Immunomic Therapeutics Reports Preclinical Data on Its MCPγV-LT and Her2/Neu DNA Vaccines at the American Association for Cancer Research (AACR) Annual Meeting 2022

On April 8, 2022 Immunomic Therapeutics, Inc., ("ITI"), a privately-held clinical-stage biotechnology company pioneering the study of LAMP-mediated nucleic acid-based immunotherapy, reported that the data from two posters are being presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 held in New Orleans, Louisiana, from April 8-13 (Press release, Immunomic Therapeutics, APR 8, 2022, View Source [SID1234611758]). These posters to be presented at the AACR (Free AACR Whitepaper) Meeting focus on the investigational nucleic acid platform, UNITE (UNiversal Intracellular Targeted Expression) for two vaccines, ITI-3000 for Merkel cell carcinoma (targeting the large T antigen of the Merkel cell polyomavirus) and Her2/Neu-LAMP DNA vaccine, both of which fuse a tumor associated antigen with lysosomal associated membrane protein 1 (LAMP-1). This proprietary lysosomal targeting technology results in enhanced antigen presentation and a balanced T cell response.

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The posters are accessible on the "Posters" section of Immunomic Therapeutics’ website at: View Source

Highlights from the presentations are as follows:

Poster Number: 10

Title: "LAMP1 targeting of the large T antigen of Merkel cell polyomavirus elicits potent CD4+ T cell responses, tumor inhibition, and provides rationale for first-in-human trial"

Authors: Claire Buchta Rosean, PhD1; David M. Koelle, MD2; Paul Nghiem, MD, PhD3, Mohan Karkada, PhD1, Teri Heiland, PhD1

1Immunomic Therapeutics Inc., Rockville, MD, USA 2Department of Medicine/Division of Allergy and Infectious Diseases, University of Washington, Seattle, WA, USA 3Department of Medicine, Department of Dermatology, University of Washington, Seattle, WA, USA

This presentation reports results of pre-clinical mouse studies of a cancer vaccine that promotes potent, antigen-specific CD4 T cell responses to MCPγV-LT. The majority of Merkel cell carcinomas (MCC), a rare and highly aggressive type of neuroendocrine skin cancer, are associated with Merkel cell polyomavirus (MCPyV) infection. MCPyV integrates into the host genome, resulting in expression of a truncated form of the viral large T antigen (LT) in infected cells, and makes LT an attractive target for therapeutic cancer vaccines.
To activate antigen-specific CD4 T cells in vivo, the investigators utilized the nucleic acid vaccine platform, UNITE (UNiversal Intracellular Targeted Expression), which fuses a tumor-associated antigen with lysosomal-associated membrane protein 1 (LAMP1). LTS220A, encoding a mutated form of MCPγV-LT that diminishes its pro-oncogenic properties, was introduced into the UNITE platform. Mice were vaccinated with 40µg of ITI-3000 or control vector via intradermal injection and electroporation. A B16F10 melanoma line stably transduced to express LTS220A (B16-LT) was injected subcutaneously in the right flank.
Vaccination with LTS220A-UNITE (ITI-3000) induced antigen-specific CD4 Th1 cells that was associated with delayed tumor growth and enhanced survival in treated mice in both the prophylactic and therapeutic settings. This effect was dependent on the CD4 T cells ability to produce IFNγ (interferon gamma), suggesting the potential mechanism of action of ITI-3000. In addition, ITI-3000 induced changes in the tumor microenvironment, including increased numbers of CD4 and CD8 T cells, reduced frequency of myeloid cells, and enhanced pro-inflammatory cytokine production.
Poster Number: 3

Title: "Harnessing soluble CD40L to enhance anti-tumor efficacy of Her2-LAMP DNA vaccine using UNITE platform"

Authors: Wei Shen1, Renhuan Xu2, Yun-Ting Kao1, Mohan Karkada1, and Teri Heiland1

1Immunomic Therapeutics, 15010 Broschart Road, Rockville, MD 20850 2ARV-Tech, Rockville, MD 20852

