CD52 inhibitors(CAMPATH-1 antigen inhibitors), ADCC(Antibody-dependent cell-mediated cytotoxicity (ADCC) effects), Complement dependent cytotoxicity (CDC) effects

Therapeutic Areas

NeoplasmsImmune System DiseasesNervous System Diseases+ [2]

Active Indication

Hematopoietic stem cell transplantationMultiple Sclerosis, Relapsing-RemittingChronic Lymphocytic Leukemia+ [2]

Inactive Indication

Acute Myeloid LeukemiaAutoimmune cytopeniasBone marrow transplant rejection+ [18]

Originator Organization

Active Organization

+ [2]

Inactive Organization

Bayer AG

Astex Pharmaceuticals, Inc.Genzyme Europe BV

+ [2]

Drug Highest PhaseApproved

First Approval Date

US (07 May 2001),

Chronic Lymphocytic LeukemiaMultiple Sclerosis

RegulationOverseas New Drugs Urgently Needed in Clinical Settings (CN), Orphan Drug (JP), Accelerated Approval (US)

Gene Sequence

Sequence Code 53009H

Source: *****

Sequence Code 53002L

Source: *****

325

Clinical Trials associated with Alemtuzumab

NCT06084780 / Not yet recruitingPhase 2IIT

Prospective Case Series of Intestinal and Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE)

The goal of this prospective phase 2 study is to assess the efficacy and safety of intestinal or multivisceral transplantation for participants with PMP not amenable to other curative-intent treatments. Participants will undergo intestinal/multivisceral transplantation. Participants will be followed for 12 months to assess efficacy and safety.

Start Date01 Oct 2024

Sponsor / Collaborator

The Case Comprehensive Cancer Center

NCT05501756 / RecruitingPhase 2IIT

Precision Alemtuzumab Dosing for Allogeneic Hematopoietic Cell Transplantation in Non-Malignant Diseases

Alemtuzumab is an antibody that reduces the strength of the immune system that is given in preparation for allogeneic hematopoietic cell transplant (HCT). In this research study the investigators want to find out if they can adjust the dose of alemtuzumab used as part of allogeneic HCT to target the level of Day 0 (the planned day of graft infusion) to an optimal therapeutic window of 0.15-0.9 ug/mL.

Start Date11 Jan 2023

Sponsor / Collaborator

Children's Hospital & Medical Center

NCT05607420 / RecruitingPhase 1/2

Open-label Dose-finding and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART20x22 in Subjects With Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma (B-NHL)

First-in-human, open-label, dose-finding and dose-expansion study of UCART20x22 administered intravenously in subjects with relapsed or refractory B-Cell Non-Hodgkin Lymphoma (B-NHL). The purpose of this study is to evaluate the safety and clinical activity of UCART20x22 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).

Start Date01 Nov 2022

Sponsor / Collaborator

Cellectis SA

100 Clinical Results associated with Alemtuzumab

100 Translational Medicine associated with Alemtuzumab

100 Patents (Medical) associated with Alemtuzumab

3,397

Literatures (Medical) associated with Alemtuzumab

01 Dec 2024·Clinical Immunology

Cladribine tablets in relapsing-remitting multiple sclerosis preferentially target B-cells

Article

Author: Baker, David ; Turner, Ben ; Wafa, Mohammad ; Schmierer, Klaus ; De Trane, Stefania ; Bestwick, Jonathan ; Marta, Monica ; Monero, Rosemary ; Ammoscato, Francesca ; Gnanapavan, Sharmilee ; Holden, David ; Adams, Ashok ; Giovannoni, Gavin ; Skonieczna, Justyna ; Bianchi, Lucia ; Andrews, Michael

Recent studies demonstrate the efficacy of B cell-targeting therapies in managing multiple sclerosis (MS) activity, emphasizing the critical role of B cells in MS pathogenesis. CladB study aimed to quantify the temporal changes in peripheral immune cells and their activity over 96 weeks of Cladribine tablets (CladT) treatment in relapsing-remitting MS (RRMS). Ten participants (3 males, 7 females) had blood samples collected at multiple intervals (Day 0, 1, 5, then weekly for 8 weeks, biweekly for up to 24 weeks, and monthly for up to 96 weeks) for immune cell analysis, compared to a historical alemtuzumab-treated cohort. Paired cerebrospinal fluid (CSF) was also taken for various analyses, alongside clinical and brain imaging assessments. CladT depleted memory B cells, while alemtuzumab rapidly depleted T and B cells. The кFLC index decreased from 164.5 ± 227.1 to 71.3 ± 84.7 at 48 weeks (p = 0.002) and to 64.4 ± 67.3 at 96 weeks (p = 0.01). The IgG index dropped from 1.1 ± 0.5 at baseline to 0.8 ± 0.4 at 48 weeks (p = 0.014) and to 0.8 ± 0.3 at 96 weeks (p = 0.02). CSF CXCL-13 decreased from 88.6 ± 68.4 pg/mL to 39.4 ± 35.2 pg/mL at 48 weeks (p = 0.037) and 19.1 ± 11.7 pg/mL at 96 weeks (p = 0.027). CSF NfL levels were reduced at 48 weeks (p = 0.01). CladT primarily depletes memory B cells and antibody-secreting cell precursors in RRMS, leading to sustained effects on intrathecal antibody production and total IgG, and a reduction in CSF CXCL-13.

01 Nov 2024·Transplantation

Late Treatment With Autologous Expanded Regulatory T-cell Therapy After Alemtuzumab Induction Is Safe and Facilitates Immunosuppression Minimization in Living Donor Renal Transplantation

Article

Author: Wood, Kathryn J. ; Hennessy, Conor ; Harden, Paul N. ; Hester, Joanna ; Alzhrani, Alaa ; Rombach, Ines ; Issa, Fadi ; Lombardi, Giovanna ; Friend, Peter ; Dutton, Susan ; Hammad, Salim ; Brook, Matthew O.

Background.The TWO Study (Transplantation Without Overimmunosuppression) aimed to investigate a novel approach to regulatory T-cell (Treg) therapy in renal transplant patients, using a delayed infusion protocol at 6 mo posttransplant to promote a Treg-skewed lymphocyte repopulation after alemtuzumab induction. We hypothesized that this would allow safe weaning of immunosuppression to tacrolimus alone. The COVID-19 pandemic led to the suspension of alemtuzumab use, and therefore, we report the unique cohort of 7 patients who underwent the original randomized controlled trial protocol. This study presents a unique insight into Treg therapy combined with alemtuzumab and is therefore an important proof of concept for studies in other diseases that are considering lymphodepletion.Methods.Living donor kidney transplant recipients were randomized to receive autologous polyclonal Treg at week 26 posttransplantation, coupled with weaning doses of tacrolimus, (Treg therapy arm) or standard immunosuppression alone (tacrolimus and mycophenolate mofetil). Primary outcomes were patient survival and rejection-free survival.Results.Successful cell manufacturing and cryopreservation until the 6-mo infusion were achieved. Patient and transplant survival was 100%. Acute rejection-free survival was 100% in the Treg-treated group at 18 mo after transplantation. Although alemtuzumab caused a profound depletion of all lymphocytes, including Treg, after cell therapy infusion, there was a transient increase in peripheral Treg numbers.Conclusions.The study establishes that delayed autologous Treg therapy is both feasible and safe, even 12 mo after cell production. The findings present a new treatment protocol for Treg therapy, potentially expanding its applications to other indications.

