On June 13, 2022 ImCheck Therapeutics reported the close of a EUR 96 million (USD 103 million) financing, co-led by Earlybird and Andera Partners (Press release, ImCheck Therapeutics, JUN 13, 2022, View Source [SID1234615917]). The Series C round (EUR 80 Million – USD 86 Million) and the last outstanding tranche of Series B converted in Series C shares (EUR 16 Million – USD 17.2 Million) solidifies ImCheck’s financial position and leadership in the gamma-delta T cell space. Invus and The Leukemia & Lymphoma Society Therapy Acceleration Program also joined the round as new investors. Existing investors including the Growth Opportunity Fund of founding investor Kurma Partners, Eurazeo, Gimv, EQT Life Sciences (previously LSP), Boehringer Ingelheim Venture Fund, Pfizer Ventures, Bpifrance through its Innobio 2 and Large Venture funds, Wellington Partners, Agent Capital, Pureos Bioventures and Alexandria Venture Investments participated.

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The proceeds will be used to support the Phase IIa expansion arms of EVICTION in solid tumors and hematologic cancers, and completion of randomized, double-blind, placebo-controlled clinical trials evaluating ImCheck’s lead candidate ICT01 in combination with a PD-1 inhibitor for multiple solid tumors. The Company also will apply the capital to investigate ICT01 in combination with other therapeutic agents, including IL-2, in the forthcoming EVICTION-2 clinical trial. The funding will accelerate the further advance toward the clinic of additional antibody candidates in immuno-oncology, auto-immune and infectious diseases. In addition, it will allow the Company to achieve pivotal study readiness for ICT01 and expand its footprint through extended clinical operations and regulatory affairs in Europe and the US.

"Since its inception, ImCheck has gained the support of a syndicate of outstanding international funds. In a highly challenging funding market, we have secured a significant fundraising through the addition of highly strategic and valuable investors from the U.S. and Europe, putting us in a position to further deliver on the immense promise of our pipeline," stated Pierre d’Epenoux, Chief Executive Officer of ImCheck Therapeutics. "We view our singular proprietary position with butyrophilins, which offer powerful immunomodulation of both the innate and adaptive immune systems, as the key to therapeutic intervention for many disease indications and we value the support we are now gaining from The Leukemia & Lymphoma Society Therapy Acceleration Program as a first investment from a cancer patient nonprofit organization."

In conjunction with the financing round, Florent Gros (Earlybird) and Raphaël Wisniewski (Andera Partners) will join the Company’s Board of Directors.

Florent Gros, Partner at Earlybird, commented,"ImCheck’s approach to immuno-oncology is highly differentiated through the clinical demonstration of gamma-delta T cell activation, an area of immunology that has huge potential and interest from the biopharmaceutical community. At Earlybird, we support companies that dare to think differently and ImCheck’s innovative concept for immunomodulation could be a game-changer for a range of indications."

Raphaël Wisniewski, Partner at Andera Partners, said, "Immune checkpoint inhibitors have heralded a new era in cancer treatments and ImCheck Therapeutics is pioneering the next generation of these immunotherapeutics. In watching their progress to date, we have seen the leadership team execute on a compelling vision for a butyrophilin-based therapeutic approach from the early development stage into a highly valuable clinical development program. We at Andera Partners are confident the company will move its groundbreaking technology forward to meet patients’ needs in a range of cancer indications with wider potential for auto-immune and infectious diseases."

ImCheck Therapeutics’ immunotherapeutic technology is capable of overcoming the tumor’s resistance to adaptive immune responses through a novel superfamily of immune checkpoint targets, butyrophilins (BTNs). BTNs can be modulated to harness a wide range of immune cells including gamma-delta T cells, CD3, CD8, NK cells and macrophages, bridging the innate and adaptive immune responses. The company’s broad pipeline is built upon immunomodulation via BTN-targeting antibodies aimed either at activating the immune system in disease indications such as cancer or infectious diseases, or down-regulating immunity in auto-immune disorders.

