On May 31, 2023 OncoNano Medicine, Inc. reported that it will present positive final results from its Phase 2 study of pegsitacianine, a pH-sensitive fluorescent nanoprobe for enhanced intra-operative visualization of cancer, in a podium presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting 2023 (Press release, OncoNano Medicine, MAY 31, 2023, View Source [SID1234632279]). Results of the study show that acidic pH is a viable marker and can be used for tumor targeting by the company’s ON-BOARD micelle platform.

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Pegsitacianine has been studied in more than 135 patients across multiple tumor types, including peritoneal metastatic, breast, head and neck, colorectal, esophageal, ovarian and prostate cancers. Results show that pegsitacianine is able to localize different tumor types regardless of their histology or anatomic location. The ON-BOARD platform, therefore, is able to target solid tumors, deploying its payload specifically within the acidic tumor microenvironment (TME).

"We are encouraged by this data as it demonstrates the potential of pegsitacianine to augment the visualization of cancer during an operation, which has the potential to be a significant advance in real-time surgical imaging," said Kartik Krishnan, M.D., Ph.D., Chief Medical Officer of OncoNano Medicine. "We look forward to taking pegsitacianine to the next levels of development and continue our mission to diagnose and treat cancer with high specificity."

The results of this Phase 2 study reveal that pegsitacianine was well tolerated and that 20 of 40 evaluable patients (50%) had pathology-confirmed disease resected, that was not resected in the planned standard of care surgery, under pegsitacianine guidance. Approximately 25% of patients experienced a pegsitacianine-related adverse event, which was a transient, selfresolving, non-anaphylactic infusion-related reaction.

"We were highly encouraged with the results observed in this Phase 2 trial and look forward to validating these findings in the upcoming Phase 3 trial," said Patrick Wagner, M.D., lead author and Director of Complex General Surgical Oncology, Allegheny Health Network Cancer Institute. "The results demonstrate that acidic pH is a viable tumor agnostic marker and highlight pegsitacianine’s ability to target different tumor types regardless of their genotype or anatomic location."

"This Phase 2 data brings new hope to both clinicians and patients," said Charles M. Balch, M.D., former EVP and Chief Executive Officer of ASCO (Free ASCO Whitepaper) and present Chair of the Scientific Advisory Board at OncoNano Medicine. "Having demonstrated the capacity to identify tumors that were previously not clinically visible to surgeons, pegsitacianine has the potential to improve the accuracy of staging and surgical outcomes, while reducing operating time for several types of abdominal cancers. Newer versions in the pipeline have great potential to expand therapeutic targets and enable new combinations of drugs previously hindered by toxicity."

Presentation Overview:

TITLE: Detection of peritoneal metastases during cytoreductive surgery using pegsitacianine, a pH sensitive imaging agent: Final results from a phase 2 study
PRESENTER: Patrick Wagner, MD, Director of Complex General Surgical Oncology, Allegheny Health Network Cancer Institute
DATE: June 5, 2023

On May 31, 2023 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported pre-clinical data from the Company’s multiple tumor-associated antigens (multiTAA)-specific T cell product candidate MT-601 in lymphoma cells, including CD19 CAR T refractory cells (Press release, Marker Therapeutics, MAY 31, 2023, View Source [SID1234632278])`.

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Although CAR T cell therapies targeting the CD19 antigen have gained acceptance as treatment for patients with relapsed/refractory lymphoma because of their significant benefit relative to the standard of care, up to 60% of patients treated with CAR T therapies relapse within a year (Chong EA et al, N Engl J Med, 2021).

MultiTAA-specific T cell therapy was investigated in the Baylor TACTAL study, which enrolled patients with both Hodgkin’s and non-Hodgkin’s lymphoma (Vasileiou et al, J Clin Oncol, 2021). The multiTAA-specific T cell product used in the TACTAL study was directed against five antigens and showed more durable clinical responses. For example, some patients who attained a complete remission (CR) with the multiTAA-specific T cell treatment were still in remission at the 72 months follow-up, suggesting a longer-lasting clinical benefit relative to published results for CD19 CAR T therapies.

Given the high probability of antigen-loss associated relapse seen with CD19 CAR T therapy, Marker believes that its multiTAA-specific T cell product may result in durable responses due to its ability to overcome antigen loss by targeting more than one antigen. Data from the TACTAL study has demonstrated that multiTAA-specific T cell products recognize cancer cells by up to five antigens presented on cancer cells and continue to kill even when cancer cells morph by downregulating the targeted antigen to escape recognition.

