On November 12, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the third quarter ended September 30, 2024 (Press release, Protara Therapeutics, NOV 12, 2024, View Source [SID1234648205]).
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"We continue to make significant progress advancing all of our ongoing programs as we strive toward our goal of bringing life-changing therapies to patients impacted by cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Notably, we remain on track to report interim data in the fourth quarter of 2024 from our ADVANCED-2 study of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC). Given its ease of use, favorable safety profile, and encouraging early clinical data, we believe TARA-002 could be an impactful addition to the NMIBC treatment landscape."
Mr. Shefferman added, "In addition to our NMIBC program, we remain on track to commence the pivotal THRIVE-3 study of intravenous (IV) Choline Chloride, for which we recently received Fast Track designation from the U.S. Food and Drug Administration (FDA), in the first quarter of 2025. Finally, we continue to expect interim data in the first half of 2025 from our ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs), a rare condition primarily impacting children for which there are no FDA approved therapies."
Recent Progress and Highlights
TARA-002 in NMIBC
The Company remains on track to report results from a pre-planned interim analysis of six-month evaluable patients in the ongoing Phase 2 open-label ADVANCED-2 trial later this quarter. The ADVANCED-2 trial is assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-unresponsive (n≈100) and BCG-Naïve (n=27). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the FDA’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment Draft Guidance for Industry.
The Company expects to report preliminary results in mid-2025 from 12-month evaluable patients in the ongoing Phase 2 open-label ADVANCED-2 trial.
The Company continues to explore systemic priming dosing prior to initiation of intravesical administration, as well as combination therapy with TARA-002 in NMIBC patients with CIS. Given TARA-002’s mechanism of action and safety profile, the Company believes it has strong potential for use in combination therapy and is working to determine various opportunities for the clinical program.
IV Choline Chloride for Patients on Parenteral Support (PS)
In October 2024, Protara announced that the FDA granted Fast Track designation to IV Choline Chloride, the Company’s investigational IV phospholipid substrate replacement therapy, in patients for whom oral or enteral nutrition is not possible, insufficient, or contraindicated. Protara is currently developing IV Choline Chloride as a source of choline for adult and adolescent patients on PS.
In September 2024, the Company announced results from THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on PS. The study found that 78% of patients who are dependent on PS were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury.
In September 2024, the U.S. Patent and Trademark Office issued the Company a second patent in this program, claiming a method for treating choline deficiency with a choline composition with a term until 2041.
The Company expects to begin dosing patients in the THRIVE-3 registrational trial in the first quarter of 2025.
TARA-002 in LMs
In September 2024, Protara announced completion of the first safety cohort of the ongoing Phase 2 STARBORN-1 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs. Of the three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002, including one with a macrocystic LM and one with a ranula (a type of maxillofacial cyst) achieved a complete response after receiving one dose of TARA-002. TARA-002 was generally well-tolerated. Enrollment is underway in additional cohorts, with initial results expected in the first half of 2025.
Third Quarter 2024 Financial Results
As of September 30, 2024, cash, cash equivalents, and investments in marketable debt securities totaled approximately $81.5 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 2026.
Research and development expenses for the third quarter of 2024 increased to $8.1 million from $6.2 million for the prior year period. This increase was primarily due to an increase in expenses related to TARA-002 of $1.5 million as well as an increase in expenses related to IV Choline Chloride of $0.4 million.
General and administrative expenses for the third quarter of 2024 decreased to $4.3 million from $4.5 million for the prior year period. This decrease was primarily due to a reduction in personnel-related expenses of $0.6 million, offset partially by increases in market development, business development, and investor relations activities of $0.3 million.
For the third quarter of 2024, Protara incurred a net loss of $11.2 million, or $0.50 per share, compared with a net loss of $9.9 million, or $0.87 per share, for the same period in 2023. Net loss for the third quarter of 2024 included approximately $0.9 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 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 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 phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 80% of patients dependent on PS are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients and has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PS patients. The U.S. Patent and Trademark Office has issued the Company a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.