On November 17, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the US Food and Drug Administration (FDA) approved Gazyva (obinutuzumab) in combination with chemotherapy, followed by Gazyva alone in those who responded, for people with previously untreated advanced follicular lymphoma (stage II bulky, III or IV) (Press release, Hoffmann-La Roche, NOV 16, 2017, View Source [SID1234522126]). The approval is based on results from the phase III GALLIUM study, which showed superior progression-free survival (PFS) for patients who received this Gazyva-based regimen compared with those who received a Rituxan (rituximab)-based regimen as an initial (first-line) therapy. Follicular lymphoma, the most common slow-growing (indolent) form of non-Hodgkin lymphoma (NHL), is incurable and becomes harder to treat each time it returns.

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"Today’s Gazyva approval is an important advance for the thousands of people diagnosed each year with follicular lymphoma who hope to delay disease progression for as long as possible," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "We’re pleased we can now offer patients with this incurable blood cancer an initial treatment option shown to improve upon Rituxan, the standard of care in this setting for more than 10 years."

The GALLIUM study showed the Gazyva-based regimen significantly reduced the risk of disease worsening or death compared to a Rituxan-based regimen by 28% (PFS as assessed by independent review committee [IRC]; HR=0.72; 95% CI 0.56-0.93; p=0.0118). The most common Grade 3-5 side effects (occurring in at least 5% of patients) observed more frequently in the Gazyva arm were low white blood cell count, infusion reactions, low white blood cell count with fever and low platelet count. The most common side effects (occurring in at least 20% of patients) observed at least 2% more frequently in the Gazyva arm included infusion reactions, low white blood cell count, upper respiratory tract infection, cough, constipation and diarrhoea.

Gazyva’s supplemental Biologics License Application based on the GALLIUM data was granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. With this approval, Gazyva is available in the US for three different indications across two common types of blood cancer.

About the GALLIUM study
GALLIUM (NCT01332968) is a global phase III open-label, multicentre, randomised two-arm study examining the efficacy and safety of Gazyva plus chemotherapy followed by Gazyva alone for up to two years, as compared head-to-head against Rituxan plus chemotherapy followed by Rituxan alone for up to two years. Chemotherapies used (CHOP, CVP or bendamustine) were selected by each participating study site prior to beginning enrolment. GALLIUM included 1,385 patients with previously untreated non-Hodgkin lymphoma (NHL), of whom 1,202 patients had advanced follicular lymphoma (stage II bulky, III or IV). Efficacy results in follicular lymphoma with a median observation time of 38 months were the following:

Safety was evaluated based on 1,385 patients with previously untreated follicular lymphoma (86%) or marginal zone lymphoma (14%). The most common Grade 3-5 side effects that occurred more often with Gazyva plus chemotherapy followed by Gazyva alone compared to Rituxan plus chemotherapy followed by Rituxan alone were low white blood cell count, infusion reactions, low white blood cell count with fever and low platelet count.

On November 16, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that Dr. David C. Dale, MD will present the first clinical data from an on-going study of X4P-001-RD in patients with WHIM Syndrome, a sub-type of a primary immunodeficiency disease, at the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, X4 Pharmaceuticals, NOV 16, 2017, View Source [SID1234522146]). Preliminary data from these abstracts are available on the ASH (Free ASH Whitepaper) conference website. The details of the poster presentation is as follows:

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X4P-001: A Novel Molecularly-Targeted Oral Therapy for WHIM Syndrome

Saturday, December 9, 2017, Abstract # 995, Presenter: David C. Dale, MD

"We are very pleased to share preliminary data showing that X4P-001-RD has a meaningful impact on the levels of circulating white blood cells in this severely immunodeficient patient population," said Paula Ragan, PhD, President and CEO of X4. "These data demonstrate the potential of X4P-001-RD to benefit patients with WHIM who otherwise have no approved treatment options and support our goal of advancing toward a pivotal study."

