Leap Therapeutics Presents Updated Data for DKN-01 in Esophagogastric Cancer Patients at SITC 2020 35th Annual Meeting

On November 9, 2020 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported the presentation of clinical data from its Phase 1b/2a clinical trial of DKN-01 in patients with advanced esophagogastric cancer (EGC) at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 35th Anniversary Annual Meeting (Press release, Leap Therapeutics, NOV 9, 2020, View Source [SID1234570363]). DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein.

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In the study, high levels of tumoral DKK1 expression correlated with improved clinical outcomes in heterogeneous EGC patients treated with DKN-01 as monotherapy or in combination with pembrolizumab or paclitaxel. Pooled data for the 69 patients in the study for whom tumoral DKK1 expression data is available demonstrated that DKK1-high patients experienced higher overall response rates (ORR), a doubling of median progression-free survival (PFS), and longer median overall survival (OS) as compared to DKK1-low patients. Additionally, DKK1-high anti-PD-1/PD-L1 refractory patients treated with DKN-01 plus pembrolizumab experienced longer PFS and OS compared to DKK1-low patients, suggesting that DKK1 tumoral expression may serve as a predictive biomarker to identify patients for treatment with DKN-01 in combination with anti-PD-1 antibodies. No notable differences were found in baseline MSS, TMB, or PD-L1 expression between DKK1-high versus DKK1-low groups.

"We continue to observe clinically meaningful activity of DKN-01 in patients with advanced previously treated esophagogastric cancer that reinforces our belief that elevated tumoral DKK1 expression is a predictive biomarker for improved outcomes, particularly for those patients treated with DKN-01 in combination with an anti-PD-1 antibody," said Samuel J. Klempner, MD, Assistant Professor, Massachusetts General Hospital Cancer Center and Harvard Medical School.

"We believe that the totality of the results from this study provides strong support for the ongoing study of DKN-01 in combination with tislelizumab, an anti-PD-1 antibody, in DKK1-high second line gastroesophageal junction and gastric cancer (GEJ/GC) patients and in combination with tislelizumab, capecitabine, and oxaliplatin in first-line GEJ/GC patients," continued Dr. Klempner.

The P102 Study in Relapsed or Refractory Esophagogastric Cancer

The P102 study (KEYNOTE-731) is a multi-part Phase 1/2 study of DKN-01 as a monotherapy and in combination with paclitaxel or KEYTRUDA (pembrolizumab) in advanced EGC patients, with a median of two previous treatments with standard therapies, representing a difficult to treat population. The study is intended to establish the safety and activity of DKN-01 as a monotherapy and in combination with paclitaxel or pembrolizumab, with efficacy endpoints of ORR, PFS, and OS. Tumoral DKK1 expression was determined retrospectively by RNAscope chromogenic in situ hybridization and correlated with clinical outcomes. Pembrolizumab was provided for the study through a clinical trial collaboration agreement with Merck (known as MSD outside the United States and Canada).

Key DKN-01/Pembrolizumab Findings from the P102 Study

·Anti-PD-1/PD-L1 refractory patients (all): The four DKK1-high patients had a significantly longer PFS of 12.8 weeks and OS of 46 weeks as compared to the five DKK1-low patients who experienced PFS of 6 weeks and OS of 16 weeks.

·Anti-PD1/PD-L1 refractory GEJ/GC patients: The three DKK1-high patients had a best response of stable disease (SD) and a longer PFS of 13.4 weeks and OS of 37.4 weeks, as compared to the two DKK1-low patients who both had progressive disease (PD) with a PFS of 3.6 weeks and OS of 11.7 weeks.

·Anti-PD-1/PD-L1 naïve GEJ/GC patients: As previously reported, DKK1-high patients experienced over 22 weeks PFS and nearly 32 weeks OS, with a 50% overall response rate and 80% disease control rate (DCR) in ten evaluable patients. DKK1-low patients experienced nearly 6 weeks PFS and over 17 weeks OS, with a 20% DCR in fifteen evaluable patients. PD-L1 Combined Positive Scores (CPS) did not predict efficacy on the combination of DKN-01 plus pembrolizumab. In multi-variate analysis, DKK1-high status correlated with longer PFS independent of PD-L1 CPS scores.

