On October 22, 2024 Oncternal Therapeutics, Inc. (Nasdaq: ONCT) (the "Company") reported updated data from its Phase 1/2 Study of ONCT-534 for the treatment of patients with relapsed or refractory metastatic Castration-Resistant Prostate Cancer (mCRPC) (Press release, Oncternal Therapeutics, OCT 22, 2024, View Source [SID1234647308]).

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Based on initial pharmacokinetic results, two additional dosing cohorts with twice daily (BID) oral dosing of ONCT-534 had been incorporated in the Phase 1/2 study ONCT-534-101 (NCT05917470). Overall, fifteen patients received ONCT-534 once daily (QD) in six dosing cohorts and six patients received ONCT-534 BID in two dosing cohorts. Based on a data cut off of September 30, 2024, the BID dosing schedule was well tolerated, with no related Grade 3 or higher toxicities. One patient, who experienced a rising PSA on ONCT-534 at 160 mg BID, had a subsequent 50% reduction in PSA after four weeks of ONCT-534 at 300 mg BID, and at the same time the CAT Scan showed a 16% reduction in target lesions compared to baseline. Enumeration and biomarker analysis of circulating tumor cells (CTCs) showed promising effects on expression of androgen receptor (AR)-regulated genes, and AR nuclear translocation in six additional patients. CTC analysis also showed that some patients who did not respond to ONCT-534 had prostate cancer that had developed neuroendocrine features, which are associated with AR independent disease.

"While we still believe the decision to discontinue the ONCT-534-101 clinical trial remains the correct one in the current biotechnology environment, the updated clinical results highlight the potential of ONCT-534 in prostate cancer. We believe there is value in exploring BID dosing further, as well as studying ONCT-534 in earlier lines of therapy for advanced prostate cancer," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "We continue to explore strategic alternatives for our product candidates, including ONCT-534, ONCT-808, zilovertamab and ONCT-216 in an ongoing effort to maximize value to our shareholders."

On October 22, 2024 NanoCell Therapeutics, Inc. ("NanoCell"), a company developing in-vivo cell engineering solutions based on its proprietary non-viral, DNA-based gene therapy platform, reported that the team will present at several upcoming investor and scientific conferences (Press release, NanoCell Therapeutics, OCT 22, 2024, View Source [SID1234647306]).

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Initial data from the company’s ongoing preclinical studies on targeted lipid nanoparticle (tLNP) technology for in vivo CAR-T cell generation will be presented at the ESGCT by Dr. Jacek Lubelski, NanoCell’s Chief Technology Officer. The presentation will highlight preclinical results demonstrating the ability of the company’s novel tLNP technology to generate persistent CAR-T cells in vivo, showing tumor control and extended survival in a human PBMC engrafted mouse model of B-cell leukemia.

At the SITC (Free SITC Whitepaper) 39th Annual Meeting and the SITC (Free SITC Whitepaper) Immune Engineering Workshop in Houston, Texas, Dr. Lubelski will present new data from the company’s non-viral vector platform research program.

NanoCell will participate in two upcoming investor conferences. Dr. Maurits Geerlings, CEO, will join a panel discussion and host investor meetings at the Longwood Healthcare Leaders Boston CEO Forum (October 28-29, 2024), followed by one-on-one investor meetings at BioEurope in Stockholm (November 4-6, 2024).

Presentation Details:

European Society of Gene & Cell Therapy (ESGCT):
Oral Presentation: "tLNPs can effectively deliver DNA to T-cells and generate long-acting CAR-T cells in vivo"
Number: OR050
Session: Parallel Session 6c: Non Viral Vectors / Nanotechnology I
Location: Meeting Room 2
Timing: Wednesday, October 23rd, 2024,
Timing: 17:30-19:30

Presentation Details:

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting:
Poster Presentation
Title: "Efficient generation of long-lasting CAR-T cells in vivo using novel non-viral vector"
Abstract Number: 284
Timing: November 8-10, 2024
Location: Houston, TX

Additional Poster Abstract Presentation:

SITC Immune Engineering Workshop:
Titel: "Efficient generation of long-lasting CAR-T cells in vivo using novel non-viral vector"
Timing: November 7, 2024
Location: Houston, TX

Investor Conferences

Event Details:
Longwood Healthcare Leaders Boston CEO Forum
Investor 1:1 Meetings and Panel
Title: Innovation in Cell Therapy
Timing: October 28-29, 2024
Venue: Mandarin Oriental Boston, 776 Boylston Street, Boston, MA, USA

Event Details:
BioEurope
Investor 1:1 Meetings
Company Overview
Timing: November 4–6, 2024
Venue: Stockholmsmässan, Mässvägen 1, 125 30 Älvsjö, Stockholm, Sweden

On October 22, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage radiopharmaceutical company focused on developing innovative treatments for cancer patients, reported data from the clinical and preclinical development of its novel first-in-class lead radiopharma program based on MNPR-101 at the European Association of Nuclear Medicine (EANM) 2024 Annual Congress held in Hamburg, Germany (Press release, Monopar Therapeutics, OCT 22, 2024, View Source [SID1234647304]). MNPR-101-Lu radiation dosimetry analytics using human data from MNPR-101-Zr show a favorable organ safety profile at high Lu-177 therapeutic dose levels. The slides for Monopar’s oral presentation can be found at the following link: View Source

