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Novel treatment of inflammatory diseases

Periodic Reporting for period 1 - NOTID (Novel treatment of inflammatory diseases)

Reporting period: 2016-12-01 to 2017-04-30

The global challenge concerns non-functioning drugs and problems followed by that. The innovation address three major problems related to society, economy and industrialization. For society as well as individuals there is health problem that we deal with. Our initial survey has revealed that for the treatment of RA there is an immediate need for drugs that act according to new and novel mechanisms. This is because today’s most efficient treatment by the so called TNFα inhibitors also show a large ratio of non-responders. In addition, these drugs suppress the immune system which means that combinations can not be used without a risk for adverse effects. Our innovation will act on a new target and according to a novel mechanism and has potential as a stand-alone or complementary drug for inflammatory diseases. The economic problem that we deal with is the fight against increasing costs for drugs. If drugs are more efficient, less drugs will be used, which should decrease the costs for RA drugs. Furthermore, if the right kind of efficient drug is used as early as possible for a patient the costs of treatment and cost for society will decrease. A major problem in commercializing our innovation is to be able to finance the further steps of development and to find the right partners for that process.

The overall objective is to develop a new biological candidate drug for safe and effective treatment of patients suffering from chronic inflammatory diseases, e.g. Rheumatoid Arthritis (RA). The approach is a therapeutic antibody that by a novel mechanism of action blocks the function of bile salt-stimulated lipase (BSSL), and thereby inhibits the inflammatory process and prevents joint destruction. The objective of the proposed innovation project as a whole (SME-instrument phase 1-3) is to plan, develop, secure and bring our innovation towards commercialization on the global market and to get to a Clinical Phase I proof of concept. In measurable terms we aim to document and reach the following results:

Functional effects
• A biological drug that passed toxicology and safety studies ready for Phase I clinical trials.
• A cell line for production of the biological drug.

Medical effects
• Provide a truely novel and complementary treatment to anti-TNFα and other biologicals.
• Offer a new mechanism that does not suppress the patients immune system.
• Inhibit and reverse the progress of the disease to minimise disability.

Economic effects
• Improve RA patients ability to work and reduce sick-leave.
• Reduce patients need for treatment at hospital.
The feasibility study comprised 5 tasks.
Task 1 concerned the elaboration of a Drug Development Plan. This task included the evaluation of partners for production of a pre-candidate drug. The task also prepared for the design of the drug development. Task 2 elaborated a financial plan and included an investigation of different business models for financing of the exploitation phase. Task 3 elaborated a strategy for intellectual property rights.
The task also handled branding issues and the future communication strategy. Task 4 focused on a strategic evaluation of an orphan drug designation. Finally we put together the results from the first four tasks and produced an updated business innovation plan.

Results achieved include the elaboration of a Drug Development Plan (task 1). We have investigated and met potential partners for production, but we have not yet decided who the partner will be. We have several good candidates. We have elaborated a financial plan (task 2) which described the financial needs over a period of four years. In the current analysis of funding needs the total sum is €13 million to reach clinical proof of concept i.e. it has been tested in humans. We have filed for a divisional patent for Inflammatory Bowel Disease as a result of Task 3. We have increased our knowledge about orphan drug designation through dialogue and we have established a clinical plan for Juvenile Idiopathic Arthritis (JIA).
The expected potential impact includes the potential of generating of 20 new jobs. Employment creation will follow the development of sales and will consist of sales staff, administration and R&D staff. Realistically 20 jobs will be created within LipUm the first three years of commercial operation. Most probably we will need a partner company for marketing and sales and depending on the markets to be launchned a realistic forecast for job creation in the partner company would be 50 employees for the European market and 100 employees for the US market.

The wider societal implication is that more patients will be treated. Costs for treatment will decrease and unnecessary treatment can be avoided. Human suffering will decrease.
BBSL molecule - the novel target