Last update 12 Dec 2024

Nelarabine

Last update 12 Dec 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Arranon, Atriance, 506-U78

+ [19]

Target

POLA1

Mechanism

POLA1 inhibitors(DNA polymerase alpha subunit inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesHemic and Lymphatic Diseases+ [1]

Active Indication

T-cell Acute Lymphoblastic Leukemia/LymphomaPrecursor T-Cell Lymphoblastic Leukemia-LymphomaRecurrent T Lymphoblastic Lymphoma+ [3]

Inactive Indication

Adult T-Cell Leukemia-LymphomaAnaplastic Large-Cell LymphomaCD30-Positive recurrent or refractory Cutaneous T-Cell Lymphoma+ [16]

Originator Organization

GSK Plc

Active Organization

National Cancer Institute

Reach Pharmaceuticals Pty Ltd.Startup

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

+ [4]

Inactive Organization

GSK Plc

Novartis AG

National Institutes of Health

Drug Highest PhaseApproved

First Approval Date

US (28 Oct 2005),

Precursor T-Cell Lymphoblastic Leukemia-Lymphoma

RegulationPriority Review (US), Accelerated Approval (US), Orphan Drug (AU), Orphan Drug (US)

Structure

Molecular FormulaC11H15N5O5

InChIKeyIXOXBSCIXZEQEQ-UHTZMRCNSA-N

CAS Registry121032-29-9

Clinical Trials associated with Nelarabine

NCT06390319 / Not yet recruitingPhase 2IIT

SJALL23T: Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)

This is a clinical trial testing whether the addition of one of two chemotherapy agents, dasatinib or venetoclax, can improve outcomes for children and young adults with newly diagnosed T-cell acute lymphoblastic leukemia and lymphoma or mixed phenotype acute leukemia.
Primary Objective
* To evaluate if the end of induction MRD-negative rate is higher in patients with T-ALL treated with dasatinib compared to similar patients treated with 4-drug induction on AALL1231.
* To evaluate if the end of induction MRD-negative rate is higher in patients with ETP or near-ETP ALL treated with venetoclax compared to similar patients treated with 4-drug induction on AALL1231.
Secondary Objectives
* To assess the event free and overall survival of patients treated with this therapy.
* To compare grade 4 toxicities, event-free survival (EFS) and overall survival (OS) of patients treated with this therapy in induction and reinduction to toxicities of similar patients treated on TOT17.

Start Date01 Dec 2024

Sponsor / Collaborator

St. Jude Children's Research Hospital, Inc.

[+1]

NCT06210750 / WithdrawnPhase 2IIT

A Randomized Phase 2 Trial to Evaluate the Efficacy of BCL-2 Inhibitor Therapy With Chemotherapy Compared to Chemotherapy Alone in Adult Patients With Newly Diagnosed T-Cell ALL and T-Cell Lymphoblastic Lymphoma

This phase II trial tests the safety and effectiveness of giving chemotherapy with or without venetoclax and/or navitoclax for the treatment of patients with newly diagnosed T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic lymphoma (T-LBL). Chemotherapy drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax and navitoclax are in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. They may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving chemotherapy with or without venetoclax and/or navitoclax may be effective treatments for patients with newly diagnosed T-ALL or T-LBL.

Start Date09 Aug 2024

Sponsor / Collaborator

National Cancer Institute

CTR20240655 / RecruitingPhase 3

奈拉滨注射液治疗儿童和成人难治或复发的T淋巴母细胞白血病（T-ALL）和T淋巴母细胞淋巴瘤（T-LBL）患者的有效性及安全性研究

[Translation] Efficacy and safety of nelarabine injection in the treatment of refractory or relapsed T-lymphoblastic leukemia (T-ALL) and T-lymphoblastic lymphoma (T-LBL) in children and adults

评估儿童和成人难治或复发的T-ALL和T-LBL患者，接受奈拉滨注射液治疗后IRC评估的2周期内的复合完全缓解率（CCR）。

[Translation]

To evaluate the composite complete response rate (CCR) within 2 cycles of IRC assessment in pediatric and adult patients with refractory or relapsed T-ALL and T-LBL who received nelarabine injection.

