On February 21, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results for the fourth quarter and full year ended December 31, 2018 and provided an update on recent corporate activities (Press release, Halozyme, FEB 21, 2019, View Source [SID1234533533]).

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"Our latest collaboration with argenx demonstrates the broad applicability of our ENHANZE drug delivery technology and is a great way to start the year," said Dr. Helen Torley, president and chief executive officer. "We anticipate multiple additional key milestones in our ENHANZE business including potential approval of Herceptin SC later this quarter and potential regulatory submissions for a subcutaneous formulation of Darzalex in the second half of the year. With a total of nine ENHANZE collaborations spanning from established products to new, innovative therapies addressing unmet needs, we remain confident in the potential for $1 billion in royalty revenue in 2027."

"Our PEGPH20 oncology program achieved a critical milestone during the fourth quarter with the completion of enrollment in our pivotal HALO-301 pancreas cancer study with approximately 500 patients. We also reached agreement with the FDA to change the primary endpoint for HALO-301 to a single primary endpoint of overall survival (OS), which we believe incrementally de-risked the study. We look forward with excitement to topline results from HALO-301, which we currently project in the second half of 2019."

Fourth Quarter 2018 and Recent Highlights Include:

In February 2019, Halozyme licensed its ENHANZE drug delivery technology to argenx providing exclusive access to ENHANZE for any product targeting the human neonatal Fc receptor FcRn, including argenx’s lead asset efgartigimod (ARGX-113), and up to two additional targets. Under the terms of the agreement, argenx paid an upfront payment of $30 million to Halozyme, and will pay Halozyme $10 million per target for future target nominations and potential future payments of up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones. Halozyme will also receive mid-single digit royalties on sales of commercialized products.

In January 2019, the U.S. Food and Drug Administration completed its review of the submitted clinical study protocol amendment and statistical analysis plan for HALO-301, which included a change in the primary endpoint to a single primary endpoint of overall survival (OS), with no additional questions or comments.

In December, enrollment in HALO-301, the company’s Phase 3 study evaluating PEGPH20 in metastatic pancreas cancer, was completed with approximately 500 subjects enrolled. The company projects the study will achieve its target of 330 OS events between August and November of 2019. Based on achieving this timeline, the company projects topline results will be available in the second half of 2019.

In December, Roche dosed the first patient in a Phase 1b/2 study of Tecentriq (atezolizumab) with ENHANZE triggering a $5 million milestone payment to Halozyme.

During the fourth quarter, BMS began recruitment for a Phase 1 study of OPDIVO (nivolumab) with ENHANZE.

ENHANZE partner Janssen continued to make progress in clinical studies for a subcutaneous co-formulation of Darzalex (daratumumab) with the recent initiation of two additional Phase 3 trials. Janssen is planning regulatory filings in the second half of 2019.

In October, Halozyme expanded its collaboration with Roche by licensing its ENHANZE drug-delivery technology for exclusive development of a new undisclosed clinical stage therapeutic target resulting in an upfront payment of $25 million.

Fourth Quarter and Full Year 2018 Financial Highlights

Revenue for the fourth quarter was $60.2 million compared to $189.6 million for the fourth quarter of 2017. The year-over-year decrease was driven by $141.4 million upfront license fees for the BMS and Alexion agreements and a $15.0 million milestone payment from Janssen recognized in 2017, compared to $30.0 million in upfront and milestone revenue for the Roche collaboration recognized in 2018. The decrease was offset by a 9 percent growth in royalties on a reported basis from partner sales. Revenue for the fourth quarter included $19.3 million in royalties and $4.2 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Revenue for the full year was $151.9 million, compared to $316.6 million in 2017.
Revenue from royalties for the full year was $79.0 million, up 24% on an as-reported basis compared to $63.5 million in 2017.

Research and development expenses for the fourth quarter were $36.7 million, compared to $41.4 million for the fourth quarter of 2017.
Research and development expenses for the full year were $150.3 million, compared to $150.6 million in 2017.

Selling, general and administrative expenses for the fourth quarter were $18.0 million, compared to $14.8 million for the fourth quarter of 2017.
Selling, general and administrative expenses for 2018 were $60.8 million, compared to $53.8 million in 2017.

Net loss for the fourth quarter was $2.1 million, or $0.01 per share, compared to net income in the fourth quarter of 2017 of $123.9 million, or $0.85 per share.
Net loss for the full year was $80.3 million, or $0.56 per share, compared to net income of $63.0 million in 2017, or $0.45 per share.

Cash, cash equivalents and marketable securities were $354.5 million at December 31, 2018, compared to $469.2 million at December 31, 2017.

