apceth Biopharma to Present new data for apceth-201 and apceth-301

On May 3, 2018 apceth Biopharma GmbH, a company with the mission of improving patients’ lives with next generation cell therapies, reported that it has been selected for 2 oral presentations on apceth-201 and apceth-301 at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2018 Annual Meeting that will take place in Chicago on May 16-19, 2018 (Press release, apceth, MAY 3, 2018, View Source [SID1234526045]).

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apceth’s first presentation is titled "Intracerebral Immunomodulation Using Genetically Engineered Mesenchymal Stem Cells Induces Long-Term Survival and Immunity in Glioblastoma" and will be given by Prof. Dr. Nils Ole Schmidt from the University Clinic Hamburg-Eppendorf. Prof. Schmidt will present the latest results regarding apceth-301 which is being developed for the treatment of solid tumors. The presentation will take place on Wednesday May 16 between 11:15 AM – 11:30 AM in Salon A-1/2 (abstract number 22).

The second presentation is titled "Human Mesenchymal Stem Cells Genetically Engineered to Express Alpha-1 Anti-Trypsin (apceth-201) Confer a Long-Term Survival Benefit in a Lethal, Haplo-Identical Mouse Model of Graft-Vs-Host-Disease" and will be given by Dr. Ulf Geumann of apceth. The presentation will take place on Thursday May 17 between 5:00PM – 5:15PM in the International Ballroom North (abstract number 351).
"We are honored to have been invited by ASGCT (Free ASGCT Whitepaper) to present the most recent data on apceth-201 and apceth-301", said Dr. Christine Guenther, CEO of apceth Biopharma. "We are very excited about the data and are currently preparing to move into clinical development."

GLYCOMIMETICS REPORTS FIRST QUARTER 2018 RESULTS

On May 3, 2018 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the first quarter ended March 31, 2018 and highlighted recent company achievements (Press release, GlycoMimetics, MAY 3, 2018, View Source [SID1234526072]). Quarter-end cash as a result of a follow-on financing in March was $242.6 million.

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"Our first-quarter 2018 accomplishments reflected both progress and transformation for GlycoMimetics. During this period, we laid a foundation – operationally and financially – from which we believe we will drive significant value creation. This foundation was built on the achievement of several key milestones, most notably, the announced design for our Phase 3 trial in relapsed/refractory acute myeloid leukemia (AML) patients, which forms the core of our comprehensive late-stage clinical development strategy for GMI-1271," said Rachel King, GlycoMimetics Chief Executive Officer.

"Our overall plan also includes a trial in Europe to test GMI-1271 in combination with a hypomethylating agent in newly diagnosed patients unfit for intensive chemotherapy. In addition, we continue to explore options for a trial in newly diagnosed patients fit for chemotherapy. Together these trials will position us, if successful, to offer a new standard treatment across the continuum of care in AML. Importantly, we now have the financial resources in place to achieve the key clinical milestones that we believe will drive value creation for the company," she added.

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) and entering passcode 1096657. 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.

Key Operational Highlights for the First Quarter of 2018:

Based on guidance from the US Food and Drug Administration (FDA), the company announced its design for a randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate GMI-1271 in individuals with relapsed/refractory AML. The single pivotal trial is planned to enroll approximately 380 adult patients at 30 to 40 centers in the United States, Canada, Europe and Australia, with enrollment expected to begin in the third quarter of 2018.

The company entered into an agreement with the Haemato Oncology Foundation for Adults in the Netherlands, or HOVON, to initiate clinical trial startup activities to evaluate GMI-1271 in adults with newly diagnosed AML but who cannot tolerate intensive chemotherapy, as well as in patients with myelodysplastic syndrome, or MDS, with a high risk of leukemia.

The company’s strategic partner Pfizer continues to enroll individuals with sickle cell disease in its Phase 3 clinical study of rivipansel for the treatment of vaso-occlusive crisis. GlycoMimetics continues to expect rivipansel to advance to an anticipated topline Phase 3 readout in the fourth quarter of 2018.