Start Date07 May 2024

Sponsor / Collaborator

Nanjing Shunxin Pharmaceutical Co., Ltd.

100 Clinical Results associated with Nelarabine

100 Translational Medicine associated with Nelarabine

100 Patents (Medical) associated with Nelarabine

252

Literatures (Medical) associated with Nelarabine

01 Jan 2025·PEDIATRIC BLOOD & CANCER

Cost–utility of nelarabine for the first‐line treatment of newly diagnosed pediatric T‐cell acute lymphoblastic leukemia in Canada

Article

Author: Gibson, Paul ; Shoukry, Roaa ; Sala, Mandy ; Bond, Elizabeth ; Moskalewicz, Alexandra ; Pechlivanoglou, Petros ; Gupta, Sumit ; Bradley, Nicole

Abstract:

Background:

The Children's Oncology Group (COG)‐AALL0434 trial investigated the addition of nelarabine to the augmented Berlin–Frankfurt–Münster (aBFM) protocol in patients (1.0–30.99 years) with newly diagnosed T‐cell acute lymphoblastic leukemia (T‐ALL). Despite demonstrating superior outcomes, nelarabine is not currently funded by many health systems, in part due to a lack of cost‐effectiveness data. We estimated the cost–utility of nelarabine for this indication from a Canadian public healthcare payer perspective.

Methods:

We developed a microsimulation model that followed hypothetical patients with newly diagnosed T‐ALL from post‐induction therapy to death. Three health states were modeled: relapse‐free, post‐relapse, and death. Efficacy was estimated using AALL0434 and retrospective data from Ontario, Canada. Costs were obtained from Canadian sources. Utility estimates and long‐term mortality risks were sourced from literature. Total healthcare costs, quality‐adjusted life‐years (QALYs), and incremental cost‐effectiveness ratio (ICER) were reported. Probabilistic and scenario analyses were conducted.

Results:

Incorporating nelarabine in the aBFM protocol increased costs by $51,670 Canadian dollars per patient, but resulted in 1.97 more QALYs and an ICER of $26,184/QALY. Most of the identified cost and benefit were accrued within the AALL0434 trial period (first 11 years post diagnosis) and while patients were in the relapse‐free health state. Across multiple scenarios, the ICER was stable under an assumed $50,000/QALY threshold.

Conclusion:

Incorporating nelarabine into aBFM was cost‐effective across different scenarios and assumptions. These results support its funding by public and private payers.

01 Dec 2024·LEUKEMIA

Longitudinal follow up of a phase 2 trial of venetoclax added to hyper-CVAD, nelarabine and pegylated asparaginase in patients with T-cell acute lymphoblastic leukemia and lymphoma

Article

Author: Wierda, William ; Issa, Ghayas ; Alvarado, Yesid ; Ferrajoli, Alessandra ; Maiti, Abhishek ; Jabbour, Elias ; Garcia-Manero, Guillermo ; Konopleva, Marina ; Kantarjian, Hagop M. ; Kadia, Tapan ; Kebriaei, Partow ; Short, Nicholas J ; Ravandi, Farhad ; Borthakur, Gautam ; Kantarjian, Hagop M ; Huang, Xuelin ; Garris, Rebecca ; Short, Nicholas J. ; Balkin, Hayley ; Jain, Nitin ; Senapati, Jayastu

