On May 2, 2019 Cambrex Corporation (NYSE: CBM), the leading small molecule company providing drug substance, drug product and analytical services across the entire drug lifecycle, reported results for the first quarter ended March 31, 2019 (Press release, Cambrex, MAY 2, 2019, View Source [SID1234535561]).

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First Quarter 2019 Highlights

Completed the acquisition of Avista Pharma Solutions ("Avista"), an early clinical phase contract development, manufacturing and testing organization, for approximately $252 million in total cash consideration. This transaction closed on January 2, 2019.

Net revenue was $159.5 million, an increase of 13% compared to the same quarter last year. Excluding the impact of currency, net revenue increased 17%.

Income from continuing operations was $10.5 million and Diluted EPS was $0.31 per share compared to $24.2 million and $0.72 per share, respectively, for the same quarter last year. Adjusted Income from continuing operations and Diluted EPS were $20.2 million and $0.60 per share compared to $25.8 million and $0.77 per share, respectively, for the same quarter last year (see table at the end of this press release).

Adjusted EBITDA was $41.1 million compared to $37.9 million in the same quarter last year (see table at the end of this press release).

Net debt excluding lease obligations was $430.0 million at the end of the quarter compared to $204.1 million at December 31, 2018. The change during the quarter was primarily the result of acquiring Avista and related expenses, positively offset by $28.7 million of Free cash flow. The Company entered into an $800 million amended credit facility in January of 2019 in connection with the Avista acquisition.

The Company continues to expect full year 2019 Net revenue, which excludes the impact of foreign currency, to be between 21% and 25% higher than 2018 Net revenue. Adjusted EBITDA is still expected to be between $150 and $160 million (see Financial Expectations – Continuing Operations section below for related explanations and additional financial guidance).

"The first quarter was a very productive quarter for Cambrex. Our financial performance was in line with our full year expectations, and we saw many examples of the synergies we anticipated when we acquired Halo and Avista. While there is still much work to be done, we took significant steps towards realizing our vision of building the leading fully integrated global small molecule CDMO. As our commercial team gains experience selling all of our services, and awareness of the full scope of our offering grows with existing and prospective customers, we expect the synergies to become more and more significant moving forward," commented Steven M. Klosk, President and Chief Executive Officer.

"We continue to invest in our facilities to ensure we have world class capabilities that position us to take advantage of ongoing positive CDMO market trends. During the quarter, we announced several new investments in our business including expanded cGMP liquid filling capacity at our Mirabel, Quebec facility, and expanded laboratories to facilitate increased generic API development capabilities at our site outside of Milan, Italy. In April, we completed construction of a new production area to manufacture highly potent drug substances at our Charles City, Iowa facility, and expect to begin production during the second quarter of 2019."

Basis of Reporting

The Company has provided a reconciliation of GAAP to adjusted (i.e. Non-GAAP) amounts at the end of this press release. Cambrex management believes that the adjustments provide useful information to investors due to the magnitude and nature of certain amounts recorded under GAAP.

As a result of the recent Halo and Avista acquisitions and consistent with how the business is managed, the Company has three reportable segments, Drug Substance ("DS"), which includes the legacy Cambrex API business excluding the High Point, North Carolina facility, Drug Product ("DP"), which includes the former Halo business, and Early Stage Development and Testing ("ESDT"), which includes the former Avista business in addition to the High Point facility.

First Quarter 2019 Operating Results – Consolidated, Continuing Operations

Net revenue was $159.5 million, an increase of $18.4 million, or 13%, from $141.1 million in the same quarter last year. Results include a 4% unfavorable impact of foreign exchange compared to the first quarter of 2018. Net revenue during the first quarter of 2019 includes the acquisitions of Halo and Avista. Compared to the same quarter last year, the Drug Substance segment revenues declined due to lower volumes.

Gross margin was 34% for the first quarter of 2019 compared to 36% in the same quarter last year.

Operating profit was $20.3 million for the first quarter of 2019 compared to $30.4 million in the same quarter last year. Operating profit for 2019 includes a $4.2 million increase in amortization expense for purchased intangibles and $6.1 million in acquisition and integration expenses. Adjusted EBITDA was $41.1 million compared to $37.9 million in the same quarter last year (see table at the end of this press release).

