ITI Reports Preclinical Data on Its MCP?V-LT and Her2/Neu DNA Vaccines at AACR 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 [SID1234611657]). 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.

Nkarta to Participate at Upcoming Investor Conference

On April 8, 2022 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported its participation at an upcoming investor conference (Press release, Nkarta, APR 8, 2022, View Source [SID1234611651]):

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21st Annual Needham Virtual Healthcare Conference
April 12, 2022
4:30 p.m. ET – presentation

A simultaneous webcast of the event will be available on the Investors section of Nkarta’s website, www.nkartatx.com, and a replay will be archived on the website for approximately four weeks.

InDex Pharmaceuticals Holding AB (publ) publishes Annual Report for 2021

On April 8, 2022 InDex Pharmaceuticals Holding AB (publ) reported the Annual Report for 2021 (Press release, InDex Pharmaceuticals, APR 8, 2022, View Source [SID1234611650]). The Annual Report is attached as a PDF and is available on the company’s website, View Source

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The Annual Report is mailed to shareholders and other stakeholders who specifically request it. Send request to [email protected], or by mail to InDex Pharmaceuticals Holding AB (publ), Berzelius väg 13, 171 65 Solna, Sweden.

Publication
The information was submitted for publication through the agency of the contact person set out above at 13:20 CET on April 8, 2022.

Emergent BioSolutions to Release First Quarter 2022 Financial Results and Conduct Conference Call on April 28, 2022

On April 8, 2022 Emergent BioSolutions Inc. (NYSE: EBS) reported that it will host a conference call on Thursday, April 28, 2022, at 5:00 pm eastern time to discuss the financial results for the first quarter of 2022, recent business developments, revenue guidance for the second quarter of 2022, and financial outlook for full year 2022 (Press release, Emergent BioSolutions, APR 8, 2022, View Source [SID1234611647]).

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This conference call can be accessed live by telephone or by webcast:

Webcast Information:
Visit View Source the live webcast.

A replay of the call can be accessed from the Investors page of the Emergent website.

Clovis Oncology Highlights Presentations on FAP-2286 and Rubraca® (Rucaparib) at the AACR Annual Meeting 2022

On April 8, 2022 Clovis Oncology, Inc. (NASDAQ: CLVS), reported that two abstracts featuring non-clinical data from studies evaluating FAP-2286 and Rubraca and a Trial-in-Progress poster detailing the Phase 1 portion of the LuMIERE study will be presented at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, being held April 8-13, 2022, in New Orleans (Press release, Clovis Oncology, APR 8, 2022, View Source [SID1234611646]).

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In a new non-clinical data analysis, FAP-2286 demonstrated potent affinity for human fibroblast activation protein (FAP) by biochemical and cell-based assays. Additionally, lutetium-177 (177Lu)-FAP-2286 showed longer tumor retention, resulting in greater tumor inhibition as compared to lutetium-177 (177Lu)-FAPI-46, a FAP-targeted radiotracer developed for therapeutic applications at the University of Heidelberg, Germany.

FAP-2286 is the first peptide-targeted radionuclide therapy (PTRT) and imaging agent targeting FAP to enter clinical development and the lead candidate in Clovis Oncology’s targeted radionuclide therapy (TRT) development program.

Approximately 50 patients will be enrolled in the Phase 1 portion of the multicenter, open-label LuMIERE trial, which is currently enrolling patients with advanced solid tumors (NCT04939610). The Phase 1 portion of the study is evaluating the safety of the investigational therapeutic agent 177Lu-FAP-2286 and will identify the recommended Phase 2 dose and schedule. The safety and tumor uptake of the imaging agent gallium-68 (68Ga)-FAP-2286 is also being evaluated. Once the Phase 2 therapeutic dose is determined, Phase 2 expansion cohorts in multiple tumor types are planned for later in 2022.

Separately, non-clinical data evaluating Rubraca efficacy in a panel of tumors with deleterious alterations in a core group of non-BRCA HRR genes showed responses similar to the efficacy observed in BRCA1/2-altered models. The goal of the studies was to investigate the in vitro and in vivo synthetic lethality activity of Rubraca in multiple cancer cell types and tumors harboring genetic or epigenetic alterations in non-BRCA HRR genes.

"The non-clinical data presented at AACR (Free AACR Whitepaper) further show the potential of FAP-2286, our first targeted radiotherapy candidate, as a therapeutic and imaging agent and we look forward to sharing initial clinical data from the Phase 1 portion of the LuMIERE study of FAP-2286 later this year," said Patrick J. Mahaffy, President and CEO of Clovis Oncology. "We also remain committed to a greater understanding how patients with tumors associated with different genetic alterations may benefit from treatment with Rubraca."

