Agios Reports Business Highlights and Third Quarter 2023 Financial Results
– First Patient Dosed in Phase 3 Portion of the RISE UP Pivotal Study of Mitapivat in Sickle Cell Disease –
– Completed Enrollment in Phase 3 ACTIVATE-KidsT Pediatric Study of Mitapivat in PK Deficiency; ACTIVATE-Kids Study Achieves >50% Enrollment –
– On Track for Data Readouts in Two Phase 3 Trials of Mitapivat in Thalassemia Next Year and Topline Data for AG-946 in LR-MDS by Year-end 2023 –
“Agios is approaching a catalyst-rich period, with three mid-to-late-stage data readouts expected by the end of next year, and a total of six by the end of 2025,” said
Third Quarter 2023 & Recent Highlights
U.S.Launch: Generated $7.4 millionin U.S.net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023. A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023. A total of 100 patients are on PYRUKYND® therapy.
- Sickle Cell Disease: Dosed first patient in the Phase 3 portion of the RISE UP pivotal study of mitapivat.
- Pediatric PK Deficiency: Completed enrollment in the Phase 3 ACTIVATE-kidsT study of mitapivat in regularly transfused pediatric patients with PK deficiency. Achieved goal of >50% enrollment in Phase 3 ACTIVATE-kids study.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following additional key milestones and priorities in the coming months:
- Sickle Cell Disease: Present data from the positive Phase 2 portion of the RISE UP study of mitapivat at an upcoming medical meeting. Advance patient enrollment in the Phase 3 portion of RISE UP.
- Lower-risk Myelodysplastic Syndromes (LR-MDS): Announce topline data from the Phase 2a study of novel PK activator AG-946 by year-end 2023.
- Thalassemia: Announce topline data from the two Phase 3 studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia in the first and second halves of 2024, respectively.
- Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat in non-regularly transfused pediatric PK deficiency next year.
- Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end 2023.
- Data Presentations: Present broad set of clinical and translational data at the 65th
American Society of Hematology(ASH) Annual Meeting & Exposition; abstracts will be available at 9 a.m. ETtoday.
Third Quarter 2023 Financial Results
Cost of Sales: Cost of sales for the third 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 Agios’ 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 pandemics 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||$||872,390||$||1,096,993|
|Accounts receivable, net||1,176||2,206|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
||Nine Months Ended
|Product revenue, net||$||7,399||$||3,516||$||19,720||$||7,430|
|Cost of sales||$||633||$||517||$||2,295||$||1,291|
|Research and development||81,841||64,966||218,037||209,612|
|Selling, general and administrative||25,822||29,123||84,598||88,902|
|Total operating expenses||108,296||94,606||304,930||299,805|
|Loss from operations||(100,897||)||(91,090||)||(285,210||)||(289,875||)|
|Royalty income from gain on sale of oncology business||—||4,443||—||9,851|
|Interest income, net||8,375||3,818||24,720||6,305|
|Other income, net||1,198||1,082||4,342||5,392|
|Net loss per share - basic and diluted||$||(1.64||)||$||(1.49||)||$||(4.61||)||$||(4.90||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||55,803,663||54,844,579||55,559,766||54,734,301|
Source: Agios Pharmaceuticals, Inc.