Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage
company developing transformative therapies for the treatment of
cancer and rare diseases, today announced financial results for the
third quarter ended September 30, 2021 and provided a business
update.
“We have made significant progress advancing our
TARA-002 clinical programs in the third quarter, most notably the
completion of our confirmatory large-scale GMP comparability work
and the clearance of our Investigational New Drug (IND) application
by the U.S. Food and Drug Administration (FDA) for our non-muscle
invasive bladder cancer (NMIBC) clinical development program. NMIBC
is one of the most recurrent and difficult to treat cancers with
limited treatment options, and we look forward to initiating our
Phase 1 clinical trial of TARA-002 in adults with high-grade NMIBC
by the end of the year,” said Jesse Shefferman, Chief Executive
Officer of Protara Therapeutics. “We are also working to align with
the FDA on the design of a clinical trial of TARA-002 in patients
with lymphatic malformations (LMs), a rare pediatric indication for
which there are currently no U.S. FDA-approved therapies.”
Recent Highlights and Upcoming
Milestones
TARA-002 in NMIBC
- In October 2021, Protara announced
that the FDA cleared the Company’s IND application for TARA-002, an
investigational cell-based therapy in development for the treatment
of NMIBC. The confirmatory, GMP-scale comparability data for
TARA-002 in relation to OK-432, the originator therapy for
TARA-002, have been completed and were reviewed by the FDA as part
of the clearance of the IND.
- The Company plans to commence a
Phase 1 clinical trial by the end of 2021 to assess the safety,
tolerability, and preliminary signs of anti-tumor activity of
TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs
- In October 2021, the Company
updated its IND submission for TARA-002 for the treatment of LMs
with completed confirmatory, GMP-scale comparability data, and
plans to engage the FDA on the design, and subsequently initiate a
clinical trial in pediatric LM patients.
IV Choline Chloride in Intestinal Failure
Associated Liver Disease (IFALD)
- In September 2021, the Company
announced the completion of a retrospective prevalence study
designed to enhance understanding of the incidence of IFALD in
patients dependent on parenteral nutrition (PN). The study found
that approximately 30% of patients who are dependent on PN have
cholestasis, despite the use of medical management in these
patients.
- The Company plans to use the
results from the completed retrospective study and ongoing
prospective study to inform next steps for the IV Choline Chloride
development program.
Corporate Update
- In October 2021, the Company hired
Mary Grendell as its new General Counsel. Ms. Grendell has an
extensive track record as a legal executive, most recently serving
as Vice President, Deputy General Counsel and Corporate Secretary,
at Intercept Pharmaceuticals. Previously, she held positions at
Mylan (now part of Viatris) and Amgen following her early legal
career at Sullivan & Cromwell and Covington & Burling. Ms.
Grendell received her J.D. from the University of Pennsylvania Law
School and her B.A. from Yale University.
Third Quarter 2021 Financial
Results
- As of September 30, 2021, cash,
cash equivalents and marketable debt securities totaled $138.4
million.
- Research and development expenses
for the third quarter of 2021 increased to $4.1 million from $2.8
million during the third quarter of 2020. The increased R&D
expenses were primarily due to increases in non-clinical, clinical
and regulatory expenses associated with TARA-002, headcount and
stock-based compensation, and other employee-related expenses.
- General and administrative expenses
for the third quarter of 2021 increased to $6.7 million from $5.3
million during the third quarter of 2020. The increase was
primarily due to increases in headcount and employee-related
expenses, development of commercial capabilities, and costs
associated with the new corporate office in New York, NY.
- For the third quarter of 2021,
Protara reported a net loss of $10.8 million, or $0.96 per share,
compared with a net loss of $8.0 million, or $1.26 per share, for
the third quarter of 2020. Net loss for the third quarter of 2021
included approximately $2.7 million of stock-based compensation
expenses.
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of NMIBC and LMs for which it has
been granted Rare Pediatric Disease Designation by the U.S.
Food and Drug Administration. TARA-002 was developed from the same
master cell bank of genetically distinct group A Streptococcus
pyogenes as OK-432, a broad immunopotentiator marketed as
Picibanil® in Japan and Taiwan by Chugai
Pharmaceutical Co., Ltd. Protara has successfully demonstrated
manufacturing comparability between TARA-002 and OK-432.
When TARA-002 is administered, it is
hypothesized that innate and adaptive immune cells within the cyst
or tumor are activated and produce a strong immune cascade.
Neutrophils, monocytes and lymphocytes infiltrate the abnormal
cells and various cytokines, including interleukins IL-6, IL-8,
IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha,
and vascular endothelial growth factor (VEGF) are secreted by
immune cells to induce a strong local inflammatory reaction and
destroy the abnormal cells.
About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the 6th most common cancer
in the United States, with NMIBC representing approximately
80% of bladder cancer diagnoses. Approximately 65,000 patients are
diagnosed with NMIBC in the United States each year.
NMIBC is cancer found in the tissue that lines the inner surface of
the bladder that has not spread into the bladder muscle.
