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Atara Biotherapeutics Presents Positive Preclinical Data on ATA3431, A Next-Generation Allogeneic CD20/CD19-Targeted CAR, at the 65th ASH Annual Meeting

On December 11, 2023 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported preclinical data on ATA3431, a next-generation allogeneic CD20/CD19-dual targeted chimeric antigen receptor (CAR) EBV T-cell therapy candidate (Press release, Atara Biotherapeutics, DEC 11, 2023, View Source [SID1234638409]). Findings support ATA3431 advancement into clinical testing, initially focused on the treatment of B-cell malignancies. The data will be presented in a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place December 9-12, 2023, in San Diego.

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"We’re very excited that ATA3431 is progressing towards an IND submission in 2025 with such a compelling and competitive profile," said Cokey Nguyen, Ph.D., Executive Vice President, Chief Scientific & Technical Officer at Atara. "Our EBV T-cell technology is well validated with over 500 patients treated across our portfolio, and allogeneic EBV T-cell based CD19 targeting is further supported by long-term academic outcomes data with an earlier-generation allogeneic EBV CD19 T-cell construct.1 Building on this robust experience, these preclinical findings provide strong proof-of-concept reinforcing the potential of ATA3431 as a best-in-class approach which we look forward to further evaluating in the clinic."

ATA3431 is an allogeneic, bispecific CAR directed against CD20 and CD19, built on Atara’s EBV T-cell platform that does not require T-cell receptor (TCR) or human leukocyte antigen (HLA) gene editing. The design consists of a tandem CD20-CD19 design, with binders oriented to optimize potency. ATA3431 also incorporates the clinically validated 1XX costimulatory domain that enhances stemness and modulates exhaustion to extend functional persistence.

Compared to an autologous CD20/CD19 CAR-T benchmark, the ATA3431 preclinical data demonstrate potent antitumor activity, long-term persistence, and superior tumor growth inhibition. Data highlights include:

In functional evaluation, ATA3431 showed stable CAR expression with a predominantly CD8+ T-cell distribution. The 1XX signaling domain and optimized manufacturing process that enriches for a less differentiated phenotype yielded a high central memory population compared with autologous CD20/CD19 bispecific CAR-T cells, achieving consistent killing of CD20+ and/or CD19+ tumor cells following repeated in vitro challenges.
ATA3431 demonstrated minimal alloreactivity against HLA mismatched targets due to the inherent ability of EBV T cells to recognize defined viral antigens. The cells also showed HLA-independent activity against CD20+/CD19+ targets in vitro.
ATA3431 mediated highly potent tumor growth inhibition in a lymphoma animal model that correlates with long-term persistence without additional exogenous cytokine support.
ATA3431 showed superior in vivo anti-tumor efficacy, survival, and functional persistence, in both CD19 high- and low-expressing lymphoma models, compared to autologous benchmark CAR-T cells with no observed treatment-related toxicities. This demonstrates ATA3431’s potential to overcome antigen escape, which is hypothesized to be a major cause of treatment resistance or disease relapse with current CD19-targeted CAR-T treatment.
Separately, an academic study presented long-term follow-up data on heavily pre-treated patients infused with an earlier generation off-the-shelf CD19 targeted EBV CAR T-cell construct in B-cell lymphoma and acute lymphoblastic leukemia. Encouraging overall survival of up to three years was observed in 12 patients with relapsed/refractory B-cell malignancies after hematopoietic cell transplant (HCT) treated with 3rd party donor derived allogeneic EBV CD19 CAR T.1

ATA3431 Poster Presentation Details:

Title: ATA3431: Allogeneic CD19/CD20 Bispecific CAR EBV T-cells for the Treatment of B-Cell Malignancies

Presenting Author: Seung Sarah Cha, PhD, Atara Biotherapeutics, Thousand Oaks, CA
Date & Time: Monday, December 11, 2023, 6-8 p.m. PST
Abstract Number: 4800
Poster Session: 703. Cellular Immunotherapies: Basic and Translational: Poster III
Location: San Diego Convention Center, Halls G-H
Next-Generation Allogeneic CAR-T Approach

