On February 25, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, reported a corporate update and reported financial results for the fourth quarter and year ended December 31, 2015 (Press release, Acceleron Pharma, FEB 25, 2016, View Source [SID:1234509189]).

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"We made significant progress over the past year in advancing our pipeline, as marked by successful clinical results across our programs and the recent launch of Phase 3 programs in MDS and beta-thalassemia with our partner Celgene," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "At the start of 2016, we raised $150 million in gross proceeds from an equity financing which will be used to fund our wholly owned programs, including our compounds for muscle diseases. Our locally acting muscle agent, ACE-083, showed unprecedented results in increasing muscle volume, and we plan to present new Phase 1 data later this year and initiate a Phase 2 trial in FSHD, a form of muscular dystrophy."

Added Dr. Knopf, "In late 2015, we introduced ACE-2494, a systemic muscle therapeutic created from our new IntelliTrap discovery platform. IntelliTrap is a powerful discovery engine, and we expect it to enable us to introduce a novel therapeutic into the clinic every 12 to 18 months."

2015 HIGHLIGHTS AND CURRENT UPDATES

DEVELOPMENT PROGRAMS

Hematology

Luspatercept in myelodysplastic syndromes (MDS)

Luspatercept is a protein therapeutic that increases hemoglobin levels and is being developed to help patients reduce or eliminate their need for red blood cell (RBC) transfusions

Initiated Phase 3 MEDALIST study in MDS with partner Celgene. MEDALIST is a 210-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in subjects with anemia due to very low, low, or intermediate-risk MDS with ring sideroblasts (≥ 15%) who require red blood cell transfusions.

Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 extension study showing that 50% of MDS patients achieved transfusion independence and 69% of patients achieved sustained increases in hemoglobin levels.

Expanded the Phase 2 MDS study with additional cohorts in lower risk MDS patients that are either erythropoietin-stimulating agent (ESA) treatment naïve or ring sideroblast negative.

Received FDA Fast Track Designation.

Luspatercept in beta-thalassemia

Initiated Phase 3 BELIEVE study in beta-thalassemia with partner Celgene. BELIEVE is a 300-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in adults who require regular red blood cell transfusions due to beta-thalassemia.

Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 studies showing luspatercept reduced transfusion burden, improved health-related quality of life measures, had beneficial effects on liver iron concentration and demonstrated a favorable safety profile.

Received FDA Fast Track Designation.

Muscle Diseases

ACE-083

Protein therapeutic designed to increase muscle mass and strength in the muscles in which it is administered

First-in-human Phase 1 study results showed an unprecedented 14.5% mean increase in muscle volume in the injected rectus femoris muscle of the quadriceps. The data were presented in the Late Breaking Clinical Trials Session of the 8th International Conference on Cachexia, Sarcopenia, and Muscle Wasting in December 2015.

Preclinical data in mice showed that ACE-083 produced significant increases in muscle mass in the injected muscle with no observed effect on either the uninjected contralateral muscle or on whole body mass. Increases in muscle mass were associated with a significant increase in muscle force and power. The data were presented at the 20th International Annual Congress of the World Muscle Society in October 2015.

ACE-2494

Systemic muscle therapeutic designed to increase muscle mass and strength in a range of muscle diseases

Introduced ACE-2494, Acceleron’s first IntelliTrap molecule. Preclinical data in mice presented at the 2015 World Muscle Society Congress showed that after 4 weeks of treatment, ACE-2494 generated substantial dose-dependent mean increases in muscle mass: 41% in rectus femoris, 53% in gastrocnemius, and 87% in pectoralis.
Oncology

Dalantercept in renal cell carcinoma (RCC)

Protein therapeutic that inhibits angiogenesis and is being developed in combination with approved VEGF-based anti-angiogenesis compounds to improve patient outcomes.

Enrollment is ongoing in Part 2 of the Phase 2 DART study, a randomized, double-blind study of dalantercept plus axitinib compared to placebo plus axitinib in patients with advanced renal cell carcinoma.

Results from Part 1 of the DART study with dalantercept plus axitinib demonstrated a median progression free survival of 8.3 months across all dose levels tested versus the historic control of 4.8 months for axitinib alone. The data were presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and ASCO (Free ASCO Whitepaper) 2015 Genitourinary Cancers Symposium.

Received FDA Fast Track Designation for dalantercept in combination with axitinib for the treatment of patients with advanced RCC following treatment with one anti-angiogenic agent.

Nephrology

Sotatercept in chronic kidney disease

Protein therapeutic that has effects on fibrosis, vascular calcification, bone mineral density and red blood cell (RBC) levels

Presented preliminary data from ongoing Phase 2a clinical studies of sotatercept in End-Stage Kidney Disease patients, including effects on hemoglobin, vascular calcification, bone mineral density and safety and tolerability at the American Society of Nephrology Kidney Week in October 2015.

Acceleron and Celgene assessing the opportunity for the development of sotatercept in the pre-dialysis chronic kidney disease (CKD) setting.

RESEARCH AND DEVELOPMENT

Introduced the Company’s IntelliTrap platform for discovery of selective and novel compounds targeting the transforming growth factor-beta superfamily of proteins.

Identified ACE-2494 as the first compound to be developed from the platform and is expected to be Acceleron’s fifth internally discovered therapeutic to enter the clinic.

Acceleron plans to have a new internally discovered compound enter the clinic every 12 to 18 months.

CORPORATE UPDATES

Raised $150 million in gross proceeds in an underwritten public offering of common stock in January 2016.

Hosted first Research & Development Day in October 2015. Company executives and a panel of outside experts briefed the investment community on Acceleron’s clinical programs in MDS, beta-thalassemia, cancer and muscular dystrophies.

