FDA Grants Orphan Drug and Rare Pediatric Disease Designation Status to Cellectis’ UCART22 product candidate for Acute Lymphoblastic Leukemia (ALL) Treatment
Cellectis (NASDAQ: CLLS) announced that the FDA has granted Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) Status to its UCART22 product candidate for treating Acute Lymphoblastic Leukemia (ALL). ALL represents 10% of leukemia cases in the US, progressing rapidly and often fatal if untreated. The designations highlight UCART22's potential to address urgent needs in ALL treatment, especially for patients ineligible for stem cell transplantation or relapsing after other therapies.
UCART22, an allogeneic CAR T-cell therapy, is being evaluated in the BALLI-01 Phase 1/2 study. Recent data showed a 67% preliminary response rate at Dose Level 2 for UCART22-P2, manufactured by Cellectis. The company expects to provide updates on BALLI-01 by year-end 2024. These FDA designations may expedite development and reduce costs for UCART22.
Cellectis (NASDAQ: CLLS) ha annunciato che la FDA ha concesso lo Stato di Farmaco Orfano (ODD) e la Designazione di Malattia Pediatrica Rara (RPDD) al suo candidato terapeutico UCART22 per il trattamento della Leucemia Linfoide Acuta (ALL). L'ALL rappresenta il 10% dei casi di leucemia negli Stati Uniti, progredendo rapidamente e risultando spesso fatale se non trattata. Le designazioni evidenziano il potenziale di UCART22 nel rispondere a esigenze urgenti nel trattamento dell'ALL, specialmente per i pazienti non idonei al trapianto di cellule staminali o che ricadono dopo altre terapie.
UCART22, una terapia con cellule T CAR allogeniche, è attualmente valutata nello studio di fase 1/2 BALLI-01. Dati recenti hanno mostrato un tasso di risposta preliminare del 67% al Livello di Dose 2 per UCART22-P2, prodotto da Cellectis. L'azienda prevede di fornire aggiornamenti sul BALLI-01 entro la fine del 2024. Queste designazioni da parte della FDA potrebbero accelerare lo sviluppo e ridurre i costi per UCART22.
Cellectis (NASDAQ: CLLS) anunció que la FDA ha otorgado el Estatuto de Medicamento Huérfano (ODD) y la Designación de Enfermedad Pediátrica Rara (RPDD) a su candidato de producto UCART22 para el tratamiento de la Leucemia Linfoblástica Aguda (ALL). La ALL representa el 10% de los casos de leucemia en los Estados Unidos, progresando rápidamente y siendo a menudo fatal si no se trata. Las designaciones destacan el potencial de UCART22 para abordar necesidades urgentes en el tratamiento de la ALL, especialmente para los pacientes inelegibles para el trasplante de células madre o que recaen después de otras terapias.
UCART22, una terapia con células T CAR alogénicas, está siendo evaluada en el estudio de fase 1/2 BALLI-01. Los datos recientes mostraron un tasa de respuesta preliminar del 67% en el Nivel de Dosis 2 para UCART22-P2, fabricado por Cellectis. La compañía espera proporcionar actualizaciones sobre BALLI-01 para finales de 2024. Estas designaciones de la FDA pueden acelerar el desarrollo y reducir costos para UCART22.
Cellectis(NASDAQ: CLLS)는 FDA가 희귀약품지정(ODD) 및 희귀소아질병지정(RPDD)를 UCART22 제품 후보에 부여했다고 발표했습니다. 이는 급성림프모구백혈병(ALL) 치료를 위한 것입니다. ALL은 미국에서 백혈병 환자의 10%를 차지하며, 치료하지 않을 경우 빠르게 진행되어 종종 치명적입니다. 이 지정들은 UCART22가 ALL 치료의 긴급한 필요에 대응할 수 있는 잠재력을 강조하며, 특히 줄기세포 이식을 받을 수 없는 환자나 다른 치료 후 재발한 환자를위한 것입니다.
