Strategic Cooperation between GeneQuantum Healthcare and Biocytogen on Development of Antitumor Bioconjugates

On October 31, 2018 GeneQuantum Healthcare and Biocytogen reported that reached a Strategic Cooperation Agreement focusing on the development of next generation bioconjugates for tumor immunotherapy in Beijing on October 29, 2018 (Press release, GeneQuantum Healthcare, OCT 31, 2018, View Source [SID1234553996]). Based on their mutually beneficial complementary advantages, both companies agreed to integrate GeneQuantum Healthcare’s world-class bio-conjugation platform and Biocytogen’s tumor immunotherapy antibody drug development and evaluation platform to jointly develop a series novel bioconjugates for tumor immunotherapy and precision medicine.

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Dr. Gang Qin from GeneQuantum Healthcare remarked: "GeneQuantum Healthcare has built a diverse and innovative platform in order to face challenges in the biopharmaceutical field. GeneQuantum Healthcare has developed the intelligent ligase-dependent conjugation (iLDC) system based on the independently developed ligase-dependent conjugation (LDC) technology, which provides a systematic solution toward manufacturing next generation bioconjugates. On the other hand, Biocytogen has developed an efficient animal model production platform to screen and develop high-throughput antibody drugs for tumor immuno-oncology. We are excited to collaborate with Biocytogen, a partner that possesses unique features and top-level technologies that are extremely valuable in the process of developing next generation bioconjugates for tumor immunotherapy and targeted therapies to meet the yet unsatisfied clinical demands cancer patients worldwide. We believe this mutual win-win cooperation between these two emerging biotech companies will set a precedent and an example for biomedical intelligent manufacturing in China."

"Through the development of customized gene-edited animal models, Biocytogen can provide clients with high-quality services, including pharmacological efficacy evaluation and R&D of antibody drugs. The unique large-scale in vivo drug efficacy evaluation platform with tailored animal models and the efficient pilot antibody drug discovery process enable Biocytogen to provide pharmaceutical companies with fast and efficient services toward novel antibody drug discovery. Biocytogen is committed to becoming a world-class comprehensive CRO company specialized in biomedical R&D to advance human health. GeneQuantum Healthcare’s independent, innovative and world-class intelligent bioconjugate development platform is a leader in the industry," Dr. Yuelei Shen from Biocytogen said. "We hope to harness the integrated advantages of both GeneQuantum Healthcare and Biocytogen to jointly develop new and efficient anti-tumor drugs to satisfy the needs of patients in China and other countries."

Calithera Biosciences to Report Third Quarter 2018 Financial Results on Wednesday, November 7, 2018

On October 31, 2019 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported that the Company’s third quarter 2018 financial results will be released on Wednesday, November 7, 2018 (Press release, Calithera Biosciences, OCT 31, 2018, View Source [SID1234535233]). Company management will host a conference call on Wednesday, November 7, 2018 at 2:00 p.m. Pacific Time/ 5:00 p.m. Eastern Time to discuss the financial results and other recent corporate highlights.

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The press release and live audio webcast can be accessed via the Investor section of the Company’s website at The conference call can be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 (international) and refer to conference ID 9368596. Please log in approximately 5-10 minutes before the event to ensure a timely connection. The archived webcast will remain available for replay on Calithera’s website for 30 days.

Intellia Therapeutics Announces Third Quarter 2018 Financial Results and Corporate Developments

On October 31, 2018 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported financial results and operational developments for the third quarter of 2018 (Press release, Intellia Therapeutics, OCT 31, 2018, View Source [SID1234530584]).

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"We made excellent progress across our in vivo and ex vivo programs in the third quarter. In our ATTR program, Intellia previously reported that our proprietary LNP delivery technology could edit genes in the liver of non-human primates sufficiently to reduce TTR protein levels to a potential therapeutic range. Our continued in vivo delivery enhancement efforts have now produced striking improvements over these liver editing and protein knockdown levels. Data from the initial application of these enhancements in our ATTR program suggest that we may be able to double our previously reported liver editing results and more significantly reduce circulating protein beyond recognized therapeutic levels, at a lower, well-tolerated dose. We are applying these enhancements to develop a superior ATTR development candidate and we believe we can leverage these enhancements to significantly improve the performance of our development candidates for follow-on in vivo programs," said Intellia President and Chief Executive Officer John Leonard, M.D.

