Protara Announces Positive Results from the Ongoing Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC
Protara Therapeutics announced positive results from its ongoing Phase 2 ADVANCED-2 trial of TARA-002 in Non-Muscle Invasive Bladder Cancer (NMIBC) patients. The trial demonstrated a 72% six-month complete response rate across BCG exposures, with notable results in both BCG-Unresponsive (100%) and BCG-Naïve (64%) patients.
Key highlights include an 80% reinduction salvage rate and 100% of patients maintaining complete response from three to six months. The study showed a favorable safety profile with no Grade 2 or greater treatment-related adverse events. The dataset included 20 patients evaluable at three months, 18 at six months, and 3 at nine months, with data cutoff on November 19, 2024.
Protara Therapeutics ha annunciato risultati positivi dal suo studio di fase 2 ADVANCED-2 in corso su TARA-002 per pazienti con carcinoma della vescica non muscolo invasivo (NMIBC). Lo studio ha dimostrato un tasso di risposta completa del 72% a sei mesi tra le esposizioni a BCG, con risultati notevoli sia nei pazienti non responsivi a BCG (100%) che in quelli naïve a BCG (64%).
I punti salienti includono un tasso di recupero della reinduzione dell'80% e il 100% dei pazienti che ha mantenuto la risposta completa da tre a sei mesi. Lo studio ha mostrato un profilo di sicurezza favorevole senza eventi avversi correlati al trattamento di grado 2 o superiore. Il dataset includeva 20 pazienti valutabili a tre mesi, 18 a sei mesi e 3 a nove mesi, con termine dei dati il 19 novembre 2024.
Protara Therapeutics anunció resultados positivos de su ensayo clínico en fase 2 ADVANCED-2 en curso sobre TARA-002 en pacientes con cáncer de vejiga no músculo invasivo (NMIBC). El ensayo mostró una tasa de respuesta completa del 72% a los seis meses entre las exposiciones a BCG, con resultados notables tanto en pacientes no responsivos a BCG (100%) como en pacientes naïve a BCG (64%).
Los aspectos más destacados incluyen una tasa de salvamento por reinducción del 80% y el 100% de los pacientes manteniendo la respuesta completa de tres a seis meses. El estudio mostró un perfil de seguridad favorable sin eventos adversos relacionados con el tratamiento de grado 2 o superior. El conjunto de datos incluyó a 20 pacientes evaluables a los tres meses, 18 a los seis meses y 3 a los nueve meses, con corte de datos el 19 de noviembre de 2024.
Protara Therapeutics는 비근육 침윤성 방광암(NMIBC) 환자를 대상으로 진행 중인 2상 ADVANCED-2 시험에서 TARA-002의 긍정적인 결과를 발표했습니다. 이 시험에서 BCG 노출에 대해 72%의 6개월 완전 반응률을 보여주었으며, BCG 비반응군(100%)과 BCG 미경험군(64%) 모두에서 주목할 만한 결과를 보였습니다.
주요 하이라이트에는 80%의 재유도 구제율과 3개월에서 6개월 사이에 완전 반응을 유지하는 환자가 100% 포함돼 있습니다. 이 연구는 2등급 이상의 치료 관련 부작용이 없는 안전성 프로필을 보여주었습니다. 데이터 세트에는 3개월에 평가 가능한 20명의 환자, 6개월에 18명, 9개월에 3명이 포함되었으며, 데이터 컷오프는 2024년 11월 19일입니다.
Protara Therapeutics a annoncé des résultats positifs de son essai de phase 2 ADVANCED-2 en cours sur TARA-002 chez des patients atteints de cancer de la vessie non musclé invasif (NMIBC). L'essai a démontré un taux de réponse complet de 72% à six mois parmi les expositions à BCG, avec des résultats notables tant chez les patients non réactifs au BCG (100%) que chez les patients naïfs de BCG (64%).
Les points clés incluent un taux de sauvetage par réinduction de 80% et 100% des patients maintenant une réponse complète de trois à six mois. L'étude a montré un profil de sécurité favorable sans événements indésirables liés au traitement de grade 2 ou plus. Le jeu de données comprenait 20 patients évaluables à trois mois, 18 à six mois et 3 à neuf mois, avec une date limite de données le 19 novembre 2024.
Protara Therapeutics gab positive Ergebnisse aus der laufenden Phase-2-Studie ADVANCED-2 zu TARA-002 bei Patienten mit nicht-muskelinvasivem Blasenkrebs (NMIBC) bekannt. Die Studie zeigte eine vollständige Ansprechrate von 72% über einen Zeitraum von sechs Monaten bei BCG-Expositionen, mit bemerkenswerten Ergebnissen sowohl bei BCG-unresponsive (100%) als auch bei BCG-naiven (64%) Patienten.
