- ALL represents 10% of all leukemia instances in america, progresses quickly, and is usually deadly inside weeks or months if left untreated1
- There’s an pressing must develop new therapies for ALL for sufferers who usually are not candidates for hematopoietic stem cell transplantation (HSCT) or relapse after
- FDA ODD and RPDD designations for UCART22 marks an necessary step in the direction of growing allogeneic CAR T merchandise that will be available for all sufferers
NEW YORK, July 25, 2024 (GLOBE NEWSWIRE) — Cellectis (the “Firm”) (Euronext Development: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology firm utilizing its pioneering gene-editing platform to develop life-saving cell and gene therapies, at present introduced that the U.S. Meals and Drug Administration (FDA) has granted Orphan Drug (ODD) and Uncommon Pediatric Illness Designation (RPDD) Standing to UCART22 product candidate for the remedy of Acute Lymphoblastic Leukemia (ALL).
ALL represents about 10% of all leukemia instances in america, progresses quickly, and is usually deadly inside weeks or months if left untreated. It’s estimated that 6,660 new instances of ALL and 1,560 deaths associated to the illness occurred within the US in 20222.
Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis mentioned: “We’re excited that the FDA granted UCART22 each ODD and RPDD Standing within the remedy of acute lymphoblastic leukemia. This determination represents further proof of the potential of UCART22 to carry a much-needed therapeutic choice to those sufferers with ALL. There’s an pressing must develop new therapies for ALL for sufferers who usually are not candidates for HSCT or relapse after CD19 directed CAR T-cell therapies and/or HSCT.”
UCART22 is an allogeneic CAR T-cell product candidate concentrating on CD22 and evaluated in BALLI-01, a Section 1/2 open-label dose-escalation and dose-expansion examine, designed to judge the protection, enlargement, persistence and medical exercise of UCART22 in sufferers with relapse/refractory ALL.
The final medical knowledge offered by Cellectis on the American Society of Hematology in December 2023 had been encouraging and recommended that UCART22-P2 (totally manufactured at Cellectis) is stronger with a preliminary response price of 67% at Dose Stage 2, in comparison with a 50% response price at Dose Stage 3 with UCART22-P1 (manufactured by an exterior CDMO). Cellectis expects to supply updates on the progress of BALLI-01 by year-end 2024.
The FDA grants ODD standing to medicines meant for the remedy, analysis or prevention of uncommon ailments or problems that have an effect on fewer than 200,000 individuals within the US, an RPDD is granted for severe or life-threatening illness wherein the intense or life-threatening manifestations, corresponding to mortality with relapsed and/or refractory illness, primarily have an effect on people aged from delivery to 18 years. Receiving ODD might assist to expedite and scale back the price of growth, approval, and commercialization of a therapeutic agent. Receiving RPDD might result in receiving a uncommon pediatric illness precedence evaluate voucher on the time of promoting approval.
About Cellectis
Cellectis is a clinical-stage biotechnology firm utilizing its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis makes use of an allogeneic method for CAR-T immunotherapies in oncology, pioneering the idea of off-the-shelf and ready-to-use gene-edited CAR T-cells to deal with most cancers sufferers, and a platform to make therapeutic gene enhancing in hemopoietic stem cells for varied ailments. As a clinical-stage biopharmaceutical firm with over 24 years of expertise and experience in gene enhancing, Cellectis is growing life-changing product candidates using TALEN®, its gene enhancing know-how, and PulseAgile, its pioneering electroporation system to harness the ability of the immune system in an effort to deal with ailments with unmet medical wants. Cellectis’ headquarters are in Paris, France, with areas in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq International Market (ticker: CLLS) and on Euronext Development (ticker: ALCLS).
Ahead-looking Statements
This press launch incorporates “forward-looking” statements inside the which means of relevant securities legal guidelines, together with the Personal Securities Litigation Reform Act of 1995. Ahead-looking statements could also be recognized by phrases corresponding to “potential” “count on,” “would,” “might,” and “counsel,”, or the destructive of those and comparable expressions. These forward-looking statements are based mostly on our administration’s present expectations and assumptions and on data presently accessible to administration. Ahead-looking statements embody statements in regards to the development, timing and progress of medical trials, the timing of our presentation of medical knowledge, and the potential of our candidate merchandise packages, the potential of UCART22 product candidate. These forward-looking statements are made in gentle of data presently accessible to us and are topic to quite a few dangers and uncertainties, together with with respect to the quite a few dangers related to biopharmaceutical product candidate growth, together with the danger of dropping the orphan drug designation whether it is established that the product now not meets the orphan drug standards earlier than market authorization is granted (if any).The precedence evaluate voucher may not be granted on the time of promoting authorization. Moreover, many different necessary components, together with these described in our Annual Report on Kind 20-F and the monetary report (together with the administration report) for the yr ended December 31, 2023 and subsequent filings Cellectis makes with the Securities Alternate Fee on occasion, in addition to different recognized and unknown dangers and uncertainties might adversely have an effect on such forward-looking statements and trigger our precise outcomes, efficiency or achievements to be materially totally different from these expressed or implied by the forward-looking statements. Besides as required by legislation, we assume no obligation to replace these forward-looking statements publicly, or to replace the explanation why precise outcomes might differ materially from these anticipated within the forward-looking statements, even when new data turns into accessible sooner or later.
For additional data on Cellectis, please contact:
Media contacts:
Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14 33, media@cellectis.com
Patricia Sosa Navarro, Chief of Employees to the CEO, +33 (0)7 76 77 46 93
Investor Relation contacts:
Arthur Stril, Interim Chief Monetary Officer, +1 (347) 809 5980, traders@cellectis.com
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1 (Sasaki et al., 2021)
2 (Siegel R.L. et al., 2022)
