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, 2022 and provided a business
update.
“We continue to make meaningful progress with
dose escalation in the ongoing Phase 1a portion of our ADVANCED-1
study of TARA-002 in non-muscle invasive bladder cancer (NMIBC),
and look forward to ultimately utilizing these data, as well as
data from our ongoing preclinical work, to design effective
later-stage clinical trials in this indication,” said Jesse
Shefferman, Chief Executive Officer of Protara Therapeutics. “We
are pleased to share that we have received initial feedback from
the U.S. Food and Drug Administration (FDA) on our proposed Phase 2
study protocol evaluating TARA-002 in Lymphatic Malformations
(LMs), and now expect to initiate the study in 2023. LMs represents
a highly underserved rare pediatric indication for which we believe
TARA-002 could serve as a much-needed treatment
option.”
Recent Highlights
TARA-002 in NMIBC
- Dose escalation remains ongoing in
the Phase 1a portion of the Company’s ADVANCED-1 clinical trial
evaluating TARA-002, an investigational cell-based
immunopotentiator, for the treatment of NMIBC. The Phase 1a dose
escalation portion of the trial is designed to identify a safe and
tolerable dose of TARA-002 to be further evaluated in the Phase 1b
expansion portion of the trial.
TARA-002 in LMs
- Protara recently received initial
feedback from the Vaccines and Related Products Division of the FDA
on the protocol for the proposed Phase 2 study evaluating TARA-002
in LMs. The Company is in the process of finalizing the trial
design and expects to commence study start-up activities in the
first half of 2023.
IV Choline Chloride in Intestinal Failure
Associated Liver Disease (IFALD)
- The Company’s prospective study to
enhance understanding of the incidence of IFALD in patients
dependent on parenteral nutrition remains ongoing. Protara expects
to use results from the prospective study, as well as its
previously completed retrospective study, to inform next steps for
the IV Choline Chloride development program.
Third Quarter 2022 Financial
Results
- As of September 30, 2022, cash,
cash equivalents and marketable debt securities were $107.1
million. The Company now expects its current cash and cash
equivalents will be sufficient to fund its planned operations into
the second half of 2024.
- Research and development (R&D)
expenses for the third quarter of 2022 decreased to $3.5 million
from $4.1 million during the third quarter of 2021. The decreased
R&D expenses were primarily due to lower regulatory costs
relative to regulatory expense in the comparable three months ended
September 30, 2021, when the Company filed its Investigational New
Drug application for TARA-002.
- General and administrative expenses
for the third quarter of 2022 decreased to $4.5 million from $6.7
million for the prior year period. The decrease was primarily due
to a decrease of $1.2 million in stock-based compensation expense,
a decrease of $0.2 million in compensation, benefits and other
employee-related expenses, and a decrease of $0.6 million in market
development expenses.
- For the third quarter of 2022,
Protara reported a net loss of $7.7 million, or $0.68 per share,
compared with a net loss of $10.8 million, or $0.96 per share, for
the same period in 2021. Net loss for the third quarter of 2022
included approximately $1.4 million of stock-based compensation
expenses.
