Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism pioneering therapies for rare and
genetically defined diseases, today reported business highlights
and financial results for the third quarter ended September 30,
2022.
“Agios is on the cusp of changing the treatment landscape for
people with rare and genetically defined diseases, with the
potential of PYRUKYND® to make a positive impact across multiple
underserved diseases. I joined the company in August because of its
differentiated portfolio, top-notch team and genuine dedication to
patients, and my excitement for the future of Agios has only grown
over the past few months,” said Brian Goff, chief executive officer
at Agios. “We are continuing to build our capabilities and
connections in rare and genetically defined diseases through our
U.S. launch of PYRUKYND®, and we expect our learnings to support
anticipated future expansion in related diseases where development
efforts are ongoing. We will build on the tremendous
accomplishments we’ve achieved in 2022 as we close out the year
with a focus on continuing to execute the launch, enrolling our
thalassemia and sickle cell disease pivotal trials and securing the
approval of PYRUKYND® for PK deficiency in the EU and Great
Britain. I am honored to lead this team as we together drive
long-term growth and value for patients, shareholders and all our
stakeholders.”
Third Quarter 2022 & Recent Highlights
- Continued to execute U.S. launch of PYRUKYND®, generating $3.5
million in U.S. net revenue for the third quarter of 2022, the
second full quarter following FDA approval. A total of 84 unique
patients have completed prescription enrollment forms, representing
an increase of 64 percent over the second quarter. A total of 56
patients are on PYRUKYND® therapy, representing a 51 percent
increase over the second quarter. Increased patient demand was
partially offset by modest inventory build in the prior quarters of
launch.
- Received positive opinion from the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency
(EMA), recommending the granting of a marketing authorization for
PYRUKYND® for the treatment of PK deficiency in adult
patients.
- Completed Medicines and Healthcare Products Regulatory Agency
(MHRA) filing for the approval of PYRUKYND® as a treatment for
adults with PK deficiency in Great Britain.
- Continued to enroll patients across five PYRUKYND® pivotal
studies in thalassemia, sickle cell disease and pediatric PK
deficiency.
- Initiated Phase 2a study of novel PK activator AG-946 in adults
with lower-risk MDS.
- Published PYRUKYND® data in top-tier medical journals,
including Phase 2 thalassemia data in The Lancet and Phase 3
ACTIVATE-T data in The Lancet Haematology.
- Appointed Cecilia Jones as Agios’ chief financial officer,
effective Sept. 26, 2022.
- Appointed Rahul Ballal, Ph.D., chief executive officer of
Imara, and Cynthia Smith, former chief commercial officer of ZS
Pharma, to Agios’ board of directors.
- Completed the sale of royalty rights on U.S. net sales of
Servier’s TIBSOVO® to Sagard Healthcare Partners for a one-time
payment of $131.8 million.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following key milestones and
priorities by the end of 2022:
- Adult PK Deficiency: Receive EU and Great
Britain regulatory decisions for PYRUKYND® in adults with PK
deficiency.
- Thalassemia: 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.
- Sickle Cell Disease: Complete enrollment in
the Phase 2 portion of the RISE UP study of PYRUKYND® in adults
with sickle cell disease.
- Data Presentations: Present broad set of
clinical and translational data at the 64th American Society of
Hematology (ASH) Annual Meeting & Exposition; abstracts will be
available at 9 a.m. ET today.
Third Quarter 2022 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.
Revenue: Net U.S. product revenue from sales of PYRUKYND® for
the third quarter of 2022 was $3.5 million. This revenue reflects
the second full quarter of PYRUKYND® launch, following FDA approval
on February 17, 2022.
Cost of Sales: Cost of sales for the third quarter of 2022 was
$0.5 million.
Non-Operating Income: Non-operating income included
approximately $4.4 million from TIBSOVO® royalties for the third
quarter of 2022. TIBSOVO® royalty income will cease in 2022 due to
the sale of these royalty rights to Sagard Healthcare Partners.
Research and Development (R&D)
Expenses: R&D expenses were $65.0
million for the third quarter of 2022 compared to $64.0
million for the third quarter of 2021.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $29.1
million for the third quarter of 2022 compared to $27.2
million for the third quarter of 2021. The year-over-year
increase in SG&A expenses was primarily attributable to an
increase in workforce-related expenses.
Net Loss from Continuing Operations: Net
loss from continuing operations was $81.7 million for the
third quarter of 2022 compared to a net loss of $84.3
million for the third quarter of 2021.
Cash Position and Guidance: Cash, cash
equivalents and marketable securities as of September 30,
2022, were $1.0 billion compared to $1.4 billion as
of September 30, 2021. This cash position does not include the
receipt of a one-time payment of $131.8 million associated with the
sale of royalty rights on U.S. net sales of Servier’s TIBSOVO®.
