On November 14, 2016 MabVax Therapeutics Holdings, Inc. (NASDAQ: MBVX), a clinical stage immuno-oncology drug development company, reported on progress from its two HuMab-5B1 antibody phase I programs evaluating the use of MVT-5873 as a therapeutic antibody and MVT-2163 as an immuno-PET imaging agent in patients with locally advanced and metastatic pancreatic cancer or other CA19-9 positive malignancies.

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MVT-5873 Interim Phase I Data in Pancreatic Cancer
The MVT-5873 phase I clinical trial initiated in February 2016 is designed to establish safety and determine the recommended phase II dose (RP2D) for MVT-5873 as both as monotherapy (Part 1 of the trial), and in combination with standard of care chemotherapy (Part 2) using nab-paclitaxel plus gemcitabine. Initiation of Part 2 requires establishing three safe dose levels for MVT-5873 as monotherapy in patients with relapsed or refractory locally advanced or metastatic pancreatic cancer. The Company reports that the safety of MVT-5873 has been established at three incremental dose levels by treating 16 patients at three clinical sites. While patients continue to be recruited to establish the RP2D, the Company also reports that Part 2 of the clinical trial is now open and will include patients with previously untreated pancreatic cancer receiving a standard of care chemotherapy as defined in the protocol.

To date, the study has consented 28 subjects with 3 in screening, 9 screen failures, and 16 subjects treated. Of the 16 patients treated, six continue to receive treatment. Patients can remain on therapy based on dose tolerability and investigator assessment of continued benefit including assessment of disease status using RECIST 1.1 criteria to evaluate tumor response rate and duration of response. Stable disease was noted for seven patients lasting from three months to eight months. The dosage safety levels established in Part 1 of the trial also support the dosage levels of MVT-5873 to be used in conjunction with the company’s MVT-2163 immuno-PET imaging agent and the MVT-1075 radioimmunotherapy product which is planned to begin phase I clinical evaluation early in 2017.

MVT-2163 Interim Phase I Data in Pancreatic Cancer
The MVT-2163 phase I trial was initiated in June of this year to evaluate the company’s next generation diagnostic PET imaging agent in patients with locally advanced or metastatic adenocarcinoma of the pancreas (PDAC) or other CA19-9 positive malignancies. MVT-2163 (89Zr-HuMab-5B1) combines the well-established PET imaging radiolabel Zirconium [89Zr] with the targeting specificity of MVT-5873. This trial is designed to establish safety, pharmacokinetics, biodistribution, and the amount of MVT-5873 to be used in co-administration to obtain optimized PET scan images. The company has demonstrated interim safety, pharmacokinetics, and biodistribution by completing the initial two cohorts of patients: the first cohort administered MVT-2163 alone and the second cohort administered MVT-2163 following a blocking dose of MVT-5873. The company reports that the initial PET images demonstrated target specificity by correlation with lesions identified by conventional computerized tomography (CT) scans. The biodistribution data obtained in the first two cohorts demonstrates improvement in PET images by pre-administration of MVT-5873, as has been observed with other antibody based PET agents. The company is actively recruiting patients and expects to establish the optimal co-administration dose of MVT-5873 early in 2017.

"Our strategy at the outset was to initiate these two clinical trials concurrently to address the key questions of safety and targeting specificity for the HuMab-5B1 antibody. We are highly encouraged by these promising early results from the MVT-5873 and MVT-2163 clinical trials," stated President and CEO J. David Hansen. He added, "We are moving ahead with the planned combination of MVT-5873 with a standard of care chemotherapy in a chemotherapy-naïve pancreatic cancer patient population and are looking forward to presenting these results next year. We are continuing to accrue patients in order to establish the RP2D for MVT-5873 as a monotherapy. In the MVT-2163 trial, dose escalation continues to confirm optimal dose and timing for the best PET scan image. Finally, the company remains on track for submitting the Investigational New Drug Application (IND) for MVT-1075 to the Food And Drug Administration (FDA) later this year, and plans to initiate the phase I trial of MVT-1075 in the first half of 2017 after receiving FDA authorization to proceed."

