On November 12, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the third quarter ended September 30, 2024, and provided a corporate update (Press release, TScan Therapeutics, NOV 12, 2024, View Source [SID1234648219]).

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"As we approach the end of the year, we remain committed to advancing our clinical-stage pipeline across both heme and solid tumor malignancies and providing an update on the ALLOHA Phase 1 trial following ASH (Free ASH Whitepaper). We are encouraged to see that none of the 16 patients on the treatment arm relapsed, including five patients at least one-year post-transplant as of the July 8th abstract cutoff date. We look forward to sharing updated data, including several additional patients, at ASH (Free ASH Whitepaper)," said Gavin MacBeath, Ph.D., Chief Executive Officer. "During the third quarter we continued to prioritize screening, enrolling, and dosing patients in the solid tumor program and remain on track to dose our first patient with multiplex therapy and provide an update on our Phase 1 study by the end of the year."

Recent Corporate Highlights

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The Company recently announced an upcoming oral presentation at the 66th ASH (Free ASH Whitepaper) Annual Meeting. The data in the abstract included 16 treatment-arm patients and 11 control-arm patients with a data cutoff of July 8, 2024. No dose limiting toxicities were observed across all treatment-arm patients and the safety profile was generally consistent with hematopoietic cell transplantation (HCT). All treatment-arm patients (16 of 16) were relapse-free and minimal residual disease (MRD)-negative as of the data cutoff, whereas three control-arm patients (3 of 11) relapsed, two of whom died from their disease. These data support both the safety and potential of TSC-100 and TSC-101 to reduce relapses and increase relapse-free survival in patients receiving reduced intensity conditioning HCT. Updated data will be presented at the annual meeting.

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The Company will host a virtual KOL event featuring Ran Reshef, M.D., M.Sc., on Tuesday, December 10th, at 8:00 a.m. ET to discuss the data presented at the ASH (Free ASH Whitepaper) Annual Meeting. The Company will also discuss its clinical development strategy for the heme program. Dr. Reshef is the Professor of Medicine and Director of the Cellular Immunotherapy Program at Columbia University Irving Medical Center. Additional details around the call will be provided closer to the event. Registration for the event can be found here.

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The Company recently increased its internal manufacturing capacity as well as identified a global contract development and manufacturing organization (CDMO) with commercial capabilities to support both the heme
and solid tumor programs. The Company is on track to transfer the commercial heme manufacturing process to the CDMO in 2025.

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The Company recently presented three posters at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting held in Houston, TX and virtually:

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Discovery of a MAGE-A4-specific TCR-T Therapy Candidate for Multiplex Treatment of Solid Tumors
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Preclinical Models for T-Plex, a Customized Multiplexed TCR-T Cell Therapy Addressing Intra-Tumor Antigen and HLA Heterogeneity
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Development of a Target Agnostic Platform to Assess the Reactivity of T Cell Receptor (TCR)-Engineered T Cell (TCR-T) Therapies to Primary Human Tissues

Copies of the presentation materials can be found under the "Publications" section of the Company’s website at tscan.com.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s two lead TCR-T therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic syndrome (MDS) undergoing allogeneic HCT (the ALLOHATM trial, NCT05473910).

G&A Expenses: General and administrative expenses for the third quarter of 2024 were $7.4 million, compared to $5.9 million for the third quarter of 2023. The increase of $1.5 million was primarily driven by an increase in personnel expenses due to increased headcount to support business activities. General and administrative expenses included non-cash stock compensation expense of $1.3 million and $0.4 million for the third quarter of 2024 and 2023, respectively.

Net Loss: Net loss was $29.9 million for the third quarter of 2024, compared to $23.0 million for the third quarter of 2023, and included net interest income of $2.7 million and $1.8 million, respectively.

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2024, were $271.1 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources will continue to fund its current operating plan into the fourth quarter of 2026.

