On November 3, 2015 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) reported unaudited consolidated financial results for the third quarter and year-to-date periods ended September 30, 2015 (Filing, 8-K, AMAG Pharmaceuticals, NOV 3, 2015, View Source [SID:1234507909]). Total revenues for the third quarter of 2015 increased to $96.2 million, compared with $25.5 million in the third quarter of 2014. This increase is primarily related to the additions of Makena (hydroxyprogesterone caproate injection) in November 2014 and Cord Blood Registry (CBR) in August 2015 to the company’s portfolio, which contributed revenue of $65.2 million and $7.2 million, respectively, to the third quarter 2015 results. After certain non-cash and one-time costs related to business development activities and acquisitions, the company reported an operating loss of $1.4 million in the third quarter of 2015, compared with operating income of $4.3 million in the same period last year. This resulted in a net loss of $20.6 million, or $0.62 per basic and diluted share, compared with net income of $1.5 million, or $0.07 per basic share and $0.06 per diluted share for the same period last year.

Non-GAAP revenue totaled $103.5 million in the third quarter of 2015, compared with $23.5 million for the same period last year.(1) The company reported adjusted EBITDA of $52.8 million in the third quarter of 2015, compared with adjusted EBITDA of $1.3 million in the third quarter of 2014.(1) Non-GAAP cash earnings for the third quarter of 2015 totaled $42.3 million, or $1.02 per diluted share, compared with $0.3 million, or $0.01 per diluted share, for the same period in 2014.(1),(2)

"While our overall portfolio experienced growth during the third quarter, we believe there is opportunity for even better performance in the future as integration activities are completed and our investments in the next generation development programs for Makena and an expanded label for Feraheme progress toward FDA approvals and launches," said William Heiden, AMAG’s chief executive officer. "With our business continuing to generate strong cash flows, we will maintain our focus on executing on the opportunities we have today, line extension approvals in the future, as well as continued portfolio expansion through acquisitions and licensing transactions."

3Q15 Business Highlights and Recent Developments

· Makena net sales totaled $65.2 million in the third quarter of 2015, representing 36% growth over the corresponding period in the prior year.(3) The growth in sales was driven by a 42% increase in volume partially offset by a decrease in net revenue per injection due to higher growth in the Medicaid versus commercial segment.

· Sales of Feraheme (ferumoxytol) injection returned to growth, increasing 3% to $23.2 million in the third quarter of 2015, compared with $22.5 million in the same period last year. Sales of MuGard grew 16% in the third quarter of 2015 over the same period last year.

· The company closed on the acquisition and financing of CBR, the world’s largest stem cell collection and storage company, on August 17, 2015. The CBR business recorded $14.5 million of non-GAAP revenue for the six-week period between closing and September 30, 2015.(1) On a pro forma basis (as if AMAG had acquired CBR at the beginning of the third quarter of 2015), total CBR revenue was $29.3 million, compared with $33.0 million in the third quarter of 2014, with the decrease being partially attributable to new customer discounts in the third quarter of 2015.(4)

· During the third quarter of 2015, the company announced plans to further expand its growing maternal health portfolio by entering into an exclusive option agreement with Velo Bio, LLC, a privately held life sciences company. The agreement gives the company the option to acquire the global rights to an orphan drug candidate in clinical development for use in the treatment of severe preeclampsia, an area of high unmet need in pregnant women.

· The Company recently announced start-up activities for a head-to-head, Phase 3 clinical trial evaluating Feraheme in adult patients with iron deficiency anemia (IDA). The study marks a step forward on the pathway to potentially broaden the use of Feraheme beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment. The company expects to begin enrolling patients in the trial in the first quarter of 2016, with potential approval of a supplemental new drug application for a broader IDA indication and launch in 2018.

· The company advanced its next generation development programs for Makena seeking to enhance the product for patients and their healthcare providers. As previously disclosed, the company has filed for approval of a single-dose, preservative-free version of Makena, with anticipated approval in the fourth quarter of 2015. In addition, the company is developing a device for subcutaneous administration of Makena by an auto-injector. The company has an exclusive agreement in place with a device partner whose technologies are covered by multiple issued patents, and has other approved products in its portfolio. AMAG is also developing a longer-acting formulation of Makena.

Third Quarter Ended September 30, 2015 (unaudited)

Financial Results (GAAP Basis)

Total revenues for the third quarter of 2015 were $96.2 million, compared with $25.5 million for the same period in 2014. Net product sales of Makena, Feraheme and MuGard totaled $88.9 million in the third quarter of

(3) Based on unaudited pro forma sales for the third quarter of 2014. Acquisition of Lumara Health closed in November 2014.
(4) Based on unaudited pro forma sales for the third quarter of 2015 and 2014. Acquisition of CBR closed on August 17, 2015.

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2015, compared with $23.0 million in the third quarter of 2014. Service revenue from CBR totaled $7.2 million in the third quarter of 2015.

Total costs and expenses for the third quarter of 2015 were $97.5 million, compared with $21.2 million for the same period in 2014. The increases in costs and expenses were primarily due to the following: (i) higher costs associated with managing the company’s expanded portfolio and infrastructure following the acquisitions of Lumara Health in November 2014 and CBR in August 2015, (ii) increased cost of goods sold from new product and higher sales volumes for each of the company’s products, (iii) non-cash charges associated with inventory step-up and amortization related to the acquisitions, (iv) a $10.0 million upfront payment for the exclusive option to acquire rights to a development stage asset to treat severe preeclampsia, and (v) $9.2 million in acquisition and restructuring costs related to CBR.

