Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat cancer and rare genetic diseases,
today reported business highlights and financial results for the
second quarter ended June 30, 2020.
“The second quarter was a productive and important time at
Agios, as we accomplished several key 2020 objectives across our
three focus areas of malignant hematology, solid tumors and rare
genetic diseases. In particular, we made significant progress on
our mitapivat clinical programs, including achieving
proof-of-concept in sickle cell disease and planning for our
pivotal development programs in both thalassemia and sickle cell
disease,” said Jackie Fouse, Ph.D., chief executive officer at
Agios. “For the remainder of 2020, we are focused on the completion
of our pivotal trials ACTIVATE and ACTIVATE-T for mitapivat in
pyruvate kinase deficiency and securing regulatory feedback on the
pivotal programs in both thalassemia and sickle cell disease to
enable their initiation next year, the submission of a supplemental
new drug application for TIBSOVO® in cholangiocarcinoma in the
first quarter of 2021, driving enrollment in our ongoing clinical
trials and continued strong commercial execution.”
SECOND QUARTER 2020 HIGHLIGHTS
Rare Genetic Diseases
- Established clinical proof-of-concept for mitapivat in sickle
cell disease based on a preliminary analysis of data on eight
patients from the Phase 1 study being conducted in collaboration
with the National Institutes of Health (NIH). Seven of eight (88%)
evaluable patients experienced a hemoglobin increase, with five of
eight patients (63%) achieving a hemoglobin increase of ≥1.0 g/dL
from baseline. Additionally, the data showed improvements in
associated markers of sickling as well as a safety profile
consistent with previously reported mitapivat data or expected in
the context of sickle cell disease.
- Presented data on 13 patients from the Phase 2 study of
mitapivat in non-transfusion-dependent α- and β-thalassemia at the
European Hematology Association (EHA) Annual Congress in June.
Treatment with mitapivat induced a hemoglobin increase of ≥1.0 g/dL
in 12 of 13 (92%) evaluable patients, including four of four (100%)
α-thalassemia patients. Additionally, the data showed improvements
in associated markers of hemolysis and erythropoiesis as well as a
safety profile consistent with previously reported mitapivat
data.
- Received Orphan Drug Designation from the Food and Drug
Administration (FDA) for mitapivat in thalassemia.
Hematologic Malignancies and Solid Tumors
- TIBSOVO® net sales of $27.6 million, an increase of 22%
from the first quarter of 2020; expanded total number of unique
prescribers by 15% from the first quarter of 2020.
- Published data from the Phase 3 ClarIDHy study of TIBSOVO® in
The Lancet Oncology. As a result of this publication, the National
Comprehensive Cancer Network (NCCN) guidelines were updated to
recommend treatment with TIBSOVO® for patients with advanced
IDH1-mutant cholangiocarcinoma.
- Presented updated data from the Phase 1 dose-escalation study
of vorasidenib in IDH-mutant non-enhancing glioma at the American
Society of Clinical Oncology (ASCO) Annual Meeting in May. In
the study, vorasidenib demonstrated prolonged disease control and
encouraging preliminary activity, as well as a favorable safety
profile consistent with previously reported data.
Corporate
- Completed a $255 million purchase agreement with Royalty Pharma
for IDHIFA® (enasidenib) royalty rights and outstanding regulatory
milestone payments.
KEY UPCOMING MILESTONES
Rare Genetic Diseases
- Report data from ACTIVATE and ACTIVATE-T, the company’s two
global pivotal trials for mitapivat in adults with pyruvate kinase
(PK) deficiency, between the end of 2020 and mid-2021.
- Finalize robust pivotal development plan for mitapivat in
thalassemia, including both α-and β-thalassemia, as well as
transfusion dependent and non-transfusion dependent patient
populations, by the end of 2020.
- Initiate first-in-human study in healthy volunteers for AG-946,
a next-generation PKR activator, in Q3 2020.
Hematologic Malignancies and Solid Tumors
- Deliver full-year 2020 U.S. revenue for TIBSOVO® of $105-115
million.
