On August 2, 2018 Xspray Pharma reported that the first clinical trial material from NerPharMa S.r.l., Xspray’s strategic pharmaceutical manufacturing partner in Milan, has been delivered to the HyNap-Dasa clinical trial, the results of which are expected in Q3 2018 (Press release, Xspray, AUG 2, 2018, View Source [SID1234650103]). HyNap-Dasa is one of three product candidates that Xspray currently has in development. The company’s goal is to launch HyNap-Dasa on the US market in 2021.
Xspray has contracted NerPharMa to produce materials for Xspray’s clinical program and finished products for future commercial sale. The material delivered to the HyNap-Dasa study is the first resulting from this collaboration.

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Xspray is developing HyNap-Dasa both as a fully equivalent version of Sprycel to be registered in the US under an Abbreviated New Drug Application (ANDA), or as an improved product under the 505(b)(2) regulatory procedure. NerPharMa is a pharmaceutical contract manufacturer and subsidiary of Nerviano Medical Sciences S.r.l. of Milan, Italy.

NerPharMa’s GMP manufacturing facility has been approved by the Italian Medicines Agency (AIFA), Italy’s national authority responsible for drug regulation, and the US Food and Drug Administration (FDA).

The clinical HyNap-Dasa trial is a pilot study, testing two formulations in healthy volunteers. Study results are expected in the third quarter and will be the base of the planned pivotal study, scheduled to start in 2019.

"Technology transfer in the pharmaceutical industry is complex, but our partnership with Nerpharma over the past half-year has gone very well, and we were able to deliver clinical trial material according to plan," commented Per Andersson, CEO of Xspray Pharma.

"We’ve now proved that we can deliver GMP materials in an FDA-approved manufacturing facility, both clinical. This is critical, partly for our ongoing clinical program, but first and foremost, for our future commercial sales in the US."

"We’re delighted that the transfer of Xspray’s technology has gone well, and that we now have functional equipment at NerPharMa. I’m convinced that we’ll see more widespread uptake of Xspray’s very useful technology soon," responded Angelo Colombo, CEO of NerPharMa.

On August 2, 2018 Cambrex Corporation (NYSE: CBM), a leading manufacturer of small molecule innovator and generic Active Pharmaceutical Ingredients (APIs), reported its results for the second quarter ended June 30, 2018 (Press release, Cambrex, AUG 2, 2018, View Source [SID1234528318]).

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Highlights
Net revenue increased 13% to $152.0 million compared to $134.6 million in the same quarter last year. Excluding the impact of adopting the new revenue standard, ASC 606 – Revenue from Contracts with Customers, net revenue decreased 1%.
GAAP Diluted EPS from continuing operations increased 61% to $1.21 per share from $0.75 per share in the same quarter last year. The 2018 results reflect a lower tax rate as a result of tax reform in the United States. Excluding the impact of adopting ASC 606, Diluted EPS from continuing operations was $0.87 per share.
EBITDA increased 22% to $52.2 million compared to $42.6 million in the same quarter last year. Adjusted EBITDA, which excludes the impact of adopting ASC 606, decreased to $37.2 million from $42.6 million in the same quarter last year (see table at the end of this release).
Net cash was $171.3 million at the end of the quarter, a decrease of $16.2 million during the quarter.
The Company continues to expect full year 2018 Adjusted net revenue growth, which excludes the impact of foreign currency and adoption of ASC 606, to be between -2% and 2% compared to 2017 and Adjusted EBITDA to be between $150 and $160 million. This does not include the impact of the Halo acquisition (see Financial Expectations – Continuing Operations section below for related explanations and additional financial guidance).
Entered into a definitive agreement to acquire Halo Pharma, a leading dosage form Contract Development and Manufacturing Organization (CDMO) specializing in product development and commercial manufacturing, for approximately $425 million in total cash consideration.
"Our recent accomplishments mark significant progress toward our goals of investing in increased capacity and expanding our capabilities in order to take advantage of favorable industry trends and provide best-in-class services for our global customers. Most notably, we were pleased to announce the agreement to acquire Halo Pharma, which expands our offerings into finished dose development and manufacturing, while diversifying our customer base and accelerating our revenue growth," commented Steven M. Klosk, President and Chief Executive Officer.

"We are also encouraged by second quarter performance across our three product categories. We recently added a new late-stage clinical project to our innovator portfolio with the potential to generate between $5 million and $10 million in peak API sales. This brings the total to three new late-stage projects added during the first half of 2018, with the combined potential to generate greater than $25 million in API revenue. Sales of generic APIs and controlled substances were also strong in the quarter."

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.

Second Quarter 2018 Operating Results – Continuing Operations
Net revenue was $152.0 million, an increase of $17.5 million, or 13%, compared to the second quarter of 2017. Excluding a 2% favorable impact of foreign exchange compared to the second quarter of 2017, net revenue increased 11%. The increase in volumes is driven by higher custom development products, generic APIs and the adoption of ASC 606, which accelerated revenue recognition for a portion of Cambrex’s portfolio, enabling revenues for certain products to be recognized over time, rather than upon delivery to the customer. Cambrex elected the modified retrospective method which did not require prior periods to be restated. The increases were partially offset by lower pricing. Excluding the impact of adopting ASC 606, net revenue decreased 1%.

