On November 4, 2016 Shire plc (LSE: SHP, NASDAQ: SHPG), the leading global biotechnology company focused on serving individuals with rare diseases, reported the depth and breadth of the data it will be presenting at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, taking place December 3-6 in San Diego, California (Press release, Shire, NOV 4, 2016, View Source [SID1234516323]). The company’s robust research in hematology, oncology and genetic disease will be showcased in 4 oral presentations and 12 poster presentations.

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

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

                  Schedule Your 30 min Free Demo!

"Shire’s extensive presence at ASH (Free ASH Whitepaper) 2016 reinforces our commitment to discovering and delivering transformative treatments for patients with rare conditions," said Philip J. Vickers, Ph.D., Head of Research and Development, Shire. "We aim to continually lead the scientific dialogue for rare hematologic research and address some of the most urgent unmet needs for patients facing these challenging diseases."

HEMATOLOGY
Data in hematology, the largest portfolio for the company, will focus on research that continues to build on the proven approach of direct factor replacement as the global standard of care for hemophilia. Data presentations will showcase the continued growth of the portfolio and new strategies to improve outcomes for people with hemophilia and other bleeding conditions. The company will also present research on several of its promising early- and late-stage pipeline programs:

Target Joint Status in Patients with Hemophilia A During 18 Consecutive Months of Prophylaxis with a Pegylated Full-Length Recombinant Factor VIII with Extended Half-Life Pub #2592. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)
Modelling FVIII Levels for Prediction of Zero Spontaneous-Joint Bleeding in a Cohort of Severe Hemophilia A Subjects with Target Joints Initiated on Tertiary Prophylaxis Pub #2576. Session #322. Session Title: Disorders of Coagulation or Fibrinolysis. Poster # II. Sunday, Dec. 4, 2016 6 p.m. – 8 p.m. in Hall GH (San Diego Convention Center)
Pharmacodynamic Profile of a Recombinant ADAMTS13 (BAX930) in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome (USS)) Pub #135. Session #311. Session Title: Disorders of Platelet Number of Function: TTP and HUS. Saturday, Dec. 3, 2016 12 p.m. – 1:30 p.m. in Room 29 (San Diego Convention Center)
Appearance of High-Affinity Antibodies Precedes Clinical Diagnosis of FVIII Inhibitors – Preliminary Analysis from the Hemophilia Inhibitor PUP Study (HIPS) Pub #328. Session #322. Session Title: Disorders in Coagulation or Fibrinolysis: Hemophilia Inhibitors. Sunday, Dec. 4, 2016 9:30 a.m. – 11 a.m. in Room 33 (San Diego Convention Center)
ONCOLOGY
As part of Shire’s focus on serving individuals with rare diseases, the company is committed to addressing unmet needs in areas of oncology where patients and providers have limited treatment options. Shire has pipeline assets and research targets for rare cancers including metastatic pancreatic cancer, colorectal cancer and targets in checkpoint inhibitors and allogeneic CAR-T, as well as a biologic treatment approved as part of a multi-agent chemotherapeutic regimen for the treatment of acute lymphocytic leukemia (ALL), ONCASPARTM (pegaspargase), that is approved by the U.S. Food & Drug Administration (FDA) and all 27 EU member states. Data presented at ASH (Free ASH Whitepaper) will include a focus on pegaspargase as a therapeutic option for ALL.

GENETIC DISEASE
Shire is also dedicated to helping patients with inherited illnesses. Type I Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). It affects approximately 1 in 50,000 to 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Every type I Gaucher patient is unique and will experience varying symptoms and degrees of disease severity, making type I Gaucher disease difficult to diagnose. To this end, Shire will be hosting an engaging, case-based presentation to raise disease awareness:

Product Theater: Julie M: A Diagnostic Journey. Monday, Dec. 5, 12:15 p.m. to 1:15 p.m.
Important Information for ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated]

Indications
ADYNOVATE, [Antihemophilic Factor (Recombinant), PEGylated], is a human antihemophilic factor indicated in adolescent and adult patients (12 years and older) with hemophilia A (congenital factor VIII deficiency) for:

On-demand treatment and control of bleeding episodes
Routine prophylaxis to reduce the frequency of bleeding episodes
ADYNOVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS
ADYNOVATE is contraindicated in patients who have had prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies
Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.

