On August 13, 2024 Bolt Biotherapeutics (Nasdaq: BOLT), a clinical-stage biopharmaceutical company developing novel immunotherapies for the treatment of cancer, reported financial results for the second quarter ended June 30, 2024, and provided a business update (Press release, Bolt Biotherapeutics, AUG 13, 2024, View Source [SID1234645792]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the second quarter, we continued to make significant progress across our two programs, BDC-3042 and BDC-4182, following our strategic pipeline prioritization in May," said Willie Quinn, Chief Executive Officer. "For our lead program BDC-3042, we completed the safety evaluation period for cohort 5 with no dose-limiting toxicities. BDC-3042 continues to be well tolerated to date, and we are now enrolling patients into cohort 6. We will be presenting a poster on BDC-4182, our claudin 18.2-targeting BoltbodyTM ISAC, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting, and we look forward to sharing more data on this program in November. I’m proud that the team has not missed a beat working through our strategic pipeline prioritization and restructuring. Our strong cash position allows us to move these programs through early clinical development and provides us with cash runway through mid-2026."

Recent Highlights and Anticipated Milestones
•Advanced to cohort 6 in the Phase 1 study of BDC-3042 in patients with advanced cancers. BDC-3042 is a proprietary agonist antibody that targets Dectin-2, an immune-activating receptor expressed by tumor-associated macrophages (TAMs). This single-agent, dose-escalation Phase 1 clinical study is evaluating BDC-3042 in patients with metastatic or unresectable triple-negative breast cancer (TNBC), colorectal cancer, clear cell renal cell carcinoma, head and neck cancer, non-small cell lung cancer (NSCLC), ovarian cancer, or melanoma.
•Preparing BDC-4182 to start clinical trials in 2025. BDC-4182 is a next-generation BoltbodyTM ISAC clinical candidate targeting claudin 18.2, a novel, clinically validated target in oncology with expression in gastric/gastroesophageal junction cancer, pancreatic cancer, and other tumor types. BDC-4182 has advanced into IND-enabling activities, supported by in vitro and in vivo experiments demonstrating potent anti-tumor activity in multiple preclinical models. A poster on BDC-4182 will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting, which will take place from November 6-10, 2024, in Houston, Texas.

•Collaborations with Genmab and Toray continue to progress. The Company continues to work with its collaborators to discover and develop ISACs for the treatment of cancer. Recent developments with Genmab supported the extension of the original initial research phase of the collaboration.
•Cash, cash equivalents, and marketable securities were $97.5 million as of June 30, 2024. Cash on hand is expected to fund multiple milestones and operations through mid-2026.

Second Quarter 2024 Financial Results

•Collaboration Revenue – Collaboration revenue was $1.3 million for the quarter ended June 30, 2024, compared to $1.4 million for the same quarter in 2023. Revenue in the comparative periods was generated from services performed under R&D collaborations as we fulfill our performance obligations.

•Research and Development (R&D) Expenses – R&D expenses were $15.4 million for the quarter ended June 30, 2024, compared to $15.6 million for the same quarter in 2023.

•General and Administrative (G&A) Expenses – G&A expenses were $4.9 million for the quarter ended June 30, 2024, compared to $5.6 million for the same quarter in 2023. The decrease between the comparable periods was mainly due to a decrease in salary and related expenses primarily due to a decrease in bonus expense as a result of the restructuring plan.

•Restructuring Charges – Restructuring charges were $3.6 million for the quarter ended June 30, 2024, consisting of $2.9 million of one-time termination benefits such as severance costs and related benefits and $0.7 million of non-cash stock-based compensation expense as a result of the restructuring plan. There were no restructuring charges in the quarter ended June 30, 2023.

•Loss from Operations – Loss from operations was $22.6 million for the quarter ended June 30, 2024, compared to $19.8 million for the same quarter in 2023.

About the Boltbody Immune-Stimulating Antibody Conjugate (ISAC) Platform
Bolt Biotherapeutics’ Boltbody ISAC platform harnesses the precision of antibodies with the power of the innate and adaptive immune system to generate a productive anti-cancer response. Each Boltbody ISAC candidate comprises a tumor-targeting antibody, a non-cleavable linker, and a proprietary immune stimulant. The antibody is designed to target one or more markers on the surface of a tumor cell and the immune stimulant is designed to recruit and activate myeloid cells. Activated myeloid cells initiate a positive feedback loop by releasing cytokines and chemokines, chemical signals that attract other immune cells and lower the activation threshold for an immune response. This increases the population of activated immune system cells in the tumor microenvironment and promotes a robust immune response with the goal of generating durable therapeutic responses for patients with cancer.