Hans Henrik Christensen, Chief Financial Officer of ImCheck Therapeutics, added, "ImCheck has raised a total of EUR 154 million since inception. We truly appreciate the support from existing and new investors, which extends our cash runway until 2026. This enables us to further explore the ‘pipeline in a product’ opportunity we have with our lead clinical candidate, ICT01."

Legal counsel for the Series C transaction provided by Dentons Europe and McDermott Will & Emery. Investor relations support provided by Trophic Communications. French media and communications support provided by ATCG Partners.

On June 13, 2022 Mnemo Therapeutics, a biotechnology company developing transformational immunotherapies, reported the kick-off of their participation in the prestigious Hospital-University Research in Health (RHU) program (Press release, Mnemo Therapeutics, JUN 13, 2022, View Source [SID1234615911]). Designed by France’s National Research Agency, the RHU funding supports cutting-edge research and cross-institutional collaborations, bringing together academia, businesses, and hospitals to push the boundaries of what is possible in healthcare.

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In December of 2021, 17 proposals were selected for the RHU funding. Among them, receiving close to 10 million euros, is the EpCART project. Under the leadership of Sebastian Amigorena, Ph.D., one of Mnemo’s scientific co-founders and head of the Immune Responses and Cancer Team at Institut Curie, the EpCART project aims to clinically validate a novel approach to CAR-T immunotherapy.

"We are honored to be named a laureate for this prestigious initiative and work alongside our long-standing partners at Institut Curie, as well as the MEARY Cell and Gene Therapy Center at Saint-Louis Hospital, AP-HP," said Mnemo CEO, Robert LaCaze.

The EpCART project focuses on epigenetic reprogramming of CAR-T cells. Through the inactivation of SUV39H1, a key enzyme in the differentiation pathway of T cells, the memory phenotype of these cells can be greatly increased. This works to achieve long-lasting tumor control, addressing patient relapse.

"Current immuno-oncology therapies often suffer from immune-cell memory loss, causing therapies to become less active and persistent in their ability to attack cancer cells over time," said Dr. Amigorena. "The EpCART project mines insights into the memory of the immune system to overcome this challenge and produce a new class of CAR-T therapies with enhanced memory and persistence. We believe this will drive more durable responses for cancer patients."

Over the next five years, the EpCART project will seek to clinically validate this technology as an autologous therapy. Mnemo will work in collaboration with the other EpCART partners to conduct a Phase I-II clinical trial, investigating the activity of these innovative CAR-T therapies in a cohort of 35 patients with difficult-to-treat solid tumors.

The EpCART project will accelerate research on Mnemo’s EnfiniT Discovery Engine, an integrated drug discovery tool combining key technologies to create lasting immune memory, identify novel cancer-specific targets, and more.

"Today is an exciting step in our journey to develop transformational immunotherapies with the aim of improving the body’s ability to detect, fight, and overcome cancer," said Mnemo Co-Founder and Chief Operating Officer, Alain Maiore. "We are truly honored to collaborate with such esteemed partners and look forward to the fruits of this initiative."

On June 12, 2022 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported positive initial data from the first 22 Response Assessment for Neuro-Oncology ("RANO")-evaluable patients enrolled in the ongoing, open-label, single-arm, pivotal Phase 2 FIREFLY-1 clinical trial (Press release, Day One, JUN 12, 2022, View Source [SID1234615929]). FIREFLY-1 is evaluating tovorafenib (DAY101) as once-weekly monotherapy in patients aged 6 months to 25 years with relapsed or progressive pLGG, which is the most common brain tumor diagnosed in children and for which there are no approved therapies and there is no standard of care. The primary endpoint of the FIREFLY-1 trial is ORR by RANO criteria as assessed by blinded independent central review. Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. FIREFLY-1 is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and is designed to support the potential regulatory approval of tovorafenib.

