Orgenesis Inc (NASDAQ:ORGS) (FRA:45O) has announced multiple milestones in the development of Ranpirnase for multiple indications, including anogenital warts, adenoviral conjunctivitis and even coronavirus (COVID-19).
First, Orgenesis 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 said.
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Additionally, OrGenesis’ licensing partner, Okogen Inc, 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.
“We congratulate Okogen on the RUBY trial results and their continued progress in the development of Ranpirnase for ophthalmic indications,” OrGenesis CEO Vered Caplan said in a statement.
“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.”
On the coronavirus front, Orgenesis and 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.
Further, 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. The drug works by catalyzing the degradation of RNA and mediating the body’s regulation of cell proliferation, mutation, differentiation and death. That versatility makes it 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, the company said.
Contact Andrew Kessel at andrew.kessel@proactiveinvestors.com
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