About DKN-01

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling, a signaling pathway frequently implicated in tumorigenesis and suppressing the immune system. DKK1 has an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK ligands on tumor cells. The U.S. Food and Drug Administration has granted Orphan Drug Designation for the treatment of gastric and gastroesophageal junction cancer and Fast Track Designation in combination with tislelizumab for the treatment of patients with gastric and gastroesophageal junction adenocarcinoma whose tumors express high DKK1 protein, following disease progression on or after prior fluoropyrimidine- and platinum- containing chemotherapy and if appropriate, human epidermal receptor growth factor (HER2)/neu-targeted therapy.

IMV’s T Cell Therapy Demonstrates 86% Objective Response Rate in Combination with Merck’s Keytruda® in PD-L1 Positive Patients with r/r DLBCL

On November 9, 2020 IMV Inc. (Nasdaq: IMV; TSX: IMV), a clinical stage biopharmaceutical company pioneering a novel class of immunotherapies, reported that the Company’s T cell therapy demonstrates an 86% objective response rate in combination with Merck’s Keytruda (pembrolizumab) in patients with Program Death Ligand 1 (PD-L1) positive relapsed / refractory diffuse large B-cell lymphoma (r/r DLBCL) (Press release, IMV, NOV 9, 2020, View Source [SID1234570362]). Detailed results will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting, to be held virtually Nov. 9-14, 2020 and during a webcast hosted by IMV on November 12, 2020.

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"Biomarkers are critical for the development of precision medicine in oncology. Not only can they improve the outcome of cancer patients receiving treatments, but they also can greatly reduce the risk inherent in late-stage clinical trials and facilitate the path to market for new treatments such as our new T cell therapy," said Joanne Schindler, Chief Medical Officer at IMV. "DLBCL is the most common form of Non-Hodgkin lymphoma and relapsed/refractory patients need more accessible and better treatment options. Identifying a biomarker predictive of response for these patients was an important objective of our clinical plan and path to approval."

All clinical responses are associated with expression of the PD-L1 biomarker

All clinical responses observed so far in the study have been in PD-L1 positive subjects defined as a percentage of PD-L1+ cells scored in the tumor region of 10% or more. No benefits have been observed in the PD-L1 negative population (n=11) where all subjects experienced Progressive Disease (PD) (n=9) or a Stable Disease (SD) (n=2).

The difference between the two populations is statistically significant and indicates that PD-L1 has the potential to become a predictive biomarker and a companion diagnostic for DLBCL

treatment with the combination, to identify and recruit the patients that are the most likely to respond.

As of the data cut-off date for the presentation at SITC (Free SITC Whitepaper), 18 pre-treatment samples from patients enrolled in the SPiReL study were available for biomarker analysis. Thirty nine percent (7/18) of subjects demonstrated a positive pre-treatment tumor PD-L1 expression. Key findings for this population include:

100% of subjects with Disease Control Rate (DCR) defined as Stable Disease (SD) or Complete or Partial Response (CR or PR)

86% (6/7) of subjects with Objective Response Rate (ORR) (3 CR, and 3 PR)

The PD-L1 pathway regulates T-cell responses allowing tumors to evade detection by the immune system. PD-L1 expression has been extensively studied in relation to the prognosis of various cancers and is approved in multiple tumor types as a predictive biomarker for treatment with checkpoint inhibitors targeting the PD-1/PD-L1 pathway. In DLBCL, PD-L1 has been shown to be expressed in 26% to 75% of patients and is generally thought to be associated with a poor prognosis and shorter survival1,2.

Checkpoint inhibitors such as Keytruda and Opdivo are not approved in DLBCL and have demonstrated limited activity including in PD-L1 positive patients.1,3

Poster Presentation Details

Poster Title

Baseline PD-L1 expression and tumor immune infiltration is associated with clinical response in patients with r/r DLBCL treated with DPX-Survivac, low-dose cyclophosphamide and pembrolizumab

Presenter: Neil Berinstein, MD, FRCPC, ABIM
Hematologist at the Sunnybrook Health Science Centre, Toronto.