"Furthermore, in our insertion programs, we successfully introduced functioning genes in mice using our modular LNP delivery system of CRISPR/Cas9 in combination with our proprietary AAV insertion templates developed in collaboration with Regeneron. In our presentation at ESGCT, Intellia was the first company to demonstrate technology that can insert genes in mice to produce, and fine tune, protein levels up to and greater than those required for normal human physiology.

"In parallel to these in vivo achievements, we are rapidly advancing our ex vivo immuno-oncology efforts, beginning with the development of best-in-class CRISPR-edited T cells for WT1," added Dr. Leonard.

Third Quarter 2018 Operational Highlights and Recent Corporate Developments Include:

Transthyretin Amyloidosis Program Enhancements

Intellia announced today results from its transthyretin amyloidosis (ATTR) non-human primate (NHP) studies, conducted in collaboration with Regeneron Pharmaceuticals, Inc., related to its enhancements of the cargo components of its lipid nanoparticle (LNP)-based delivery system. These novel component enhancements, which are part of the ongoing development of its proprietary and modular in vivo delivery platform, have produced unprecedented results, achieving up to 78 percent (mean of 59 percent) liver editing in our most recent NHP study. The corresponding transthyretin (TTR) protein reduction at 21 days showed a decrease from baseline of up to 96 percent (mean reduction of 78 percent) after a single dose. This substantially improved level of liver editing, achieved with a lower dose and well-tolerated safety profile, compares with mean editing levels of 34 percent in Intellia’s previously reported NHP studies.

Based on these new data, the Company is pursuing confirmatory studies with the goal of integrating enhanced cargo components in its Investigational New Drug (IND)-enabling studies and submission of an IND (previously planned for the end of 2019, but now targeted for 2020) for ATTR. In addition, the Company intends to apply these technology improvements to the rest of its in vivo product pipeline.

"We are elated to have achieved such impressive and compelling editing and protein knockdown results in our NHP studies. We believe that this approach, while introducing a relatively short delay to our previous IND timeline, will ultimately yield the best possible treatment option for patients – one that we hope will clearly advance the standard of care well beyond approved and potential therapies for the treatment of ATTR," added Dr. Leonard.

Advancing Complex Genome Editing Capabilities
Intellia advanced its complex genome editing capabilities with the first robust demonstration of CRISPR-mediated, targeted insertion of transgenes in the liver of mice. The Company used its modular LNP delivery system of CRISPR/Cas9 in combination with Intellia’s proprietary modular adeno-associated virus (AAV) to insert donor template DNA into the albumin locus of mice. In collaboration with Regeneron, the Company used F9 as a model gene, which encodes Factor IX (FIX) protein, which is the clotting factor deficient in patients with hemophilia B. Using a proprietary bi-directional template platform technology, researchers showed that they can detect hybrid mAlb-hF9 transcripts in over 50 percent of hepatocytes following a single dose and measured circulating human FIX protein levels of more than 30,000 ng/mL. These levels are higher than those required in a clinical setting and correspond to levels predicted to be up to 40 to 300 times higher than those capable of preventing spontaneous bleeding episodes in hemophilia B patients, relative to wildtype or a hyperfunctional version of F9, respectively (sources: George, et al, NEJM, 2017; Simioni et al, NEJM, 2009). Furthermore, the Company observed that varying either the LNP or AAV dose modulated FIX levels. Protein levels remained stable after dosing throughout the 12-week observation period.

