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
second quarter ended June 30, 2023 and provided a business update.
“Following the 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), we have seen rapid enrollment in the expansion portion of
the trial and anticipate sharing preliminary results in the first
half of 2024,” said Jesse Shefferman, Chief Executive Officer of
Protara Therapeutics. “We are building on this momentum, and plan
to initiate two additional trials for TARA-002 later this year
while continuing to maintain a disciplined approach to investments.
We are in a solid financial position with cash runway into
2025.”
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
carcinoma in situ (CIS), including one heavily pre-treated Bacillus
Calmette-Guérin (BCG)-unresponsive patient who achieved a complete
response (CR).
- In May 2023, the Company dosed the
first patients in its Phase 1b ADVANCED-1EXP study, an open-label
expansion trial evaluating intravesical TARA-002 at the
40KE1 dose in 12 CIS patients, including BCG-naïve,
BCG-unresponsive, and BCG-inadequately treated patients. Dosing
continues to progress in the trial, with preliminary results
expected in the first half of 2024. The primary endpoint of the
trial is the CR rate at three months.
- In the fourth quarter of 2023, 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, including 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, and 75 patients
with BCG-unresponsive CIS (± Ta/T1).
TARA-002 in Lymphatic Malformations (LMs)
- In April 2023, the Company received
regulatory clearance from the U.S. Food and Drug Administration
(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 at the ten pediatric centers
of excellence participating in the trial, and initiation is
expected in the fourth quarter of 2023.
IV Choline Chloride Program
- Protara is concluding its
prospective prevalence study to enhance understanding of the
incidence of choline deficiency in patients dependent on parenteral
nutrition. 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.
Second Quarter 2023 Financial
Results
- As of June 30, 2023, cash,
cash equivalents and restricted cash were $80.4 million. The
Company expects its current cash and cash equivalents will be
sufficient to fund its planned operations into 2025.
- Research and development expenses
for the second quarter of 2023 increased to $7.2
million from $3.1 million during the second quarter
of 2022, primarily reflecting an increase in expenses related to
clinical and non-clinical trial activities for TARA-002.
- General and administrative expenses
for the second quarter of 2023 decreased to $4.9
million from $5.6 million for the prior year period,
primarily due to lower employee related expenses.
- For the second quarter of 2023,
Protara reported a net loss of $11.3 million,
or $1.00 per share, compared with a net loss of $8.5
million, or $0.76 per share, for the same period in 2022. Net
loss for the second 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
IV Choline Chloride is an investigational,
intravenous (IV) phospholipid substrate replacement therapy
initially in development for patients receiving parenteral
nutrition (PN). 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. IV
Choline Chloride has been granted Orphan Drug Designation by the
FDA for 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.
References
1. Klinische Einheit, or KE, is a German term indicating a
specified weight of dried cells in a vial.
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.
PROTARA THERAPEUTICS, INC. AND
SUBSIDIARIESCondensed Consolidated Balance
Sheets(in thousands, except share and per share data) |
|
|
|
|
As of |
|
|
|
June 30,2023 |
|
|
December 31, 2022 |
|
|
|
(unaudited) |
|
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
33,978 |
|
|
$ |
24,127 |
|
Marketable debt securities |
|
|
46,382 |
|
|
|
60,243 |
|
Prepaid expenses and other current assets |
|
|
3,905 |
|
|
|
1,776 |
|
Total current assets |
|
|
84,265 |
|
|
|
86,146 |
|
Restricted cash,
non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities,
non-current |
|
|
- |
|
|
|
17,886 |
|
Property and equipment,
net |
|
|
1,443 |
|
|
|
1,592 |
|
Operating lease right-of-use
asset |
|
|
5,807 |
|
|
|
6,277 |
|
Other assets |
|
|
2,881 |
|
|
|
644 |
|
Total assets |
|
$ |
95,141 |
|
|
$ |
113,290 |
|
|
|
|
|
|
|
|
|
|
Liabilities and Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
1,852 |
|
|
$ |
1,586 |
|
Accrued expenses |
|
|
2,131 |
|
|
|
3,237 |
|
Operating lease liability |
|
|
949 |
|
|
|
917 |
|
Total current liabilities |
|
|
4,932 |
|
|
|
5,740 |
|
Operating lease liability,
non-current |
|
|
4,984 |
|
|
|
5,467 |
|
Total liabilities |
|
|
9,916 |
|
|
|
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
June 30, 2023 and December 31, 2022, 8,027 shares issued and
outstanding as of June 30, 2023 and December 31, 2022. |
|
|
- |
|
|
|
- |
|
Common stock, $0.001 par value, authorized 100,000,000 shares:
Common stock, 11,307,962 and 11,267,389 shares issued and
outstanding as of June 30, 2023 and December 31, 2022,
respectively. |
|
|
11 |
|
|
|
11 |
|
Additional paid-in capital |
|
|
265,853 |
|
|
|
262,724 |
|
Accumulated deficit |
|
|
(180,303 |
) |
|
|
(159,964 |
) |
Accumulated other comprehensive income (loss) |
|
|
(336 |
) |
|
|
(688 |
) |
Total stockholders’ equity |
|
|
85,225 |
|
|
|
102,083 |
|
Total liabilities and stockholders’ equity |
|
$ |
95,141 |
|
|
$ |
113,290 |
|
PROTARA THERAPEUTICS, INC. AND
SUBSIDIARIESUnaudited Condensed Consolidated
Statements of Operations and Comprehensive Loss(in
thousands, except share and per share data) |
|
|
|
For the Three Months EndedJune
30, |
|
|
For the Six Months Ended June
30, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
7,247 |
|
|
$ |
3,084 |
|
|
$ |
12,390 |
|
|
$ |
8,353 |
|
General and administrative |
|
|
4,893 |
|
|
|
5,621 |
|
|
|
9,482 |
|
|
|
11,226 |
|
Total operating expenses |
|
|
12,140 |
|
|
|
8,705 |
|
|
|
21,872 |
|
|
|
19,579 |
|
Loss from operations |
|
|
(12,140 |
) |
|
|
(8,705 |
) |
|
|
(21,872 |
) |
|
|
(19,579 |
) |
Other income (expense), net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest and investment income |
|
|
846 |
|
|
|
166 |
|
|
|
1,533 |
|
|
|
285 |
|
Other income (expense), net |
|
|
846 |
|
|
|
166 |
|
|
|
1,533 |
|
|
|
285 |
|
Net loss |
|
$ |
(11,294 |
) |
|
$ |
(8,539 |
) |
|
$ |
(20,339 |
) |
|
$ |
(19,294 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share attributable to common stockholders, basic and
diluted |
|
$ |
(1.00 |
) |
|
$ |
(0.76 |
) |
|
$ |
(1.80 |
) |
|
$ |
(1.71 |
) |
Weighted-average shares outstanding, basic and diluted |
|
|
11,307,842 |
|
|
|
11,255,215 |
|
|
|
11,305,867 |
|
|
|
11,252,686 |
|
Other comprehensive income (loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net unrealized (loss) gain on marketable debt securities |
|
|
133 |
|
|
|
(180 |
) |
|
|
352 |
|
|
|
(911 |
) |
Other comprehensive income (loss) |
|
|
133 |
|
|
|
(180 |
) |
|
|
352 |
|
|
|
(911 |
) |
Comprehensive Loss |
|
$ |
(11,161 |
) |
|
$ |
(8,719 |
) |
|
$ |
(19,987 |
) |
|
$ |
(20,205 |
) |
Company Contact:Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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