About Lymphatic Malformations
LMs are rare, congenital malformations of
lymphatic vessels resulting in the failure of these structures to
connect or drain into the venous system. Most LMs are present in
the head and neck region and are diagnosed in early childhood
during the period of active lymphatic growth, with more than 50%
detected at birth and 90% diagnosed before the age of 3 years. The
most common morbidities and serious manifestations of the disease
include compression of the upper aerodigestive tract, including
airway obstruction requiring intubation and possible tracheostomy
dependence; intralesional bleeding; impingement on critical
structures, including nerves, vessels, lymphatics; recurrent
infection, and cosmetic and other functional disabilities.
About IV Choline Chloride and Intestinal
Failure-associated Liver Disease (IFALD)
IV Choline Chloride is an investigational,
intravenous (IV) phospholipid substrate replacement therapy
initially in development for patients receiving PN who have IFALD.
Choline is a known important substrate for phospholipids that are
critical for healthy liver function. Because PN patients cannot
sufficiently absorb adequate levels of choline and no available PN
formulations contain sufficient amounts of choline to correct this
deficiency, PN patients often experience a prolonged progression to
hepatic failure and death, with the only known intervention being a
dual small bowel/liver transplant. If approved, IV Choline Chloride
would be the first approved therapy for IFALD. It has been granted
Orphan Drug Designations (ODDs) by the FDA for the treatment of
IFALD and the prevention of choline deficiency in PN patients.
About Protara Therapeutics, Inc.
Protara is committed to identifying and
advancing transformative therapies for people with cancer and rare
diseases with limited treatment options. Protara’s portfolio
includes its lead program, TARA-002, an investigational cell-based
therapy being developed for the treatment of non-muscle invasive
bladder cancer and lymphatic malformations, and IV Choline
Chloride, an investigational phospholipid substrate replacement
therapy for the treatment of intestinal failure-associated liver
disease. For more information, visit www.protaratx.com.
Forward-Looking Statements
Statements contained in this press release
regarding matters that are not historical facts are "forward
looking statements" within the meaning of the Private Securities
Litigation Reform Act of 1995. Protara may, in some cases, use
terms such as “predicts,” “believes,” “potential,” “proposed,”
“continue,” “designed,” “estimates,” “anticipates,” “expects,”
“plans,” “intends,” “may,” “could,” “might,” “will,” “should” or
other words or expressions referencing future events, conditions or
circumstances that convey uncertainty of future events or outcomes
to identify these forward-looking statements. Such forward-looking
statements include but are not limited to, statements regarding
Protara’s intentions, beliefs, projections, outlook, analyses or
current expectations concerning, among other things: Protara’s
business strategy, including its development plans for its product
candidates and plans regarding the timing or outcome of existing or
future clinical trials; statements related to expectations
regarding interactions with the FDA, including anticipated
alignment with the FDA on clinical trial design for TARA-002 in
pediatric LM patients; Protara’s financial footing; statements
regarding the anticipated safety or efficacy of Protara’s product
candidates; and Protara’s outlook for the remainder of the year.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. Factors that contribute
to the uncertain nature of the forward-looking statements include:
risks that Protara’s financial guidance may not be as expected, as
well as risks and uncertainties associated with: Protara’s
development programs, including the initiation and completion of
non-clinical studies and clinical trials and the timing of required
filings with the FDA and other regulatory agencies; the impact of
the COVID-19 pandemic on Protara’s business and the global economy;
general market conditions; changes in the competitive landscape;
changes in Protara’s strategic and commercial plans; Protara’s
ability to obtain sufficient financing to fund its strategic plans
and commercialization efforts; having to use cash in ways or on
timing other than expected; the impact of market volatility on cash
reserves; the loss of key members of management; and the risks and
uncertainties associated with Protara’s business and financial
condition in general, including the risks and uncertainties
described more fully under the caption “Risk Factors” and elsewhere
in Protara's filings and reports with the United States Securities
and Exchange Commission. All forward-looking statements contained
in this press release speak only as of the date on which they were
made and are based on management's assumptions and estimates as of
such date. Protara undertakes no obligation to update any
forward-looking statements, whether as a result of the receipt of
new information, the occurrence of future events or otherwise,
except as required by law.
