ASC Therapeutics Receives Key Regulatory Designations in U.S. and Europe to Advance its Second-Generation Gene Therapy for Hemophilia A

MILPITAS, Calif.--(BUSINESS WIRE)-- ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic, metabolic, and other rare diseases has received from the United States (U.S.) Food and Drug Administration (FDA) the Fast Track Designation for ASC618, a second-generation gene therapy of hemophilia A. In addition, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for an Orphan Medical Product Designation of ASC618.

In conjunction with the U.S. FDA IND clearance in 2021 and U.S. FDA Orphan Drug Designation in 2020 for ASC618, these regulatory achievements will significantly facilitate the scientific and clinical development of ASC 618’s gene replacement therapies for hemophilia A, reflecting:

• A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies.
• The viral construct’s critical role with decreasing the therapeutic dose and minimizing cellular stress by increasing synthesis and secretion of factor VIII.

Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, “We are very pleased with the FDA’s and EMA’s regulatory decisions regarding ASC618. This brings us one step closer for providing a truly novel therapeutic approach for hemophilia A, providing potentially a functional cure for patients who currently require life-long care.”

Oscar Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, “These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams. Pre-clinical studies have shown that ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII.”

About ASC618

ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males¹. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant²; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells.

ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration. The study design is available at https://www.clinicaltrials.gov/ct2/show/NCT04676048

About ASC Therapeutics

ASC Therapeutics is a biopharmaceutical company pioneering the development of gene replacement therapies, in-vivo gene editing and allogeneic cell therapies for hematological, metabolic, and other rare diseases. Led by a management team of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on four technology platforms: 1) In-vivo gene therapy of inherited blood clotting disorders, initially focusing on ASC618 for hemophilia A, for which U.S. FDA IND clearance was received; 2) In-vivo gene therapy in metabolic disorders, initially focusing on Maple Syrup Urine Disease, in collaboration with the Universities of Massachusetts and Pennsylvania; 3) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 4) Allogeneic cell therapy, with the first indication with a Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease. To learn more please visit https://www.asctherapeutics.com/.

References

¹ Steven W. Pipe, Gil Gonen-Yaacovi & Oscar G. Segurado. Hemophilia A gene therapy: current and next-generation approaches, Expert Opinion on Biological Therapy. 2022; DOI: 10.1080/14712598.2022.2002842

^{2 }Brown HC, Wright JF, Zhou S, et al. Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev. 2014;1:14036

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