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
fourth quarter and year ended December 31, 2021 and provided a
business update.
“Following a productive 2021, we are well poised
to advance our pipeline in 2022, in particular, we are excited to
have commenced our Phase 1 study of TARA-002 in non-muscle invasive
bladder cancer (NMIBC), a significant step forward in our mission
to bring a new immunotherapy to this patient population,” said
Jesse Shefferman, Chief Executive Officer of Protara Therapeutics.
“Discussions with the U.S. Food and Drug Administration (FDA)
remain ongoing 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. In addition, we continue to assess potential future
indications and combinations for TARA-002.”
Mr. Shefferman added, “Supported by a strong
balance sheet, which includes ample runway to support our planned
operations into mid-2024, we remain steadfast in our commitment to
bringing meaningful new therapeutic options to pressing areas of
high unmet need.”
Recent Highlights
TARA-002 in NMIBC
- In October 2021, the Company
announced that the FDA cleared its Investigational New Drug (IND)
application for TARA-002, an investigational cell-based therapy in
development for the treatment of NMIBC. A Phase 1 clinical trial
has commenced 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, current Good Manufacturing Practices
(cGMP) comparability data. The Company is engaged with the FDA to
align on a development plan for TARA-002 in LMs.
IV Choline Chloride in Intestinal Failure
Associated Liver Disease (IFALD)
- The Company’s prospective
prevalence study to enhance understanding of the incidence of IFALD
and choline deficiency in patients dependent on parenteral
nutrition remains ongoing. The Company plans to use results from
the prospective study, as well as its previously completed
retrospective study, to inform next steps for the IV Choline
Chloride development program.
Corporate Updates
- In January 2022, Protara announced
the appointment of Jathin Bandari, M.D., as Chief Medical Officer.
Dr. Bandari is a practicing urologic oncologist, recently serving
at the University of Rochester where he specializes in both
minimally invasive urologic oncology and advanced open pelvic
retroperitoneal cancer surgery, and where he maintains a faculty
appointment. Dr. Bandari joined Protara in April 2020 and most
recently was Vice President, Head of Clinical Development, and
Interim Chief Medical Officer.
Fourth Quarter and Full Year 2021
Financial Results
- As of December 31, 2021, cash, cash
equivalents and marketable debt securities totaled $130.7 million.
The Company expects its cash, cash equivalents, and marketable debt
securities will be sufficient to fund its planned operations into
mid-2024.
- Research and development expenses
for the fourth quarter of 2021 increased to $4.1 million from $3.7
million for the prior year period, and for the full year increased
to $21.1 million compared to $12.0 million for 2020. The fourth
quarter and full year increases 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 fourth quarter of 2021 increased to $6.2 million from $5.3
million for the prior year period, and for the full year increased
to $26.4 million compared to $22.5 million for 2020. The fourth
quarter and full year increases were due to increases in headcount
and employee-related expenses, market development capabilities, and
costs associated with the new office in New York.
- For the fourth quarter of 2021,
Protara reported a net loss of $10.2 million, or $0.91 per share,
compared with a net loss of $8.8 million, or $0.79 per share, for
the same period in 2020. Net loss for the year ended December 31,
2021 was $47.3 million, or $4.21 per share, compared with a net
loss of $34.0 million, or $4.70 per share, for the year ended
December 31, 2020. Net loss for the fourth quarter included
approximately $2.0 million of stock-based compensation expenses.
Net loss for the year ended December 31, 2021 included $10.4
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
(NMIBC)
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)
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 parenteral
nutrition (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 potential 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 as well as
the impact on Protara’s contract research organizations, study
sites or other clinical partners; 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; the impact of
general U.S. and foreign, economic, industry, market, regulatory or
political conditions; 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.