On August 13, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported financial results for the second quarter of 2024 and provided a business update (Press release, Mural Oncology, AUG 13, 2024, View Source [SID1234645815]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We’ve seen resurgent interest across the industry in cytokines as powerful tools to fight cancer and Mural is in a unique position to deliver promising drug candidates that have the potential to overcome the limitations of prior approaches," said Caroline Loew, Ph.D., Chief Executive Officer of Mural Oncology. "Since becoming an independent company late last year, we’ve rapidly worked to shape and grow a nimble organization focused on delivering meaningful new immunotherapy treatments to cancer patients. We believe each of our programs is engineered with a differentiated approach that we hope will play out significantly in the clinic starting early next year."

Recent Corporate Highlights and Upcoming Milestones

Mural appointed George Golumbeski, Ph.D., to its board of directors in July. Dr. Golumbeski currently serves as a partner at DROIA Ventures, a specialist biotech investment firm focused on therapeutics for oncology and genetic disease. Prior to DROIA, he served as President and Head of Corporate Development for GRAIL and Executive Vice President of Business Development for Celgene. He has nearly 30 years of extensive experience with strategic collaborations, M&A, in-licensing, out-licensing, and alliance management.
Mural’s late-stage clinical trials of nemvaleukin alfa continue to progress toward readouts in the first half of 2025. The company is focused on two foundational indications for nemvaleukin, where the majority of patients do not have any currently approved therapies.

ARTISTRY-7 is a potentially registrational, phase 3 clinical trial evaluating nemvaleukin in combination with pembrolizumab compared to investigators’ choice of chemotherapy in patients with platinum-resistant ovarian cancer. Patient enrollment in this trial is now complete. Mural continues to expect to report interim overall survival (OS) results based on approximately 75% of events in the first quarter of 2025. The company anticipates reporting final OS results in the second quarter of 2026.
Mural expects to report top-line data results from cohort 2 of ARTISTRY-6 in the first half of 2025. This is a potentially registrational, phase 2 clinical trial evaluating nemvaleukin as a monotherapy in patients with mucosal melanoma.
Mural is also evaluating a less-frequent intravenous (LFIV) dose of nemvaleukin in patients with cutaneous melanoma in cohort 3 (monotherapy) and cohort 4 (combination therapy) in ARTISTRY-6. The company expects preliminary data readouts in the monotherapy cohort in the first half of 2025, and in the combination cohort with pembrolizumab in the second half of 2025.
In June, Mural presented data from ARTISTRY-3, an evaluation of the LFIV dosing of nemvaleukin, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. This data from ARTISTRY-3 informed the LFIV dose currently being used in cohort 3 and cohort 4 of ARTISTY-6. In the ARTISTRY-3 trial, the company evaluated escalating LFIV infusions, all of which were generally well tolerated. The safety profile in all dosing schedules evaluated was consistent with nemvaleukin’s known mechanism of action, and no dose limiting toxicities were observed. Although administering higher doses per cycle than in previous trials evaluating nemvaleukin, no new safety signals were identified. The desired pharmacodynamic (PD) effects were also seen across all evaluated doses. Expansion of antitumor CD8+ T cells and natural killer (NK) cells was observed concurrent with minimal expansion of immunosuppressive regulatory T cells (Tregs).

Mural’s preclinical interleukin-18 (IL-18) and IL-12 programs remain on track, with nominations for both development candidates expected this year.