01 Nov 2024·Annals of Hematology

Antileukaemic rescue by dose-dense donor-lymphocyte infusions in T-PLL after allogeneic stem cell transplantation – a case report

Article

Author: Krüger, William H ; Späth, Christian ; Neumann, Thomas

AbstractT-Cell Prolymphocytic Leukaemia (T-PLL) is an aggressive disease with a poor prognosis and only curable by allogeneic stem cell transplantation. We describe the case of a male suffering from T-PLL. Therapy was alemtuzumab followed by an allograft from an unrelated donor. T-PLL relapsed after allogeneic stem cell transplantation. Discontinuation of immunosuppression had no effected and three increasing doses of donor lymphocytes were given within one month. The patient developed acute GvHD of the lover (grade III). GvHD was successfully treated by steroids and ruxolitinib and graft-versus-leukaemia effects induced a complete remission of T-PLL. 18,5 months after transplantation the patient is well and alive without GvHD under immunosuppression with ruxolitinib. Flow cytometry of peripheral blood was negative for residual leukemic cells.

News (Medical) associated with Alemtuzumab

18 Sep 2024

·www.businesswire.com

New Long-Term Zeposia (ozanimod) Data Demonstrate Durable Efficacy and Consistent Safety in Relapsing Forms of Multiple Sclerosis

