On February 18, 2016 (Heat Biologics, Inc. ("Heat") (Nasdaq:HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported financial results for the fiscal year ended December 31, 2015 (Press release, Heat Biologics, FEB 18, 2016, View Source [SID:1234509093]).

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"2015 proved to be a significant year for Heat as we reported positive Phase 1 data for HS-410 in bladder cancer, as well as initiated a Phase 1b trial evaluating HS-110 in lung cancer in combination with a PD-1 checkpoint inhibitor," stated Jeff Wolf, Heat’s Founder and Chief Executive Officer. "We expect to continue the momentum into 2016 with important milestones throughout the year highlighted by topline data for our Phase 2 trial evaluating HS-410 for non-muscle invasive bladder cancer (NMIBC) anticipated in the fourth quarter."

Recent Developments & Fourth Quarter 2015 Corporate Highlights

On February 10, 2016, Heat announced that the U.S. FDA lifted the partial clinical hold on its HS-410 Phase 2 clinical trial and patient enrollment was resumed after one week; clinical timelines were materially unchanged. The partial clinical hold came after Heat concluded that the cell line on which HS-410 is based had been previously misidentified and immediately notified FDA of this conclusion. The FDA placed Heat’s HS-410 Phase 2 clinical trial on partial clinical hold while they reviewed updated documentation. The partial clinical hold did not relate to concerns regarding the safety or efficacy of HS-410.

In January 2016, Heat reported three-month interim data from the unblinded, monotherapy cohort of the company’s ongoing Phase 2 trial of HS-410 for the treatment of high-risk, NMIBC. Images of the bladder taken from patients treated with HS-410 alone (e.g., no Bacillus Calmette-Guérin (BCG)) showed changes that resemble T cell-rich structures, indicating that systemic administration with HS-410 leads to a localized immune response within the bladder that cannot be attributed to standard of care. The interim data further validated HS-410’s mechanism of action. One of the treated patients had carcinoma in situ (CIS) – the patient population believed to be least responsive to BCG – and that patient experienced complete response. Heat continues to enroll the 25-patient arm and expects to report further monotherapy HS-410 data later this year.

In November 2015, Heat announced results from its Phase 1 clinical trial, evaluating the safety and immune response of HS-410 after surgery and treatment with standard of care, BCG, in patients with NMIBC. HS-410 had a positive safety profile with no serious adverse events. HS-410 elicited a broad-based (polyclonal) expansion of patient T cells and a high level of CD8+ tumor-infiltrating lymphocytes (TILs). Additionally, based on tissue samples taken from each patient, HS-410 shared 15 or more tumor antigens with those expressed on the patients’ cancer cells.

In October 2015, Heat completed enrollment of the 75 patients in the company’s blinded, randomized, placebo-controlled arms of its Phase 2 clinical trial of HS-410 for the treatment of NMIBC. In these three arms, Heat is evaluating the ability of HS-410 in combination with BCG to prevent cancer recurrence. The company plans to report topline efficacy, immune-response and safety data in the fourth quarter of 2016.

Fiscal Year 2015 Financial Highlights

Research and development expenses decreased to $2.6 million in 2015 from $2.9 million in 2014, a decrease of $0.3 million. The decrease is attributable to a reduction in pre-manufacturing costs associated with preparing clinical trial material and professional and consulting expenses, offset by increases in compensation costs, lab supplies and other fees.

Clinical and regulatory expenses increased to $14.1 million in 2015 from $5.3 million in 2014, an increase of $8.8 million. The increase is attributable to increases in clinical trial execution expenses, personnel costs and expenses related to the production of our clinical trial material.

General and administrative expenses increased to $4.4 million in 2015 from $4.0 million in 2014, an increase of $0.4 million. The increase is attributable to increased personnel and professional service fees.

Net loss for 2015 was $20.3 million compared to a net loss of $11.8 million for 2014.

Cash, cash equivalents and short-term investments totaled approximately $11.6 million at December 31, 2015 compared to $14.4 million at December 31, 2014.