Gross margins were flat at 43% versus the same quarter last year. Excluding the impact of adopting ASC 606, gross margin was 37%.

Selling, general and administrative expenses were $16.0 million, compared to $18.1 million in the same quarter last year. This decrease was primarily due to lower personnel related costs.

Research and development expenses were $4.1 million, compared to $4.5 million in the same quarter last year. This decrease was primarily driven by the timing of spending on the development of generic drug products.

Operating profit was $44.7 million compared to $35.0 million in the same quarter last year. The increase was primarily the result of higher gross profit and lower operating expenses as described above. Excluding the impact of adopting ASC 606, operating profit was $29.7 million. Adjusted EBITDA was $37.2 million compared to $42.6 million in the same quarter last year (see table at the end of this press release).

Income tax expense was $8.7 million resulting in an effective tax rate of 18% compared to $9.2 million and an effective tax rate of 27% in the same quarter last year. The reduction in the effective tax rate reflects the impact of tax reform in the United States.

Income from continuing operations was $40.9 million or $1.21 per share compared to $25.1 million or $0.75 per share in the same quarter last year. Excluding the impact of adopting ASC 606, Diluted EPS from continuing operations was $0.87 per share.

Adjusted income from continuing operations was $24.7 million or $0.74 per share, compared to $25.4 million or $0.76 per share in the same quarter last year (see table at the end of this press release).

Capital expenditures were $8.9 million and depreciation and amortization was $7.5 million compared to $10.7 million and $7.6 million, respectively, in the same quarter last year.

Net cash was $171.3 million at the end of the second quarter, a decrease of $16.2 million during the quarter.

The following table shows the Company’s current expectations for its full year 2018 financial performance versus its expectations from the previous quarter. These expectations do not reflect the impact of the acquisition of Halo Pharma on 2018 results. The Company expects to issue revised guidance in our third quarter call once the acquisition has closed.

tations are for continuing operations and exclude the impact of any potential acquisitions, divestitures, restructuring activities, outcomes of tax disputes and the adoption of ASC 606 which became effective January 1, 2018. Adjusted net revenue growth expectations exclude the impact of foreign exchange and the adoption of ASC 606. EBITDA, Adjusted EBITDA and Adjusted income from continuing operations per share for 2018 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, net of cash during the year. Adjusted effective tax rate excludes certain effects of share-based payments that were possibly deferred under the previous guidance. 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.

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 second quarter 2018 is filed with the SEC.

Conference Call and Webcast
A conference call to discuss the Company’s second quarter 2018 results will begin at 8:30 a.m. Eastern Time on August 2, 2018 and can be accessed by calling 1-888-208-1711 for domestic and +1-323-794-2575 for international. Please use the passcode 3913181 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 August 9, 2018 by calling 1-888-203-1112 for domestic and +1-719-457-0820 for international. Please use the passcode 3913181 to access the replay.

On August 2, 2018 Selecta Biosciences, Inc. (Nasdaq:SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by mitigating unwanted immune responses, reported that CFO and Head of Corporate Strategy John Leaman, M.D., will be present at the Canaccord Genuity 38th Annual Growth Conference in Boston, Mass. at 2 p.m. ET on Thursday, August 09, 2018 (Press release, Selecta Biosciences, AUG 2, 2018, View Source [SID1234528384]). A live and archived webcast of the presentation will be available on the Investors & Media section of the Selecta website at www.selectabio.com.

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On August 2, 2018 Acceleron Pharma Inc. (Nasdaq:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, reported financial results for the second quarter ended June 30, 2018.

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"We have had a very successful start to 2018, and we look forward to carrying this momentum forward throughout the rest of the year and beyond. We are extremely pleased that luspatercept, our lead product candidate and first-in-class erythroid maturation agent, achieved positive and highly statistically significant Phase 3 results in both the MEDALIST and BELIEVE trials, confirming our confidence in its clinical profile in myelodysplastic syndromes and beta-thalassemia," said Habib Dable, President and Chief Executive Officer of Acceleron. "We and our collaboration partner, Celgene, look forward to presenting the Phase 3 data at an upcoming medical congress and are focused on the execution of key regulatory activities, including US and EU submission in the first half of 2019. We believe luspatercept is a potential platform treatment to transform the lives of patients suffering from a range of hematologic diseases associated with anemia.
Added Mr. Dable: "Our neuromuscular and pulmonary programs achieved critical milestones, putting us in a position for important Phase 2 readouts for ACE-083 and sotatercept in 2019 and 2020, respectively."

Development Program Highlights

Hematology

Luspatercept:

Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis (MF)
Luspatercept is a first-in-class erythroid maturation agent (EMA) designed to treat the late-stage erythroid maturation defect that results in chronic anemia and the need for regular red blood cell transfusions in adults with serious hematologic diseases. Luspatercept is part of the global collaboration between Acceleron and Celgene.

