On October 16, 2019 Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain (Press release, Intellia Therapeutics, OCT 16, 2019, View Source [SID1234542299]).

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Intellia’s data includes important updates about the company’s programs and platform development activities:

Oral Presentation:

"In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression"

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellia’s gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellia’s similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.
Abstract number: OR48
Session 5b: New delivery systems and technologies
Presentation date/time: Friday, October 25, 2019, 11:30 a.m. – 1:30 p.m. CET
Location: Room 113-115

Poster Presentations:

"In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics"

This poster presentation will highlight Intellia’s approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel O’Connell, Ph.D.
Poster ID Number: P655
Date: Wednesday, October 23, 2019

"Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia"

This poster presentation focuses on Intellia’s ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San Raffaele
Poster ID Number: P111
Date: Wednesday, October 23, 2019

"Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout"

This poster presentation focuses on the company’s T cell engineering technology, which is being applied in its Wilms’ Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.
Poster ID Number: P162
Date: Thursday, October 24, 2019

"CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria"

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.
Poster ID Number: P552
Date: Thursday, October 24, 2019

On October 16, 2019 Eli Lilly and Company (NYSE: LLY) reported top-line results from its Phase 3 SEQUOIA trial evaluating pegilodecakin plus FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in patients with metastatic pancreatic cancer whose disease had progressed during or following a first-line gemcitabine-containing regimen (Press release, Eli Lilly, OCT 16, 2019, View Source [SID1234542298]). The SEQUOIA trial did not meet its primary endpoint of overall survival.

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The most common Grade 3/4 adverse events occurring at a higher rate (>5% difference) on the pegilodecakin-plus-FOLFOX arm compared to the FOLFOX arm were neutropenia, thrombocytopenia, fatigue and anemia. Detailed efficacy and safety results will be submitted for presentation at a future medical meeting.

Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

"More than 56,700 Americans – mothers, daughters, fathers, sons, colleagues and friends – will be diagnosed with pancreatic cancer this year alone," said Julie Fleshman, JD, MBA, president and CEO of the Pancreatic Cancer Action Network (PanCAN). "Because this is an aggressive disease and the current scope of treatment options is limited, there remains an urgent need for meaningful solutions to improve outcomes for pancreatic cancer patients."

"Pancreatic cancer has proven to be one of the most difficult tumor types to treat and there have been very few recent treatment advancements in the later-line metastatic setting. We are grateful to the patients, investigators and researchers who participated in the study," said Maura Dickler, M.D., vice president, late phase development, Lilly Oncology. "While we are disappointed by the outcome of the SEQUOIA study, we look forward to the upcoming results in lung cancer, learning from those results and increasing our understanding of pegilodecakin’s novel mechanism of action in cancer immunotherapy."

Lilly gained pegilodecakin with the acquisition of ARMO BioSciences in June 2018. SEQUOIA was initiated by ARMO in March 2017 based on results of the Phase 1/1b IVY study, which evaluated pegilodecakin – used as a single agent and in combination with chemotherapy and with checkpoint inhibitor therapy – across multiple tumor types including pancreatic, non-small cell lung and renal cell cancers. Results from the IVY trial were also the basis for the ongoing Phase 2 CYPRESS 1 and CYPRESS 2 studies of pegilodecakin in combination with checkpoint inhibitors in non-small cell lung cancer (NSCLC). The CYPRESS studies were initiated by ARMO in March 2018 and results, expected in early 2020, will inform future studies of pegilodecakin in NSCLC. For the next stage of pegilodecakin’s clinical development, Lilly is focused on assessing biomarkers and conducting studies in NSCLC and other tumor types including renal cell carcinoma, where the molecule has shown promising activity.

Notes to Editors

About SEQUOIA
SEQUOIA is a global, multi-center, randomized Phase 3 study designed to compare the efficacy and safety of pegilodecakin in combination with FOLFOX (folinic acid, 5-FU, oxaliplatin) compared to FOLFOX alone in participants with metastatic adenocarcinoma of the pancreas whose disease had progressed on one prior gemcitabine-containing regimen. The primary endpoint of the study is overall survival and key secondary endpoints are progression-free survival and objective response rate. SEQUOIA was initiated by ARMO BioSciences in March 2017 and enrolled 567 patients.

