– BLA filing for axatilimab in chronic
GVHD granted Priority Review; PDUFA action date set for
August 28, 2024 –
– NDA for revumenib in R/R KMT2Ar acute
leukemia submitted under FDA's RTOR program; PDUFA date assignment
expected in 1Q24 –
– Enrollment in AUGMENT-101 mNPM1 patient
cohort expected to complete in late 1Q/early 2Q; topline data in
4Q24 –
– Initiated revumenib Phase 1
combination trial with 7+3 chemotherapy in newly diagnosed acute
leukemias –
– Axatilimab Phase 2 IPF trial underway
–
– $600 million
in cash and cash equivalents expected to provide runway through
2026 –
– Company to host conference call today at
4:30 p.m. ET –
WALTHAM,
Mass., Feb. 27, 2024 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX), a clinical-stage biopharmaceutical
company developing an innovative pipeline of cancer therapies,
today reported its financial results for the fourth quarter and
full year ended December 31, 2023,
and provided a clinical and business update.
"2023 was a landmark year for Syndax marked by execution across
all of our major clinical, regulatory, and operational milestones,"
said Michael A. Metzger, Chief
Executive Officer. "With the axatilimab BLA filing under FDA
Priority Review and receipt of a PDUFA action date for revumenib
anticipated later this quarter, preparations are well underway for
the potential launch of both first- and best-in-class drugs later
this year. We are committed to maximizing the full potential
of these therapies beyond their initial approvals through expansion
into earlier lines of therapy in combination with standard of care
agents and into new indications. With a strong cash position that
is expected to fund operations through 2026, Syndax is well
positioned for continued success and long-term growth."
Recent Pipeline Progress and Anticipated Milestones
Revumenib
- On December 29, 2023, the Company
submitted a New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) for revumenib, a potent, selective small
molecule menin inhibitor, for the treatment of adult and pediatric
relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute
leukemia. The NDA submission is being reviewed under the FDA's
Real-Time Oncology Review (RTOR) program, and the Company expects
to receive a Prescription Drug User Fee Act (PDUFA) action date
from the FDA this quarter.
- The Company expects to complete enrollment in the AUGMENT-101
pivotal trial cohort of patients with R/R mutant nucleophosmin
(mNPM1) acute myeloid leukemia (AML) later this quarter or early in
the second quarter of 2024. Topline data is expected in the fourth
quarter of 2024 and could support a supplemental NDA (sNDA) filing
for revumenib in R/R mNPM1 AML in the first half of 2025.
- Positive results from the Phase 1 and Phase 2 portions of the
Company's AUGMENT-101 trial were featured throughout multiple
sessions at the 65th American Society of Hematology
(ASH) Annual Meeting in December
2023, including results from the pivotal portion of the
trial, which were highlighted as a late-breaking oral
presentation.
- At the ASH Annual Meeting and during the Company's accompanying
investor event, investigators presented compelling data from
multiple Phase 1 combination trials of revumenib in mNPM1 and
KMT2Ar acute leukemia across the treatment landscape. The trials
are expanding to validate recommended Phase 2 doses, with
additional data expected in the second half of 2024. These trials
include:
- BEAT AML: Evaluating the combination of revumenib with
venetoclax and azacitidine in front-line AML patients. This trial
is being conducted as part of the Leukemia & Lymphoma Society's
Beat AML® Master Clinical Trial.
- SAVE: Evaluating the all-oral combination of revumenib with
venetoclax and decitabine/cedazuridine in R/R AML or mixed
phenotype acute leukemias. The trial is being conducted by
investigators from the MD Anderson Cancer Center.
- AUGMENT-102: Evaluating the combination of revumenib with
fludarabine and cytarabine in patients with R/R acute
leukemias.
- In February 2024, the Company
initiated a Phase 1 trial of revumenib in combination with 7+3
chemotherapy followed by maintenance treatment in newly diagnosed
patients with mNPM1 or KMT2Ar acute leukemias.
- The Company plans to initiate a pivotal trial of revumenib in
combination with venetoclax and azacitidine in newly diagnosed
mNPM1 or KMT2Ar acute leukemia patients unable to receive intensive
chemotherapy by year-end 2024.