Optimal frontline use of active agents in T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) is prudent to improve outcomes. We report the long-term follow-up of the phase 2 trial of HyperCVAD with nelarabine and pegylated asparaginase (Original cohort). In the latest protocol iteration venetoclax was added to the induction/consolidation regimen (Venetoclax cohort). Eligible patients were adults with untreated T-ALL/LBL or after minimal therapy and with adequate organ function. Primary endpoint of this analysis was improvement in 2-year progression free survival (PFS) and overall survival (OS) with venetoclax. From Aug 2007 to Dec 2024, 145 patients, at a median age of 35.4 years, were treated; 46 (33.8%) were in the venetoclax cohort. At median follow-up (mFU) of 62.4 months, 5-year PFS, duration of response (DOR), and OS were 63.7%, 72.0% and 66.2% respectively. In the venetoclax cohort (mFU 24.4 months) 2-year PFS (87.9% versus 64.1%, p = 0.03) and 2-year DOR (93.6% versus 69.2%, p = 0.005) were superior to the original cohort (mFU 89.4 months) and 2-year OS appeared better (87.8% versus 73.9%, p = 0.16). Febrile neutropenia was the most common serious adverse event, seen in 60% patients. The addition of venetoclax to HyperCVAD-nelarabine-pegylated asparaginase was tolerable and led to improvement in DOR and PFS.

01 Nov 2024·Clinical Lymphoma Myeloma & Leukemia

Incidence and Clinical Presentation of Severe Neurotoxicity from Nelarabine in Patients with T-Cell Acute Lymphoblastic Leukemia

Article

Author: Jain, Nitin ; Nicholas, Short ; Ravandi, Farhad ; Jabbour, Elias ; Sasaki, Koji ; Woodman, Karin ; Kantarjian, Hagop ; Kugler, Eitan ; Braish, Julie S ; Braish, Julie S.

BACKGROUND:

Nelarabine is a purine analog with demonstrated efficacy in the treatment of T-cell Lymphoblastic Leukemia and Lymphoma (T-ALL/LBL). Despite its efficacy and excellent blood-brain barrier penetration, it has a significant side effect profile which is namely concerning for neurotoxicity. Reported neurotoxicity has varied from mild peripheral neuropathy to debilitating grade 4 neurologic complications including Guillain-Barre like syndrome and myelopathy.

PATIENTS AND METHODS:

We conducted a single centered, retrospective case series to study patients who developed severe neurotoxicity after receiving nelarabine as part of T-ALL treatment. One hundred thirty-five patients were identified. Thirteen patients were reviewed for severe neurotoxicity (defined as ≥grade 3), and of those five patients were deemed to have neurotoxicity secondary to nelarabine exposure.

RESULTS:

Five patients (4%) developed severe neurotoxicity as manifested by Guillain-Barre like syndrome or myelopathy within a timeframe of eight to fifty-eight days from last nelarabine dose. Upon diagnosis, patients received formal neurologic evaluation by our neuro-oncology specialists including imaging, cerebrospinal fluid testing, and electromyography. Patients were treated with IVIG, and steroids upon diagnosis, but the majority of neuro-deficits were irreversible.

CONCLUSION:

Our study shows that nelarabine is generally well-tolerated, and the incidence of severe neurotoxicity is rare. Given the potential risk of severe neurotoxicity, we propose capped dose of nelarabine 1000 mg/day, neurological assessment before subsequent dosing, and avoidance of simultaneous IT therapy during nelarabine administration.

News (Medical) associated with Nelarabine

28 Jun 2024

·www.biospace.com

Shorla Oncology Announces FDA Approval for TEPYLUTE, A Novel Formulation to Treat Breast and Ovarian Cancer

