On August 3, 2016 Geron Corporation (Nasdaq:GERN) reported financial results for the three and six months ended June 30, 2016 and recent events (Press release, Geron, AUG 3, 2016, View Source;p=irol-newsArticle&ID=2192600 [SID:1234514221]).

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For the second quarter of 2016, the company reported a net loss of $8.6 million, or $0.05 per share, compared to $9.4 million, or $0.06 per share, for the comparable 2015 period. Net loss for the first six months of 2016 was $17.5 million, or $0.11 per share, compared to $18.7 million, or $0.12 per share, for the comparable 2015 period. The company ended the second quarter of 2016 with $136.4 million in cash and investments and has not incurred any impairment charges on its marketable securities portfolio.

Revenues for the three and six months ended June 30, 2016 were $211,000 and $960,000, respectively, compared to $251,000 and $788,000 for the comparable 2015 periods. Revenues for the three and six month periods ending June 30, 2016 and 2015 included royalty and license fee revenues under various non-imetelstat license agreements.

Total operating expenses for the three and six months ended June 30, 2016 were $9.1 million and $18.9 million, respectively, compared to $9.7 million and $19.7 million for the comparable 2015 periods. Research and development expenses for the three and six months ended June 30, 2016 were $4.6 million and $9.6 million, respectively, compared to $4.8 million and $9.8 million for the comparable 2015 periods. General and administrative expenses for the three and six months ended June 30, 2016 were $4.5 million and $9.3 million, respectively, compared to $4.0 million and $8.6 million for the comparable 2015 periods. Operating expenses for the three and six months ended June 30, 2015 also included restructuring charges of $941,000 and $1.3 million, respectively, in connection with the company’s organizational resizing announced in March 2015.

The decrease in research and development expenses for the three and six month periods ending June 30, 2016, compared to the same periods in 2015, primarily reflects the net result of reduced personnel-related costs resulting from the March 2015 organizational resizing and lower costs for the manufacturing of imetelstat drug product, partially offset by higher costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen Biotech, Inc. (Janssen). The company expects research and development expenses to increase during the remainder of the year as the clinical development of imetelstat continues in collaboration with Janssen. The increase in general and administrative expenses for the three and six month periods ending June 30, 2016, compared to the same periods in 2015, primarily reflects higher non-cash stock-based compensation expense and increased allocation of facilities and other overhead costs to general and administrative activities.

Interest and other income for the three and six months ended June 30, 2016 was $293,000 and $549,000, respectively, compared to $145,000 and $294,000 for the comparable 2015 periods. The increase in interest and other income for the three and six month periods ending June 30, 2016, compared to the same periods in 2015, primarily reflects higher yields on the company’s marketable securities portfolio.

Recent Company Events

In July 2016, three U.S. patents related to imetelstat were issued by the U.S. Patent and Trademark Office. U.S. 9,375,485 has claims covering the use of telomerase inhibitor compounds, including imetelstat, for alleviating at least one symptom of myelofibrosis or myelodysplastic syndromes, including chronic myelomonocytic leukemia, and is expected to remain in force until at least March 2033. U.S. 9,388,415 and U.S. 9,388,416 have claims covering methods for using imetelstat to inhibit the activity of telomerase and using imetelstat to inhibit cancer cell proliferation, as well as methods for using imetelstat to treat cancer, and are expected to remain in force until at least September 2024. These patents are related to Geron’s existing imetelstat composition of matter patent U.S. 7,494,982, which issued in 2009 and is expected to remain in force until at least December 2025. Further extension of patent terms may be available for regulatory review periods.

Geron’s portfolio of patents related to imetelstat and related products whose mechanism of action is telomerase inhibition have been licensed to Janssen under an exclusive worldwide license and collaboration agreement for all human disorders or medical conditions.

On August 3, 2016 Synthetic Biologics, Inc. (NYSE MKT: SYN), a clinical stage company focused on developing therapeutics to protect the gut microbiome while targeting pathogen-specific diseases, reported an operational update and reported financial results for the three months ended June 30, 2016(Press release, Synthetic Biologics, AUG 3, 2016, View Source [SID:1234514220]).