Financial Outlook for 2019

Halozyme updated its 2019 financial guidance, first provided on January 9, 2019, to reflect the recent argenx collaboration and license agreement:

Net revenue of $205 million to $215 million, excluding revenue from any additional, new ENHANZE global collaboration and licensing agreements;

Operating expenses of $265 million to $275 million, or $225 million to $235 million excluding an expected increase in cost of goods sold. Excluding the cost of goods sold the modest increase in expenses is driven by ENHANZE partner support, and support of the potential commercialization of PEGPH20;

Operating cash burn of $45 million to $55 million;

Debt repayment of approximately $90 million, the company expects to pay off the remainder of the royalty-backed debt by the end of the first quarter of 2020;

Year-end cash, cash equivalents and marketable securities balance of $210 million to $220 million.

Webcast and Conference Call
Halozyme will webcast its Quarterly Update Conference Call for the fourth quarter of 2018 today, Thursday, February 21 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a replay will be available following the close of the call. To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (877) 410-5657 (domestic callers) or (334) 323-7224 (international callers) using passcode 387156. A telephone replay will be available after the call by dialing (877) 919-4059 (domestic callers) or (334) 323-0140 (international callers) using replay ID number 68892505.

On February 21, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported that it will present at the following conference (Press release, MorphoSys, FEB 21, 2019, View Source [SID1234533550]):

8th Annual Leerink Partners Global Healthcare Conference
Date: February 28, 2019, 2:30 pm EST (8:30 pm CET, 7:30 pm GMT)
Venue: New York, NY, USA
Presenter: Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG
Dr. Julia Neugebauer, Director Corporate Communications & IR

PDF versions of the presentations will be provided at www.morphosys.com. The link to the webcasts will be filed under www.morphosys.com/conference-calls.

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On February 21, 2019 City of Hope reported that it has received its third lymphoma Specialized Programs of Research Excellence (SPORE) grant from the National Cancer Institute (NCI), one of four current NCI-supported lymphoma SPOREs (Press release, City of Hope, FEB 21, 2019, View Source [SID1234533556]). The grant covers a five-year period and totals $12.5 million.

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SPOREs – a cornerstone of the NCI’s efforts to promote collaborative, interdisciplinary translational cancer research – involve both basic and clinical/applied scientists working together to support projects that will result in new and diverse approaches to the prevention, early detection, diagnosis and treatment of human cancers. This interdisciplinary research is currently advanced in the Toni Stephenson Lymphoma Center, which is the foundation of City of Hope’s Hematologic Malignancies and Stem Cell Transplantation Institute.

"For many years now, City of Hope has led the way in pioneering bone marrow transplantation, immunotherapy and other innovative treatment options for lymphoma patients, both those who are being diagnosed with the disease for the first time and those who have experienced a relapse," said Stephen J. Forman, M.D., City of Hope Francis & Kathleen McNamara Distinguished Chair in Hematology and Hematopoietic Cell Transplantation and leader of the Hematologic Malignancies and Stem Cell Transplantation Institute. "This renewal of lymphoma SPORE will make it possible for us to continue developing leading-edge therapies in our laboratories that will ultimately reach a patient’s bedside."

"City of Hope will do this by developing novel therapeutics and prognostics representing the forefront of knowledge gained from observations in molecular biology and cellular immunology at City of Hope," said Larry W. Kwak, M.D., Ph.D., vice president/deputy director of City of Hope’s comprehensive cancer center, director of the Toni Stephenson Lymphoma Center and the Dr. Michael Friedman Professor in Translational Medicine. "Six clinical trials are proposed in this grant, five of which utilize agents (cellular products, small molecules, radiolabeled antibodies) that will be produced at City of Hope in its Good Manufacturing Practice Manufacturing Core and have been developed from the institution’s preclinical laboratory studies."

The grant is led by Forman and Kwak as multi-principal investigators. This is the 11th year City of Hope has received funding from the NCI for a lymphoma SPORE grant.

The Toni Stephenson Lymphoma Center has helped to create the collaborative, fast-paced culture in which the projects in the new SPORE will be advanced. It is home to many of the accomplished faculty and leading experts who are conducting clinical trials that have come about with SPORE funding, and has a track record of developing new therapies for many types of lymphoma.

Established by Emmet and Toni Stephenson, and their daughter Tessa Stephenson Brand, the Toni Stephenson Lymphoma Center brings together physicians and scientists in a highly-interdisciplinary infrastructure to accelerate City of Hope’s research on the biological mechanisms of lymphoma, identify new molecular targets and produce immunotherapies to treat the disease. In addition, the Stephenson Pilot Grant program funds new ideas for lymphoma or treatment platforms that can be innovatively applied across disease sites, including lymphoma. The cores in the SPORE award are also supported in part by Stephenson funds.

