability, our ability to manage our research and development programs that are based on novel technologies, our ability to control key elements relating to the development and commercialization of therapeutic product candidates with third parties, the timing of completion of clinical trials and studies, the availability of additional data, outcomes of clinical trials of our product candidates, the potential uses and benefits of our product candidates, our ability to manage potential disruptions as a result of the coronavirus outbreak, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans differentiation technology as therapeutic treatment for diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to further our CGT development projects, either directly or through our JV partner agreements, and to fulfill our obligations under such agreements, our license agreements with other institutions, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2020, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.
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