UPCOMING 2016 PROGRAM MILESTONES AND EVENTS

We anticipate the following milestones and events in 2016:

Hematology

Luspatercept in MDS

Will update long-term treatment results at major medical conferences.

Initial data on ring sideroblast negative and ESA treatment naïve (front-line) patients (YE 2016).
Luspatercept in beta-thalassemia

Update long-term treatment results at major medical conferences.

Muscle Diseases

ACE-083

Present Phase 1 data from two new cohorts (Mid 2016).

Initiate Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD) patients (H2 2016).
ACE-2494

Submit IND for first-in-human study for ACE-2494 (YE 2016).
Oncology

Dalantercept in RCC

Present preliminary Part 2 DART study results (dalantercept in combination with axitinib) (YE 2016).
Nephrology

Sotatercept in CKD

Provide an update on development strategy (H2 2016).
Financial Results

Cash Position – Cash, cash equivalents and investments were $136.0 million as of December 31, 2015. Net cash used in operating activities in 2015 was $44.2 million. We believe that our existing cash, cash equivalents and investments, including the net proceeds of $140.4 million from our January 2016 offering, will be sufficient to fund our projected operating requirements into the second half of 2019.

Revenue – Collaboration revenue for the year was $18.1 million. This includes license and milestone amortization of $1.2 million and cost sharing reimbursement revenue from our Celgene partnership of $16.9 million related to expenses incurred by the Company in support of our partnered programs.

Costs and expenses – Total costs and expenses for the year were $79.0 million. This includes R&D expenses of $58.4 million and G&A expenses of $20.6 million.

Net Loss – The Company’s net loss for the year ended December 31, 2015 was $63.9 million.

On February 25, 2016 Emergent BioSolutions Inc. (NYSE:EBS) reported financial results for the quarter and twelve months ended December 31, 2015 (Press release, Emergent BioSolutions, FEB 25, 2016, View Source;p=RssLanding&cat=news&id=2143557 [SID:1234509229]).

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2015 FINANCIAL HIGHLIGHTS

Total revenues: Q4 2015 of $168.1 million, +14% Y/Y; twelve months 2015 of $522.8 million, +16% Y/Y;
GAAP net income: Q4 2015 of $33.3 million, or $0.71 per diluted share, +11% Y/Y; twelve months 2015 of $62.9 million, or $1.41 per diluted share, +71% Y/Y;

Adjusted net income: Q4 2015 of $37.5 million, or $0.78 per diluted share, +8% Y/Y; twelve months 2015 of $75.6 million, or $1.60 per diluted share, +40% Y/Y;

EBITDA: Q4 2015 of $58.5 million, or $1.22 per diluted share, +10% Y/Y; twelve months 2015 of $130.1 million, or $2.75 per diluted share, +41% Y/Y; and

Adjusted EBITDA: Q4 2015 of $61.7 million, or $1.28 per diluted share, +7% Y/Y; twelve months 2015 of $137.4 million, or $2.91 per diluted share, +30% Y/Y.

2016 FORECAST:

Full Year: revenue of $600 to $630 million; GAAP net income of $75 to $85 million, non-GAAP adjusted net income of $90 to $100 million, and EBITDA of $150 to $160 million

1Q 2016: revenue of $105 to $120 million

"Our strong fourth quarter in 2015 continued our history of growth over the last three years both financially and operationally. Since 2012, we nearly doubled our revenues to over $520 million, achieved a net income CAGR of 38% and expanded our portfolio to nine products," said Daniel J. Abdun-Nabi, president and chief executive officer of Emergent BioSolutions. "2016 will bring a renewed focus on addressing public health threats as we spin off Aptevo Therapeutics, our biosciences business, and enter our next phase of growth. We will continue to establish ourselves as a market leader with a global impact and work to achieve our vision of protecting and enhancing 50 million lives by 2025."

2015 BUSINESS ACCOMPLISHMENTS

Announced plan to implement tax-free spin-off of Aptevo Therapeutics (the Company’s Biosciences business) into a separate, publicly traded company, targeted for mid-2016;

Launched a new platform technology, Emergard, the Company’s military-grade auto-injector device for chemical threats being sold in international markets;

Received three approvals from the U.S. Food and Drug Administration (FDA):

Expansion of the BioThrax (Anthrax Vaccine Adsorbed) label to include post-exposure prophylaxis (PEP) against anthrax disease; the first vaccine to be licensed using the FDA Animal Rule,

IXINITY, a recombinant factor IX treatment for Hemophilia B, and

Anthrasil, an immune globulin for the treatment for inhalational anthrax.

Secured over $95 million in new multi-year contract and grant funding, including the following:

$20 million in multiple contracts with BARDA to manufacture Ebola monoclonal antibodies, including the Company’s first awarded task order under the Center for Innovation in Advanced Development and Manufacturing program,

A $44 million CDC contract to further supply the strategic national stockpile with the Company’s Vaccinia Immune Globulin product, and

A $31 million BARDA contract for the advanced development of NuThrax (anthrax vaccine adsorbed with CPG 7909 adjuvant), the Company’s next generation anthrax vaccine candidate.

Initiated a Phase 1 clinical trial for MOR209/ES414, an immunotherapeutic protein built on our ADAPTIR platform technology and targeting prostate cancer, which is being developed in collaboration with MorphoSys AG; and

Continued progress towards achieving licensure of Building 55.

2015 FINANCIAL PERFORMANCE

(I) Quarter Ended December 31, 2015 (unaudited)

Revenues

Product Sales

For Q4 2015, product sales were $132.6 million, an increase of 17% as compared to 2014. The increase primarily reflects increased sales of BioThrax during the quarter.

