On March 13, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the full year and fourth quarter ended December 31, 2023 and provided a business update (Press release, Protara Therapeutics, MAR 13, 2024, View Source [SID1234641115]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"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.