PRINCETON, N.J.--( BUSINESS WIRE )-- Bristol Myers Squibb (NYSE: BMY) today announced new data from the Phase 3 DAYBREAK trial demonstrating that decreased rates of brain volume loss were sustained in the open-label extension (OLE) for patients treated with Zeposia (ozanimod) for relapsing forms of multiple sclerosis. These findings showed that patients receiving continuous Zeposia treatment for up to five years experienced low and stable rates of whole brain volume (WBV) loss through Month 60 (annualized least squares mean [LSM] % change from parent trial baseline: RADIANCE, −0.27; SUNBEAM, −0.35). Additionally, findings from a separate DAYBREAK OLE safety analysis demonstrated declining or stable incidence rates of treatment-emergent adverse events (TEAEs), with relatively low rates of infections, serious infections and opportunistic infections over more than eight years of treatment with Zeposia . These data and 12 additional abstracts will be presented at the 40 th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Copenhagen, Denmark taking place September 18-20, 2024. “If not treated early upon diagnosis, multiple sclerosis can lead to significant, irreversible brain volume loss and cognitive decline,” said Jeffrey Cohen, MD, Mellen Center for Multiple Sclerosis, Cleveland Clinic, Cleveland, Ohio and a paid consultant for Bristol Myers Squibb. “These new analyses reinforce the well-established safety and efficacy profile of Zeposia as an effective oral therapy, especially for newly diagnosed patients living with relapsing forms of multiple sclerosis.” Reductions in brain volume loss with Zeposia treatment The DAYBREAK OLE trial included 2,257 patients from the SUNBEAM and RADIANCE Phase 3 trials and evaluated rates of brain volume loss (Poster #P1623). Switching from interferon beta-1a (IFN-β) to Zeposia treatment consistently reduced rates of WBV loss (annualized LSM% change from RADIANCE baseline to Month 24 and DAYBREAK baseline to Month 24: −0.48 and −0.19, respectively, with a similar pattern observed in SUNBEAM). Additionally, similar reductions were observed for change in thalamic volume loss. High annualized LSM% reductions in cortical grey matter volume (CGMV) were observed with IFN-β (annualized change at Month 12 relative to SUNBEAM baseline: −1.02; annualized change at Month 24 relative to RADIANCE baseline: −0.59), but this trend reversed 12 months after switching to Zeposia in DAYBREAK (annualized LSM% increase relative to DAYBREAK baseline: patients from SUNBEAM, 0.10; patients from RADIANCE, 0.20), with low annualized LSM% CGMV loss observed thereafter. Established Zeposia safety profile confirmed with more than eight years of DAYBREAK data The final DAYBREAK OLE safety analysis (Poster #P1609) included 762 patients who were treated with continuous Zeposia with a median exposure of 83.9 months. Incident rates per 1,000 person-years decreased over time from the Phase 3 trials to Month 60 or more of the DAYBREAK OLE trial. Decreases were observed for overall TEAEs (896.1 versus 101.7), infections (300.5 versus 142.6), opportunistic infections (12.0 versus 4.9), cardiac (22.8 versus 9.5), hepatic (77.0 versus 15.7) and pulmonary disorders (11.3 versus 4.7), respectively. “The data presented at ECTRIMS further reinforce the long-term safety and efficacy of Zeposia and add to the robust body of evidence demonstrating its potential impact on decreasing disease progression over time,” said Alyssa Johnsen, MD, PhD , senior vice president and head of clinical development, Immunology, Cardiovascular and Neuroscience, Bristol Myers Squibb. “Building on our expertise with Zeposia , we are expanding our pipeline as we continue to look for new ways to advance the field of neuroscience. New modalities and disease targets fuel our goal of delivering medicines that elevate standards of care across neurological diseases, including multiple sclerosis.” Bristol Myers Squibb thanks the patients and investigators who participated in the Zeposia clinical trials. About DAYBREAK DAYBREAK was a Phase 3, multi-center, long-term open-label extension study to evaluate the safety and efficacy of Zeposia (ozanimod) administered orally to patients with relapsing forms of multiple sclerosis (RMS). Eligible patients from the RADIANCE, SUNBEAM and RPC01-1001 trials diagnosed with RMS were enrolled to receive treatment until the end of the DAYBREAK. Patients in the trial received Zeposia 0.92 mg (equivalent to 1 mg). About SUNBEAM SUNBEAM was a pivotal, Phase 3, multicenter, randomized, double-blind, double-dummy, active-controlled trial evaluating the efficacy, safety and tolerability of two doses of oral Zeposia (0.92 mg and 0.46 mg, equivalent to 1 mg and 0.5 mg, respectively) against weekly intramuscular Avonex ® for at least a 12-month treatment period. The study included 1,346 people living with RMS across 152 sites in 20 countries. The primary endpoint of the trial was annualized relapse rates during the treatment period. The secondary MRI endpoints included the number of new or enlarging hyperintense T2-weighted brain MRI lesions over 12 months, number of gadolinium-enhanced brain MRI lesions at Month 12 and percent change from baseline in whole brain volume at Month 12. Cortical grey and thalamic volume changes were also prospectively assessed versus active comparator. About RADIANCE RADIANCE Part B was a pivotal, Phase 3, multicenter, randomized, double-blind, double-dummy, active-controlled trial evaluating the efficacy, safety and tolerability of oral Zeposia 0.92 mg (equivalent to 1 mg) against weekly intramuscular Avonex ® (interferon beta-1a) over a 24-month treatment period. The study included 1,320 people living with RMS across 150 sites in 21 countries. The primary endpoint of the trial was annualized relapse rates over 24 months. The secondary MRI endpoints included the number of new or enlarging hyperintense T2-weighted brain MRI lesions over 24 months. About Multiple Sclerosis Multiple sclerosis (MS) is a disabling, unpredictable disease in which the immune system attacks the protective myelin sheath that covers the nerves. The myelin damage disrupts communication between the brain and the rest of the body. Ultimately, the nerves themselves may deteriorate—a process that's currently irreversible. MS affects 700,000 people in Europe and approximately 2.9 million people worldwide. Relapsing forms of MS (RMS), including clinically isolated syndrome, relapsing remitting disease and active secondary progressive disease, is characterized by clearly defined attacks of worsening neurologic function. These attacks—often called relapses, flare-ups or exacerbations—are followed by partial or complete recovery periods. During these recovery periods, also called remissions, symptoms improve partially or completely with no apparent progression of disease. However, smoldering neuroinflammation can be present from the earliest stages of MS, which is underlying and continuous disease activity occurring simultaneously in different areas of the brain that contributes to disability accumulation. Since MS relapses are unpredictable, patients can feel frustrated, stressed or scared when they occur. RMS is the most common disease course at the time of diagnosis. Approximately 85% of patients are initially diagnosed with RMS, compared with 10%-15% diagnosed with progressive forms of the disease. Bristol Myers Squibb: Delivering Breakthrough Science for Meaningful Interventions in Neuroscience Neurological conditions represent some of the greatest challenges of our time because of their impact on society, including patients, caregivers, families and healthcare systems. At Bristol Myers Squibb, we are committed to advancing our robust pipeline of potential medicines for neurological disorders with the goal of modifying disease and improving quality of life. Leveraging genetics, biomarkers and predictive sciences, we target key pathways involved in the initiation and progression of neurological diseases to develop therapies with the potential to optimize patient outcomes. About Zeposia (ozanimod) Zeposia (ozanimod) is an oral, sphingosine 1-phosphate (S1P) receptor modulator that binds with high affinity to S1P receptors 1 and 5. Zeposia blocks the capacity of lymphocytes to egress from lymph nodes, reducing the number of lymphocytes in peripheral blood. The mechanism by which Zeposia exerts therapeutic effects in multiple sclerosis (MS) is unknown but may involve the reduction of lymphocyte migration into the central nervous system. Zeposia is approved in numerous countries around the world for the treatment of adults with relapsing forms of MS and adults with moderately to severely active ulcerative colitis. U.S. FDA APPROVED INDICATIONS ZEPOSIA ® (ozanimod) is indicated for the treatment of: Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. Moderately to severely active ulcerative colitis (UC) in adults. IMPORTANT SAFETY INFORMATION Contraindications: Patients who in the last 6 months, experienced myocardial infarction, unstable angina, stroke, transient ischemic attack (TIA), decompensated heart failure requiring hospitalization, or Class III/IV heart failure or have a presence of Mobitz type II second-degree or third-degree atrioventricular (AV) block, sick sinus syndrome, or sino-atrial block, unless the patient has a functioning pacemaker Patients with severe untreated sleep apnea Patients taking a monoamine oxidase (MAO) inhibitor Infections: ZEPOSIA may increase the susceptibility to infections. Life-threatening and rare fatal infections have occurred in patients receiving ZEPOSIA. Obtain a recent (i.e., within 6 months or