1stOncology™: Cancer Intelligence Service – Page 16339 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Abeona Therapeutics Announces Fourth Quarter and Full Year 2015 Summary Financial Results and Recent Operational Highlights

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

"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.

Soricimed Granted Orphan-Drug Designation from the U.S. FDA for Treatment of Ovarian Cancer

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]).

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.

Rigel Announces Fourth Quarter and Year End 2015 Financial Results

On March 8, 2016 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the fourth quarter and year ended December 31, 2015(Press release, Rigel, MAR 8, 2016, View Source;p=RssLanding&cat=news&id=2146948 [SID:1234509426]).

"Fostamatinib is our top priority for 2016 with the first results of our Phase 3 studies in immune thrombocytopenic purpura anticipated in the middle of the year," said Raul Rodriguez, president and chief executive officer of Rigel. "Also this year, we expect the readouts of the two other fostamatinib clinical programs, the Phase 2 in IgA nephropathy and the recently announced Phase 2 proof-of-concept study in autoimmune hemolytic anemia," he added.

For the fourth quarter of 2015, Rigel reported a net loss of $12.7 million, or $0.14 per share, compared to a net loss of $22.3 million, or $0.25 per share, in the fourth quarter of 2014. Weighted average shares outstanding for the fourth quarters of 2015 and 2014 were 89.0 million and 87.8 million, respectively.

Contract revenues from collaborations of $8.5 million in the fourth quarter of 2015 were primarily comprised of the amortization of the $30.0 million upfront payment from Bristol-Myers Squibb (BMS), as well as a license fee from a third party pursuant to a license agreement executed in October 2015. Contract revenues from collaborations of $8.3 million in the fourth quarter of 2014 were comprised of non-refundable payments from AstraZeneca AB (AZ) as a result of its continued development of R256 in asthma.

Rigel reported total costs and expenses of $21.3 million in the fourth quarter of 2015, compared to $30.6 million in the fourth quarter of 2014. Costs and expenses in the fourth quarter of 2014 included a loss on executing a sublease, as well as stock-based compensation expense and severance costs related to the retirement of Rigel’s former chief executive officer.

For the year ended December 31, 2015, Rigel reported contract revenues from collaborations of $28.9 million and a net loss of $51.5 million, or $0.58 per basic and diluted share, compared to contract revenues from collaborations of $8.3 million and a net loss of $90.9 million, or $1.04 per basic and diluted share, in 2014. Contract revenues from collaborations in 2015 were mainly comprised of $16.6 million from the amortization of the $30.0 million upfront payment from BMS and upfront payments received from other collaborators. Contract revenues from collaborations in 2014 were comprised of non-refundable payments from AZ as a result of its continued development of R256 in asthma.

As of December 31, 2015, Rigel had cash, cash equivalents and short-term investments of $126.3 million, compared to $143.2 million as of December 31, 2014. Rigel expects this amount to be sufficient to fund operations into the third quarter of 2017.

8-K – Current report

On March 8, 2016 Dynavax Technologies Corporation (NASDAQ: DVAX) reported financial results for the fourth quarter and year ended December 31, 2015 (Filing, Q4/Annual, Dynavax Technologies, 2015, MAR 8, 2016, View Source [SID:1234509418]).

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.

Cerus Corporation Reports Fourth Quarter and Year End 2015 Results

On March 8, 2016 Cerus Corporation (NASDAQ:CERS) reported financial results for the fourth quarter and year ended December 31, 2015 (Press release, Cerus, MAR 8, 2016, View Source [SID:1234509417]).

Recent company highlights include:
U.S. Progress –

Signed INTERCEPT platelet and plasma supply agreements with the American Red Cross, Roswell Park Cancer Institute, Blood Systems, Inc., OneBlood, Inc., LifeShare Blood Centers, ARUP, Inc., Rhode Island Blood Center, Shepeard Community Blood Center, Mississippi Valley Regional Blood Center, Community Blood Center

Received CMS permanent P-codes for outpatient hospital billing of pathogen-reduced platelets and plasma

AABB authorized use of the INTERCEPT Blood System for platelets to reduce the risk of transfusion-associated graft versus host disease

Began enrollment in the Phase IV PIPER Study, which Cerus is conducting as its FDA-required post-approval haemovigilance study

Announced a collaboration agreement with Haemonetics for the use of Acrodose platelet kits with the INTERCEPT Blood System for platelets

International Progress –
Regulatory approval obtained for use of the INTERCEPT Blood System for platelets and plasma in Brazil, Peru, and Colombia

Appointed new leadership for the Europe, Middle East and Africa region

Signed INTERCEPT platelet and plasma supply agreement with the National Transfusiology Center of Mongolia