The company reported an operating loss of $1.4 million and a net loss of $20.6 million, or $0.62 per basic and diluted share, for the third quarter of 2015, compared with operating income of $4.3 million and net income of $1.5 million, or $0.07 per basic share and $0.06 per diluted share, for the same period in 2014.

Financial Results (Non-GAAP Basis)(1),(2)

Non-GAAP revenues totaled $103.5 million, up from $23.5 million in the third quarter of 2014. Non-GAAP CBR revenue totaled $14.5 million in the third quarter of 2015. The difference between GAAP and non-GAAP revenue for CBR represents purchase accounting adjustments related to deferred revenue.

Total costs and expenses on a non-GAAP basis totaled $50.6 million resulting in a gross margin of 93% and adjusted EBITDA margin of 51% for the third quarter of 2015. This compares to costs and expenses of $22.3 million in the same period of 2014, which resulted in a gross margin of 90% and adjusted EBITDA margin of 5%. Non-GAAP adjusted EBITDA for the third quarter of 2015 was $52.8 million, compared with $1.3 million for the same period in 2014.

After deducting cash interest expense, the company generated third quarter non-GAAP cash earnings of $42.3 million, or $1.27 per non-GAAP basic share and $1.02 per non-GAAP diluted share. The weighted average diluted shares used in calculating the non-GAAP cash earnings per diluted share for the third quarter of 2015 includes the impact of the convertible debt and related bond hedge and warrants.

Balance Sheet Highlights

As of September 30, 2015, the company’s cash and investments totaled approximately $442.9 million and total debt (face value) was $1.05 billion.

"The two transformative acquisitions that we completed in the past twelve months have allowed us to significantly grow our top line while delivering strong cash flows and profitability for our shareholders," said Frank Thomas, AMAG’s president and chief operating officer. "As we continue to integrate the businesses, we are anticipating even better financial and operating performance, allowing us to serve our patients more effectively and efficiently."

Nine Months Ended September 30, 2015 (unaudited)

Financial Results (GAAP Basis)

Total revenues for the nine months ended September 30, 2015 were $309.5 million, compared with $71.1 million for the same period in 2014. This increase is primarily related to the addition of Makena in November 2014 and CBR in August 2015, which contributed $184.3 million and $7.2 million, respectively, in product revenue to the year-to-date 2015 results. In addition, the company recognized $39.2 million of collaboration revenue in 2015 related to the termination of the company’s ex-US ferumoxytol marketing agreement with Takeda Pharmaceutical Company Limited.

Net income for the first nine months of 2015 totaled $25.6 million, compared with a net loss of $7.2 million for same period in 2014. Basic net income per share was $0.84, compared with a net loss per basic share of $0.33 in 2014. Diluted net income per share was $0.73 in 2015, compared with a net loss per diluted share of $0.33 in 2014. The weighted average diluted shares used in calculating diluted net income per share for the first nine months of 2015 followed the if-converted method of accounting for the convertible debt.

Financial Results (Non-GAAP Basis)(1),(2)

Non-GAAP adjusted EBITDA for the nine months ended September 30, 2015 was $152.1 million, compared with a loss before interest, taxes, depreciation and amortization of $0.8 million for the same period in 2014. After deducting cash interest expense, the company generated non-GAAP cash earnings of $127.2 million, or $3.31 per non-GAAP diluted share.

Updating 2015 Financial Outlook(6)

The company is updating its full year 2015 guidance to include revenue from CBR and reflect the business performance for the first nine months and the outlook for the remainder of 2015.

On November 3, 2015 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biotechnology company developing engineered monoclonal antibodies for the treatment of autoimmune diseases, asthma and allergic disease, and cancer, reported financial results for the third quarter ended September 30, 2015 and provided a review of recent business highlights (Filing, 8-K, Xencor, NOV 3, 2015, View Source [SID:1234507935]).

"The advancements made this quarter for both our internal and partnered XmAb programs represent progress across the full breadth of our Fc engineering technology. For our XmAb bispecific antibody platform, we are working toward the initiation of clinical trials for XmAb14045 and XmAb13676, which are planned for the first half and second half of 2016, respectively and in September we announced our research collaboration with Amgen to apply our bispecific platform to their antibodies," said Bassil Dahiyat, Ph.D., president and chief executive officer of Xencor. "In the months ahead, we plan to initiate clinical testing of XmAb5871 in IgG4-Related Disease (IgG4-RD) and report full data results from our ongoing Phase 1a trial of XmAb7195 in the first half of 2016. We are on strong financial footing, with sufficient cash to advance development of our clinical programs and platform through 2019."

Business Highlights

XmAb 5871: A first-in-class monoclonal antibody that targets CD19 with its variable domain and that uses Xencor’s proprietary XmAb immune inhibitory Fc domain to target FcγRIIb, a receptor that inhibits B-cell function.

· Xencor plans to file an investigational new drug (IND) application this year and initiate enrollment in a Phase 2, open-label, pilot study of XmAb5871 in IgG4-RD in early 2016. The trial, designed to assess control of disease activity, will enroll approximately 15 subjects for up to 24 weeks and will utilize the IgG4-RD Responder Index to measure treatment activity (Carruthers 2012, International Journal of Rheumatology). Xencor expects to report preliminary data by the end of 2016.