Share Count: As of September 30, 2024, the Company had issued and outstanding shares of 53,354,124, which consists of 49,077,536 shares of voting common stock and 4,276,588 shares of non-voting common stock, and outstanding pre-funded warrants to purchase 65,587,945 shares of voting common stock at an exercise price of $0.0001 per share.

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Plans to open expansion cohorts at the proposed recommended Phase 2 dose level to further characterize safety and evaluate translational and efficacy endpoints by the end of 2024.
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One-year clinical and translational data on initial patients to be reported by the end of 2024.
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Initiate a registration trial, pending feedback from regulatory authorities, and plans to report two-year clinical and translational data in 2025.

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of therapeutic TCR-Ts that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-Ts to overcome tumor heterogeneity and prevent resistance that may arise from either target or HLA loss (screening protocol: NCT05812027; treatment protocol: NCT05973487).

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Actively screening, enrolling, and dosing patients across the TCR-T therapy candidates.
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Update on solid tumor program expected by the end of 2024.
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Investigational new drug (IND) filing for TCR targeting MAGE-A4 on HLA-A*02:01 (TSC-202-A0201) planned by the end of the year.
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Response data for multiplex therapy anticipated in 2025.

Third Quarter 2024 Financial Results

Revenue: Revenue for the third quarter of 2024 was $1.0 million, compared to $3.9 million for the third quarter of 2023. The decrease was primarily due to timing of research activities pursuant to the Company’s collaboration agreement with Amgen which commenced in May 2023.

R&D Expenses: Research and development expenses for the third quarter of 2024 were $26.3 million, compared to $22.7 million for the third quarter of 2023. The increase of $3.5 million was primarily driven by an increase in clinical studies expense associated with the ongoing enrollment of our ALLOHA Phase 1 heme trial and start-up activities and initial enrollment in our Phase 1 solid tumor clinical trial, as well as an increase in personnel expenses due to additional headcount in support of our expanded research and development activities. Research and development expenses included non-cash stock compensation expense of $1.2 million and $0.9 million for the third quarter of 2024 and 2023, respectively.

On November 12, 2024 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported a business update and announced financial results for the third quarter ended September 30, 2024 (Press release, Lisata Therapeutics, NOV 12, 2024, View Source [SID1234648161]).

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"We are pleased to share the progress made in the third quarter of 2024, highlighted by the advancement of our robust development portfolio centered around our novel product candidate, certepetide," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Lisata. "While we await preliminary results from Cohort A of the Phase 2b ASCEND trial expected this quarter, we continue to explore the broad application of certepetide’s unique mechanism of action. Our development portfolio now encompasses multiple clinical and preclinical trials evaluating certepetide for the treatment of various solid tumors, including pancreatic, cholangiocarcinoma, glioblastoma, colon, appendiceal, and melanoma. In addition, certepetide is being evaluated in a preclinical non-cancerous setting for endometriosis. All our studies are designed to yield data during the coming year, and we look forward to a data-rich 2025."

Development Portfolio Highlights

Certepetide as a treatment for solid tumors in combination with other anti-cancer agents

Certepetide is an investigational drug designed to selectively activate the C-end rule active transport mechanism in a tumor specific manner, resulting in systemically co-administered anti-cancer agents more efficiently penetrating and accumulating in the tumor. Additionally, certepetide has been shown to modify the tumor microenvironment, diminishing its immunosuppressive nature, enhancing cytotoxic T cell concentration and inhibiting the metastatic cascade. Lisata and its collaborators have amassed significant non-clinical data demonstrating enhanced delivery of various existing and emerging anti-cancer therapies, including chemotherapies, immunotherapies, and RNA-based therapeutics. To date, certepetide has also demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing clinical trials designed to demonstrate its ability to enhance the effectiveness of standard-of-care ("SoC") chemotherapy for pancreatic cancer as well as the combination of chemotherapy and immunotherapy in a variety of solid tumors. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.) as well as Orphan Drug Designation for glioma, osteosarcoma, and cholangiocarcinoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). Currently, certepetide is the subject of multiple ongoing or planned Phase 2a and 2b clinical studies being conducted globally in a variety of solid tumor types in combination with a variety of anti-cancer regimens, including:

