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
third quarter ended September 30, 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 Brian Goff, chief
executive officer at Agios. “We are excited to report dosing of the
first patient in the Phase 3 portion of the pivotal RISE UP study
of our leading PK activator, mitapivat, in sickle cell disease and
look forward to sharing more detailed data from the positive Phase
2 portion of RISE UP at an upcoming medical meeting. We look
forward to future data readouts across our industry-leading
pipeline of PK activators, including the Phase 2a study of AG-946
in lower-risk MDS by the end of this year and both Phase 3 studies
of mitapivat in thalassemia next year.”
Third Quarter 2023 & Recent Highlights
- PYRUKYND® U.S. Launch: Generated
$7.4 million in 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. ET today.
Third Quarter 2023 Financial Results
Revenue: Net U.S. product revenue from sales of PYRUKYND® for
the third quarter of 2023 was $7.4 million, compared to $3.5
million for the third quarter of 2022.
Cost of Sales: Cost of sales for the third quarter of 2023 was
$0.6 million.
Research and Development (R&D) Expenses: R&D expenses
were $81.8 million for the third quarter of 2023, compared to $65.0
million for the third quarter of 2022. The year-over-year increase
was primarily driven by the $17.5 million upfront payment to
Alnylam for the TMPRSS6 asset.
Selling, General and Administrative (SG&A) Expenses:
SG&A expenses were $25.8 million for the third quarter of 2023
compared to $29.1 million for the third quarter of 2022. The
year-over-year decrease was primarily attributable to lower
stock-based compensation expense and reduced professional fees.
Net Loss: Net loss was $91.3 million for the third quarter of
2023 compared to $81.7 million for the third quarter of 2022.
Cash Position and Guidance: Cash, cash equivalents and
marketable securities as of September 30, 2023, were $872.4 million
compared to $1.1 billion as of December 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
potential 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 InformationAgios will host a
conference call and live webcast with slides today at 8:00 a.m. ET
to discuss third quarter 2023 financial results and recent business
activities. 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 AgiosAgios 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 and MDS-associated anemia. In addition to
its clinical pipeline, Agios is advancing a preclinical TMPRSS6
siRNA as a potential treatment for polycythemia vera, and a
preclinical PAH stabilizer as a potential treatment for
phenylketonuria (PKU). 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 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 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) |
|
|
|
|
|
|
September 30,2023 |
December 31,2022 |
Cash, cash equivalents, and marketable securities |
|
$ |
872,390 |
|
$ |
1,096,993 |
Accounts receivable, net |
|
|
1,176 |
|
|
2,206 |
Inventory |
|
|
17,274 |
|
|
8,492 |
Total assets |
|
|
1,007,258 |
|
|
1,238,718 |
Stockholders' equity |
|
|
886,843 |
|
|
1,100,814 |
|
|
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
|
|
|
|
Three Months EndedSeptember 30, |
|
Nine Months EndedSeptember 30, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Revenues: |
|
|
|
|
|
Product revenue, net |
$ |
7,399 |
|
|
$ |
3,516 |
|
|
$ |
19,720 |
|
|
$ |
7,430 |
|
Milestone revenue |
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
2,500 |
|
Total revenue |
|
7,399 |
|
|
|
3,516 |
|
|
|
19,720 |
|
|
|
9,930 |
|
Operating expenses: |
|
|
|
|
|
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 |
$ |
(91,324 |
) |
|
$ |
(81,747 |
) |
|
$ |
(256,148 |
) |
|
$ |
(268,327 |
) |
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 |
|
Contacts:
Investor Contact
Chris Taylor, VP Investor Relations and Corporate
CommunicationsAgios PharmaceuticalsIR@agios.com
Media Contact
Dan Budwick1AB Mediadan@1abmedia.com
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