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 third quarter
ended September 30, 2020.
“The third quarter marked a highly productive
time for the Company, notably with the Food and Drug
Administration’s (FDA) confirmation of initial comparability
between TARA-002 and OK-432, and separately the Company’s
identification of an acceptable development path for TARA-002 in
non-muscle invasive bladder cancer (NMIBC), an oncology indication
with high unmet need,” said Jesse Shefferman, Chief Executive
Officer of Protara Therapeutics. “We believe TARA-002 has the
potential to serve as a much needed intervention for NMIBC patients
lacking alternative therapeutic options, and we look forward to the
expected commencement of our clinical program in NMIBC in 2021.
Finally, we have requested a meeting with the FDA Division of
Vaccines and Related Products, the review division which the
Investigational New Drug (IND) application was initially opened by
the University of Iowa, to discuss a potential Biological License
Application (BLA) filing for TARA-002 in lymphatic malformations
(LMs).”
Recent Highlights
TARA-002
In September 2020, Protara announced the
following updates for the TARA-002 program:
- FDA Confirmation
of Initial Comparability
Between TARA-002 and OK-432. Following a pre-IND
engagement with the Office of Tissues and Advanced
Therapies (OTAT) division of the Center for Biologics
Evaluation and Research (CBER), the FDA agreed that Protara
has successfully demonstrated initial manufacturing comparability
between TARA-002 and OK-432 and that the in-process and release
protocols employed by Protara to demonstrate initial comparability
are appropriate to utilize for GMP-Scale comparability testing.
Good Manufacturing Practice (GMP) scale-up is currently in process
and the Company expects to complete three such large-scale batch
runs to confirm comparability in mid-2021.
- Clinical Development Path
in NMIBC. The Company reached alignment with the FDA on a
proposed clinical development plan to evaluate TARA-002 in patients
with NMIBC. Advancement into the clinic will be supported by
existing and ongoing non-clinical studies as well as historical
clinical safety and efficacy data for OK-432. Subject to the
successful completion of select non-clinical studies to
characterize local toxicity of intravesical administration of
TARA-002, as well as acceptance of an IND filing, the Company plans
to commence a Phase 1 study in 2021 to assess the safety and
tolerability of TARA-002 in patients with NMIBC, including patients
with carcinoma in situ (CIS), with results expected in 2022.
- Regulatory Path in
LMs. Protara has requested a meeting with the FDA Division
of Vaccines and Related Products Applications to discuss the
regulatory path for TARA-002 in LMs. The Company plans to utilize
the robust dataset for OK-432 in LMs to support a BLA filing for
TARA-002 in LMs.
IV Choline Chloride
- Prevalence Study
Underway to Assess
Incidence of Liver Disease in Patients Dependent on
Parenteral Nutrition
(PN). The Company recently
commenced a prevalence study to assess the incidence of liver
disease in patients dependent on PN in the home care setting. The
Company believes the study will enhance its understanding of the
population of patients who may potentially benefit from IV Choline
Chloride, its investigational phospholipid substrate replacement
therapy currently in development for the treatment of patients
receiving PN who have intestinal failure-associated liver disease
(IFALD).
Corporate Update
- Raised
$151
Million in Concurrent Public
Offerings. Protara recently announced the closing of
concurrent but separate underwritten public offerings of 4,600,000
shares of its common stock, at a public offering price of $16.87
per share, and 4,148 shares of non-voting Series 1 Convertible
Preferred Stock, at a public offering price of $16,873.54 per
share. In addition, the underwriters exercised their overallotment
option in full to purchase an additional 690,000 shares of common
stock at a public offering price of $16.87. Aggregate net proceeds
to Protara were approximately $151 million, after deducting
underwriting discounts and offering expenses.
Third Quarter 2020
Results from Operations
- As of September 30, 2020, cash
and restricted cash were $166.0 million.
- Protara reported a net loss
of $8.0 million for the third quarter of 2020 as compared
to a net loss of $2.4 million for the three months
ended September 30, 2019. The third quarter of 2020 included
approximately $2.8 million of stock-based compensation
expense.
- Research and Development expenses
were $2.8 million for the third quarter of 2020, an
increase of $1.7 million as compared to the three months
ended September 30, 2019. The increase was primarily due to an
increase of (i) $0.6 million in personnel and related costs, (ii)
$0.8 million in chemistry manufacturing and controls (CMC) expenses
and (iii) $0.3 million in regulatory expenses.
- General and Administrative expenses
were $5.3 million for the third quarter of 2020, which
represented an increase of $4.0 million as compared to
the three months ended September 30, 2019. The increase
was primarily related to an increase of (i) $2.6 million in
stock-based compensation expense, (ii) $0.6 million in insurance
expense and (iii) $0.7 million in personnel and related costs.
A Form 10-Q containing the full financial
statements was filed this morning and is available for viewing on
Protara’s website
at www.protaratx.com or www.sec.gov.
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of lymphatic malformations (LMs) and
non-muscle invasive bladder cancer (NMIBC). 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 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. TARA-002 has been granted Rare
Pediatric Disease Designation by the U.S. Food and Drug
Administration for the LMs indication.
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. The current standard of care for high-grade NMIBC
includes intravesical Bacillus Calmette-Guerin (BCG), which has
been the subject of multiple global supply shortages in the past
decade.
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 2 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, Protara’s development plans for its product
candidates , including its plans regarding the timing or outcome of
existing or future non-clinical studies and clinical trials,
Protara’s expectations regarding interactions or upcoming filings
with the FDA, statements regarding the anticipated safety or
efficacy of Protara’s product candidates and, Protara’s financial
footing. 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.