On February 18, 2016 Momenta Pharmaceuticals, Inc. (Nasdaq:MNTA) reported its financial results for the fourth quarter and year ended December 31, 2015 (Press release, Momenta Pharmaceuticals, FEB 18, 2016, View Source [SID:1234509091]).

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For the fourth quarter of 2015, the Company reported total revenues of $22.4 million, including $15.6 million in product revenues from Sandoz’s sale of Glatopa (glatiramer acetate injection). For the year ended December 31, 2015, the Company reported total revenues of $89.7 million, including $43.4 million in product revenues from Sandoz’s sales of Glatopa and $20.0 million in milestone payments earned upon sole FDA approval and launch of Glatopa. Momenta reported a net loss of $(29.2) million, or $(0.43) per share for the fourth quarter compared to a net loss of $(16.0) million, or $(0.31) per share for the same period in 2014. For the year ended December 31, 2015, the Company reported a net loss of $(83.3) million, or $(1.32) per share compared to a net loss of $(98.6) million, or $(1.91) per share for the same period in 2014. At December 31, 2015, the Company had cash, cash equivalents, and marketable securities of $350.0 million compared to $191.5 million at December 31, 2014.

"The year 2015 proved to be pivotal in our company’s growth and development. Glatopa, our second complex generic to receive FDA marketing approval, is the first and only substitutable generic product for multiple sclerosis on the market today. Glatopa’s approval provided further validation of the strength of our proprietary analytic platform and physicochemical and biologic characterization capabilities, and has set the stage for our biosimilar and novel drug programs," said Craig A. Wheeler, President and Chief Executive Officer of Momenta Pharmaceuticals. "In addition, Glatopa’s approval allowed us to complete a successful financing that significantly strengthened our balance sheet and helped us secure a strong biosimilars collaboration partner in Mylan. We believe we are now well-positioned to execute on our biosimilar and novel drug programs in 2016 and to continue to build our business for long-term growth and sustainability."

Fourth Quarter Highlights and Recent Events

Complex Generics:

Glatopa, generic version of daily COPAXONE 20 mg (glatiramer acetate injection)

In the fourth quarter of 2015, Momenta recorded $15.6 million in product revenues from Sandoz’s Glatopa sales. Since the launch of Glatopa in June 2015, Momenta has recorded $43.4 million in product revenues from Sandoz’s sales of Glatopa reflecting $52.5 million in profit share net of a deduction of $9.1 million for reimbursement to Sandoz of the Company’s share of pre-launch Glatopa-related legal expenses. In addition, in 2015 the Company earned $20.0 million in milestone payments from Sandoz upon receiving sole FDA approval for Glatopa and upon first commercial sale of Glatopa.
The ANDA submitted by Sandoz for a three-times-a-week generic COPAXONE 40 mg (glatiramer acetate injection) is under FDA review and has received a target action date.
A district court trial challenging Teva’s four Orange Book-listed patents for COPAXONE 40 mg (glatiramer acetate injection) is scheduled for September 26, 2016.
Enoxaparin Sodium Injection

Momenta earned $2.2 million and $5.1 million in product revenues on Sandoz’s net sales of enoxaparin sodium injection in the fourth quarter of 2015 and for the year ended December 31, 2015, respectively.
In November 2015, the Court of Appeals for the Federal Circuit (CAFC) vacated the District Court’s summary judgment decision with respect to Amphastar, finding Amphastar’s use of Momenta’s U.S. Pat. 7,575,886 not to be protected under the "safe harbor" provisions in the Hatch-Waxman Act, and remanded the case back to the District Court. On February 17, 2016, the CAFC denied Amphastar’s petition for rehearing.
Biosimilars:

