On April 4, 2018 Compugen Ltd. (NASDAQ: CGEN), a leader in predictive discovery and development of first-in-class therapeutics for cancer immunotherapy, reported it entered into an exclusive license agreement with MedImmune, the global biologics research and development arm of AstraZeneca, to enable the development of bi-specific and multi-specific immuno-oncology antibody products (Press release, Compugen, APR 4, 2018, View Source [SID1234525602]).

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Under the terms of the agreement, Compugen will provide an exclusive license to MedImmune for the development of bi-specific and multi-specific antibody products derived from a Compugen pipeline program. MedImmune has the right to create multiple products under this license and will be solely responsible for all research, development and commercial activities under the agreement. Compugen will receive a $10 million upfront payment and is eligible to receive up to $200 million in development, regulatory and commercial milestones for the first product as well as tiered royalties on future product sales. If additional products are developed, additional milestones and royalties would be due to Compugen. Compugen will retain all other rights to its entire pipeline of programs as monotherapies and in combination with other products.

"We are excited to announce our agreement with MedImmune, a global leader in the development of antibody-based oncology therapeutics," said Anat Cohen-Dayag, PhD, President and CEO of Compugen. "This licensing deal allows us to monetize specific scientific advances in our programs, while we continue to advance our lead programs into clinical trials." Dr. Cohen-Dayag added, "We are committed to our strategy of selectively collaborating with biopharmaceutical companies for the development of first-in-class products against our diverse, computationally-derived portfolio of targets."

"This agreement with Compugen will support our abilities to generate novel immunotherapy targets which, coupled with MedImmune’s expertise in antibody engineering, can advance our goal of delivering treatments to meaningfully improve the lives of cancer patients," said Ronald Herbst, Vice President, Oncology Research & Development, MedImmune.

Conference Call and Webcast Information

Compugen management will host a conference call today, April 2, 2018, at 8:30 a.m. ET to discuss the license agreement. To access the live conference call by telephone, please dial 1-800-994-4498 from the U.S. or +972-3-918-0608 internationally. The conference call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

About Bi-Specific and Multi-Specific Products

Antibodies are naturally occurring components of the immune system that bind specifically to a target protein via two identical arms. Through genetic engineering, antibodies can be modified to bind to different proteins through the two separate arms by exchanging one arm with that of another antibody with a different target specificity (bi-specific antibodies). Alternatively, additional features can be engineered into an antibody to allow binding to three or more target proteins simultaneously (multi-specific antibodies). These engineered forms of antibodies are increasingly being developed as therapeutics, as they enable multiple mechanisms of action for treating disease within a single molecule.

On April 4, 2018 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that patients have successfully begun treatment in its U.S. Phase I/II clinical trial of Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML") (Press release, Moleculin, APR 4, 2018, View Source [SID1234525177]).

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The first patient enrolled in Moleculin’s Annamycin clinical trial was treated at The University Hospitals Cleveland Medical Center Seidman Cancer Center on March 28, 2018.

"It is exciting to now have this trial fully under way," commented Walter Klemp, Chairman and CEO of Moleculin. "We are also pleased that the same Cancer Center has begun treatment of the second patient as well, so we are hopeful that the pace of recruitment will also meet our expectations. We continue to work toward opening additional U.S. sites to increase patients’ access to this clinical trial."

On April 4, 2018 Pfizer Inc. reported that the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application and granted priority review for dacomitinib, a pan-human epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR-activating mutations (Press release, Pfizer, APR 4, 2018, View Source [SID1234525178]). The European Medicines Agency has also accepted the Marketing Authorization Application for dacomitinib for the same indication.

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The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is in September 2018.

"While significant progress has been made in the treatment of patients with non-small cell lung cancers harboring EGFR-activating mutations, it remains a challenging disease and new treatment options are needed," said Mace Rothenberg, M.D., chief development officer, Oncology, Pfizer Global Product Development. "In the pivotal clinical trial that supports these applications, dacomitinib showed clinically meaningful improvement in progression-free survival over gefitinib, one of the first EGFR-targeted therapies to demonstrate activity in this disease. These filing acceptances are an important step toward increasing treatment options for patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer."

Dacomitinib is the second investigational Pfizer lung cancer medicine to receive regulatory acceptance within two months, reinforcing Pfizer’s commitment to patients with NSCLC where there continues to be a significant unmet need.

The submissions are based on results from the Phase 3 ARCHER 1050 study, a global head-to-head trial investigating dacomitinib (n=227) compared to gefitinib (n=225) that showed dacomitinib may offer a clinically meaningful improvement over gefitinib. Patients that received dacomitinib in the study experienced a median progression-free survival (PFS) of 14.7 months compared with 9.2 months in patients treated with gefitinib, as measured by Blinded Independent Central Review (BICR). This difference represented a 41 percent reduction in the risk of disease progression or death for patients treated with dacomitinib compared with gefitinib (HR = 0.59 [95% CI: 0.47,0.74], p <0.0001) as a first-line treatment in locally advanced or metastatic NSCLC with EGFR-activating mutations.