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Synthetic Biologics, Inc. www.syntheticbiologics.com (PRNewsFoto/Synthetic Biologics, Inc.)
"In the second quarter we continued the transition from an early-stage clinical development company to a late-stage clinical development company focused on the commercialization of our two lead GI microbiome-focused drug candidates," said Jeffrey Riley, Chief Executive Officer. "We held an End of Phase 2 meeting with FDA and received guidance for advancement to a pivotal clinical trial for SYN-010, designed to treat an underlying cause of the symptoms associated with irritable bowel syndrome with constipation (IBS-C)." Mr. Riley continued, "The approval of the generic name ‘ribaxamase’ for SYN-004, the announcement of positive clinical outcomes from a second Phase 2a clinical trial and robust enrollment in our global Phase 2b proof-of-concept clinical trial continued to fuel momentum for our program designed to protect the gut microbiome and prevent C. difficile infection (CDI), antibiotic-associated diarrhea (AAD) and the emergence of antibiotic resistant organisms. To date, we have enrolled 374 patients and anticipate announcing topline results from our ongoing Phase 2b clinical trial for SYN-004 in early 2017."

Microbiome-Focused Clinical Program Progress

SYN-010 – Treatment of irritable bowel syndrome with constipation (IBS-C):

Held End of Phase 2 meeting with FDA and received guidance for clinical study design and requirements for Phase 3 development
Plan to initiate Phase 2b/3 pivotal clinical trial (1Q 2017)
Presented detailed data during Digestive Disease Week 2016 supporting previously reported positive outcomes from two Phase 2 clinical trials of SYN-010, including:
Data demonstrating an inverse correlation between breath methane Area Under Curve (AUC) and complete spontaneous bowel movements (CSBMs) in study participants diagnosed with IBS-C
Data demonstrating clear improvements in abdominal pain, bloating and quality of life measures (IBS-SSS) in study participants who were administered SYN-010
Announced results from a separate randomized, open-label Pharmacokinetic (PK) study demonstrating SYN-010 avoided desired drug release in the stomach and delivered the antimethanogenic lovastatin lactone into the lower small intestine and colon while reducing systemic exposure to the cholesterol-lowering lovastatin beta-hydroxyacid metabolite

SYN-004 (ribaxamase) – Prevention of CDI, AAD and the emergence of antibiotic-resistant organisms:

Received approval from United States Adopted Names Council (USAN) for the generic name "ribaxamase" for SYN-004
Continued enrollment in global Phase 2b proof-of-concept clinical trial intended to evaluate the ability of ribaxamase to prevent CDI, C. difficile-associated diarrhea (CDAD) and AAD in patients hospitalized with a lower respiratory tract infection and receiving intravenous (IV) ceftriaxone
Enrolled 374 patients to date across global clinical sites; enrollment expected through 3Q 2016
Anticipate announcing topline results from Phase 2b proof-of-concept clinical trial (1Q 2017)
Announced positive results from second Phase 2a clinical trial demonstrating a correlation of the 150 mg dose of ribaxamase, both alone and in the presence of the proton pump inhibitor, esomeprazole and the successful degradation of IV ceftriaxone to levels that were near or below detectable without impacting ceftriaxone plasma concentrations
The 150 mg dose strength of ribaxamase was well tolerated by all participants
Operational Update – Expanded Leadership Team

Deb Mathews, PharmD, joined the Company as Vice President, Medical Affairs, bringing broad experience and strong leadership of clinical and medical affairs as the Company begins to implement commercialization strategies
Isaac J. Bright, MD, joined the Company in the newly created position of Vice President, Corporate Development, to lead all strategic corporate and business development efforts for the Company’s two lead microbiome-focused drug candidates
Second Quarter 2016 Financial Results

General and administrative expenses decreased by 3% to $2.1 million for the second quarter of 2016, from $2.2 million for the second quarter of 2015. This decrease is primarily the result of lower legal fees offset by an increase in stock-based compensation and increased employee costs associated with the transition of the administrative and financial office to our Maryland headquarters. The charge related to stock-based compensation expense was $507,000 for the second quarter of 2016, compared to $335,000 for the second quarter of 2015.

Research and development expenses decreased by 5% to $7.2 million for the second quarter of 2016, from $7.5 million for the second quarter of 2015. This decrease is primarily the result of decreased Phase 2 program costs associated with clinical development programs, manufacturing and research activities within our microbiome-focused pipeline. Research and development expenses also include a charge related to non-cash stock-based compensation expense of $400,000 for the second quarter of 2016, compared to $252,000 for the second quarter of 2015.

