On November 2, 2018 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the third quarter ended September 30, 2018 and highlighted recent company achievements (Press release, GlycoMimetics, NOV 2, 2018, View Source [SID1234530626]). Quarter-end cash and cash equivalents at September 30, 2018 were $219.8 million.

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"During the third quarter, we focused our activity on planning and initiating GlycoMimetics’ comprehensive clinical program to evaluate uproleselan across the spectrum of AML. We expect to announce enrollment of the first patient in our own Phase 3 pivotal study of "upro" in relapsed/refractory patients within a very short period of time, as initiation activity is well underway at many sites, and enrollment is now open. Support among investigators for this trial as well as for our two consortia-led trials is strong. We believe that the data contained in the abstracts selected for oral and poster presentations at the ASH (Free ASH Whitepaper) meeting reinforce the benefits already shared at prior medical meetings and bolster our confidence in upro’s potential to change the treatment paradigm in AML, whether patients have AML that is relapsed, refractory, or newly diagnosed," said Rachel King, GlycoMimetics Chief Executive Officer.

Key Operational Highlights for the Third Quarter of 2018:

The company’s strategic partner Pfizer continued to enroll individuals with sickle cell disease (SCD) in its Phase 3 clinical study of rivipansel for the treatment of vaso-occlusive crisis (VOC). Pfizer advised GlycoMimetics in August that enrollment was approximately 75% complete and is estimated to be completed in early 2019, with top-line data expected to be available in the second quarter of 2019.
The GMI-sponsored pivotal Phase 3 trial of uproleselan in relapsed/refractory AML is being initiated across multiple investigative sites in the US, and work continues to expand to clinical sites across Europe, Canada and Australia.
Planning advanced for the National Cancer Institute (NCI) collaborative study of uproleselan in newly diagnosed patients fit for chemotherapy; a protocol including an interim analysis of event-free survival was finalized and posted on clinicaltrials.gov.
Planning continued for the collaborative Haemato Oncology Foundation for Adults in the Netherlands (HOVON) European study of uproleselan in newly diagnosed patients unfit for chemo with a goal of trial initiation in 2019.
Third Quarter 2018 Financial Results:

Cash position: As of September 30, 2018, GlycoMimetics had cash and cash equivalents of $219.8 million as compared to $123.9 million as of December 31, 2017. In March 2018, GlycoMimetics completed a public offering of 8,050,000 shares of common stock, yielding net proceeds of $128.4 million.
R&D Expenses: The Company’s research and development expenses increased to $9.7 million for the quarter ended September 30, 2018 as compared to $5.8 million for the prior year quarter. The increase was primarily due to an increase in clinical trial expenses related to the start-up of the Phase 3 clinical trial of uproleselan and higher manufacturing expenditures for uproleselan clinical supplies for our planned Phase 3 clinical trial and to meet our supply obligations for clinical trials of uproleselan conducted by or in collaboration with third parties.
G&A Expenses: The Company’s general and administrative expenses increased to $2.8 million for the quarter ended September 30, 2018 as compared to $2.4 million for the prior year quarter. The increase was primarily due to higher patent and other legal expenses.
Shares Outstanding: Shares outstanding as of September 30, 2018 were 43,137,227.
The company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) with passcode 9176334. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’sel an) 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. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed/refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, which have been derived from results from third party clinical trials evaluating standard chemotherapy, as well as lower than expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects. The U.S. Food and Drug Administration (FDA) has granted uproleselan Breakthrough Therapy Designation for the treatment of adult AML patients with relapsed/refractory (R/R) disease. GlycoMimetics is currently implementing a comprehensive development program across the clinical spectrum of AML. This includes a company sponsored Phase 3 trial in R/R AML and two consortia-sponsored trials in newly diagnosed patients. One consortium trial is being sponsored by the NCI and will enroll newly diagnosed patients fit for intensive chemotherapy. The other trial is sponsored by the HOVON group in Europe and will enroll newly diagnosed patients unfit for intensive chemotherapy.

About Rivipansel

Rivipansel, the most advanced drug candidate in the GlycoMimetics pipeline, is a glycomimetic drug candidate that acts as a pan-selectin antagonist, meaning it binds to all three members of the selectin family – E-, P- and L-selectin. The first potential indication for rivipansel is VOC of SCD, one of the most severe complications of SCD which can result in acute ischemic organ injury at one or more sites. By reducing cell adhesion, activation and inflammation that are believed to contribute to reduced blood flow through the microvasculature during VOC, GlycoMimetics believes that rivipansel could be the first drug to interrupt the underlying cause of VOC, thereby potentially enabling patients to leave the hospital more quickly. Pfizer is conducting a Phase 3 clinical trial for rivipansel in SCD.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma (MM) or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GlycoMimetics has completed a Phase 1 clinical trial of GMI-1359 in healthy volunteers.