This presentation reports results of preclinical mouse studies on a HER2/Neu-LAMP DNA vaccine that included a bicistronic DNA construct in which both the tumor antigen (HER2) fused with LAMP-1 (lysosomal- associated membrane protein 1) and sCD40L were expressed separately. CD40 ligand (CD40L; CD154) is a transmembrane protein expressed on the surface of activated T cells, particularly on CD4 T cells, which stimulates CD40-dependent activation of antigen-presenting cells (APCs), resulting in enhancement of T cell and antibody responses.
To elicit cellular and humoral responses, the investigators employed the proprietary UNITE (UNiversal Intracellular Targeted Expression)-VAX platform, which utilizes a plasmid DNA expressing TAAs and LAMP to deliver TAAs to the MHC II compartment, thus potentially enhancing both antibody generation and CD4+ T cell response. Mice were immunized intradermally with 20µg of control vector, HER2-LAMP, or bicistronic-HER2-LAMP-sCD40L.
Vaccination with HER2-LAMP-sCD40L was associated with the detection of soluble forms of CD40L in the supernatant of HER2-LAMP-sCD40L transfected 293T cells: this soluble CD40L may function to enhance vaccine immunogenicity. The HER2-LAMP-sCD40L DNA vaccine elicited a robust HER2- specific cellular and humoral response; of note, CD4 T cells were elevated as compared with CD8 T cells, suggesting that sCD40L preferentially acts on CD4 T cells in vivo. The HER2-LAMP-sCD40L DNA vaccine exhibited significant antitumor effect in a murine breast tumor model.
"Based on these preclinical findings, we are planning a first-in-human Phase 1 open-label study to evaluate the safety, tolerability, and immunogenicity of ITI-3000 in Merkel cell carcinoma patients," stated Dr. William Hearl, Chief Executive Officer of Immunomic Therapeutics. "This study will be our second clinical study utilizing our proprietary nucleic acid vaccine platform UNITE and underscores the potential for our platform to design and introduce novel cancer vaccines for hard-to-treat cancers."

"We are highly encouraged by the data from the preclinical development for Her2-LAMP-sCD40L vaccine," stated Teri Heiland, PhD, Chief Scientific Officer of Immunomic Therapeutics. "The prolonged survival noted in the murine model suggests that this novel bicistronic vaccine may be an effective strategy to promote anti-tumor efficacy in vivo for multiple HER2-expressing cancer types."

About UNITE

ITI’s investigational UNITE platform, UNiversal Intracellular Targeted Expression, leverages the ability to engineer chimeric proteins, directing antigen presenting cells to present antigens to the immune system through a targeted pathway and driving a robust immune response. UNITE vaccines are distinct in that they combine two components: nucleic acid constructs that encode a specific antigen and an endogenous Lysosomal Associated Membrane Protein (LAMP-1) sequence. The UNITE platform harnesses LAMP-1 as a means of presenting the vaccine target to the immune system, resulting in antibody production, inflammatory cytokine release, and establishing critical immunological memory, something that other vaccine approaches commonly lack. This approach could put UNITE technology at the crossroads of immunotherapies in multiple indications, including cancer, human allergy, animal health, and infectious disease. Preclinical data is currently being developed to explore whether LAMP-1 nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and used to create immune responses in tumor types that otherwise do not provoke an immune response.

Asana BioSciences Announces Dosing of First Patient in Phase 1 Trial of ASN004, a Novel 5T4-Antibody-Drug-Conjugate

On April 8, 2022 Asana BioSciences, LLC, a clinical stage biopharmaceutical company, reported that the first patient has been dosed in a Phase 1 trial for ASN004, an antibody drug conjugate (ADC) targeting the 5T4 oncofetal antigen (trophoblast glycoprotein), that is expressed in a wide range of malignant tumors but shows very limited expression in normal tissues (Press release, Asana BioSciences, APR 8, 2022, View Source [SID1234611757]). Higher 5T4 expression is associated with worse clinical outcome in non-small cell lung, head and neck, gastric, pancreatic, colorectal, and ovarian cancers.

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ASN004 incorporates a novel single-chain Fv-Fc antibody linked to a clinically validated Auristatin F hydroxypropylamide cytotoxic payload, and drug to antibody ratio (DAR) of approximately 10-12. In preclinical cancer models, a single dose of ASN004 achieved complete and durable tumor regression leading to tumor-free survivors.

"5T4 is an ideal antigen target for payload delivery to tumor with high internalization rate dynamics combined with the high DAR and represents a clear opportunity for precision guided therapy as needed" said Dr. Anthony Tolcher, Director of Clinical Research, Next Oncology, San Antonio, Texas and the Principal Investigator of the Phase 1, multicenter, dose-finding study, designed to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-tumor activity of ASN004, in patients with advanced solid tumors in the US (NCT04410224).

"We are delighted to initiate the clinical development of ASN004. The broad tumor expression profile of 5T4 qualifies it as a promising target for cancer therapies, with potential to be the best-in-class ADC," said Sandeep Gupta, PhD, Founder, President and Chief Executive Officer at Asana BioSciences. "ASN004 is the 6th novel program that Asana has successfully brought into the clinic, affirming our mission to provide new and better treatment options to patients," added Dr. Gupta.

