Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a GI-focused healthcare company, today announced that it is expanding its pipeline by entering into a collaboration and license option agreement with COUR Pharmaceutical Development Company, Inc. (“COUR”), a biotechnology company developing novel immune-modifying nanoparticles to treat autoimmune diseases. This agreement gives Ironwood an option to acquire an exclusive license to develop and commercialize, in the U.S., COUR’s investigational therapy CNP-104 (the “License”), which if successful, could transform the treatment of Primary Biliary Cholangitis – otherwise known as PBC - a rare autoimmune disease targeting the liver that affects an estimated 133,000 people in the U.S. Currently there is no cure, and medical care is focused on disease management. PBC can lead to irreversible damage and scarring of the liver tissue, ultimately requiring liver transplant.

CNP-104 is being developed utilizing COUR’s nanoparticle platform (CNP), a novel, proprietary system, which combines disease-specific pathogenic antigens with state-of-the-art pharmaceutical nanoparticles that mimic normal removal of dead or dying cells from the body. COUR’s platform has shown proof of technology in clinical and preclinical settings, further demonstrating the opportunity for the platform to treat PBC.

"Collaborating with COUR fits squarely within our business development framework and guiding principles; moving our strategy forward through expansion of our pipeline, leveraging our deep relationships with gastroenterologists and advancing innovation via highly differentiated opportunities like CNP-104,” said Tom McCourt, president and chief executive officer of Ironwood. “COUR’s expertise in immune reprogramming and Ironwood’s development and commercial strength and reach in the GI disease area, will help introduce a potentially new therapy to patients in significant need of new options. This option agreement highlights our focus on prudently allocating capital to value enhancing opportunities while maintaining the flexibility to simultaneously execute on our other strategic priorities.”

As previously announced on October 4, 2021, the U.S. Food and Drug Administration (FDA) accepted COUR’s investigational new drug application (IND) for CNP-104. With the FDA’s acceptance of the IND application, COUR expects to initiate a clinical trial for CNP-104 in 2021 to evaluate the safety, tolerability, pharmacodynamics and efficacy of CNP-104 in PBC patients (the “Initial Study”). After reviewing the data from the Initial Study, the Company has the right to exercise the option.

"We are delighted to collaborate with COUR to drive clinical progress behind CNP-104, which we believe has the potential to significantly shift the treatment paradigm in PBC, said Michael Shetzline, M.D., Ph.D., chief medical officer, senior vice president and head of research and drug development at Ironwood. “With our expertise in GI, we are well positioned to further develop a potentially first-to-market treatment that offers patients the opportunity to address the underlying pathology in PBC in a way no current therapies can.”

“We are excited to collaborate with Ironwood given their commercial infrastructure and history of GI innovation,” said COUR CEO John Puisis. “Their success in commercializing and growing LINZESS and their deep relationships within the GI community make them an ideal partner for COUR as it relates to PBC. This agreement will help expand COUR’s autoimmunity portfolio and further enhance COUR’s reach in immune mediated diseases.”

Pursuant to the terms of the agreement, Ironwood will pay COUR approximately $20 million in upfront and near-term payments associated with the Initial Study. After reviewing the data from the Initial Study, if Ironwood exercises its option, COUR will be eligible to receive royalties in the high single digits to low double digits percentage of the aggregated annual net sales in the U.S. of products containing CNP-104, and additional payments of up to $475 million, inclusive of an option exercise payment and commercial milestones over the term of the agreement.