after discontinuation of prior MS or UC therapy) complete blood count (CBC) including lymphocyte count before initiation of ZEPOSIA. Delay initiation of ZEPOSIA in patients with an active infection until the infection is resolved. Consider interruption of treatment with ZEPOSIA if a patient develops a serious infection. Continue monitoring for infections up to 3 months after discontinuing ZEPOSIA. Herpes zoster was reported as an adverse reaction in ZEPOSIA-treated patients. Herpes simplex encephalitis and varicella zoster meningitis have been reported with sphingosine 1-phosphate (S1P) receptor modulators. Patients without a healthcare professional-confirmed history of varicella (chickenpox), or without documentation of a full course of vaccination against varicella zoster virus (VZV), should be tested for antibodies to VZV before initiating ZEPOSIA. A full course of vaccination for antibody-negative patients with varicella vaccine is recommended prior to commencing treatment with ZEPOSIA. Cases of fatal cryptococcal meningitis (CM) were reported in patients treated with another S1P receptor modulator. If CM is suspected, ZEPOSIA should be suspended until cryptococcal infection has been excluded. If CM is diagnosed, appropriate treatment should be initiated. In the MS and UC clinical studies, patients who received ZEPOSIA were not to receive concomitant treatment with antineoplastic, non-corticosteroid immunosuppressive, or immune-modulating therapies used for treatment of MS and UC. Concomitant use of ZEPOSIA with any of these therapies would be expected to increase the risk of immunosuppression. When switching to ZEPOSIA from immunosuppressive medications, consider the duration of their effects and their mode of action to avoid unintended additive immunosuppressive effects. Use of live attenuated vaccines should be avoided during and for 3 months after treatment with ZEPOSIA. If live attenuated vaccine immunizations are required, administer at least 1 month prior to initiation of ZEPOSIA. Progressive Multifocal Leukoencephalopathy (PML): PML is an opportunistic viral infection of the brain that typically occurs in patients who are immunocompromised, and that usually leads to death or severe disability. PML has been reported in patients treated with S1P receptor modulators, including ZEPOSIA, and other MS and UC therapies and has been associated with some risk factors. If PML is suspected, withhold ZEPOSIA and perform an appropriate diagnostic evaluation. If confirmed, treatment with ZEPOSIA should be discontinued. Immune reconstitution inflammatory syndrome (IRIS) has been reported in MS patients treated with S1P receptor modulators who developed PML and subsequently discontinued treatment. IRIS presents as a clinical decline in the patient’s condition that may be rapid, can lead to serious neurological complications or death, and is often associated with characteristic changes on MRI. The time to onset of IRIS in patients with PML was generally within a few months after S1P receptor modulator discontinuation. Monitoring for development of IRIS and appropriate treatment of the associated inflammation should be undertaken. Bradyarrhythmia and Atrioventricular Conduction Delays: Since initiation of ZEPOSIA may result in a transient decrease in heart rate and atrioventricular conduction delays, dose titration is recommended to help reduce cardiac effects. Initiation of ZEPOSIA without dose escalation may result in greater decreases in heart rate. If treatment with ZEPOSIA is considered, advice from a cardiologist should be sought for those individuals: with significant QT prolongation with arrhythmias requiring treatment with Class 1a or III anti-arrhythmic drugs with ischemic heart disease, heart failure, history of cardiac arrest or myocardial infarction, cerebrovascular disease, and uncontrolled hypertension with a history of Mobitz type II second-degree or higher AV block, sick sinus syndrome, or sino-atrial heart block Liver Injury: Elevations of aminotransferases may occur in patients receiving ZEPOSIA. Obtain liver function tests, if not recently available (i.e., within 6 months), before initiation of ZEPOSIA. Patients who develop symptoms suggestive of hepatic dysfunction should have hepatic enzymes checked and ZEPOSIA should be discontinued if significant liver injury is confirmed. Fetal Risk: There are no adequate and well-controlled studies in pregnant women. Based on animal studies, ZEPOSIA may cause fetal harm. Women of childbearing potential should use effective contraception to avoid pregnancy during treatment and for 3 months after stopping ZEPOSIA. Women who become pregnant while taking ZEPOSIA for MS may enroll in the ZEPOSIA pregnancy registry by calling 1-877-301-9314 or visiting www.zeposiapregnancyregistry.com . Increased Blood Pressure: Increase in systolic pressure was observed after about 3 months of treatment and persisted throughout treatment. Blood pressure should be monitored during treatment and managed appropriately. Certain foods that may contain very high amounts of tyramine could cause severe hypertension in patients taking ZEPOSIA. Patients should be advised to avoid foods containing a very large amount of tyramine while taking ZEPOSIA. Respiratory Effects: ZEPOSIA may cause a decline in pulmonary function. Spirometric evaluation of respiratory function should be performed during therapy, if clinically indicated. Macular Edema: S1P modulators have been associated with an increased risk of macular edema. Patients with a history of uveitis or diabetes mellitus are at increased risk. Patients with a history of these conditions should have an ophthalmic evaluation of the fundus, including the macula, prior to treatment initiation and regular follow-up examinations. An ophthalmic evaluation is recommended in all patients at any time if there is a change in vision. Continued use of ZEPOSIA in patients with macular edema has not been evaluated; potential benefits and risks for the individual patient should be considered if deciding whether ZEPOSIA should be discontinued. Posterior Reversible Encephalopathy Syndrome (PRES): Rare cases of PRES have been reported in patients receiving a S1P receptor modulator. If a ZEPOSIA-treated patient develops unexpected neurological or psychiatric symptoms or any symptom/sign suggestive of an increase in intracranial pressure, a complete physical and neurological examination should be conducted. Symptoms of PRES are usually reversible but may evolve into ischemic stroke or cerebral hemorrhage. Delay in diagnosis and treatment may lead to permanent neurological sequelae. If PRES is suspected, treatment with ZEPOSIA should be discontinued. Unintended Additive Immunosuppressive Effects From Prior Immunosuppressive or Immune-Modulating Drugs: When switching from drugs with prolonged immune effects, the half-life and mode of action of these drugs must be considered to avoid unintended additive immunosuppressive effects while at the same time minimizing risk of disease reactivation. Initiating treatment with ZEPOSIA after treatment with alemtuzumab is not recommended. Severe Increase in Multiple Sclerosis (MS) Disability After Stopping ZEPOSIA: In MS, severe exacerbation of disease, including disease rebound, has been rarely reported after discontinuation of a S1P receptor modulator. The possibility of severe exacerbation of disease should be considered after stopping ZEPOSIA treatment so patients should be monitored upon discontinuation. After stopping ZEPOSIA in the setting of PML, monitor for development of immune reconstitution inflammatory syndrome (PML-IRIS). Immune System Effects After Stopping ZEPOSIA: After discontinuing ZEPOSIA, the median time for lymphocyte counts to return to the normal range was 30 days with approximately 90% of patients in the normal range within 3 months. Use of immunosuppressants within this period may lead to an additive effect on the immune system, therefore caution should be applied when initiating other drugs 4 weeks after the last dose of ZEPOSIA. Most Common Adverse Reactions that occurred in the MS clinical trials of ZEPOSIA-treated patients (≥4%): upper respiratory infection, hepatic transaminase elevation, orthostatic hypotension, urinary tract infection, back pain, and hypertension. In the UC clinical trials, the most common adverse reactions that occurred in ≥4% of ZEPOSIA-treated patients and greater than in patients who received placebo were upper respiratory infection, liver test increased, and headache. Use in Specific Populations: Hepatic Impairment: Dosage adjustment in patients with mild or moderate hepatic impairment (Child-Pugh class A or B) is required, and use of ZEPOSIA in patients with severe hepatic impairment (Child-Pugh class C) is not recommended. For additional safety information, please see the full Prescribing Information and Medication Guide . About Bristol Myers Squibb Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn , Twitter , YouTube , Facebook and Instagram . Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that results of future post-marketing studies will be consistent with the results of this study, that Zeposia (ozanimod) for the indication described in this release may not be commercially successful, any marketing approvals, if granted, may have significant limitations on their use, and that continued approval of Zeposia for such indication may be contingent upon verification and description of clinical benefit in additional confirmatory trials. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2023, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise. corporatefinancial-news

