– Topline data from AUGMENT-101 KMT2Ar
patients expected in the third quarter of 2023 –
–Topline data from the pivotal AGAVE-201 trial
on track for mid-2023 –
– Two U.S. registrational filings expected by
the end of 2023 –
– Company to host conference call today at
4:30 p.m. ET –
WALTHAM,
Mass., Feb. 28, 2023 /PRNewswire/ -- Syndax
Pharmaceuticals, Inc. (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, 2022 and provided a business update.
"With two pivotal data readouts expected later this year and
potential regulatory filings to follow shortly thereafter, we
expect 2023 will be an extraordinary year for Syndax," said
Michael A. Metzger, Chief Executive
Officer. "The fourth quarter was marked by significant progress on
the clinical, regulatory and operational fronts and we expect this
positive momentum to continue in 2023. We had a strong presence at
the American Society of Hematology (ASH) Annual Meeting where we
presented positive revumenib results from the Phase 1 portion of
the AUGMENT-101 trial and the axatilimab Phase 1/2 trial data were
published in the Journal of Clinical Oncology. These data
further support the potential for both our pipeline agents to be
first- and best-in-class therapies that could meaningfully change
treatment paradigms."
"For revumenib, we are on track to begin reporting topline data
from the AUGMENT-101 pivotal trial in the third quarter of this
year, with the first data expected to be in patients with KMT2A
rearranged (KMT2Ar) acute leukemia and expect to file a New Drug
Application (NDA) by year-end 2023. For axatilimab, we also remain
on track to report topline results from our pivotal AGAVE-201 trial
in chronic graft versus host disease (cGVHD) in mid-2023, with a
Biologics License Application (BLA) filing expected to follow by
year-end 2023. We look forward to providing updates on all of our
progress as we continue to strive toward our mission of realizing a
future in which people with cancer live longer and better than ever
before."
Recent Pipeline Progress and Anticipated Milestones
Revumenib
- The pivotal Phase 2 portion of AUGMENT-101 is enrolling
relapsed/refractory (R/R) patients across distinct trial
populations: patients with nucleophosmin mutant (mNPM1) acute
myeloid leukemia (AML), patients with KMT2Ar AML, and patients with
KMT2Ar acute lymphocytic leukemia (ALL), each of which may serve as
the basis for regulatory filings. Following the receipt of
Breakthrough Therapy designation from the FDA for revumenib for the
treatment of R/R acute leukemia harboring a KMT2A rearrangement,
regardless of age or tumor type, and based on discussions with the
FDA, the Company will pool data from the AUGMENT-101 cohorts
enrolling R/R KMT2Ar AML and R/R KMT2Ar ALL to file a single NDA
for the treatment of adult and pediatric KMT2Ar acute leukemia. The
Company has completed enrollment of a sufficient number of KMT2Ar
acute leukemia patients to support this filing strategy and expects
to share topline data from the KMT2Ar cohort in the third quarter
of 2023 and submit an NDA filing by the end of 2023. The Company
also expects to complete enrollment of the NPM1 AML cohort in the
second half of 2023.
- During two oral presentations at the 64th ASH Annual
Meeting in December 2022, the Company
reported updated positive data from the Phase 1 portion of the
ongoing Phase 1/2 AUGMENT-101 trial. As of the March 2022 data cutoff date, 60 patients with R/R
mutant NPM1 or KMT2Ar acute leukemia were efficacy evaluable. In
the efficacy evaluable population, the overall response rate was
53% (32/60) with a CR/CRh rate of 30% (18/60), and 78% (14/18) of
patients with CR/CRh attaining minimal residual disease (MRD)
negativity. Additional analyses from the trial indicate that at
doses which met the protocol defined criteria for a recommended
Phase 2 dose, the CR/CRh rate was 27% in both the KMT2Ar (10/37)
and the mutant NPM1 (3/11) patient populations. A total of 38%
(12/32) of responders proceeded to transplant. The median time to
response in the trial was 1.9 months, and the median duration of
CR/CRh response was 9.1 months in the efficacy evaluable population
as of data cutoff. Revumenib was well-tolerated, and there were no
discontinuations due to treatment related adverse events.