In the fourth quarter of 2015, Momenta and Baxalta announced the initiation of a pivotal clinical trial for M923, a biosimilar candidate of HUMIRA (adalimumab). The companies also announced that M923 met the primary endpoints in a study to evaluate the pharmacokinetics of M923 compared to both US and EU sourced HUMIRA reference products. The companies are targeting first regulatory submission in 2017 and a first commercial launch in 2018.
In January 2016, Momenta announced a global collaboration with Mylan N.V. to develop, manufacture and commercialize six of the Company’s biosimilar candidates, including M834, a biosimilar candidate of ORENCIA (abatacept). Under the collaboration agreement, Mylan has agreed to pay Momenta an upfront cash payment of $45 million. In addition, each Company will share equally in the cost and profits with respect to products, with Mylan funding its share of expenses, in part, through up to $200 million in contingent milestone payments. On February 9, 2016, the companies received clearance for the collaboration under the Hart-Scott-Rodino Antitrust Improvements Act.
In January 2016, the U.S. Patent and Trademark Office (PTAB) instituted Momenta’s request for an Inter Partes Review proceeding to challenge Bristol Myers Squibb’s U.S. formulation Pat. 8,476,239 for ORENCIA. The Company expects a decision from the PTAB in January 2017.
Novel Drugs:

Necuparanib (novel oncology candidate)

Following the institution of a protocol amendment in December 2015, the Company resumed patient enrollment in its ongoing Phase 2 trial of necuparanib in pancreatic cancer. In November 2015, Momenta had put a temporary hold on patient enrollment in its ongoing Phase 2 trial of necuparanib following receipt of recommendations from the Company’s independent Data Safety Monitoring Board (DSMB) to develop guidelines for diagnosing and managing thrombocytopenia, based on a limited number of specific toxicities observed in the study. The Company expects to have clinical data in the second half of 2017.
Momenta continues to collect data from the Phase 1 study of necuparanib and plans to publish and/or present updated results at a medical conference in 2016.
Autoimmune Drugs

Momenta’s three novel autoimmune candidates are in preclinical development. These candidates include a hyper-sialylated IVIg (hsIVIg), a high potency alternative to IVIg, and two recombinant molecules: M230, a Selective Immunomodulator of Fc receptors (SIF3) and M281, an anti-FcRn monoclonal antibody. The Company is advancing the recombinant candidates with a goal of initiating clinical trials in mid-2016 for M281, and in 2017 for M230. The Company is continuing its efforts to identify and explore potential collaboration opportunities for the further development and commercialization of its hsIVIg program.

Fourth Quarter and Year End 2015 Financial Results

Total revenues for the fourth quarter of 2015 were $22.4 million (including Glatopa product revenue of $15.6 million), compared to $21.2 million for the same period in 2014. For the year ended December 31, 2015, total revenues were $89.7 million (including Glatopa product revenue of $43.4 million), compared to $52.3 million for 2014. Glatopa was launched in June 2015.

Enoxaparin product revenue decreased from $4.7 million for the fourth quarter of 2014 to $2.2 million for the same period in 2015. For the year ended December 31, 2015 , total enoxaparin product revenue was $5.1 million compared to $19.9 million for 2014. The decrease in enoxaparin product revenue is due to the change in collaboration economics from a royalty payment to 50% profit share, decreased unit sales due to lower market share and continued competitive pricing.

Collaborative research and development revenue for the fourth quarter of 2015 was $4.6 million compared to the $16.4 million recorded in the same quarter last year. The decrease is primarily due to the $12.0 million M923 technical development milestone earned under the Baxalta Agreement in the fourth quarter of 2014. For the year ended December 31, 2015, collaborative research and development revenues were $41.1 million, compared to $32.3 million for 2014. The increase is primarily due to $20.0 million in milestone payments earned upon sole FDA approval and first commercial sale of Glatopa in 2015.

