On August 29, 2024 Artiva Biotherapeutics, Inc. (Nasdaq: ARTV), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, reported financial results for the second quarter ended June 30, 2024, and highlighted recent progress (Press release, Artiva Biotherapeutics, AUG 29, 2024, View Source [SID1234646197]).

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"Artiva is at an inflection point as we advance AlloNK in clinical development across multiple autoimmune indications and transition to a publicly traded company," said Fred Aslan, M.D., Artiva’s Chief Executive Officer. "With two trials in autoimmunity underway, we are focused on the potential of AlloNK in delivering effective, safe, and accessible cell therapies for patients with devastating autoimmune disease."

Recent Business Highlights

AlloNK

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Treatment initiated in the first patient in an investigator-initiated basket trial (IIT) exploring AlloNK in combination with rituximab in patients with rheumatoid arthritis, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists (IRIS), a community rheumatology clinic, demonstrating the potential to bring AlloNK to patients in a community setting in addition to academic and transplant centers. The study is assessing the safety, tolerability, and clinical activity of AlloNK (also known as AB-101).

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Announced treatment of the first LN patient in Artiva sponsored Phase 1/1b trial in April 2024. To Artiva’s knowledge, this marks the first patient to receive an allogeneic, off-the-shelf NK cell therapy in a U.S. clinical trial for treatment of an autoimmune disease. In the Phase 1/1b trial, AlloNK will be administered in combination with rituximab or obinutuzumab. The trial was expanded to include patients with SLE without kidney involvement.

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Expect to report initial data on autoimmune indications from at least one of the Phase 1/1b trial or the basket IIT in the first half of 2025.

Corporate and Financial Updates

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Significantly extended cash runway through the completion of Artiva’s initial public offering (IPO). Strengthened balance sheet with approximately $179.0 million in gross proceeds from the IPO in July 2024, which extends cash runway at least through the end of 2026 and through key clinical data milestones.

Second Quarter 2024 Financial Results

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Cash, Cash Equivalents and Short-Term Investments. As of June 30, 2024, Artiva had cash, cash equivalents, and short-term investments of $46.6 million. In addition, Artiva completed its IPO in July 2024 in which it sold 14,920,000 shares of its common stock, including partial exercise of the overallotment option, for gross proceeds of $179.0 million.

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Collaboration Revenue. Collaboration revenue was $0 for the three months ended June 30, 2024, compared to $3.5 million for the three months ended June 30, 2023. Revenues in 2023 were related to the Merck Sharpe & Dohme Corp. collaboration which was terminated in October 2023.

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Research and Development Expenses. Research and development expenses were $12.3 million for the three months ended June 30, 2024, compared to $11.3 million for the three months ended June 30, 2023.

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General and Administrative Expenses. General and administrative expenses were $3.9 million for the three months ended June 30, 2024, compared to $4.1 million for the three months ended June 30, 2023.

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Other Income (Expense), net. Other expense, net, was $1.7 million for the three months ended June 30, 2024, compared to other income, net, of $0.5 million for the three months ended June 30, 2023. Other expense, net, for the three months ended June 30, 2024, includes a loss on change in fair value of simple agreements for future equity (SAFEs) of $2.4 million.

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Net Loss. Net loss totaled $17.8 million for the three months ending June 30, 2024, as compared to $11.3 million for the three months ending June 30, 2023, with non-cash stock-based compensation expense of $1.5 million and $2.5 million for the three months ended June 30, 2024 and 2023, respectively.

On August 29, 2024 GRAIL, Inc. (Nasdaq: GRAL) a healthcare company whose mission is to detect cancer early when it can be cured, reported that detailed findings of the performance of its Galleri multi-cancer early detection (MCED) test in prostate cancer were published in JCO Precision Oncology (Press release, Grail, AUG 29, 2024, View Source [SID1234646214]). The data support the clinical performance of the Galleri test to preferentially screen for aggressive, clinically significant prostate cancer as compared to slow-growing (indolent) cases in the Circulating Cell-free Genome Atlas (CCGA) and PATHFINDER studies.

