Newly Published Pre-Clinical Data Show Intratumoral Injections of Messenger RNA Encoding Three Immune Modulators Stimulate Durable Anti-Cancer Responses in Treated and Distal Tumors

On January 30, 2019 Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported the publication of pre-clinical data that shows the therapeutic potential of mRNA-2752, an investigational mRNA immuno-oncology therapy that encodes a novel combination of three immunomodulators designed to activate the immune system to recognize and eradicate tumors that are resistant to checkpoint inhibitors (Press release, Moderna Therapeutics, JAN 30, 2019, View Source [SID1234532967]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study, published in the scientific journal Science Translational Medicine, found that the local delivery of mRNA encoding the secreted cytokines IL23 and IL36γ and the membrane-bound T-cell co-stimulator OX40L, induced a broad immune response promoting tumor regression in both injected lesions and distant un-injected tumors in mice. When combined with checkpoint inhibitors, mRNA-2752 boosted complete response rates in immunosuppressive and in immunologically barren tumor models that are otherwise unresponsive to checkpoint inhibitors.

"These pre-clinical data are important because they show how we can utilize multiple mRNAs encoding for immune modulators in a single therapy to activate a robust, systemic immune response against cancer in immunosuppressive and in so-called ‘cold’ tumors that are resistant to checkpoint inhibitors," said Joshua Frederick, Ph.D., Moderna’s head of oncology research. "We were pleased to discover the cooperation of the components encoded by this mRNA mixture in engaging innate immune cells, innate-like lymphocytes and effector T cells, ultimately resulting in complete tumor regressions and protective immunity in our mouse models of cancer."

"Unlike conventional biologics, we believe mRNA therapies can uniquely alter the tumor microenvironment to make cancers more susceptible to checkpoint inhibitors via a paracrine effect by producing high, local therapeutic concentrations of membrane-bound and secreted immunomodulators, both of which are believed to play a critical role in the immune response against cancer," said Tal Zaks, M.D., Ph.D., chief medical officer at Moderna. "This important study highlights why we are excited to have started our Phase 1 clinical study for mRNA-2752, as we believe the combination of these immune signals has the potential to help patients for whom checkpoint inhibitors alone have been insufficient."

The study showed that in a MC38-R mouse cancer model that is considered immunosuppressive and found to be unresponsive to checkpoint inhibitor immunotherapy, a single dose of the Triplet administered intratumorally led to complete responses (defined as the absence of all detectable cancer). After multiple injections in the immunosuppressive tumor model, complete response rates increased to a majority of the treated animals. In addition, a single dose of the Triplet led to near-complete control of both injected tumors and distal untreated tumors. The addition of anti-PD-L1, anti-PD-1 or anti-CTLA-4 checkpoint inhibitors to a single dose of the Triplet improved complete response rates over either mRNA or antibody treatment alone.

Moderna has advanced mRNA-2752 into a Phase 1 study (ClinicalTrials.gov Identifier: NCT03739931) and has started dosing patients with advanced or metastatic solid tumor malignancies or lymphoma. The open label, multi-center study is evaluating the safety and tolerability of mRNA-2752 as a monotherapy or in combination with either AstraZeneca’s durvalumab (anti-PD-L1 antibody) or tremelimumab (anti-CTLA-4 antibody) and will assess anti-tumor activity, protein expression in tumors and pharmacokinetics and exploratory endpoints that include assessment of immunological response.

A link to the publication, Durable anti-cancer immunity from intratumoral administration of IL-23, IL-36γ and OX40L mRNAs (S. L. Hewitt, et. al.), can be found here.

Eureka Therapeutics Announces US FDA Clearance of IND Application for Phase 1/2 Trial of ET140202 Artemis T-Cell Therapy, for the Treatment of Liver Cancer

On January 30, 2019 Eureka Therapeutics, Inc., a clinical stage biotechnology company developing antibody-TCR (AbTCR) T-cell therapies, reported that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for ET140202 ARTEMIS T-cell therapy in AFP-positive patients with advanced hepatocellular carcinoma (HCC), the most common type of liver cancer (Press release, Eureka Therapeutics, JAN 30, 2019, View Source [SID1234532966]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company plans to initiate its Phase 1/2 US multicenter clinical trial in the first half of 2019. The Phase 1 dose escalation portion of the trial will assess the safety, tolerability, pharmacokinetics and preliminary anti-tumor activity of ET140202. A dose expansion phase is planned to follow the selection of a recommended Phase 2 dose.

As previously reported in September 2018, Eureka presented data from a first-in-human study of ET140202 in China. The findings from the First Affiliated Hospital of Xi’An Jiaotong University demonstrated a favorable safety profile of ET140202 T-cell therapy in six patients with no observed cytokine release syndrome (CRS) or drug-related neurotoxicity. In addition, one patient in the i.v. arm of the study had a complete response. Overall, tumor regression was observed in three out of six patients.

"This is an exciting time for Eureka as we prepare to initiate our US clinical trial in patients with hepatocellular carcinoma," said Cheng Liu, Ph.D., President and Chief Executive Officer of Eureka Therapeutics. "HCC is an area of significant unmet medical need and patient options are currently limited. We intend to advance ET140202 as rapidly as possible in the US and to build upon the experience from our promising proof-of-concept study in China."

ABOUT LIVER CANCER

A highly unmet medical need, liver cancer is the second most common cause of cancer-related deaths, with roughly 600,000 patient deaths every year worldwide. Hepatocellular carcinoma (HCC) is the predominant type of liver cancer that occurs in approximately 31,500 newly diagnosed patients per year in the United States. General 5-year survival rate of liver and intrahepatic bile duct cancer in the United States is only 18%.