On November 14, 2016 RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) ("RedHill" or the "Company"), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, reported its financial results for the quarter ended September 30, 2016 (Press release, RedHill Biopharma, NOV 14, 2016, View Source [SID1234516721]).

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The Company will host a conference call on Monday, November 14, 2016, at 9:00 am EST to review the financial results and business highlights, dial-in details are included below.

Financial highlights for the quarter ended September 30, 20161

Research and Development Expenses for the third quarter of 2016 were $7.0 million, an increase of $3.1 million, compared to $3.9 million in the third quarter of 2015 and an increase of $1.0 million, compared to $6.0 million in the second quarter of 2016. Research and Development Expenses for the nine months ended September 30, 2016 were $17.7 million, an increase of $4.9 million, compared to $12.8 million in the comparable period of 2015. The increase in 2016 resulted primarily from the ongoing Phase III MAP US study with RHB-104 (Crohn’s disease), the ongoing Phase II and Phase III studies with BEKINDA (IBS-D and gastroenteritis, respectively) and from preparations for several Phase I/II studies with YELIVA(TM) for multiple indications.

General, Administrative and Business Development Expenses in the third quarter of 2016 were $1.4 million, an increase of $0.7 million, compared to $0.7 million in the third quarter of 2015 and an increase of $0.2 million, compared to $1.2 million in the second quarter of 2016. General, Administrative and Business Development Expenses for the nine months ended September 30, 2016 were $3.8 million, an increase of $1.4 million, compared to $2.4 million in the comparable period of 2015. The increase was mainly due to enhanced business development and investor relations activities.

Operating Loss in the third quarter of 2016 was $8.5 million, an increase of $3.9 million, compared to $4.6 million in the third quarter of 2015 and an increase of $1.3 million, compared to $7.2 million in the second quarter of 2016. Operating Loss for the nine months ended September 30, 2016 were $21.6 million, an increase of $6.4 million, compared to $15.2 million in the comparable period of 2015. The increase was mainly due to increases in Research and Development Expenses, as detailed above.

Net Cash Used in Operating Activities in the third quarter of 2016 was $7.4 million, an increase of $3.7 million, compared to $3.7 million in the third quarter of 2015 and an increase of $1.7 million, compared to $5.7 million in the second quarter of 2016. Net Cash Used in Operating Activities for the nine months ended September 30, 2016 was $18.1 million, an increase of $6.3 million, compared to $11.8 million in the comparable period of 2015. The increase mainly reflects the increase in Operating Loss, as detailed above.

Net Cash Used in Investment Activities in the third quarter of 2016 was $10.7 million, compared to Net Cash Used in Investment Activities of $2.4 million in the third quarter of 2015. Net Cash Used in Investment Activities for the nine months ended September 30, 2016 was $3.2 million, an increase of $4.3 million, compared to Net Cash Provided by Investment Activities of $1.1 million in the comparable period of 2015. The increase in Net Cash Used in Investment Activities was due to an increase in bank deposits and marketable securities in 2016.

Cash Balance2 as of September 30, 2016 was $40.5 million, a decrease of $17.6 million, compared to $58.1 million as of December 31, 2015. The decrease was a result of the ongoing operations, mainly related to research and development activities.

"We are very pleased with the important operational milestones achieved during this quarter, including the significant enhancements to the RHB-104 Phase III development program for Crohn’s disease and the initiation of several clinical studies with our novel Phase II oncology drug YELIVA(TM)" said Mr. Micha Ben Chorin, RedHill’s CFO. "We maintained a debt-free balance sheet and a cash position of $40.5 million at the end of the third quarter of 2016 as we continue to advance several Phase III and Phase II gastrointestinal programs toward important data points in the coming months. We also continue to advance our strategic plan to build a U.S. specialty GI pharmaceutical company."

Conference Call and Webcast Information:

The Company will host a conference call on Monday, November 14, 2016, at 9:00 am EST to review the financial results and business highlights.

To participate in the conference call, please dial the following numbers 5-10 minutes prior to the start of the call: United States: +1-877-280-2342; International: +1-646-254-3367; and Israel: +972-3-721-9510. The access code for the call is 2341628.