Mural’s enhanced IL-18 is engineered to deliver a more sustained immune response for cancer treatment. Native IL-18 is a potent immune-stimulating cytokine, but its efficacy is blunted by IL-18 binding protein (IL-18BP), a high affinity decoy receptor that binds with and neutralizes IL-18, thereby rendering it ineffective. Native IL-18 is also limited by its short half-life. Mural’s IL-18 variant contains mutations that eliminate binding to IL-18BP while minimally impacting the native IL-18 structure. The company has also fused IL-18 to protein scaffolds to extend the half-life and increase IL-18’s exposure. Together, Mural believes these have demonstrated a more durable immunological effect in preclinical studies.
The company’s enhanced IL-12 is engineered to leverage native IL-12’s anti-tumor potency while mitigating its hallmark toxicity. Native IL-12 is a highly potent pro-inflammatory cytokine, but because of its very narrow therapeutic index, it can also be incredibly toxic with systemic exposure. Mural’s IL-12 variant splits the molecule into two inactive monomers, and these individual subunits are then separately fused to antibody fragments and sequentially injected, which deliver and concentrate IL-12 specifically in the tumor microenvironment with the goal of limiting systemic exposure. In preclinical studies, Mural believes its engineered IL-12 achieved the desired reduction in serum while maintaining tumor concentrations providing the potential to reduce systemic toxicities.
Financial Results for the Quarter Ended June 30, 2024

Cash Position: As of June 30, 2024, cash, cash equivalents, and marketable securities were $204.7 million.
R&D Expenses: Research and development expenses were $27.5 million for the second quarter of 2024 compared to $42.5 million for the second quarter of 2023. The decrease in R&D expenses was primarily due to different team composition compared to the personnel allocated to us by Alkermes, our former parent, prior to the separation, as well as decreased spend on the ARTISTRY-1 and ARTISTRY-2 trials as activities related to these trials wound down in 2023 and decreased spend on the ARTISTRY-7 trial due to the timing of patient enrollment.
G&A Expenses: General and administrative expenses were $6.7 million for the second quarter of 2024 compared to $4.7 million for the second quarter of 2023. The increase in expenses was primarily due to costs associated with operating as a standalone company after the separation. This includes employee-related expenses and professional fees.
Net Loss: Net loss was $31.6 million for the second quarter of 2024 compared to $50.2 million for the second quarter of 2023.
Financial Guidance

The company reaffirms guidance that its cash, cash equivalents, and marketable securities are expected to fund its operations into the fourth quarter of 2025.
As noted previously, management forecasts lower operating expenses in 2025 versus 2024 due to the timing of clinical trial expenses.
About Nemvaleukin
Nemvaleukin alfa (nemvaleukin) is a novel, engineered cytokine designed to leverage antitumor effects of the IL-2 pathway while mitigating the hallmark toxicities that limit its use. Nemvaleukin selectively binds to the intermediate-affinity IL-2 receptor (IL-2R) and is sterically occluded from binding to the high-affinity IL-2R. Because of this molecular design, nemvaleukin treatment leads to preferential expansion of antitumor CD8+ T cells and natural killer cells, with minimal expansion of immunosuppressive regulatory T cells. Nemvaleukin is currently being evaluated in two potentially registrational late-stage trials.

On August 14, 2024 – Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a clinical-stage biotechnology company developing novel LAG-3 immunotherapies for cancer and autoimmune disease, reported that the first participant has been successfully dosed in the first-inhuman Phase I trial of IMP761 (Press release, Immutep, AUG 13, 2024, View Source [SID1234645831]). This first-in-class agonist LAG-3 antibody is designed to restore balance to the immune system by enhancing the "brake" function of LAG-3 to silence dysregulated self-antigen-specific memory T cells that cause many autoimmune diseases.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The single and multiple ascending dose, placebo-controlled, double-blind Phase I study is being conducted by the Centre for Human Drug Research (CHDR), a world-class institute in Leiden, the Netherlands, specializing in cutting-edge early-stage clinical drug research. The study aims to enrol 49 healthy volunteers, to assess safety, pharmacokinetics (PK) and pharmacodynamics (PD).

CHDR will implement its unique keyhole limpet haemocyanin (KLH) challenge model allowing for the evaluation of IMP761’s pharmacodynamic activity at the earliest stages of clinical development. Immutep anticipates the first safety data from the Phase I study to be available before end of the year with assessment of PK/PD relationships to follow in the first half of CY2025.

The immune checkpoint LAG-3 has been identified as a promising target for agonist LAG-3 immunotherapy to treat rheumatoid arthritis, Type 1 diabetes, and multiple sclerosis, among other autoimmune diseases.1,2,3 In preclinical studies, IMP761 has led to a large decrease in inflammatory cytokines and demonstrated its effectiveness in suppressing antigen-specific T cell–mediated immune responses.

