On December 5, 2024 Wugen, Inc., a clinical-stage U.S. biotechnology company developing allogeneic, off-the-shelf cell therapies for the treatment of hematological and solid tumor malignancies, reported upcoming scientific presentations, including updates from the company’s Phase 1/2 study of WU-CART-007 (Press release, Wugen, DEC 5, 2024, View Source [SID1234648831]). Researchers will present Phase 1/2 updates and studies for its investigational cell therapies this week at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, Dec. 7-10 in San Diego.

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Results from Phase 1/2 showed continued anti-leukemic activity and clinically manageable safety in adults and adolescents. WU-CART-007 is an investigational, potential first-in-class, allogeneic, anti-CD7 CAR-T cell therapy under evaluation for treatment of patients with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL).

Based on findings to date with WU-CART-007, including this week’s data, Wugen will initiate the first pivotal Phase 2 study for an off-the-shelf CD7-targeted CAR-T cell therapy.

"Maturing evidence, including the Phase 1/2 study being presented, suggest WU-CART-007 has the potential to improve current standard of care for patients with these blood cancers. T-ALL/LBL are malignancies associated with high rates of relapse and mortality in patients, many of whom are young," said Armin Ghobadi, M.D, professor of medicine and clinical director of Center for Gene and Cellular Immunotherapy (CGCI) at the Washington University School of Medicine in St. Louis.

"At this year’s ASH (Free ASH Whitepaper) meeting, we will present clinical data from adult and adolescent patients that support our soon to open pivotal trial for patients with relapsed or refractory T-ALL/LBL," said Wugen Chief Medical Officer, Cherry Thomas, M.D. "We are looking forward to working with experienced investigators to provide meaningful clinical benefit to patients with limited treatment options."

The pivotal Phase 2 study is a single-arm trial evaluating the efficacy and safety of WU-CART-007 in patients with R/R T-ALL/LBL and T-ALL/LBL. The study will involve two groups: a R/R cohort and subsequently an exploratory minimal residual disease (MRD)-positive cohort. The trial will enroll pediatric and adult patients at oncology centers in the United States, Europe, Asia and Australia.

Wugen Presentations at ASH (Free ASH Whitepaper)

3450 WU-CART-007 (WT-7), an Allogeneic CAR T-Cell Targeting CD7 in Relapsed/Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/Lymphoma (T-ALL/LBL): Phase 2 Results

Presenter: Armin Ghobadi, M.D., Washington University School of Medicine, St. Louis

Presentation type: Poster

Time/location: Sunday, Dec. 8, 6:00-8:00 p.m. PT; Halls G,H
Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II

3461 WU-CART-007 (WT-7) Is an Effective Treatment for Adolescent Patients with Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL) – Subgroup Analysis of WU-CART-007 1001

Presenter: Shannon L. Maude, M.D., Division of Oncology, Center for Childhood Cancer Research and Cancer Immunotherapy Program, Children’s Hospital of Philadelphia, Philadelphia

Presentation type: Poster

Time/location: Sunday, Dec. 8, 6:00-8:00 p.m. PT; Halls G,H
Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster II

Wugen researchers will also present the following abstracts for the company’s investigational memory natural killer cell therapy, WU-NK-101:

916 W-NK1 Choreographs Innate and Adaptive Immune Responses to Provide a Robust and Durable Anti-AML Response

Presenter: Tom Leedom, Wugen, Inc., St. Louis

Presentation type: Oral

Time/location: Monday, Dec. 9, 3:30 p.m. PT; Marriott Grand Ballroom 8-9, Marriott Marquis San Diego Marina

Session: 703. Cellular Immunotherapies other than CAR-T Cells: Basic and Translational: Enhancing NK Cell Therapeutics Hematology Disease Topics & Pathways: Research, Translational Research 2:45-4:15 p.m. PT

4257 WUN101-01: First in Human (FIH) Phase 1 Study of WU-NK-101 (W-NK1) in Patients with Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)

Presenter: Amanda F. Cashen, M.D., Washington University School of Medicine, St. Louis

Presentation type: Poster

Time/location: Monday, Dec. 9, 6:00-8:00 p.m. PT; Halls G, H

Session: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster III

Hematology Disease Topics & Pathways: Research, Clinical trials, Translational Research, Clinical Research