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Initial data from the first 25 patients enrolled in the trial demonstrate:

64% ORR and 91% CBR (partial response/unconfirmed partial response + stable disease) in the 22 RANO-evaluable patients:

14 partial responses (13 confirmed responses and 1 unconfirmed response)

6 patients with stable disease

All patients with stable disease (n=6) were noted to have tumor shrinkage, ranging between 19% and 43%

Responses were observed in patients with both BRAF fusions and BRAF V600E mutations who received prior MAPK-targeted therapy

The median-time-to-response was 2.8 months

A heavily-pretreated population, with a median of 3 prior lines of therapy (range: 1-9)

All patients who responded remain on therapy (n=14) and no patients have discontinued treatment due to treatment-related adverse events.

Initial safety data, based on the first 25 patients, indicated monotherapy tovorafenib to be generally well-tolerated. The majority of adverse events (AEs) were grade 1 or 2 in nature; the most common ( ≥25% any grade) treatment related AEs were increase in blood creatine phosphokinase, rash, and hair color changes. Treatment-related AEs of grade 3 or greater occurred in nine patients (36%).

"These initial findings underscore the potential of tovorafenib monotherapy to become a significant and transformative new option for relapsed/progressive pLGG, a pediatric brain tumor with no approved treatments today," said Samuel Blackman, M.D., Ph.D., co-founder and chief medical officer of Day One. "With the registrational cohort fully enrolled, patient follow-up is ongoing, and we look forward to the topline data from the complete study population in the first quarter of 2023. Based on these positive initial data, we plan to begin the pivotal Phase 3 FIREFLY-2 clinical trial evaluating tovorafenib as a front-line therapy in pLGG to evaluate whether tovorafenib can provide benefit early in the disease development."

"These initial data demonstrate significant anti-tumor activity in children with relapsed/progressive pLGG, including children who are refractory to available therapies. Pediatric low-grade glioma is a truly challenging disease in which children face years of aggressive regimens that can carry a long-term impact on learning, cognition, and quality of life," said Roger Packer, M.D., senior vice president, Center for Neuroscience and Behavioral Medicine, and director, Brain Tumor Institute, Children’s National Hospital.

Day One plans to present additional interim trial results from FIREFLY-1 at an upcoming medical conference in the second half of 2022. Day One anticipates releasing topline results for the full FIREFLY-1 pivotal study population in the first quarter of 2023. If the data are supportive, Day One expects to submit a new drug application (NDA) to the United States Food and Drug Administration (FDA) in the first half of 2023.

Expanding Development of Tovorafenib in Front-Line pLGG

Based on these initial FIREFLY-1 data, Day One plans to expand the development of tovorafenib as a front-line therapy for patients newly diagnosed with pLGG. The global, pivotal Phase 3, registrational clinical trial ("FIREFLY-2/LOGGIC") will evaluate once-weekly monotherapy tovorafenib in newly-diagnosed patients with pLGG. The FIREFLY-2/LOGGIC study is designed to evaluate the efficacy and safety of tovorafenib in patients with newly-diagnosed pLGG harboring a known activating BRAF alteration. The study is a randomized, monotherapy, open-label trial aiming to enroll approximately 400 patients aged 6 months to 25 years across approximately 100 sites globally, including in the U.S., Europe and Asia. Participants will be randomized to either tovorafenib (Arm 1) or an investigator’s choice of one of three standard of care chemotherapy options (Arm 2). The primary endpoint will be the ORR based upon RANO criteria as reported by Blinded Independent Central Review. Secondary endpoints will include safety, progression-free survival, duration of response, functional outcomes, and quality of life measures.

Day One will conduct the FIREFLY-2/LOGGIC trial in collaboration with the Low-Grade Glioma in Children (LOGGIC) consortium, a group of internationally recognized experts in pLGG research, and an extensive network of pediatric oncology centers, including Hopp Children’s Cancer Center Heidelberg (KiTZ), the German Cancer Research Center (DKFZ) and the Brain Tumor Group of the European Society for Paediatric Oncology (SIOPE BTG). Day One expects to dose the first patient in FIREFLY-2/LOGGIC trial in the third quarter of 2022.

