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 LeukemiaAdult T-Cell Leukemia-LymphomaAnemia, Aplastic+ [37]

Originator Organization

Genzyme Corp.

Active Organization

Sanofi

Cellectis SASanofi-Aventis Korea Co., Ltd.

+ [3]

Inactive Organization

Bayer AG

Astex Pharmaceuticals, Inc.

Bayer HealthCare Pharmaceuticals, Inc.

+ [1]

License Organization

Bayer Pharma AG

Genzyme Corp.

Sanofi

+ [1]

Drug Highest PhaseApproved

First Approval Date

United States (07 May 2001),

Chronic Lymphocytic LeukemiaMultiple Sclerosis

RegulationOrphan Drug (United States), Orphan Drug (Japan), Orphan Drug (South Korea), Overseas New Drugs Urgently Needed in Clinical Settings (China)

Structure/Sequence

Sequence Code 53002L

Source: *****

Sequence Code 53009H

Source: *****

342

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 Feb 2026

Sponsor / Collaborator

The Case Comprehensive Cancer Center

NCT07214064

/ Not yet recruitingPhase 2IIT

Safety and Feasibility of a Venetoclax- Augmented Treosulfan-Based Reduced Intensity Conditioning Before Allogeneic Stem Cell Transplantation in AML, MDS/AML and Higher Risk MDS

Start Date01 Jan 2026

Sponsor / Collaborator-

NCT06959771

/ RecruitingPhase 1/2IIT

Base Editing Hematopoietic Stem Cell and BE T Cell Gene Therapy for CD40L-HyperIgM Syndrome-Single Patient Study

Background:
X-linked hyper-IgM (HIGM) syndrome is caused by a mutation in the CD40 ligand (CD40L) gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Researchers want to know if these base-edited stem cells and T cells can help people with CD40L-HIGM syndrome.
Objective:
To test base-edited stem cells and base-edited T cells in 1 person with CD40L-HIGM syndrome.
Eligibility:
A single male with CD40L-HIGM syndrome.
Design:
A single participant is planned to receive a single dose of edited stem cells and supportive treatment with edited T cells. Participant stem and T cells will undergo base editing to repair the mutation.
In preparation for the gene therapy, the participant will receive busulfan chemotherapy and alemtuzumab. After treatment, the participant will have follow-up visits every few months in the first 2 years after treatment. Long-term visits will continue annually for 15 years.

Start Date16 Jul 2025

Sponsor / Collaborator

National Institute of Allergy & Infectious Diseases

100 Clinical Results associated with Alemtuzumab

100 Translational Medicine associated with Alemtuzumab

100 Patents (Medical) associated with Alemtuzumab

2,804

Literatures (Medical) associated with Alemtuzumab

01 Apr 2026·JOURNAL OF NEUROIMMUNOLOGY

B cell tolerance checkpoint function in multiple sclerosis and transient CD52 depletion

Article

Author: Stathopoulos, Panos ; Karadima, Georgia ; O'Connor, Kevin C ; Kilidireas, Konstantinos ; Vakrakou, Aglaia G ; Tzanetakos, Dimitrios ; Michaletou, Chrysoula ; Anagnostouli, Maria ; Baltoumas, Fotis ; Tzanoudaki, Marianna ; Dalakas, Marinos C ; Stefanis, Leonidas ; Zografou, Chrysoula ; Pavlopoulos, Georgios ; Alexaki, Anastasia ; Kyriakaki, Galini ; Dame, Theodoros

Transient CD52 immune cell depletion with the monoclonal antibody alemtuzumab is highly effective in treating relapsing-remitting multiple sclerosis but often leads to secondary autoimmunity. Whether these effects are linked to an alteration of B cell tolerance mechanisms is currently not known. To evaluate peripheral B cell tolerance checkpoint integrity in patients and controls, we constructed 138 recombinant mAbs from single mature naïve B cells and tested their poly- and autoreactivity. We examined three healthy donors (HDs), three immunotherapy-naïve MS patients, and six patients treated with alemtuzumab at comparable time points post-treatment (mean ± SD 3.8 ± 0.39 years). Moreover, we investigated B cell receptor (BCR) repertoire parameters associated with tolerance mechanisms in the same subject groups. Polyreactive and autoreactive fraction means did not differ significantly among the three subgroups. Presence of a high or low autoreactive fraction of naïve B cells in patients treated with alemtuzumab did not correlate with secondary autoimmunity at the time of sampling and with future MS activity, and therefore most likely reflects stochastic variation in the context of immune reconstitution. In BCR repertoire analysis, alemtuzumab-treated patients showed lower mean naïve complementarity-determining region 3 (CDR3) net charge compared to HDs (P = 0.0036), an interesting yet isolated finding warranting further investigation. Overall, transient CD52 depletion did not seem to affect major changes in peripheral B cell tolerance checkpoint function as assessed with naïve B cell cloning and BCR NGS, while observations in the described setting may also apply to other immune reconstitution therapies.