Clinical ResultPhase 3Drug ApprovalVaccine

06 Aug 2024

·www.globenewswire.com

Cellectis Provides Financial Results for the Second Quarter 2024

ODD and RPDD granted by the FDA and ODD granted by the European Commission to UCART22 for the treatment of ALLODD granted by the FDA to CLLS52 (alemtuzumab) for ALL treatmentCash position of $273 million as of June 30, 20241; cash runway projection into 2026 NEW YORK, Aug. 06, 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 provided business updates and reported financial results for the six-month period ending June 30, 2024. "Over the past months, we have achieved a significant milestone with the granting of ODD designations by the Food and Drug Administration and the European Commission, complemented by the FDA’S Rare Pediatric Disease Designation. We have overcome major challenges, which reflects our ongoing commitment to innovation. Driven by an unwavering belief in our ability to revolutionize the healthcare field, we continue our pursuit of advancement with the confidence that our work will lead to the launch of a life-saving drug product. Our determination is the engine of our future success” said André Choulika, Ph.D., Chief Executive Officer at Cellectis. ________________________ 1 Cash position includes cash, cash equivalents, restricted cash and fixed-term deposits classified as current financial assets. Restricted cash was $5 million as of June 30, 2024. Fixed-term deposits classified as current financial assets were $119 million as of June 30, 2024. Pipeline Highlights UCART Clinical Programs On June 4, 2024, Cellectis received Orphan Drug Designation (ODD) from the European Commission (EC) for UCART22, for the treatment of acute lymphoblastic leukemia (ALL). The Orphan Drug Designation in the European Union is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. This designation may allow certain regulatory, financial, and commercial incentives to develop medicines for rare diseases where there are no satisfactory treatment options.On July 25, 2024, the FDA designated UCART22 as a drug for a Rare Pediatric Disease (RPDD). This designation may allow to obtain a “Priority Review Voucher” at the time of Biologics License Application (BLA). The FDA also granted ODD to UCART22 product candidate for ALL treatment. Receiving ODD by the FDA may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent.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. These designations for UCART22 mark an important step towards developing allogeneic CAR T products that would be readily available for all patients.On August 1, 2024, the FDA granted ODD to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity.Cellectis continues to focus on the enrollment of patients in the BALLI-01 study, evaluating UCART22 in relapsed or refractory B-cell acute lymphoblastic leukemia, in the NatHaLi-01 study, evaluating UCART20x22 in relapsed or refractory B-cell non-Hodgkin lymphoma, and in the AMELI-01 study, evaluating UCART123 in relapsed or refractory acute myeloid leukemia. We expect to provide updates in the advancements of BALLI-01 until the end of the year 2024. Research Data & Preclinical Programs Non-viral Gene Therapy Approach for Sickle Cell Disease On June 12, 2024, Cellectis announces the publication of a scientific article in Nature Communications.Cellectis leverages TALEN® technology and a non-viral gene repair template delivery to develop a clinically relevant gene editing process in hematopoietic stem and progenitor cells (HSPCs). This process enables efficient HBB gene correction with high precision, specificity and minimal genomic adverse events. Applying this HBB gene correction process to SCD patient-HSPCs results in over 50% expression of normal adult hemoglobin in mature red blood cells and in the correction of sickle phenotype, without inducing β-thalassemic phenotype. Edited HSPCs engraft efficiently in an immunodeficient murine model and maintain clinically relevant levels of HBB gene correction events. This comprehensive preclinical data package sets the stage for the therapeutic application of autologous gene corrected HSPCs to address SCD. Partnerships Licensed Allogeneic CAR T-cell Development Programs Anti-CD19 Programs Allogene’s investigational oncology products utilize Cellectis technologies. We have initiated an arbitration proceeding through the Centre de Médiation et d'Arbitrage de Paris. We are requesting that the arbitral tribunal issue a decision (i) terminating the Servier License Agreement, and (ii) requiring Servier to pay us fair financial compensation for losses incurred due to the lack of development of the licensed products and for non-payment of milestone payments for milestones that have been achieved under the Servier License Agreement. In May 2024, Allogene announced the execution of an Amendment and Settlement Agreement (the "Servier Amendment"), which amended the license agreement between Servier and Allogene, under which Servier exclusively sublicensed to Allogene its rights under the License Agreement between Cellectis and Servier (the "Servier License"), for the development and commercialization of allogeneic anti-CD19 CAR T cell product candidates in the U.S. (the "Allogene Sublicense"). Allogene disclosed that, pursuant to the Servier Amendment to the Allogene Sublicense, the licensed territory was expanded to include the European Union and the United Kingdom, and Allogene was granted an option to further extend its licensed territory to include China and Japan subject to certain conditions. Corporate Updates Collaboration and Investment Agreements with AstraZeneca On May 6, 2024, Cellectis announced the completion of the subsequent investment of $140M in Cellectis by AstraZeneca (LSE/STO/Nasdaq: AZN) (the “Additional Investment”).AstraZeneca subscribed for 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 convertible preferred share, issued by the Board of Directors of Cellectis.On the completion date of the Additional Investment, AstraZeneca owned approximately 44% of the share capital and 30% of the voting rights of the Company (based on the number of voting rights outstanding at the time).Following the Additional Investment, Mr. Marc Dunoyer and Dr. Tyrell Rivers have been nominated members of the board of directors of Cellectis, designated by AstraZeneca. Annual Shareholders Meeting On June 28, 2024, Cellectis held a shareholders’ general meeting at the Biopark auditorium in Paris, France.At the meeting, during which approximately 40% of shares were exercised, resolutions 1 through 28 were adopted and resolution 29 was rejected, consistent with the recommendations of the management. The detailed results of the vote and the resolutions are available on Cellectis’ website: https://www.cellectis.com/en/investors/general-meetings/ Financial Results The interim condensed consolidated financial statements of Cellectis have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board (“IFRS”). As from June 1, 2023, and the deconsolidation of Calyxt, which corresponded to the Plants operating segment, we view our operations and manage our business in a single operating and reportable segment corresponding to the Therapeutics segment. For this reason, we are no longer presenting financial measures broken down between our two reportable segments – Therapeutics and Plants. In the appendices of this Q2 2024 financial results press release, Calyxt's results are isolated under "Income (loss) from discontinued operations" for the 6-month period ended June 30, 2023, and are no longer included for the 6-month period ended June 30, 2024, due to the deconsolidation. Cash: As of June 30, 2024, Cellectis had $273 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current-financial assets. This compares to $156 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current-financial assets as of December 31, 2023. This $117 million increase is mainly due to cash payments from Cellectis to suppliers of $26 million, including $18 million to R&D suppliers and $8 million to SG&A suppliers, Cellectis’ wages, bonuses and social expenses paid of $24 million, the payments of lease debts of $5 million and the repayment of the “PGE” loan of $3 million, partially offset by the $16 million cash received from EIB pursuant to the disbursement of the €15 million Tranche B, $5 million of cash-in from our financial investments, $14 million of cash-in from our revenue, $140 million cash received from AstraZeneca as part of its equity investment in Cellectis. With cash and cash equivalents of $149 million and $119 million term deposit classified as current financial assets as of June 30, 2024, the Company believes its cash and cash equivalents and deposits will be sufficient to fund its operations into 2026 and therefore for at least twelve months following the unaudited interim condensed consolidated financial statements' publication. Revenues and Other Income: Consolidated revenues and other income were $16.0 million for the six months ended June 30, 2024 compared to $5.6 million for the six months ended June 30, 2023. This $10.4 million increase between the six months ended June 30, 2023 and 2024 was mainly attributable to (i) recognition of a $12.3 million revenue in 2024 based (a) on the progress of our performance obligation rendered under the first research plan of the Joint Research and Collaboration Agreement (the “JRCA”) signed with AstraZeneca Ireland Limited (AZ Ireland) and (b) the reaching of a development milestone under the License Agreement signed with Servier, while revenues recognized for the six months ended June 30, 2023 were immaterial, (ii) a decrease of research tax credit of $1.1 million due to a decrease of eligible expenses, and (iii) the recognition in the six-month periods ended June 30, 2023 of $0.8 million representing the portion of an initial payments from BPI corresponding to a grant pursuant to our grant and repayable advance agreement with BPI signed in March 2023. R&D Expenses: Consolidated R&D expenses were $45.8 million for the six months ended June 30, 2024, compared to $43.6 million for the six months ended June 30, 2023. R&D personnel expenses decreased by $0.8 million from $20.0 million in 2023 to $19.2 million in 2024 primarily due to a decrease in the average unit fair value of stock options and free share awards vesting between the two periods. R&D purchases, external expenses and other increased by $3.1 million (from $23.6 million in 2023 to $26.7 million in 2024) mainly related to increase in manufacturing activities to support our R&D pipeline. SG&A Expenses: Consolidated SG&A expenses were $9.0 million for the six months ended June 30, 2024 compared to $8.9 million for the six months ended June 30, 2023. SG&A personnel expenses decreased by $0.2 million (from $4.0 million in 2023 to $3.8 million in 2024), with a $0.4 million increase in salaries being offset by a $0.6 million decrease in stock-based compensation expenses. SG&A purchases, external expenses and other increased by $0.3 million (from $4.9 million in 2023 to $5.2 million in 2024). Other operating income and expenses: Other operating income and expenses were a $0.7 million net income for the six months ended June 30, 2024 compared to a $0.1 million net expense for the six months ended June 30, 2023. Other operating income and expenses decreased by $0.8 million primarily due to the recognition of costs related to a litigation of $0.5 million in 2023. Net financial gain (loss): We had a consolidated net financial gain of $18.0 million for the six months ended June 30, 2024, compared to a $10.2 million loss for the six months ended June 30, 2023. This $28.3 million difference reflects mainly (i) a $14.3 million gain in change in fair value of SIA derivative instrument, (ii) a $3.2 million increase in gain from our financial investments, (iii) a $4.3 million gain in change in fair value of EIB tranche A and B, , (iv) a $5.5 million decrease of the loss in fair value of our investment in Cibus and (vi) the loss in fair value measurement on Cytovia convertible note recognized in the six months period ended June 30, 2023 of $6.8 million, partially offset by (i) a $1.3 million interest expense on EIB Tranche A and Tranche B loans and (ii) a $0.7 million increase in foreign exchange loss, and (iii) a decrease in net foreign exchange gain of $3.5 million. Net income (loss) from discontinued operations: Net income from discontinued operations of $8.4 million for the six months ended June 30, 2023 corresponded to Calyxt’s results. Since Calyxt has been deconsolidated since June 1, 2023, there is no longer any "Income (loss) from discontinued operations" for the six months ended June 30, 2024. Net Income (loss) Attributable to Shareholders of Cellectis: Consolidated net loss attributable to shareholders of Cellectis was $19.6 million (or a $0.24 loss per share) for the six months ended June 30, 2024, compared to a $41.8 million loss (or a $0.78 loss per share) for the six months ended June 30, 2023, of which $57.6 million was attributed to Cellectis continuing operations. The $29.5 million change in net loss was primarily driven by (i) an increase in revenues and other income of $10.4 million, (ii) a decrease of $1.5 million in non-cash stock based compensation expense due to a decrease in the average unit fair value of stock options and free share awards vesting between the two periods, (iii) a $28.3 million change from a net financial loss of $10.2 million to a net financial gain of $18.0 million and (iv) a decrease in net other operating expense of $0.8 million, and (v) a $8.4 million decrease in net income from discontinued operations attributable to shareholders of Cellectis, partially offset by (i) an increase of $3.3 million in purchases, external expenses and other, and a (ii) an increase of $0.4 million in wages. Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: Consolidated adjusted net loss attributable to shareholders of Cellectis was $17.9 million (or a $0.22 loss per share) for the six months ended June 30, 2024, compared to a net loss of $36.7 million (or a $0.68 loss per share) for the six months ended June 30, 2023. Please see "Note Regarding Use of Non-IFRS Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis. We currently foresee focusing our cash spending at Cellectis for 2024 in the following areas: Supporting the development of our pipeline of product candidates, including the manufacturing and clinical trial expenses of UCART22, UCART20x22, UCART123 and potential new product candidates, andOperating our state-of-the-art manufacturing capabilities in Paris (France), and Raleigh (North Carolina, USA); andContinuing strengthening our manufacturing and clinical departments. CELLECTIS S.A.STATEMENT OF CONSOLIDATED FINANCIAL POSITION (unaudited)($ in thousands) As of December 31, 2023 June 30, 2024ASSETS Non-current assets Intangible assets 671 653 Property, plant, and equipment 54,681 50,370 Right-of-use assets 38,060 33,671 Non-current financial assets 7,853 16,650 Total non-current assets 101,265 101,344 Current assets Trade receivables 569 9,741 Subsidies receivables 20,900 14,958 Other current assets 7,722 7,587 Current deferred tax assets 710 Cash and cash equivalent and Current financial assets 203,815 272,806 Total current assets 233,005 305,803 TOTAL ASSETS 334,270 407,147 LIABILITIES Shareholders’ equity Share capital 4,365 5,897 Premiums related to the share capital 522,785 606,146 Currency translation adjustment (36,690) (38,077)Retained earnings (304,707) (405,729)Net income (loss) (101,059) (19,627)Total shareholders’ equity - Group Share 84,695 148,610 Non-controlling interests 0 0 Total shareholders’ equity 84,695 148,610 Non-current liabilities Non-current financial liabilities 49,125 58,348 Non-current lease debts 42,948 38,362 Non-current provisions 2,200 2,194 Non-current deferred tax liabilities 158 0 Total non-current liabilities 94,431 98,904 Current liabilities Current financial liabilities 5,289 5,119 Current lease debts 8,502 8,357 Trade payables 19,069 18,213 Deferred revenues and deferred income 110,325 117,754 Current provisions 1,740 884 Current deferred tax liabilities 122 Other current liabilities 10,219 9,184 Total current liabilities 155,144 159,633 TOTAL LIABILITIES AND SHAREHOLDERS’ EQUITY 334,270 407,147 UNAUDITED STATEMENTS OF CONSOLIDATED OPERATIONSFor the three-month period ended June 30, 2024($ in thousands, except per share amounts) For the three-month period ended June 30, 2023* 2024 Revenues and other income Revenues 178 8,061 Other income 1,823 1,442 Total revenues and other income 2,001 9,504 Operating expenses Research and development expenses (22,200) (23,518)Selling, general and administrative expenses (3,950) (3,882)Other operating income (expenses) 528 686 Total operating expenses (25,622) (26,714) Operating income (loss) (23,621) (17,211) Financial gain (loss) (5,844) (8,251) Income tax (258) 193 Income (loss) from continuing operations (29,724) (25,270)Income (loss) from discontinued operations 13,083 0 Net income (loss) (16,641) (25,270)Attributable to shareholders of Cellectis (11,707) (25,270)Attributable to non-controlling interests (4,934) 0 Basic and diluted net income (loss) attributable to shareholders of Cellectis, per share ($/share) (0.20) (0.28)Diluted net income (loss) attributable to shareholders of Cellectis, per share ($/share) (0.20) (0.28)Basic and diluted net income (loss) attributable to shareholders of Cellectis from discontinued operations, per share ($ /share) 0.32 0.00 Diluted net income (loss) attributable to shareholders of Cellectis from discontinued operations, per share ($ /share) 0.32 0.00 Number of shares used for computing Basic 55,583,768 89,852,142 Diluted 55,583,768 89,852,142 *These amounts reflect Calyxt's adjustments as presented in Cellectis 2023 20F (Note 3) Cellectis S.A.UNAUDITED STATEMENTS OF CONSOLIDATED OPERATIONSFor the six-month period ended June 30, 2024($ in thousands, except per share amounts) For the six-month period ended June 30, 2023* 2024 Revenues and other income Revenues 317 12,589 Other income 5,242 3,412 Total revenues and other income 5,560 16,002 Operating expenses Research and development expenses (43,614) (45,841)Selling, general and administrative expenses (8,914) (8,986)Other operating income (expenses) (83) 721 Total operating expenses (52,612) (54,107) Operating income (loss) (47,053) (38,105) Financial gain (loss) (10,246) 18,023 Income tax (258) 455 Income (loss) from continuing operations (57,557) (19,627)Income (loss) from discontinued operations 8,392 0 Net income (loss) (49,165) (19,627)Attributable to shareholders of Cellectis (41,781) (19,627)Attributable to non-controlling interests (7,384) 0 Basic net income (loss) attributable to shareholders of Cellectis, per share ($/share) (0.78) (0.24)Diluted net income (loss) attributable to shareholders of Cellectis, per share ($/share) (0.78) (0.24)Basic net income (loss) attributable to shareholders of Cellectis from discontinued operations, per share ($ /share) 0.29 0.00 Diluted net income (loss) attributable to shareholders of Cellectis from discontinued operations, per share ($ /share) 0.29 0.00 Number of shares used for computing Basic 53,541,010 80,881,026 Diluted 53,541,010 80,881,026 *These amounts reflect Calyxt's adjustments as presented in Cellectis 2023 20F (Note 3) Note Regarding Use of Non-IFRS Financial Measures Cellectis S.A. presents adjusted net income (loss) attributable to shareholders of Cellectis in this press release. Adjusted net income (loss) attributable to shareholders of Cellectis is not a measure calculated in accordance with IFRS. We have included in this press release a reconciliation of this figure to net income (loss) attributable to shareholders of Cellectis, which is the most directly comparable financial measure calculated in accordance with IFRS. Because adjusted net income (loss) attributable to shareholders of Cellectis excludes non-cash stock-based compensation expense—a non-cash expense, we believe that this financial measure, when considered together with our IFRS financial statements, can enhance an overall understanding of Cellectis’ financial performance. Moreover, our management views the Company’s operations, and manages its business, based, in part, on this financial measure. In particular, we believe that the elimination of non-cash stock-based expenses from Net income (loss) attributable to shareholders of Cellectis can provide a useful measure for period-to-period comparisons of our core businesses. Our use of adjusted net income (loss) attributable to shareholders of Cellectis has limitations as an analytical tool, and you should not consider it in isolation or as a substitute for analysis of our financial results as reported under IFRS. Some of these limitations are: (a) other companies, including companies in our industry which use similar stock-based compensation, may address the impact of non-cash stock- based compensation expense differently; and (b) other companies may report adjusted net income (loss) attributable to shareholders or similarly titled measures but calculate them differently, which reduces their usefulness as a comparative measure. Because of these and other limitations, you should consider adjusted net income (loss) attributable to shareholders of Cellectis alongside our IFRS financial results, including Net income (loss) attributable to shareholders of Cellectis. RECONCILIATION OF IFRS TO NON-IFRS NET INCOME (unaudited)For the three-month period ended June 30, 2024($ in thousands, except per share data) For the three-month period ended June 30, 2023* 2024 Net income (loss) attributable to shareholders of Cellectis (11,707) (25,270)Adjustment: Non-cash stock-based compensation expense attributable to shareholders of Cellectis 3,140 830 Adjusted net income (loss) attributable to shareholders of Cellectis (8,567) (24,440)Basic adjusted net income (loss) attributable to shareholders of Cellectis ($/share) (0.15) (0.27)Basic adjusted net income (loss) attributable to shareholders of Cellectis from discontinued operations ($ /share) (0.04) 0.00 Weighted average number of outstanding shares, basic (units) (1) 55,583,768 89,852,142 Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share) (1) (0.15) (0.27)Diluted adjusted net income (loss) attributable to shareholders of Cellectis from discontinued operations ($/share) (0.04) 0.00 Weighted average number of outstanding shares, diluted (units) (1) 55,583,768 89,852,142 *These amounts reflect Calyxt's adjustments as presented in Cellectis 2023 20F (Note 3) RECONCILIATION OF IFRS TO NON-IFRS NET INCOME (unaudited)For the six-month period ended June 30, 2024($ in thousands, except per share data) For the six-month period ended June 30, 2023* 2024 Net income (loss) attributable to shareholders of Cellectis (41,781) (19,627)Adjustment: Non-cash stock-based compensation expense attributable to shareholders of Cellectis 5,119 1,717 Adjusted net income (loss) attributable to shareholders of Cellectis (36,662) (17,910)Basic adjusted net income (loss) attributable to shareholders of Cellectis ($/share) (0.68) (0.22)Basic adjusted net income (loss) attributable to shareholders of Cellectis from discontinued operations ($ /share) (0.09) 0.00 Weighted average number of outstanding shares, basic (units) (1) 53,541,010 80,881,026 Diluted adjusted net income (loss) attributable to shareholders of Cellectis ($/share) (1) (0.68) (0.22)Diluted adjusted net income (loss) attributable to shareholders of Cellectis from discontinued operations ($/share) (0.09) 0.00 Weighted average number of outstanding shares, diluted (units) (1) 53,541,010 80,881,026 *These amounts reflect Calyxt's adjustments as presented in Cellectis 2023 20F (Note 3) 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 25 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 ”future,” “projection,” “will,” “ may,” “would,” “expect,” and “believe” 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, the potential of our candidate products programs and CLLS52, the outcome of the arbitration proceedings against Servier, and the sufficiency of cash to fund operations. 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). With respect to our cash runway, our operating plans, including product candidates 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.      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 Relations contact: Arthur Stril, Interim Chief Financial Officer, +1 (347) 809 5980, investors@cellectis.com Attachment 20240805_Cellectis Reports Financial Results for Second Quarter 2024 - V0508[7]