Poster is available on Company’s website and on the SITC (Free SITC Whitepaper) conference platform since November 9 at 8.00am EST. The final poster presentation will include additional data collected between the date of the abstract submission and the presentation itself. The poster is available under the Scientific Publications & Posters section on IMV’s website.

Company will discuss the data during a live webcast on November 12 at 8.00am EST with a presentation of the results by Neil Berinstein, MD, FRCPC, ABIM, Principal Investigator of the SPiReL study. Webcast registration will be available under "Events, Webcasts and Presentations" in the Investors section of IMV’s website. The video recording will be available for replay shortly thereafter.

About the SPiReL Study

1 Xu-Monette, Zijun Y et al. "PD-1 expression and clinical PD-1 blockade in B-cell lymphomas" Blood vol. 131,1 (2018): 68-83. doi:10.1182/blood-2017-07-740993
2 Suzuki Y, Kohno K, Matsue K, et al. PD-L1 (SP142) expression in neoplastic cells predicts a poor prognosis for patients with intravascular large B-cell lymphoma treated with rituximab-based multi-agent chemotherapy. Cancer Med. 2020;9(13):4768-4776. doi:10.1002/cam4.3104
3 Ansell SM, et al. Nivolumab for Relapsed/Refractory Diffuse Large B-Cell Lymphoma in Patients Ineligible for or Having Failed Autologous Transplantation: A Single-Arm, Phase II Study. J Clin Oncol. 2019 Feb 20;37(6):481-489. doi: 10.1200/JCO.18.00766

"SPiReL" is a Phase 2 non-randomized, open label, efficacy, and safety study of a novel immunotherapy combination with DPX-Survivac and pembrolizumab. Intermittent low dose cyclophosphamide is given as an immune modulator. Subjects with r/r incurable DLBCL and survivin expression are eligible for participation. The primary outcome is to document the objective response rate using modified Cheson criteria to the combination treatment. Secondary objectives include safety, duration of response and time to next treatment. Exploratory endpoints include T cell response, tumour immune cell infiltration, and biomarker analysis. To date, 24 subjects have been enrolled.

About DPX-Survivac

DPX-Survivac is the lead candidate in IMV’s new class of immunotherapy that generates targeted and sustained cancer cell killing capabilities in vivo. Treatments with the DPX-Survivac T cell therapy have demonstrated a favorable safety profile across all clinical studies.

IMV’s T cell therapy, DPX-Survivac, consists of survivin-based peptides formulated in IMV’s proprietary delivery platform (DPX). IMV’s lead compound is designed to generate a sustained cytotoxic T cell response against cancer cells presenting survivin peptides on their surface.

Survivin, recognized by the National Cancer Institute (NCI) as a promising tumor-associated antigen, is broadly over-expressed in most cancer types, and plays an essential role in antagonizing cell death, supporting tumor-associated angiogenesis, and promoting resistance to chemotherapies. IMV has identified over 20 cancer indications in which survivin can be targeted by DPX-Survivac.

DPX-Survivac has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as maintenance therapy in advanced ovarian cancer, as well as Orphan Drug designation status from the U.S. FDA and the European Medicines Agency (EMA) in the ovarian cancer indication.

Targovax announces poster at the 2020 Society for Immunotherapy of Cancer (SITC) Annual Meeting

On November 9, 2020 Targovax ASA (OSE: TRVX), a clinical stage immuno-oncology company developing oncolytic viruses to target hard-to-treat solid tumors, reported that the poster "A randomised open-label phase I/II study adding ONCOS-102 to pemetrexed/cisplatin in patients with unresectable malignant pleural mesothelioma – 12-month analysis of biomarkers and clinical outcomes" are now available at the Company’s website (Press release, Targovax, NOV 9, 2020, View Source [SID1234570361]).

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The abstract presents the 12-month analysis of biomarkers and clinical outcome from the phase I/II trial in malignant pleural mesothelioma where ONCOS-102 is added to standard of care chemotherapy (pemetrexed / cisplatin). This analysis supports the data presented in June.

