On March 08, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported it will present new data on its proprietary variant classification program that is used to classify variants in cancer risk genes (Press release, Myriad Genetics, MAR 8, 2016, View Source [SID:1234509407]). The study will be highlighted at the American College of Medical Genetics (ACMG) and Genomics annual clinical genetics meeting in Tampa, Fla.

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"As the world leader in multi-gene panel testing for hereditary cancer, we have a long track record and commitment to advancing the science of variant classification," said Johnathan Lancaster, chief medical officer, Myriad Genetic Laboratories. "Our goal is to provide physicians with the highest quality results possible for every test we perform."

Myriad’s myVision variant classification program is comprised of proprietary techniques that allow for the most accurate classification for hereditary cancer variants including: Pheno, M-Co, InSite and LitView. The Pheno technique is a history weighting algorithm that could only be developed by Myriad after sequencing the DNA of more than 400,000 patients.

In this study, the Pheno algorithm was used to analyze variants of unknown significance associated with high cancer risk genes including BRCA1, BRCA2, MLH1, MSH2 and MSH6. Additionally, the algorithm was updated to analyze variants of unknown significance in moderate cancer risk genes including ATM, CHEK2 and PALB2. The results of this study showed that Pheno was >99.5 percent accurate for upgrading and downgrading variants of uncertain significance to more definitive clinical classifications.

"As the myRisk Hereditary Cancer 25-gene panel test becomes more integrated into clinical practice, there will be a need to classify a greater number of variants," said Lancaster. "Variants of uncertain significance are particularly problematic for physicians because they leave questions as to whether variations in a patient’s DNA are of concern. This study demonstrates the ability of Pheno to accurately classify variants from a broader range of genes, which should help reduce anxiety for more patients and their families."

Details about the featured Myriad presentations at ACMG are below. Follow Myriad on Twitter via @MyriadGenetics and stay up-to-date with the meeting by using the hashtag #ACMGMtg16.

Myriad Presentations

Title: Reclassification of uncertain variants identified in high and moderate cancer risk genes using history weighting analysis.
Date: Friday, March 11, 2016: 10:30 a.m. to 12:00 p.m. ET.
Location: Poster 110.
Presenter: Karla Bowles, Myriad Genetic Laboratories.

Title: Detailed review of four patients affected with cancer that were previously unaffected at the time of single syndrome testing and subsequently had pathogenic variants identified by a 25-gene panel.
Date: Thursday, March 10, 2016: 10:30 a.m. to 12:00 p.m. ET.
Location: Poster 109.
Presenter: Allison Anguiano, Myriad Genetic Laboratories.
About Pheno and myVision Variant Classification Program

Pheno is a family history-weighting tool that compares the severity of personal and family histories of patients who carry a specific variant to that of individuals who carry known deleterious mutations and to individuals in whom no mutation was detected. Pheno is a proprietary component of the myVision Variant Classifcation Program, which is the most advanced informatics program in the industry, overseeing the classification and reclassification of genetic variants, and is part of Myriad’s commitment to patients and their families that lasts a lifetime. For more information about Myriad’s variant classification program visit: View Source

About Myriad myRisk Hereditary Cancer Testing

The Myriad myRisk Hereditary Cancer test uses next-generation sequencing technology to evaluate 25 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma. For more information visit: View Source

On March 8, 2016 Abeona Therapeutics, Inc. (NASDAQ: ABEO), a 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 fourth quarter and full fiscal year ended December 31, 2015 (Press release, Abeona Therapeutics, MAR 8, 2016, View Source;p=RssLanding&cat=news&id=2146834 [SID:1234510443]). The Company will provide a business update for investors and other stakeholders on a conference call, Wednesday, March 9th, at 4:45 pm (Eastern). Tim Miller, Ph.D., President and CEO 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 4Q15 financials, and a discussion of business highlights.

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"The past year has led to significant advancements in our goal of building a leadership position in the field of gene therapy and plasma protein therapies towards transforming the lives of patients with rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "In 2015, we expanded our pipeline with two clinical stage AAV gene therapies for Sanfilippo syndrome types A and B, added a third AAV gene therapy product in Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) (also known as juvenile Batten disease), signed a license to an innovative CRISPR-Cas9 gene editing platform in rare blood disorders, with an initial focus in Fanconi anemia, strengthened our team, and added substantial financial resources to our balance sheet. In 2016, our priorities include driving our AAV gene therapy and alpha-1 protease inhibitor programs into the clinic, and advancing our gene editing programs including defining of regulatory pathways to bring our CRISPR product candidates to patients."

Tim Miller, Ph.D., President and CEO, stated, "2016 will be an exciting, transformative year for Abeona Therapeutics as we position ourselves to enter multiple human clinical trials with our pipeline of innovative product candidates. As recently announced, the FDA allowance of the IND for the Phase 1/2 clinical study of ABO-102 for patients with Sanfilippo syndrome type A (MPS IIIA) moves our programs into the clinic here in the US, and we look forward to working with our collaborators to expand this program into Europe and Australia later this year. We believe that our gene therapy programs in Sanfilippo syndrome type B (ABO-101) and Juvenile Neuronal Ceroid Lipofuscinosis (ABO-201) will follow shortly. Lastly, we would like to thank our dedicated researchers and clinical collaborators, as well as the many dedicated patient foundations, for their tireless efforts and commitment to advancing new treatment options for these devastating unmet medical needs."

