Harbour BioMed Offshoot Secures $395M Deal for Preclinical CAH Therapy

HBM Alpha Therapeutics (HBMAT), a joint venture between Harbour BioMed and Boston Children's Hospital, has inked a lucrative deal worth up to $395 million for the global rights (excluding Greater China) to its preclinical corticotropin-releasing hormone (CRH) therapy. The agreement, announced on February 25, 2025, marks a significant development in the treatment landscape for congenital adrenal hyperplasia (CAH) and other CRH-related disorders.

Deal Details and Therapy Potential

The therapy, known as HAT001 at HBMAT and HBM9013 at Harbour BioMed, is an anti-CRH-neutralizing antibody that has shown promising preclinical results. According to Harbour BioMed's post-market release, HAT001 has demonstrated "strong preclinical efficacy in downregulating CRH-mediated induction of adrenocorticotropic hormone," positioning it as a potential rival to Neurocrine's recently approved Crenessity.

The unnamed "business partner" acquiring the rights will provide HBMAT with up to $395 million through a combination of upfront payments, development and regulatory milestones, and commercial milestone payments. Additionally, HBMAT will be eligible for tiered royalties on potential future sales and has the opportunity to receive a warrant for minority interest in the partner company.

CAH Treatment Landscape and Market Implications

This deal comes on the heels of Neurocrine's FDA approval for Crenessity in December, which marked the first significant advancement in CAH treatment in decades. CAH encompasses various autosomal recessive diseases caused by mutations in genes encoding enzymes essential for adrenal hormone synthesis.

Both Crenessity and HAT001 aim to address a critical need in CAH treatment by potentially reducing patients' reliance on high-dose glucocorticoid replacement therapies, which can lead to serious side effects such as weight gain, heart problems, and bone loss with long-term use.

HBMAT's approach with HAT001 is particularly noteworthy, as it seeks to "effectively cause reversible pharmacologic adrenalectomy, converting CAH patient treatment into that for primary adrenal insufficiency." This could allow for treatment with much lower, physiological doses of glucocorticoids, potentially improving long-term outcomes for CAH patients.

Strategic Moves and Industry Expertise

The deal represents what Harbour BioMed's CEO, Dr. Jingsong Wang, describes as a "partial exit" from HBMAT, which he called "the first global NewCo we incubated." While the identity of the business partner remains undisclosed, Dr. Wang emphasized their "expertise in drug development" as a key factor in the decision, stating that they are "well-positioned to help bring this therapy to patients worldwide."

This strategic move highlights the ongoing competition and innovation in the CAH treatment space, as well as the pharmaceutical industry's continued interest in developing therapies for rare genetic disorders. As HAT001 progresses towards human studies, it will be closely watched by industry observers and patients alike, potentially representing the next significant advancement in CAH treatment following Neurocrine's recent breakthrough.