(in millions) Three Months Ended
December 31,
2015 2014 % Change
Product Sales
BioThrax $ 111.9 $ 87.9 27 %
Other biodefense 12.5 14.5 (13 )%
Total Biodefense $ 124.4 $ 102.3 22 %

Total Biosciences $ 8.2 11.0 (25 )%
Total Product Sales $ 132.6 $ 113.4 17 %

Contract Manufacturing

For Q4 2015, revenue from the Company’s contract manufacturing operations was $10.5 million, an increase of 10% as compared to 2014. The increase was primarily due to the timing of fill/finish services to third parties.

Contracts, Grants and Collaborations
For Q4 2015, contracts, grants and collaborations revenue was $24.9 million, unchanged as compared to 2014.

Operating Expenses

Cost of Product Sales and Contract Manufacturing

For Q4 2015, cost of product sales and contract manufacturing was $39.8 million, an increase of 22% as compared to 2014. The increase was primarily attributable to increased sales of BioThrax to the CDC.

Research and Development

For Q4 2015, gross research and development (R&D) expenses were $32.5 million, a decrease of 17% as compared to 2014. The decrease primarily reflects lower contract service costs associated with product candidates and technology platform development activities associated with the Biosciences division.

For Q4 2015, net R&D expenses were $7.6 million, a decrease of 46% as compared to 2014, reflecting a decrease in unfunded development spending in our Biosciences division, including spending on IXINITY, a product that we launched in Q2 2015. Net R&D expenses, which are more representative of the Company’s actual out-of-pocket investment in product development, are calculated as gross research and development expenses less contracts, grants and collaboration revenues.

(in millions) Three Months Ended
December 31,
2015 2014 % Change
Research and Development Expenses (Gross) $ 32.5 $ 39.0 (17 )%
Adjustments:
Contracts, grants and collaborations revenues 24.9 25.0 N/A
Net Research and Development Expenses $ 7.6 $ 14.0 (46 )%

Selling, General and Administrative

For Q4 2015, selling, general and administrative expenses were $46.0 million, an increase of 44% as compared to 2014. The increase was primarily attributable to a one-time $3.5 million reserve for potential write-off of accounts receivable within the Biosciences segment, a charge to write-off certain obsolete fixed assets, and increased information technology costs associated with the implementation of a new ERP system, as well as costs associated with the spin-off of Aptevo Therapeutics and professional services to support the Company’s strategic growth initiatives.

Net Income

For Q4 2015, GAAP net income was $33.3 million, an increase of 11% as compared to 2014. For Q4 2015 and 2014, GAAP net income per diluted share is computed using the if-converted method. This method requires GAAP net income to be adjusted to reflect the impact of interest expense and amortization of debt issuance cost, both net of tax, associated with the Company’s 2.875% Convertible Senior Notes due 2021. As a result, GAAP net income per diluted share for Q4 2015 is adjusted in the amount of $0.9 million, from $33.3 million to $34.2 million, and diluted shares outstanding were 48.1 million. GAAP net income per diluted share for Q4 2014 is adjusted in the amount of $0.6 million, from $30.1 million to $30.7 million, and diluted shares outstanding were 46.4 million.

(II) Twelve Months Ended December 31, 2015 (unaudited)

Revenues

Product Sales

For the twelve months of 2015, product sales were $356.9 million, an increase of 14% as compared to 2014. The increase primarily reflects increased sales of BioThrax in 2015.

(in millions) Twelve Months Ended
December 31,
2015 2014 % Change
Product Sales
BioThrax $ 293.9 $ 245.9 20 %
Other biodefense 35.0 35.9 (3 )%
Total Biodefense $ 328.9 $ 281.8 17 %

Total Biosciences $ 28.0 $ 30.1 (7 )%
Total Product Sales $ 356.9 $ 311.9 14 %

Contract Manufacturing
For the twelve months of 2015, revenue from the Company’s contract manufacturing operations was $43.0 million, an increase of 39% as compared to 2014. The increase was primarily due to a full year of revenues from the Company’s fill/finish facility in Baltimore, plasma based manufacturing from the Company’s Winnipeg facility and contract manufacturing services related to the production of an MVA Ebola vaccine candidate.

Contracts, Grants and Collaborations

For the twelve months of 2015, contracts, grants and collaborations revenue was $122.9 million, an increase of 15% as compared to 2014. The increase was primarily due to development funding for Anthrasil and for our CIADM program.

Operating Expenses

Cost of Product Sales and Contract Manufacturing

For the twelve months of 2015, cost of product sales and contract manufacturing was $124.3 million, an increase of 5% as compared 2014. The increase was primarily attributable to the increase in the number of BioThrax doses delivered to the CDC.

Research and Development

For the twelve months of 2015, gross R&D expenses were $154.0 million, an increase of 2% as compared to 2014. The increase was primarily attributable to higher contract service costs for product candidates and manufacturing development in the Biodefense segment.

Net R&D expenses for the twelve months of 2015 were $31.1 million, a decrease of 29% as compared to 2014, reflecting a decrease in unfunded development spending in our Biosciences division, including spending on IXINITY.

(in millions) Twelve Months Ended
December 31,
2015 2014 % Change
Research and Development Expenses (Gross) $ 154.0 $ 150.8 2 %
Adjustments:
Contracts, grants and collaboration revenues 122.9 107.3 15 %
Net Research and Development Expenses $ 31.1 $ 43.5 (29 )%

Selling, General and Administrative

For the twelve months of 2015, selling, general and administrative expenses were $148.5 million, an increase of 21% as compared to 2014. The increase was primarily attributable to additional post-acquisition selling, general and administrative costs largely associated with the operations acquired in Q1 2014, including IXINITY launch costs, as well as costs associated with the spin-off of Aptevo Therapeutics and costs associated with professional services to support the Company’s strategic growth initiatives.