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"All screening tests run the risk of overdiagnosis. In the case of prostate cancer, this is largely due to the high prevalence of low-grade, indolent cancers," said Brandon Mahal, M.D., a radiation oncologist at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine, and lead author of the study. "The results of this study demonstrate that the use of MCED tests in a population-based screening program is unlikely to contribute to overdiagnosis of slow-growing prostate cancers that may not need treatment. That being said, clinically validated MCED tests like Galleri reveal that when a prostate cancer signal is detected, it usually indicates aggressive disease and additional diagnostic evaluation is necessary."

The published data is from an analysis of 420 prostate cancer patients identified in the independent clinical validation portion (substudy 3) of the multi-center, case-control observational study Circulating Cell-free Genome Atlas (CCGA) study and 18 cases from the prospective intended-use PATHFINDER study. The data were previously presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in March 2024.

Results from this analysis showed that of the prostate cancers that were detected by the MCED test, most were clinically significant (93% were intermediate or high grade and 67% were stage III or IV). For detected prostate cancers, the cancer signal of origin (CSO) prediction accuracy was > 90%. Detectability for stage I and II cancers were 4.2% across both studies combined, which is expected with low shedding prostate cancer tumor fraction and is consistent with cfDNA literature in prostate cancer. Test sensitivity of prostate cancer for all stages was 11.2% in substudy CCGA-3. Notably, the MCED test detected no low-grade cancers, 1.9% of intermediate-grade cancers, and only 4.2% of stage I and II cancers across both studies combined. Of the detected cases, 93% were Gleason grade groups 3-5. This analysis demonstrates the MCED test preferentially detects high-grade, clinically significant prostate cancer. This is important because an MCED test, when used in addition to standard-of-care screening, should not exacerbate overdiagnosis of indolent cancers. These findings also suggest that individuals with a cancer signal detected and a prostate CSO prediction should undergo a prompt diagnostic evaluation to determine the presence of aggressive disease for which treatment is generally indicated1.

"This prostate cancer analysis underscores the power of Galleri in a general population of men at-risk for prostate cancer as it is able to detect biologically significant cancers that need treatment without potentially contributing to the burden of overdiagnosis," said Dr. Eric Klein, Distinguished Scientist at GRAIL and an author on the study. "The very high accuracy of the cancer signal origin prediction for prostate cancer indicates the need for a prompt diagnostic work-up in those with a cancer signal detected."

On August 29, 2024 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported that management will participate in two upcoming investor conferences (Press release, Arvinas, AUG 29, 2024, View Source [SID1234646198]):

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Wells Fargo Healthcare Conference on Thursday, September 5, 2024.

Ian Taylor, Ph.D., President, R&D, and Randy Teel, Ph.D., Chief Business Officer, will participate in a fireside chat. A live audio webcast of the presentation will be available here and on the Events + Presentations section of the Company’s website.

Cantor Global Healthcare Conference on Thursday, September 19, 2024.

Noah Berkowitz, MD, Ph.D. Chief Medical Officer, and Andrew Saik, Chief Financial Officer, will participate in a fireside chat. A live audio webcast of the presentation will be available here and on the Events + Presentations section of the Company’s website.

On August 29, 2024 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported its 2024 Interim Results (Press release, Carsgen Therapeutics, AUG 29, 2024, View Source [SID1234646215]).

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Business Highlights

Zevor-cel was approved by NMPA in China.
Patient enrollment for the confirmatory Phase II trial of satri-cel (CT041) in China has been completed.
Multiple adjuvant studies for solid tumor CAR T-cell therapies are underway.
Clinical data on satri-cel, zevor-cel, and CT071 were presented at academic conferences.
Rapid development of differentiated allogeneic CAR T-cell product pipelines.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the past six months, we have made significant progresses in technological innovation, product development, and business operations. Zevor-cel was successfully launched in China and has been included in nearly 20 provincial and municipal healthcare plans. The pivotal Phase II trial of satri-cel in China has completed patient enrollment. We are also actively exploring the potential of satri-cel in adjuvant therapy, aiming for greater clinical benefit for patients. Additionally, we are rapidly advancing CT071, which is manufactured with our CARcelerate platform, while continuing to develop multiple allogeneic CAR T-cell products based on the THANK-uCAR platform."

