Picibanil

Protara Therapeutics reported that 13 of 18 bladder cancer patients who received its experimental cell therapy saw no signs of their cancer at six months. Two of three patients who received Protara’s treatment, called TARA-002, and were followed for nine months maintained a complete response, according to data shared Thursday by the biotech at the Society of Urologic Oncology’s annual meeting. The open-label Phase 2 study included patients with high-risk non-muscle invasive bladder cancer, meaning that their cancer is in the bladder lining but has not grown into the bladder muscle. The patients in the study were either unresponsive to or had not tried Bacillus Calmette-Guérin (BCG), the standard treatment for this cancer. Protara’s shares $TARA jumped 120% from a closing price of $3.64 on Wednesday to about $7.74 on Thursday morning. As of the Nov. 19 cutoff, the study had 20 patients with at least three months of follow-up, 14 of whom had a response at any time in the study. “The activity of TARA-002 across BCG exposures, coupled with its ease of use and low procedural burden for physicians, make it an exciting potential treatment option for NMIBC patients,” Brian Mazzarella, an investigator on the study and VP of research for Urology America, said in a statement. In five patients who previously received BCG but were unresponsive, four had a complete response to TARA-002 at any time in the study, while 10 of 15 of those who were in the pivotal cohort of BCG-naïve patients had a complete response at any time. In the ADVANCED-2 study, there were no grade 2 or greater adverse events, and no patients discontinued treatment due to adverse events, Protara said. TARA-002 uses an inactivated form of the bacteria Streptococcus pyogenes , which comes from the same master cell bank as Chugai’s Picibanil, a treatment that is approved in Japan and Taiwan to treat certain cancers and benign cysts. In bladder cancer, the goal of the therapy is to both kill tumor cells and stoke an immune response against the cancer. Protara is one of several biopharma companies developing new treatments for this form of bladder cancer. The standard treatment, BCG, is manufactured by Merck and has faced repeated shortages in the past decade. Earlier this year, the FDA approved ImmunityBio’s Anktiva. CG Oncology also reported Phase 3 data from its own experimental oncolytic virus treatment on Thursday morning. And enGene is likewise developing a treatment , with plans to apply for FDA approval in 2026.

NEW YORK, Nov. 15, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that data from an interim analysis of the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) will be featured during a poster session at the upcoming 25th Annual Meeting of the Society of Urologic Oncology (SUO) taking place December 4, 2024 to December 6, 2024, in Dallas, Texas. The presentation will include safety data featured in the abstract published today on the SUO website, as well as updated safety and new efficacy data from approximately 20 enrolled patients, the majority of whom are six-month evaluable. ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial 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. Trial subjects received an induction with or without a reinduction course of six weekly intravesical instillations of TARA-002, followed by a maintenance course of three weekly installations every three months in the BCG-Unresponsive cohort. Details of the poster presentation are as follows: Title: ADVANCED-2: Phase 2 Open-Label Study to Evaluate Safety and Anti-Tumor Activity of Intravesical Instillation of TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer Poster Number: 119Poster Category: NMIBCSession Title: Bladder Cancer Session Date and Time: Thursday, December 5, 2024, 1:15 p.m. – 2:15 p.m. CT 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 and also approved in Taiwan. 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 Protara Therapeutics, Inc. Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com. 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All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law. Company Contact: Justine O'MalleyProtara TherapeuticsJustine.OMalley@protaratx.com646-817-2836