About Pancreatic Cancer
Metastatic pancreatic cancer is one of the deadliest major cancers, with just three percent of patients in the U.S. living five years after the cancer is diagnosed. In the U.S., pancreatic cancer is the third leading cause of cancer death and is expected to become the second leading cause of cancer-related death in the next decade.1 Globally, pancreatic cancer is the seventh leading cause of cancer-related death.2

About Pegilodecakin
Pegilodecakin is an immunotherapy which stimulates the body’s natural defenses against cancer and expands tumor-attacking T cells. This class of T cells can infiltrate and destroy cancer cells. Pegilodecakin, a pegylated IL-10, has shown clinical activity as a single agent in renal cancer and promising results in combination with both chemotherapy and checkpoint inhibitor therapy across several tumor types including non-small cell lung cancer and renal cell carcinoma.

On October 16, 2019 ArQule, Inc. (Nasdaq: ARQL) reported it will report financial results for the third quarter of 2019 before the market opens on Wednesday, October 30, 2019 (Press release, ArQule, OCT 16, 2019, View Source [SID1234542297]). The Company will hold a conference call and webcast on the same day at 9:00 a.m. ET to discuss these results and provide a general business update.

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The live webcast can be accessed in the "Investors and Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. and entering the conference ID: 4289763. A replay will be available two hours after the completion of the call and can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events and Presentations."

On October 16, 2019 ArQule, Inc. (Nasdaq: ARQL) reported that it will be presenting data on the company’s AKT inhibitor, ARQ 751, in two poster presentations at the 2019 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) being held from October 26th to October 30th, 2019 in Boston, Massachusetts (Press release, ArQule, OCT 16, 2019, View Source [SID1234542296]).

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Presentations will detail clinical data demonstrating the correlation between biomarkers and patient response to treatment with ARQ 751 as well as preclinical in vivo and in vitro findings supporting the potential of ARQ 751 in combination with a variety of therapeutic agents.

Details on the presentations are as follows:

Presentation Title: The use of biomarkers and ctDNA in a phase 1 trial of ARQ 751
Abstract Number: A034
Presenter: Shubham Pant, M.D., MD Anderson Cancer Center
Date: October 27, 2019
Poster Viewing Time: 12:30 p.m.- 4:00 p.m. EDT
Location: Hall D, Hynes Convention Center

Presentation Title: In vitro and in vivo combination of ARQ 751 with PARP inhibitors, CDK4/6 inhibitors, Fulvestrant and Paclitaxel
Abstract Number: C076
Presenter: Yi Yu, Ph.D., ArQule
Date: October 29, 2019
Poster Viewing Time: 12:30 p.m.- 4:00 p.m. EDT
Location: Hall D, Hynes Convention Center

Additional details can be found on the conference website. A copy of presentation materials can be accessed by visiting the Publications & Presentations section of the ArQule website after the presentations conclude.

About ARQ 751
ARQ 751 is orally bioavailable, selective small molecule inhibitor of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

On October 16, 2019 Abbott (NYSE: ABT) reported financial results for the third quarter ended Sept. 30, 2019 (Press release, Abbott, OCT 16, 2019, View Source [SID1234542295]).

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Third-quarter worldwide sales of $8.1 billion increased 5.5 percent on a reported basis and 7.6 percent on an organic* basis.
Reported diluted EPS from continuing operations under GAAP was $0.53 in the third quarter. Adjusted diluted EPS from continuing operations, which excludes specified items, was $0.84, an increase of 12.0 percent versus prior year1.
Abbott narrowed its full-year 2019 outlook for diluted EPS from continuing operations on a GAAP basis to $2.06 to $2.08, and full-year adjusted diluted EPS from continuing operations to $3.23 to $3.25, reflecting 12.5 percent growth versus the prior year at the midpoint of the range2.
FreeStyle Libre achieved worldwide sales of $496 million in the quarter, an increase of 63.1 percent on a reported basis and 67.6 percent on an organic basis versus the prior year.
During the third quarter, FreeStyle Libre obtained public reimbursement coverage in Ontario and Quebec, becoming the first and only sensor-based glucose monitoring system to be listed by any provincial health plan in Canada.
Worldwide sales of MitraClip were $176 million in the quarter, an increase of 30.4 percent on a reported basis and 31.9 percent on an organic basis, including U.S. growth of 45.7 percent versus the prior year. During the third quarter, Abbott announced U.S. FDA approval of its next-generation MitraClip device, which offers enhancements and more sizes to offer doctors further options.
In September, Abbott announced data from its COAPT Trial that shows that MitraClip is projected to increase life-expectancy and quality of life compared to guideline-directed medical therapy alone in heart failure patients with secondary mitral regurgitation, or a leaky mitral heart valve.
During the third quarter, Abbott received U.S. FDA approval for its Alinity-S diagnostics system, the latest technology for screening and protecting the U.S. blood and plasma supply.
"We’re performing exceptionally well across several areas," said Miles D. White, chairman and chief executive officer, Abbott. "We’re right on track to achieve ongoing EPS and organic sales growth at the upper-end of our initial guidance ranges for the year."

