On October 31, 2023 Curocell, South Korea based CAR-T specialized company (CEO: Gunsoo Kim), reported that it has completed Phase 2 clinical trial for its next-generation CD19 CAR-T therapy "Anbal-cel" that targets relapsed or refractory DLBCL (Diffuse Large B-cell Lymphoma) (Press release, Curocell, OCT 31, 2023, View Source [SID1234636585]).

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This announcement is a significant milestone and is paving the way towards the launch and approval of Korea’s first CAR-T therapy. It has also provied typo new hope for the treatment of patients with DLBCL, an aggressive form of cancer that is growing in prevalence within Korea.

The Phase 2 clinical trial evaluated the safety, efficacy and tolerability of "Anbal-cel" in patients with relapsed or refractory DLBCL. It was conducted over a period of 20 months starting in February 2022, enrolling a total of 80 patients from six hospitals across Korea, including Samsung Medical Center, Asan Medical Center, Seoul National University Hospital, National Cancer Center, Chonnma typo National University Hospital, and Pusan National University Hospital.

"Anbal-cel" is a next-generation CAR-T therapy based on Curocell’s OVIS technology that significantly inhibits the expression of PD-1 and TIGIT, two types of immune checkpoint receptors, which are known to inhibit anti-tumor function/activity of CAR-T cells. This technology has substantially improved the therapeutic performance of CAR-T therapy in patients with relapsed or refractory DLBCL with a poor prognosis.

Curocell made international headlines during the company’s oral presentation of its Phase 2 clinical trial interim results at the International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland back in June. The interim results analyzed the efficacy and safety of its CAR-T treatment in 41 patients participating in the Phase 2 trial. The complete response rate (CRR: Rate at which the cancer enters into full remission) observed in the interim results was 71%, showing an improved therpeutic effect compared to the 40 to 50% CRR range for three FDA-approved CAR-T therapies currently on the market.

Curocell plans to officially announce the final results of its Phase 2 clinical trial for "Anbal-cel" in the first half of next year and submit an BLA application to Korea’s Ministry of Food and Drug Safety for approval next September. Starting from the expected year of approval in 2025, the company is planning to launch the supply of commercial products from Korea’s only large-scale GMP facility dedicated to the production of CAR-T boasting state-of-the-art equipment.

CEO of Curocell, Gunsoo Kim said that, "I am extremely proud that we were able to complete Korea’s first clinical Trial or study for CAR-T therapy in such a short period of time, which I believe is one of the most remarkable achievement in the history of drug development in Korea." He added, "We will make every effort to obtain approval for Anbal-cel in 2025 and ensure sustained growth for the company through future expansion in our overseas business."

The clinical trial for "Anbal-cel" was officially selected by the Korea Drug Development Fund (KDDF) in 2021 as the target of a government grant. It was conducted with the support of the National New Drug Development Project Team, funded by the Ministry of Science, ICT, and Future Planning, the Ministry of Trade, Industry and Energy, and the Ministry of Health and Welfare. (Project Number: HN21C0653)

On October 31, 2023 PacBio (NASDAQ: PACB) reported financial results for the quarter ended September 30, 2023 (Press release, Pacific Biotech, OCT 31, 2023, View Source [SID1234636584]).

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Third quarter results

Revenue of $55.7 million, a 72% increase compared with $32.3 million in the prior-year period.
Instrument revenue of $34.7 million compared with $11.4 million in the prior-year period. Instrument revenue in the third quarter of 2023 included revenue recognized from 52 RevioTM sequencing systems.
Consumables revenue of $16.9 million compared with $16.1 million in the prior-year period.
Service and other revenue of $4.1 million compared with $4.8 million in the prior-year period.
Gross profit for the third quarter of 2023 was $17.9 million, representing a 32% increase compared with $13.5 million for the third quarter of 2022 and a gross margin of 32% in the third quarter of 2023 compared to 42% for the third quarter of 2022. Non-GAAP gross profit for the third quarter of 2023 was $18.1 million and represented a non-GAAP gross margin of 32% in the third quarter of 2023, compared to a non-GAAP gross profit of $13.7 million in the third quarter of 2022, which represented a non-GAAP gross margin of 42% (see accompanying tables for reconciliations of GAAP and non-GAAP measures).

