On May 16, 2016 Abeona Therapeutics Inc. (NASDAQ: ABEO), a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, reported summary financial results for the first quarter (Press release, Abeona Therapeutics, MAY 16, 2016, View Source;p=RssLanding&cat=news&id=2168743 [SID:1234512440]). The Company will provide a business update for investors and other stakeholders on a conference call, Tuesday, May 17th, at 10 am (Eastern). Tim Miller, Ph.D., President and Chief Executive Officer and Jeffrey Davis, Chief Operating Officer, together with other executives, will conduct the call. Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international). The call will consist of an overview of the Company’s 1Q16 financials, and a discussion of business highlights.

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"The first quarter of this year has led to significant advancements in our goal of building a leadership position in the field of gene therapy and plasma protein therapies for rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "We thank our collaborators, shareholders and staff as we prepare to launch human clinical trials in up to four different rare diseases over the next 12 months."

Tim Miller, PhD, stated, "In the first quarter, Abeona hit important regulatory milestones with the FDA allowance of our Phase 1/2 clinical study in Sanfilippo syndrome type A (MPS IIIA) and the European approvals of the Genetically Modified Organisms (GMO) and Ethical Committee (CEIC) regulatory filings. Additionally, Abeona and its academic collaborators presented meaningful pre-clinical data at the World Symposium of Lysosomal Storage Diseases in San Diego, and on its proprietary CRISPR/Cas9 platform at the 2nd Annual CRISPR Precision Gene Editing Congress in Boston, MA."

Recent Abeona Operating Highlights

The Interministerial Council of Genetically Modified Organisms has approved the Genetically Modified Organism (GMO) Voluntary Release and Ethical Committee (CEIC) regulatory filings for both Phase 1/2 Gene Therapy Clinical Studies to treat patients with AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH) for patients with Sanfilippo syndrome type A (MPS IIIA) or type B (MPS IIIB)
FDA allowed an Investigational New Drug (IND) for Systemic AAV Phase 1/2 Clinical Study With ABO-102 Gene Therapy for Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Abeona highlighted new preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) data at WORLDSymposium() 2016 which demonstrated encouraging in vivo efficacy in preclinical JNCL (also known as Juvenile Batten disease) model
Abeona partnered with Therapure Biopharma to continue its efforts in developing rare plasma proteins in SDF Alpha for inherited COPD
Abeona presented compelling data at the 2nd Annual CRISPR Precision Gene Editing Congress in Boston, MA which showed that CRISPR/Cas9 gene repair resulted in normalization of the FANCC gene in Fanconi anemia (FA)
First Quarter Summary Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2016 were $37.4 million, compared to $40.1 million as of December 31, 2015. Net cash used in operating activities in 1Q16 was $2.5 million as compared to $3.2 million in the same period in 2015, a decrease of $647 thousand.
Revenues: Revenues were $235 thousand for the first quarter of 2016, compared to $258 thousand in in the first quarter of 2015. 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 $0.17 for the first quarter of 2016, compared to a loss per share of $0.10 in comparable period in 2015.

On May 16, 2016 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company with three clinical-stage development programs focused on the emerging field of regenerative medicine, reported financial results for the first quarter ended March 31, 2016 (Press release, BioTime, MAY 16, 2016, View Source;p=RssLanding&cat=news&id=2168708 [SID:1234512445]). The company also announced plans to expand the AST-OPC1 Phase 1/2a trial in spinal cord injury patients following recent clearance for the expansion by the U.S. Food and Drug Administration (FDA) based on the favorable safety profile observed so far with AST-OPC1 in the current study.

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"Recent FDA clearance for expanding the AST-OPC1 clinical study in spinal cord injury patients and the recent capital raise needed to fund this expansion are key developments for Asterias," said Steve Cartt, President and Chief Executive Officer. "This important trial expansion should increase the statistical confidence of the safety and efficacy readouts, expand the range of spinal cord injury patients being evaluated, and better position the product for a potential accelerated regulatory pathway should we observe positive efficacy signals and continued safety in the study."

