Drug Development
Traditional drug discovery Current targeted drug R&D
1. Focused 2. Few indications 3. Serendipitous expansion
Missed indication
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Search space of traditional approaches
Biovista approach
Biovista’s Clinical Outcome Search Space™ (COSS)
1. About 23,000 indications 2. About 6,000 adverse events 3. About 90,000 drugs and compounds 4. About 20,000 human genes
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ples are Celgene’s Thalomid®, which is reposi- tioned thalidomide, and its derivative Revlimid® (lenalidomide). These two repositioned drugs rep- resent a combined global revenue stream of more than $2.8 billion for Celgene7. Although one should not assume that such success for a particu- lar repositioned drug will automatically mean the same success for all repositioned drugs, it becomes very hard to argue against the financial potential of repositioning as a strategy. Potential for market success depends on numerous factors, including market need, competition, differentiation, an excellent product, IP barriers, payer acceptance, compliance and a successful market strategy. These factors apply for repositioned drugs in the same way as they do for NCE/NME drugs as well, and it is thus important to remember that there is no inherent property of repositioned drugs that would limit their market potential.
of their originally-targeted indications, can lever- age their inherently reduced development risk into potentially new indications. They can do so if they can be proven to be effective in the new indications and also sufficiently differentiated against standard of care. When such drugs enter clinical trials, they compete with non-repositioned drugs not in terms of safety, but in terms of efficacy. Since safety accounts for approximately 30% of drug failures in clinical trials, this is a significant development advantage that repositioned drugs enjoy.
2. The money savings advantage. According to a recent report based on a survey of 30 pharmaceu- tical and biotechnology forms, the cost to re- launch a repositioned drug averages $8.4 million, whereas to relaunch a new formulation of an exist- ing drug in its original indication costs an average $41.3 million6. In both cases, the drug has reached the market. The difference between the costs of market attainment for a repositioned drug versus a new drug, however, is simply staggering. Given that the latter averages more than $1.3 billion, suc- cessfully bringing a repositioned drug to market seems to cost approximately 160 million times less than the current standard of NCE/NME develop- ment. Even if this differential is off by a hundred million or more, from the purely financial perspec- tive, repositioning is in a completely different league of investment needed to create a new drug product in the market.
3. The market potential advantage. Not all drugs are blockbusters, but some that are achieved this status as repositioned drugs. Two excellent exam-
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4. The return on investment potential. If it takes an average $8.4 million to launch a successful reposi- tioned drug, and there is no limit to market returns as the Celgene case shows, then all things being equal, the disparity in upfront investment means that repositioned drugs will always represent a bet- ter return on investment than NCE/NME drugs. However, exactly like with NCE/NME drugs, it is very important to keep in mind that this should also be a portfolio strategy: it is prudent to have a reasonable stable of repositioned drugs under development as a portfolio, to allow for attrition due to potential lack of efficacy (but not safety), when any drug is tested in clinical trials.
5. The out-licensing potential. Pharmaceutical companies are said to be exploring new models to out-license some of their clinical drug candidates that may have been shelved for whatever reason, even though they have met their end points and have proven themselves to be safe. If such drugs were to be repositioned, then the pharmaceutical company increases the attraction these drugs have, and gives itself more options to find interested buy- ers. For example, the pharmaceutical company may retain the original use rights to the drug, and out-license the rights to the new indication only. Or, the company may retain the rights to the new indication and out-license the original use if the lat- ter has become a non-strategic one, whereas now the new use falls within the company’s areas of interest. With either scenario, repositioning grants a pharmaceutical company specific and novel busi- ness development possibilities for out-licensing that it otherwise would not have.
Drug Discovery World Spring 2011
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