· Xencor plans in 2016 to initiate clinical development of XmAb5871 in an additional autoimmune disease and initiate a bioequivalence trial with a subcutaneous formulation.

XmAb 7195: A first in class monoclonal antibody that targets IgE with its variable domain and uses Xencor’s XmAb immune inhibitor Fc domain to target FcγRIIb, resulting in three distinct mechanisms of action for reducing IgE levels.

· Xencor plans to report complete IgE reduction and safety data from the ongoing Phase 1a trial of XmAb7195 for the treatment of asthma in the first half of 2016.

· Xencor plans to initiate a Phase 1 trial with a subcutaneous formulation of XmAb7195 in 2016.

Internal Bispecific Oncology Pipeline: Xencor’s initial bispecific programs are tumor-targeted antibodies that contain both a tumor antigen binding domain and a cytotoxic T cell binding domain (CD3). These bispecific antibodies activate T cells for highly potent and targeted killing of malignant cells. Their XmAb Fc domains confer long circulating half-lives, stability and ease of manufacture.

· Xencor remains on track to initiate clinical trials for its first two bispecific oncology candidates, XmAb14045, for the treatment of acute myeloid leukemia, and XmAb13676, for the treatment of B-cell malignancies, in the first and second half of 2016, respectively.

· Xencor plans to start clinical trials for additional bispecific oncology candidates in 2017.

Partnered XmAb Programs

· In September 2015, Xencor and Amgen entered into a research and license agreement to develop and commercialize five bispecific molecules based on Amgen antibodies against predefined targets, and Xencor’s preclinical T cell engager program directed at CD38 and CD3 for multiple myeloma. Xencor received a $45 million upfront payment, and is eligible to receive up to $1.7 billion in clinical, regulatory and sales milestone payments in total and royalties on sales.

· In September 2015, Xencor reported that its partner, CSL Limited, through its licensee Janssen Biotech Inc., initiated a Phase 2 clinical trial of CSL362 (now called JNJ-56022473), which uses Xencor’s XmAb Cyotoxic Fc Domain, for the potential treatment of patients with acute myeloid leukemia (AML). The trial initiation triggered a milestone payment to Xencor.

Third Quarter Ended September 30, 2015 Financial Results

Cash equivalents and marketable securities totaled $197.6 million as of September 30, 2015, compared to $54.7 million on December 31, 2014. The increase reflects the net proceeds of $115.0 million received from completion of Xencor’s follow-on financing in the first quarter and net proceeds from partners and collaborators during the first three quarters of 2015 including an upfront payment of $45.0 million received from Amgen in the third quarter.

Revenues for the third quarter ended September 30, 2015 were $3.5 million, compared to $0.8 for the same period in 2014. Revenues for the nine months ended September 30, 2015 were $6.0 million, compared to $3.9 million for the same period in 2014. Revenues in the three and nine month period ended September 30, 2015 were earned primarily from the Company’s Novo Nordisk and Alexion collaborations and also reflect a milestone payment received from our CSL collaboration, compared to revenue for the same periods in 2014, which was primarily earned from Xencor’s 2010 Amgen collaboration which terminated in the fourth quarter of 2014.

Research and development expenditures for the third quarter ended September 30, 2015 were $10.6 million, compared to $5.0 million for the same period in 2014. Total research and development expenses for the nine months ended September 30, 2015 were $23.3 million, compared to $13.5 million for the same period in 2014. The increased research and development spending for the three and nine months ended September 30, 2015 over the same periods in 2014 is primarily due to increased spending on Xencor’s bispecific technology and development pipeline, including its initial bispecific oncology candidates, XmAb14045 and XmAb13676.

General and administrative expenses in the third quarter ended September 30, 2015 were $3.2 million, compared to $2.2 million for the same period in 2014. Total general and administrative expenses for the first nine months of 2015 were $8.5 million compared to $5.5 million for the same period in 2014. The increased spending in the general and administrative area is due to increased staffing in Xencor’s legal and accounting departments and additional spending in professional fees for legal and business development activities.

Non-cash, share-based compensation expense for the first nine months of 2015 was $3.4 million compared to $1.1 million in the first nine months of 2014.

Net loss for the third quarter ended September 30, 2015 was $10.0 million, or $(0.25) on a fully diluted per share basis, compared to a net loss of $6.3 million or $(0.20) on a fully diluted per share basis, for the same period in 2014. For the nine months ended September 30, 2015, net loss was $25.3 million, or $(0.66) on a fully diluted per share basis, compared to a net loss of $15.1 million, or $(0.48) on a fully diluted per share basis for the same period in 2014. The increased loss for the three and nine months ended September 30, 2015 over the same periods in 2014 is due to increased spending in both the research and development and general and administrative areas and the increase in stock based compensation charges.

The total shares outstanding as of September 30, 2015 was 40,477,003, which reflects the additional 8,625,000 shares issued in the Company’s follow-on financing in the first quarter of 2015.

Based on current operating plans, Xencor expects to have sufficient cash to fund research and development programs and operations through 2019.

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On November 03, 2015 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company at the forefront of developing and commercializing novel immuno-oncology product candidates, including both cellular immunotherapy and checkpoint inhibitor platforms, to improve the lives of patients with cancer, reported consolidated financial results for the third quarter of 2015 and progress in its proprietary and partnered clinical development programs (Press release, NewLink Genetics, NOV 3, 2015, View Source [SID:1234507912]).

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"Our dynamic and experienced team has made significant progress in driving our broad clinical development programs," said Charles Link, M.D., Chairman and Chief Executive Officer. "We look forward to reporting on our strong pipeline of clinical data over the coming year."