On November 2, 2018 Coherus BioSciences, Inc. (NASDAQ: CHRS), reported that the U.S. Food and Drug Administration (FDA) has approved UDENYCA (pegfilgrastim-cbqv), the first pegfilgrastim biosimilar approved by both the FDA and the European Commission (EC) for patients with cancer receiving myelosuppressive chemotherapy (Press release, Coherus Biosciences, NOV 2, 2018, View Source [SID1234531675]). UDENYCA is Coherus’ first drug to receive FDA or EC approval.

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"We are excited to announce that Coherus has received FDA approval for UDENYCA. I want to thank the Coherus team, our strategic partners, and the U.S. Food and Drug Administration for this extraordinary achievement," said Denny Lanfear, Chairman, CEO and President of Coherus BioSciences. "The list price of Neulasta has nearly tripled since approval in 2002 and now represents a $4 billion annual cost burden in the U.S. We believe that competition is essential in controlling burdensome price increases, and UDENYCA will play an important role in curbing that spend when launched. Our in-depth understanding of the market will allow us to deliver significant value to patients, payors, and providers in the U.S., including 340B hospitals, small clinics and small hospitals."

"For a number of reasons we believe the oncology marketplace is ideal for biosimilars, and we are committed to a vigorous product launch," said Chris Thompson, Senior Vice President of Sales. "Our oncology-focused, highly capable and fully-staffed commercial team is in place. We are confident that our U.S.-based manufacturing network has the finished goods in inventory to meet our highest expected demand for an extended period."

The approval of UDENYCA was supported by a comprehensive analytical similarity package, as well as pharmacokinetic, pharmacodynamic and immunogenicity studies, including over 600 healthy subjects.

"UDENYCA’s robust clinical package includes a dedicated immunogenicity similarity study in over 300 healthy subjects," said Barbara Finck, M.D., Chief Medical Officer of Coherus BioSciences. "In support of that study, and as part of our commitment to ensuring patient safety, we deployed a battery of sensitive immunogenicity assays. This effort not only supported the biosimilarity of UDENYCA, but also advanced the understanding of the immunogenic response of pegfilgrastim products."

The European Commission approved UDENYCA on September 21, 2018.

The company will provide additional details with respect to pricing and launch timing on the November 8 earnings call.

About UDENYCA
UDENYCA (pegfilgrastim-cbqv), formerly CHS-1701, is a PEGylated growth colony-stimulating factor indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia. UDENYCA drug substance manufacturing is located in Boulder, Colorado. Pegfilgrastim is one of the largest selling oncology biologics with worldwide revenues in excess of $4.5 billion in 2017.

INDICATION
UDENYCA is a leukocyte growth factor indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia.

Limitations of Use
UDENYCA is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATION: Patients with a history of serious allergic reaction to human granulocyte colony-stimulating factors such as pegfilgrastim or filgrastim products.

WARNINGS AND PRECAUTIONS:

Fatal splenic rupture: Evaluate patients who report left upper abdominal or shoulder pain for an enlarged spleen or splenic rupture.
Acute respiratory distress syndrome (ARDS): Evaluate patients who develop fever, lung infiltrates, or respiratory distress. Discontinue UDENYCATM in patients with ARDS.
Serious allergic reactions, including anaphylaxis: Permanently discontinue UDENYCATM in patients with serious allergic reactions.
Fatal sickle cell crises: Have occurred.
Glomerulonephritis: Evaluate and consider dose-reduction or interruption of UDENYCATM if causality is likely.
Adverse Reactions: Most common adverse reactions (≥ 5% difference in incidence compared to placebo) are bone pain and pain in extremity.

To report SUSPECTED ADVERSE REACTIONS, contact Coherus BioSciences at 1-800-4-UDENYCA (1-800-483-3692) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

On November 2, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immuno-oncology biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers, reported financial results and recent corporate highlights for the third quarter ended September 30, 2018 (Press release, Checkpoint Therapeutics, NOV 2, 2018, View Source [SID1234530627]).

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"In the third quarter of 2018, we announced positive interim safety and efficacy data from our ongoing Phase 1/2 clinical trial of CK-101, a third-generation EGFR inhibitor being evaluated in advanced non-small cell lung cancer (NSCLC), which was a significant milestone for the company," said James F. Oliviero, President and Chief Executive Officer of Checkpoint. "Our plans remain on-track for the rest of the year as we continue enrollment to establish the optimal dose of CK-101, after which we plan to initiate a Phase 3 trial in 2019 in treatment-naïve EGFR mutation-positive NSCLC patients. We also look forward to reporting early next year interim data from the expansion cohort phase of our Phase 1 clinical trial of CK-301, a fully human anti-PD-L1 antibody, in selected recurrent or metastatic cancers."