•ASCEND: Phase 2b double-blind, randomized, placebo-controlled clinical trial evaluating two dosing regimens of certepetide in combination with SoC chemotherapy (gemcitabine/nab-paclitaxel) in patients with metastatic pancreatic ductal adenocarcinoma ("mPDAC"). The trial is being conducted at 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Trials Group ("AGITG") in collaboration with the University of Sydney and with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney as the Coordinating Centre. Following the completion of enrollment in the fourth quarter of 2023, data from an interim analysis of the 95 Cohort A patients (single dose of certepetide administered with SoC) will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in January of 2025. Data from the 60 patients in Cohort B patients (single dose of certepetide administered with SoC plus a second dose of certepetide four hours after the first) is expected in mid-2025 with a final analysis of both Cohorts available thereafter.

•BOLSTER: Phase 2a double-blind, placebo-controlled, multi-center, randomized trial in the U.S. evaluating certepetide in combination with SoC in first- and second-line cholangiocarcinoma ("CCA"). The Company achieved complete enrollment in first-line CCA nearly six months ahead of plan, accelerating anticipated topline data readout to mid-2025. Based on this rapid enrollment rate and the pressing need to improve treatment outcomes in patients that have progressed after first-line CCA treatment, a second cohort has been added to the BOLSTER trial evaluating subjects in second-line CCA. Lisata previously announced that the first patient has been treated in the second-line CCA cohort, with enrollment completion expected in the first half of 2025.
•CENDIFOX: Phase 1b/2a open-label trial in the U.S. of certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon and appendiceal cancers. The trial has completed enrollment in the pancreatic cohort and expects to complete enrollment in the remaining two cohorts by the end of 2024.
•Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for mPDAC. During the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting, Qilu Pharmaceutical presented an abstract sharing preliminary data from the study which corroborated previously reported findings from the Phase 1b/2a trial of certepetide plus gemcitabine and nab-paclitaxel conducted in Australia in patients with mPDAC. As previously reported, Qilu has begun treating patients in their Phase 2 placebo-controlled trial in mPDAC.
•iLSTA: Phase 1b/2a randomized, single-blind, single-center, safety and pharmacodynamic trial in Australia evaluating certepetide in combination with the checkpoint inhibitor, durvalumab, plus SoC gemcitabine and nab-paclitaxel chemotherapy versus SoC alone in patients with locally advanced non-resectable PDAC. With 24 of the 30 patients enrolled, enrollment remains on track to be completed by the first half of 2025.
•A Lisata-funded Phase 2a, double-blind, placebo-controlled, randomized, proof-of-concept study evaluating certepetide in combination with SoC temozolomide versus temozolomide alone in patients with newly diagnosed glioblastoma multiforme ("GBM") is being conducted across multiple sites in Estonia and Latvia and is targeted to enroll 30 patients with a randomization of 2:1 in favor of the certepetide treatment group. Enrollment completion is expected in the second half of 2025.
•FORTIFIDE: Phase 1b/2a, double-blind, placebo-controlled, three-arm, randomized study in the U.S. to evaluate the safety, tolerability, and efficacy of a 4-hour continuous infusion of certepetide in combination with SoC in subjects with second-line mPDAC who have progressed on FOLFIRINOX. As part of this study, Lisata has engaged Haystack Oncology to use its MRD technology to measure circulating tumor DNA levels at multiple timepoints in patients throughout the study as an exploratory endpoint for analyzing the early therapeutic effect of certepetide. The Company expects to enroll the first patient in the study by the first quarter of 2025.
As recently announced, Lisata has entered into multiple research collaborations, including a sponsored research agreement with the University of Cincinnati to assess certepetide in combination with bevacizumab (a VEGF inhibitor) in a preclinical murine model for the treatment of endometriosis. Lisata is also partnering with Valo Therapeutics ("ValoTx") to investigate the benefits of combining certepetide with ValoTx’s platform technology, PeptiCRAd, and a checkpoint inhibitor in a preclinical murine model for the treatment of melanoma.