Intellia also applied the hybrid LNP-AAV delivery approach to its wholly owned in vivo preclinical program in alpha-1 antitrypsin deficiency (AATD). Alpha-1 antitrypsin (AAT), a protein that protects the lungs and is mutated or absent in patients with AATD, is produced by the SERPINA1 gene. To produce AAT protein, Intellia combined CRISPR/Cas9 delivery by LNP with the SERPINA1 DNA template delivered by AAV into the albumin locus. The targeted insertion generated gene expression levels in mice that correspond to levels that protect against the loss of pulmonary capacity in humans. This milestone underscores the utility of Intellia’s modular LNP-based delivery system and shows significant progress in developing an in vivo genome editing solution for AATD.

Progressing Transgenic T Cell Receptor Technology
Intellia and its research collaborator, Ospedale San Raffaele (OSR), presented data at the 26th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) showing progression in the Company’s lead ex vivo program for the treatment of acute myeloid leukemia (AML), utilizing transgenic T cell receptor (TCR) technology. In vitro data demonstrated that CRISPR/Cas9 editing resulted in over 90 percent knockout of endogenous TCRs and insertion of Wilms’ Tumor 1 (WT1) epitope-specific TCRs. The resulting engineered T cells were fully functional and killed a significant percentage of leukemic blasts. The Company continues to advance its first ex vivo development candidate, which is undergoing in vitro and in vivo functional testing.

Expanded Board of Directors and Management Team
Intellia announced today the appointment of Jesse Goodman, M.D., to its board of directors. Dr. Goodman brings more than three decades of expertise in medical research and public health as a professor, practicing clinician and former director of the U.S. Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER).

"We are thrilled to have someone with the broad scientific, public health, regulatory and clinical credentials of Jesse join Intellia’s board of directors," said Intellia Chairman Perry Karsen. "During his time at the FDA, Jesse demonstrated exceptional leadership in ensuring that the agency and companies could work together to advance scientific innovation and responsibly enhance patient access to safe and effective therapies. We expect his contributions will be invaluable as Intellia begins transitioning to a biotech company approaching clinical trials with patients."

Dr. Goodman is currently a professor of medicine at Georgetown University, where he directs the Center on Medical Product Access, Safety and Stewardship (COMPASS). As an attending physician in infectious diseases, he also is an active clinician and educator. Additionally, he serves as an independent non-executive director for GlaxoSmithKline plc.; as president and trustee of the U.S. Pharmacopeial Convention; and on the Regulatory Working Group of the Coalition on Epidemic Preparedness Innovation (CEPI). Dr. Goodman was chief scientist at the FDA from 2009 through 2014, during which time he led preparations for and responses to major public health threats, including emerging infectious diseases, disasters and terrorism. He previously also served as the FDA’s deputy commissioner for science and public health and as director of CBER. Dr. Goodman has a bachelor of arts in biology from Harvard University; earned a master’s in public health from the University of Minnesota; and received his doctor of medicine from the Albert Einstein College of Medicine. He completed his residency and fellowship training at the Hospital of the University of Pennsylvania and at the University of California at Los Angeles. Dr. Goodman is board-certified in internal medicine, infectious diseases and oncology, and has been elected to the American Society for Clinical Investigation and to the U.S. National Academy of Medicine.

Earlier this week, Intellia announced that Glenn Goddard had been named its executive vice president and chief financial officer. Mr. Goddard joins as a member of Intellia’s executive management team, and will oversee all financial functions and investor relations, as well as information technologies and facilities.

Strengthened Intellectual Property Position
Yesterday, the U.S. Patent and Trademark Office (USPTO) granted U.S. Patent No. 10,113,167 to The Regents of the University of California, the University of Vienna and Emmanuelle Charpentier, Ph.D., co-owners of foundational intellectual property relating to CRISPR/Cas9 genome editing technology. The patent covers optimized guide RNA formats (including single-guide and dual-guide formats) that may be used in any environment, including eukaryotic cells (such as human, animal and plant cells). The optimized formats modify the part of a guide RNA that interacts with the CRISPR/Cas9 nuclease. The previously awarded U.S. Patent No. 10,000,772 covers methods of using optimized guide RNAs to edit genes in certain environments, including in eukaryotic cells. Intellia sublicenses this intellectual property from The Regents of the University of California and the University of Vienna under a license agreement with Caribou Biosciences Inc.

