Medical Need announced today that it has entered into an exclusive supply and distribution agreement with Swiss company Gene Signal, relating to their product GS-101 (aganirsen) eye-drops, under development for the treatment of several ophthalmological conditions. The lead indication is for the treatment of corneal neovascularization, where one phase III trial has been completed, demonstrating good safety and efficacy. A second confirmatory trial is under way.
Under the agreement, Medical Need gets the exclusive rights to GS-101 in Denmark, Finland, Iceland, Norway and Sweden up until regulatory approval, and will be responsible for the distribution, provision and sale of the product in that territory.
“GS-101 is a very exciting product under development for a number of indications with high unmet medical needs”, said Dr. Peder Walberg, CEO of Medical Need. “With its novel mechanism of action, the product provides an exciting new treatment option for patients suffering from abnormal growth of blood vessels in the eye, a common problem within ophthalmology. In addition, offering the possibility to avoid injections into the eye, which is the current standard of care for some of these conditions, will be a great benefit for the patients.”
“We welcome our partnership to make GS-101 available to patients in the Nordic area” said Eric Viaud, CEO of Gene Signal. “ We see this as an important milestone in our efforts to make the fruits of our Intellectual Property (IP) available to patients suffering from ophthalmic ocular disease” he added.
About Corneal Transplantation and Corneal Neovascularisation
Corneal transplantation is performed when a patient’s own cornea is damaged, inflamed or deformed, often resulting in loss of visual acuity and/or pain. During the procedure, all or part of the patient’s cornea is replaced with the cornea from a donor. Corneal transplantation normally has a high success rate. This is attributable to the privileged immune status of the cornea, which reduces rejection episodes common in other organ transplant situations. However, when pathological blood vessels invade the cornea, a condition known as pathologic corneal neovascularisation (CNV), this compromises the privileged immune status so the risk of graft rejection becomes much higher. In such situations, such as transplantation due to infection or trauma, reducing corneal neovascularisation in connection with corneal transplant could reduce the risk of graft rejection. This could in turn save both eye sight and free up grafts for additional patients, which is important due to the global shortage of corneal grafts.
Aganirsen (GS-101), is a novel antisense oligonucleotide, which acts by inhibiting transcription of insulin receptor substrate 1 (IRS-1) which is over-expressed in pathological angiogenesis. IRS-1 in turn stimulates the production of VEGF-1, a well-known promotor of neovascularization. Unlike inhibiting VEGF-1 however, reducing IRS-1 has been demonstrated to target pathological vessels specifically, without inhibiting normal vessel growth. Contrary to other products which need to be injected into the eye, Aganirsen is administered topically by the patients themselves, as an eye-drop. Depending on the formulation, the substance is indicated primarily for the front (cornea) or back of the eye (retina).
GS-101 is initially being developed for the treatment of corneal graft rejection as the first indication. In the I-CAN Phase III trial, the first randomized trial of a topical inhibitor of corneal angiogenesis, aganirsen was shown to be safe and well-tolerated and to significantly reduce the relative area of corneal neovascularisation, reducing the need for corneal transplantation in patients suffering from viral keratitis and central neovascularization. The study results were published in the journal Ophtalmology. A confirmatory trial will be started in 2016. The product is also planned to be investigated in a Phase II trial for the treatment of ischemic central retinal vein occlusion (iCRVO) as well as a Phase II trial in age-related macular degeneration (AMD) and diabetic macular oedema (DME).
The product holds four orphan drug designations in Europe, for the Prevention of Corneal Graft Rejection, treatment of Neovascular Glaucoma, Ischemic Central Retinal Vein Occlusion and Retinopathy of Prematurity.
About antisense oligonucleotides
Antisense oligonucleotides are small complementary nucleotide segments, which bind to and silence mRNA, thereby inhibiting the transcription of a particular protein. Thanks to the small molecular size of the oligonucleotide, they hold many advantages over other biologics which are normally larger proteins or monoclonal antibodies. Antisense oligonucleotides have the ability to readily diffuse across cell membranes, have low immunogenicity and can be produced by simple chemical synthesis, without the need for a complex biological production process.
About Gene Signal
Gene Signal International SA is a Swiss-based biotechnology company pioneering the development of innovative therapies for angiogenesis-based diseases. Its product candidates are a new class of oligonucleotides, proteins and monoclonal antibodies which are derived from genes that are exclusively involved in the angiogenesis process. At least four official candidates are in development for eleven indications in ophthalmology, dermatology, vascular disorders and cancer. The company was founded in 2000, is privately owned, and is led by a team of highly qualified scientific and commercial talents. Its headquarters are in Lausanne (EPFL Swiss Federal Institute of Technology), Switzerland, with research programs based in France (Bioparc Genopole, Evry) and product development in Canada (Montreal). For more information, please visit: www.genesignal.com
About Medical Need
Medical Need Europe AB is a privately held pharmaceutical company headquartered in Sweden, focusing on the registration, distribution, marketing and sale of orphan drugs and niche specialty pharmaceuticals, as well as provision of unlicensed medicines on a named patient basis, for the treatment of rare diseases with a high unmet medical need.,
The company is currently active within several rare and niche disorders, such as; Urea Cycle Disorders, Niemann-Pick type C, defects of bile acid synthesis, Congenital-Sucrase-Isomaltase Deficiency, progressive multiple sclerosis (MS), hematological stem cell transplantation, multiple myeloma, multi-drug resistant tuberculosis, chronic remitting pouchitis and Ménières disease. For more information, please visit: www.medicalneed.com