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
full year and fourth quarter ended December 31, 2023 and provided a
business update.
“We expect 2024 to be a milestone-rich year for
Protara and believe we are well positioned to continue to execute
on advancing our pipeline to deliver meaningful new therapies to
patients with cancer and rare diseases,” said Jesse Shefferman,
Chief Executive Officer of Protara Therapeutics. “Notably, we
anticipate sharing Phase 1b proof of concept (POC) and preliminary
Phase 2 data for our lead TARA-002 indication, non-muscle invasive
bladder cancer (NMIBC), later this year, which we believe will
continue to support its potential to play an important role in the
evolving NMIBC treatment landscape. Progress also continues for our
program of TARA-002 in lymphatic malformations (LMs), a highly
underserved pediatric population, with dosing in our Phase 2 study
now underway.”
Recent Progress and
Highlights
TARA-002 in NMIBC
- The Company remains on track to
report preliminary results from the ongoing Phase 1b ADVANCED-1EXP
POC, open-label expansion trial in the first half of 2024. The
trial is evaluating intravesical TARA-002 at the 40KE1 dose in up
to 12 carcinoma in situ (CIS) patients, including Bacillus
Calmette-Guérin (BCG)-naïve, BCG-unresponsive, and BCG-inadequately
treated patients. The primary endpoint will assess the activity
levels at the preliminary three-month assessment timepoint,
including complete response (CR) rates and immuno-dynamic activity
in CIS and CIS +Ta/T1 patients.
- The Company expects to share
preliminary results from a pre-planned risk-benefit analysis of the
ongoing Phase 2 open-label ADVANCED-2 trial in the second half of
2024. The analysis is expected to include approximately 10 patients
who are six-month evaluable. The ongoing ADVANCED-2 trial is
assessing intravesical TARA-002 in at least 102 NMIBC patients with
CIS (± Ta/T1) who are BCG-naïve (n=27) and BCG-unresponsive
(n=75-100). Trial subjects will receive an induction course of six
weekly intravesical instillations, and following mandatory biopsy
at three months, will either receive a reinduction course of six
weekly intravesical instillations of TARA-002, or the first
maintenance course of three weekly installations every three months
for 24 months.
TARA-002 in LMs
- Dosing continues to progress 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 CR (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 for Patients on Parenteral
Nutrition (PN)
- 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.
Fourth Quarter and Full Year 2023
Financial Results
- As of December 31, 2023, cash, cash
equivalents, and investments in marketable debt securities totaled
$65.6 million. The Company expects its cash, cash equivalents, and
investments in marketable debt securities will be sufficient to
fund its planned operations and data milestones into the second
quarter of 2025.
- Research and development expenses
for the fourth quarter of 2023 increased to $6.4 million from $5.0
million for the prior year period, and for the full year increased
to $25.0 million compared to $16.8 million for 2022. The fourth
quarter and full year increases were primarily due to an increase
in expenses related to clinical trials and non-clinical activities
for TARA-002.
- General and administrative expenses
for the fourth quarter of 2023 decreased to $4.7 million from $5.0
million for the prior year period, and for the full year decreased
to $18.6 million compared to $20.7 million for 2022. The fourth
quarter and full year decreases were primarily due to a reduction
in personnel costs and lower directors and officers liability
insurance premiums which were partially offset by an increase in
professional costs.
- For the fourth quarter of 2023,
Protara incurred a net loss of $10.2 million, or $0.90 per share,
compared with a net loss of $39.0 million, or $3.46 per share, for
the same period in 2022. Net loss for the year ended December 31,
2023 was $40.4 million, or $3.57 per share, compared with a net
loss of $66.0 million, or $5.86 per share, for the year ended
December 31, 2022. Net loss in the fourth quarter of 2022 included
a non-cash goodwill impairment charge of $29.5 million associated
with the accounting for the reverse merger transaction in January
2020. Net loss for the fourth quarter of 2023 included
approximately $1.5 million of stock-based compensation expenses.
Net loss for the year ended December 31, 2023 included
approximately $6.1 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 pro-inflammatory response with
release of cytokines such as tumor necrosis factor (TNF)-alpha,
interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly
kills tumor cells and triggers a host immune response by inducing
immunogenic cell death, which further enhances the antitumor immune
response.
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 Intravenous (IV) Choline
Chloride
IV Choline Chloride is an investigational, IV
phospholipid substrate replacement therapy initially in development
for patients receiving PN. Choline is a known important substrate
for phospholipids that are critical for healthy liver function and
also plays an important role in modulating gene expression, cell
membrane signaling, brain development and neurotransmission, muscle
function, and bone health. PN patients are unable to synthesize
choline from enteral nutrition sources, and there are currently no
available PN formulations containing choline. Approximately 80
percent of PN-dependent patients are choline-deficient and have
some degree of liver damage, which can lead to hepatic failure. IV
Choline Chloride has been granted Orphan Drug Designation by the
FDA for the prevention of choline deficiency in PN patients. The
Company was issued a patent claiming a choline composition with a
term expiring in 2041.
About Protara
Therapeutics, Inc.