Company Contact:
Blaine DavisProtara
TherapeuticsBlaine.Davis@protaratx.com646-844-0337
PROTARA THERAPEUTICS, INC. |
|
CONDENSED
CONSOLIDATED BALANCE SHEETS |
|
|
|
|
|
|
|
|
|
As of |
|
|
|
September 30, 2020 |
|
December 31, 2019 |
|
|
|
(unaudited) |
|
|
|
Assets |
|
|
|
|
|
Current assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
165,904,797 |
|
|
$ |
564,124 |
|
|
Restricted cash |
|
|
50,000 |
|
|
|
- |
|
|
Deferred offering costs |
|
|
- |
|
|
|
121,712 |
|
|
Prepaid expenses and other current assets |
|
|
1,160,257 |
|
|
|
78,057 |
|
|
Total current assets |
|
|
167,115,054 |
|
|
|
763,893 |
|
|
|
|
|
|
|
|
Non-current assets: |
|
|
|
|
|
Property and equipment, net |
|
|
760,548 |
|
|
|
458,591 |
|
|
Goodwill |
|
|
29,367,213 |
|
|
|
- |
|
|
Other assets |
|
|
1,664,442 |
|
|
|
- |
|
|
Total assets |
|
$ |
198,907,257 |
|
|
$ |
1,222,484 |
|
|
|
|
|
|
|
|
Liabilities and Stockholders' Equity
(Deficit) |
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
1,912,553 |
|
|
$ |
715,653 |
|
|
Accrued expenses |
|
|
1,569,348 |
|
|
|
2,634,790 |
|
|
Short-term debt |
|
|
370,793 |
|
|
|
- |
|
|
Right-of-use liability, current |
|
|
34,079 |
|
|
|
- |
|
|
Total current
liabilities |
|
|
3,886,773 |
|
|
|
3,350,443 |
|
|
|
|
|
|
|
|
Non-current liabilities: |
|
|
|
|
|
Right-of-use liability, long-term |
|
|
394,721 |
|
|
|
- |
|
|
Total
liabilities |
|
|
4,281,494 |
|
|
|
3,350,443 |
|
|
|
|
|
|
|
|
Commitments and Contingencies (Note 6) |
|
|
|
|
|
|
|
|
|
|
|
Stockholders' Equity (Deficit) |
|
|
|
|
|
Preferred Stock, $0.001 par value, authorized 10,000,000
shares: |
|
|
|
|
|
Series 1 Convertible Preferred Stock, 8,028 and 0 shares authorized
at September 30, 2020 |
|
|
|
|
|
and December 31, 2019, respectively, 8,027 and 0 shares issued and
outstanding as of |
|
|
|
|
|
September 30, 2020 and December 31, 2019, respectively. |
|
|
8 |
|
|
|
- |
|
|
Common Stock, $0.001 par value, authorized 100,000,000 shares: |
|
|
|
|
|
Common Stock, 10,521,840 and 2,627,533 common shares issued and
outstanding as of |
|
|
|
|
|
September 30, 2020 and December 31, 2019, respectively. |
|
|
10,522 |
|
|
|
2,628 |
|
|
Additional Paid in Capital |
|
|
232,567,265 |
|
|
|
10,651,073 |
|
|
Accumulated Deficit |
|
|
(37,952,032 |
) |
|
|
(12,781,660 |
) |
|
Total
Stockholders' Equity (Deficit) |
|
|
194,625,763 |
|
|
|
(2,127,959 |
) |
|
Total Liabilities and
Stockholders' Equity (Deficit) |
|
$ |
198,907,257 |
|
|
$ |
1,222,484 |
|
|
|
|
|
|
|
|
PROTARA
THERAPEUTICS, INC. |
|
CONDENSED
CONSOLIDATED STATEMENTS OF OPERATIONS |
|
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
For the Three Months Ended September 30, |
|
For the Nine Months Ended September 30, |
|
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
|
|
|
|
|
|
Operating
expense: |
|
|
|
|
|
|
|
|
|
Research & development |
|
$ |
2,796,214 |
|
|
$ |
1,098,617 |
|
|
$ |
8,330,727 |
|
|
$ |
3,163,179 |
|
|
General & administrative |
|
|
5,265,965 |
|
|
|
1,255,466 |
|
|
|
17,156,952 |
|
|
|
2,147,635 |
|
|
Total operating expenses |
|
|
8,062,179 |
|
|
|
2,354,083 |
|
|
|
25,487,679 |
|
|
|
5,310,814 |
|
|
|
|
|
|
|
|
|
|
|
|
Operating loss |
|
|
(8,062,179 |
) |
|
|
(2,354,083 |
) |
|
|
(25,487,679 |
) |
|
|
(5,310,814 |
) |
|
|
|
|
|
|
|
|
|
|
|
Other
income, net |
|
|
|
|
|
|
|
|
|
Interest income, net |
|
|
(92,094 |
) |
|
|
- |
|
|
|
(317,307 |
) |
|
|
- |
|
|
Total other income, net |
|
|
(92,094 |
) |
|
|
- |
|
|
|
(317,307 |
) |
|
|
- |
|
|
|
|
|
|
|
|
|
|
|
|
Net
Loss |
|
$ |
(7,970,085 |
) |
|
$ |
(2,354,083 |
) |
|
$ |
(25,170,372 |
) |
|
$ |
(5,310,814 |
) |
|
|
|
|
|
|
|
|
|
|
|
Weighted Average Shares Outstanding, basic and diluted |
|
6,324,295 |
|
|
|
2,564,429 |
|
|
|
5,910,849 |
|
|
|
2,560,444 |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per
share, basic and diluted |
|
$ |
(1.26 |
) |
|
$ |
(0.92 |
) |
|
$ |
(4.26 |
) |
|
$ |
(2.07 |
) |
|
|
|
|
|
|
|
|
|
|
|
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