Agios Reports Fourth Quarter and Full Year 2021 Financial Results
– Received FDA Approval of PYRUKYND® (mitapivat) for the Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase (PK) Deficiency; First Approved Therapy for this Rare Blood Disorder –
– First Patients Dosed in All Three Pivotal Trials of PYRUKYND® in Thalassemia and Sickle Cell Disease –
“With last week’s FDA approval of PYRUKYND® for the treatment of hemolytic anemia in adults with PK deficiency, we have delivered the first therapy for a rare, debilitating, lifelong disease – and we have set the stage for Agios’ next chapter as a leader in the genetically defined disease space,” said
Fourth Quarter 2021 & Recent Highlights
- Received approval from the
U.S. Food and Drug Administration(FDA) for PYRUKYND®, the first therapy for the treatment of hemolytic anemia in adults with PK deficiency and Agios’ first genetically defined disease medicine.
- Dosed first patients in all three pivotal trials of PYRUKYND® in thalassemia and sickle cell disease.
- Hosted investor day on
Nov. 17to share updates on the company’s research and development pipeline, including progress on the PKM2, BCAT2 and PAH programs, and provide insights into the commercial launch strategy.
- Presented the following key data at the 63rd
American Society of Hematology(ASH) Annual Meeting & Exposition:
- Long-term efficacy data of PYRUKYND® in adults with PK deficiency who participated in the Phase 3 ACTIVATE and ACTIVATE-T trials
- Long-term efficacy and safety data of PYRUKYND® in adults with thalassemia who do not receive regular transfusions
- Efficacy, safety and translational data of PYRUKYND® in sickle cell disease from ongoing collaborator-led studies
- Phase 1 healthy volunteer study data of AG-946, the company’s novel PK activator
Anticipated 2022 Key Milestones & Priorities
Agios expects to execute on the following key milestones and priorities in 2022:
Pyruvate Kinase (PK) Deficiency
European Medicines Agency(EMA) regulatory decision for PYRUKYND® in adults with PK deficiency by year-end.
- Initiate Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® in not regularly transfused and regularly transfused pediatric patients with PK deficiency, respectively, in mid-2022.
- 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, by year-end.
Sickle Cell Disease
- Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in sickle cell disease by year-end.
- Initiate the sickle cell disease cohort of the Phase 1 study of novel PK activator AG-946 in the first half of 2022.
Expansion and Acceleration of PK Activation Portfolio
- Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk myelodysplastic syndrome (MDS) by year-end.
- Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
Fourth Quarter and Full Year 2021 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.
Research and Development (R&D) Expenses: R&D expenses for continuing operations were $73.3 million for the fourth quarter of 2021 compared to $59.4 million for the fourth quarter of 2020, and $257 million for the year ended Dec. 31, 2021 compared to $220.8 million for the year ended
Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $31.5 million for the fourth quarter of 2021 compared to
Non-Operating Income: Non-operating income included approximately
Net Loss: Net loss was $98.6 million for the fourth quarter of 2021 compared to a net loss of $84.5 million for the fourth quarter of 2020, and
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of Dec. 31, 2021, were $1.3 billion compared to $670.5 million as of Dec. 31, 2020. The company 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 fourth quarter and year end 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 9873227. 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.
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 genetically defined 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 Agios’ plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including PYRUKYND® (mitapivat) and AG-946; the potential benefits of Agios’ products and product candidates; Agios’ key milestones and guidance for 2022; its financial guidance regarding the period in which it will have capital available to fund its operations; 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. 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 failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with
|Consolidated Balance Sheet Data|
|Cash, cash equivalents and marketable securities||$||1,286,393||$||670,537|
|Assets held for discontinued operations||-||50,460|
|Liabilities held for discontinued operations||-||299,728|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
|(In thousands, except share and per share data)||2021||2020||2021||2020|
|Cost and expenses:|
|Research and development||$||73,299||$||59,423||$||256,973||$||220,811|
|Selling, general and administrative||31,528||25,909||121,445||115,105|
|Total cost and expenses||104,827||85,332||378,418||335,916|
|Loss from operations||(104,827||)||(85,332||)||(378,418||)||(335,916||)|
|Gain on sale of oncology business||2,643||-||6,639||-|
|Interest income, net||332||791||836||6,611|
|Other income, net||3,268||-||14,433||-|
|Net loss from continuing operations||(98,584||)||(84,541||)||(356,510||)||(329,305||)|
|Net income (loss) from discontinued operations, net of tax||3,957||(13,116||)||1,961,225||1,935|
|Net income (loss)||$||(94,627||)||$||(97,657||)||$||1,604,715||$||(327,370||)|
|Net loss from continuing operations per share - basic and diluted||$||(1.81||)||$||(1.22||)||$||(5.90||)||$||(4.77||)|
|Net income (loss) from discontinued operations per share - basic and diluted||$||0.07||$||0.19||$||32.45||$||0.03|
|Net income (loss) per share - basic and diluted||$||(1.74||)||$||(1.41||)||$||26.55||$||(4.74||)|
|Weighted-average number of common shares used in computing net loss per share from continuing operations, net income (loss) per share from discontinued operations and net income (loss) per share – basic and diluted||54,335,230||69,271,163||60,447,346||68,997,879|
Senior Director, Investor Relations
Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.