The adverse events (AEs) observed with dacomitinib in the study were consistent with findings from previous trials. The most common AEs were diarrhea (87%), nail changes (62%), rash/dermatitis acneiform (49%), and mouth sores (44%). The most common Grade 3 AEs with dacomitinib were rash (14%) and diarrhea (8%). Grade 4 AEs occurred in 2 percent of dacomitinib-treated patients. There was one case of Grade 5 diarrhea and one case of Grade 5 liver disease. The discontinuation rate due to treatment-related AEs for dacomitinib was 10 percent compared to 7 percent for gefitinib.

The ARCHER 1050 results were published in Lancet Oncology, shared as an oral late-breaker presentation at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and featured in the ASCO (Free ASCO Whitepaper) press program. A final assessment of overall survival from ARCHER 1050 will be presented at a medical meeting later this year.

About Dacomitinib

Dacomitinib is an investigational, oral, once-daily, irreversible pan-human epidermal growth factor receptor tyrosine kinase inhibitor (TKI). It has not received regulatory approval in any country.

In 2012, Pfizer and SFJ Pharmaceuticals Group entered into a collaborative development agreement to conduct ARCHER 1050 across multiple sites.

About Non-Small Cell Lung Cancer

Lung cancer is the leading cause of cancer deaths worldwide.1 NSCLC accounts for about 85 percent of lung cancer cases and remains difficult to treat, particularly in the metastatic setting.2 Approximately 75 percent of NSCLC patients are diagnosed late with metastatic or advanced disease where the five-year survival rate is only five percent.2,3,4

EGFR is a protein that helps cells grow and divide. When the EGFR protein is mutated it can cause cancer cells to form. EGFR mutations occur in 10 to 35 percent of NSCLC tumors globally, yet the disease is associated with low survival rates and disease progression remains a challenge. 5,6

About Pfizer in Lung Cancer

Pfizer Oncology is committed to addressing the unmet needs of patients with lung cancer, the leading cause of cancer-related deaths worldwide and a particularly difficult-to-treat disease. Pfizer strives to address the diverse and evolving needs of patients with non-small cell lung cancer (NSCLC) by developing efficacious and tolerable therapies, including biomarker-driven therapies and immuno-oncology (IO) agents and combinations. By combining leading scientific insights with a patient-centric approach, Pfizer is continually advancing its work to match the right patient with the right medicine at the right time. Through our growing research pipeline and collaboration efforts, we are committed to delivering renewed hope to patients living with NSCLC.

On April 3, 2018 Cellectis (Euronext Growth: ALCLS – Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), and Allogene Therapeutics, Inc. (Allogene), a biotechnology company focused on the rapid advancement of allogeneic CAR T therapies targeting blood cancers and solid tumors, reported that Allogene intends to assume from Pfizer the global strategic collaboration to develop "off-the-shelf" CAR T immunotherapies for oncology (Press release, Cellectis, APR 3, 2018, View Source [SID1234525157]). This agreement was initially formed with Cellectis in June 2014.

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Earlier today, Allogene and Pfizer announced that the two companies have entered into an asset contribution agreement for Pfizer’s allogeneic CAR T-cell therapy portfolio, which includes 16 preclinical assets and UCART19. Subject to certain closing conditions, Allogene will assume the strategic collaboration and license agreement with Cellectis, with exclusive rights to develop and commercialize previously defined allogeneic UCART programs directed at select targets. Cellectis will remain eligible to receive clinical and commercial milestone payments of up to $2.8 billion, or $185 million per target for 15 targets and tiered royalties in the high single digits on net sales of any products that are commercialized by Allogene under the agreement. This new alliance, with Allogene’s dedicated team, is expected to lead to a strong acceleration of CAR T therapies.

Allogene, co-founded and led by former executives of Kite Pharma, is well-positioned to catalyze the next revolution in cell therapy with its leaders’ unrivaled experience in the clinical development of autologous CAR T therapy. Arie Belldegrun M.D., FACS, Founder and former Chairman, President and Chief Executive Officer of Kite, will serve as Executive Chairman of Allogene, and David Chang, M.D., Ph.D., former Executive Vice President, Research and Development and Chief Medical Officer of Kite, will serve as President and Chief Executive Officer of Allogene.

Cellectis’ CAR T platform provides a proprietary, allogeneic approach that uses engineered T-cells from a healthy donor for use in multiple patients. This is distinct from autologous approaches that use a patient’s own T-cells to target tumor cells.

"Allogeneic represents the next transformative step in medicine, as it will potentially allow patients all over the world to quickly receive these potentially life-saving therapies in the most efficient and cost-effective way possible. The last four years of partnership with Pfizer have been very rewarding and productive. Pfizer was among the early adopters of the allogeneic approach, seeing that gene-edited CART programs are the future of immunotherapy to treat cancer. We sincerely thank everyone involved in this collaboration, from the top management to the scientists, for their foresight and their belief in our common portfolio," said Dr. André Choulika, Ph.D., Cellectis’ Chairman & Chief Executive Officer. "We strongly believe Allogene, together with Cellectis, will from the best possible team to continue this collaboration. Our expertise in allogeneic CART and gene-editing combined with the leadership of Drs. Arie Belldegrun and David Chang and their exemplary track record and execution in cell therapy paves the way for the future in off-the-shelf products enhanced by gene editing."