- Receive European Medicines Agency CHMP opinion for TIBSOVO® in
relapsed or refractory acute myeloid leukemia (AML) with an IDH1
mutation by the end of 2020.
- Report mature overall survival data from ClarIDHy Phase 3 study
in Q3 2020; if data are supportive, file supplemental new drug
application (sNDA) for TIBSOVO® in previously treated
IDH1-mutant cholangiocarcinoma in Q1 2021.
Research
- Achieve at least one new development candidate by year-end
2020.
SECOND QUARTER 2020 FINANCIAL RESULTS
Revenue: Total revenue for the second quarter
of 2020 was $37.3 million, which includes $27.6 million of net
product revenue from sales of TIBSOVO®, $6.4 million in
collaboration revenue and $3.3 million in royalty revenue from net
global sales of IDHIFA® under our collaboration agreement with
Celgene, now a wholly owned subsidiary of Bristol Myers Squibb.
This compares to revenue of $26.2 million for the second quarter of
2019. TIBSOVO® net product revenue increased 101% from the same
period last year.
Cost of Sales: Cost of sales were $0.7 million
for the second quarter of 2020 compared to $0.3 million for the
second quarter of 2019.
Research and Development (R&D) Expenses:
R&D expenses were $90.9 million for the second quarter of 2020
compared to $107.4 million for the second quarter of 2019. The
decrease in R&D expense was primarily attributable to milestone
payments related to AG-636 and an undisclosed early-stage research
program in the second quarter of 2019, winding down the ClarIDHy
Phase 3 study of TIBSOVO® and HOVON startup expenses incurred in
the second quarter of 2019, and lower spend across ongoing TIBSOVO®
clinical studies as a result of slowed enrollment and reduced
activities due to the COVID-19 pandemic.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $36.0 million for the
second quarter of 2020 compared to $32.4 million for the second
quarter of 2019. The increase in SG&A expense was primarily
attributable to the initial gated infrastructure build of the
company’s European operations offset by reduced travel and industry
engagement given restrictions in place to combat the COVID-19
pandemic.
Net Loss: Net loss was $90.5 million for the
second quarter of 2020 compared to $109.9 million for the second
quarter of 2019.
Cash Position and Guidance: Cash, cash
equivalents and marketable securities as of June 30, 2020 were $794
million, including the amount received under the Royalty Pharma
agreement, compared to $624 million as of June 30, 2019. The
company expects that its cash, cash equivalents and marketable
securities as of June 30, 2020, together with anticipated product
revenue, anticipated interest income and anticipated expense
reimbursements under our collaboration and license agreements, but
excluding any additional program-specific milestone payments, will
enable the company to fund its anticipated operating expenses and
capital expenditure requirements, including its pivotal development
programs for mitapivat in thalassemia and sickle cell disease,
through the end of 2022.
CONFERENCE CALL INFORMATIONAgios will host a
conference call and live webcast with slides today at 8:00 a.m. ET
to discuss its second quarter 2020 financial results and recent
business activities. To participate in the conference call, please
dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international)
and refer to conference ID 2955575. 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 the Agios/Celgene Collaboration In 2010,
Agios and Celgene Corporation, now a wholly owned subsidiary of
Bristol Myers Squibb, entered into a collaboration agreement
focused on cancer metabolism. Under the terms of the agreement,
Celgene has worldwide development and commercialization rights for
IDHIFA® (enasidenib). Celgene and Agios are currently
co-commercializing IDHIFA® in the U.S., and Agios
continues to conduct certain clinical development activities within
the IDHIFA® development program. Agios is eligible to receive
a $25 million payment upon achievement of a specified ex-U.S.
commercial milestone event, as well as reimbursement for costs
incurred for its co-commercialization efforts and development
activities.
