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 third quarter ended September 30, 2023 and provided
a business update.
“Progress continues across our TARA-002 clinical
development programs, with patient dosing now underway in the
ADVANCED-2 trial in patients with non-muscle invasive bladder
cancer (NMIBC) and the STARBORN-1 trial in pediatric patients with
lymphatic malformations (LMs),” said Jesse Shefferman, Chief
Executive Officer of Protara Therapeutics. “We remain keenly
focused on execution across all our ongoing clinical studies and
remain on track to report preliminary results from the expansion
portion of the ADVANCED-1 trial of TARA-002 in patients with NMIBC
in the first half of 2024. With a cash runway into the second
quarter of 2025, we believe we are well positioned to achieve key
milestones in our TARA-002 development programs.”
Recent Highlights
TARA-002 in NMIBC
- In September 2023, the Company
announced dosing of the first patient in ADVANCED-2, a Phase 2
open-label trial evaluating intravesical TARA-002 in up to 102
NMIBC patients with carcinoma in situ (CIS) (± Ta/T1) who are
Bacillus Calmette-Guérin (BCG)-naïve (n=27) and BCG-unresponsive
(n=75). Trial subjects will receive an induction with or without a
reinduction course of six weekly intravesical instillations of
TARA-002, followed by a maintenance course of three weekly
installations every three months in the BCG-unresponsive
cohort.
- As previously announced, patient
dosing is underway in the Phase 1b ADVANCED-1EXP study, an
open-label expansion trial evaluating intravesical TARA-002 at the
40KE1 dose in up to 12 CIS patients, including BCG-naïve,
BCG-unresponsive, and BCG-inadequately treated patients. The
Company remains on track to report preliminary results in the first
half of 2024. The primary endpoint of the trial is the complete
response rate at three months.
TARA-002 in LMs
- In October 2023, the Company
announced dosing of the first patient in STARBORN-1, a Phase 2
clinical trial of TARA-002 in pediatric patients with macrocystic
and mixed-cystic LMs. Including an age de-escalation safety
lead-in, the trial will enroll approximately 30 patients who will
receive up to four injections of TARA-002 spaced approximately six
weeks apart. The primary endpoint of the trial is the proportion of
participants with macrocystic and mixed cystic LMs who demonstrate
clinical success, defined as having either a complete response (90%
to 100% reduction from baseline in total LM volume) or substantial
response (60% to less than 90% reduction in total LM volume) as
measured by axial imaging.
IV Choline Chloride Program
- The Company continues to engage
with the U.S. Food and Drug Administration and plans to use both
regulatory feedback and results from its prevalence study to inform
next steps for the IV Choline Chloride development program.
Third Quarter 2023 Financial
Results
- As of September 30, 2023,
cash, cash equivalents and investments were $74.0 million. The
Company expects its current cash, cash equivalents and investments
will be sufficient to fund its planned operations into the second
quarter of 2025.
- Research and development expenses
for the third quarter of 2023 increased to $6.2
million from $3.5 million during the third quarter
of 2022, primarily reflecting an increase in expenses related to
non-clinical and clinical trial activities for TARA-002 of $2.2
million as well as $0.4 million of increased employee expenses
inclusive of stock-based compensation.
- General and administrative expenses
for both the third quarter of 2023 and 2022 were $4.5 million.
- For the third quarter of 2023,
Protara reported a net loss of $9.9 million,
or $0.87 per share, compared with a net loss of $7.7
million, or $0.68 per share, for the same period in 2022. Net
loss for the third quarter of 2023 included approximately $1.4
million of stock-based compensation expenses.
