Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism pioneering therapies for rare diseases,
today reported business highlights and financial results for the
fourth quarter and year ended December 31, 2023.
“The past 12 months have been remarkable for
Agios. We reported positive data across our industry-leading
pipeline of PK activators, including Phase 3 data in
non-transfusion-dependent thalassemia, Phase 2 data in sickle cell
disease and clinical proof-of-concept in lower-risk MDS, and
expanded our preclinical pipeline by in-licensing a novel siRNA
program from Alnylam,” said Brian Goff, chief executive officer at
Agios. “In 2024, we expect two additional Phase 3 readouts,
including the Phase 3 study of mitapivat in transfusion-dependent
thalassemia, and are actively preparing for a potential U.S. launch
in thalassemia in 2025. Together with our strong cash position,
Agios is poised for significant near- and long-term growth as we
progress toward of our vision of becoming a leading rare disease
company.”
Fourth Quarter 2023 & Recent
Highlights
- PYRUKYND® U.S. Launch: Generated $7.1 million in U.S. net
revenue for the fourth quarter of 2023, a 4 percent decrease from
the third quarter of 2023, primarily driven by lower customer
inventory levels at the end of the fourth quarter of 2023,
partially offset by favorable gross-to-net adjustments. A total of
178 unique patients have completed prescription enrollment forms,
representing an increase of 11 percent over the third quarter of
2023. A total of 109 patients are on PYRUKYND® therapy, a 9 percent
increase from the third quarter of 2023.
- Thalassemia: Announced positive topline data from the Phase 3
ENERGIZE study of mitapivat in non-transfusion-dependent
thalassemia. The study achieved its primary endpoint of hemoglobin
response and achieved both key secondary endpoints associated with
change from baseline in FACIT-Fatigue Score and hemoglobin
concentration.
- Sickle Cell Disease: Presented positive results from the Phase
2 portion of the RISE UP pivotal study of mitapivat at the 65th
American Society of Hematology (ASH) Annual Meeting and Exposition.
The study achieved its primary endpoint of hemoglobin response and
an improvement in annualized rates of sickle cell pain crises was
observed.
- Lower-risk Myelodysplastic Syndromes: Announced clinical
proof-of-concept data in the open-label Phase 2a study of AG-946
for the treatment of anemia in lower-risk myelodysplastic syndromes
(LR-MDS).
- Earlier-stage Pipeline: Filed an Investigational New Drug
Application (IND) for AG-181, Agios’ PAH stabilizer for the
treatment of phenylketonuria (PKU).
- Data presentations: Presented broad set of clinical and
translational data at the 65th ASH Annual Meeting & Exposition,
including positive data from the Phase 2 portion of the RISE UP
study of mitapivat in sickle cell disease, as noted above.
Anticipated 2024 Milestones
- Thalassemia: Following the announcement of positive topline
data from the Phase 3 ENERGIZE study of mitapivat in
non-transfusion-dependent thalassemia in January 2024, Agios plans
to report topline data from the Phase 3 ENERGIZE-T study of
mitapivat in transfusion-dependent thalassemia (mid-year) and file
for FDA approval of mitapivat in thalassemia (year-end)
- Sickle Cell Disease: Complete enrollment in the Phase 3 portion
of the RISE UP study of mitapivat (year-end)
- Pediatric PK Deficiency: Complete enrollment in the Phase 3
ACTIVATE-kids study of mitapivat (mid-year). Report topline data
from Phase 3 ACTIVATE kids-T study (year-end)
- Lower-risk Myelodysplastic Syndromes: Dose first patient in
Phase 2b study of AG-946 (mid-year)
- Earlier-stage Pipeline: Dose the first patient in the Phase 1
study of AG-181 for the treatment of PKU (early 2024)
Fourth Quarter and Full Year 2023
Financial Results
Revenue: Net U.S. product revenue from sales of
PYRUKYND® was $7.1 million for the fourth quarter of 2023 compared
to $4.3 million for the fourth quarter of 2022, and $26.8 million
for the year ended Dec. 31, 2023 compared to $11.7 million for the
year ended Dec. 31, 2022.
Cost of Sales: Cost of sales was $0.6 million for
the fourth quarter of 2023 and $2.9 million for the full year ended
Dec. 31, 2023.
Research and Development (R&D) Expenses:
R&D expenses were $77.5 million for the fourth quarter of 2023
compared to $70.3 million for the fourth quarter of 2022, and
$295.5 million for the year ended Dec. 31, 2023 compared to $279.9
million for the year ended Dec. 31, 2022. These changes reflect an
increase in development costs for mitapivat and the up-front
payment associated with the license agreement with Alnylam,
partially offset by a reduction in expenses associated with the
evolution of our research organization and the sale of our oncology
business to Servier.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $35.3 million for the fourth
quarter of 2023 compared to $32.8 million for the fourth quarter of
2022, and $119.9 million for the year ended Dec. 31, 2023 compared
to $121.7 million for the year ended Dec. 31, 2022.
Net Income (Loss): Net loss was $95.9 million for
the fourth quarter of 2023 compared to a net income of $36.5
million for the fourth quarter of 2022, and net loss was $352.1
million for the year ended Dec. 31, 2023 compared to $231.8 million
for the year ended Dec. 31, 2022. The increase in net loss is due
to the $127.9 million sale to Sagard in the fourth quarter of 2022
of our rights to future contingent payments associated with
royalties on U.S. net sales of TIBSOVO®.