Research and development expenses for the fourth quarter of 2015 were $37.6 million, compared to $26.2 million for the same period in 2014. The increase of $11.4 million, or 44%, from the 2014 period was due to increases of $9.3 million in third-party research and process development costs for our biosimilar and novel autoimmune drug programs, $1.6 million in personnel-related expenses and $0.5 million in necuparanib Phase 2 clinical trial expenses. For the year ended December 31, 2015, research and development expenses were $126.0 million compared to $106.5 million for the year ended 2014. The increase of $19.5 million in research and development expenses, or 18%, from 2014 to 2015 resulted from increases of $17.6 million in third-party research and process development costs for our biosimilar and novel autoimmune drug programs, $3.3 million in necuparanib Phase 2 clinical trial expenses and $1.4 million in nonclinical studies for our novel autoimmune and early stage biosimilar programs. The increases were partly offset by decreases of $2.5 million for research supplies and $0.3 million in personnel-related expenses.

General and administrative expenses for the quarter ended December 31, 2015, were $14.4 million, compared with $11.1 million for the same period in 2014. The increase of $3.3 million, or 30%, was due to a $2.1 million increase in professional fees and a $1.2 million increase in personnel-related expenses. For the year ended December 31, 2015, general and administrative expenses were $48.1 million, compared to $45.2 million for the year ended 2014. The increase of $2.9 million, or 6%, resulted from increases of $2.3 million in professional fees, $0.8 million in facility related costs and $0.4 million in depreciation expense. The increases were partly offset by a $0.6 million decrease primarily in share-based compensation expenses associated with performance-based stock awards.

At December 31, 2015, Momenta had $350.0 million in cash, cash equivalents and marketable securities.

Financial Guidance

Today, Momenta provided guidance that it expects its operating expenses, excluding stock-based compensation and net of collaborative revenues, to be approximately $45 – $55 million per quarter for the first half of 2016.

On February 18, 2016 Laboratory Corporation of America Holdings (LabCorp or the "Company") (NYSE: LH) reported results for the fourth quarter and year ended December 31, 2015 and 2016 guidance (Press release, LabCorp, FEB 18, 2016, View Source;p=RssLanding&cat=events&id=5215848 [SID:1234509089]).

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"The year was transformative for LabCorp," said David P. King, chairman and chief executive officer. "We significantly expanded the Company’s capabilities, global presence and avenues for future growth while reinforcing our position as the world’s leading healthcare diagnostics company. We concluded the year with strong fourth quarter results including continued organic volume growth in LabCorp Diagnostics, accelerated revenue growth in Covance Drug Development, double-digit adjusted EPS growth, and robust free cash flow."

Consolidated Results

Fourth Quarter Results

Net revenue for the quarter was $2.24 billion, an increase of 48.4% over last year’s $1.51 billion. The acquisition of Covance contributed $669.5 million in net revenue during the quarter, driving 44.3% year-over-year growth. The remainder of the increase of $62.6 million, or 4.1%, was driven by organic volume growth, price, mix and tuck-in acquisitions, partially offset by currency. Organic revenue growth in the quarter, excluding currency, was 3.9%.

Operating income for the quarter was $243.5 million, compared to $219.0 million in the fourth quarter of 2014. The Company recorded restructuring charges and special items of $86.4 million during the fourth quarter of 2015, compared to $15.6 million during the same period in 2014. Adjusted operating income (excluding amortization of $38.3 million, restructuring and special items) for the quarter was $368.2 million, or 16.4% of net revenue, compared to $250.0 million, or 16.5%, in the fourth quarter of 2014. The increase in adjusted operating income was primarily due to the Covance acquisition, organic volume growth, productivity, price and mix, partially offset by personnel costs. The decline in margin was due to the mix impact from the acquisition of Covance.

The Company recorded net earnings in the quarter of $114.2 million, or $1.11 per diluted share, compared to $119.6 million, or $1.39 per diluted share, last year. Adjusted EPS (excluding amortization, restructuring and special items) were $1.98 in the quarter, an increase of 20.0% compared to $1.65 in the fourth quarter of 2014.