Income tax expense was $3.5 million resulting in an effective tax rate of 25% compared to $5.8 million and an effective tax rate of 19% in the same quarter last year. Income tax expense for the first quarter of 2018 would have been approximately 20% excluding the immediate recognition of certain effects of share-based compensation. The increase in the tax rate for 2019 as compared to 2018 is primarily due to higher state taxes as a result of state tax reform and the Company’s expanded state tax presence due to recent acquisitions, as well as the geographic mix of income.

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Income from continuing operations was $10.5 million, or $0.31 per diluted share, compared to $24.2 million, or $0.72 per diluted share, in the same quarter last year.

Adjusted Income from continuing operations was $20.2 million, or $0.60 per share, compared to $25.8 million, or $0.77 per share, in the same quarter last year.

Capital expenditures were $19.2 million and depreciation and amortization was $14.7 million compared to $23.8 million and $7.5 million, respectively, in the same quarter last year.

Net debt was $430.0 million at the end of the quarter compared to $204.1 million at December 31, 2018. The change during the quarter was primarily the result of acquiring Avista and related transaction and integration expenses, positively offset by $28.7 million of Free cash flow.

First Quarter 2019 Operating Results – Drug Substance ("DS") segment

Net revenue was $112.1 million compared to $136.6 in the same quarter last year, an 18% decrease. This includes a 4% unfavorable impact of foreign exchange compared to the first quarter of 2018. The decrease was due to lower volumes of certain branded APIs, primarily from Cambrex’s largest product, partially offset by higher sales of clinical phase products and generic APIs.

Gross profit in the first quarter of 2019 was $43.4 million compared to $50.0 million in the same quarter last year. Gross margin increased to 39% from 37% in the same quarter last year. The increase in margin was primarily driven by a favorable impact from foreign currency, favorable product mix related to generic APIs and lower inventory charges due to batch failures in 2018 partially offset by lower production volumes.

Selling, general and administrative ("SG&A") expenses were $9.4 million in the first quarter of 2019 compared to $11.5 million in the same quarter last year. The decrease was mainly due to lower consulting costs associated with a 2018 operational excellence initiative and the impact of foreign currency.

Operating profit was $31.2 million compared to $35.4 million in the first quarter of 2018. The decrease was due to lower gross profit partially offset by lower operating expenses as described above.

First Quarter 2019 Operating Results – Drug Product ("DP") segment

Net revenue in the first quarter of 2019 was $24.5 million. The former Halo business that comprises what is now the DP segment was acquired in September of 2018.

Gross profit was $6.0 million and gross margin was 25% in the first quarter of 2019.

SG&A expenses were $6.2 million in the first quarter of 2019, which includes amortization of purchased intangibles of $3.0 million.

Operating loss was $0.3 million in the first quarter of 2019 which includes amortization of purchased intangibles of $3.0 million.

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First Quarter 2019 Operating Results – Early Stage Development and Testing ("ESDT") segment

The Company’s Cambrex High Point ("CHP") facility is included in the ESDT segment for both periods presented. The former Avista businesses that comprise the majority of what is now the ESDT segment were acquired in January of 2019. The 2018 results for this segment include only the High Point facility, formerly part of Cambrex’s legacy API business.

Net revenue in the first quarter of 2019 was $22.9 million compared to $4.5 million in the same quarter last year. The increase is due to the acquisition of Avista and a 31% increase in CHP’s first quarter 2019 revenue.

Gross profit was $5.5 million in the first quarter of 2019 compared to $0.9 million in the same quarter last year. Gross margin was 24% in the first quarter of 2019 compared to 20% in the same period last year.

SG&A expenses were $6.5 million in the first quarter of 2019, which includes amortization of purchased intangibles of $1.4 million.

Operating loss was $2.5 million in the first quarter of 2019, which includes $1.2 million in integration expenses and $1.4 million of amortization of purchased intangible assets. Operating loss for the first quarter of 2018 was $0.4 million.