Net Income

For the twelve months of 2015, GAAP net income was $62.9 million, an increase of 71% as compared to 2014. For the twelve months of 2015 and 2014, GAAP net income per diluted share is computed using the if-converted method. This method requires GAAP net income to be adjusted to reflect the impact of interest expense and amortization of debt issuance cost, both net of tax, associated with the Company’s 2.875% Convertible Senior Notes due 2021. As a result, GAAP net income per diluted share for the twelve months of 2015 is adjusted in the amount of $3.9 million, from $62.9 million to $66.8 million, and diluted shares outstanding were 47.3 million. GAAP net income per diluted share for the twelve months of 2014 is adjusted in the amount of $3.6 million, from $36.7 million to $40.3 million, and diluted shares outstanding were 45.8 million.

2016 FINANCIAL OUTLOOK

(I) Full Year 2016

For the full year of 2016, the Company reaffirms its forecast for total revenues of $600 to $630 million, driven by growth in BioThrax sales of $305 to $320 million, continued domestic and international sales of the other Biodefense division products, and continued robust development funding through contracts and grants revenues. The Company also forecasts full year 2016 GAAP net income of $75 to $85 million, non-GAAP adjusted net income of $90 to $100 million, and EBITDA of $150 to $160 million (see "Reconciliation of GAAP Net Income to Adjusted Net Income and EBITDA" for a definition of terms and a reconciliation table). The Company’s outlook for 2016 includes the impact of a successful spin-off of Aptevo Therapeutics in mid-2016 and continuous delivery of BioThrax to the CDC under an anticipated follow-on, multiyear procurement contract, but does not include any estimates for BioThrax deliveries from Building 55, the Company’s large scale BioThrax manufacturing facility, or any estimates for potential new corporate development or other M&A transactions.

(II) Q1 2016

For the first quarter of 2016, the Company reaffirms its forecast for total revenues of $105 to $120 million.

RECONCILIATION OF GAAP NET INCOME TO ADJUSTED NET INCOME, EBITDA AND ADJUSTED EBITDA

This press release contains three financial measures (Adjusted Net Income, EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization), and adjusted EBITDA) that are considered "non-GAAP" financial measures under applicable Securities & Exchange Commission rules and regulations. These non-GAAP financial measures should be considered supplemental to and not a substitute for financial information prepared in accordance with generally accepted accounting principles. The Company’s definition of these non-GAAP measures may differ from similarly titled measures used by others. Adjusted Net Income adjusts for specified items that can be highly variable or difficult to predict, or reflect the non-cash impact of charges resulting from purchase accounting. EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and provision for income taxes. Adjusted EBITDA also excludes specified items that can be highly variable and the non-cash impact of certain purchase accounting adjustments. The Company views these non-GAAP financial measures as a means to facilitate management’s financial and operational decision-making, including evaluation of the Company’s historical operating results and comparison to competitors’ operating results. These non-GAAP financial measures reflect an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results and the reconciliations to the corresponding GAAP financial measure, may provide a more complete understanding of factors and trends affecting the Company’s business.

The determination of the amounts that are excluded from these non-GAAP financial measures are a matter of management judgment and depend upon, among other factors, the nature of the underlying expense or income amounts. Because non-GAAP financial measures exclude the effect of items that will increase or decrease the Company’s reported results of operations, management strongly encourages investors to review the Company’s consolidated financial statements and publicly filed reports in their entirety.
Reconciliation of GAAP Net Income to Adjusted Net Income

(in millions, except per share value) Three Months Ended
December 31,
2015 2014 Source
GAAP Net Income $ 33.3 $ 30.1 NA
Adjustments:
Spin-off and acquisition-related costs
(transaction & integration) 2.0 0.6 SG&A
Non-cash amortization charges 2.7 2.3 COGS, SG&A,
Other Income
Impact of purchase accounting on inventory step-up – 1.0 COGS
Restructuring activities 1.2 2.6 SG&A
Tax effect (1.8 ) (2.0 ) NA
Total Adjustments 4.2 4.5 NA
Adjusted Net Income
$
37.5
$
34.6
NA
Adjusted Net Income per Diluted Share $ 0.78 $ 0.75

(in millions, except per share value) Twelve Months Ended
December 31,
2015 2014 Source
GAAP Net Income $ 62.9 $ 36.7 NA
Adjustments:
Spin-off and acquisition-related costs
(transaction & integration) 5.5 8.1 SG&A
Non-cash amortization charges 10.8 9.5 COGS, SG&A,
Other Income
Write-off of syndicated loans – 1.8 Other Income
Impact of purchase accounting on inventory step-up 0.6 3.0 COGS
Restructuring activities 1.2 2.6 SG&A
Tax effect (5.4 ) (7.5 ) NA
Total Adjustments 12.7 17.5 NA
Adjusted Net Income
$
75.6
$
54.2
NA
Adjusted Net Income per Diluted Share $ 1.60 $ 1.18

Reconciliation of GAAP Net Income to EBITDA and Adjusted EBITDA

(in millions, except per share value) Three Months Ended
December 31,
2015 2014
GAAP Net Income $ 33.3 $ 30.1
Adjustments:
+ Depreciation & Amortization 9.1 7.8
+ Provision For Income Taxes 14.5 14.2
+ Total Interest Expense 1.6 1.2
Total Adjustments 25.2 23.2
EBITDA
$
58.5
$
53.3