Recent Abeona Highlights

Sanfilippo syndrome gene therapy programs: On February 29, 2016, Abeona announced the FDA allowance of 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). On January 11, 2016, we announced that initial regulatory approvals from European bodies — the Genetically Modified Organism (GMO) Voluntary Release regulatory filings, and the ethical committee regulatory filings — for both the ABO-101 and ABO-102 programs in Spain. Abeona plans to commence both programs for the upcoming human clinical trials to be conducted at Cruces University Hospital in Bilbao, Spain. Both the ABO-101 and ABO-102 programs have received Orphan Drug and Pediatric Rare Disease designations from the FDA.
SDF-Alpha plasma protein program: Abeona has completed optimization of the downstream chromatography steps for our SDF-Alpha (alpha-1 protease inhibitor) for inherited COPD. Additional provisional patent applications have been filed to provide the Company with expanded intellectual property protection. The Company has expanded its CMO relationships to ensure it has the ability to manufacture clinical material for future trials. The Company confirms that its proprietary SDF platform provides significantly enhanced yields of alpha-1 protease inhibitor, at levels up to 10 times of that achievable with the industry standard Cohn processes, and with purity levels consistent with that achieved by other commercial processes.
Advanced pipeline programs: Together with its academic collaborators, the Company continued to progress its pre-clinical programs in juvenile Batten disease and its CRISPR-Cas9 program in Fanconi anemia (FA) and other rare blood disorders. Juvenile Batten disease is the most common form of a group of disorders known as neuronal ceriod lipofuscinosis (NCL), a lysosomal storage disease that affects the nervous system in children and for which there are no approved treatment options. Fanconi anemia is a rare pediatric blood disease characterized by multiple physical abnormalities, bone marrow failure and a higher than normal risk of cancer.
Fourth Quarter and Full 2015 Summary Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2015 were $40.1 million, compared to $11.6 million as of December 31, 2014. The increase was primarily driven by net proceeds from multiple equity financings partially offset by cash used to fund operations. Cash used in operations in 2015 was $10.4 million.
Revenues: Revenue was $215 thousand and $1.0 million for the fourth quarter of 2015 and the full year 2015, respectively, compared to $234 thousand and $925 thousand in comparable periods in 2014. 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 $0.07 and $0.53 for the fourth quarter of 2015 and the full year 2015, respectively, compared to a loss per share of $2.05 and $15.26 in comparable periods in 2014.
About ABO-101 (AAV NAGLU) and ABO-102 (AAV SGSH) are next generation adeno-associated viral (AAV)-based gene therapies for MPS IIIA and MPS IIIB, respectively. These gene therapies involve a one-time delivery of a genetically modified virus to deliver a normal copy of the defective gene to cells of the central nervous system (CNS) and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in preclinical animal models of Sanfilippo syndrome, ABO-101 and ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in animals with Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose significantly restored normal cell and organ function, corrected cognitive defects that remained for months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with Sanfilippo syndrome Type A and B,. In addition, safety studies conducted in animal models of Sanfilippo syndromes have demonstrated that delivery of ABO-101 and ABO-102 are well tolerated with minimal side effects.

About Abeona: Abeona Therapeutics, Inc.

On March 8, 2016 OncoSec Medical Incorporated ("OncoSec") (NASDAQ: ONCS), a company developing DNA-based intratumoral cancer immunotherapies, reported key corporate objectives as well as financial results for the second quarter and year to date ended January 31, 2016(Press release, OncoSec Medical, MAR 8, 2016, View Source [SID:1234509408]).

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CORPORATE OBJECTIVES
"As we enter the next quarter, we are confident in our team’s mission to deliver safer and more effective intratumoral immunotherapies to provide long-term benefits for cancer patients. The fundamental goal of our technology, ImmunoPulse IL-12, is to promote a systemic, tumor-specific immune response. We believe this holds the greatest potential to provide meaningful clinical benefit to patients and investment value to OncoSec’s shareholders," said Punit Dhillon, President and CEO of OncoSec. "Our objectives over the next year are focused on establishing clinical response data to support the combination rationale for ImmunoPulse IL-12 with anti-PD-1/PD-L1 as well as identifying a new lead candidate to expand our ImmunoPulse platform and deliver multiple immune molecules in a single treatment."