Third Quarter 2024 Financial Highlights

For the three months ended September 30, 2024, operating expenses totaled $5.3 million, compared to $6.0 million for the three months ended September 30, 2023, representing a decrease of $0.6 million or 10.5%.
Research and development expenses were approximately $2.5 million for the three months ended September 30, 2024, compared to $3.4 million for the three months ended September 30, 2023, representing a decrease of $0.8 million or 24.8%. This was primarily due to a reduction in clinical research organization expenses associated with the Company’s Phase 2a BOLSTER trial as a result of trial protocol modifications and lower equity expenses. In addition, there were start-up expenses in the prior year related to the GBM study.

General and administrative expenses were approximately $2.8 million for the three months ended September 30, 2024, compared to $2.6 million for the three months ended September 30, 2023, representing an increase of $0.2 million or 8.1%. This was primarily due to higher consulting expenses.
Overall, net losses were $4.9 million for the three months ended September 30, 2024, compared to $5.3 million for the three months ended September 30, 2023.

On November 12, 2024 IO Biotech (Nasdaq: IOBT), a clinical-stage biopharmaceutical company developing novel, off-the-shelf, immune-modulating therapeutic cancer vaccines based on its T-win platform, reported financial results for the third quarter ended September 30, 2024 (Press release, IO Biotech, NOV 12, 2024, View Source [SID1234648177]).

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"We continue to add to the body of evidence that our novel investigational therapeutic cancer vaccine, IO102-IO103, has the potential to bring clinical benefit to patients with strong signals of activity now observed in patients with three types of metastatic solid tumors," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "As we look to the primary endpoint data readout from our Phase 3 pivotal trial in the first half of next year, we plan to be prepared to submit a Biologics License Application (BLA) to the FDA in 2025 and potentially make our first therapeutic cancer vaccine available for patients in the US with advanced melanoma in 2026."

Dr. Zocca continued, "Our T-win platform generates off-the-shelf therapeutic cancer vaccine candidates with a unique mechanism of action, capable of both targeting immune-suppressive cells and cancer cells. This dual action is what drives the strong activity we see when we combine IO102-IO103 with an anti-PD-1 therapy. In addition to IO102-IO103, the T-win platform has generated other novel candidates including IO112, targeting arginase 1 with a unique mechanism of action. We are excited about the strength of the data supporting the potential of IO112 and plan to submit an Investigational New Drug (IND) application to the FDA for this program in 2025."

Recent Business Highlights

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The per-protocol interim analysis in the pivotal Phase 3 trial (IOB-013/KN-D18), which is evaluating IO102-IO103 in combination with KEYTRUDA (pembrolizumab) in advanced melanoma, was completed by the IDMC in the third quarter of 2024. Based on review of safety and efficacy data, the IDMC recommended the trial continue without modifications and noted that no new safety signals were observed. The outcome of the primary endpoint of PFS is projected to be available in the first half of 2025, potentially followed by a BLA submission in 2025 depending on the PFS outcome.

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Encouraging data for the fully enrolled squamous cell carcinoma of the head and neck (SCCHN) and non-small cell lung cancer (NSCLC) cohorts of the Phase 2 basket trial (IOB-022/KN-D38) evaluating IO102-IO103 in combination with pembrolizumab in the first-line treatment of patients with metastatic disease were presented at the 2024 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September and the 2024 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November, respectively. [https://bit.ly/3zgzsoY; https://bit.ly/4fsVCno]

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The company continues to progress its perioperative Phase 2 basket trial (IOB-032/PN-E40) studying treatment with IO102-IO103 in combination with pembrolizumab dosed before (neo-adjuvant) and after (adjuvant) surgery with curative intent in patients with resectable melanoma or SCCHN. Enrollment continues in both the single arm SCCHN cohort (cohort B) and the randomized melanoma cohort (cohort C), in which patients are randomized either to IO102-IO103 in combination with pembrolizumab or to pembrolizumab alone.

