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 12, 2016 Cellectar Biosciences, Inc. (NASDAQ:CLRB), an oncology-focused biotechnology company, reported its financial results for the first quarter of 2016 (Filing, Q1, Cellectar Biosciences, 2016, MAY 16, 2016, View Source [SID:1234512452]).

During the first quarter of 2016, the company reported research and development expenses of $1.0 million, a reduction of $0.6 million from the first quarter of 2015. This improvement is attributable to the company’s shift in strategic focus on its therapeutic compound research and development efforts and the streamlined clinical trial approach it implemented during the second half of 2015.

Cellectar’s general and administrative expenses for first quarter 2016 totaled $1.0 million, similar to the prior year period. Loss from operations was $2.0 million, compared to $2.6 million during the same period last year.

The Company ended the first quarter with $1.9 million in cash and cash equivalents, compared to $3.9 million in cash and cash equivalents on December 31, 2015. When added to the approximately $7.2 million generated from the recently completed public offering, the company estimates that its available cash and cash equivalents should fund its planned operations into the first quarter of 2017. However, the company expects that additional capital will be required to complete its planned clinical and preclinical development.

"We continue to successfully execute our operating plan which included positive CLR 131 phase 1 data for the treatment of relapsed or refractory multiple myeloma, advanced our chemotherapeutic phospholipid drug conjugate program and launched our research collaboration with Pierre Fabre," said Jim Caruso. "We look forward to sharing these results in our conference call this afternoon and discussing our plans to further advance the company."

Cellectar will be holding a conference call at 5:00 PM ET today to review these results, as well as the company’s development plans. The call can be accessed by calling 888-646-8293. The call will also be webcast and replays will be available, both via the Investor Relations section of the company’s website: investor.cellectarbiosciences.com.

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On May 16, Pfizer Inc. (NYSE:PFE) and Anacor Pharmaceuticals, Inc. (NASDAQ:ANAC) reported that they have entered into a definitive merger agreement under which Pfizer will acquire Anacor for $99.25 per Anacor share, in cash, for a total transaction value, net of cash, of approximately $5.2 billion, which assumes the conversion of Anacor’s outstanding convertible notes (Press release, Pfizer, MAY 16, 2016, View Source [SID:1234512410]). The Boards of Directors of both companies have unanimously approved the transaction. Anacor’s flagship asset, crisaborole, a differentiated non-steroidal topical PDE4 inhibitor with anti-inflammatory properties, is currently under review by the U.S. FDA for the treatment of mild-to-moderate atopic dermatitis, commonly referred to as eczema.

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"We believe the acquisition of Anacor represents an attractive opportunity to address a significant unmet medical need for a large patient population with mild-to-moderate atopic dermatitis, which currently has few safe topical treatments available," said Albert Bourla, Group President of Pfizer’s Global Innovative Pharma and Global Vaccines, Oncology and Consumer Healthcare Businesses. "Crisaborole is a differentiated asset with compelling clinical data that, if approved, has the potential to be an important first-line treatment option for these patients and the physicians who treat them."

"Anacor will be a strong fit with Pfizer’s innovative business, further supporting our strategic focus on Inflammation and Immunology, and is expected to enhance near-term revenue growth for the innovative business. Our dedicated Inflammation and Immunology group has strong existing in-market franchises with Enbrel and Xeljanz, as well as a robust mid-stage pipeline, and this acquisition has the potential to add a near-term U.S. product launch. We believe we are well positioned to maximize crisaborole’s commercial potential through our strong relationships with pediatricians and primary care physicians," continued Bourla.

In both of its Phase 3 pivotal studies, crisaborole achieved statistically significant results on all primary and secondary endpoints and in March 2016, the FDA accepted for review Anacor’s New Drug Application seeking approval of crisaborole for the potential treatment of mild-to-moderate atopic dermatitis in children and adults. The Prescription Drug User Fee Act (PDUFA) goal date for the completion of the FDA’s review is January 7, 2017. If approved, Pfizer believes peak year sales for crisaborole have the potential to reach or exceed $2.0 billion.

"Today marks the beginning of an exciting new chapter for Anacor, which we believe will deliver significant value to our shareholders," said Paul L. Berns, Anacor’s Chairman and Chief Executive Officer. "We have a deep respect for Pfizer, and it is clear that they share our commitment to addressing the significant unmet medical needs in inflammatory disease. We are proud of the innovative company that our team has built and are confident that Pfizer will help accelerate Anacor’s important mission given the strength of its global platform and resources."

Atopic dermatitis is a common, relapsing, chronic, inflammatory skin disorder, with patients displaying a chronic rash characterized by inflammation and itching, often occurring in folds of the skin with symptoms lasting up to 14 days or more. Approximately 18 to 25 million people in the United States suffer from this condition, including between 8 and 18% of infants and children. Atopic dermatitis has been considerably underdiagnosed due to the lack of approved effective systemic agents, and limitations of current topical agents. There have been no new molecular entities for atopic dermatitis in the last 15 years.

Anacor also holds the rights to Kerydin, a topical treatment for onychomycosis (toenail fungus) that is distributed and commercialized by Sandoz Inc. in the U.S.

Pfizer anticipates financing the transaction through existing cash. Pfizer does not expect the transaction to impact its current 2016 financial guidance. Pfizer expects the transaction to be slightly dilutive to Adjusted Diluted Earnings Per Share (EPS)(1) in 2017 with accretion to Adjusted Diluted EPS(1) beginning in 2018 and increasing thereafter.

Under the terms of the merger agreement, a subsidiary of Pfizer will commence a cash tender offer to purchase all of the outstanding shares of Anacor common stock for $99.25 per share in cash. The closing of the tender offer is subject to customary closing conditions, including U.S. antitrust clearance and the tender of a majority of the outstanding shares of Anacor common stock. The merger agreement contemplates that Pfizer will acquire any shares of Anacor that are not tendered into the offer through a second-step merger, which will be completed promptly following the closing of the tender offer. Pfizer expects to complete the acquisition in the third-quarter 2016.

Pfizer’s financial advisors for the transaction were Centerview Partners and Guggenheim Securities, and Wachtell, Lipton, Rosen & Katz acted as its legal advisor. Citi served as Anacor’s financial advisor, and Davis Polk & Wardwell, LLP served as its legal advisor.

On May 16, 2016 Hemispherx Biopharma (NYSE MKT: HEB) reported its financial results for the three months ended March 31, 2016 (Filing, Q1, Hemispherx Biopharma, 2016, MAY 16, 2016, View Source [SID:1234512453]). The net loss was approximately $2,164,000 or $(0.01) per share as compared to a net loss of $3,445,000 or ($0.02) per share for the same three month period in 2015. Cash, cash equivalents and marketable securities were approximately $7,849,000 at March 31, 2016 as compared to $8,910,000 as of December 31, 2015.

Hemispherx Biopharma recently made changes to its senior management team and implemented austerity measures which included the reduction of executive compensation and elimination of non-essential contractors and personnel. These measures have resulted in a significant reduction in costs and expenses. The company is now focusing on commercial success by seeking co-development partners and working closely with the research and regulatory communities to bring disease fighting technologies to the world.

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