Cash Position and Guidance: Cash, cash equivalents
and marketable securities as of Dec. 31, 2023, were $806.4 million
compared to $1.1 billion as of Dec. 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
cash inflows which could extend runway beyond 2026 including
potential royalties or monetization of 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 fourth quarter and full year 2023 financial
results and recent business highlights. 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.
About Agios 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 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 deep scientific
expertise in classical hematology and leadership in the field of
cellular metabolism and rare hematologic diseases, Agios is
advancing a robust clinical pipeline of investigational medicines
with programs in alpha- and beta-thalassemia, sickle cell disease,
pediatric PK deficiency, MDS-associated anemia and phenylketonuria
(PKU). In addition to its clinical pipeline, Agios is advancing a
preclinical TMPRSS6 siRNA as a potential treatment for polycythemia
vera. 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,
TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans,
strategies and expectations for its preclinical, clinical and
commercial advancement of its drug development, including
PYRUKYND®, AG-946 and AG-181, its PAH stabilizer; Agios’ strategic
vision and goals, including its key milestones for 2024; 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 U.S. FDA, the EMA or other
regulatory authorities, investigational review boards at clinical
trial sites and publication review bodies; Agios’ ability to obtain
and maintain requisite regulatory approvals and to enroll patients
in its planned clinical trials; unplanned cash requirements and
expenditures; competitive factors; Agios' ability to obtain,
maintain and enforce patent and other intellectual property
protection for any product candidates it is developing; Agios’
ability to establish and maintain key collaborations; uncertainty
regarding any milestone or royalty payments related to the sale of
its oncology business or its in-licensing of TMPRSS6 siRNA, and the
uncertainty of the timing of any such payments; uncertainty of the
results and effectiveness of the use of Agios’ cash and cash
equivalents; and general economic and market conditions. These and
other risks are described in greater detail under the caption "Risk
Factors" included in Agios’ public filings with the Securities and
Exchange Commission. Any forward-looking statements contained in
this press release speak only as of the date hereof, and Agios
expressly disclaims any obligation to update any forward-looking
statements, whether as a result of new information, future events
or otherwise, except as required by law.
|
Consolidated
Balance Sheet Data(in
thousands)(Unaudited) |
|
|
December 31,
2023 |
|
December 31,
2022 |
Cash, cash equivalents, and marketable securities |
$ |
806,363 |
|
$ |
1,096,993 |
Accounts
receivable, net |
2,810 |
|
2,206 |
Inventory |
19,076 |
|
8,492 |
Total
assets |
937,118 |
|
1,238,718 |
Stockholders' equity |
811,019 |
|
1,100,814 |
|
|
|
|
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Years Ended December 31, |
|
2023 |
|
2022 |
|
2021 |
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
Product revenue, net |
$ |
26,823 |
|
|
$ |
11,740 |
|
|
$ |
— |
|
Milestone revenue |
|
— |
|
|
|
2,500 |
|
|
|
— |
|
Total revenue |
|
26,823 |
|
|
|
14,240 |
|
|
|
— |
|
Operating expenses |
|
|
|
|
|
|
|
|
|
|
|
Cost of sales |
$ |
2,881 |
|
|
$ |
1,704 |
|
|
$ |
— |
|
Research and development |
|
295,526 |
|
|
|
279,910 |
|
|
|
256,973 |
|
Selling, general and administrative |
|
119,903 |
|
|
|
121,673 |
|
|
|
121,445 |
|
Total operating expenses |
|
418,310 |
|
|
|
403,287 |
|
|
|
378,418 |
|
Loss from operations |
|
(391,487 |
) |
|
|
(389,047 |
) |
|
|
(378,418 |
) |
Gain on sale of contingent payments |
|
— |
|
|
|
127,853 |
|
|
|
— |
|
Royalty income from gain on sale of oncology business |
|
— |
|
|
|
9,851 |
|
|
|
6,639 |
|
Interest income, net |
|
33,344 |
|
|
|
12793 |
|
|
|
836 |
|
Other income, net |
|
6,055 |
|
|
|
6,749 |
|
|
|
14,433 |
|
Net loss from continuing operations |
|
(352,088 |
) |
|
|
(231,801 |
) |
|
|
(356,510 |
) |
Net income from discontinued operations, net of tax |
|
— |
|
|
|
— |
|
|
|
1,961,225 |
|
Net (loss) income |
$ |
(352,088 |
) |
|
$ |
(231,801 |
) |
|
$ |
1,604,715 |
|
Net loss from continuing operations per share - basic and
diluted |
$ |
(6.33 |
) |
|
$ |
(4.23 |
) |
|
$ |
(5.90 |
) |
Net income from discontinued operations per share - basic and
diluted |
$ |
— |
|
|
$ |
— |
|
|
$ |
32.45 |
|
Net (loss) income per share - basic and diluted |
$ |
(6.33 |
) |
|
$ |
(4.23 |
) |
|
$ |
26.55 |
|
Weighted-average number of common shares used in computing net loss
per share from continuing operations, net income per share from
discontinued operations and net (loss) income per share – basic and
diluted |
|
55,651,487 |
|
|
|
54,789,435 |
|
|
|
60,447,346 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Contacts:
Investor Contact
Chris Taylor, VP Investor Relations and Corporate
Communications Agios Pharmaceuticals IR@agios.com
Media Contact
Dan Budwick 1AB dan@1abmedia.com
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