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
first quarter ended March 31, 2023 and provided a business update.
“We are off to a strong start in 2023, notably
with the recent presentation of positive preliminary data from the
dose escalation portion of the ADVANCED-1 trial of TARA-002 in
patients with high-grade non-muscle invasive bladder cancer (NMIBC)
and expect to sustain this productive momentum in the months
ahead,” said Jesse Shefferman, Chief Executive Officer of Protara
Therapeutics. “Supported by these exciting preliminary findings, we
are keenly focused on continued execution across the NMIBC program,
including the recently announced Phase 1b/2 ADVANCED-2 trial of
TARA-002 in patients with high-grade carcinoma in situ (CIS), which
we expect to commence in the second half of this year. With final
regulatory clearance from the U.S. Food and Drug Administration
(FDA) on our Phase 2 trial in lymphatic malformations (LMs), we
look forward to initiating STARBORN-1 in the near future and
progressing our program to bring a much-needed therapy to these
pediatric patients who have no approved treatments today. We remain
committed to unlocking the full potential of TARA-002 and look
forward to sharing our progress throughout the coming
quarters.”
Recent Highlights
TARA-002 in NMIBC
- In April 2023 at the American
Urological Association Annual Meeting, the Company announced
positive preliminary results from the Phase 1a dose-escalation
component of the ADVANCED-1 clinical trial of TARA-002, its
investigational cell-based therapy, for the treatment of patients
with high-grade NMIBC. The clinical data indicate that TARA-002 was
generally well tolerated and anti-tumor activity was observed,
including tumor regression in all three evaluable patients with
CIS, including one heavily pre-treated Bacillus Calmette-Guérin
(BCG)-unresponsive patient who achieved a complete response.
- The Company plans to initiate
ADVANCED-2, a Phase 1b/2 open-label trial evaluating intravesical
TARA-002 in up to 102 patients with high-grade CIS. The Phase 1b
trial is expected to enroll 27 patients with CIS (± Ta/T1),
BCG-Naïve or BCG-experienced, who have not received intravesical
BCG for at least 24 months prior to CIS diagnosis. The Phase 2
trial is expected to enroll 75 patients with BCG-unresponsive CIS
(± Ta/T1). ADVANCED-2 is expected to initiate in the second half of
2023.
TARA-002 in Lymphatic Malformations (LMs)
- In April 2023, the Company received regulatory clearance from
the FDA to commence STARBORN-1, a Phase 2 clinical trial of
TARA-002 in pediatric patients with macrocystic and mixed-cystic
LMs. Trial start-up activities are well underway in the ten
pediatric centers of excellence participating in the trial, and
initiation is expected in the fourth quarter of 2023.
IV Choline Chloride in Intestinal Failure
Associated Liver Disease (IFALD)
- Protara’s prospective study to
enhance understanding of the incidence of IFALD in patients
dependent on parenteral nutrition is ongoing, with results expected
in the third quarter of 2023. The Company continues to engage with
the FDA and plans to use both regulatory feedback and results from
the prospective study to inform next steps for the IV Choline
Chloride development program.
First Quarter 2023 Financial
Results
- As of March 31, 2023, cash,
cash equivalents and restricted cash were $89.5 million. The
Company expects its current cash and cash equivalents will be
sufficient to fund its planned operations into 2025.
- Research and development (R&D)
expenses for the first quarter of 2023 decreased to $5.1
million from $5.3 million during the first quarter
of 2022.
- General and administrative expenses
for the first quarter of 2023 decreased to $4.6
million from $5.6 million for the prior year period.
This decrease was primarily due to decreases of $0.5 million in
employee related expenses (including $0.3 million of stock-based
compensation expense), $0.3 million resulting from a reduction in
directors and officers liability insurance premiums, as well as
$0.2 million related to a reduction in market development
activities.
- For the first quarter of 2023,
Protara reported a net loss of $9.0 million,
or $0.80 per share, compared with a net loss
of $10.8 million, or $0.96 per share, for the same period
in 2022. Net loss for the first quarter of 2023 included
approximately $1.6 million of stock-based compensation
expenses.