Third Quarter 2024 Financial Results

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Net loss for the three months ended September 30, 2024, was $24.0 million, compared to $21.7 million for the three months ended September 30, 2023.

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Research and development expenses were $20.2 million for the three months ended September 30, 2024, compared to $17.7 million for the three months ended September 30, 2023. The increase was primarily related to timing of clinical trial-related activities for the company’s IO102-IO103 therapeutic cancer vaccine candidate, including the continued execution of the company’s pivotal Phase 3 clinical trial. The company recognized $0.6 million in research and development equity-based compensation for the three months ended September 30, 2024, compared to $2.1 million for the three months ended September 30, 2023.

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General and administrative expenses were $6.3 million for the three months ended September 30, 2024, compared to $5.8 million for the three months ended September 30, 2023. The company recognized $1.0 million in general and administrative equity-based compensation for the three months ended September 30, 2024, compared to $0.9 million for the three months ended September 30, 2023.

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Cash and cash equivalents as of September 30, 2024 were $80.2 million, compared to $143.2 million at December 31, 2023. During the three months ended September 30, 2024, the company used cash, cash equivalents and restricted cash of $20.8 million. The company continues to expect that it will have sufficient cash to run the company into the fourth quarter of 2025.

About IO102-IO103

IO102-IO103 is an investigational off-the-shelf therapeutic cancer vaccine designed to kill both tumor cells and immune-suppressive cells in the tumor microenvironment (TME) by stimulating activation and expansion of T cells against indoleamine 2,3-dioxygenase (IDO) positive and/or programmed death-ligand 1 (PD-L1) positive cells. The company is currently conducting a pivotal Phase 3 trial (IOB-013/KN-D18; NCT05155254) investigating IO102-IO103 in combination with pembrolizumab versus pembrolizumab alone in patients with advanced melanoma, a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709) investigating IO102-IO103 in combination with pembrolizumab as first line treatment in patients with solid tumors, and a Phase 2 basket trial (IOB-032/PN-E40; NCT05280314) investigating IO102-IO103 in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors.

The clinical trials are sponsored by IO Biotech and conducted in collaboration with Merck, which is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About the IOB-013/KN-D18 Pivotal Phase 3 Clinical Trial

IOB-013/KN-D18 (Clinical Trials.gov: NCT05155254) is an open label, randomized Phase 3 pivotal clinical trial evaluating IO102-IO103 in combination with pembrolizumab versus pembrolizumab alone in patients with previously untreated, unresectable or metastatic (advanced) melanoma. A total of 407 patients have been enrolled from more than 100 centers across the United States, Europe, Australia, Turkey, Israel and South Africa. The primary endpoint of the study is progression free survival, an event-driven analysis conducted when 226 events, defined as disease progression or death, have been reported in the study. Secondary endpoints include overall response rate (ORR), overall survival (OS), durable objective response rate (DRR), complete response rate (CRR), duration of response (DoR), time to complete response (TTCR), disease control rate (DCR), and incidence of AEs and SAEs (safety and tolerability). Biomarkers in the blood and tumor tissue will also be assessed. IO Biotech is sponsoring the Phase 3 trial and Merck is supplying pembrolizumab.

About IOB-022/KN-D38 Phase 2 Solid Tumor Basket Trial

IOB-022/KN-D38 (NCT05077709) is a non-comparative, open label trial to investigate the safety and efficacy of IO102-IO103 in combination with pembrolizumab in first-line advanced cancers in non-small cell lung cancer (NSCLC) and squamous cell carcinoma of the head and neck (SCCHN). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab. IO Biotech maintains global commercial rights to IO102-IO103.