About IMP761

IMP761, a first-in-class immunosuppressive LAG-3 agonist antibody, has the potential to address the root cause of many autoimmune diseases by specifically silencing autoimmune memory T cells that accumulate at disease sites and restoring balance to the immune system. As published in the Journal of Immunology, encouraging pre-clinical in vivo and in vitro studies show IMP761 inhibits peptide-induced T cell proliferation, activation of human primary T cells, and an antigen-specific delayed-type hypersensitivity (DTH) reaction. Additional preclinical data in oligoarticular juvenile idiopathic arthritis (o-JIA) published in Pediatric Research details how IMP761 led to a decrease in a broad spectrum of effector cytokines in just 48 hours. This study also showed children with o-JIA have a skewed LAG-3 metabolism and suggested they can benefit from agonistic LAG-3 activity.

On August 13, 2024 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Candel Therapeutics, AUG 13, 2024, View Source [SID1234645793]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The second quarter of 2024 represented a pivotal period for Candel, characterized by robust clinical advancements and key regulatory successes, that further validate our innovative approach to cancer immunotherapy," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel. "Our encouraging overall survival phase 2 data for CAN-2409 highlights the potential of our lead candidate to address a significant unmet need for non-small cell lung cancer patients, who are non-responsive to immune checkpoint inhibitor treatment, and for patients with borderline resectable pancreatic cancer. In addition, the FDA granting orphan drug designation for

CAN-3110 in recurrent high-grade glioma underscores the promise of this first-in-class, novel asset developed for difficult-to-treat cancers."

Dr. Tak continued, "Our inclusion in the Russell 3000 Index also marks a significant milestone in Candel’s growth and offers an opportunity to increase our recognition within the investment community. These achievements, coupled with a successful R&D event at ASCO (Free ASCO Whitepaper), have set a strong foundation as we approach several key readouts in the latter half of 2024."

Second Quarter 2024 & Recent Highlights

•Program Updates
oCAN-2409 – Pancreatic Cancer

▪In early April, announced positive updated survival data from the phase 2 randomized controlled clinical trial of CAN-2409 plus valacyclovir (prodrug), together with standard of care (SoC) chemoradiation, in borderline resectable pancreatic ductal adenocarcinoma (PDAC).

▪Data showed notable improvements in estimated median overall survival (mOS) of 28.8 months after experimental treatment with CAN-2409 versus 12.5 months in control group.

▪At 24 months, survival rate was 71.4% in CAN-2409 treated patients after chemoradiation and prior to surgery versus 16.7% in the control group. At 36 months, estimated survival was 47.6% in the CAN-2409 group after chemoradiation and prior to surgery versus 16.7% in the control group.

▪No new safety signals were observed, providing further support that multiple injections of CAN-2409 have been generally well-tolerated to date, with no dose-limiting toxicities and no cases of pancreatitis reported.

▪Analysis of resected tumors showed the formation of dense aggregates of immune cells, including CD8+, cytotoxic tumor infiltrating lymphocytes and dendritic cells, within the tumor microenvironment after CAN-2409 administration, confirming the activation of a robust antitumoral immune response.

▪Received orphan drug designation from the FDA for CAN-2409 for the treatment of pancreatic cancer.

oCAN-2409 – Non-Small Cell Lung Cancer

▪Presented topline overall survival data from the phase 2 clinical trial of CAN-2409 plus valacyclovir in combination with continued immune checkpoint inhibitor (ICI) therapy in patients with stage III/IV NSCLC inadequately responding to ICI therapy at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting.
▪Data showed mOS of 20.6 months in patients with progressive disease despite ICI treatment compared to published results of less than 12 months with SoC docetaxel-based chemotherapy in similar patient populations.1
▪CAN-2409 treatment resulted in activation of the systemic immune response after two administrations of CAN-2409, including increased numbers of circulating cytotoxic and memory T cells associated with subsequent prolonged survival.
▪As of the April 1, 2024 data cut-off date, CAN-2409 treatment in NSCLC continued to exhibit a favorable safety and tolerability profile.
oCAN-3110 – Recurrent High-Grade Glioma
▪Received orphan drug designation from the FDA for CAN-3110 for the treatment of rHGG.
▪Presented a Trial-in-Progress poster at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on the ongoing phase 1b clinical trial exploring multiple doses of CAN-3110 in patients with rHGG.
oenLIGHTEN Discovery Platform
▪Presented preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting unveiling the second candidate from the enLIGHTEN Discovery Platform, a first-in-class multimodal immunotherapy candidate to induce tertiary lymphoid structures (TLS), being developed as a novel therapeutic for solid tumors.
•Corporate Updates