Dr. Link added, "GDC-0919, the checkpoint inhibitor program partnered with Genentech continues to make great progress. Both companies are putting substantial resources behind this program, and we continue to be excited about the development plan and the progress of our collaboration."

"As we anticipate the data readout for the IMPRESS trial in 2016, NewLink Genetics continues to advance its investment in manufacturing and pre-commercial activities relating to algenpantucel-L, our HyperAcute Cellular Immunotherapy product candidate for patients with resected pancreatic cancer," said Nicholas Vahanian, M.D., President and Chief Medical Officer. "We await the promise of our cellular immunotherapy agents to educate the immune system to destroy tumor cells in pancreatic and other cancers."

Financial Results for the Three-Month Period Ended September 30, 2015

Cash Position: NewLink Genetics ended the quarter on September 30, 2015, with cash, cash equivalents, and certificates of deposit totaling $200.4 million, compared to $202.8 million for the year ending December 31, 2014 .

R&D Expenses: Research and development expenses in the third quarter of 2015 were $22.5 million, compared to $10.9 million during the comparable period in 2014. The increase is primarily due to clinical trial expenses related to NewLink Genetics’ broad pipeline of product candidates, as well as expenses for manufacturing and research related to the Ebola vaccine candidate. The majority of the Ebola-related expenses are subject to reimbursement under government contracts.

G&A Expenses: General and administrative expenses in the third quarter of 2015 were $7.4 million, compared to $4.9 million during the comparable period in 2014. The increase was primarily due to an increase in share-based compensation expense, consulting and legal fees, and medical affairs and pre-commercial activities.

Net Loss: NewLink Genetics reported a net loss of $15.9 million, or ($0.55) per diluted share, for the third quarter of 2015, compared to a net loss of $5.6 million, or ($0.20) per diluted share, for the comparable period in 2014.

NewLink Genetics ended the quarter with 28,774,911 shares outstanding.

Financial Guidance

NewLink Genetics expects to have more than $160 million in cash and equivalents on December 31, 2015.

Conference Call and Program Updates:

The Company has scheduled a conference call for 8:30 a.m. ET today to discuss these results and to provide an update on clinical and business development activities. Dial-in information for the conference call is set forth at the end of this press release. Programs to be discussed include:

IDO Checkpoint Inhibitor Programs

NewLink Genetics entered into an exclusive worldwide license and collaboration agreement with Genentech, a member of the Roche Group, for the development of the IDO checkpoint inhibitor GDC-0919 and an expanded pipeline of potential IDO/TDO inhibitor candidates in 2014. This product candidate is currently in Phase 1 clinical development for patients with advanced solid tumors.

Key preclinical data is being presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in Bethesda, Maryland on November 6, 2015.

Phase 1 data on GDC-0919 was presented at the ECC/ESMO meeting in Vienna. Key preliminary data showed that GDC-0919 had a favorable safety profile and preliminary evidence of disease stabilization and peripheral pharmacodynamic modulation. Details from the poster presentation are available in the press release found at View Source
The collaboration’s clinical development team has advanced GDC-0919 into a combination study with atezolizumab that is actively enrolling patients. In addition, the collaboration is planning combination studies of GDC-0919 with OX-40 agonists.
NewLink will be eligible to receive in excess of $1 billion in milestone payments based on achievement of certain predetermined milestones as well as escalating double-digit royalties on potential commercial sales of multiple products by Genentech.
Indoximod, NewLink Genetics’ proprietary IDO pathway inhibitor, is in multiple Phase 1 and Phase 2 clinical trials for patients with breast, prostate, pancreatic and brain cancers as well as melanoma. We will provide additional details about our trials on the conference call. Additionally, there will be multiple opportunities for the Company to provide further updates during 2015 and 2016 at academic meetings and associated programs.

HyperAcute Cellular Immunotherapy Programs

NewLink Genetics’ proprietary HyperAcute Cellular Immunotherapy programs may prove to have broad potential for patients across a spectrum of cancer indications, including use in combination with checkpoint inhibitors.

Algenpantucel-L is NewLink Genetics’ HyperAcute Cellular Immunotherapy product candidate for patients with pancreatic cancer. The product is currently being studied in a Phase 3 clinical trial called IMPRESS, or IMmunotherapy for Pancreatic RESectable Cancer Study, in patients with surgically resected pancreatic cancer. The study is powered to show an improvement in overall survival after 442 events, and we continue to expect that final results will be reported in 2016.

PILLAR, or Pancreatic Immunotherapy with Algenpantucel-L for Locally Advanced Non-Resectable Disease, is our Phase 3 clinical trial studying the efficacy of algenpantucel-L for patients with borderline resectable or locally advanced unresectable pancreatic cancer. We expect to complete enrollment in this study in 2015.

Tergenpumatucel-L

Tergenpumatucel-L, NewLink Genetics’ HyperAcute Cellular Immunotherapy product candidate for patients with non-small cell lung cancer (NSCLC), remains in Phase 2. We are eager to learn more about this product in our recently begun trial evaluating tergenpumatucel-L in combination with indoximod and chemotherapy for patients with advanced NSCLC.

Dorgenmeltucel-L

Dorgenmeltucel-L, NewLink Genetics’ HyperAcute Cellular Immunotherapy product candidate for patients with melanoma, continues in a trial evaluating efficacy in combination with the checkpoint inhibitors ipilimumab, nivolumab, and pembrolizumab for patients with advanced melanoma.

