Allogene Therapeutics Receives FDA Orphan-Drug Designation for ALLO-605, its First TurboCAR™ T Cell Product Candidate, for the Treatment of Multiple Myeloma

 

Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) products for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to ALLO-605, the Company’s next-generation AlloCAR T product candidate targeting BCMA for the treatment of multiple myeloma.

ALLO-605 is the Company’s first TurboCAR™ product candidate. TurboCAR™ is a proprietary, next generation platform technology based on a programmable cytokine signaling, designed to control T cell exhaustion and to improve T cell function and potency. These properties may enable CAR T products to succeed in more difficult to treat hematologic malignancies and solid tumors. The FDA granted Fast Track designation to ALLO-605 in Q2 2021 based on the potential for the product candidate to address an unmet need for patients who have failed other standard multiple myeloma therapies. The Phase 1 study evaluating ALLO-605 is ongoing.

 

“Orphan-drug designation marks an important step towards developing our anti-BCMA portfolio for patients with multiple myeloma and making allogeneic CAR T products readily available for patients,” said Rafael Amado, M.D., Executive Vice President of Research and Development and

 

Chief Medical Officer. “We look forward to providing an update on our BCMA clinical assets by the end of the year with an eye toward prioritizing a strategy for the next stage of development.”

 

Orphan-drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. ODD granted therapies entitle companies to development incentives including tax credits for clinical testing and prescription drug user fee exemptions. If a product that has ODD subsequently receives the first FDA approval for the designated disease, the FDA may not approve any other applications to market the same biologic for the same indication for seven years, except in limited circumstances. ODD does not convey any advantage in, or shorten the duration of, the regulatory review or approval process.

About ALLO-605

ALLO-605, a next-generation AlloCAR T™ known as a TurboCAR™, is an investigational product that targets the B-cell maturation antigen (BCMA) for the treatment of patients with relapsed/refractory multiple myeloma and other BCMA-positive malignancies. This study uses ALLO-647, Allogene's proprietary monoclonal antibody (mAb), as a part of its differentiated lymphodepletion regimen. ALLO-605 incorporates Allogene’s proprietary TurboCAR technology, which allows for cytokine activation signaling to be engineered selectively into CAR T cells. Preclinical results with ALLO-605 were presented at the American Society of Hematology (ASH) annual meeting in December 2020. In June 2021, ALLO-605 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the potential treatment of relapsed/refractory multiple myeloma. The Phase 1 study evaluating ALLO-605 is underway.

About Allogene Therapeutics

Allogene Therapeutics, with headquarters in South San Francisco, is a clinical-stage biotechnology company pioneering the development of allogeneic chimeric antigen receptor T cell (AlloCAR T™) products for cancer. Led by a management team with significant experience in cell therapy, Allogene is developing a pipeline of “off-the-shelf” CAR T cell products with the goal of delivering readily available cell therapy on-demand, more reliably, and at greater scale to more patients.

https://ir.allogene.com/news-releases/news-release-details/allogene-therapeutics-receives-fda-orphan-drug-designation-allo

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