Cornerstone Pharmaceuticals, Inc. Announces Corporate Name Change to Rafael Pharmaceuticals, Inc.

On June 5, 2017 Cornerstone Pharmaceuticals, Inc., a clinical-stage, oncology-focused pharmaceutical company, reported that it is changing its corporate name to Rafael Pharmaceuticals, Inc., effective immediately (Press release, Rafael Pharmaceuticals, JUN 5, 2017, View Source [SID1234521069]). The name change signifies a new era in the company’s evolution during which the company aims to usher in a new paradigm in anticancer treatment with its breakthrough metabolic cancer drugs. The new name is derived from Raphael, the biblical angel of healing.

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"Over the past few months we’ve gained a lot of momentum across several facets and this is an ideal moment to effectuate our name change," said Howard Jonas, Executive Chairman of Rafael Pharmaceuticals. "We’ve strengthened our company’s foundation by making key hires, by adding renowned scientists and clinicians to our advisory boards, and by adding leaders in the pharmaceutical and scientific research communities to our Board of Directors. Moreover, we’ve made substantial progress towards commercializing our proprietary Altered Energy Metabolism Directed (AEMD) drug platform. Most notably, we recently completed successful End-of-Phase I Type B Meetings with the FDA, and now have approval from the FDA to initiate pivotal trials for CPI-613 in acute myeloid leukemia (AML) and pancreatic cancer."

Novartis announces clinical collaboration with Bristol-Myers Squibb to evaluate potential treatments in metastatic colorectal cancer

On June 5, 2017 Novartis reported it has entered into a clinical research collaboration in which Bristol-Myers Squibb will investigate the safety, tolerability, and efficacy of Mekinist (trametinib) in combination with Opdivo (nivolumab) and Opdivo + Yervoy (ipilimumab) regimen as a potential treatment option for metastatic colorectal cancer in patients with microsatellite stable tumors where the tumors are proficient in mismatch repair (MSS mCRC pMMR) (Press release, Novartis, JUN 5, 2017, View Source [SID1234519452]).

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Under the terms of the agreement, the study will be conducted by Bristol Myers-Squibb and is expected to establish recommended dose regimens and the preliminary anti-tumor activity of the combination therapies. Both Bristol Myers-Squibb and Novartis will evaluate the results to determine optimal approaches and potential clinical development of these combinations.

"Novartis has a longstanding heritage in exploring the combination of medicines to broaden our knowledge of mutational driven cancers and develop innovative treatments," said Vas Narasimhan, MD, Head, Global Drug Development and Chief Medical Officer, Novartis. "Along with our ongoing internal immuno-oncology efforts, the expansion of our collaboration with Bristol-Myers Squibb further advances our collective goals to advance the science and to support patients in need."

About Colorectal Cancer
Worldwide, colorectal cancer is the third most common type of cancer in men and the second most common in women, with approximately 1.4 million new diagnoses in 2012. Of these, nearly 750,000 were diagnosed in men, and 614,000 in women[1]. Globally in 2012, approximately 694,000 deaths were attributed to colorectal cancer[1]. In the U.S. alone, an estimated 135,430 patients will be diagnosed with cancer of the colon or rectum in 2017, and approximately 50,000 are estimated to die of their disease[2]. There is wide variation in 5-year survival rates across disease stages, with 5-year survival rates for patients with metastatic or stage IV colorectal cancer around 11%[3]. The incidence of microsatellite stability (MSS) in colorectal tumors varies by stage, with 80% to 85% of exhibiting MSS[4].

About Mekinist and Important Safety Information
MEKINIST is a kinase inhibitor indicated, as a single agent or in combination with dabrafenib, for the treatment of patients with unresectable or metastatic melanoma with BRAF V600E or V600K mutations as detected by an FDA-approved test. MEKINIST is not indicated for treatment of patients who have received prior BRAF-inhibitor therapy. MEKINIST can cause serious side effects including new primary malignancies (cutaneous and non-cutaneous), hemorrhage, inflammation of the colon and perforation of the intestines, blood clots in the legs, cardiomyopathy, eye toxicity, interstitial lung disease, serious fevers, serious skin toxicity, hyperglycemia, and harm to a fetus. Some of these side effects can be fatal in rare cases. The most common side effects when MEKINIST is used as a single-agent are rash, diarrhea, and lymphedema.

About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.

Opdivo’s leading global development program is based on Bristol-Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has enrolled more than 25,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.

In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 60 countries, including the United States, the European Union and Japan. In October 2015, Bristol-Myers Squibb’s Opdivo and Yervoy (link is external) combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.

About Yervoy
Yervoy, which is a recombinant, human monoclonal antibody, blocks the cytotoxic T- lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activation. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation. The mechanism of action of Yervoy effect in patients with melanoma is indirect, possibly through T-cell mediated anti-tumor immune responses. On March 25, 2011, the FDA approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is now approved in more than 40 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types. This includes Phase 3 trials in prostate and lung cancers.