- 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 KMT2Ar acute leukemias.
- The Australasian Leukaemia and Lymphoma Group (ALLG) has
initiated the INTERCEPT trial of revumenib as monotherapy in
patients with AML who are minimal residual disease-positive
following initial treatment. 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.
- A proof-of-concept clinical trial of revumenib in patients with
unresectable metastatic microsatellite stable colorectal cancer has
initiated and the Company expects to report initial topline data
from the trial by year-end 2023.
Axatilimab
- The Company and its partner, Incyte, expect to report topline
data from the pivotal AGAVE-201 trial evaluating axatilimab in
patients with cGVHD following two or more prior lines of therapy in
mid-2023, with the expectation for a BLA filing by year-end
2023.
- The Company and Incyte announced that results from the Phase
1/2 trial of axatilimab in patients with recurrent or refractory
cGVHD following two or more prior lines of therapy were published
in the Journal of Clinical Oncology. The article, titled
"Axatilimab for chronic graft-versus-host disease after failure
of at least two prior systemic therapies: results of a Phase 1/2
study," is available online.
- The Company plans to initiate a 52-week, randomized,
double-blind and placebo-controlled Phase 2b trial to assess the efficacy, safety and
tolerability of axatilimab in patients with idiopathic pulmonary
fibrosis (IPF) in the first half of 2023. 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 Incyte to initiate a trial testing
axatilimab in combination with ruxolitinib in steroid naive cGVHD.
The Phase 1 trial is expected to begin later this year.
Corporate Updates
- In December 2022, the Company
announced the appointment of Steve
Sabus as Chief Commercial Officer. Mr. Sabus brings to
Syndax nearly thirty years of commercial experience launching drugs
and building sales and marketing organizations within the
biopharmaceutical industry.
Fourth Quarter and Full Year 2022 Financial Results
As of December 31, 2022, Syndax had cash, cash equivalents,
short-term and long-term investments of $481.3 million and 69.3 million common shares and
prefunded warrants outstanding.
Fourth quarter 2022 research and development expenses increased
to $31.8 million from $23.9 million, and for the full year increased to
$118.5 million compared to
$88.2 million for 2021. The increase
was primarily due to increased clinical activities as well as
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 fourth quarter 2022
increased to $10.2 million from
$6.9 million and for the full year
increased to $33.3 million compared
to $25.2 million for 2021. The
increase is primarily due to increased pre-commercialization
activities as well as employee related expenses and professional
fees.
For the three months ended December 31, 2022, Syndax
reported a net loss attributable to common stockholders of
$39.2 million, or $0.62 per share, compared to a net gain
attributable to common stockholders of $96.2
million, or $1.81 per share,
for the prior year period. For the year ended December 31,
2022, Syndax reported a net loss attributed to common stockholders
of $149.3 million or $2.46 per share, compared to a net gain
attributable to common stockholders of $24.9
million or $0.48 per share for
the prior year.
Financial Update and Guidance
In December 2022, Syndax issued
7,840,909 shares of its common stock at a price to the public of
$22.00 per share. This includes the
exercise in full by the underwriters of their option to purchase up
to 1,022,727 additional shares of common stock. As a result, Syndax
received aggregate net proceeds of $162.0
million after deducting underwriting discounts and
commissions and estimated offering expenses payable by Syndax.
For the first quarter of 2023, the Company expects research and
development expenses to be $30 to
$35 million and total operating
expenses to be $40 to $45 million. For the full year of 2023, the
Company expects research and development expenses to be
$160 to $175
million and total operating expenses to be $225 to $240
million.
