On November 15, 2017 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, reported financial results for the third quarter and recent business highlights (Press release, Abeona Therapeutics, NOV 15, 2017, View Source;p=RssLanding&cat=news&id=2317120 [SID1234522085]). The Company will provide investors an update on recent and ongoing business activities and an overview of its 3Q17 financials on Monday, November 20th, at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international).

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"The third quarter was marked with achievements across multiple clinical programs, including initiating enrollments at our global clinical sites for ABO-102 for MPS IIIA and reporting additional data that underscored the durability and clinical benefit of the gene therapy. Our Epidermolysis Bullosa program achieved FDA Breakthrough Therapy designation, completed its Phase 1/2 clinical trial and continues to advance as we finalize the clinical protocol before initiating the pivotal Phase 3 trial next year. We were pleased to have recently initiated screening in our MPS IIIB program and look forward to commencing enrollments shortly," stated Timothy J. Miller, Ph.D., President and CEO. "In addition, work in optimizing our AIM vector platform demonstrated exciting progress, including enhanced tissue tropisms compared to naturally occurring AAV capsids."

3rd Quarter Summary Financial Results:

Cash position: Cash and cash equivalents as of September 30, 2017 were $56.5 million, compared to $58.3 million as of June 30, 2017. Net cash used in operating activities in the nine months ended September 30, 2017 was $17.6 million as compared to $9.6 million in the same period in 2016. Cash and cash equivalents includes approximately $5 million from exercised warrants in the third quarter. Subsequent to the end of the third quarter, the Company closed a public offering of common stock with gross proceeds of $92 million. Total cash as of October 31, 2017 was $142.6 million.
Revenues: Revenues were $219 thousand for the third quarter of 2017, compared to $184 thousand in the third quarter of 2016. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share: Loss per share was $0.13 for the third quarter of 2017, compared to a loss per share of $0.08 in the comparable period in 2016.
Abeona Recent Highlights:

November 9, 2017: Enrolled First Subject at Spain Clinical Site in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
October 19, 2017: Announced Closing of $92 Million Underwritten Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares
October 16, 2017: Announced a Grant of up to $13.85 Million from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies
October 11, 2017: Hosted inaugural R&D day and announced enrollment of First Two Patients in Global Expansion of Phase 1/2 Clinical Trial in MPS IIIA
October 6, 2017: Announced Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa
Gene therapy demonstrated durable and significant reduction of underlying disease pathology across multiple clinical measures in Cohort 1 (n=3) compared to a natural history control group (n=8-12)
Systemic biopotency demonstrated time- and dose-dependent reductions of disease causing Heparan Sulfate in the Cerebrospinal fluid (CSF) and liver volumes
Preservation of deep brain architecture observed after intravenous administration
Stabilization of neurocognitive assessment scores at one year post-injection
October 4, 2017: Announced Dedication of Commercial Gene Therapy Manufacturing Facility in Cleveland, Ohio
September 28, 2017: Announced Collaboration with Brammer Bio for Commercial Translation of ABO-102
August 29, 2017: Received FDA Breakthrough Therapy Designation for EB-101 Autologous Cell Therapy in Epidermolysis Bullosa
July 25, 2017: Announced Appointment of Juan Ruiz, M.D., Ph.D. as Chief Medical Officer
July 18, 2017: Received Guidance from FDA to Commence Pivotal Phase 3 for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa

"We have made great progress in the quarter towards becoming a key player in the development of novel breakthrough gene and cell therapies for rare genetic diseases," stated Steven H. Rouhandeh, Executive Chairman. "The recent investment from high-quality investors and leading foundations is another achievement that demonstrates our internal capabilities and commitment to the advancement of our robust pipeline and next generation vector platform, including MPS III gene therapy products. We look forward to further strengthening our efforts with key hires, advancing clinical capabilities, and commercial expansion in the coming quarters."

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.

On November 15, 2017 EUSA Pharma and AVEO Oncology (NASDAQ:AVEO) reported the first commercial launch of FOTIVDA (tivozanib) with the initiation of product sales in Germany (Press release, AVEO, NOV 15, 2017, View Source;p=RssLanding&cat=news&id=2317048 [SID1234522076]). In the European Union, Norway and Iceland, tivozanib is indicated for the first line treatment of adult patients with advanced renal cell carcinoma (aRCC) and for adult patients who are vascular endothelial growth factor receptor (VEGFR) and mTOR pathway inhibitor-naïve following disease progression after one prior treatment with cytokine therapy for aRCC.i Tivozanib is an oral, once-daily, potent and highly-selective vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI). EUSA Pharma is the licensee for tivozanib in Europe, North and South Africa, Latin America and Australasia.

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Lee Morley, EUSA Pharma’s Chief Executive Officer said, "We are delighted that we have been able to grant access to FOTIVDA for patients in one of Europe’s key markets so soon after our regulatory approval. We look forward to working with physicians in Germany to ensure the profile and benefits of FOTIVDA are known and understood. We will of course continue to work with health authorities across all European markets to ensure early access to FOTIVDA as a therapeutic option in the ongoing fight against aRCC."

"The first-ever commercial launch of FOTIVDA is a tremendous accomplishment for AVEO, our partner EUSA Pharma, and, most importantly, patients with aRCC who now have access to a new and differentiated therapeutic option," said Michael Bailey, president and chief executive officer of AVEO. "Over the course of its development, FOTIVDA’s efficacy and tolerability profile among VEGF TKIs has been recognized by investigators as an important potential option for their patients, making this long-anticipated milestone a gratifying achievement. We continue to leverage this profile as we work towards exploring the full extent of FOTIVDA’s use in the emerging aRCC market. We also look forward to continuing to expand its availability in Europe, through our partner, EUSA, and potentially in North America, where we plan to file for FDA approval pending the results of our pivotal study, TIVO-3."

FOTIVDA was approved by the European Commission in August 2017. Approval was primarily based on data from a global, open-label, randomized, multi-center Phase 3 trial (TIVO-1)i,ii which evaluated the efficacy and tolerability of tivozanib compared to a currently available comparator VEGFR-TKI treatment (sorafenib) in 517 patients with advanced RCC. Patients treated with tivozanib experienced superior PFS (11.9 vs. 9.1 months in the overall population [HR, 0.797; 95% CI, 0.639 to 0.993; P =.042] and 12.7 vs. 9.1 months in treatment naïve patients [HR, 0.756; 95% CI, 0.580 to 0.985; P =.037]) versus sorafenib.ii There was also an improved side effect profile with tivozanib, with only 14% (versus 43% with sorafenib) requiring a dose reduction due to adverse events (AEs). In addition, fewer people on tivozanib experienced burdensome side effects, such as diarrhea (23% vs 33%) and hand-foot syndrome (14% vs 54%).ii

About RCC in Europe

RCC is the most common form of kidney cancer,iii which accounts for an estimated 49,000 deaths in Europe each year.iv It is expected to be one of the fastest increasing cancers over the next ten years.v Tyrosine Kinase Inhibitor (TKI) vascular endothelial growth factor (VEGF) inhibitors are the standard of care treatment for advanced RCC in Europe, however, patients on current treatments can often experience significant side effects.ii,vi

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.i,ii Tivozanib has been investigated in several tumors types, including renal cell, colorectal and breast cancers.