- The ADVANCED-1EXP trial is underway, bringing us one step closer to understanding the potential of TARA-002 for treating Non-Muscle Invasive Bladder Cancer (NMIBC) with Carcinoma in Situ (CIS). We look forward to the preliminary results in the first half of 2024.
- Come 4th Quarter of 2023, TARA-002 is set to be examined in the ADVANCED-2 trial, evaluating its potential in treating Non-Muscle Invasive Bladder Cancer (NMIBC) patients who are either BCG-unresponsive or BCG-naïve with carcinoma in situ (CIS). The time is near for TARA-002 to deliver its potentially life-saving promise to patients worldwide.
- TARA-002 is on the cusp of taking a major leap forward, with plans set for a Phase 2 trial of the revolutionary medication in Lymphatic Malformations during the 4th quarter of 2023. This marks a significant milestone on the road to improving outcomes for individuals suffering from this debilitating condition.
- As of June 30, 2023, our company had an impressive reserve of $80 million, comprised of cash, cash equivalents, and investments. With our current capital, we anticipate our operations will be amply funded through 2025.
Protara Therapeutics, Inc. (Nasdaq: TARA) announced financial results and an update on its progress for the second quarter of 2023 on August 03. Protara is driving the development of groundbreaking therapies for diseases such as cancer and rare conditions, and its end of quarter financials show a promising future ahead.
Protara Therapeutics is advancing quickly with the successful preliminary results of the dose expansion portion of the ADVANCED-1 trial of TARA-002 in high-grade non-muscle invasive bladder cancer (NMIBC) patients. Rapid enrollment for the trial has enabled Protara to plan for the sharing of preliminary results in the first half of 2024 and the launch of two additional trials for TARA-002 later this year. With a disciplined approach to investments, Protara is well-positioned with enough cash on hand to sustain the company into 2025.
Recent Highlights
TARA-002 in NMIBC
- At the American Urological Association Annual Meeting in April 2023, the Company announced a break-through with their investigational cell-based therapy, TARA-002 – positive preliminary results for treating patients with high-grade NMIBC. In the Phase 1a dose-escalation component of the ADVANCED-1 clinical trial, TARA-002 was found to be generally well tolerated and displayed anti-tumor activity, including complete responses in heavily pre-treated and BCG-unresponsive patients. Even patients diagnosed with carcinoma in situ observed tumor regression.
- In May 2023, the Company commenced a Phase 1b ADVANCED-1EXP study, and subsequently dosed the first patients with intravesical TARA-002 at the 40KE1 dose. The open-label expansion study consists of 12 CIS patients; including BCG-naïve, BCG-unresponsive, and BCG-inadequately treated individuals. As the dosing progresses, preliminary results are expected to be released in the first half of 2024. The trial’s primary endpoint is the CR rate at three months.
- In the final quarter of 2023, the Company will spearhead ADVANCED-2, an exciting Phase 1b/2 open-label trial which will assess the efficacy of intravesical TARA-002 in up to 102 patients with high-grade CIS, including 27 who have never been treated with BCG for at least two years prior to their diagnosis and 75 with BCG-unresponsive CIS (± Ta/T1). We look forward to the potential discovery of a valuable new treatment.
TARA-002 in Lymphatic Malformations (LMs)
- With a green-light from the U.S. Food and Drug Administration, the STARBORN-1 clinical trial of TARA-002 is set to kick off this fourth quarter of 2023! Ten leading pediatric centers of excellence have joined forces to launch the innovative Phase 2 study to tackle macrocystic and mixed-cystic LMs in pediatric patients. The start-up activities are in full swing, and the trial is set to transform the ways in which we understand and treat this condition.
IV Choline Chloride Program
- Through a prospective prevalence study designed to examine the incidence of choline deficiency in patients receiving parenteral nutrition, Protara is nearing its conclusion in order to better understand this widespread issue. With plans to use both feedback from the FDA and the results of the study to inform the development program of IV Choline Chloride, Protara is focused on finding ways to ensure optimal nutrition for those in need.
Second Quarter 2023 Financial Results
- As of June 30, 2023, our liquid assets stood at a total of $80.4 million. We are confident that our resources are adequate to sustain our operations through 2025.