Polaris Lead Therapeutic ADI‑PEG 20 in Combination with First Line Chemotherapy Is Active in Argininosuccinate Synthetase Deficient Mesothelioma

On June 5, 2017 Polaris Group reported that its lead therapeutic ADI‑PEG 20 (pegylated arginine deiminase) in combination with pemetrexed and cisplatin (ADIPemCis) is active in argininosuccinate synthetase (ASS1) deficient malignant pleural mesothelioma (MPM), including non-epithelioid MPM, which carries an unfavorable prognosis and historically responds poorly to chemotherapy (Press release, Polaris Pharmaceuticals, JUN 5, 2017, View Source [SID1234526285]). The results were generated from a phase 1b dose escalation and expansion study (TRAP), led by Dr. Peter Szlosarek of Barts Cancer Center, Queen Mary University of London, and presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s 2017 annual meeting.

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Thirty one (31) MPM patients (11 epithelioid and 20 non-epithelioid) were enrolled and treated with fixed-dose pemetrexed and cisplatin (PemCis), the standard first-line chemotherapy for MPM, in combination with increasing doses of ADI‑PEG 20. The treatment appeared to be safe and well tolerated. The partial response rate was 35.5% (95% CI 19.2%-54.6%) with a disease control rate of 93.5% (95% CI 78.6%-99.2%). Median progression free survival was 5.6 months (95% CI 4-6) and median overall survival was 10.1 months (95% CI 6.7-17.7).

"Based on the encouraging efficacy signal from the TRAP study, we have initiated a randomized, double blind, phase 2/3 trial comparing ADI‑PEG 20+PemCis with PemCis in patients with non-epithelioid MPM," said John Bomalaski, M.D., Executive Vice President, Medical Affairs at Polaris Pharmaceuticals, Inc. "With ADI‑PEG 20+PemCis, we hope to bring an effective treatment to this subset of MPM patients, who have a dire need for medical intervention."

About ADI‑PEG 20

ADI‑PEG 20 is a biologic being developed by Polaris Group to treat cancers carrying a major metabolic defect that renders them unable to internally synthesize arginine. Because arginine is essential for protein synthesis and survival of cells, these cancer cells become dependent upon the external supply of arginine to survive and grow. ADI‑PEG 20 is designed to deplete the external supply of arginine, causing arginine-dependent cancer cells to die while leaving the patient’s normal cells unharmed. Multiple cancers have been reported to have a high degree of arginine-dependency and can potentially be treated with ADI‑PEG 20.

Novartis announces ground-breaking collaboration with IBM Watson Health on outcomes-based care in advanced breast cancer

On June 5, 2017 Novartis reported a first-of-its-kind collaboration with IBM Watson Health on an initiative to optimize cancer care and improve patient outcomes (Press release, Novartis, JUN 5, 2017, View Source [SID1234519453]). The two companies will collaborate to explore development of a cognitive solution that uses real-world data and advanced analytical techniques with the aim to provide better insights on the expected outcomes of breast cancer treatment options.

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With one of the broadest portfolios and largest number of advanced breast cancer compounds in development, breast cancer is a priority oncology focus area for Novartis. Scientific understanding of advanced breast cancer is improving and the treatment landscape is expanding, but new therapies and real-world evidence can raise more questions around the optimal treatment plan for individual patients.

This collaboration will join Novartis expertise in breast cancer with IBM Watson Health skill in data analytics and machine learning to determine which combinations and sequences may lead to the best patient outcomes. The initiative primarily will use real-world patient data.

"Through this collaboration with IBM Watson Health, we will use real-world breast cancer data and cognitive computing to identify solutions that may help physicians better understand which therapy may be best for which patients or advise clinical practice guidelines, with the goal of improving patient outcomes and experiences," said Bruno Strigini, CEO, Novartis Oncology. "We hope this collaboration also uncovers care efficiencies that can be applied beyond breast cancer."

"At Watson Health, we are tackling some of the world’s greatest challenges at the nexus of science and data, and we are proud to work with Novartis to better individualize advanced breast cancer treatment approaches in an effort to enhance patient care," said Anil Jain, MD, chief medical informatics officer, IBM Watson Health. "As the industry shifts toward value-based care, it’s critical for clinicians to understand the real-world outcomes of therapeutics on subsets of their patients, and our goal ultimately is to put those insights into oncologists’ hands. With the deep expertise of Novartis in breast cancer, we are confident that this collaboration may provide transformative tools for clinicians and patients to make more informed treatment decisions."

Watson is the first commercially available cognitive computing capability representing a new era in computing. The system, delivered through the cloud, analyzes high volumes of data, understands complex questions posed in natural language and proposes evidence-based answers. Watson continuously learns, gaining in value and knowledge over time, from previous interactions.

For more than 25 years, Novartis has been at the forefront of driving scientific advancements for breast cancer patients. Novartis has four marketed products in breast cancer: Kisqali* (ribociclib), Afinitor (everolimus), Tykerb/Tyverb (lapatinib) and Femara (letrozole). Novartis also has the largest number of breast cancer compounds in development, including BYL719 (alpelisib), an alpha-specific PI3K inhibitor; LSZ102, an oral SERD; LJM716, a fully human, anti-HER3 IgG1 antibody; and MCS110, a colony-stimulating factor 1 (CSF1) inhibitor.

