On November 5, 2015 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 third quarter ended September 30, 2015 (Press release, Agios Pharmaceuticals, NOV 5, 2015, View Source;p=RssLanding&cat=news&id=2107178 [SID:1234507987]).
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"We have made significant progress this year toward realizing our goal of what’s possible for patients with our IDH inhibitors, by deploying a comprehensive development strategy of speed and breadth with AG-221 and AG-120 in AML and other cancers," said David Schenkein, M.D., chief executive officer at Agios. "In addition, we are pleased to have selected our fifth molecule, AG-519, for clinical development in PK deficiency. This coupled with DRIVE PK, our ongoing Phase 2 study of AG-348, may optimize our potential to help people with this rare genetic disorder."
KEY UPCOMING MILESTONES IN CANCER METABOLISM
Agios anticipates the following milestones from its IDH clinical development programs in collaboration with Celgene:
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein
Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-221 in advanced IDH2-mutant positive hematologic malignancies at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place December 5-8, 2015 in Orlando.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
Present first data from the ongoing Phase 1 dose-escalation trial of AG-120 in advanced IDH1-mutant positive solid tumors 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) on November 8, 2015 in Boston.
Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-120 in advanced IDH1-mutant positive hematologic malignancies at the ASH (Free ASH Whitepaper) Annual Meeting.
Initiate a global registration-enabling Phase 3 study in patients with acute myeloid leukemia (AML) harboring an IDH1 mutation in the first half of 2016.
AG-221 and AG-120 front-line AML combination trials
Initiate a Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy in newly diagnosed AML patients eligible for intensive chemotherapy by the end of 2015.
Initiate a Phase 1/2 combination study of either AG-221 or AG-120 with VIDAZA (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016.
KEY UPCOMING MILESTONES IN RARE GENETIC METABOLIC DISORDERS
AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency
Present data from the Phase 1 healthy volunteers study of AG-348 and new findings from the Natural History Study of PK deficiency (being conducted with Boston Children’s Hospital) at the ASH (Free ASH Whitepaper) Annual Meeting.
AG-519: a novel, oral activator of PKR for the treatment of PK deficiency
Initiate an integrated single ascending dose (SAD) and multiple ascending dose (MAD) placebo-controlled Phase 1 study in healthy volunteers in the first quarter of 2016.
RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM
Agios has provided the following updates on its clinical development programs in collaboration with Celgene:
AG-221
IDHENTIFY, the Phase 3 study of AG-221, was initiated in October. 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. This study is being conducted by Celgene.
The expansion phase of the Phase 1 trial of AG-221 is on track and continues to enroll. It includes four cohorts with 25 patients each and a fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or reinduction treatment, or have relapsed after allogeneic transplantation.
The ongoing Phase 1 trial of AG-221 in IDH2-mutated advanced solid tumors and angioimmunoblastic T-cell lymphoma continues to enroll patients.
AG-120
The expansion phase of the Phase 1 trial of AG-120 is on track and continues to enroll. It includes three expansion cohorts of a total of 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML.
The ongoing Phase 1 trial of AG-120 in IDH1-mutated advanced solid tumors continues to enroll.
AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins
Two Phase 1, open-label, dose-escalation and expansion studies are on track and continue to enroll – the first in advanced IDH mutant-positive solid tumors and the second in patients with advanced IDH mutant-positive hematologic malignancies whose cancer has progressed on a prior IDHm inhibitor therapy.
RECENT DEVELOPMENT UPDATES IN RARE GENETIC DISORDERS OF METABOLISM
AG-348
DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency, is on track and enrolling.
A natural history study of PK deficiency is also ongoing and patient enrollment is on track.
AG-519
Agios selected a fifth molecule for clinical development, AG-519, a novel, oral activator of PKR for the treatment of PK deficiency.
THIRD QUARTER 2015 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of September 30, 2015 were $408.0 million, compared to $467.4 million as of December 31, 2014. The decrease was driven by cash used to fund operating activities of approximately $101.2 million, which was offset by funding of approximately $54.8 million made by Celgene during the nine months ended September 30, 2015 related to our collaboration agreements.
Collaboration revenue was $5.5 million for the third quarter of 2015, compared to $33.9 million for the comparable period in 2014. In July 2014, the company amended its collaboration agreement with Celgene. As a result, for the third quarter of 2014 the company recognized a total of $25.9 million under the previous accounting guidance and upon the modification in addition to $8.0 million in revenue subsequent to the modification through September 30, 2014.
Research and development (R&D) expense was $36.0 million, including $4.9 million of stock- based compensation expense in the third quarter of 2015, compared to $25.5 million, including $1.4 million in stock-based compensation expense for the comparable period in 2014. The increase in R&D expense was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development.
General and administrative (G&A) expense was $9.9 million, including $4.5 million of stock-based compensation expense, in the third quarter of 2015, compared to $5.2 million, including $1.4 million of stock-based compensation expense, for the comparable period in 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 third quarter of 2015 was $40.3 million, compared to net income of $3.7 million for the comparable period in 2014. The third quarter of 2014 includes additional revenue recognition related to the amendment of the company’s collaboration agreement with Celgene.
FINANCIAL GUIDANCE FOR THE FULL YEAR 2015
Agios is reiterating that it expects to end 2015 with more than $350.0 million of cash, cash equivalents and marketable securities. The anticipated year end 2015 cash position does not include any additional program-specific milestone payments. Agios expects that its cash, cash equivalents and marketable securities would be sufficient to fund its operating expenses and capital expenditure requirements until late 2017.