Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage
company developing transformative therapies for the treatment of
cancer and rare diseases with significant unmet needs, today
announced financial results for the second quarter ended June 30,
2021 and provided a business update.
“We continue to make significant progress
executing against our development plans for TARA-002 in non-muscle
invasive bladder cancer (NMIBC) and lymphatic malformations (LMs),
and we look forward to achieving multiple important milestones in
the second half of this year,” said Jesse
Shefferman, Chief Executive Officer
of Protara Therapeutics. “With the successful completion of
required non-clinical studies for TARA-002, we remain on track to
submit our Investigational New Drug (IND) application and initiate
our Phase 1 clinical study by year-end. In addition, our Good
Manufacturing Practice (GMP) scale up and comparability work remain
on track to be completed by year-end, and we look forward to
working with the U.S. Food and Drug Administration (FDA) on a
clinical trial of TARA-002 in patients with LMs, a rare pediatric
indication for which there are currently no U.S. FDA-approved
therapies.”
Recent Highlights and Upcoming
Milestones
TARA-002 Comparability
- The Company remains on track to
complete confirmatory, large-scale GMP manufacturing comparability
by year-end.
TARA-002 in NMIBC
- Protara successfully completed
required non-clinical IND-enabling studies to characterize local
toxicity of intravesical administration of TARA-002. The Company
remains on track to submit an IND application in the second half of
2021 and, subject to the FDA acceptance of the IND application, the
Company plans to commence a Phase 1 trial by the end of 2021 to
assess the safety and tolerability of TARA-002 in patients with
NMIBC, including patients with carcinoma in situ (CIS).
TARA-002 in LMs
- Following the completion of
confirmatory, large-scale GMP manufacturing comparability, the
Company plans to align with 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)
- The Company is currently executing
a prevalence study in partnership with a large home health
organization in the U.S. to enhance understanding of the
appropriate patient population and will use this information to
define the next steps for the development program.
Corporate Update
- In June 2021, the Company announced
the appointment of Jane Huang, M.D., to its Board of Directors. Dr.
Huang is an experienced biotech executive and proven leader
throughout the development lifecycle of multiple oncology
therapeutics globally and currently serves as Chief Medical
Officer, Hematology at BeiGene, Ltd.
Second Quarter 2021 Financial
Results
- As of June 30, 2021, cash, cash
equivalents and investments totaled $145 million.
- Research and development expenses
for the second quarter of 2021 increased to $5.9 million from $2.5
million during the second quarter of 2020. The increased R&D
expenses were primarily due to increases in manufacturing and
regulatory expenses associated with TARA-002.
- General and administrative expenses
for the second quarter of 2021 increased to $6.9 million from $4.8
million during the second quarter of 2020. The increase was
primarily due to increases in stock-based compensation, headcount,
and costs associated with the new Company headquarters in New York,
NY.
- For the second quarter of 2021,
Protara reported a net loss of $12.8 million, or $1.14 per share,
compared with a net loss of $7.1 million, or $1.22 per share, for
the second quarter of 2020. Net loss for the second quarter of 2021
included approximately $3.0 million of stock-based compensation
expenses.
