CymaBay Therapeutics to Present at the H.C. Wainwright BioConnect Investor Conference at NASDAQ

On April 17, 2023 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in the H.C. Wainwright BioConnect Investor Conference at NASDAQ (Press release, CymaBay Therapeutics, APR 17, 2023, View Source [SID1234630148]).

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H.C. Wainwright BioConnect Investor Conference at NASDAQ
Date: Tuesday, May 2
Time: 9:00 am Eastern Time
Format: Presentation
Webcast: View Source

Corvus Pharmaceuticals Presents New CPI-818 Data Demonstrating the Potential of ITK Inhibition as a Treatment for Solid and Hematologic Cancers at the American Association for Cancer Research (AACR) Annual Meeting

On April 17, 2023 Corvus Pharmaceuticals, Inc. (Nasdaq: CRVS), a clinical-stage biopharmaceutical company, reported new data for CPI-818, the Company’s ITK inhibitor, demonstrating its potential to treat a variety of solid and hematological cancers based on a novel immunotherapy mechanism of action (Press release, Corvus Pharmaceuticals, APR 17, 2023, View Source [SID1234630147]). The data will be presented today in a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, which is taking place April 14-19, 2023 in Orlando, FL.

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"The preclinical and laboratory data presented at AACR (Free AACR Whitepaper) demonstrates the potential of targeting ITK with CPI-818 to modulate T cell differentiation and enhance the immune system’s ability to kill both solid and hematological cancers," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "CPI-818 inhibited tumor growth in five different tumor models including colon, renal and melanoma cancers, and in both T and B cell lymphomas. This work builds upon the immunologic properties and anti-tumor activity already seen in our ongoing Phase 1/1b clinical trial in T cell lymphoma, and we now believe we can extend this approach to a wide range of solid tumors. Our data presented at AACR (Free AACR Whitepaper) shows that CPI-818’s novel mechanism of action involves multiple synergistic features including Th1 skewing, increases in T cell cytolytic capacity and reduction in T cell exhaustion. These properties are a result of the myriad of functions that ITK plays in T cell biology. Together, we believe these characteristics position CPI-818 to potentially lead the next generation of tumor immunotherapy if approved."

Dr. Miller added, "We are excited by the long-term potential of CPI-818 across a broad range of cancer indications, but in the near-term our main focus is continuing enrollment in our T cell lymphoma (TCL) Phase 1/1b clinical trial and meeting with the FDA to discuss a potential registration randomized Phase 3 clinical trial."

CPI-818 Preclinical Data Presented at AACR (Free AACR Whitepaper)
The CPI-818 preclinical data was presented by Lih-Yun Hsu, Ph.D., Director of Immunology, Corvus Pharmaceuticals, in a poster session (abstract #1813) today at the AACR (Free AACR Whitepaper) Annual Meeting. The key highlights from the poster, which is also available on the Publications and Presentations page of the Corvus website, include:

CPI-818 monotherapy (7 days oral administration) provided statistically significant inhibition of growth in established tumors in the following cancer models: CT26 colon cancer, RENCA kidney cancer, B16 melanoma, EL4 TCL and A20 B cell lymphoma.

Mechanism studies revealed that CD8 T cells were primarily involved in inhibiting growth in the CT26 colon cancer model and that CD8, CD4 T cells and NK cells were primarily involved in inhibiting growth in the EL4 TCL model.
Studies also showed that CPI-818 increased the cytolytic capacity of tumor infiltrating lymphocytes. These cells produce interferon gamma, tumor necrosis factor (TNF) and perforin, which are cytokines and effector molecules produced by killer T cells.

The preclinical data demonstrated that CPI-818 enhances the anti-tumor efficacy of anti-PD1 and anti-CTLA4 therapy in animal models, including at suboptimal doses of these therapies. The triplet combination led to complete tumor elimination in 19 of 20 animals with established CT26 colon cancer tumors.

