On November 28, 2018 HOOKIPA Pharma Inc. ("HOOKIPA"), a clinical stage biopharmaceutical company developing a new class of immuno-therapeutics targeting infectious diseases and cancers based on its proprietary technology platform, reported that it entered into a research collaboration and license agreement with DarwinHealth to develop novel immunotherapies based on the systematic discovery and prioritization of the next generation of immunogenic, tumor-specific cryptic antigens (Press release, Hookipa Biotech, NOV 28, 2018, View Source [SID1234531680]). DarwinHealth is a precision-focused cancer medicine company, utilizing systems-biology derived algorithms to identify appropriate therapies for cancer patients and to systematically discover and prioritize the next generation of immunogenic, tumor-specific antigens.

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Under the terms of the agreement, DarwinHealth will utilize a combined single-cell transcriptome analysis and bioinformatics-based approach using both mouse and human cancerous and non-cancerous tissues to identify the next generation of shared "off-the-shelf" tumor-specific antigens. HOOKIPA will perform the validation experiments and be granted exclusive rights to the development, manufacturing and commercialization of products arising from the collaboration.

"Immunotherapy is one of the areas of greatest potential for future cancer treatment. Arguably, all antigen-specific immunotherapy is limited by the scarcity of known antigens. The current group of shared tumor self-antigens has been established for many years, and while more recent efforts have been aimed at identifying patient-specific neo-antigens, systematic attempts to identify the next generation of tumor self-antigens have not been pursued as aggressively," said Dr. Igor Matushansky, Chief Medical Officer and Global Head of Research and Development of HOOKIPA. "This is why the primary objective of our next generation, antigen discovery program – entitled HIDE [Human Immunotranscript Discovery initiativE]" – with DarwinHealth is to identify a novel constellation of shared self-antigens for multiple tumor subtypes. Our goal is that following a successful completion of the two-year program, antigens with validated immunogenicity will be deployed clinically as antigen-specific, vector-mediated immunotherapy using our TheraT* vector."

"DarwinHealth utilizes proprietary, systems biology-generated algorithms to match cancer patients with the drugs and drug combinations that are most likely to produce a successful treatment outcome. These same algorithms can also be used to prioritize investigational drugs and compound combinations of unknown potential against a full spectrum of human malignancies, as well as novel cancer targets," explained Andrea Califano, Dr. co-founder of DarwinHealth and Clyde and Helen Wu Professor of Chemical Systems Biology and Chair, Department of Systems Biology at Columbia University, "and importantly, for immuno-oncology applications, DarwinHealth can apply proprietary bioinformatics- and experimentally-based methodologies to identify human, cryptic immunogenic transcripts (crypto-antigens) that can be optimally delivered using HOOKIPA’s highly innovative vector."

Commenting on this immuno-oncology-focused collaboration, Gideon Bosker, CEO and co-founder of DarwinHealth said: "Combining HOOKIPA´s proprietary viral vector technology with DarwinHealth´s novel, systems-based approach to identifying next generation, shared antigens shows immense promise, potentially enabling cancer research to leap forward and transform patients´ lives."

On November 28, 2018 Oncorus, Inc., an oncolytic virus company focused on driving innovation to transform outcomes for cancer patients, reported the nomination of ONCR-177, a next-generation locally administered oncolytic virus clinical candidate for multiple solid tumor indications (Press release, Oncorus, NOV 28, 2018, View Source [SID1234531681]). ONCR-177 utilizes Oncorus’ proprietary, next-generation oncolytic herpes simplex virus (oHSV) platform. In addition, Oncorus has created a unique synthetic oncolytic virus platform, which will enable the company to develop multiple oncolytic viruses for repeated, systemic administration. Proprietary innovations in potency and safety engineered by Oncorus into both platforms enable best-of-class potential for the company’s portfolio programs and the opportunity to pursue multiple cancer types.

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"While we’ve seen early promise of the ability of oncolytic virus therapies to improve the response rates of checkpoint inhibitors, many patients still do not respond to these agents. Our team is committed to changing this fact," said Theodore (Ted) Ashburn, M.D., Ph.D., President and Chief Executive Officer of Oncorus. "We are driving multiple proprietary innovations across two distinct platforms that are enabling a portfolio of oncolytic virus product candidates with best-in-class potential. We intend to become the leaders in realizing the full potential of this exciting treatment modality to dramatically transform outcomes for cancer patients."

Dr. Ashburn will give an update on the company’s pipeline and business at a presentation today at 7:30 AM EST at the Piper Jaffray 30th Annual Health Care Conference in New York City, highlighting key accomplishments from 2018 to date and outlining near- and long-term value drivers for the company.

