Agnovos Healthcare LLC, a company using regenerative medicine to treat localized effects of bone disease, has received the FDA’s nod for an IDE clinical trial to assess its AGN1 local osteo-enhancement procedure (LOEP) small-volume (SV) kit in patients with vertebral compression fractures (VCFs). The investigational device is intended to treat stable but painful VCFs via a minimally invasive procedure.

The AGN1 LEOP SV kit won breakthrough device designation from the FDA in March of 2020 for the treatment of stable VCFs. The kit, which comes in the form of two single-use trays, contains a calcium-based, resorbable, triphasic implant material that is designed to align the pace of resorption with bone formation, providing treated vertebrae with immediate and durable protection.

The AGN1 implant material is already available in a CE-marked kit to promote new bone in pelvis and other extremities. The CE mark does not cover VCF treatment, so Agnovos plans a second study in Europe to secure that indication in CE-mark jurisdictions.

“We are pleased that FDA has approved the IDE for the STAND study,” said Tanner Howe, Agnovos’ president and CEO. “We believe that the AGN1 LOEP SV kit has the potential to transform the treatment landscape for vertebral compression fractures. It is designed to work differently than traditional vertebral augmentation, because the AGN1 implant material is intended to be resorbed and replaced by new bone.”

STAND clinical trial

The randomized, single-blinded, noninferiority STAND study will compare the treatment of VCFs using AGN1 LOEP SV vs. vertebral augmentation with polymethyl methacrylate (PMMA) bone cement. The study aims to enroll 246 subjects, according to The primary outcome measures are change in VCF-related pain from baseline as measured by a 100 mm Visual Analog Scale, change in function as measured by the Oswestry Disability Index and incidence of device- or procedure-related serious adverse events at 12 months.

New York-based Agnovos plans to launch the study by the fourth quarter of this year, said James Pilachowski, vice president of global marketing, adding that the company is still working with the FDA to define a post-study regulatory pathway.

“Since the proprietary AGN1 implant material included in the kit is resorbable and intended to be replaced by bone, the treatment may help to reduce the risk of adjacent vertebral body fractures described in literature,” Pilachowski told BioWorld. “This study is focused on determining impact of treatment on pain reduction, function and safety.”

Prior studies in cadavers showed that the AGN1 LOEP SV kit enhanced the biomechanical properties of vertebral bodies, the thick oval portions of bone forming the front of vertebrae, without reducing mobility. In a study published in Journal of Orthopaedic Research in 2019 using cadaveric femurs from older women, treatment with the AGN1 implant material increased failure load on average by 20.5%. In a subset of osteoporotic femurs, AGN1 increased failure load by 26% and work to failure by 45%. None of the patients experienced significant stiffness as a result of the treatment.

Large market opportunity

An estimated 1.5 million VCFs occur in the U.S. each year, causing often debilitating pain, reduced mobility and an increased risk of additional fractures. The condition typically occurs when the front part of a vertebra is compressed due to trauma. Treatments include physical therapy and medications to alleviate the pain. Vertebral implants are used to provide stability and reduce pain while the fracture is healing. According to a report by Allied Market Research, the global market for VCF devices is expected to reach about $1.12 billion by 2022.

Founded in 2012 by James Howe, a retired orthopedic surgeon, Agnovos is initially focused on osteoporosis, which increases the risk for bone fractures. The privately held company’s first product, Ossure LOEP for hip, earned a CE mark in 2017. Since then, the OSSURE LOEP kit “has been used as indicated to form bone in more than 100 proximal femurs in a combination of clinical studies and commercial cases,” Pilachowski said.

To date, Agnovos has raised more than $200 million to commercialize its AGN1 platform technology. Pilachowski said the next 24-36 months will be “focused on collecting the clinical evidence needed to bring the CE-marked kit to the U.S. We also expect to continue to gather clinical evidence for current and future indications in spine and other anatomical locations.”