Operating expenses totaled $100.4 million for the third quarter of 2023, compared to $88.2 million for the third quarter of 2022. Non-GAAP operating expenses totaled $90.9 million for the third quarter of 2023, compared to $83.8 million for the third quarter of 2022. Operating expenses for the third quarter of 2023 and the third quarter of 2022 included non-cash share-based compensation of $18.6 million and $18.0 million, respectively.

Net loss for the third quarter of 2023 was $66.9 million, compared to a net loss of $77.0 million for the third quarter of 2022. Non-GAAP net loss was $67.9 million for the third quarter of 2023, compared to $72.5 million for the third quarter of 2022.

Net loss per share for the third quarter of 2023 was $0.26 compared to net loss per share of $0.34 for the third quarter of 2022. Non-GAAP net loss per share for the third quarter of 2023 was $0.27 compared to $0.32 for the third quarter of 2022.

Cash, cash equivalents, and investments, excluding short- and long-term restricted cash, at September 30, 2023, totaled $767.8 million, compared to $772.3 million at December 31, 2022.

Updates since PacBio’s last earnings release

Launched PacBio WGS Variant Pipeline, a standardized computational method consolidating over ten separate secondary and tertiary analysis tools into a single user-friendly workflow, further enabling users with all levels of bioinformatics experience to access HiFi whole genome sequencing (WGS).
Collaborated with automation providers Hamilton, Integra, Revvity, and Tecan to create fully automated sample preparation protocols for Revio and Sequel II/IIe systems.
Collaborated with GeneDx and the University of Washington to study the potential capabilities of HiFi long-read WGS to increase diagnostic rates in pediatric patients with genetic conditions.
Announced PacBio Capital, a program that allows customers greater flexibility and a streamlined process for leasing PacBio sequencing systems.
Appointed David Meline, former CFO of Moderna Inc., Amgen Inc., and 3M Company, to PacBio’s Board of Directors.
"PacBio had another successful quarter as we continued to drive Revio adoption and exceeded $50 million in quarterly revenue for the first time in company history," said Christian Henry, President and Chief Executive Officer. "We also had record consumable revenue in the quarter, with Revio consumables exceeding Sequel II/IIe consumables, underscoring our customers’ enthusiasm and ability to ramp up sequencing volumes on the new platform. Additionally, we shipped the first Onso systems, and I’m encouraged by the initial customer reception of the platform and the Sequencing by Binding chemistry."

Quarterly Conference Call Information

Management will host a quarterly conference call to discuss its third quarter ended September 30, 2023, results today at 4:30 p.m. Eastern Time. Investors may listen to the call by dialing 1-888-349-0136, if outside the U.S., by dialing 1-412-317-0459, requesting to join the "PacBio Q3 Earnings Call". The call will be webcast live and available for replay at PacBio’s website at View Source

On October 31, 2023 HDT Bio Corp., a clinical-stage private company developing advanced RNA products to treat and prevent infectious diseases and cancer, reported that Dr. Steven Reed, Chief Executive Officer of HDT Bio, will present at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2023 Annual Meeting in San Diego, California, on November 3 (Press release, HDT Bio, OCT 31, 2023, View Source [SID1234636583]). Dr. Reed will deliver a presentation on the AMPLIFY vaccine platform as a novel technology for cancer immunoprevention.

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HDT’s AMPLIFY vaccine platform is based on its self-amplifying RNA (repRNA) combined with LION, its patented nanoparticle-based formulation technology. LION not only stabilizes, protects, and transports RNA during formulation and administration, but also enhances safety. This approach, compared to current RNA vaccines, allows for simplified manufacturing processes, improved temperature stability, and heightened safety, thereby enabling multivalency for more effective and efficient vaccine products.

"Cancer is a formidable adversary. Our presence at the SITC (Free SITC Whitepaper) 2023 Annual Meeting underscores our commitment to advancing the field of immunoprevention," said Dr. Steven Reed, CEO of HDT Bio. "With the AMPLIFY vaccine platform and products derived thereof, we are committed to transforming the landscape of cancer care, marking a monumental leap towards a future where cancer is no longer a looming threat but a preventable condition."