Ebola Vaccine

During the third quarter, we announced that NewLink Genetics was awarded an $8.1 million base contract with future options totaling $5.2 million by the Defense Threat Reduction Agency of the United States Department of Defense to support various development activities of the investigational rVSV-ZEBOV (Ebola) vaccine candidate. Additionally, the Biomedical Advanced Research and Development Authority of the United States Department of Health and Human Services exercised an $18 million option on NewLink Genetics’ existing contract.

NewLink has exclusively licensed research, development, manufacturing and commercialization of the rVSV-ZEBOV (Ebola) vaccine to Merck. This vaccine candidate was originally developed by the Public Health Agency of Canada (PHAC).

40 potential new medicines and vaccines offer significant opportunity to drive long-term performance and deliver new benefits to patients and consumers

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At a presentation to investors in New York today, GSK described a deep portfolio of innovation, focussed across six core areas of scientific research and development: HIV & Infectious diseases, Oncology, Immuno-Inflammation, Vaccines, Respiratory and Rare Diseases (Press release, GlaxoSmithKline, NOV 3, 2015, View Source [SID:1234511948]). Around 40 new potential medicines and vaccines were profiled, supporting the Group’s outlook for growth in the period 2016-2020 and the significant opportunity the Group has to create value beyond 2020.

The portfolio represents some of the latest scientific achievements from GSK’s R&D organisation and its more than 1,500 company and academic collaborations. The company believes approximately 80% of the medicines and vaccines presented have the potential to be "first-in-class" with novel mechanisms of action. As a result, many of these potential medicines and vaccines may offer benefits beyond current standards of care and, in some cases, could radically transform how patients are treated.

In developing this portfolio, the company’s scientists have focussed on:

Targeting immune mechanisms that could alter the fundamental course of diseases, modifying disease progression and presenting opportunities to achieve remission and functional cures.
Developing simplified treatment regimens and a new generation of long-acting medicines to provide long-term control and improve treatment outcomes for patients.
Using next generation technology platforms to increase understanding of fundamental disease mechanisms, to develop new approaches to disease management and control.

At the event, notable advances within the portfolio were outlined, including potential:

Leading-edge molecules in the field of epigenetics and immuno-oncology for the treatment of cancer;
The next generation of respiratory medicines beyond inhaled treatments;
A portfolio of new antibodies for inflammatory diseases including rheumatoid arthritis, autoimmune diseases and osteoarthritis;
New options for long-term control and prevention of HIV;
Opportunities designed to cure or induce long-term remission in both Hepatitis B and C;
Breakthrough cell and gene therapies for treatment of rare diseases;
A novel maternal immunisation platform for vaccines.

GSK also profiled a number of significant material opportunities in late-stage development, including: Nucala (mepolizumab)* for treatment of severe eosinophilic asthma, Shingrix (zoster)*, a candidate vaccine for the prevention of shingles, sirukumab for the treatment of rheumatoid arthritis, daprodustat for anaemia, cabotegravir for HIV, a candidate combination vaccine for the prevention of bacterial meningitis and a new inhaled triple therapy for treatment of COPD.

In total GSK has the potential to file up to 20 assets with regulators before 2020. Seven of these assets are in advanced late-stage development (with the potential to launch before 2020) with the remainder, being in earlier development, notably in the areas of oncology, immuno-inflammation and respiratory disease. In 2016/2017 GSK has the potential to start phase II development of ~30 new molecular entities (NMEs) and product line extensions (PLEs) and to start phase III development of ~20 NMEs and PLEs.

During the period 2021-2025, GSK has the potential to file up to 20 additional innovative assets, now in clinical development.

Commenting on the event, Sir Andrew Witty, CEO GSK said: "Earlier this year we set out our expectations for the Group to generate sustained sales and earnings performance over the next 5 years. With the recent transaction, we have significantly strengthened our Vaccines and Consumer Healthcare businesses.

"Today, we have profiled around 40 innovative potential new medicines and vaccines which will support future growth in our Pharmaceuticals and Vaccines businesses. Several of these assets are in advanced late-stage development and, for the first time, we have also outlined the scale of new opportunities GSK has in earlier stages of development, notably in areas such as oncology and immuno-inflammation.

"The level of innovation in this portfolio is substantial. We believe this is critical in today’s operating environment as payors look to balance pressures of pricing and demand. It also provides us with confidence that this portfolio can generate significant value for shareholders and deliver widespread benefits to patients and consumers."
HIV and infectious diseases

The burden from infectious diseases continues to grow, presenting significant public health challenges. GSK’s leadership in HIV began with the development of the world’s first breakthrough medicine for HIV patients, Retrovir (zidovudine), in the 1980s. Successful development continues as demonstrated by the recent launches of new dolutegravir-based products, Tivicay (dolutegravir) and Triumeq (dolutegravir/abacavir/lamivudine). Dolutegravir was discovered through a collaboration between GSK and Shionogi. The next stage of development for dolutegravir is investigating its potential as a two-drug regimen. A phase III study is ongoing as part of a collaboration with Janssen, to investigate dolutegravir in combination with rilpivirine, as a potential maintenance therapy for adult patients with HIV who have already achieved viral suppression with a three drug regimen.