"We have built mutual trust and respect over the years in the CAR T industry and this collaboration will be the starting point for a long-term partnership," said David Chang, M.D., Ph.D., President and Chief Executive Officer of Allogene. "Our mission at Allogene is to catalyze the next revolution in cancer treatment through the development of allogeneic CAR T therapies directed at blood cancers as well as solid tumors. We believe this collaboration fuels our mission and we look forward to partnering with Cellectis to accelerate the development of allogeneic cell therapies."

On April 3, 2018 Eleven Biotherapeutics, Inc. (NASDAQ: EBIO), a late-stage clinical company developing next-generation antibody-drug conjugate (ADC) therapies for the treatment of cancer, reported that preliminary efficacy and safety data from its ongoing Phase 3 VISTA trial of Vicinium in patients with non-muscle invasive bladder cancer (NMIBC) who have been previously treated with bacillus Calmette-Guérin (BCG) have been selected for presentation during a plenary session currently scheduled for Monday, May 21, 2018 at 11:00 a.m. PT at the American Urological Association Annual Meeting taking place in San Francisco (Press release, Eleven Biotherapeutics, APR 3, 2018, View Source [SID1234525376]). Vicinium is a fusion protein, designed to be a next-generation ADC, that targets the epithelial cell adhesion molecule (EpCAM) antigens on the surface of bladder cancer cells to deliver a potent cytotoxin into those cells.

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Data, as reported in the abstract published online today, are from a subgroup of the first 75 evaluable carcinoma in situ patients. Within these patients, preliminary findings show that greater than 95 percent expressed EpCAM on the surface of tumor cells, the target of Vicinium. Efficacy data as measured by complete response rates at three-months will be reported during the plenary session. Vicinium has been well-tolerated. Treatment-related adverse events (AEs) were reported in 46 percent of patients, the majority of which were Grade 1 or 2. The most common of these were dysuria (12%), UTI or pollakiuria (10%) and hematuria (7%), which are often associated with catheter-delivered treatments into the bladder and bladder cancer itself. As of the December 2017 data cut off, three treatment-related serious AEs were reported, including renal failure (CTCAE Grade 5) with cholestatic hepatitis (Grade 4) in one patient and acute kidney injury (Grade 3) in a second patient who recovered.

In March 2018, recruitment was completed in the VISTA trial, a single-arm registration study designed in accordance with U.S. Food and Drug Administration’s final guidance for developing treatments for BCG-unresponsive NMIBC.
"When treatment with today’s standard of care, BCG, is no longer an option, the next treatment option is typically removal of the patient’s bladder, a challenging, life-altering procedure that many patients elect not to undergo," said Stephen Hurly, president and chief executive officer of Eleven Biotherapeutics. "Vicinium has demonstrated that it is a well-tolerated and active agent in patients with BCG unresponsive NMIBC in studies to-date. We are excited to be presenting the first preliminary data from our Phase 3 VISTA trial of Vicinium for patients with NMIBC at this year’s AUA meeting. During our plenary presentation, we will share initial efficacy findings and data supporting the favorable safety we have observed so far with Vicinium. We believe Vicinium holds tremendous potential as a treatment for bladder cancer, and we look forward to sharing these and additional data later in the year."
The company plans to host a conference call in conjunction with the data presentation, with details to follow.
About the VISTA Clinical Trial
The VISTA trial is an open-label, multicenter, single-arm Phase 3 clinical trial evaluating the efficacy and tolerability of Vicinium in patients with high-risk non-muscle invasive bladder cancer (NMIBC) that is carcinoma in situ (CIS, cancer found on the inner lining of the bladder that has not spread into muscle or other tissue) or papillary (cancer that has grown from the bladder lining out into the bladder but has not spread into muscle or other tissue), who have been previously treated with bacillus Calmette-Guérin (BCG). The primary endpoint of the trial is the complete response rate in patients with CIS with or without papillary disease and durability of that response. Patients in the study receive locally administered Vicinium twice a week for six weeks, followed by once-weekly treatment for another six weeks, then treatment every other week for up to two years. Topline data assessing responses and durability of responses at three-months on treatment are expected in mid-2018, with 12-month data anticipated in mid-2019.

About Vicinium
Vicinium, Eleven Biotherapeutics’ lead product candidate, is a next-generation antibody-drug conjugate developed using the company’s proprietary Targeted Protein Therapeutics platform. Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered linker to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical studies conducted by Eleven Biotherapeutics, EpCAM has been shown to be overexpressed in non-muscle invasive bladder cancer (NMIBC) cells with minimal to no EpCAM expression observed on normal bladder cells. Eleven Biotherapeutics is currently conducting the Phase 3 VISTA trial, designed to support the registration of Vicinium for the treatment of NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Topline, three-month data from the trial are expected in mid-2018. Additionally, Eleven Biotherapeutics believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.