ADVERSE REACTIONS
Common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf

Important Information for ADVATE [Antihemophilic Factor (Recombinant)]

Indications
ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant antihemophilic factor indicated for use in children and adults with hemophilia A (congenital factor VIII deficiency) for:

Control and prevention of bleeding episodes
Perioperative management
Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
ADVATE is not indicated for the treatment of von Willebrand disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS
ADVATE is contraindicated in patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or hamster protein or other constituents of the product.

WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE. Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.

ADVERSE REACTIONS
Serious adverse reactions seen with ADVATE are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII. The most common adverse reactions observed in clinical trials (frequency ≥5% of subjects) were pyrexia, headache, cough, nasopharyngitis, arthralgia, vomiting, upper respiratory tract infection, limb injury, nasal congestion, and diarrhea.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ADVATE_USA_ENG.pdf

Important Information for ONCASPARTM (pegaspargase)

Indications
ONCASPAR is an asparagine specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for treatment of patients with:

First line acute lymphoblastic leukemia
Acute lymphoblastic leukemia and hypersensitivity to asparaginase
DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS
ONCASPAR is contraindicated in patients who have:

History of serious allergic reactions to ONCASPAR
History of serious thrombosis with prior L-asparaginase therapy
History of pancreatitis with prior L-asparaginase therapy
History of serious hemorrhagic events with prior L-asparaginase therapy
WARNINGS & PRECAUTIONS
Anaphylaxis and Serious Reactions
Anaphylaxis and serious allergic reactions can occur therefore, patients should be observed for one hour after administration. Discontinue ONCASPAR in patients with serious allergic reactions.

Thrombosis
Serious thrombotic events, including sagittal sinus thrombosis can occur Discontinue ONCASPAR in patients with serious thrombotic events.

Pancreatitis
Pancreatitis can occur. Evaluate patients with abdominal pain for evidence of pancreatitis. Discontinue ONCASPAR in patients with pancreatitis.

Glucose Intolerance
Glucose intolerance can occur. In some cases, glucose intolerance is irreversible. Monitor serum glucose.

Coagulopathy
Increased prothrombin time, increased partial thromboplastin time, and hypofibrinogenemia can occur. Monitor coagulation parameters at baseline and periodically during and after treatment.

Hepatotoxicity and Abnormal Liver Function
Hepatotoxicity and abnormal liver function can occur. Perform appropriate monitoring.

ADVERSE REACTIONS
Most common adverse reactions (≥2%) are allergic reactions (including anaphylaxis), hyperglycemia, pancreatitis, central nervous system (CNS) thrombosis, coagulopathy, hyperbilirubinemia, and elevated transaminases.

Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.

For Full Prescribing Information, visit http://www.shirecontent.com/PI/PDFs/ONCASPAR_USA_ENG.pdf

Important Information for VPRIV (velaglucerase alfa for injection)

Indications
VPRIV is a hydrolytic lysosomal glucocerebroside-specifi enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

DETAILED IMPORTANT RISK INFORMATION

CONTRAINDICATIONS
None

WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, have occurred with VPRIV. The most commonly observed symptoms of hypersensitivity reactions were headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia/body temperature increased. Additional hypersensitivity reactions of chest discomfort, dyspnea, and pruritus have been reported with VPRIV. As with any intravenous protein product, hypersensitivity reactions are possible, therefore appropriate medical support including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate appropriate medical treatment. Discontinue VPRIV if hypersensitivity symptoms occur.

ADVERSE REACTIONS
Most common adverse reactions during clinical studies in ≥10% of patients were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated PTT, fatigue/asthenia, and pyrexia.

For Full Prescribing Information, visit http://pi.shirecontent.com/PI/PDFs/Vpriv_USA_ENG.pdf

On November 4, 2016 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the third quarter of 2016 and provided a business update (Press release, Regeneron, NOV 4, 2016, View Source [SID1234516295]).

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

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

                  Schedule Your 30 min Free Demo!

