Agreements

Date: 2014-09-02

Type of information: Licensing agreement

Compound: patent portfolio for the therapeutic use of GABA (gamma-aminobutyric acid)

Company: Diamyd Medical (Sweden) University of California in Los Angeles (UCLA) (USA - CA)

Therapeutic area: Metabolic diseases - Inflammatory diseases - Autoimmune diseases - Rheumatic diseases

Type agreement: licensing

Action mechanism:

Disease: type 1 diabetes, type 2 diabetes, metabolic syndrome, rheumatoid arthritis

Details:

• • On May 14, 2013, Diamyd Medical has announced that the company has concluded a new exclusive licensing agreement with the University of California in Los Angeles (UCLA) relating to a patent portfolio for the therapeutic use of GABA (gamma-aminobutyric acid) in connection with type 1 diabetes and other inflammatory disorders such as type 2 diabetes, metabolic syndrome and rheumatoid arthritis. The agreement covers, for example, one patent application for a combination therapy including GABA and GAD, which is the active substance in the company’s diabetes vaccine, Diamyd®. Professors Tian and Kaufman at UCLA have shown that GABA in combination with GAD acts synergistically in an animal model of type 1 diabetes and that GABA alone can ameliorate type 2 diabetes and rheumatoid arthritis.
• The dominating hypothesis among diabetes researchers today is that the key to curing type 1 diabetes is to simultaneously tackle the disease from several fronts by combining various therapeutics or by providing treatment before onset of the disease. Many, and possibly even the majority, of leading scientists also believe that future treatment of type 1 diabetes must involve an autoantigen, such as GAD, which is the active substance in the company’s diabetes vaccine, Diamyd®. In the continued development of the diabetes vaccine, the company is thus focusing on combining treatment with Diamyd® with other therapeutics and/or treating earlier in the disease process to achieve a strong beta-cell preserving effect.
• Activation of GABA receptors on white blood cells can impede the development of type 1 diabetes, insulin resistance and rheumatoid arthritis in animal models. Combination therapy with GABA and GAD has been shown to act synergistically and, for example, to prolong the survival of transplanted insulin producing beta cells in type 1 diabetes animal models (Tian et al. PLoS One 2011; 6(9):e25337). It is thus relevant to examine whether GABA treatment can boost the beta-cell preserving effect of the company’s GAD-based diabetes vaccine to such an extent that market approval for the diabetes vaccine can be secured. If the combination therapy and the patent application for it are approved, patent protection for the company’s diabetes vaccine Diamyd® would be indirectly obtained until about 2030.
• Diamyd has independently pursued the development of the diabetes vaccine to global Phase III trials and delivered one of Sweden’s largest biotech agreements ever. Two Swedish researcher-initiated Phase II trials of Diamyd® are currently in progress. In February 2013, a clinical study by the name of DIABGAD-1 was launched, in which the diabetes vaccine Diamyd® is being combined with relatively high doses of vitamin D and the anti-inflammatory drug ibuprofen. The purpose of the treatment is to preserve the body’s own ability to produce insulin and control the blood sugar level in children and adolescents newly diagnosed with type 1 diabetes. About a third of the planned 60 participants have already been enrolled in the study.
• A study is also under way to evaluate whether Diamyd® can prevent type 1 diabetes among children at high risk of developing the disease. The study called DiAPREV-IT includes 50 children from the age of four and was fully enrolled in 2012. The first results are expected to be compiled three years after the last participant was enrolled, thus enabling a presentation in 2015.

Financial terms:

Latest news:

• • On September 9, 2016, Diamyd Medical announced that the company expands activities to both type 1 and type 2 diabetes as well as rheumatoid arthritis with proprietary GABA drug product. Results from previously published preclinical studies conducted at University of California in Los Angeles (UCLA) show beneficial therapeutic effects of GABA in the diseases. These results are supported by a number of other publications showing GABA's beneficial effect on the pancreas and its immunomodulatory effect. Since May 2013, when Diamyd Medical signed an exclusive license agreement with UCLA regarding a patent portfolio on the therapeutic use of GABA, the company has further developed the value with activities including, as previously announced, the initiation of a clinical study with GABA and Diamyd® in cooperation with the University of Alabama.
• By producing its own GABA, Diamyd Medical will have full control of product development. The first development stage of a GABA drug product will, with a preferred provider for GMP production and with preclinical studies, determine formulation technology and dosage. At a later stage the production can be scaled up in alignment with the clinical development strategy including formulation, administration and dosage of the drug.
• • On September 2, 2014, Diamyd Medical announced that researchers at UCLA have confirmed earlier findings that combinations of GABA and Antigen Based Therapy (ABT) works synergistically as a treatment in the NOD mouse model of type 1 diabetes. A recent paper (Tian, Dang, Kaufman, et al. Diabetes 2014;63:3128–3134) reports that newly diabetic NOD mice have successfully been treated and some even cured using a combination regimen consisting of GABA and Antigen Based Therapy (ABT). The ABT used in the study was pro-insulin formulated in alum, while similar results were obtained with GAD65 in alum as the ABT in a previous paper using an islet transplantation NOD model (Tian, Dang, Kaufman. PLoS One 2011;6:e25337). GAD65 is the active substance in Diamyd®, the Company’s ABT which is currently being tested in children for the treatment and prevention of type 1 diabetes.The findings provide proof-of-principle that combining ABT with an anti-inflammatory agent, such as GABA, can effectively restore normoglycemia in newly diabetic mice. The therapy is reported to preserve insulin producing cells as well as to promote their replication. It is concluded in the new paper, that the use of GABA in combination with an ABT may hold promise for type 1 diabetes intervention in humans. A group at the University of Alabama, Birmingham, in collaboration with Diamyd Medical, is planning a randomized, double-blind, placebo-controlled clinical study with the combination GABA and Diamyd® as ABT in patients with new onset type 1 diabetes. Discussions with the FDA regarding the study design are ongoing.

Is general: Yes