Annette Bakker is frustrated.
As president of the Children’s Tumor Foundation, a nonprofit focused on neurofibromatosis and schwannomatosis, Bakker has been trying to work with pharma companies to release their shelved drug assets for repositioning, which would allow research on halted programs to continue externally.
Yet many of them are convinced they’re already doing it, even when they’re not.
“I have to admit that I have been banging my head a little bit,” she said. “Recently I discovered that it might be because we’re not talking about the same thing.”
Annette Bakker, president, Children’s Tumor Foundation
Permission granted by Annette Bakker
Now, Bakker is working to explain to the industry the important difference between drug repurposing and drug repositioning.
Pharma companies repurpose drugs regularly. These are drugs already approved by the FDA and then used for a new purpose, whether for a new indication or off label.
“Drug repositioning is different,” Bakker said, and it involves taking a second look at experimental drugs that have been shelved for nonscientific reasons, such as not winning the race to market.
One of the best examples of drug repositioning is mirdametinib, one of Pfizer’s shelved assets, Bakker said. By taking another look at the drug at the urging of the Children’s Tumor Foundation, Pfizer eventually spun out another company, SpringWorks Therapeutics, Bakker said. SpringWorks is now developing mirdametinib for neurofibromatosis and other conditions.
Still, drug repositioning is widely misunderstood by the very people she wants to work with, per Bakker.
“What frustrates me is that (the terms) ‘drug repurposing’ and ‘drug repositioning’ have been used interchangeably,” she said, adding that companies might believe they are doing one without understanding the difference.
“There is a complete underappreciation of shelved assets for rare disease — we are looking at drugs that otherwise may not be developed at all.”
Annette Bakker
President, Children’s Tumor Foundation
These assets are often “technically available for out-licensing,” — but in reality, they’re just shelved, she pointed out.
“Some of them get out-licensed and that’s fine. But the majority of them just die … and nothing happens with those drugs,” Bakker said. “There is, in fact, a whole slew of drugs that today are shelved by companies that could actually have value for patients with neurofibromatosis or other rare diseases.”
For instance, in neurofibromatosis, “there is a huge overlap with the cancer pathways.”
“There is a real opportunity here (to) say, these are the molecular targets we are looking for — do you have drugs in your ‘fridge’ that you are not developing and that we could discuss to outsource and develop?” she said.
There are plenty of reasons a company might be reluctant to do this beyond misunderstanding the terminology: a lack of resources and expertise, scattered data or the stigma of “failure” in simply discussing shelved assets.
Solutions might include things like the “nonprofit marketplace” that the Children’s Tumor Foundation has worked on with The Milken Institute and other organizations. Bakker also suggests creating incentives for companies to participate.
“At the moment there is no incentive for a company to release their shelved assets,” Bakker said. “To get mirdametinib out of Pfizer, it took two years and 200 people, and all of them were volunteers.”
Bakker isn’t sure what those incentives might look like, although a company’s environmental, social and governance score could come into play.
“Instead of measuring how many trees companies are planting, (include) how many drugs you have released that you have re-commercialized for a rare disease as one the ESG measures,” she said. “I think we need to get a couple of really creative people here that understand the problem … and think about what that incentive could look like.”
In the meantime, the Children’s Tumor Foundation and other organizations interested in drug repositioning are looking for shelved compounds with a few things in common. They need to be safe and tolerable, with at least phase 1 and preferably phase 2 data; have some intellectual property protection; have been deprioritized for strategic commercial reasons, not scientific ones; and have been discontinued for fewer than five years.
Bakker hopes to open the door for more conversations about this topic.
“There is a complete underappreciation of shelved assets for rare disease — we are looking at drugs that otherwise may not be developed at all,” she said. “What I would like to do is at least get (companies) to the table to talk to me … I just would like to start having conversations, and I feel that for the moment, we rarely have this conversation.”
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