01 Mar 2026·Multiple Sclerosis and Related Disorders

Highly active multiple sclerosis - An important, yet inaccurate concept

Article

Author: Albuquerque, Flavia Timbó ; de Menezes, Felipe Toscano Lins ; Lopes, Alexandre Bussinger ; Bichuetti, Denis Bernardi ; de Oliveira, Enedina Maria Lobato ; de Souza, Nilton Amorim

BACKGROUND:

Multiple sclerosis (MS) is a chronic demyelinating disease that often leads to disability in young adults. Among its various phenotypes, highly active MS (HA-MS) presents with frequent relapses, incomplete recovery, and a high burden of MRI lesions. Despite its clinical importance, there is no universally accepted definition of HA-MS, which complicates early identification and treatment decisions.

OBJECTIVE:

This study aimed to evaluate whether Brazilian neurologists can recognize HA-MS and whether there is agreement regarding its defining characteristics. It also explored how this recognition influences treatment choices.

METHODS:

A cross-sectional, self-administered online survey was distributed to neurologists affiliated with the Brazilian Academy of Neurology (ABN). The questionnaire was open from March to September 2023. Responses were analyzed using descriptive statistics and chi-square or Fisher's exact tests.

RESULTS:

A total of 206 neurologists completed the survey. Most respondents agreed that signs of high disease activity influence therapeutic decisions (98.1%). The most frequently cited indicators of HA-MS were annualized relapse rate (37.4%) and the number of gadolinium-enhancing lesions on MRI (32.0%). The Rush et al. definition was most commonly endorsed (53.9%), while Tintoré's was least favored (85.9%). High-efficacy therapies-particularly Natalizumab, Ocrelizumab, and Alemtuzumab-were the preferred treatment options. Neuroimmunologists were significantly more likely to prescribe advanced therapies and less likely to use first-line agents compared to general neurologists.

CONCLUSION:

Brazilian neurologists show substantial agreement in identifying clinical and radiological signs of HA-MS and favor early, high-efficacy treatment strategies. These findings highlight the need for clear, standardized criteria to guide consistent diagnosis and timely therapeutic intervention, ultimately improving patient care in MS.

01 Mar 2026·Multiple Sclerosis and Related Disorders

Real-world COVID-19 immunity in multiple sclerosis (CoVaR-MS): A longitudinal follow up study

Article

Author: Jenkinson, David ; Jordan, Kelvin P ; Salim, Omar ; Abraham, Roby J ; Banavathi, Krishna ; Goddard, Sarah ; Kalra, Seema

COVID-19 vaccination in Multiple Sclerosis (MS) has continued clinical relevance. Complex dosing and variability in immunosuppression/ immunomodulation from disease modifying treatments (DMTs) necessitate real-world studies of COVID-19 immunity. We aimed to evaluate if PwMS develop adequate and persistent serological and cellular COVID-19 immunity after vaccination. We conducted a real-world longitudinal study (n = 235) including PwMS (n = 205; Alemtuzumab, Dimethyl fumarate, Fingolimod, Interferon, Natalizumab, Ocrelizumab, no-DMT) and Healthy volunteers (HV) (n = 30). Serological and cellular immunity was tested at ≤18 and ≤24-month after completed COVID-19 vaccine-course. 97 % of both HV and PwMS were immune at ≤18-month, and 96.9 % PwMS and 100 % HV at ≤24-month, (OR 0.33, 95 % CI (0.09, 0.93), p = 0.05)). Both groups showed similar cellular immunity at both timepoints. Vaccine Adverse effects incidence was similar too. PwMS on Ocrelizumab were significantly less likely to have low serological immunity (p < 0.001 at both timepoints). They were more likely to have intact T cell responses than HV (OR 5.4, 95 % CI 1.28-22.60). Despite this, they had higher infections per person year. PwMS on Fingolimod also had low serological immunity (Odd's ratio 0.33; CI 0.11-0.97) which stayed low despite boosters and only 6 % showed intact cellular immunity, probably due to lymphopenia. Other DMT-treated PwMS (Natalizumab, Dimethyl Fumarate, Interferon, and no no-DMT) showed comparable COVID-19 serological and cellular immunity as HV. We report COVID-19 immunity clearly over time and across various DMT-groups. Ocrelizumab lowered serological but maintained cellular immunity; whilst Fingolimod lowered both. Data support boosters in PwMS, more so on Ocrelizumab and Fingolimod.