TEPYLUTE is a ready-to-dilute formulation of thiotepa that reduces preparation time and enables accurate dosing CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has approved the company’s New Drug Application (NDA) for TEPYLUTE, a ready-to-dilute formulation to treat breast and ovarian cancer in an easier to prepare, injectable product that enables dosing accuracy.1 ‘’This approval fulfills an unmet need by addressing the shortcomings and handling complexities of the current lyophilized powder formulation,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “We have taken a vital oncology drug and made it easier for oncology clinics and hospitals to use, while also reducing medical personnel exposure to a hazardous drug.” TEPYLUTE, formerly SH-105, is the third FDA-approved drug for Shorla, and a significant milestone for the company as it seeks approval for several cancer-fighting drugs for the U.S. market. “The approval of TEPYLUTE represents an important milestone for Shorla as our first in-house developed NDA,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology. TEPYLUTE is a liquid form of a well-established, standard of care oncology drug, thiotepa. The new formulation eliminates the need for complex and time-consuming reconstitution. It provides consistent dosing accuracy and allows for “just in time” preparation.2 “Among TEPYLUTE’s many benefits, it removes the necessity to reconstitute which can introduce additional risks of drug preparation errors,” emphasized Rayna Herman, Chief Commercial Officer. “We look forward to providing an update on our launch plans for TEPYLUTE in the near future.” The American Cancer Society estimates that more than 300,000 women will be diagnosed with breast cancer in the U.S in 2024.3 About 19,680 women will be diagnosed with ovarian cancer in the United States.4 Shorla Oncology is currently marketing two products with a robust pipeline including SH-201, the first palatable oral liquid treatment for certain forms of leukemia and other cancers. In April, the company announced the FDA had accepted SH-201 for an NDA review with an expected action date of November 30, 2024. About Shorla Oncology Shorla Oncology is a privately- held, U.S. and Ireland- based commercial stage specialty pharmaceutical company established by Sharon Cunningham and Orlaith Ryan. The company has an advanced pipeline of innovative oncology drugs for orphan and pediatric cancers. Shorla is focused on indications where existing treatments are limited, in shortage or the drug applications are inadequate for the target population. The company’s growing portfolio brings accessible, affordable and life-saving treatments to patients, delivering a major contribution to patient care. Shorla currently markets two products, Nelarabine for the treatment of T-cell Leukemia and JYLAMVO for the treatment of acute lymphoblastic leukemia and other indications. For further information, please visit ________________________ 1 Carausu M, Carton M, Darlix A, Pasquier D, Leheurteur M, Debled M, Mouret-Reynier MA, Goncalves A, Dalenc F, Verret B, Campone M. Breast cancer patients treated with intrathecal therapy for leptomeningeal metastases in a large real-life database. ESMO Open 6: 100150. 1 Reinhardt H, Otte P, Eggleton AG, Ruch M, Wöhrl S, Ajayi S, Duyster J, Jung M, Hug MJ, Engelhardt M. Avoiding chemotherapy prescribing errors: analysis and innovative strategies. Cancer. 2019 May 1;125(9):1547-57. 2 TEPYLUTE Prescribing information and data on Shorla Oncology. 3 Breast Cancer Statistics | How Common Is Breast Cancer? | American Cancer Society, Accessed June 27, 2024 4 Ovarian Cancer Statistics | How Common is Ovarian Cancer | American Cancer Society, Accessed June 27, 2024

Drug ApprovalNDA

14 Jun 2024

·www.globenewswire.com

Phase 2 Findings Show Wugen’s Investigational Allogeneic CAR-T, WU-CART-007, Was Highly Effective and Surpassed Standard of Care in Treating Hard-to-Treat T-ALL/LBL; Data Presented Today at European Hematology Association (EHA) 2024 Hybrid Congress