First Quarter 2018 Financial Results:

Cash position: As of March 31, 2018, GlycoMimetics had cash and cash equivalents of $242.6 million as compared to $123.9 million as of December 31, 2017. GlycoMimetics successfully completed a follow-on public offering of 8,050,000 shares netting proceeds of approximately $128.4 million.

R&D Expenses: The Company’s research and development expenses increased to $9.0 million for the quarter ended March 31, 2018 as compared to $5.9 million for the first quarter of 2017. The increase was due to on-going costs related to manufacturing and process development for GMI-1271.

G&A Expenses: The Company’s general and administrative expenses increased to $2.9 million for the quarter ended March 31, 2018 as compared to $2.1 million for the quarter ended March 31, 2017. The increase was due to higher patent, legal and non-cash stock-based compensation expenses.

Shares Outstanding: Shares outstanding as of March 31, 2018 were 42,490,110.

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. In a Phase 1/2 clinical trial, GMI-1271 was evaluated in both newly diagnosed elderly and relapsed/refractory patients with acute myeloid leukemia (AML). In both populations, patients treated with GMI-1271 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 FDA has granted GMI-1271 Breakthrough Therapy designation for the treatment of adult AML patients with relapsed/refractory disease.

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 vaso-occlusive crisis (VOC) of sickle cell disease (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.

Syros to Report First Quarter 2018 Financial Results on Thursday, May 10, 2018

On May 3, 2018 Syros Pharmaceuticals (NASDAQ:SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of genes, reported that it will host a live conference call and webcast at 8:30 a.m. ET on Thursday, May 10, 2018 to report its first quarter 2018 financial results and provide a corporate update (Press release, Syros Pharmaceuticals, MAY 3, 2018, View Source [SID1234526090]).

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To access the live conference call, please dial 866-595-4538 (domestic) or 636-812-6496 (international) and refer to conference ID 2967666. A webcast of the call will also be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 30 days following the presentation.

AMAG PHARMACEUTICALS ANNOUNCES FIRST QUARTER 2018 FINANCIAL RESULTS
AND RAISES FULL YEAR FINANCIAL GUIDANCE

On May 3, 2018 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) reported unaudited consolidated financial results for the quarter ended March 31, 2018, provided a business update, and increased its full year 2018 financial guidance (Press release, AMAG Pharmaceuticals, MAY 3, 2018, View Source [SID1234526018]).

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Total GAAP revenue for the first quarter of 2018 increased to $146.4 million, 5% higher than the same period last year. The year-over-year increase was driven by the addition of Intrarosa (prasterone) to the product portfolio, as well as net sales growth from Makena (hydroxyprogesterone caproate injection) and Cord Blood Registry (CBR). The company reported an operating loss of $44.9 million in the first quarter of 2018, compared with an operating loss of $40.0 million in the same period last year. Non-GAAP adjusted EBITDA totaled $40.6 million in the first quarter of 2018, compared with $57.6 million in the first quarter of 2017.1

"Strong execution led to the achievement of a number of significant regulatory and business milestones in the first quarter of 2018, including two FDA approvals that provide an opportunity to broaden and extend the Makena and Feraheme brands," stated Bill Heiden, AMAG’s president and chief executive officer. "We’ve received positive initial market feedback on both the Makena subcutaneous auto-injector and Feraheme broad label launches, and continue to see good progress on the Intrarosa launch. During the first quarter, we also filed the new drug application for bremelanotide and we are initiating programs to educate healthcare providers about diagnosing hypoactive sexual desire disorder. These accomplishments, solid first quarter financial results, and our expectation for continued strong commercial execution allowed us to increase our 2018 full year financial guidance."

