– Commercial Organization Launch Ready Ahead of
June 21, 2019 PDUFA Action Date for Single Agent TIBSOVO® for IDH1m
Newly Diagnosed AML –
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, 2019.
“We continued to deliver significant progress across our
oncology and rare genetic disease programs during the first
quarter. Importantly, our U.S. commercial team is driving TIBSOVO®
toward solid performance for its first full year on the market, and
we are on track to achieve our internal forecast,” said Jackie
Fouse, Ph.D., chief executive officer at Agios. “We made progress
on expansion opportunities for TIBSOVO® across the frontline AML
setting. FDA accepted our sNDA in newly diagnosed AML, the
HOVON/AMLSG Phase 3 intensive chemotherapy combination study
initiated, and we received Breakthrough Therapy Designation for the
combination of TIBSOVO® and azacitadine. We also advanced our
mitapivat program. Our two pivotal trials in pyruvate kinase
deficiency remain on track to complete enrollment this year, we
have dosed the first patient in our Phase 2 thalassemia study, and
we now expect a NIH-sponsored study in sickle cell disease to
initiate this year.”
FIRST QUARTER 2019 HIGHLIGHTS & RECENT
PROGRESS
- Received FDA acceptance and Priority Review for the
supplemental new drug application (sNDA) for single agent
TIBSOVO® for the treatment of patients with newly diagnosed
acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1
(IDH1) mutation who are not eligible for standard therapy. The
Prescription Drug User Fee Act (PDUFA) action date was set for June
21, 2019.
- Presented updated data from the ongoing Phase 1 combination
trial of TIBSOVO® with azacitidine in patients with newly diagnosed
AML with an IDH1 mutation at the 17th International Symposium on
Acute Leukemias.
- Received Breakthrough Therapy designation from FDA for TIBSOVO®
in combination with azacitidine for the treatment of newly
diagnosed AML patients with an IDH1 mutation who are ≥75 years old
or who have comorbidities that preclude use of intensive induction
chemotherapy.
- Supported the cooperative groups HOVON and AMLSG on the
initiation of the Phase 3 randomized, placebo-controlled study of
TIBSOVO® or IDHIFA® in combination with induction therapy and
consolidation therapy followed by maintenance therapy in patients
with newly diagnosed AML with an IDH1 mutation.
- Presented preclinical data for AG-270, a first-in-class
methionine adenosyltransferase 2a (MAT2A) inhibitor, at
the American Association for Cancer
Research meeting.
- Dosed the first patient in a Phase 2 proof-of-concept study for
mitapivat in thalassemia.
- Announced two newly created commercial leadership roles to
support the commercialization of the company’s medicines in the
U.S. and Europe. Darrin Miles, who has been with the company since
2015, most recently as vice president, oncology program leadership,
was promoted to senior vice president, U.S. commercial and global
marketing. In addition, a search is underway for the role of senior
vice president, international.
KEY UPCOMING MILESTONES
The company plans to achieve the following key milestones in the
remainder of 2019:
Cancer:
- Potential U.S. approval and launch of single agent TIBSOVO® for
newly diagnosed AML with an IDH1 mutation not eligible for standard
therapy by June 21, 2019.
- Submit an sNDA to the FDA for TIBSOVO® for second line or later
IDH1 mutant cholangiocarcinoma by year-end.
- Initiate a registration-enabling Phase 3 study of vorasidenib
in low-grade glioma with an IDH1 mutation by year-end.
- Determine recommended dose of AG-270 in methylthioadenosine
phosphorylase (MTAP)-deleted tumors; initiate expansion arms,
including a single-agent arm in a variety of MTAP-deleted tumors
and two combination arms combining AG-270 and standard-of-care in
non-small cell lung cancer and pancreatic ductal adenocarcinoma in
the third quarter.
- Begin dosing patients in the Phase 1 dose-escalation trial of
AG-636, an inhibitor of the metabolic enzyme dihydroorotate
dehydrogenase (DHODH), in lymphoma in the first half of 2019.
Rare Genetic Diseases:
- Complete enrollment in two global pivotal trials for mitapivat
in adults with pyruvate kinase (PK) deficiency by year-end 2019:
- ACTIVATE-T: A single-arm trial of up to 40 regularly transfused
patients; enrollment was increased from 20 to 40 based on
demand
- ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80
patients who do not receive regular transfusions
- Achieve proof-of-concept for mitapivat in thalassemia in the
second half of 2019.
ANTICIPATED 2019 DATA PRESENTATIONS
- The following abstracts have been accepted for presentation at
2019 American Society of Clinical Oncology (ASCO) Annual Meeting:
- Updated data from a Phase 1 study of single agent TIBSOVO® in
IDH1 mutant newly diagnosed AML ineligible for standard
therapies.
- Updated data from the Phase 1 combination study of TIBSOVO® and
azacitidine in newly diagnosed AML with an IDH1 mutation.
- Results from the first cohort in a Phase 1 perioperative study
of TIBSOVO® and vorasidenib in recurrent IDH1 mutant low-grade
glioma.
- Topline data from the Phase 3 ClarIDHy study of TIBSOVO® in
IDH1 mutant second line or later cholangiocarcinoma to be reported
in the first half and full data to be presented in the second half
of 2019.
- Data from the dose-escalation portion of the ongoing Phase 1
study of AG-270 in patients with MTAP-deleted tumors expected in
the second half of 2019.