Financial Highlights

($ in millions, except per share data)

Three Months Ended
September 30,

2016

2015*

% Change
EYLEA U.S. net product sales

$
854

$
734

16
%
Total revenues

$
1,220

$
1,137

7
%
GAAP net income

$
265

$
210

26
%
GAAP net income per share – diluted

$
2.27

$
1.82

25
%
Non-GAAP net income(2)

$
365

$
276

32
%
Non-GAAP net income per share – diluted(2)

$
3.13

$
2.38

32
%

* See Table 3 of this press release for an explanation of revisions made to 2015 non-GAAP amounts previously reported.

"In the third quarter, we saw continued U.S. sales growth with EYLEA in retinal diseases and with Praluent in hypercholesterolemia," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "We are preparing for a potential approval and launch for Dupixent in atopic dermatitis and continuing to advance our pipeline at all stages."

Business Highlights

Marketed Product Update

EYLEA (aflibercept) Injection for Intravitreal Injection

In the third quarter of 2016, net sales of EYLEA in the United States increased 16% to $854 million from $734 million in the third quarter of 2015. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.
Bayer commercializes EYLEA outside the United States. In the third quarter of 2016, net sales of EYLEA outside of the United States(1) were $471 million, compared to $371 million in the third quarter of 2015. In the third quarter of 2016, Regeneron recognized $171 million from its share of net profit from EYLEA sales outside the United States, compared to $131 million in the third quarter of 2015.
Praluent (alirocumab) Injection for the Treatment of Elevated Low-Density Lipoprotein (LDL) Cholesterol

In the third quarter of 2016, global net sales of Praluent were $38 million, compared to $4 million in the third quarter of 2015. Product sales for Praluent are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Praluent. Praluent was launched in the United States in the third quarter of 2015 and in certain countries in the European Union commencing in the fourth quarter of 2015.
In the second quarter of 2016, the U.S. Food and Drug Administration (FDA) accepted for review a supplemental Biologics License Application (sBLA) for a monthly dosing regimen of Praluent, with a target action date of January 24, 2017. In addition, a regulatory application for a monthly dosing regimen of Praluent was filed in the European Union.
In July 2016, the Japanese Ministry of Health, Labour and Welfare granted marketing and manufacturing authorization for Praluent for the treatment of uncontrolled LDL cholesterol, in certain adult patients with hypercholesterolemia at high cardiovascular risk.
In August 2016, the Company and Sanofi presented data from the Phase 3 ODYSSEY ESCAPE study in patients with heterozygous familial hypercholesterolemia (HeFH) who were undergoing LDL apheresis therapy. The trial demonstrated that adding Praluent to existing therapy reduced LDL cholesterol by approximately 50% from baseline (compared to a 2% increase for placebo). The trial also achieved its primary endpoint, demonstrating that patients who added Praluent to their existing treatment regimen significantly reduced the frequency of their apheresis therapy by 75%, compared to placebo.
The ODYSSEY OUTCOMES trial remains ongoing, and is assessing the potential of Praluent to demonstrate cardiovascular benefit. An independent Data Monitoring Committee will conduct a second interim analysis for futility and overwhelming efficacy (hazard ratio < 0.802 corresponding to p < 0.0001) for the primary endpoint with consistency across subgroups and regions, positive trends for secondary end points including all-cause mortality, and no excess non-cardiovascular mortality. This second interim analysis is expected by the end of this month.
Pipeline Progress

Regeneron has sixteen product candidates in clinical development. These consist of EYLEA and fifteen fully human monoclonal antibodies generated using the Company’s VelocImmune technology, including five in collaboration with Sanofi. In addition to EYLEA and Praluent, highlights from the antibody pipeline include:

Sarilumab, the Company’s antibody targeting IL-6R for rheumatoid arthritis, is currently being studied in the global Phase 3 SARIL-RA program.

On October 28, 2016, the Company and Sanofi announced that the FDA issued a Complete Response Letter (CRL) regarding the Biologics License Application (BLA) for sarilumab. The CRL refers to certain deficiencies identified during a routine good manufacturing practice inspection of the Sanofi fill and finish facility in Le Trait, France. Satisfactory resolution of these deficiencies is required before the BLA can be approved. Sanofi submitted a comprehensive corrective action plan to the FDA, is implementing the corrective actions, and is working closely with the FDA towards a timely resolution. The CRL does not identify any concerns relating to the safety or efficacy of sarilumab.
In July 2016, the European Medicines Agency (EMA) accepted for review the Marketing Authorization Application (MAA) for sarilumab. In addition, in October 2016, an application for marketing approval for sarilumab was submitted in Japan.
Dupixent (dupilumab), the Company’s antibody that blocks signaling of IL-4 and IL-13, is currently being studied in atopic dermatitis, asthma, nasal polyps, and eosinophilic esophagitis.