Investigator-initiated research

2066 Phase 1 Dose-Escalation and Dose-Expansion Study to Evaluate the Safety and Tolerability of Anti-CD7 Allogeneic CAR T-Cells (WU-CART-007) in Patients with CD7+ T-Cell Non-Hodgkin Lymphoma

Presenter: Michael H. Kramer, M.D., Ph.D., Washington University School of Medicine, Saint Louis

Presentation type: Poster

Time/location: Sat., Dec. 7, 5:30-7:30 p.m. PT; Halls G, H

Session: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster I;

About WU-CART-007

WU-CART-007 is an allogeneic, off-the-shelf, fratricide-resistant CD7-targeted CAR-T cell therapy engineered to overcome the technological challenges of harnessing CAR-T cells to treat CD7+ hematological malignancies. Wugen is deploying CRISPR/Cas9 gene editing technology to delete CD7 and the T cell receptor alpha constant (TRAC), preventing CAR-T cell fratricide and mitigating the risk of graft-versus-host-disease (GvHD). WU-CART-007 is manufactured using healthy donor-derived T cells to eliminate the risk of malignant cell contamination historically observed in the autologous CAR-T setting. WU-CART-007 is currently being evaluated in a global Phase 1/2 clinical trial for the treatment of relapsed or refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL)/lymphoblastic lymphoma (LBL). More information on the Phase 1/2 trial is available on clinicaltrials.gov, identifier NCT# 04984356 and on the Phase 2 pivotal trial on clinicaltrials.gov, identifier NCT06514794.

WU-CART-007 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration and Priority Medicines (PRIME) Scheme designation in the European Union for the treatment of relapsed/refractory (R/R) T cell acute lymphoblastic leukemia (T-ALL) and T cell lymphoblastic lymphoma (T-LBL). RMAT and PRIME designations provide increased agency support to expedite the development and review of promising therapies for patients in need.

On December 5, 2024 Sysmex Corporation (HQ: Kobe, Japan; President: Kaoru Asano) and Japan Tissue Engineering Co., Ltd. (HQ: Gamagori, Aichi Prefecture; President & CEO: Ken-ichiro Hata; hereinafter "J-TEC") reported that they signed a basic agreement ("the Agreement") on December 3, 2024, with the aim of advancing (i.e., mechanizing and automating) manufacturing capabilities for regenerative medical products by utilizing innovative technologies, in order to accelerate the industrialization of regenerative medicine and cell therapy and to enhance sustainability (Press release, Sysmex, DEC 5, 2024, View Source [SID1234648830]). Going forward, both companies will start concrete strategic collaboration based on this Agreement.
One of the major challenges in the industrialization of regenerative medicine and cell therapy is the difficulty of manufacturing products from living cells. In particular, autologous cell products1 derived from patients’ own cells need to be manufactured to accommodate the non-uniform character of the cells from each patient. As a result, it is extremely challenging to mechanize and automate the manufacturing processes in a standardized manner. This creates a bottleneck in expanding the scale of production and enhancing efficiency in the regenerative medicine and cell therapy industry, and the transfer of manual manufacturing techniques therefore becomes an issue that directly affects the sustainability of the business.

Sysmex has contributed to the evaluation of cell functions by providing quality control testing2 capable of non-destructively analyzing cell characteristics, as well as Internet of Things (IoT) and other robotic technologies, to academic institutions and pharmaceutical companies that develop regenerative medical products. The company has also been working on automating manufacturing processes for the pipeline of our group companies and strategic partners.

J-TEC was the first company in Japan to develop and obtain approval of regenerative medical products, making it a pioneer in regenerative medicine. In particular, it has been working to commercialize and utilize autologous cell products in society. Based on the experience and knowledge gained in this process, the company has established a platform to stably supply products while maintaining high quality.

Both companies have been discussing ways to leverage their individual strengths to solve issues in the regenerative medicine and cell therapy industry, based on the technology and know-how they have developed through their respective business activities. As part of this, a basic agreement was signed on December 3, 2024, with the aim of enhancing manufacturing capabilities for regenerative medicine and cell therapy through innovative technologies.

