– NDA submission initiated for revumenib
in R/R KMT2Ar acute leukemia under RTOR –
– mNPM1 final efficacy data from the
Phase 1 portion of AUGMENT-101 demonstrates a 36% CR/CRh
rate –
– Revumenib and axatilimab U.S.
registrational filings on track for year-end 2023 completion and
potential 2024 approvals –
– Axatilimab to be featured in plenary
session at 65th ASH Annual Meeting –
– Revumenib pivotal monotherapy results,
combination with venetoclax and post-transplant maintenance data to
be highlighted at 65th ASH Annual Meeting –
– Company to host conference call today
at 4:30 p.m. ET –
WALTHAM,
Mass., Nov. 2, 2023 /PRNewswire/ -- Syndax
Pharmaceuticals (Nasdaq: SNDX), a clinical-stage biopharmaceutical
company developing an innovative pipeline of cancer therapies,
today reported its financial results for the quarter ended
September 30, 2023, and provided a
business update.
"Syndax continues to make excellent progress against our key
milestones and corporate priorities and is well positioned to
potentially launch two first- and best-in-class blockbuster
therapies in 2024," said Michael A.
Metzger, Chief Executive Officer. "We are also pleased to
announce final Phase 1 mNPM1 data with a 36% CR/CRh rate, which
demonstrates revumenib's ability as a monotherapy with excellent
safety and tolerability to drive patients with mNPM1 AML to durable
MRD negative remissions. We recently shared topline pivotal
data for revumenib in R/R KMT2Ar acute leukemia, our second
positive pivotal result over the past few months. We look forward
to providing additional monotherapy and combination data, which
further highlight the compelling clinical profile and utility of
each asset, next month at the ASH Annual Meeting."
New Data Announcement
The Company announced today positive data from the Phase 1
portion of the AUGMENT-101 trial of revumenib in a total of 14
patients with relapsed or refractory (R/R) mutant nucleophosmin
(mNPM1) acute myeloid leukemia (AML) who met the recommended Phase
2 dose (RP2D) criteria. The final dataset includes three additional
patients that were enrolled in the Phase 1 trial to complete the
pharmacokinetic characterization of revumenib. In this analysis,
the overall response rate (ORR)1 was 50% (7/14)
with a complete remission (CR) or a CR with partial hematological
recovery (CRh) rate of 36% (5/14); 100% (5/5) of CR/CRh patients
were minimal residual disease (MRD) negative. 43% (3/7) of
responders proceeded to transplant, all after achieving a CR or
CRh. 60% (3/5) of CR/CRh patients maintained a response beyond six
months. At the time of the analysis, four patients remained in
response, with two patients in response over twenty-two months.
Revumenib was well tolerated, and the safety profile was consistent
with what was previously reported in the AUGMENT-101 trial. There
were no Grade 4 or 5 QTc prolongation or greater than Grade 2
differentiation syndrome events, and no patients discontinued due
to treatment-related adverse events (TREAs).
Recent Pipeline Progress and Anticipated Milestones
Revumenib
- In October 2023, the Company
announced positive topline data from the pivotal AUGMENT-101 trial
of revumenib, Syndax's first-in-class menin inhibitor, in patients
with R/R KMT2A-rearranged (KMT2Ar) acute leukemia. The trial met
its primary endpoint at the protocol-defined interim analysis stage
with a CR/CRh rate of 23% (13/57; 95% confidence interval [CI]:
[12.7, 35.8, one-sided p-value = 0.0036]) in the pooled KMT2Ar
acute leukemia cohort. The CR/CRh rate in patients with KMT2Ar AML
was 24.5% (12/49). 39% (14/36) of patients who achieved a CR/CRh
underwent hematopoietic stem cell transplant (HSCT); eight of whom
went to transplant prior to achieving CR/CRh and therefore, were
not included in the reported CR/CRh rate. The CR/CRh responses in
both the overall population and the AML subset were durable with a
6.4-month (95% CI: 3.4, NR) median duration as of the July 2023 data cut-off, with 46% (6/13) remaining
in response. MRD status was assessed in 10 of the 13 patients who
achieved a CR/CRh, 70% (7/10) of whom were MRD negative. Revumenib
was well tolerated, and the overall safety profile was consistent
with the Company's previously reported data. TRAEs leading to dose
reductions and treatment discontinuation were low. Based on the
Independent Data Monitoring Committee (IDMC) recommendation, the
Company stopped the trial to further accrual in the KMT2Ar
cohorts.
The AUGMENT-101 pivotal data will be presented at the upcoming 65th
American Society of Hematology (ASH) Annual Meeting on Sunday, December 10, 2023. Of note, the abstract
for this session only includes data from the Phase 1 portion of the
AUGMENT-101 trial, however the presentation will describe the
pivotal dataset.
