āshibio, a privately held, clinical-stage biotechnology company developing novel therapeutics for bone and connective tissue disorders, announced the presentation of preliminary efficacy data from Part 1 of its ongoing Phase 2/3 ANDECAL study (ANDECAL-1) evaluating andecaliximab for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare genetic disease characterized by progressive heterotopic ossification (HO), or abnormal bone formation in muscle and soft tissues. These data were presented at the 2026 Annual Meeting of the American Society of Bone and Mineral Research (ASBMR) in Boston, Mass.

Preliminary efficacy findings from ANDECAL-1

In a plenary poster presentation at ASBMR,1 Edward Hsiao, MD, PhD, Professor and Director of the UCSF Metabolic Bone Clinic at the University of California, San Francisco, presented baseline characteristics of patients with FOP in ANDECAL-1. The poster presentation included the first disclosure of efficacy results from ANDECAL-1 with preliminary data demonstrating an 86.2% reduction in new flare-up rate (compared to 6 months prior to baseline) with andecaliximab at a dose of 300mg/week.

Of the 12 enrolled participants in ANDECAL-1, the dose-finding and safety portion of the trial, 11 (92%) carried R206H, the most common variant of the FOP-causing ACVR1 gene. All had new or ongoing disease flare-ups (FUs) within 2.5 months prior to baseline; all were ambulatory and needed help with activities of daily living (ADLs). Participants in ANDECAL-1 had a greater number of recent new FUs prior to baseline compared to participants in the FOP Natural History Study (NHS). The mean (standard deviation [SD]) time since onset of the participants’ most recent FU was 0.38 (0.8) years in ANDECAL-1 versus 1.5 (2.6) years in the NHS. Ninety-two percent of ANDECAL-1 participants had at least one new FU in the 12 months before baseline, compared to 66.7% in the NHS; during this time, the mean number of FUs per participant was 3.25 in ANDECAL-1, versus 2.5 in the NHS.

“The baseline characteristics reveal greater FOP disease activity with a larger number of new flare-ups in ANDECAL-1 participants prior to andecaliximab, compared to enrollees in a prior FOP natural history study,” Dr. Hsiao observed. “The findings underscore the complexity of FOP, a disease in which activity can vary widely. The more severe disease activity we have observed in ANDECAL-1 will provide a true test of efficacy for andecaliximab and will be a major consideration as we interpret the full results.”

Advancing andecaliximab as a potential treatment for FOP and NHHO

Andecaliximab is an investigational antibody being developed by āshibio for heterotopic ossification, including FOP and non-hereditary heterotopic ossification (NHHO), a much more prevalent condition affecting approximately 100,000 patients in US, Europe, and Japan. NHHO is a severely disabling condition that can occur following spinal cord injury, hip arthroplasty, and other forms of trauma, and for which there are no approved therapies.

The safety database of andecaliximab includes >1,000 patients treated in prior clinical trials showing andecaliximab to be safe and well tolerated with subcutaneous (SC) or intravenous (IV) administration. A pooled safety analysis of ~350 participants receiving SC andecaliximab was presented at the 2024 Annual Meeting of ASBMR and included an analysis of treatment emergent adverse event (TEAE) during the double-blind treatment period in prior trials.2 Treatment-related TEAEs more common with andecaliximab compared with placebo (difference of ≥1 percentage point) were injection site erythema (2.1% versus 1.1%), headache (1.4% versus 0%), anemia (1.1% versus 0%), and injection site pain (1.1% versus 0%).

“The preliminary data from ANDECAL-1 are encouraging, demonstrating a substantial reduction in the rate of new flare-ups in patients with FOP treated with andecaliximab,” said Pankaj Bhargava, MD, Chief Executive Officer of āshibio. “These data support MMP9 as a novel target in FOP and further increase our conviction that andecaliximab may be uniquely positioned as a potential treatment for FOP and NHHO. The favorable safety profile of andecaliximab is differentiated from currently approved FOP treatments, and we look forward to advancing our clinical development program for all forms of heterotopic ossification.”

About āshibio

āshibio is a privately held, clinical-stage biotechnology company developing a pipeline of novel therapeutics for bone and connective tissue disorders. The company is advancing andecaliximab for the treatment of heterotopic ossification (HO), including fibrodysplasia ossificans progressiva (FOP) and non-hereditary heterotopic ossification (NHHO). āshibio is also developing vantictumab for autosomal dominant osteopetrosis type 2 (ADO2), a genetic disorder characterized by impaired osteoclast function. āshibio intends to advance vantictumab into clinical trials in patients with ADO2 in the first half of 2028. For more information, visit www.ashibio.com.

References

1 Hsiao EC, Mukaddam MA, Pignolo RJ, et al. Baseline characteristics in Part 1 of the ongoing ANDECAL Study, a two-part phase 2/3 study of andecaliximab (anti-matrix metalloproteinase-9 mAb) in participants with fibrodysplasia ossificans progressive (FOP). Presented at American Society for Bone and Mineral Research (ASBMR) Annual Meeting, Boston, Mass., October 9, 2026, Presentation number Fri-001.

2 Wenkert D, Smith V, Bhargava P. Inhibition of MMP9 as a novel treatment strategy for fibrodysplasia ossificans progressiva (FOP): Safety analysis of the anti-MMP9 antibody andecaliximab in development for FOP. Presented at American Society for Bone and Mineral Research (ASBMR) Annual Meeting, Toronto, September 28, 2024, Presentation number Sat-LB 596.

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