Atara is focused on applying Epstein-Barr virus (EBV) T-cell biology, featuring experience in over 500 patients treated, and novel chimeric antigen receptor (CAR) technologies to meet the current limitations of autologous and allogeneic CAR therapies head-on by advancing a potential best-in-class CAR-T pipeline in oncology and autoimmune disease. Unlike gene-edited approaches aimed at inactivating T-cell receptor (TCR) function to reduce the risk for graft-vs-host disease, EBV T cells maintain expression of native TCRs that promote in vivo functional persistence while also demonstrating inherently low alloreactivity due to their recognition of defined viral antigens and partial human leukocyte antigen (HLA) matching. A molecular toolkit of clinically-validated technologies—including the 1XX costimulatory domain designed for better cell fitness and less exhaustion while maintaining stemness— offers a differentiated approach to addressing significant unmet need with the next generation CAR T.

Affimed Announces Positive Data for AFM24 in Combination with the PD-L1
Checkpoint Inhibitor Atezolizumab in Heavily Pre-treated EGFR-Wildtype Non-Small Cell Lung Cancer Patients

On December 11, 2023 Affimed N.V. (Nasdaq: AFMD) ("Affimed or the "Company"), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported interim safety and efficacy data on its innate cell engager (ICE) AFM24 from the ongoing AFM24-102 combination study with atezolizumab, an anti-PD-L1 checkpoint inhibitor, in patients with advanced EGFR-expressing solid tumors (Press release, Affimed, DEC 11, 2023, View Source [SID1234638408]). The data update as of December 6th, 2023, includes 15 patients from the EGFR-wildtype NSCLC cohort with a median of 2 prior lines of therapy. Importantly, all patients were pretreated with and ultimately progressed while on PD-[L]1 targeting therapy.

The combination of AFM24 with atezolizumab showed encouraging signals of clinical activity, including 1 unconfirmed CR, 3 PRs (1 confirmed, 2 unconfirmed) and 7 patients exhibiting SD. All eleven patients with a confirmed response, unconfirmed response or stable disease (73%) are continuing treatment, with 4 patients exceeding 3 months of therapy; 2 patients improved from SD at the first scan to PR at the second scan based on RECIST criteria.

"Most patients with advanced NSCLC will need additional treatment after first-line therapy, and currently available options for patients in the 2L+ setting provide only modest response rates and short progression-free survival," said Dr. Andreas Harstrick, Chief Medical Officer at Affimed. "Given the severity of this cancer and the urgent need for new treatments, we are very encouraged by the early safety and efficacy results demonstrated by the combination of AFM24 and atezolizumab in this cohort. We look forward to seeing the data in this cohort mature as well as to sharing data from the EGFR-mutant NSCLC cohort, anticipated in the first half of 2024."

Affimed’s ICE AFM24, in combination with atezolizumab, has the potential to reactivate the innate and consequently the adaptive immune system to recognize and destroy EGFR-positive NSCLC tumors. Considering the low ORR reported on atezolizumab monotherapy in checkpoint inhibitor—relapsing and refractory patients, Affimed believes the clinical activity observed in AFM24-102 is likely due to the synergy of AFM24 with atezolizumab.

AFM24 has demonstrated a positive safety and tolerability profile as both a monotherapy and in combination therapy. The combination with atezolizumab has not led to unexpected toxicity, and the toxicity observed to date is in line with the toxicity profile of the individual agents alone. The majority of patients experienced only mild to moderate treatment-related adverse events.

Affimed also announced that it has discontinued enrollment in AFM24-102 into the gastric cancer cohort and the basket cohort evaluating pancreatic cancer, biliary tract cancer and hepatocellular carcinoma. While clinical activity was observed in both cohorts, neither cohort is likely to achieve response rates that would meet the Company’s efficacy hurdle and the Company’s strategic focus is to advance the NSCLC program as fast as possible.

The conference call will be available via phone and webcast. The live audio webcast of the call will be available in the "Webcasts" section on the "Investors" page of the Affimed website at View Source To access the call by phone, please use link https://register.vevent.com/register/BIb2258d6c5f5a474cad74869a7b7b1bb5, and you will be provided with dial-in details and a pin number.

