– Updated positive data from Phase 1 portion of AUGMENT-101
trial demonstrates CR/CRh rate of 30% and a median durability of
response of 9.1 months –
– Enrollment complete in pivotal Phase 2 AGAVE-201 trial of
axatilimab in patients with cGVHD –
– Company remains on track for two U.S. registrational
filings by the end of 2023 –
– Company to host conference call today at 4:30 p.m. ET –
WALTHAM,
Mass., Nov. 3, 2022 /PRNewswire/ -- Syndax
Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies, today reported its financial results and provided
a business update for the third quarter ended September 30, 2022.
"As we near the end of this transformational year, we are
particularly excited to share that updated data from the Phase 1
portion of the ongoing AUGMENT-101 trial of revumenib will be
featured during two oral presentations at the American Society of
Hematology (ASH) Annual Meeting in December. The rate of patients
achieving a complete response (CR/CRh) and the nine-month median
duration of CR/CRh response as of the data cutoff, further support
the robust clinical profile of revumenib," said Michael A. Metzger, Chief Executive Officer.
"The data continue to underscore the potential for revumenib to
serve as a first- and best-in-class treatment option for patients
with relapsed/refractory (R/R) nucleophosmin 1 (NPM1) mutations and
mixed lineage leukemia rearrangements (MLLr). Enrollment continues
in AUGMENT-101 and we anticipate reporting topline data from at
least one of the pivotal cohorts starting in the third quarter of
2023, with a potential New Drug Application (NDA) filing expected
by the end of 2023."
Mr. Metzger added, "We also continue to make significant
progress on the development of axatilimab and are pleased to
announce that we have completed enrollment in our pivotal AGAVE-201
trial in chronic graft versus host disease (cGVHD). We look forward
to announcing topline results from the trial in mid-2023, with a
potential biologics license application (BLA) filing expected to
follow by the end of 2023."
Recent Pipeline Progress and Anticipated Milestones
Revumenib
- Earlier today, the Company announced updated positive data from
the Phase 1 portion of the ongoing AUGMENT-101 trial of revumenib
in patients with R/R NPM1 mutant or MLLr (also referred to as
KMT2Ar) acute leukemia which highlighted a 30% (18/60) CR/CRh rate
and a median duration of CR/CRh response of 9.1 months.
Additionally, of the 12 patients who achieved a complete response
on revumenib treatment and then went on to receive a stem cell
transplant, nine (75%) remained in remission as of the data cutoff
date, with a median follow-up of 12.3 months. Three patients were
treated with revumenib maintenance in the compassionate use setting
following stem cell transplant or non-myeloablative stem cell
boost, two of whom (67%) remained in remission for over one year.
To date, there have been no discontinuations due to
treatment-related adverse events. Data reported today will be
featured in two oral sessions at the ASH Annual Meeting on
Saturday, December 10, 2022. Copies
of both abstracts are available on the ASH website at
www.hematology.org.
The Company also announced today that it will host a conference
call and webcast to discuss the ASH data presentations on
Sunday, December 11, 2022 at
8 a.m. CT / 9
a.m. ET. Joining the call will be members of the Syndax
management team as well as Principal Investigators from the
AUGMENT-101 trial. For additional information on how to access the
event, please visit the Investor section of the Company's website
at www.syndax.com.
- The pivotal Phase 2 portion of AUGMENT-101 is ongoing,
enrolling patients across each of three distinct trial populations:
patients with NPM1 mutant acute myeloid leukemia (AML), patients
with MLLr AML, and patients with MLLr acute lymphocytic leukemia
(ALL). Based on discussions with the U.S. Food and Drug
Administration, AUGMENT-101 may serve as the basis for regulatory
filings in each of the three distinct populations. The Company
expects completion of enrollment in the first trial to extend into
the first quarter of 2023 and to report topline data from at least
one of the trials starting in the third quarter of 2023. The
Company continues to expect to submit its first NDA filing for
revumenib by the end of 2023.