News (Medical) associated with Alemtuzumab

15 Jan 2026

·www.prnewswire.com

Hemophagocytic Lymphohistiocytosis Market Poised for Phenomenal Expansion in the US at a CAGR of 24.5% During the Forecast Period (2025-2034) | DelveInsight

The hemophagocytic lymphohistiocytosis market is expected to increase due to the launch of emerging therapies, such as plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), tadekinig alfa (r-hIL-18BP) (AB2 Bio), and others, as well as label expansions of current therapies. This is because of the higher patient burden, approval of newer therapies with high cost, and broader acceptance and availability of newly approved therapies. LAS VEGAS, Jan. 15, 2026 /PRNewswire/ -- DelveInsight's Hemophagocytic Lymphohistiocytosis Market Insights report includes a comprehensive understanding of current treatment practices, hemophagocytic lymphohistiocytosis emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophagocytic Lymphohistiocytosis Market Summary The market size for hemophagocytic lymphohistiocytosis was found to be USD 140 million in the United States in 2024. The HLH market remains highly unmet, with conventional therapies leading to high mortality (20–30% before HSCT) and only about 60% long-term survival due to toxicity, treatment failure, and relapse risk. Among the 7MM countries, there were 5,100 incident cases of HLH in the US in 2024. Leading hemophagocytic lymphohistiocytosis companies, such as TJ Biopharma, AB2 Bio, Electra Therapeutics, Novartis, and others, are developing new hemophagocytic lymphohistiocytosis treatment drugs that can be available in the hemophagocytic lymphohistiocytosis market in the coming years. The promising hemophagocytic lymphohistiocytosis therapies in clinical trials include Plonmarlimab (TJM2/TJ003234), Tadekinig alfa (r-hIL-18BP), ELA026, MAS 825, and others. Discover the new hemophagocytic lymphohistiocytosis treatment @ Key Factors Driving the Growth of the Hemophagocytic Lymphohistiocytosis Market Rising HLH Incidence: The increasing incidence of HLH, often triggered by underlying infections, autoimmune diseases, malignancies, or immuno-oncology treatments, has expanded the pool of patients needing diagnosis and therapy. Advances in Diagnostics and Early Detection: Improvements in diagnostic technologies, including genetic testing, immunologic profiling, and next-generation sequencing, enable earlier and more accurate detection of HLH. This allows timely intervention, which is critical given the rapid, life-threatening course of HLH. GAMIFANT Maintains First-in-Class Position in HLH: GAMIFANT (Sobi) remains the only approved therapy for primary HLH (since 2018) and has recently expanded its label, gaining US FDA approval in June 2025 for MAS associated with Still's disease. Following this approval, GAMIFANT has demonstrated stronger-than-projected market uptake, with double-digit growth in Constant Exchange Rates (CER). The company plans to pursue EU regulatory submission in 2026, indicating continued strategic expansion of its HLH and MAS portfolio. Launch of Emerging HLH Drugs: The dynamics of the HLH market are expected to change in the coming years due to the launch of emerging therapies such as plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), tadekinig alfa (r-hIL-18BP) (AB2 Bio), and others. Hemophagocytic Lymphohistiocytosis Market Analysis Management of HLH necessitates a comprehensive, multimodal strategy that integrates immunosuppressive, cytotoxic, and targeted therapies to control hyperinflammation, limit pathological immune activation, and restore immune homeostasis. Therapeutic decisions are guided by disease severity and the underlying trigger, with early, aggressive intervention essential to prevent irreversible organ damage. High-dose corticosteroids, particularly dexamethasone, constitute a foundational component of HLH treatment due to their potent anti-inflammatory effects and ability to suppress cytokine-mediated tissue injury. Etoposide-based regimens remain a cornerstone of first-line therapy, as they effectively eradicate