Andecaliximab

– āshibio advances lead program into safety and efficacy trials – – Andecaliximab is the first in a planned pipeline of investigational therapies for patients with bone and connective tissue disorders – – Milestone follows Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) – BRISBANE, CA, USA I January 23, 2025 I āshibio , a privately held, clinical-stage biotechnology company developing novel therapeutics for the treatment of severe bone and connective tissue disorders, today announced the dosing of the first participant in its ANDECAL study, a Phase 2/3 clinical trial evaluating the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of andecaliximab in patients with fibrodysplasia ossificans progressiva (FOP). The dosing milestone for andecaliximab, āshibio’s lead product candidate, marks advancement of the company’s first clinical trial in a set of indications characterized by heterotopic ossification (HO), a pathological condition that causes abnormal bone formation in soft tissues such as muscles, tendons and ligaments. “Heterotopic ossification, or HO, is a devastating condition with significant unmet need. We are initiating development in fibrodysplasia ossificans progressiva, a rare genetic disease characterized by severe and progressive HO leading to profound disability and shortened life span,” said āshibio Chief Executive Officer Pankaj Bhargava, MD. “People living with FOP lack adequate treatment options, and dosing of the first participant in the ANDECAL trial is a significant milestone in our mission to address their needs. Andecaliximab has extensive prior human clinical experience and safety data. Our team is dedicated to advancing this potential first-in-class treatment for HO, starting with FOP and expanding to other forms of non-genetic HO.” Andecaliximab is a humanized antibody that specifically inhibits the matrix metalloproteinase-9 (MMP-9) enzyme, which researchers have identified as a potentially novel therapeutic target for FOP. The discovery that the MMP-9 enzyme could serve as a novel target for FOP was first published in the Journal of Bone and Mineral Research (JBMR) in February 2024. The researchers evaluated a unique patient who harbors the classic mutation of FOP yet has minimal symptoms of the disease. The patient, who is now 36 years old, carries two mutations in the MMP-9 gene, which likely protect him from the devastating heterotopic ossification that most individuals living with FOP face. The effect of blocking MMP-9 was confirmed in mouse models of FOP in which knockout experiments and pharmacological treatments targeting MMP-9 showed substantial reduction of HO. “This unique person revealed something that could significantly change the lives of people with FOP,” said Frederick S. Kaplan, MD, one of the study’s senior authors and the Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania. “We hope that ongoing efforts will validate what we found and provide a path toward quality treatment for individuals with FOP.” Edward Hsiao, MD, PhD, Professor in the Division of Endocrinology and Metabolism and Director of the Metabolic Bone Clinic at the University of California, San Francisco, enrolled the first participant dosed in the ANDECAL study. “We are excited to initiate enrollment in the ANDECAL trial. I am grateful to the UCSF team and my colleague Dr. Kelly Wentworth for all their efforts in making this study available to our patients with FOP,” said Dr. Hsiao. “The advancement of andecaliximab into the clinic is a significant milestone in our mission to improve the lives of those living with FOP and other forms of HO,” said āshibio Chief Medical Officer, Deborah Wenkert, MD. “We are deeply grateful to the participants for taking part in this important study, and to the investigators for their partnership in helping to advance this potential treatment to address the unmet needs of individuals living with FOP.” The U.S. Food and Drug Administration (FDA) has granted andecaliximab a Rare Pediatric Disease Designation (RPDD) for the treatment of FOP. An RPDD designation is granted to potential treatments for serious or life-threatening rare diseases with fewer than 200,000 patients in the U.S., and which primarily affect individuals under 18 years of age. 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 by the team at MPM BioImpact, 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. For more information, visit www.ashibio.com . About the ANDECAL trial The ANDECAL study is a Phase 2/3 trial consisting of two parts. Part 1, now underway at sites in the United States, is a randomized study evaluating the safety, PK, and PD of two different doses of andecaliximab, along with a preliminary efficacy assessment, in individuals living with FOP. Part 1 consists of two cohorts: Cohort 1a is being conducted in individuals with FOP aged 15 years and older who will undergo a NaF PET scan. Cohort 1b is being conducted in individuals with FOP who are aged 12 years and older and experiencing flare-ups. Following completion of Part 1, āshibio plans to initiate Part 2, a double-blind, placebo-controlled registration study, at sites in the U.S. and selected countries globally. SOURCE: āshibio