Marker recently started to evaluate the safety and efficacy of MT-601, a multiTAA-specific T cell product that recognizes six antigens, including WT-1, a TAA that was not part of the targeted antigens in the TACTAL study, in patients with non-Hodgkin’s lymphoma who have either relapsed after receiving anti-CD19 CAR T cell treatment or are ineligible for anti-CD19 CAR T cell treatment (ClinicalTrials.gov Identifier: NCT05798897). Marker believes that killing induced by a product directed against six TAAs instead of five TAAs can potentially provide a more durable solution. Prior to starting the clinical trial Marker assessed the killing capacity of MT-601 in vitro in a lymphoma cell line and demonstrated that MT-601 kills lymphoma cells regardless of their CD19 expression levels.

"We have recently developed a long-term in vitro model to monitor the interaction of T cells with cancerous cells. Data from a lymphoma cell line utilizing this model demonstrated that MT-601 inhibited growth of lymphoma cells as well as the growth of CD19 CAR resistant lymphoma cells," said Eric A. Smith, Ph.D., Director of Research and Development at Marker Therapeutics. Marker has posted further details about this preclinical study on the Investor Relations section of its website.

Dr. Smith continued "Specifically, we have developed an in vitro model which reproduces the CD19 antigen-negative tumor that causes relapse and observed the following:

· In this in vitro model, 98% of lymphoma cells were eliminated after initial administration of a CD19-targeting CAR T cell product.
· While the CAR T cells significantly controlled lymphoma cell growth, we observed that 3 weeks after the start of anti-CD19 CAR T cell administration, a population of lymphoma cells that were resistant to CD19 CAR T cell administration started to grow.
· These CD19 CAR resistant lymphoma cells were tested for CD19 expression and were shown to be negative for the CD19 surface antigen, which explained why they were no longer controlled with a second administration of anti-C19 CAR T cells, thus recapitulating the antigen-negative relapse observations in CAR relapsed/refractory lymphoma patients.
· However, when MT-601, with its broad antigen recognition (Survivin, NY-ESO-1, WT-1, PRAME, MAGE-A4, SSX2) was added to this anti-CD19 CAR T cell resistant cell population complete growth inhibition was observed.

These data highlight that MT-601 has the potential to eliminate tumors that are CD19 CAR T cell refractory, indicating that MT-601 might offer a viable therapeutic option for lymphoma patients that have relapsed from previous CAR T cell interventions."

"We are encouraged by the promising results of our pre-clinical study of MT-601," said Juan F. Vera, M.D., President and Chief Executive Officer at Marker Therapeutics. "These pre-clinical findings reinforce previous TACTAL clinical observations, highlighting the potential benefit of our innovative multiTAA-specific T cell therapy in CD19 CAR T refractory lymphoma patients. Our Investigational New Drug (IND) application for MT-601 for the treatment of patients with relapsed non-Hodgkin lymphoma has been cleared by the FDA and we are excited to further explore the potential benefits of MT-601 in our multicenter Phase 1 clinical trial, which was initiated in the first quarter of 2023. We are continuing our detailed review of the scope of clinical opportunities provided by MT-601 and look forward to providing an update about our clinical strategy and anticipated milestones as soon as possible."

About multiTAA-specific T cells

Marker’s multi-tumor associated antigen (multiTAA)-specific T cell platform is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s blood capable of recognizing a broad range of tumor antigens. Clinical trials that enrolled more than 180 patients with various hematological malignancies and solid tumors showed that the multiTAA-specific T cell product was well tolerated, demonstrated durable clinical responses, and consistent epitope spreading. The latter is typically not observed with other T cell therapies and enables the patient’s own T cells to expand, potentially contributing to a lasting anti-tumor effect. Unlike other cell therapies which require hospitalization and close monitoring, multiTAA-specific T cells are designed to be administered in an outpatient setting.

On May 31, 2023 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) ("Lexicon") reported that it has commenced an underwritten public offering to offer and sell, subject to market and other conditions, shares of its common stock, par value $0.001 (Press release, Lexicon Pharmaceuticals, MAY 31, 2023, View Source [SID1234632277]). In addition, Lexicon intends to grant the underwriters a 30-day option to purchase additional shares of common stock. There can be no assurance as to whether or when the proposed offering may be completed, or as to the actual size or terms of the proposed offering.

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Citigroup, Jefferies and Piper Sandler are acting as joint book-running managers for the proposed offering.

Concurrently with the closing of the underwritten public offering, Lexicon intends to sell shares of its common stock in a private placement to an affiliate of Invus, L.P., Lexicon’s largest stockholder, pursuant to its preemptive right under Lexicon’s Fifth Amended and Restated Certificate of Incorporation. Lexicon intends to grant the private placement purchaser an option to purchase, on a pro rata basis, additional shares of common stock to the extent the underwriters exercise their option to purchase additional common stock. Any shares of common stock offered pursuant to the concurrent private placement would not be registered under the Securities Act of 1933, as amended (the "Securities Act"). The closing of the underwritten public offering is not conditioned on the closing of the concurrent private placement.

