tw0122
2年前
FDA Grants Orphan Drug and Rare Pediatric Disease Designation Status to Cellectis’ UCART22 product candidate for Acute Lymphoblastic Leukemia (ALL) Treatment
Source: GlobeNewswire Inc.
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 10% of all leukemia cases in the United States, progresses rapidly, and is typically fatal within weeks or months if left untreated. It is estimated that 6,660 new cases of ALL and 1,560 deaths related to the disease occurred in the US in 20222.
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 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 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????
Monksdream
3年前
Cellectis (Euronext Growth: ALCLS – NASDAQ: CLLS) today anounced that, following the consultation of its works council, it has now signed a binding Subsequent Investment Agreement with AstraZeneca (LSE/STO/Nasdaq: AZN) regarding the contemplated additional equity investment of $140M by AstraZeneca, which was previously announced on November 1, 2023. The additional investment will be made by way of subscription of 10,000,000 “class A” convertible preferred shares and 18,000,000 “class B” convertible preferred shares, in each case at a price of $5.00 per share (the “Additional Investment”).
tw0122
3年前
straZeneca to Invest $245 Million in Deal With Cellectis
Source: Dow Jones News
By Ben Otto
British drugmaker AstraZeneca plans to invest $245 million in a deal with biotechnology company Cellectis to develop next-generation therapeutics in oncology, immunology and rare diseases.
AstraZeneca said Wednesday that it will initially make a $105 million payment to Paris-based Cellectis in the fourth quarter, comprising an $80 million equity investment, for a 22% stake in the company, and $25 million in cash.
AstraZeneca plans to make a further $140 million equity investment, subject to regulatory and other approvals, with a closing in early 2024, taking its total stake in Cellectis to 44%.
AstraZeneca said it plans to leverage Cellectis's gene-editing technologies and manufacturing capabilities to design novel cell and gene therapy products.
Cellectis will be eligible to receive an investigational new drug option fee and various milestone payments ranging from $70 million to up to $220 million for each of 10 candidate products, plus tiered royalties, AstraZeneca said.
AstraZeneca will retain an option for an exclusive license for the products developed under the research collaboration agreement.
Monksdream
3年前
Cellectis SA NASDAQ: CLLS
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Health Care : Biotechnology | Small Cap Value | Based in FranceCompany profile
Cellectis SA is a France-based company active in the field of genome engineering and genomic surgery. The Company specializes in the research, development, and commercialization of rational genome engineering technologies. The Company develops immunotherapies that aim to force the immune system to target and eradicate cancer cells. It has developed an expertise in combining meganucleases with engineered targeting Deoxyribonucleic Acid (DNA) matrices into Meganuclease Recombination Systems (MRS), used for gene excision, correction or replacement. Cellectis SA markets its technologies mainly for use in the research field, in pharmaceutical drug discovery programs, in the agronomics, bioproduction, and biotherapeutics fields. Cellectis SA operates several subsidiaries. The Company operates in France and the United States, among others.
Frankestin
3年前
file:///C:/Users/bozh/Downloads/UCART123_AMELI-01_Phase_1_Oral_Presentation_ASGCT_2023_Final_Upload.pdf
file:///C:/Users/bozh/Downloads/051123_PosterASGCT2023_BAOa.pdf
Evidence of UCART123 anti-tumor activity was observed in four patients out of fifteen at DL2 or above with best overall responses in the FCA arm. Two out of eight patients (25%) at DL2 in the FCA arm achieved meaningful response: * A patient who failed five prior lines of therapy experienced a durable minimal residual disease (MRD) negative complete response (CR) with full count recovery at Day 56 that continues beyond one year.
* A patient with stable disease achieved greater than 90% bone marrow blast reduction (60% to 5%) at Day 28.
The preliminary data show that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity.
Patient Enrollment in a 2-Dose Regimen Arm
Overall, these preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML. Based on observed UCART123 expansion patterns and cytokine profiles, pursuant to an amended protocol, a second dose of UCART123 is given after 10-14 days to allow for additional UCART123 expansion and clinical activity without the use of additional lymphodepletion. The UCART123 cell expansion from the second dose of UCART123, in the setting of reduced disease burden, is expected to be safe and allow for clearance of residual disease.
“These clinically meaningful preliminary data from the AMELI-01 study are very encouraging for patients and for the future of allogeneic CART-cell therapy. AML is a disease with an urgent need for alternative treatment options for patients, and we are excited to be moving the study forward,” said Dr. Mark Frattini, M.D., Ph.D., Chief Medical Officer at Cellectis. “We have now implemented a two-dose regimen arm for our AMELI-01 trial and we look forward to sharing future updates as they become available.”
n4807g
9年前
with the GILD acquisition of KITE the potential for CAR-T, and more specifically Alogeneic CAR-T increases dramatically. FWIW CLLS is one of the leaders (if not the leader) in this sphere. Additionally, their partnership with Pfizer opens tantalizing possibilities for the future if the technology, and their therapies prove valid.
Something worth reading:
http://www.pfizer.com/news/press-release/press-release-detail/pfizer_and_cellectis_enter_into_global_strategic_cancer_immunotherapy_collaboration
and of particular interest.....
Under the terms of the agreement, Pfizer has exclusive rights to pursue development and commercialization of CAR-T therapies, in the field of oncology, directed at a total of fifteen targets selected by Pfizer. Both companies will work together on preclinical research and Pfizer will be responsible for the development and potential commercialization of any CAR-T therapies for the Pfizer-selected targets. In addition, the agreement provides for a total of twelve targets selected by Cellectis. Both companies will work together on preclinical research on four Cellectis-selected targets and Cellectis will work independently on eight additional targets. Cellectis will be responsible for clinical development and commercialization of CAR-T therapeutics for the Cellectis-selected targets. Pfizer has right of first refusal to the four Cellectis-selected targets.
I am long CLLS and accumulating shares.
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