About WHIM Syndrome

WHIM syndrome is a primary immunodeficiency disease ("PID") caused by genetic mutations in the CXCR4 receptor gene resulting in susceptibility to certain types of infections. WHIM is an abbreviation for the characteristic clinical symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis. Within the overall category of primary immunodeficiencies, there are between 15,000 and 100,000 patients in the US that are classified with PID of unknown origin — of which WHIM is one.1,2,3 WHIM syndrome is a rare disorder and the precise prevalence or incidence of patients that have the genetic mutation responsible for WHIM syndrome is unknown. Because patients are highly susceptible to infections, WHIM syndrome is associated with significant morbidity beginning in early childhood and continuing throughout life. Current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immunoglobulin or G-CSF. There is no approved therapy for the treatment of WHIM syndrome.

About X4P-001-RD for Primary Genetic Immunodeficiency Disease

X4P-001-RD, an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, is being developed for use as a life-long treatment for patients with WHIM syndrome and other primary genetic immunodeficiencies. X4P-001-RD is currently being studied in a Phase 2/3 trial in patients with WHIM syndrome. Within the bone marrow, a normally functioning CXCR4 receptor controls the release of neutrophils and leukocytes into the blood stream, thereby ensuring normal immune surveillance functions throughout the body. In patients with WHIM syndrome, mutations to the CXCR4 receptor cause aberrant signaling leading to retention of neutrophils and leukocytes in the bone marrow and inadequate immune surveillance and function.4,5 X4P-001-RD is designed to normalize the signaling for the mutant CXCR4 receptor to promote the release of neutrophils and leukocytes, thereby restoring healthy immunity.

On November 15, 2017 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies reported that it will participate in two upcoming investor conferences (Press release, Halozyme, NOV 15, 2017, View Source [SID1234522087]).

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Dr. Helen Torley, president and chief executive officer will represent Halozyme in a question and answer session at the 29th Annual Piper Jaffray Healthcare Conference in New York on Wednesday, November 29 at 8:00 a.m. ET / 5:00 a.m. PT.

On Thursday, December 14, Jim Mazzola, vice president corporate communication and investor relations will present a company overview at the BMO Capital Markets 2017 Prescriptions for Success Healthcare Conference in New York at 3:30 p.m. ET / 12:30 p.m. PT.

Webcasts of both sessions can be accessed through the "Investors" section of www.halozyme.com, and a recording will be made available for 90 days following each event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.

On November 15, 2017 AstraZeneca reported that the US Food and Drug Administration (FDA) has approved a new indication for Faslodex (fulvestrant), expanding the indication to include use with abemaciclib, a CDK4/6 inhibitor, for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced or metastatic breast cancer (MBC) in women with disease progression after endocrine therapy (Press release, AstraZeneca, NOV 15, 2017, View Source [SID1234522075]).

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Dave Fredrickson, Executive Vice President, Head of the Oncology Business Unit at AstraZeneca, said: "Faslodex has long been an effective monotherapy option for women with hormone receptor positive breast cancer, which is the most common type of advanced breast cancer. Today’s decision builds upon the recent approval for Faslodex in the first-line advanced setting and is supported by strong evidence to use this medicine within a combination therapy for advanced breast cancer. Combining Faslodex with abemaciclib provides patients with another effective, non-chemotherapy option to combat this disease."

Peter A. Kaufman, MD of the Norris Cotton Cancer Center at Dartmouth-Hitchcock Medical Center, said: "This new indication for Faslodex offers another treatment option for women living with HR+, HER2- advanced or metastatic breast cancer with disease progression after endocrine therapy. The study supporting this indication demonstrated that Faslodex used in combination with abemaciclib significantly improves progression-free survival compared to Faslodex and placebo."

The FDA approval is based on data from the Phase III MONARCH 2 trial, which met the study’s primary endpoint of PFS.

The trial included 669 women with HR+, HER2- advanced breast cancer. The results showed a statistically significant increase in investigator-assessed median PFS of 7.1 months (16.4 months vs 9.3 months) in patients who received Faslodex 500 mg and abemaciclib 150 mg over Faslodex and placebo (HR: 0.553; 95% CI: 0.449-0.681; p<0.0001).