Recent Corporate Highlights:

In September 2018, Checkpoint announced positive interim safety and efficacy data from its Phase 1/2 clinical trial of CK-101, a third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) being evaluated in advanced NSCLC. The data were presented in an oral presentation at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer in Toronto. CK-101 was well tolerated across multiple dose groups and safe. Durable anti-tumor activity was observed, particularly in treatment-naïve EGFR mutation-positive NSCLC patients.
In October 2018, Checkpoint appointed Christian Béchon to its Board of Directors.
Financial Results:

Cash Position: As of September 30, 2018, Checkpoint’s cash and cash equivalents totaled $29.6 million, compared to $19.2 million at December 31, 2017, an increase of $10.4 million year-to-date.
R&D Expenses: Research and development expenses for the third quarter of 2018 were $7.8 million, compared to $5.0 million for the third quarter of 2017, an increase of $2.8 million.
G&A Expenses: General and administrative expenses for the third quarter of 2018 were $1.5 million, compared to $1.3 million for the third quarter of 2017, an increase of $0.2 million.
Net Loss: Net loss attributable to common stockholders for the third quarter of 2018 was $9.3 million, or $0.32 per share, compared to a net loss of $5.9 million, or $0.26 per share, for the third quarter of 2017.

On November 2, 2018 Takeda Pharmaceutical Company Limited (TOKYO: 4502) (Press release, Takeda, NOV 2, 2018, View Source [SID1234530660]):

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+4.2% growth in underlying revenue driven by growth drivers, with growth in each region

Underlying sales increased by + 4.2%, with Takeda’s growth drivers (gastroenterology, oncology, neuroscience and emerging markets) continuing to grow +9.8 %.
The key products Entyvio (+ 33.1%) and Ninlaro (+ 38.0%) contributed significantly to the growth in sales, as well as the products acquired from Ariad in 2017. Each region achieved positive growth compared to last year (USA + 9.2%, Japan + 4.1%, Europe & Canada + 4.3%, Emerging Markets + 2.4%).
Reported revenues decreased by -0.1%. Although our growth drivers maintained their strong growth, foreign currency exchange rates (-1.0 pp) and disposals (-3.2 pp) had a negative impact. The impact of the divestments included the sale of additional products to the Teva JV during the 2017 fiscal year, as well as Multilab and Techpool during the fiscal year 2018.
Underlying core earnings of + 31.8% with a margin of + 5.1 pp resulting from strict OPEX discipline

Earnings from underlying core businesses increased +31.8%, reflecting revenue growth and a 5.1pp margin increase, of which two-thirds (3.3ppm) ) were stimulated by OPEX improvements. This was the result of the global OPEX initiative fully integrated into working methods at Takeda.
Reported operating profit fell 26.6%. It was impacted by two strong non-recurring gains recorded in fiscal 2017: the sale of Wako shares for 106.3 billion yen and the sale of additional products to the Teva JV. In addition, Takeda recorded non-recurring expenses for fiscal year 2018 related to the proposed acquisition of Shire. Excluding these non-recurring items, operating income increased by +64.5%.
Core EPS was up + 32.7% and reported EPS decreased 26.9% to 162 yen per share, impacted by disposals and Shire-related costs.
The product pipeline has reached several milestones in the first half of fiscal year 2018

Maintenance of multiple myeloma after a Ninlaro stem cell transplant (TOURMALINE-MM3 study), Alukbris ALK + first line metastatic non-small cell lung cancer (ALTA-1L study), Adcetris first-line CD30 + T cell peripheral lymphoma (ECHELON-2 study) and the subcutaneous formulation in Entyvio ulcerative colitis (VISIBLE 1 study) all met their primary endpoints.
Seven new molecular entities have been integrated into the Phase 1 pipeline since April 2018.
Non-core asset disposal plan is on track

Free cash flow from operations since the beginning of the year decreased by -29.7% primarily as a result of the impact of the sale of additional products to the Teva JV during the 2017 fiscal year.
The sale of securities and real estate generated 44.2 billion yen in cash and the sale of non-core activities Techpool and Multilab generated an additional 27.2 billion yen.
The net debt / EBITDA ratio is 1.7x, an improvement of 1.8x for the fourth quarter of fiscal year 2017 and 2.7x for the fourth quarter of fiscal year 2016.
Christophe Weber, President and Chief Executive Officer, commented:

"Strategic priorities and a high quality of execution led to a solid performance in the first half of fiscal year 2018, as we are committed to meeting our key priorities to increase the portfolio, strengthen the pipeline, boost profitability. Our growth drivers contribute significantly to both earnings and earnings, and I am pleased to report that two-thirds of the 510 basis points of underlying core earnings margin improvement can be explained by the discipline in terms of costs following the global OPEX initiative.
In the first half of the year, we also achieved several important financial and regulatory milestones towards the proposed acquisition of Shire plc. I would like to emphasize that Takeda’s current strategy is working and that Takeda’s board of directors, Takeda’s management team and I are confident that the acquisition of Shire will allow Takeda to significantly accelerate the transformation. of the company to become a global leader in biopharmaceutical, R & D-based, values-based, headquartered in Japan. "

Basic earnings represent adjusted net income to exclude the tax expense, our share of capital gains or losses accounted for by the equity method, financial income and expenses, other costs and income. operating, amortization and impairment of intangible assets associated with revenue and other items that management perceives as unrelated to our core business, such as the impact of purchase accounting and transaction costs.

Underlying growth compares two periods (quarters or years) of financial results on a common basis and is used by management to evaluate the business. These financial results are calculated in constant currencies and exclude the impacts of divestitures and other amounts that are exceptional items, unusual or unrelated to our ongoing operations.

Attributable to the owners of the company.

Takeda Upgrades Velcade’s Full-Year Forecast, Dynamics of Growth Factors and OPEX Discipline

Upward revisions to underlying guidelines and reported forecasts.

Underlying guidance for fiscal year 2018: Underlying earnings guidance increased

Previous projections (% growth)
(May 14, 2018) Revised Projections (% Growth)
(October 31, 2018)
Underlying turnover Low single digit growth rate Low single digit growth rate
Earnings from underlying core businesses High single digit growth Average growth%
Underlying basic EPS Low double-digit growth rate High growth (around mid-20s)
Annual dividend per share 180 yen 180 yen
Projections maintain an additional competitor with no therapeutic equivalence in Velcade with the launch of subcutaneous and intravenous administration in the United States in March 2019, an increase of 35.5 billion yen compared to previous projections (Figure 1). global business for fiscal 2017: 129.6 billion yen, fiscal 2018: 111.0 billion yen) *
Increased underlying core earnings margin in the upper range of + 100-200bps.
These underlying projections exclude the estimated financial impact of fiscal year 2018 related to Takeda’s proposed acquisition of Shire plc.
* (application of the constant exchange rate based on the rate of the fiscal year 2018)

The revised projections in the table above include the costs incurred in the first half of fiscal year 2018 related to Takeda’s proposed acquisition of Shire plc (pre-tax earnings impact: 19.8 billion yen, net profit for the impact on the year: 16.5 billion yen); however, these do not include anticipated Shire costs for the second half of the fiscal year. In addition, the projections do not include any expected Shire earnings in the event that the closing of the acquisition is finalized during fiscal year 2018.
Takeda expects the share of Shire-related costs anticipated for fiscal year 2018 to be between 40 billion yen and 60 billion yen. This does not include integration costs, interest expense and other financial charges, since the magnitude of the impact of fiscal year 2018 will be related to the closing period of the transaction.
(Reference)

A revised financial projection that excludes costs incurred in the first half of fiscal year 2018 related to Takeda’s proposed acquisition of Shire plc is presented below. Previous projections for May 14, 2018 do not include expenses related to Shire.

Forecasts excluding the estimated financial impact of the proposed acquisition of Shire will be announced by Takeda as soon as a reasonable assumption has been confirmed.
For more information on the results of the first half of Takeda’s fiscal year 2018, as well as other financial information, please visit View Source

On November 2, 2018 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported an upcoming poster presentation at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place from December 1-4, 2018 in San Diego, CA (Press release, Bio-Path Holdings, NOV 2, 2018, View Source [SID1234530611]). In addition, ASH (Free ASH Whitepaper) abstracts will appear in the November supplemental issue of Blood.

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Dr. Maro Ohanian, Assistant Professor of the Department of Leukemia at The University of Texas M.D. Anderson Cancer Center, will present interim data from the ongoing Phase 2 trial of prexigebersen (BP1001), the Company’s lead drug candidate, for the treatment of acute myeloid leukemia. Co-author Dr. Ana Tari Ashizawa, Vice President of Research and Development at Bio-Path, will also be available at the poster presentation

Details for the presentation are as follows:

Date: Saturday, December 1, 2018

Presentation Time: 6:15 PM – 8:15 PM Pacific Time

Location: San Diego Convention Center, Hall GH

Session Name: 613. Acute Myeloid Leukemia: Clinical Studies: Poster I

Title: Interim Safety and Efficacy of Lower Intensity Induction Therapy with Intravenous Prexigebersen (BP1001) in Patients with Untreated Acute Myeloid Leukemia (AML)