cytotoxic lymphocytes and macrophages that are excessively activated and drive disease pathology. Immunosuppressive agents such as cyclosporine are commonly employed to suppress T-cell activation and reduce cytokine release, thereby dampening immune overactivation. Targeted biologic therapies, including rituximab for EBV-associated HLH and monoclonal antibodies such as alemtuzumab or antithymocyte globulin in refractory disease, enable more selective immune modulation. When infections serve as triggering factors, concurrent antimicrobial therapy, including antivirals and antibiotics, is critical to address both the precipitating cause and the downstream inflammatory cascade. Collectively, these treatment approaches aim to achieve disease stabilization and control, allowing eligible patients to proceed to definitive curative options such as hematopoietic stem cell transplantation (HSCT). GAMIFANT demonstrated strong commercial momentum in Q3 2025, generating USD 66.6 million in revenue, a 98% year-over-year increase. Revenue growth was driven by rising patient adoption, a favorable product mix, and the successful U.S. launch for macrophage activation syndrome (MAS) in Still's disease, making GAMIFANT the first approved therapy for both pediatric and adult patients. This milestone highlights GAMIFANT's broader applicability across the HLH–MAS disease spectrum and reinforces its long-term growth potential. Regulatory momentum remains positive, while active compassionate-use programs in Europe continue to reflect sustained clinician demand. Looking ahead, GAMIFANT revenues are expected to grow at a low double-digit CAGR at constant exchange rates, supported by global market expansion, improved patient access, and continued uptake across both MAS and HLH indications. Hemophagocytic Lymphohistiocytosis Competitive Landscape The pipeline of HLH is very scarce, with only three emerging therapies: plonmarlimab (TJ Biopharma), ELA026 (Electra Therapeutics), MAS825 (Novartis), and tadekinig alfa (r-hIL-18BP) (AB2 Bio). Despite all being in late-stage development, this underscores the scarcity of therapeutic innovation in this space. AB2 Bio's Tadekinig alfa is an innovative recombinant human IL-18 binding protein (IL-18BP) that neutralizes IL-18, a key pro-inflammatory cytokine. Under normal physiological conditions, endogenous IL-18BP is present in excess, maintaining circulating free IL-18 at undetectable levels. In certain autoinflammatory disorders, this regulatory balance is disrupted, leading to elevated systemic free IL-18 and severe pathological hyperinflammation. Tadekinig alfa re-establishes this balance by sequestering excess free IL-18, thereby attenuating inflammatory responses. This mechanism represents a novel and promising therapeutic strategy for autoimmune and inflammatory diseases characterized by elevated systemic IL-18 levels. The therapy is currently being evaluated in a Phase III clinical trial. Electra Therapeutics' ELA026 is a monoclonal antibody developed to treat severe inflammatory diseases driven by dysregulated myeloid cells and T-lymphocytes. It selectively and rapidly depletes the circulating inflammatory myeloid cells and T cells responsible for disease pathology, while preserving the CD47/SIRPα immune checkpoint pathway. ELA026 is presently in Phase II/III clinical development. The anticipated launch of these emerging therapies are poised to transform the hemophagocytic lymphohistiocytosis market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hemophagocytic lymphohistiocytosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about the hemophagocytic lymphohistiocytosis drugs @ Hemophagocytic Lymphohistiocytosis Drugs Market Recent Developments in the Hemophagocytic Lymphohistiocytosis Market In October 2025, Electra Therapeutics announced that the first patients had been dosed in the SURPASS study, a pivotal Phase II/III clinical trial evaluating ELA026, the first investigational therapy designed to broadly treat secondary HLH. In June 2025, Sobi announced that the US FDA approved emapalumab for the treatment