Significant progress achieved with pipeline
focused on rare hematologic diseases and cancers
Positive Phase 1 results in patients with
sickle cell disease (SCD) presented at European Hematology
Association (EHA) Virtual Congress supporting etavopivat’s
potential to significantly impact RBC health and lifespan
Phase 1 trial of FT-7051 enrolling men with
metastatic castration-resistant prostate cancer (mCRPC); initial
results to be presented in October at the NCI/AACR/EORTC Virtual
International Conference on Molecular Targets and Cancer
Therapeutics
Olutasidenib data in relapsed/refractory acute
myeloid leukemia (R/R AML) presented at the American Society of
Clinical Oncology (ASCO) and EHA Virtual Congress; new drug
application (NDA) preparation ongoing
Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a
clinical-stage biopharmaceutical company focused on rare
hematologic diseases and cancers, today reported financial results
for the second quarter ended June 30, 2021. The company also
highlighted recent progress and upcoming milestones for its
pipeline programs.
“During the second quarter, we presented positive results from
our ongoing Phase 1 trial demonstrating etavopivat’s highly
differentiated profile and multimodal mechanism of action to
improve markers of sickle cell disease and red blood cell health
that are associated with vaso-occlusion,” said Frank Lee, president
and chief executive officer of Forma. “These results, in addition
to the progress on our other clinical programs this quarter,
position us well to deliver on our mission of transforming the
lives of patients with rare hematologic diseases and cancers.”
Key Business and Clinical
Highlights
PKR Program in Sickle Cell Disease (SCD):
- Clinical data presented at EHA Virtual Congress support
potential of investigational agent etavopivat to significantly
impact RBC health and lifespan. Updated results were presented
from the two week multiple ascending dose (MAD) cohorts and initial
open-label extension (OLE) results administering etavopivat for up
to 12 weeks, including:
- Sustained increases in hemoglobin levels. In the MAD
cohorts 73% of patients (11/15) achieved a hemoglobin increase of ≥
1 g/dL at the end of two-weeks of treatment. In the OLE, hemoglobin
levels increased >1g/dL in 88% of patients (7/8) receiving once
daily treatment for at least two weeks, and this increase was
sustained in those patients receiving continued treatment for up to
12 weeks.
- Improvements in RBC oxygenation and deformability. RBC’s
from 14 patients in the MAD cohorts showed increased
hemoglobin-oxygen affinity, a significant shift in the point of
sickling (POS), and improved deformability.
- Significant reduction in hemolysis with markers approaching
normal levels. Reticulocyte counts were reduced in 100% of
patients (15/15), with normalization in some patients at the end of
2 weeks of treatment. The majority of patients demonstrated a
marked decrease in lactate dehydrogenase levels (LDH) and indirect
bilirubin levels as compared to baseline levels.
- Reduction in systemic biomarkers related to inflammation and
hypercoagulability. Initial results from the OLE showed
improvement in systemic biomarkers such as lower levels of
TNF-alpha, a marker of inflammation and decreases in prothrombin
1.2 and D-dimer, markers of coagulation activation.
- Etavopivat was well tolerated with a safety profile
consistent with underlying sickle cell disease. Etavopivat was
well tolerated at doses up to 600mg daily (150% of the maximum dose
in the ongoing Phase 2/3 Hibiscus Study).
CPB/p300 Program in Prostate Cancer:
- FT-7051 Phase 1 clinical trial enrollment is ongoing. In
January 2021, Forma announced the first patient dosed in the
ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment
of mCRPC. The trial is a multicenter, open-label evaluation of the
safety and tolerability, pharmacokinetics/pharmacodynamics (PK/PD),
and preliminary anti-tumor activity, of FT-7051 in men with mCRPC
who have progressed despite prior therapy with at least one
anti-androgen therapy. The adaptive trial design is intended to
accelerate the dose escalation to potentially therapeutic doses and
yield important safety information, as well as to identify
biomarkers of clinical benefit such as PSA response. Genetic
mutation analysis will be conducted to correlate genetic changes
with resistance to standard-of-care and will also evaluate
expression of the AR-v7 splice variant, for which there are no
approved therapies.
IDH1 Program in AML and Glioma:
- Phase 2 registrational results for olutasidenib in R/R AML
were presented at scientific conferences. Olutasidenib data in
R/R AML were presented at both the annual ASCO and EHA meetings in
June 2021. The primary efficacy evaluable population, comprised of
123 patients, received 150 mg of olutasidenib twice daily for at
least six months prior to the planned interim analysis. The primary
endpoint, a composite complete remission (CR) or CR plus CR with
partial hematologic recovery (CRh), was achieved in 33.3% (30% CR
and 3% CRh) of patients. While the median duration of response was
not yet reached, in a sensitivity analysis with hematopoietic stem
cell transplant considered as the end of a response, the median
duration was 13.8 months. The median overall survival (OS) was 10.5
months. Although a median OS has not yet been reached for the
CR/CRh population, 18-month survival is estimated at 87% for that
response category, and median survival is 15.0 months for
non-CR/CRh responders. In addition, among patients with a CR who
were transfusion-dependent at baseline, 56-day transfusion
independence was achieved in 100% of patients as measured by
platelets and 80% as measured by RBC’s. Olutasidenib was
well-tolerated, and adverse events were consistent with the late
stage disease in this heavily pre-treated patient population. Based
upon these results, Forma is preparing an NDA for the R/R AML
indication.