First Quarter 2019 Operating Results – Corporate Headquarters

SG&A expenses were $2.9 million in the first quarter of 2019 compared to $4.0 million in the same quarter last year. The decrease is primarily the result of lower due diligence costs related to mergers and acquisition activities.

Consolidated acquisition and integration expenses were $6.1 million for the first quarter of 2019, of which $4.9 million is reflected in the Corporate Headquarters cost center and $1.2 million is reflected in the ESDT segment. There were no acquisition and integration expenses during the first quarter of 2018.

Operating loss at Corporate in the first quarter of 2019 was $8.1 million compared to $4.6 million in the same quarter last year.

Financial Expectations – Continuing Operations

The following table shows the Company’s current expectations for its full year 2019 financial performance versus its expectations from the previous quarter. The expectations in the table below reflect expected results from the business including the recent acquisitions of Halo and Avista. All expectations for 2019, including growth relative to 2018, are based on actual and expected ASC 606 results for both years.

Consistent with the Company’s usual guidance practices, these financial expectations are for continuing operations and exclude the impact of any potential future acquisitions and related transaction and integration expenses, including expenses related to the recent acquisitions of Halo Pharma and Avista Pharma Solutions, divestitures, restructuring activities, certain tax items discussed below, and the impact of foreign currency on Net revenue.

EBITDA, Adjusted EBITDA and Adjusted Income from continuing operations per share for 2019 will be computed on a basis consistent with the reconciliation of the current quarter financial results in the tables at the end of this press release. Free cash flow is defined as the change in debt (excluding lease obligations, M&A and integration expenses), net of cash during the year. Adjusted effective tax rate excludes the immediate recognition of certain benefits of share-based compensation and certain other items adjusted for in the non-GAAP reconciliation tables at the end of this release.

The tax rate will be sensitive to the Company’s geographic mix of income, changes in the tax laws or rates within the countries in which the Company operates and the effects of certain share-based payments.

Cambrex expects M&A and related integration expenses for 2019 to be between $9 and $11 million. These amounts are excluded from the guidance for consolidated Adjusted EBITDA, Adjusted income from continuing operations per share and Free cash flow included above. These expenses include approximately $3.8 million of transaction and $2.3 million of integration expenses incurred in conjunction with the acquisition of Avista in early January and expected on-going integration expenses across most functional areas for the remainder of 2019. This estimate also includes certain anticipated expenses related to IT systems, but does not include costs to upgrade the two newly acquired businesses to Cambrex’s ERP software, which are planned to occur over the course of 2019 and 2020.

Expectations above include preliminary estimates for Depreciation and Amortization expense associated with purchase accounting for the Avista acquisition. Cambrex expects to finalize the purchase accounting for Avista during the second quarter of 2019. As a result, expected Depreciation and Amortization expense and its impact on expected Adjusted income from continuing operations per share in the table above could change when purchase accounting is finalized.

Refer to the tables at the end of this press release which include items typically adjusted to arrive at the Company’s non-GAAP results.

The financial information contained in this press release is unaudited, subject to revision and should not be considered final until the Company’s Form 10-Q for first quarter 2019 is filed with the Securities and Exchange Commission ("SEC").

Conference Call and Webcast

A conference call to discuss the Company’s first quarter 2019 results will begin at 8:30 a.m. Eastern Time on May 2, 2019 and can be accessed by calling 1-800-682-0995 for domestic and +1-334-323-0522 for international. Please use the passcode 2251796 and call approximately 10 minutes prior to the start time. A webcast will be available in the Investors section on the Cambrex website located at www.cambrex.com. A telephone replay of the conference call will be available through May 9, 2019 by calling 1-888-203-1112 for domestic and +1-719-457-0820 for international. Please use the passcode 2251796 to access the replay.

On May 2, 2019 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the first quarter ended March 31, 2019 and highlighted recent company achievements (Press release, GlycoMimetics, MAY 2, 2019, View Source [SID1234535557]). Quarter-end cash was $195.6 million.