Third Quarter 2018 Financial Results

Collaboration Revenue

Collaboration revenue was $7.4 million for the third quarter of 2018, compared to $7.3 million during the third quarter of 2017. The increase in collaboration revenue in 2018 was primarily driven by amounts recognized under Intellia’s collaboration agreement with Regeneron.

Since inception through September 30, 2018, the Company has received $122.7 million in funding from the collaborations with Novartis Institutes for Biomedical Research, Inc. (Novartis) and Regeneron, excluding amounts received for equity investments, and had an accounts receivable balance of $2.8 million as of September 30, 2018.

Operating Expenses

Research and development expenses increased by $5.8 million to $23.2 million during the third quarter of 2018, compared to $17.5 million during the third quarter of 2017. This increase was driven primarily by the advancement of Intellia’s research programs, research personnel growth to support these programs, as well as the expansion of the development organization, and includes laboratory supplies and research materials such as reagents.

General and administrative expenses increased by $2.6 million to $8.3 million during the third quarter of 2018, compared to $5.7 million during the third quarter of 2017. This increase was driven primarily by increased salary and related headcount-based expenses to support Intellia’s larger research and development organization and administrative obligations.

The Company’s net loss was $22.7 million for the third quarter of 2018, compared to $15.4 million during the third quarter of 2017.

Balance Sheet

Cash and cash equivalents were $293.2 million as of September 30, 2018, compared to $340.7 million as of December 31, 2017.

Financial Guidance

The Company’s primary uses of capital will continue to be for research and development programs, laboratory and related supplies, compensation costs for current and future employees, consulting, intellectual property related costs and general operating costs.

As of September 30, 2018, the Company had an accumulated deficit of $182.0 million. The Company expects losses to increase as it continues to incur significant research and development expenses related to the advancement of Intellia’s therapeutic programs and ongoing operations. Based on Intellia’s research and development plans and expectations related to the progress of the Company’s programs, the Company expects that the cash and cash equivalents as of September 30, 2018, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2020, excluding any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements with Novartis and Regeneron or any strategic use of capital not currently in the base-case planning assumptions.

Conference Call to Discuss Third Quarter 2018 Earnings and Corporate Developments

The Company will present third quarter 2018 results and corporate developments in a conference call on Oct. 31, 2018 at 8 a.m. ET. The investor presentation may be downloaded starting at 7:30 a.m. ET from the Events and Presentations page of the Investor Relations section of Intellia’s website at

To join the call:

U.S. callers should dial 866-548-4713 and use conference ID# 5893807, approximately five minutes before the call.
International callers should click here to access dial-in information and use conference ID# 5893807, approximately five minutes before the call.
A replay of the call will be available on Intellia’s website, beginning on Oct. 31, 2018 at 12 p.m. ET.

XBiotech Highlights Third Quarter Developments for 2018

On October 31, 2018 XBiotech Inc. (NASDAQ: XBIT) reported an update on recent company developments in its third quarter Form 10-Q filing with the SEC (Press release, XBiotech, OCT 31, 2018, View Source;p=RssLanding&cat=news&id=2374511 [SID1234530567]). The Company reports that it continues to make important progress in its clinical and pre-clinical programs in addition to other corporate initiatives.

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John Simard, XBiotech’s President & CEO, commented, "I am pleased with our performance during Q3 and I am positive about the prospects for substantive advances in our programs."

Further information with respect to the Company’s recent developments and other items is set forth in the Form 10-Q filed today. A summary of key events included in the Management’s Discussion and Analysis section of the Form 10-Q is provided below:

Clinical Programs
Atopic Dermatitis
In April 2018, XBiotech announced the launch of a Phase 2, open label clinical trial to evaluate the Company’s True Human monoclonal antibody, bermekimab, in patients with moderate to severe Atopic Dermatitis (AD). The study utilizes the Company’s new pre-filled syringes to deliver bermekimab therapy via subcutaneous injection. This ongoing study is evaluating the safety and efficacy of bermekimab in two groups of AD patients: those receiving 4 weekly 200mg treatments and those receiving 8 weekly 400mg treatments. The relatively short duration treatment regimen compares with the 16 week treatment for the only approved biological therapy in AD. During Q3, we announced successful completion and positive interim findings for the first cohort of this study, which involved nine patients who received the low dose (200mg/weekly) and four treatments with bermekimab. The Company reported that the 200mg subcutaneous injections were well-tolerated and that bermekimab treatment was associated with statistically significant improvement in several endpoints assessing efficacy. Enrollment was completed during Q3, totaling 37 patients, with 28 patients in the second, higher-dose cohort. Completion of the study and results analysis is expected around year end 2018 or early 2019.