UCART22는 알로겐 CAR T-세포 요법으로 현재 BALLI-01 1/2상 연구에서 평가되고 있습니다. 최근 데이터에 따르면 UCART22-P2에 대한 2단계 용량에서 67%의 초기 반응률이 나타났습니다. 이 제품은 Cellectis에서 제조했습니다. 이 회사는 2024년 말까지 BALLI-01에 대한 업데이트를 제공할 것으로 예상합니다. 이러한 FDA의 지정은 UCART22의 개발을 가속화하고 비용을 절감할 수 있습니다.
Cellectis (NASDAQ: CLLS) a annoncé que la FDA a accordé le Statut de Médicament Orphelin (ODD) et la Désignation de Maladie Pédiatrique Rare (RPDD) à son candidat produit UCART22 pour le traitement de la Leucémie Lymphoblastique Aiguë (ALL). L'ALL représente 10 % des cas de leucémie aux États-Unis, évoluant rapidement et étant souvent mortelle si elle n'est pas traitée. Ces désignations soulignent le potentiel d'UCART22 pour répondre aux besoins urgents en matière de traitement de l'ALL, notamment pour les patients non éligibles à la transplantation de cellules souches ou en rechute après d'autres thérapies.
UCART22, une thérapie par cellules T CAR allogéniques, est actuellement évaluée dans l'étude BALLI-01 de phase 1/2. Les données récentes ont montré un taux de réponse préliminaire de 67 % au niveau de dose 2 pour UCART22-P2, fabriqué par Cellectis. La société prévoit de fournir des mises à jour sur BALLI-01 d'ici la fin de 2024. Ces désignations de la FDA pourraient accélérer le développement et réduire les coûts pour UCART22.
Cellectis (NASDAQ: CLLS) gab bekannt, dass die FDA dem Produktkandidaten UCART22 den Status als Waisenarzneimittel (ODD) und die Bezeichnung für seltene pädiatrische Erkrankungen (RPDD) für die Behandlung der Akuten lymphoblastischen Leukämie (ALL) zuerkannt hat. ALL macht 10% der Leukämiefälle in den USA aus, schreitet schnell voran und ist im Falle einer Nichtbehandlung oft tödlich. Die Auszeichnungen heben das Potenzial von UCART22 hervor, dringende Bedürfnisse in der ALL-Behandlung zu adressieren, insbesondere für Patienten, die nicht für eine Stammzelltransplantation in Frage kommen oder nach anderen Therapien erneut erkranken.
UCART22, eine allogene CAR-T-Zelltherapie, wird derzeit in der BALLI-01 Phase 1/2 Studie evaluiert. Neueste Daten zeigten eine vorläufige Ansprechrate von 67% auf Dosisstufe 2 für UCART22-P2, das von Cellectis hergestellt wird. Das Unternehmen plant, bis Ende 2024 Updates zur BALLI-01-Studie bereitzustellen. Diese FDA-Auszeichnungen könnten die Entwicklung beschleunigen und die Kosten für UCART22 senken.
- FDA granted Orphan Drug and Rare Pediatric Disease Designation Status to UCART22 for ALL treatment
- UCART22-P2 showed 67% preliminary response rate at Dose Level 2 in BALLI-01 study
- ODD and RPDD may expedite development, reduce costs, and potentially lead to priority review voucher
- ALL represents only 10% of leukemia cases in the United States
- 1,560 deaths related to ALL occurred in the US in 2022
The FDA's granting of Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to Cellectis' UCART22 for Acute Lymphoblastic Leukemia (ALL) treatment is a significant development in the field of cell therapy. These designations highlight the urgent need for innovative treatments in ALL, particularly for patients who aren't candidates for stem cell transplantation or have relapsed after other therapies.