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"The first quarter of 2019 was one of focused activity in the clinical development arena. We continued to identify and initiate new sites and enroll participants in our Company-sponsored Phase 3 trial in relapsed or refractory AML patients. We also worked closely with our two consortia partners to expand our late-stage uproleselan program, culminating in our announcement that the NCI consortium dosed its first patient in its trial in late April. During the same period, we worked with clinical collaborators at Duke Cancer Institute to plan our next trial for GMI-1359, a dual antagonist of E-selectin and CXCR-4, and defined individuals with breast cancer and bone metastases as our initial target study population," said Rachel King, GlycoMimetics Chief Executive Officer.

Key First-Quarter 2019 and Recent Operational Highlights:

The GlycoMimetics-sponsored pivotal Phase 3 trial of uproleselan in relapsed/refractory AML continues to enroll patients in the US and Australia. Clinical sites across the US, Europe, Canada and Australia continue to be identified and activated.

The NCI-sponsored clinical trial evaluating uproleselan in newly diagnosed older adults with AML has initiated enrollment.

Study start-up activities continued for the collaborative Haemato Oncology Foundation for Adults in the Netherlands (HOVON) European study of uproleselan in newly diagnosed patients unfit for chemotherapy.

The Company announced plans to initiate a proof-of-concept clinical trial of GMI-1359 in individuals with breast cancer whose tumors have spread to bone. The trial will evaluate safety and biomarkers of cancer cell mobilization in individuals with hormone receptor positive metastatic breast cancer.

Data was published in Nature Cell Biology that strongly suggests that E-selectin is key to tumor growth and metastasis to bone and provides further support for the upcoming clinical trial of GMI-1359 in individuals with metastatic breast cancer.

Dr. Eric Feldman has joined the GlycoMimetics executive team as Vice President, Clinical Development, and Christian Dinneen-Long has joined as Vice President, Corporate Counsel.

A planned transition is taking place within the Company’s Board of Directors. Current Board Chair Jim Barrett, who has held the role since the Company’s inception, will not seek re-election as he retires from the Board of Directors and scales back participation in several organizations. Current GlycoMimetics Board Member Tim Pearson will become Board Chair as of the close of the Company’s annual meeting on May 17, 2019.

First Quarter 2019 Financial Results:

Cash position: As of March 31, 2019, GlycoMimetics had cash and cash equivalents of $195.6 million as compared to $209.9 million as of December 31, 2018.

R&D Expenses: The Company’s research and development expenses increased to $11.8 million for the quarter ended March 31, 2019 as compared to $9.0 million for the first quarter of 2018. These increases were primarily the result of the Company’s Phase 3 clinical trial in relapsed or refractory AML patients.

G&A Expenses: The Company’s general and administrative expenses increased to $3.4 million for the quarter ended March 31, 2019 as compared to $2.9 million for the quarter ended March 31, 2018. The increase was due to higher patent, legal and non-cash stock-based compensation expenses.

Shares Outstanding: Shares outstanding as of March 31, 2019 were 43,180,169.

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 6537429. 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 progressing a comprehensive development program across the clinical spectrum of AML.

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 Inc., the exclusive licensee of rivipansel for clinical development and worldwide commercialization, 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 involved in tumor trafficking and metastatic spread. 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 or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. In the second half of 2019, the Company plans to initiate an exploratory clinical trial in individuals with breast cancer whose tumors have spread to bone.

On May 2, 2019 Geron Corporation (Nasdaq: GERN) reported financial results for the first quarter ended March 31, 2019 (Press release, Geron, MAY 2, 2019, View Source [SID1234535556]). As of the quarter-end, the Company had approximately $170 million in cash and marketable securities, which is sufficient to support the commencement of late-stage development for imetelstat in lower risk myelodysplastic syndromes (MDS).

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"We continue to make good progress on our 2019 objectives," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "We are on track to assume sponsorship of the IND for imetelstat by the end of the second quarter, as expected, which will enable us to open enrollment of the planned Phase 3 portion of IMerge by mid-year. In addition, our expanding in-house leadership and drug development team will support the accomplishment of our 2019 objectives and beyond."

First Quarter 2019 Results

For the first quarter of 2019, the Company reported a net loss of $10.1 million, or $0.05 per share, compared to $7.2 million, or $0.04 per share, for the comparable 2018 period.