Corporate
- In June 2021, Forma announced the appointment of John E.
Bishop, Ph.D., as chief technology officer. Dr. Bishop leads
chemistry, manufacturing and control (CMC)-related functions and
quality, encompassing Forma’s early pipeline through commercial
product. Dr. Bishop’s background includes extensive expertise with
CMC development in oncology and hematology. Prior to joining Forma,
Dr. Bishop served as senior vice president of pharmaceutical
sciences at Epizyme, Inc., where he was a member of the executive
team and held overall responsibility for the CMC and quality
assurance functions.
Upcoming Milestones
- Scientific conference presentation of updated Phase 1
etavopivat results in SCD. Updated results of safety, clinical
activity, and biomarkers from the 12-week OLE are expected to be
presented at a scientific congress in late 2021. Up to 20 patients
are being administered etavopivat 400mg once daily and assessed for
hematologic and hemolytic response, improvements in RBC oxygenation
and deformability, and systemic markers of SCD.
- Initiation of etavopivat trials in thalassemia and pediatric
sickle cell patients. Enrollment in a Phase 2 trial of
etavopivat in thalassemia patients is expected to begin prior to
the end of the year, with results anticipated in 2022. The trial
may enroll up to 60 patients with either thalassemia or SCD who are
receiving chronic red blood cell transfusions, or thalassemia
without chronic red blood cell transfusions. A trial in pediatric
sickle cell disease patients is planned to begin in the first half
of 2022.
- Scientific conference presentation of initial Phase 1
FT-7051 clinical results in mCRPC. An abstract from this
ongoing trial has been accepted for presentation at the
NCI/AACR/EORTC Virtual International Conference on Molecular
Targets and Cancer Therapeutics taking place Oct. 7-10, 2021. The
presentation will include preclinical data and initial clinical
results on safety, tolerability and PK/PD from patients undergoing
dose escalation.
- Possibility of COVID-19 impact remains. The COVID-19
pandemic remains a factor in the successful completion of these
milestones and ongoing clinical trials. Many clinical trials across
the biopharma industry, including Forma’s, have been impacted by
the COVID-19 pandemic. Clinical trial sites implementing new
policies in response to COVID-19 may result in potential delays to
enrollment of clinical trials or changes in the ability to access
sites participating in clinical trials.
Financial Results
- Cash Position: Cash, cash equivalents and marketable
securities were $570.8 million as of June 30, 2021, as compared to
$645.6 million as of Dec. 31, 2020. Current cash runway is
projected through the third quarter of 2024.
- Research and Development (R&D) Expenses: R&D
expenses were $31.6 million for the quarter ended June 30, 2021,
compared to $20.5 million for the quarter ended June 30, 2020. The
increase was primarily attributable to etavopivat development, as
well as increases in staff and stock-based compensation.
- General and Administrative (G&A) Expenses: G&A
expenses were $12.5 million for the quarter ended June 30, 2021,
compared to $6.4 million for the quarter ended June 30, 2020. The
increase in was primarily attributable to increased stock-based
compensation, executive and staff hiring, professional fees, and
insurance.
- Net Loss: Net loss was $43.6 million for the quarter
ended June 2021, compared to net loss of $25.4 million for the
quarter ended June 30, 2020.
Forma will conduct a conference call and
webcast Aug.13 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss
second quarter 2021 results and business updates. The call can be
accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386
internationally, with conference ID 9155938.
The live webcast will be available in the
“News & Investors” section of Forma’s website
www.formatherapeutics.com.
About Forma Therapeutics
Forma Therapeutics is a clinical-stage biopharmaceutical company
focused on the research, development and commercialization of novel
therapeutics to transform the lives of patients with rare
hematologic diseases and cancers. Our R&D engine combines deep
biology insight, chemistry expertise and clinical development
capabilities to create drug candidates with differentiated
mechanisms of action focused on indications with high unmet need.
