Xencor Doses First Patient in Phase 1 Study of XmAb®22841 for the Treatment of Patients with Advanced Solid Tumors

On June 3, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune diseases, asthma and allergic diseases, reported that the first patient has been dosed in a Phase 1 clinical study to evaluate the safety and tolerability of XmAb22841, both as a monotherapy and in combination with pembrolizumab, in patients with advanced solid tumors (Press release, Xencor, JUN 3, 2019, View Source [SID1234536793]). XmAb22841 is a bispecific antibody that simultaneously targets the immune checkpoint receptors CTLA-4 and LAG-3.

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"Our TME activators seek to improve the tolerability and clinical benefit of immunotherapy by activating T cells specifically in the tumor microenvironment, where many checkpoint receptors are highly expressed on immune cells. Toward that goal, XmAb22841 is designed with XmAb bispecific technology to target those cytotoxic T cells that simultaneously co-express the immune checkpoints CTLA-4 and LAG-3," said Paul Foster, M.D., senior vice president and chief medical officer at Xencor. "We intend to advance XmAb22841 in combination with anti-PD1 immunotherapy to potentially drive better responses through triple checkpoint blockade."

The Phase 1 dose-escalation and expansion study, which explores XmAb22841 as a monotherapy and in combination with pembrolizumab will characterize the safety and tolerability, pharmacokinetics, pharmacodynamics, immunogenicity and preliminary anti-tumor activity of intravenous administration in patients with select advanced solid tumors. For more information about the study, please visit View Source (identifier: NCT03849469).

About XmAb22841

XmAb22841 is a bispecific antibody that simultaneously targets immune checkpoint receptors CTLA-4 and LAG-3 and is designed to promote tumor-selective T-cell activation. Xencor’s XmAb bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture. XmAb bispecific Fc domains have been engineered to eliminate Fc gamma receptor (FcγR) binding, with the intent to prevent activation and/or depletion of T cells via engagement by FcγR-expressing cells. XmAb22841 is being evaluated in a Phase 1 study for the treatment of advanced solid tumors, as a monotherapy and in combination with pembrolizumab.

Heat Biologics Announces Promising Interim Phase 2 Lung Cancer Data Suggesting that HS-110 Plus Nivolumab May Restore Clinical Benefit After Checkpoint Inhibitor Treatment Failure

On June 3, 2019 Heat Biologics, Inc. (NASDAQ: HTBX), a biopharmaceutical company developing therapies designed to activate a patient’s immune system against cancer, reported compelling new interim results from its ongoing Phase 2 study investigating HS-110 in combination with Bristol-Myers Squibb’s anti-PD-1 checkpoint inhibitor, nivolumab (Opdivo) (Press release, Heat Biologics, JUN 3, 2019, View Source [SID1234536792]). The updated results were obtained from Cohort B patients whose data has matured an additional 3 months since last reported at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium in February of this year. This data may represent the first Phase 2 data showing clinical activity in non-small cell lung cancer (NSCLC) patients whose disease has progressed after prior treatment with a checkpoint inhibitor (CPI). The Cohort B results were presented yesterday at the2019American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting poster session.

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COL(ret) George E Peoples, MD, FACS, Heat’s Chief Medical Advisor, noted, "These latest Cohort B data provides us even greater confidence that the addition of HS-110 to nivolumab may restore anti-tumor activity in patients whose disease has progressed after treatment with a CPI. Of particular note, 4 out of 5 evaluable patients in Cohort B with PD-L1 negative tumors achieved disease stabilization and 4 out of 7 evaluable patients with low CD8+ TIL levels in their tumors achieved disease stabilization. We are encouraged by these positive results and look forward to reporting additional data later this year."

Jeff Hutchins, Ph.D., Heat’s Chief Scientific and Operating Officer said, "The fact that we saw tumor shrinkage in 35% of patients and disease control in 55% of patients whose disease has progressed after treatment with a CPI supports our mechanistic hypothesis that the broad, T-cell mediated immune response activated by HS-110 may improve clinical outcomes for patients who have lost the benefit of treatment with a checkpoint inhibitor. It is also important to note that the occurrence of dermal injection site reactions is associated with statistically significant improved progression free survival and overall survival, providing further support for the mechanism of action of HS-110."