Hidradenitis Suppurativa
During Q3, XBiotech announced enrollment of the first patient in its Phase 2, open label clinical study to evaluate bermekimab in patients with moderate to severe Hidradenitis Suppurativa (HS). U.S. recruitment in this study was completed in Q3 with enrollment rates exceeding expectations. The very robust enrollment is viewed by the Company as evidencing the strong desire for new treatment options in HS and the potential marketability for HS bermekimab therapy should it successfully advance through marketing approval. Importantly, the clinical study is evaluating bermekimab in two distinct patient populations: those that have failed anti-TNF therapy and in patients that have had no prior anti-TNF treatment history (the only approved biological drug to treat HS is the anti-TNF drug, Humira.). HS patients are being treated with 13 weekly subcutaneous injections of bermekimab. It was previously demonstrated in the Company’s double blind, placebo-controlled study of patients with HS that bermekimab, when administered as an intravenous therapy, was effective for treating HS patients who failed or were ineligible for anti-TNF therapy. The Company projects completion of the current study and a results read-out during the first quarter of 2019.

Pancreatic Cancer
XBiotech is evaluating bermekimab in combination with chemotherapy for the treatment of patients with pancreatic cancer. A clinical study headed by Dr. Andrew Hendifar, MD is being conducted at Cedars-Sinai Medical Center, Los Angeles, CA to examine the safety of bermekimab in combination with Onivyde (nanoliposomal irinotecan) and 5-fluorouracil (5FU)/folinic acid (leucovorin) for pancreatic cancer patients with wasting syndrome. Enrollment is expected to be completed around year end 2018. Bermekimab is believed to block tumor-related inflammation that is involved in neovascularization and tissue matrix remodeling, which is a crucial process for the growth and spread of tumors. As well, bermekimab may block tumor related inflammation that causes central nervous system (CNS) mediated metabolic dysregulation that can lead to wasting. The molecular target of bermekimab, IL-1⍺, may also be involved in tumor metastasis. In a Phase III randomized study in advanced cancer patients, patients that achieved the primary endpoint (as defined in Hickish et al., Lancet Oncology 2017) had lower incidence of disease progression. In another Phase III cancer study, bermekimab treatment was associated with a significant increase in lean body mass. Patients with pancreatic cancer often suffer from aggressive disease progression and wasting. The disease is known for its rapid mortality and high degree of morbidity. Bermekimab may also improve the anti-tumor activity of the cytotoxic chemotherapy and help patients endure longer exposure to these agents by reducing the inflammatory response caused by the treatment. Although the mechanism of cachexia in pancreatic cancer is not fully understood, bermekimab could help reduce this debilitating symptom by enabling patients to maintain and/or gain lean body mass during cancer treatment.

Research & Development
During Q3 XBiotech continued to consolidate operations at its Winnebago campus facility. All personnel and operations with the exception of in vivo research activities were successfully transferred and are now operating at the Winnebago campus. During Q3, the Company completed design and received city permits for constructing an annex building to house a new animal research facility at its Winnebago campus. This new facility will mark the final stage of the Company’s move to consolidate operations and transfer its R&D group from the East Riverside Drive location to the Winnebago campus. XBiotech’s new annex facility will include a state-of-the-art "biobubble" for working with infectious diseases, will house a Class II containment work space for pathogenic virus culture and propagation, and will include a lab for screening human blood donations and conducting the first stage of discovery work for identifying new antibody therapies. R&D activities in the new facility are planned to commence during Q1 2019.

