Agios Reports Third Quarter 2018 Financial Results
– Supplemental New Drug Application Submission for Single Agent TIBSOVO® (ivosidenib) in Newly Diagnosed IDH1m AML Patients Not Eligible for Standard Treatment Planned by the End of
– Enrollment for Phase 3 AGILE Trial of Ivosidenib Combination with Azacitidine in Newly Diagnosed AML Patients Not Eligible for Intensive Chemotherapy Expected to Complete in 2020 with Revised Primary Endpoint –
– Mitapivat Pivotal Trials ACTIVATE and ACTIVATE-T in Adults with PK Deficiency Expected to Complete Enrollment in 2019 –
– TIBSOVO® Launch in R/R IDH1m AML On-track; Reported Revenue of
"On the heels of the third quarter U.S. approval of TIBSOVO®, our first wholly owned precision medicine, we remain focused on executing on our remaining 2018 milestones and continuing to create value from our portfolio," said
THIRD QUARTER & RECENT 2018 HIGHLIGHTS
- Launched TIBSOVO® (ivosidenib) for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by a test approved by the
U.S. Food and Drug Administration( FDA).
- Awarded the U.S. Prix Galien Award for Best Pharmaceutical Product of 2018 for IDHIFA® (enasidenib), an isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation as detected by a test approved by the
FDA. Each year, the Prix Galien USA Committee recognizes outstanding achievements in improving the human condition through the development of innovative therapies.
- Reached agreement with the
FDAto submit a supplemental new drug application (sNDA) for single agent TIBSOVO® in newly diagnosed AML patients with an IDH1 mutation who are not eligible for standard treatment.
- Reached agreement with the
FDAthat event free survival (EFS) is an acceptable primary endpoint for the Phase 3 AGILE trial of ivosidenib combination with azacitidine in newly diagnosed AML patients with an IDH1 mutation who are ineligible for intensive chemotherapy. Full enrollment for AGILE is now expected to complete in 2020 vs. previous guidance of 2021.
- Received global rights to AG-881, a brain-penetrant, pan-IDH inhibitor that was previously part of a joint worldwide collaboration with
- Submitted an investigational new drug (IND) application for AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies.
- Announced that effective
February 1, 2019, CEO David Schenkein, M.D., will transition to the role of executive chairman of the board of directors and Jacqualyn (“Jackie”) Fouse, Ph.D., will succeed Dr. Schenkein as Agios’ next chief executive officer.
KEY UPCOMING MILESTONES
The company expects to achieve the following near-term milestones:
- Submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TIBSOVO® for the treatment of adult patients with R/R AML with an IDH1 mutation by year end 2018.
- Support, in conjunction with
Celgene, the initiation of HOVON 150, an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy followed by a maintenance therapy period in frontline AML patients with an IDH1 or IDH2 mutation, respectively, by year end 2018.
- Submit 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 by the end of January 2019.
Rare Genetic Diseases:
- Initiate a Phase 2 proof-of-concept trial of mitapivat in thalassemia by year end 2018.
FOURTH QUARTER CLINICAL DATA PRESENTATIONS
- Updated data from Phase 1 trial of AG-881 in solid tumors, including glioma, has been accepted as an oral presentation at the 2018
Society for Neuro-Oncology(SNO) Annual Meeting on November 15-18in New Orleans.
- Updated data from the ongoing Phase 1 trial of single agent ivosidenib in IDH1m hematologic malignancies in a subset of patients with newly diagnosed AML not eligible for standard therapy has been accepted as an oral presentation at the 2018
American Society of Hematology(ASH) Annual Meeting and Exposition on December 1-4in San Diego.
- 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 also been accepted as an oral presentation at ASH.
- Updated data in myelodysplastic syndrome (MDS) from the ongoing Phase 1 study of single agent ivosidenib in IDH1m hematologic malignancies has been accepted as a poster presentation at ASH.
THIRD QUARTER 2018 FINANCIAL RESULTS
Revenue for the quarter ended September 30, 2018 was
Cost of sales for the quarter ended September 30, 2018 were
Research and development (R&D) expenses were $82.6 million, including $13.4 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $72.9 million, including $7.6 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 and IND enabling activities for AG-636, our DHODH inhibitor. R&D expense also increased as a result of ongoing research efforts across our discovery platform programs.
Sales, general and administrative (SG&A) expenses were $31.1 million, including $10.8 million of stock-based compensation expense, for the quarter ended September 30, 2018, compared to $17.5 million, including $4.6 million of stock-based compensation expense, for the quarter ended September 30, 2017. The increase in SG&A expense was primarily attributable to the growth in our U.S. commercial organization to support the launch of TIBSOVO® and personnel costs related to increased headcount.
Net loss for the quarter ended September 30, 2018 was $94.7 million, compared to a net loss of $77.1 million for the quarter ended September 30, 2017.
Cash, cash equivalents and marketable securities as of September 30, 2018 were
The company expects that its cash, cash equivalents and marketable securities as of September 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
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-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 IDHIFA® (enasidenib), TIBSOVO® (ivosidenib), AG-881, mitapivat, AG-270 and AG-636; the potential benefits of Agios' product candidates; its key milestones for 2018; 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,” “believe,” “could,” “estimate,” “expect,” “hope,” “intend,” “may,” “milestone,” “path,” “plan,” “possible,” “potential,” “predict,” “prepare,” “project,” “strategy,” “will,” “would,” 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 collaborator, Celgene, 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. FDA and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios' ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios' ability to maintain key collaborations, such as its agreements with Celgene and CStone Pharmaceuticals; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
|Consolidated Balance Sheet Data
|Cash, cash equivalents and marketable securities||$||878,400||$||567,750|
|Accounts receivable, net||2,631||-|
|Collaboration receivable – related party||3,395||2,448|
|Royalty receivable – related party||1,863||1,222|
|Deferred revenue – related party||108,435||163,640|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Product revenue, net||$||4,465||$||-||$||4,465||$||-|
|Collaboration revenue – related party||8,732||10,643||42,478||32,497|
|Collaboration revenue – other||-||-||12,440||-|
|Royalty revenue – related party||2,001||715||4,991||715|
|Cost and expenses:|
|Cost of sales||695||-||695||-|
|Research and development, net||82,561||72,917||247,515||215,465|
|Sales, general and administrative||31,104||17,458||82,287||48,411|
|Total cost and expenses||114,360||90,375||330,497||263,876|
|Loss from operations||(99,162||)||(79,017||)||(266,123||)||(230,664||)|
|Net loss per share – basic and diluted||(1.63||)||(1.59||)||(4.45||)||(4.94||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||58,033,386||48,459,424||57,158,492||45,851,203|
Associate Director, Investor Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.