GSK is exploring new therapies for patients that could potentially enable long-term HIV control through infrequent dosing. The long-acting integrase strand inhibitor, cabotegravir, is at the forefront of this work and is currently in phase II development. Clinical data supporting the progression of cabotegravir development for both treatment and prevention of HIV was presented and included a positive headline data readout from the LATTE2 trial. Data from this phase IIb trial is expected to be presented at a scientific conference in 2016. Cabotegravir is expected to enter phase III development in 2016.

In a collaboration, the details of which will be announced later this week, GSK will work with the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, to optimise and develop broadly neutralising antibodies (bnAbs), to recognise their potential to enable infrequent dosing in the long acting treatment and prevention of HIV.

Through its collaborations, and by applying the latest scientific breakthroughs, GSK is aggressively pursuing research programmes focused on curing patients with other infectious diseases.

A new collaboration with Regulus Therapeutics will undertake a clinical combination study investigating the potential of GSK’s NS5B polymerase inhibitor, 2878175, currently in phase I development, and Regulus’ miR-122 antagonist, RG-101, to offer a single treatment cure for hepatitis C. The company’s collaboration with Isis Pharmaceuticals, which began in 2010 to develop new therapies using antisense technology, is also exploring use of the antisense oligonucleotide, GSK3228836, as a functional cure/long term remission for hepatitis B, with a phase II study planned for 2016.

GSK began its research into antibiotics over 40 years ago and, while the number of large pharmaceutical companies involved in this area has reduced in recent years, a dedicated research team at GSK continues to focus on discovering the next generation of medicines to treat bacterial infections. The company’s topoisomerase inhibitor, gepotidacin (GSK2140944), has a novel mechanism of action and the potential to address multiple indications. It has been developed in collaboration with BARDA and DTRA. The asset is currently in phase II development with a phase III study planned to begin in 2016.
Oncology

GSK has focussed its oncology discovery efforts to target the fundamental drivers of cancer, exploring new technologies and approaches to stimulate anti-tumour immunity, reprogram cancer cells and improve long-term survival. Development timelines for oncology drugs can be compressed, which offers potential for several of these assets to be filed with regulators in the next 3 to 5 years.

Epigenetics, the ‘control system’ that helps regulate the DNA of cells and determines cell function – including the initiation and progression of cancer – holds significant potential for future cancer therapies. GSK made a significant research commitment to the field of epigenetics in 2008 and has a number of strategic biotech and world-leading academic collaborations.

GSK has an industry-leading epigenetics pipeline including a potential first in class BET inhibitor, GSK525762 – currently in phase I clinical development – which has the potential to treat many indications including solid tumours and heme malignancies. GSK2879552, an LSD1 inhibitor, is also in ongoing phase I clinical studies to treat small cell lung cancer (SCLC) and acute myeloid leukaemia. The phase I studies have shown an early signal of significant progression-free survival for some patients with SCLC.

GSK also has a pipeline of potential next generation immuno-oncology therapies to stimulate anti-tumour immunity in patients. Its collaboration with Adaptimmune, is exploring use of GSK 3377794, a T-cell receptor (TCR) therapy in phase I/II development across multiple indications including sarcoma, myeloma, NSCLC, melanoma and ovarian cancer.

Monoclonal antibody GSK3174998, an OX40 agonist antibody being developed in collaboration with MD Anderson, is one of four OX-40s currently in development across the industry. GSK has begun a development programme in eight solid tumours and heme malignancies, and announced today that in 2016 a study will commence exploring the asset’s potential for use in combination with Merck’s anti-PD-1 therapy, pembrolizumab, in solid tumours.

A first in class ICOS agonist antibody, GSK3359609, being developed in collaboration with INSERM, is focused on enhancing patients’ anti-tumour T-cell response and is expected to enter the clinic in Q1 2016, providing a potential universal mechanism across multiple cancers either alone or in combination treatments.

Targeting the key biologic pathways thought to control cancer stem cells is also a key area for the company’s oncology research. Tarextumab, being developed in collaboration with OncoMed, is a first-in-class anti-cancer stem cell therapy in phase II development for the treatment of pancreatic and small cell lung cancer.
Immuno-inflammation

GSK’s growth of research in this area, and the multiple opportunities being explored, reflect the company’s progress in understanding the underlying cause of immune-related disease and the potential for broad therapeutic utility from single pathway interventions.

The company today highlighted a broad portfolio of innovative immune-modulating therapies in clinical development, focused on potentially altering the course of disease and inducing sustainable remission.

GSK3196165, a granulocyte macrophage colony-stimulating factor (GMCSF) antibody in-licensed from MorphoSys AG and in phase II development in rheumatoid arthritis (RA), has shown a good magnitude of effect with a fast onset of action in this indication and further potential for early use to induce remission. Understanding from this programme has also unlocked a clinical development path for disease modification and analgesic activity in hand osteoarthritis (HOA). An expedited phase II trial in this indication is anticipated in 2016.

GSK also profiled a portfolio of potential first in class antibodies for inflammatory diseases, with four assets already in the clinic and set to enter phase II in 2016: GSK2618960, an anti-IL-7R antibody for Sjögren’s syndrome; GSK3050002, an anti-CCL20 antibody for psoriatic arthritis in collaboration with Morphotek/ Eisai; GSK2831781, a cell depleting anti-LAG3 antibody for T-cell driven immuno-inflammation indications, and GSK2330811, an anti-OSM antibody for systemic sclerosis.

RIP1 kinase inhibitor, GSK2982772, is a novel class oral therapeutic with phase I and preclinical data that support the potential for this drug to have activity in multiple potential indications. Phase II studies in RA, ulcerative colitis and psoriasis will progress in parallel in 2016.

