On August 9, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported second quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled for Monday, August 12 at 10:00 a.m. ET (Press release, Abeona Therapeutics, AUG 9, 2019, View Source [SID1234538540]). Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (international) or via webcast at View Source

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"The second quarter was highlighted by progress made in both of our MPS III programs," said João Siffert, M.D., Chief Executive Officer. "Data from our Transpher A study showed that children with MPS IIIA who were treated early with ABO-102 preserved neurocognitive development within the normative range 12-18 months post treatment. Our MPS IIIB program has also progressed, with enrollment of additional patients in cohort 2 of the Transpher B study. Our team remains highly focused on our lead programs, including the start of our VIITAL Phase 3 clinical trial in recessive dystrophic epidermolysis bullosa, continued enrollment in the MPS III programs, and preparations to start the clinical trial in CLN1 disease."

Second Quarter Financial Results:

Cash, cash equivalents and marketable securities as of June 30, 2019, were $62.5 million compared to $68.3 million as of March 31, 2019. The decrease in cash was driven primarily by the net cash used in operating activities of $15.2 million.

Research and development expenses for the second quarter ended June 30, 2019 were $16.3 million compared to $7.9 million in the same period of 2018. The increase in R&D expense was primarily attributable to increased in-house manufacturing activities and related headcount costs.

General and administrative expenses for the second quarter ended June 30, 2019 were $5.6 million compared to $4.6 million in the same period of 2018. The increase in G&A expenses was primarily due to increased headcount and related facility costs.

Net loss was $0.49 per share for the second quarter of 2019 compared to $0.26 per share in the same period of 2018.

Second Quarter and Recent Highlights:

-July 25, 2019: Announced positive interim data from the Phase 1/2 AAV9 gene therapy clinical trial in MPS IIIA showing preservation of neurocognitive function for the three youngest patients treated with ABO-102, as well as robust and sustained improvements in biomarkers of the disease. No product-related serious adverse events were reported to date.
-June 26, 2019: Appointed Dr. Victor Paulus as Senior Vice President of Regulatory Affairs and Jodie Gillon as Vice President of Patient Advocacy and Clinical Affairs
-June 18, 2019: Received FDA Fast Track Designation for ABO-202 AAV9 gene therapy in CLN1 disease
-May 21, 2019: Announced FDA clearance of Investigational New Drug application for ABO-202 AAV9 gene therapy in CLN1 disease
-May 14, 2019: Announced treatment of first patient in second cohort of Phase 1/2 clinical trial for ABO-101 AAV9 gene therapy in MPS IIIB
-May 1, 2019: Reported preclinical data demonstrating broad therapeutic potential of AIM gene therapy in retinal diseases at Association for Research in Vision and Ophthalmology Annual Meeting
-April 30, 2019: Reported preclinical data demonstrating therapeutic potential of ABO-401 for treatment of cystic fibrosis at American Society of Gene and Cell Therapy annual meeting
-April 4, 2019: Received FDA Fast Track Designation for ABO-101 AAV9 gene therapy for MPS IIIB

Steven H. Rouhandeh, Abeona’s Executive Chairman, said, "Abeona has continued the development of its breakthrough gene and cell therapies for rare genetic diseases through 2019 with important regulatory and clinical achievements secured. We look forward to progressing our MPS programs, and to starting of our Phase 3 VIITAL trial in EB before year end."

On August 9, 2019 GeneCentric Therapeutics, Inc. reported that it has entered the second phase of an academic-industry collaboration with Jose Zevallos MD, MPH, Chief of the Division of Head and Neck Oncologic Surgery in the Department of Otolaryngology at Washington University School of Medicine in St. Louis (Press release, GeneCentric Therapeutics, AUG 9, 2019, View Source [SID1234538556]).

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This collaboration is funded by a National Cancer Institute (NCI) R01 grant, awarded in August 2017 to Dr. Zevallos. The grant supports the investigation of novel approaches to improve outcomes for patients with head and neck squamous cell carcinoma (HNSCC). Titled "Development of a Four-Class, Molecular Subtyping Diagnostic for HPV-Negative Head and Neck Cancer," the grant is focused on the development of a clinic-ready tumor subtyping diagnostic test, in collaboration with GeneCentric Therapeutics, that will guide treatment decisions for patients with HPV-negative HNSCC.

The HPV-negative subtyper being applied under this NCI grant is a modified version of GeneCentric’s previously-developed Head and Neck Cancer Subtype Profiler (HNSP). Following successful completion of phase 1 development of a reduced gene-set signature, GeneCentric is poised to commence phase 2 validation studies with Dr. Zevallos and his team. The last stage of the NCI grant, phase 3, entails final confirmation of the HNSCC diagnostic test and commercialization.

"This research has the potential to impact treatment for a broad range of patients with head and neck cancer. Our aim is to better identify cancers that are resistant to radiation therapy. We also hope the test will more accurately identify patients with occult lymph node metastasis and help surgeons make more informed decisions on when surgery to remove neck tumors should be offered to patients," said Dr. Zevallos of the NCI sponsored partnership with GeneCentric.