Highlights for Cohort B patients arepresented below:

HS-110 in combination with nivolumab demonstrates clinical activity i’difficult to treat’ low CD8+ TIL (≤10%) and PD-L1 negative (<1%) tumors:
4 out of 5 evaluable patients with PD-L1 negative tumors achieved disease stabilization, 1 of which was a RECIST partial response.
4 out of 7 evaluable patients with low CD8+ TIL tumors achieved disease stabilization, 2 of which were RECIST partial responses.
The addition of HS-110 to nivolumab may restore clinical benefit to patients whose disease has progressed after CPI treatment failure:
Tumor shrinkage observed in 35% of patients
Disease control rate of 55%
Median Progression-Free Survival (mPFS) of 2.7 months
Median Overall Survival (mOS) not yet reached
The occurrence of any grade dermal Injection Site Reaction during treatment (Y/N) is associated with improved Progression-Free Survival and Overall Survival:
mPFS: NR vs 1.8 months; HR 0.17 (95% CI, 0.03-0.84); p=0.013
mOS: NR vs 5 months; HR 0.13 (95% CI, 0.02-0.71); p=0.002
Treatment with HS-110 in combination with nivolumab was well tolerated, with no additional toxicities beyond those observed with single agent CPI therapy.

Trial results are summarized in the official 2019 ASCO (Free ASCO Whitepaper)Annual Meeting poster.

Trial Design

The Phase 2 trial is designed to evaluate the safety and efficacy of HS-110 combined with an immune checkpoint inhibitor for the treatment of advanced non-small cell lung cancer. Cohort B consists of patients who received a minimum of 4 months of treatment with a checkpoint inhibitor (CPI) as part of their prior therapy, but subsequently had documented progressive disease. Patients receive weekly HS-110 (1 x 107 cells) administered as 5 intradermal 0.1 mL injections for 18 weeks in combination with bi-weekly nivolumab 240 mg IV administered until confirmed disease progression or unacceptable toxicity, whichever occurs first. The primary endpoint is objective response rate (ORR); secondary endpoints include overall survival (OS), progression-free survival (PFS), disease control rate (DCR) and duration of response (DOR). Exploratory endpoints include correlation of clinical outcomes to baseline CD8+ TILs, PD-L1 expression, peripheral blood tumor mutation burden and ELISPOT analysis.

Findings from Can-Fite’s Phase II Liver Cancer Study were Presented at ASCO Annual Meeting Late-Breaking Session

On June 3, 2019 Can-Fite BioPharma Ltd. (NYSE MKT: CANF) (TASE:CFBI), a biotechnology company with a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that data from its recently completed Phase II trial in patients with hepatocellular cancer (HCC), the most common form of liver cancer, was presented at the late-breaking abstract session of the 54th Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), the world’s largest clinical cancer research meeting (Press release, Can-Fite BioPharma, JUN 3, 2019, View Source [SID1234536791]).

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Prof. Salomon M. Stemmer, Principal Investigator of Can-Fite’s Phase II trial, delivered the presentation on Sunday June 2, 2019 at the Developmental Immunotherapy and Tumor Immunobiology session. The presentation is entitled: "A phase II, randomized, double-blind, placebo-controlled trial evaluating efficacy and safety of namodenoson (CF102), an a3 adenosine receptor agonist (A3AR), as a second-line treatment in patients with Child-Pugh B (CPB) advanced hepatocellular carcinoma (HCC)." While the Phase II study did not achieve its primary endpoint of overall survival in the whole population (n=78), superiority in overall survival was found in the largest study subpopulation of patients who were classified Child Pugh B7 (n=56) based on severity of disease compared to the placebo treated group. Median survival in the Namodenoson group (n = 34) was 6.8 months, versus 4.3 months for the placebo group (n = 18) (Hazard Ratio = 0.77, p = 0.40). (See Figure 1 )

The most impressive finding was that 44% of the patients with Child Pugh B7 treated with Namodenoson were alive at one year compared to 18% in the placebo group. In the overall patient population, among patients who had at least one assessment post baseline, disease control was significant in the Namodenoson group, 26% versus 10% in the control group after four months of treatment, P value 0.013. Among the other positive findings that were presented is the 9% partial response in the Namodenoson treated group vs. 0% in the placebo group. An example of a patient demonstrating an excellent tumor shrinkage was presented. (See Figure 2)

A3AR expression level at baseline was 1.98±0.36 in comparison to 1 unit in healthy subjects and was not changed substantially during the treatment period, demonstrating that continuous treatment with Namodenoson does not result in de-sensitization or loss of the target. Consistent with its previously demonstrated favorable safety profile, Namodenoson showed an adverse event profile that was comparable to that of placebo.

"No systemic therapies have shown clinical benefit in Child Pugh B patients with advanced HCC, underscoring the importance of developing an effective drug to serve this unmet medical need," stated Can-Fite CEO Pnina Fishman. "We believe evidence of Namodenoson’s efficacy to prolong life in Child Pugh B7 liver cancer patients, without any deterioration in quality of life due to the drug’s strong safety data, make it a promising anti cancer agent to be moved into Phase III. We were pleased that ASCO (Free ASCO Whitepaper) selected Prof. Stemmer’s presentation so he could share these important findings at ASCO (Free ASCO Whitepaper) with the world’s leading clinical cancer researchers and physicians."