This expanded indication for Faslodex is the second FDA approval for Faslodex in combination with a CDK4/6 inhibitor. Faslodex has been licensed in the US since 2016 for use with the CDK4/6 inhibitor, palbociclib, for the treatment of women with HR+, HER2-negative MBC, whose cancer has progressed after endocrine therapy.

NOTES TO EDITORS
About MONARCH 2

MONARCH 2 is a Phase III, international, randomised, double-blind, placebo-controlled, multicenter study, sponsored by Eli Lilly and Company, of Faslodex with abemaciclib vs Faslodex with placebo conducted in women with HR+, HER2- advanced or metastatic breast cancer, whose disease progressed on or after neoadjuvant or adjuvant endocrine therapy, ≤12 months from the end of adjuvant endocrine therapy, or while receiving first-line endocrine therapy for metastatic disease. The study included 669 women randomly assigned to receive intramuscular injection of 500 mg Faslodex with abemaciclib or placebo orally twice daily in a 2:1 ratio. Pre/perimenopausal women were enrolled in the study and received the gonadotropin-releasing hormone agonist goserelin acetate for at least four weeks prior to and for the duration of the study. Patients remained on treatment until development of progressive disease or unmanageable toxicity.

Patients enrolled in this study had a median age of 60 years (range, 32 to 91). The majority of patients in the study were white (56%). All patients had an ECOG (Eastern Cooperative Oncology Group) performance status of 0 or 1.

Approximately 59% of patients in each of the treatment arms, Faslodex in combination with abemaciclib and Faslodex with placebo, received endocrine therapy as their first therapy for advanced breast cancer; the remaining 38% of patients in the experimental and in the control treatment arms received this regimen as their second endocrine therapy for advanced breast cancer. 55.8% had visceral disease and 26.9% had bone-only disease. Twenty-five percent of patients had primary endocrine resistance, and 2.7% had locally advanced disease.

Detailed results of the MONARCH 2 trial are published online in the Journal of Clinical Oncology.

About Advanced Breast Cancer or Metastatic Breast Cancer (MBC)

Advanced/metastatic breast cancer refers to Stages III and IV breast cancer. Stage III disease may be referred to as locally-advanced breast cancer. MBC is the most advanced stage of breast cancer (Stage IV), and occurs when cancer cells have spread beyond the initial tumor site to other parts of the body outside of the breast.

Despite treatment options increasing during the past three decades, there is currently no cure for patients diagnosed with MBC and the 5-year relative survival rate for this patient population is currently 26.9%. Thus, the primary aim of treatment is to slow progression of the disease for as long as possible, improving, or at least maintaining, a patient’s quality of life.

It is estimated that in 2017, there will be approximately 153,000 women in the US living with MBC, and this number is projected to increase to approximately 160,000 by the year 2020.

About Faslodex (fulvestrant)

Faslodex is indicated for the treatment of oestrogen receptor positive, locally advanced or metastatic breast cancer in postmenopausal women not previously treated with endocrine therapy, or with disease relapse on or after adjuvant anti-oestrogen therapy, or disease progression on anti-oestrogen therapy.

In the US, EU and Japan, Faslodex is also approved in combination with palbociclib for the treatment of women with HR+, HER2-negative advanced or metastatic breast cancer, whose cancer has progressed after endocrine medicine. Faslodex represents a hormonal treatment approach that helps to slow tumour growth by blocking and degrading the oestrogen receptor – a key driver of disease progression.

Faslodex is approved in over 80 countries as a monotherapy to treat ER+ advanced breast cancer patients. It is currently being evaluated in combination with medicines from various drug classes for the treatment of women with HR+ advanced breast cancer.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody-Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On November 15, 2017 Xenetic Biosciences, Inc. (NASDAQ: XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported its unaudited financial results for the quarter ended September 30, 2017 (Press release, Xenetic Biosciences, NOV 15, 2017, View Source [SID1234522089]).

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Xenetic also provided a corporate update and anticipated milestones for the Company’s lead product candidate, XBIO-101 (sodium cridanimod), a small-molecule immunomodulator and interferon inducer which, in preliminary studies, has been shown to increase progesterone receptor ("PrR") expression in endometrial tumor tissue, and an update on its proprietary PolyXen platform technology.

