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
second quarter ended 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 Brian
Goff, chief executive officer at Agios. “We announced positive data
from the Phase 2 portion of the RISE UP study of mitapivat in
sickle cell disease, completed enrollment in three clinical
studies, licensed a compelling preclinical program from Alnylam,
and continued to strengthen our commercial capabilities to support
future anticipated launches. We look forward to the readout of the
Phase 2a study of AG-946 in lower-risk MDS by the end of this year
and the readouts of the Phase 3 studies of mitapivat in thalassemia
next year.”
Second Quarter 2023 & Recent Highlights
- PYRUKYND® U.S. Launch: Generated
$6.7 million in 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
Owens to the board of directors. Kaye Foster assumed 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 million milestone 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
Revenue: Net U.S. product revenue from sales of PYRUKYND® for
the second quarter of 2023 was $6.7 million, compared to $3.1
million for the second quarter of 2022. PYRUKYND® received FDA
approval on February 17, 2022.
Cost of Sales: Cost of sales for the second quarter of 2023 was
$1.1 million.
Research and Development (R&D) Expenses: R&D expenses
were $68.9 million for the second quarter of 2023 compared to $74.5
million for the second quarter of 2022. The year-over-year decrease
was primarily driven by a decrease in workforce related expenses as
a result of reduced headcount related to the evolution of our
research organization.
Selling, General and Administrative (SG&A) Expenses:
SG&A expenses were $30.4 million for the second quarter of 2023
compared to $28.3 million for the second quarter of 2022. The
year-over-year increase was primarily attributable to an increase
in stock-based compensation expense.
Net Loss: Net loss was $83.8 million for the second quarter of
2023 compared to $91.8 million for the second quarter of 2022.
Cash Position and Guidance: Cash, cash equivalents and
marketable securities as of June 30, 2023, were $946.9 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 second 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 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 has TMPRSS6 siRNA as a potential
treatment for polycythemia vera, a PAH stabilizer in preclinical
development as a potential treatment for phenylketonuria (PKU), and
deep scientific expertise in classical hematology. For more
information, please visit the company’s website at
www.agios.com.
Cautionary Note Regarding Forward-Looking
StatementsThis 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 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 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 proceeds
from the transaction with Servier; 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) |
|
|
|
|
|
|
|
|
|
|
June 30, 2023 |
December 31, 2022 |
Cash, cash equivalents, and marketable securities |
|
|
|
$ |
946,923 |
$ |
1,096,993 |
Accounts receivable, net |
|
|
|
|
2,251 |
|
2,206 |
Inventory |
|
|
|
|
15,671 |
|
8,492 |
Total assets |
|
|
|
|
1,085,153 |
|
1,238,718 |
Stockholders' equity |
|
|
|
|
964,236 |
|
1,100,814 |
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
2023 |
|
|
2022 |
|
|
|
2023 |
|
|
2022 |
|
Revenues: |
|
|
|
|
|
Product
revenue, net |
$ |
6,712 |
|
$ |
3,082 |
|
|
$ |
12,321 |
|
$ |
3,914 |
|
Milestone revenue |
|
— |
|
|
2,500 |
|
|
|
— |
|
|
2,500 |
|
Total revenue |
|
6,712 |
|
|
5,582 |
|
|
|
12,321 |
|
|
6,414 |
|
Operating expenses: |
|
|
|
|
|
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 |
$ |
(83,806 |
) |
$ |
(91,806 |
) |
|
$ |
(164,824 |
) |
$ |
(186,580 |
) |
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 |
|
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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