-- Researchers Shared Findings Including a Clinically Manageable Safety Profile and Anti-leukemic Activity with WU-CART-007 in Patients with R/R T-ALL/LBL and Previously Treated with Existing Therapies -- U.S. Biotech Wugen to Initiate Follow-Up Study Exploring Its Anti-CD7, Off-the-Shelf, Allogeneic CAR-T Cell Therapy in Pediatric and Minimal Residual Disease (MRD) Patients in Q424 -- Company Presented Additional New Data at EHA on WU-CART-007 and WU-NK-101, Its Off-the-Shelf Investigational Memory Natural Killer Cell Therapy ST. LOUIS and SAN DIEGO, June 14, 2024 (GLOBE NEWSWIRE) -- Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, announces positive Phase 2 results for the company’s investigational anti-CD7 CAR-T therapy, WU-CART-007 (“W-T7”), in patients with relapsed/refractory (R/R) t-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL). In his presentation today at the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, lead investigator Ibrahim Aldoss, M.D., City of Hope in Duarte, CA, shared data from this Phase 2 cohort expansion study showing a clinically manageable safety profile as well as evidence of anti-leukemic activity with WU-CART-007 (“W-T7”) in patients with R/R T-ALL/LBL previously treated with existing therapies for their cancers. “Relapsed and refractory T-ALL/LBL is a highly aggressive disease with high rates of relapse and mortality in both children and adults,” Dr. Aldoss said. “These results are exciting and support the potential of this allogeneic CAR-T as a new treatment for patients who still face a refractory illness despite having received the best medicines we have for T-ALL/LBL right now.” “The need for effective, allogeneic treatments that are available at the point of care without delays is urgent for patients with hard-to-treat blood cancers,” said Wugen Chief Medical Officer Jan Davidson-Moncada, M.D., Ph.D. “Wugen is confident we can overcome specific challenges that have existed for CAR-T therapies in targeting T-cell diseases. We are eager to conduct further studies of WU-CART-007, which was invented by Wugen’s Co-founder and Chief Scientific Officer Matt Cooper, Ph.D., to target CD7+ malignancies.” Key Phase 2 Findings The presentation at EHA featured results with the recommended dose (RP2D) of WU-CART-007 administered in 13 patients at a median age 30 years who were heavily pre-treated and had a high burden of disease at the start of the study. A substantial proportion of patients (38%) had progressed or relapsed after allogeneic stem cell transplant. Outcomes following WU-CART-007 treatment in the study surpassed current standard of care1 for R/R T-ALL/LBL and included: Composite complete remission rate (CRc) of 73% (8/11) (primary endpoint).Overall response rate of 91% (10/11; 2 partial response (PR) in patients with extramedullary disease, R/R T-LBL).Duration of response (DOR) (secondary endpoint): Median duration (mDOR) was 6.2 months (95% CI: 1.8, NE);Median follow up was 8.5 months (95% CI: 2.7, NE); 46% (5/11) patients remained in continuous remission at 4.3 to 8.6 months. Seven patients (5 at RP2D) successfully transplanted. Pharmacokinetic analysis exhibited rapid expansion and persistence of an allogeneic cell therapy: At RP2D, WU-CART-007 expansion peaked at Day 10 (223,799 copies/g of DNA) and can be detected out to Day 90. No patient developed drug product-specific anti-HLA antibodies.No anti-drug antibody detected on any patients to date. Phase 1 dose escalation data from this first-in-human global Phase 1/2 clinical trial of WU-CART-007 for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL) were presented last December at the American Society of Hematology meeting. See those findings here. Additional Wugen Data at EHA Researchers presented other new findings with WU-CART-007 and WU-NK 101 in these posters at EHA: Title: Preliminary Effect of Enhanced Lymphodepletion (ELD) on WU-CART-007 in T-ALL/LBLPresenters: Ouiam Bakkacha M.D. and Ben Capoccia Ph.D., Wugen, St. Louis, MOAbstract: P408 Title: WU-NK-101 is an Off-The-Shelf Memory NK Cell with a Time-Resilient, Anti-Tumor Phenotype that can be Detected in R/R AML Patient PBMC After InfusionPresenter: Laura Arthur, Ph.D., Wugen, St. Louis, MOAbstract: P1422 More information is available on the EHA 2024 Hybrid Congress website. About WU-CART-007WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration and received PRIME (“Priority Medicines”) designation in the European Union for the treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need. About WU-NK-101WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This cell population has a superior phenotype, proliferation capacity, and metabolic fitness, making it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML). Wugen is planning to initiate solid tumor studies of WU-NK-101 in combination with cetuximab. Studies of WU-NK-101 to date have shown promising robust in vivo activity in various tumor indications, retention of anti-cancer activity in TME, resistance to immune suppression, and enhanced activity with checkpoint inhibitors. WU-NK-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. About Wugen A spin-out of Washington University in St. Louis, Wugen, Inc. is a clinical-stage U.S. biotechnology company developing the next generation of off-the-shelf CAR-T and memory natural killer (NK) cell therapies for cancer. For more information, please visit www.wugen.com. Investor Contact:Mark LewisWugenMlewis@wugen.com+1 314-501-1968 Media Contact:Christine FanelleScient PRchristine@scientpr.com+1 215-595-5211 1 Nelarabine USPI

