On June 1, 2020 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, reported that the U.S. Food and Drug Administration has cleared the investigational new drug (IND) application for its Phase 1/2 study evaluating the triple combination of COM701, Compugen’s first-in-class anti-PVRIG antibody, Opdivo (nivolumab), Bristol Myers Squibb’s PD-1 immune checkpoint inhibitor and BMS-986207, Bristol Myers Squibb’s investigational anti-TIGIT antibody, in patients with advanced solid tumors (Press release, Compugen, JUN 1, 2020, View Source [SID1234558790]).

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The triple combination study is designed to evaluate the simultaneous blockade of three immune checkpoint pathways, PVRIG, TIGIT and PD-1, and will accelerate the clinical evaluation of Compugen’s DNAM axis hypothesis and biomarker-driven approach in patients with advanced
solid tumors, including those that may be refractory or non-responsive to standard-of-care immune checkpoint inhibitors. The study is expected to commence in the second half of 2020.

"This study complements our clinical strategy designed to evaluate the blockade of PVRIG as a monotherapy and in combination with intersecting DNAM axis components to fully elucidate the role of this potentially foundational axis for cancer immunotherapy," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "The encouraging clinical data from the ongoing COM701 Phase 1 study evaluating our discovered target PVRIG, and the emerging clinical validation of the TIGIT pathway, leave us increasingly enthusiastic about our science-driven clinical approach evaluating these two complementary yet distinct pathways in combination with PD-1. Importantly, as the only company with wholly-owned clinical candidates against both PVRIG and TIGIT, we are uniquely positioned to address the role of the DNAM axis."

Henry Adewoye, M.D., Senior Vice President and Chief Medical Officer of Compugen, added, "This study evaluating COM701 in combination with BMS-986207 and nivolumab fits our overall clinical development strategy of advancing novel therapies in cancers with high unmet medical need. We have previously reported partial responses with COM701 as monotherapy and in combination with nivolumab in patients with extremely challenging to treat cancer types, such as MSS platinum resistant primary peritoneal cancer and MSS-CRC. These preliminary clinical data support our preclinical work indicating that PVRIG and PD-1 are distinct pathways and that inhibition of the two may result in clinical benefit to patients. The triple combination will further test our science-driven hypothesis that PVRIG, TIGIT, and PD-1 are non-redundant inhibitory pathways and that their simultaneous blockade is expected to further enhance anti-tumor immune responses and broaden the patient population responsive to cancer immunotherapies. We look forward to collaborating with Bristol Myers Squibb on this important study."

Under this IND, the Company intends to initiate an open-label Phase 1/2 trial designed to evaluate the safety, tolerability and preliminary antitumor activity of COM701 in combination with Opdivo and BMS-986207 during dose escalation, as well as anti-tumor activity in selected tumor types in the expansion cohorts. Dose levels for Opdivo and BMS-986207 combinations have already been determined through prior testing by Bristol Myers Squibb, allowing for dose escalation of COM701 with fixed doses of Opdivo and BMS-986207.

About COM701
COM701 is a humanized antibody that binds with high affinity to PVRIG, a novel immune checkpoint discovered computationally by Compugen, and blocks the interaction with its ligand, PVRL2. TIGIT, an immune checkpoint discovered computationally by Compugen in 2009, and PVRIG constitute parallel immune checkpoint pathways that counteract DNAM, a costimulatory receptor on T cells and NK cells. Preclinical data suggest that the blockade of PVRIG induces a robust anti-tumor immune response and demonstrates synergistic activity when used in combination with inhibitors of TIGIT and/or PD-1. Currently, COM701 is being evaluated in a Phase 1 clinical study. Data from the ongoing study have shown that COM701 is well-tolerated and demonstrated preliminary signals of anti-tumor activity in a heavily pretreated patient population.