C. difficile
XBiotech continues to make progress with its key pre-clinical R&D programs. In vivo testing of the C. difficile prophylaxis is ongoing. The Company’s scientists have established what we believe is a more challenging model for the disease. The strain of C. difficile now being used in pre-clinical development work produces a more aggressive and lethal form of disease. We believe that the new model will better establish the potential for our True Human antibody to prevent or treat human infections with C. difficile.

The Company continues to progress with developing its True Human antibodies for the treatment and prevention of influenza. XBiotech has focused on developing antibodies that target hemagglutinin and neuraminidase, two key surface moieties of the influenza virus. These key antibodies have been genetically introduced in cell lines to enable manufacturing production. A highly specific selection process has been ongoing during Q3 to identify True Human antibodies that target precise regions of the Hemagglutinin. This process, which is expected to allow the antibody therapy to be effective in a broader range of virus strains, is proceeding as planned and is expected to continue through 2018.

Anti-Tumor Antibody 12D7G
XBiotech’s manufacturing development group has progressed with advancing the 12-D7G antibody cell line production system. During the quarter, the gene for 12D7G was synthesized and inserted into a cell line, which was used to produce the first set of stable clones for potential manufacturing. The candidate production cells are undergoing further selection and a high-producing cell line suitable for clinical or commercial production is expected by year end. The True Human antibody 12D7G binds to a tumor-related protein, NYESO-1, for which it has the potential to stimulate the body to produce a highly specific immune response against tumors. 12D7G is a candidate immunotherapy that could be used to enhance the specificity and potential efficacy of checkpoint inhibitor therapies.


XBiotech continues to optimize its upstream and downstream manufacturing processes. Process development work is ongoing to increase bioreactor volume and running time for its cell culture systems, to enhance yields and reduce costs. Ongoing optimization of our manufacturing technology reflects our continued effort to establish the lowest possible cost of goods. We believe that lowering the cost of goods for monoclonal antibodies opens up new and larger potential areas of unmet medical need. We believe that considerable opportunities exist for safe and effective medicines derived from human antibody immunity; and that some of these opportunities will require competitive pricing that will be enabled by reduced cost of goods.

This year the Company invested in plant and equipment infrastructure and product formulation development to enable the launch of syringes that are pre-filled with bermekimab for use in subcutaneous injection. A new filling machine capable of loading syringes with bermekimab was installed at the Winnebago manufacturing center. The filling line operation was also transferred from the East Riverside Drive facility to the new filling suite at XBiotech’s Winnebago campus. The first release of bermekimab-filled syringes (pre-filled and ready for use in the clinic) occurred during Q3. These syringes are already being used successfully in two ongoing clinical trials. The Company is increasing the output capacity for the syringes with a target capability to fill 1,200 syringes per day, with an annual production capacity of about 300,000 units by 2019. We believe this will be adequate output to support a market launch should an approval be granted.

During the third quarter XBiotech began to reestablish and optimize manufacturing production of its monoclonal antibody, 514G3, for the treatment or prevention of Staphylococcus aureus (S. aureus) infections including MRSA. The Company expects to improve yields and processing efficiencies, and to reduce overall cost of production for 514G3 to support its initiatives to potentially use 514G3 as a prophylaxis.

Strategic Direction

XBiotech is in clinical development with bermekimab for therapeutic indications including oncology, cardiovascular medicine, and dermatology. The Company’s anti-infective antibody, 514G3, is in clinical development to treat S. aureus infections. Other anti-infectious disease antibodies are in pre-clinical development and are not expected to enter clinical studies before 2020.