Agios 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 InformationAgios will host a
conference call and live webcast with slides today at 8:00
a.m. ET to discuss third quarter 2022 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 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 rare
and genetically defined 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, 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 multiple investigational therapies
in preclinical development and an industry-leading research team
with unmatched expertise in cellular metabolism and genetics. 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 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 impact of the COVID-19
pandemic on 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 future approved products, and launching, marketing and
selling 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, including with respect to the
regulatory submissions for PYRUKYND® (mitapivat), 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 and 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
collaborations; 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 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, or SEC,
including the risks and uncertainties set forth under the heading
Risk Factors in our filings with the SEC. While the list of factors
presented here is considered representative, this list should not
be considered to be a complete statement of all potential risks and
uncertainties. Any forward-looking statements contained in this
press release are made only as of the date hereof, and we undertake
no obligation to update forward-looking statements to reflect
developments or information obtained after the date hereof and
disclaim any obligation to do so other than as may be required by
law.
Consolidated Balance Sheet Data |
(in thousands) |
(Unaudited) |
|
September 30,2022 |
December 31,2021 |
Cash, cash equivalents, and marketable securities |
$ |
1,026,032 |
$ |
1,286,393 |
Accounts receivable, net |
|
1,818 |
|
— |
Inventory |
|
5,176 |
|
— |
Total assets |
|
1,180,320 |
|
1,437,736 |
Stockholders' equity |
|
1,050,170 |
|
1,291,975 |
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
|
|
|
|
|
|
Three Months Ended September30, |
|
Nine Months Ended September30, |
|
|
2022 |
|
|
2021 |
|
|
|
2022 |
|
|
2021 |
|
Revenues: |
|
|
|
|
|
Product revenue, net |
$ |
3,516 |
|
$ |
— |
|
|
$ |
7,430 |
|
$ |
— |
|
Milestone revenue |
|
— |
|
|
— |
|
|
|
2,500 |
|
|
— |
|
Total revenue |
|
3,516 |
|
|
— |
|
|
|
9,930 |
|
|
— |
|
Cost and expenses: |
|
|
|
|
|
Cost of sales |
$ |
517 |
|
$ |
— |
|
|
$ |
1,291 |
|
$ |
— |
|
Research and development |
|
64,966 |
|
|
64,000 |
|
|
|
209,612 |
|
|
183,674 |
|
Selling, general and administrative |
|
29,123 |
|
|
27,152 |
|
|
|
88,902 |
|
|
89,917 |
|
Total cost and expenses |
|
94,606 |
|
|
91,152 |
|
|
|
299,805 |
|
|
273,591 |
|
Loss from operations |
|
(91,090 |
) |
|
(91,152 |
) |
|
|
(289,875 |
) |
|
(273,591 |
) |
Royalty income from gain on sale of oncology business |
|
4,443 |
|
|
1,996 |
|
|
|
9,851 |
|
|
3,996 |
|
Interest income, net |
|
3,818 |
|
|
256 |
|
|
|
6,305 |
|
|
504 |
|
Other income, net |
|
1,082 |
|
|
4,641 |
|
|
|
5,392 |
|
|
11,165 |
|
Net loss from continuing operations |
|
(81,747 |
) |
|
(84,259 |
) |
|
|
(268,327 |
) |
|
(257,926 |
) |
Net (loss) income from discontinued operations, net of tax |
|
— |
|
|
(4,507 |
) |
|
|
— |
|
|
1,957,268 |
|
Net (loss) income |
$ |
(81,747 |
) |
$ |
(88,766 |
) |
|
$ |
(268,327 |
) |
$ |
1,699,342 |
|
Net loss from continuing operations per share - basic and
diluted |
$ |
(1.49 |
) |
$ |
(1.48 |
) |
|
$ |
(4.90 |
) |
$ |
(4.13 |
) |
Net (loss) income from
discontinued operations per share - basic and diluted |
$ |
— |
|
$ |
(0.08 |
) |
|
$ |
— |
|
$ |
31.31 |
|
Net
(loss) income per share - basic and diluted |
$ |
(1.49 |
) |
$ |
(1.56 |
) |
|
$ |
(4.90 |
) |
$ |
27.19 |
|
Weighted-average number of common shares used in computing net loss
per share from continuing operations, net (loss) income from
discontinued operations and net (loss) income per share – basic and
diluted |
|
54,844,579 |
|
|
57,048,175 |
|
|
|
54,734,301 |
|
|
62,503,087 |
|
Contacts
Investors:Holly Manning, 617-844-6630Senior
Director, Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Senior
Director, Corporate CommunicationsJessica.Rennekamp@agios.com
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