CARsgen® Announces 2024 Interim Results

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.

Results of GRAIL’s Galleri® Multi-Cancer Early Detection Blood Test in Prostate Cancer Published in JCO Precision Oncology

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."

UroGen Pharma to Present at Upcoming Investor Conferences

On August 29, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it will participate in fireside chats at the following investor conferences in September (Press release, UroGen Pharma, AUG 29, 2024, View Source [SID1234646212]):

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H.C. Wainwright 26th Annual Global Investment Conference – September 9-11th, 2024
Fireside Chat: September 9th, 2024 at 9:00am ET
Location: New York, NY
Webcast Registration Link: Here

2024 Cantor Global Healthcare Conference – September 17-19th, 2024
Fireside Chat: September 18, 2024 at 3:40pm ET
Location: New York, NY
Webcast Registration Link: Here

A webcast from the conference will also be available via the Investors section of UroGen’s website, www.urogen.com. A replay of the webcast will be available for approximately 90 days.

Ultragenyx to Participate in Investor Conferences in September

On August 29, 2024 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported participation in three upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, AUG 29, 2024, View Source [SID1234646211]).

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Morgan Stanley 22nd Annual Global Healthcare Conference (New York, NY)

Wednesday, September 4, 2024, Emil Kakkis, M.D., Ph.D., CEO and President, will participate in a fireside chat and host 1×1 meetings.
Cantor Global Healthcare Conference (New York, NY)

Tuesday, September 17, 2024, Emil Kakkis and Howard Horn, Chief Financial Officer, will participate in a fireside chat and host 1×1 meetings.
Bank of America Global Healthcare Conference (London, UK)

Wednesday, September 18, 2024, Eric Crombez, M.D., Chief Medical Officer and Joshua Higa, Vice President, Investor Relations, will participate in a fireside chat and host 1×1 meetings.
The live and archived webcast of the fireside chats will be accessible from the company’s website at View Source

Sana Biotechnology to Present at September 2024 Investor Conferences

On August 29, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported that it will webcast its presentations at three investor conferences in September. The presentations will feature a business overview and update (Press release, Sana Biotechnology, AUG 29, 2024, View Source [SID1234646209]).

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Sana will present at the Morgan Stanley 22nd Annual Global Healthcare Conference at 11:30 a.m. ET on Wednesday, September 4, 2024.
Sana will present at the 2024 Wells Fargo Healthcare Conference at 3:00 p.m. ET on Thursday, September 5, 2024.
Sana will present at the HC Wainwright 26th Annual Global Investment Conference at 12:30 p.m. ET on Monday, September 9, 2024.

The webcasts will be accessible on the Investor Relations page of Sana’s website at View Source A replay of each presentation will be available at the same location for 30 days following the conference.