On March 8, 2016 Dynavax Technologies Corporation (NASDAQ: DVAX) reported financial results for the fourth quarter and year ended December 31, 2015 (Press release, Dynavax Technologies, MAR 8, 2016, View Source [SID:1234509405]).

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The Company had $196.1 million in cash, cash equivalents and marketable securities as of December 31, 2015.

Total revenues for the year ended December 31, 2015, decreased by $7.0 million or 63 percent compared to the same period in 2014, primarily due to a $5.2 million decrease in collaboration revenue due to winding down of work performed for the AZD1419 program and expiration of our collaboration agreement with GSK in 2014.

Operating expenses increased by $6.8 million or seven percent during 2015 compared to 2014, primarily due to costs related to HBV-23, the Phase 3 clinical study of HEPLISAV-B completed in October 2015, preparation for the commercial launch of HEPLISAV-B in the United States and clinical trial expense for SD-101, Dynavax’s cancer immunotherapeutic product candidate.

The net loss allocable to common stockholders for the year ended December 31, 2015 was $106.8 million, or $3.25 per share, compared to $90.7 million, or $3.45 per share for the year ended December 31, 2014.

"During 2015, we completed HBV-23 and significantly strengthened the Company’s cash position. Earlier this year we reported that this third pivotal study had met both co-primary endpoints. We plan to resubmit the HEPLISAV-B BLA (Biologics License Application) to the FDA by the end of this month. Based on our expectation of a six-month review, if our application is approved we expect to launch this product in the fourth quarter of this year," said Eddie Gray, chief executive officer of Dynavax.

On March 8, 2016 – Soricimed Biopharma Inc. ("Soricimed"), a clinical-stage pharmaceutical company discovering and developing peptide-based cancer therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to peptide SOR-C13 for the treatment of ovarian cancer (Press release, Soricimed Biopharma, MAR 8, 2016, View Source [SID:1234509843]).

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Orphan drug status is granted following review of the rarity and severity of the medical condition, as well as the potential benefit of the product treating this condition. Orphan drug status qualifies Soricimed for various development incentives, including tax credits and reduced filing fees for clinical trials undertaken in the U.S. If approved for commercialization by the FDA, SOR-C13 may qualify for seven years of marketing exclusivity in the United States.

According to the American Cancer Society, ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system, with an incidence rate of 11.9 per 100,000 and a death rate of 7.7 per 100,000. In some cases, orphan drugs can be made available to patients before marketing approval on a compassionate use basis.

"Receiving orphan drug status is significant", stated Paul Gunn, President and CEO at Soricimed. "It is an important regulatory milestone, offering special incentives to Soricimed through the development stage of SOR-C13." Soricimed announced that it had reached its target enrollment and treatment duration in its Phase 1 trial of SOR-C13 in patients with solid tumor cancers who had failed other treatments. Topline results were released last month.

About SOR-C13: SOR-C13 is a first-in-class peptide in development for the treatment of cancer. SOR-C13 binds with high selectivity and affinity to TRPV6, a calcium channel that is highly elevated in prostate, breast, lung and ovarian cancer and is correlated with poor outcomes. TRPV6-mediated Ca2+ entry is responsible for maintaining a high tumour proliferation rate, as well as increasing tumour cell survival and fortifying mechanisms that withstand cell destruction. By binding to this channel, SOR-C13 starves cancer cells of calcium that is needed for cell growth and division. As demonstrated in animal models, the result is an inhibition of tumor growth. Due to the high specificity of SOR-C13 for its target and its unique mechanism of action this drug candidate may result in fewer and less severe side effects compared to standard cancer chemotherapy. SOR-C13 is the first drug candidate targeting TRPV6 to have entered clinical development anywhere in the world.

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 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 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.