OncoSec’s development milestones and value drivers over the next 12 months include:

Complete patient enrollment in the Phase II combination trial of ImmunoPulse IL-12 with anti-PD-1 in patients with metastatic melanoma
Present preliminary clinical and biomarker data from our Phase II melanoma clinical trials at upcoming scientific conferences; data to be used to finalize development strategy
Identify novel "multi-gene" combination ImmunoPulse candidate
Complete triple negative breast cancer pilot study as proof-of-concept and present interim data
Present data from our preclinical programs, including studies with our existing industry collaborators
FINANCIAL RESULTS
For the second quarter of fiscal 2016 and the six months ended January 31, 2016, OncoSec reported a net loss of $7.0 million and $14.1 million, or $0.42 per share and $0.89 per share, respectively, compared to a net loss of $4.6 million and $8.7 million, or $0.38 per share and $0.71 per share, respectively, for the same periods last year. The increase in net loss for the year ended January 31, 2016, compared with the same period in 2015, resulted primarily from (i) an increase of $3.1 million in personnel costs, inclusive of non-cash stock-based compensation (ii) an increase of $1.3 million in clinical studies costs due to the progression of patient treatments in all of our clinical programs, (iii) an increase of $0.5 million related to outside services primarily associated with discovery research and next generation electroporation device development and (iv) an increase of $0.5 million in facility costs which consists primarily of rental expense due to the relocation of our Corporate headquarters, which includes onsite laboratory space. There were no revenues for the three and six months ended January 31, 2016 or January 31, 2015.

Research and development expenses were $4.1 million and $7.8 million for the second quarter of fiscal 2016 and the six months ended January 31, 2016, respectively, compared to $2.9 million and $5.4 million for the same periods in 2015. General and administrative expenses were $2.9 million and $6.3 million for the second quarter of fiscal 2016 and the six months ended January 31, 2016, compared to $1.7 million and $3.3 million for the same period in 2015.

At January 31, 2016, OncoSec had $28.8 million in cash and cash equivalents, as compared to $32.0 million of cash and cash equivalents at July 31, 2015. OncoSec expects these funds to be sufficient to allow it to continue to operate its business for at least the next 12 months.

On March 08, 2016 XBiotech Inc. (NASDAQ:XBIT) reported that the Company has now submitted its Marketing Authorization Application (MAA) for its candidate therapy (Xilonix) for advanced colorectal cancer to the European Medicines Agency (EMA) (Press release, XBiotech, MAR 8, 2016, View Source [SID1234556367]). Submission of the MAA for Xilonix is necessary for the Company to gain approval to sell the drug in European Union member nations. The Company, which only recently was granted eligibility to submit its MAA through the Centralised Procedure, has made a remarkably quick turnaround for delivery of the extensive MAA documentation needed for the EMA review.

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John Simard, CEO of XBiotech, commented, "With submission of the Marketing Authorization Application, we now look forward to the review process."

Few agents are reliably able to mediate durable responses in metastatic disease. And many agents have significant trade-offs in terms of side effects. In patients with advanced disease, often weakened from successive rounds of cytotoxic therapy, the risk-benefit associated with further therapy is often questionable. Based on novel objective response (OR) criteria, developed by XBiotech in conjunction with the EMA’s Scientific Advice Working Group, the candidate therapy subject of the MAA was recently evaluated in a Phase III clinical study for the ability to control disease-related symptoms that inversely correlate with survival. The Company believes using the symptom-based objective response criteria will allow better assessment of overall benefit from therapy in advanced cancer patients.

On March8, 2016 Sanofi Pasteur and Merck, known as MSD outside the United States and Canada, reported their intent to end their joint vaccines operations in Europe (Press release, Merck & Co, MAR 8, 2016, View Source [SID:1234509409]). Upon concluding their joint venture, both companies plan to integrate their respective European vaccine businesses into their operations, independently manage their product portfolios and pursue their own distinct growth strategies in Europe.

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The joint venture Sanofi Pasteur MSD, owned on a 50/50 basis by Sanofi Pasteur and MSD, was created in 1994 to develop and commercialize vaccines originating from both companies’ pipelines to improve and promote public health in 19 European countries. Over the past twenty years, Sanofi Pasteur MSD has launched numerous innovative vaccines originating from Sanofi Pasteur and MSD’s development pipelines, addressing key unmet medical needs and helping to protect millions of lives.

Sanofi Pasteur and MSD jointly issued the following statement: "We are proud of Sanofi Pasteur MSD’s successful 20-year history. Our joint venture has achieved considerable success over the past two decades from a public health and commercial perspective. After carefully considering our individual strategic priorities, alongside the economic and regulatory environments for vaccine operations in the European Union, we have mutually agreed that it is in our best interests to manage our vaccine product portfolios independently. We believe that focusing our efforts on opportunities unique to our respective companies will better position us to drive growth, execute in a more efficient manner and optimize vaccine coverage. By bringing vaccines more rapidly to market, both companies would deliver greater value to all stakeholders."

Sanofi Pasteur and MSD will ensure that any impact on employees as a result of the proposed changes to the business model will be managed responsibly. We are also focused on a smooth and orderly transition while achieving our public healthcare goals and upholding our commitments to our customers and business partners.

Sanofi Pasteur and MSD expect the project to be completed by the end of 2016, subject to local labor laws and regulations and regulatory approvals.