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of non-muscle invasive bladder cancer
(NMIBC) and lymphatic malformations (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
initial 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 non-muscle invasive bladder cancer
(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
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 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 plans with respect to certain clinical trials, GMP
scale up and comparability work and anticipated timing, Protara’s
development plans for its product candidates and related
expectations regarding interactions or upcoming filings with the
FDA, including its planned IND filing for TARA-002 in NMIBC and
plans regarding the timing or outcome of existing or future
non-clinical studies and clinical trials, 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 sales, revenue, expense
and other 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 |
|
|
June 30, 2021 |
|
December 31, 2020 |
|
|
(unaudited) |
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
46,836 |
|
|
$ |
168,598 |
|
Restricted cash |
|
|
- |
|
|
|
50 |
|
Marketable debt securities, current |
|
|
37,830 |
|
|
|
- |
|
Prepaid expenses and other current assets |
|
|
3,153 |
|
|
|
787 |
|
Total current assets |
|
|
87,819 |
|
|
|
169,435 |
|
|
|
|
|
|
Non-current
assets: |
|
|
|
|
Restricted cash, non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities, non-current |
|
|
60,378 |
|
|
|
- |
|
Property and equipment, net |
|
|
1,769 |
|
|
|
1,240 |
|
Operating lease right-of-use asset |
|
|
7,648 |
|
|
|
1,060 |
|
Goodwill |
|
|
29,517 |
|
|
|
29,517 |
|
Other assets, non-current |
|
|
1,016 |
|
|
|
1,160 |
|
Total assets |
|
$ |
188,892 |
|
|
$ |
203,157 |
|
|
|
|
|
|
Liabilities and
Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
1,403 |
|
|
$ |
914 |
|
Accrued expenses |
|
|
1,439 |
|
|
|
1,913 |
|
Operating lease liability, current |
|
|
826 |
|
|
|
88 |
|
Total current liabilities |
|
|
3,668 |
|
|
|
2,915 |
|
|
|
|
|
|
Non-current
liabilities: |
|
|
|
|
Operating lease liability, non-current |
|
|
6,819 |
|
|
|
999 |
|
Total liabilities |
|
|
10,487 |
|
|
|
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
June 30, |
|
|
|
|
2021 and December 31, 2020, 8,027 shares issued and outstanding as
of |
|
|
|
|
June 30, 2021 and December 31, 2020. |
|
|
- |
|
|
|
- |
|
Common Stock, $0.001 par value, authorized 100,000,000 shares: |
|
|
|
|
Common Stock, 11,233,856 and 11,211,840 shares issued and |
|
|
|
|
outstanding as of June 30, 2021 and December 31, 2020,
respectively. |
|
|
11 |
|
|
|
11 |
|
Additional Paid in Capital |
|
|
251,502 |
|
|
|
245,992 |
|
Accumulated Deficit |
|
|
(73,007 |
) |
|
|
(46,760 |
) |
Accumulated Other Comprehensive Income (Loss) |
|
|
(101 |
) |
|
|
- |
|
Total Stockholders'
Equity |
|
|
178,405 |
|
|
|
199,243 |
|
Total Liabilities and
Stockholders' Equity |
|
$ |
188,892 |
|
|
$ |
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 June 30, |
|
For the six months ended June 30, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
|
|
|
|
|
|
|
Operating expense: |
|
|
|
|
|
|
|
|
Research & development |
|
$ |
5,887 |
|
|
$ |
2,470 |
|
|
$ |
12,927 |
|
|
$ |
5,534 |
|
General & administrative |
|
|
6,905 |
|
|
|
4,796 |
|
|
|
13,445 |
|
|
|
11,891 |
|
Total operating expenses |
|
|
12,792 |
|
|
|
7,266 |
|
|
|
26,372 |
|
|
|
17,425 |
|
|
|
|
|
|
|
|
|
|
Operating loss |
|
|
(12,792 |
) |
|
|
(7,266 |
) |
|
|
(26,372 |
) |
|
|
(17,425 |
) |
|
|
|
|
|
|
|
|
|
Other income, net |
|
|
|
|
|
|
|
|
Interest income, net |
|
|
(10 |
) |
|
|
(126 |
) |
|
|
(125 |
) |
|
|
(225 |
) |
Total other income, net |
|
|
(10 |
) |
|
|
(126 |
) |
|
|
(125 |
) |
|
|
(225 |
) |
|
|
|
|
|
|
|
|
|
Net loss |
|
|
(12,782 |
) |
|
|
(7,140 |
) |
|
|
(26,247 |
) |
|
|
(17,200 |
) |
|
|
|
|
|
|
|
|
|
Other comprehensive gain
(loss): |
|
|
|
|
|
|
|
|
Unrealized gains (losses) on available- |
|
|
|
|
|
|
|
|
for-sale marketable debt securities |
|
|
63 |
|
|
|
- |
|
|
|
(101 |
) |
|
|
- |
|
Total other comprehensive gain (loss) |
|
|
63 |
|
|
|
- |
|
|
|
(101 |
) |
|
|
- |
|
|
|
|
|
|
|
|
|
|
Comprehensive Loss |
|
$ |
(12,719 |
) |
|
$ |
(7,140 |
) |
|
$ |
(26,348 |
) |
|
$ |
(17,200 |
) |
|
|
|
|
|
|
|
|
|
Weighted Average Shares
Outstanding, basic and diluted |
|
|
11,232,010 |
|
|
|
5,843,203 |
|
|
|
11,229,484 |
|
|
|
5,701,855 |
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(1.14 |
) |
|
$ |
(1.22 |
) |
|
$ |
(2.34 |
) |
|
$ |
(3.02 |
) |
|
|
|
|
|
|
|
|
|
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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