Lexicon currently intends to use the net proceeds that it will receive from the proposed offering, together with its existing cash and cash equivalents and short-term investments, to fund the commercial launch of INPEFA (sotagliflozin). Lexicon expects to use any remaining net proceeds that it will receive from the proposed offering to fund the continued research and development of its drug candidates and for working capital and other general corporate purposes.

A shelf registration statement on Form S-3 relating to the proposed offering was filed with the U.S. Securities and Exchange Commission ("SEC") on August 6, 2021 and declared effective by the SEC on September 14, 2021 (File No. 333-258564). A preliminary prospectus supplement and accompanying prospectus relating to the proposed offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the preliminary prospectus supplement and accompanying prospectus may also be obtained from Citigroup, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717 (Tel: 800-831-9146); Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 821-7388; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at [email protected].

On May 31, 2023 IconOVir Bio, Inc. (IconOVir), a preclinical-stage biotechnology company pioneering the next generation of oncolytic virus (OV) therapy to improve the treatment of patients with cancer, reported that Mark McCamish, M.D., Ph.D., President and Chief Executive Officer of IconOVir, will present a corporate overview at the Jefferies Healthcare Conference on Wednesday, June 7, 2023 at 4:30 p.m. ET (1:30 p.m. PT) (Press release, IconOVir Bio, MAY 31, 2023, View Source [SID1234632275]).

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On May 31, 2023 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported positive preliminary data from its ongoing Phase 2 study of HB-200 in combination with pembrolizumab in patients with recurrent/metastatic Human Papillomavirus 16-positive (HPV16+) head and neck cancer (Press release, Hookipa Biotech, MAY 31, 2023, View Source [SID1234632272]). New data show a 43 percent objective response rate (ORR) with HB-200 in combination with pembrolizumab in checkpoint inhibitor (CPI)-naïve patients, doubling the 19 percent response rate for pembrolizumab alone. HOOKIPA plans to share the full data at a medical conference later this year and is preparing to start a pivotal trial of HB-200 in combination with pembrolizumab in the 1st-line setting in 2024. The company will host a webcast call June 1st at 8:00 a.m. EDT.

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"Given the unmet medical need for patients with recurrent/metastatic HPV16+ head and neck cancers, we are thrilled to share the preliminary data on HB-200 in combination with pembrolizumab as they show a robust improvement in objective response rate and prolonged tumor control compared to pembrolizumab alone," said Joern Aldag, Chief Executive Officer at HOOKIPA. "Efficacy, immunogenicity and safety data observed in our HB-200 program to-date support our decision to progress to a pivotal trial of HB-200 in combination with pembrolizumab as 1st-line treatment for these patients. The data also underscore the scalability of our arenaviral platform across a range of cancers."

HB-200 results (NCT04180215)
As of March 31, 2023, 35 patients with HPV16+ recurrent/metastatic head and neck cancers received HB-200 in combination with pembrolizumab as part of the Phase 2 study: 20 patients were treated with HB-200 and pembrolizumab in the 1st-line setting and 15 in the 2nd-line setting. All received HB-200 intravenously every three weeks for the first five doses and every six weeks thereafter. HB-200 means 2-vector therapy with alternating application of HB-201 (LCMV), HB-202 (PICV) vectors, encoding HPV16 E6/E7 antigens.

1st-line combination data
HB-200 in combination with pembrolizumab demonstrated promising anti-tumor activity with a 43 percent objective response rate (6 of 14 patients with confirmed responses by investigator assessment under RECIST 1.1) among CPI-naïve patients with recurrent/metastatic HPV16+ PD-L1+ head and neck cancer. These data represent a doubling of the 19 percent objective response rate reported with pembrolizumab alone.1 As of March 31, 2023, 14 patients with at least two imaging assessments were included in the interim efficacy analysis. Six patients responded (one with confirmed complete response and five with confirmed partial responses), with a 71 percent disease control rate (10 of 14 patients), meaning stable disease or a complete or partial response. While recruitment is ongoing, based on these data, HOOKIPA is preparing to start a pivotal trial of HB-200 in combination with pembrolizumab as 1st-line treatment of recurrent/metastatic HPV16+ PD-L1+ head and neck cancers in 2024.