Other income was $3.5 million for the second quarter of 2016, compared to other expense of $3.9 million for the second quarter of 2015. Other income for the second quarter of 2016 is due to non-cash expense of $3.5 million from the change in fair value of warrants. The decrease in the fair value of the warrants was due to the decrease in our stock price from the year ended December 31, 2015. Non-cash expense related to the increase of fair value of warrants for the second quarter of 2015 was $3.9 million.

On August 3, 2016 NantHealth, Inc. (Nasdaq: NH), a leading next-generation, evidence-based, personalized healthcare company, reported that Sanford Health, one of the largest health systems in the nation, has entered into a commercial license agreement for the use of its eviti, the evidence-based treatment intelligence and web-based oncology decision support platform, which was recently named #1 Clinical Decision Support solution for 2016 by Black Book Market Research (Press release, NantHealth, AUG 3, 2016, View Source [SID:1234514219]).

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Under terms of the agreement, Sanford Health’s network of oncologists will have access to NantHealth’s eviti Connect decision support solution, providing them with evidence-based regimens, condition-specific clinical trial options and the potential for more efficient preauthorization and expedited reimbursement. Further, the eviti service holds the promise of delivering OMICS data for bio-specific therapy. Sanford Health, based in the Dakotas, joins over 75 percent of U.S. oncology practices who have used the eviti platform to make better clinical decisions earlier in the treatment process.

eviti Connect will be deployed to streamline system-wide processes for the prescribing of high-quality, cost-effective treatments while improving the flow of information between Sanford Health and its providers. All of the participants in the cancer care process benefit from eviti’s collaborative, real-time approach to improving care and lowering risks and costs.

In order for oncologists to serve as many cancer patients as possible, select treatment plans with the best possible outcome, and with the most responsible economic profile, the technology solution must have the rapid-learning and data compilation capabilities of true super-computing. eviti’s clinical decision support is easy to use while computing massive amounts of complex data to quickly present appropriate treatment options, including the latest peer-reviewed and proprietary immunotherapy clinical trials. eviti applications go beyond the capabilities of most traditional electronic medical records systems to present treatment options personalized for each patient’s need.

"Sanford Health Plan is committed to offering oncologists in our networks the support they need to provide the best, most advanced patient care possible," said Kirk Zimmer, Executive Vice President, Sanford Health Plan. "eviti gives physicians real-time access to the widest range of evidenced-based treatment options available, and further, to ensure that the care they provide meets the clinical requirements for reimbursement. This comprehensive approach stands to improve clinical outcomes, while reducing claims denials and potentially costs."

The eviti platform provides physicians real-time access to its comprehensive, unbiased Evidence-Based Medical Library, which encompasses over 2,700 of the most appropriate, evidence-based treatment regimens covering all cancers and cancer subtypes and all modalities. Each regimen incorporates level of evidence, expected clinical outcomes, treatment costs, toxicities and any supporting literature. This evidence-based medical library is meticulously compiled from peer-reviewed literature, oncology associations, and government agencies. The library is maintained by a full-time team of oncologists and oncology nurses, clinical informatics professionals, and an advisory board of nationally recognized oncology experts.

"We are pleased to enter into this partnership with Sanford Health and look forward to working with the organization to enable their oncologists to more quickly see and evaluate the spectrum of appropriate treatment options in order to make even better, and more informed treatment decisions," said Patrick Soon-Shiong, M.D., CEO of NantHealth. "Today, independent studies show that nearly 32 percent of oncology treatment plans deviate from evidence-based standards without medical justification. While this is understandable given the rapidly growing number of treatment options available to patients and their caregivers, we must do better. Our goal is to provide a single, reliable source of the most current information available to physicians, enabling them to provide the right care at the right time—and to immediately know whether that care will be covered."

On August 3, 2016 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported financial results for the second quarter ended June 30, 2016(Press release, NanoString Technologies, AUG 3, 2016, View Source [SID:1234514216]).

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Second Quarter Financial Highlights

Total revenue of $22.6 million, 73% year-over-year growth
Total product and service revenue of $17.5 million, 40% year-over-year growth
Consumables revenue of $10.3 million, including $1.2 million of Prosigna IVD kits, 39% year-over-year growth
Instrument revenue of $6.4 million, 46% year-over-year growth
Collaboration revenue of $5.1 million

"We had a successful second quarter characterized by strong commercial execution and rapid progress in delivering against diagnostic milestones in our biopharma collaborations," said president and chief executive officer Brad Gray. "Demand for our nCounter platform was robust across all products and geographies. Our nCounter SPRINT Profiler accounted for approximately half of units sold, driving 46% year-on-year growth in instrument revenue and underscoring the value of SPRINT in reaching new customers and expanding our installed base."