Recent Corporate Highlights:

Appointed Jeffrey F. Eisenberg as Chief Executive Officer;
Entered into Right to Sublicense Agreement related to the Company’s PolyXen Technology with Baxalta Inc., a wholly-owned subsidiary of Shire plc; and
Commenced patient dosing in the Phase 2 clinical study of XBIO-101 in conjunction with progestin therapy for the treatment of endometrial cancer.
"I believe we are well positioned to build on the momentum of our recent corporate and clinical achievements. We remain focused on the solid execution of our Phase 2 study of XBIO-101 for the treatment of endometrial cancer, with the goal of announcing interim data before the end of next year. Further, our recent right to sublicense agreement with Baxalta not only leverages our PolyXen platform technology and provides a source of non-dilutive capital, but also positions Xenetic for value driving opportunities in the near and long-term," stated Jeffrey Eisenberg, Chief Executive Officer of Xenetic Biosciences.

XBIO-101 Program Update

Patient dosing recently commenced for the Company’s Phase 2 clinical study of XBIO-101 in conjunction with progestin therapy for the treatment of endometrial cancer. The study targets a population of patients who have either failed progestin monotherapy or who have been identified as having progesterone receptor negative ("PrR-") tumors.

The primary objective of this open-label, multi-center, single-arm, two-period Phase 2 study is to assess the anti-tumor activity of XBIO-101 in conjunction with progestin therapy as measured by Overall Disease Control Rate in women with recurrent or persistent endometrial carcinoma not amenable to surgical treatment or radiotherapy who have either failed progestin monotherapy or who have been identified as PrR-. Secondary objectives include assessments of efficacy and safety/tolerability parameters.

The study is expected to enroll up to 72 women with recurrent or persistent endometrial cancer not amenable to surgical treatment or radiotherapy but suitable to be treated with progestins. All subjects determined to be PrR- at screening, as well as those subjects who experience disease progression after at least 4 weeks of progestin monotherapy, will receive XBIO-101 in combination with continued progestin treatment. Subjects will receive treatment until disease progression as defined according to RECIST 1.1 criteria.

The Company expects to announce interim data from the Phase 2 study before the end of 2018.

PolyXen Platform Technology Update

The Company recently announced that it has entered into a Right to Sublicense Agreement (the "Right to Sublicense Agreement") with Baxalta Incorporated, Baxalta US Inc., and Baxalta GmbH (collectively, with their affiliates, "Baxalta"), wholly-owned subsidiaries of Shire plc (LSE: SHP, NASDAQ: SHPG). Pursuant to the Right to Sublicense Agreement, Xenetic granted to Baxalta the right to grant a nonexclusive sublicense to certain patents related to the Company’s PolyXen technology that were previously exclusively licensed to Baxalta pursuant to an agreement between the Company and Baxalta in connection with products relating to the treatment of blood and bleeding disorders.

As part of the Right to Sublicense Agreement, Baxalta paid Xenetic a one-time payment of $7.5 million and is expected to make single digit royalty payments based upon net sales of the products covered under the related sublicense throughout the term of the agreement.

Additionally, Xenetic expects to continue to pursue business development activities to explore partnerships utilizing its PolyXen delivery platform.

Summary of Financial Results for Third Quarter 2017

Net loss for the nine months ended September 30, 2017, was $8.0 million compared to a net loss of approximately $53.8 million for the same period in 2016. The decrease in net loss was primarily due to a decrease of in-process research and development expense, as well as a decrease in share-based compensation expense related to warrants previously issued in 2016. These decreases were offset by an increase in general operating costs and costs related to the initiation of our XBIO-101 Phase 2 clinical study.

The Company ended the quarter with approximately $0.7 million of cash. With the addition of the $7.5 million milestone payment under the Right to Sublicense Agreement, as of the date of this release, the Company’s current cash position is approximately $8.0 million. Based on management’s current projections, the Company has sufficient cash to fund its operations through the second quarter of 2018.