Through open innovation based on the strengths of both companies, they will contribute to the sustainable development of Japan’s regenerative medicine and cell therapy industry by realizing the mechanization and automation of manufacturing processes for regenerative medical products, which is a key issue in the regenerative medicine and cell therapy industry.
Terminology
1
Autologous cell products:
Regenerative medical products manufactured from patients’ own living cells.

2
Quality testing that allows non-destructive cell analysis:
Testing that enables the analysis of cell quality without destroying the cells themselves. Since the cells do not need to be destroyed, it allows for the efficient and accurate testing of cell products in a short period of time.

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On December 5, 2024 Pfizer Inc. (NYSE: PFE) reported that it will highlight the latest advancements from its growing hematology and breast cancer portfolios at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (December 7-10) and the San Antonio Breast Cancer Symposium (SABCS, December 10-13) (Press release, Seagen, DEC 5, 2024, View Source [SID1234648829]). Data from more than 100 company-sponsored, investigator-sponsored, and collaborative research abstracts, including 13 oral presentations and four poster spotlights, will be shared across the company’s approved medicines and expanding portfolio of potential breakthroughs for patients with blood and breast cancers, as well as rare blood disorders.

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"Our robust presence at ASH (Free ASH Whitepaper) and SABCS reinforces Pfizer’s legacy of scientific innovation for people living with blood disorders and breast cancer," said Chris Boshoff, Chief Oncology Officer and Executive Vice President, Pfizer. "We are pleased to share the latest updates for some of our key approved medicines, including ADCETRIS, ELREXFIO, and IBRANCE, which continue to generate compelling data as foundations of care in their respective indications. We are also excited to present new results in hemophilia and from our expanding pipeline of innovative, next-generation therapy candidates for both blood and breast cancers, including new data on combination approaches across our core scientific modalities."

Key ASH (Free ASH Whitepaper) Presentations

Data from more than 75 company-sponsored, investigator-sponsored, and collaborative research abstracts will be presented at ASH (Free ASH Whitepaper), including updated analyses from the pivotal ECHELON-3 trial supporting the clinical benefit of ADCETRIS (brentuximab vedotin) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). New data for ELREXFIO (elranatamab-bcmm) in relapsed/refractory multiple myeloma (RRMM) will also be presented from the pivotal MagnetisMM-3 trial, as well as Phase 1 combination data from the MagnetisMM-20 trial. Pfizer will also present updates from its growing Hematology-Oncology pipeline, which includes next-generation CD30 antibody-drug conjugates and other novel and differentiated molecules, including the first presentation of combination data for SEA-CD70 in high-risk myelodysplastic syndromes.

Additionally, Pfizer will present results across its portfolio of investigational and approved medicines in benign hematology.

ADCETRIS: Additional analyses from the Phase 3 ECHELON-3 trial will be presented, highlighting the durability of complete responses and consistent benefit of ADCETRIS in combination with lenalidomide and rituximab in patients with relapsed/refractory DLBCL, including enrollment of elderly patients, those who are refractory to most recent treatment, and those who have received prior CAR-T therapy. These findings also underscore the overall survival (OS) advantage over lenalidomide and rituximab plus placebo in patients who have received at least two prior lines of therapy. In addition, updated two-year follow-up data from a Phase 2 study investigating the combination of ADCETRIS, nivolumab, doxorubicin, and dacarbazine in newly diagnosed early-stage classical Hodgkin lymphoma (cHL) will be presented highlighting promising efficacy and safety of this investigational novel combination.
ELREXFIO: A post hoc analysis of the Phase 2 MagnetisMM-3 trial continues to show deep and durable responses after longer-term follow-up of nearly three years, and these responses were also maintained with a reduction to once-monthly dosing in RRMM. Data will also be shared from the ongoing Phase 1b MagnetisMM-20 trial that indicate encouraging clinical efficacy and predictable safety signals with ELREXFIO in combination with carfilzomib and dexamethasone after a median of two prior lines of therapy (range: one to three).
SEA-CD70 (PF-08046040): Encouraging preliminary data from the ongoing Phase 1 study with PF-08046040, also known as SEA-CD70, a nonfucosylated monoclonal antibody targeting CD70 that is designed to enhance effector function, will be shared for the first time from the combination dose-optimization cohort with azacitidine in patients with higher-risk myelodysplastic syndromes (MDS). SEA-CD70 is being developed with the goal of being a best-in-class foundational medicine either alone or as combination treatment in myeloid malignancies.
Key SABCS Presentations