Revenues for the first quarter of 2019 were $57,000 compared to $318,000 for the comparable 2018 period. Revenues for 2019 and 2018 included royalty and license fee revenues under various non-imetelstat license agreements. The decrease in revenues for the three months ended March 31, 2019 compared to the same period in 2018 primarily reflects a reduction in the number of active license agreements as a result of patent expirations on the underlying technology.

Total operating expenses for the first quarter of 2019 were $11.4 million compared to $7.8 million for the comparable 2018 period.

Research and development expenses for the first quarter of 2019 were $5.9 million compared to $2.4 million for the comparable 2018 period. The increase in research and development expenses for first quarter of 2019 compared to the same period in 2018 primarily reflects higher imetelstat development costs under the former collaboration agreement with Janssen Biotech, Inc. (Janssen) due to Geron’s payment responsibility increasing from 50% to 100% following the termination of the collaboration agreement; new costs for the imetelstat program transition from Janssen to Geron, including for consultants and the Company’s contract research organization (CRO); and increased expenses related to additional development headcount.

General and administrative expenses for the first quarter of 2019 were $5.5 million compared to $5.3 million for the comparable 2018 period. The increase in general and administrative expenses in the first quarter of 2019 compared to same period in 2018 primarily reflects recruitment expenses for new members for the Company’s board of directors.

Interest and other income for the first quarter of 2019 was $1.2 million compared to $394,000 for the comparable 2018 period. The increase in interest and other income for 2019 compared to 2018 primarily reflects higher yields on the Company’s marketable securities portfolio.

Conference Call for Imetelstat Program Update

In lieu of a first quarter conference call, Geron will host a conference call to provide an imetelstat program update at 9:00 a.m. ET on Thursday, May 16, 2019.

Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The conference ID is 1264477. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat include a Phase 2/3 trial called IMerge in lower risk myelodysplastic syndromes (MDS) and a Phase 2 trial called IMbark in Intermediate-2 or High-risk myelofibrosis. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for the treatment of patients with transfusion-dependent anemia due to lower risk MDS who are non-del(5q) and refractory or resistant to an erythroid stimulating agent.

On May 2, 2019 Pacira BioSciences, Inc. (Nasdaq: PCRX) reported financial results for the first quarter of 2019 (Press release, Pacira Pharmaceuticals, MAY 2, 2019, View Source;p=RssLanding&cat=news&id=2396834 [SID1234535554]).

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"During the quarter, Pacira achieved significant sales growth while executing several key strategic initiatives, contributing to strong momentum we expect to continue for the rest of 2019," said Dave Stack, chairman and chief executive officer of Pacira BioSciences. "We continue to drive our focused strategy and expect to deliver another year of record EXPAREL sales. We are seeing accelerated penetration in soft tissue procedures, driven by the strong and growing body of evidence around regional pain management approaches that utilize field blocks and nerve blocks as the core of a multimodal non-opioid strategy. In addition, our significant partnership with Johnson & Johnson in orthopedic procedures continues to solidify the role of EXPAREL as the foundation of non-opioid solutions for rotator cuff, spine, joint reconstruction, and hip fracture procedures. Our active customer base is steadily expanding with ambulatory and oral surgery centers both remaining key drivers of new EXPAREL patients."

Mr. Stack continued: "Finally, through our recent MyoScience acquisition, we were pleased to add the iovera° system to our non-opioid franchise as we broaden our strategic reach in this critical area of pain management."

First Quarter 2019 Financial Results

Total revenues were $91.3 million in the first quarter of 2019, a 22.4 percent increase over the $74.6 million reported for the first quarter of 2018.

EXPAREL net product sales were $90.6 million in the first quarter of 2019, a 22.4 percent increase over the $74.0 million reported for the first quarter of 2018 with one less selling day in the first quarter of 2019.

Total operating expenses were $90.2 million in the first quarter of 2019, compared to $81.5 million in the first quarter of 2018.

GAAP net loss was $2.8 million, or $0.07 per diluted share, in the first quarter of 2019, compared to a GAAP net loss of $10.7 million, or $0.26 per diluted share, in the first quarter of 2018.

Non-GAAP net income of $9.3 million, or $0.22 per diluted share, in the first quarter of 2019, compared to non-GAAP net income of $0.9 million, or $0.02 per diluted share, in the first quarter of 2018.

