Baudax Bio, Inc. Receives FDA Orphan Drug
Designation for Ti-168 in Hemophilia A with Inhibitors Treatment.
United
States: Baudax
Bio, Inc. a biotechnology company specializing in T cell receptor
("TCR") therapies utilizing human regulatory T cells
("Tregs"), clinical stage Neuromuscular Blocking Agents
("NMBs"), and an associated reversal agent, announced that the U.S.
Food and Drug Administration (FDA) has granted orphan drug designation to its
leading clinical candidate, TI-168. This designation is for the purpose of
treating Hemophilia A with inhibitors. TI-168 represents their next-generation,
FVIII-specific Treg therapy, engineered to provide reliable and effective
treatment for Hemophilia A patients with FVIII inhibitors.
According to the President &
CEO of Baudax Bio, Inc.,
“We are very pleased with the FDA’s decision to grant orphan drug designation
to TI-168, which we believe highlights the urgent need for innovation and new
therapeutic options for Hemophilia A patients. We believe this is an important therapeutic
area, with established preclinical proof of concept in TI-168 through successes
observed in Hemophilia A with inhibitors in animal models. With an
Investigational New Drug (IND) application already FDA-cleared, we believe we
can activate the phase 1/2a clinical trial of TI-168 for treatment of
Hemophilia A with inhibitors with a modest initial budget and advance this
therapy to further clinical investigation in early 2024.
According to TechSci Research, Hemophilia A is caused from
mutations leading to reduced factor VIII (FVIII) production or function,
requiring standard treatment through replacement therapies, providing the
missing clotting factor. Although effective in reducing bleeding events, about
one- third of patients develop inhibitors against the supplied FVIII, impacting
treatment efficacy. Hence, leading to the requirement of alternative and
adequate treatment.
In the fast-evolving realm of
immunotherapy, Regulatory T-cell (Treg) therapies are proving to be a
game-changer. Treg cells (Regulatory T cells) play a pivotal role in
maintaining immune balance, and Treg cell therapies are at the forefront of
medical innovation. Baudax Bio is a biotech company specializing in innovative
products for certain autoimmune conditions, including some orphan drug
conditions, as well as acute care and related settings. The company is focused
on advancing the development of TI-168, a next-generation Treg therapy
specifically targeting Hemophilia A with FVIII inhibitors. This therapy
combines a patented Treg culture method with a TeraImmune-designed
FVIII-specific TCR, demonstrating success in controlling FVIII ADA in a
hemophilic animal model. The lead program, TI-168, has shown promising
pre-clinical data, and the FDA has approved an IND for a Phase 1/2a clinical
trial to treat Hemophilia A with inhibitors.
In the coming years, the
company will advance the development of TeraImmune’s innovative immune-cell
therapies, leveraging a dual Treg manufacturing platform consisting of both
natural regulatory Tregs isolated from patients and induced Tregs converted
from a patient’s T-effector (“Teff”) cells. This in turn will not only
revolutionize the hemophilia treatment but also pave the way for developing
treatments of different immunological disorders using Treg.