License out/inFinancial StatementCell TherapyGene TherapyOrphan Drug

02 Aug 2024

·www.globenewswire.com

FDA Grants Orphan Drug Designation to Cellectis’ CLLS52 (alemtuzumab) For ALL Treatment

Aug. 01, 2024 -- 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 Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as part of the lymphodepletion regimen associated with UCART22, evaluated in the BALLI-01 clinical trial in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). “We are excited that the FDA granted CLLS52 (alemtuzumab) ODD designation status. The importance of adding alemtuzumab to the lymphodepletion regimen has been demonstrated in Cellectis’ BALLI-01 study, where the addition of this lymphodepletion agent to the fludarabine and cyclophosphamide regimen was associated with sustained lymphodepletion and significantly higher UCART22 cell expansion allowing for greater clinical activity,” said Mark Frattini, M.D., Ph.D. Chief Medical Officer at Cellectis. Cellectis is the inventor of the combination of CD52 knockout UCART cells with a lymphodepleting regimen containing an anti-CD52 antibody such as alemtuzumab. The CD52 knockout aims to render the UCART product candidates resistant to alemtuzumab as part of the lymphodepleting regimen. Cellectis’ UCART22 product candidate has the CD52 gene inactivated by TALEN® gene editing technology. 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. Receiving ODD may help to expedite and reduce the cost of development, approval, and commercialization of a therapeutic agent. 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 25 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). The content above comes from the network. if any infringement, please contact us to modify.