About IOB-032/PN-E40 Phase 2 Solid Tumor Basket Trial

IOB-032/PN-E40 (NCT05280314) is a Phase 2 basket trial investigating the IO102-IO103 therapeutic cancer vaccine in combination with pembrolizumab as neo-adjuvant/adjuvant treatment of patients with solid tumors. The study completed enrollment of 15 patients with melanoma in cohort A and continues to enroll 15 patients with SCCHN in cohort B as single arm cohorts receiving combination of IO102-IO103 with pembrolizumab, whereas in cohort C, melanoma patients will be randomized 1:1 to either the combination of IO102-IO103 with pembrolizumab or pembrolizumab alone. In the neo-adjuvant period, for all cohorts, treatment is every 3 weeks (Q3W) for 3 cycles (melanoma) or 2-3 cycles (SCCHN). Patients entering the study will be scheduled for surgery and begin neoadjuvant treatment 4-9 weeks prior. Surgery will be followed by adjuvant treatment with the same regimen for 15 cycles. Cohort C patients with poor pathological response to pembrolizumab alone in the neo-adjuvant phase (>10% residual viable tumor) may cross over to combination treatment post-surgery. The primary endpoint is major pathological response at surgery (≤10% residual viable tumor; central assessment). IO Biotech is sponsoring the Phase 2 trial and Merck is supplying pembrolizumab.

On November 12, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the third quarter ended September 30, 2024 (Press release, Protara Therapeutics, NOV 12, 2024, View Source [SID1234648205]).

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"We continue to make significant progress advancing all of our ongoing programs as we strive toward our goal of bringing life-changing therapies to patients impacted by cancer and rare diseases," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "Notably, we remain on track to report interim data in the fourth quarter of 2024 from our ADVANCED-2 study of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC). Given its ease of use, favorable safety profile, and encouraging early clinical data, we believe TARA-002 could be an impactful addition to the NMIBC treatment landscape."

Mr. Shefferman added, "In addition to our NMIBC program, we remain on track to commence the pivotal THRIVE-3 study of intravenous (IV) Choline Chloride, for which we recently received Fast Track designation from the U.S. Food and Drug Administration (FDA), in the first quarter of 2025. Finally, we continue to expect interim data in the first half of 2025 from our ongoing Phase 2 STARBORN-1 trial of TARA-002 in lymphatic malformations (LMs), a rare condition primarily impacting children for which there are no FDA approved therapies."

Recent Progress and Highlights

TARA-002 in NMIBC

The Company remains on track to report results from a pre-planned interim analysis of six-month evaluable patients in the ongoing Phase 2 open-label ADVANCED-2 trial later this quarter. The 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.
The Company expects to report preliminary results in mid-2025 from 12-month evaluable patients in the ongoing Phase 2 open-label ADVANCED-2 trial.
The Company continues to explore systemic priming dosing prior to initiation of intravesical administration, as well as combination therapy with TARA-002 in NMIBC patients with CIS. Given TARA-002’s mechanism of action and safety profile, the Company believes it has strong potential for use in combination therapy and is working to determine various opportunities for the clinical program.
IV Choline Chloride for Patients on Parenteral Support (PS)

In October 2024, Protara announced that the FDA granted Fast Track designation to IV Choline Chloride, the Company’s investigational IV phospholipid substrate replacement therapy, in patients for whom oral or enteral nutrition is not possible, insufficient, or contraindicated. Protara is currently developing IV Choline Chloride as a source of choline for adult and adolescent patients on PS.
In September 2024, the Company announced results from THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on PS. The study found that 78% of patients who are dependent on PS were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury.
In September 2024, the U.S. Patent and Trademark Office issued the Company a second patent in this program, claiming a method for treating choline deficiency with a choline composition with a term until 2041.
The Company expects to begin dosing patients in the THRIVE-3 registrational trial in the first quarter of 2025.
TARA-002 in LMs

In September 2024, Protara announced completion of the first safety cohort of the ongoing Phase 2 STARBORN-1 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs. Of the three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002, including one with a macrocystic LM and one with a ranula (a type of maxillofacial cyst) achieved a complete response after receiving one dose of TARA-002. TARA-002 was generally well-tolerated. Enrollment is underway in additional cohorts, with initial results expected in the first half of 2025.
Third Quarter 2024 Financial Results