Operating cash flow for the fourth quarter was $384.6 million, compared to $213.7 million in the fourth quarter of 2014. The increase in operating cash flow was due to the acquisition of Covance as well as favorable working capital. Capital expenditures totaled $85.1 million, compared to $46.3 million in the fourth quarter of 2014. As a result, free cash flow (operating cash flow less capital expenditures) was $299.5 million, compared to $167.4 million in the fourth quarter of 2014.

At the end of the quarter, the Company’s cash balance and total debt were $716.4 million and $6.4 billion, respectively. During the quarter, the Company invested $43.3 million in tuck-in acquisitions and paid down $250.0 million of debt. The Company’s liquidity at the end of the quarter was approximately $1.7 billion, consisting of cash and available credit.

Full Year Results
The following full year consolidated results of the Company include Covance as of February 19, 2015; prior to February 19, 2015, these consolidated results exclude Covance.

Net revenue was $8.51 billion, an increase of 41.5% over last year’s $6.01 billion. The acquisition of Covance contributed $2.20 billion from the February 19, 2015 closing date, driving 36.7% year over year net revenue growth. The remainder of the increase of $289.5 million, or 4.8%, was due to strong organic volume growth, tuck-in acquisitions, price, and mix, partially offset by currency. Organic revenue growth in 2015, excluding currency, was 4.6%.

Operating income for 2015 was $1,002.9 million, compared to $910.4 million in 2014. Operating income was reduced by $279.5 million in restructuring charges and special items (primarily costs associated with the acquisition of Covance and Project LaunchPad, the Company’s enterprise-wide business process improvement initiative) recorded during 2015, compared to $41.2 million in 2014. Adjusted operating income (excluding amortization of $164.5 million, restructuring and special items) was $1.45 billion, or 17.0% of net revenue, compared to $1.03 billion, or 17.1%, in 2014. The increase in adjusted operating income was primarily due to the acquisition of Covance, organic volume growth, price, mix and productivity, partially offset by currency and personnel costs. The decline in margin was due to the mix impact from the acquisition of Covance.

The Company’s pre-tax earnings were reduced by restructuring and special items of $334.4 million ($279.5 million impacted operating income, $52.6 million impacted interest expense, and $2.3 million impacted other, net), or $245.7 million after-tax. As a result, the Company recorded net earnings in 2015 of $436.9 million, or $4.34 per diluted share, compared to $511.2 million, or $5.91 per diluted share, last year. Adjusted EPS (excluding amortization, restructuring and special items) were $7.91 in 2015, an increase of 16.3% compared to $6.80 in 2014.

Operating cash flow for 2015 was $982.4 million, compared to $739.0 million in 2014, driven by the Covance acquisition and improved earnings. Capital expenditures totaled $255.8 million, compared to $203.5 million in 2014, due to the Covance acquisition. As a result, free cash flow (operating cash flow less capital expenditures) was $726.6 million, compared to $535.5 million last year. Free cash flow in 2015 was negatively impacted by approximately $110 million in net non-recurring items relating to the acquisition of Covance. Excluding these items, free cash flow would have been $836.6 million in 2015.

The following segment results are presented on a pro forma basis for all periods as if the acquisition of Covance closed on January 1, 2014 and exclude amortization, restructuring, special items and unallocated corporate expenses. In the fourth quarter of 2015, the Company refined its methodology for the calculation of unallocated corporate expenses, which impacts segment results and has been applied to prior periods for comparative purposes. As a result, LabCorp Diagnostics’ operating expenses increased by $8.5 million in the quarter, and would have increased by $7.6 million in the fourth quarter of 2014. In addition, unallocated corporate expenses were reduced by $8.5 million in the quarter, and would have been reduced by $7.6 million in the fourth quarter of 2014. Reconciliations of segment results to historically reported results are included in the Condensed Pro Forma Segment Information tables and notes.