The company also profiled two late-stage assets in this therapy area. Sirukumab is an anti-IL-6 antibody currently in phase III development with Janssen Biologics to treat rheumatoid arthritis and with potential for further GSK development programmes in giant cell arteritis and asthma.

When intravenous Benlysta (belimumab) was approved in 2011 it was the first treatment for systemic lupus erythematosis (SLE) in 50 years and has established itself as a key therapy option. New data presented today from a 3rd consecutive successful pivotal study show efficacy in a subcutaneous formulation of belimumab, which has potential to help patients manage their disease. Filing for this subcutaneous formulation is planned for Q4 2015/ Q1 2016.
Metabolic

Daprodustat (GSK1278863), a low dose prolyl hydroxylase inhibitor (PHI) in phase II development for the treatment of anaemia in patients with chronic kidney disease, would be an oral tablet to potentially replace the injectable current standard of care (rhEPO), and has potential for improved cardiovascular safety. A phase III study in this indication is expected to begin in 2016 and further development programmes are in phase I for the treatment of diabetic foot ulcer and in muscle injury.
Vaccines

The company’s leadership in vaccines R&D is reflected through its short, mid and long term clinical development programmes.

Shingrix (zoster), GSK’s candidate shingles vaccine, represents a significant advance in vaccination to help prevent shingles, displaying high and potentially lasting efficacy across all age groups from 50 to above 80 years old. Global filings are expected in 2H 2016.

The company has the broadest portfolio of approved and candidate meningococcal meningitis vaccines. This includes its commercialised Menveo (MenACWY) tetravalent and Bexsero (MenB) vaccines and a full pentavalent combination candidate vaccine, MenABCWY, which may become the optimal option for disease prevention and is currently in phase II development, with phase III planned for 2017.

The development of a vaccine against Respiratory syncytial virus(RSV) is a key public health priority. RSV is a common cause of bronchiolitis and pneumonia in infants and can lead to hospitalisation and an enhanced risk of severe asthma. No vaccine is currently available. GSK has two novel approaches to RSV vaccination in phase II clinical development: a paediatric RSV vaccine that uses a genetically engineered recombinant chimpanzee adenovirus (CHAd155) – the same vector that is used in GSK’s Ebola vaccine candidate; and a recombinant glycoprotein maternal RSV vaccine that, given to pregnant women, may provide infants with protective maternally-derived RSV neutralising antibodies.

Maternal immunisation is now a clinically validated strategy to prevent diseases that afflict very young infants in the first weeks of life. In addition to RSV, GSK is further advancing its new maternal immunisation vaccines portfolio with a vaccine candidate to prevent Group B Strep (GBS), a leading cause of pneumonia, meningitis and sepsis in newborns.Beyond GBS and RSV, GSK is also considering this approach for the prevention of pertussis and influenza diseases using its currently available vaccines, thereby building potentially the most comprehensive maternal immunisation vaccines portfolio in development.

Epidemiological studies show an association between some bacterial infections in the lung and exacerbation episodes in COPD patients. GSK is investigating a candidate vaccine concept currently in a phase II clinical proof of concept study, for the prevention of exacerbations in COPD patients.
Respiratory

Following the recent launches of Relvar/Breo Ellipta, Anoro Ellipta, Arnuity Ellipta and IncruseEllipta, GSK’s commitment to developing the most innovative inhaled respiratory medicines continues through the ongoing phase III development with Theravance of the unique once-daily closed triple combination in the Ellipta device of fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) for patients with chronic obstructive pulmonary disease (COPD). Filings are expected in 2016 (EU) and 2018 (USA).

Building on its heritage as a leader in respiratory research, the company today unveiled a next-generation of treatments for respiratory disease, beyond the current approach with inhaled medicines.

While current options for the treatment of mild to moderate asthma enable patients to achieve good control of their symptoms, there remains significant unmet need in severe patients. GSK’s diverse portfolio of targeted and extended-duration biologicals offer the potential to alter the fundamental course of disease, with Nucala (mepolizumab), its subcutaneous anti-IL-5 mAb, leading the portfolio – a first in class medicine with a strong profile, significantly reducing exacerbations in patients with severe eosinophilic asthma. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending marketing authorisation for Nucala on 24 September 2015. An FDA decision is expected on 4 November 2015. Following Nucala, other assets in the asthma biologic pipeline include sirukumab, which is expected to enter phase II in 2016, a long-acting anti-IL-5 mAb, expected to begin phase I/II studies in 2017 and an inhaled anti-TSLP domain antibody (dAb) expected to enter the clinic in 2016.

GSK2245035, an intranasal TLR7 agonist in phase II development, is supported by clinical data demonstrating prolonged suppression of allergic response and reaffirms GSK’s continued innovation into allergic asthma, exploring the potential to achieve disease remission.

By targeting the underlying drivers of disease, two novel assets offer the potential to delay or halt progression of COPD, a disease that affects 329 million people worldwide and is expected to become the 3rd leading cause of death by 2030 – GSK2269557, an inhaled PI3Kδ inhibitor and danirixin (GSK1325756), an oral CXCR2 antagonist are both in phase II development.