The use of monoclonal antibodies as prophylaxis (i.e. to prevent disease) has not generally been considered a large or highly profitable business. The cost to produce antibodies is generally too large for them to be ideal for use in disease prevention, especially when the incidence of most diseases is not frequent enough among the general population to justify relatively high unit costs. To date, therefore, most marketed monoclonal antibodies have been for therapeutic use only (to treat patients already afflicted by disease). We believe there are two crucial components for the ideal medical and commercial success of monoclonal antibodies for use in prophylaxis: a high incidence of the target disease in the population; and strong evidence of significant clinical benefit as a result of therapy. We believe that the aforementioned situation potentially exists for XBiotech’s product candidates being administered prophylactically to patients with end-stage renal disease (ESRD) who are undergoing maintenance hemodialysis. In patients undergoing hemodialysis, there is a high relative incidence of cardiovascular and infectious disease-related morbidity and mortality compared to the general population. With bermekimab and 514G3, the Company has product candidates that could potentially reduce the incidence of cardiovascular and infectious disease mortality and morbidity, respectively, in this population.

Cardiovascular Risk in ESRD
Major adverse cardiovascular events (MACE) account for a 20% annual mortality rate in ESRD patients undergoing hemodialysis; only 35% of patients on maintenance hemodialysis will survive five years1. MACE is by far the leading cause of death and morbidity in this population. Clinical data, a body of scientific research, and recent discoveries (from Dr. Peter Libby’s group: Folco et al. Arterioscler Thromb Vasc Biol. 2018;38:1901-1912) suggest a potential role for bermekimab in reducing MACE in ESRD patients. More than 500,000 patients are currently undergoing maintenance hemodialysis in the United States for the treatment of ESRD. Reducing cardiovascular risk in patients with ESRD is thus a major unmet medical need—for which there are no approved therapies. The successful application of bermekimab as a prophylaxis in patients receiving maintenance hemodialysis would thus represent a very important breakthrough for management of this disease.

S. aureus infections in ESRD
The incidence of S. aureus infection in patients undergoing hemodialysis is about one hundred times that of the general population, and the bacteria accounts for about 10% of all-cause mortality in the hemodialysis population. Each year an estimated 37,000 hemodialysis patients will develop S. aureus infections. Of the 500,000 patients currently receiving hemodialysis in the United States, it is expected that about 40,000 will die from S. aureus infections over their hemodialysis careers2.

We believe that should we be able to demonstrate 514G3 to reduce incidence of mortality and morbidity due to S. aureus that it would be a groundbreaking approach to reducing burden of infectious disease in patients undergoing maintenance hemodialysis.

XBiotech has two ongoing clinical studies in dermatology, to assess bermekimab therapy in patients with Atopic dermatitis (AD) and hidradenitis suppurativa (HS). The Company believes that there continues to be considerable unmet medical need for effective treatment of both AD and HS. Moreover, recent FDA approvals of other biological agents to treat AD and HS have provided clinical study endpoints that we believe reduce uncertainty for our clinical study designs; therefore, these endpoints are included in XBiotech’s current studies. Based on outcomes of the ongoing studies, XBiotech expects to initially focus on only one indication (AD or HS) to conduct pivotal registration stud(ies). The market for dupilumab (the recently approved AD biological drug) has been estimated to be as much as US$5 billion3, and Humira (the recently approved biological drug for HS) is expected to generate US$21 billion in sales by 20204. Marketing approvals in either AD or HS indications for bermekimab would represent a very significant entry into the market.

XBiotech continues to analyze data generated from its Phase III studies in oncology, particularly with respect to stratifying the groups to better understand which patients are likely to benefit most from bermekimab therapy. In Q3, these analyses have provided important information suggesting that patient selection based on biomarkers could significantly enhance response rates (response as defined in Hickish et al., Lancet Oncology 2017) to bermekimab therapy by nearly 50%. Analysis and manuscript preparation of these findings is being performed by our scientific board members, including leading oncologist Dr. Razelle Kurzrock, and others. The findings will be submitted for peer review and described in their entirety upon successful publication. The Company believes that these findings may provide a rationale for seeking registration or conducting further clinical work based on a biomarker-defined subgroup. The Company plans to seek FDA guidance based on these findings.

Other Developments
Scientific Board
During the quarter, XBiotech announced the addition of two leading physicians and researchers to its Scientific Advisory Board (SAB): Dr. Alice Gottlieb and Dr. Peter Libby. Both of these extraordinary scientists are world-recognized leaders in their fields.

