AVROBIO, Inc. Announces FDA Clearance of Investigational New Drug Application for AVR-RD-01 Gene Therapy for the Treatment of Fabry Disease
The plato™ platform represents a significant advance towards a commercial-stage gene therapy solution designed to treat thousands of patients
“We are very pleased that FAB-201 remains on track to expand into sites
in the U.S in the second half of 2019,” said
Clinical data presented at WORLDSymposium in
AVR-RD-01 is an ex vivo lentiviral gene therapy candidate being investigated as a single-dose therapy with the potential to provide durable and potentially life-long therapeutic benefit for patients with Fabry disease. AVR-RD-01 is designed to employ a state-of-the-art lentiviral vector system that is an efficient gene transfer technology for the permanent integration of functional copies of the gene into the patient’s own stem cells. In patients with Fabry disease, hematopoietic stem cells are collected from the patient, and then transduced with lentiviral vector carrying a functional version of the GLA gene that encodes active α-galactosidase A (AGA) – the enzyme that is deficient in Fabry disease – to create AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient with the goal of restoring normal GLA gene expression such that functional AGA enzyme is sufficiently produced by the patient’s own body.
FAB-201 is an open-label, multinational study of the efficacy and safety of AVR-RD-01 in approximately 8 to 12 treatment-naïve males, aged 16 years and older, with classic Fabry disease. The subjects will have a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not yet received treatment with enzyme replacement therapy (ERT) within the past ten years and/or chaperone therapy at the time of screening.
The study will expand into the U.S. in the second half of 2019 and includes five study periods: Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up at 48 weeks. Patients will undergo a conditioning regimen administration with busulfan intravenously for four days to achieve myeloablation prior to the transplant. After study completion, consenting subjects will continue periodic safety and efficacy assessments for approximately 14 years, for a total of 15 years post-transplant follow-up.
About the plato™ platform
platform, AVROBIO’s commercial-scale platform for anticipated future
worldwide commercialization and pipeline expansion activities, consists
of a proprietary state-of-the-art four-plasmid vector system, automation
of a closed cell manufacturing process and a conditioning regimen that
utilizes therapeutic drug monitoring (TDM). The plato platform is
designed to enhance the potency, safety, efficacy, and long-term
durability of AVROBIO’s gene therapies, and may additionally provide the
capability to address central nervous system (CNS) manifestations that
accompany many lysosomal storage diseases. The platform is additionally
being incorporated in the planned Phase 1/2 clinical trial of AVR-RD-02
This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy, prospective products and goals, the therapeutic potential of our product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization and pipeline expansion activities, the design, commencement, enrollment and timing of ongoing or planned clinical trials, and the timing, scope and likelihood of regulatory filings and approvals. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.
Any forward-looking statements in this press release are based on
AVROBIO’s current expectations, estimates and projections about our
industry as well as management’s current beliefs and expectations of
future events only as of the date of this release and are subject to a
number of risks and uncertainties that could cause actual results to
differ materially and adversely from those set forth in or implied by
such forward-looking statements. These risks and uncertainties include,
but are not limited to, the risk that any one or more of AVROBIO’s
product candidates will not be successfully developed or commercialized,
the risk of cessation or delay of any ongoing or planned clinical trials
Christopher F. Brinzey
The Yates Network