The FDA previously designated Dupixent as a Breakthrough Therapy for the treatment of adult patients with inadequately controlled moderate-to-severe atopic dermatitis, and in September 2016, accepted the BLA for priority review with a target action date of March 29, 2017.
In October 2016, the FDA granted Breakthrough Therapy designation for Dupixent for the treatment of moderate to severe (12 to less than 18 years of age) and severe (6 months to less than 12 years of age) atopic dermatitis in pediatric patients who are not adequately controlled with, or who are intolerant to, topical medication.
In October 2016, additional data from LIBERTY AD SOLO 1 and SOLO 2 atopic dermatitis studies of Dupixent were presented at the European Academy of Dermatology and Venereology conference and simultaneously published in the New England Journal of Medicine.
The pivotal Phase 3 LIBERTY ASTHMA QUEST study of dupilumab for the treatment of asthma completed enrollment during the third quarter of 2016.
Fasinumab, the Company’s antibody targeting Nerve Growth Factor (NGF), is being studied in patients with pain due to osteoarthritis and chronic low back pain.

In October 2016, the FDA placed the Phase 2b study of fasinumab in chronic low back pain on clinical hold and requested an amendment of the study protocol after observing a case of adjudicated arthropathy in a patient receiving high dose fasinumab who had advanced osteoarthritis at study entry. The Company completed an unplanned analysis which showed clear evidence of efficacy with improvement in pain scores in all fasinumab groups compared to placebo at the 8- and 12-week time points, and preliminary safety results are generally consistent with what has been previously reported with the class. The Company and Teva plan to design a pivotal Phase 3 study in chronic low back pain that excludes patients with advanced osteoarthritis.
In October 2016, the Company announced that at the 36-week analysis of the Phase 2/3 clinical study of fasinumab in patients with moderate-to-severe osteoarthritis pain of the hip or knee, the incidence of adjudicated arthropathies was found to be potentially dose-dependent, with a higher rate of patients experiencing arthropathies in the higher dose groups. In the ongoing fasinumab osteoarthritis pivotal Phase 3 program, the Company and Teva are planning to advance only the lower doses from the Phase 2/3 study, subject to discussion with the FDA and other health authorities.
Nesvacumab, the Company’s antibody to Ang2 co-formulated with aflibercept for intravitreal injection, is currently being studied in patients with wet AMD and diabetic macular edema (DME). The Phase 2 RUBY study of nesvacumab/aflibercept for the treatment of DME completed enrollment during the fourth quarter of 2016.

Rinucumab, the Company’s antibody to PDGFR-beta co-formulated with aflibercept for intravitreal injection, is currently being studied in patients with neovascular age-related macular degeneration (wet AMD). In September 2016, the Company announced top-line results from the Phase 2 CAPELLA study. These data showed that at 12 weeks, rinucumab in combination with aflibercept did not add to the improvement in best corrected visual acuity (BCVA) that was demonstrated with intravitreal aflibercept injection monotherapy, the primary endpoint of the study. Results in the EYLEA monotherapy arm of the CAPELLA study were consistent with the efficacy and safety seen in Phase 3 pivotal studies of EYLEA in wet AMD.

REGN3500 entered Phase 1 clinical development for the treatment of inflammatory diseases in the third quarter of 2016. Sanofi exercised its right to opt-in to co-develop REGN3500.

Business Development Update

In July 2016, the Company and Adicet Bio, Inc. entered into a license and collaboration agreement to develop next-generation engineered immune-cell therapeutics with fully human chimeric antigen receptors and T-cell receptors directed to disease-specific cell surface antigens in order to enable the precise engagement and killing of tumor cells.
In September 2016, the Company and Teva Pharmaceuticals International GmbH (Teva), a wholly owned subsidiary of Teva Pharmaceutical Industries Ltd., entered into a collaboration agreement to develop and commercialize fasinumab. Under the terms of the agreement, the Company will lead global development and commercialization in the United States, and Teva will lead development and commercialization in territories outside the United States (excluding certain Asian countries that are subject to a separate collaboration agreement previously entered into between the Company and Mitsubishi Tanabe Pharma Corporation).
Third Quarter 2016 Financial Results

Product Revenues: Net product sales were $857 million in the third quarter of 2016, compared to $738 million in the third quarter of 2015. EYLEA net product sales in the United States were $854 million in the third quarter of 2016, compared to $734 million in the third quarter of 2015.