Data from 30 company-sponsored, investigator-sponsored, and collaborative research abstracts will be presented at SABCS, including nine real-world analyses affirming IBRANCE (palbociclib) as a first-line standard-of-care treatment for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer (MBC). The company will also present new data from its expanding pipeline of innovative, next-generation therapy candidates that have the potential to address critical unmet patient needs across all subtypes and stages of breast cancer, including new and updated Phase 1 data for atirmociclib, vepdegestrant, and the novel KAT6 inhibitor, PF-07248144.

IBRANCE : In P-VERIFY, the largest-ever real-world comparative overall survival analysis of first-line CDK4/6 inhibitors plus aromatase inhibitor (AI) therapy in HR+/HER2- MBC, numerically similar overall survival rates were observed across CDK4/6 inhibitor groups at 12, 24, and 30 months.
Atirmociclib (PF-07220060): Updated data will be presented from a Phase 1/2a study of atirmociclib, a next-generation, highly selective CDK4 inhibitor, in combination with letrozole as a potential first-line treatment for patients with HR+/HER2- MBC. Atirmociclib is being developed as a potential future CDK inhibitor backbone therapy in HR+ MBC, and a Phase 3 study in the first-line setting is anticipated to start by early 2025.
Vepdegestrant: For the first time, initial Phase 1b data will be shared from the Phase 1b/2 TACTIVE-U trial evaluating the combination of vepdegestrant, a potential first-in-class PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, in combination with abemaciclib in patients with ER+/HER2- locally advanced or MBC. These data reinforce the potential of vepdegestrant as a new backbone endocrine therapy, and Pfizer and Arvinas anticipate topline Phase 3 data evaluating vepdegestrant as a monotherapy in the first quarter of 2025.
KAT6 (PF-07248144) : Updated efficacy and safety data will be presented from a Phase 1 dose-expansion study of PF-07248144, a novel KAT6 inhibitor, in heavily pretreated ER+/HER2- MBC. These early data continue to provide strong clinical proof of concept for this novel target as a potential new treatment approach for ER+/HER2- MBC, and Pfizer anticipates initiating a Phase 3 study for PF-07248144 in the post-CDK4/6 inhibitor setting in mid-2025.
Additional information on key Pfizer-sponsored abstracts at ASH (Free ASH Whitepaper) and SABCS, including date and time of presentation, follow in the chart below. A complete list of Pfizer-sponsored accepted abstracts is available here: View Source

Blood Cancers

Updated Analysis of Brentuximab Vedotin, Nivolumab, Doxorubicin, and Dacarbazine for Nonbulky, Early-Stage Classical Hodgkin Lymphoma (Abstract #460)

Abramson J

Oral Presentation

Sunday, December 8, 9:30-11:00 AM PST

Presentation Time: 10:15 AM PST

Efficacy of Elranatamab (ELRA) in Combination with Carfilzomib (CFZ) and Dexamethasone (DEX) in the Phase 1b MagnetisMM-20 Trial in Relapsed or Refractory Multiple Myeloma (RRMM) (Abstract #1024)

Tomasson H

Oral Presentation

Monday, December 9, 4:30-5:30 PM PST

Presentation Time: 5:15 PM PST

PF-08046040 (SEA-CD70), a Nonfucosylated CD70-Directed Antibody, in Combination with Azacitidine for Patients with Myelodysplastic Syndromes (MDS): A Phase 1 Dose-Finding and Dose Expansion Study (Abstract #1840)

Poster Presentation

Saturday, December 7, 5:30-7:30 PM PST

Durability of Complete Responses in Patients from the ECHELON-3 Study (Abstract #3101)

Yasenchak C

Poster Presentation

Sunday, December 8, 6:00-8:00 PM PST

MagnetisMM-3: Long-Term Update and Efficacy and Safety of Less Frequent Dosing of Elranatamab in Patients with Relapsed or Refractory Multiple Myeloma (Abstract #4738)

Prince M

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Outcomes in Older Patients with Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) from the ECHELON-3 Study (Abstract #4483)