Conference Call and Webcast Information

Day One will host a conference call and webcast tomorrow, June 13, 2022, at 8:00 a.m. Eastern Time. To participate by telephone, please dial 844-713-6132 (Domestic) or 1-213-320-2543 (International). The conference ID number is 3568447. The webcast will be made available for replay on the Company’s website beginning approximately two hours after the event and will be available for 30 days following the live presentation.

About Pediatric Low-Grade Glioma

Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children, accounting for 30% – 50% of all central nervous system tumors. BRAF wild-type fusions are the most common cancer-causing genomic alterations in pediatric low-grade gliomas. These genomic alterations are also found in several adult and pediatric solid tumors. Currently approved BRAF inhibitors are only active in tumors harboring BRAF V600 mutations, exhibit limited activity in brain tumors, and cannot be used in patients harboring BRAF fusions.

Pediatric low-grade glioma can impact a child’s health in many ways depending on tumor size and location, including vision loss and motor dysfunction. There are no approved therapies for pLGG, and current treatment approaches are associated with significant acute and life-long adverse effects. While most children with pLGG survive their cancer, children who do not achieve remission following surgery may face years of increasingly aggressive therapies that can have lasting effects on learning, cognition, and quality of life. Due to the indolent nature of pLGG, patients receive multiple years of systemic therapy.

About Tovorafenib

Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib has been studied in over 250 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) among pediatric, adolescent and young adult patients with pediatric low-grade glioma (pLGG), which is an area of considerable unmet need with no approved therapies. Tovorafenib is also being evaluated alone or as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1). Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission (EC) for the treatment of glioma.

About the Pacific Pediatric Neuro-Oncology Consortium

The Pacific Pediatric Neuro-Oncology Consortium (PNOC) is an international consortium with study sites within the United States, Canada, Europe and Australia dedicated to bringing new therapies to children and young adults with brain tumors.

On June 12, 2022 Gracell Biotechnologies Inc. ("Gracell" or the "Company",NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing highly efficacious and affordable cell therapies for the treatment of cancer, reported the updated clinical data from a multicenter study evaluating GC012F, the Company’s autologous CAR-T therapeutic candidate dual-targeting B-cell maturation antigen (BCMA) and CD19, for the treatment of relapsed/refractory multiple myeloma (RRMM). Gracell shared the data at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress (EHA2022 Congress), held from June 9-12 in Vienna, Austria (Press release, Gracell Biotechnologies, JUN 12, 2022, View Source [SID1234615914]).

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"The updated data of the final dataset of 29 treated patients underscore the deep responses achieved with GC012F, including a 100% MRD negativity rate in all patients treated," commented Dr. Martina Sersch, Chief Medical Officer of Gracell. "GC012F for the treatment of RRMM continues to demonstrate a favorable safety profile and a promising mDOR of 15.7 months in mostly high risk, heavily pretreated patients, including those with extramedullary disease and those who were previously exposed to PI, IMiDs and anti-CD38 agents. In addition, we presented at EHA (Free EHA Whitepaper) the first-in-human data of GC012F in B-NHL, a potential second indication. We look forward to continuing developing this lead asset and providing a new treatment option to the patients with high unmet needs."

Additionally, on June 10, Gracell presented the first clinical results of the first-in-human phase 1 investigator-initiated study (IIT) in China evaluating the safety and tolerability of GC012F in B-cell non-Hodgkin’s lymphoma patients, and longer-term follow-up results of a safety and efficacy study from an IIT evaluating allogeneic TruUCAR-T GC502 in patients with B-cell acute lymphoblastic leukemia. Gracell announced these data in a press release on May 12.

BCMA/CD19 Dual-Targeting FasTCAR-T GC012F for the Treatment of RRMM
From October 2019 to January 2022, 29 heavily pretreated RRMM patients were enrolled and treated in this single-arm, open label, multicenter IIT with a single infusion of GC012F at three dose levels: 1×105 cells/kg (DL1), 2×105 cells/kg (DL2), and 3×105 cells/kg (DL3). 90% (26/29) patients were high risk based on mSMART 3.0 criteria and patients had received a median of five prior lines of therapy.