Cash provided by operations was $3.5 million in the first quarter of 2019, compared to cash used in operations of $2.7 million in the first quarter of 2018. Pacira ended the first quarter of 2019 with cash, cash equivalents and short-term investments ("cash") of $412.4 million. On April 9, 2019, we completed our acquisition of MyoScience for $120.0 million in cash.
Recent and Upcoming Business Highlights

MyoScience acquisition advances leadership in non-opioid pain management with FDA-approved iovera° system. In April 2019, Pacira completed its acquisition of MyoScience, Inc., a privately held medical technology company, adding the innovative iovera° system to the Pacira commercial offering. The iovera° system is a novel, non-opioid treatment that alleviates pain through a cryoanalgesia mechanism, which applies intensely focused cold therapy to a specific nerve to interrupt its ability to transmit a pain signal. Upon completion of the acquisition, Pacira changed its corporate name to Pacira BioSciences, Inc.
Results from Phase 4 study of EXPAREL in patients undergoing Cesarean section accepted for oral presentation at Society for Obstetric Anesthesia and Perinatology Annual Meeting. The podium presentation, entitled "Liposomal Bupivacaine Transversus Abdominis Plane Block for Pain after Cesarean Delivery: Results from a Multicenter, Randomized, Double-Blind, Controlled Trial" will be delivered on May 4 in Phoenix, Arizona.
New study of EXPAREL for third molar extractions shows significantly reduced opioid use; data accepted for presentation at International Association of Dental Research Meeting. The poster presentation, "Postsurgical Pain Management after Third-Molar Extraction" will be delivered on June 20, 2019 in Vancouver, BC, Canada.
New analysis shows enhanced recovery pathway that includes EXPAREL facilitates rapid discharge, high satisfaction and limited opioid use in Medicare patients undergoing total joint replacement. In March 2019, Pacira announced new data showing that a patient-optimizing, opioid-sparing enhanced recovery after surgery pathway that includes intraoperative infiltration with EXPAREL results in high rates of early discharge and patient satisfaction among Medicare-insured patients undergoing total knee or hip arthroplasty. Findings also demonstrate that the vast majority of patients do not require more than a 7-day opioid prescription following discharge.
2019 Financial Guidance

Pacira updated its 2019 financial guidance to include net product sales of iovera° (from the April 9, 2019 acquisition date onward) and reiterated all remaining 2019 financial guidance as follows:

EXPAREL net product sales of $400 million to $410 million.

iovera° net product sales of $8 million to $10 million.

Non-GAAP gross margins of 75% to 76%.

Non-GAAP research and development (R&D) expense of $60 million to $70 million.

Non-GAAP selling, general and administrative (SG&A) expense of $165 million to $175 million.

Stock-based compensation of $30 million to $35 million.
See "Non-GAAP Financial Information" and "Reconciliations of GAAP to Non-GAAP 2019 Financial Guidance" below.

On May 2, 2019 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the first quarter ended March 31, 2019 (Press release, bluebird bio, MAY 2, 2019, View Source [SID1234535552]).

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"In the first quarter, we came one step closer to bringing ZYNTEGLO, the first gene therapy for transfusion-dependent β-thalassemia (TDT), to patients with a positive opinion from the CHMP and potential approval in Europe anticipated in the second quarter," said Nick Leschly, chief bluebird. "We look forward to providing more detail on our commercial plans and launch expectations at our upcoming Analyst Day, including how our initial country-by-country launch in TDT will lay the foundation for our future launches. Our Analyst Day will also delve into our progress toward our stated goals of 1-2 new INDs per year beginning in 2020, and a deep pipeline by 2022. While the research and commercial engines are revving, our clinical development programs continue to progress at full speed, with the next data from our programs in TDT and sickle cell disease (SCD) anticipated in mid-year, and studies in earlier lines of multiple myeloma adding to the robust ide-cel (bb2121) development plan. Our team of bluebirds and our partners have been working relentlessly to get us to this point – and we’re only just getting started on our journey to help patients and their families."

