Protara Therapeutics Announces Second Quarter 2022 Financial Results and Business Overview
- Enrollment Progressing in Phase 1 ADVANCED-1 Study of TARA-002 for the Treatment of Non-Muscle Invasive Bladder Cancer -
- Strong Cash, Cash Equivalents and Investments Position of
“We are pleased with the continued progress of the ADVANCED-1 study of TARA-002 in non-muscle invasive bladder cancer (NMIBC),” said
TARA-002 in NMIBC
- Enrollment is progressing in the Company’s Phase 1 ADVANCED-1 clinical trial evaluating TARA-002, an investigational cell-based immunopotentiator, for the treatment of NMIBC.
June 2022, a Trials in Progress poster related to the ADVANCED-1 Phase 1 trial was featured at the American Society of Clinical Oncology Annual Meeting.
TARA-002 in LMs
- Protara continues to engage with the Vaccines and Related Products Division of the
U.S. Food and Drug Administration(FDA) regarding a development path for TARA-002 in LMs. Following receipt of preliminary guidance from the FDA and in consultation with the Company’s external scientific advisory board, Protara plans to initiate a Phase 2 clinical trial of TARA-002 in LMs, subject to alignment with the agency on a trial protocol.
- Protara announced that the
European Medicines Agency(EMA) recently granted orphan drug designation (ODD) to TARA-002 for the treatment of LMs.
May 2022, the Company announced results of a retrospective analysis of OK-432, the originator compound for TARA-002, for the treatment of LMs. Results from the analysis, which were presented during a poster presentation at the International Society for the Study of Vascular Anomalies World Congress2022, showed that OK-432 met the primary efficacy endpoint of clinical success (defined as complete [90%-100%] or substantial [60%-89%] reduction in LM volume measured radiographically) in treating LMs and demonstrated a favorable safety profile.
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.
April 2022, the U.S. Patent and Trademark Officeissued to the Company a patent claiming a sterile aqueous choline salt composition with a term expiring in 2041.
Second Quarter 2022 Financial Results
- As of
June 30, 2022, cash, cash equivalents and marketable debt securities were $112.8 million. The Company expects its current cash and cash equivalents will be sufficient to fund its planned operations into mid-2024.
- Research and development (R&D) expenses for the second quarter of 2022 decreased to
$3.1 millionfrom $5.9 millionduring the second quarter of 2021. The decreased R&D expenses were primarily due to decreases in manufacturing expenses associated with TARA-002.
- General and administrative expenses for the second quarter of 2022 decreased to
$5.6 millionfrom $6.9 millionfor the prior year period. The decrease was primarily due to decreases in stock-based compensation.
- For the second quarter of 2022, Protara reported a net loss of
$8.5 million, or $0.80per share, compared with a net loss of $12.8 million, or $1.14per share, for the same period in 2021. Net loss for the second quarter of 2022 included approximately $3.8 millionof stock-based compensation expenses.
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
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
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.
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.
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 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
Unaudited Condensed Consolidated Balance Sheets
(in thousands, except share and per share data)
|Cash and cash equivalents||$||33,815||$||35,724|
|Marketable debt securities||64,643||55,505|
|Prepaid expenses and other current assets||2,633||1,883|
|Total current assets||101,091||93,112|
|Restricted cash, non-current||745||745|
|Marketable debt securities, non-current||14,349||39,467|
|Property and equipment, net||1,640||1,719|
|Operating lease right-of-use asset||6,731||7,171|
|Liabilities and Stockholders’ Equity|
|Operating lease liability||885||855|
|Total current liabilities||3,493||4,298|
|Operating lease liability, non-current||5,933||6,384|
|Commitments and contingencies (Note 8)|
|Series 1 Convertible Preferred Stock, 8,028 shares authorized at
|Common stock, 11,257,177 and 11,235,731 shares issued and outstanding as of
|Additional paid-in capital||259,875||256,126|
|Accumulated other comprehensive income (loss)||(1,122||)||(211||)|
|Total stockholders’ equity||145,458||161,914|
|Total liabilities and stockholders’ equity||$||154,884||$||172,596|
Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss
(in thousands, except share and per share data)
|For the Three months Ended
||For the Six months Ended
|Research and development||$||3,084||$||5,887||$||8,353||$||12,927|
|General and administrative||5,621||6,905||11,226||13,445|
|Total operating expenses||8,705||12,792||19,579||26,372|
|Loss from operations||(8,705||)||(12,792||)||(19,579||)||(26,372||)|
|Other income (expense), net:|
|Interest and investment income||166||10||285||125|
|Other income (expense), net||166||10||285||125|
|Net loss per share attributable to common stockholders, basic and diluted||$||(0.80||)||$||(1.14||)||$||(1.71||)||$||(2.34||)|
|Weighted-average shares outstanding, basic and diluted||11,255,215||11,232,010||11,252,686||11,229,484|
|Other comprehensive income (loss):|
|Net unrealized (loss) gain on marketable debt securities||(180||)||63||(911||)||(101||)|
|Other comprehensive income (loss)||(180||)||63||(911||)||(101||)|
Source: Protara Therapeutics