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated as a second line treatment for hepatocellular carcinoma, with a recently completed Phase II trial and planned Phase III trial in this indication. The drug is currently in an ongoing Phase II trial as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

HiFiBiO Therapeutics and Leading Academic Institutions Demonstrate Breakthrough Single-Cell Approach for Novel Biomarker Discovery to Combat Cancer

On June 3, 2019 HiFiBiO Therapeutics, a pioneer in innovative biotherapeutics with a unique single-cell analytics platform for extensive immune profiling, reported results from a breakthrough translational study in collaboration with scientists at world-leading academic institutions. A manuscript, "High-Throughput Single-Cell ChIP-seq Identifies Heterogeneity of Chromatin States in Breast Cancer," was released online ahead of publication in Nature Genetics.

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In this study, scientists from HiFiBiO Therapeutics, ESPCI Paris and the Institut Curie applied HiFiBiO Therapeutics’ proprietary single-cell CelliGO platform and unique Drug Intelligent Science (DIS) capabilities to probe why certain disease cells are resistant to available therapeutics. The team demonstrated a novel high-throughput single-cell ChIP-seq (scChIP-seq) approach that overcomes the current limitations of chromatin immunoprecipitation followed by sequencing (ChIP-seq) technologies to better understand the role chromatin heterogeneity plays in cellular differentiation and development. The team used the scChIP-seq approach to discover new biomarkers for triple negative breast cancer, which led to several patent applications.

"This publication is a major milestone for HiFiBiO Therapeutics and another example of how we are applying our proprietary single-cell technology platform to develop new solutions that can address unmet medical needs and transform patient care," said Liang Schweizer, PhD, CEO and President of HiFiBiO Therapeutics. "The new HiFiBiO scChIP-seq approach enables us and our collaborators to target more precise patient subsets and generate higher and longer lasting responses to the innovative therapeutics under development."

The new scChIP-seq approach profiles chromatin landscapes of thousands of cells in tumors and other complex biological systems with high accuracy at single-cell resolution. Using patient-derived xenograft models of acquired resistance to chemotherapy and targeted therapy for triple negative breast cancer patients, the team identified a subset of cells within the untreated drug-sensitive tumors that share a common chromatin signature with resistant cells, undetectable by using bulk approaches. This signature has the potential to lead to the discovery of new drug targets and biomarkers for patient stratification.

"We are committed to partnering with leading academic institutions, as well as pharmaceutical and biotechnology companies. We strongly believe that our proprietary CelliGO platform and unique DIS capabilities can accelerate the development of high-quality antibody drugs and enable novel biomarker discovery for better patient stratification and clinical success," said Annabelle Gérard, PhD, Senior author of the publication and Head of External Innovation at HiFiBiO Therapeutics. "Together with scientists at ESPCI Paris and the Institut Curie, we have demonstrated a new approach that enables the scientific community to decipher novel biology, understand mechanisms of action, develop more effective therapeutics, and design targeted clinical trials for patients with cancer and other complex diseases."

To read the full manuscript, please visit View Source

Roche presents data from across its breast cancer portfolio at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting

On June 3, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported results from a number of abstracts highlighting studies including the treatment of both PD-L1-positive triple-negative and HER2-positive breast cancer at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting, 31 May – 4 June, in Chicago, United States (Press release, Hoffmann-La Roche, JUN 3, 2019, View Source [SID1234536789]). Data include results from the second overall survival (OS) interim analysis from the phase III IMpassion130 study evaluating Tecentriq (atezolizumab) plus chemotherapy (Abraxane [paclitaxel protein-bound particles for injectable suspension (albumin-bound); nab-paclitaxel]) for the initial (first-line) treatment of adults with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC).1 In HER2-positive breast cancer, results of an end-of-study analysis of the phase III CLEOPATRA study will also be presented, evaluating the long-term efficacy and safety of Perjeta (pertuzumab) in combination with Herceptin (trastuzumab) and docetaxel chemotherapy (the Perjeta-based regimen) in patients with previously untreated HER2-positive metastatic breast cancer (mBC).2

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"Our science-driven approach has been transforming standards of care in breast cancer for more than 20 years, and we are excited to present data from a Perjeta study, that showed an unprecedented survival benefit for patients living with advanced HER2-positive disease," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We are continuing our transformative work in breast cancer, presenting updated interim overall survival results from our phase III Tecentriq combination, the first positive immunotherapy study in PD-L1-positive metastatic triple-negative breast cancer, a disease with high unmet need."

