āshibio Doses First Patient in Phase 1b Trial of Andecaliximab in Patients with Spinal Cord Injury (SCI) at Risk of Heterotopic Ossification (HO)

- Company expands lead program into non-hereditary heterotopic ossification (NHHO), a condition with no approved treatments -

- Initial NHHO study targets patients with traumatic SCI who have high rates of HO, which causes abnormal bone growth in soft tissues -

BRISBANE, Calif.--(BUSINESS WIRE)--āshibio, a privately held, clinical-stage biotechnology company developing novel therapeutics for the treatment of severe bone and connective tissue disorders, today announced that the first participant has been dosed in its Phase 1b clinical trial of andecaliximab in patients with spinal cord injury (SCI). The study, called ANDECA-HO, is evaluating patients who are at risk of developing heterotopic ossification (HO), a condition that causes abnormal bone formation in muscles, tendons, ligaments, and other soft tissues.

The trial signals a strategic expansion of the andecaliximab clinical development program into non-hereditary heterotopic ossification (NHHO). NHHO is a severely debilitating condition that can occur in people who have experienced SCI, traumatic brain injury, hip arthroplasty, burns, blast injuries, and other forms of trauma. āshibio is conducting its first NHHO study in patients with SCI and plans to expand to other NHHO conditions in the future. Researchers estimate that clinically significant HO affects approximately 20% of SCI patients in the United States each year, an estimated 15,000 patients per year.

The ANDECA-HO study is āshibio’s second clinical trial for andecaliximab, the company’s lead product candidate for the potential treatment of HO. On January 23, 2025, the company announced the dosing of the first patient in the ANDECAL trial, a Phase 2/3 study of andecaliximab in patients with fibrodysplasia ossificans progressiva (FOP), a genetic disease characterized by severe HO starting in early childhood. āshibio is investigating andecaliximab in patients with FOP and NHHO, due to the common pathology of HO in both conditions.

“Heterotopic ossification can prevent individuals with spinal cord injury from being in a sitting position, fitting into a wheelchair, or transferring from a bed. HO increases the risk of complications such as bed sores and infections, and increases the SCI survivor’s reliance on others for activities of daily living, negatively impacting their quality of life,” said āshibio Chief Medical Officer Deborah Wenkert, MD. “Abnormal bone formation compresses nerves and blood vessels, leading to loss of function and poorer health outcomes. We hope the ANDECA-HO study brings us a step closer to delivering new options to patients living with or at risk of HO.”

Andecaliximab is a humanized antibody that specifically inhibits the matrix metalloproteinase-9 (MMP9) enzyme. Researchers have demonstrated MMP9 expression in human and rodent NHHO lesions, and believe that MMP9 promotes HO in patients with SCI due to its greater expression in areas of inflammation and its role in degrading certain matrix proteins. Previous clinical trials of andecaliximab, involving more than 1,000 patients, have characterized its safety and tolerability profile.

“MMP9 was discovered as a novel target based on clinical observations in a unique patient with FOP and preclinical research demonstrating the critical role of MMP9 in heterotopic ossification,”¹ said Pankaj Bhargava, MD, Chief Executive Officer of āshibio. “Preclinical studies conducted by our team have further demonstrated that MMP9 is essential for the development of NHHO, a condition with no approved therapies that is often cited as an area of high unmet need by physiatrists specializing in spinal cord injury medicine,” added Dr. Bhargava.

“Inhibiting MMP9 in individuals with spinal cord injury may help reduce heterotopic ossification that can severely limit mobility, hinder recovery, and reduce quality of life,” noted ANDECA-HO Principal Investigator Andrew Park, MD, Physician Scientist at Craig Hospital in Englewood, Colo., who dosed the first patient in the SCI study. “We are excited to initiate dosing in this trial, which gives us an important opportunity to assess the safety and activity of andecaliximab in a patient population that has long awaited a safe and effective treatment option.”

The Phase 1b trial is an open-label study assessing the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of andecaliximab in up to 10 adults (aged 18-89 years) with a history of traumatic SCI. This study will support a future randomized trial in patients with HO related to SCI and other conditions.

For additional trial details, visit the study page on clinicaltrials.gov.

About āshibio

āshibio is a privately held, clinical-stage biotechnology company developing a pipeline of novel therapeutics for the treatment of bone and connective tissue disorders. Founded in 2022, āshibio exited stealth mode in June 2024 with $40 million in seed and Series A financing. The company has initiated a Phase 2/3 trial of its lead asset, andecaliximab, in patients with fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder characterized by progressive heterotopic ossification (HO), a pathological condition characterized by abnormal bone formation in muscle and soft tissues. āshibio has also initiated a development program in non-hereditary heterotopic ossification (NHHO), a severely disabling condition for which there are no approved therapies. The first study in the NHHO program is a Phase 1b trial of andecaliximab in patients with spinal cord injury at risk of HO. For more information, visit www.ashibio.com.

References:

1. Lounev V, Groppe JC, Brewer N, Wentworth KL, Smith V, Xu M, Schomburg L, Bhargava P, Al Mukaddam M, Hsiao EC, Shore EM, Pignolo RJ, Kaplan FS. Matrix metalloproteinase-9 deficiency confers resilience in fibrodysplasia ossificans progressiva in a man and mice. Journal of Bone and Mineral Research, 2024 May 2;39(4):382-398