Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular
metabolism and PK activation pioneering therapies for rare
diseases, today reported business highlights and financial results
for the first quarter ended March 31, 2024.
“We were delighted to report positive data from the Phase 3
ENERGIZE study of mitapivat in non-transfusion-dependent
thalassemia and look forward to announcing topline data from the
Phase 3 ENERGIZE-T study in transfusion-dependent thalassemia in
the second quarter of this year,” said Brian Goff, chief executive
officer at Agios. “Mitapivat has the potential to become the first
therapy approved for all thalassemia subtypes, and our commercial
organization is actively preparing for a potential launch next
year. Beyond thalassemia, we look forward to the RISE UP Phase 3
readout in sickle cell disease next year, with potential for
approval in 2026, as we progress toward our vision of becoming a
leading rare disease company with a potential multi-billion-dollar
franchise in PK activation.”
First Quarter 2024 and Recent Highlights
- PYRUKYND® Revenues: Generated $8.2
million in net revenue for the first quarter of 2024, a 15 percent
sequential increase from the fourth quarter of 2023, primarily
driven by increased patient demand. A total of 188 unique patients
have completed prescription enrollment forms, representing an
increase of 6 percent over the fourth quarter of 2023. A total of
120 patients are on PYRUKYND® therapy, a 10 percent increase from
the fourth quarter of 2023.
- Thalassemia: Announced positive
results from the Phase 3 ENERGIZE study of mitapivat in adults with
non-transfusion-dependent alpha- or beta-thalassemia.
- Earlier-Stage Pipeline: Dosed the
first participants in the Phase 1 study of AG-181 for the treatment
of phenylketonuria (PKU).
- Environment, Social, and Governance
(ESG): Published 2024 ESG Report, which provides corporate
sustainability disclosures for the period Jan. 1, 2023 to Dec. 31,
2023.
- Other: Servier announced FDA filing
acceptance and priority review for a new drug application (NDA) for
vorasidenib for the treatment of IDH-mutant diffuse glioma. The FDA
has assigned a Prescription Drug User Fee Act (PDUFA) action date
of August 20, 2024. 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 2024:
- Thalassemia: Two key milestones for
the year, including reporting topline data from the Phase 3
ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia
(Q2) and filing 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
(now mid-year).
- Lower-risk Myelodysplastic
Syndromes: Dose first patient in Phase 2b study of AG-946
(mid-year).
- Other: Potential approval of
Servier’s vorasidenib for the treatment of IDH-mutant diffuse
glioma. The FDA has assigned a PDUFA action date of August 20,
2024. Agios retains certain economic rights, as described
above.
First Quarter 2024 Financial Results
Revenue: Net product revenue from sales of PYRUKYND® for the
first quarter of 2024 was $8.2 million, compared to $5.6 million
for the first quarter of 2023.
Cost of Sales: Cost of sales for the first quarter of 2024 was
$0.6 million.
Research and Development (R&D) Expenses: R&D expenses
were $68.6 million for the first quarter of 2024, compared to $67.3
million for the first quarter of 2023. The year-over-year
comparison reflects an increase in process development expenses,
offset by a decrease in workforce-related expenditures.
Selling, General and Administrative (SG&A) Expenses:
SG&A expenses were $31.0 million for the first quarter of 2024
compared to $28.4 million for the first quarter of 2023. The
year-over-year increase was primarily attributable to an increase
in commercial-related activities as we prepare for the potential
approval of PYRUKYND® in thalassemia.
Net Loss: Net loss was $81.5 million for the first quarter of
2024 compared to $81.0 million for the first quarter of 2023.
Cash Position and Guidance: Cash, cash equivalents and
marketable securities as of March 31, 2024, were $714.3 million
compared to $806.4 million as of December 31, 2023. 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 first quarter 2024 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) |
|
|
|
|
March 31, 2024 |
December 31, 2023 |
Cash, cash equivalents, and marketable securities |
$ |
714,292 |
|
|
$ |
806,363 |
|
Accounts receivable, net |
|
3,453 |
|
|
|
2,810 |
|
Inventory |
|
23,070 |
|
|
|
19,076 |
|
Total assets |
|
849,709 |
|
|
|
937,118 |
|
Stockholders' equity |
|
743,922 |
|
|
|
811,019 |
|
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
|
|
Three Months Ended March 31, |
|
|
2024 |
|
|
|
2023 |
|
Revenues: |
|
|
|
Product revenue, net |
$ |
8,189 |
|
|
$ |
5,609 |
|
Total revenue |
|
8,189 |
|
|
|
5,609 |
|
Operating expenses: |
|
|
|
Cost of sales |
$ |
627 |
|
|
$ |
554 |
|
Research and development |
|
68,620 |
|
|
|
67,301 |
|
Selling, general and administrative |
|
31,014 |
|
|
|
28,367 |
|
Total operating expenses |
|
100,261 |
|
|
|
96,222 |
|
Loss from operations |
|
(92,072 |
) |
|
|
(90,613 |
) |
Interest income, net |
|
8,889 |
|
|
|
8,091 |
|
Other income, net |
|
1,634 |
|
|
|
1,504 |
|
Net loss |
$ |
(81,549 |
) |
|
$ |
(81,018 |
) |
Net loss per share - basic and diluted |
$ |
(1.45 |
) |
|
$ |
(1.47 |
) |
Weighted-average number of
common shares used in computing net loss per share – basic and
diluted |
|
56,383,475 |
|
|
|
55,265,390 |
|
Contacts:
Investor ContactChris Taylor, VP Investor
Relations and Corporate CommunicationsAgios
PharmaceuticalsIR@agios.com
Media ContactDan Budwick1ABdan@1abmedia.com
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