of adult and pediatric (newborn and older) patients with HLH/MAS in known or suspected Still's disease, including sJIA, with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. What is Hemophagocytic Lymphohistiocytosis? Hemophagocytic lymphohistiocytosis is a rare, life-threatening immune system disorder characterized by extreme and uncontrolled immune activation. In HLH, immune cells, particularly macrophages and lymphocytes, become overactive and release excessive amounts of inflammatory cytokines, leading to a "cytokine storm" that causes widespread tissue damage and organ failure. The condition can be inherited (primary HLH) due to genetic defects affecting immune regulation, or acquired (secondary HLH) and triggered by infections, malignancies, autoimmune diseases, or other immune challenges. Clinically, HLH often presents with persistent high fever, enlarged liver and spleen, cytopenias, liver dysfunction, and markedly elevated inflammatory markers. Without prompt diagnosis and treatment, HLH can rapidly progress and is frequently fatal. Hemophagocytic Lymphohistiocytosis Epidemiology Segmentation The hemophagocytic lymphohistiocytosis epidemiology section provides insights into the historical and current hemophagocytic lymphohistiocytosis patient pool and forecasted trends for the leading markets. The commonest single disease entities were lymphoma, SLE, and sJIA. There has been evidence of clinically diagnosed HIV, EBV, or CMV infection either in the health record or on the death certificate in 2.3%, 8.9%, and 3.3%, respectively. The hemophagocytic lymphohistiocytosis treatment market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into: Total Incident Cases of HLH Total Incident Cases of HLH by Type Incident Cases of HLH by Gender Incident Cases of Familial HLH by Mutation Incident Cases of Acquired HLH by Etiology Total Treated Cases of HLH Scope of the Hemophagocytic Lymphohistiocytosis Market Report Therapeutic Assessment: Hemophagocytic Lymphohistiocytosis current marketed and emerging therapies Hemophagocytic Lymphohistiocytosis Market Dynamics: Key Market Forecast Assumptions of Emerging Hemophagocytic Lymphohistiocytosis Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Hemophagocytic Lymphohistiocytosis Market Access and Reimbursement Download the report to understand what is the latest research on hemophagocytic lymphohistiocytosis @ New Hemophagocytic Lymphohistiocytosis Medication Table of Contents Related Reports Hemophagocytic Lymphohistiocytosis Clinical Trial Analysis Hemophagocytic Lymphohistiocytosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key HLH companies, including Chia Tai Tianqing Pharmaceutical Group, Bellicum Pharmaceuticals, Atara Biotherapeutics, Incyte Corporation, Alpine Immune Sciences, Expression Therapeutics, among others. Systemic Lupus Erythematosus Market Systemic Lupus Erythematosus Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SLE companies, including Biogen, Novartis, MorphoSys, Idorsia Pharmaceuticals, Viatris, RemeGen, UCB Pharma, Genentech, Bristol Myers Squibb, AbbVie, among others. Systemic Lupus Erythematosus Clinical Trial Analysis Systemic Lupus Erythematosus Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key SLE companies, including Roche, Biogen, Viatris Inc., Novartis, Qilu Pharmaceutical, ImmuPharma, Beijing Mabworks Biotech Co., Ltd., Zenas Biopharma, AbbVie, Beijing InnoCare Pharma Tech Co., Ltd., Alumis Inc., Galapagos NV, Palleon Pharmaceuticals, Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Johnson & Johnson, Merck KGaA, Resolve Therapeutics, ILTOO Pharma, Argenx, Cartesian Therapeutics, Keymed Biosciences Co.Ltd, BioSenic, Eisai, Vertex Pharmaceuticals, Kyverna Therapeutics, Miltenyi Biomedicine, Astrazeneca, Otsuka Pharmaceutical, Fresh Tracks Therapeutics, among others. Macrophage Activation Syndrome Market Macrophage Activation Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MAS companies, including Electra Therapeutics, TJ Biopharma, AB2 Bio, Novartis, Deepcure, Sobi, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo

Drug ApprovalImmunotherapyPhase 3

02 Dec 2025

·www.biopharmadive.com

The ‘clever’ tool increasingly getting bigger biotech deals signed

Not six months ago, bankers and lawyers had significant reservations whether they could get larger biotechnology deals over the finish line.Questions around interest rates, tariff policies and the direction of the Food and Drug Administration had turned an already down biotech market into a turbulent one in which stocks would swing by the double-digits weekly sometimes daily making it even harder for buyers and sellers to agree on price.Facing such turmoil, dealmakers have made greater use out of an old tool known as contingent value rights. And the payoff is clear. Since July, at least two dozen biotech acquisitions worth $50 million or more have been signed, a boost from the first half of this year and a significant acceleration from the back half of 2024. Data compiled by BioPharma Dive show a third of those deals employed CVRs.A CVR is essentially a promise to a sellers shareholders that their payout will get bigger if the companys assets go on to hit certain milestones. In an industry as fraught as biotech, where once-promising drugs can quickly become obsolete from a single study failure or regulatory setback, CVRs act as risk mitigators.Theyre also useful for hammering out price. Buyers get to pay less initially, while sellers get affirmation about the potential worth of their medicines. Its a clever workaround, said Kevin Eisele, managing director at the investment firm William Blair.The growing popularity is illustrated in a mid-October report from Jefferies that examined biotech mergers and acquisitions worth more than half a billion dollars. The report found that not only has the number of buyouts with CVRs increased substantially in recent years, but so, too, has the proportion of the deal tied to these rights. In 2025,CVRs have, on average, accounted for 37% of the total size of those larger transactions that incorporated them, according to Jefferies.In some cases, the CVR offers a payout far higher than a deals upfront cost. Day One Biopharmaceuticals last month agreed to buy struggling cancer drug developer Mersana Therapeutics for $25 per share. But Mersana investors could take home an additional $30.25 per share from a CVR associated with the companys most advanced research program.Even more stark is Eli Lillys bid for gene therapy developer Adverum Biotechnologies, which carries a CVR that could be worth 2.5 times the upfront payment of $3.56 per share.Dennis Ding, an analyst at Jefferies, wrote how this uptick in CVR use is an added positive for biotech dealmaking, namely for companies that one could argue seem expensive despite not yet being totally derisked ahead of clinical or regulatory catalysts.Biotech M&A deals with a CVR announced in the second half of this yearBuyerTargetUpfrontCVRDay One BiopharmaceuticalsMersana Therapeutics$129MUp to $30.25PfizerMetsera$10BUp to $20.65Eli LillyAdverum Biotechnologies$75MUp to $8.91Chugai PharmaceuticalRenalys Pharma$98MUp to 16BAlkermesAvadel Pharmaceuticals$2.1BUp to $1.5Novo NordiskAkero Therapeutics$4.7BUp to $6Roche89Bio$2.4BUp to $6MannKindscPharmaceuticals$303MUp to $1SOURCE: BioPharma Dive dataDealmakers first began using CVRs in the late 1980s. These tools, as lawyers from Sidley Austin explained in a 2023 post to a prominent Harvard Law School forum, are particularly attractive during times of economic uncertainty. Thats been the case in the life sciences over the past four years, as a historic dive in the biotech stock market has left many drug developers cash-strapped and on tenterhooks.Jefferies data reflects this. In both 2015 and 2016, two $500 million-plus deals had CVRs. The following two years saw none. There were five to 10 annually between 2019 and 2022. Then, in 2023, CVRs were in 14 of the 27 deals tallied by Ding and his team.Companies need to raise capital. And in a market where thats challenging, a CVR provides a nice ability to get an upside on [an asset] you have confidence in, said Daniel Rees, a partner at Latham & Watkins who also serves as global vice chair of the firms M&A and Private Equity Practice.It's a well-trodden path, and were seeing it increase, said Latham partner Charles Ruck.Ruck added that, from his experience, there isnt much downside to a CVR. They can be complicated to structure especially if theyre related to research or sales goals, rather than a binary event like an FDA approval but hes never heard of a CVR keeping a deal from happening.They have, however, caused problems after acquisitions complete.In 2019, Sanofi forked over $315 million to former shareholders of Genzyme,a company it acquired for roughly $20 billion,to settle claims that it purposely slowed development of the multiple sclerosis drug Lemtrada to avoid paying a $7-per-share CVR.Two years later, a bank representing former Celgene investors filed a similar lawsuit against Bristol Myers Squibb, alleging the pharmaceutical giant dragged its feet trying to win approval of a cancer cell therapy that was part of a $9-per-share CVR. Had all the components of that CVR been met, Bristol Myers would have had to shell out an additional $6.4 billion.A U.S. district judge dismissed that casethis September, only for the Celgene shareholders to slap Bristol Myers with a renewed, larger lawsuitlast month.Such cases underscore that while CVRs float future payouts, sellers must have confidence that the acquirer will make a good faith attempt to hit the associated goals. Rees noted that, with a sales-based CVR, the identity of who you're selling to becomes even more important. Because you're looking at: can they achieve those sales targets that you may not be as focused on if it's just a regulatory approval.Eisele expects that CVRs, despite being a niche product just several years ago, now look poised to stick around.There's a lot of focus on discipline on both sides of the table, Eisele said. Acquirers want to show they're not overspending on targets, while the targets are under liquidity pressures from existing investors after long periods of underperformance and want to get something out of the investment.CVRs have helped bridge that gap. '