First Quarter 2018 and Recent Business Highlights:

Secured two U.S. Food and Drug Administration (FDA) approvals

Broad intravenous (IV) iron deficiency anemia (IDA) label for Feraheme (ferumoxytol injection)

Makena subcutaneous (SC) auto-injector

Submitted a new drug application (NDA) to the FDA for bremelanotide for the treatment of hypoactive sexual desire disorder (HSDD) in premenopausal women

Maintained 50% Makena market share and launched the SC auto-injector (March 2018)

47% of new patient enrollments through the Makena Care Connection were prescribed the SC auto-injector in the week of April 23

Eight patents covering Makena SC auto-injector were listed in the FDA Orange Book, the last of which expires in 2036

Increased Intrarosa weekly market share to 2.8%, with approximately 50,000 total prescriptions written by more than 6,600 healthcare providers since the July 2017 launch

High gross-to-net adjustments (low net price) continued, but are expected to improve throughout 2018

1 See summaries of GAAP to non-GAAP adjustments at the conclusion of this press release.

1


Initiated first wave of multi-faceted Intrarosa digital direct-to-consumer program

Launched Feraheme with broad IV IDA label and already capturing additional share

Four successive quarters of year-over-year growth in new family enrollments at CBR, which are a strong indicator of upcoming new customer collections and revenues

Ended the quarter with $370.6 million of cash and investments, an increase of more than $40 million from year end

Increased 2018 full year financial guidance

First Quarter Ended March 31, 2018 (unaudited)
Financial Results (GAAP Basis)
Total revenues for the first quarter of 2018 increased 5% to $146.4 million, compared with $139.5 million in the first quarter of 2017. Intrarosa, which was commercially launched in July 2017, contributed $2.2 million in net sales during the first quarter of 2018. Net product sales of Makena increased 4% to $90.0 million in the first quarter of 2018, compared with $86.5 million in the same period last year. Sales of Feraheme and MuGard decreased 3% to $25.2 million in the first quarter of 2018, compared with $26.1 million in the first quarter of 2017. The temporary shortage of saline caused by the 2017 hurricane destruction in Puerto Rico, a key manufacturing site for saline, negatively impacted Feraheme sales in the early weeks of the quarter. Service revenue from CBR increased 8% to $29.0 million in the first quarter of 2018, compared with $26.9 million in the same period last year, due in part to continued growth of new family enrollments.

Costs and expenses, including costs of product sales and services, totaled $191.2 million in the first quarter of 2018, compared with $179.5 million for the same period in 2017. This increase was primarily due to higher costs of products sold of $36.3 million, of which $31.5 million was an increase in amortization expense primarily related to the Makena intramuscular intangible asset, and higher selling, general and administrative (SG&A) expenses related to launch activities for the Feraheme expanded label, the Makena SC auto-injector and Intrarosa. The increase in total costs and expenses was partially offset by lower research and development expenses and lower acquired in-process research and development (IPR&D) expenses. Acquired IPR&D expense in 2017 consisted of a one-time, upfront payment of $60 million to Palatin for bremelanotide. Acquired IPR&D expense in 2018 consisted of a $20 million charge to recognize the contingent liability associated with the FDA acceptance milestone that the company expects to pay to Palatin.

The operating loss in the first quarter of 2018 was $44.9 million, compared to an operating loss of $40.0 million for the same period last year. The company reported a net loss of $54.2 million, or $1.59 loss per basic and diluted share, for the first quarter of 2018, compared with net loss of $36.6 million, or $1.06 loss per basic and diluted share for the same period in 2017.

Financial Results (Non-GAAP Basis)1
Going forward, the company will present GAAP revenue only, as historically the only difference between GAAP and non-GAAP revenue was an adjustment to purchase accounting related to CBR deferred revenue, which is now minimal.

Total costs and expenses on a non-GAAP basis totaled $105.7 million in the first quarter of 2018, compared with $83.2 million in the first quarter of 2017. This increase was primarily due to higher SG&A expenses related to investments the company is making to support the launches of the Feraheme broad label, Makena SC auto-injector and Intrarosa.

Non-GAAP adjusted EBITDA for the first quarter of 2018 was $40.6 million, compared to $57.6 million in the first quarter of 2017. Adjusted EBITDA for the first quarter of 2018 was in line with the company’s expectations and previously stated plans to invest in the continued development and commercialization of its newer and expanded products to create long-term shareholder value.