About TARA-002
TARA-002 is an investigational cell therapy in
development for the treatment of NMIBC and 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 has successfully demonstrated 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, tumor necrosis factor
(TNF)-alpha, granulocyte colony-stimulating factor, and
granulocyte-macrophage colony-stimulating factor are secreted by
immune cells to induce a strong local 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 identifying and
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 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, 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, including potential alignment
with the FDA on, and finalization of, clinical trial design for
TARA-002 in pediatric LM patients; Protara’s financial footing;
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; the impact of the COVID-19 pandemic on Protara’s business
and the global economy as well as the impact on Protara’s contract
research organizations, study sites or other clinical partners;
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 or
political 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.Unaudited Condensed Consolidated Balance
Sheets(in thousands, except share and per share data)
|
|
As of |
|
|
|
September 30,2022 |
|
|
December 31, 2021 |
|
Assets |
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
47,498 |
|
|
$ |
35,724 |
|
Marketable debt securities |
|
|
48,819 |
|
|
|
55,505 |
|
Prepaid expenses and other current assets |
|
|
2,350 |
|
|
|
1,883 |
|
Total current assets |
|
|
98,667 |
|
|
|
93,112 |
|
Restricted cash,
non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities,
non-current |
|
|
10,760 |
|
|
|
39,467 |
|
Property and equipment,
net |
|
|
1,662 |
|
|
|
1,719 |
|
Operating lease right-of-use
asset |
|
|
6,506 |
|
|
|
7,171 |
|
Goodwill |
|
|
29,517 |
|
|
|
29,517 |
|
Other assets |
|
|
734 |
|
|
|
865 |
|
Total assets |
|
$ |
148,591 |
|
|
$ |
172,596 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
456 |
|
|
$ |
954 |
|
Accrued expenses |
|
|
2,354 |
|
|
|
2,489 |
|
Operating lease liability |
|
|
901 |
|
|
|
855 |
|
Total current liabilities |
|
|
3,711 |
|
|
|
4,298 |
|
Operating lease liability,
non-current |
|
|
5,702 |
|
|
|
6,384 |
|
Total liabilities |
|
|
9,413 |
|
|
|
10,682 |
|
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, 2022 and December 31, 2021, 8,027 shares issued and
outstanding as of September 30, 2022 and December 31, 2021. |
|
|
- |
|
|
|
- |
|
Common stock, $0.001 par value, authorized 100,000,000 shares:
Common stock, 11,267,389 and 11,235,731 shares issued and
outstanding as of September 30, 2022 and December 31, 2021,
respectively. |
|
|
11 |
|
|
|
11 |
|
Additional paid-in capital |
|
|
261,294 |
|
|
|
256,126 |
|
Accumulated deficit |
|
|
(120,997 |
) |
|
|
(94,012 |
) |
Accumulated other comprehensive income (loss) |
|
|
(1,130 |
) |
|
|
(211 |
) |
Total stockholders’ equity |
|
|
139,178 |
|
|
|
161,914 |
|
Total liabilities and stockholders’ equity |
|
$ |
148,591 |
|
|
$ |
172,596 |
|
PROTARA THERAPEUTICS,
INC.Unaudited 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, |
|
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
3,466 |
|
|
$ |
4,093 |
|
|
$ |
11,819 |
|
|
$ |
17,020 |
|
General and administrative |
|
|
4,508 |
|
|
|
6,737 |
|
|
|
15,734 |
|
|
|
20,182 |
|
Total operating expenses |
|
|
7,974 |
|
|
|
10,830 |
|
|
|
27,553 |
|
|
|
37,202 |
|
Loss from operations |
|
|
(7,974 |
) |
|
|
(10,830 |
) |
|
|
(27,553 |
) |
|
|
(37,202 |
) |
Other income (expense),
net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest and investment income |
|
|
283 |
|
|
|
53 |
|
|
|
568 |
|
|
|
178 |
|
Other income (expense), net |
|
|
283 |
|
|
|
53 |
|
|
|
568 |
|
|
|
178 |
|
Net loss |
|
|
(7,691 |
) |
|
|
(10,777 |
) |
|
|
(26,985 |
) |
|
|
(37,024 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.68 |
) |
|
$ |
(0.96 |
) |
|
$ |
(2.40 |
) |
|
$ |
(3.30 |
) |
Weighted-average shares outstanding, basic and diluted |
|
|
11,265,475 |
|
|
|
11,235,507 |
|
|
|
11,256,995 |
|
|
|
11,231,513 |
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net unrealized (loss) gain on marketable debt securities |
|
|
(8 |
) |
|
|
62 |
|
|
|
(919 |
) |
|
|
(39 |
) |
Other comprehensive income
(loss) |
|
|
(8 |
) |
|
|
62 |
|
|
|
(919 |
) |
|
|
(39 |
) |
Comprehensive Loss |
|
$ |
(7,699 |
) |
|
$ |
(10,715 |
) |
|
$ |
(27,904 |
) |
|
$ |
(37,063 |
) |
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
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