Our work has generated a broad proprietary portfolio of programs
with the potential to provide profound patient benefit. For more
information, please visit www.FormaTherapeutics.com or follow us on
Twitter @FORMAInc and LinkedIn.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including, without limitation, express or implied
statements regarding the company’s beliefs and expectations
regarding its: business plans and objectives; future plans for
etavopivat, FT-7051 and olutasidenib, including expectations
regarding timing, success and data announcements of our current
ongoing clinical trials; initial results for the etavopivat open
label extension cohort of our Phase 1 clinical trial; therapeutic
potential, clinical benefits, mechanisms of action and safety of
our product candidates, planned regulatory submissions, including
an NDA for olutasidenib, and upcoming milestones for the company’s
other product candidates; growth as a company; presentation of
additional data at upcoming scientific conferences, and other
preclinical data and potential data publications in 2021; the
potential commercial and collaboration opportunities, including
potential future collaborators and parties, as well as value and
market, for our product candidates; uses and need of capital,
expenses and other 2021 financial results currently or in the
future, and the potential impact of COVID-19 on patient retention
and enrollment, future operations, clinical trials or
investigational new drug (IND) applications. The words “may,”
“will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “estimate,” “predict,” “project,” “potential,”
“continue,” “target” and similar expressions are intended to
identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs and are subject to
a number of risks, uncertainties and important factors that may
cause actual events or results to differ materially from those
expressed or implied by any forward-looking statements contained in
this press release, including, without limitation, those risks and
uncertainties associated with the following: the impact of the
COVID-19 pandemic on the company’s business, operations, patient
enrollment and retention , strategy, goals and anticipated
milestones; the therapeutic potential of etavopivat, FT-7051, and
olutasidenib, the timing and completion of our Phase 1 clinical
study in etavopivat and final audit and quality controlled
verification of initial data and related analyses, and the timing
associated with the initiation or continuation of any trials and
success of ongoing clinical trials of etavopivat and FT-7051;
Forma’s ability to execute on its strategy; the submission and
acceptance of a new drug application (NDA) for submission to the
U.S. Food and Drug Administration (FDA) for olutasidenib; positive
results from a clinical study may not necessarily be predictive of
the results of future or ongoing clinical studies; any one or more
of Forma’s product candidates may not be successfully developed and
commercialized; regulatory developments in the United States and
foreign countries; Forma’s ability to protect and maintain our
intellectual property position; the impact of COVID-19 affecting
countries or regions in which we have operations or do business,
including potential negative impacts on our employees, customers,
supply chain and production as well as global economies and
financial markets; Forma’s ability to fund operations; Forma’s
ability to identify satisfactory collaboration opportunities, as
well as those risks and uncertainties set forth more fully under
the caption "Risk Factors" in our Quarterly Report on Form 10-Q for
the quarter ended June 30, 2021, filed with the United States
Securities and Exchange Commission (SEC) and subsequent filings
with the SEC. Forma disclaims any obligation to publicly update or
revise any such statements to reflect any change in expectations or
in events, conditions or circumstances on which any such statements
may be based, or that may affect the likelihood that actual results
will differ from those set forth in the forward-looking statements.
Any forward-looking statements contained in this press release
represent Forma’s views only as of the date hereof and should not
be relied upon as representing its views as of any subsequent date.
Forma explicitly disclaims any obligation to update any
forward-looking statements.
Selected Financial
Information
(in thousands except share and
per share data)
(unaudited)
Statement of Operations Items:
For the Three Months Ended
June 30,
For the Six Months Ended June
30,
2021
2020
2021
2020
Collaboration revenue
$
—
$
—
$
—
$
—
Operating expenses: Research and development
31,587
20,511
57,930
43,721
General and administrative
12,471
6,448
22,338
15,381
Restructuring charges
—
(20
)
—
63
Total operating expenses
44,058
26,939
80,268
59,165
Loss from operations
(44,058
)
(26,939
)
(80,268
)
(59,165
)
Other income (expense): Gain on Hit Discovery divestiture
—
—
—
23,312
Interest income
309
895
571
1,536
Other income (expense), net
272
(2,634
)
268
(2,616
)
Total other income (expense), net
581
(1,739
)
839
22,232
Loss before taxes
(43,477
)
(28,678
)
(79,429
)
(36,933
)
Income tax expense (benefit)
108
(3,238
)
116
(22,723
)
Net loss and comprehensive loss
$
(43,585
)
$
(25,440
)
$
(79,545
)
$
(14,210
)
Accretion of cumulative dividends on Series D redeemable
convertible preferred stock
—
(1,800
)
—
(3,736
)
Net loss allocable to shares of common stock, basic and diluted
$
(43,585
)
$
(27,240
)
$
(79,545
)
$
(17,946
)
Net loss per share of common stock, basic and diluted
$
(0.92
)
$
(4.58
)
$
(1.68
)
$
(4.23
)
Weighted-average shares of common stock outstanding, basic and
diluted
47,339,464
5,943,165
47,317,361
4,245,622
Selected Balance Sheet Items:
June 30, 2021
December 31, 2020
Cash, cash equivalents, and marketable securities
$
570,793
$
645,588
Total assets
$
613,369
$
680,971
Accounts payable, accrued expenses, and other current liabilities
$
29,104
$
31,399
Total stockholders’ equity
$
579,395
$
648,244
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210813005019/en/
Media Contact: Adam Silverstein, +1 917-697-9313 Porter
Novelli adam.silverstein@porternovelli.com
Investor Contact: Mario Corso, +1 781-366-5726 Forma
Therapeutics mcorso@formatherapeutics.com
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