NanoString Highlights Spatial Biology Research at the 2022 American Association of Cancer Research (AACR) Conference

On April 8, 2022 NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, reported the highlights of over 125 studies enabled by NanoString platforms that will be presented at the 2022 meeting of the American Association of Cancer Research (AACR) (Free AACR Whitepaper), which will be held April 8-13, 2022 (Press release, NanoString Technologies, APR 8, 2022, View Source [SID1234611756]).

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At AACR (Free AACR Whitepaper) 2022, over 110 posters and 15 oral presentations will showcase unique biological insights generated using NanoString Technologies’ platforms. This is the largest number of AACR (Free AACR Whitepaper) abstracts in a single year for NanoString and demonstrates the increasing adoption of spatial biology in cancer research. These abstracts include at least 36 studies exploring spatial biology using the CosMx Spatial Molecular Imager (SMI) and GeoMx Digital Spatial Profiler (DSP), and 59 studies exploring gene expression using the nCounter Analysis System.

"NanoString customers continue to make tremendous contributions to our understanding of cancer and the development of cures," said Brad Gray, president and CEO of NanoString. "We are particularly excited to highlight research studies that leverage our latest product offerings in spatial biology. These include simultaneous detection of RNA and protein on a single slide using GeoMx DSP and 100-plex protein imaging using the CosMx SMI."

Several studies and presentations that feature the use of NanoString’s spatial biology platforms are highlighted below.

Spotlight Theater, April 12, 10:00-11:00 am CST

NanoString will host a spotlight theater during AACR (Free AACR Whitepaper) on April 12 from 10:00-11:00 am CST, featuring Joseph Beechem, Ph.D., senior vice president of R&D and chief scientific officer for NanoString, presenting "Spatial Whole Transcriptome Profiling and Single Cell High-Plex Imaging: Quantum Leaps Forward in Studying Cancer." Arutha Kulasinghe, Ph.D., National Health and Medical Research Council (NHMRC) Research Fellow, Queensland University of Technology, will present "Spatial genomics in immunotherapy…and COVID-19." And Hargita Kaplan, director of Translational Medicine, Clinical Laboratory Operations, Atreca, Inc., will present "GeoMx DSP WTA Platform TCR Alpha Program Collaboration."

Poster: Multi-omic dissection of immunotherapy response in groups in non-small cell lung cancer
Arutha Kulasinghe, Ph.D., NHMRC Research Fellow, Queensland University of Technology
This study utilized spatial transcriptomics methods, including the GeoMx DSP and multiplex IHC, to define the tumor/stroma compartment specific proteome and transcriptome from a cohort of 2nd line immunotherapy treated non-small cell lung cancer patients.

Poster: Development of a custom high-plex GeoMx digital spatial profiler breast cancer protein biomarker assay
Christopher Corless, M.D., Ph.D., Oregon Health & Science University
This study’s goal was to develop a high-plex assay to simultaneously quantitate 27 established and novel breast cancer (BC)-related immune protein and phosphoprotein biomarkers using the GeoMx DSP. The custom assay performance was compared to standard, immunohistochemistry-based clinical BC biomarker assays (e.g., ER, PR, HER2) across the spectrum of BC subtypes and in multiple laboratories with statistically significant concordance.

Poster: A single-cell, spatial multi-omics atlas and cellular interactome of all major skin cancer types
Quan Ngyuen, Ph.D., University of Queensland
Alongside other multi-omics tools, CosMx SMI was used to generate the first spatial single-cell atlas and cellular interactome of all major skin cancer types (squamous cell carcinoma, basal cell carcinoma, and melanoma). Spatial transcriptomics with CosMx SMI reveals cell-type organization into distinct functional tissue layers and identifies cancer-, patient-, and region-specific differences in cell-cell interaction.

Poster: Subcellular characterization of over 100 proteins in FFPE tumor biopsies with CosMx Spatial Molecular Imager
Zach Lewis, Ph.D., NanoString Technologies
108 proteins were imaged in a breast cancer biopsy using the CosMx Spatial Molecular Imager, representing one of the highest-plex protein imaging studies. CosMx encoding and chemistry enable high-throughput, short-turnaround protein imaging studies on the same instrument as the CosMx single-molecule RNA assay.

Poster: Multi-omic analysis of whole transcriptome and high plex protein assays on a single FFPE slide
Shilah Bonnet, Ph.D., NanoString Technologies NanoString Technologies
The newly developed GeoMx Spatial Proteogenomic workflow enables the co-detection of protein (>100-plex) and RNA (up to 21,000-plex) from a single FFPE slide. The performance of the Proteogenomic workflow has been confirmed on various cell pellets and tissue types.