The conference call will be broadcasted live and available for replay on the Company’s website, View Source, for 30 days. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

Recent operational highlights:

On July 5, 2016, RedHill and its co-development partner, IntelGenx Corp. (IntelGenx), announced the signing of an exclusive license agreement with Grupo JUSTE S.A.Q.F (Grupo JUSTE), for the commercialization of RIZAPORT oral thin-film for acute migraines. Under the terms of the agreement, RedHill granted Grupo JUSTE the exclusive rights to register and commercialize RIZAPORT in Spain and a right of first refusal for a predefined term for additional territories. RedHill and IntelGenx received an upfront payment and are entitled to receive additional milestone payments upon the achievement of certain predefined regulatory and commercial targets, as well as tiered royalties. Further financial terms of the agreement were not disclosed. Grupo JUSTE recently filed a national Marketing Authorization Application for RIZAPORT in Spain and commercial launch in Spain is expected to take place in the second half of 2017.

On July 13, 2016, RedHill announced the signing of a research collaboration agreement with the U.S. National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), intended to evaluate RedHill’s proprietary experimental therapy for the treatment of Ebola virus disease. The new research collaboration follows encouraging results from preliminary non-clinical studies conducted in conjunction with the NIAID using RedHill’s proprietary experimental therapy. If successful, this study is intended to provide supportive data for discussions with the FDA for potential use of the Animal Rule pathway for approval. Ebola virus disease is a severe and often fatal illness, which can cause severe hemorrhagic fever in humans and has a mortality rate ranging from 25% to 90%3. There is currently no FDA-approved treatment for Ebola virus disease.

On July 21, 2016, RedHill announced that it had received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a new patent covering RHB-105. The patent application, entitled "Pharmaceutical Compositions For The Treatment Of Helicobacter Pylori"expands RedHill’s patent portfolio covering RHB-105 and is expected to be valid until 2034, once granted.

On August 1, 2016, RedHill announced that the last patient completed the final scheduled follow-up visit in the Phase IIa proof-of-concept clinical study evaluating RHB-104 in patients treated for relapsing-remitting multiple sclerosis (the CEASE-MS study). The analysis of the study is ongoing, with top-line final results expected in Q4/2016. Previously announced interim results after completion of the 24-week RHB-104 treatment period of the study demonstrated positive safety and efficacy signals and support further clinical development.

On August 29, 2016, RedHill announced that it had received from the European Patent Office a Notice of Intention to Grant for a new patent covering the use of RHB-104 in the treatment of multiple sclerosis. Upon grant by the European Patent Office, the patent is expected to be valid until 2032 and can be officially validated in up to 38 European countries.

On September 8, 2016, RedHill announced that a Phase Ib/II clinical study evaluating YELIVA(TM) in patients with refractory or relapsed multiple myeloma was initiated. The open-label, dose escalation Phase Ib/II study is being conducted at Duke University Medical Center and will enroll up to 77 patients with refractory or relapsed multiple myeloma who have previously been treated with proteasome inhibitors and immunomodulatory drugs. The study is supported by a $2 million grant from the National Cancer Institute Small Business Innovation Research Program awarded to Apogee Biotechnology Corp., in conjunction with Duke University, with additional support from RedHill.

On September 12, 2016, RedHill announced a research collaboration with Stanford University School of Medicine for the evaluation of YELIVA(TM). The research collaboration is intended to complement RedHill’s planned Phase Ib clinical study to evaluate YELIVA(TM) as a radioprotectant for prevention of mucositis in head and neck cancer patients undergoing therapeutic radiotherapy. Results from the research collaboration are expected in mid-2017.

On September 21, 2016, RedHill and IntelGenx, announced that they had entered into a binding term sheet with Pharmatronic Co., granting Pharmatronic Co. the exclusive license to commercialize RIZAPORT in the Republic of Korea (South Korea). Under the term sheet, RedHill and IntelGenx are to receive an upfront payment and will be eligible to receive additional milestone payments upon achievement of certain predefined regulatory and commercial targets, as well as tiered royalties. Subject to the satisfaction of the remaining conditions, the parties will endeavor to enter into a definitive agreement within 60 days of the execution of the term sheet. Further financial terms of the term sheet were not disclosed.

