NEW YORK - The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Protara Therapeutics, Inc.'s (NASDAQ:TARA) investigational therapy, Intravenous (IV) Choline Chloride, for patients requiring parenteral support. This designation is aimed at expediting the development and review of drugs that treat serious conditions and fill an unmet medical need.
IV Choline Chloride is being developed as a source of choline for adult and adolescent patients who cannot receive nutrition orally or enterally. The company's CEO, Jesse Shefferman, highlighted the urgency of addressing choline deficiency in these patients, which can lead to severe health issues such as hepatic injury and neuropsychological impairment. According to Protara, around 80% of patients on parenteral support suffer from choline deficiency.
The THRIVE-3 clinical trial is scheduled to begin in the first quarter of 2025 to assess the safety and efficacy of IV Choline Chloride. The trial will be a seamless Phase 2b/3 study, including dose confirmation and a double-blinded, randomized, placebo-controlled phase. The primary endpoint will be the change in plasma choline concentration from baseline compared to placebo.
The American Society for Parenteral and Enteral Nutrition and the European Society for Clinical Nutrition and Metabolism have both recommended IV choline for patients on parenteral support. Currently, there are no approved IV choline products for these patients globally, and Protara aims to address this gap.
Protara Therapeutics focuses on developing transformative therapies for cancer and rare diseases. Their portfolio includes TARA-002, an investigational cell-based therapy, and IV Choline Chloride for patients on parenteral support.
The FDA's Fast Track program allows for more frequent interactions with the FDA during drug development, eligibility for accelerated approval and priority review, and the ability to submit sections of a New Drug Application on a rolling basis.
This news is based on a press release statement from Protara Therapeutics.
In other recent news, Protara Therapeutics has been making significant strides in the biopharmaceutical landscape. The company has reported encouraging results from its ongoing clinical trials, particularly for its investigational drug, TARA-002. Analyst firm Oppenheimer has maintained an Outperform rating on Protara shares, following positive updates from the company's research programs. Similarly, TD Cowen has also upheld a Buy rating on the stock, signaling confidence in the company's strategic direction.
Protara has revealed a high prevalence of choline deficiency among patients dependent on parenteral support, underscoring the urgent need for treatment options. In response, the company is advancing its intravenous choline chloride into a pivotal study in the first quarter of 2025. Additionally, Protara's TARA-002 has shown promising results in early-stage trials for patients with non-muscle invasive bladder cancer.
Furthermore, the company has reported positive results from its LM registrational trial, where two out of three patients showed a complete response after a single dose of TARA-002. As Protara continues its research and development, these recent developments highlight the company's commitment to addressing unmet medical needs through innovative therapies.
InvestingPro Insights
Protara Therapeutics' recent FDA Fast Track designation for IV Choline Chloride comes at a critical time for the company, as reflected in its financial metrics. According to InvestingPro data, Protara's market capitalization stands at a modest $39.4 million, indicating its current position as a small-cap biopharmaceutical company. This valuation aligns with the company's focus on developing therapies for niche markets, such as patients requiring parenteral support.
InvestingPro Tips reveal that Protara "holds more cash than debt on its balance sheet," which is crucial for a company in the resource-intensive phase of drug development. This financial cushion could be vital in supporting the upcoming THRIVE-3 clinical trial scheduled for early 2025. However, investors should note that the company is "quickly burning through cash," a common characteristic of biotech firms in the development stage.
The stock's volatility, as highlighted by another InvestingPro Tip, reflects the high-risk, high-reward nature of the biopharmaceutical sector, especially for companies with products in clinical trials. This volatility is evident in Protara's price movements, with a 34.98% price total return over the past year, despite a 27.65% decline in the last six months.
It's worth noting that analysts have revised their earnings upwards for the upcoming period, which could be interpreted as a positive signal in light of the recent FDA Fast Track designation. However, the company is not expected to be profitable this year, which is not unusual for biotech firms focused on research and development.
For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and insights. There are 7 more InvestingPro Tips available for Protara Therapeutics, which could provide valuable context for understanding the company's financial health and market position as it advances its clinical programs.
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