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Monopar’s presentation, accepted as a "Top-Rated Oral Presentation" within the Scientific Program, illustrates the potential of the urokinase plasminogen activator receptor (uPAR) as a promising radiopharma target in solid tumors. Preclinical and clinical data show favorable biodistribution, tumor uptake, and low off-target binding of Monopar’s uPAR-targeted radiopharmaceuticals MNPR-101-Zr and MNPR-101-Lu. "We were able to optimize our uPAR-targeted radiopharmaceuticals in preclinical studies, and the data show these efforts have translated directly into humans with encouraging tumor uptake. Even at the highest Lu-177 therapeutic antibody dose we are aware of in the clinic, we estimate a favorable radiation dosimetry safety profile for off-target effects such as bone marrow exposure," said Andrew Cittadine, Monopar’s Chief Operating Officer.

Further information about the MNPR-101-Lu Phase 1a trial is available at www.ClinicalTrials.gov under study identifier NCT06617169. Further information about the MNPR-101-Zr Phase 1 imaging and dosimetry clinical trial is available at www.ClinicalTrials.gov under study identifier NCT06337084.

On October 22, 2024 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 that they will be showcasing pre-clinical data that permit the design of an efficient and specific TALE base editors (TALEB) as well as a process to enhance the efficacy of non-viral gene insertion in hematopoietic stem and progenitor cells (HSPCs) at the European Society of Cell and Gene Therapy 31st annual congress, that will take place on October 22-25, 2024, in Roma, Italy (Press release, Cellectis, OCT 22, 2024, View Source [SID1234647303]).

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The data will be presented in two posters:

Controlling C-to-T editing with TALE base editors

Presenter: Alexandre Juillerat, Ph.D., Vice-President Gene Editing & NY Lab Head at Cellectis

Date/Time: Thursday, October 24 from 2:00pm to 3:30pm CET

Poster number: P0666

TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI). The C-to-T class of TALEB edits double strand DNA by converting a cytosine (C) to a thymine (T) via the formation of an uracil intermediate.
Cellectis recently developed a strategy that allows the comprehensive characterization of C-to-T conversion efficiencies within the editing window. This method also takes advantage of a highly precise and efficient TALEN-mediated ssODN knock-in in primary T cells to assess how target composition and spacer variations affect TALEB activity/efficiency.
The datasets obtained in this study enhanced our understanding of TALEB and permitted the design of efficient and specific tools that could be compatible with the potential development of therapeutic applications.

Circular Single-Stranded DNA Enables Efficient TALEN-Mediated Gene Insertion in Long Term HSC

Presenter: Julien Valton, Ph.D., Vice-President Gene Therapy at Cellectis

Date/Time: Thursday, October 24 from 2:00pm to 3:30pm CET

Poster number: P0585

Non-viral alternatives such as linear single-stranded DNA (LssDNA) and circular single-stranded DNA (CssDNA) are emerging as promising options to vectorized DNA donor template for nuclease-mediated gene insertion in hematopoietic stem and progenitor cells (HSPCs) used for gene therapy applications.
Capitalizing on its TALEN technology, Cellectis has devised a gene editing process that incorporates non-viral DNA donor template delivery (LssDNA or CssDNA) to enhance gene insertion in HSPCs.
The circularization of ssDNA increases gene insertion rates in long term HSCs and has the potential to enhance their engraftment capacity in preclinical murine model, thereby to facilitate the advancement of next-generation cell therapies. This research marks a crucial step towards enhancing the efficacy of non-viral gene therapy.

On October 22, 2024 BullFrog AI, Inc. (NASDAQ: BFRG; BFRGW) ("BullFrog AI" or the "Company"), a technology-enabled drug development company using artificial intelligence (AI) and machine learning to enable the successful development of pharmaceuticals and biologics, reported its VP Artificial Intelligence, Enrique García-Rivera, Ph.D., will present at Google’s inaugural Cancer AI Symposium on October 30, 2024, at the Boston Center for the Arts (Press release, Bullfrog AI, OCT 22, 2024, View Source [SID1234647302]).

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Dr. García-Rivera’s presentation will showcase BullFrog AI’s groundbreaking use of artificial intelligence in drug discovery and development. Highlighting the Company’s proprietary bfLEAP platform, the presentation will explore how multimodal biological data—including genomics, transcriptomics, and clinical data—are integrated to accelerate the drug development process.

A key aspect of the presentation will be how bfLEAP is applied to cancer research, leveraging AI to identify complex biological patterns that contribute to disease progression and treatment resistance. Dr. García-Rivera will also reference BullFrog AI’s collaboration with the Lieber Institute for Brain Development as a prime example of how bfLEAP is being utilized across various therapeutic areas. The collaboration, which focuses on neurological disorders such as bipolar disorder, underscores the platform’s versatility and potential to drive innovation across multiple domains, including cancer.

Dr. García-Rivera will also introduce BullFrog AI’s novel "AlgoLLM" system for gene prioritization, which revolutionizes the identification of high-priority targets in drug development by harnessing large language models. This innovative AI tool is critical for streamlining the discovery of actionable insights in neuropsychiatric disorders and other diseases, advancing the Company’s mission to develop more effective and personalized treatments.