- Two trials, BEAT-AML and AUGMENT-102, are ongoing and will
assess the safety, tolerability, and preliminary anti-leukemic
efficacy of revumenib, and establish an appropriate Phase 2 dose
when used in combination with other approved agents. BEAT-AML is a
front-line combination trial of revumenib with venetoclax and
azacitidine being conducted as part of the Leukemia &
Lymphoma Society's Beat AML® Master Clinical
Trial. AUGMENT-102 is a trial assessing revumenib in combination
with chemotherapy in patients with R/R mNPM1 or MLLr acute
leukemias.
- The Company expects the Australasian Leukaemia and Lymphoma
Group (ALLG) to initiate the INTERCEPT trial of revumenib as
monotherapy in patients with AML who are minimal residual
disease-positive following initial treatment, in the fourth quarter
of 2022. The trial is a part of the INTERCEPT AML Master Clinical
Trial, a collaborative clinical trial investigating novel therapies
to target early relapse and clonal evolution as pre-emptive therapy
in AML. Revumenib is the first menin inhibitor to be included in
the INTERCEPT AML Master Clinical Trial.
- The Company remains on track to initiate a proof-of-concept
clinical trial of revumenib in patients with unresectable
metastatic microsatellite stable colorectal cancer in the fourth
quarter of 2022.
Axatilimab
- The Company and its partner, Incyte, today announced completion
of enrollment in the pivotal AGAVE-201 trial evaluating axatilimab
in patients with cGVHD following two or more prior lines of
therapy. The trial is evaluating the safety and efficacy of three
dosing regimens of axatilimab. The primary endpoint will assess
objective response rate based on the 2014 NIH consensus criteria
for cGVHD, with key secondary endpoints including duration of
response and improvement in modified Lee Symptom Scale score. The
Company expects to report topline data in mid-2023, with the
expectation for a BLA filing later in 2023.
- The Company plans to initiate a Phase 2b trial to assess the efficacy, safety and
tolerability of axatilimab in patients with idiopathic pulmonary
fibrosis (IPF) in the fourth quarter of 2022. This 52-week,
randomized, double-blind and placebo-controlled trial is expected
to enroll approximately 170 patients. The primary endpoint will
assess the change from baseline in forced vital capacity, which is
the current registrational endpoint in IPF.
- The Company is working with its partner, Incyte, to initiate a
trial testing axatilimab in combination with ruxolitinib in steroid
naive cGVHD. The Phase 1 trial is in preparation and is expected to
begin in the first quarter of 2023.
Third Quarter 2022 Financial Results
As of September 30, 2022, Syndax had cash, cash equivalents
and short-term investments of $337.8 million and 61.3 million
common shares and pre-funded warrants outstanding.
Third quarter 2022 research and development expenses increased
to $26.9 million from $25.6 million for the prior
year period. The increase was primarily due to increased employee
related expenses and professional fees partially offset by
decreased clinical and manufacturing expenses, in large part the
result of axatilimab cost sharing benefits.
General and administrative expenses for the third quarter 2022
increased to $8.2 million from $6.8 million for the
prior year period. The increase is primarily due to increased
employee related expenses and professional fees.
For the three months ended September 30, 2022, Syndax
reported a net loss attributable to common stockholders
of $35.4 million, or $0.58 per share, compared to a
net loss attributable to common stockholders of $20.6 million, or $0.40 per share, for
the prior year period.
Financial Update and Guidance
For the full year of 2022, the Company is lowering its
expectations of both research and development and total operating
expenses. The Company now expects research and development expenses
to be $115 to $125 million and total operating expenses to be
$145 to $155
million. This is a reduction from the Company's prior
guidance for the full year of 2022 of $130 to $140
million in research and development expenses and
$160 to $170
million in total operating expenses. The updated
guidance reflects the benefits derived from the shared expenses for
our axatilimab cGVHD development program.
In September, Syndax made a debt repayment in connection with
the termination of a Loan Agreement. This loan repayment has
no adverse impact on Syndax's cash guidance and the Company
continues to expect to have sufficient capital to fund operations
into the second half of 2024.