The following Clovis-sponsored posters and supplemental information will be available as of Friday, April 8 at 1:00 p.m. CDT at View Source

FAP-2286

Abstract #3317: Comparative biodistribution and radiotherapeutic efficacy of the fibroblast activation protein (FAP)-targeting agents FAP-2286 and FAPI-46

Lead Author: Dirk Zboralski, Ph.D., 3B Pharmaceuticals GmbH, Berlin, Germany
Poster Session: Preclinical Radiotherapeutics
Date/Time: April 12, 1:30 – 5:00 p.m. CDT
Key Takeaways: FAP-2286 demonstrated potent affinity for human FAP by biochemical and cell-based assays. 177Lu-FAP-2286 showed longer tumor retention, resulting in greater tumor inhibition as compared to 177Lu-FAPI-46. The prolonged tumor retention of FAP-2286 correlated with a higher intracellular accumulation.
Abstract #CT251: LuMIERE: A phase 1/2 study investigating safety, pharmacokinetics, dosimetry, and preliminary antitumor activity of 177Lu-FAP-2286 in patients with advanced or metastatic solid tumors

Lead Author: Jonathan McConathy, M.D., Ph.D., University of Alabama School of Medicine, Birmingham, AL.
Poster Session: Phase I Trials in Progress 2
Date/Time: April 13, 9:00 a.m. – 12:30 p.m. CDT
Key Takeaways: Peptide-targeted radionuclide therapy directed toward FAP with the agent FAP-2286 has demonstrated antitumor activity in preclinical studies. LuMIERE (NCT04939610) is an ongoing phase 1/2, multicenter, open-label study evaluating safety and tolerability, pharmacokinetics (PK), dosimetry, and preliminary activity of the therapeutic agent 177Lu-FAP-2286 in patients with a FAP-expressing solid tumor. Safety and tumor uptake of the imaging agent 68Ga-FAP-2286 are also being evaluated.
For more information about FAP-2286, Targeted Radionuclide Therapy (TRT), or Clovis’ TRT development program, click here.

Rubraca

Abstract #1260: Nonclinical evaluation of rucaparib in tumors with mutations in non-BRCA1/2 homologous recombination repair (HRR) genes

Lead Author: Liliane Robillard, M.S., Clovis Oncology, Inc., Boulder, CO.
Poster Session: Biomarkers Predictive of Therapeutic Benefit 2
Date/Time: April 11, 9:00 a.m. – 12:30 p.m. CDT
Key Takeaways: In vitro small interfering ribonucleic acid (siRNA) knockdown of a subset of HRR genes showed synthetic lethality with rucaparib treatment in ovarian and prostate cancer cell lines. Rucaparib efficacy observed in patient-derived tumor xenograft (PDX) models with deleterious alterations in a core group of non-BRCA HRR genes was similar to the efficacy observed in
BRCA1/2
-altered models across different solid tumors, with enhanced sensitivity in tumors with biallelic alterations.
About FAP-2286

FAP-2286 is a clinical candidate under investigation as a peptide-targeted radionuclide therapy (PTRT) and imaging agent targeting fibroblast activation protein (FAP). FAP-2286 consists of two functional elements; a targeting peptide that binds to FAP and a site that can be used to attach radioactive isotopes for imaging and therapeutic use. High FAP expression has been shown in pancreatic ductal adenocarcinoma, salivary gland, mesothelioma, colon, bladder, sarcoma, squamous non–small cell lung, squamous head and neck cancers, and cancer of unknown primary. High FAP expression was detected in both primary and metastatic tumor samples and was independent of tumor stage or grade. Clovis holds US and global rights for FAP-2286 excluding Europe, Russia, Turkey, and Israel.

FAP-2286 is an unlicensed medical product.

About Targeted Radionuclide Therapy

Targeted radionuclide therapy is an emerging class of cancer therapeutics, which seeks to deliver radiation directly to the tumor while minimizing delivery of radiation to normal tissue. Targeted radionuclides are created by linking radioactive isotopes, also known as radionuclides, to targeting molecules (e.g., peptides, antibodies, small molecules) that can bind specifically to tumor cells or other cells in the tumor environment. Based on the radioactive isotope selected, the resulting agent can be used to image and/or treat certain types of cancer. Agents that can be adapted for both therapeutic and imaging use are known as "theranostics." Clovis, together with licensing partner 3B Pharmaceuticals, is developing a pipeline of novel, targeted radiotherapies for cancer treatment and imaging, including its lead candidate, FAP-2286, an investigational peptide-targeted radionuclide therapeutic (PTRT) and imaging agent, as well as three additional discovery-stage compounds.