EBITDA per Diluted Share $ 1.22 $ 1.15
Additional Adjustments:
+ Spin-off and acquisition-related costs
(transaction & integration) 2.0 0.6
+ Impact of purchase accounting on inventory step-up – 1.0
+ Restructuring activities 1.2 2.6
Total Additional Adjustments 3.2 4.2
Adjusted EBITDA
$
61.7
$
57.5

Adjusted EBITDA per Diluted Share $ 1.28 $ 1.24

(in millions, except per share value) Twelve Months Ended
December 31,
2015 2014
GAAP Net Income $ 62.9 $ 36.7
Adjustments:
+ Depreciation & Amortization 33.8 31.0
+ Provision Income Taxes 26.9 16.3
+ Total Interest Expense 6.5 8.2
Total Adjustments 67.2 55.5
EBITDA
$
130.1
$
92.2

EBITDA per Diluted Share $ 2.75 $ 2.01
Additional Adjustments:
+ Spin-off and acquisition-related costs
(transaction & integration) 5.5 8.1
+ Impact of purchase accounting on inventory step-up 0.6 3.0
+ Restructuring activities 1.2 2.6
Total Additional Adjustments 7.3 13.7
Adjusted EBITDA
$
137.4
$
105.9

Adjusted EBITDA per Diluted Share $ 2.91 $ 2.31

On February 25, 2016 Alkermes plc (NASDAQ: ALKS) reported financial results for the twelve months ended Dec. 31, 2015 and provided financial expectations for 2016 (Press release, Alkermes, FEB 25, 2016, View Source;p=RssLanding&cat=news&id=2143181 [SID:1234509192]).

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"Alkermes has a diversified CNS business poised for significant growth over the coming years. In 2015, we continued to successfully execute on our business plan, highlighted by the robust revenue growth of VIVITROL and the launch of our novel, long-acting antipsychotic ARISTADA for the treatment of schizophrenia," said Richard Pops, Chief Executive Officer of Alkermes. "Looking ahead to 2016, we expect to achieve continued revenue growth and to make significant advances across our pipeline. We will continue to enroll the pivotal clinical studies of ALKS 3831 for schizophrenia and ALKS 8700 for multiple sclerosis; obtain the first clinical data for ALKS 7119, our CNS candidate for Alzheimer’s agitation, and RDB 1450, our immuno-oncology candidate; and report results from the FORWARD-5 efficacy study of ALKS 5461 for major depressive disorder by year-end."

"Our financial results in 2015 were driven by the strong performance of VIVITROL, the approval and launch of ARISTADA into a rapidly growing long-acting antipsychotic market, and the continued strength of our base business," commented James Frates, Chief Financial Officer of Alkermes. "In 2016, we expect our business to continue to grow, led by VIVITROL and ARISTADA. Together with our solid royalty and manufacturing base business, these proprietary products are expected to drive revenue growth of 15 to 20 percent."

Quarter Ended Dec. 31, 2015 Financial Highlights

Total revenues for the quarter were $163.1 million. This compared to $175.2 million for the same period in the prior year, or $156.7 million excluding $18.5 million of revenues from the products associated with the Gainesville manufacturing facility that was divested in April 2015 ("the Gainesville Divestiture").

Net loss according to generally accepted accounting principles in the U.S. (GAAP) was $69.4 million, or a basic and diluted GAAP loss per share of $0.46, for the quarter and reflected increased investment in the company’s advancing late-stage pipeline and commercial infrastructure. This compared to GAAP net income of $30.5 million, or a basic GAAP earnings per share (EPS) of $0.21 and a diluted GAAP EPS of $0.20 for the same period in the prior year, or GAAP net income of $25.2 million, or a basic EPS of $0.17 and a diluted EPS of $0.16, excluding $5.3 million of GAAP net income related to the Gainesville Divestiture.

Non-GAAP net loss was $22.6 million, or a non-GAAP basic and diluted loss per share of $0.15 for the quarter. This compared to non-GAAP net income of $16.8 million, or a non-GAAP basic and diluted EPS of $0.11 for the same period in the prior year, or non-GAAP net income of $9.0 million, or a non-GAAP basic and diluted EPS of $0.06, excluding $7.8 million of non-GAAP net income related to the Gainesville Divestiture.

Quarter Ended Dec. 31, 2015 Financial Results

Revenues

Net sales of VIVITROL were $38.2 million, compared to $29.7 million for the same period in the prior year, representing an increase of 29%. On a unit basis, sales grew 43% compared to the same period in the prior year. Compared to the third quarter of 2015, VIVITROL grew 7% on a unit basis, driven by increased adoption by treatment systems, while net sales grew 1% as the company increased accruals for Medicaid rebates to reflect the increasing volume of VIVITROL units covered by Medicaid.

Net sales of ARISTADA were $4.6 million, following its launch in October 2015.

Manufacturing and royalty revenues from RISPERDAL CONSTA, INVEGA SUSTENNA/XEPLION and INVEGA TRINZA were $75.1 million, compared to $70.3 million for the same period in the prior year.

Manufacturing and royalty revenues from AMPYRA/FAMPYRA1 were $19.1 million, compared to $24.3 million for the same period in the prior year, due primarily to the timing of shipments.

Royalty revenue from BYDUREON was $12.2 million, compared to $9.8 million for the same period in the prior year.

Costs and Expenses

Operating expenses were $230.2 million for the quarter ended Dec. 31, 2015, reflecting increased investment in the company’s development pipeline and the launch of ARISTADA. This compared to $190.8 million for the same period in the prior year, or $177.4 million excluding $13.4 million of operating expenses related to the Gainesville Divestiture.