Acquisition

03 Nov 2025

·www.globenewswire.com

Cellectis to Present a Development Update for eti-cel at ASH 2025

Preliminary data recently shared for eti-cel (UCART20x22) show an 86% ORR and a 57% CR rate (n=7), underscoring its potential to improve outcomes in r/r NHLPreclinical data demonstrated that combining eti-cel with low-dose IL-2 may deepen and extend anti-tumor activity in patients with r/r NHLEti-cel full Phase 1 dataset, including low-dose IL-2 combination cohorts, expected to be presented in 2026Correlation between alemtuzumab exposure and response with lasme-cel (UCART22) allows optimization of efficacy without an increase in toxicities NEW YORK, Nov. 03, 2025 (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 the acceptance of two abstracts for poster presentation at the American Society of Hematology (ASH) 2025 annual meeting taking place from December 6 to 9, 2025, in Orlando, FL. First poster – Development update on eti-cel The first poster provides a development update on eti-cel product candidate (UCART20x22), an allogeneic dual CAR-T targeting CD20 and CD22 being developed in Phase 1 of the NATHALI-01 clinical trial, for patients with relapsed/refractory non Hodgkin lymphoma (r/r NHL). In addition, the poster outlines the addition of low dose interleukin-2 (IL-2) to further deepen and extend anti-tumor activity of eti-cel in patients with r/r NHL, supported by compelling preclinical data. Cellectis unveiled preliminary results on eti-cel, which demonstrate an encouraging overall response rate (ORR) of 86% and a complete response (CR) rate of 57% at the current dose level (n=7), with 4 out of 7 patients achieving a complete response. The preliminary high rate of complete responses underscores the potential of this innovative approach to transform outcomes for r/r NHL patients. Cellectis expects to present the full Phase 1 dataset for eti-cel, including low-dose IL-2 combination cohorts, in 2026. “We are excited by the progress and evolution of the eti-cel program with the addition of IL-2, which promises to build on the encouraging preliminary response rates observed in the Phase 1 program,” said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. “We look forward to sharing the full Phase 1 dataset including the IL-2 cohorts expected in 2026.” Poster title: Trial in progress: 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) NATHALI-01 Presenter: Vivian Dai, Senior Director, Clinical Research Scientist at Cellectis Date/Time: December 7, 2025 at 6:00 PM – 8:00 PM ET Room: OCCC – West Halls B3-B4 Second poster – Correlation between alemtuzumab exposure and response with lasme-cel The second poster highlights the correlation between alemtuzumab exposure and depth of response in the difficult-to-treat patients who have received lasme-cel (UCART22) in the course of the Phase 1 of BALLI-01, a clinical trial testing this allogeneic CAR-T product candidate targeting CD22 in relapsed/refractory acute lymphoblastic leukemia (ALL). Additionally, the data identifies a threshold exposure level of alemtuzumab above which achieving a complete response/complete response with incomplete hematologic recovery (CR/CRi) is more likely without any increase in toxicities. “We strongly believe in the critical role of alemtuzumab in optimizing responses in these heavily pretreated patients,” said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. “These data have confirmed this and demonstrated that we could further enhance the high CR/CRi and minimal residual disease (MRD)-negative rates observed in our Phase 1 program. We look forward to starting enrollment in our pivotal Phase 2 program in Q4 2025.” Poster title: Increased alemtuzumab exposure correlates with improved responses in heavily pretreated R/R ALL patients: Analysis of the BALLI-01 trial Presenter: Xenia Naj, Ph.D., Director Translational Sciences at Cellectis Date/Time: December 8, 2025, 6:00 PM - 8:00 PM Room: OCCC - West Halls B3-B4 These abstracts can now be accessed here About Cellectis   Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. The company 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 develop gene therapies in other therapeutic indications. With its in-house manufacturing capabilities, Cellectis is one of the few end-to-end gene editing companies that controls the cell and gene therapy value chain from start to finish.     Cellectis’ headquarters are in Paris, France, with locations in New York and Raleigh, NC. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS). To find out more, visit www.cellectis.com and follow Cellectis on LinkedIn and X. Cautionary Statement   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 “designed to,” “believe,” “could,” “expect,” “expected,” “look forward,” “may,” “promise,” 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, include statements regarding the potential of the Phase 2 BALLI-01 trial to be a registrational phase, the 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 sufficiency of cash to fund operations, the potential benefit of our product candidates and technologies. These forward-looking statements are made in light of information currently available to us and are subject to significant risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F as amended and in our annual financial report (including the management report) for the year ended December 31, 2024 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, which are available on the SEC’s website at www.sec.gov, 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, Chief Financial Officer & Chief Business Officer, investors@cellectis.com Attachment PR_ASH2025_ ENGLISH

Phase 1ImmunotherapyCell TherapyASHClinical Result

100 Deals associated with Alemtuzumab

External Link

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	Japan	Sanofi KK	26 Sep 2014
Multiple Sclerosis, Relapsing-Remitting	European Union	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	Iceland	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	Liechtenstein	Sanofi	12 Sep 2013
Multiple Sclerosis, Relapsing-Remitting	Norway	Sanofi	12 Sep 2013
Chronic Lymphocytic Leukemia	United States	Genzyme Corp.	07 May 2001
Multiple Sclerosis	United States	Genzyme Corp.	07 May 2001

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Multiple sclerosis relapse	Phase 3	United Kingdom	Genzyme Corp.	24 Aug 2007
Acute Myeloid Leukemia	Phase 3	Israel	Bayer AG	01 Jul 2004
Myelodysplastic Syndromes	Phase 3	Israel	Bayer AG	01 Jul 2004
B-Cell Leukemia	Phase 3	United States	Genzyme Corp.	01 Jul 2001
CD22 Positive B-cell Acute Lymphoblastic Leukemia	Phase 2	United States	Cellectis SA	14 Oct 2019
CD22 Positive B-cell Acute Lymphoblastic Leukemia	Phase 2	France	Cellectis SA	14 Oct 2019
Philadelphia positive acute lymphocytic leukaemia	Phase 2	United States	Genzyme Corp.	01 Sep 2009
Recurrent Adult Acute Lymphoblastic Leukemia	Phase 2	United States	Genzyme Corp.	01 Sep 2009
Refractory acute lymphoid leukemia	Phase 2	United States	Genzyme Corp.	01 Sep 2009
Chronic graft-versus-host disease	Phase 2	Canada	Bayer AG	01 Oct 2008