Total Revenues: Total revenues, which include product revenues described above, increased by 7% to $1,220 million in the third quarter of 2016, compared to $1,137 million in the third quarter of 2015. Total revenues also include Sanofi and Bayer collaboration revenues of $336 million in the third quarter of 2016, compared to $382 million in the third quarter of 2015. Collaboration revenues in the third quarter of 2016 decreased primarily due to lower reimbursable research and development expenses and an increase in the Company’s share of losses primarily from the commercialization of Praluent and pre-commercialization activities for sarilumab and Dupixent under the Company’s antibody collaboration with Sanofi, partly offset by an increase in the Company’s net profit from commercialization of EYLEA outside the United States.

Refer to Table 4 for a summary of collaboration revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $543 million in the third quarter of 2016, compared to $426 million in the third quarter of 2015. The higher R&D expenses in the third quarter of 2016 were principally due to the $25 million up-front payment made in connection with the July 2016 license and collaboration agreement with Adicet, higher development costs, including manufacturing drug supplies, primarily related to fasinumab, Dupixent, and REGN2810, and higher headcount to support the Company’s increased R&D activities, partly offset by lower development costs primarily related to Praluent and sarilumab. In addition, in the third quarter of 2016, R&D-related non-cash share-based compensation expense was $81 million, compared to $64 million in the third quarter of 2015.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $270 million in the third quarter of 2016, compared to $210 million in the third quarter of 2015. The increase was primarily due to higher commercialization-related expenses in connection with EYLEA and Praluent, and higher headcount. In addition, in the third quarter of 2016, SG&A-related non-cash share-based compensation expense was $49 million, compared to $36 million in the third quarter of 2015.

Cost of Goods Sold (COGS): GAAP COGS was $30 million in the third quarter of 2016, compared to $67 million in the third quarter of 2015. COGS primarily consists of costs in connection with producing U.S. EYLEA commercial supplies, various start-up costs in connection with the Company’s Limerick, Ireland commercial manufacturing facility, and royalties. COGS decreased principally due to a decrease in royalties since the Company’s obligation to pay Genentech based on U.S. sales of EYLEA ended in May 2016.

Cost of Collaboration and Contract Manufacturing (COCM): GAAP COCM was $14 million in the third quarter of 2016, compared to $42 million in the third quarter of 2015. COCM decreased primarily due to lower royalties since the Company’s obligation to pay Genentech based on sales of EYLEA outside the United States also ended in May 2016.

Income Tax Expense: In the third quarter of 2016, GAAP income tax expense was $101 million and the effective tax rate was 27.6%, compared to $183 million and 46.5% in the third quarter of 2015. The effective tax rate for the third quarter of 2016 was positively impacted, compared to the U.S. federal statutory rate, by the tax benefit associated with stock-based compensation, the domestic manufacturing deduction, the federal tax credit for increased research activities, and changes to tax reserves, partly offset by the negative impact of losses incurred in foreign jurisdictions with rates lower than the federal statutory rate and the non-tax deductible Branded Prescription Drug Fee. As described in Table 3 of this press release, the Company adopted Accounting Standards Update 2016-09 (ASU 2016-09), Compensation – Stock Compensation, Improvements to Employee Share-Based Payment Accounting, during the second quarter of 2016. ASU 2016-09 requires companies to recognize all excess tax benefits and tax deficiencies in connection with stock-based compensation as income tax expense or benefit in the income statement (previously, excess tax benefits were recognized in additional paid-in capital on the balance sheet).

GAAP and Non-GAAP Net Income: The Company reported GAAP net income of $265 million, or $2.53 per basic share and $2.27 per diluted share, in the third quarter of 2016, compared to GAAP net income of $210 million, or $2.04 per basic share and $1.82 per diluted share, in the third quarter of 2015.