Zu den wichtigsten Punkten gehören eine Salvagerate von 80% bei der Reinduktion und dass 100% der Patienten die vollständige Ansprechrate von drei bis sechs Monaten aufrechterhalten. Die Studie wies ein günstiges Sicherheitsprofil ohne behandlungsbezogene Nebenwirkungen der Grad 2 oder höher auf. Der Datensatz umfasste 20 Patienten, die nach drei Monaten evaluiert wurden, 18 nach sechs Monaten und 3 nach neun Monaten, mit einem Datenschnitt am 19. November 2024.
- 72% six-month complete response rate across all patient groups
- 100% complete response rate in BCG-Unresponsive patients at six months
- 80% reinduction salvage rate
- 100% of patients maintained complete response from three to six months
- Favorable safety profile with no Grade 2 or greater treatment-related adverse events
- Lower response rate (64%) in BCG-Naïve patients compared to BCG-Unresponsive patients
- long-term data with only three patients evaluable at nine months
Insights
The Phase 2 ADVANCED-2 trial results for TARA-002 demonstrate exceptional promise in treating Non-Muscle Invasive Bladder Cancer. The 72% six-month complete response rate across BCG exposures is particularly impressive, with a remarkable 100% response rate in BCG-Unresponsive patients. The 80% reinduction salvage rate and durability of response are clinically significant.
The safety profile is notably strong, with no Grade 2 or greater treatment-related adverse events - a important advantage over existing treatments. The most common side effects are mild and transient, typical of bacterial immunopotentiation. This favorable safety profile, combined with high efficacy, positions TARA-002 as a potentially transformative therapy in the NMIBC treatment landscape.
These positive clinical results could significantly impact Protara Therapeutics' market position. With a market cap of
The expanded international site presence should accelerate patient enrollment, potentially reducing time to market. The favorable safety profile and ease of administration could drive strong physician adoption and market penetration if approved. Investors should note the planned mid-2025 data readout as a key catalyst for potential stock price movement.
- TARA-002 demonstrates
72% six-month landmark complete response rate and70% complete response rate at any time across BCG exposures 100% six-month landmark complete response rate and80% complete response rate at any time observed in BCG-Unresponsive patients64% six-month landmark complete response rate and67% complete response rate at any time observed in BCG-Naïve patients80% reinduction salvage rate and compelling durability observed with100% of patients maintaining a complete response from three months to six months across BCG exposures- Favorable safety and tolerability profile with no Grade 2 or greater treatment-related adverse events
- Company to host conference call and webcast today at 8:30 a.m. ET
NEW YORK, Dec. 05, 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 results from its ongoing Phase 2 open-label ADVANCED-2 trial. The trial is assessing intravesical TARA-002, the Company’s investigational cell-based therapy, in high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive or BCG-Naïve. The complete response (CR) rate across BCG exposures was
The dataset includes 20 patients who were evaluable at three months, 18 patients who were evaluable at six months and three patients who were evaluable at nine months with a data cutoff of November 19, 2024. In the pivotal cohort of the ADVANCED-2 trial in BCG-Unresponsive patients, the CR rate was
“These impressive TARA-002 results demonstrate meaningful activity in a difficult to treat patient population,” said Brian Mazzarella, MD, Vice President of Research for Urology America, and ADVANCED-2 study investigator. “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.”
“We are thrilled with these positive six-month data, which reinforce TARA-002’s potential in NMIBC, while offering a compelling product profile for physicians and patients,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We believe these encouraging data together with our international site expansion will accelerate patient enrollment, and we look forward to reporting initial data from 12-month evaluable patients in mid-2025.”
The majority of adverse events were Grade 1 and transient with no Grade 2 or greater TRAEs as assessed by study investigators. No patients discontinued treatment due to adverse events. The most common adverse events were in line with typical responses to bacterial immunopotentiation, such as flu-like symptoms. 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.
Conference Call and Webcast
Protara will host a conference call and webcast to discuss the data today at 8:30 am ET. The live call can be accessed by registering as a participant here. Upon registration, participants will receive conference call dial-in information. A live webcast of the event can be accessed by visiting the Events and Presentations section of the Company’s website: https://ir.protaratx.com. The webcast will be archived for a limited time following the presentation.
About ADVANCED-2
The Phase 2 open-label ADVANCED-2 trial is 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.
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 and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. 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-1b, IL-6, IL-12, granulocyte-macrophage colony-stimulating factor (GM-CSF) and natural killer cells. 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 sixth most common cancer in the United States, with NMIBC representing approximately
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.
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials (including reporting initial data from 12-month evaluable patients in mid-2025); statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year and future periods. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. 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'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836
FAQ
What are the Phase 2 ADVANCED-2 trial results for TARA-002 in NMIBC patients?
What is the safety profile of TARA-002 in the Phase 2 ADVANCED-2 trial?
When will Protara (TARA) report 12-month data for the ADVANCED-2 trial?
