Tmunity boosts pipeline with childhood brain cancer treatment from UCSF

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The T-cell receptor, discovered and engineered by UCSF’s Hideho Okada, M.D., Ph.D., targets a genetic mutation that is found in 70% of children with the rare but fatal brain tumor diffuse intrinsic pontine glioma. (Sean Pavone)

In January, Tmunity Therapeutics laid out a plan to file two new INDs a year—starting in 2019. Now, the biotech is poised to exceed that “aggressive” goal, thanks to a new partnership with the University of California, San Francisco (UCSF). If all goes well, a new T-cell receptor (TCR) treatment for a fatal type of brain cancer could be in the clinic by 2021, said Tmunity CEO Usman “Oz” Azam. 

Under the deal, Tmunity will work with UCSF’s Hideho Okada, M.D., Ph.D., on a treatment for diffuse intrinsic pontine glioma (DIPG), a rare and aggressive type of brain tumor that predominantly affects children. Because of its location toward the midbrain and the pons—part of the brainstem—DIPG is difficult to reach and treat surgically. 

“Neurosurgical interventions are highly risky and don’t often help patients. They tend to be inoperable, and if they are [operable], there’s very limited surgery that can be done,” Azam told FierceBiotech. “To date, no modality has been developed to potentially reduce the size of that tumor, or cure it. … It is highly unusual to survive more than a year after diagnosis.” 

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The TCR, discovered and engineered by Okada, targets a genetic mutation that is found in 70% of DIPG cases. Tmunity envisions inserting this receptor onto a patient’s own T cells before putting them back into the patient to attack the cancer. Specifically, the receptor binds to neoantigen, or a protein, that results from the K27M mutation in a particular histone. 

“In layman’s terms, it is a genetic abnormality detected in 70% of these kids that can be highly targeted. That is very important in cancer biology—if you have a unique genetic mutation, in theory you can drug it,” Azam said. 

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Okada’s lab has tested the treatment in animal models, and Tmunity will take the reins on developing, manufacturing and commercializing it. Born out of an extensive collaboration and licensing agreement with the University of Pennsylvania, Tmunity had been conducting its early phase 1 work and some manufacturing at the university’s facilities. It now has its own GMP manufacturing facility, where it has started growing cells. 

“The hope is that, in the next two years, we can attempt to get this into man. That is our vision. There’s a lot of heavy lifting to do in the next 18 months to get the regulatory framework ready for an IND. Hopefully, by 2021, we will be in the clinic treating patients,” Azam said. 

As for the two INDs that are still to come this year, Tmunity is keeping those programs under wraps, though Azam did say in a previous interview that they are both targeting solid tumors.

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