Key 2018 Accomplishments:

Nomination of lead clinical candidate, ONCR-177. Oncorus recently nominated a lead clinical candidate, ONCR-177, a locally administered oncolytic virus therapy. ONCR-177 features key innovations engineered into Oncorus’ next-generation oHSV platform, including the largest number of immunomodulatory payloads in the class. These payloads include IL-12, FLT3L, CCL4 and antagonists of the clinically proven checkpoint inhibitors, CTLA-4 and PD-1, to enable the recruitment and activation of T cells, natural killer (NK) cells and dendritic cells and increase the likelihood of productive anti-tumor responses. ONCR-177 is also a fully-replication competent virus in that it retains the ability to expresses γ34.5, which allows the virus to replicate in the presence of host antiviral immune responses, a feature unique among oHSVs that have been developed to date or are being developed currently. ONCR-177 also features Oncorus’ proprietary microRNA-attenuation strategy, which leverages the differential expression of microRNA sequences to allow robust viral replication in tumor cells, while preventing replication in healthy tissue. In addition, Oncorus has developed a complementary and orthogonal approach to protecting neurons from viral infection by engineering mutations in UL37, an HSV protein, which prevent both axonal retrograde transport and latency.
The company intends to file an investigational new drug application (IND) and start a first-in-human study by the end of 2019 in order to evaluate ONCR-177 in multiple solid tumor indications.

"The promise of oncolytic viruses as a therapeutic tool in the fight against cancer has been clear for some time. However, a fundamental challenge to date has been the need to sacrifice potency to ensure safety," said Christophe Quéva, Ph.D., Oncorus’ Chief Scientific Officer. "Our team has been working diligently to enhance our oHSV platform in a number of innovative ways to successfully overcome this ‘potency versus safety’ tradeoff. We are very encouraged by our progress to date and look forward to seeing how our innovations improve outcomes in clinical studies involving ONCR-177."

Achieved in vivo proof-of-concept for synthetic virus program for repeat, systemic administration. Oncorus has made notable progress advancing its research programs for systemic intravenous delivery for its breakthrough synthetic virus platform. Systemic delivery of oncolytic viruses offers the promise of targeting indications not amenable to locally administered oncolytic virus therapy such as cancers of the lung. The company recently achieved in vivo proof-of-concept for its systemic virus platform and aims to nominate a lead clinical candidate synthetic virus by the end of 2019.
Solidified leadership with key hires. Oncorus made key management hires in 2018 that helped expand the company’s expertise, solidify its leadership team, and position it for rapid growth. In September, the company announced that Theodore (Ted) T. Ashburn, M.D., Ph.D., was appointed President and CEO. Dr. Ashburn brings a wealth of experience in immuno-oncology drug development and commercialization to Oncorus. Mitchell H. Finer, Ph.D., Oncorus’ co-founder, founding CEO and MPM Capital Managing Director, assumed the role of Executive Chairman. Other key hires include John Goldberg, M.D., Senior Vice President, Clinical Development; Brett Belongia, Ph.D., Senior Director of CMC; Brian Haines, Ph.D., Senior Director Pharmacology, Toxicology; and Tooba Cheema, Ph.D., Director of Biomarkers and Translational Medicine.
Closed the third and final tranche of Series A round of financing. Oncorus closed a third and final tranche of its Series A financing, which raised a total of $61.4 million. The company’s blue-chip investor syndicate includes founding investor MPM Capital, as well as MPM’s UBS Oncology Impact Fund (OIM), Deerfield Management, Arkin Bio Ventures, Long March, Astellas and Celgene.
Continued to build a robust intellectual property (IP) estate. Oncorus continued to pursue a broad IP strategy and augment its current patent estate surrounding its proprietary innovations for both its oHSV and synthetic virus platforms and respective applications.
2019 Goals:

Oncorus’ key goals and upcoming value drivers include:

Filing an IND and initiating a first-in-human Phase 1 study involving ONCR-177
Nominating a lead synthetic virus clinical candidate for repeat systemic administration, and
Continuing to expand its team with the recruitment of key talent across multiple disciplines
"It’s an exciting time to be in the oncolytic virus therapy space, given the continued progress of this modality and its potential to become an important addition to the arsenal of immuno-oncology therapies available to cancer patients," said Dr. Finer. "2018 has been a year of important growth for Oncorus on both the scientific and business fronts. We have clear momentum as we approach 2019 and look forward to transitioning into a clinical stage company, progressing our portfolio of best-in-class products and, above all, realizing the promise of oncolytic virus therapy for cancer patients."

