Agios Reports Business Highlights and Third Quarter 2019 Financial Results
– TIBSOVO® Net Revenue of
– Reported Positive Phase 3 ClarIDHy Results for TIBSOVO® in Previously Treated IDH1 Mutant Cholangiocarcinoma; sNDA Submission Planned by Year-End –
– Presented Data from Single Agent Dose-Escalaton Arm of the Phase 1 Study of AG-270 in MTAP Deleted Tumors; Combination Arms with Taxanes in Non-Small Cell Lung Cancer and Pancreatic Cancer Initiated –
– Published Updated DRIVE PK Data for Mitapivat in PK Deficiency in
“The third quarter was marked by strong commercial execution, now a full year since the U.S. launch of TIBSOVO®, and several meaningful clinical updates spanning our early and late stage oncology programs that advance our strategy,” said
THIRD QUARTER 2019 HIGHLIGHTS & RECENT PROGRESS
- Presented data from the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors at the
AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeuticsin October. The data demonstrated that AG-270 induces reductions in the biomarkers of methionine adenosyltransferase 2A (MAT2A) inhibition, notably plasma concentrations of S-adenosylmethionine (SAM) and tumor levels of symmetrically demethylated arginine (SDMA), at well tolerated doses.
- Presented results from the Phase 3 ClarIDHy study of TIBSOVO® in previously treated isocitrate dehydrogenase-1 (IDH1) mutant cholangiocarcinoma at the
European Society for Medical Oncology Congressin September, demonstrating significant improvement in progression free survival compared to placebo.
- Initiated two combination arms for the Phase 1 study of AG-270 in MTAP-deleted tumors, one evaluating AG-270 in combination with docetaxel in MTAP-deleted second-line non-small cell lung cancer and another in combination with nab-paclitaxel and gemcitabine in MTAP-deleted first or second-line pancreatic ductal adenocarcinoma.
- Published new data from the core and extension phases of the DRIVE PK Phase 2 study of mitapivat in adults with pyruvate kinase (PK) deficiency in the
September 5, 2019issue of the New England Journal of Medicine, demonstrating sustained increases in hemoglobin for up to 35 months.
KEY UPCOMING MILESTONES
The company plans to achieve the following key milestones in the remainder of 2019:
- Submit a supplemental new drug application to the
FDAfor TIBSOVO® for previously treated IDH1 mutant cholangiocarcinoma by year-end.
- Initiate the registration-enabling Phase 3 INDIGO study of vorasidenib in Grade 2 non-enhancing glioma with an IDH mutation by year-end. The study will evaluate 366 patients in 1:1 double-blind randomization to either 50 mg of vorasidenib once daily or placebo. The primary endpoint is progression free survival.
Rare Genetic Diseases:
- Complete enrollment in two global pivotal trials for mitapivat in adults with PK deficiency by year-end:
- ACTIVATE-T: A single-arm trial of up to 40 regularly transfused patients
- ACTIVATE: A 1:1 randomized, placebo-controlled trial of up to 80 patients who do not receive regular transfusions
- Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
REMAINING 2019 DATA PRESENTATIONS
- Updated data from the perioperative study of TIBSOVO® and vorasidenib in low-grade glioma have been accepted for presentation at the
Society for Neuro-OncologyAnnual Meeting taking place in Phoenixfrom November 22-24, 2019.
- Data from the IDH and PKR programs have been accepted for presentation at the
American Society of HematologyAnnual Meeting taking place in Orlando, Fla.from December 7-10, 2019, including new data from the extension phase of the Phase 2 DRIVE PK study of mitapivat in adults with PK deficiency and important translational data from the Phase 1 combination study of TIBSOVO® and azacitidine in frontline acute myeloid leukemia (AML).
THIRD QUARTER 2019 FINANCIAL RESULTS
Revenue: Total revenue for the third quarter of 2019 was
Cost of Sales: Cost of sales were
Research and Development (R&D) Expenses: R&D expenses were
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 and adjacent areas of biology. 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®, IDHIFA®, vorasidenib, 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 inthis 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 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; 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
|Condensed Consolidated Balance Sheet Data|
|Cash, cash equivalents and marketable securities||$||540,476||$||805,421|
|Accounts receivable, net||7,106||5,076|
|Collaboration receivable – related party||1,838||2,462|
|Royalty receivable – related party||2,600||2,234|
|Deferred revenue – related party||66,670||92,519|
|Condensed Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
|Nine Months Ended
|Product revenue, net||$||17,422||$||4,465||$||40,287||$||4,465|
|Collaboration revenue – related party||5,516||8,732||32,414||42,478|
|Collaboration revenue – other||420||-||2,202||12,440|
|Royalty revenue – related party||2,666||2,001||7,569||4,991|
|Cost and expenses:|
|Cost of sales||393||695||1,030||695|
|Research and development, net||101,672||82,561||304,646||247,515|
|Selling, general and administrative||33,019||31,104||97,200||82,287|
|Total cost and expenses||135,084||114,360||402,876||330,497|
|Loss from operations||(109,060||)||(99,162||)||(320,404||)||(266,123||)|
|Net loss per share – basic and diluted||$||(1.81||)||$||(1.63||)||$||(5.27||)||$||(4.45||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||58,803,534||58,033,386||58,661,607||57,158,492|
Investor & Media Contact:
Associate Director, Investor Relations
Source: Agios Pharmaceuticals, Inc.