ABOUT ET140202

ET140202 utilizes Eureka’s proprietary ARTEMIS T-cell receptor platform engineered with a proprietary human TCR-mimic (TCRm) antibody to target an AFP-peptide/HLA-A2 complex on HCC cancer cells. Using its proprietary E-ALPHA antibody discovery platform, Eureka developed a TCRm antibody to selectively bind an AFP peptide displayed on the cell surface by the HLA-A2 major histocompatibility complex (MHC).

Can-Fite’s Drugs’ Potential Ability to Treat Cytokine Release Syndrome in Cancer Immunotherapy Published in Scientific Journal

On January 30, 2019 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported that Drug Design, Development and Therapy has published an article titled, "Targeting the A3 Adenosine Receptor to Treat Cytokine Release Syndrome in Cancer Immunotherapy (Press release, Can-Fite BioPharma, JAN 30, 2019, View Source [SID1234532965])." The article presents data from numerous studies that show adenosine’s role in inhibiting inflammatory cytokine production. Can-Fite’s Piclidenoson, a Phase III drug candidate, and Namodenson, a Phase II drug candidate, both target the A3 adenosine receptor (A3AR), which the Company believes may treat cytokine release syndrome (CRS) while also promoting an anti-cancer effect.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

CRS is a potentially life threatening side effect of cancer immunotherapies including CAR-T. The market for CAR-T drugs is estimated to reach approximately $5.4 Billion in 2024 according to Evaluate Pharma.

"While CAR-T and other cancer immunotherapies are saving lives, as their use increases, there is growing concern about the drugs’ life threatening side effects including the high incidence of CRS. With the publication of this article in Drug Design, Development and Therapy, we are advancing the scientific community towards delivering immunotherapies that offer a high degree of efficacy with a greater degree of safety for the patient. Our platform technology, through Namodenoson, has already displayed its anti-cancer effects in humans, and therefore it is a candidate to not only protect patients from CRS, but to also boost the body’s fight against cancer," stated Dr. Pnina Fishman, Can-Fite’s CEO. "We look forward to implementing our development strategy for our drugs in the treatment of CRS."

Can Fite’s platform technology selectively targets A3AR, which plays a central role in mediating the mechanism of inflammation by reducing elevated levels of pro-inflammatory cytokines such as IL-6, IL-1β, NF-Kβ, TNF-α, and more.

BioInvent Receives FDA Orphan Designation for BI-1206 for Mantle Cell Lymphoma

On January 30, 2019 BioInvent International AB (OMXS: BINV) reported that the U.S. Food and Drug Administration (FDA) has granted the Company orphan designation for its proprietary antibody BI-1206 for the treatment of mantle cell lymphoma (MCL) (Press release, BioInvent, JAN 30, 2019, View Source [SID1234532964]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Orphan designation is intended to support companies developing treatments that target rare medical conditions and are expected to provide significant therapeutic advantage over existing medicines. The orphan designation from the FDA provides certain incentives to BioInvent, including a 7-year market exclusivity in the U.S once a New Drug Application (NDA) or Biologics License Application (BLA) has been approved, and also availability of grants exclusively for orphan drug products.

BioInvent is currently conducting a dose escalation, consecutive-cohort, open-label Phase I/IIa study of BI-1206 with approximately 30 patients across sites in the EU and the U.S. The trial evaluates BI-1206 in combination with rituximab in patients with indolent relapsed or refractory B-cell non-Hodgkin’s lymphoma (NHL). MCL is one of the targeted sub-indications, along with follicular lymphoma (FL) and marginal zone lymphoma (MZL).

"This orphan designation for BI-1206 is very good news for BioInvent, and most importantly for patients suffering from this very serious condition. There is a significant unmet medical need, as there are presently few treatment options for patients suffering from mantle cell lymphoma. We are looking forward to generating data from our Phase I/IIa trial to support the use of BI-1206 in combination with rituximab in this indication," says Martin Welschof, CEO of BioInvent.

First patient enrolled in 3rd dose cohort of Phase Ib/II study SENSITIZE

On January 30, 2019 4SC AG (4SC, FSE Prime Standard: VSC) reported the enrollment of the first patient in the third dose cohort of the ongoing Phase Ib/II study SENSITIZE (Press release, 4SC, JAN 30, 2019, View Source [SID1234532963]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The SENSITIZE study (ClinicalTrials.gov identifier: NCT03278665) is enrolling up to 40 patients suffering from unresectable advanced-stage cutaneous melanoma who are refractory or non-responding to prior treatment with anti-PD-1 antibodies (checkpoint inhibitors).

In the first part of the study, three patient cohorts will be treated at three different dose levels of domatinostat in combination with pembrolizumab. In the second part, additional patients will be treated with the recommended dosing regimen defined in the first dose-finding part of the study. 4SC anticipates first data to be available in H1 2019.

Jason Loveridge, Ph.D., CEO of 4SC: "Enrolling the first patient in the third dose cohort of the SENSITIZE study again is a significant milestone for 4SC and the study is continuing steadily. We are convinced that the combination of domatinostat with checkpoint inhibitors is the best route to most rapidly progress domatinostat towards market approval. With this, as well as the investigator-sponsored Phase II study EMERGE in advanced gastrointestinal cancer, we continue to build momentum and execute on our plan, which is intended to lead to the initiation of a potentially pivotal trial in 2019."