At the data cutoff of June 8, 2022, the 29 patients had been evaluated for response with a median follow-up duration of 11 months, ranging from 4.9 to 34.5 months. Patients continue to be followed for deepening responses. The response rate at different dose levels was 100% (2/2) in DL1, 80% (8/10) in DL2, and 100% (17/17) in DL3. All patients 100% (29/29) achieved minimal residual disease (MRD) negativity. 75.9% (22/29) of all patients treated achieved MRD- sCR. Median duration of response (DOR) at data cut off was 15.7 months (95% CI: 7.6-33.1).

The safety profile of GC012F was consistent with previous findings with mostly low grade of cytokine release syndrome (CRS). 93% (27/29) of events were Grade 0-2 and 7% (2/29) of events were Grade 3. No Grade 4 or 5 CRS, or any Grade immune effector cell-associated neurotoxicity syndrome (ICANS) were observed. Patients continue to be monitored for safety and efficacy including best overall response (BOR) and DOR. Due to the COVID-19 related lockdowns at the lead site of the study in Shanghai, China, follow-up assessment visits of certain patients were delayed and Gracell is looking forward to providing further updates in future publications for DOR, ORR, and BOR in all treated patients.

In November 2021, GC012F was granted Orphan Drug Designation for the treatment of multiple myeloma by the U.S. Food and Drug Administration.

Details of the presentation are as follows:

Abstract title: Updated results of a multicenter first-in-human study of BCMA/CD19 dual-targeting FasTCAR-T GC012F for patients with relapsed/refractory multiple myeloma (RRMM)
Session title: Relapsed/refractory myeloma: BCMA-directed therapies
Presentation time: Sunday, June 12 from 11:30 AM – 12:45 PM CEST
Presentation location: Hall A2-A3
For more information about the EHA (Free EHA Whitepaper)2022 Hybrid Congress, visit www.ehaweb.org.

Clinical update conference call and webcast details
Monday, June 13, 2022 @ 8:00AM ET
Investor domestic dial-in: (833) 693-0545
Investor international dial-in: +1(661) 407-1586
Conference ID: 1820109
Live webcast link: View Source
A replay of the webcast will be available on ir.gracellbio.com shortly after the conclusion of the event for 90 days.

About GC012F
GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being evaluated in IIT studies in China for the treatment of multiple myeloma and B-cell non-Hodgkin’s lymphoma. GC012F simultaneously targets CD19 and BCMA to drive fast, deep and durable responses, which can potentially improve efficacy and reduce relapse in multiple myeloma and B-NHL patients.

About FasTCAR
CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With next-day manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, and, together with fast release time, enables enhanced accessibility of cell therapies for cancer patients.

On June 12, 2022 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that it has released results from a Phase I study of the company’s novel Bcl-2-selective inhibitor lisaftoclax (APG-2575) in Chinese patients with relapsed/refractory non-Hodgkin lymphoma (r/r NHL) at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Hybrid Congress (EHA 2022) (Press release, Ascentage Pharma, JUN 12, 2022, View Source;ascentage-pharma-releases-encouraging-results-of-bcl-2-inhibitor-lisaftoclax-apg-2575-in-chinese-patients-with-relapsedrefractory-non-hodgkin-lymphoma-301566251.html [SID1234615913]).

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The EHA (Free EHA Whitepaper) Congress is the largest gathering of the hematology field in Europe. It showcases the most cutting-edge research and state-of-the-art innovative therapies, attracting over 10,000 clinical experts and researchers from more than 100 countries every year.

The data presented at this year’s EHA (Free EHA Whitepaper) Congress show that lisaftoclax was well tolerated at doses of up to 800 mg/day, without evidence of tumor lysis syndrome (TLS). In addition, lisaftoclax demonstrated preliminary efficacy in a range of relapsed/refractory hematologic malignancies, including chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone lymphoma (MZL), mantle cell lymphoma (MCL), and T-cell NHL, having achieved 4 complete responses (CRs) and 8 partial responses (PRs) in 32 efficacy evaluable patients.