On October 5, 2016, RedHill announced that a Phase II clinical study evaluating YELIVA(TM) in patients with advanced hepatocellular carcinoma (HCC) was initiated at the Hollings Cancer Center at the Medical University of South Carolina (MUSC), subject to IND clearance by the FDA. The study is supported by a grant from the U.S. National Cancer Institute (NCI) awarded to MUSC, with additional support from RedHill. The HCC study protocol is still under FDA review. Enrolment in the study is expected to begin following successful completion of the FDA’s review process, anticipated by end of 2016.

On October 6, 2016, RedHill provided an update on the RHB-104 Phase III Crohn’s disease development program, planned enhancements to the MAP US first Phase III study and expected milestones, including:

An increase in the total number of patients planned to be enrolled in the study from 270 to 410 and the addition of an open-label extension study offering all patients who complete 26 weeks of study participation and remain out of remission (Crohn’s disease active index (CDAI) > 150) the opportunity to receive treatment with RHB-104 for a 52-week period.

A first safety-focused independent DSMB meeting is on track to take place in the fourth quarter of 2016. A second independent DSMB meeting is expected in the second quarter of 2017 and will include safety and interim efficacy analysis, with evaluation of an early stop for success for overwhelming efficacy, under pre-specified efficacy criteria.

Two small-scale, open-label ex-U.S. clinical studies with RHB-104, each with up to 20 Crohn’s disease patients, are planned to be initiated and are intended to provide additional supportive clinical data to potential future marketing applications, as well as to evaluate RHB-104’s efficacy in newly diagnosed and treatment-naïve Crohn’s disease patients, and as an add-on therapy to current standard-of-care.

RedHill will remain blinded to the interim and ongoing results from the Phase III study and no changes are planned to the MAP US Phase III study’s primary endpoint or 90% power.

Assuming enrollment of all 410 planned subjects, completion of patient recruitment in the MAP US Phase III study is expected by the end of 2017.
On October 18, 2016, RedHill announced that it had received from the Japan Patent Office a Notice of Allowance for a new patent covering RHB-104 for the treatment of multiple sclerosis, which is expected to be valid until 2032, once granted.

On November 1, 2016, RedHill announced that it had intended to offer its American Depositary Shares (ADSs), each representing 10 of its ordinary shares, in an underwritten public offering. RedHill subsequently announced, on November 2, 2016, that it had withdrawn the proposed underwritten public offering of its ADSs due to market conditions.

On November 1, 2016, RedHill announced that it had entered into a non-binding term sheet with a pharmaceutical company as part of its potential strategic vertical integration plan to build a U.S. specialty pharmaceutical company by establishing a commercial presence and capabilities. Under the term sheet, RedHill would be granted the right to exclusively promote a specialty gastrointestinal product in certain territories in the U.S. The parties would share revenues generated in such territories based on an agreeable split between the parties. RedHill is not required to make any upfront or milestone payments under the term sheet. Although RedHill’s goal is to complete the transaction pertaining to the commercial asset in the fourth quarter of 2016, the term sheet is non-binding and there is no certainty as to the execution nor timing of execution of a definitive agreement between RedHill and its potential partner. There is no assurance that satisfactory due diligence will be completed or that the parties will obtain all necessary corporate approvals.

On November 3, 2016, RedHill announced that top-line results from both the ongoing Phase III clinical study with BEKINDA 24 mg for acute gastroenteritis and gastritis (the GUARD study) and the ongoing Phase II clinical study with BEKINDA 12 mg for diarrhea-predominant irritable bowel syndrome (IBS-D) are expected in mid-2017. Over two-thirds of the planned total of 320 subjects have been enrolled in the Phase III GUARD study with BEKINDA 24 mg for acute gastroenteritis and gastritis in the U.S. Approximately half of the planned total of 120 subjects have been enrolled in the Phase II clinical study with BEKINDA 12 mg for the treatment of IBS-D in the U.S.

