The biopharmaceutical company Lipum is developing a biological drug with a novel mechanism of action for the treatment of rheumatoid arthritis and other chronic inflammatory diseases. The company's drug candidate SOL-116 is an antibody that blocks Bile Salt-Stimulated Lipase, an enzyme that has been shown to play a significant role in the inflammatory process. SOL-116 will soon be tested in humans for the first time in a Phase I study in patients with rheumatoid arthritis (RA) and healthy volunteers.
SOL-116 has versatile potential
Lipum has chosen to focus its development work on the treatment of rheumatism in adults and children. However, SOL-116 has potential for the treatment of several other chronic inflammatory diseases and is therefore continuously evaluated for additional indications. One example of this is that Lipum is part of a research consortium with the aim of evaluating new treatments to reduce inflammation in cardiovascular diseases and breast cancer. The project is coordinated by Cardiovascular Research Center (CVRC) vid Örebro University which was recently granted 10 million SEK from The Knowledge Foundation.
TPP – guiding document during the development journey
Something that Lipum is working on extra intensively now that they are about to enter the clinical phase with SOL-116 is updating their Target Product Profile (TPP), which is a valuable planning tool for the development of a drug:
»TPP is something that we are working on and gradually refining during development and as new information from the outside world comes in. Especially intensively now that we are about to enter the clinic. « – Einar Pontén, CEO Lipum
According to FDA (US Food and Drug Administration) The TPP describes the desired profile and characteristics of a product that targets one or more diseases. A TPP is often helpful to the project team and the entire organization as it serves as a goal and provides a structure for planning throughout the drug development process. The most important thing is that the product's benefits (product differentiation) are clearly stated so that the focus is on documenting them.
Central elements of a TPP
The key point in a TPP is to define the patient target group, i.e. the indication, that you plan to treat and what clinical outcomes you plan for – should the new preparation be better or equal to the existing treatment?
Defining the patient target group early on is extremely important – do you plan to treat the entire group or just a subgroup? It is also very important to understand the clinical needs within the indication. How good the new product must be greatly influences how the clinical studies required to get the product approved by the authorities, as well as to meet the requirements that payers have for a new product to be implemented in daily healthcare, should be designed.
The TPP also describes the expected side effect profile, dosage form and dosage. A favorable side effect profile may be the great opportunity in certain indications where currently there are only preparations with many side effects that patients do not tolerate.
Several advantages of a TPP
There are clear advantages to developing a TPP early, at the latest when entering the clinical phase. In addition to the structure of the planning, it also controls which areas to monitor commercially, such as competitors, study design and price levels within different indications. A well-thought-out TPP facilitates internal decision-making, and creates favorable conditions for interactions with external stakeholders such as regulatory authorities (e.g. FDA, EMA) and potential partners and investors. One or more TPPs mean that you are much better prepared to present and discuss your project.
Interview with Sven Undeland
A key person in Lipum's team in developing a TPP for SOL-116 is Sven Undeland. Sven has been active in the pharmaceutical industry for more than 30 years and has previously held leading roles in, among other things: Orexo, AstraZeneca, Sanofi-Aventis and Pharmacia. As a consultant, Sven Undeland offers strategic support to life science companies, including in their product strategy and TPP development, which he tells more about in an interview with BioStock.
First of all, how is a Target Product Profile designed?
– I work with a “one pager”, but it can look different within different companies. But having only a one-pager forces the team to really focus on the central parts where the product can make a difference, improved properties compared to today's treatments.
– You can work with multiple TPPs if you see multiple opportunities (indications, subgroups).
– It is also a strength to have a minimal TPP, i.e. the minimum you must achieve to have a positive business case. If you do not achieve this, you should stop the project or at least rework the TPP and your plans to test whether you have a product.
Why is it important to develop a TPP early in the development of a drug?
– It “forces” the team to obtain information from the outside world within the intended disease to be treated. Doing this “homework” early provides many advantages; more efficient planning, controlling external monitoring, etc. It is also important to update the TPP during development with both external and internal information.
How has the process been in developing a TPP for SOL-116?
– I place a lot of emphasis on obtaining information from outside, internal desktop TPPs have a low probability of becoming a good steering tool for the team. Much is available on the “net” and in databases that we have access to, but it is also very important to do primary research/interviews with various experts and participate in congresses, etc.
If we look specifically at Lipum's candidate SOL-116 – what does that TPP look like?
–We have developed a TPP for the lead indication, RA, and will develop more TPPs for other inflammatory indications.
– In the external analysis we have conducted, including interviews with international opinion leaders, we have gained a clear picture of what clinical needs exist and what characteristics Lipum should focus on documenting.
»If the TPP for RA can be met, SOL-116 is a +1 BUSD product. The other inflammatory diseases we are looking at also have very high potential.«
Finally, what is your view on the potential of Lipum's drug candidate SOL-116?
– The RA indication has a very large market value of approximately 25 BUSD for the 7 major markets. If the TPP for RA can be met, SOL-116 is a +1 BUSD product. The other inflammatory diseases we are looking at also have very high potential. The most important thing is that we do good initial work and can describe the opportunities that SOL-116 has for potential partners and investors.