On July 30, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2020 (Press release, Agios Pharmaceuticals, JUL 30, 2020, View Source [SID1234562577]).

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"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 Jackie Fouse, Ph.D., chief executive officer at Agios. "For the remainder of 2020, we are focused on the completion of our pivotal trials ACTIVATE and ACTIVATE-T for mitapivat in pyruvate kinase deficiency and securing regulatory feedback on the pivotal programs in both thalassemia and sickle cell disease to enable their initiation next year, the submission of a supplemental new drug application for TIBSOVO in cholangiocarcinoma in the first quarter of 2021, driving enrollment in our ongoing clinical trials and continued strong commercial execution."

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) (Free EHA Whitepaper) 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) (Free ASCO Whitepaper) 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.
Corporate

Completed a $255 million purchase 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 $105-115 million.
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.
Research

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 $37.3 million, which includes $27.6 million of net product revenue from sales of TIBSOVO, $6.4 million in collaboration revenue and $3.3 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene, now a wholly owned subsidiary of Bristol Myers Squibb. This compares to revenue of $26.2 million for the second quarter of 2019. TIBSOVO net product revenue increased 101% from the same period last year.

Cost of Sales: Cost of sales were $0.7 million for the second quarter of 2020 compared to $0.3 million for the second quarter of 2019.

Research and Development (R&D) Expenses: R&D expenses were $90.9 million for the second quarter of 2020 compared to $107.4 million for the second quarter of 2019. The decrease in R&D expense was primarily attributable to milestone payments related to AG-636 and an undisclosed early-stage research program in the second quarter of 2019, winding down the ClarIDHy Phase 3 study of TIBSOVO and HOVON startup expenses incurred in the second quarter of 2019, and lower spend across ongoing TIBSOVO clinical studies as a result of slowed enrollment and reduced activities due to the COVID-19 pandemic.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $36.0 million for the second quarter of 2020 compared to $32.4 million for the second quarter of 2019. The increase in SG&A expense was primarily attributable to the initial gated infrastructure build of the company’s European operations offset by reduced travel and industry engagement given restrictions in place to combat the COVID-19 pandemic.

Net Loss: Net loss was $90.5 million for the second quarter of 2020 compared to $109.9 million for the second quarter of 2019.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2020 were $794 million, including the amount received under the Royalty Pharma agreement, compared to $624 million as of June 30, 2019. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2020, together with anticipated product 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, including its pivotal development programs for mitapivat in thalassemia and sickle cell disease, through the end of 2022.

CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss its second quarter 2020 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 2955575. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

About the Agios/Celgene Collaboration
In 2010, Agios and Celgene Corporation, now a wholly owned subsidiary of Bristol Myers Squibb, entered into a collaboration agreement focused on cancer metabolism. Under the terms of the agreement, Celgene has worldwide development and commercialization rights for IDHIFA (enasidenib). Celgene and Agios are currently co-commercializing IDHIFA in the U.S., and Agios continues to conduct certain clinical development activities within the IDHIFA development program. Agios is eligible to receive a $25 million payment upon achievement of a specified ex-U.S. commercial milestone event, as well as reimbursement for costs incurred for its co-commercialization efforts and development activities.