On November 3, 2022 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare and genetically defined diseases, reported business highlights and financial results for the third quarter ended September 30, 2022 (Press release, Agios Pharmaceuticals, NOV 3, 2022, View Source [SID1234622990]).
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"Agios is on the cusp of changing the treatment landscape for people with rare and genetically defined diseases, with the potential of PYRUKYND to make a positive impact across multiple underserved diseases. I joined the company in August because of its differentiated portfolio, top-notch team and genuine dedication to patients, and my excitement for the future of Agios has only grown over the past few months," said Brian Goff, chief executive officer at Agios. "We are continuing to build our capabilities and connections in rare and genetically defined diseases through our U.S. launch of PYRUKYND, and we expect our learnings to support anticipated future expansion in related diseases where development efforts are ongoing. We will build on the tremendous accomplishments we’ve achieved in 2022 as we close out the year with a focus on continuing to execute the launch, enrolling our thalassemia and sickle cell disease pivotal trials and securing the approval of PYRUKYND for PK deficiency in the EU and Great Britain. I am honored to lead this team as we together drive long-term growth and value for patients, shareholders and all our stakeholders."
Third Quarter 2022 & Recent Highlights
Continued to execute U.S. launch of PYRUKYND, generating $3.5 million in U.S. net revenue for the third quarter of 2022, the second full quarter following FDA approval. A total of 84 unique patients have completed prescription enrollment forms, representing an increase of 64 percent over the second quarter. A total of 56 patients are on PYRUKYND therapy, representing a 51 percent increase over the second quarter. Increased patient demand was partially offset by modest inventory build in the prior quarters of launch.
Received positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending the granting of a marketing authorization for PYRUKYND for the treatment of PK deficiency in adult patients.
Completed Medicines and Healthcare Products Regulatory Agency (MHRA) filing for the approval of PYRUKYND as a treatment for adults with PK deficiency in Great Britain.
Continued to enroll patients across five PYRUKYND pivotal studies in thalassemia, sickle cell disease and pediatric PK deficiency.
Initiated Phase 2a study of novel PK activator AG-946 in adults with lower-risk MDS.
Published PYRUKYND data in top-tier medical journals, including Phase 2 thalassemia data in The Lancet and Phase 3 ACTIVATE-T data in The Lancet Haematology.
Appointed Cecilia Jones as Agios’ chief financial officer, effective Sept. 26, 2022.
Appointed Rahul Ballal, Ph.D., chief executive officer of Imara, and Cynthia Smith, former chief commercial officer of ZS Pharma, to Agios’ board of directors.
Completed the sale of royalty rights on U.S. net sales of Servier’s TIBSOVO to Sagard Healthcare Partners for a one-time payment of $131.8 million.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following key milestones and priorities by the end of 2022:
Adult PK Deficiency: Receive EU and Great Britain regulatory decisions for PYRUKYND in adults with PK deficiency.
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.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in adults with sickle cell disease.
Data Presentations: Present broad set of clinical and translational data at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition; abstracts will be available at 9 a.m. ET today.
Third 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 third quarter of 2022 was $3.5 million. This revenue reflects the second full quarter of PYRUKYND launch, following FDA approval on February 17, 2022.
Cost of Sales: Cost of sales for the third quarter of 2022 was $0.5 million.
Non-Operating Income: Non-operating income included approximately $4.4 million from TIBSOVO royalties for the third quarter of 2022. TIBSOVO royalty income will cease in 2022 due to the sale of these royalty rights to Sagard Healthcare Partners.
Research and Development (R&D) Expenses: R&D expenses were $65.0 million for the third quarter of 2022 compared to $64.0 million for the third quarter of 2021.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $29.1 million for the third quarter of 2022 compared to $27.2 million for the third quarter of 2021. The year-over-year increase in SG&A expenses was primarily attributable to an increase in workforce-related expenses.
Net Loss from Continuing Operations: Net loss from continuing operations was $81.7 million for the third quarter of 2022 compared to a net loss of $84.3 million for the third quarter of 2021.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2022, were $1.0 billion compared to $1.4 billion as of September 30, 2021. This cash position does not include the receipt of a one-time payment of $131.8 million associated with the sale of royalty rights on U.S. net sales of Servier’s TIBSOVO. 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 third 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.