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 fourth quarter and year ended
December 31, 2020 and provided a business update.
“We believe 2021 will be a transformative year
for Protara, and we are entering it with strong momentum,” said
Jesse Shefferman, Chief Executive Officer of Protara
Therapeutics. “We remain on track to commence a Phase 1 study of
TARA-002 in patients with non-muscle invasive bladder cancer
(NMIBC), a pressing area of unmet need, by the end of the year. We
believe that TARA-002 has the opportunity to play a meaningful role
in the current NMIBC treatment landscape.”
Mr. Shefferman continued, “We are in discussions
with the U.S. Food and Drug Administration’s (FDA) Division of
Vaccines and Related Products to establish a path forward to file
our Biological License Application (BLA) for TARA-002 in lymphatic
malformations (LMs). We are encouraged by the progress to date and,
at the FDA’s request, have submitted the full Clinical Study Report
(CSR) of a randomized Phase 2 study of OK-432 (the originator
compound of TARA-002) in LMs led by the University of Iowa. We look
forward to continuing our dialogue with the FDA.”
Recent Highlights and Upcoming
Milestones
TARA-002 in NMIBC
- Protara remains on track to
complete select non-clinical studies to characterize local toxicity
of intravesical administration of TARA-002 in the first half of
2021, with an Investigational New Drug (IND) application submission
anticipated in the second half of 2021. Subject to FDA acceptance
of the IND application, the Company plans to commence a Phase 1
study 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
- Protara plans to utilize the robust
dataset for OK-432 (the originator compound of TARA-002) in LMs to
support a potential filing. In connection with Protara’s request to
discuss a potential BLA submission for TARA-002 in LMs, the FDA
Division of Vaccines and Related Products has requested a CSR
summarizing the totality of a randomized Phase 2 study of OK-432 in
LMs led by the University of Iowa. The Company has submitted the
CSR to the FDA and continues to prepare for a potential BLA filing
in the second half of 2021, or to initiate additional clinical work
as required.
IV Choline Chloride in intestinal failure
associated liver disease (IFALD)
- Following a successful meeting with
the FDA in 2020 regarding the registration package for IV Choline
Chloride, the Company is currently undertaking 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 February 2021, the Company
announced the appointment of Cynthia Smith to its Board of
Directors. Ms. Smith brings to Protara over 20 years of diverse
leadership experience within the healthcare industry, most recently
serving as Chief Commercial Officer at ZS Pharma.
Fourth Quarter and Full Year 2020
Financial Results
- As of December 31, 2020, cash, cash
equivalents and restricted cash were $169.4 million.
- Research and development expenses
for the fourth quarter of 2020 increased to $3.7 million from $0.7
million for the prior year period, and for the full year increased
to $12.0 million compared to $3.9 million for 2019. The fourth
quarter and full year increases were primarily due to increases in
personnel and related costs, manufacturing and regulatory expenses
as the company advanced its clinical programs supporting
TARA-002.
- General and administrative expenses
for the fourth quarter of 2020 increased to $5.3 million from $1.8
million for the prior year period, and for the full year increased
to $22.5 million compared to $4.0 million for 2019. The fourth
quarter and full year increases were primarily due to increases in
stock-based compensation expense, insurance expense and personnel
and related costs supporting the company’s growth.
- For the fourth quarter of 2020,
Protara reported a net loss of $8.8 million, or $0.79 per share,
compared with a net loss of $2.5 million, or $0.96 per share, for
the same period in 2019. Net loss for the year ended December 31,
2020 was $34.0 million, or $4.70 per share, compared with a net
loss of $7.8 million, or $3.04 per share, for the year ended
December 31, 2019. Net loss for the fourth quarter included
approximately $2.3 million of stock-based compensation expenses.