The preclinical data also demonstrated that CPI-818 reduced the expression of T cell exhaustion markers in animals treated with anti-PD1 and anti-CTLA4 therapy. T cell exhaustion is a phenomenon seen in tumors and chronic infections where prolonged exposure to antigens results in exhausted or ineffective T cell function and inability to eliminate tumors or infections. The down-regulation of these T cell exhaustion markers suggests that the inhibition of ITK by CPI-818 potentially produces favorable changes in the tumor microenvironment that could enhance anti-tumor immune system activity.

In vitro studies with normal human naïve CD4+ T cells demonstrated that CPI-818 suppressed T cell differentiation into Th2 cells and their production of Th2 derived cytokines IL4, IL5, IL9, IL10 and IL17, however it did not affect differentiation into Th1 cells or their production of the cytokine interferon gamma (IFNg). These findings were the result of Th1 skewing.

CPI-818 is currently being studied in a Phase 1/1b clinical trial as a single agent therapy in patients with relapsed TCL. The Company recently incorporated a minimum absolute lymphocyte count (ACL) as an eligibility criterion for enrollment in the clinical trial and anticipates presenting updated data from this trial at a medical meeting in the second quarter 2023. Based on the current enrollment rate of this clinical trial, the Company believes that the number of patients treated in this clinical trial would provide adequate safety and preliminary efficacy data to inform the design of a potential registration Phase 3 randomized clinical trial. As recommended by the FDA, the Company plans to meet with the FDA to discuss such a clinical trial; it is anticipated that this meeting will take place later this year.

Cogent Biosciences Presents Preclinical Data at AACR Annual Meeting Highlighting Precision Therapy Pipeline and Announces Initiation of Part 2 of the Registration-Enabling APEX Trial with Bezuclastinib in Advanced Systemic Mastocytosis

On April 17, 2023 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported updated preclinical data from the Company’s pipeline programs, including its novel EGFR-sparing brain-penetrant ErbB2 inhibitor and its next-generation selective fibroblast growth factor receptor 2 (FGFR2) program (Press release, Cogent Biosciences, APR 17, 2023, View Source [SID1234630146]). The data are being presented today in poster sessions at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2023 Annual Meeting. Cogent also announced the initiation of Part 2 of the Company’s ongoing APEX trial with bezuclastinib in Advanced Systemic Mastocytosis (AdvSM).

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"We are pleased to share our progress highlighting the Cogent Research Team in their ongoing effort to discover and advance potential best-in-class novel therapies for rare disease populations with high unmet medical need," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "Separately, based on bezuclastinib’s impressive and consistent clinical activity, safety and tolerability, we are also excited to announce the initiation of Part 2 of the APEX trial in AdvSM at a once-daily dose of 150 mg. We remain on track to provide clinical updates in the second half of 2023 from both APEX and SUMMIT, our trial of bezuclastinib in NonAdvSM patients, as well as updated clinical results from the PEAK lead-in trial in GIST patients this quarter."

AACR Poster Details

Title: Identification of a novel EGFR sparing brain penetrant ErbB2 inhibitor with activity against oncogenic ErbB2 mutations
Session Category: Molecular/Cellular Biology and Genetics
Session Title: Cell Cycle Progression, Checkpoint, and Telomeres
Session Date and Time: Monday Apr 17, 2023 9:00 AM – 12:30 PM ET
Location: Poster Section 10
Poster Board Number: 21
Published Abstract Number: 1440

Cogent is developing a potential best-in-class EGFR-sparing, brain-penetrant ErbB2 inhibitor that includes coverage of key mutations (YVMA, S310F, V842I, L755S) inadequately addressed by currently approved therapies. Activating mutations in the ErbB2 gene have been identified in multiple cancers and demonstrate a tumorigenic role similar to that of ErbB2 amplification. The poster presented today describes a series of novel compounds which potently inhibit several key ErbB2 mutations, including YVMA insertions, while sparing inhibition of EGFR. An exemplar compound from these series demonstrates advantages versus tucatinib, an approved benchmark compound, on tumor growth inhibition in a peripheral ErbB2 L755S driven mutant model, as well as in an ErbB2 driven intracranial model. Recent program advances with a novel chemotype have further improved ErbB2 mutational potency and selectivity, increased estimated brain penetrance to 40% and improved human whole blood stability to nearly 24 hours, suggesting a favorable profile for optimal clinical efficacy.

