Agios Reports Second Quarter 2018 Financial Results
– TIBSOVO® Launch Underway Post FDA Approval for IDH1m R/R AML; Second Medicine Approved from Agios’ Discovery Platform in 12 Months –
– Entered License Agreement with
– Mitapivat (AG-348) Pivotal Program (ACTIVATE and ACTIVATE-T) Initiated for Pyruvate Kinase Deficiency –
– Company in Strong Financial Position with Q2 2018 Ending Cash,
“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
SECOND QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS
- Received full approval from the
U.S. Food and Drug Administration( FDA) on July 20, 2018for 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 FDAapproved test.
- Entered into an exclusive license agreement with
CStone Pharmaceuticalsto develop and commercialize ivosidenib in Greater China, resulting in a $12 millionupfront payment and the potential for $412 millionin 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) 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 the
New 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:
- 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.
- 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) Annual Meeting and Exposition on December 1-4in San Diego.
- Updated data in untreated AML from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH.
- Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of ivosidenib in IDH1m hematologic malignancies has been submitted to ASH.
SECOND QUARTER 2018 FINANCIAL RESULTS
Revenue for the quarter ended
Research and development (R&D) expenses were $86.7 million, including $9.7 million of stock-based compensation expense, for the quarter ended
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 $83.1 million for the quarter ended June 30, 2017.
Cash, cash equivalents and marketable securities as of
The company expects that its cash, cash equivalents and marketable securities as of
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.
About TIBSOVO® (ivosidenib)
TIBSOVO® (ivosidenib) is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an
About Agios/Celgene Collaboration
IDHIFA® (enasidenib) and AG-881 are part of Agios' global strategic collaboration with
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including IDHIFA® (enasidenib), TIBSOVO® (ivosidenib), AG-881, mitapivat (AG-348)
|Consolidated Balance Sheet Data
|Cash, cash equivalents and marketable securities||$||936,629||$||567,750|
|Collaboration receivable – related party||19,326||2,448|
|Royalty receivable – related party||1,573||1,222|
|Deferred revenue – related party||113,540||163,640|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended June 30,||Six Months Ended June 30,|
|Collaboration revenue – related party||$||26,401||$||11,346||$||33,746||$||21,854|
|Collaboration revenue – other||12,440||-||12,440||-|
|Royalty revenue – related party||1,573||-||2,990||-|
|Research and development, net||86,730||79,816||164,954||142,548|
|General and administrative||26,633||16,130||51,183||30,953|
|Total operating expenses||113,363||95,946||216,137||173,501|
|Loss from operations||(72,949)||(84,600)||(166,961)||(151,647)|
|Net loss per share – basic and diluted||(1.19)||(1.78)||(2.81)||(3.35)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||57,721,786||46,745,760||56,713,795||44,525,478|
Senior Manager, Investor Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.