Clinical ResultCell TherapyImmunotherapyOrphan DrugASH

14 Jun 2024

·www.biospace.com

Researchers Shared Findings Including a Clinically Manageable Safety Pro Anti-leukemic Activity with WU-CART-007 in Patients with R/R T-ALL/LBL and Previously Treated with Existing Therapies U.S. Biotech Wugen to Initiate Follow-Up Study Exploring Its Anti-CD7, Off-the-Shelf, Allogeneic CAR-T Cell Therapy in Pediatric and Minimal Residual Disease (MRD) Patients in Q424 Company Presented Additional New Data at EHA on WU-CART-007 and WU-NK-101, Its Off-the-Shelf Investigational Memory Natural Killer Cell Therapy ST. LOUIS and SAN DIEGO, June 14, 2024 (GLOBE NEWSWIRE) -- Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, announces positive Phase 2 results for the company’s investigational anti-CD7 CAR-T therapy, WU-CART-007 (“W-T7”), in patients with relapsed/refractory (R/R) t-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL). In his presentation today at the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, lead investigator Ibrahim Aldoss, M.D., City of Hope in Duarte, CA, shared data from this Phase 2 cohort expansion study showing a clinically manageable safety pro well as evidence of anti-leukemic activity with WU-CART-007 (“W-T7”) in patients with R/R T-ALL/LBL previously treated with existing therapies for their cancers. “Relapsed and refractory T-ALL/LBL is a highly aggressive disease with high rates of relapse and mortality in both children and adults,” Dr. Aldoss said. “These results are exciting and support the potential of this allogeneic CAR-T as a new treatment for patients who still face a refractory illness despite having received the best medicines we have for T-ALL/LBL right now.” “The need for effective, allogeneic treatments that are available at the point of care without delays is urgent for patients with hard-to-treat blood cancers,” said Wugen Chief Medical Officer Jan Davidson-Moncada, M.D., Ph.D. “Wugen is confident we can overcome specific challenges that have existed for CAR-T therapies in targeting T-cell diseases. We are eager to conduct further studies of WU-CART-007, which was invented by Wugen’s Co-founder and Chief Scientific Officer Matt Cooper, Ph.D., to target CD7+ malignancies.” Key Phase 2 Findings The presentation at EHA featured results with the recommended dose (RP2D) of WU-CART-007 administered in 13 patients at a median age 30 years who were heavily pre-treated and had a high burden of disease at the start of the study. A substantial proportion of patients (38%) had progressed or relapsed after allogeneic stem cell transplant. Outcomes following WU-CART-007 treatment in the study surpassed current standard of care1 for R/R T-ALL/LBL and included: Composite complete remission rate (CRc) of 73% (8/11) (primary endpoint). Overall response rate of 91% (10/11; 2 partial response (PR) in patients with extramedullary disease, R/R T-LBL). Duration of response (DOR) (secondary endpoint): Median duration (mDOR) was 6.2 months (95% CI: 1.8, NE); Median follow up was 8.5 months (95% CI: 2.7, NE); 46% (5/11) patients remained in continuous remission at 4.3 to 8.6 months. Seven patients (5 at RP2D) successfully transplanted. Pharmacokinetic analysis