Note: To avoid delays, we encourage participants to dial into the conference call 15 minutes ahead of the scheduled start time. A replay of the webcast will be accessible at the same link for 30 days following the call.

About the AFM24-102 Phase 1/2a Study

AFM24-102 is a Phase 1/2a open-label, non-randomized, multicenter, dose escalation, and expansion study evaluating AFM24 in combination with atezolizumab in patients with selected EGRF-expressing advanced solid malignancies whose disease has progressed after treatment with previous anticancer therapies (NCT05109442).

The Company also announced data from the phase 1/2 data study of acimtamig in combination with allogeneic NK in relapsed/refractory Hodgkin Lymphoma patients conducted at the University of Texas MD Anderson Cancer Center. The Company will host a call today at 1:30 p.m. PST / 4:30 p.m. EST / 22:30 CET to discuss both the acimtamig and AFM24 clinical data updates and to provide an update on the status of the acimtamig LuminICE-203 study, AFM13-104.

About AFM24

AFM24 is a tetravalent, bispecific innate cell engager (ICE) that activates the innate immune system by binding to CD16A on innate immune cells and EGFR, a protein widely expressed on solid tumors, to kill cancer cells. Generated by Affimed’s fit-for-purpose ROCK platform, AFM24 represents a distinctive mechanism of action that uses EGFR as a docking site to engage innate immune cells for tumor cell killing through antibody-dependent cellular cytotoxicity and antibody-dependent cellular phagocytosis.

In addition to studying AMF24 in combination with the checkpoint inhibitor atezolizumab, Affimed is also evaluating options for a combination of AFM24 with an allogeneic off-the-shelf NK cell product that the Company expects to be well suited for heavily pretreated patient populations.

2seventy bio to Host Investor Call at 8:00am ET on Tuesday, December 12 to Discuss Data from KarMMa-3 and KarMMa-2 Studies of Abecma (idecabtagene vicleucel)

On December 11, 2023 2seventy bio, Inc. (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, reported that the Company will host a conference call on Tuesday, December 12, 2023, at 8:00am ET (Press release, 2seventy bio, DEC 11, 2023, View Source [SID1234638407]). The event will highlight data presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place from December 9-12, 2023, in San Diego.

Participants can access the conference call live via webcast which will be available on the Investors and Media page of the company’s website at View Source A replay of the call will be available on the 2seventy bio website for 30 days following the event.

ASH Presentation Details
Oral Presentation: Idecabtagene Vicleucel (ide-cel) Versus Standard Regimens in Patients (pts) with Triple-Class Exposed (TCE) Relapsed and Refractory Multiple Myeloma (RRMM): Updated Analysis from KarMMa-3
Presenter: Paula Rodriguez-Otero
Date & Time: Monday, December 11, 4:45pm PT

Poster Presentation: Efficacy and Safety of Idecabtagene Vicleucel (ide-cel) in Patients with Clinical High-Risk Newly Diagnosed Multiple Myeloma (MM) with an Inadequate Response to Frontline Autologous Stem Cell Transplantation (ASCT): KarMMa-2 Cohort 2c Extended Follow-up
Presenters: Madhav Dhodapkar; Melissa Alsina
Date & Time: Saturday, December 9, 5:30 – 7:30pm PT

Novartis presents new 48-week results from Phase III APPLY-PNH trial showing sustained efficacy and long-term safety of Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH)

On December 11, 2023 Novartis reported results from the extension period of the pivotal Phase III APPLY-PNH trial of oral monotherapy Fabhalta (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who had residual anemia (hemoglobin <10 g/dL) despite previous anti-C5 therapy (Press release, Novartis, DEC 11, 2023, https://www.novartis.com/news/media-releases/novartis-presents-new-48-week-results-from-phase-iii-apply-pnh-trial-showing-sustained-efficacy-and-long-term-safety-fabhalta-iptacopan-adults-paroxysmal-nocturnal-hemoglobinuria-pnh [SID1234638372]). Continuous Fabhalta treatment (200 mg twice daily) for 48 weeks enabled sustained hemoglobin-level increases to near-normal (12 g/dL or more), blood transfusion avoidance, and reduced patient-reported fatigue in the majority of patients; comparable benefits emerged in those patients switching from anti-C5 therapy to Fabhalta in the extension1. Data will be presented at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (ASH) (Free ASH Whitepaper).

