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with an immune deficiency with a precise genetic defect that made it impossible for him to have functioning CRAC channels, Velicelebi says. The boy was given a bone marrow transplant years earlier, and was able to develop a functioning immune system. The boy is otherwise healthy, which suggests that a drug developer inhibiting CRAC channels ought to be able to inhibit inflammation without harming other major organs.
That might not sound like much, but it’s an important vote of confidence for a drug with a mechanism that’s never been tested before in people.
“This is a human validation of the target, which is what pharma is looking for,” Velicelebi says.
CalciMedica has developed its lead candidate, and two backups. They’ve all shown an ability to be given orally in rats, to inhibit CRAC channels, to reduce inflammation, and to remain stable in the bloodstream for once-daily dosing. The company has decided to go after psoriasis as its first application partly because it ought to work against overactive T cells that contribute to skin lesions, it’s easy to get a biopsy to show early effectiveness, and because it’s easier to enroll patients in clinical trials, Velicelebi says.
Plenty of other companies are trying to do similar things. Several Big Pharma companies have internal programs to develop drugs against CRAC channels, based on anecdotal feedback Velicelebi says she has gathered. Several others are clearly interested, because they have entered partnership talks with CalciMedica under confidentiality agreements, she says. The only other active competitor among biotech companies is Lexington, MA-based Synta Pharmaceuticals (NASDAQ: SNTA) which is pursuing CRAC targets through a collaboration with Switzerland-based drug giant Roche.
There are a number of other strategies out there for orally-delivered autoimmune treatments. South San Francisco-based Rigel Pharmaceuticals (NASDAQ: RIGL) has one of the best-known programs, in partnership with AstraZeneca, to develop an inhibitor of a target called syk. Pfizer has made news from mid-stage clinical trials with its oral Jak-3 inhibitor. Plymouth, MI-based Lycera, which hasn’t yet entered clinical trials, has attracted $36 million in venture capital for its concept of developing drugs against other targets, ATPase and Th17. Wilmington, DE-based Incyte (NASDAQ: INCY), along with Eli Lilly, is also going after Jak inhibitors.
CalciMedica has a long way to go before it can prove that its drug is safe and effective in people. Ultimately, the clinical trials for the kind of uses it has in mind can only be run by the biggest drug companies with the biggest budgets. If the drug is shown to be safe in early clinical trials, then it will be time to have more talks with the Big Pharma companies who can take it through the long journey of clinical trials needed for FDA approval. After that, it will be time to talk more seriously about how many people might benefit, and to what extent.
“There is room in the market for lots of different mechanisms,” Velicelebi says. “We need to remember that we’re trying to bring forward a new option for millions of people who suffer from autoimmune diseases. The more treatments out there, the better.”