On May 16, 2016 Transgenomic, Inc. (NASDAQ:TBIO), reported new concordance study data confirming the superior performance of its ICE COLD-PCR (ICP) enrichment technology over standard biopsied tissue PCR results for detection of tumor mutations in cancer patients (Press release, Transgenomic, MAY 16, 2016, View Source [SID:1234512439]). The data show that plasma-based liquid biopsy testing using ICP is 100% concordant with an established approach that uses standard PCR. The matched patient data further indicate that the ICP-enabled liquid biopsies detected more tumor mutations than tissue biopsy testing using standard PCR. Transgenomic is in the process of submitting the study to a peer-reviewed scientific publication.

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The concordance study was reviewed by Anil Vachani, MD, MS, Associate Professor of Medicine at the Hospital of the University of Pennsylvania and the Veteran’s Administration Medical Center, and a Director of Clinical Research at the University of Pennsylvania Perelman School of Medicine. Dr. Vachani’s research includes the study of methods to optimize the use of targeted cancer therapies. Dr. Vachani noted, "High sensitivity detection of genetic biomarkers from cell free DNA has the potential to provide guidance to clinical oncologists for personalized, precision cancer treatment. Liquid biopsies are a source of genetic information when there is no available tumor tissue and they make it possible to monitor treatment effectiveness and the emergence of drug resistance on an ongoing basis, thereby enabling more effective real-time treatment decisions. This can be critical for patients with metastatic disease. Based on these initial encouraging results, ICP may be a useful technology for helping to facilitate the broad use of cell free DNA liquid biopsies in cancer. I look forward to seeing the results of further confirmatory studies now underway."

In this study, Transgenomic analyzed 22 matched samples from chemo-naïve late-stage colorectal cancer (CRC) patients. The purpose was to compare the concordance between tissue biopsy results using traditional PCR and Sanger sequencing with plasma liquid biopsy results obtained using ICE COLD-PCR and Sanger sequencing. The study also compared mutation detection results from the matched tissue/FFPE samples analyzed with Sanger sequencing and either standard PCR or ICE COLD-PCR.

The concordance between the conventional tumor biopsy results and the matched ICP-enriched plasma sample liquid biopsy results was 100%. In addition, the ICP-enriched biopsies identified seven mutations missed by the conventional PCR approach–four additional mutations were identified in the ICP-enriched plasma samples and three additional mutations were identified in tissue samples that were analyzed with ICE COLD-PCR.

Transgenomic President and CEO Paul Kinnon commented, "We are delighted with the latest results from our studies, which show 100% concordance between ICE COLD-PCR-based liquid biopsy results and tissue-based results from the same patients, as well as ICP’s superior performance in identifying tumor mutations that were missed by conventional PCR methods. This new data further validates the clinical utility of ICP as a robust method to detect and monitor actionable mutations from a liquid biopsy sample, an essential component needed for adoption of precision medicine. We expect to submit additional data to peer reviewed scientific settings in the coming months."

Transgenomic’s ICP cancer assays exponentially amplify targeted regions of exons that have been determined to be predictive and/or prognostic for many types of cancer, including colorectal cancer, melanoma, breast cancer and non-small cell lung cancer. The 17-target mutation panel matches the set of mutation data currently used routinely in clinical settings for identifying and selecting therapeutic or clinical trial options for patients being treated for multiple cancer types. Additional mutations are being added as the science advances.

ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.

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.

Oncbiomune has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, OncBioMune Pharmaceuticals, 2017, MAY 16, 2016, View Source [SID1234522122]).

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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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