FIRST QUARTER 2019 FINANCIAL RESULTS
Revenue: Total revenue for the first quarter of
2019 was $30.2 million, which includes $17.9 million in
collaboration revenue and $2.2 million in royalty revenue from net
global sales of IDHIFA® under our collaboration agreement
with Celgene, and $9.1 million of net product revenue from
U.S. sales of TIBSOVO®. This compares to revenue of $8.8 million
for the first quarter of 2018, which included $7.3 million in
collaboration revenue and $1.4 million in royalty revenue from net
global sales of IDHIFA® under our collaboration agreement
with Celgene.
Cost of Sales: We began U.S. sales of TIBSOVO®
in the third quarter of 2018. Cost of sales were $0.3 million for
the first quarter of 2019.
Research and Development (R&D) Expenses:
R&D expenses were $95.6 million for the first quarter of
2019 compared to $78.2 million for the first quarter of 2018. The
increase in R&D expense was primarily attributable to clinical
trial activity related to TIBSOVO® frontline trials, the mitapivat
pivotal program in PK deficiency and Phase 2 study in thalassemia,
and start-up activities for AG-636.
Selling, General and Administrative (SG&A)
Expenses: SG&A expenses were $31.8 million for the
first quarter of 2019 compared to $24.6 million for the first
quarter of 2018. The increase in SG&A expense was primarily
attributable to costs to support commercialization of
TIBSOVO® and personnel costs related to increased
headcount.
Net Loss: Net loss was $93.1 million for the
first quarter of 2019 compared to $90.8 million for the first
quarter of 2018.
Cash Position and Guidance: Cash, cash
equivalents and marketable securities as of March 31, 2019
were $707.8 million compared to $805.4 million as
of December 31, 2018. The net decrease of $97.6 million in
cash position was primarily driven by net expenditures to fund
operations, including a onetime cash expense of $19.2 million for
bonus payouts during the first quarter. The company expects that
its cash, cash equivalents and marketable securities as of March
31, 2019, together with anticipated product and royalty 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 through at least the end of 2020.
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 2019 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
9573049. 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 focused on discovering and
developing novel investigational medicines to treat cancer and rare
genetic diseases through scientific leadership in the field of
cellular metabolism and adjacent areas of biology. In addition to
an active research and discovery pipeline across both therapeutic
areas, Agios has two approved oncology precision medicines and
multiple first-in-class investigational therapies in clinical
and/or preclinical development. All Agios programs focus on
genetically identified patient populations, leveraging our
knowledge of metabolism, biology and genomics. For more
information, please visit the company's website at
www.agios.com.
About Agios/Celgene Collaboration IDHIFA®
(enasidenib) and AG-270 are part of our collaboration with Celgene
Corporation. 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. AG-270 is part of a 2016 global
research collaboration agreement with Celgene focused on metabolic
immuno-oncology. Celgene has the option to participate in a
worldwide 50/50 cost and profit share with Agios, under which Agios
is eligible for up to $169 million in clinical and regulatory
milestone payments for the program.
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),
IDHIFA® (enasidenib), vorasidenib, mitapivat, AG-270 and AG-636;
the potential benefits of Agios' product candidates; its key
milestones for 2019; 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 benefit
of its strategic plans and focus. The words “anticipate,” “expect,”
“hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,”
“will,” 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: 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 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, such as its
agreements with Celgene and CStone Pharmaceuticals; 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, 2019 |
|
December 31, 2018 |
Cash, cash equivalents and marketable
securities |
$ |
707,791 |
|
$ |
805,421 |
Accounts receivable, net |
4,355 |
|
5,076 |
Collaboration receivable – related party |
2,319 |
|
2,462 |
Royalty receivable – related party |
2,200 |
|
2,234 |
Inventory |
2,328 |
|
869 |
Total assets |
822,131 |
|
858,457 |
Deferred revenue – related party |
77,128 |
|
92,519 |
Stockholders' equity |
620,257 |
|
687,537 |
|
Condensed
Consolidated Statements of Operations Data(in thousands, except
share and per share data)(Unaudited) |
|
Three Months
Ended March 31, |
|
2019 |
|
2018 |
Revenues: |
|
|
|
Product revenue, net |
$ |
9,138 |
|
$ |
- |
Collaboration revenue – related
party |
17,919 |
|
7,345 |
Collaboration revenue – other |
970 |
|
- |
Royalty revenue – related
party |
2,200 |
|
1,417 |
Total Revenue |
30,227 |
|
8,762 |
|
|
|
|
Cost and expenses: |
|
|
|
Cost of sales |
334 |
|
- |
Research and development, net |
95,585 |
|
78,224 |
Selling, general and
administrative |
31,791 |
|
24,550 |
Total cost and expenses |
127,710 |
|
102,774 |
Loss from operations |
(97,483) |
|
(94,012) |
Interest income |
4,405 |
|
3,187 |
Net loss |
$ |
(93,078) |
|
$ |
(90,825) |
Net loss per share – basic and diluted |
$ |
(1.59) |
|
$ |
(1.63) |
Weighted-average number of common shares used in
computing net loss per share – basic and diluted |
58,453,918 |
|
55,694,603 |
|
|
|
|
Investor & Media Contact:Holly Manning,
617-844-6630Associate Director, Investor
RelationsHolly.Manning@agios.com
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