Protara is a clinical-stage biotechnology
company committed to advancing transformative therapies for people
with cancer and rare diseases. Protara’s portfolio includes its
lead candidate, TARA-002, an investigational cell-based therapy in
development for the treatment of non-muscle invasive bladder cancer
(NMIBC) and lymphatic malformations (LMs). The Company is
evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients
with carcinoma in situ (CIS) who are unresponsive or naïve to
treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2
trial in pediatric patients with LMs. Additionally, Protara is
developing IV Choline Chloride, an investigational phospholipid
substrate replacement for patients on parenteral nutrition who are
otherwise unable to meet their choline needs via oral or enteral
routes. 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; failure to attract and retain
management and key personnel; 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. Consolidated Balance Sheets(in
thousands, except share and per share data) |
|
|
|
December 31, |
|
|
|
2023 |
|
|
2022 |
|
Assets |
|
|
|
|
|
|
Current
assets: |
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
39,586 |
|
|
$ |
24,127 |
|
Marketable debt securities |
|
|
25,994 |
|
|
|
60,243 |
|
Prepaid expenses and other current assets |
|
|
3,125 |
|
|
|
1,776 |
|
Total current assets |
|
|
68,705 |
|
|
|
86,146 |
|
Restricted cash, non-current |
|
|
745 |
|
|
|
745 |
|
Marketable debt securities, non-current |
|
|
- |
|
|
|
17,886 |
|
Property and equipment, net |
|
|
1,296 |
|
|
|
1,592 |
|
Operating lease right-of-use asset |
|
|
5,264 |
|
|
|
6,277 |
|
Other assets |
|
|
2,944 |
|
|
|
644 |
|
Total assets |
|
$ |
78,954 |
|
|
$ |
113,290 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
2,434 |
|
|
$ |
1,586 |
|
Accrued expenses |
|
|
2,732 |
|
|
|
3,237 |
|
Operating lease liability |
|
|
983 |
|
|
|
917 |
|
Total current liabilities |
|
|
6,149 |
|
|
|
5,740 |
|
Operating lease liability, non-current |
|
|
4,484 |
|
|
|
5,467 |
|
Total liabilities |
|
|
10,633 |
|
|
|
11,207 |
|
Commitments and
contingencies (Note 10) |
|
|
|
|
|
|
|
|
Stockholders’
Equity: |
|
|
|
|
|
|
|
|
Preferred stock, $0.001 par value, authorized 10,000,000
shares: |
|
|
|
|
|
|
|
|
Series 1 convertible preferred stock, 8,028 shares authorized at
December 31, 2023 and 2022, 7,991 and 8,027 shares issued and
outstanding as of December 31, 2023 and 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 December 31, 2023 and 2022, respectively. |
|
|
11 |
|
|
|
11 |
|
Additional paid in capital |
|
|
268,725 |
|
|
|
262,724 |
|
Accumulated deficit |
|
|
(200,384 |
) |
|
|
(159,964 |
) |
Accumulated other comprehensive income (loss) |
|
|
(31 |
) |
|
|
(688 |
) |
Total stockholders’ equity |
|
|
68,321 |
|
|
|
102,083 |
|
Total liabilities and stockholders’ equity |
|
$ |
78,954 |
|
|
$ |
113,290 |
|
|
PROTARA THERAPEUTICS, INC. AND
SUBSIDIARIESConsolidated Statements of Operations
and Comprehensive Loss(in thousands, except share and per
share data) |
|
|
|
(Unaudited) |
|
|
(Audited) |
|
|
|
For the Three Months EndedDecember
31, |
|
|
For the Years Ended December
31, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
6,381 |
|
|
$ |
4,989 |
|
|
$ |
24,989 |
|
|
$ |
16,808 |
|
General and administrative |
|
|
4,660 |
|
|
|
5,003 |
|
|
|
18,624 |
|
|
|
20,737 |
|
Loss on impairment of goodwill |
|
|
- |
|
|
|
29,517 |
|
|
|
- |
|
|
|
29,517 |
|
Total operating expenses |
|
|
11,041 |
|
|
|
39,509 |
|
|
|
43,613 |
|
|
|
67,062 |
|
Loss from operations |
|
|
(11,041 |
) |
|
|
(39,509 |
) |
|
|
(43,613 |
) |
|
|
(67,062 |
) |
Other income (expense),
net: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest and investment income |
|
|
820 |
|
|
|
543 |
|
|
|
3,193 |
|
|
|
1,110 |
|
Other income (expense), net |
|
|
820 |
|
|
|
543 |
|
|
|
3,193 |
|
|
|
1,110 |
|
Net loss |
|
$ |
(10,221 |
) |
|
$ |
(38,966 |
) |
|
$ |
(40,420 |
) |
|
$ |
(65,952 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.90 |
|
|
$ |
(3.46 |
) |
|
$ |
(3.57 |
) |
|
$ |
(5.86 |
) |
Weighted-average shares outstanding, basic and diluted |
|
|
11,364,903 |
|
|
|
11,267,389 |
|
|
|
11,331,338 |
|
|
|
11,259,615 |
|
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net unrealized gain (loss) on marketable debt securities |
|
|
134 |
|
|
|
(442 |
) |
|
|
657 |
|
|
|
(477 |
) |
Other comprehensive income
(loss) |
|
|
134 |
|
|
|
(443 |
) |
|
|
657 |
|
|
|
(477 |
) |
Comprehensive Loss |
|
$ |
(10,087 |
) |
|
$ |
(38,524 |
) |
|
$ |
(39,763 |
) |
|
$ |
(66,429 |
) |
|
Company Contact:
Justine O'MalleyProtara
TherapeuticsJustine.OMalley@protaratx.com646-817-2836
Protara Therapeutics (NASDAQ:TARA)
Gráfica de Acción Histórica
De Abr 2024 a May 2024
Protara Therapeutics (NASDAQ:TARA)
Gráfica de Acción Histórica
De May 2023 a May 2024