Over the next five years, City of Hope doctors and researchers, as well as scientists from other institutions, will focus on the following projects for the SPORE grant:

CAR T therapy and vaccine combination for non-Hodgkin’s lymphoma

After patients with non-Hodgkin’s lymphoma (NHL) have received a blood stem cell transplant with their own stem cells or that of a donor, there could still be enough cancerous cells not killed by the procedure for the disease to relapse. City of Hope is on a quest to improve current treatment for these patients, as well as those with NHL who are unable to receive a transplant. A treatment option for these patients is combining a transplant with chimeric antigen receptor (CAR) T cell therapy; during the last round of lymphoma SPORE funding, City of Hope started a clinical trial for NHL patients that used a treatment combining a transplant with CD19 CAR T cells. (Researchers published the positive treatment results in the journal Blood and also presented on the research at the American Society of Hematology (ASH) (Free ASH Whitepaper) conference.) The therapy works by taking a person’s immune cells – T cells – and adding a CAR that helps target and wipe out cancerous cells. The institution is now looking for new ways to improve the treatment.

One option that will be tested is adding CD19-specific CAR, which targets the CD19 protein in cancerous B cells, to a virus-specific T cell that can be stimulated to multiply using a vaccine. One such example is the well-studied cytomegalovirus (CMV) – it has the greatest number of T cells in the human body targeting the virus. City of Hope researchers plan to add the Triplex vaccine – developed at City of Hope – to the CAR T cells. Triplex already has been shown to be safe in a City of Hope phase 2 trial in transplant patients. Because Triplex can stimulate T cells to multiply, researchers hope that the vaccine will boost the number and longevity of CMV-CD 19 CAR T cells in these trials in patients’ bodies so they may better fight against their disease.

NHL patients will be able to enroll in three trial designs. In the first trial, patients will receive chemotherapy followed by the Triplex vaccine. For a second trial, patients will receive a hematopoietic stem cell transplant (HSCT) with their own stem cells and the vaccine. In the third trial, patients will undergo HSCT with stem cells from a donor followed by the Triplex vaccine.

The team leading these City of Hope clinical trials includes Forman, who is also scientific director of City of Hope’s T Cell Immunotherapy Laboratory; City of Hope doctors Tanya Siddiqi, M.D., assistant clinical professor of hematology and hematopoietic cell transplantation, and Leslie Popplewell, M.D., associate clinical professor of hematology and hematopoietic cell transplantation, who will lead the autologous transplant trial, and Ryotaro Nakamura, M.D., associate professor of hematology and hematopoietic cell transplantation, who will lead the allogeneic trial. Don J. Diamond, Ph.D., a City of Hope professor in the Department of Hematology & Hematopoietic Cell Transplantation, developed Triplex, and Xiuli Wang, Ph.D., a City of Hope research professor in the same department, developed the CAR T cells for this project.

Two trials for relapsed/treatment-resistant Hodgkin’s lymphoma

Patients with high-risk Hodgkin’s lymphoma who have relapsed or resisted treatment currently only have a 20 to 50 percent chance of achieving a cure. City of Hope is leading two compatible clinical trials for these patients; the therapies are expected to treat the disease that would then enable them to receive a bone marrow transplant. Led by Alex Herrera, M.D., City of Hope assistant professor in hematology and hematopoietic cell transplantation, the first is a phase 2 trial of response-adapted sequential therapy using a combination therapy – nivolumab, an immunotherapy drug known as a PD-1 inhibitor, and ICE (ifosfamide, carboplatin, etoposide phosphate) chemotherapy. Nivolumab works by preventing the PD-1 protein from doing its job, which is to suppress the immune system from fighting cancerous cells. By putting PD-1 in check, a person’s immune system can better fight Hodgkin’s lymphoma. This approach, which utilizes an innovative PET response-adapted treatment sequence, is designed to increase the proportion of patients who have achieved a complete response before they receive a transplant. The trial also gauges the impact of PD-1 inhibitors on a Hodgkin’s lymphoma tumor microenvironment, the area surrounding tumor cells, by using new methods to better evaluate what’s occurring in that area.

A second aim of the project, led by Eileen Smith, City of Hope associate director of the Clinical Research Program, Department of Hematology & Hematopoietic Cell Transplantation, is to start a phase 2 trial that uses an anti-CD25 antibody immunoconjugate, a targeted therapy that uses a CD25 antigen to kill tumorous cells. The antibody will augment high-dose chemotherapy, an autologous stem cell transplant and radiation targeting a tumor’s microenvironment; preliminary data from a similar phase 1 trial showed that the regimen is feasible, remarkably well tolerated and has promising efficacy.

David M. Colcher, Ph.D., City of Hope professor in molecular imaging and therapy, is also a scientific principal investigator.