- Research and development expenses for the second quarter of 2023 experienced a dramatic jump to $7.2 million, a 135% increase from the $3.1 million invested during the same period of the preceding year. This can mainly be attributed to more extensive clinical and non-clinical trial activity for TARA-002.
- The second quarter of 2023 saw a marked decrease in general and administrative expenses, down from $5.6 million for the prior year period to $4.9 million. This decrease is mainly attributed to reduced employee-related expenses.
- Protara reported a striking net loss of $11.3 million, or $1.00 per share, in the second quarter of 2023 compared to the previous year’s quarterly loss of $8.5 million, or $0.76 per share. This loss included an additional $1.6 million attributed to stock-based compensation expenses.
About TARA-002
TARA-002 is an experimental cell therapy being developed as a potential treatment for NMIBC and LMs; its potential has been recognized by the U.S. Food and Drug Administration, who granted it Rare Pediatric Disease Designation. TARA-002, sourced from the same master cell bank of Streptococcus pyogenes strains used in the approved Picibanil® immunopotentiator, was subjected to rigorous testing to ensure manufacturing was comparable to OK-432. All results have indicated the therapies are the same, giving promise to TARA-002 for the future.
The powerful immune cascade triggered by the administration of TARA-002 activates innate and adaptive immune cells within the cyst or tumor. Neutrophils, monocytes and lymphocytes rush in to wage a battle of inflammation, whilst interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma and tumor necrosis factor (TNF)-alpha are released to help them in their cause. By joining forces, these determined cells are ready to take down the abnormal cells and bring about a healthier body.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
Bladder cancer is an unfortunately common cancer in the United States, placing among the top six most commonly diagnosed cancers in our country with approximately 65,000 cases of Non-Muscle Invasive Bladder Cancer (NMIBC) alone diagnosed each year. NMIBC is a type of cancer that only affects the lining of the inner bladder wall without spread into the bladder muscle. Thankfully, diagnosis and treatment of this type of bladder cancer is relatively easy and offers a high rate of success.
About Lymphatic Malformations (LMs)
LMs, congenital malformations of the lymphatic vessels, often present in the head and neck region and are diagnosed in early childhood. These malformations can lead to severe morbidities and affect the ability to breathe, causing intubation and tracheostomy dependence. Intralesional bleeding, impingement on structures such as nerves and vessels, recurrent infection, and other functional and cosmetic disabilities can also occur. For babies diagnosed with LMs, proactive and timely medical management is essential to reduce the potentially severe complications of the condition.
About IV Choline Chloride
IV Choline Chloride is an innovative, intravenous phospholipid replacement therapy specifically developed for patients receiving parenteral nutrition (PN). Choline is an essential substrate for phospholipids that are vital for healthy liver function. Unfortunately, PN patients lack the ability to absorb the necessary choline and commercial PN products contain inadequate amounts to meet their needs, leading to a prolonged progression to hepatic failure and death.
IV Choline Chloride has earned approval as an Orphan Drug from the U.S. FDA for the prevention of choline deficiency in PN patients, and could be the key to saving countless PN patients from an otherwise grim prognosis.
About Protara Therapeutics, Inc.
Protara is devoted to revolutionizing the world of treatments for cancer and rare diseases. Our leading program, TARA-002, is currently being studied as a potential method of treating non-muscle invasive bladder cancer and lymphatic malformations. Furthermore, we are also researching IV Choline Chloride, an innovative phospholipid substrate replacement to aid people who rely on parenteral nutrition. We are hopeful that our work will result in major advancements for those affected by cancer and rare diseases.
Forward-Looking Statements
At Protara, we look to the future with optimism. We are developing products and clinical trials, guided by our business strategy and financial position, all with the aim of bringing safe and effective treatments to the public. Our outlook is filled with promise, with potential positive interactions with the FDA, progress in efficacy trials, and a successful financial outlook for the remainder of the year.
However, we also acknowledge that our forward-looking statements involve inherent risks. Market conditions, competitive landscapes, cash flow, management changes, and general economic issues may all affect the reality of our goals and outcomes. We remain dedicated to our mission and optimistic for the future.