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Hematopoietic stem cell transplantation	98	Alemtuzumab	rsamprjtrw(pojbndrjrz) = pnibmqzigo bncnenxmef (zxxvhzqifc ) View more	Positive	04 Feb 2026
				Post-transplant cyclophosphamide (PTCy)	rsamprjtrw(pojbndrjrz) = vaeiczrvmb bncnenxmef (zxxvhzqifc ) View more
Tandem Meetings ASTCT and CIBMTR2026	Not Applicable	Hematopoietic stem cell transplantation	28	Alemtuzumab	vnfsqowliw(lgmxhyuwtd) = hzlajvjkwx lodwskkhmd (fxgvzaijgs ) View more	Positive	04 Feb 2026
				Post-Transplant Cyclophosphamide (PTCy)	vnfsqowliw(lgmxhyuwtd) = baxkafokup lodwskkhmd (fxgvzaijgs ) View more
ASH2025	Not Applicable	T-Cell Prolymphocytic Leukemia	49	Alemtuzumab (transplanted before 2018)	nfcymkdydb(ycwnfuaszg) = poixyigpiv lgifofjumw (hihxvmwxuw ) View more	Positive	06 Dec 2025
				Alemtuzumab (transplanted in 2018 or later)	nfcymkdydb(ycwnfuaszg) = vyprncfchb lgifofjumw (hihxvmwxuw ) View more
ASH2025	Not Applicable	Hematologic Neoplasms	2,683	conventional GVHD prophylaxis + immunosuppressive agent	zelpcnzmyu(sahisabanw) = ynuecaavtq gozgldmcyl (wfifsjtwzq, 0.95 - 1.31) View more	Positive	06 Dec 2025
				conventional GVHD prophylaxis	-
UKALL14 (ASH2025)	Phase 2	Adult Acute Lymphocytic Leukemia	89	Fludarabine+Melphalan+Alemtuzumab	hcdfdjkhgf(yuumyuiqrp) = jlbjdjolps ctgkdwmrly (phjynhrpwz ) View more	Negative	06 Dec 2025
				Cyclophosphamide+8Gy TBI+Alemtuzumab	hcdfdjkhgf(yuumyuiqrp) = jntpghaqmw ctgkdwmrly (phjynhrpwz ) View more
ASH2025	Not Applicable	Refractory Severe Aplastic Anemia	62	Alemtuzumab-based regimen	yzqzgrinbz(grlxctiwtd) = mulyyssqyd iopkoeoyuw (xvunsmedoi, 1 - 19) View more	Positive	06 Dec 2025
				ATG-based regimen	yzqzgrinbz(grlxctiwtd) = efytxlmssq iopkoeoyuw (xvunsmedoi, 5 - 27) View more
NCT04150497 (BALLI-01, Company_Website) Manual	Phase 1/2	Precursor B-Cell Lymphoblastic Leukemia-Lymphoma \| Refractory B Acute Lymphoblastic Leukemia Third line CD22 Positive	40	UCART22 + Alemtuzumab	zscqskyjbc(idfrdqotom) = jmmxtzixum ssvdvbhosq (kdcynqszqk ) View more	Positive	16 Oct 2025
				UCART22 + Alemtuzumab (Process 1)	zscqskyjbc(idfrdqotom) = hwwqjcbabr ssvdvbhosq (kdcynqszqk ) View more
NCT00920972 \| NCT03128996 (Pubmed \| Transplant Cell Ther)	Phase 2	Refractory Severe Aplastic Anemia	22	Alemtuzumab+fludarabine+melphalan±thiotepa (MSD/unrelated/haploidentical)	qmtexfsnpi(ohhnurlnuk) = Complications before day +180 were primarily transient viral reactivations. Thyroid and ovarian hypofunction were the commonest effects noted at long-term follow-up. grrypvejuh (kfqnxcjpmq )	Positive	01 Sep 2025
NCT02711202 (CTgov)	Not Applicable	Neoplasms	20	Remicade+Tacrolimus+Sirolimus+MabCampath, (Sirolimus)	oaqycbxdwy = mgfvgbbqpu feqicwtxan (ugdtrdizxf, fykakjmlms - vmmljdemii) View more	-	04 Jun 2025
				Remicade+Tacrolimus+MabCampath, (Tacrolimus)	oaqycbxdwy = yttmrmqykj feqicwtxan (ugdtrdizxf, ebssggpnyw - zdokuilqeq) View more
4075 (ASH2024)	Not Applicable	Anemia, Aplastic	20	Fludarabine	cykbndczoz(ieubcdpsfy) = owmbwvbayz twovbtmvwa (fiqqwrlgwr ) View more	Positive	09 Dec 2024
				Cyclophosphamide	cykbndczoz(ieubcdpsfy) = jperievggk twovbtmvwa (fiqqwrlgwr )

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