Calendar Year 2015 Financial Highlights

Total revenues were $628.3 million in calendar 2015, which included VIVITROL net sales of $144.4 million and ARISTADA net sales of $4.6 million. This compared to total revenues of $618.8 million for calendar 2014. Please see the tables at the end of this press release for a detailed breakdown of the revenues from our key commercial products. Excluding the Gainesville Divestiture, 2015 total revenues were $608.6 million in calendar 2015, compared to total revenues of $545.8 million in calendar 2014.

GAAP net loss was $227.2 million, or a basic and diluted GAAP loss per share of $1.52, for calendar 2015 and reflected increased investment in the company’s advancing late-stage pipeline and the launch of ARISTADA in October 2015. This compared to a GAAP net loss of $30.1 million, or a basic and diluted GAAP loss per share of $0.21, for calendar 2014. Excluding the Gainesville Divestiture, GAAP net loss was $231.7 million, or a basic and diluted loss per share of $1.55, in calendar 2015, compared to a GAAP net loss of $53.7 million, or a basic and diluted GAAP loss per share of $0.37, in calendar 2014.

Non-GAAP net loss was $53.2 million, or a non-GAAP basic and diluted loss per share of $0.36, for calendar 2015. This compared to non-GAAP net income of $54.6 million, or a non-GAAP basic EPS of $0.38 and a non-GAAP diluted EPS of $0.35, for calendar 2014. Excluding the Gainesville Divestiture, non-GAAP net loss was $59.5 million, or a non-GAAP basic and diluted loss per share of $0.40, in calendar 2015, compared to a non-GAAP net income of $19.4 million, or a basic and diluted EPS of $0.13, in calendar 2014.

At Dec. 31, 2015, Alkermes recorded cash and total investments of $798.8 million, compared to $801.6 million at Dec. 31, 2014. At Dec. 31, 2015, the company’s total debt outstanding was $349.9 million.

Financial Expectations for 2016

The following outlines the company’s financial expectations for 2016, which include continued investment in the pipeline and a full year of expenses related to the ARISTADA commercial launch. The following statements are forward-looking, and actual results may differ materially. Please see "Note Regarding Forward-Looking Statements" at the end of this press release for risks that could cause results to differ materially from these forward-looking statements.

Revenues: The company expects total revenues to range from $700 million to $750 million, a 15% to 20% increase from 2015 excluding revenues derived from the Gainesville Divestiture, driven by continuing growth of VIVITROL and the ongoing launch of ARISTADA. Included in this total revenue expectation, Alkermes expects VIVITROL net sales to range from $180 million to $200 million. For ARISTADA, the company expects to provide net product revenue guidance during 2016 after gaining additional experience from the launch.

Cost of Goods Manufactured and Sold: The company expects cost of goods manufactured and sold to range from $125 million to $135 million.

Research and Development (R&D) Expenses: The company expects R&D expenses to range from $370 million to $400 million.
Selling, General and Administrative (SG&A) Expenses: The company expects SG&A expenses to range from $360 million to $390 million.

Amortization of Intangible Assets: The company expects amortization of intangibles to be approximately $60 million.

Net Interest Expense: The company expects net interest expense to be approximately $10 million.

Income Tax Expense: The company expects income tax expense of up to $10 million.

GAAP Net Loss: The company expects a GAAP net loss to be in the range of $225 million to $255 million, or a basic and diluted loss per share of $1.48 to $1.68, based on a weighted average basic and diluted share count of approximately 152 million shares outstanding.

Non-GAAP Net Loss: The company expects a non-GAAP net loss to be in the range of $25 million to $55 million, and non-GAAP basic and diluted loss per share to be between $0.16 and $0.36.

Capital Expenditures: The company expects capital expenditures to be approximately $45 million.

On February 25, 2016 Geron Corporation (Nasdaq: GERN) reported financial results for the fourth quarter and year ended December 31, 2015 and recent events (Filing, Q4/Annual, Geron, 2015, FEB 25, 2016, View Source [SID:1234509230]).

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Fourth Quarter and Year-End 2015 Results

For 2015, the company reported net income of $46,000, or $0.00 per share, compared to a net loss of $35.7 million, or $(0.23) per share, for 2014. For the fourth quarter of 2015, the company reported a net loss of $8.5 million, or $(0.05) per share, compared to a net loss of $8.9 million, or $(0.06) per share, for the comparable 2014 period. The company ended 2015 with $146.7 million in cash and investments.

Revenues for 2015 were $36.4 million compared to $1.2 million for 2014. Revenues for the fourth quarter of 2015 were $220,000 compared to $178,000 for the comparable 2014 period. Revenues for 2015 included the full recognition of the $35.0 million upfront payment from Janssen Biotech, Inc. (Janssen) as collaboration revenue upon the company’s transfer of the imetelstat license rights and completion of technology transfer-related activities outlined under the imetelstat collaboration agreement with Janssen in the third quarter of 2015. The upfront cash payment was received in December 2014 and recorded as deferred revenue at that time.

Total operating expenses for 2015 were $36.9 million compared to $37.5 million for 2014. Total operating expenses for the fourth quarter of 2015 were $8.9 million compared to $9.2 million for the comparable 2014 period. Operating expenses for 2015 included restructuring charges of $1.3 million in connection with the company’s organizational resizing announced in March 2015.

Research and development expenses for 2015 were $17.8 million compared to $20.7 million for 2014. Research and development expenses for the fourth quarter of 2015 were $4.0 million compared to $4.4 million for the comparable 2014 period. The decrease in research and development expenses in 2015 compared to 2014 primarily reflects the net result of lower personnel related costs due to the organizational resizing and reduced manufacturing costs for imetelstat drug product, partially offset by higher costs for the clinical development of imetelstat in collaboration with Janssen.