* See note on organic growth below.

THIRD-QUARTER BUSINESS OVERVIEW
Note: Management believes that measuring sales growth rates on an organic basis is an appropriate way for investors to best understand the underlying performance of the business.

Organicsales growth:

Excludes the prior year first, second and third-quarter results for a non-core business within U.S. Adult Nutrition, which was discontinued during the third quarter 2018; and
Excludes the impact of foreign exchange.

Note: In order to compute results excluding the impact of exchange rates, current year U.S. dollar sales are multiplied or divided, as appropriate, by the current year average foreign exchange rates and then those amounts are multiplied or divided, as appropriate, by the prior year average foreign exchange rates.

Third-quarter 2019 worldwide sales of $8.1 billion increased 5.5 percent on a reported basis. On an organic basis, worldwide sales increased 7.6 percent. Refer to tables titled "Non-GAAP Reconciliation of Adjusted Historical Revenue" for a reconciliation of adjusted historical revenue.

Worldwide Nutrition sales increased 2.0 percent on a reported basis in the third quarter. On an organic basis, sales increased 3.8 percent. Refer to tables titled "Non-GAAP Reconciliation of Adjusted Historical Revenue" for a reconciliation of adjusted historical revenue.

Worldwide Pediatric Nutrition sales increased 0.5 percent on a reported basis in the third quarter, including an unfavorable 0.9 percent effect of foreign exchange, and increased 1.4 percent on an organic basis. In the U.S., sales growth was led by Abbott’s market-leading toddler brands, PediaSure and Pedialyte. International sales declined 2.4 percent on a reported basis and 0.7 percent on an organic basis, driven by challenging market dynamics in Greater China, partially offset by growth across several countries in Southeast Asia and Latin America.

Worldwide Adult Nutrition sales increased 3.9 percent on a reported basis in the third quarter and increased 6.9 percent on an organic basis. International Adult Nutrition sales increased 7.5 percent on a reported basis and 10.4 percent on an organic basis in the third quarter. Sales performance in the quarter was led by strong growth of Ensure, Abbott’s market-leading complete and balanced nutrition brand, and Glucerna, Abbott’s market-leading diabetes-specific nutrition brand.

Worldwide Diagnostics sales increased 4.7 percent on a reported basis in the third quarter, including an unfavorable 1.9 percent effect of foreign exchange, and increased 6.6 percent on an organic basis.

Core Laboratory Diagnostics sales increased 8.3 percent on a reported basis and 10.6 percent on an organic basis in the third quarter. Sales performance was led by above-market growth in the U.S. and internationally, where Abbott is achieving continued strong adoption of its Alinity family of innovative and highly differentiated diagnostic instruments. During the third quarter, Abbott received U.S. FDA approval for its Alinity-S blood and plasma screening diagnostics instrument and several testing assays.

Molecular Diagnostics sales decreased 7.8 percent on a reported basis in the third quarter, including an unfavorable 1.4 percent effect of foreign exchange, and decreased 6.4 percent on an organic basis. Internationally, sales growth in the quarter was negatively impacted by lower non-governmental organization purchases in Africa.

Point of Care Diagnostics sales increased 6.3 percent on a reported basis in the third quarter, including an unfavorable 0.4 percent effect of foreign exchange, and increased 6.7 percent on an organic basis. Sales growth was led by Abbott’s market-leading i-STAT handheld system in the U.S. and internationally.

Rapid Diagnostics sales decreased 0.7 percent on a reported basis in the third quarter, including an unfavorable 1.5 percent effect of foreign exchange, and increased 0.8 percent on an organic basis. Organic sales growth was led by infectious disease testing in developed markets and cardio-metabolic testing globally, partially offset by lower than expected infectious disease testing sales in Africa.

