Agios Reports Business Highlights and First Quarter 2023 Financial Results
– On Track for PYRUKYND® (mitapivat) Data Readouts of the Phase 2 Portion of the RISE UP Study in Sickle Cell Disease in Mid-2023 and the Phase 3 ENERGIZE and ENERGIZE-T Studies in Thalassemia in 2024
“In the first quarter of the year, Agios made significant progress executing across our industry-leading pipeline of PK activators, with clinical studies spanning thalassemia, sickle cell disease, lower-risk MDS and pediatric PK deficiency,” said
First Quarter 2023 & Recent Highlights
U.S.Launch: Generated $5.6 millionin U.S.net revenue for the first quarter of 2023, the fourth full quarter following FDA approval. A total of 127 unique patients have completed prescription enrollment forms, representing an increase of 21 percent over the fourth quarter of 2022. A total of 89 patients are on PYRUKYND® therapy, representing a 14 percent increase over the fourth quarter of 2022.
- Thalassemia: Closed screening of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively.
- Leadership: Appointed
Jeffrey Capelloto the board of directors. Paul Clancywill step down from the board of directors at the end of his term, effective June 13, 2023.
- Environmental, Social, and Governance (ESG): Published 2023 ESG Report, which provides corporate sustainability disclosures for the period
January 1, 2022to December 31, 2022.
- Other: Servier’s Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma met both its primary endpoint and key secondary endpoints. As part of the divestiture of Agios’ oncology business to
Servier, Agios retains rights to a potential $200 millionmilestone 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:
- Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively, by mid-year.
- Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKYND® and go/no-go to Phase 3 decision by mid-year.
- Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® by year-end.
- Lower-risk Myelodysplastic Syndromes (LR-MDS): Complete enrollment of the Phase 2a study of novel PK activator AG-946 by year-end.
- Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end.
First Quarter 2023 Financial Results
Cost of Sales: Cost of sales for the first quarter of 2023 was
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
Agios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's leadership in the field of cellular metabolism, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios has a PAH stabilizer in preclinical development as a potential treatment for phenylketonuria (PKU) and deep scientific expertise in classical hematology. 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 the potential benefits of PYRUKYND® (mitapivat), AG-946 and its PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, AG-946 and its PAH stabilizer; Agios’ strategic vision and goals, including its key milestones for 2023; and the potential benefits of Agios’ 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 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 or other public health emergencies 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|
|Accounts receivable, net||1,778||2,206|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
|Product revenue, net|
|Cost of sales|
|Research and development||67,301||70,123|
|Selling, general and administrative||28,367||31,515|
|Total operating expenses||96,222||101,977|
|Loss from operations||(90,613)||(101,145)|
|Royalty income from gain on sale of oncology business||—||2,704|
|Interest income, net||8,091||694|
|Other income, net||1,504||2,973|
|Net loss per share - basic and diluted|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||55,265,390||54,555,467|
Source: Agios Pharmaceuticals, Inc.