General and administrative expenses for 2015 were $17.8 million compared to $16.8 million for 2014. General and administrative expenses for the fourth quarter of 2015 were $4.9 million compared to $4.8 million for the comparable 2014 period. The increase in general and administrative expenses in 2015 compared to 2014 primarily reflects higher non-cash stock-based compensation expense.

Interest and other income for 2015 was $677,000 compared to $373,000 for 2014. Interest and other income for the fourth quarter of 2015 was $196,000 compared to $100,000 for the comparable 2014 period. The increase in interest and other income for 2015 compared to 2014 primarily reflects higher cash and investment balances with the receipt of the $35.0 million upfront payment from Janssen and higher yields on the company’s marketable securities portfolio.

Company Events

Publications and Presentations

● The New England Journal of Medicine (NEJM) published two papers in which data from clinical studies of two hematologic myeloid malignancies, essential thrombocythemia (ET) and myelofibrosis (MF), suggest imetelstat may have disease-modifying activity by inhibiting the malignant progenitor cell clones responsible for the underlying diseases in a relatively select manner. The papers are available online in the September 3rd issue at www.NEJM.org.

● Three presentations describing clinical and non-clinical data on imetelstat were made at the 57th annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015:

○ Telomerase Inhibitor Imetelstat Therapy in Refractory Anemia with Ring Sideroblasts with or without Thrombocytosis (Abstract #55; oral presentation)

Data were presented for safety and efficacy as of the May 10, 2015 data cut. Nine patients with a form of the myelodysplastic syndromes (MDS) known as refractory anemia with ring sideroblasts (MDS-RARS) were enrolled in the study cohort, classified as having either intermediate-1 or intermediate-2 risk disease by the International Prognostic Scoring System (IPSS). Six of nine (66.7%) patients had prior treatment with erythropoiesis stimulating agents (ESAs).

Three of the eight (37.5%) patients who were dependent on red blood cell transfusions at study entry became transfusion independent, defined as not requiring transfusions for at least eight weeks. The median duration of transfusion independence was 28 weeks (range: nine weeks to 37 weeks).

Adverse events were similar in nature to the adverse events reported in the MF clinical study published in the NEJM.

○ Dynamics of Mutations in Patients with ET Treated with Imetelstat (Abstract #57; oral presentation)

Data from further mutational analyses of patient samples from the clinical study in ET showed that imetelstat treatment suppressed allele burdens of multiple gene mutations in addition to the JAK2V617F, CALR and MPL mutations.

○ Activity of the Telomerase Inhibitor GRN163L (Imetelstat) on Acute Myeloblastic Leukemia Blasts Is Enhanced by DNA Methyltransferase Inhibitors Irrespective of TERT Promoter Methylation Status (Abstract #1267; poster presentation)

Data from an in vitro study showed that imetelstat has activity against samples derived from patients with high risk leukemias, and that activity was enhanced by the demethylating agent 5-azacytadine, which is currently used in the treatment of some patients with high risk MDS or acute myeloid leukemia.

Clinical Development by Janssen

● IMbarkTM. In September 2015, the first patient was dosed in a Phase 2 clinical trial to evaluate imetelstat in patients with MF. The trial, referred to as IMbarkTM, will assess the efficacy, safety and tolerability of two dose levels of single-agent imetelstat and is designed to enroll approximately 200 patients (approximately 100 patients per dosing arm) with intermediate-2 or high risk MF, as defined by the Dynamic International Prognostic Scoring System, who have relapsed after or are refractory to Janus Kinase (JAK) inhibitor treatment. Patients will be assigned randomly, on a 1:1 ratio, to one of two dosing arms – 9.4 mg/kg or 4.7 mg/kg every three weeks. Patients will be blinded to the dosing arm assignment. Dose reductions for adverse events are allowed and will follow protocol-specified algorithms. An internal review of data from the trial is planned after approximately 20 patients per arm have been randomized and followed for at least 12 weeks in order to assess the adequacy of one or both of the initial dosing arms. As a result of this internal review, which is expected to occur in the second half of 2016, one or both dosing arms could continue as planned, be stopped or modified, or alternative doses could be selected.

The co-primary efficacy endpoints for IMbarkTM are spleen response rate and symptom response rate. Spleen response rate is defined as the percentage of patients who achieve ≥ 35% reduction in spleen volume from baseline at the Week 24 visit, as measured by imaging scans and assessed at a central imaging facility and by an Independent Review Committee. Symptom response rate is defined as the percentage of patients who have ≥ 50% reduction in Total Symptom Scores from baseline at the Week 24 visit, based on patient-reported outcomes on a modified Myelofibrosis Symptom Assessment Form version 2.0 electronic diary. The primary efficacy analysis of the co-primary endpoints will occur after all treated patients have been followed for at least 24 weeks, and the data cut for this analysis is expected to occur in the second half of 2017. Formal clinical data from this trial is expected to be presented at a medical conference to be determined in the future.

Further information about the trial, including participating medical centers around the world, can be found at View Source

● IMergeTM. In January 2016, the first patient was dosed in a Phase 2/3 clinical trial to evaluate imetelstat in patients with MDS. The trial, referred to as IMergeTM, will evaluate imetelstat in transfusion dependent patients with IPSS Low or Intermediate-1 risk MDS who have relapsed after or are refractory to prior treatment with an ESA.

