Agios Reports Business Highlights and Second Quarter 2023 Financial Results
- Announced Positive Results from Phase 2 Portion of the RISE UP Pivotal Study of Mitapivat in Sickle Cell Disease; On Track to Enroll First Patient in Phase 3 Portion of the Study in Q4 2023
- Completed Enrollment in Phase 3 ENERGIZE and ENERGIZE-T Studies of Mitapivat in Thalassemia and Phase 2a Study of AG-946 in Lower-Risk MDS
- Announced Exclusive Worldwide License Agreement with Alnylam Pharmaceuticals to License Alnylam’s Novel siRNA for the Potential Treatment of Polycythemia Vera
U.S.PYRUKYND® (mitapivat) Net Revenue of $6.7 Millionin Q2; $947 Millionof Cash, Cash Equivalents and Marketable Securitiesas of June 30, 2023
“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
Second Quarter 2023 & Recent Highlights
U.S.Launch: Generated $6.7 millionin 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 Owensto the board of directors. Kaye Fosterassumed 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) 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 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:
- 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
Cost of Sales: Cost of sales for the second 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 the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the
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, TMPRSS6 siRNA 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||$||946,923||$||1,096,993|
|Accounts receivable, net||2,251||2,206|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Product revenue, net||$||6,712||$||3,082||$||12,321||$||3,914|
|Cost of sales||$||1,108||$||435||$||1,662||$||774|
|Research and development||68,895||74,523||136,196||144,646|
|Selling, general and administrative||30,409||28,264||58,776||59,779|
|Total operating expenses||100,412||103,222||196,634||205,199|
|Loss from operations||(93,700||)||(97,640||)||(184,313||)||(198,785||)|
|Royalty income from gain on sale of oncology business||—||2,704||—||5,408|
|Interest income, net||8,254||1,793||16,345||2,487|
|Other income, net||1,640||1,337||3,144||4,310|
|Net loss per share - basic and diluted||$||(1.51||)||$||(1.68||)||$||(2.97||)||$||(3.41||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||55,604,330||54,799,680||55,435,796||54,678,249|
Source: Agios Pharmaceuticals, Inc.