On June 1, 2020 Alligator Bioscience (Nasdaq Stockholm: ATORX), reported that the company received an additional USD 0.5 million in a second installment of the upfront payment from the Chinese company Biotheus Inc (Press release, Alligator Bioscience, JUN 1, 2020, View Source [SID1234558789]). A first installment of USD 0.5 million was received in August 2019 in connection with Alligator entering into a licensing agreement with Biotheus with a total value up to USD 142 million. The agreement concerns the Chinese rights to an immune-activating antibody from the antibody library ALLIGATOR-GOLD, with the intention of creating up to three new bispecific molecules.

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"It is very gratifying that Biotheus has chosen to continue the collaboration after conducting a thorough scientific and technical evaluation of our antibody. It serves as an additional validation of our antibody library and of our capacity to develop immune-activating antibodies, while giving Alligator further presence in the important Chinese pharmaceutical market," said Per Norlén, CEO Alligator Bioscience.

The license agreement includes exclusive rights to an immune-activating antibody directed to a receptor within the Tumor Necrosis Factor Receptor-superfamily (TNFR-SF), a collective name for a number of target molecules with similar functions, and applies to the China, Hong Kong, Taiwan and Macau markets.

In addition to the received upfront payment totaling USD 1 million, the license agreement gives Alligator the right to receive additional milestones and option fees at a potential total value of up to USD 141 million, as well as royalties on future sales and share of any sub-license revenue.

This information is such information as Alligator Bioscience AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. This information was submitted for publication, through the agency of the contact person set out above, at 1:30 p.m. CEST on June 1, 2020.

On June 1, 2020 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Intellia Therapeutics, Inc. (NASDAQ: NTLA) reported an expansion of their existing collaboration to provide Regeneron with rights to develop products for additional in vivo CRISPR/Cas9-based therapeutic targets and for the companies to jointly develop potential products for the treatment of hemophilia A and B (Press release, Intellia Therapeutics, JUN 1, 2020, View Source [SID1234558786]). Regeneron also receives non-exclusive rights to independently develop and commercialize ex vivo gene edited products. Intellia will receive an upfront payment of $70 million, and Regeneron will make an additional equity investment in Intellia of $30 million at $32.42 per share.

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Regeneron and Intellia have worked together to make significant advances with Intellia’s CRISPR/Cas9 platform to enable the targeted insertion of therapeutic proteins and antibodies. This collaboration expansion allows the companies to leverage more fully their jointly-developed targeted transgene insertion capabilities and potentially accelerate efforts to discover and develop new therapeutics, including products for hemophilia A and B. In preclinical studies, the companies demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates, which generated normal or higher levels of circulating human Factor IX. Factor IX is a blood-clotting protein that is missing or defective in hemophilia B patients. These results suggest that transgene insertion may provide a functional Factor 9 gene, which encodes for this important protein.

"The Regeneron team works hard to push the boundaries of science and technology, and we believe the precise in vivo gene insertion capabilities jointly developed with Intellia could be a promising therapeutic platform with significant potential in many diseases, including those that have been historically difficult to treat," said George D. Yancopoulos, M.D., Ph.D., Co-Founder, President and Chief Scientific Officer, Regeneron. "We’re pleased to expand our work with Intellia, a like-minded group of scientists focused on maximizing the potential of CRISPR/Cas9 in order to help as many patients as possible."

"We’re excited to work with Regeneron on what could potentially be a cure for hemophilia A and B in this expansion of our successful collaboration that builds on our leading insertion capabilities," said Intellia’s Chief Executive Officer and President, John M. Leonard, M.D. "We believe that our CRISPR/Cas9-based technology addresses the limitations of current replacement and gene therapy approaches, and importantly, may provide a durable, potentially life-long solution to these genetic diseases."

Under the amended agreement, the term of the companies’ existing collaboration is extended until April 2024, with Regeneron having an option to renew for an additional two years. Regeneron will have rights to discover and develop CRISPR/Cas9-based therapeutic products for an additional five in vivo liver targets, for a total of up to 15 targets. As currently set forth in the existing collaboration, the parties will jointly research these in vivo targets, and thereafter certain targets may be developed by Regeneron or Intellia, or co-developed under certain conditions. Per the terms of the original agreement, Regeneron will pay potential royalties and milestone payments for the in vivo products it independently develops. In addition, Regeneron will receive a royalty-bearing, non-exclusive license to certain Intellia intellectual property to develop and commercialize up to 10 ex vivo CRISPR/Cas9 products in defined cell types.