On November 10, 2016, RedHill announced that it has concluded a positive Type B Meeting with the U.S. Food and Drug Administration (FDA) discussing the chemistry, manufacturing and controls (CMC) aspects of the RHB-105 Phase III development program. The confirmatory Phase III study with RHB-105 for H. pylori infection is planned to be initiated in H1/2017, after completion of the ongoing supportive PK program. Subject to a successful outcome, the confirmatory Phase III study and the supportive PK program are expected to complete the package required for a U.S. NDA for RHB-105, including clinical data and CMC.

On November 14, 2016 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer and other hypoxia-related diseases, reported financial results for the three months ended September 30, 2016 and provided an overview of recent corporate highlights (Filing, Q3, RestorGenex, 2016, NOV 14, 2016, View Source [SID1234516713]). The third quarter results will be filed on the Quarterly Report on Form 10-Q with the SEC.

David Kalergis, Chairman and Chief Executive Officer, stated, "I am very excited about the progress that we have made in advancing the clinical development of our lead candidate, trans sodium crocetinate (TSC). The completion of the reverse stock split and subsequent uplisting to the Nasdaq Capital Market is an important step for the Company and I am extremely pleased that we have reached this stage of growth. The successful completion of our three month animal toxicology studies is also an important milestone in support of Diffusion’s readiness to conduct a Phase 3 pivotal trial of TSC in newly diagnosed GBM patients. Our newly assembled Scientific Advisory Board will serve as a valuable resource as we prepare to begin this Phase 3 trial, and will also guide our research as we seek to develop TSC for therapeutic use in other hypoxia-related diseases."

Corporate Highlights

In August 2016, Diffusion announced a 1-for-10 reverse stock split in preparation for its proposed uplisting to Nasdaq Capital Market.

In September 2016, Diffusion announced the successful completion of animal toxicity studies in preparation for a Phase 3 pivotal trial of TSC in newly diagnosed GBM patients.

In September 2016, the Company also established a Scientific Advisory Board of distinguished experts to serve as a resource for the development of TSC in its many areas on therapeutic use for indications involving hypoxic conditions.

In November 2016, the Company subsequently announced that its shares of common stock were approved for listing on the Nasdaq Capital Market, effective November 9, 2016.

Three Months Ended September 30, 2016 Financial Results

Research and development expenses were $1.9 million for the three months ended September 30, 2016, compared to $0.9 million for the three months ended September 30, 2015. This increase was primarily a result of the $1.0 million non-cash impairment charge upon the abandonment of the future development efforts of the RES-440 IPR&D asset acquired from RestorGenex.

General and administrative expenses were $3.9 million for the three months ended September 30, 2016, compared to $0.4 million for the three months ended September 30, 2015. The increase was primarily attributable to a $2.5 million non-cash litigation settlement with an investor in September 2016 and an increase in incremental costs in connection with operating as a public company.

Net loss was $5.4 million, or $0.53 per share, for the three months ended September 30, 2016, compared to a net loss of $1.4 million, or $0.64 per share, for the three months ended September 30, 2015. The increase in the net loss was due primarily to higher expenses associated with the increased research and development expenses and general and administrative expenses.

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On November 14, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the third quarter ended September 30, 2016 (Press release, Onconova, NOV 14, 2016, View Source [SID1234516681]).

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"Onconova continues to reach important milestones in the development of rigosertib for patients with myelodysplastic syndromes (MDS). In July, our partner SymBio Pharmaceuticals announced the enrollment of the first patient in Japan for our INSPIRE pivotal trial for rigosertib in 2nd-line higher-risk MDS (HR-MDS). In September, we announced the results of a successful End-of-Phase 2 meeting with the FDA for oral rigosertib in combination with azacitidine for 1st-line HR-MDS patients," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "We are pleased with the progress of our oral rigosertib development program, as well as the INSPIRE trial that is now running on four continents with more than 150 trial-sites in 15 countries."