*Kisqali is only commercially available in the United States.

ZIOPHARM Oncology Announces Positive Updated Results of Ad-RTS-hIL-12 + Veledimex in Recurrent Glioblastoma at the 2017 ASCO Annual Meeting

On June 5, 2017 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new immunotherapies, reported updated results from its Phase 1 multicenter study of Ad-RTS-hIL-12 + veledimex including the 20 mg expansion cohort in patients with recurrent or progressive glioblastoma (GBM) at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting June 2-6 in Chicago (Press release, Ziopharm, JUN 5, 2017, View Source [SID1234519448]).

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In a poster presentation titled "Expanded phase I study of intratumoral Ad-RTS-hIL-12 + oral veledimex: tolerability and survival in recurrent glioblastoma," the Company reported results from 25 patients with recurrent or progressive Grade III or IV glioma enrolled in three veledimex dosing cohorts (20 mg, n = 15; 30 mg, n = 4; and 40 mg, n = 6). Subjects with recurrent or progressive Grade III or IV glioma undergoing resection were injected intratumorally with Ad 2 x 1011 viral particles and received daily oral activator veledimex for 15 doses.

As of May 24, 2017, the cutoff date for the ASCO (Free ASCO Whitepaper) presentation, median overall survival (mOS) of all patients receiving intratumoral Ad-RTS-hIL-12 with 20 mg of orally-administered veledimex was maintained at 12.5 months, with a mean follow-up time of 9.2 months. The majority of patients in the 20 mg cohort had 2 or more recurrences prior to entry in the study, indicating very advanced disease.

"With the ability to control and turn IL-12 expression on and off from within the brain tumor, Ad-RTS-hIL-12 + veledimex offers the potential to safely direct one of the most potent anti-cancer immune cytokines against one of the most aggressive and lethal cancers," said E. Antonio Chiocca, MD, PhD, Harvey W. Cushing Professor of Neurosurgery, Department of Surgery, Harvard Medical School, Surgical Director, Center for Neuro-oncology, Dana-Farber Cancer Institute, Chairman, Neurosurgery, Brigham and Women’s Hospital and Co-Director, Institute for the Neurosciences, Brigham and Women’s Hospital.

Dr. Chiocca added, "These data suggest that intra-tumor expression of IL-12 is well tolerated by patients with recurrent glioblastoma. There are also highly encouraging observations that activation of the immune system in the patients may result in anti-tumor effects. I look forward to understanding Ad-RTS-IL-12 + veledimex’s full potential in this challenging disease in a larger study."

Based on the ratio of CD8+/FOXP3+ (effector/suppressor) T cells measured in peripheral blood 14 to 28 days after viral injection, survival appears correlated with IL-12-mediated cellular immune activation. Consistent with this observation, steroid use in the first 15 days after injection of the virus appears to have a deleterious effect on patient survival, presumably due to interference with immune activation. Drug-related toxicities, which were primarily non-neurologic, showed a dose response to veledimex, were consistent with those previously reported, and importantly, continue to be reversed upon cessation of the activator ligand, with no drug-related deaths. As previously reported, a strong, dose-dependent correlation between veledimex dose, veledimex blood brain barrier penetration, IL-12 and IFN-gamma production was also observed.

"We are now seeing a correlation between survival and cellular immune modulation, in addition to maintenance of the survival benefit in the expanded 20 mg veledimex dose cohort," said Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM. "We have uncovered that the effects of an IL-12-driven immune activation is dampened by the concurrent use of high-dose steroids. This is reflected in the survival of the patients, where patients who received less than 10 mg of dexamethasone have a much better survival than those on elevated systemic steroids. We are excited about Ad-RTS-hIL-12 + veledimex moving into a pivotal study this year, following completion of our discussions with regulators."

A copy of the poster presentation is available at www.ziopharm.com.

Details for the poster presentation at ASCO (Free ASCO Whitepaper) 2017:

Title: Expanded phase I study of intratumoral Ad-RTS-hIL-12 + oral veledimex: tolerability and survival in recurrent glioblastoma
Abstract Number: 2044
Session: Central Nervous System Tumors
Date and Time: Monday, June 5, 2017, 1:15 — 4:45 p.m. CT

Ad-RTS-hIL-12 + veledimex

Ad-RTS-hIL-12 + veledimex is a novel, viral gene therapy candidate for the controlled expression of interleukin-12 (IL-12), a pro-inflammatory cytokine critical for stimulating anti-cancer immune responses.

Recurrent Glioblastoma (GBM)

Glioblastoma represents approximately 15% of all primary brain tumors and remains a high unmet clinical need that affects roughly 74,000 people worldwide annually.i,ii GBM is an aggressive form of brain cancer with recurrence rates near 90%, and prognosis for patients is poor with treatment often combining multiple approaches including surgery, radiation, and chemotherapy. Patients with recurrent GBM typically have a mOS of 6-7 months, and overall survival in patients who have failed temozolomide, bevacizumab or equivalent salvage chemotherapy, is approximately 3-5 months.iii,iv