Harbour Offshoot Signs $395M Deal for Preclinical Crenessity Rival

Harbour BioMed has entered into a significant agreement concerning its preclinical therapy, HAT001, a treatment option for congenital adrenal hyperplasia (CAH), valued at $395 million. The deal involves Harbour BioMed’s antibody subsidiary, HBM Alpha Therapeutics (HBMAT), and an undisclosed business partner, granting the latter exclusive global rights to develop and commercialize the therapy outside of China.

HBMAT, a joint venture formed in 2019 with Harbour BioMed and Boston Children's Hospital, aims to leverage Harbour’s antibody expertise and the hospital's research in endocrine disorders. The therapy, known as HAT001 at HBMAT and HBM9013 at Harbour, is an anti-corticotropin-releasing hormone (CRH) neutralizing antibody, designed to aid in the treatment of CAH. CAH encompasses a group of genetic disorders impacting the synthesis of adrenal hormones due to enzyme deficiencies.

The importance of HAT001’s development is underscored by recent advancements in the CAH treatment landscape. In December, Neurocrine Biosciences received FDA approval for Crenessity, an oral medication for CAH that reduces excess adrenocorticotropic hormone, thereby easing the need for high doses of glucocorticoid therapy. Long-term glucocorticoid usage is associated with serious side effects such as weight gain, cardiovascular issues, and bone density loss.

Like Crenessity, HAT001 aims to minimize patients' dependency on glucocorticoids. Harbour BioMed envisions that HAT001 will induce a reversible pharmacologic adrenalectomy, transforming the treatment of CAH into a more manageable condition akin to primary adrenal insufficiency, which requires lower doses of glucocorticoids. Preclinical studies have already demonstrated promising efficacy in reducing CRH-mediated adrenocorticotropic hormone induction, paving the way for upcoming human trials.

In exchange for the therapy's rights outside of the Greater China region, the prospective partner will provide HBMAT with up to $395 million through a mix of upfront payments and future development, regulatory, and commercial milestone payments, along with tiered royalties if the therapy reaches the market. Additionally, HBMAT may receive a minority interest warrant in the partner company.

Jingsong Wang, M.D., Ph.D., CEO of Harbour BioMed, described the transaction as Harbour’s “partial exit” from HBMAT, highlighting the collaborative venture as the first global NewCo they established. While the identity of the business partner remains undisclosed, Wang emphasized the partner’s strong expertise in drug development, which positions them well to advance HAT001 for patient use worldwide.

This agreement represents a crucial step for Harbour BioMed in its strategic advancement of innovative therapies for endocrine disorders, while potentially widening the reach and impact of its antibody technology on a global scale. The collaboration underscores the ongoing evolution of treatment options for rare genetic diseases and highlights the potential for new therapeutic pathways in addressing longstanding medical challenges associated with CAH.