On February 18, 2016 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported business highlights and financial results for the fourth quarter and year ended December 31, 2015 (Press release, Agios Pharmaceuticals, FEB 18, 2016, View Source;p=RssLanding&cat=news&id=2140406 [SID:1234509085]). In addition, Agios highlighted select corporate milestones for its preclinical and clinical development programs.

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"2015 marked a year of significant achievements for Agios as we rapidly advanced our IDH inhibitors in AML, presented initial data in solid tumors and selected our fifth molecule for clinical development," said David Schenkein, M.D., chief executive officer at Agios. "We are in a strong position entering 2016, focusing on rapid and broad late-stage clinical development for our lead IDH mutant inhibitors, executing clinical trials of our PKR activators and advancing our research programs. We look forward to several important milestones, beginning with presenting the first data from both of our PKR activators in the first half of the year. These milestones will bring us closer to our vision of helping people with cancer and rare genetic disorders."

2016 EXPECTED MILESTONES IN CANCER METABOLISM PROGRAMS

AG-221, AG-120 and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation.

IDH Mutant Inhibitors in Hematologic Malignancies:

Complete enrollment of both 125-patient expansion cohorts for the Phase 1/2 study of AG-221 and Phase 1 study of AG-120 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) in the second half of 2016

Initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016

Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016

Initiate a Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016

Continue to enroll patients in the following ongoing clinical trials:
Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML
Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies

IDH Mutant Inhibitors in Solid Tumors:

Present data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low grade glioma in the second half of 2016

Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016

Continue to enroll patients in the following ongoing clinical trials:
Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors

Cancer Metabolism Research:

Present preclinical findings on a new research program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers at the Keystone Symposia on New Frontiers in Understanding Tumor Metabolism taking place February 21-25, 2016 in Banff, Alberta, Canada

Initiate preclinical development activities for the first molecule in the next wave of novel investigational medicines

2016 EXPECTED MILESTONES IN RARE GENETIC METABOLIC DISORDERS PROGRAMS

Plan to submit the first data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with pyruvate kinase (PK) deficiency, for presentation at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June 2016

Plan to submit the first data from the Phase 1 study of AG-519 in healthy volunteers for presentation at EHA (Free EHA Whitepaper) in June 2016. Preclinical findings about the molecule will also be submitted for presentation at EHA (Free EHA Whitepaper).

Outline the clinical development plans for Agios’ PKR activators in beta-thalassemia in the second half of 2016
Present new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016

FOURTH QUARTER 2015 HIGHLIGHTS OF CANCER METABOLISM PROGRAMS

Agios has provided the following updates on its clinical development programs in collaboration with Celgene:

IDH Mutant Inhibitors in Hematologic Malignancies:

New data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1/2 study for AG-221 and the Phase 1 study of AG-120 were presented in December at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH) (Free ASH Whitepaper). Read the full AG-221 data here and the AG-120 data here.

In December, Agios initiated a Phase 1b, multicenter, international, open-label study of AG-221 or AG-120 in combination with induction and consolidation therapy in patients with newly diagnosed AML with an IDH mutation who are eligible for intensive chemotherapy.

IDH Mutant Inhibitors in Solid Tumors:

The first data from the ongoing Phase 1 dose-escalation trial of AG-120 in advanced IDH1-mutant positive solid tumors were presented in an oral presentation at AACR (Free AACR Whitepaper)-EORTC-NCI AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in November. Read the full data here.

RECENT CORPORATE AND FINANCIAL UPDATES

Agios recently announced the appointment of Steve Hoerter to chief commercial officer. Mr. Hoerter has more than 20 years of extensive pharmaceutical and biotechnology commercial experience and most recently served as executive vice president and chief commercial officer at Clovis Oncology, Inc. Prior to Clovis, Mr. Hoerter held senior commercial roles at Genentech and Roche.

In January 2016, Agios received a $25 million milestone payment from Celgene for achievement of the first patient dosed in the Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML. This is an international, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of AG-221 versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy.

FULL YEAR 2015 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of December 31, 2015 were $375.9 million, compared to $467.4 million as of December 31, 2014. The decrease was driven by cash used to fund operating activities of approximately $161.8 million, which was offset by funding of approximately $64.7 million from Celgene during the year ended December 31, 2015 related to our collaboration agreements.

Collaboration revenue was $59.1 million for the year ended December 31, 2015, compared to $65.4 million for the prior year. Beginning in the first quarter of 2015, the company began offsetting research and development expense for amounts received from Celgene for reimbursement of certain development costs incurred on Celgene’s behalf related to AG-221 which were presented as gross collaboration revenue during 2014.