oHosted successful NSCLC Research and Development panel during the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting, featuring prominent scientific and medical thought leaders discussing the topline overall survival data from the phase 2 clinical trial of CAN-2409 in NSCLC.

oAnnounced inclusion in the Russell 3000 Index, effective July 1, 2024, as part of FTSE Russell’s annual reconstitution of its U.S. equity indexes.
Anticipated Milestones

•Updated phase 1b data (Arm C) for CAN-3110 in rHGG expected in H2 2024.
•Phase 2b topline data for CAN-2409 in low-to-intermediate-risk, localized, non-metastatic prostate cancer expected in Q4 2024.
•Phase 3 topline disease-free survival data for CAN-2409 in localized intermediate/high-risk prostate cancer expected in Q4 2024.
Financial Results for Second Quarter Ended June 30, 2024

Research and Development Expenses: Research and development expenses were $5.0 million for the second quarter of 2024 compared to $5.9 million for the second quarter of 2023. The decrease was primarily due to lower clinical development costs driven by a reduction in regulatory, manufacturing and clinical trial costs for CAN-2409 programs and lower payroll-related expenses following the corporate restructuring in the fourth quarter of 2023. These decreases were partially offset by increased stock-based compensation expense. Research and development expenses included non-cash stock compensation expense of $1.3 million for the second quarter of 2024 compared to $0.3 million for the second quarter of 2023.

General and Administrative Expenses: General and administrative expenses were $3.6 million for both the second quarter of 2024 and the second quarter of 2023. There was a small decrease, primarily due to lower insurance costs and recruiting costs. These decreases were partially offset by increased professional and consulting fees. General and administrative expenses included non-cash stock compensation expense of $0.6 million for the second quarter of 2024 compared to $0.4 million for the second quarter of 2023.

Net Loss: Net loss for the second quarter of 2024 was $22.2 million, compared to a net loss of $9.6 million for the second quarter of 2023, and included other expense, net of $13.7 million and $35,000, respectively, primarily due to the change in the fair value of the Company’s warrant liability.

Cash Position: Cash and cash equivalents, as of June 30, 2024, were $21.5 million, as compared to $35.4 million as of December 31, 2023. Based on current plans and assumptions, the Company expects that its existing cash and cash equivalents will be sufficient to fund its current operating plan into the first quarter of 2025.

On August 13, 2024 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies, reported financial results for the second quarter ended June 30, 2024 (Press release, Nkarta, AUG 13, 2024, View Source [SID1234645816]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Patients remain our focus, and early execution on our clinical trials across disease areas is a testament to that commitment," said Paul J. Hastings, CEO of Nkarta. "NKX019 has the potential to reach people living with a wide range of autoimmune diseases, and we will continue to evaluate ways to maximize our impact in this field. Our cellular engineering enables us to evaluate a reduced toxicity lymphodepletion regimen, to limit hospitalization and patient burden, and spare the complications of other agents."

Continued execution in clinical development of NKX019 for autoimmune diseases

Received clearance from FDA of second Investigational New Drug (IND) application for NKX019 in autoimmune disease, supporting the planned initiation of Ntrust-2, a clinical trial of NKX019 for the treatment of systemic sclerosis, myositis and vasculitis. Patients enrolled in Ntrust-2 will receive three doses of NKX019 on Days 0, 3, and 7 following lymphodepletion (LD) with single-agent cyclophosphamide (cy).
Initiation of an investigator-sponsored trial (IST) of NKX019 in systemic lupus erythematosus (SLE) with or without LN by researchers at Columbia University Irving Medical Center. Patients enrolled in the IST will receive three doses of NKX019 on Days 0, 7, and 14 following LD with single-agent cy.
Autoimmune milestones 2024-2025