The Company reported non-GAAP net income of $365 million, or $3.48 per basic share and $3.13 per diluted share, in the third quarter of 2016, compared to non-GAAP net income of $276 million, or $2.67 per basic share and $2.38 per diluted share, in the third quarter of 2015.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

On November 4, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported that three abstracts relating to the Company’s lead product candidate, rigosertib were accepted for presentation at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in San Diego, California, which takes place December 3-6, 2016 (Press release, Onconova, NOV 4, 2016, View Source [SID1234516294]).

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

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

                  Schedule Your 30 min Free Demo!

Details for the presentations are listed below.

Combination Therapy Phase 2 Data
Abstract Number: 3167
Title: Combination of Oral Rigosertib and Injectable Azacitidine in Patients with Myelodysplastic Syndromes (MDS): Results from a Phase II Study
Session Name: 637. Myelodysplastic Syndromes — Clinical Studies: Poster II
Date: Sunday, December 4, 2016
Presentation Time: 6:00 — 8:00 PM PST
Location: Hall GH (San Diego Convention Center)
Presenter: Shyamala C. Navada, MD, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

Safety Analysis Summary for >500 Patients Treated with Rigosertib in Clinical Trials
Abstract Number: 2011
Title: Comprehensive Analysis of Safety: Rigosertib in 557 Patients with Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)
Session Name: 637. Myelodysplastic Syndromes — Clinical Studies: Poster I
Date: Saturday, December 3, 2016
Presentation Time: 5:30 — 7:30 PM PST
Location: Hall GH (San Diego Convention Center)
Presenter: Guillermo Garcia-Manero, MD, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

Computational Analysis of Subgroup Data from Previous Clinical Trials
Abstract Number: 4324
Title: Computational Analysis of Genomic Abnormalities from a Phase 3 Trial of Rigosertib in Higher-Risk MDS: Simulation of a Predictive Signature for Clinical Response
Session Name: 637. Myelodysplastic Syndromes — Clinical Studies: Poster III
Date: Monday, December 5, 2016
Presentation Time: 6:00 — 8:00 PM PST
Location: Hall GH (San Diego Convention Center)
Presenter: Guillermo Garcia-Manero, MD, Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX

On November 4, 2016 Kura Oncology, Inc. (NASDAQ:KURA), a clinical stage biopharmaceutical company, reported preclinical findings and data from the company’s ongoing Phase 2 trial in HRAS mutant solid tumors at the 9th European Scientific Oncology Conference (ESOC-9) in Marbella, Spain. Antonio Gualberto, M.D., Ph.D., chief medical officer, delivered a presentation titled, "Targeting the RAS-ERK Pathway (Press release, Kura Oncology, NOV 4, 2016, View Source;p=RssLanding&cat=news&id=2219741 [SID1234516287])."

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

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

                  Schedule Your 30 min Free Demo!

Kura is currently evaluating tipifarnib, a farnesyl transferase inhibitor, as a potential treatment for patients with HRAS mutant solid tumors. In his presentation, Dr. Gualberto provided details on three patients with HRAS mutant squamous head and neck cancer (SCCHN), treated with tipifarnib. Two of the three SCCHN patients experienced a confirmed partial response (PR) and have been on study for 15 months and 8 months, while a third patient experienced disease stabilization for 7 months. The two partial responses were observed after 6 and 2 cycles of treatment, respectively. All three SCCHN patients had received prior treatment with cetuximab alone or in combination with chemotherapy but appeared to derive lesser benefit with those regiments than with the subsequent tipifarnib treatment.

"Despite the fact that HRAS was identified as an oncogene more than 40 years ago, there have been no effective therapeutic approaches in the clinic," said Dr. Gualberto. "These data may constitute the first demonstration of mechanism-based inhibition of HRAS in cancer patients, and support further investigation of tipifarnib in squamous head and neck cancer, an indication of high unmet need."

Dr. Gualberto’s slide presentation is available in the Scientific Presentations and Papers section of Kura Oncology’s website at www.kuraoncology.com.

On November 4, 2016 GlycoMimetics, Inc. (NASDAQ: GLYC) reported progress on its clinical development programs and its financial results for the quarter ended September 30, 2016 (Filing, Q3, GlycoMimetics, 2016, NOV 4, 2016, View Source [SID1234516276]).

