Jnana Therapeutics raises $107M for PKU program and announces new Roche partnership

PKU phenylketonuria

Jnana Therapeutics has entered into a second collaboration and license agreement with Roche for the discovery of small molecule drugs for the treatment of cancer, immune-mediated and neurological diseases. 

The collaboration covers multiple targets from a diverse range of target classes to address diseases with high unmet need.

The company also announced the close of its $107 million series C financing to progress the company’s lead program in phenylketonuria (PKU).

Roche partnership

“We are excited to partner with Roche, one of the largest healthcare companies and a strong collaborator, for a second time,” said Joanne Kotz, co-founder and CEO of Jnana Therapeutics. 

“Roche has been a valuable strategic partner and we are proud that our success to date has led to the opportunity to broaden our work together in immune-mediated and neurological diseases, as well as to extend our collaboration into cancer.”

Under the terms of the agreement, Jnana will receive an upfront payment of $50 million, significant near-term milestone payments, and additional potential future payments that could exceed $2 billion, as well as tiered royalties. Jnana will conduct discovery and preclinical activities against multiple cancer, immune-mediated and neurological disease targets, and Roche will be responsible for development and commercialization of any resulting products.

Targeting SLC metabolite transporters

“We have made great progress in our existing partnership with Roche, which is focused on targeting SLC metabolite transporters, and have also made exciting progress in our internal pipeline against an array of target classes including transcription factors,” said Joel C. Barrish, co-founder, president and CSO of Jnana Therapeutics. 

“We are delighted to now bring the full power of our RAPID platform to our second partnership with Roche as we advance a diverse set of compelling therapeutic targets that have proven challenging to drug.”

“We have been impressed by Jnana’s team and their RAPID drug discovery platform, as part of our first collaboration that focused on SLC metabolite transporters,” said James Sabry, global head of Roche Pharma Partnering. 

“As fostering truly symbiotic partnerships with our partners where we learn and grow together is a key focus of our overall partnering strategy, we are excited to now build on our successful existing collaboration with Jnana to address new target classes as we expand our efforts to discover new medicines for patients with cancer, immune and neurological diseases.”

About Jnana Therapeutics’ series C financing

Jnana’s series C financing was led by Bain Capital Life Sciences with participation from existing investors including RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures and Pfizer Ventures.

The proceeds from the funding will be used to progress the company’s lead program in phenylketonuria, a rare genetic metabolic disease, through a clinical proof-of-concept (POC) study, and to advance additional wholly owned, potential first-in-class medicines in immune-mediated diseases and cancer. 

Jnana’s RAPID chemoproteomics platform enabled the identification of JNT-517, a small-molecule inhibitor of the phenylalanine transporter SLC6A19 that acts at a novel, cryptic allosteric site, as well as a growing pipeline of programs against challenging-to-drug targets such as transcription factors.

Jnana Therapeutics announces dosing of first volunteer

Jnana Therapeutics also announced the dosing of the first healthy volunteers in a phase 1 study evaluating JNT-517, its clinical candidate for the potential treatment of PKU. After the phase 1a portion in healthy volunteers, JNT-517 will be evaluated in a phase 1b study in individuals with PKU, with the potential to demonstrate clinical POC. In addition, the company announced that JNT-517 has been granted Rare Pediatric Disease Designation by the US Food and Drug Administration.

“With its unique mechanism of action of blocking phenylalanine reabsorption in the kidney, JNT-517 has the potential to be the first therapy for all individuals living with PKU, regardless of genetic background or age,” Barrish said.

“The Rare Pediatric Disease Designation by the FDA underscores the promise of JNT-517 to accelerate innovation for these patients with limited treatment options.”

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