As designed, the trial consists of two parts, and a total of approximately 200 patients are expected to be enrolled. Part 1 of the trial is planned as a Phase 2, open-label, single-arm design to assess the efficacy and safety of imetelstat. Up to 30 patients are expected to be enrolled in Part 1, all of whom will receive imetelstat and be followed for safety, hematologic improvement and reduction in transfusion requirement. Before proceeding to Part 2, the data from Part 1 must support a positive assessment of the benefit/risk profile of imetelstat in these patients. The internal review of data from Part 1 to support advancing to Part 2 is expected to occur in the second half of 2016. Part 2 of the trial is planned as a Phase 3 double-blind, randomized, placebo-controlled design to compare the efficacy of imetelstat against placebo. Approximately 170 patients are expected to be enrolled in Part 2, who will be assigned randomly, in a 2:1 ratio, to receive either imetelstat or placebo.

The primary efficacy endpoint is designed to be the rate of red blood cell transfusion-independence lasting at least eight weeks, defined as the proportion of patients without any red blood cell transfusion during any consecutive eight weeks since entry to the trial. A primary efficacy analysis is planned to occur 12 months after the last patient is enrolled.

Further information about the trial, including participating medical centers around the world, can be found at View Source

Regulatory Designations

● The United States Food and Drug Administration has granted orphan drug designation to imetelstat for the treatment of MF and for the treatment of MDS. In addition, the European Medicine Agency has granted orphan drug designation to imetelstat for the treatment of MF.

On February 25, 2016 AstraZeneca and Acerta Pharma BV, a company in which AstraZeneca has a majority equity investment, reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) adopted three positive opinions recommending acalabrutinib (ACP-196) for designation as an orphan medicinal product (Filing, 6-K, AstraZeneca, FEB 25, 2016, View Source [SID:1234509193]). The three positive opinions are for the treatment of chronic lymphocytic leukaemia (CLL) / small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL) and lymphoplasmacytic lymphoma (Waldenström’s macroglobulinaemia, MG).

Sean Bohen, Executive Vice-President of Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "Today’s three positive opinions recommending acalabrutinib for designation as an orphan medicinal product are important milestones. They reinforce the strategic rationale for our investment in Acerta, demonstrating clear progress in developing a potential best-in-class medicine that could transform treatment for patients across a range of blood cancers. The positive opinions underscore the continued need for the development of new therapies in these serious and life-threatening conditions and support our commitment to bring new medicines to patients as quickly as possible."

CLL is a slow-growing blood and bone marrow cancer that accounts for approximately one in four cases of leukaemia.i,ii Most CLL patients experience disease progression despite initial response to therapy and may require additional treatment. iii SLL is a clinically similar disease localized to the lymph nodes.iv

MCL is an aggressive non-Hodgkin’s lymphoma (NHL) typically associated with very poor outcomes.v MCL represents around 5% of all NHLs.vi The name comes from the fact that the tumour cells originate in the mantle zone of the lymph node. vi
WG is a rare, slow-growing cancer predominantly affecting older individuals, with a mean age of 60 at diagnosis.vii, viii and median survival from five to nearly eleven years. vii, viii

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The COMP adopts an opinion on Orphan Drug Designation, after which the opinion is submitted to the European Commission (EC) for endorsement. Orphan Drug Designation is a status assigned to a medicine intended for use in rare diseases.ix To be granted orphan status by the EC, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life threatening and has a prevalence of up to five in 10,000 in the European Union. Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan status provides companies with development and market exclusivity incentives for designated compounds and medicines.

In addition to ongoing Phase II/III trials in CLL, MCL and WG, acalabrutinib is currently being tested in Phase I/II trials in monotherapy as well as in combination with immunotherapy or chemotherapies in a range of other blood cancers and solid tumours.

i Chronic Lymphocytic Leukemia. Leukemia & Lymphoma Society Website. View Source Accessed February 19, 2016.
ii What are the key statistics for chronic lymphocytic leukemia? American Cancer Society Website. View Source . Accessed February 19, 2015.
iii Veliz M, Pinilla-Ibarz J. Treatment of relapsed or refractory chronic lymphocytic leukemia. Cancer Control. 2012; 19(1):37-53.
iv Chronic lymphocytic leukemia/Small lymphocytic lymphoma, National Cancer Institute Website. View Source Accessed February 19, 2016.
v Campo E and Rule S. Mantle cell lymphoma: evolving management strategies. Blood. 2015 Jan 1;125(1):48-55.
vi Mantle Cell Lymphoma, Lymphoma.org Website. View Source;b=6300157 Accessed February 19, 2016.
vi Lymphoplasmacytic lymphoma. National Cancer Institute. Surveillance, Epidemiology, and End Results program. View Source Accessed February 19, 2016.
vii Dimopoulos MA, Kastritis E, Ghobrial IM. Waldenström’s macroglobulinemia: a clinical perspective in the era of novel therapeutics. Ann Oncol. 2016 Feb;27(2):233-40.
viii Oza and Rajkumar. Waldenstrom macroglobulinemia: prognosis and management. Blood Cancer Journal (2015) 5, e394; doi:10.1038/bcj.2015.28
ix European Medicines Agency web site. "Orphan Designation." View Source;mid=WC0b01ac05800240ce. Accessed February 17, 2016.

About Acalabrutinib
Acalabrutinib is a highly selective, irreversible, second generation BTK inhibitor, with approximately 1,000 patients treated to date in clinical studies across the entire development programme. More than 600 patients have been treated with acalabrutinib monotherapy. Phase I/II data showing a favourable safety profile and efficacy in relapsed/refractory chronic lymphocytic leukaemia patients was presented at the American Society of Haematology Annual Meeting & Exposition in December 2015, with simultaneous publication in the New England Journal of Medicine.
Potentially registrational studies in haematological malignancies are ongoing. In addition, a head-to-head study versus ibrutinib in high risk chronic lymphocytic leukaemia patients is currently ongoing.
Acalabrutinib is also currently being tested in multiple Phase I/II studies in solid tumours, as monotherapy or in combination with immune checkpoint inhibitors or other standard of care regimens.