About GeneCentric Therapeutics Head and Neck Cancer Subtyping Platform (HNSP)

Head and neck cancer, a group of cancers that starts within the mouth, nose, throat, larynx, sinuses, or salivary glands, is the seventh most common of all cancers. Because the majority begin in squamous cells which line the moist surfaces inside the head and neck areas, such cancers are also referred to as Head and Neck Squamous Cell Carcinomas (HNSCC).

These cancers comprise a heterogeneous disease with multiple tumor types, and correspondingly varying prognoses and treatment responses. Advanced HNSCC is associated with a 40-50 percent recurrence rate following primary treatment. Human papilloma virus (HPV) infection is an important risk factor, but infection is present in only a subset of cases and knowledge of HPV status alone is not sufficient to guide treatment. Better classification tools are needed to inform therapeutic choices and improve survival. To address this critical need, GeneCentric has applied its Cancer Subtyping Platform (CSP) to develop a comprehensive profile of head and neck tumor subtypes that can guide drug development, clinical trials and patient treatment. The GeneCentric Head and Neck cancer Subtype Profiler (HNSP) consists of five distinct subtypes, including one specific to HPV-related HNSCC, as determined through our research.

On August 9, 2019 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on identifying, in-licensing and developing high-potential marketed and development-stage drugs and drug candidates, reported financial results and recent corporate highlights for the second quarter ended June 30, 2019 (Press release, Fortress Biotech, AUG 9, 2019, View Source [SID1234538541]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "We are pleased to have achieved several important milestones in the second quarter, including positive data for three of our late-stage product candidates in development across our partner companies: IV tramadol for post-surgical pain management; MB-107 gene therapy for the treatment of X-linked severe combined immunodeficiency (XSCID); and cosibelimab, an anti-programmed death ligand-1 (PD-L1) antibody for the treatment of multiple advanced cancers. Looking ahead to the remainder of 2019, we anticipate multiple potentially value-creating catalysts, including a New Drug Application filing for IV tramadol and additional important clinical data readouts for many of our product candidates. Our world-class business development team continues to focus on expanding our diverse pipeline with additional high-quality biotech and specialty pharmaceutical assets, further de-risking our product portfolio."

Financial Results:

·As of June 30, 2019, Fortress’ consolidated cash, cash equivalents, short-term investments (certificates of deposit), and restricted cash totaled $170.5 million, compared to $137.5 million as of March 31, 2019, and $99.2 million as of December 31, 2018, an increase of $33.0 million for the quarter and an increase of $71.3 million year-to-date.
·Fortress’ net revenue totaled $9.3 million for the second quarter of 2019, compared to $6.8 million for the second quarter of 2018.
· Research and development expenses were $18.5 million for the second quarter of 2019, of which $18.0 million was related to Fortress partner companies. This compares to $17.5 million for the second quarter of 2018, of which $15.1 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in research and development were $0.8 million for both the second quarter of 2019 and 2018.
·Research and development expenses from license acquisitions totaled $0.2 million for the second quarter of 2019, compared to a nominal amount for the second quarter of 2018.
·General and administrative expenses were $13.4 million for the second quarter of 2019, of which $9.3 million was related to Fortress partner companies. This compares to $13.1 million for the second quarter of 2018, of which $7.7 million was related to Fortress partner companies. Non-cash, stock-based compensation expenses included in general and administrative expenses were $2.6 million for the second quarter of 2019, compared to $2.4 million for the second quarter of 2018.
·Net loss attributable to common stockholders was $13.1 million, or $0.24 per share, for the second quarter of 2019, compared to a net loss attributable to common stockholders of $21.6 million, or $0.50 per share, for the second quarter of 2018. For the first six months of 2019, net loss attributable to common stockholders was $11.7 million or $0.23 per share, compared to $42.6 million or $0.99 per share for the first six months of 2018.

Recent Corporate Highlights1:

Marketed Dermatology Products

·In the second quarter of 2019, our marketed products generated net revenue of $8.2 million, compared to $6.7 million in the second quarter of 2018.
· We are anticipating the launch of a second prescription oral antibiotic drug for acne during the current quarter, Q3 2019.
·This new asset, coupled with our salesforce expansion to 34 territory managers, will allow us to reach over 5,000 dermatologists across the country. This combination is expected to fuel the growth of our dermatology portfolio in 2019 and beyond.
·Our dermatology products are marketed by our partner company, Journey Medical Corporation.

IV Tramadol

·In June 2019, we announced that our second pivotal Phase 3 trial of IV tramadol achieved the primary endpoint of a statistically significant improvement in Sum of Pain Intensity Difference over 24 hours (SPID24) compared to placebo in patients with postoperative pain following abdominoplasty surgery. In addition, the trial met all of its key secondary endpoints. The study also included a standard-of-care IV opioid as an active comparator, IV morphine 4 mg. In this study, IV tramadol also demonstrated similar efficacy and safety to that of IV morphine.
·IV Tramadol is currently in development at our partner company, Avenue Therapeutics, Inc.