Fighting STAT3 in non-Hodgkin’s lymphoma

Non-Hodgkin’s lymphoma (NHL) is the sixth most common cancer in the United States, with more than 70,000 estimated new cases each year. Growing evidence links B cell NHLs to persistent activation of STAT3, a gene that drives tumor cell growth and anti-tumor immune suppression. But there is currently no drug approved by the U.S. Food and Drug Administration that stops the activation of STAT3. Hua Yu, Ph.D., City of Hope’s Billy and Audrey L. Wilder Professor in Tumor Immunotherapy and co-leader, of the Cancer Immunotherapeutics Program, and Marcin Kortylewski, Ph.D., associate professor, Department of Immuno-Oncology, and team have already demonstrated that an immunotherapy developed at City of Hope (CpG-STAT3siRNA) turns off STAT3, and stimulates the immune system to attack tumors, in addition to killing B cell lymphoma tumor cells and making radiation therapy more effective in animal models.

The new lymphoma SPORE grant – as well as funding from The Marcus Foundation – makes it possible for City of Hope to start a phase 1 trial for that drug in patients. The trial will test its safety in patients, and whether the therapy can be injected within tumors; patients will also receive low-dose radiation therapy to augment the new drug.

A more effective, second generation therapy will also be tested in animal models – the goal is to also develop that drug for a clinical trial. That drug is also expected to have a two-pronged effect – attack lymphoma cells and spur the immune system to fight cancer, a crucial step because cancerous cells stop the immune system from killing them.

"B cell lymphomas can be very difficult to treat, which is why City of Hope continues to develop and produce innovative targeted therapies for lymphoma that can also restore the immune system to fight cancer," Yu said. "Our new therapies would be the first drugs to both kill tumor cells and activate the immune system."

In addition, STAT3 is common in other cancers – Yu hopes that the new therapy will be effective, so it can also be used against leukemia, pancreatic and other cancers.

Elizabeth Budde, M.D., Ph.D., an assistant professor in the Department of Hematology & Hematopoietic Cell Transplantation at City of Hope, is the project’s clinical principal investigator.

Understanding a serious complication for Hodgkin’s lymphoma patients receiving an autologous stem cell transplant

Hodgkin’s lymphoma (HL) and non-Hodgkin’s lymphoma (NHL) patients who receive stem cell transplantation can develop a deadly complication that may arise after the procedure. Between 6 to 8 percent of HL/NHL transplant patients can develop therapy-related myelodysplasia/acute myeloid leukemia (t-MDS/AML), which is more common in older adults and is the leading cause of nonrelapse mortality in this group. It is generally believed that blood stem cells exposed to high doses of chemotherapy and other cytotoxic therapies suffer genetic damage that leads to t-MDS/AML but some patients could also have a genetic predisposition to the disease.

Researchers at University of Alabama at Birmingham, City of Hope, University of Minnesota, University of Nebraska, St. Jude Children’s Research Hospital and Dana Farber Cancer Institute are developing a prediction model that includes clinical and genetic details to determine the probability of a patient’s risk of developing t-MDS/AML. The research will help identify which patients are at risk for developing t-MDS/AML and what a medical team can do to personalize treatment and help prevent a patient from developing the disease.

Smita Bhatia, M.D., M.P.H., and Ravi Bhatia, M.D., both at University of Alabama at Birmingham, and Stephen J. Forman, M.D., of City of Hope are leading the research. Mukta Arora, M.D., M.S. (at University of Minnesota), Julie Vose, M.D. (at University of Nebraska), Ben Ebert, M.D., Ph.D. (at Dana Farber Cancer Institute), and Yutaka Yasui, Ph.D. (at St. Jude Children’s Research Hospital) are also working on the project.

On February 21, 2019 ArQule, Inc. (Nasdaq: ARQL) reported that Paolo Pucci, Chief Executive Officer, and Brian Schwartz, M.D., Chief Medical Officer and Head of Research and Development, of ArQule will present at the 8th Annual Leerink Partners Global Healthcare Conference on February 28, 2019, at 11:30 a.m. ET at the Lotte New York Palace in New York City (Press release, ArQule, FEB 21, 2019, View Source [SID1234533534]).

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The live webcast of the presentation will be available via the "Investors & Media" section of ArQule’s website, www.arqule.com, under "Events & Presentations." A replay of the webcast will be available shortly after the conclusion of the presentation.

On February 21, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases, reported that it will host a conference call and webcast to report its fourth quarter and full year 2018 financial results and provide a corporate update on Wednesday, February 27, 2019 (Press release, Audentes Therapeutics, FEB 21, 2019, View Source [SID1234533551]).

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The conference call will be held at 4:30 pm EST, after the market closes. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investor + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 1196899 to access the call.

A replay of the live webcast will be available on the Audentes website for approximately 30 days.