Recent Business Highlights:

Progress in Oral Rigosertib Combination with Azacitidine for 1st-line HR-MDS

Following evaluation of 54 enrolled patients in the Phase 2 Trial 09-08 of oral rigosertib plus azacitidine, Onconova conducted an End-of-Phase 2 meeting during which updated results from this trial were discussed with the FDA. Based on these discussions, and guidance from the Agency, Onconova will design a randomized, controlled Phase 3 clinical trial comparing the combination of oral rigosertib plus azacitidine to azacitidine plus placebo in hypomethylating agent (HMA) naïve HR-MDS patients. This sizable population of MDS patients not previously treated with HMAs is a poorly met medical need where the front-line treatments (HMAs) are effective for only a fraction of the indicated patients. Notably, in contrast to the INSPIRE trial, where the primary efficacy endpoint was Overall Survival (OS), the new pivotal trial will employ Response Rate (RR) as the approval endpoint, permitting more rapid completion and evaluation of the study. The RR will be a composite of complete remission (CR) and partial remission (PR).
Progress in INSPIRE Pivotal Trial of IV Rigosertib in 2nd-line HR-MDS

The global Phase 3 INSPIRE trial of IV rigosertib in patients who have failed to respond to or progressed with an HMA therapy is now enrolling in the United States, Europe, Australia, and Japan. As of October 3, 2016, 157 sites were open for the INSPIRE trial to recruit patients.
The INSPIRE trial was recently reviewed in a pre-planned first meeting of the Drug Safety Monitoring Board. Following a review of the safety data of enrolled patients, the Board recommended continuation of the trial without any modifications.
Key Opinion Leader Meeting on Novel Approaches to Targeting RAS

Onconova hosted an investor event featuring two pioneers in the area of RAS biology, Dr. Channing J. Der of the University of North Carolina, and Dr. E. Premkumar Reddy of Mount Sinai School of Medicine. The meeting focused on novel approaches for targeting the RAS pathway and highlighted the therapeutic potential for rigosertib as a novel RAS-directed therapy. A replay of the webcast for this event can be found by clicking the following link: View Source
Recent Rigosertib Publications

Two peer-reviewed articles describing clinical and non-clinical studies with rigosertib in MDS were published in Expert Review of Anticancer Therapy (link to article) and Expert Opinion on Orphan Drugs (link to article).
Upcoming Events

Presentation of updated data from 09-08 combination therapy trial at ASH (Free ASH Whitepaper) Annual Meeting: December 2016
Presentations highlighting safety and tolerability in more than 500 patients, and analysis of previously completed randomized clinical trial of rigosertib at ASH (Free ASH Whitepaper) Annual Meeting: December 2016
Completion of site activation for INSPIRE trial: 1Q2017
Third Quarter 2016 Financial Results

Cash, cash equivalents and marketable securities as of September 30, 2016, totaled $25.8 million, compared to $19.8 million as of December 31, 2015.
Total net revenue was $1.7 million for the third quarter of 2016 and $5.4 million for the nine months ended September 30, 2016, compared to $1.6 million and $1.9 million, respectively, for the comparable periods in 2015.
Research and development expenses were $4.0 million for the third quarter of 2016 and $15.4 million for the nine months ended September 30, 2016, compared to $5.3 million and $21.3 million, respectively, for the comparable periods in 2015.
General and administrative expenses were $2.0 million for the third quarter of 2016 and $7.2 million for the nine months ended September 30, 2016, compared to $2.2 million and $7.8 million, respectively, for the comparable periods in 2015.

On November 11, 2016 Compugen Ltd. (NASDAQ: CGEN), a leading predictive drug discovery company, reported new data for its preclinical CGEN-15029 program demonstrating the potential for the development of new cancer immunotherapy treatments for solid tumors, including the potential for drug combination with current immune checkpoint blockers (Filing, 6-K, Compugen, NOV 14, 2016, View Source [SID1234516652]). CGEN-15029 is the internal designation for PVRIG, a novel immune checkpoint identified by Compugen utilizing its in silico predictive discovery infrastructure. The Company plans to file an IND in 2017 for COM701, its lead antibody targeting PVRIG.

