HMOX1

– 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.

BOSTON--( BUSINESS WIRE )--Please replace the release with the following corrected version due to revisions to the second and fourth bullet points in the body of release. The updated release reads: BPGbio Receives FDA Rare Pediatric Disease Designation for its Investigational Treatment for Epidermolysis Bullosa This marks the company’s second Rare Pediatric Disease Designation for BPM31510 franchise in the last two months BPM31510T targets mitochondrial machinery to promote wound healing in EB patients BPGbio, Inc ., a leading biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis , today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation for BPM31510T, its investigational treatment for epidermolysis bullosa (EB). This designation coincides with EB Awareness Week, observed annually from October 25-31, highlighting the urgent need for innovative EB treatments. EB, a rare genetic connective tissue disorder, affects roughly one in 20,000 children in the United States, leaving them with delicate skin prone to blistering, chronic wounds, scarring, and increased cancer risks. Severe cases of EB can result in life-threatening complications, including an aggressive form of squamous cell carcinoma (SCC). Currently, there is no cure and treatment options are limited. “The EB community is thrilled by this designation, as EB patients and families remain hopeful for more effective treatments,” said Brett Kopelan , Executive Director, DEBRA of America , the largest EB patient advocacy organization in the U.S. “We deeply appreciate BPGbio’s commitment to advancing research and expanding options for the EB community.” The FDA’s Rare Pediatric Disease Designation is intended for companies developing treatments for pediatric diseases that currently lack approved therapies. This designation provides BPGbio eligibility for a priority review voucher upon BPM31510T’s approval for EB. It is the second Rare Pediatric Disease Designation that BPGbio has received for BPM31510, with the initial designation awarded last month for primary CoQ10 deficiency . BPM31510T has also received the FDA’s Orphan Drug Designation. “EB is difficult to manage clinically and on a day-to-day basis due to fragile skin resulting in painful and persistent blistering wounds that can, in some subtypes, progress into squamous cell carcinoma, an aggressive form of skin cancer, which is the leading cause of mortality in the severe form of EB,” said Robert S. Kirsner, M.D., Ph.D. , Chairman and Harvey Blank Professor of Dermatology at the University of Miami Miller School of Medicine, and Vice President of the Wound Healing Society. “In its Phase I clinical trial, BPM31510T was well-tolerated and demonstrated potential efficacy as an easy-to-use, topical therapy for EB. This designation, and the continued clinical development for BPM31510T, offers new hope to patients suffering from EB and their families.” BPM31510T, developed to support wound healing in EB patients, targets cellular and mitochondrial processes that promote wound healing. By influencing key pathways involved in wound healing—including inflammation, cell proliferation, and tissue remodeling—BPM31510T has been shown to support wound care in EB across multiple subtypes. Following successful preclinical and Phase I studies, BPGbio is partnering with DEBRA of America to advance BPM31510T into Phase II/III trials, anticipated to begin in 2025. “Receiving a second Rare Pediatric Disease Designation for our BPM31510 franchise underscores the power of our NAi Interrogative Biology Platform,” said Niven R. Narain , Ph.D., President and CEO, BPGbio. “While conditions like EB, CoQ10 deficiency, and cancer, may seem unrelated, they share mitochondrial dysfunction as a root cause. BPGbio is committed to further developing BPM31510 in the mitochondria medicine space through strategic partnerships to bring the benefits of therapeutics to underserved patients.” BPM31510 is BPGbio’s leading clinical candidate in topical, intravenous, and oral formulations. BPM31510T has completed a phase 1 trial for EB and phase 2 for SCC; BPM31510IV is being studied in ongoing Phase 2B for glioblastoma multiforme (orphan drug) and has completed a Phase 2A for pancreatic cancer (orphan drug); BPM31510IV has recently received FDA Rare Pediatric Disease designation for its potential treatment for primary coenzyme Q10 deficiency . The company is planning a potential pivotal trial of BPM31510IV targeting multiple CoQ10 deficiency mutations at multiple U.S. and EU sites; BPM31510 Oral A Phase 2 trial for Sarcopenia is currently being planned for BPM 31510 Oral. “We are honored to offer hope for children who suffer from debilitating wounds and pain,” said Vijay Modur , M.D., Ph.D., Chief Medical Officer, BPGbio. “Preliminary evidence from Phase 1 clinical trial suggests BPGbio’s BPM31510 appears to be well-tolerated and potentially efficacious in improving wound healing. We’re looking forward to validating these results further through Phase II/III trials.” About BPM31510 BPM31510IV is BPGbio’s lead candidate in late-stage development for aggressive solid tumors such as glioblastoma multiforme (GBM) and pancreatic cancer . Other topical and oral formulations of the investigational agent are also being developed as a potential treatment for several rare diseases. The compound has demonstrated a tolerable safety profile and shown potential clinical benefits across multiple disease indications. Validated by BPGbio’s NAi Interrogative Biology platform , BPM31510 induces a hallmark shift in the tumor microenvironment (TME) by modulating mitochondrial oxidative phosphorylation in aggressive tumors, leading to cancer cell death. In many mitochondrial diseases, restoring CoQ 10 levels can overcome the effect of mutations in genes that lead to mitochondrial dysfunction. BPM31510 has been granted Orphan Drug Designation by the FDA for GBM, pancreatic cancer, and epidermolysis bullosa (EB), as well as Rare Pediatric Disease Designation for primary CoQ10 deficiency and EB. About BPGbio BPGbio is a leading biology-first AI-powered clinical stage biopharma focused on mitochondrial biology and protein homeostasis. The company has a deep pipeline of AI-developed therapeutics spanning oncology, rare disease and neurology, including several in late-stage clinical trials. BPGbio’s novel approach is underpinned by NAi , its proprietary Interrogative Biology Platform, protected by over 400 US and international patents; one of the world’s largest clinically annotated non-governmental biobanks with longitudinal samples; and exclusive access to the most powerful supercomputer in the world. With these tools, BPGbio is redefining how patient biology can be modeled using bespoke Bayesian AI specifically designed for solving large-scale biology challenges. Headquartered in greater Boston, the company is at the forefront of a new era in medicine, combining biology, multi-modal data, and AI to transform the way we understand, diagnose, and treat disease. For more information, visit www.bpgbio.com .