CORRECTING and REPLACING 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

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 CoQ₁₀ 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.

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