Title (361): A randomised open-label phase I/II study adding ONCOS-102 to pemetrexed/cisplatin in patients with unresectable malignant pleural mesothelioma – 12-month analysis of biomarkers and clinical outcomes
Presenter:

Prof. Luis Paz-Ares, Principal Investigator of the trial and Chair of the Medical Oncology Department at the Hospital Doce de Octubre, Madrid

Poster session:

Location:

Wednesday 11. November 23:15-23:45 CET (17:15–17:45 EST)

Friday 13. November 22:40-23:10 CET (16:40-17:10 EST)

Virtual Poster Hall

The poster is also available on Targovax’s website.

Ambrx Closes $200 million in Crossover Financing with Leading Healthcare Investors to Advance its Clinical and Preclinical Pipeline of Precision Biologics

On November 9, 2020 Ambrx Inc., a clinical-stage biopharmaceutical company focused on developing Precision Biologics using an expanded genetic code reported the successful closing of an oversubscribed US$200 million crossover financing round (Press release, Ambrx, NOV 9, 2020, View Source [SID1234570360]).

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New investors include Fidelity Management & Research Company LLC, funds and accounts managed by BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. BofA Securities acted as the sole placement agent for the offering.

"This financing represents our most significant capital raise to date and includes several leading global and US based healthcare funds. Since mid-2018, our new management team, set on a new long term vision, has committed to building company fundamentals and transforming Ambrx from a technology developer to a full-fledged biopharmaceutical company," said Dr. Feng Tian, CEO and Chairman of Ambrx. "The conclusion of this financing and the strong data emerging from our ongoing clinical programs, as well as our deep preclinical pipeline of proprietary drug candidates, positions the company for rapid growth."

Ambrx technology uses an expanded genetic code to incorporate synthetic amino acids into protein, all completed within a living cell. These synthetic amino acids enable the creation of Precision Biologics, an exciting new class of therapeutics with broad application and potential. This includes next generation antibody drug conjugates (ADCs), bispecifics, and targeted immuno-oncology therapies for cancer as well as smart cytokines to modulate the immune system, and long acting therapeutic peptides for metabolic and cardiovascular disease.

The company’s innovation engine continues to expand the clinical and preclinical pipeline by discovering new and valuable programs that take full advantage of the Ambrx technologies. These novel programs have the potential to address a wide range of diseases with new or improved mechanisms of action, providing the company with a rich set of programs to develop internally or in partnership with leading pharmaceutical companies.

VBI Vaccines Announces e-Poster Presentation at the Society for Neuro-Oncology (SNO) 2020 Virtual Meeting

On November 9, 2020 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported that its abstract highlighting data from Part B of the ongoing Phase 1/2a study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate, in recurrent glioblastoma (GBM) was accepted for e-poster presentation at the 25th Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO), taking place November 19-21, 2020 (Press release, VBI Vaccines, NOV 9, 2020, View Source [SID1234570359]).

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Poster #: CTIM-07

Title: Identification of a baseline biomarker associated with tumor responses in a Phase 1/2a trial of a therapeutic CMV vaccine against recurrent glioblastoma (GBM)

Session: Clinical trials: Immunologic

Presenter: Andrew B. Lassman, M.D., Chief of Neuro-oncology at Columbia University Irving Medical Center and Associate Director for Clinical Infrastructure at Herbert Irving Comprehensive Cancer Center, and principal investigator of the ongoing Phase 1/2a study

Date: Available November 19-21, 2020

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in 38 patients with recurrent GBM:

● Phase 1 (Part A)

●Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) in recurrent GBM patients with any number of prior recurrences
●This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg

●Phase 2a (Part B)

●Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase
●This phase is a two-arm study, enrolling 10 first-recurrent GBM patients in each arm, assessing 10.0 µg of VBI-1901 in combination with either GM-CSF or GSK’s proprietary AS01B adjuvant system as immunomodulatory adjuvants
●Enrollment of the 10 patients in the GM-CSF arm and the 10 patients in the AS01B arm are complete

VBI-1901 is administered intradermally when adjuvanted with GM-CSF and intramuscularly when adjuvanted with the AS01B adjuvant system. Patients in both phases of the study receive the vaccine immunotherapeutic every four weeks until tumor progression.

About VBI-1901 and GBM

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and is exceptionally lethal.