Agios Reports Fourth Quarter and Full Year 2018 Financial Results
– Strong Launch of TIBSOVO® Continues with Net Revenue of
– Phase 3 ClarIDHy Trial of TIBSOVO® in Second Line or Later Cholangiocarcinoma Fully Enrolled –
– AG-270 Preclinical Data Accepted for Presentation at AACR; Data from Perioperative Study of TIBSOVO® and Vorasidenib in Low Grade Glioma Submitted for Presentation at
– Dr. Jackie Fouse Assumed Role as Chief Executive Officer on
“I’m excited to join the Agios team on the heels of a transformational year for the company. During 2018, we launched our first wholly owned oncology medicine, expanded our clinical programs across both oncology and rare genetic diseases, and continued to advance our robust research pipeline,” said
KEY UPCOMING MILESTONES
The company plans to achieve the following key milestones in 2019:
FDAapproval of the supplemental new drug application (sNDA) for single agent TIBSOVO® (ivosidenib) for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy and subsequent launch in this indication in the U.S.
- Submit an sNDA to the
FDAfor TIBSOVO® for second line or later IDH1 mutant cholangiocarcinoma by year-end.
- Initiate a registration-enabling Phase 3 study of vorasidenib (AG-881) in low-grade glioma with an IDH1 mutation by year-end.
- Determine recommended dose of AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in methylthioadenosine phosphorylase (MTAP)-deleted tumors; initiate expansion arms, including a single-agent arm in a variety of MTAP-deleted cancers and a combination arm in a solid tumor in the first half of 2019.
- Initiate a Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in lymphoma in the first half of 2019.
Rare Genetic Diseases:
- Complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019:
- ACTIVATE-T: A single-arm trial of approximately 20 regularly transfused patients
- ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions
- Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
ANTICIPATED KEY 2019 DATA PRESENTATIONS
- Updated data from the ongoing Phase 1 combination trial of TIBSOVO® with azacitidine in patients with newly diagnosed AML with an IDH1 mutation to be presented at the 17th International Symposium on Acute Leukemias taking place
February 24-27, 2019in Munich.
- Preclinical data for AG-270 accepted for presentation at the
American Association for Cancer Research(AACR) meeting taking place March 29-April 3, 2019in Atlanta.
- Data from the perioperative 'window' trial with TIBSOVO® and vorasidenib in IDHm low-grade glioma submitted for presentation at the 2019
American Society of Clinical Oncology( ASCO) Annual Meeting taking place May 31-June 4, 2019in Chicago.
- Topline data from the Phase 3 ClarIDHy study of TIBSOVO® in IDH1 mutant second line or later cholangiocarcinoma to be reported in the first half and full data to be presented in the second half of 2019.
- Data from the dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors expected in the second half of 2019.
FOURTH QUARTER 2018 HIGHLIGHTS & RECENT PROGRESS
- Submitted an sNDA to the
FDAfor TIBSOVO® for the treatment of patients with newly diagnosed AML with an IDH1 mutation who are not eligible for standard therapy.
- Submitted and received validation for a Marketing Authorization Application to the European Medicines Agency for TIBSOVO® for the treatment of adult patients with R/R AML with an IDH1 mutation.
- Completed enrollment in the Phase 3 ClarIDHy study of TIBSOVO® in IDH1 mutant second line or later cholangiocarcinoma.
- Initiated a Phase 2 proof-of-concept trial of mitapivat in thalassemia.
FDAclearance of an IND application for AG-636, a DHODH inhibitor.
FOURTH QUARTER AND FULL YEAR 2018 FINANCIAL RESULTS
Revenue: Total revenue for the fourth quarter of 2018 was
Cost of Sales: We began U.S. sales of TIBSOVO® in the third quarter of 2018. Cost of sales were
Research and Development (R&D) Expenses: R&D expenses were $93.8 million for the fourth quarter of 2018 compared to
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were
Net Loss: Net loss was
Cash Position and Guidance: 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 Agios/Celgene Collaboration
IDHIFA® (enasidenib) and AG-270 are part of our 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 TIBSOVO® (ivosidenib), IDHIFA® (enasidenib), vorasidenib (AG-881), mitapivat, AG-270 and AG-636; the potential benefits of Agios' product candidates; its key milestones for 2019; its plans regarding future data presentations; its financial guidance regarding the period in which it will have capital available to fund its operations; and the potential benefit of its strategic plans and focus. The words “anticipate,” “expect,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios' current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios or its collaborators is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios' product candidates will successfully continue. There can be no guarantee that any positive developments in Agios' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including: Agios' results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S.
|Consolidated Balance Sheet Data
|Cash, cash equivalents and marketable securities||$||805,421||$||567,750|
|Accounts receivable, net||5,076||-|
|Collaboration receivable – related party||2,462||2,448|
|Royalty receivable – related party||2,234||1,222|
|Deferred revenue – related party||92,519||163,640|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended
|Product revenue, net||$||9,376||$||-||$||13,841||$||-|
|Collaboration revenue – related party||18,183||8,577||60,661||41,074|
|Collaboration revenue – other||230||-||12,670||-|
|Royalty revenue – related party||2,224||1,222||7,215||1,937|
|Cost and expenses:|
|Cost of sales||702||-||1,397||-|
|Research and development, net||93,809||77,216||341,324||292,681|
|Selling, general and administrative||31,858||22,713||114,145||71,124|
|Total cost and expenses||126,369||99,929||456,866||363,805|
|Loss from operations||(96,356||)||(90,130||)||(362,479||)||(320,794||)|
|Net loss per share – basic and diluted||$||(1.58||)||$||(1.81||)||$||(6.03||)||$||(6.75||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||58,189,254||48,772,901||57,418,300||46,587,631|
Senior Director, Investor & Public Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.