Bartlett N

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Outcomes by Refractory Status and Prior Therapies Received in Patients with Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) from the ECHELON-3 Study (Abstract #4489)

Hahn U

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Hemophilia

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 AFFINE Gene Therapy Trial (Abstract #1053)

Leavitt AD

Oral Presentation

Monday, December 9, 4:30-6:00 PM PST

Presentation Time: 5:00 PM PST

Descriptive Characterization of Bleeding Events in Participants with Severe Hemophilia Α or B without Inhibitors, Receiving Prophylactic Marstacimab Treatment (Abstract #716)

Matino D

Oral Presentation

Monday, December 9, 10:30 AM-12:00 PM PST

Presentation Time: 10:45 AM PST

Sickle Cell Disease

Qualitative Interview Study to Characterize the Treatment Experiences of Participants with Sickle Cell Disease and Assess Perceptions of Red Blood Cell Transfusions (Abstract #3691)

Kosa K

Poster Presentation

Sunday, December 8, 6:00-8:00 PM PST

Breast Cancer

Comparative overall survival of CDK4/6is plus an aromatase inhibitor (AI) in HR+/HER2- MBC in the US real-world setting

Rugo et al

Poster Spotlight Presentation (PS2-03)

Thursday, December 12, 7:00-8:30 AM CST

PF-07248144, a first-in-class KAT6 inhibitor, in patients with HR+ HER2− metastatic breast cancer: Updated results from phase 1 dose expansion study

Mukohara et al

Poster Presentation (P4-10-28)

Thursday, December 12, 5:30-7:00 PM CST

Vepdegestrant, a PROteolysis TArgeting Chimera (PROTAC) Estrogen Receptor (ER) Degrader, Plus Abemaciclib in ER-Positive/Human Epidermal Growth Factor Receptor 2 (HER2)-Negative Advanced or Metastatic Breast Cancer: TACTIVE-U Preliminary Phase 1b Results

Hilton et al

Poster Presentation ​(P4-12-03)

Thursday, December 12, 5:30-7:00 PM CST

The next-generation CDK4-selective inhibitor atirmociclib (PF-07220060) in combination with letrozole as first-line treatment in patients with HR+/HER2+ metastatic breast cancer

Giordana et al

Poster Presentation (P5-07-28)

Friday, December 13, 12:30-2:00 PM CST

On December 5, 2024 Purple Biotech Ltd. ("Purple Biotech" or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that overcome tumor immune evasion and drug resistance, reported the closing of its previously announced registered direct offering of 472,668 of the Company’s American Depositary Shares ("ADSs"), each ADS representing 200 ordinary shares, at a purchase price of $6.00 per ADS (Press release, Purple Biotech, DEC 5, 2024, View Source [SID1234648828]).

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The gross proceeds to the Company from the offering are approximately $2.8 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. Purple Biotech intends to use the net proceeds from the offering to fund the development of its oncology therapeutic candidates and for general working capital and corporate purposes.

The ADSs described above were offered by Purple Biotech pursuant to a "shelf" registration statement on Form F-3 (File No. 333-268710) previously filed with the U.S. Securities and Exchange Commission (the "SEC") on December 8, 2022, and declared effective by the SEC on May 22, 2023. The offering of the ADSs in the offering was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 865-5711 or e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On December 5, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported results from its ongoing Phase 2 open-label ADVANCED-2 trial (Press release, Protara Therapeutics, DEC 5, 2024, View Source [SID1234648827]). 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 72% (13/18) at six months and 70% (14/20) at any time with 100% (9/9) of patients maintaining a CR from three months to six months. In addition, two of three patients maintained a CR at nine months. These results will be featured today during a poster session at the 25th Annual Meeting of the Society of Urologic Oncology (SUO) in Dallas, Texas.

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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 100% (4/4) at six-months and 80% (4/5) at any time. In the proof-of-concept cohort of BCG-Naïve patients, the CR rate was 64% (9/14) at six months and 67% (10/15) at any time. TARA-002 demonstrated a favorable safety and tolerability profile with no Grade 2 or greater treatment-related adverse events (TRAEs), and no patients discontinued due to adverse events.

"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: View Source 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 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.