Recent Business Highlights

Grew installed base to over 410 nCounter Analysis Systems at June 30, 2016
Achieved milestones in the Merck and Medivation & Astellas collaborations valued at $13.5 million, of which $5.0 million was received in the second quarter and $8.5 million was received in the third quarter
Over 30 abstracts presented at the June 2016 annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) demonstrating the value of the nCounter platform, including several highlighting investigational assays for immuno-oncology and lymphoma
Received positive Medical Coverage Policy decisions from Aetna and Cigna for the Prosigna Breast Cancer Gene Signature Assay
Presented first proof-of-concept data for digital immunohistochemistry (IHC), a novel technology to simultaneously count multiple protein targets in the spatial context of tumor tissue biopsies, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) conference

Second Quarter Financial Results

Revenue for the three months ended June 30, 2016 increased by 73% to $22.6 million, as compared to $13.1 million for the second quarter of 2015. Instrument revenue was $6.4 million, up 46% versus the prior year period, resulting from strong demand for all products in all regions. Consumables revenue, excluding Prosigna, was $9.1 million for the second quarter of 2016, 32% higher than in the comparable 2015 quarter. Prosigna IVD kit revenue was $1.2 million for the quarter, an increase of 110% over the second quarter of 2015. Collaboration revenue totaled $5.1 million, benefiting from the achievement of multiple milestones in diagnostic development collaborations. Gross margin on product and service revenue was 55% for the second quarter of 2016, up from 53% for the second quarter of 2015.

Research and development expense increased by 52% to $8.8 million for the second quarter of 2016 versus $5.8 million for the second quarter of 2015, reflecting increased costs associated with biopharma collaborations announced earlier this year and new products and technologies under development for the life science research market. Selling, general and administrative expense was $15.5 million for the second quarter of 2016 compared to $12.8 million for the prior year period.

Net loss for the three months ended June 30, 2016 declined to $10.8 million, or a loss of $0.55 per diluted share, compared with $12.4 million, or a loss of $0.66 per diluted share, for the second quarter of 2015.

Outlook for 2016

The company raised its financial outlook for 2016 as follows:

Total revenue was increased to the range of $89 million to $93 million, as compared to the previous range of $86 million to $90 million
Gross margin on product and service revenues in the range of 54% to 55%, unchanged
Operating expenses in the range of $94 million to $99 million, unchanged
Operating loss guidance was reduced to the range of $37 million to $40 million, as compared to the previous range of $40 million to $43 million
Net loss per share range was reduced to $2.15 to $2.30, as compared to the previous range of $2.30 to $2.45
Cash from collaborations in 2016 in the range of $40 million to $45 million, unchanged

On August 3, 2016 MacroGenics, Inc. (NASDAQ:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported a corporate progress update and reported financial results for the quarter ended June 30, 2016 (Press release, MacroGenics, AUG 3, 2016, View Source [SID:1234514214]).

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"MacroGenics continues to make progress across its broad pipeline of clinical compounds, including margetuximab, our Fc-optimized anti-HER2 monoclonal antibody, our two clinical programs targeting B7-H3 as well as several bispecific product candidates based on our DART platform," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Of note during the second quarter, we presented promising Phase 1 clinical data for one of our DART programs, MGD010, at EULAR 2016. We look forward to providing data on other programs later this year, including additional clinical data from our enoblituzumab monotherapy study."

"In terms of our preclinical projects, we expect to continue our pace of generating promising clinical development candidates based on MacroGenics’ technology platforms. We remain on track to submit one IND later this year and two additional INDs in 2017," commented Dr. Koenig. "Finally, during the second quarter of 2016, we were very pleased to enter into a second collaboration with Janssen Biotech, Inc. for the development of MGD015, a DART program being developed for the treatment of various hematological malignancies and solid tumors."

