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.

Delcath Systems has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Delcath Systems, 2017, NOV 14, 2017, View Source [SID1234522073]).

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On November 14, 2017 Cleveland BioLabs, Inc. (NASDAQ:CBLI) reported financial results and development progress for the third quarter ended September 30, 2017 (Press release, Cleveland BioLabs, NOV 14, 2017, View Source [SID1234522045]).

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Cleveland BioLabs reported a net loss of $(1.3) million, excluding minority interests, for the third quarter of 2017, or $(0.11) per share, compared to net income, excluding minority interests, of $1.1 million, or $0.10 per share, for the same period in 2016. The increase in net loss was primarily due to an increase in the downward non-cash adjustment to our warrant liabilities and decreased revenues and expenses due to the completion of our development contracts with the Russian Federation Ministry of Industry and Trade ("MPT"), which was partially offset by reduced operating costs aligned with our streamlined focus primarily on pursuing a pre- Emergency Use Authorization ("pre-EUA") with the U.S. Food and Drug Administration ("FDA") and a Marketing Authorization Application ("MAA") with the European Medicines Agency ("EMA") for entolimod as a medical radiation countermeasure ("MRC").

As of September 30, 2017, the Company had $10.1 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is expected to fund operations for at least one year beyond the filing date of our Form 10-Q.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The pursuit of approval by the FDA and EMA and commercialization for entolimod as a medical radiation countermeasure are continuing to be the company’s most important priorities and goals. Per FDA’s request during the past quarter, we collated and submitted manufacturing information (Module 3) to the agency. We also initiated the in vivo biocomparability study in non-human primates that had been previously requested by the agency as part of its review of our pre-EUA application; this study is ongoing. Following completion of this study and discussion of the study results with the FDA, we expect the agency to resume review of our pre-EUA dossier."

"We are also pleased to announce submission in the European Union of a MAA for use of entolimod as a MRC. Our application was recently validated by the EMA and will now undergo agency review. Filing of the MAA represents a significant milestone for the company and another major step toward making entolimod available worldwide as a life-saving treatment of acute radiation syndrome ("ARS")," continued Dr. Kogan. "I am proud of the dedicated team at CBLI that prepared the MAA and shares the company’s commitment to developing an effective and practical ARS therapy for mass-casualty radiation and nuclear disaster scenarios."

Further Financial Results

Revenue for the third quarter of 2017 decreased to $0.3 million compared to $1.1 million for the third quarter of 2016. The net decrease was primarily attributable to reduced revenue from our development contracts with MPT which completed in 2016 and reduced revenue due to completion of manufacturing activities from our Joint Warfighter Medical Research Program ("JWMRP") contract from the Department of Defense ("DoD") for the continued development of the entolimod as a medical radiation countermeasure.

Research and development costs for the third quarter of 2017 decreased to $0.9 million compared to $1.1 million for the third quarter of 2016. The reduction in research and development costs is due to completion

of a clinical study of the safety and tolerability of entolimod as a neo-adjuvant therapy in treatment-naïve patients with primary colorectal cancer and completion of associated preparatory research studies, offset by an increase in entolimod for biodefense applications for continued preclinical development along with drug manufacturing activities associated with our JWMRP contract and expenses associated with our regulatory activities in support of filing a MAA with EMA.

General and administrative costs for the third quarter of 2017 decreased to $0.6 million compared to $0.8 million for the third quarter of 2016. This decrease was primarily attributable to reductions in personnel and other operating costs in connection with cost savings efforts to streamline operations.