About Non-Muscle Invasive Bladder Cancer
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
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 three 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 TARA-002 in LMs
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 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 |
|
|
|
September 30,2023 |
|
|
December 31, 2022 |
|
|
|
(unaudited) |
|
|
|
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
33,768 |
|
|
$ |
24,127 |
|
Marketable debt securities |
|
|
40,266 |
|
|
|
60,243 |
|
Prepaid expenses and other current assets |
|
|
3,779 |
|
|
|
1,776 |
|
Total current assets |
|
|
77,813 |
|
|
|
86,146 |
|
Restricted cash,
non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities,
non-current |
|
|
- |
|
|
|
17,886 |
|
Property and equipment,
net |
|
|
1,401 |
|
|
|
1,592 |
|
Operating lease right-of-use
asset |
|
|
5,567 |
|
|
|
6,277 |
|
Other assets |
|
|
2,941 |
|
|
|
644 |
|
Total assets |
|
$ |
88,467 |
|
|
$ |
113,290 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
2,504 |
|
|
$ |
1,586 |
|
Accrued expenses |
|
|
3,305 |
|
|
|
3,237 |
|
Operating lease liability |
|
|
966 |
|
|
|
917 |
|
Total current liabilities |
|
|
6,775 |
|
|
|
5,740 |
|
Operating lease liability,
non-current |
|
|
4,736 |
|
|
|
5,467 |
|
Total liabilities |
|
|
11,511 |
|
|
|
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
September 30, 2023 and December 31, 2022, 7,991 and 8,027 shares
issued and outstanding as of September 30, 2023 and December 31,
2022, respectively. |
|
|
- |
|
|
|
- |
|
Common stock, $0.001 par value, authorized 100,000,000 shares:
Common stock, 11,364,903 and 11,267,389 shares issued and
outstanding as of September 30, 2023 and December 31, 2022,
respectively. |
|
|
11 |
|
|
|
11 |
|
Additional paid-in capital |
|
|
267,273 |
|
|
|
262,724 |
|
Accumulated deficit |
|
|
(190,163 |
) |
|
|
(159,964 |
) |
Accumulated other comprehensive income (loss) |
|
|
(165 |
) |
|
|
(688 |
) |
Total stockholders’ equity |
|
|
76,956 |
|
|
|
102,083 |
|
Total liabilities and stockholders’ equity |
|
$ |
88,467 |
|
|
$ |
113,290 |
|
|
|
|
|
|
|
|
|
|
The accompanying notes are an integral part of
these unaudited condensed consolidated financial statements.
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 EndedSeptember
30, |
|
|
For the Nine Months Ended September
30, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
6,218 |
|
|
$ |
3,466 |
|
|
$ |
18,608 |
|
|
$ |
11,819 |
|
General and administrative |
|
|
4,482 |
|
|
|
4,508 |
|
|
|
13,964 |
|
|
|
15,734 |
|
Total operating expenses |
|
|
10,700 |
|
|
|
7,974 |
|
|
|
32,572 |
|
|
|
27,553 |
|
Loss from operations |
|
|
(10,700 |
) |
|
|
(7,974 |
) |
|
|
(32,572 |
) |
|
|
(27,553 |
) |
Other income (expense),
net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest and investment income |
|
|
840 |
|
|
|
283 |
|
|
|
2,373 |
|
|
|
568 |
|
Other income (expense), net |
|
|
840 |
|
|
|
283 |
|
|
|
2,373 |
|
|
|
568 |
|
Net loss |
|
$ |
(9,860 |
) |
|
$ |
(7,691 |
) |
|
$ |
(30,199 |
) |
|
$ |
(26,985 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.87 |
) |
|
$ |
(0.68 |
) |
|
$ |
(2.67 |
) |
|
$ |
(2.40 |
) |
Weighted-average shares outstanding, basic and diluted |
|
|
11,347,887 |
|
|
|
11,265,475 |
|
|
|
11,320,027 |
|
|
|
11,256,995 |
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net unrealized gain (loss) on marketable debt securities |
|
|
171 |
|
|
|
(8 |
) |
|
|
523 |
|
|
|
(919 |
) |
Other comprehensive income
(loss) |
|
|
171 |
|
|
|
(8 |
) |
|
|
523 |
|
|
|
(919 |
) |
Comprehensive Loss |
|
$ |
(9,689 |
) |
|
$ |
(7,699 |
) |
|
$ |
(29,676 |
) |
|
$ |
(27,904 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
The accompanying notes are an integral part of
these unaudited condensed consolidated financial statements.
Company Contact:Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
Protara Therapeutics (NASDAQ:TARA)
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