Title: In vivo characterization of a selective FGFR2 inhibitor with potency against gatekeeper and molecular brake mutations
Session Category: Molecular/Cellular Biology and Genetics
Session Title: Cell Cycle Progression, Checkpoint, and Telomeres
Session Date and Time: Monday Apr 17, 2023, 9:00 AM – 12:30 PM ET
Location: Poster Section 10
Poster Board Number: 20
Published Abstract Number: 1439

FGFR inhibitors are well-established oncogenic drivers in multiple diseases, but approved medicines fail to capture the full landscape of FGFR altered tumor types, with FGFR1-mediated hyperphosphatemia serving as the most common dose-limiting toxicity for pan-FGFR inhibitors. The poster presented today provides the first published evidence of a reversible, selective FGFR2 inhibitor with coverage of activating and emerging resistance mutations that spares inhibition of FGFR1. Preclinical data demonstrate a profile that delivers equipotent coverage across both key gatekeeper and molecular brake mutations (V564X, N549X) in FGFR2, while avoiding any evidence of FGFR1-linked hyperphosphatemia at efficacious plasma concentrations. In addition, as a reversible inhibitor, the Cogent program retains enzymatic potency against potential cysteine 491 mutations which are known to emerge as key resistance mutations in patients treated with covalent inhibitors.

APEX Part 2 Design Highlights

APEX is an ongoing Phase 2 trial evaluating bezuclastinib in patients with AdvSM. Part 2 will enroll approximately 65 patients treated at a once-daily 150 mg optimized dose and if successful, is designed to support regulatory submission. Enrollment is expected to be complete by the end of 2024. Several additional patient cohorts are anticipated during Part 2 of the APEX trial designed to demonstrate the breadth of AdvSM patients who may benefit from bezuclastinib, including:

Up to 20 patients with systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) treated concomitantly with bezuclastinib and AHN directed therapies, including azacitidine.
Up to 15 patients with inevaluable mIWG disease without C-findings.
Approximately 10 patients at a dose of 300 mg once-daily to explore the effect of exceeding IC90 KIT D816V engagement in AdvSM patients.
The predicted clinical exposure of the optimized 150 mg formulation of bezuclastinib is expected to surpass that of the previous formulation of bezuclastinib dosed at 100 mg twice-daily in APEX Part 1. Clinical data from approximately 30 patients from APEX Part 1 will be included in a presentation at a scientific meeting in the second half of 2023. Currently, clinical activity, safety and tolerability of patients dosed in APEX Part 1 remains consistent with results presented at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2022.

Cellectis Presents Preclinical Data on TALEN®-edited MUC1 CAR T-cells to Enhance Efficacy in Targeting Triple Negative Breast Cancer at the American Association for Cancer Research (AACR) Annual Meeting

On April 17, 2023 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported preclinical data on TALEN-edited MUC1 CAR T-cells at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 (Press release, Cellectis, APR 17, 2023, View Source [SID1234630145]). The data showed the capability of armored allogeneic MUC1 CAR T-cells to excel in the immune suppressive tumor micro-environment suggesting that they could be an effective option in treating relapsed and refractory triple negative breast cancer (TNBC) patients with limited therapeutic options.

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Tumor-associated MUC1 antigen is overexpressed in a large number of TNBC patients offering an effective discriminatory target for CAR T-cell therapy.

Cellectis’ MUC1 CAR T-cells are allogeneic and target Mucin 1 for TNBC and a variety of epithelial cancers. As other solid tumor targets can be plagued by safety concerns due to off-tumor expression, MUC1 is of high interest as its expression in normal epithelium is restricted to apical membranes. Additionally, MUC1 heavy glycosylation in normal tissue contrasts with Cellectis’ MUC1 CAR that is designed to recognize hypoglycosylated MUC1 present in cancer cells. Cellectis’ MUC1 CAR T-cells incorporate up to four TALEN-mediated knockouts and two knock-ins.