On November 28, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that nine abstracts, including two oral presentations, relating to the company’s hematology/oncology portfolio were accepted for the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego from December 1-4 (Press release, Jazz Pharmaceuticals, NOV 28, 2018, View Source;p=RssLanding&cat=news&id=2378498 [SID1234531666]).

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"We look forward to showcasing our commitment to the hematology/oncology community at ASH (Free ASH Whitepaper) by advancing the science and addressing the clinical needs of patients with blood cancers and with complications of stem cell transplantation," said Allen Yang, M.D., Ph.D., head of clinical development and acting chief medical officer of Jazz Pharmaceuticals. "We’ve made great progress in the last year with the initiation of new clinical trials for Vyxeos and Defitelio, the marketing authorization of Vyxeos in the European Union, and a collaboration with MD Anderson Cancer Center to evaluate potential treatment options for hematologic malignancies."

Jazz Pharmaceuticals data at the 2018 ASH (Free ASH Whitepaper) Annual Meeting will highlight the following:
Results from a post-hoc analysis from the pivotal Phase 3 randomized trial of Vyxeos (daunorubicin and cytarabine) liposome for injection, also known as the molecule name CPX-351, in a subgroup of older patients with newly diagnosed high risk (secondary) AML with myelodysplasia-related changes (AML-MRC) that evaluated the efficacy of Vyxeos compared to conventional 7+3 chemotherapy on overall survival and remission rates as well as the safety profile in older adults with AML-MRC. Additionally, data will be presented from an exploratory analysis which evaluated the impact of hematopoietic cell transplantation (HCT) on survival in patients treated with Vyxeos compared to 7+3 in the Phase 3 trial.

Vyxeos abstracts include:

Efficacy and Safety of CPX-351 versus 7+3 in a Subgroup of Older Patients with Newly Diagnosed Acute Myeloid Leukemia with Myelodysplasia-Related Changes (AML-MRC) Enrolled in a Phase 3 Study [Abstract #1425; Saturday, December 1, 6:15 PM – 8:15 PM PST]
Population Pharmacokinetic (PK)/Pharmacodynamic (PD) Modeling of Myelosuppression in Patients with Hematologic Malignancies for CPX-351 and Standard-of-Care 7+3 Therapy [Abstract #4037; Monday, December 3, 6:00 PM – 8:00 PM PST]
The Impact of Hematopoietic Cell Transplantation on Survival: An Exploratory Analysis of a Phase 3 Study of CPX-351 versus 7+3 in Older Patients with Newly Diagnosed, High-Risk/Secondary AML [Abstract #2706; Sunday, December 2, 6:00 PM – 8:00 PM PST]
Final Safety and Efficacy Results from the CPX-351 Early Access Program (EAP) for Older Patients with High-Risk/Secondary Acute Myeloid Leukemia (sAML) [Abstract #1434; Saturday, December 1, 6:15 PM – 8:15 PM PST]
Impact of Post–Hematopoietic Cell Transplant (HCT) Survival on Cost-effectiveness of CPX-351 versus 7+3 in the Treatment of Therapy-Related AML or AML-MRC in the United States (online only)
A Phase I/Pilot Study of CPX-351 [Daunorubicin and Cytarabine Liposome for Injection (Vyxeos)] for Children, Adolescents and Young Adults with Recurrent or Refractory Acute Leukemia [Abstract #336; Sunday, December 2, 10:45 AM PST(oral presentation)]
In addition, data from an expanded access program and post-hoc analyses of clinical trials for Defitelio (defibrotide sodium) will be presented, including an oral presentation of a pooled analysis of survival based on timing of initiation in adults with Veno-Occlusive Disease/ Sinusoidal Obstruction Syndrome (VOC/SOS) following hematopoietic stem cell transplant (HSCT).