Beyond asthma and COPD, GSK is using its long-term leadership in respiratory R&D to actively explore new diseases, including idiopathic pulmonary fibrosis and acute lung injury. GSK2862277, an inhaled TNFR1 dAb, is already in phase II clinical development for acute lung injury.
Rare Diseases

GSK is developing potential breakthrough cell and gene therapies for the treatment of rare diseases. In May 2015 the company filed for European approval of the gene therapy, GSK2696273, to treat patients with adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID). This is the first autologous stem cell gene therapy product to be submitted for marketing application review worldwide, and represents the first in a set of innovative rare disease programmes from GSK’s collaboration with the Telethon and Ospedale San Raffaele Institute in Italy. Further gene therapy products are in clinical development in the rare diseases, metachromatic leukodystrophy (MLD), Wiskott-Aldrich Syndrome (WAS) and beta thalassemia.

Through this development programme and filing, GSK believes it has a leadership position in this novel and demanding area of drug development. The company believes cell and gene therapy has the potential to be an important additional modality for tackling the underlying cause of serious disease.

Two innovative treatments for amyloidosis, a complex multi-component disease with a high mortality burden, are in development through GSK collaborations with a biotech partner and academia, demonstrating the strength and value of GSK’s research collaborations.

The combination of a small molecule and a monoclonal antibody, CPHPC + anti-SAP mAb, directly targets amyloid deposits that cause disease, and is in phase II development through a collaboration with Pentraxin Therapeutics.

GSK2998728, a transthyretin (TTR) RNA-targeted compound, is in phase III development with Isis Pharmaceuticals to treat familialamyloid polyneuropathy (FAP) and wild-type TTR amyloidosis cardiomyopathy (TTRCM).
GSK’s clinical research and development projects

Today the company has published on its website an updated pipeline chart, which details more than 90 clinical research projects that GSK is conducting. This, along with further details from today’s investor event, including copies of all presentations, is available at www.gsk.com.
2016-2020 Outlook

At its Investor Day on 6 May 2015, GSK outlined a series of expectations for its performance over the five year period 2016-2020. This included an expectation that new Pharmaceutical and Vaccine products, launched in the last three years, together with contributions from current pipeline assets, Nucala (mepoluzimab) and Shingrix (zoster), are expected to generate sales of at least £6 billion per annum by 2020 on a CER basis. GSK expects core EPS to grow at a CAGR of mid-to-high single digits on a CER basis over the five year period 2016-2020. The introduction of a generic alternative to Advair in the US was factored into the Group’s assessment of its future performance. For more information see: www.gsk.com/en-gb/investors/investor-event.

On November 2, 2015 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported financial results for the third quarter ended September 30, 2015 (Press release, NanoString Technologies, NOV 2, 2015, View Source [SID:1234507888]).

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Third Quarter Financial Highlights

Total revenue of $15.7 million, 27% year-over-year growth
Total product and service revenue of $13.9 million, 23% year-over-year growth
Consumables revenue of $9.0 million, including $0.7 million of Prosigna IVD kits, 44% year-over-year growth
Instrument revenue of $4.3 million, 6% year-over-year decline
"Positive third quarter results reflect increasing momentum as we continue to strengthen our leadership position in the field of precision oncology," said President and Chief Executive Officer, Brad Gray. "We are continuing to expand our target markets through new product innovation, such as the recent introductions of our nCounter SPRINT Profiler and our first 3D Biology product, which enables immuno-oncology researchers to make new biological observations while maximizing the data generated from valuable samples."

Recent Business Highlights

Grew installed base to over 320 nCounter Analysis Systems at September 30, 2015
Launched nCounter SPRINT Profiler, a more affordable system designed to meet the needs of the individual researcher
Launched nCounter RNA:Protein PanCancer Immune Profiling Panel, the company’s first 3D Biology application, which expands the company’s immuno-oncology portfolio and provides a powerful tool for researchers
Expanded collaboration with Celgene to include additional countries, such as China and Russia
Received favorable final local coverage determination by Palmetto GBA’s MolDx program for Prosigna, which became effective on Oct 1, 2015
Inclusion of Prosigna in the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) clinical practice guidelines, which recognized Prosigna’s value in determining the potential benefit from chemotherapy in breast cancer patients

Third Quarter Financial Results

Revenue for the three months ended September 30, 2015 rose 27% to $15.7 million, from $12.3 million for the third quarter of 2014. Instrument revenue was $4.3 million, down 6% from the prior year period, reflecting lower sales volumes in the Asia Pacific region. Consumables revenue, excluding Prosigna, was $8.4 million for the third quarter of 2015, 40% higher than in the comparable 2014 quarter. Prosigna revenue was $662,000 for the quarter, and collaboration revenue totaled $1.8 million. Gross margin on product and service revenue was 55% for the third quarter of 2015, up from 53% in the third quarter of 2014.

Research and development expense was $5.8 million for the third quarter of 2015, roughly flat versus $6.0 million for the third quarter of 2014. Selling, general and administrative expense was $12.0 million for the third quarter of 2015 compared to $12.5 million for the prior year period, reflecting efficiencies gained through streamlining the sales and marketing organization.

Net loss for the three months ended September 30, 2015 was $9.5 million, or a loss of $0.49 per diluted share, compared with $12.1 million, or a loss of $0.67 per diluted share, for the third quarter of 2014.

Outlook for 2015

The company’s financial outlook for 2015 includes:

Total revenue in the range of $60 million to $63 million, unchanged
Gross margin of approximately 53%, previously 53% to 55%
Operating expenses in the range of $76 million to $78 million, previously $77 million to $81 million
Operating loss in the range of $40 million to $44 million, previously $41 million to $47 million