Mr. Cartt continued, "In addition, the capital we recently raised, despite the very challenging capital markets, significantly strengthened our cash position. Combined with continued non-dilutive funding from leading scientific institutions, this will allow Asterias to immediately progress our three clinical-stage programs toward important milestones targeted for late 2016 and into 2017."

As of May 15, 2016, the company’s cash, cash equivalents and available-for-sale securities totaled over $33 million. This includes a recent $2.5 million grant payment from the California Institute of Regenerative Medicine (CIRM) related to progress in the ongoing AST-OPC1 study.

Corporate Highlights

On May 13, 2016, Asterias raised approximately $16.2 million in net proceeds from a public offering of shares of its common stock and warrants. Asterias has granted the underwriters a 30-day option to purchase up to an additional 772,059 shares of common stock and/or additional warrants to purchase up to 386,029 shares of common stock to cover over-allotments, if any. The underwriters exercised their over-allotment option to purchase 386,029 additional warrants. If the over-allotment option to purchase the additional shares is exercised in full, additional net proceeds from the offering to Asterias will be approximately $2.4 million.
In April, Howard I. Scher M.D., one of the world’s leading oncology experts, was appointed to the Board of Directors of Asterias. Dr. Scher is internationally recognized for his expertise in clinical oncology. He has extensive experience in the design of clinical trials for novel anti-cancer agents, including monoclonal antibodies and other biologic therapies, cytotoxics, and drugs that target specific signaling pathways.
Research and Development Highlights

AST-OPC1 (oligodendrocyte progenitor cells)

Patient recruitment is ongoing for the second cohort in a Phase 1/2a clinical trial of AST-OPC1 in complete cervical spinal cord injury, in which five patients will be administered a dose of 10 million AST-OPC1 cells. This cohort is the first of two dose cohorts receiving doses in the predicted efficacious range based on preclinical studies. Asterias has been granted FDA clearance to expand patient enrollment in the Phase 1/2a clinical trial from 13 patients to up to 35 patients, based on the continued favorable safety profile observed in the ongoing clinical study. The company believes that the trial expansion should increase the statistical confidence of the safety and efficacy readouts, expand the range of spinal cord injury patients being evaluated, and better position the product for a potential accelerated regulatory pathway should the company observe positive efficacy signals and continued safety in the study. This trial is being funded in part by a $14.3 million grant from CIRM.
In February, the FDA granted Orphan Drug Designation for AST-OPC1 for the treatment of acute spinal cord injury. Orphan Drug Designation qualifies the sponsor of the drug certain benefits and incentives, including seven years of marketing exclusivity following regulatory approval, and financial incentives such as potential tax credits for certain activities and waiver of certain administrative fees.
In April, Asterias presented an overview of the AST-OPC1 therapeutic development program in spinal cord injury at the American Spinal Injury Association Annual Meeting and at the Stem Cell Summit 2016 meeting.
AST-VAC1 (antigen-presenting autologous dendritic cells)

In February, Asterias successfully completed the End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for AST-VAC1, the company’s investigational cancer immunotherapy and lead clinical program targeting maintenance of relapse-free-survival in acute myeloid leukemia (AML) patients. The company currently is evaluating plans for progressing the AST-VAC1 program towards a pivotal Phase 3 trial which would begin in late 2017.
Clinical data from the Phase 2 trial of AST-VAC1 in AML was presented during an oral session at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 19th Annual Meeting on May 5, 2016. The data was first presented at the 2015 annual meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper).
AST-VAC2 (antigen-presenting allogeneic dendritic cells)

AST-VAC2 is Asterias’ innovative allogeneic (non-patient-specific) immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. The company’s research partner, Cancer Research UK (CRUK), will execute the first clinical trial of AST-VAC2. As part of this partnership, CRUK will perform cGMP manufacture of AST-VAC2 at its Biotherapeutics Development Unit, and will submit a Clinical Trial Authorisation application to the UK regulatory authorities for a Phase 1/2a clinical trial in non-small cell lung cancer. The trial will be sponsored, managed and funded by the CRUK Centre for Drug Development. Asterias anticipates receiving approval from UK regulatory authorities for clinical development of AST-VAC2 by the end of 2016 and beginning enrollment for the Phase 1/2a clinical trial in the first quarter of 2017. The trial will examine the safety, immunogenicity and activity of AST-VAC2 in non-small cell lung cancer patients and could potentially position the product for development in numerous cancer indications.
First Quarter Financial Update