Recognition of pathogen reduction as a Zika intervention –
The U.S. Food and Drug Administration’s (FDA) guidance document regarding Zika allows for the use of pathogen reduction to reduce transfusion risk and maintain local blood availability

The World Health Organization (WHO) guidelines for maintaining a safe and adequate blood supply during Zika virus outbreaks includes pathogen reduction technology for safeguarding platelet and plasma supplies in areas with active transmission

"The current Zika outbreak highlights the need for a proactive solution to secure the safety and availability of national blood supplies. The FDA and WHO have now recognized that pathogen reduction is an integral component to ensure that patients are protected from possible transfusion transmission of Zika, as well as dengue and chikungunya viruses," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "Looking ahead, with over 60% of the U.S. market now under contract and a long term agreement with the American Red Cross, we expect to begin to transition these contracts into revenue in 2016, and in Europe, we seek to initiate our launch planning for our INTERCEPT red cell system pursuant to our planned CE Mark submission in the second half of the year."

Revenue
Product revenue for the fourth quarter of 2015 was $9.7 million compared to revenue for the same period in 2014 of $9.6 million. This reflects a 22% year over year increase in INTERCEPT disposable kit demand. Revenue for the year ended December 31, 2015 was $34.2 million, compared to $36.4 million for the year ended December 31, 2014. INTERCEPT disposable kit demand over the full year time period grew by 15%, in line with our expectations.

Looking ahead, the Company expects 2016 global revenue in the range of $37 million to $40 million.

Gross Margins
Gross margins for the fourth quarter of 2015 were 36%, compared to 31% for the fourth quarter of 2014. Gross margins for the year ended December 31, 2015, were 31%, compared with 42% in the same period in 2014.

The Company recorded period charges for expiring inventory and certain minimum contractual purchase commitments which negatively impacted margins by approximately 10% and 7% for the quarter and year ended December 31, 2015, respectively. These types of charges impacted margins by less than 1% during the same periods of 2014. In addition, margins for the year ended December 31, 2015 were negatively impacted by the decline in the value of the Euro relative to the Company’s reporting currency, the US dollar, negatively impacting reported gross margins by approximately 5% when comparing the year ended December 31, 2015 to the comparable period in 2014.

Operating Expenses
Total operating expenses were $18.5 million and $71.8 million for the quarter and year ended December 31, 2015, respectively, compared to $15.9 million and $59.7 million for the quarter and year ended December 31, 2014, respectively. The continued buildout of a U.S. based commercial team following December 2014 FDA approvals drove the increase in operating expenses throughout 2015. In addition, label claim expansion activities over 2015 drove increased costs which were offset by the decline in activities incurred in 2014 to obtain those December approvals.

Operating and Net Loss
Operating losses during the fourth quarter of 2015 were $15.0 million, compared to $12.9 million during the fourth quarter of 2014, and $61.1 million compared to $44.5 million for years ended December 31, 2015 and 2014, respectively.

Net loss for the fourth quarter of 2015 was $14.8 million, or $0.15 per diluted share, compared to a net loss of $20.2 million, or $0.26 per diluted share, for the fourth quarter of 2014. Net loss for the year ended December 31, 2015, was $55.9 million, or $0.61 per diluted share, compared to a net loss of $38.8 million, or $0.61 per diluted share, for the same period of 2014.

Net losses for the fourth quarter of 2015 were impacted by the operating losses discussed above and mark-to-market adjustments of the Company’s then outstanding warrants to fair value, which contributed to the Company’s net losses by $1.1 million during the quarter ended December 31, 2015, compared to a contribution of $6.6 million to net losses during the comparable period in 2014. Net losses for the fourth quarter of 2015 were also favorably impacted by approximately $0.2 million of foreign exchange gains during the fourth quarter of 2015, compared to losses of $0.4 million during the corresponding prior period.

Net losses for the year ended December 31, 2015 were impacted by the operating losses discussed above and mark-to-market adjustments of the Company’s then outstanding warrants to fair value, which reduced the Company’s net losses by $3.6 million during the year ended December 31, 2015 compared to a reduction to net losses of $7.7 million during the comparable period in 2014. Net losses for the year were also impacted by foreign exchange losses of $0.4 million during the year ended December 31, 2015, compared to $1.3 million of foreign exchange losses during the year ended December 31, 2014.

At December 31, 2015, the Company had no remaining outstanding warrants and as such, does not expect mark-to-market adjustments going forward.

Cash, Cash Equivalents and Investments
At December 31, 2015, the Company had cash, cash equivalents and short-term investments of $107.9 million compared to $51.3 million at December 31, 2014. Included in the 2015 short-term investments are approximately $11.2 million of marketable equity securities, which had no recorded value at December 31, 2014.

At December 31, 2015, the Company had approximately $20 million in outstanding debt under its loan agreement with Oxford Finance.