- The Company announced today that it has initiated a New Drug
Application (NDA) for revumenib for the treatment of R/R KMT2Ar
acute leukemia under the FDA's Real-time Oncology Review (RTOR)
program. 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. The Company expects to complete the
NDA submission by year-end 2023.
- The AUGMENT-101 pivotal trial is enrolling patients with mNPM1
AML which could support a second indication in acute leukemia for
revumenib. The Company expects to complete enrollment of the cohort
in late 1Q24 or early 2Q24 and report topline data in
4Q24.
- The Company also announced data from patients in the
AUGMENT-101 trial who received revumenib as post-transplant
maintenance, including patients who have been treated with
revumenib maintenance for over a year, resulting in long-term
responses and conversion to MRD negative status. The data will be
presented at the upcoming ASH Annual Meeting on Monday, December 11, 2023. A copy of the abstract
is available on the ASH website at www.hematology.org.
- The Company has several trials of revumenib ongoing across the
treatment landscape in mNPM1 and KMT2Ar acute leukemias that
include the following:
- BEAT-AML: Evaluating the combination of revumenib with
venetoclax and azacitidine in front-line AML patients, being
conducted as part of the Leukemia & Lymphoma
Society's Beat AML® Master Clinical Trial. The
Company expects to present preliminary safety and efficacy data in
4Q23.
- 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. Early results
from the trial will be featured in an oral presentation at the
ASH Annual Meeting on Saturday, December 9,
2023. A copy of the abstract is available on the ASH website
at www.hematology.org.
- AUGMENT-102: Evaluating the combination of revumenib with
chemotherapy in patients with R/R acute leukemias. The Company
expects to provide an update on initial safety data along with the
RP2D from the trial in 4Q23.
- INTERCEPT: Evaluating revumenib as a monotherapy in patients
with AML who are minimal residual disease-positive following
initial treatment as part of the INTERCEPT AML Master Clinical
Trial.
- The Company plans to initiate a trial of revumenib with 7+3
cytarabine and daunorubicin chemotherapy followed by maintenance
treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute
leukemias in late 4Q23 or early 1Q24.
- A proof-of-concept clinical trial of revumenib in patients with
unresectable metastatic microsatellite stable colorectal cancer is
enrolling patients, and the Company expects to provide an update
on the Phase 1 trial in 1Q24.
Axatilimab
- In July 2023, the Company and its
partner, Incyte, announced positive topline data from the
pivotal AGAVE-201 trial of axatilimab, Syndax's anti-CSF-1R
antibody, in patients with chronic graft-versus-host disease
(cGVHD) following two or more prior lines of therapy. All
three dose cohorts, 0.3 mg/kg every two weeks, 1.0 mg/kg every two
weeks and 3.0 mg/kg every four weeks, met the primary endpoint.
The ORR within the first six months of treatment at the 0.3
mg/kg dose was 74%, and 60% of these patients were still responding
at one year. Furthermore, axatilimab was generally well tolerated,
and the most common adverse events were consistent with on-target
effects and prior trials. Syndax and Incyte expect to submit a
Biologics License Application (BLA) filing by year-end 2023.
The pivotal AGAVE-201 trial results will be featured at the
plenary session at the ASH Annual Meeting on Sunday, December 10, 2023. A copy of the abstract
is available on the ASH website at www.hematology.org.
- The Company expects to initiate a randomized, double-blind and
placebo-controlled Phase 2 trial that assesses the efficacy, safety
and tolerability of axatilimab in patients with idiopathic
pulmonary fibrosis (IPF) by year-end 2023.
- Incyte and Syndax expect to initiate a trial assessing
axatilimab in combination with ruxolitinib in cGVHD in
mid-2024.
Third Quarter 2023 Financial Results
As of September 30, 2023, Syndax had cash, cash
equivalents, short and long-term investments of $379.3 million and 69.9 million common shares and
prefunded warrants outstanding.
Third quarter 2023 research and development expenses increased
to $39.1 million from $26.9 million for the comparable prior year
period. The 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.
Third quarter 2023 general and administrative expenses increased
to $17.3 million from $8.2 million for the comparable prior year
period. The increase is primarily due to employee-related expenses
and professional fees.
For the three months ended September 30, 2023, Syndax
reported a net loss attributable to common stockholders of
$51.1 million, or $0.73 per share, compared to a net loss
attributable to common stockholders of $35.4
million, or $0.58 per share,
for the comparable prior year period.
