- Ivosidenib (AG-120) NDA Submission for IDH1m
R/R AML Planned by Year End 2017; First Phase 1 Expansion Data
Expected in the Second Half of 2017 -
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
fourth quarter and year ended December 31, 2016. In addition, Agios
highlighted select corporate milestones and data presentations for
its preclinical and clinical development programs.
“Our 2016 accomplishments, including the enasidenib NDA
submission with our collaboration partner Celgene and clear
proof-of-concept data for our PK deficiency program, demonstrate
our ability to transform our scientific discoveries into important
precision medicines,” said David Schenkein, M.D., chief executive
officer at Agios. “In 2017, we are focused on making the transition
to a commercial stage company by delivering our lead cancer
programs to patients, bringing our first rare genetic disease
program into pivotal development and advancing our next research
program, focused on MTAP-deleted cancers, into the clinic.”
KEY UPCOMING MILESTONES
The company expects to achieve the following key milestones:
IDH Mutant Inhibitors in Hematologic Malignancies
- Potential approval of enasidenib in the United States for IDH2m
positive relapsed/refractory (R/R) acute myeloid leukemia (AML) in
collaboration with Celgene by the end of 2017.
- Submit a new drug application (NDA) to the U.S. FDA for
ivosidenib (AG-120) for IDH1m positive R/R AML by the end of
2017.
- Initiate a global, registration-enabling Phase 3 study (AGILE)
combining ivosidenib (AG-120) and VIDAZA® in newly diagnosed AML
patients with an IDH1 mutation ineligible for intensive
chemotherapy in the first half of 2017.
IDH Mutant Inhibitors in Solid Tumors
- Complete the dose-escalation phase of the ongoing Phase 1 study
of AG-881 in IDHm positive glioma in the first half of 2017.
Rare Genetic Diseases
- Finalize design for a global pivotal trial of AG-348 in
pyruvate kinase (PK) deficiency in the third quarter of 2017.
- Initiate a global pivotal trial of AG-348 in PK deficiency in
the first half of 2018.
Cancer Metabolism Research:
- Submit an Investigational New Drug (IND) application for the
development candidate targeting methylthioadenosine phosphorylase
(MTAP)-deleted tumors by the end of 2017. MTAP is a metabolic
enzyme that is deleted in approximately 15 percent of all
cancers.
ANTICIPATED 2017 DATA PRESENTATIONS
- First data from the expansion phase of the ongoing Phase 1
study of ivosidenib (AG-120) in R/R AML in the second half of
2017
- First data from the ongoing Phase 1b combination study of
enasidenib or ivosidenib (AG-120) with standard-of-care intensive
chemotherapy in newly diagnosed AML in the second half of 2017
- First data from the cholangiocarcinoma expansion cohort of the
ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m
positive solid tumors in the first half of 2017
- Updated data from the glioma expansion of the ongoing Phase 1
study of ivosidenib (AG-120) in advanced IDH1m positive solid
tumors in the second half of 2017
- Updated data from AG-348 Phase 2 DRIVE PK study in PK
deficiency in both the first and second half of 2017
- Updated preclinical data for the program targeting MTAP-deleted
tumors at the Keystone Tumor Metabolism Meeting taking place March
5-9, 2017 in Whistler, British Columbia
FOURTH QUARTER 2016 HIGHLIGHTS
- Supported Celgene’s submission of an NDA for enasidenib in
IDH2m positive R/R AML.
- Initiated a global, registration-enabling randomized Phase 3
study (ClarIDHy) for ivosidenib (AG-120) in IDH1m positive advanced
cholangiocarcinoma. The FDA also granted ivosidenib Fast Track
Designation for the treatment of patients with previously treated,
unresectable or metastatic cholangiocarcinoma with an IDH1
mutation.
- Completed the dose-escalation phase of the Phase 1 study of
AG-881 in IDHm positive hematologic malignancies. The study is now
closed for enrollment.
- Selected a development candidate targeting MTAP-deleted tumors
to enter IND-enabling studies.
FULL YEAR 2016 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as
of December 31, 2016 were $573.6 million, compared
to $375.9 million as of December 31, 2015. This
increase was driven by cash received under our collaboration
agreements with Celgene totaling $258.2 million, which includes a
$200 million upfront payment from the May 2016 collaboration
agreement, $25 million related to initiation of the enasidenib
Phase 3 IDHENTIFY study and $33.2 million of program funding, net
proceeds of $162.1 million received from the company’s September
2016 public offering, and $7.9 million from stock award activities.
These items were offset by a decrease in cash related to
expenditures to fund operating activities and purchases of fixed
assets of $230.6 million during the year ended December 31,
2016.
Collaboration revenue was $69.9 million for the year
ended December 31, 2016, compared to $59.1
million for the prior year.
Research and development (R&D) expenses were $220.2
million, including $25.4 million of stock-based
compensation expense, for the year ended December 31, 2016,
compared to $141.8 million, including $17.4
million in stock-based compensation expense, for the year
ended December 31, 2015. The increase in R&D expenses was
primarily due to increased costs to support advancement of the
company's lead investigational medicines toward later-stage
development. Celgene is responsible for all development
costs for enasidenib and certain development costs for AG-881 and
reimburses the company for development costs incurred for these
investigational medicines.