On June 1, 2020 VBL Therapeutics (Nasdaq: VBLT), reported the presentation of the positive outcome of pre-planned interim analysis results from the OVAL Phase 3 pivotal clinical trial in platinum-resistant ovarian cancer at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 virtual annual meeting (Press release, VBL Therapeutics, JUN 1, 2020, View Source [SID1234558783]).

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The analysis compared the CA-125 objective response rate (ORR) according to GCIG criteria in the treatment and control arms among the first 60 evaluable patients. The CA-125 ORR in those patients was 53%. Assuming a balanced randomization, the response rate in the treatment arm (VB-111 in addition to weekly paclitaxel) was 58% or higher. In patients who had post-dosing fever, which is a marker for VB-111 treatment, the response rate was 69%.

The CA-125 response rate observed in the Phase 3 interim analysis is at least as good as the response rate seen in the prior Phase 2 study, which enrolled a similar patient population and showed overall survival benefit. In the previously reported Phase 2 study of VB-111 in platinum resistant ovarian cancer, 58% of the patients treated with VB-111 and paclitaxel demonstrated a CA-125 response. Those patients with a CA-125 response demonstrated a median overall survival of 808 days, versus 351 days for those patients without a CA-125 response.

"The OVAL interim data are very encouraging as they demonstrate the potential benefit of VB-111 over standard-of-care in a randomized-controlled setting," said Tami Rachmilewitz, M.D., Vice President Clinical Development of VBL Therapeutics. "With over 25% of the patients already enrolled in the study, we look forward to further advancing the OVAL study by expansion to Europe and Japan later this year."

For VBL’s presentation at ASCO (Free ASCO Whitepaper) see: Link

About the OVAL (VB-111-701/GOG-3018) study (NCT03398655)
OVAL is an international Phase 3 randomized, double-blind, placebo-controlled potential registration clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with the GOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)
VB-111 is a first-in-class, targeted anti-cancer gene-therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an "all comers" Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received an Orphan Designation for the treatment of ovarian cancer from the European Commission. VB-111 has also received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).

On June 1, 2020 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the first quarter ended March 31, 2020, and provided a business update on recent corporate and clinical developments (Press release, Protalix, JUN 1, 2020, View Source [SID1234558782]). The Company’s management will discuss the financial results and provide a clinical, corporate and financial highlights on a conference call and live webcast scheduled for Monday, June 1, 2020 at 8:30 am Eastern Daylight Time (EDT).

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"The first quarter of 2020 has most certainly been transformational for Protalix, despite the COVID-19 pandemic that affected the global markets," said Dror Bashan, Protalix’s President and Chief Executive Officer. "I am proud to say that despite the pandemic, Protalix was able to keep the company running smoothly and adapt quickly to the changing environment."

"During the quarter, we were able to close a $43.7 million private placement," he continued. "Furthermore, the topline results from the completion of our Phase III BRIDGE study and the subsequent BLA submission for PRX-102 announced in May prove that Protalix has actually gained momentum by leaning into this unprecedented challenge. I am convinced now more than ever that our team is positioned for long-term success and look forward to continuing our momentum through the rest of this year and into 2021."

Conference Call and Webcast Information

The Company will host a conference call on Monday, June 1, 2020, at 8:30 am, Eastern Daylight Time, to review the clinical, corporate and financial highlights. To participate in the conference call, please dial the following numbers prior to the start of the call:

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

First Quarter 2020 and Recent Business Highlights

Clinical and Regulatory Advancements

·On May 28, 2020, the Company and its development and collaboration partner, Chiesi Global Rare Diseases, a unit of Chiesi Farmaceutici S.p.A., or Chiesi, announced the submission on May 27, 2020 of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for pegunigalsidase alfa, or PRX-102, for the treatment of adult patients with Fabry disease via the FDA’s Accelerated Approval pathway. PRX-102 was granted Fast Track designation by the FDA in January 2018. Upon the BLA approval, if approved, the Company will be eligible to receive a milestone payment from Chiesi.

