Agios Reports Business Highlights and Second Quarter 2020 Financial Results
– Second Quarter TIBSOVO® Net Revenue of $27.6 Million; Company Reiterates 2020 TIBSOVO® Net U.S. Revenue Guidance of $105–115 Million –
– Significant Clinical Progress for First-in-Class PKR Activator Mitapivat: Established Proof-of-Concept in Sickle Cell Disease; Presented Phase 2 Data in α- and β-Thalassemia; Pivotal Programs to be Initiated in 2021 –
“The second quarter was a productive and important time at Agios, as we accomplished several key 2020 objectives across our three focus areas of malignant hematology, solid tumors and rare genetic diseases. In particular, we made significant progress on our mitapivat clinical programs, including achieving proof-of-concept in sickle cell disease and planning for our pivotal development programs in both thalassemia and sickle cell disease,” said
SECOND QUARTER 2020 HIGHLIGHTS
Rare Genetic Diseases
- Established clinical proof-of-concept for mitapivat in sickle cell disease based on a preliminary analysis of data on eight patients from the Phase 1 study being conducted in collaboration with the
National Institutes of Health(NIH). Seven of eight (88%) evaluable patients experienced a hemoglobin increase, with five of eight patients (63%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline. Additionally, the data showed improvements in associated markers of sickling as well as a safety profile consistent with previously reported mitapivat data or expected in the context of sickle cell disease.
- Presented data on 13 patients from the Phase 2 study of mitapivat in non-transfusion-dependent α- and β-thalassemia at the
European Hematology Association(EHA) Annual Congress in June. Treatment with mitapivat induced a hemoglobin increase of ≥1.0 g/dL in 12 of 13 (92%) evaluable patients, including four of four (100%) α-thalassemia patients. Additionally, the data showed improvements in associated markers of hemolysis and erythropoiesis as well as a safety profile consistent with previously reported mitapivat data.
- Received Orphan Drug Designation from the
Food and Drug Administration(FDA) for mitapivat in thalassemia.
Hematologic Malignancies and Solid Tumors
- TIBSOVO® net sales of
$27.6 million, an increase of 22% from the first quarter of 2020; expanded total number of unique prescribers by 15% from the first quarter of 2020.
- Published data from the Phase 3 ClarIDHy study of TIBSOVO® in The Lancet Oncology. As a result of this publication, the National Comprehensive Cancer Network (NCCN) guidelines were updated to recommend treatment with TIBSOVO® for patients with advanced IDH1-mutant cholangiocarcinoma.
- Presented updated data from the Phase 1 dose-escalation study of vorasidenib in IDH-mutant non-enhancing glioma at the American Society of Clinical Oncology (ASCO) Annual Meeting in May. In the study, vorasidenib demonstrated prolonged disease control and encouraging preliminary activity, as well as a favorable safety profile consistent with previously reported data.
- Completed a
$255 millionpurchase agreement with Royalty Pharma for IDHIFA® (enasidenib) royalty rights and outstanding regulatory milestone payments.
KEY UPCOMING MILESTONES
Rare Genetic Diseases
- Report data from ACTIVATE and ACTIVATE-T, the company’s two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency, between the end of 2020 and mid-2021.
- Finalize robust pivotal development plan for mitapivat in thalassemia, including both α-and β-thalassemia, as well as transfusion dependent and non-transfusion dependent patient populations, by the end of 2020.
- Initiate first-in-human study in healthy volunteers for AG-946, a next-generation PKR activator, in Q3 2020.
Hematologic Malignancies and Solid Tumors
- Deliver full-year 2020 U.S. revenue for TIBSOVO® of
- Receive European Medicines Agency CHMP opinion for TIBSOVO® in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by the end of 2020.
- Report mature overall survival data from ClarIDHy Phase 3 study in Q3 2020; if data are supportive, file supplemental new drug application (sNDA) for TIBSOVO® in previously treated IDH1-mutant cholangiocarcinoma in Q1 2021.
- Achieve at least one new development candidate by year-end 2020.
SECOND QUARTER 2020 FINANCIAL RESULTS
Revenue: Total revenue for the second quarter of 2020 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
About the Agios/Celgene Collaboration
Agios is focused on discovering and developing novel investigational medicines to treat malignant hematology, solid tumors and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across these three therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development. For more information, please visit the company's website at www.agios.com.
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 tablets), IDHIFA® (enasidenib), mitapivat, vorasidenib, AG-270, and AG-946; the potential benefits of Agios’ product candidates; its key milestones and guidance for 2020; 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 benefits of its strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” 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, without limitation: risks and uncertainties related to the impact of the COVID-19 pandemic to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; 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
|Consolidated Balance Sheet Data|
|Cash, cash equivalents, and marketable securities||$||794,413||$||717,806|
|Accounts receivable, net||12,023||8,952|
|Collaboration receivable – related party||2,537||1,539|
|Royalty receivable – related party||1,650||2,900|
|Deferred revenue – related party||—||61,513|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Product revenue, net||$||27,581||$||13,727||$||50,255||$||22,865|
|Collaboration revenue – related party||5,735||8,979||65,832||26,898|
|Collaboration revenue – other||692||812||1,685||1,782|
|Royalty revenue – related party||3,339||2,703||6,673||4,903|
|Cost and expenses:|
|Cost of sales||675||303||1,208||637|
|Research and development||90,917||107,389||182,173||202,974|
|Selling, general and administrative||35,951||32,390||74,452||64,181|
|Total cost and expenses||127,543||140,082||257,833||267,792|
|Loss from operations||(90,196||)||(113,861||)||(133,388||)||(211,344||)|
|Interest income, net||1,769||3,990||4,705||8,395|
|Non-cash interest expense for the sale of future revenue||(2,051||)||—||(2,051||)||—|
|Net loss per share – basic and diluted||$||(1.31||)||$||(1.87||)||$||(1.90||)||$||(3.46||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||68,958,091||58,722,244||68,784,109||58,589,167|
Director, Investor Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.