"The new APPLY-PNH data are an expansion of the robust outcomes we saw in the randomized phase and demonstrate that patients with PNH who took Fabhalta experienced meaningful hemoglobin improvement over the longer term – nearly a year," said principal co-investigator Antonio Risitano, M.D., Ph.D., President of the International PNH Interest Group and Head of the Hematology and Hematopoietic Transplant Unit, Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria at the AORN San Giuseppe Moscati, Avellino, Italy. "Additionally, the new data confirm that these benefits may occur within weeks after switching from anti-C5s. The APPLY-PNH findings continue to confirm Fabhalta as a promising therapeutic option for people living with PNH."

Patients completing the 24-week randomized treatment period of APPLY-PNH could elect to enter the extension, continuing Fabhalta (61/62 patients; one patient discontinued due to pregnancy) or switching from anti-C5s to Fabhalta (34/35 patients; one patient discontinued based on investigator decision) through week 481,2.

In the continuous Fabhalta group, outcomes achieved in the randomized period were sustained at 48 weeks: mean hemoglobin level continued to be near-normal (12.2 g/dL), nearly all patients (91.9%) remained free of transfusions (Weeks 2-48), and improvements in patient-reported fatigue were observed (adjusted mean change from baseline: 9.80-point increase in Functional Assessment of Chronic Illness Therapy – Fatigue [FACIT-F] score)1.

In the anti-C5-to-Fabhalta group, similar benefits emerged after switch: mean hemoglobin levels increased to near-normal (from 9.1 g/dL at 24 weeks to 12.1 g/dL at 48 weeks), transfusion avoidance was achieved for almost all patients (94.1%, Weeks 26-48), and improvements in patient-reported fatigue were observed after switching to Fabhalta (adjusted mean change from baseline between Week 48 and Week 24: 10.79-point increase in FACIT-F score)1.

"Coming on the heels of Fabhalta’s recent approval in PNH, these extended data from the APPLY-PNH phase III trial reinforce Fabhalta’s utility as an important new oral monotherapy for people living with PNH," said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. "We are eager to bring this novel treatment to even more people living with rare complement-driven disorders as we pursue several additional indications for Fabhalta."

Fabhalta had a similar safety profile at 48 weeks vs. 24 weeks1,2. Three patients had major adverse vascular events (MAVEs), all considered unrelated to Fabhalta (one serious transient ischemic attack [TIA] occurred in the randomized period and was reported previously)1,2. In the extension, there was one non-serious TIA and one serious portal vein thrombosis (PVT; this patient had a history of PVT and discontinued heparin prior to the MAVE)1. Six patients of 62 receiving continuous Fabhalta for 48 weeks had clinical breakthrough hemolysis (BTH); one patient in the anti-C5-to-Fabhalta extension arm had clinical BTH after switching (compared to six of 35 patients while on anti-C5s prior to switch)1,2. All cases of clinical BTH resolved without changing Fabhalta dosing1. During the 48-week study period, the most frequently reported treatment-emergent adverse events (TEAEs) in the Fabhalta arm were COVID-19 (29.0% of patients), headache (19.4%), and diarrhea (16.1%)1. Throughout the full 48 weeks on Fabhalta, 9.7% of patients experienced any severe TEAE and 14.5% experienced any serious TEAE, none of which was suspected to be related to Fabhalta treatment; there were no serious hemolysis TEAEs on Fabhalta1,2. There were no serious infections caused by N. meningitidis, S. pneumoniae, or H. influenzae and no treatment discontinuations because of TEAEs1,2.

PNH is a rare, chronic, and serious complement-mediated blood disorder, in which a large proportion of patients can remain anemic and some dependent on blood transfusions despite currently available standard of care, anti-C5 treatments7-10.

