Several steps closer to the goal
When we presented the new Diamyd Medical in May 2013, we established three key strategic factors to be able to prevent, treat and cure type 1 diabetes and other forms of autoimmune diabetes. These comprised continuing development of our Antigen Based Therapy (ABT), the GAD-based diabetes vaccine Diamyd®, by strengthening the efficacy of the treatment in various ways, building alliances with industrial partners and broadening of the business in the field of diabetes.
Using these key factors as a base, we have worked intensively with a goal-oriented approach to our efforts. We have continued the development of Diamyd® with a focus on combination therapy and we are well underway with the planning and starting of new clinical trials together with teams of researchers around the world. To benefit from increasing interest in the diabetes vaccine generated by our work, we are now focusing our energy on bringing on board industrial partners. Finally, through investments, we have broadened Diamyd Medical’s focus areas and, accordingly, the Company’s base as a diabetes company.
Combination therapy is the key
Over the last years, it has become increasingly apparent that the autoimmune process that causes type 1 diabetes is extremely complex. The consensus among researchers is that the disease must be attacked from several different angles simultaneously through combining several drugs in much the same manner as for many forms of cancer. This is confirmed by the increasing number of research grants available for developing combination therapies and the involvement of global pharmaceutical companies.
Our main approach for identifying an effective treatment is to combine Diamyd® with other agents that can strengthen the efficacy and provide the diabetes vaccine with the right conditions to act. One such study is DIABGAD-1, where Diamyd® is being tested in combination with ibuprofen and vitamin D. The aim is also to stop the autoimmune process early before it leads to fully developed type 1 diabetes. A prevention study with Diamyd®, DiAPREV-IT is ongoing, where the intent is to see if the diabetes vaccine can prevent or delay the development of clinical symptoms of type 1 diabetes in children. All 50 children participating in the study will have been monitored for a five-year period by the end of 2016, when the results of the study will be available.
New clinical trials
Last year, we set a goal of having three new clinical trials with Diamyd® in process within three years – we are now nearing this goal at an increasing pace. We are planning several new investigator-initiated trials together with teams of researchers around the world. Our collaboration with externally funded researchers enables the evaluation of more alternatives than if we were to independently drive and fund all development.
Recently, one such study received approval from the Swedish Medical Products Agency. The DIAGNODE study is the first of its kind, where a low dose of Diamyd® is administered directly into lymph nodes in combination with vitamin D treatment. The concept is comparable to the development in allergy therapy, where the administration of allergen into lymph nodes significantly has improved the efficacy. This is an exciting approach that, in its first phase, will be tested at Linköping University on a small group of adult patients recently diagnosed with type 1 diabetes.
Another example comprises a study with Diamyd® in combination with gamma amino butyric acid (GABA) on children and adolescents with newly diagnosed type 1 diabetes. This is being planned in collaboration with a research group at the University of Alabama in the US. The idea of combining GABA with an ABT for type 1 diabetes gained further support in September from a paper in a scientific journal, where scientists at the University of California in Los Angeles (UCLA) confirms that combination therapy with GABA and ABT acts synergistically when treating non-obese diabetic (NOD) mice, which are used as a model for type 1 diabetes.
Furthermore, the research team conducting the DiAPREV-IT study have well-advanced plans in place for a further, similar, but larger, prevention study in which the diabetes vaccine is also combined with vitamin D.
Vaccine available for studies
A prerequisite for any new studies is that approved study drug is available and new batches of Diamyd® and matching placebo were produced during the fiscal year. We have a well-established manufacturing process in place, which together with a proven favorable safety profile means that Diamyd® is approved for clinical trials with young children.
It is strategically important that we keep the production process updated and ensure that GAD protein and ready-for-use Diamyd® are available. We recently decided to have Protein Sciences manufacture new GAD protein. Manufacture will be paid, in part, with Diamyd shares. Accordingly, Protein Sciences will become a strategic shareholder in Diamyd Medical. Deepening the relationship with Protein Sciences is extremely positive for ensuring manufacturing long-term.
Being able to rapidly initiate new studies, including Phase III, and having an established manufacturing process comprise substantial value and are strong points in discussions with industrial players.
Focus on business development
Antigen Based Therapy is considered a critical component for any successful future combination therapies for type 1 diabetes and, in our assessment, Diamyd® is the ABT at the forefront of development worldwide. This is confirmed by the many inquiries we receive asking to test Diamyd® in combination with other drugs. To benefit from this increasing interest, we are now focusing our energy on identifying industrial partners. As a door opener, we are presenting proposals for specific combination therapies with Diamyd® to selected major pharmaceutical companies that have the rights to any of the other components involved. The goal is to enter into collaboration agreements to secure resources for the development of our drug projects all the way to market approval.
Focus areas broadened
During the fiscal year, we broadened Diamyd Medical’s focus areas through two extremely interesting investments. The first and largest investment was in Cellaviva AB, which is establishing Sweden’s first commercial stem cell bank for private family saving of stem cells in umbilical cord blood and other sources of stem cells. This investment allows Diamyd Medical to enter the field of stem cells and the, for us, extremely interesting field of Personalized Regenerative Medicine (PRM).
The second investment was in Companion Medical, a newly started medical technology company in San Diego, in the US, which develops advanced technical devices for people living with insulin-treated diabetes. In addition, we also gained the distribution rights, in the Nordic region, for Companion’s future products. The investment makes it possible, in the long term, to enter the medical technology diabetes market.
Exciting year ahead
As early as the start of 2015, we plan to be able to present the six-month results from the DIABGAD-1 combination trial. The first results focus on immunological markers that can provide early indication of where the disease is heading.
We are proud to report significant progress over the past year and we will continue in the same spirit. I am convinced that, during the next year, we will reach several key milestones.
Stockholm, November 6, 2014
President and CEO Diamyd Medical AB