General and administrative (G&A) expenses were $50.7
million, including $16.7 million of stock-based
compensation expense, for the year ended December 31, 2016,
compared to $36.0 million, including $14.5
million of stock-based compensation expense, for the year
ended December 31, 2015. The increase in G&A expense was
largely due to increased headcount and other professional expenses
to support growing operations.
Net loss for the year ended December 31,
2016 was $198.5 million, compared to a net loss
of $117.7 million for the year ended December 31,
2015.
CASH GUIDANCE
Based on its current operating plans, the company expects that
its existing cash, cash equivalents and marketable securities as of
December 31, 2016, together with anticipated interest income, and
anticipated payments from Celgene under our collaboration
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 2018.
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides
today at 8:00 a.m. ET to discuss fourth quarter and full year 2016
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 61135222.
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 focused on discovering and
developing novel investigational medicines to treat cancer and rare
genetic diseases through scientific leadership in the field of
cellular metabolism. In addition to an active research and
discovery pipeline across both therapeutic areas, Agios has
multiple first-in-class investigational medicines 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 Enasidenib
and AG-881 are part of Agios' global strategic collaboration with
Celgene Corporation focused on cancer metabolism. Under the terms
of the 2010 collaboration agreement, Celgene has worldwide
development and commercialization rights for enasidenib. Agios
continues to conduct clinical development activities within the
enasidenib development program and is eligible to receive up to
$120 million in payments assuming achievement of certain milestones
and royalties on net sales. Agios and Celgene intend to
co-commercialize enasidenib in the U.S. For AG-881, the companies
have a joint worldwide development and 50/50 profit share
collaboration, and Agios is eligible to receive regulatory
milestone payments of up to $70 million. The program focused on
MTAP deleted cancers is part of a 2016 global co-development and
co-commercialization 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
Statement
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
Agios’ plans, strategies and expectations for its and its
collaborator’s preclinical, clinical and commercial advancement of
its drug development programs including enasidenib, ivosidenib
(AG-120), AG-881 and AG-348; the potential benefits of Agios'
product candidates; its key milestones for 2017; 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,” “believe,” “estimate,”
“expect,” “intend,” “may,” “plan,” “predict,” “project,” “would,”
“could,” “potential,” “possible,” “hope,” “strategy,” “milestone,”
“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 collaborator,
Celgene, 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 the U.S. FDA and 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, such as its agreements with
Celgene; and general economic and market conditions. These and
other risks are described in greater detail under the caption "Risk
Factors" included in Agios' Quarterly Report on Form 10-Q for the
quarter ended September 30, 2016, and other filings that Agios may
make with the Securities and Exchange Commission in the future. 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) |
|
|
|
|
|
|
|
|
|
December 31,
2016 |
|
December 31, 2015 |
|
|
|
|
|
|
|
|
Cash, cash
equivalents and marketable securities |
$ |
573,564 |
|
$ |
375,907 |
Collaboration receivable – related party |
|
4,886 |
|
|
8,225 |
Total assets |
|
|
|
619,094 |
|
|
420,065 |
Deferred
revenue – related party |
|
|
190,210 |
|
|
24,364 |
Stockholders’ equity |
|
358,591 |
|
|
345,118 |
|
|
|
|
|
|
|
|
|
|
|
|
Consolidated Statements of Operations
Data (in thousands, except share and per share
data)(Unaudited) |
|
|
|
|
Three Months Ended December 31, |
|
Years Ended December 31, |
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
Collaboration revenue –
related party |
$ |
22,648 |
|
|
$ |
6,218 |
|
|
$ |
69,892 |
|
|
$ |
59,119 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
64,678 |
|
|
|
36,933 |
|
|
|
220,163 |
|
|
|
141,827 |
|
General
and administrative |
|
15,379 |
|
|
|
10,182 |
|
|
|
50,714 |
|
|
|
35,992 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total operating
expenses |
|
80,057 |
|
|
|
47,115 |
|
|
|
270,877 |
|
|
|
177,819 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from
operations |
|
(57,409 |
) |
|
|
(40,897 |
) |
|
|
(200,985 |
) |
|
|
(118,700 |
) |
Interest income |
|
923 |
|
|
|
276 |
|
|
|
2,514 |
|
|
|
968 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
|
(56,486 |
) |
|
|
(40,621 |
) |
|
|
(198,471 |
) |
|
|
(117,732 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share–
basic and diluted |
$ |
(1.34 |
) |
|
$ |
(1.08 |
) |
|
$ |
(5.07 |
) |
|
$ |
(3.15 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average number
of common shares used in net loss per share applicable to common
stockholders – basic and diluted |
|
42,110,541 |
|
|
|
37,660,033 |
|
|
|
39,126,400 |
|
|
|
37,429,262 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Contacts
Investors:
Kendra Adams, 617-844-6407
Senior Director, Investor & Public Relations
Kendra.Adams@agios.com
Renee Leck, 617-649-8299
Senior Manager, Investor & Public Relations
Renee.Leck@agios.com
Media:
Holly Manning, 617-844-6630
Associate Director, Corporate Communications
Holly.Manning@agios.com
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