Commitment to a Cure
Corporate Presentation
May 2024
NASDAQ: CLLS
EURONEXT GROWTH: ALCLS.PA
Forward-Looking Statements
This presentation 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 "designed to", "anticipate," "expected," "on track," "plan," "scheduled," "should", and "will," "would", or the negative of these and similar expressions.
These forward-looking statements, which are based on our management's current expectations and assumptions and on information currently available to management, including information provided or otherwise publicly reported by our licensed partners. Forward-looking statements about advancement, timing and progress of clinical trials (including with respect to patient enrollment and follow-up), the timing of our presentation of data and submission of regulatory filings, the adequacy of our supply of clinical vials, the operational capabilities at our manufacturing facilities, the sufficiency of cash to fund operations, the potential benefit of our product candidates and technologies, the potential payments for which Cellectis is eligible under the agreements signed between Cellectis and each of its partners, including AstraZeneca, Servier, Allogene and Iovance and the financial position of Cellectis.
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.
With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. 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.
2
Cellectis at a Glance
3 Clinical Trials
70+ patients dosed
in Cellectis-sponsored trials
Global GMP
Facilities
End-to-end manufacturing
autonomy
Near-Term Clinical
Catalysts
Multiple near-term UCART
clinical data updates
$156M
*as of December 31, 2023
Cash Runway into 2026**
Diversified Partnerships with Industry Leaders
~220 patients dosed to date
- Revenues > $6B in milestones + royalties
- 5 clinical trials sponsored by Cellectis' licensed partners
*Cash position includes cash, cash equivalents, restricted cash and fixed-termdeposits classified as current -financialassets. Restricted cash was $5 million as of December 31, 2023. Fixed-term | 3 |
deposits classified as current-financial assets was $15 million as of December 31, 2023. |
** cash runway includes the additional investment by AstraZeneca of $140 million, completed on May 3, 2024.
A Highly-Experienced Executive Committee
André Choulika, Ph.D. | Steven Doares, Ph.D. | Phillippe Duchateau, Ph.D. | Mark Frattini, M.D., Ph.D. | Kyung Nam-Wortman | ||||||||||||||||
Founder & CEO | SVP, US Manufacturing | Chief Scientific Officer | Chief Medical Officer | EVP, Chief Human | ||||||||||||||||
& Site Head | Resources Officer | |||||||||||||||||||
Stephan Reynier | David Sourdive, Ph.D. | Arthur Stril | Marie-Bleuenn Terrier |
Chief Regulatory & | EVP CMC & Manufacturing | Interim Chief Financial Officer | General Counsel |
Pharmaceutical Compliance Officer | & Co-Founder |
4
UCARTs are Designed to be "Off-The-Shelf"
Scalable
Manufacturing
Reduced cost
Scalable manufacturing:
1 batch = 100s doses
Robustness
The goal is to provide
potency and consistency
to each patient
Market Access
Immediately available to all eligible patients
Control Production, Costs and Patients Safety
5
Strategic Partnership with AstraZeneca
Cell & Gene Therapy R&D Collaboration
- Develop up to 10 novel products in oncology, immunology and rare diseases
- $25M upfront
- Milestones from $70M to $220M per product with tiered royalties
- AstraZeneca to cover Cellectis' research costs
Investment Agreements
- $220M equity investment (subscribed for 16 million ordinary shares and 28 million convertible preferred shares at $5.00 per share)
Note:AstraZeneca has nominated two directors to the board of directors of Cellectis. Further, certain business decisions are subject to AstraZeneca's approval. For more | |
information on the agreements signed with AstraZeneca, please refer to the Annual Report filed on Form 20-F and the management report for the year ended December | 6 |
31, 2023 |
Experts in Gene-Editing Use TALEN®
High rates of gene editing for
knock outs and knock ins
Efficient | |||
In clinic since 2015 | Mature | Safe | Protein / DNA interaction with |
~220 patients dosed | 32 base pairs recognition | ||
WHY | |||
TALEN®? | |||
Euchromatin & | Genome | Precise | Targets desired site with a |
heterochromatin | Outreach | maximum 7 bps range | |
Base Editors
TALEN® is a registered trademark owned by Cellectis
TALE Base Editing | 7 |
Cellectis' UCART Candidate Platform
TALEN® Powered
CD52 KO | TRAC KO | |
Resistance to | Minimizes risk of | |
alemtuzumab | GvHD |
CD52 | TCR |
CAR-T cell
CAR
CAR20+CAR22
Targeting CD20 & CD22
For B-cell Malignancies
CAR22
Targeting CD22
For B-cell ALL
CAR123
Targeting CD123
For AML
ALL: acute lymphoblastic leukemia; AML: acute myeloid leukemia; CAR: chimeric antigen receptor; GvHD: Graft versus Host Disease; KO: Knock-Out; TCR: T-cell receptor; TRAC: T-cell receptor | 8 |
alpha constant |
Differentiated Targets & Near-Term Catalysts
Candidate / Target | Indication | Study | Preclinical | Phase 1 Dose | Phase 1 Dose | Phase 2 | Upcoming Expected Milestones | |
Escalation | Expansion | Pivotal2 | ||||||
Owned | UCART22 | ALL | BALLI-01 | Data update with Recommended | ||||
CD22 | NCT04150497 | Phase 2 Dose (RP2D) | ||||||
UCART123 | AML | AMELI-01 | Data update with 2-dose regimen | |||||
Fully | CD123 | NCT03190278 | ||||||
UCART20x22 | NHL | NatHaLi-01 | Data update with Recommended | |||||
Dual Target | NCT05607420 | Phase 2 Dose (RP2D) | ||||||
CD20, CD22 | ||||||||
Licensed to: | ||||||||
CEMACABTAGENE | LBCL | ALPHA3 | ||||||
ANSEGEDLEUCEL | ||||||||
Partners | CD191 | |||||||
CEMACABTAGENE | CLL | ALPHA2 | U.S. rights | |||||
ANSEGEDLEUCEL | ||||||||
NCT04416984 | ||||||||
CD191 | ||||||||
ALLO-7153 | UNIVERSAL | |||||||
Licensed | MM | |||||||
BCMA | NCT04093595 | |||||||
ALLO-6053 | MM | IGNITE | ||||||
BCMA | NCT05000450 | |||||||
ALLO-3164 | RCC | TRAVERSE | ||||||
CD70 | NCT04696731 | |||||||
- cemacabtagene ansegedleucel has been developed under a collaboration agreement between Servier and Allogene based on an exclusive license granted by Cellectis to Servier. Servier grants to Allogene exclusive rights to cemacabtagene ansegedleucel in the U.S. The ALPHA3 and ALPHA2 studies target Large B-Cell Lymphoma (LBCL) and Chronic Lymphocytic Leukemia (CLL), respectively.
- Phase 3 may not be required if Phase 2 is registrational.
- ALLO-715and ALLO-605 utilize TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for allogeneic products directed
at the BCMA target. Allogene holds global development and commercial rights for this investigational candidate. | 9 |
4 ALLO-316 utilizes TALEN® gene-editing technology pioneered and owned by Cellectis. Allogene has an exclusive license to the Cellectis technology for |
allogeneic products directed at the CD70 target. Allogene holds global development and commercial rights for this investigational candidate. ALL, Acute Lymphoblastic Leukemia; AML, Acute Myeloid Leukemia; NHL, Non-Hodgkin's Lymphoma; RCC, Renal Cell Carcinoma
Cellectis' UCART Platform
10
Attachments
- Original Link
- Original Document
- Permalink
Disclaimer
Cellectis SA published this content on 12 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 May 2024 10:07:09 UTC.