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 28, 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: SNDXQ422
Domestic Dial-in Number: 800-245-3047
International Dial-in Number: 203-518-9765
Live webcast:
https://www.veracast.com/webcasts/OpenEx/General/SNDXQ4.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, 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, 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 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; 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
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31,
|
|
|
December 31,
|
|
(In
thousands)
|
2022
|
|
|
2021
|
|
Cash, cash equivalents,
short and long-term investments
|
$
|
481,271
|
|
|
$
|
439,936
|
|
Total assets
|
$
|
497,236
|
|
|
$
|
449,657
|
|
Total
liabilities
|
$
|
29,787
|
|
|
$
|
41,289
|
|
Total stockholders'
equity (deficit)
|
$
|
467,449
|
|
|
$
|
408,368
|
|
|
|
|
|
|
|
|
|
Common stock
outstanding
|
|
68,111,385
|
|
|
|
54,983,105
|
|
Common stock and common
stock equivalents*
|
|
77,460,706
|
|
|
|
66,011,976
|
|
|
|
|
|
|
|
|
|
*Common stock and
common stock equivalents:
|
|
|
|
|
|
|
Common stock
|
|
68,111,385
|
|
|
|
54,983,105
|
|
|
Common stock warrants
(pre-funded)
|
|
1,142,856
|
|
|
|
3,975,024
|
|
|
|
Common stock and
pre-funded stock warrants
|
|
69,254,241
|
|
|
|
58,958,129
|
|
|
Options to purchase
common stock
|
|
7,981,677
|
|
|
|
6,921,514
|
|
|
Restricted Stock
Units
|
|
224,788
|
|
|
|
132,333
|
|
|
|
Total common stock and
common stock equivalents
|
|
77,460,706
|
|
|
|
66,011,976
|
|
SYNDAX
PHARMACEUTICALS, INC.
|
|
(unaudited)
|
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended
December 31,
|
|
|
Twelve Months
Ended
December 31,
|
|
(In thousands,
except share and per share data)
|
2022
|
|
|
2021
|
|
|
2022
|
|
|
2021
|
|
License fee
revenue
|
$
|
-
|
|
|
$
|
126,576
|
|
|
$
|
-
|
|
|
$
|
139,709
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
31,841
|
|
|
|
23,900
|
|
|
|
118,499
|
|
|
|
88,248
|
|
|
General and
administrative
|
|
10,192
|
|
|
|
6,927
|
|
|
|
33,258
|
|
|
|
25,241
|
|
Total operating
expenses
|
|
42,033
|
|
|
|
30,827
|
|
|
|
151,757
|
|
|
|
113,489
|
|
Loss from
operations
|
|
(42,033)
|
|
|
|
95,749
|
|
|
|
(151,757)
|
|
|
|
26,220
|
|
Other income (expense),
net
|
|
2,839
|
|
|
|
449
|
|
|
|
2,419
|
|
|
|
(1,294)
|
|
Net loss
|
$
|
(39,194)
|
|
|
$
|
96,198
|
|
|
$
|
(149,338)
|
|
|
$
|
24,926
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable
to common stockholders
|
$
|
(39,194)
|
|
|
$
|
96,198
|
|
|
$
|
(149,338)
|
|
|
$
|
24,926
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common
|
|
|
|
|
|
|
|
|
|
|
|
|
stockholders--basic and
diluted
|
$
|
(0.62)
|
|
|
$
|
1.81
|
|
|
$
|
(2.46)
|
|
|
$
|
0.48
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average number
of common stock
|
|
|
|
|
|
|
|
|
|
|
|
|
used to compute net
loss per share attributable
|
|
|
|
|
|
|
|
|
|
|
|
|
to common
stockholders--basic and diluted
|
|
63,192,750
|
|
|
|
53,176,335
|
|
|
|
60,760,906
|
|
|
|
52,064,809
|
|
View original
content:https://www.prnewswire.com/news-releases/syndax-pharmaceuticals-reports-fourth-quarter-and-full-year-2022-financial-results-and-provides-clinical-and-business-update-301758656.html
SOURCE Syndax Pharmaceuticals, Inc.