"We are very excited to share these encouraging preclinical data at AACR (Free AACR Whitepaper) that dissect how different attributes (knock-out or knock-in) contribute to the efficacy of our CAR T-cell product candidate" said Laurent Poirot, Ph.D., Senior Vice President Immunology at Cellectis. "We are convinced that allogeneic MUC1 CART-cells can be an effective option in the treatment of relapsed and refractory triple negative breast cancer."

The poster presentation at AACR (Free AACR Whitepaper) highlights the following preclinical data:

Intratumoral delivery of antigen-specific CAR T-cells resulted in effective control of tumor growth.
Results demonstrate superior activity of armored MUC1-CAR T-cells not only in tumor clearance but also in the recovery of normal glands.
Thus, innovative strategies can be used to allow CAR T-cell efficiency in the hostile tumor microenvironment while preserving safety.

Title: Deciphering the benefits of variable delivery routes and molecular armoring to enhance efficacy of MUC1-CAR T-cells in targeting triple-negative breast cancer

Session Title: Adoptive Cell Therapy 1

Presenter: Piril, Erler, Ph.D., Scientist II, Immuno-Oncology, Cellectis

Session Date and time: Sunday April 16, 2023, 1:30-5:00 PM ET
Location: Section 37
Poster Board Number: 14
Abstract Presentation Number: 899

Candel Therapeutics Receives FDA Fast Track Designation for CAN‑2409 in Non-Small Cell Lung Cancer

On April 17, 2023 Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing and commercializing viral immunotherapies to help patients fight cancer, reported that the U.S. Food and Drug Administration (FDA) granted fast track designation for its lead asset CAN-2409, an investigational viral immunotherapy, plus valacyclovir in combination with pembrolizumab in order to improve survival or delay progression in patients with stage III/IV non-small cell lung cancer (NSCLC) who are resistant to first line PD-(L)1 inhibitor therapy and who do not have activating molecular driver mutations or have progressed on directed molecular therapy (Press release, Candel Therapeutics, APR 17, 2023, View Source [SID1234630144]). Fast track designation is a process designed to facilitate the development and expedite the review of medicines to treat serious conditions and fulfill an unmet medical need. An investigational medicine that receives fast track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan and, if relevant criteria are met, eligibility for accelerated approval and priority review.

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"We are pleased with the FDA’s decision to grant fast track designation for CAN-2409, which reinforces our belief that our investigational medicine has meaningful potential to treat those living with late-stage lung cancer," said Paul Peter Tak, MD, PhD, FMedSci, President and CEO of Candel. "Despite progress made in recent years, there remains a significant unmet need for patients with lung cancer who have an inadequate response to standard of care immune checkpoint inhibitors. Fast track designation is intended to bring promising medicines to patients sooner and the receipt of this designation by the FDA reinforces our belief that CAN-2409 has the potential to improve outcomes for patients who lack other treatment options."

CAN-2409 is an investigational viral immunotherapy designed to stimulate an individualized, systemic immune response to the patient’s specific tumor. CAN-2409 plus valacyclovir in combination with continued PD-1/PD-L1 agents is being evaluated in an ongoing, open-label phase 2 clinical trial (NCT04495153) in patients with late-stage NSCLC.

During its R&D Day in December 2022, the Company reported data from 26 patients with NSCLC in its ongoing phase 2 clinical trial demonstrating evidence of local and systemic anti-tumor activity and showed a disease control rate of 77 percent (20/26) in patients entering the trial with disease progression despite previous immune checkpoint inhibitor treatment. Importantly, CAN-2409 demonstrated a favorable change in the trajectory of tumor growth in all patients for whom pre-enrollment scans were available as of October 21, 2022.

The Company expects to present updated clinical data from its phase 2 clinical trial in the third quarter of 2023.