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 the dosing of the first participant in its ANDECAL study, a Phase 2/3 clinical trial evaluating the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of andecaliximab in patients with fibrodysplasia ossificans progressiva (FOP). The dosing milestone for andecaliximab, āshibio’s lead product candidate, marks advancement of the company’s first clinical trial in a set of indications characterized by heterotopic ossification (HO), a pathological condition that causes abnormal bone formation in soft tissues such as muscles, tendons and ligaments. “Heterotopic ossification, or HO, is a devastating condition with significant unmet need. We are initiating development in fibrodysplasia ossificans progressiva, a rare genetic disease characterized by severe and progressive HO leading to profound disability and shortened life span,” said āshibio Chief Executive Officer Pankaj Bhargava, MD. “People living with FOP lack adequate treatment options, and dosing of the first participant in the ANDECAL trial is a significant milestone in our mission to address their needs. Andecaliximab has extensive prior human clinical experience and safety data. Our team is dedicated to advancing this potential first-in-class treatment for HO, starting with FOP and expanding to other forms of non-genetic HO.” Andecaliximab is a humanized antibody that specifically inhibits the matrix metalloproteinase-9 (MMP-9) enzyme, which researchers have identified as a potentially novel therapeutic target for FOP. The discovery that the MMP-9 enzyme could serve as a novel target for FOP was first published in the Journal of Bone and Mineral Research (JBMR) in February 2024. The researchers evaluated a unique patient who harbors the classic mutation of FOP yet has minimal symptoms of the disease. The patient, who is now 36 years old, carries two mutations in the MMP-9 gene, which likely protect him from the devastating heterotopic ossification that most individuals living with FOP face. The effect of blocking MMP-9 was confirmed in mouse models of FOP in which knockout experiments and pharmacological treatments targeting MMP-9 showed substantial reduction of HO. “This unique person revealed something that could significantly change the lives of people with FOP,” said Frederick S. Kaplan, MD, one of the study’s senior authors and the Isaac and Rose Nassau Professor of Orthopaedic Molecular Medicine at the University of Pennsylvania. “We hope that ongoing efforts will validate what we found and provide a path toward quality treatment for individuals with FOP.” Edward Hsiao, MD, PhD, Professor in the Division of Endocrinology and Metabolism and Director of the Metabolic Bone Clinic at the University of California, San Francisco, enrolled the first participant dosed in the ANDECAL study. “We are excited to initiate enrollment in the ANDECAL trial. I am grateful to the UCSF team and my colleague Dr. Kelly Wentworth for all their efforts in making this study available to our patients with FOP,” said Dr. Hsiao. “The advancement of andecaliximab into the clinic is a significant milestone in our mission to improve the lives of those living with FOP and other forms of HO,” said āshibio Chief Medical Officer, Deborah Wenkert, MD. “We are deeply grateful to the participants for taking part in this important study, and to the investigators for their partnership in helping to advance this potential treatment to address the unmet needs of individuals living with FOP.” The U.S. Food and Drug Administration (FDA) has granted andecaliximab a Rare Pediatric Disease Designation (RPDD) for the treatment of FOP. An RPDD designation is granted to potential treatments for serious or life-threatening rare diseases with fewer than 200,000 patients in the U.S., and which primarily affect individuals under 18 years of age. 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 by the team at MPM BioImpact, 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. For more information, visit www.ashibio.com . About the ANDECAL trial The ANDECAL study is a Phase 2/3 trial consisting of two parts. Part 1, now underway at sites in the United States, is a randomized study evaluating the safety, PK, and PD of two different doses of andecaliximab, along with a preliminary efficacy assessment, in individuals living with FOP. Part 1 consists of two cohorts: Cohort 1a is being conducted in individuals with FOP aged 15 years and older who will undergo a NaF PET scan. Cohort 1b is being conducted in individuals with FOP who are aged 12 years and older and experiencing flare-ups. Following completion of Part 1, āshibio plans to initiate Part 2, a double-blind, placebo-controlled registration study, at sites in the U.S. and selected countries globally.