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of NMIBC and of 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 approved in Taiwan by Chugai Pharmaceutical
Co., Ltd. Protara has successfully shown 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-2, IL-6,
IL-8, IL-10, IL-12, interferon (IFN)-gamma, and tumor necrosis
factor (TNF)-alpha are secreted by immune cells to induce a strong
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 advancing transformative therapies for
people with cancer and rare diseases. 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 for patients
dependent on parenteral nutrition. 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; Protara’s financial position;
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; 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, political or public health 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. AND SUBSIDIARIES |
|
Condensed Consolidated Balance Sheets |
|
(in thousands, except share and per share data) |
|
|
|
|
As of |
|
|
March 31,2023 |
|
|
December 31, 2022 |
|
Assets |
(unaudited) |
|
|
|
|
Current assets: |
|
|
|
|
|
Cash and cash equivalents |
$ |
21,035 |
|
|
$ |
24,127 |
|
Marketable debt securities |
|
65,575 |
|
|
|
60,243 |
|
Prepaid expenses and other current assets |
|
8,006 |
|
|
|
1,776 |
|
Total current assets |
|
94,616 |
|
|
|
86,146 |
|
Restricted cash,
non-current |
|
745 |
|
|
|
745 |
|
Marketable debt securities,
non-current |
|
2,893 |
|
|
|
17,886 |
|
Property and equipment,
net |
|
1,521 |
|
|
|
1,592 |
|
Operating lease right-of-use
asset |
|
6,044 |
|
|
|
6,277 |
|
Other assets |
|
586 |
|
|
|
644 |
|
Total assets |
$ |
106,405 |
|
|
$ |
113,290 |
|
|
|
|
|
|
|
|
|
Liabilities and Stockholders’ Equity |
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
Accounts payable |
$ |
4,081 |
|
|
$ |
1,586 |
|
Accrued expenses |
|
1,396 |
|
|
|
3,237 |
|
Operating lease liability |
|
933 |
|
|
|
917 |
|
Total current liabilities |
|
6,410 |
|
|
|
5,740 |
|
Operating lease liability,
non-current |
|
5,227 |
|
|
|
5,467 |
|
Total liabilities |
|
11,637 |
|
|
|
11,207 |
|
Commitments and contingencies (Note 8) |
|
|
|
|
|
|
|
Stockholders’ Equity: |
|
|
|
|
|
|
|
Preferred stock, $0.001 par value, authorized 10,000,000 shares:
Series 1 Convertible Preferred Stock, 8,028 shares authorized at
March 31, 2023 and December 31, 2022, 8,027 shares issued and
outstanding as of March 31, 2023 and December 31, 2022. |
|
- |
|
|
|
- |
|
Common stock, $0.001 par value, authorized 100,000,000 shares:
Common stock, 11,306,753 and 11,267,389 shares issued and
outstanding as of March 31, 2023 and December 31, 2022,
respectively. |
|
11 |
|
|
|
11 |
|
Additional paid-in capital |
|
264,235 |
|
|
|
262,724 |
|
Accumulated deficit |
|
(169,009 |
) |
|
|
(159,964 |
) |
Accumulated other comprehensive income (loss) |
|
(469 |
) |
|
|
(688 |
) |
Total stockholders’ equity |
|
94,768 |
|
|
|
102,083 |
|
Total liabilities and stockholders’ equity |
$ |
106,405 |
|
|
$ |
113,290 |
|
|
|
|
|
|
|
|
|
PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES |
|
Unaudited Condensed Consolidated Statements of Operations
and Comprehensive Loss |
|
(in thousands, except share and per share data) |
|
|
|
|
For the Three Months Ended March
31, |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
Research and development |
$ |
5,143 |
|
|
$ |
5,269 |
|
General and administrative |
|
4,589 |
|
|
|
5,605 |
|
Total operating expenses |
|
9,732 |
|
|
|
10,874 |
|
Loss from operations |
|
(9,732 |
) |
|
|
(10,874 |
) |
Other income (expense), net: |
|
|
|
|
|
|
Interest and investment income |
|
687 |
|
|
|
119 |
|
Other income (expense), net |
|
687 |
|
|
|
119 |
|
Net loss |
|
(9,045 |
) |
|
|
(10,755 |
) |
|
|
|
|
|
|
|
Net loss per share attributable to common stockholders, basic and
diluted |
$ |
(0.80 |
) |
|
$ |
(0.96 |
) |
Weighted-average shares outstanding, basic and diluted |
|
11,303,869 |
|
|
|
11,250,127 |
|
Other comprehensive income (loss): |
|
|
|
|
|
|
Net unrealized (loss) gain on marketable debt securities |
|
219 |
|
|
|
(731 |
) |
Other comprehensive income (loss) |
|
219 |
|
|
|
(731 |
) |
Comprehensive Loss |
$ |
(8,826 |
) |
|
$ |
(11,486 |
) |
|
|
|
|
|
|
|
|
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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