ImmunotherapyOrphan DrugGene TherapyCell TherapyClinical Study

100 Deals associated with Alemtuzumab

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KEGG	Wiki	ATC	Drug Bank
D02802	Alemtuzumab	L04AA34	DB00087

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hematopoietic stem cell transplantation	JP	Sanofi KK	26 Sep 2014
Multiple Sclerosis, Relapsing-Remitting	EU	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	IS	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	NO	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	LI	Sanofi	12 Sep 2013
Chronic Lymphocytic Leukemia	US	Genzyme Corp.	07 May 2001
Multiple Sclerosis	US	Genzyme Corp.	07 May 2001

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Chronic Lymphocytic Leukemia	Phase 2	IS	Genzyme Europe BV	06 Jul 2001
Chronic Lymphocytic Leukemia	Phase 2	EU	Genzyme Europe BV	06 Jul 2001
Chronic Lymphocytic Leukemia	Phase 2	NO	Genzyme Europe BV	06 Jul 2001
Chronic Lymphocytic Leukemia	Phase 2	LI	Genzyme Europe BV	06 Jul 2001
Acute Myeloid Leukemia	Preclinical	IL	Bayer AG	01 Jul 2004
Myelodysplastic Syndromes	Preclinical	IL	Bayer AG	01 Jul 2004

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02626715 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Hematopoietic stem cell transplantation \| Myelodysplastic Syndromes	21	Reduced-Intensity Conditioning Regimen (Reduced-Intensity Conditioning)	staxujyplz(izpvqbgsva) = tpsfnjolwz qybbhswbkm (aaxzywaiko, guwcbtfyyn - agkxdbdgmg) View more	-	16 Jul 2024
				Myeloablative Conditioning Regimen (Myeloablative Conditioning)	staxujyplz(izpvqbgsva) = pnrkzuppfe qybbhswbkm (aaxzywaiko, srkwjiuywx - ysuyyqguvl) View more
EHA2024	Not Applicable	Anemia, Aplastic Second line CD52	57	Alemtuzumab	(rhjxlcsgrz) = Adverse events were of low grade and infectious events were infrequent gbxckgdidt (vjzpkohnvj )	Positive	14 May 2024
NCT00520130 (Pubmed) Manual	Phase 1/2	Graft vs Host Disease	83	High-dose Alemtuzumab+Cyclosporine	(cspjkeqpfn) = fnyhabwuww xapvpozvqo (sphejnavxn ) View more	Positive	26 Apr 2024
				Tacrolimus+Methotrexate+Sirolimus	(cspjkeqpfn) = cddgmvfgsk xapvpozvqo (sphejnavxn ) View more
ISN WCN2024	Not Applicable	Glomerulonephritis, IGA	40	Basiliximab induction	utbniwnnef(rxlzmelkba) = 55% patients lost their graft with an average time to graft loss of 8.2 years. Graft loss at 1, 5 and 10 years was 2%, 18% and 38% respectively. The use of steroids was associated with a trend to delayed graft loss, although this was statistically insignificant. There is no association between duration of steroid therapy and time to graft loss fhhfzkcswn (lxpafhspbb ) View more	Positive	01 Apr 2024
				Alemtuzumab induction
NCT00061568 (CTgov)	Phase 1/2	Beta-Thalassemia \| Anemia, Diamond-Blackfan \| Anemia, Sickle Cell	130	Peripheral blood hematopoietic progenitor cell (PBPC) transplant+Sirolimus+Alemtuzumab (Participants With Severe Beta-globin Disorders in Allogeneic Peripheral Blood Stem Cell Transplants)	phikgupvyy(acrlsuwxdj) = bslyzbkcsd weqbsrlhmk (iedwtngagu, pvwoxusvok - aamzepizhn) View more	-	29 Feb 2024
				Peripheral blood hematopoietic progenitor cell Apheresis (Human Leukocyte Antigens (HLA) Matched Related Stem Cell Donor)	yyszyoddsz(adcchietth) = dxlshybdsc lpfatptvdj (uysmlmluap, dnqyxakjxd - huszimdmxk) View more
NCT02385110 (CTgov)	Phase 2	Lymphohistiocytosis, Hemophagocytic \| Leukemia	18	Phone Call+etoposide+Dexamethasone+Methotrexate+Alemtuzumab (Group 1: Alemtuzumab + Etoposide + Dexamethasone)	lzigtvfpbi(chymbssozy) = kigoeaghyf dilodaypum (dgyobgvxrf, qxnajzfpfw - eolgcxjbys) View more	-	17 Jan 2024
				Phone Call+etoposide+tocilizumab+Dexamethasone+Methotrexate+Alemtuzumab (Group 2: Etoposide + Dexamethasone + Tocilizumab)	lzigtvfpbi(chymbssozy) = fqzixntphb dilodaypum (dgyobgvxrf, fqoovqtwel - tgshlsoofn) View more
ECTRIMS2023	Not Applicable	Multiple Sclerosis, Relapsing-Remitting	-	Cladribine	(rvhmncbgrq): HR = 1.08 (95% CI, 0.47 - 2.47)	-	30 Sep 2023
				Fingolimod
ECTRIMS2023	Not Applicable	CD52	39	Alemtuzumab	oqjwsljwzx(wqhmtvdvhl) = rigoktgmfg pxwtvgxnmy (vempudvcqo ) View more	Positive	30 Sep 2023
ECTRIMS2023	Not Applicable	Multiple Sclerosis GFAP \| NfL	51	Alemtuzumab	szbsspyuvu(eguilshxgo) = irtmknzkzy kbyoknzpra (bihvlnogxr ) View more	Positive	30 Sep 2023
				alemtuzumab	rvbsmscsif(qzvhhpajwa) = mhfiepahco qblaqxoyrq (lpsaooqgyc, 248 - 9080)
ECTRIMS2023	Not Applicable	Multiple Sclerosis CD52	391	Alemtuzumab	thtgmdmcaj(kaqenciodq) = 38% autoimmune thyroiditis mlfomlznpm (vhgvrbdehp ) View more	Positive	30 Sep 2023

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