MB-102 (CD123 CAR T)

·In July 2019, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MB-102 (CD123 CAR T) for the treatment of acute myeloid leukemia (AML).
·In August 2019, we announced that the FDA has approved the Investigational New Drug (IND) application to initiate a multicenter Phase 1/2 clinical trial of MB-102 (CD123 CAR T) in AML, blastic plasmacytoid dendritic cell neoplasm (BPDCN) and high-risk myelodysplastic syndrome (MDS).
·MB-102 is currently in development at our partner company, Mustang Bio, Inc.

MB-108 (Oncolytic Virus C134)

·In May 2019, the FDA granted Orphan Drug Designation to MB-108 (oncolytic virus C134) for the treatment of malignant glioma, a type of brain cancer with a median survival of less than 18 months.
·MB-108 is currently in development at our partner company, Mustang Bio, Inc.

On August 8, 2019, Stemline Therapeutics, Inc. ("Stemline" or the "Company") reported that it has entered into an underwriting agreement (the "Underwriting Agreement") with J.P. Morgan Securities LLC, as representative of the several underwriters named therein (the "Underwriters") (Filing, 8-K, Stemline Therapeutics, AUG 9, 2019, View Source [SID1234538565]). Pursuant to the Underwriting Agreement, the Company agreed to sell to the Underwriters, in a firm commitment underwritten public offering, 5,000,000 shares (the "Firm Shares") of the Company’s common stock, $.0001 par value per share ("Common Stock"), at a price to the public of $15.25 per share, less underwriting discounts and commissions. In addition, pursuant to the Underwriting Agreement, the Company has granted the Underwriters an option, exercisable for 30 days, to purchase up to an additional 750,000 shares of Common Stock (the "Additional Shares," together with the Firm Shares, the "Shares"). The transactions contemplated by the Underwriting Agreement are expected to close on August 13, 2019, subject to the satisfaction of customary closing conditions. A copy of the Underwriting Agreement is attached hereto as Exhibit 1.1 and is incorporated by reference herein.

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J.P. Morgan Securities LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering.

The gross proceeds to the Company are expected to be approximately $76,250,000, assuming no exercise of the option to purchase Additional Shares and before deducting underwriting discounts and commissions and estimated expenses payable by the Company associated with the offering.

The Underwriting Agreement contains customary representations, warranties and agreements by the Company, customary conditions to closing, indemnification obligations of the Company and the Underwriters, including for liabilities under the Securities Act of 1933, as amended, other obligations of the parties and termination provisions.

Alston & Bird LLP, counsel to the Company, delivered an opinion as to the validity of the Shares, a copy of which is attached hereto as Exhibit 5.1 and is incorporated by reference herein.

On August 9, 2019 Lion TCR Pte. Ltd., a clinical-stage Biotech company focused on development of innovative engineered T cell immunotherapy against viral-related cancer and chronic Hepatitis B infection, reported on 29 July 2019 that it has signed an agreement with Dalian Lvshun District Government at the 10th Singapore-Liaoning Economic and Trade Council Meeting (SLETC) to develop cell manufacturing and research facilities for cancer treatment (Press release, Lion TCR, AUG 9, 2019, View Source [SID1234538769]). The 5,000-sqm facility, built by Dalian Municipal Government, will support Lion TCR’s development and commercialisation of cell therapy products in China in addition to Lion TCR’s facility at the China-Singapore Guangzhou Knowledge City. Lion TCR shall utilize GMP (Good Manufacturing Practice) certified entities for the production of advanced cellular immunotherapy products for clinical trials and for future commercial products. The facility design and process will comply with both China CDE and internationally recognized GMP guidelines.

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The 10th SLETC was chaired by Mr. Masagos Zulkifli, Singapore’s Minister for the Environment and Water Resources, and Mr. Tang Yijun, Liaoning Governor, the Co-Chairmen of SLETC. At the council meeting, 11 Singapore companies including Lion TCR signed project agreements to provide greater connectivity, sustainability, technology and healthcare in Liaoning. Singapore is Liaoning’s third largest foreign investor with a total investment of about S$19 billion (95 billion yuan). Minister Masagos told the Party Secretary and Mayor of Dalian City at their meeting: "Singapore looks forward to deepening cooperation with Dalian in environmental protection, new energy, interconnection, biomedical, petrochemical, marine fishery, food processing, sustainable development and other fields."

Mr. Stephen Lim, CEO of Lion TCR commented that "Lion TCR looks forward to using this Dalian GMP cell therapy facilities as a manufacturing base for our China commercialisation needs. As a stand-alone building is required for cell therapy GMP application in China, this offer to build with option for Lion to purchase by the Dalian Government is a great help to Lion as it frees our immediate cashflow for continual research and on-going clinical trials."

Mr. Masagos Zulkifli, Singapore’s Minister for the Environment and Water Resources, Singapore Co-Chair of Singapore-Liaoning Economic and Trade Council (fourth from left); Mr. Stephen Lim, CEO of Lion TCR (third from left); Dr. Victor Li, Chairman of Lion TCR (third from right).