Jnana Raises $107 Million to Advance Lead PKU Program and Signs Multi-Billion Deal with Roche

Rare Daily Staff

Jnana Therapeutics, a company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, closed a $107 million financing and 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.

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

The funds will be used to advance Jnana’s lead program in proof-of-concept clinical trials for the treatment of phenylketonuria (PKU), an inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood, and if left untreated, these toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications.

Jnana’s lead candidate JNT-517 targets the SLC transporter SLC6A19, which is responsible for kidney reabsorption of Phe back into the bloodstream. Inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU. 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. JNT-517 has been granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration.

The first collaboration with Roche, signed in July 2020 and valued at more than $1 billion, was for the discovery of small molecule drugs directed at the solute carrier family of metabolite transporters as a broad, innovative approach for modulating cellular metabolism to treat immune-mediated and neurological diseases.

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

“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,” said Joanne Kotz, co-founder and CEO of Jnana Therapeutics.

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.

“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.”

Photo: Joanne Kotz, co-founder and CEO of Jnana Therapeutics