TNBC is a form of breast cancer representing approximately 15% of all cases.3,4 Metastatic TNBC is one of the most aggressive and difficult-to-treat forms of breast cancer.3,4 HER2-positive disease is another particularly aggressive form of breast cancer accounting for approximately 15-20% of all cases of breast cancer.5

IMpassion130 study results
Results presented at ASCO (Free ASCO Whitepaper) were consistent with the first interim analysis reported at the European Society for Medical Oncology congress in October 2018.1,6 At the second interim analysis, statistical significance was not met for overall survival (OS) in the intention-to-treat (ITT) population (median OS=21.0 vs 18.7 months; HR=0.86, 95% CI: 0.72-1.02, p=0.078).1 However, Tecentriq and nab-paclitaxel showed a clinically meaningful OS improvement of seven months vs placebo and nab-paclitaxel in patients who were tested positively for PD-L1 expression on tumour-infiltrating immune cells (median OS=25.0 vs 18.0 months; HR=0.71, 95% CI: 0.54-0.93) with more mature data.1 OS results in the PD-L1-positive population were not formally tested due to the prespecified hierarchical analysis plan of the study. Over half (51% (43-59%)) of PD-L1-positive metastatic TNBC patients in the Tecentriq arm were alive at two years, compared with 37% (29-45%) in the control arm. Follow-up will continue until the next planned analysis.1

Additional analyses of patient-reported outcomes from IMpassion130 showed that the combination was well-tolerated, similarly to nab-paclitaxel alone.7 Tecentriq plus nab-paclitaxel showed gains in clinical benefit without compromising patients health-related quality of life (HRQoL), day-to-day functioning and without increasing patients toxicity burden compared to nab-paclitaxel alone.7 HRQoL evaluates the overall impact of disease and treatment on patient’s quality of life in terms of disease related symptoms, treatment side effects and function/well-being. An expanded safety analysis showed that the Tecentriq plus nab-paclitaxel arm appeared consistent with the known safety profiles from the primary data with no new safety signals observed.8

CLEOPATRA study results
After a follow-up of eight years, results from the phase III CLEOPATRA study, investigating patients with previously untreated HER2-positive mBC, showed a median OS benefit of 57.1 months in the Perjeta arm compared with 40.8 months in the control arm (placebo, Herceptin and chemotherapy), an absolute improvement of 16.3 months, with a 31% overall reduction in the risk of death (HR=0.69, 95% CI 0.58-0.82, p<0.0001).2

These new data are consistent with previously published results and confirm that the unprecedented OS benefit of the Perjeta-based regimen was maintained after an additional four years of follow-up.2 Significantly, over a third (37%) of HER2-positive mBC patients in the Perjeta arm were alive at eight years, compared with 23% in the control arm.2 Safety data from the long-term follow-up study were consistent, and showed that the cardiac and overall safety profiles of the Perjeta-based regimen were maintained.2

Additionally, data from the Chinese phase III PUFFIN study were presented at this year’s meeting further confirming the use of the Perjeta-based regimen in HER2-positive mBC, as results were consistent with those seen in the CLEOPATRA study.9

Roche’s breadth of expertise continues to advance breast cancer research with the goal of expanding treatment options to improve the lives of patients living with the disease worldwide.

About the IMpassion130 study
The IMpassion130 study is a phase III, multicentre, randomised, double-blind study evaluating the efficacy, safety and pharmacokinetics of Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel in people with unresectable locally advanced or metastatic TNBC who have not received prior systemic therapy for mBC. The study enrolled 902 people who were randomised equally (1:1). The co-primary endpoints are progression-free survival (PFS) per investigator assessment (RECIST 1.1) in the ITT population and in the PD-L1-positive population and OS in the ITT population. Secondary endpoints include objective response rate and duration of response.

About the CLEOPATRA Study
CLEOPATRA (CLinical Evaluation Of Pertuzumab And TRAstuzumab) was an international, phase III, randomised, double-blind, placebo-controlled study. The study compared the combination of Perjeta, Herceptin and docetaxel chemotherapy with placebo, Herceptin and chemotherapy in 808 people with previously untreated HER2-positive mBC, or with HER2-positive mBC that had come back after prior therapy in the adjuvant or neoadjuvant setting. The primary endpoint of the study was PFS as assessed by an independent review committee. Secondary endpoints included OS and safety profile.

About Roche in breast cancer
Roche has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have contributed to bringing breakthrough innovations in HER2-positive breast cancer. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for all forms of early and advanced breast cancer, including triple-negative and hormone receptor-positive.

Our targeted medicines Herceptin, Perjeta and Kadcyla are continuing to transform the treatment of early and advanced HER2-positive breast cancer and, through our Tecentriq and ipatasertib clinical programmes, we are committed to bring new treatment combinations to people with triple-negative breast cancer, ultimately improving outcomes.