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

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

                  Schedule Your 30 min Free Demo!

"For GlycoMimetics, the third quarter was highlighted by continued achievements in our clinical development programs, particularly with regard to GMI-1271, our clinical-stage E-selectin antagonist. We announced initiation of a Phase 1 clinical trial of GMI-1271 in multiple myeloma, expanding potential uses of the drug candidate. We continue to enroll the Phase 2 portion of the GMI-1271 AML trial in both newly diagnosed and relapsed/refractory patients. We also announced the initiation of a Phase 1 clinical trial of our next drug candidate, GMI-1359, in healthy volunteers. After the close of the quarter, we announced the acceptance of multiple abstracts for six posters and one oral presentation at ASH (Free ASH Whitepaper) in December 2016. With our new trial initiations and continued enrollment in our ongoing trials, we expect to be in a position to provide additional news on the clinical development of GMI-1271 and GMI-1359 in late 2016 and throughout 2017," said Rachel King, GlycoMimetics’ Chief Executive Officer.

Key Operational Highlights:
·

GlycoMimetics dosed the first patient in a Phase 1 clinical trial of GMI-1271 for multiple myeloma (MM) in September 2016. The multi-center, open-label dose escalation trial, which has begun in Ireland, is designed to measure the efficacy, safety and pharmacokinetics of GMI-1271 in combination with bortezomib-based chemotherapy among patients who have been diagnosed with MM and have not responded well to standard chemotherapy.

·

GlycoMimetics initiated dosing in a Phase 1 clinical trial of its next drug candidate, GMI-1359, in healthy volunteers. GMI-1359 is a small molecule drug candidate that simultaneously inhibits both E-selectin and CXCR4. In this first-in-humans trial, volunteer participants will receive a single injection of GMI-1359, after which they will be evaluated for safety, tolerability, pharmacokinetics

and pharmacodynamics over 16 days. The randomized, double-blind escalating dose study is being conducted at a single site in the United States.

·

We continue to recruit and dose patients in the Phase 2 portion of our clinical study evaluating GMI-1271 in AML in both newly diagnosed and relapsed/refractory patients at 8 active sites in the United States, Ireland and Australia. Having recently been granted fast track status by the FDA for GMI-1271 in this indication, GlycoMimetics plans to continue to engage with the FDA to discuss clinical and manufacturing planning as the program progresses.

·

GlycoMimetics also recently announced that six posters and one oral presentation on data from three of the company’s clinical programs will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s Annual Meeting in December 2016.

Third Quarter 2016 Financial Results:

·

Cash position: As of September 30, 2016, the Company had cash and cash equivalents of $45.3 million as compared to $46.8 million as of December 31, 2015.

·

Revenue: Revenue for the three-month periods ended September 30, 2016 and 2015 was not material. There were no milestone or royalty payments due from Pfizer during the three months ended September 30, 2016 or 2015.

·

R&D Expenses: The Company’s research and development expenses increased to $5.9 million for the quarter ended September 30, 2016 as compared to $5.0 million for the third quarter of 2015. The increase was due to higher costs associated with the clinical trials for GMI-1271 in AML and MM and for GMI-1359 in healthy volunteers, partially offset by a decrease in expenses related to manufacturing and process development for GMI-1271.

·

G&A Expenses: The Company’s general and administrative expenses decreased to $2.0 million for the quarter ended September 30, 2016 as compared to $2.1 million for the third quarter of 2015. The decrease was related to slightly lower legal expenses, patent fees and commercial research fees.

·

Shares Outstanding: Shares outstanding as of September 30, 2016 were 23,063,430.

About GMI-1271
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper), GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.
About GMI-1359
GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. Since E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow, we believe that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that affect the bone marrow such as AML and MM, as compared to targeting CXCR4 alone. In December 2015 at the annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), we presented preclinical data suggesting that GMI-1359 may enhance the ability of chemotherapy to target and improve survival rates in patients with a high-risk form of mutated AML.
About Rivipansel
GlycoMimetics’ most advanced drug candidate, rivipansel, a pan-selectin antagonist, is being developed for the treatment of vaso-occlusive crisis in sickle cell disease and is being evaluated in a Phase 3 clinical trial being conducted by its strategic collaborator, Pfizer.