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PROTARA THERAPEUTICS, INC. |
CONSOLIDATED BALANCE SHEETS |
(in thousands, except share and per share data) |
|
|
As of |
|
|
December 31,2021 |
|
December 31,2020 |
|
|
|
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
35,724 |
|
|
$ |
168,598 |
|
Restricted cash |
|
|
- |
|
|
|
50 |
|
Marketable debt securities |
|
|
55,505 |
|
|
|
- |
|
Prepaid expenses and other current assets |
|
|
1,883 |
|
|
|
787 |
|
Total current assets |
|
|
93,112 |
|
|
|
169,435 |
|
Restricted cash, non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities, non-current |
|
|
39,467 |
|
|
|
- |
|
Property and equipment, net |
|
|
1,719 |
|
|
|
1,240 |
|
Operating lease right-of-use asset |
|
|
7,171 |
|
|
|
1,060 |
|
Goodwill |
|
|
29,517 |
|
|
|
29,517 |
|
Other assets |
|
|
865 |
|
|
|
1,160 |
|
Total assets |
|
$ |
172,596 |
|
|
$ |
203,157 |
|
|
|
|
|
|
Liabilities and
Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
954 |
|
|
$ |
914 |
|
Accrued expenses |
|
|
2,489 |
|
|
|
1,913 |
|
Operating lease liability, current |
|
|
855 |
|
|
|
88 |
|
Total current liabilities |
|
|
4,298 |
|
|
|
2,915 |
|
Operating lease liability, non-current |
|
|
6,384 |
|
|
|
999 |
|
Total liabilities |
|
|
10,682 |
|
|
|
3,914 |
|
|
|
|
|
|
Commitments and
Contingencies (Note 11) |
|
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Stockholders'
Equity |
|
|
|
|
Preferred stock, $0.001 par value, authorized 10,000,000
shares: |
|
|
|
|
Series 1 convertible preferred stock, 8,028 shares authorized at
December 31, 2021 and 2020, respectively 8,027 shares issued and
outstanding as of December 31, 2021 and 2020, respectively. |
|
|
- |
|
|
|
- |
|
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 December 31, 2021 and 2020, respectively. |
|
|
11 |
|
|
|
11 |
|
Additional paid in capital |
|
|
256,126 |
|
|
|
245,992 |
|
Accumulated deficit |
|
|
(94,012 |
) |
|
|
(46,760 |
) |
Accumulated other comprehensive income (loss) |
|
|
(211 |
) |
|
|
- |
|
Total stockholders' equity |
|
|
161,914 |
|
|
|
199,243 |
|
Total Liabilities and Stockholders' Equity |
|
$ |
172,596 |
|
|
$ |
203,157 |
|
|
|
|
|
|
|
|
|
|
PROTARA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE
LOSS |
(in thousands, except share and per share data) |
|
|
For the Years Ended December 31, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
Research and development |
|
$ |
21,088 |
|
|
$ |
11,982 |
|
General and administrative |
|
|
26,401 |
|
|
|
22,462 |
|
Total operating expenses |
|
|
47,489 |
|
|
|
34,444 |
|
Loss from operations |
|
|
(47,489 |
) |
|
|
(34,444 |
) |
|
|
|
|
|
Other income (expense),
net |
|
|
|
|
Interest and investment income |
|
|
237 |
|
|
|
500 |
|
Interest expense |
|
|
- |
|
|
|
(34 |
) |
Other income (expense), net |
|
|
237 |
|
|
|
466 |
|
|
|
|
|
|
Net loss |
|
|
(47,252 |
) |
|
|
(33,978 |
) |
|
|
|
|
|
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(4.21 |
) |
|
$ |
(4.70 |
) |
Weighted average shares
outstanding, basic and diluted |
|
|
11,232,576 |
|
|
|
7,233,913 |
|
Other comprehensive income
(loss): |
|
|
|
|
Net unrealized (loss) gain on marketable debt securities |
|
(211 |
) |
|
|
- |
|
Other comprehensive income (loss) |
|
|
(211 |
) |
|
|
- |
|
Comprehensive Loss |
|
$ |
(47,463 |
) |
|
$ |
(33,978 |
) |
|
|
|
|
|
|
|
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Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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