Pipeline Update

Margetuximab. Recent highlights related to the Company’s Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2, or HER2, include:

SOPHIA Study: MacroGenics’ Phase 3 pivotal study in patients with HER2-positive metastatic breast cancer is ongoing, as the Company continues to initiate sites and enroll patients. This study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 relapsed/refractory patients. Approximately three-quarters of the anticipated study sites have been activated as of June 30, 2016. The Company expects to provide an update on patient enrollment and the expected timing of trial completion after all sites are activated and actively enrolling.
Phase 1b/2 Gastric Cancer Study: MacroGenics continues to recruit and dose patients in a Phase 1b/2 clinical trial of margetuximab in combination with pembrolizumab, an anti-PD-1 therapy, in patients with advanced HER2-positive gastric cancer. Treatment options for these patients are limited and this proposed combination regimen would avoid chemotherapy while exploiting the potential for enhancing the antitumor immune response. This trial is being conducted in collaboration with Merck and is currently recruiting patients in the United States, with plans to expand into Asia later this year.
B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action and take advantage of this antigen’s broad expression across multiple solid tumor types. Current ongoing clinical-stage development programs include:

Enoblituzumab: The Company continues to recruit patients in three ongoing studies of enoblituzumab, an Fc-optimized monoclonal antibody that targets B7-H3. These studies include one monotherapy study and two combination studies with each of ipilimumab and pembrolizumab. As previously reported, the monotherapy study was expanded to include additional prostate and bladder cancer cohorts.
MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types.
DART Product Candidates. There are currently six DART molecules in Phase 1 clinical development, including MGD006 (CD123 x CD3, also known as S80880), MGD007 (gpA33 x CD3), MGD009 (B7-H3 x CD3), MGD010 (CD32B x CD79B), MGD011 (CD19 x CD3, also known as JNJ-64052781) and PF-06671008 (P-cadherin x CD3). Highlights during the second quarter include:

MGD010 Clinical Data Presentation: MacroGenics presented clinical data from its Phase 1 study of MGD010 at the Annual European Congress of Rheumatology (EULAR 2016) in London, England. MGD010, a DART molecule, was designed to simultaneously target the B-cell surface proteins, CD32B and CD79B, and is being developed for the treatment of autoimmune disorders. Data from the first-in-human, double-blind, placebo-controlled study in healthy volunteers showed that MGD010 was well tolerated at all dose levels. MGD010 demonstrated linear pharmacokinetics and modulation of B-cell function without depletion.
PF-06671008 Phase 1 Study Commencement: Pfizer commenced the Phase 1 clinical study of PF-06671008, which targets P-cadherin and CD3. The dosing of the first patient in the study triggered a $2 million milestone payment to MacroGenics under the companies’ 2010 agreement.
MacroGenics expects to submit IND applications for MGA012, a monoclonal antibody, in 2016 as well as two DART molecules in 2017. These two DART molecules are:

MGD013: MacroGenics is developing MGD013 to simultaneously block two immune checkpoint molecules, PD-1 and LAG-3.
MGD014: MGD014 is a DART molecule that is being developed to eliminate latent HIV infection.
Beyond MGD013 and MGD014, MacroGenics continues to generate and evaluate multiple other candidates that target a range of immune regulatory and other molecules using its proprietary platforms.

Second Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2016, were $265.6 million, compared to $339.0 million as of December 31, 2015. The MGD015 collaboration agreement with Janssen closed during the second quarter of 2016; however, the $75 million upfront payment from them was not received until early in the third quarter.
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $80.7 million for the quarter ended June 30, 2016, compared to $6.7 million for the quarter ended June 30, 2015. This increase is primarily due to the $75 million in revenue recognized under the Janssen MGD015 collaboration announced in the second quarter of 2016 and the $2 million milestone received from Pfizer.
R&D Expenses: Research and development expenses were $33.3 million for the quarter ended June 30, 2016, compared to $22.7 million for the quarter ended June 30, 2015. This increase was due primarily to increased activity in the Company’s preclinical immune checkpoint programs, including MGD013, the initiation of a Phase 1 clinical trial of MGD009 and the initiation of two Phase 1 clinical trials combining enoblituzumab with other compounds.
G&A Expenses: General and administrative expenses were $7.2 million for the quarter ended June 30, 2016, compared to $5.3 million for the quarter ended June 30, 2015. This increase is primarily due to increased professional fees, recruiting costs and stock-based compensation expense.
Net Income/Loss: Net income was $40.5 million for the quarter ended June 30, 2016, compared to net loss of $21.4 million for the quarter ended June 30, 2015. The change from net loss to net income was primarily due to the recognition of the $75 million upfront payment from Janssen.
Shares Outstanding: Shares outstanding as of June 30, 2016 were 34,694,039.