Details of the presentation are as follows:

Title: Novel Technologies for Cancer Immunoprevention
Presenter: Steven Reed, Ph.D.
Category: Immunoprevention of Cancer
Session: Concurrent Session 106c
Date: Friday, November 3 at 2:15 p.m. Pacific Time
Location: Ground Level, Exhibit Hall D, San Diego Convention Center

On October 31, 2023 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR) reported that preclinical research on LYMPHIR ("denileukin diftitox" or "E7777") was published today in Frontiers in Immunology1, a leading peer-reviewed journal in the immunology field (Press release, Citius Pharmaceuticals, OCT 31, 2023, View Source [SID1234636582]). The article reported positive results from a preclinical study that showed that denileukin diftitox in combination with an anti-PD-1 checkpoint inhibitor was more effective in the treatment of solid tumors (liver and colon) than either therapy alone. Data from this study contributed to the design and dosing regimen of two Phase 1 investigator-initiated studies currently underway at the University of Pittsburgh and the University of Minnesota.

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"We are excited to share the published results of this important animal study with the broader scientific community. The compelling results of this study showed that LYMPHIR administered in combination with an anti-PD-1 inhibitor, either sequentially or concurrently, led to significantly increased anti-tumor activity and prolonged survival compared to either treatment alone. Based on the strong signal of the study, two ongoing Phase 1 trials were initiated by investigators at leading cancer research institutions. We intend to share interim data from these studies when it becomes available. Importantly, the results of the preclinical study highlight LYMPHIR’s additional potential as a combination therapy in the treatment of solid tumors, which may allow Citius to explore substantially larger market opportunities for LYMPHIR in the future," stated Leonard Mazur, Chairman and CEO of Citius.

In recent years, one of the most important advances in solid tumor management has been the use of anti-PD-1/PDL1 antibody therapy. However, the effectiveness of treatment has varied, with not all tumors or patients responding with equivalent sensitivity to the treatment. The primary role of Treg-targeted cancer immunotherapy is to transiently deplete Treg cells. It is believed that regulatory T cells (Tregs) in the tumor microenvironment play an important role in patient resistance to anti-PD-1 immunotherapy. Targeting Tregs with LYMPHIR during treatment with anti-PD1/PDL1 checkpoint inhibitors may change the dynamics of the immune microenvironment, including anti-PD-1 sensitivity, in situations where Tregs are prominent. Consequently, the innovative preclinical study evaluated whether adding LYMPHIR improved the efficacy of anti-PD-1 antibody therapy. Syngeneic murine solid tumor models in colon cancer CT-26 and liver cancer H22 were used to conduct the study.

"This landmark study demonstrated that an increase in T-reg cell infiltration induced by anti-PD-1 treatment can be counterbalanced by their depletion by LYMPHIR, suggesting potential synergistic activity in a solid tumor model," stated Dr. Myron Czuczman, Chief Medical Officer of Citius. "We look forward to future results of ongoing human studies evaluating the potential benefit of Treg depletion by LYMPHIR in combination with checkpoint inhibitors and other immunomodulatory agents."

Key Study Findings

Targeting Tregs using LYMPHIR combined with anti-PD-1 (either sequentially or concurrently) demonstrated significant anti-tumor activity, and consistently targeted and transiently depleted Tregs in the tumor microenvironment
Combination treatment was more effective than monotherapy with either drug alone
Combination therapy was well-tolerated and significantly enhanced long-term survival in solid tumor-bearing animals
Mahdi, H. Woodall-Jappe, M., Singh, P., Czuczman, S., Targeting Regulatory T cells by E7777 enhances CD8 T-cell-mediated anti-tumor activity and extends survival benefit of anti-PD-1 in solid tumor models. Frontiers in Immunology. (Published online ahead of print, 2023 October 27).
About the Investigator Initiated Trials