Oral: AACR (Free AACR Whitepaper) NextGen Star, Will Hwang, MD, PhD
April 11, 11:26-11:41 am CST
As an AACR (Free AACR Whitepaper) NextGen Star, Will Hwang, M.D., Ph.D., radiation oncologist and research fellow, Massachusetts General Hospital Cancer Center/Harvard Medical School, will present, "Multicellular spatial community featuring a novel neuronal-like malignant phenotype is enriched in pancreatic cancer after neoadjuvant chemotherapy and radiotherapy."

Repare Therapeutics to Present New Clinical and Preclinical Data at the 2022 AACR Annual Meeting

On April 8, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported it has been selected for an oral presentation of clinical data from its ongoing Phase 1/2 TRESR (Treatment Enabled by SNIPRx) trial of RP-3500, a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase), as well as two additional poster presentations at the upcoming 2022 AACR (Free AACR Whitepaper) Annual Meeting held in New Orleans on April 8-13, 2022 (Press release, Repare Therapeutics, APR 8, 2022, View Source [SID1234611754]).

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The Company also announced that it will host a conference call with accompanying slides for analysts and investors on Monday, April 11, 2022 at 6:30 p.m. Eastern Time. The call and presentation will include RP-3500 data presented at the 2022 AACR (Free AACR Whitepaper) Annual Meeting as well as additional data, representing an update from data in the Company’s abstract (referenced below), which reflects an earlier cut-off date.

"As with any new therapeutic class, differentiation among early product candidates is what ultimately determines success in the clinic and a path toward registration," commented Lloyd M. Segal, President and Chief Executive Officer of Repare. "In the context of data published as recently as today, we are confident that RP-3500 is a highly differentiated and potentially leading ATR inhibitor. We continue to draw on a wealth of data from the comprehensive Phase 1 monotherapy module of our Phase 1/2 TRESR trial of RP-3500 to begin establishing its therapeutic potential. We look forward to seeing these data presented at AACR (Free AACR Whitepaper) and discussing RP-3500’s ongoing and future development plans."

Oral Presentation Details on RP-3500 Phase 1/2 TRESR Trial Results:

Title: Genomic and pathologic determinants of response to RP-3500, an ataxia telangiectasia and Rad3-related inhibitor (ATRi), in patients (pts) with DNA damage repair (DDR) loss-of-function (LOF) mutant tumors in the Phase 1/2 TRESR trial
Presenter: Dr. Timothy Yap, MBBS, Ph.D., FRCP, Medical Director, Institute for Applied Cancer Science, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, MD Anderson Cancer Center, Houston, Texas
Abstract Number: CT030
Session Title: Clinical Trials Minisymposium – Patient Selection Strategies for Molecularly Targeted Agents in Clinical Trials
Date/ Time: Monday, Apr 11, 2022 at 3:05 PM – 3:15 PM CT

Poster Presentation Details on SNiPDx Panel for Synthetic Lethal Drug Discovery:

Title: Detection of biallelic loss of DNA repair genes in formalin-fixed, paraffin embedded (FFPE) tumor samples using a novel tumor-only sequencing panel with error correction
Presenter: Dominik Glodzik, Ph.D., Repare Therapeutics, Instructor in Biomedical Informatics, Harvard Medical School
Abstract Number: 2801
Session Title: Diagnostic Biomarkers
Date/ Time: Tuesday, April 12, 2022 at 9:00 AM CT

Poster Details on Preclinical Data for PKMYT1 Inhibitor RP-6306:

Title: RP-6306, a novel PKMYT1 inhibitor, demonstrates synthetic lethality as monotherapy and in combination with gemcitabine in CCNE1 amplified cancer cells
Presenter: Jimmy Fourtounis, Repare Therapeutics
Abstract Number: 5650
Session Title: Cell Cycle
Date/ Time: Friday, April 8, 2022 at 12:00 – 1:00 PM CT

Company Conference Call:

The Company will host a conference call with accompanying slides for analysts and investors on Monday, April 11, 2022 at 6:30 p.m. Eastern Time to further discuss the RP-3500 data presented at the 2022 AACR (Free AACR Whitepaper) Annual Meeting. Repare’s executive management team will be joined by Timothy Yap, MBBS, PhD, FRCP, Principal Investigator and Medical Director, Institute for Applied Cancer Science, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, MD Anderson Cancer Center, Houston, TX.

