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
first quarter ended March 31, 2020. In addition, Agios provided an
update on its response to the global COVID-19 pandemic and the
expected impact on its business operations.
“Though there is no modern playbook for a crisis like the
COVID-19 pandemic, we moved quickly to reduce the risk of our
team’s and communities’ exposure to the virus and took action to
enable uninterrupted access to our commercial and clinical
medicines for the patients who are counting on us,” said Jackie
Fouse, Ph.D., chief executive officer at Agios.
“Despite the challenges we faced in the first quarter in the
wake of the COVID-19 pandemic, we made significant clinical and
commercial progress, including completing enrollment in the
ACTIVATE pivotal trial of mitapivat in PK deficiency, delivering
strong TIBSOVO® performance and achieving key U.S. and EU
regulatory milestones. We have also made important resource
allocation decisions with the goal of delivering on our key
business objectives while conserving cash and increasing our
financial flexibility,” continued Dr. Fouse. “Through these
efforts, we remain on track to achieve our Agios 2025 strategic
vision. As we have all worked to adapt to these unexpected and
trying circumstances, the teamwork and resilience demonstrated by
my colleagues has truly been remarkable and provides confidence
that we will come out of these challenging times even stronger than
we were before.”
FIRST QUARTER 2020 HIGHLIGHTS & RECENT
PROGRESS
- TIBSOVO® net sales increased by 16% and total number of unique
prescribers expanded by 25% from the fourth quarter of 2019; this
growth was driven largely by uptake in both the newly diagnosed and
relapsed and refractory acute myeloid leukemia (AML) segments.
- Enrollment was completed in ACTIVATE, the ongoing pivotal trial
of mitapivat in adults with pyruvate kinase (PK) deficiency who do
not regularly receive transfusions.
- In March, mitapivat was issued a positive opinion on its
application for orphan drug designation by the European Medicines
Agency (EMA) Committee for Orphan Medicinal Products for the
treatment of adults with PK deficiency.
- In March, Agios received clearance from the U.S. Food and Drug
Administration (FDA) for its investigational new drug application
for AG-946, a next generation PKR activator.
- Updated data from the Phase 1 study of vorasidenib in
non-enhancing low-grade glioma have been accepted for presentation
at the 2020 American Society of Clinical Oncology (ASCO)
Annual Meeting in May, which is being held virtually.
- Data from the Phase 2 study of mitapivat in thalassemia have
been accepted for presentation at the European Hematology
Association (EHA) Annual Congress in June, which will be held
virtually.
COVID-19 RESPONSE & IMPACT ON BUSINESS
OUTLOOK
TIBSOVO®
To date, patient and physician demand for TIBSOVO® (ivosidenib
tablets) – an oral therapy for AML – has not been negatively
impacted by the COVID-19 pandemic. AML is a serious disease that
progresses rapidly if untreated, and in most cases treatment delays
are not recommended. Professional oncology guidelines for the
treatment of cancer patients during the pandemic favor effective
oral options with well tolerated safety profiles.
Based on current inventory levels and a robust supply chain
strategy, Agios does not anticipate interruptions to supply of
TIBSOVO®, even if extended disruptions to manufacturing operations
were to occur as a result of the COVID-19 pandemic.
In addition, Agios has taken steps to ensure new and existing
TIBSOVO® patients can access the medication. TIBSOVO® can be
delivered directly to patients’ residences to ensure they can
receive their medication even if they are homebound for a prolonged
period of time. The myAgiosTM patient assistance program has
remained a constant resource for patients, including those who have
lost health insurance due to unemployment.
Based on these considerations, Agios continues to expect its
2020 TIBSOVO® net U.S. revenue to be between $105 and 115
million.
Clinical Programs
Because hospitals globally have shifted resources to treat
patients with COVID-19, Agios expects some delays in its clinical
programs based on anticipated challenges related to data
collection, access to trial sites and patient enrollment.