A Phase 1 Study is underway at the University of Pittsburgh Medical Center (UPMC), Hillman Cancer Center. This study is an open label, Phase 1/1b study to investigate the safety and efficacy of a combined regimen of pembrolizumab with T-regulatory cell depletion via denileukin diftitox in patients diagnosed with recurrent or metastatic solid tumors. (Title: The efficacy of T-regulatory cell depletion with E7777 combined with immune checkpoint inhibitor, pembrolizumab, in recurrent or metastatic solid tumors: Phase I/II Study. NCT05200559). The study consists of two parts. Part I is a dose escalation study of four cohorts (3,6,9,12 mcg of LYMPHIR) and is expected to enroll 18-30 patients. Part II is a dose expansion study of approximately 40 patients to evaluate the safety and tolerability of the recommended combination dose of LYMPHIR and pembrolizumab (to include ovarian cancer and MSI-H cancer cohorts). The study will also investigate the alteration of the immune microenvironment within tumors and peripheral blood. Secondary endpoints include the objective response (complete response plus partial response), progression-free survival, and overall survival.

A Phase 1 trial has also been initiated at the University of Minnesota, Masonic Cancer Center. This study is a single-arm open-label trial which has an estimated enrollment of 20 participants who will be administered denileukin diftitox prior to receiving Chimeric Antigen Receptor (CAR-T) therapies. The Phase 1 study consists of two components: dose finding to establish a maximum tolerated dose (MTD) of denileukin diftitox in combination with CAR-T therapies and an extension component to provide an estimate of efficacy at that MTD. (Title: Phase I/II Trial Using E7777 to Enhance Regulatory T-Cell Depletion Prior to CAR-T Therapy for Relapsed/Refractory B-Cell Lymphoma (DLBCL) NCT04855253).

On October 31, 2023 Compugen Ltd. (Nasdaq: CGEN) (TASE:CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported that it will present new data on its lead pre-clinical asset COM503, a potential first-in-class anti-IL18BP antibody in an oral presentation at the 38th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper), taking place 3-5 November 2023, San Diego, CA (Press release, Compugen, OCT 31, 2023, View Source [SID1234636581]). Abstracts have been released by SITC (Free SITC Whitepaper) today.

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"We are very excited to be presenting new pre-clinical data on COM503, a novel approach to harness cytokine biology to treat cancer, discovered using our proprietary computational discovery platform," said Anat Cohen-Dayag., Ph.D., President, and CEO at Compugen. "Cytokines are powerful therapeutic tools; however, there is a challenge of giving them systemically at levels high enough to reach and modulate the tumor microenvironment without causing systemic side effects. This is the reason for some recent clinical failures in this space."

Eran Ophir, Ph.D., Chief Scientific Officer at Compugen, added, "At SITC (Free SITC Whitepaper), during both oral and poster presentations, we will present evidence supporting our approach to harness IL-18 biology to fight cancer and address the challenges that led to past failures with the systemic dosing of cytokines. We show that following antibody blockade of IL-18BP, endogenous IL-18 levels in human tumors are sufficient to provoke an anti-tumor immune response. In addition, we show that administering an anti-IL18BP antibody is expected to have a better therapeutic window than administering an engineered IL-18 cytokine. Our data suggest that our anti-IL18BP antibody approach has a leading edge in inhibiting tumor growth while avoiding peripheral toxicity associated with administration of a cytokine. We look forward to discussing the data at SITC (Free SITC Whitepaper) over the coming days and the IND filing in 2024."

Key data that will be presented at SITC (Free SITC Whitepaper) include:

Antibody inhibition of IL-18BP prevented tumor growth across multiple mouse tumor models.
Antibody induced inhibition of IL-18BP resulted in a significant increase in functional immune-cells such as the effector T-cells and induced a T cell clonal expansion in the tumor, as well as an immune memory response.
Engineered IL-18 cytokine generated peripheral inflammatory responses evident by increased serum cytokines in contrast with an anti-IL-18BP antibody approach which modulated the tumor microenvironment without affecting the periphery.
Oral presentation details:

Abstract Title: Harnessing natural IL-18 activity through IL-18BP blockade reshapes the tumor microenvironment for potent anti-tumor immune response
Abstract number: 550
Session: Cytokines in Cancer
Date: Friday, November 3, 2023
Time: 3:30 PM – 5:10 PM PDT
The data will also be presented as a poster on Saturday, November 4, 2023

The abstract is available on the publication section of Compugen’s website at www.cgen.com as well as part of the JITC supplement. The poster and presentation will be made available on the publication section of Compugen’s website at www.cgen.com following presentation.