Research and development (R&D) expenses were $141.8 million, including $17.4 million of stock-based compensation expense, for the year ended December 31, 2015, compared to $100.4 million, including $6.7 million in stock-based compensation expense, for the year ended December 31, 2014. The increase in R&D expenses was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development. Celgene is responsible for all development costs for AG-221 and certain development costs for AG-120 and AG-881 and reimburses the company for development costs incurred for these investigational medicines.

General and administrative (G&A) expenses were $36.0 million, including $14.5 million of stock-based compensation expense, for the year ended December 31, 2015, compared to $19.1 million, including $4.8 million of stock-based compensation expense, for the year ended December 31, 2014. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the year ended December 31, 2015 was $117.7 million, compared to a net loss of $53.5 million for the year ended December 31, 2014.

FINANCIAL GUIDANCE FOR THE FULL YEAR 2016

Agios announced today that it expects to end 2016 with more than $180 million of cash, cash equivalents and marketable securities. The anticipated year-end 2016 cash position does not include any additional program-specific milestone payments. The company expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017.

On February 08, 2016 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported its consolidated financial results for the year ended December 31, 2015 (Press release, Innate Pharma, FEB 17, 2016, View Source [SID:1234509346]). The consolidated financial statements are attached to this press release.

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Hervé Brailly, Chief Executive Officer and co-founder of Innate Pharma, commented: "2015 was a very important year for Innate Pharma. We signed a landmark co-development and commercialization agreement in April with AstraZeneca which we can leverage on to bring us to the next steps of our corporate progress, i.e. late stage drug development and marketing. The initial payment also gives us financial flexibility which we are using to expand the base of the Company. We are growing the organization, hiring new talent and investing in our proprietary clinical and preclinical pipeline to ensure the future growth of Innate. One tangible move in this direction was the recent acquisition of CD39, a new, preclinical first-in-class checkpoint inhibitor program, and our investment in innovative technologies such as the NK bispecific engagers illustrated by the recently announced collaboration with Sanofi.

As we await important clinical data for lirilumab, we intend to continue to broaden and consolidate our unique positioning in the very promising area of immuno-oncology".

On February 17, 2016 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported treatment of the first patient in a Phase 1 study of its product candidate bb2121 in patients with relapsed/refractory multiple myeloma (Filing, 8-K, bluebird bio, FEB 17, 2016, View Source [SID:1234509086]). bb2121 is a chimeric antigen receptor T cell (CAR T) therapy targeting B cell maturation antigen (BCMA), and bluebird bio is developing bb2121 in collaboration with Celgene Corporation. bluebird bio also announced today that Celgene has exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies.

"bb2121 is bluebird bio’s first oncology program to enter the clinic, and the treatment of this first patient marks an important milestone for us as we build a broad, fully integrated T cell immunotherapy franchise," said Nick Leschly, chief bluebird. "We are pleased that Celgene has exercised their option to license bb2121. We believe our combined manufacturing, development and commercial expertise will enable us to rapidly advance bb2121 through clinical trials."

"Despite many recent advances in the field, multiple myeloma remains incurable, with almost all patients becoming refractory to therapy eventually," said James N. Kochenderfer, M.D., National Cancer Institute, an investigator for the CRB-401 study. "BCMA is one of the most exciting targets in multiple myeloma, and we are eager to explore the potential of bb2121 to become an important new treatment option for patients living with multiple myeloma."

bluebird bio and Celgene amended and restated their collaboration agreement in June 2015 to focus on developing product candidates targeting BCMA during a three-year collaboration term. By exercising its exclusive option under the terms of the agreement, Celgene will be responsible for worldwide development and commercialization of bb2121 after Phase 1. bluebird bio is responsible for the development of bb2121 through the completion of the CRB-401 Phase 1 study and has an option to share in the development, promotion and profits in the United States. bluebird bio will receive a $10 million option exercise payment from Celgene, and bluebird bio is also eligible to receive specified development and regulatory and commercial milestone payments and royalty payments on net sales.

About the CRB-401 Study

The primary objective of the CRB-401 study is to evaluate the maximum tolerated dose of bb2121 and determine the recommended Phase 2 dose. The secondary objective is patient response, measured using the International Myeloma Working Group (IMWG) Response Criteria for Multiple Myeloma. The first portion of the study includes a dose-escalation phase in which cohorts of patients will receive ascending doses of bb2121 to determine the maximum tolerated dose and establish a recommended Phase 2 dose. The second portion of the study is a dose expansion phase where patients will receive bb2121 to further evaluate the safety, tolerability and clinical activity at the recommended Phase 2 dose.

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On February 17, 2016 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG), a specialty pharmaceutical company with a diverse portfolio of products in the areas of maternal health, anemia management and cancer supportive care, reported unaudited consolidated financial results for the fourth quarter and full year ended December 31, 2015 (Filing, 8-K, AMAG Pharmaceuticals, FEB 17, 2016, View Source [SID:1234509079]).