Dr. Alice Gottlieb, M.D., Ph.D. is internationally recognized for her expertise and trail-blazing work in the development of biological therapies to treat skin diseases. She has played leading roles in the clinical development of therapies including etanercept, infliximab, ustekinumab and secukinumab. Dr. Gottlieb is working to help guide development of bermekimab for the treatment of skin diseases. She currently serves as Study Chair for XBiotech’s ongoing Phase 2 study in hidradenitis suppurativa.

Dr. Peter Libby, M.D. is the Mallinckrodt Professor of Medicine at Harvard Medical School and clinical cardiologist at Brigham and Women’s Hospital. Dr. Libby is collaborating with XBiotech on basic research into the mechanism of inflammation and the use of bermekimab to treat cardiovascular disease. Pioneering research by Dr. Libby and his team was recently published in the journal of the American Heart Association, Arteriosclerosis, Thrombosis, and Vascular Biology. The breakthrough findings of this research showed that bermekimab could potentially be used to prevent heart attacks and strokes.

Intellectual Property
XBiotech continues to aggressively expand its already extensive patent portfolio. During the quarter the Company was awarded 4 new patents and 7 patent allowances. A Canadian patent and Russian patent both cover bermekimab. Two European patents were awarded, one covering the use of IL-1α-specific antibodies for the treatment of dermatological pathologies and the other the use of IL-1α-specific antibodies for the treatment of cancer-associated cachexia. Patent applications allowed in Q3 include one each in Australia, Canada, Israel, Mexico, the Philippines, Russia, and South Korea. The allowed Australian application relates to using IL-1α-specific antibodies to treat arthritis. The allowed Canadian application relates to using IL-1α-specific antibodies to reduce a subset of blood cells that contribute to inflammatory diseases. The allowed Israeli and Mexican applications each cover key aspects of the Company’s innovative True HumanTM antibody discovery platform. The allowed Russian application relates to using IL-1α-specific antibodies to treat vascular diseases and complications thereof, and the allowed Philippines application relates to the Company’s antibody for treating S.aureus infections, 514G3.

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

GSK delivers Q3 sales of £8.1 billion, +3% AER, +6% CER

On October 31, 2018 GlaxoSmithKline reported 2018 third quarter results (Press release, GlaxoSmithKline, OCT 31, 2018, View Source [SID1234530551]).

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Financial highlights
• Group sales £8.1 billion. Pharmaceuticals £4.2 billion, +1% AER, +3% CER; Vaccines £1.9 billion, +14% AER, +17% CER; Consumer Healthcare £1.9 billion,-1% AER, +3% CER
• Adjusted Group operating margin of 31.2%,-0.3 percentage points AER; +0.2 percentage points CER. Pharmaceuticals 32.2%; Vaccines 43.0%; Consumer Healthcare 22.0%
• Total EPS 28.8p +16% AER, +23% CER
• Adjusted EPS 35.5p +10% AER, +14% CER
• YTD free cash flow £2,375 million (2017: £1,668 million)
• 19p dividend declared for the quarter. Continue to expect 80p for FY 2018
• Now expect 2018 Adjusted EPS growth of 8-10% CER

Product and pipeline highlights
• Total New Respiratory product sales £645 million, +39% AER, +40% CER; Ellipta sales £500 million, +34% AER, +35% CER; Nucala £145 million, +59% AER, +62% CER
• Tivicay and Triumeq sales £1.1 billion +12% AER, +13% CER. Juluca sales £37 million
• Shingrix sales £286 million. Now expect £700-750 million sales for FY 2018
• New two-drug regimen dolutegravir (DTG) and lamivudine (3TC) for HIV filed in US and Europe
• Positive phase III study results received for new HIV therapy cabotegravir+rilpivirine (FLAIR/ATLAS)
• Clinical study initiated for use of BCMA in second line treatment of multiple myeloma
• New phase II efficacy and safety data for aGM-CSF in rheumatoid arthritis presented at ACR and supports further clinical development
• R&D prioritisation continues with resources reinvested in priority projects following termination of several pipeline programmes as data thresholds not met
• Positive phase II data for candidate TB vaccine published in NEJM