Financial Update and Guidance
For the full year of 2023, the Company expects research and
development expenses to be $160 to
$165 million and total operating
expenses to be $225 to $230 million. This is a reduction from prior
guidance of $160 to $175 million for research and development
expenses and $225 to $240 million for total operating expenses.
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, Thursday, November 2, 2023.
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: SNDX3Q23
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast:
https://www.veracast.com/webcasts/syndax/events/SNDX3Q23.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 Syndax
Syndax 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 "may," "will," "expect," "plan," "anticipate,"
"estimate," "intend," "could," "believe" 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 potential submission of
an NDA and BLA by year-end, the potential use of our product
candidates to treat various cancer indications and fibrotic
diseases, and Syndax's expected full year research and development
expenses as well as 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.
References
1 Overall response rate includes CR, CRh, CRp,
CRi, MLFS, and PR
CR = Complete remission
CRh = Complete remission with partial hematologic recovery
CRp = Complete remission with incomplete platelet recovery
CRi = Complete remission with incomplete count recovery
MLFS = Morphologic leukemia-free state
PR = Partial response
Syndax Contact
Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827
SNDX-G
SYNDAX
PHARMACEUTICALS, INC.
|
|
(unaudited)
|
|
CONDENSED
CONSOLIDATED BALANCE SHEETS
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30,
|
|
|
December 31,
|
|
(In
thousands)
|
2023
|
|
|
2022
|
|
Cash, cash equivalents,
short and long-term investments
|
$
|
379,261
|
|
|
$
|
481,271
|
|
Total assets
|
$
|
399,224
|
|
|
$
|
497,236
|
|
Total
liabilities
|
$
|
40,038
|
|
|
$
|
29,787
|
|
Total stockholders'
equity
|
$
|
359,186
|
|
|
$
|
467,449
|
|
|
|
|
|
|
|
|
|
Common stock
outstanding
|
|
69,638,168
|
|
|
|
68,111,385
|
|
Common stock and common
stock equivalents*
|
|
83,992,083
|
|
|
|
77,460,706
|
|
|
|
|
|
|
|
|
|
*Common stock and
common stock equivalents:
|
|
|
|
|
|
|
Common stock
|
|
69,638,168
|
|
|
|
68,111,385
|
|
|
Common stock warrants
(pre-funded)
|
|
285,714
|
|
|
|
1,142,856
|
|
|
|
Common stock and
pre-funded stock warrants
|
|
69,923,882
|
|
|
|
69,254,241
|
|
|
Options to purchase
common stock
|
|
13,543,965
|
|
|
|
7,981,677
|
|
|
Restricted Stock
Units
|
|
524,236
|
|
|
|
224,788
|
|
|
|
Total common stock and
common stock equivalents
|
|
83,992,083
|
|
|
|
77,460,706
|
|
SYNDAX
PHARMACEUTICALS, INC.
|
|
(unaudited)
|
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
September 30,
|
|
|
Nine Months
Ended
September 30,
|
|
(In thousands,
except share and per share data)
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
$
|
39,087
|
|
|
$
|
26,901
|
|
|
$
|
107,906
|
|
|
$
|
86,658
|
|
|
General and
administrative
|
|
17,268
|
|
|
|
8,240
|
|
|
|
44,143
|
|
|
|
23,066
|
|
Total operating
expenses
|
|
56,355
|
|
|
|
35,141
|
|
|
|
152,049
|
|
|
|
109,724
|
|
Loss from
operations
|
|
(56,355)
|
|
|
|
(35,141)
|
|
|
|
(152,049)
|
|
|
|
(109,724)
|
|
Other income (expense),
net
|
|
5,209
|
|
|
|
(262)
|
|
|
|
15,162
|
|
|
|
(420)
|
|
Net loss
|
$
|
(51,146)
|
|
|
$
|
(35,403)
|
|
|
$
|
(136,887)
|
|
|
$
|
(110,144)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable
to common stockholders
|
$
|
(51,146)
|
|
|
$
|
(35,403)
|
|
|
$
|
(136,887)
|
|
|
$
|
(110,144)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common
|
|
|
|
|
|
|
|
|
|
|
|
|
stockholders--basic and
diluted
|
$
|
(0.73)
|
|
|
$
|
(0.58)
|
|
|
$
|
(1.97)
|
|
|
$
|
(1.84)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average number
of common stock
|
|
|
|
|
|
|
|
|
|
|
|
|
used to compute net
loss per share attributable
|
|
|
|
|
|
|
|
|
|
|
|
|
to common
stockholders--basic and diluted
|
|
69,855,766
|
|
|
|
60,670,294
|
|
|
|
69,645,888
|
|
|
|
59,941,384
|
|
View original
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SOURCE Syndax Pharmaceuticals, Inc.