In February, Agios established a clinical trial task force to
ensure the safety of patients in its clinical trials and to support
patients on a case-by-case basis to enable their continued
participation in the studies. Where appropriate, Agios has
instituted home visits, telemedicine approaches, the use of local
laboratories and courier shipments of investigational
medicines.
The status of each Agios clinical program, along with the
expected impact of COVID-19, is outlined below. The ultimate extent
of the impact of the COVID-19 pandemic on clinical trial
enrollment, continuation and data collection will depend on future
developments, which are highly uncertain.
Rare Genetic Diseases
Clinical Trial |
Status & Program Updates |
ACTIVATE: Ongoing pivotal trial of mitapivat in
adults with PK deficiency who do not regularly receive
transfusions |
- Enrollment is complete with 80 patients.
- Topline data are now expected between the end of 2020 and
mid-2021 versus previous guidance of the end of 2020; the potential
delay is due to anticipated challenges with clinical trial site
access after the last patient has completed the study.
|
ACTIVATE-T: Ongoing pivotal trial of mitapivat in
adults with PK deficiency who regularly receive transfusions |
- Enrollment is complete with 27 patients.
- Topline data are now expected between the end of 2020 and
mid-2021 versus previous guidance of the end of 2020; the potential
delay is due to anticipated challenges with clinical trial site
access after the last patient has completed the study.
|
Thalassemia Phase 2: Ongoing proof-of-concept
trial of mitapivat in alpha- and beta-thalassemia |
- Enrollment is complete with 20 patients.
- Data on 13 patients will be presented at EHA in June.
|
Sickle Cell Disease Phase 2: Ongoing
proof-of-concept trial of mitapivat in sickle cell disease being
run under a Cooperative Research and Development
Agreement (CRADA) with the U.S. National Institutes of Health
(NIH) |
- New enrollment is paused as a result of the COVID-19
pandemic.
- The decision on proof-of-concept for mitapivat in sickle cell
disease remains on track for mid-2020.
- Data are expected to be submitted by NIH for presentation at
the American Society of Hematology (ASH) Annual Meeting in
December.
|
AG-946 Phase 1: First-in-human study of
next-generation PKR activator in healthy volunteers |
- Study initiation is expected in mid-2020.
|
Hematologic Malignancies
Clinical Trial |
Status & Program Updates |
TIBSOVO® Phase 1: Relapsed and Refractory AML EU
Filing |
- The EU regulatory process for TIBSOVO® in relapsed and
refractory AML remains on track with a CHMP opinion expected by the
end of 2020.
|
AGILE: Ongoing Phase 3 trial of TIBSOVO® in
combination with azacitidine in frontline AML |
- Enrollment completion is now expected in 2021 versus previous
guidance of the end of 2020; this delay is due to COVID-19 related
delays in site start-up activities and enrollment
interruptions.
|
HOVON150/AMLSG29: Ongoing intergroup-sponsored
Phase 3 trial of TIBSOVO® or IDHIFA® (enasidenib) in combination
with standard induction and consolidation chemotherapy in frontline
AML |
- Enrollment has slowed as a result of COVID-19 related delays in
site start-up activities and enrollment interruptions.
- Agios intends to provide an update on expected timing of
enrollment completion when site activation is complete and patient
enrollment has returned to expected levels.
|
Myelodysplastic Syndrome (MDS): Ongoing expansion
arm of the Phase 1 trial of TIBSOVO® in hematologic
malignancies |
- Enrollment completion is now expected in 2021 versus previous
guidance of the end of 2020; this delay is due to COVID-19 related
delays in site start-up activities and enrollment
interruptions.
|
AG-636 Phase 1: First-in-human dose-escalation
trial of DHODH inhibitor in lymphoma |
- As a result of limited enrollment in the study, Agios will stop
in-house development of AG-636 and evaluate partnering
options.
|
Solid Tumors
Clinical Trial |
Status & Program Updates |
ClarIDHy: Ongoing Phase 3 trial of TIBSOVO® in
previously treated cholangiocarcinoma |
- Agios still expects mature overall survival data from the study
in mid-2020, but anticipates delays in collecting the data from
trial sites and executing the data cleaning process.