To access the call, please dial (877) 870-4263 (U.S. and Canada) or (412) 317-0790 (international) at least 10 minutes prior to the start time and ask to be joined to the Repare Therapeutics call. A live video webcast will be available in the Investor section of the Company’s website at View Source A webcast replay will also be archived for at least 30 days.

About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

Scorpion Therapeutics Presents Preclinical Proof-of-Concept Data Supporting the Development of Its Potentially Best-in-Class Mutant-Selective PI3Kα Inhibitor for the Treatment of Solid Tumors at the AACR Annual Meeting 2022

On April 8, 2022 Scorpion Therapeutics, Inc. ("Scorpion Therapeutics" or the "Company"), a pioneering oncology company redefining the frontier of precision medicine through its Precision Oncology 2.0 strategy, reported preclinical proof-of-concept data for STX-478, the Company’s first development candidate from its STX-H1047-PI3Kα program (Press release, Scorpion Therapeutics, APR 8, 2022, View Source [SID1234611753]). STX-478, formerly ST-814, is a highly differentiated, allosteric and central nervous system ("CNS")-penetrant small molecule specifically designed to inhibit the H1047X-mutant form of phosphoinositide 3-kinase alpha ("PI3Kα"), one of the most frequent variations in PI3Kα and a validated oncogenic disease driver across a variety of solid tumors. The data will be presented on Wednesday, April 13 in a late-breaking poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2022 in New Orleans, Louisiana.

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"We are pleased to announce preclinical proof-of-concept data for STX-478, a potential best-in-class mutant-selective PI3Kα inhibitor and our first development candidate," said Axel Hoos, M.D., Ph.D., chief executive officer of Scorpion Therapeutics. "These data are a perfect exemplar of our approach to deliver potential best-in-class molecules against validated targets through our Precision Oncology 2.0 strategy, which aims to optimize drug design to achieve target product profiles that directly address unmet patient needs. In only 18 months since program initiation, we believe we have developed a highly differentiated inhibitor of PI3Kα that overcomes the two major limitations of currently available agents – lack of selectivity for mutant PI3Kα and limited CNS penetrance – to potentially deliver superior efficacy across an array of tumor types. We are now focused on progressing STX-478 through IND-enabling studies and look forward to submitting our first IND application in the first half of 2023."

PI3Kα is one of the most highly mutated targets in cancer and mutations at the H1047X residue represent the highest frequency variants in PI3Kα. More than 55,000 people in the United States annually are diagnosed with cancers driven by mutations at this residue.1 The frequency of PI3Kα mutations in driving tumor progression has made the target a high priority for drug discovery. However, currently available treatments are limited by their inhibition of the normal, or wild-type, version of PI3Kα in healthy tissues, which can lead to significant metabolic side effects, including hyperglycemia, that hinder the ability of patients to tolerate these therapies. In addition, despite the fact that up to 50% of all solid tumor patients will develop significant morbidity and mortality from brain metastases, existing options have little to no CNS penetrance, and are therefore unable to treat tumors that have progressed into the brain.

In the preclinical data presented at AACR (Free AACR Whitepaper), STX-478 demonstrated exceptional selectivity for PI3Kα against the kinome, with activity against PI3Kα kinase domain mutants, including the most commonly occurring variant, H1047R. Additionally, STX-478 demonstrated activity across a spectrum of PI3KαH1047X cell line-derived xenograft models and tumor types, while avoiding the metabolic dysfunction induced by currently marketed therapies and achieving superior tumor growth inhibition. Pharmacokinetic analyses suggest that STX-478 benefits from outstanding drug-like properties, with adequate CNS exposure to treat brain tumors or metastases. The predicted long half-life and minimum peak-to-trough plasma concentrations support once-daily dosing and a favorable therapeutic index in patients.

"Through our proprietary drug-hunting platform, we have developed an atomic-level understanding of the activating mutations in PI3Kα and successfully designed STX-478 to selectively target mutant PI3Kα, while avoiding the normal, or wild-type, form in healthy tissues," said Darrin Stuart, Ph.D., chief scientific officer of Scorpion Therapeutics. "Despite widespread recognition of PI3Kα as a clinically validated oncogene, currently marketed agents are not wild-type sparing and therefore can be associated with significant dose-limiting toxicities. The new data to be presented at AACR (Free AACR Whitepaper) demonstrate the potential of STX-478 to deliver superior safety, tolerability and efficacy, and we look forward to advancing our small molecule to deliver better solutions to patients with cancer."

Based on these data, Scorpion Therapeutics named STX-478 as the lead development candidate from its STX-H1047-PI3Kα program in March 2022. STX-478 is currently advancing through preclinical development with an investigational new drug application submission expected in the first half of 2023.