- Enrollment is ongoing in a Phase 1 proof-of-concept clinical
trial of revumenib in patients with unresectable metastatic
microsatellite stable colorectal cancer. The Company expects to
provide an update on the trial in the second quarter of 2024.
Axatilimab
- The FDA has accepted the Biologics License Application (BLA)
filing for axatilimab, an anti-CSF-1R antibody, in patients with
chronic graft-versus-host disease (GVHD) after failure of at least
two prior lines of systemic therapy. The application has been
granted Priority Review and assigned a PDUFA action date of
August 28, 2024.
- Results from the pivotal AGAVE-201 trial were featured in the
Plenary Scientific Session at the 65th ASH Annual
Meeting in December 2023.
- The Company exercised its option under its collaboration
agreement with Incyte to co-commercialize axatilimab in the U.S.
and will provide 30% of the commercial effort.
- Enrollment is ongoing in a randomized, double-blinded,
placebo-controlled Phase 2 trial of axatilimab in patients with
idiopathic pulmonary fibrosis (IPF).
- Incyte plans to initiate two combination trials with axatilimab
in chronic GVHD in mid-2024, including a Phase 2 combination trial
with ruxolitinib and a Phase 3 combination trial with
steroids.
Corporate Update
In December of 2023, Syndax closed an underwritten public
offering of 12,432,431 shares of its common stock at $18.50 per share. This total included the
exercise in full by the underwriters of their option to purchase up
to 1,621,621 additional shares of common stock. Additionally, the
Company issued and sold 2,719,744 shares pursuant to its ATM
facility. Aggregate net proceeds from these offerings were
approximately $258.1 million after
deducting underwriting discounts and sales agent commissions and
estimated offering expenses payable by Syndax.
Fourth Quarter and Full Year 2023 Financial Results
As of December 31, 2023, Syndax had cash, cash equivalents,
and short and long-term investments of $600.5 million and 85.1 million common shares and
prefunded warrants outstanding.
Fourth quarter 2023 research and development expenses increased
to $55.1 million from $31.8 million, and for the full year increased to
$163.0 million compared to
$118.5 million for 2022. The
year-over-year increase in research and development expenses was
primarily due to increased employee-related expenses and
professional fees as well as increased clinical and manufacturing
expenses.
Fourth quarter 2023 selling, general and administrative expenses
increased to $22.8 million from $10.2 million, and for the full year increased to
$66.9 million compared to
$33.3 million for 2022. The
year-over-year increase in selling, general and administrative
expenses was primarily due to increased employee-related expenses
and professional fees as well as increased commercialization
activities for revumenib and axatilimab.
For the three months ended December 31, 2023, Syndax
reported a net loss attributable to common stockholders of
$72.5 million, or $1.00 per share, compared to a net loss
attributable to common stockholders of $39.2
million, or $0.62 per share,
for the comparable prior year period. For the year ended
December 31, 2023, Syndax reported a net loss attributable to
common stockholders of $209.4 million
or $2.98 per share, compared to a net
loss attributable to common stockholders of $149.3 million or $2.46 per share for the comparable prior year
period.
Financial Guidance
For the first quarter of 2024, the Company expects research and
development expenses to be $56 to
$62 million and total operating
expenses to be $82 to $88 million. For the full year of 2024, the
Company expects research and development expenses to be
$240 to $260
million and total operating expenses to be $355 to $375
million, which includes an estimated $43 million in non-cash stock compensation
expense.
The Company believes that it has sufficient cash runway to fund
its research, clinical development and commercial operations
through 2026.
Conference Call and Webcast
In connection with the earnings release, Syndax's management
team will host a conference call and live audio webcast at
4:30 p.m. ET today, Tuesday, February 27, 2024.
The live audio webcast and accompanying slides may be accessed
through the Events & Presentations page in the Investors
section of the Company's website. Alternatively, the conference
call may be accessed through the following:
Conference ID: Syndax4Q23
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast:
https://www.veracast.com/webcasts/syndax/events/SNDX4Q23.cfm
For those unable to participate in the conference call or
webcast, a replay will be available on the Investors section of the
Company's website at www.syndax.com approximately 24 hours after
the conference call and will be available for 90 days following the
call.