- Agios now expects to file a supplemental new drug application
(sNDA) for TIBSOVO® in previously treated cholangiocarcinoma
between the end of 2020 and mid-2021 versus previous guidance of
the end of 2020.
|
INDIGO: Ongoing Phase 3 trial of vorasidenib in
low-grade glioma |
- Enrollment has slowed as a result of COVID-19 related delays in
site start-up activities and enrollment interruptions.
- Agios intends to provide an update on expected timing of
enrollment completion when site activation is complete and patient
enrollment has returned to expected levels.
|
AG-270 Phase 1: Ongoing dose-escalation arms
evaluating AG-270 in combination with taxanes in non-small cell
lung cancer and pancreatic cancer |
- Enrollment has slowed as a result of COVID-19 related delays at
trial sites.
- A go/no-go decision is still expected no later than 2022.
|
Research
- Agios remains on track to name a new development candidate by
the end of the year.
- Agios conducted a prioritization exercise and made the decision
to pause certain research programs, including its program in
Friedreich's Ataxia.
Cash Conservation Actions
In order to conserve cash while supporting the execution of
critical business objectives during this period of uncertainty,
Agios has made decisions to cease in-house development of AG-636;
delay select research programs, including the Friedreich’s Ataxia
program, and longer-term clinical studies; limit staff hiring and
significantly reduce contract workforce; and pause certain
infrastructure projects. Additional savings are anticipated across
the business as a result of reduced spending levels that will occur
naturally due to the COVID-19 pandemic, such as travel expenses and
clinical trial spend.
FIRST QUARTER 2020 FINANCIAL RESULTS
Revenue: Total revenue for the first quarter of
2020 was $87.1 million, which includes $22.7 million of net product
revenue from sales of TIBSOVO®, $61.1 million in collaboration
revenue and $3.3 million in royalty revenue from net global sales
of IDHIFA® under the collaboration agreement with Celgene. This
compares to revenue of $30.2 million for the first quarter of
2019.
The year-over-year increase in total revenue was primarily due
to a $42.2 million increase in collaboration revenue. As previously
announced, Celgene declined to extend the metabolic immuno-oncology
research collaboration with Agios. In addition, Celgene declined
its option to designate one undisclosed research program for
continued development and opt-in right. As a result, Agios
recognized the majority of the deferred revenue during the first
quarter of 2020. The remainder of the deferred revenue balance will
be recognized in the second quarter of 2020 through the expiration
of the research term in mid-May. The revenue increase was also
driven by an increase of $13.5 million of net sales of
TIBSOVO®.
Cost of Sales: Cost of sales were $0.5 million
for the first quarter of 2020 compared to $0.3 million for the
first quarter of 2019.
Research and Development (R&D) Expenses:
R&D expenses were $91.3 million for the first quarter of
2020 compared to $95.6 million for the first quarter of 2019. The
decrease in R&D expenses was primarily due to the recognition
of milestones for the initiation of the HOVON150/AMLSG29 study in
the first quarter of 2019 and decreased activity for the ClarIDHy
study and the Phase 1 hematologic malignancy study of TIBSOVO®.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $38.5 million for the
first quarter of 2020 compared to $31.8 million for the first
quarter of 2019. The increase in SG&A expenses were driven by
higher personnel costs, including stock-based compensation expense,
related to our workforce.
Net Loss: Net loss was $40.3 million for the
first quarter of 2020 compared to $93.1 million for the first
quarter of 2019.
Cash Position and Guidance: Cash, cash
equivalents and marketable securities as of March 31, 2020
were $613.1 million compared to $707.8 million as
of March 31, 2019.