1. Zevor-cel Market Launch and Commercialization Progress in China

Zevor-cel (zevorcabtagene autoleucel, CT053) is a fully human autologous CAR T-cell product targeting B-cell maturation antigen (BCMA). It was approved by the National Medical Products Administration (NMPA) on February 23, 2024 for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least three lines of therapy, (including a proteasome inhibitor and an immunomodulatory agent). CARsgen has been collaborating with Huadong Medicine for the commercialization of zevor-cel in mainland China. As of July 31, 2024, zevor-cel had been included in nearly 20 provincial or municipal government-subsidized insurance programs and private health insurance products; certification and regulatory filings had been completed in over 100 healthcare institutions across 19 provinces or cities nationwide; we had received a total of 52 orders from Huadong Medicine.

The Group’s revenue was over RMB6 million for the six months ended June 30, 2024 mainly from zevor-cel in which the primary revenue of zevor-cel was calculated on the basis of ex-works price, rather than on the basis of end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Besides, the Company received a milestone payment of RMB75 million from Huadong Medicine for zevor-cel for the six months ended June 30, 2024. Due to the inherent time cycle of CAR-T manufacturing, this results in a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries.

2. Progresses in Development of Differentiated CAR T Pipeline

Satricabtagene autoleucel (CT041) is an autologous humanized CAR T-cell product targeting Claudin18.2. Patient enrollment for the confirmatory Phase II trial (NCT04581473) in China for the treatment of gastric cancer/gastroesophageal junction cancer (GC/GEJ) has been completed. Our collaboration with Moderna, Inc. is ongoing to investigate Moderna’s investigational Claudin18.2 mRNA product in combination with satri-cel in preclinical studies.

CARsgen is actively expanding the application of CAR T-cell therapies in post-surgery treatments for solid tumors, including an ongoing Phase I clinical trial for pancreatic cancer (CT041-ST-05, NCT05911217), an investigator-initiated trial that is currently in the start-up phase for GC/GEJ, and a Phase I clinical trial for hepatocellular carcinoma (CT011-HCC-03, NCT06560827).

In the hematological malignancies field, the focus of the pipeline includes CT071, a fully human autologous CAR T-cell product targeting GPRC5D. CT071 was developed using CARsgen’s CARcelerate platform for the treatment of MM and primary plasma cell leukemia (pPCL). CARcelerate is a proprietary platform developed by CARsgen that shortens the manufacturing time to approximately 30 hours, resulting in younger and potentially more potent CAR T cells compared to conventional manufacturing processes. An investigator-initiated trial (NCT05838131) for R/R MM and R/R PCL and another investigator-initiated trial (NCT06407947) for the treatment of newly diagnosed multiple myeloma (NDMM) are currently underway in China. An IND was cleared by the FDA in November 2023 for the treatment of patients with R/R MM and R/R pPCL.

In addition to autologous products, CARsgen is also advancing differentiated allogeneic CAR T-cell products based on the THANK-uCAR platform. These include: CT0590, for the treatment of R/R MM and PCL; KJ-C2320, for the treatment of acute myeloid leukemia (AML); KJ-C2219, targeting CD19 and CD20, for the treatment of B-cell-related hematologic malignancies and autoimmune diseases; and KJ-C2114, for the treatment of solid tumors.

3. Clinical Data Disclosure

Updated results of the pivotal Phase II registrational trial of zevor-cel in China were reported as an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2024 annual meeting. Updated data from the investigator-initiated trial (CT041-CG4006, NCT03874897) of satri-cel were published in Nature Medicine in June and presented orally at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. A summary of safety and efficacy in patients with refractory metastatic pancreatic cancer (PC) (CT041-CG4006 & CT041-ST-01 Ib) was published in Journal of Clinical Oncology. The results from the satri-cel phase 1b clinical trial (CT041-ST-02, NCT04404595) in the U.S. were presented at the 2024 ASCO (Free ASCO Whitepaper) GI meeting. Results from the investigator-initiated trial (NCT05838131) of CT071 for the treatment of R/R MM were presented as a poster at the EHA (Free EHA Whitepaper) 2024 annual meeting. Data updates for CT071 and CT0590 are to be disclosed in the second half of 2024.

On August 29, 2024 Circio reported its First half-year report 2024 (Press release, Circio, AUG 29, 2024, View Source [SID1234646236]).

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