Balance Sheet Highlights

As of March 31, 2018, the company’s cash and investments totaled $370.6 million and total debt (principal amount outstanding) was $816.4 million.

"AMAG cleared a number of de-risking milestones during the first quarter of 2018, namely the approval and launch of the subcutaneous auto-injector in advance of potential generic competition and the approval and launch of the broad Feraheme label. These events combined with continued execution across the company give us conviction to increase our financial guidance for the full year," said Ted Myles, AMAG’s chief financial officer. "We believe we are well positioned, financially and operationally, to achieve our 2018 company goals, including the updated guidance. We will continue to invest in 2018 to build and grow a broad portfolio of differentiated long-lived assets that we believe will generate significant shareholder value."

Conference Call and Webcast Access
AMAG Pharmaceuticals, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the company’s first quarter 2018 financial results, recent business highlights and 2018 outlook.

Dial-in Number
U.S./Canada dial-in number: (877) 412-6083
International dial-in number: (702) 495-1202
Conference ID: 6978298

Replay dial-in number: (855) 859-2056
Replay International dial-in number: (404) 537-3406
Conference ID: 6978298

A telephone replay will be available from approximately 11:00 a.m. ET on May 3, 2018 through midnight on May 9, 2018.

The webcast with slides will be accessible through the Investors section of AMAG’s website at www.amagpharma.com. A replay of the webcast will be archived on the website for 30 days.

Use of Non-GAAP Financial Measures
AMAG has presented certain non-GAAP financial measures, including non-GAAP revenue, non-GAAP costs and expenses and non-GAAP adjusted EBITDA (earnings before income taxes, depreciation and amortization). These non-GAAP financial measures exclude certain amounts, revenue, expenses or income, from the corresponding financial measures determined in accordance with accounting principles generally accepted in the U.S. (GAAP). Management believes this non-GAAP information is useful for investors, taken in conjunction with AMAG’s GAAP financial statements, because it provides greater transparency regarding AMAG’s operating performance. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of AMAG’s operating results as reported under GAAP, not as a substitute for GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these

non-GAAP financial measures and the most comparable GAAP financial measures are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

Apricus Biosciences Provides Corporate Update and First Quarter 2018 Financial Results

On May 3, 2018 Apricus Biosciences, Inc. (Nasdaq:APRI), a biopharmaceutical company seeking to advance innovative medicines in urology and rheumatology, reported financial results for the first quarter of 2018 and provided a corporate update on its near-term priorities (Press release, Apricus Biosciences, MAY 3, 2018, View Source;p=RssLanding&cat=news&id=2346899 [SID1234526046]).

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"Since our recent end-of-review meeting on the NDA for Vitaros with the FDA, we have been focused on pursuing U.S. Vitaros partnership discussions with interested parties. Our objective is to enable continued development and potential approval of the Vitaros product and receive financial terms commensurate with this development stage asset in exchange for a sublicense or assignment of our U.S. development and/or commercialization rights. In parallel, the Company is evaluating strategic alternatives, which may include a sale of the company, a business combination, a merger or reverse merger or a license, and in order to maximize shareholder value, the Company has engaged Canaccord Genuity LLC to assist in that process," said Richard Pascoe, Chief Executive Officer.

First Quarter Financial Results

Net loss during the quarter ended March 31, 2018 was $2.3 million, or loss per share of $0.14, compared to net income of $8.1 million, or earnings per share of $1.04, during the first quarter of 2017. Net income during the quarter ended March 31, 2017 was primarily due to the $11.8 million gain recorded upon the sale of our ex-U.S. Vitaros rights and assets to Ferring.

For all periods presented, financial statement activity related to our ex-U.S. Vitaros business has been presented as discontinued operations. As of March 31, 2018, the Company’s cash totaled $5.7 million, compared to $6.3 million as of December 31, 2016, which is expected to fund operations through the end of 2018. The Company’s cash balance as of March 31, 2018 does not include net proceeds of approximately $2.9 million from the Company’s public equity offering, which closed on April 2, 2018.