"2015 was a pivotal year for AMAG as we drove revenue growth of Makena and Feraheme, expanded our presence in maternal health through the acquisition of Cord Blood Registry and made an investment in a promising therapy for the potential treatment of severe preeclampsia," said William Heiden, AMAG’s chief executive officer. "Growing revenues and integrating these transactions, combined with advancing our next generation programs for Makena and Feraheme, have positioned us well for continued strong growth."

Full Year 2015 Business Highlights:

· Increased net product sales of Makena by 52% to $251.6 million, compared with pro forma net product sales of $165.8 million(2) in 2014. This growth in sales was driven by a 56% increase in volume as more at-risk pregnant women were treated with Makena.

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· Made progress toward commercialization of a single-dose, preservative-free formulation of Makena. The company responded to questions contained in the FDA’s complete response letter in November 2015 and anticipates an approval of its manufacturing supplement in the first quarter of 2016 with commercial launch in the second quarter of 2016.

· Advanced the development of the next generation program for Makena with an auto-injector device for subcutaneous administration of Makena through a partnership with Antares Pharma, Inc., an experienced drug device company.

· Expanded the maternal health portfolio through the acquisition of Cord Blood Registry (CBR), the world’s largest private newborn stem cell bank serving pregnant women and their families, and the purchase of an option to acquire worldwide rights to an orphan drug candidate being developed for severe preeclampsia.

· Returned Feraheme (ferumoxytol) to growth in the second half of the year, increasing sales by 5% to $88.5 million in 2015, compared with $84.4 million in 2014.(3)

(1) See summaries of non-GAAP adjustments for the three and twelve months ended December 31, 2015 and 2014 at the conclusion of this press release.
(2) Unaudited. Includes net product sales of Makena as though Lumara Health had been acquired at the beginning of 2014. Lumara Health was purchased on November 12, 2014.

· Initiated start-up activities for a head-to-head, Phase 3 clinical trial evaluating the safety of Feraheme compared to Injectafer (ferric carboxymaltose injection) in adults with iron deficiency anemia (IDA). This study is intended to support an sNDA filing to broaden the use of Feraheme beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment.

Fourth Quarter Ended December 31, 2015 (unaudited)

Financial Results (GAAP Basis)

Total revenues for the fourth quarter of 2015 were $108.7 million, compared with $53.3 million for the same period in 2014. Net product sales of Makena were $67.4 million in the fourth quarter of 2015, compared with $22.5 million(4) in the same period last year. Sales of Feraheme and MuGard totaled $23.5 million in the fourth quarter of 2015, compared with $24.5 million in the fourth quarter of 2014, which included a favorable $1.8 million release of product return reserves. Service revenue from CBR totaled $17.0 million in the fourth quarter of 2015.

Total costs and expenses for the fourth quarter of 2015 were $86.1 million, compared with $56.6 million for the same period in 2014. The increase in costs and expenses was primarily due to higher costs associated with managing the company’s expanded portfolio and infrastructure following the acquisitions of Lumara Health in November 2014 and CBR in August 2015.

The company reported operating income of $22.7 million and net income of $7.2 million, or $0.21 per basic share and $0.20 per diluted share, for the fourth quarter of 2015, compared with an operating loss of $3.4 million and net income of $143.0 million, or $5.98 per basic share and $4.67 per diluted share, for the same period in 2014. In the fourth quarter of 2014, the company recognized a non-cash income tax benefit of $153.2 million associated with the release of reserves on certain tax attributes (i.e., net operating losses) as a result of the Lumara Health transaction. The weighted average diluted shares used in calculating diluted net income per share in 2015 and 2014 followed the if-converted method of accounting for the convertible debt.

Financial Results (Non-GAAP Basis)(1),(5)

Non-GAAP revenues totaled $120.6 million, up from $51.0 million in the fourth quarter of 2014. Non-GAAP CBR revenue totaled $28.8 million in the fourth quarter of 2015. The difference between GAAP and non-GAAP revenue for CBR represents purchase accounting adjustments related to deferred revenue.

Total costs and expenses on a non-GAAP basis totaled $59.2 million resulting in a gross margin of 92% and adjusted EBITDA margin of 51% for the fourth quarter of 2015. This compares to costs and expenses of $36.4 million in the same period of 2014, which resulted in a gross margin of 89% and adjusted EBITDA margin of 29%. Non-GAAP adjusted EBITDA for the fourth quarter of 2015 was $61.3 million, compared with $14.5 million for the same period in 2014.

(3) Excludes a favorable $1.8 MM release of product return reserves in 2014.
(4) AMAG purchased Lumara Health maternal health business on November 12, 2014.
(5) See share count reconciliation at the conclusion of this press release.