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 3,
2022.
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: SYNDAXQ3
Domestic Dial-in Number: 800-225-9448
International Dial-in Number: 203-518-9708
Live
webcast: https://www.veracast.com/webcasts/OpenEx/General/S03S7x.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 Pharmaceuticals, Inc.
Syndax Pharmaceuticals is a clinical stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. Highlights of the Company's pipeline include
revumenib (SNDX-5613), a highly selective inhibitor of the
Menin–MLL binding interaction, and axatilimab, a monoclonal
antibody that blocks the colony stimulating factor 1 (CSF-1)
receptor, both currently in pivotal trials. 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," "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 use of our product candidates to treat
various cancer indications and fibrotic diseases, and Syndax's
expected fourth quarter and full year research and development
expenses, and expected 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; the
COVID-19 pandemic may disrupt our business and that of the third
parties on which we depend, including delaying or otherwise
disrupting our clinical trials and preclinical studies,
manufacturing and supply chain, or impairing employee productivity;
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
|
|
|
|
|
|
|
|
|
|
September
30,
|
|
December
31,
|
(In
thousands)
|
2022
|
|
2021
|
Cash, cash equivalents,
short and long-term investments
|
$
337,838
|
|
$
439,936
|
Total assets
|
$
359,687
|
|
$
449,657
|
Total
liabilities
|
$
21,585
|
|
$
41,289
|
Total stockholders'
equity (deficit)
|
$
338,102
|
|
$
408,368
|
|
|
|
|
|
|
Common stock
outstanding
|
60,122,661
|
|
54,983,105
|
Common stock and common
stock equivalents*
|
68,877,618
|
|
66,011,976
|
|
|
|
|
|
|
*Common stock and
common stock equivalents:
|
|
|
|
|
Common stock
|
60,122,661
|
|
54,983,105
|
|
Common stock warrants
(pre-funded)
|
1,142,856
|
|
3,975,024
|
|
|
Common stock and
pre-funded stock warrants
|
61,265,517
|
|
58,958,129
|
|
Options to purchase
common stock
|
7,386,063
|
|
6,921,514
|
|
Restricted Stock
Units
|
226,038
|
|
132,333
|
|
|
Total common stock and
common stock equivalents
|
68,877,618
|
|
66,011,976
|
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)
|
2022
|
|
2021
|
|
2022
|
|
2021
|
License fee
revenue
|
$
-
|
|
$
12,375
|
|
$
-
|
|
$
13,133
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
26,901
|
|
25,606
|
|
86,658
|
|
64,348
|
|
General and
administrative
|
8,240
|
|
6,801
|
|
23,066
|
|
18,314
|
Total operating
expenses
|
35,141
|
|
32,407
|
|
109,724
|
|
82,662
|
Loss from
operations
|
(35,141)
|
|
(20,032)
|
|
(109,724)
|
|
(69,529)
|
Other (expense) income,
net
|
(262)
|
|
(607)
|
|
(420)
|
|
(1,743)
|
Net
loss
|
$
(35,403)
|
|
$
(20,639)
|
|
$
(110,144)
|
|
$
(71,272)
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable
to common stockholders
|
$
(35,403)
|
|
$
(20,639)
|
|
$
(110,144)
|
|
$
(71,272)
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common
|
|
|
|
|
|
|
|
|
stockholders--basic and
diluted
|
$
(0.58)
|
|
$
(0.40)
|
|
$
(1.84)
|
|
$
(1.38)
|
|
|
|
|
|
|
|
|
|
|
Weighted-average number
of common stock
|
|
|
|
|
|
|
|
|
used to compute net
loss per share attributable
|
|
|
|
|
|
|
|
|
to common
stockholders--basic and diluted
|
60,670,294
|
|
51,962,320
|
|
59,941,384
|
|
51,690,173
|
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SOURCE Syndax Pharmaceuticals, Inc.