About Revumenib
Revumenib is a potent, selective, small molecule inhibitor of
the menin-KMT2A binding interaction that is being developed for the
treatment of KMT2A-rearranged, also known as mixed lineage leukemia
rearranged or MLLr, acute leukemias including ALL and AML, and
NPM1-mutant AML. Positive topline results from the Phase 2
AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial
met its primary endpoint were recently presented at the
65th American Society of Hematology Annual Meeting and
data from the Phase 1 portion of AUGMENT-101 in acute leukemia
was published in Nature. Revumenib was granted
Orphan Drug Designation by the FDA and European
Commission for the treatment of patients with AML, and Fast
Track designation by the FDA for the treatment of adult and
pediatric patients with R/R acute leukemias harboring a KMT2A
rearrangement or NPM1 mutation. Revumenib was granted BTD by the
FDA for the treatment of adult and pediatric patients with R/R
acute leukemia harboring a KMT2A rearrangement.
About Axatilimab
Axatilimab is an investigational monoclonal antibody that
targets colony stimulating factor-1 receptor, or CSF-1R, a cell
surface protein thought to control the survival and function of
monocytes and macrophages. In pre-clinical models, inhibition of
signaling through the CSF-1 receptor has been shown to reduce the
number of disease-mediating macrophages along with their monocyte
precursors, which has been shown to play a key role in the fibrotic
disease process underlying diseases such as chronic
graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis
(IPF). Positive topline results from the Phase 2 AGAVE-201 trial
showing the trial met its primary endpoint were
recently presented at the 65th American Society of
Hematology Annual Meeting and Phase 1/2 data of axatilimab in
chronic GVHD were published in the Journal of
Clinical Oncology. Axatilimab was granted Orphan Drug
Designation by the U.S. Food and Drug Administration for
the treatment of patients with chronic GVHD and IPF.
In September 2021, Syndax and Incyte entered into an
exclusive worldwide co-development and co-commercialization license
agreement for axatilimab. Syndax has exercised its option under the
collaboration agreement to co-commercialize axatilimab in the U.S.
and will provide 30% of the commercial effort. Axatilimab is being
developed under an exclusive worldwide license from UCB entered
into between Syndax and UCB in 2016.
About RTOR
RTOR provides a more efficient review process for oncology drugs
to ensure that safe and effective treatments are available to
patients as early as possible, while improving review quality and
engaging in early iterative communication with the applicant.
Specifically, it allows for close engagement between the sponsor
and the FDA throughout the submission process and it enables the
FDA to review individual sections of modules of a drug application
rather than requiring the submission of complete modules or a
complete application prior to initiating review. Additional
information about RTOR can be found
at: https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program
About Syndax
Syndax Pharmaceuticals is a clinical stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. Highlights of the Company's pipeline include
revumenib, a highly selective inhibitor of the menin–KMT2A binding
interaction, and axatilimab, a monoclonal antibody that blocks the
colony stimulating factor 1 (CSF-1) receptor. For more information,
please visit www.syndax.com or follow the Company
on Twitter and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Words such as "anticipate," "believe," "could," "estimate,"
"expects," "intend," "may," "plan," "potential," "predict,"
"project," "should," "will," "would" or the negative or plural of
those terms, and similar expressions (as well as other words or
expressions referencing future events, conditions or circumstances)
are intended to identify forward-looking statements. These
forward-looking statements are based on Syndax's expectations and
assumptions as of the date of this press release. Each of these
forward-looking statements involves risks and uncertainties. Actual
results may differ materially from these forward-looking
statements. Forward-looking statements contained in this press
release include, but are not limited to, statements about the
progress, timing, clinical development and scope of clinical
trials, the reporting of clinical data for Syndax's product
candidates, the sufficiency of the Company's cash runway to fund
its research, clinical development and commercial operations
through 2026, the potential use of its product candidates to treat
various cancer indications and fibrotic diseases, and Syndax's
expected first quarter and full year research and development
expenses, and expected first quarter and full year total operating
expenses. Many factors may cause differences between current
expectations and actual results, including: unexpected safety or
efficacy data observed during preclinical or clinical trials;
clinical trial site activation or enrollment rates that are lower
than expected; changes in expected or existing competition; changes
in the regulatory environment; failure of Syndax's collaborators to
support or advance collaborations or product candidates; and
unexpected litigation or other disputes. Other factors that may
cause Syndax's actual results to differ from those expressed or
implied in the forward-looking statements in this press release are
discussed in Syndax's filings with the U.S. Securities and
Exchange Commission, including the "Risk Factors" sections
contained therein. Except as required by law, Syndax assumes no
obligation to update any forward-looking statements contained
herein to reflect any change in expectations, even as new
information becomes available.