2nd-line plus combination data
Data on HB-200 in combination with pembrolizumab in the 2nd-line plus setting are trending positively in this small initial cohort, but they are preliminary and need further maturation. As of March 31, 2023, five patients with at least two imaging assessments were included in the interim efficacy analysis based on RECIST 1.1. Preliminary results show one confirmed partial response and three patients with stable disease. Preliminary median progression-free survival in this small cohort was 5.3 months. Enrollment is ongoing, and a decision on a potential path forward in the 2nd-line plus setting will be made in 2024.

Monotherapy data
Given the limited options for heavily pre-treated patients with recurrent/metastatic HPV16+ head and neck cancers, HOOKIPA continues to evaluate HB-200 as monotherapy among these patients in the ongoing Phase 1/2 trial. Follow-up data show HB-200 monotherapy demonstrated a preliminary median overall survival of 14.2 months in the intent-to-treat population, indicating potential for prolonged clinical benefit in this heavily pre-treated population. These data are based on 11 patients who received HB-200 as monotherapy at the same dose being evaluated in combination with pembrolizumab, with a median follow-up period of 12.8 months. Additional patients are also being followed to continue to assess median overall survival in a larger cohort.

Immunogenicity
Importantly, new follow-up data from heavily pre-treated patients show an association between the induction of unprecedented levels of functional T cells after treatment with HB-200 monotherapy and clinical benefit. T cell increases were rapid and sustained for at least 8 months, as of data cut-off. Patients who achieved disease control after treatment with HB-200 monotherapy generally had greater CD8+ T cell infiltration in tumors compared to patients whose disease progressed, suggesting an association of HB-200-induced T cells and clinical benefit.

Safety and tolerability profile
Results from the Phase 1/2 study showed that HB-200 was generally well tolerated among 132 patients treated. The safety profile was similar for patients who received HB-200 monotherapy or HB-200 in combination with pembrolizumab. This favorable tolerability profile highlights the potential of HB-200 – and arenaviral immunotherapies in general – to be combined with other immunotherapies where tumor antigen-specific T cells are needed. Only seven percent of patients showed serious adverse events related to the treatment with HB-200. Only two percent of patients discontinued due to such events.

Webcast: HOOKIPA will host a live webcast on June 1, 2023 at 8:00 a.m. EDT. Joern Aldag, Chief Executive Officer, Katia Schlienger M.D., Ph.D., Chief Medical Officer, and Klaus Orlinger, Ph.D., Chief Scientific Officer will provide an overview of the HB-200 data and future plans for HOOKIPA’s oncology program. Alan Ho, M.D., Ph.D., a study investigator, will also offer commentary on the unmet medical need for patients with head and neck cancer.

Dial In: +1 646 876 9923
UK Dial In: +44 208 080 6591
Austria Dial In: +43 72 011 5988
Webinar ID: 899 3030 4628
Webcast: Link

The webcast and the presentation will be available within the Investors & Media section of HOOKIPA’s website at View Source An archived replay will be accessible for 30 days following the event.

About HB-200
HB-200 is HOOKIPA’s lead oncology candidate engineered with the company’s proprietary replicating arenaviral vector platform. It comprises two single-vector compounds with arenaviral backbones based on lymphocytic choriomeningitis virus and pichinde virus. Both express the same transgene encoding an E7E6 fusion protein derived from HPV16. HB-200 is an alternating 2-vector immunotherapy designed to further focus the immune response against the encoded antigen. HB-200 in combination with pembrolizumab received Fast Track Designation from the U.S. Food and Drug Administration for the treatment of 1st-line recurrent/metastatic HPV16+ head and neck cancers.

About the HB-200 trial (NCT04180215)
This Phase 1/2 clinical trial is an open-label trial evaluating HB-200 for the treatment of advanced HPV16+ cancers. Phase 1 assessed various dose levels, regimen, and modes of administration in a post-standard of care setting. Based on safety and tolerability, initial anti-tumor activity and T cell response data, HB-200 advanced for further development in Phase 2.

The Phase 2 part of the trial is open-label with primary endpoints of efficacy based on objective response and disease control rate as defined by RECIST 1.1 and iRECIST. The trial is evaluating HB-200 in combination with pembrolizumab in the 1st-line and 2nd-line plus settings, as well as HB-200 alone in the post-standard of care setting. HOOKIPA anticipates sharing an additional data update at an upcoming medical conference.

About Human Papillomavirus-driven Cancers
Human Papillomavirus, or HPV, is a common viral infection estimated to cause about 5 percent of the worldwide cancer burden. This includes up to 60 percent of head and neck, 89 percent of cervical, 78 percent of vaginal, 88 percent of anal, 67 percent of vulvar and 50 percent of penile cancers.

While there are numerous HPV types associated with cancer, HPV16 is the most common cause of cancer. Most HPV infections are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This uptake can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to become cancerous.