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Today’s presentation, titled "Computational identification, functional characterization and antibody blockade of a new immune checkpoint in the TIGIT family of interacting molecules" was made by John Hunter, Ph.D., Site Head and Vice President, Antibody R&D, Compugen USA, Inc. The presentation included the discovery by Compugen of PVRIG and its expression pattern in the context of cancer, demonstrating that PVRIG is expressed in infiltrating immune cells in solid tumors, specifically on cells with potent anti-tumor activity, such as effector T-cells and natural killer cells. Also disclosed was the identification of PVRL2 as PVRIG’s ligand, and the functional characterization and anti-tumor in vitro and in vivo efficacy of blocking antibodies developed by Compugen targeting the interaction of PVRIG with PVRL2. Binding of PVRIG to PVRL2 is of particular therapeutic interest, as it links PVRIG to the axis of a second immune checkpoint, TIGIT, which is recently gaining traction in the field of immuno-oncology.

In his presentation, Dr. Hunter presented additional data demonstrating that antibodies developed by Compugen, which block the interaction between PVRIG and PVRL2, enhance activation of the immune system by activating both primary CD4+ T-cells and tumor-derived CD8+ T-cells.

Consistent with the placement of PVRIG in the TIGIT axis, antibody blockade of both TIGIT and PVRIG had an additive effect on human T-cell stimulation, indicating the potential of the two to generate enhanced immune response against the cancer. Moreover, antibodies targeting the mouse PVRIG developed by the Company were assessed in vivo for effects on tumor growth inhibition in mouse models, commonly used to study immune checkpoint inhibitors. In these studies, antibodies that block the mouse PVRIG/PVRL2 interaction, similar to those generated against the human target, were shown to inhibit tumor growth when used in combination with PD1 pathway blockade. These results were further reinforced when tumor growth was tested in knock-out (KO) mice. Tumor growth was significantly reduced in KO mice, where the PVRIG gene was removed. Consistent with the antibody combination data, this effect was even further enhanced in the KO mice when they were treated with anti-PDL1 blocking antibodies.

Collectively, the experimental data strongly suggest that PVRIG, which was initially predicted computationally by the Company to serve as a novel immune checkpoint target, presents a new opportunity for the development of cancer immunotherapy treatments, including the potential for drug combination with current immune checkpoint blockers.

In June 2016, the Company selected COM701, a high affinity antagonist antibody against PVRIG, as the lead therapeutic candidate for the program. COM701, which is currently in preclinical development by the Company, demonstrates potent and reproducible enhancement of T-cell activation, consistent with the desired mechanism-of-action required to generate anti-tumor immune responses.

Anat Cohen-Dayag, Ph.D., CEO and President of Compugen, explained, "While antibody blockade of the CTLA4 and PD1 pathways has emerged as an effective treatment modality for certain types of cancer, the majority of patients do not derive long-term benefits, suggesting a need for additional approaches such as new immune checkpoints targeting new pathways and providing new mechanisms to activate the immune response against the tumor. Employing our unique predictive infrastructure to define new immune checkpoint targets, we identified PVRIG, among other novel immune checkpoint target candidates in our target pipeline."

Dr. Cohen-Dayag continued, "We are very pleased to see the rapidly increasing amount of preclinical data demonstrating the potential utility of COM701, an antibody targeting PVRIG, as a new cancer immunotherapy treatment. These results highlight, once more, the power and uniqueness of our computational predictive approach – from computer prediction of novel drug targets to preclinical validation. This capability, along with our significantly enhanced development infrastructure, allows us now to pursue and advance a number of novel immuno-oncology programs with potentially different mechanisms-of-action, thus providing us with a diversified early-stage internal target pipeline, in addition to the two programs that are the subject of an ongoing pharma collaboration."

About PVRIG
PVRIG (designated internally as CGEN-15029) is one of the novel B7/CD28-like immune checkpoint target candidates discovered by Compugen utilizing its predictive discovery infrastructure. The CGEN-15029 target was predicted in silico and experimentally confirmed to be a receptor-like immune checkpoint protein expressed on immune cells. In June 2016, COM701, a high affinity antagonist antibody against CGEN-15029, was selected as the lead therapeutic candidate for the program.