Established Pharmaceuticals sales increased 4.4 percent on a reported basis in the third quarter, including an unfavorable 3.5 percent effect of foreign exchange, and increased 7.9 percent on an organic basis.

Key Emerging Markets include India, Brazil, Russia and China along with several additional emerging countries that represent the most attractive long-term growth opportunities for Abbott’s branded generics product portfolio. Sales in these geographies increased 2.8 percent on a reported basis in the third quarter and increased 6.8 percent on an organic basis, which excludes an unfavorable 4.0 percent effect of foreign exchange. Organic sales growth was led by strong growth across several geographies, including India, China and Brazil.

Other sales increased 9.2 percent on a reported basis in the third quarter, including an unfavorable 1.7 percent effect of foreign exchange, and increased 10.9 percent on an organic basis.

Includes drug-eluting stents, balloon catheters, guidewires, vascular imaging/diagnostics products, vessel closure,

carotid and other coronary and peripheral products.

Note: Insertable Cardiac Monitor (ICM) sales, which had previously been reported in Electrophysiology, are now included in Rhythm Management. Historical periods have been adjusted to reflect this change.

Worldwide Medical Devices sales increased 8.9 percent on a reported basis in the third quarter and increased 10.6 percent on an organic basis, led by double-digit growth in Electrophysiology, Heart Failure, Structural Heart and Diabetes Care.

In Electrophysiology, growth was led by strong performance in cardiac diagnostic and ablation catheters, which are used to help physicians accurately and effectively treat atrial fibrillation, a form of irregular heartbeat.

In Heart Failure, strong double-digit growth was driven by market adoption of Abbott’s HeartMate 3 left ventricular assist device, which has been shown to improve survival and clinical outcomes in patients with advanced heart failure.

Growth in Structural Heart was led by MitraClip, Abbott’s market-leading device for the minimally invasive treatment of mitral regurgitation, or a leaky mitral heart valve. In the third quarter, Abbott announced U.S. FDA approval of its next-generation MitraClip device, MitraClip G4, which offers an expanded range of clip sizes, an alternative leaflet grasping feature and facilitation of procedure assessment in real time to offer doctors further options when treating mitral valve disease.

In Diabetes Care, sales increased 29.6 percent on a reported basis and 33.1 percent on an organic basis in the third quarter. Sales growth in the quarter was led by FreeStyle Libre, Abbott’s revolutionary continuous glucose monitoring system, with worldwide sales of $496 million, an increase of 63.1 percent on a reported basis and 67.6 percent on an organic basis versus the prior year. During the third quarter, FreeStyle Libre obtained public reimbursement coverage in Ontario and Quebec, becoming the first and only sensor-based glucose monitoring system to be listed by any provincial health plan in Canada.

ABBOTT’S GUIDANCE FOR 2019
Abbott is narrowing its guidance for 2019 diluted earnings per share from continuing operations under Generally Accepted Accounting Principles (GAAP) to $2.06 to $2.08. Abbott forecasts net specified items for the full year 2019 of $1.17 per share. Specified items include intangible amortization expense, acquisition-related expenses, charges associated with cost reduction initiatives and other expenses. Excluding specified items, projected adjusted diluted earnings per share from continuing operations would be $3.23 to $3.25 for the full year 2019.

Abbott is issuing fourth-quarter 2019 guidance for diluted earnings per share from continuing operations under GAAP of $0.59 to $0.61. Abbott forecasts specified items for the fourth quarter 2019 of $0.35 per share primarily related to intangible amortization, acquisition-related expenses, cost reduction initiatives and other expenses. Excluding specified items, projected adjusted diluted earnings per share from continuing operations would be $0.94 to $0.96 for the fourth quarter.

ABBOTT DECLARES 383RD CONSECUTIVE QUARTERLY DIVIDEND
On Sept. 12, 2019, the board of directors of Abbott declared the company’s quarterly dividend of $0.32 per share. Abbott’s cash dividend is payable Nov. 15, 2019, to shareholders of record at the close of business on Oct. 15, 2019.

Abbott has increased its dividend payout for 47 consecutive years and is a member of the S&P 500 Dividend Aristocrats Index, which tracks companies that have annually increased their dividend for at least 25 consecutive years.