Cellectis Receives Orphan Drug Designation for UCART22, its Allogeneic CAR T Product for Patients with Acute Lymphoblastic Leukemia

Rhea-AI Summary

Cellectis announced that the European Commission granted Orphan Drug Designation (ODD) to UCART22, its allogeneic CAR T product for treating Acute Lymphoblastic Leukemia (ALL).

UCART22 targets CD22 and is being evaluated in the Phase 1/2 BALLI-01 study, focusing on patients with relapsed/refractory ALL.

ALL accounts for 12% of all leukemia cases and progresses rapidly. The 10-year prevalence in the EU is estimated at 1.9 per 100,000 persons.

The ODD was based on preliminary clinical data showing UCART22's significant benefit in heavily pretreated patients. In December 2023, Cellectis reported encouraging data with a 67% response rate at Dose Level 2 for UCART22-P2.

ODD provides regulatory, financial, and commercial incentives.

Positive

• European Commission granted Orphan Drug Designation (ODD) to UCART22.
• Positive preliminary clinical data shows significant benefit for heavily pretreated patients.
• Encouraging response rates: 67% at Dose Level 2 for UCART22-P2.
• ODD provides regulatory, financial, and commercial incentives.
• Cellectis's in-house manufacturing shows higher potency for UCART22-P2.

Negative

• ALL progresses rapidly and is typically fatal within weeks or months if untreated.
• Patients with relapsed/refractory ALL have treatment options.
• Preliminary data, not yet definitive for long-term success.

Insights

Medical Research Analyst

The Orphan Drug Designation (ODD) granted to Cellectis’ UCART22 for treating Acute Lymphoblastic Leukemia (ALL) is a significant milestone in the biotechnology and pharmaceutical sectors. The ODD provides several benefits including market exclusivity for 10 years in the European Union (EU), which can be a considerable advantage for Cellectis. This exclusivity reduces competition and potentially increases profitability for UCART22, especially since ALL has limited effective treatment options.

ALL, representing 12% of all leukemia cases, is an aggressive and fatal disease if untreated. The preliminary data showing a 67% response rate for the UCART22-P2 variant is particularly encouraging as it indicates efficacy even in heavily pretreated, relapsed/refractory patients. This suggests a compelling therapeutic benefit, especially for patients who have not responded to other treatments like CD19-directed CAR T-cell therapies. The ODD will also facilitate faster regulatory approvals and reduce certain development costs due to various incentives provided by the European Medicines Agency (EMA).

In summary, the designation underscores the potential of UCART22 in addressing a critical unmet medical need and positions Cellectis favorably in the CAR T-cell therapy market.

Financial Analyst

The Orphan Drug Designation for UCART22 is a positive development for Cellectis from a financial perspective. This designation not only provides market exclusivity but also can lead to millions in potential revenue, given the lack of current effective treatment options for relapsed/refractory ALL. The exclusivity period will shield UCART22 from generic competition, allowing Cellectis to capitalize on pricing power.

Furthermore, the designation enables Cellectis to benefit from various incentives, including tax credits, reduced regulatory fees and grants for clinical trials. These financial incentives will likely reduce the overall cost of development, improving the company's return on investment for UCART22.

Investors should also note that positive preliminary clinical data, such as the 67% response rate at Dose Level 2, may enhance investor confidence and drive up the stock price. This is particularly relevant in light of the product being fully manufactured in-house, which could streamline production processes and reduce costs.

Overall, the ODD can significantly impact Cellectis’ financial health positively, making it an attractive opportunity for investors focused on biotech stocks.

Market Research Analyst

The granting of the Orphan Drug Designation to UCART22 places Cellectis in a unique position within the competitive landscape of CAR T-cell therapies. With limited treatment options available for relapsed/refractory ALL, UCART22 fills a critical gap. The CAR T-cell market is rapidly evolving and securing this designation gives Cellectis a competitive edge, particularly with the EU's regulatory and market exclusivity advantages.

UCART22’s promising clinical outcomes, specifically the higher response rate of 67% at Dose Level 2 compared to conventional treatments, highlight its potential market demand. The ability to manufacture in-house further differentiates Cellectis from competitors and can lead to improved margins and control over quality and supply chain.

For retail investors, understanding the implications of market exclusivity and potential revenue streams from this designation can offer insights into the company’s future growth prospects. Furthermore, the anticipated updates by year-end 2024 on BALLI-01 progress could serve as key catalysts for stock movements.

In the broader market context, Cellectis' advancements with UCART22 reflect the growing importance and potential of allogeneic CAR T therapies in treating rare and severe diseases.

NEW YORK, June 04, 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, announced today that the European Commission (EC) has granted an Orphan Drug Designation (ODD) to its product candidate UCART22, for the treatment of Acute Lymphoblastic Leukemia (ALL).

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.

ALL represents 12% of all leukemia cases, progresses rapidly, and is typically fatal within weeks or months if left untreated¹. In 2024, the 10-year prevalence is estimated at 1.9 in 100,000 persons in the European Union (EU). Based on the preliminary clinical data generated with UCART22 in heavily pretreated patients who were relapsed or refractory to approved medicinal products, the European Medicines Agency (EMA) considered that the significant benefit of UCART22 has been demonstrated.

“Patients with relapsed/refractory ALL have limited, if any, treatment options, especially for those who have failed prior CD19 directed CAR T-cell therapy and allogeneic stem cell transplant,” said Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. “The Orphan Drug Designation for UCART22 marks an important step towards developing allogeneic CAR T products that would be readily available for all patients.”

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 in-house) is more potent with a preliminary response rate of 67% at Dose Level 2, compared to a 50% response rate at Dose Level 3 with UCART22-P1 (manufactured by an external CDMO). Cellectis expects to provide updates on the progress of BALLI-01 by year-end 2024.

The Orphan Drug Designation in the EU is granted by the EC based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. Medicines intended for the treatment, diagnosis or prevention of seriously debilitating or life-threatening conditions that affect fewer than five in 10,000 people in the EU are eligible for the designation. The Orphan Drug Designation allows companies certain regulatory, financial, and commercial incentives to develop medicines for rare diseases where there are no satisfactory treatment options.

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1 (Dong et al., 2020)

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 “expect,” “would,” 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. 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). 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
Ashley R. Robinson, LifeSci Advisors, +1 617 430 7577 

Attachment

• PRESS RELEASE_ODD_UCART22(1)

FAQ

What is UCART22?

UCART22 is an allogeneic CAR T-cell product candidate developed by Cellectis targeting CD22 for treating Acute Lymphoblastic Leukemia (ALL).

What is the significance of the Orphan Drug Designation for UCART22?

The Orphan Drug Designation grants regulatory, financial, and commercial incentives to Cellectis for developing UCART22 to treat ALL.

What are the latest clinical results for UCART22?

The latest clinical data presented in December 2023 showed a 67% response rate at Dose Level 2 for UCART22-P2.

What is the current phase of the UCART22 trial?

UCART22 is currently being evaluated in a Phase 1/2 open-label dose-escalation and dose-expansion study.

How common is Acute Lymphoblastic Leukemia (ALL) in the EU?

ALL has an estimated 10-year prevalence of 1.9 per 100,000 persons in the European Union.

What does the Orphan Drug Designation (ODD) mean for CLLS stock?

The ODD may positively impact CLLS stock due to the regulatory, financial, and commercial incentives it provides.