·On May 11, 2020, the Company announced positive topline results following the completion of its Phase III BRIDGE clinical trial of PRX-102 for the treatment of Fabry disease. The Phase III BRIDGE clinical trial, a 12-month open-label, single arm switch-over study evaluating the safety and efficacy of PRX-102, 1 mg/kg infused every two weeks, met its main objectives for safety and efficacy, and topline analysis indicated substantial improvement in renal function as measured by mean annualized estimated Glomerular Filtration Rate (eGFR slope) in patients switched from agalsidase alfa to PRX-102.

·On February 6, 2020, Protalix and Chiesi announced the receipt of an agreement letter from the FDA for the Initial Pediatric Study Plan (iPSP) for PRX-102 for the treatment of Fabry disease, outlining an agreed-upon approach to address the needs of pediatric Fabry patients.

Corporate & Financial Developments

·On March 16, 2020, the Company announced that it has agreed to conduct a feasibility study with Kirin Holdings Company, Limited, or Kirin, to evaluate the production of a novel complex protein utilizing ProCellEx. The Company received a non-refundable payment of $1.0 million and Kirin will provide research funding for the Company’s scientists to conduct cell line engineering and protein expression studies on the target protein.

·On March 12, 2020, the Company entered into securities purchase agreements with certain existing and new institutional and other accredited investors in a private placement. Pursuant to such agreements, the Company issued and sold to the purchasers an aggregate of approximately 17.6 million unregistered shares of its common stock at a price per share of $2.485, or aggregate net committed proceeds equal to approximately $41.3 million. Each share of the Company’s common stock issued in the transaction was accompanied by a warrant to purchase an additional share of common stock at an exercise price equal to $2.36.

Financial Results

For the three months ended March 31, 2020, compared to the three months ended March 31, 2019

·The Company recorded revenues from selling goods of $5.0 million during the three months ended March 31, 2020, an increase of $1.5 million, or 43%, compared to revenues of $3.5 million for the same period of 2019. The increase resulted primarily from an increase of $0.8 million in sales of drug product to Brazil as well as an increase of $0.7 million in sales of drug substance to Pfizer Inc.

·Revenues from license and R&D services for the three months ended March 31, 2020, were $16.6 million, an increase of $9.7 million, or 140%, compared to revenues of $6.9 million for the same period of 2019. Revenues from the license agreements represent the revenues recognized in connection with previously announced agreements with Chiesi. The increase is primarily due to revenues recognized in connection with the progress of the Company’s clinical trial that have been performed, and with revenues recognized in connection with an updated costs estimation throughout the trials until completion in the amount of $6.7 million.

·Cost of goods sold was $3.4 million for the three months ended March 31, 2020, an increase of $1.4 million, or 68%, from cost of goods sold of $2.0 million for the same period of 2019. The increase is primarily due to an increase in sales of goods.

·Research and development expenses were $10.3 million for the three months ended March 31, 2020, a decrease of $1.4 million, or 12%, compared to $11.7 million of research and development expenses for the same period of 2019. The decrease was primarily due to a decrease in costs related to manufacturing of our drug in development.

·Selling, general and administrative expenses were $3.2 million for the three months ended March 31, 2020, an increase of $1.0 million, or 43%, compared to $2.2 million for the same period of 2019. The increase resulted primarily from a $0.6 million increase in compensation related costs and a $0.2 million increase in professional fees.

·Net income for the three months ended March 31, 2020 was $1.7 million, or $0.10 per share, basic and diluted, compared to a net loss of $7.3 million, or $0.50 per share, basic and diluted, for the same period of 2019.