As a result of the company’s cash conservation actions, the
company expects that its cash, cash equivalents and marketable
securities as of March 31, 2020, together with anticipated product
and royalty revenue, anticipated interest income, and anticipated
expense reimbursements under our collaboration and license
agreements, but excluding any additional collaboration-related
payments, will enable the company to fund its anticipated operating
expenses and capital expenditure requirements through June
2022.
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 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 referring to conference ID
1151878. 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 2025” Strategic VisionThe “Agios
2025” strategic vision delineates the company’s view for growth
with established and expanding franchises focused on treating
hematologic malignancies, solid tumors and rare genetic diseases.
As part of this vision, Agios expects to achieve the following
milestones by the end of 2025:
- 4 marketed medicines discovered and
developed at Agios
- Approvals in 8+ indications spanning
hematologic malignancies, solid tumors and rare genetic
diseases
- 6+ molecules in the clinic generated by
the company’s internal research discovery engine
- Cash-flow positive within the six-year
timeframe
About Agios/Celgene Collaboration IDHIFA®
(enasidenib) is part of our collaboration with Celgene Corporation,
a wholly owned subsidiary of Bristol-Myers Squibb Company. Under
the terms of our 2010 collaboration agreement focused on cancer
metabolism, Celgene has worldwide development and commercialization
rights for IDHIFA®. Agios continues to conduct certain clinical
development activities within the IDHIFA® development program and
is eligible to receive reimbursement for those development
activities and up to $80 million in remaining milestone payments,
and royalties on any net sales. Celgene and Agios are currently
co-commercializing IDHIFA® in the U.S. Celgene will reimburse Agios
for costs incurred for its co-commercialization efforts.
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.
Condensed Consolidated Balance Sheet
Data(in
thousands)(Unaudited)
|
March
31, |
|
December
31, |
|
2020 |
|
2019 |
Cash, cash
equivalents and marketable securities |
$ |
613,124 |
|
$ |
717,806 |
Accounts receivable, net |
|
11,813 |
|
|
8,952 |
Collaboration receivable – related party and other |
|
4,610 |
|
|
3,467 |
Royalty receivable – related party |
|
3,300 |
|
|
2,900 |
Inventory |
|
9,778 |
|
|
7,331 |
Total assets |
|
799,738 |
|
|
890,741 |
Deferred revenue – related party |
|
4,748 |
|
|
61,513 |
Stockholders' equity |
|
625,299 |
|
|
640,528 |
Condensed Consolidated Statements of
Operations Data(in thousands, except share and per
share data)(Unaudited)
|
Three Months
Ended March 31, |
|
|
2020 |
|
|
|
2019 |
|
Revenues: |
|
|
|
Product revenue, net |
$ |
22,674 |
|
|
$ |
9,138 |
|
Collaboration revenue – related party and other |
|
61,090 |
|
|
|
18,889 |
|
Royalty revenue – related party |
|
3,334 |
|
|
|
2,200 |
|
Total Revenue |
|
87,098 |
|
|
|
30,227 |
|
|
|
|
|
Cost and expenses: |
|
|
|
Cost of sales |
|
533 |
|
|
|
334 |
|
Research and development, net |
|
91,256 |
|
|
|
95,585 |
|
Selling, general and administrative |
|
38,501 |
|
|
|
31,791 |
|
Total cost and expenses |
|
130,290 |
|
|
|
127,710 |
|
Loss from operations |
|
(43,192 |
) |
|
|
(97,483 |
) |
Interest income |
|
2,936 |
|
|
|
4,405 |
|
Net loss |
$ |
(40,256 |
) |
|
$ |
(93,078 |
) |
Net loss per share – basic and diluted |
$ |
(0.59 |
) |
|
$ |
(1.59 |
) |
Weighted-average number of common shares used in computing net
loss per share – basic and diluted |
|
68,608,279 |
|
|
|
58,453,918 |
|
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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