Life-science companies have changed the world with the breakthrough vaccines and therapeutics they created — and continue to create — to combat Covid-19. And thanks to the new technologies they developed during the pandemic, new medicines for other diseases could be just around the corner. But shelved assets — existing compounds gathering dust on companies’ shelves — may represent another round of medical miracles just waiting to be tested in clinical trials.
The development of new treatments inevitably involves ideas that don’t pan out. Though pharmaceutical companies generally patent these ideas, when further research and development into them ceases they become shelved assets that get no more attention.
But “shelved” doesn’t have to mean “permanently abandoned.” In the hands of other researchers with different ideas, they might have the makings of lifesaving therapies.
A handful of biotech companies have already shared their shelved assets. But many large companies hang on to them, even though they have no current intention of developing them further. Their reasons for doing so range from a lack of resources necessary to transfer the asset over to an interested party to a culture that rewards bringing assets in — and not letting them out.
It takes years and billions of dollars to bring a new drug from early lab research through clinical trials to regulatory approval. Many initially promising medications are winnowed out during this process: some turn out to have negative side effects, others don’t work as intended. But drug companies sometimes pull the plug because of unfavorable market dynamics. A recent literature review by Yale researchers found that “strategic business decisions” were the second-most common reason for companies to suspend development of experimental medicines, trailing only “a lack of efficacy” but ranking ahead of “safety problems.” An earlier study found that, between 2013 and 2015, 24% of the drugs that entered clinical trials were stopped for reasons unrelated to safety or efficacy.
In a world of limited resources, it can make good business sense to prioritize one line of research over another in pursuit of the best return on investment. But a trial drug that didn’t work out for one company could still be viable for another. Different focuses, expertise, and balances of risk can all come into play.
Biotech startups will often take chances that big players won’t. Many want a shot at turning shelved assets into big winners as treatments. But to do so, they need access to the patents and other relevant intellectual property.
Large pharmaceutical companies can launch this next wave of innovation by sharing their shelved assets with other companies. This can be a win-win-win situation: If the new team develops a successful therapy, the big fish can share in the profits and thousands of lives are made better or even saved.
Some big firms have begun to understand this potential. Pfizer, with the help of non-profit and private investors, founded SpringWorks Therapeutics, which assesses the potential for shelved assets to develop into new treatments for rare tumors and cancers. This spin-out now has multiple drugs in late-stage trials.
The potential here is global in scope. Japanese behemoth Takeda just licensed one of its previously shelved assets, sapanisertib, to Calithera Biosciences, a small biotech based in San Francisco. Calithera plans to test the drug’s efficacy against lung cancer.
Many researchers are excited about the possibility of combining sapanisertib with brigatinib, one of Takeda’s on-the-market cancer drugs, to treat neurofibromatosis 2, a rare genetic condition that causes tumors to grow along children’s nerves, leading to hearing loss, vision loss, and balance issues. The combined compounds may suppress the proliferation of neurofibromatosis 2 tumor cells.
This effort is personal for me. My organization — the Children’s Tumor Foundation — participates in the Milken Institute’s BRIDGE Initiative, which is aimed at transforming old medicines into new treatments. This approach is particularly important for children with cancer, since many of them, like those with neurofibromatosis 2, don’t have any FDA-approved drugs available to them.
Repurposing shelved drugs that have already been proven safe in humans can save companies millions of dollars and years of research and development, allowing potential treatments to reach kids — and adults — with cancer and other diseases as quickly as possible.
Other companies should join these efforts by licensing shelved assets or spinning out new companies to develop and commercialize them. They have a real opportunity here to capitalize from their unused discoveries — and make a difference in the lives of millions of people along the way.
Annette Bakker is the president of the Children’s Tumor Foundation and a coauthor of the Milken Institute’s report, “Creating a Nonprofit Marketplace for Shelved Drugs: Lessons from a Pilot Project.” The foundation has received funding from Takeda and SpringWorks to support several events.
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