Total revenues were $1.6 million for the first quarter. Revenues are comprised of grant income as well as royalty revenues on product sales by licensees. Research and development expenses were $6.3 million for the first quarter. General and administrative expenses were $6.3 million for the first quarter. Net loss was $10.3 million for the three months ended March 31, 2016, or $0.27 per share, including a deferred income tax benefit of $902,000. For the first quarter, net cash used in operating activities was $4.2 million.

On May 11, 2016 Lytix Biopharma reported LTX-315’s ability to enhance infiltration of CD8 positive T-cells in murine and human tumors, and T-cell clonality in B16 melanomas (Press release, Lytix Biopharma, MAY 14, 2016, View Source [SID:1234514842]). These abilities make LTX-315 an ideal partner for beeing combined with other types of immunotherapy. The strong synergy between LTX-315 and anti-CTLA-4 in preclinical cancer model was also presented.

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On May13, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "the Company"), reported that an article has been published detailing the immuno-ablative mechanism of action of PV-10, the Company’s novel investigational drug for cancer (Press release, Provectus Pharmaceuticals, MAY 13, 2016, https://www.pvct.com/pressrelease.html?article=20160513.1 [SID:1234512371]).

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The article, titled, "Intralesional Rose Bengal in Melanoma Elicits Tumor Immunity Via Activation of Dendritic Cells by the Release of High Mobility Group Box 1," appears as an advance publication in Oncotarget, an Open-Access journal, and can be accessed by visiting:

View Source

The article documents results of a multi-year, multidisciplinary translational medicine program led jointly by Shari Pilon-Thomas and Amod A. Sarnaik of Moffitt Cancer Center in Tampa, Florida. The authors report detailed data on the mode in which intralesional injection of PV-10 (rose bengal) selectively kills tumor cells and the immunologic signaling that results from tumor ablation, starting with release of High Mobility Group Box 1 (HMGB1, a Damage-Associated Molecular Pattern molecule released by dying cancer cells that can serve as an immunological adjuvant to promote phagocytosis, antigen-presentation, and dendritic cell activation). The authors then follow this signaling through antigen uptake and dendritic cell activation, T cell priming and activation in peripheral blood, and culminating in a tumor-specific immune response marked by T cell infiltration and regression of uninjected tumors.

Eric Wachter, CTO of Provectus, observed, "The Moffitt researchers have systematically documented each of the key steps in the immuno-oncology cycle described by Chen and Mellman in their landmark review article (Oncology Meets Immunology: the Cancer-Immunity Cycle. Immunity 2013; 39: 1-10). In an exemplary demonstration of translational medicine, this team identified important immunologic markers in model systems and verified key facets of these in clinical trial participants, and similarly identified other markers in clinical trial participants and substantiated these in mouse models. While a number of their main observations were previously reported at scientific meetings, these are presented here in detailed, integrated fashion for the first time."

Shari Pilon-Thomas of Moffitt, stated, "Concordance of tumor-specific T cells in peripheral blood of clinical trial participants and mice led us to look for triggers of T cell activation. Working back from these observations, we found that HMGB1 release was common in mouse and man after tumor ablation with PV-10. These results support PV-10 ablation and the resulting tumor necrosis as the upstream trigger for systemic anti-tumor response."

Wachter noted, "This paper is a watershed event in the development of PV-10, walking the reader through all the steps of immune activation after PV-10 injection, from immunogenic cell death and signaling via release of HMGB1, dendritic cell recruitment and infiltration into draining lymph nodes, activation of tumor-specific T cells, and killing of uninjected tumors upon infiltration by these T cells."

