On August 4, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported business highlights and financial results for the second quarter ended June 30, 2022 (Press release, Agios Pharmaceuticals, AUG 4, 2022, View Source [SID1234617451]).

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"As we look ahead to the next chapter for the company, Agios is operating from a position of strength. We are executing our commercial launch of PYRUKYND, the first therapy for a rare, debilitating, lifelong blood disorder; we also have five pivotal trials underway, multiple early-stage studies planned or ongoing, a promising preclinical pipeline and a strong balance sheet providing optionality for the future growth of the business," said Jackie Fouse, Ph.D., chief executive officer at Agios. "As I reflect on my legacy as CEO of Agios, I am proud of the bold and strategic decisions we have made to maximize and accelerate our impact for people with genetically defined diseases. I am tremendously grateful to the Agios team for their unwavering resiliency in the face of unprecedented challenges and heartfelt dedication to patients and each other. I look forward to my new role at Agios as board chair and to supporting Brian as he leverages his expertise to expand Agios’ genetically defined disease capabilities, foster the company’s differentiated culture and drive our next phase of impact."

Second Quarter 2022 & Recent Highlights

Continued to execute U.S. launch of PYRUKYND, generating $3.1 million in U.S. net revenue for the second quarter of 2022, the first full quarter following FDA approval.
Initiated Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively.
Announced that effective August 8, 2022, Dr. Fouse will transition to the role of chair of Agios’ board of directors and Brian Goff will assume the role of Agios’ chief executive officer and member of the board of directors.
Evolved Agios’ research approach to focus on advancing the company’s existing validated preclinical programs and in-licensing or acquiring well-characterized, high-potential assets.
Expanded role of Sarah Gheuens, M.D., Ph.D., to chief medical officer and head of research and development, incorporating research and discovery sciences in addition to her existing chief medical officer responsibilities.
Presented clinical and translational data at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress, including new data supporting the potential benefits of PYRUKYND treatment in adults with PK deficiency.
Key Upcoming Milestones & Priorities

Agios expects to execute on the following key milestones and priorities in 2022:

Adult PK Deficiency: Receive European Medicines Agency (EMA) regulatory decision for PYRUKYND in adults with PK deficiency by year-end.
Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease by year-end.
Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
Data Presentations: Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
Second Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND for the second quarter of 2022 was $3.1 million. This revenue reflects the first full quarter of PYRUKYND launch, following FDA approval on February 17, 2022. In addition, Agios recognized revenue of $2.5 million dollars in the second quarter of 2022 as an up-front payment associated with the licensing of intellectual property for the company’s Friedreich’s Ataxia preclinical program.

Cost of Sales: Cost of sales for the second quarter of 2022 was $0.4 million.

Non-Operating Income: Non-operating income included approximately $2.7 million from TIBSOVO (ivosidenib) royalties for the second quarter of 2022.

Research and Development (R&D) Expenses: R&D expenses were $74.5 million for the second quarter of 2022 compared to $62.0 million for the second quarter of 2021. The year-over-year increase in R&D was driven primarily by increased headcount and workforce-related expenses, planned increased activity associated with the PAH preclinical program, start-up costs for the AG-946 Phase 2a MDS study, increased spend for the AG-946 Phase 1 trial, and start-up costs for the PYRUKYND pivotal studies in sickle cell disease and pediatric PK deficiency.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.3 million for the second quarter of 2022 compared to $29.2 million for the second quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to the completion of the reimbursable transition services Agios provided to Servier, which concluded in the first quarter of 2022, related to the sale of the oncology business.

Net Loss from Continuing Operations: Net loss from continuing operations was $91.8 million for the second quarter of 2022 compared to a net loss of $82.8 million for the second quarter of 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2022, were $1.1 billion compared to $1.7 billion as of June 30, 2021. The year-over-year decrease is attributable to operating expenses and 5.7 million shares of common stock that the company repurchased for $273.4 million during the third and fourth quarters of 2021. Agios expects that its cash, cash equivalents and marketable securities will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2022 financial results and recent business activities. 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.