Boehringer, Ochre Bio Team in Potential $1B Deal to Develop Regenerative Liver Drugs

Boehringer Ingelheim_iStock, Sundry Photography

Pictured: Boehringer Ingelheim's headquarters in Silicon Valley/iStock, Sundry Photography

Boehringer Ingelheim has struck a deal with Ochre Bio, announcing Monday an agreement potentially worth more than $1 billion to collaborate on treatments for liver diseases such as late-stage metabolic dysfunction-associated steatohepatitis cirrhosis.

Madrigal Pharmaceuticals recently won FDA approval for Rezdiffra (resmetirom) in metabolic dysfunction-associated steatohepatitis (MASH) and rivals are following close behind. However, Rezdiffra and other leading R&D programs target patients before they develop cirrhosis. There remains a need for therapies that can slow or reverse the progression of disease in later-stage patients, either to help them survive until a donor organ is available or eliminate the need for a transplant altogether.

Boehringer has identified Ochre as a partner that can help discover such therapies. The German pharma company has agreed to pay $35 million in upfront and near-term research-based milestone payments to collaborate with Ochre. The near-term fees are part of a broader financial package that could be worth more than $1 billion if the project hits its clinical, regulatory and commercial milestones.

In return for the outlay, Boehringer has secured the chance to work with Ochre to identify and validate regenerative targets for chronic liver diseases. The goal is to boost the liver’s ability to self-repair, which is diminished in patients with late-stage disease, while preventing or reversing disease progression.

Ochre has used funding from investors including Khosla Ventures to build its R&D platform. Working out of sites in the U.K., Taiwan and New York, Ochre is developing RNA therapies to treat liver disease. The biotech’s technology is built on a deep phenotyping approach that uses cellular genomics, tissue imaging and machine learning to reveal interactions between genes and cells. Ochre is applying its technology to liver samples, living micro-liver slices and whole living perfused livers.

Insights generated using the platform will help inform development of RNA therapies. The development side of Ochre’s operation is focused on two challenges: getting RNA to the right cell type and modulating the duration of effect.

GalNAc conjugates, a sugar molecule that can recognize and bind to a cell surface protein which is abundantly expressed on liver cells (hepatocytes), can already get RNA to the liver but they only target one of several cell types in the organ. New approaches are needed to deliver RNA to cells other than hepatocytes. Ochre is conjugating its RNA therapies to different ligands to enable delivery to other types of liver cells, such as Kupffer cells.

At the same time, Ochre is exploring how chemical modifications modulate the duration of effect. The ability to alter the duration of effect sets Ochre apart from rivals that are using CRISPR to treat liver disease by permanently editing genes. CRISPR lacks the flexibility of Ochre’s RNA-based approach but enables one-shot therapies that can free patients from the need for ongoing treatment.

Nick Paul Taylor is a freelance pharmaceutical and biotech writer based in London. He can be reached on LinkedIn.

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