The potential of UCART22, an allogeneic CAR T-cell therapy targeting CD22, is underscored by preliminary data from the BALLI-01 trial. With a
The ODD and RPDD statuses could accelerate UCART22's development and potentially reduce costs, which is critical in bringing novel therapies to market faster. For investors, this could translate to a shorter time-to-market and potentially increased market exclusivity, enhancing the product's commercial viability.
The FDA's dual designation for Cellectis' UCART22 represents a significant milestone that could have substantial financial implications. Orphan Drug Designation offers several benefits, including tax credits for clinical trial costs, waiver of the Prescription Drug User Fee and seven years of market exclusivity upon approval. These incentives can significantly reduce development costs and enhance the product's profitability potential.
The Rare Pediatric Disease Designation adds another layer of value, as it could lead to a priority review voucher upon approval. These vouchers are highly valuable, often selling for
Investors should note that while these designations are positive signals, they don't guarantee approval or commercial success. The allogeneic CAR-T market is competitive, with several players vying for position. Cellectis' success will depend on UCART22's efficacy, safety profile and ability to differentiate from competitors. The company's financial health and ability to fund ongoing clinical trials will be important factors to monitor.
- ALL represents
10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated1 - There is an urgent need to develop new therapies for ALL for patients who are not candidates for hematopoietic stem cell transplantation (HSCT) or relapse after
- FDA ODD and RPDD designations for UCART22 marks an important step towards developing allogeneic CAR T products that would be readily available for all patients
NEW YORK, July 25, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug (ODD) and Rare Pediatric Disease Designation (RPDD) Status to UCART22 product candidate for the treatment of Acute Lymphoblastic Leukemia (ALL).
ALL represents about
Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis said: “We are excited that the FDA granted UCART22 both ODD and RPDD Status in the treatment of acute lymphoblastic leukemia. This decision represents additional evidence of the potential of UCART22 to bring a much-needed therapeutic option to these patients with ALL. There is an urgent need to develop new therapies for ALL for patients who 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 targeting CD22 and evaluated in BALLI-01, a Phase 1/2 open-label dose-escalation and dose-expansion study, designed to evaluate the safety, expansion, persistence and clinical activity of UCART22 in patients with relapse/refractory ALL.
The last clinical data presented by Cellectis at the American Society of Hematology in December 2023 were encouraging and suggested that UCART22-P2 (fully manufactured at Cellectis) is more potent with a preliminary response rate of
The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US, an RPDD is granted for serious or life-threatening disease in which the serious or life-threatening manifestations, such as mortality with relapsed and/or refractory disease, primarily affect individuals aged from birth to 18 years. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent. Receiving RPDD may lead to receiving a rare pediatric disease priority review voucher at the time of marketing approval.
About Cellectis
Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 24 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN®, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis’ headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).
Forward-looking Statements
This press release contains “forward-looking” statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as “potential” “expect,” “would,” “may,” and “suggest,”, or the negative of these and similar expressions. These forward-looking statements are based on our management’s current expectations and assumptions and on information currently available to management. Forward-looking statements include statements about the advancement, timing and progress of clinical trials, the timing of our presentation of clinical data, and the potential of our candidate products programs, the potential of UCART22 product candidate. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development, including the risk of losing the orphan drug designation if it is established that the product no longer meets the orphan drug criteria before market authorization is granted (if any).The priority review voucher may also not be granted at the time of marketing authorization. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2023 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
For further information 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 Staff to the CEO, +33 (0)7 76 77 46 93
Investor Relation contacts:
Arthur Stril, Interim Chief Financial Officer, +1 (347) 809 5980, investors@cellectis.com
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1 (Sasaki et al., 2021)
2 (Siegel R.L. et al., 2022)
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FAQ
What designations did the FDA grant to Cellectis' UCART22 for ALL treatment?
What is the preliminary response rate of UCART22-P2 in the BALLI-01 study?
When does Cellectis (CLLS) expect to provide updates on the BALLI-01 study?
What percentage of leukemia cases in the US does Acute Lymphoblastic Leukemia (ALL) represent?