Full 48-week results from the Phase III APPOINT-PNH trial in treatment-naïve PNH patients will be presented at a congress in 2024.

About APPLY-PNH
APPLY-PNH (NCT04558918) was a Phase III, randomized, multinational, multicenter, active-comparator controlled, open-label trial to evaluate the efficacy and safety of twice-daily, oral Fabhalta monotherapy (200 mg) for the treatment of PNH by assessing if switching to Fabhalta was superior to continuing on anti-C5 therapies (US-approved and non-US-approved eculizumab or ravulizumab) in adult patients presenting with residual anemia (Hb <10 g/dL) despite a stable regimen of anti-C5 treatment in the last six months prior to randomization4,11. The trial enrolled 97 patients who were randomized in an 8:5 ratio to either twice-daily, oral Fabhalta monotherapy, or intravenous anti-C5 therapies (continuing with the same regimen as they were on prior to randomization)4,11.

About paroxysmal nocturnal hemoglobinuria (PNH) 
PNH is a rare, chronic and serious complement-mediated blood disorder7. People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into RBCs, white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system7,8. This leads to intravascular hemolysis (destruction of RBCs within blood vessels) and extravascular hemolysis (destruction of RBCs mostly in the spleen and liver), which cause anemia (low levels of circulating RBCs), thrombosis (formation of blood clots), fatigue and other debilitating symptoms7,8.
 
It is estimated that approximately 10-20 people per million worldwide live with PNH7. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old12,13,14.
 
PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab): despite treatment with anti-C5s, a large proportion of people with PNH remain anemic, and some dependent on blood transfusions7-10,15.

About Fabhalta (iptacopan)
Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway14,16,17. Fabhalta is FDA-approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
 
Discovered at Novartis, Fabhalta is currently in development for a range of complement-mediated diseases including immunoglobulin A nephropathy (IgA nephropathy), C3 glomerulopathy (C3G), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and atypical hemolytic uremic syndrome (aHUS).
 
Based on disease prevalence, unmet needs and data from Phase II studies, Fabhalta has received FDA approval in PNH, FDA Breakthrough Therapy Designation in C3G, orphan drug designations from the FDA and EMA in PNH and C3G, EMA PRIME designation for C3G, and EMA orphan drug designation in IgAN.

Innate Pharma Presents Positive Results From TELLOMAK Phase 2 Study With Lacutamab in Patients With Sézary Syndrome at ASH 2023

On December 10, 2023 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported positive final results from the Phase 2 TELLOMAK study in Sézary Syndrome (SS) (Press release, Innate Pharma, DEC 10, 2023, View Source [SID1234638397]). The results were presented at the ASH (Free ASH Whitepaper) 2023 Annual Meeting, in San Diego, California.

As of May 1, 2023, data cutoff, patients in the Sézary Syndrome cohort (cohort 1, n=56) received a median of 5 prior systemic therapies, including mogamulizumab, and had a median follow-up of 14.4 months.

The data demonstrated that lacutamab showed robust clinical activity and an overall favorable safety profile. The global confirmed objective response rate (ORR) was 37.5% (21/56), including 2 complete responses (CR) and 19 partial responses (PR). Overall response rate (ORR) in the skin was 46.4% (26/56), including 5 CR and 21 PR and ORR in the blood was 48.2% (27/56) with 15 CR and 12 PR. Median progression-free survival was 8.0 months (95% CI 4.7-21.2). In patients who achieved a global response, the median duration of response is 12.3 months (95% CI 5.2-NE).

Best Global Response

N=56

Best Response in Skin

N=56

Best Response in Blood

N=56

Best Response in LN

N=461

Best Response (N, %)

2 (3.6)

5 (8.9)

15 (26.8)

3 (6.5)

19 (33.9)

21 (37.5)

12 (21.4)

6 (13.0)

28 (50.0)

27 (48.2)

24 (42.9)

28 (60.9)

7 (12.5)

3 (5.4)

5 (8.9)

5 (10.9)

4 (8.7)

ORR% [95%CI]

37.5%

[26.0-50.6]

46.4%

[34.0-59.3]

48.2%

[35.7-61.0]

19.6%

[10.7-33.2]

Table 1: Efficacy results in SS patients (n=56)

____________________________________
1 includes patients not involved at baseline who progressed in the LN

"The rapid and durable responses observed in the Phase 2 TELLOMAK trial which enrolled heavily pretreated patients, confirms that treatment with lacutamab achieves clinically meaningful outcomes for patients with Sézary Syndrome after at least two prior systemic therapies," commented Dr. Sonia Quaratino, Chief Medical Officer of Innate Pharma. "Enrollment to TELLOMAK study is completed and long-term follow-up will provide more mature data on the key study endpoints in due course."