Defitelio abstracts include:

Incidence of Post-Hematopoietic Stem Cell Transplantation (HSCT) Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Without Hyperbilirubinemia at Diagnosis and Efficacy of Defibrotide in an Expanded-Access Program [Abstract #2080; Saturday, December 1, 6:15 PM – 8:15 PM PST]
A Pooled Analysis of Survival by Defibrotide Timing of Initiation in Adults with Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) Following Hematopoietic Stem Cell Transplant (HSCT) [Abstract #815; Monday, December 3, 3:45 PM PST (oral presentation)]
Cost-Effectiveness of Defibrotide for the Treatment of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Multi-Organ Dysfunction (MOD) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Canada [Abstract #4702; Monday, December 3, 6:00 PM – 8:00 PM PST]
About Vyxeos
Vyxeos (daunorubicin and cytarabine) is a liposome formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion, and is approved for the treatment of two types of secondary AML in adult patients, newly diagnosed therapy-related acute myeloid leukaemia (t-AML) and AML with myelodysplasia-related changes (AML-MRC). Vyxeos is the first product developed with the company’s proprietary CombiPlex platform, which enables the design and rapid evaluation of various combinations of therapies. Vyxeos received U.S. FDA approval and orphan drug exclusivity in August 2017 and EU EMA marketing authorization in August 2018. Vyxeos received Orphan Drug Designation for the treatment of AML by the U.S. FDA in September 2008 and by the European Commission in January 2012 (with retention of the designation reaffirmed in July 2018). Vyxeos received Promising Innovative Medicine (PIM) designation from the Medicines and Healthcare Products Regulatory Agency in the United Kingdom.

Important Safety Information
Vyxeos has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute Vyxeos for other daunorubicin- and/or cytarabine- containing products.

Vyxeos should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine or any of its ingredients.

Vyxeos can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with Vyxeos. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

Vyxeos can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles or legs
unusual tiredness
Vyxeos may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:
trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
Vyxeos contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

Vyxeos can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

Vyxeos can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving Vyxeos. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of Vyxeos.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Please see full Prescribing Information for Vyxeos including BOXED Warning, and visit www.Vyxeos.com for additional information.

About Defitelio
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. FDA marketing approval on March 30, 2016 for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication.

In Europe, defibrotide is marketed under the name Defitelio* (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

*This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC. (View Source)

Important Safety Information

Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

Please see full Prescribing Information for Defitelio and visit www.Defitelio.com for additional information.

On November 28, 2018 Cold Genesys, Inc. reported that it has entered into a clinical collaboration with Merck (known as MSD outside the United States and Canada) to evaluate the combination of Cold Genesys’ lead oncolytic immunotherapy candidate CG0070 with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab), in a Phase 2 clinical study (Press release, Cold Genesys, NOV 28, 2018, View Source [SID1234531682]).

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The trial is planned to evaluate the preliminary safety and efficacy of CG0070 plus KEYTRUDA in patients with Non-Muscle Invasive Bladder Cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG), an unmet medical need, as no treatment for this indication has been approved by the FDA in approximately 20 years.

CG0070, an investigational oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene, destroys bladder tumor cells through their defective retinoblastoma (Rb) pathway, which is present in many cancers. Several CG0070 clinical studies including a completed Phase 2 study (BOND2) have demonstrated a favorable safety profile and encouraging efficacy in patients with NMIBC following BCG failure.

"We are delighted to be collaborating with Merck on this innovative combination approach," said Arthur Kuan, CEO of Cold Genesys. "CG0070, which has demonstrated clinical safety and efficacy in over 100 patients for the treatment of NMIBC, may potentially exhibit additional effect when combined with KEYTRUDA, which also has demonstrated single agent activity in the indication."

Cold Genesys will sponsor the study. Additional details of the collaboration were not disclosed.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About CG0070

CG0070, a selectively replicative oncolytic immunotherapy based on a modified adenovirus type 5 backbone that contains a cancer-selective promoter and a GM-CSF transgene, destroys bladder tumor cells through their defective Rb pathway. CG0070 was designed to work in two complementary ways. First, it replicates inside the tumor’s cells with dysfunctional Rb pathways, causing tumor cell lysis and immunogenic cell death. Then, the rupture of the cancer cells can release tumor-derived antigens, along with GM-CSF, which can stimulate a systemic anti-tumor immune response that involves the body’s own white blood cells. In advanced clinical studies, CG0070 has been shown to be a safe and efficacious agent in NMIBC following BCG failure. The scientific rationale and clinical results to date of CG0070 make it a promising agent to be developed for a variety of solid tumor types to be used alone or in combination with immune checkpoint modulators.

On November 28, 2018 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that Julian Adams, Ph.D., the company’s chief executive officer, will participate in a fireside chat at the BMO Capital Markets 2018 Prescriptions for Success Healthcare Conference on Wednesday, December 12th at 1:20 p.m. ET in New York, NY (Press release, Gamida Cell, NOV 28, 2018, View Source [SID1234531683]).

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A live webcast of the presentation will be available on the Investors section of the Gamida Cell website, at www.gamida-cell.com and will be available for at least 30 days following the event.