|
PROTARA THERAPEUTICS, INC. |
CONDENSED CONSOLIDATED BALANCE SHEETS |
(in thousands, except share and per share data) |
|
|
As of |
|
|
September 30, 2021 |
|
December 31, 2020 |
|
|
(unaudited) |
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
40,742 |
|
|
$ |
168,598 |
|
Restricted cash |
|
|
- |
|
|
|
50 |
|
Marketable debt securities, current |
|
|
53,016 |
|
|
|
- |
|
Prepaid expenses and other current assets |
|
|
1,937 |
|
|
|
787 |
|
Total current assets |
|
|
95,695 |
|
|
|
169,435 |
|
|
|
|
|
|
Non-current
assets: |
|
|
|
|
Restricted cash, non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities, non-current |
|
|
44,652 |
|
|
|
- |
|
Property and equipment, net |
|
|
1,729 |
|
|
|
1,240 |
|
Operating lease right-of-use asset |
|
|
7,413 |
|
|
|
1,060 |
|
Goodwill |
|
|
29,517 |
|
|
|
29,517 |
|
Other assets, non-current |
|
|
946 |
|
|
|
1,160 |
|
Total assets |
|
$ |
180,697 |
|
|
$ |
203,157 |
|
|
|
|
|
|
Liabilities and
Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
928 |
|
|
$ |
914 |
|
Accrued expenses |
|
|
1,920 |
|
|
|
1,913 |
|
Operating lease liability, current |
|
|
840 |
|
|
|
88 |
|
Total current liabilities |
|
|
3,688 |
|
|
|
2,915 |
|
|
|
|
|
|
Non-current
liabilities: |
|
|
|
|
Operating lease liability, non-current |
|
|
6,603 |
|
|
|
999 |
|
Total liabilities |
|
|
10,291 |
|
|
|
3,914 |
|
|
|
|
|
|
Commitments and
Contingencies (Note 6) |
|
|
|
|
|
|
|
|
|
Stockholders'
Equity |
|
|
|
|
Preferred Stock, $0.001 par value, authorized 10,000,000
shares: |
|
|
|
|
Series 1 Convertible Preferred Stock, 8,028 shares authorized at
September 30, 2021 and December 31, 2020, 8,027 shares issued and
outstanding as of September 30, 2021 and December 31, 2020. |
|
|
- |
|
|
|
- |
|
Common Stock, $0.001 par
value, authorized 100,000,000 shares: |
|
|
|
|
Common Stock, 11,235,731 and 11,211,840 shares issued and
outstanding as of September 30, 2021 and December 31, 2020,
respectively. |
|
|
11 |
|
|
|
11 |
|
Additional Paid in Capital |
|
|
254,218 |
|
|
|
245,992 |
|
Accumulated Deficit |
|
|
(83,784 |
) |
|
|
(46,760 |
) |
Accumulated Other Comprehensive Income (Loss) |
|
|
(39 |
) |
|
|
- |
|
Total Stockholders'
Equity |
|
|
170,406 |
|
|
|
199,243 |
|
Total Liabilities and Stockholders' Equity |
|
$ |
180,697 |
|
|
$ |
203,157 |
|
|
PROTARA THERAPEUTICS, INC. |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND
COMPREHENSIVE LOSS |
(unaudited) |
(in thousands, except share and per share data) |
|
|
For the three months ended September 30, |
|
For the nine months ended September 30, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
|
|
|
|
|
|
|
Operating expense: |
|
|
|
|
|
|
|
|
Research & development |
|
$ |
4,093 |
|
|
$ |
2,796 |
|
|
$ |
17,020 |
|
|
$ |
8,330 |
|
General & administrative |
|
|
6,737 |
|
|
|
5,266 |
|
|
|
20,182 |
|
|
|
17,157 |
|
Total operating expenses |
|
|
10,830 |
|
|
|
8,062 |
|
|
|
37,202 |
|
|
|
25,487 |
|
|
|
|
|
|
|
|
|
|
Operating loss |
|
|
(10,830 |
) |
|
|
(8,062 |
) |
|
|
(37,202 |
) |
|
|
(25,487 |
) |
|
|
|
|
|
|
|
|
|
Other income, net |
|
|
|
|
|
|
|
|
Interest income, net |
|
|
(53 |
) |
|
|
(92 |
) |
|
|
(178 |
) |
|
|
(317 |
) |
Total other income, net |
|
|
(53 |
) |
|
|
(92 |
) |
|
|
(178 |
) |
|
|
(317 |
) |
|
|
|
|
|
|
|
|
|
Net loss |
|
|
(10,777 |
) |
|
|
(7,970 |
) |
|
|
(37,024 |
) |
|
|
(25,170 |
) |
|
|
|
|
|
|
|
|
|
Other comprehensive gain
(loss): |
|
|
|
|
|
|
|
|
Unrealized gains (losses) on available-for-sale marketable debt
securities |
|
|
62 |
|
|
|
- |
|
|
|
(39 |
) |
|
|
- |
|
Total other comprehensive gain (loss) |
|
|
62 |
|
|
|
- |
|
|
|
(39 |
) |
|
|
- |
|
|
|
|
|
|
|
|
|
|
Comprehensive Loss |
|
$ |
(10,715 |
) |
|
$ |
(7,970 |
) |
|
$ |
(37,063 |
) |
|
$ |
(25,170 |
) |
|
|
|
|
|
|
|
|
|
Weighted Average Shares
Outstanding, basic and diluted |
|
|
11,235,507 |
|
|
|
6,324,295 |
|
|
|
11,231,513 |
|
|
|
5,910,849 |
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(0.96 |
) |
|
$ |
(1.26 |
) |
|
$ |
(3.30 |
) |
|
$ |
(4.26 |
) |
|
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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