In the 11 efficacy evaluable patients with CLL (all of whom were heavily pretreated and had failed prior therapies such as chemoimmunotherapies and Bruton’s tyrosine kinase [BTK] inhibitors, and the majority had at least 1 type of adverse prognostic factors such as 17p deletion/TP53 mutation), there were 8 efficacy evaluable patients in cohorts received 200 mg or higher doses, including 3 CRs and 4 PRs, thus demonstrating an ORR of 87.5%. In the 6 efficacy evaluable patients with MCL, the ORR was 33.3% (1 CR). In the 4 efficacy evaluable patients with MZL, the ORR was 50%. In the 3 efficacy evaluable patients with T-cell NHL, the ORR was 33.3%.

Lisaftoclax is a novel, orally administered small-molecule Bcl-2-selective inhibitor being developed by Ascentage Pharma to treat hematologic malignancies and solid tumors by selectively blocking the antiapoptotic protein Bcl-2 and hence restoring the normal apoptosis process in cancer cells. Lisaftoclax is the first Bcl-2 inhibitor developed and entering clinical development in China, and is also the second such agent entering pivotal trials globally. Lisaftoclax is being studied in multiple clinical studies encompassing a range of solid tumors and hematologic malignancies, and has shown high clinical potential.

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, commented, "Lisaftoclax is a key drug candidate in Ascentage Pharma’s apoptosis-targeted pipeline. It has shown favorable tolerability and efficacy in multiple clinical studies in patients with solid tumors and hematologic malignancies. The drug candidate’s therapeutic potential was further validated by the data released at EHA (Free EHA Whitepaper) 2022. We will press ahead with the clinical development of lisaftoclax in efforts to bring a much-needed new treatment option to patients."

Highlights of this abstract on lisaftoclax are as follows:

Preliminary Results of a Phase 1 Study of Novel Bcl-2 Inhibitor Lisaftoclax (APG-2575) in Chinese Patients (pts) with Relapsed or Refractory (r/r) Non-Hodgkin Lymphomas (NHLs)

Abstract: #P1106

Highlights:

This multicenter, single-agent, Phase I trial consists of a dose escalation and a dose expansion and is the first-in-human study of lisaftoclax in adults with R/R NHL in China. The objective of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of lisaftoclax in Chinese patients with R/R NHL.
Patients were dosed with lisaftoclax orally once daily in 28-day cycles. A daily ramp-up schedule was used in patients with CLL or NHL with medium/high-risk TLS.
As of January 1, 2022, 40 patients had been treated with lisaftoclax (dose range 20-800 mg), including 20 who remain in the trial. NHL subtypes included: 12 CLL, 9 follicular lymphoma (FL), 7 MCL, 4 MZL, 4 T-cell lymphoma, 2 diffuse large B-cell lymphoma, and 2 Waldenström macroglobulinemia.
With a median treatment duration of 4 cycles, 4 of 32 evaluable patients achieved CR and 8 achieved PR, resulting in an ORR of 37.5%. In the 11 evaluable patients with CLL, the ORR was 63.6%, the CR rate was 27.3%, and the PR rate was 36.4%. In patients with CLL treated at doses ≥ 200 mg, the ORR was 87.5%. In the 6 evaluable patients with MCL, 1 patient achieved CR and 1 achieved PR. In the 4 evaluable patients with MZL, 2 achieved PR. In the 3 evaluable patients with T-cell NHL, 1 achieved PR.
Lisaftoclax was generally well tolerated. Most treatment-emergent adverse events (TEAEs) were grade 1-2 (67.5%). Dose-limiting toxicity (DLT) and laboratory/clinical TLS were not observed in any of the dose cohorts, and there were no dose-reduction or discontinuation due to intolerance.
Conclusions:

Lisaftoclax showed promising antitumor activity and favorable responses in subtypes of NHL including CLL/SLL, MZL, MCL, T-cell NHL. In addition, lisaftoclax demonstrated a favorable safety profile, with no TLS or DLT observed during the dose ramp-up from 20 mg – 800 mg/day.