Agios Pharmaceuticals Inc (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2015.  In addition, Agios highlighted select corporate milestones for its preclinical and clinical development programs.

“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 placeFebruary 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 theEuropean Hematology Association (EHA) in June 2016
  • Plan to submit the first data from the Phase 1 study of AG-519 in healthy volunteers for presentation at EHA in June 2016. Preclinical findings about the molecule will also be submitted for presentation at EHA.
  • 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 Annual Meeting (ASH). 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-EORTC-NCI International Conference on Molecular Targets and Cancer Therapeutics 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 endedDecember 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. (Original Source)

Shares of Agios Pharmaceuticals closed yesterday at $41.93. AGIO has a 1-year high of $126.35 and a 1-year low of $33.50. The stock’s 50-day moving average is $47.30 and its 200-day moving average is $68.77.

On the ratings front, Agios has been the subject of a number of recent research reports. In a report issued on January 20, Credit Suisse analyst Kennen MacKay initiated coverage with a Buy rating on AGIO and a price target of $75, which implies an upside of 78.9% from current levels. Separately, on December 7, Leerink Swann’s Seamus Fernandez downgraded the stock to Hold and has a price target of $62.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Kennen MacKay and Seamus Fernandez have a total average return of -9.0% and 1.3% respectively. MacKay has a success rate of 15.4% and is ranked #2944 out of 3640 analysts, while Fernandez has a success rate of 48.8% and is ranked #1365.

Agios Pharmaceuticals Inc is engaged in the development of medicines to treat cancer metabolism and inborn errors of metabolism, which are a subset of orphan genetic metabolic diseases.