Initiation of patient enrollment in Ntrust-2 clinical trial expected by year-end 2024.
Preliminary clinical data from Ntrust-1 and Ntrust-2 clinical trials planned for 2025.
Mid-year update for NKX019 in non-Hodgkin lymphoma (NHL)

Nkarta presented follow-up data from its Phase 1 clinical trial of NKX019 in relapsed/refractory NHL at the Pan Pacific Lymphoma Conference in July 2024, including follow-up on 4 patients who were retreated and re-entered complete response (CR), demonstrating the safety and encouraging effectiveness (4/4 CR) of retreatment.
Nkarta previously opened a cohort with a compressed (7-day) dosing schedule, where patients with large B-cell lymphoma (LBCL) who have progressed following CAR T therapy receive NKX019 on Days 0, 3, and 7 following LD with fludarabine (flu) and cy.
Nkarta has completed enrollment of patients into the 7-day dosing cohort in LBCL following CAR T and expects to announce data from this cohort in late-2024.
Future development of NKX019 in NHL will be contingent on favorable outcomes from the seven patients that have been treated in the new cohort.
Leadership Updates

In July 2024, Nadir Mahmood, Ph.D., joined Nkarta as President, and David R. Shook, M.D., was promoted to Chief Medical Officer, Head of Research & Development, with both executives reporting to Paul J. Hastings, Chief Executive Officer.
In June 2024, George Vratsanos, M.D., FACR, an accomplished biopharmaceutical executive with scientific and clinical expertise in immunology and autoimmunity, joined Nkarta’s Board of Directors.
Second Quarter 2024 and Recent Financial Highlights

Nkarta had cash, cash equivalents, restricted cash, and investments in marketable securities of $426.7 million as of June 30, 2024.
Research and development (R&D) expenses were $23.1 million for the second quarter of 2024. Non-cash stock-based compensation expense included in R&D expense was $2.2 million for the second quarter of 2024.
General and administrative (G&A) expenses were $7.6 million for the second quarter of 2024. Non-cash stock-based compensation expense included in G&A expense was $2.2 million for the second quarter of 2024.
Net loss was $25.0 million, or $0.34 per basic and diluted share, for the second quarter of 2024. This net loss includes non-cash charges of $6.7 million that consisted primarily of share-based compensation and depreciation expenses.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into late 2027.
About NKX019
NKX019 is an allogeneic, cryopreserved, off-the-shelf immunotherapy candidate that uses natural killer (NK) cells derived from the peripheral blood of healthy adult donors. It is engineered with a humanized CD19-directed CAR for enhanced cell targeting and a proprietary, membrane-bound form of interleukin-15 (IL-15) for greater persistence and activity without exogenous cytokine support. CD19 is a biomarker for normal B cells as well as those implicated in autoimmune disease and B cell-derived malignancies.

About Ntrust Clinical Trials in Autoimmune Disease
Ntrust-1 and Ntrust-2 are multi-center, open label, dose escalation clinical trials that build on academic studies of durable, drug-free remissions in patients with autoimmune disease after CD19-targeted cell therapy. Both trials will assess the safety of NKX019 in people living with autoimmune diseases as well as its ability to enable long-term remissions via a "reset" of the immune system through the elimination of pathogenic B cells. Per the trial protocols, patients receive three-dose cycles of NKX019 at 1 billion or 1.5 billion cells per dose following single-agent lymphodepletion with cyclophosphamide, an agent with an established safety profile across autoimmune diseases. Leveraging the engineering of NKX019, no patients in either trial will receive supplemental cytokines or antibody-based therapeutics. This approach is designed to evaluate the single-agent activity of NKX019 and facilitate a more rapid path to regulatory approval.

In the Ntrust-1 study, patients with refractory lupus nephritis receive NKX019 on Days 0, 7 and 14. Patients in Ntrust-1 may also receive additional cycles to restore response.

Once initiated, Ntrust-2 will enroll patients with systemic sclerosis (scleroderma), idiopathic inflammatory myopathy (myositis), and ANCA-associated vasculitis into parallel cohorts, and NKX019 will be dosed on Days 0, 3, and 7, a regimen that may be advantageous across all Nkarta clinical trials. Each trial is designed to initially enroll up to 12 patients.