Prof. Pierluigi Porcu, Director, Division of Hematologic Malignancies and Hematopoietic Stem Cell Transplantation, Sidney Kimmel Cancer Center, Jefferson Health, Philadelphia, and Principal Investigator in the TELLOMAK study, added: "Sézary Syndrome patients treated with more than two prior systemic therapies including mogamulizumab, represent a high unmet medical need population with poor quality of life. It is promising to see lacutamab achieving remarkable efficacy along with favorable safety in this heavily pre-treated population. We thank the investigators, clinical research coordinators, patients and caregivers involved in the TELLOMAK program."

Innate Pharma will host a virtual KOL event, featuring Prof. Pierluigi Porcu, on lacutamab, highlighting results from ASH (Free ASH Whitepaper) oral presentation on Tuesday, December 12, 2023 at 7:00AM PST (4:00PM CET).

Virtual KOL Event Details

Tuesday, December 12, 2023 at 7:00 AM PST (4:00PM CET)

The live webcast will be available at the following link:

View Source

Participants may also join via telephone using the following registration link:

View Source

This information can also be found on the Investors section of the Innate Pharma website, www.innate-pharma.com. A replay of the webcast will be available on the Company website for 90 days following the event.

About Lacutamab

Lacutamab is a first-in-class anti-KIR3DL2 humanized cytotoxicity-inducing antibody that is currently in clinical trials for treatment of cutaneous T-cell lymphoma (CTCL), an orphan disease, and peripheral T cell lymphoma (PTCL). Rare cutaneous lymphomas of T lymphocytes have a poor prognosis with few efficacious and safe therapeutic options at advanced stages.

KIR3DL2 is an inhibitory receptor of the KIR family, expressed by approximately 65% of patients across all CTCL subtypes and expressed by up 90% of patients with certain aggressive CTCL subtypes, in particular, Sézary syndrome. It is expressed by up to 50% of patients with mycosis fungoides and peripheral T-cell lymphoma (PTCL). It has a restricted expression on normal tissues.

Lacutamab is granted European Medicines Agency (EMA) PRIME designation and US Food and Drug Administration (FDA) granted Fast Track designation for the treatment of patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies. Lacutamab is granted orphan drug status in the European Union and in the United States for the treatment of CTCL.

About TELLOMAK

TELLOMAK (NCT03902184) is a global, open-label, multi-cohort Phase 2 clinical trial recruiting patients with Sézary syndrome and mycosis fungoides (MF) in the United States and Europe. Specifically:

Cohort 1: lacutamab being evaluated as a single agent in approximately 60 patients with Sézary syndrome who have received at least two prior systemic therapies, including mogamulizumab. The Sézary syndrome cohort of the study could enable the registration of lacutamab in this indication.
Cohort 2: lacutamab being evaluated as a single agent in patients with MF that express KIR3DL2, as determined at baseline with a Simon 2-stage design.
Cohort 3: lacutamab being evaluated as a single agent in patients with MF that do not express KIR3DL2, as determined at baseline, with a Simon-2 stage design.
All comers: lacutamab being evaluated as a single agent in patients with both KIR3DL2 expressing and non-expressing MF to explore the correlation between the level of KIR3DL2 expression and treatment outcomes utilizing a formalin-fixed paraffin embedded (FFPE) assay under development as a companion diagnostic.
The trial is now fully enrolled. The primary endpoint of the trial is objective global response rate. Key secondary endpoints are progression-free survival, duration of response, overall survival, quality of life, pharmacokinetics and immunogenicity and adverse events.