Recent Highlights

TDT

CHMP POSITIVE OPINION – In March 2019, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization for ZYNTEGLO (autologous CD34+ cells encoding β A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. If approved, ZYNTEGLO, formerly referred to as LentiGlobin for TDT, will be the first commercially available gene therapy to treat TDT. The CHMP’s positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant marketing authorization for ZYNTEGLO in the European Union (EU).
CALD

ALD-104 – In April 2019, bluebird bio treated the first patient in ALD-104, the company’s international, non-randomized, open-label, multi-site Phase 3 study of Lenti-D Drug Product after myeloablative conditioning using busulfan and fludarabine in patients ≤17 years of age with cerebral adrenoleukodystrophy (CALD). The study will enroll approximately 20 patients. The primary endpoint of the study is the proportion of patients who are alive and do not have any of the 6 major functional disabilities (MFDs) at Month 24.
MULTIPLE MYELOMA

NEJM PUBLICATION – In May 2019, bluebird bio and Celgene announced that the New England Journal of Medicine (NEJM) has published interim results from CRB-401, the ongoing phase 1 study of idecabtagene vicleucel (ide-cel, formerly known as bb2121), the companies’ lead investigational BCMA-targeted chimeric antigen receptor (CAR) T-cell therapy candidate for patients with relapsed and refractory multiple myeloma.
COMPANY

MANAGEMENT APPOINTMENT – In April 2019, bluebird bio announced that Joanne Smith-Farrell has been appointed chief business officer. In this role, Joanne will lead corporate development and strategy, alliance management, and she will also continue to serve as our oncology franchise leader. Joanne has been promoted to chief business officer after joining bluebird in April 2017 as our senior vice president, corporate development and strategy.
bRT OPENING – In March 2019, bluebird bio announced the official opening of its first wholly owned manufacturing facility in Durham, N.C., that will produce lentiviral vector for the company’s investigational gene and cell therapies, including: ide-cel and bb21217 for the treatment of multiple myeloma and potentially LentiGlobin for the treatment of TDT and SCD. bluebird bio purchased the facility in November 2017.
Upcoming Anticipated Milestones

TDT
European approval of ZYNTEGLO in patients with TDT and non-β0/β0 genotypes in Q2
Submission of Biologics Licensing Application to the U.S. FDA for ZYNTEGLO in patients with TDT and non-β0/β0 genotypes by the end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-2 (HGB-207) clinical study in patients with TDT and non-β0/β0 genotypes by mid-2019 and by end of 2019
Presentation of ZYNTEGLO clinical data from the Northstar-3 (HGB-212) clinical study in patients with TDT and the β0/β0 genotype by mid-2019 and by end of 2019
SCD
Initiation of Phase 3 HGB-210 study of LentiGlobin in patients with SCD by end of 2019
Presentation of LentiGlobin clinical data from the HGB-206 clinical study in patients with SCD by mid-2019 and by end of 2019
Multiple Myeloma
Presentation of ide-cel clinical data from the registration-enabling KarMMa study and CRB-401 study in patients with relapsed/refractory multiple myeloma by end of 2019
Presentation of bb21217 clinical data from the CRB-402 clinical study in patients with relapsed/refractory multiple myeloma by the end of 2019
First Quarter 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2019 and December 31, 2018 were $1.73 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of normal operating activities and cash used to purchase property, plant and equipment as the company continues the buildout of its manufacturing facility in Durham, North Carolina.
Revenues: Total revenues were $12.5 million for the three months ended March 31, 2019 compared to $16.0 million for the three months ended March 31, 2018. The decrease was primarily attributed to decreased manufacturing services under the company’s agreement with Celgene Corporation, offset by increased license and royalty revenue.
R&D Expenses: Research and development expenses were $122.6 million for the three months ended March 31, 2019 compared to $97.1 million for the three months ended March 31, 2018. The increase was primarily driven by costs incurred to advance and expand the company’s pipeline.
G&A Expenses: General and administrative expenses were $60.3 million for the three months ended March 31, 2019 compared to $34.9 million for the three months ended March 31, 2018. The increase was largely attributable to overall growth of the pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $164.4 million for the three months ended March 31, 2019 compared to $115.1 million for the three months ended March 31, 2018.