About the LuMIERE Clinical Study

LuMIERE is a Phase 1/2 study evaluating FAP-2286 as a peptide-targeted radionuclide therapy (PTRT) targeting fibroblast activation protein, or FAP, in patients with advanced solid tumors. The Phase 1 portion of the LuMIERE study is evaluating the safety of the investigational therapeutic agent and will identify the recommended Phase 2 dose and schedule of lutetium-177 labeled FAP-2286 (177Lu-FAP-2286). FAP-2286 labeled with gallium-68 (68Ga-FAP-2286) will be utilized as an investigational imaging agent to identify patients with FAP-positive tumors appropriate for treatment with the therapeutic agent. Once the Phase 2 dose is determined, Phase 2 expansion cohorts are planned in multiple tumor types.

About Rubraca (rucaparib)

Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.

Rubraca is an unlicensed medical product outside of the US and Europe

Rubraca US FDA Approved Indications

Ovarian Cancer

Rubraca is indicated for the maintenance treatment of adult women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.

Rubraca is indicated for the treatment of adult women with a deleterious BRCA mutation (germline and/or somatic)-associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies. Select patients for therapy based on an FDA-approved companion diagnostic for Rubraca.

Prostate Cancer

Rubraca is indicated for the treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy. This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Select Important Safety Information

Myelodysplastic Syndrome (MDS)/Acute Myeloid Leukemia (AML) occur in patients treated with Rubraca, and are potentially fatal adverse reactions. In 1146 treated patients, MDS/AML occurred in 20 patients (1.7%), including those in long term follow-up. Of these, 8 occurred during treatment or during the 28 day safety follow-up (0.7%). The duration of Rubraca treatment prior to the diagnosis of MDS/AML ranged from 1 month to approximately 53 months. The cases were typical of secondary MDS/cancer therapy-related AML; in all cases, patients had received previous platinum-containing regimens and/or other DNA damaging agents.

Do not start Rubraca until patients have recovered from hematological toxicity caused by previous chemotherapy (≤ Grade 1). Monitor complete blood counts for cytopenia at baseline and monthly thereafter for clinically significant changes during treatment. For prolonged hematological toxicities (> 4 weeks), interrupt Rubraca or reduce dose and monitor blood counts weekly until recovery. If the levels have not recovered to Grade 1 or less after 4 weeks or if MDS/AML is suspected, refer the patient to a hematologist for further investigations, including bone marrow analysis and blood sample for cytogenetics. If MDS/AML is confirmed, discontinue Rubraca.

Based on its mechanism of action and findings from animal studies, Rubraca can cause fetal harm when administered to a pregnant woman. Apprise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months following the last dose of Rubraca. For males on Rubraca treatment who have female partners of reproductive potential or who are pregnant, effective contraception should be used during treatment and for 3 months following the last dose of Rubraca.

Most common adverse reactions in ARIEL3 (≥ 20%; Grade 1-4) were nausea (76%), fatigue/asthenia (73%), abdominal pain/distention (46%), rash (43%), dysgeusia (40%), anemia (39%), AST/ALT elevation (38%), constipation (37%), vomiting (37%), diarrhea (32%), thrombocytopenia (29%), nasopharyngitis/upper respiratory tract infection (29%), stomatitis (28%), decreased appetite (23%), and neutropenia (20%).

Most common adverse reactions in Study 10 and ARIEL2 (≥ 20%; Grade 1-4) were nausea (77%), asthenia/fatigue (77%), vomiting (46%), anemia (44%), constipation (40%), dysgeusia (39%), decreased appetite (39%), diarrhea (34%), abdominal pain (32%), dyspnea (21%), and thrombocytopenia (21%).

Co-administration of rucaparib can increase the systemic exposure of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, which may increase the risk of toxicities of these drugs. Adjust dosage of CYP1A2, CYP3A, CYP2C9, or CYP2C19 substrates, if clinically indicated. If co-administration with warfarin (a CYP2C9 substrate) cannot be avoided, consider increasing frequency of international normalized ratio (INR) monitoring.

Because of the potential for serious adverse reactions in breast-fed children from Rubraca, advise lactating women not to breastfeed during treatment with Rubraca and for 2 weeks after the last dose.