Protara Therapeutics, Inc. (NASDAQ:TARA) is a clinical-stage biopharmaceutical company committed to advancing transformative therapies for the treatment of cancer and rare diseases. The company's portfolio includes two development programs utilizing TARA-002, an investigational cell therapy, and Intravenous (IV) Choline Chloride, an investigational phospholipid substrate replacement therapy.
Business Overview
Protara's lead oncology program is TARA-002 in non-muscle invasive bladder cancer (NMIBC), which represents approximately 80% of bladder cancer diagnoses in the United States. The company is conducting a Phase 1 open-label clinical trial, known as the ADVANCED-1 trial, to evaluate TARA-002 in treatment-naïve and treatment-experienced NMIBC patients with carcinoma in situ (CIS) and high-grade papillary tumors (Ta). In April 2023, Protara announced positive preliminary data from the Phase 1a dose escalation component of the ADVANCED-1 trial, indicating favorable tolerability and anti-tumor activity of TARA-002 in NMIBC patients.
In addition to the ADVANCED-1 trial, Protara is pursuing higher dosing at an 80KE dose and systemic priming prior to initiation of intravesical administration, as well as the combination of TARA-002 with a checkpoint inhibitor in NMIBC patients with CIS. The company is also conducting non-clinical studies on TARA-002 to better characterize the mechanism of action and define other cancer targets.
Protara is also developing IV Choline Chloride, an investigational phospholipid substrate replacement therapy, for patients receiving parenteral nutrition (PN). In April 2023, the company announced alignment with the FDA on a registrational path forward for IV Choline Chloride in patients dependent on PN. Protara intends to advance the development of IV Choline Chloride as a source of choline for adult and adolescent patients on long-term PN.
Furthermore, Protara is pursuing TARA-002 in lymphatic malformations (LMs), which are rare, non-malignant cysts of the lymphatic vascular system. In October 2023, the company initiated STARBORN-1, a Phase 2 clinical trial to evaluate the safety and efficacy of intracystic injection of TARA-002 for the treatment of macrocystic and mixed-cystic LMs in participants six months to less than 18 years of age.
Financials
Protara has not generated any revenue from product sales, as neither TARA-002 nor IV Choline Chloride have been approved for use. The company has incurred substantial operating losses, reporting a net loss of $11.1 million for the three months ended March 31, 2023, compared to a net loss of $9.0 million for the same period in 2022. Protara's annual net loss for the year ended December 31, 2022 was $40.4 million, with annual revenue of $0 and annual operating cash flow of -$37.6 million.
As of March 31, 2023, Protara had approximately $55.2 million in cash, cash equivalents, and marketable debt securities. On April 10, 2023, the company completed a private placement, raising approximately $42.0 million in net proceeds, which the company believes will be sufficient to satisfy its estimated liquidity needs for at least twelve months from the date of filing the quarterly report.
Protara's research and development expenses for the three months ended March 31, 2023 were $7.7 million, an increase of $2.6 million compared to the same period in 2022. This increase was primarily due to higher expenses related to clinical trial and non-clinical activities for TARA-002, as well as an increase in personnel-related expenses, partially offset by a reduction in clinical development activities for IV Choline Chloride.
General and administrative expenses for the three months ended March 31, 2023 were $4.1 million, a decrease of $0.5 million compared to the same period in 2022. This decrease was primarily due to a reduction in personnel-related expenses, including stock-based compensation.
Liquidity
Protara's liquidity and capital resources are critical to its ability to continue its development efforts. The company will need to raise additional capital in the future to fully realize its strategic plans, including the conduct of ongoing and future clinical trials and further research and development costs. As a result of volatility in the capital markets, economic conditions, and other factors, Protara may face challenges in obtaining additional financing on favorable terms, or at all.
Outlook
In terms of guidance, Protara 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 2023. The analysis is expected to include approximately 10 patients who are six-month evaluable.
Conclusion
Overall, Protara Therapeutics is a promising biopharmaceutical company with a diverse pipeline of investigational therapies targeting cancer and rare diseases. The company's focus on advancing transformative therapies, coupled with its strong cash position and ongoing clinical development efforts, make it an interesting investment opportunity for those willing to take on the risks associated with a clinical-stage biopharmaceutical company.