On August 3, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported business highlights and financial results for the second quarter ended June 30, 2023 (Press release, Agios Pharmaceuticals, AUG 3, 2023, View Source [SID1234633731]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"Since our last quarterly update, Agios has made tremendous progress executing across our industry-leading pipeline of PK activators, and today we are further expanding our portfolio beyond PK activation through focused business development," said Brian Goff, chief executive officer at Agios. "We announced positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease, completed enrollment in three clinical studies, licensed a compelling preclinical program from Alnylam, and continued to strengthen our commercial capabilities to support future anticipated launches. We look forward to the readout of the Phase 2a study of AG-946 in lower-risk MDS by the end of this year and the readouts of the Phase 3 studies of mitapivat in thalassemia next year."
Second Quarter 2023 & Recent Highlights
PYRUKYND U.S. Launch: Generated $6.7 million in U.S. net revenue for the second quarter of 2023, a 20 percent increase over the first quarter of 2023. A total of 147 unique patients have completed prescription enrollment forms, representing an increase of 16 percent over the first quarter of 2023. A total of 99 patients are on PYRUKYND therapy, representing an 11 percent increase over the first quarter of 2023.
Sickle Cell Disease: Announced positive results from the Phase 2 portion of the RISE UP pivotal study of mitapivat in sickle cell disease.
Thalassemia: Completed enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in not regularly transfused and regularly transfused adults with thalassemia, respectively.
Lower-Risk Myelodysplastic Syndromes (LR-MDS): Completed enrollment of the Phase 2a study of AG-946 in LR-MDS.
Business Development: Announced an exclusive worldwide license agreement with Alnylam Pharmaceuticals for a novel siRNA for the potential treatment of polycythemia vera.
Leadership: Appointed Catherine Owens to the board of directors. Kaye Foster assumed the role of lead independent director.
Other: Data from Servier’s Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma were presented during the plenary session at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and published in the New England Journal of Medicine. As part of the divestiture of Agios’ oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following additional key milestones and priorities by the end of 2023:
Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of mitapivat.
Sickle Cell Disease: Enroll first patient in Phase 3 portion of RISE UP study of mitapivat, with the 100 mg dose selected from the successful Phase 2 portion.
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announce data from the Phase 2a study of novel PK activator AG-946.
Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU).
Second Quarter 2023 Financial Results
Revenue: Net U.S. product revenue from sales of PYRUKYND for the second quarter of 2023 was $6.7 million, compared to $3.1 million for the second quarter of 2022. PYRUKYND received FDA approval on February 17, 2022.
Cost of Sales: Cost of sales for the second quarter of 2023 was $1.1 million.
Research and Development (R&D) Expenses: R&D expenses were $68.9 million for the second quarter of 2023 compared to $74.5 million for the second quarter of 2022. The year-over-year decrease was primarily driven by a decrease in workforce related expenses as a result of reduced headcount related to the evolution of our research organization.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $30.4 million for the second quarter of 2023 compared to $28.3 million for the second quarter of 2022. The year-over-year increase was primarily attributable to an increase in stock-based compensation expense.
Net Loss: Net loss was $83.8 million for the second quarter of 2023 compared to $91.8 million for the second quarter of 2022.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2023, were $946.9 million compared to $1.1 billion as of December 31, 2022. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue, interest income and vorasidenib milestone will enable the company to fund its operating expenses and capital expenditures at least into 2026. This does not include potential royalties from vorasidenib, commercializing mitapivat outside of the U.S. through one or more partnerships, or other potential strategic business or financial agreements.
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 2023 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.