Protara/TARA

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Clinical-stage biopharmaceutical company Protara Therapeutics Inc. (TARA:NASDAQ), which is developing transformative therapies for treating cancer and rare diseases, announced updated results from its trials of a drug for high-risk non-muscle invasive bladder cancer (NMIBC).

The results are from the company's ongoing Phase 2 open-label ADVANCED-2 trial assessing intravesical TARA-002, its investigational cell-based therapy in NMIBC patients with carcinoma in situ, or CIS (± Ta/T1), who are Bacillus Calmette-Guérin (BCG)-unresponsive or BCG-naïve.

Those results were featured over the weekend during an interactive poster session at the American Urological Association 2025 Annual Meeting in Las Vegas.

"For patients with high-risk NMIBC, there are few effective and durable therapies available other than radical cystectomy, which we know is quite difficult for patients to tolerate," said Dr. Tom Jayram, M.D., director of the Advanced Therapeutics Center at Urology Associates and ADVANCED-2 study investigator. "TARA-002 has shown impressive efficacy, safety profile, and 12-month durability in its Phase 2 trial. In the clinic, we have seen TARA-002 become easily integrated into workflow without major hurdles for the patients or staff. This combination of clinical activity and ease of use makes me optimistic about TARA-002 having a meaningful impact in clinical practice."

The data show that TARA-002 demonstrated a promising efficacy signal, particularly in BCG-unresponsive patients, achieving a 100% high-grade complete response (CR) rate at any time (5/5 patients), with durability extending to 67% (2/3 patients) at 12 months.

Among BCG-naïve patients, the high-grade CR rate at any time was 76% (16/21), with a 62.5% CR rate at six and nine months, and 43% maintained at 12 months.

Potential to 'Make a Meaningful Difference' in Lives

According to a release by the company on April 26, the majority of adverse events during the study were Grade 1 and transient with no Grade 3 or greater treatment-related adverse events (TRAEs) as assessed by study investigators. No patients discontinued treatment due to TRAEs.

The most common adverse events were in line with typical responses to bacterial immunopotentiation, such as flu-like symptoms, Protara said. The most common urinary symptoms reflect urinary tract instrumentation effects, such as bladder spasm, burning sensation, and urinary tract infection. Most bladder irritations resolved shortly after administration or within a few hours to a few days.

"The durable results shared today continue to support our conviction that TARA-002 has the potential to make a meaningful difference in the lives of patients with NMIBC," Chief Executive Officer Jesse Shefferman said. "Notably, we are particularly pleased with the competitive 12-month CR rates observed in the registrational BCG-Unresponsive cohort as well as the BCG-Naïve cohort. We look forward to continuing to advance this important trial as we work toward our mission of bringing transformative therapies to patients."

"The company said it expects to present an interim update with results from approximately 25 six-month evaluable BCG-unresponsive patients by the end of 2025," noted analysts Leland Gershell and Rohan Mathur in an updated research note on the company for Oppenheimer & Co. on April 27.

"Recall that the BCG-unresponsive cohort is positioned to drive FDA registration, and TARA reiterated its expectation to report 6-month data on ~25 patients in this cohort by year-end," wrote the analysts, who rated the stock Outperform. "We are optimistic that this larger and more mature dataset will further solidify the case for -002."

The analysts continued, "Key opinion leader (KOL) input we received at the conference that considered the reveal along with -002's low-burden handling/administration increases our enthusiasm for this candidate's prospects within the evolving NMIBC therapeutic landscape."

'Significant Room for Multiple Players'

Analysts Andres Y. Maldonado and Andrew S. Fein of HCW Wainwright & Co. also wrote in an April 28 research note that they had attended Protara's session at the conference.

"Overall, we continue to view TARA-002 as the best-in-class earlier-stage asset capable of filling gaps that cretostimogene and TAR-200 may leave behind, while also carving an additional path in the maintenance setting," wrote the analysts, who rated TARA as a Buy with a US$23 per share target price.

Following the poster presentation, the analysts said they met with Protara’s management team, who "emphasized TARA-002’s overall efficacy profile and positioned the program as a meaningful entrant into the intravesical immunotherapy space."

Maldonado and Fein wrote that managers acknowledged that CG Oncology's cretostimogene and Johnson & Johnson's (JNJ:NYSE) TAR-200 have dominated much of the early attention in NMIBC but pointed out that the 12-month CR rates for both programs top out around 46%, "leaving significant room for multiple players."

"Moreover, management noted that in real-world practice, the 46% landmark CR rates for these agents are expected to decline further, continuing to (expand) the opportunity for TARA-002 to establish itself in a market with substantial unmet need," the analysts said. "Importantly, management also reiterated that these are still early days for TARA-002, and while the data to date are encouraging, continued maturation of 12- and 24-month durability will be critical to fully realize the therapy's commercial potential."

The Catalyst: Cancer Is 2nd Leading Cause of Death Worldwide

According to the World Health Organization (WHO), cancer is presently the second leading cause of death worldwide, research by Roots Analysis said.

"As per GLOBOCAN, the number of patients diagnosed with various types of cancer in the U.S. crossed the 19 million-mark in 2020," the firm wrote. "By 2030, it is estimated that the aforementioned number is likely to grow to over 22 million."

Conventional approaches, such as chemotherapy, surgery and radiation therapy, have been found to be inadequate, especially for late-stage cancers.

According to a report by Straits Research, the global cancer biologics market size was valued at US$114.8 billion in 2023 and is projected to reach US$205.7 billion by 2032, registering a CAGR of 6.7% during the forecast period (2024-2032).

The global cancer biologics market share is predicted to expand significantly over the forecast period as regulatory bodies approve more biologic drugs for cancer therapy. 

"Cancer biologics, or biopharmaceuticals or biologics, are medicines used to treat cancer," the report said. "These medications are made from living organisms or their components, such as proteins, antibodies, nucleic acids, and living cells. Unlike standard chemotherapy medications, which are produced chemically, cancer biologics are designed to target specific molecular pathways involved in cancer growth, progression, and immune response."

The emphasis on tailored medications, which represents a change from the old one-size-fits-all strategy, is considerably driving market growth, Straits noted. Personalized medicine, which is personalized to each cancer patient's unique genetic and molecular traits, provides more targeted and effective therapies, especially given cancer's heterogeneity.

Ownership and Share Structure

According to Refinitiv, about 4% of the company is owned management and insiders, and 81% is owned by institutions. The rest is retail.

Top shareholders include Janus Henderson Investors with 9.82%, RA Capital Management LP with 8.81%, Adage Capital Management LP with 5.83%, TD Securities Inc. with 5.41%, and Opaleye Management Inc. with 4.48%.

Its market cap is US$$135.8 million with 38.58 million shares outstanding. It trades in a 52-week range of US$1.60 and US$10.48.