After deducting cash interest expense, the company generated fourth quarter 2015 non-GAAP cash earnings of $46.5 million, or $1.34 per non-GAAP basic share and $1.12 per non-GAAP diluted share. In the fourth quarter of 2014, non-GAAP cash earnings totaled $10.2 million, or $0.33 per non-GAAP diluted share. The weighted average diluted shares used in calculating the non-GAAP cash earnings per diluted share for the fourth quarter of 2015 includes the impact of the convertible debt and related bond hedge and warrants.

Full Year Ended December 31, 2015 (unaudited)

Financial Results (GAAP Basis)

Total revenues in 2015 were $418.3 million, compared with $124.4 million in 2014. This increase is primarily related to the addition of Makena in November 2014 and CBR in August 2015, which contributed $251.6 million and $24.1 million, respectively, in product revenue to the 2015 results. In addition, the company recognized $52.3 million of collaboration revenue in 2015 related to the company’s ex-US ferumoxytol marketing agreement with Takeda Pharmaceutical Company Limited, compared with $14.4 million of collaboration revenue in 2014. The marketing agreement was terminated in 2015, resulting in the recognition of all previously deferred revenue.

Net income totaled $32.8 million in 2015, compared with $135.8 million in 2014. Basic net income per share was $1.04, compared with $6.06 in 2014. Diluted net income per share was $0.93 in 2015, compared with $5.45 in 2014. In 2014, the company recognized a non-cash income tax benefit of $153.2 million associated with the release of reserves on certain tax attributes (e.g., net operating losses) as a result of the Lumara Health transaction. The weighted average diluted shares used in calculating diluted net income per share in 2015 and 2014 followed the if-converted method of accounting for the convertible debt.

Financial Results (Non-GAAP Basis)(1),(5)

Non-GAAP revenues totaled $397.4 million in 2015, up from $116.2 million in 2014. Non-GAAP CBR revenue totaled $43.3 million since the acquisition in August 2015. Non-GAAP adjusted EBITDA totaled $213.4 million in 2015, compared with $14.3 million in 2014. After deducting cash interest expense, the company generated non-GAAP cash earnings of $173.7 million in 2015, or $4.43 per non-GAAP diluted share. In 2014, non-GAAP cash earnings totaled $7.7 million, or $0.30 per non-GAAP diluted share. The weighted average diluted shares used in calculating the non-GAAP cash earnings per diluted share in 2015 included the impact of the convertible debt and related bond hedge and warrants.

Balance Sheet Highlights

As of December 31, 2015, the company’s cash and investments totaled approximately $466.3 million and total debt (face value) was approximately $1.0 billion.

"The two transformative acquisitions that we completed in the past eighteen months fueled our significant top- and bottom-line growth in 2015, resulting in adjusted EBITDA of more than $210 million in 2015," said Frank Thomas, AMAG’s president and chief operating officer. "These strong cash flows, combined with more than $460 million in cash and investments on the balance sheet, positions us well for future acquisitions that will allow us to leverage our commercial organization and add new, innovative products to our portfolio to drive future growth."

2016 Goals

The company’s goals for 2016 include the following:
· Drive significant net product sales growth (+40%) over 2015

· Grow non-GAAP adjusted EBITDA to more than $255 million

· Continue to execute on the Makena next generation development program, including:

· Receiving a favorable decision from the FDA in 1Q 2016 for the single-dose, preservative-free formulation of Makena with a 2Q 2016 commercial launch

· Completing chemistry, manufacturing and controls (CMC) and pilot pharmacokinetics (PK) work to support the initiation of a bio-equivalence study for the Makena subcutaneous auto injecter by the end of 2016

· Enroll patients in a head-to-head Phase 3 clinical trial in 2016 evaluating the safety of Feraheme compared to Injectafer in adults with IDA

· Complete preclinical work and initiate clinical program for severe preeclampsia Velo option

· Further expand the company’s product portfolio through acquisitions or in-licensing of products or companies

Use of Non-GAAP Financial Measures

AMAG has presented certain non-GAAP financial measures, including non-GAAP revenue, non-GAAP adjusted EBITDA (earnings before income taxes, depreciation and amortization), non-GAAP net income, or

(6) See reconciliation of 2016 financial guidance of non-GAAP CBR revenue, non-GAAP adjusted EBITDA and non-GAAP cash earnings at the conclusion of this press release.

cash earnings, non-GAAP diluted net income, or cash earnings, per share, and non-GAAP weighted average diluted shares. These non-GAAP financial measures exclude certain amounts, revenue, expenses or income, from the corresponding financial measures determined in accordance with accounting principles generally accepted in the U.S. (GAAP). Management believes this non-GAAP information is useful for investors, taken in conjunction with AMAG’s GAAP financial statements, because it provides greater transparency regarding AMAG’s operating performance. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of AMAG’s operating results as reported under GAAP, not as a substitute for GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.