exhibited rapid expansion and persistence of an allogeneic cell therapy: At RP2D, WU-CART-007 expansion peaked at Day 10 (223,799 copies/g of DNA) and can be detected out to Day 90. No patient developed drug product-specific anti-HLA antibodies. No anti-drug antibody detected on any patients to date. Phase 1 dose escalation data from this first-in-human global Phase 1/2 clinical trial of WU-CART-007 for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL) were presented last December at the American Society of Hematology meeting. See those findings here. Additional Wugen Data at EHA Researchers presented other new findings with WU-CART-007 and WU-NK 101 in these posters at EHA: Title: Preliminary Effect of Enhanced Lymphodepletion (ELD) on WU-CART-007 in T-ALL/LBL Presenters: Ouiam Bakkacha M.D. and Ben Capoccia Ph.D., Wugen, St. Louis, MO Abstract: P408 Title:WU-NK-101 is an Off-The-Shelf Memory NK Cell with a Time-Resilient, Anti-Tumor Phenotype that can be Detected in R/R AML Patient PBMC After Infusion Presenter: Laura Arthur, Ph.D., Wugen, St. Louis, MO Abstract: P1422 More information is available on the EHA 2024 Hybrid Congress website. About WU-CART-007 WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T-cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T-cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). Additional information is available on clinicaltrials.gov, identifier NCT# 04984356. WU-CART-007 has received Orphan Drug, Fast Track, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration and received PRIME (“Priority Medicines”) designation in the European Union for the treatment of relapsed/refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need. About WU-NK-101 WU-NK-101 is a novel immunotherapy harnessing the power of memory natural killer (NK) cells to treat liquid and solid tumors. Memory NK cells are hyper-functional, long-lasting immune cells that exhibit enhanced anti-tumor activity and a cytokine-induced memory-like (CIML) phenotype. This cell population has a superior phenotype, proliferation capacity, and metabolic fitness, making it better suited for cancer therapy than other NK cell therapies. Wugen is applying its proprietary MonetaTM platform to advance WU-NK-101 as a commercially scalable, off-the-shelf cell therapy for cancer. WU-NK-101 is currently in development for acute myelogenous leukemia (AML). Wugen is planning to initiate solid tumor studies of WU-NK-101 in combination with cetuximab. Studies of WU-NK-101 to date have shown promising robust in vivo activity in various tumor indications, retention of anti-cancer activity in TME, resistance to immune suppression, and enhanced activity with checkpoint inhibitors. WU-NK-101 has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of AML. About Wugen A spin-out of Washington University in St. Louis, Wugen, Inc. is a clinical-stage U.S. biotechnology company developing the next generation of off-the-shelf CAR-T and memory natural killer (NK) cell therapies for cancer. For more information, please visit . Investor Contact: Mark Lewis Wugen Mlewis@wugen.com +1 314-501-1968 Media Contact: Christine Fanelle Scient PR christine@scientpr.com +1 215-595-5211 1 Nelarabine USPI