Net loss for the year ended December 31, 2020 included $9.7 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 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. 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 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, Protara’s development plans for its product
candidates and related expectations regarding interactions or
upcoming filings with the FDA, including its plans regarding the
timing or outcome of existing or future non-clinical studies and
clinical trials, Protara’s financial footing, the impact of the
COVID-19 pandemic and related governmental responses on Protara’s
business and clinical programs, 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. |
CONSOLIDATED BALANCE SHEETS |
(in thousands, except share and per share data) |
|
|
As of December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
168,598 |
|
|
$ |
564 |
|
Restricted cash |
|
|
50 |
|
|
|
- |
|
Deferred offering costs |
|
|
- |
|
|
|
122 |
|
Prepaid expenses and other current assets |
|
|
787 |
|
|
|
78 |
|
Total current assets |
|
|
169,435 |
|
|
|
764 |
|
|
|
|
|
|
Non-current
assets: |
|
|
|
|
Restricted cash, long-term |
|
|
745 |
|
|
|
- |
|
Property and equipment, net |
|
|
1,240 |
|
|
|
459 |
|
Goodwill |
|
|
29,517 |
|
|
|
- |
|
Other assets |
|
|
2,220 |
|
|
|
- |
|
Total assets |
|
$ |
203,157 |
|
|
$ |
1,223 |
|
|
|
|
|
|
Liabilities and
Stockholders' Equity (Deficit) |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
914 |
|
|
$ |
716 |
|
Accrued expenses |
|
|
1,913 |
|
|
|
2,635 |
|
Operating lease liability, current |
|
|
88 |
|
|
|
- |
|
Total current liabilities |
|
|
2,915 |
|
|
|
3,351 |
|
|
|
|
|
|
Non-current
liabilities: |
|
|
|
|
Operating lease liability, long-term |
|
|
999 |
|
|
|
- |
|
Total liabilities |
|
|
3,914 |
|
|
|
3,351 |
|
|
|
|
|
|
Commitments and
Contingencies (Note 7) |
|
|
|
|
|
|
|
|
|
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 December 31, 2020 and 2019, respectively, 8,027 and 0
shares issued and outstanding as of December 31, 2020 and
2019, respectively. |
|
|
- |
|
|
|
- |
|
Common Stock, $0.001 par value, authorized 100,000,000 shares: |
|
|
|
|
Common Stock, 11,211,840 and 2,627,533 shares issued and
outstanding as of December 31, 2020 and 2019,
respectively. |
|
|
11 |
|
|
|
3 |
|
Additional Paid in Capital |
|
|
245,992 |
|
|
|
10,651 |
|
Accumulated Deficit |
|
|
(46,760 |
) |
|
|
(12,782 |
) |
Total Stockholders' Equity
(Deficit) |
|
|
199,243 |
|
|
|
(2,128 |
) |
Total Liabilities and Stockholders' Equity
(Deficit) |
|
$ |
203,157 |
|
|
$ |
1,223 |
|
|
|
|
|
|
PROTARA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS |
(in thousands, except share and per share data) |
|
|
|
|
|
|
|
For the Years Ended December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
Operating expense: |
|
|
|
|
Research & development |
|
$ |
11,982 |
|
|
$ |
3,878 |
|
General & administrative |
|
|
22,462 |
|
|
|
3,952 |
|
Total operating expenses |
|
|
34,444 |
|
|
|
7,830 |
|
|
|
|
|
|
Operating loss |
|
|
(34,444 |
) |
|
|
(7,830 |
) |
|
|
|
|
|
Other income, net |
|
|
|
|
Interest income, net |
|
|
(466 |
) |
|
|
- |
|
Total other income, net |
|
|
(466 |
) |
|
|
- |
|
|
|
|
|
|
Net Loss |
|
$ |
(33,978 |
) |
|
$ |
(7,830 |
) |
|
|
|
|
|
Weighted Average Shares
Outstanding, basic and diluted |
|
|
7,233,913 |
|
|
|
2,577,493 |
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(4.70 |
) |
|
$ |
(3.04 |
) |
|
|
|
|
|
PROTARA THERAPEUTICS, INC. |
CONSOLIDATED STATEMENTS OF OPERATIONS |
(in thousands, except share and per share data) |
(unaudited) |
|
|
|
|
|
|
|
For the Three Months Ended December 31, |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
Operating expense: |
|
|
|
|
Research & development |
|
$ |
3,651 |
|
|
$ |
715 |
|
General & administrative |
|
|
5,305 |
|
|
|
1,804 |
|
Total operating expenses |
|
|
8,956 |
|
|
|
2,519 |
|
|
|
|
|
|
Operating loss |
|
|
(8,956 |
) |
|
|
(2,519 |
) |
|
|
|
|
|
Other income, net |
|
|
|
|
Interest income, net |
|
|
(148 |
) |
|
|
- |
|
Total other income, net |
|
|
(148 |
) |
|
|
- |
|
|
|
|
|
|
Net Loss |
|
$ |
(8,808 |
) |
|
$ |
(2,519 |
) |
|
|
|
|
|
Weighted Average Shares
Outstanding, basic and diluted |
|
|
11,174,340 |
|
|
|
2,627,533 |
|
|
|
|
|
|
Net loss per share, basic and
diluted |
|
$ |
(0.79 |
) |
|
$ |
(0.96 |
) |
|
|
|
|
|
|
|
|
|
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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