About AgiosAgios is focused on discovering and
developing novel investigational medicines to treat malignant
hematology, solid tumors and rare genetic diseases through
scientific leadership in the field of cellular metabolism. In
addition to an active research and discovery pipeline across these
three therapeutic areas, Agios has two approved oncology precision
medicines and multiple first-in-class investigational therapies in
clinical and/or preclinical development. 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 its and its
collaborator’s preclinical, clinical and commercial advancement of
its drug development programs including TIBSOVO® (ivosidenib
tablets), IDHIFA® (enasidenib), mitapivat, vorasidenib, AG-270, and
AG-946; the potential benefits of Agios’ product candidates; its
key milestones and guidance for 2020; its plans regarding future
data presentations; its financial guidance regarding the period in
which it will have capital available to fund its operations; and
the potential benefits of its 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 or its collaborators 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 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; 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, 2020 |
|
December 31, 2019 |
Cash, cash equivalents, and marketable securities |
$ |
794,413 |
|
|
$ |
717,806 |
|
Accounts receivable, net |
|
12,023 |
|
|
|
8,952 |
|
Collaboration receivable –
related party |
|
2,537 |
|
|
|
1,539 |
|
Royalty receivable – related
party |
|
1,650 |
|
|
|
2,900 |
|
Inventory |
|
11,231 |
|
|
|
7,331 |
|
Total assets |
|
976,141 |
|
|
|
890,741 |
|
Deferred revenue – related
party |
|
— |
|
|
|
61,513 |
|
Stockholders' equity |
|
558,465 |
|
|
|
640,528 |
|
|
|
|
|
|
|
|
|
Consolidated Statements of Operations Data |
(in thousands, except share and per share
data) |
(Unaudited) |
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
2020 |
|
2019 |
|
2020 |
|
2019 |
Product revenue, net |
$ |
27,581 |
|
|
$ |
13,727 |
|
|
$ |
50,255 |
|
|
$ |
22,865 |
|
Collaboration revenue – related
party |
|
5,735 |
|
|
|
8,979 |
|
|
|
65,832 |
|
|
|
26,898 |
|
Collaboration revenue –
other |
692 |
|
|
812 |
|
|
1,685 |
|
|
|
1,782 |
|
Royalty revenue – related
party |
3,339 |
|
|
2,703 |
|
|
6,673 |
|
|
|
4,903 |
|
Total revenue |
37,347 |
|
|
26,221 |
|
|
124,445 |
|
|
|
56,448 |
|
Cost and expenses: |
|
|
|
|
|
|
|
Cost of sales |
675 |
|
|
303 |
|
|
1,208 |
|
|
|
637 |
|
Research and development |
90,917 |
|
|
107,389 |
|
|
182,173 |
|
|
|
202,974 |
|
Selling, general and administrative |
35,951 |
|
|
32,390 |
|
|
74,452 |
|
|
|
64,181 |
|
Total cost and expenses |
127,543 |
|
|
140,082 |
|
|
257,833 |
|
|
|
267,792 |
|
Loss from operations |
(90,196 |
) |
|
(113,861 |
) |
|
(133,388 |
) |
|
|
(211,344 |
) |
Interest income, net |
1,769 |
|
|
3,990 |
|
|
4,705 |
|
|
|
8,395 |
|
Non-cash interest expense for the
sale of future revenue |
(2,051 |
) |
|
— |
|
|
(2,051 |
) |
|
|
— |
|
Net loss |
$ |
(90,478 |
) |
|
$ |
(109,871 |
) |
|
$ |
(130,734 |
) |
|
$ |
(202,949 |
) |
Net loss per share – basic and
diluted |
$ |
(1.31 |
) |
|
$ |
(1.87 |
) |
|
$ |
(1.90 |
) |
|
$ |
(3.46 |
) |
Weighted-average number of common
shares used in computing net loss per share – basic and
diluted |
68,958,091 |
|
|
58,722,244 |
|
|
68,784,109 |
|
|
|
58,589,167 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Contacts
Investors:Holly Manning, 617-844-6630Director,
Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Associate
Director, Corporate CommunicationsJessica.Rennekamp@agios.com
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