Clinical ResultImmunotherapyFast TrackCell TherapyOrphan Drug

100 Deals associated with Nelarabine

External Link

KEGG	Wiki	ATC	Drug Bank
D05134	Nelarabine	L01BB07	DB01280

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
T-cell Acute Lymphoblastic Leukemia/Lymphoma	JP	Sandoz Pharmaceuticals AG	19 Oct 2007
Precursor T-Cell Lymphoblastic Leukemia-Lymphoma	US	Sandoz, Inc.	28 Oct 2005

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Recurrent T Lymphoblastic Lymphoma	Phase 3	CN	Chia Tai Tianqing Pharmaceutical Group Co., Ltd.	01 May 2024
Refractory T Acute Lymphoblastic Leukemia	Phase 3	CN	Chia Tai Tianqing Pharmaceutical Group Co., Ltd.	01 May 2024
T-cell acute lymphoblastic leukemia in relapse	Phase 2	US	Novartis AG	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	US	GSK Plc	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	AU	GSK Plc	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	AU	Novartis AG	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	AT	GSK Plc	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	AT	Novartis AG	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	CA	Novartis AG	01 Jun 2010
T-cell acute lymphoblastic leukemia in relapse	Phase 2	CA	GSK Plc	01 Jun 2010

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2023	Not Applicable	Precursor T-Cell Lymphoblastic Leukemia-Lymphoma	133	Nelarabine (NEL)	saxybuxfjq(ermmyhtdsm) = vztvqxvpgy uesuawquyb (xsniibwpju )	-	11 Dec 2023
ASH2023	Not Applicable	Precursor T-Cell Lymphoblastic Leukemia-Lymphoma	133	Pegylated Asparginase (PEG)	saxybuxfjq(ermmyhtdsm) = dnfyjixmhm uesuawquyb (xsniibwpju )	-	11 Dec 2023
GRAALL-2014/T (ASH2023)	Phase 2	Precursor T-Cell Lymphoblastic Leukemia-Lymphoma Consolidation	325	Nelarabine	qjwmgmcjbq(eafazngevf) = cvqovggkga ulzxevhetc (howbznkdqz, 20 - 37) View more	-	11 Dec 2023
NCT02763384 (CTgov)	Phase 2	Refractory T Acute Lymphoblastic Leukemia \| Adult Lymphoblastic Lymphoma	12	BL-8040+Nelarabine	wequcyjzia(ppkoilksza) = eyhvzmnebn inuezrqtpw (fmlpqqlvth, ostkenrgmo - bkrdgrhmyx) View more	-	31 Oct 2023
Pubmed	Phase 1	Neoplasms	-	Nelarabine	xytmxdgfhg(gnlcxfzpwe) = niiwkdnocq jqskyzmlqp (snjfqdcwru ) View more	-	29 Nov 2022
EHA2022	Not Applicable	T-cell Acute Lymphoblastic Leukemia/Lymphoma	120	HyperCVAD + Nelarabine	qfpwpnqoda(yxynyzbppg) = elevated liver enzymes in 25 pts (21%) aggibbkloq (tbptsmlyxm ) View more	-	12 May 2022
EHA2022	Not Applicable	T-cell Acute Lymphoblastic Leukemia/Lymphoma	120	HyperCVAD + Nelarabine + PEGylated Asparaginase		-	12 May 2022
NCT00981799 (CTgov)	Phase 1/2	Recurrent T Lymphoblastic Lymphoma \| T-cell acute lymphoblastic leukemia in relapse	23	Etoposide+cyclophosphamide+Filgrastim+Nelarabine+Methotrexate (Nelarabine Dose Level 1)	jvgfnomlll(ldzeqvgcci) = mdaxkkxjwh mqpkefhwsm (acyropxtes, pfvzslqpkz - zrvpuloyuj) View more	-	01 Oct 2020
NCT00981799 (CTgov)	Phase 1/2		23	Etoposide+cyclophosphamide+Filgrastim+Nelarabine+Methotrexate (Nelarabine Dose Level 2)	jvgfnomlll(ldzeqvgcci) = zrpaobtklr mqpkefhwsm (acyropxtes, mylwyxijey - yispceergi) View more	-	01 Oct 2020
EHA2019	Phase 4	T-cell Acute Lymphoblastic Leukemia/Lymphoma	118	Nelarabine	iwjqupkpdm(bvuhqtrjga) = yuyxstfidd nireiqandj (aitmspxtzr ) View more	-	16 Jun 2019
EHA2019	Not Applicable	Precursor T-Cell Lymphoblastic Leukemia-Lymphoma	9	Nelarabine	hlaxonrnlg(ztneekbowx) = ahaxmdhccj shjmzhhaun (zcapnfhebd ) View more	-	16 May 2019
EHA2015	Not Applicable	Refractory T Acute Lymphoblastic Leukemia	506	Nelarabine	lafpwcndxz(suagxxafir) = mbtacyased kuvonnvmxs (ajfoxkgeja )	-	21 May 2015
AACR2013	Not Applicable	Adult T-Cell Leukemia-Lymphoma	9	Nelarabine	gdsldazztm(xwegamfwrp) = xefipxrxce ivrahacdrz (lrrznzvhgv ) View more	Positive	15 Apr 2013

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