Wachter added, "This mechanism of action informed the design of the two active PV-10 clinical trials: NCT02288897 in patients with locally advanced cutaneous melanoma (melanoma limited to the skin) to test the hypothesis that PV-10 alone can produce a systemic immune response that translates to longer progression free survival (PFS); and NCT02557321 in patients with later stage melanoma to test whether combination of PV-10 with the recently approved systemic immunotherapy, pembrolizumab, can ‘induce and boost’ an immune response against melanoma."

On May 13, 2016 Enumeral Biomedical Holdings, Inc. (OTCQB: ENUM) ("Enumeral" or the "Company"), a biotechnology company focused on the discovery and development of novel antibody-based immunotherapies to help the immune system fight cancer and other diseases, reported its financial results for the three months ended March 31, 2016 (Press release, ENUMERAL BIOMEDICALORATION, MAY 13, 2016, View Source;p=irol-newsArticle&ID=2168350 [SID:1234512372]).

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Recent Business Highlights

In April 2016, Enumeral entered into a License and Transfer Agreement with Pieris Pharmaceuticals, Inc. and Pieris Pharmaceuticals GmbH. Pursuant to this agreement, Pieris is licensing from Enumeral specified intellectual property related to Enumeral’s anti-PD-1 antibody program ENUM 388D4 for the potential development and commercialization by Pieris of novel multispecific therapeutic proteins comprising fusion proteins based on Pieris’ Anticalins class of therapeutic proteins and Enumeral antibodies in the field of oncology.
In April 2016, Enumeral presented research findings on the Company’s novel class of anti-PD-1 antibodies in a poster at the AACR (Free AACR Whitepaper) Annual Meeting 2016 in New Orleans, noting that Enumeral’s anti-PD-1 antibody ENUM 244C8 appears to elicit cytokine secretion from cell types associated with innate immunity in ex vivo assays using lung biopsy samples from human patients.
First Quarter 2016 Financial Results

Cash and Cash Equivalents: Cash and cash equivalents as of March 31, 2016 were $1,837,304, as compared to $3,596,262 as of December 31, 2015. As of the date of this press release, Enumeral believes that it only has sufficient liquidity to fund operations through June 2016. If Enumeral is unable to raise additional capital on acceptable terms and on a timely basis, Enumeral may be required to downsize or wind down its operations through liquidation, bankruptcy, or a sale of its assets. The Company is currently exploring a range of potential transactions, which may include public or private equity offerings, debt financings, collaborations and licensing arrangements, and/or other strategic alternatives, including a merger, sale of assets or other similar transactions.
Revenue: Revenue increased by $159,735 to $434,457 for the three months ended March 31, 2016, as compared to $274,722 for the three months ended March 31, 2015. This increase in revenue is attributable to the Company’s collaboration agreement with Merck and its grant from the National Cancer Institute.

Research and Development Expenses: Research and development ("R&D") expenses increased by $239,539 to $1,470,043 for the three months ended March 31, 2016, as compared to $1,230,504 for the three months ended March 31, 2015. This increase is primarily due to increased facility expenses in connection with the Company’s office relocation and an increase in stock-based compensation expense associated with stock option grants issued during the three months ended March 31, 2016.
General and Administrative Expenses: General and administrative ("G&A") expenses decreased by $230,609 to $1,194,334 for the three months ended March 31, 2016, as compared to $1,424,943 for the three months ended March 31, 2015. This decrease is primarily due to a decrease in legal costs and a decrease in exit costs associated with the termination of the Company’s former lease in connection with the Company’s office relocation.

Other Income (Expense): Other income decreased by $3,997,651 to $674,868 for the three months ended March 31, 2016, as compared to $4,672,519 for the three months ended March 31, 2015. This decrease is primarily due to a decrease in non-cash income related to the change in the fair value of derivative liabilities associated with the warrants issued in connection with the Company’s July 2014 private placement transaction. The Company expects that future changes in the fair value of the derivative liabilities will be due primarily to fluctuations in the value of the Company’s common stock and potential exercises of outstanding warrants.

Net Income (Loss): Net income (loss) changed by $3,846,846 to ($1,555,052) for the three months ended March 31, 2016, as compared to $2,291,794 for the three months ended March 31, 2015. This change is primarily due to the decrease in other income described above.