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
SYNDAX
PHARMACEUTICALS, INC.
|
|
(unaudited)
|
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31,
|
|
|
December 31,
|
|
(In
thousands)
|
2023
|
|
|
2022
|
|
Cash, cash equivalents,
short and long-term investments
|
$
|
600,527
|
|
|
$
|
481,271
|
|
Total assets
|
$
|
612,880
|
|
|
$
|
497,236
|
|
Total
liabilities
|
$
|
58,684
|
|
|
$
|
29,787
|
|
Total stockholders'
equity
|
$
|
554,196
|
|
|
$
|
467,449
|
|
|
|
|
|
|
|
|
|
Common stock
outstanding
|
|
84,826,632
|
|
|
|
68,111,385
|
|
Common stock and common
stock equivalents*
|
|
96,316,640
|
|
|
|
77,460,706
|
|
|
|
|
|
|
|
|
|
*Common stock and
common stock equivalents:
|
|
|
|
|
|
|
Common stock
|
|
84,826,632
|
|
|
|
68,111,385
|
|
|
Common stock warrants
(pre-funded)
|
|
285,714
|
|
|
|
1,142,856
|
|
|
|
Common stock and
pre-funded stock warrants
|
|
85,112,346
|
|
|
|
69,254,241
|
|
|
Options to purchase
common stock
|
|
10,684,858
|
|
|
|
7,981,677
|
|
|
Restricted Stock
Units
|
|
519,436
|
|
|
|
224,788
|
|
|
|
Total common stock and
common stock equivalents
|
|
96,316,640
|
|
|
|
77,460,706
|
|
SYNDAX
PHARMACEUTICALS, INC.
|
|
(unaudited)
|
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
December 31,
|
|
|
Year Ended
December 31,
|
|
(In thousands,
except share and per share data)
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
$
|
55,126
|
|
|
$
|
31,841
|
|
|
$
|
163,032
|
|
|
$
|
118,499
|
|
|
Selling, general and
administrative
|
|
22,779
|
|
|
|
10,192
|
|
|
|
66,922
|
|
|
|
33,258
|
|
Total operating
expenses
|
|
77,905
|
|
|
|
42,033
|
|
|
|
229,954
|
|
|
|
151,757
|
|
Loss from
operations
|
|
(77,905)
|
|
|
|
(42,033)
|
|
|
|
(229,954)
|
|
|
|
(151,757)
|
|
Other income (expense),
net
|
|
5,432
|
|
|
|
2,839
|
|
|
|
20,594
|
|
|
|
2,419
|
|
Net loss
|
$
|
(72,473)
|
|
|
$
|
(39,194)
|
|
|
$
|
(209,360)
|
|
|
$
|
(149,338)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable
to common stockholders
|
$
|
(72,473)
|
|
|
$
|
(39,194)
|
|
|
$
|
(209,360)
|
|
|
$
|
(149,338)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common
|
|
|
|
|
|
|
|
|
|
|
|
|
stockholders--basic and
diluted
|
$
|
(1.00)
|
|
|
$
|
(0.62)
|
|
|
$
|
(2.98)
|
|
|
$
|
(2.46)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average number
of common stock
|
|
|
|
|
|
|
|
|
|
|
|
|
used to compute net
loss per share attributable
|
|
|
|
|
|
|
|
|
|
|
|
|
to common
stockholders--basic and diluted
|
|
72,520,784
|
|
|
|
63,192,750
|
|
|
|
70,370,519
|
|
|
|
60,760,906
|
|
View original
content:https://www.prnewswire.com/news-releases/syndax-pharmaceuticals-reports-fourth-quarter-and-full-year-2023-financial-results-and-provides-clinical-and-business-update-302073241.html
SOURCE Syndax Pharmaceuticals, Inc.