The MEDALIST and BELIEVE Phase 3 trials in patients with lower-risk MDS and transfusion-dependent beta-thalassemia, respectively, met all primary and key secondary endpoints.

Data will be submitted to a future medical meeting for presentation in late 2018.

Acceleron and Celgene plan to submit regulatory filings in the United States and Europe in the first half of 2019.

Updated results from the ongoing Phase 2 trials in MDS and beta-thalassemia were presented at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2018.

The initiation of the COMMANDS Phase 3 trial in patients with lower-risk MDS who are treatment naïve is planned for the third quarter of 2018.

Enrollment and treatment are ongoing in the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia and the Phase 2 trial in MF.

Neuromuscular Disease

ACE-083:

Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) Disease
ACE-083 is a locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause focal muscle weakness. ACE-083 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

Preliminary results from Part 1 of the ACE-083 Phase 2 trial in patients with CMT disease were highlighted in oral and poster presentations at the 2018 Peripheral Nerve Society (PNS) Annual Meeting in July.

Part 2 of the Phase 2 trial in patients with CMT was recently initiated with preliminary results expected by the end of 2019.

The Company plans to present results from all Part 1 dose cohorts in the FSHD Phase 2 trial in October 2018.

Enrollment and treatment are ongoing in Part 2 of the Phase 2 trial in patients with FSHD and preliminary results are expected in the second half of 2019.

ACE-083 received FDA Fast Track status and Orphan Drug designation in FSHD.

ACE-2494:
ACE-2494 is designed to have a systemic effect on muscle mass and strength for diseases that cause muscle weakness throughout the body. ACE-2494 utilizes the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

Enrollment and treatment are ongoing in the Phase 1 healthy volunteer trial with preliminary results expected in the first half of 2019.

Pulmonary Disease
Sotatercept:

Pulmonary Arterial Hypertension (PAH)

Sotatercept acts as a ligand trap for members of the TGF-beta superfamily directly involved in the BMP signaling pathway, which is proven critical for maintaining healthy pulmonary vasculature. In multiple preclinical studies in PAH, sotatercept significantly decreased pulmonary vessel muscularization, improved pulmonary arterial pressures, and decreased indicators of right heart failure.

The Company initiated the PULSAR Phase 2 trial in patients with PAH with preliminary results expected in the first half of 2020.

The Company plans to initiate an exploratory imaging study in Q1 2019 to provide additional understanding of endpoints in anticipation of a potential pivotal trial in the future.

In November 2018, the Company will host a PAH Research and Development Deep Dive event in New York City.

The event will include internal and external expert presentations to discuss disease background, current treatment landscape, key disease pathways including BMP signaling, Acceleron’s clinical development activities, and the latest sotatercept preclinical results.

Corporate Highlights

Robert K. Zeldin, M.D., was appointed Chief Medical Officer (CMO). He brings 20 years of industry experience and joined from Ablynx, where he served as Chief Medical Officer. Prior to Ablynx, Dr. Zeldin served in senior roles at Novartis, Merck, and the U.S. Food & Drug Administration’s Center for Biologics Evaluation and Research.

Janethe Pena, M.D., Ph.D., recently joined us as Vice President of Pulmonary to lead the company’s clinical development efforts in this area. Dr. Pena most recently served as Vice President and Group Head of Pulmonology Clinical Development at Bayer Pharmaceuticals. At Bayer, she was responsible for the pulmonary portfolio, including leading clinical trials with riociguat (Adempas) in different pulmonary hypertension indications and the overall program life cycle management.
Financial Results

Cash position – Cash, cash equivalents and investments as of June 30, 2018 were $332.3 million. As of December 31, 2017, the Company had cash, cash equivalents and investments of $372.9 million. The Company believes that existing cash, cash equivalents and investments will be sufficient to fund projected operating requirements into 2021.

Revenue – Collaboration revenue for the second quarter was $3.7 million. The revenue is all from Acceleron’s partnership with Celgene and is primarily related to expenses incurred by the Company in support of luspatercept.

Costs and expenses – Total costs and expenses for the second quarter were $33.6 million. This includes R&D expenses of $25.9 million and G&A expenses of $7.7 million.

Net loss – The Company’s net loss for the second quarter ended June 30, 2018 was $28.9 million.
Conference Call and Webcast
The Company will host a webcast and conference call to discuss its second quarter financial results for 2018 and provide an update on recent corporate activities on August 2, 2018, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and referring to the "Acceleron Second Quarter 2018 Earnings Call."

The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On August 2, 2018 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, reported that it will announce financial results for the second quarter ended June 30, 2018 on August 9, 2018 before the Nasdaq market open (Press release, Loxo Oncology, AUG 2, 2018, View Source [SID1234528319]). At 8:00 a.m. ET that day, Loxo Oncology management will host a conference call to discuss these financial results, in addition to recent updates on development and corporate activities.

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A live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.loxooncology.com. The conference call can be accessed by dialing (877) 930-8065 (domestic) or (253) 336-8041 (international) and referring to conference ID 7291605. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.