Fourth Quarter Pro Forma Segment Results

LabCorp Diagnostics
Net revenue for the quarter was $1.55 billion, an increase of 4.3% over $1.49 billion for the fourth quarter of 2014. The increase in net revenue was the result of organic volume growth (measured by requisitions), Beacon LBS, price, mix and tuck-in acquisitions, partially offset by currency. The increase in net revenue of 4.3% includes the benefit from Beacon LBS of 1.1%, and unfavorable foreign currency translation of 0.8%. Total volume (measured by requisitions) increased by 1.8% (organic volume of 1.6% and acquisition volume of 0.2%). Revenue per requisition increased by 2.2%.

Adjusted operating income (excluding amortization, restructuring and special items) for the quarter was $293.3 million, or 18.9% of net revenue, compared to adjusted operating income of $273.3 million, or 18.4% of net revenue, in the fourth quarter of 2014. The increase was primarily due to volume, price, mix, and productivity, partially offset by personnel costs. Improvement in productivity was driven by Project LaunchPad, which generated approximately $20 million in net benefits during the quarter.

Covance Drug Development
Net revenue for the quarter was $691.4 million, an increase of 4.7% over $660.1 million for the fourth quarter of 2014. The stronger U.S. Dollar negatively impacted year-over-year revenue growth by approximately 230 basis points. Excluding currency, net revenue increased 7.0% year-over-year due to increased demand.

Adjusted operating income (excluding amortization, restructuring and special items) was $110.4 million, or 16.0% of net revenue, compared to adjusted operating income of $89.8 million, or 13.6% of net revenue, in the fourth quarter of 2014. The increase was primarily due to demand, productivity and cost synergies, partially offset by personnel costs. The Company generated approximately $15 million in cost synergies during the quarter.

Net orders (gross orders less cancellations and reductions) in the quarter were $816 million, representing a net book-to-bill of 1.18. Backlog at December 31, 2015 was approximately $6.7 billion.

Outlook for 2016
The following guidance assumes foreign exchange rates effective as of January 31, 2016 for the full year.

Net revenue growth of 7.5% to 9.5% over 2015 net revenue of $8.51 billion, which includes the impact from approximately 100 basis points of negative currency.

Net revenue growth in LabCorp Diagnostics of 3.5% to 5.5% over 2015 pro forma revenue of $6.21 billion, which includes the impact from approximately 50 basis points of negative currency.

Net revenue growth in Covance Drug Development of 2% to 5% over 2015 pro forma revenue of $2.63 billion. Excluding the impact from approximately 200 basis points of negative currency and the anticipated expiration of the Sanofi site support agreement, net revenue is expected to increase 6.6% to 9.6%.

Adjusted EPS of $8.45 to $8.85, an increase of approximately 7% to 12% over the prior year.

Free cash flow (operating cash flow less capital expenditures) of $900 million to $950 million, an increase of approximately 24% to 31% over the prior year.

"We are enthusiastic about our prospects for 2016," Mr. King added. "We are focused on improving health and improving lives through execution of our strategic priorities: delivering world class diagnostics, bringing innovative medicines to patients faster, and changing the way care is provided. This focus will drive top-line growth and margin expansion, resulting in attractive year-over-year EPS growth and free cash flow."

Use of Adjusted Measures
The Company has provided in this press release and accompanying tables "adjusted" financial information that has not been prepared in accordance with GAAP, including Adjusted EPS, Adjusted Operating Income, Free Cash Flow, and certain segment information. The Company believes these adjusted measures are useful to investors as a supplement to, but not as a substitute for, GAAP measures, in evaluating the Company’s operational performance. The Company further believes that the use of these non-GAAP financial measures provides an additional tool for investors in evaluating operating results and trends, and growth and shareholder returns, as well as in comparing the Company’s financial results with the financial results of other companies. However, the Company notes that these adjusted measures may be different from and not directly comparable to the measures presented by other companies. Reconciliations of these non-GAAP measures to the most comparable GAAP measures are included in the tables accompanying this press release.

The Company today is furnishing a Current Report on Form 8-K that will include additional information on its business and operations. This information will also be available in the investor relations section of the Company’s website at www.labcorp.com. Analysts and investors are directed to the Current Report on Form 8-K and the website to review this supplemental information.

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.