On August 2, 2018 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2018 (Press release, Agios Pharmaceuticals, AUG 2, 2018, View Source [SID1234528359]). In addition, Agios highlighted select corporate milestones and clinical data from its development programs.
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"The first half of 2018 has been productive across all aspects of our business, culminating in the recent approval and launch of our second internally discovered medicine," said David Schenkein, M.D., chief executive officer at Agios. "This achievement sets us well on the path to becoming a sustainable, multiproduct company with a thriving research engine on track to submit its 7th IND and a broad clinical development program with multiple trials planned or underway to expand our oncology and rare genetic disease portfolios."
SECOND QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS
Received full approval from the U.S. Food and Drug Administration (FDA) on July 20, 2018 for TIBSOVO (ivosidenib) for the treatment of patients with relapsed or refractory AML (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA approved test.
Entered into an exclusive license agreement with CStone Pharmaceuticals to develop and commercialize ivosidenib in Greater China, resulting in a $12 million upfront payment and the potential for $412 million in development and commercial milestones.
Initiated ACTIVATE, a global, placebo-controlled, pivotal trial for mitapivat (AG-348) in approximately 80 adults with PK deficiency who do not receive regular blood transfusions. A second pivotal trial (ACTIVATE-T) in PK deficiency patients who receive regular blood transfusions is ongoing.
Presented new and updated data from the IDH programs at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago. Links to the data presentations, including updated data from the Phase 1 trial combining ivosidenib and azacitidine in the frontline AML setting can be found here.
Secured publication of the ivosidenib Phase 1 data in patients with IDH1m advanced hematological malignancies in theNew England Journal of Medicine.
Supported publication of the results from the Pyruvate Kinase Deficiency Natural History Study in the journal Blood.
Disclosed active research programs in three rare genetic diseases: phenylketonuria, erythroid porphyria and Friedreich’s ataxia, as part of a preclinical pipeline update at the company’s Investor Day in May.
KEY UPCOMING MILESTONES
The company expects to achieve the following remaining milestones in 2018:
Cancer:
Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO (ivosidenib) for the treatment of patients with R/R AML and an IDH1 mutation in the fourth quarter of 2018.
Support, in conjunction with Celgene, the initiation of HO150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
Rare Genetic Diseases:
Initiate a Phase 2 proof of concept trial of mitapivat (AG-348) in thalassemia in the fourth quarter of 2018.
Research:
Submit an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
EXPECTED FOURTH QUARTER CLINICAL DATA PRESENTATIONS
Updated data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation has been submitted to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition on December 1-4 in San Diego.
Updated data in untreated AML from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH (Free ASH Whitepaper).
SECOND QUARTER 2018 FINANCIAL RESULTS
Revenue for the quarter ended June 30, 2018 was $40.4 million, which includes $26.4 million of collaboration revenue and $1.6 million of royalty revenue from net U.S. sales of IDHIFA under our collaboration agreements with Celgene, and $12.4 million of collaboration revenue under our agreement with CStone. Revenue for the quarter ended June 30, 2017 was $11.3 million and consisted solely of collaboration revenue under our agreements with Celgene. The year over year increase in collaboration revenue for the second quarter was primarily driven by the $15.0 million milestone related to Celgene’s filing of an MAA to the EMA for IDHIFA and $12.4 million related to the delivery of the license under the CStone Agreement.
Research and development (R&D) expenses were $86.7 million, including $9.7 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $79.8 million, including $8.2 million in stock-based compensation expense, for the comparable period in 2017. The increase in R&D expense was primarily attributable to start-up costs for the mitapivat (AG-348) pivotal program in PK deficiency, including the initiation of the ACTIVATE-T trial. R&D expense also increased as a result of IND enabling activities for AG-636, our DHODH inhibitor.
General and administrative (G&A) expenses were $26.6 million, including $6.8 million of stock-based compensation expense, for the quarter ended June 30, 2018, compared to $16.1 million, including $4.0 million of stock-based compensation expense, for the quarter ended June 30, 2017. The increase in G&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO.
Net loss for the quarter ended June 30, 2018 was $68.7 million, compared to a net loss of $83.1 million for the quarter ended June 30, 2017.
Cash, cash equivalents and marketable securities as of June 30, 2018 were $936.6 million, compared to $567.8 million as of December 31, 2017. The increase in cash was driven by the net proceeds of $516.2 million from the January follow on offering, $8.9 million of cost reimbursements under our collaboration agreements with Celgene and $22.0 million received from employee stock transactions. This was offset by expenditures to fund operations of $178.1 million during the six months ended June 30, 2018.
The company expects that its cash, cash equivalents and marketable securities as of June 30, 2018, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2018 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 1497883. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.