As of September 30, 2024, cash, cash equivalents, and investments in marketable debt securities totaled approximately $81.5 million. The Company expects its cash, cash equivalents, and investments in marketable debt securities will be sufficient to fund its planned operations and data milestones into 2026.
Research and development expenses for the third quarter of 2024 increased to $8.1 million from $6.2 million for the prior year period. This increase was primarily due to an increase in expenses related to TARA-002 of $1.5 million as well as an increase in expenses related to IV Choline Chloride of $0.4 million.
General and administrative expenses for the third quarter of 2024 decreased to $4.3 million from $4.5 million for the prior year period. This decrease was primarily due to a reduction in personnel-related expenses of $0.6 million, offset partially by increases in market development, business development, and investor relations activities of $0.3 million.
For the third quarter of 2024, Protara incurred a net loss of $11.2 million, or $0.50 per share, compared with a net loss of $9.9 million, or $0.87 per share, for the same period in 2023. Net loss for the third quarter of 2024 included approximately $0.9 million of stock-based compensation expenses.
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. 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 Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function that also play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function, and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 80% of patients dependent on PS are choline-deficient and have some degree of liver damage, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first U.S. Food and Drug Administration (FDA) approved IV choline formulation for PS patients and has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PS patients. The U.S. Patent and Trademark Office has issued the Company a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041.

On November 12, 2024 Turnstone Biologics Corp. ("Turnstone" or the "Company") (Nasdaq: TSBX), a clinical-stage biotechnology company developing a differentiated approach to treat and cure patients with solid tumors by pioneering selected tumor-infiltrating lymphocyte (Selected TIL) therapy, reported financial results for the third quarter ended September 30, 2024, and provided recent business highlights (Press release, Turnstone Biologics, NOV 12, 2024, View Source [SID1234648220]).

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"Earlier this quarter, we announced a corporate restructuring and the decision to focus resources on our Phase 1 program, TIDAL-01, which we believe puts us on the path to significant value creation. Importantly, our extended cash runway into the second quarter of 2026 enables us to achieve potential key clinical milestones and we remain steadfast in advancing our differentiated approach to TIL therapy for cancer patients with high unmet need," said Sammy Farah, M.B.A., Ph.D., Turnstone’s President and Chief Executive Officer. "Recently, we reported initial clinical data from our STARLING trial which showcased durable anti-tumor activity in a heavily pre-treated late-line microsatellite stable colorectal cancer patient population, including the achievement of a complete response. The patient remains in remission with progression-free survival extending beyond one year. Our encouraging initial clinical data, combined with results that continue to emerge from within the field in support of selection-based approaches, strengthen the competitive profile of our next-generation selected TIL technology and its potential to treat solid tumors. We look forward to sharing our next clinical update in 1H 2025."

Third Quarter 2024 and Recent Business Highlights

Presented at the 2024 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting – Tumor Infiltrating Lymphocytes (TIL) Symposium, supporting ongoing clinical efforts. On November 6, 2024, Turnstone delivered a presentation titled "TIDAL-01: Enriching for a More Potent TIL Population with Selected TIL therapy" at the SITC (Free SITC Whitepaper) TIL Symposium in Houston, TX. The presentation showcased Turnstone’s initial clinical data from its Phase 1 TIDAL-01 program, including the achievement of a complete response in a third-line microsatellite stable metastatic colorectal cancer ("MSS mCRC") patient. Additionally, two posters were presented at the SITC (Free SITC Whitepaper) Annual Meeting with preclinical data that demonstrated the practicality of selecting and expanding tumor-reactive TIL as a potential treatment option for patients with solid tumors.

Reported positive initial data from the STARLING Phase 1 Trial of TIDAL-01 in MSS mCRC. In August, initial results were shared from the first 4 evaluable MSS mCRC patients from the STARLING Phase 1 study of TIDAL-01. The trial yielded a 25% overall response rate ("ORR") with durable clinical benefit and 50% disease control rate ("DCR") in a heavily pre-treated, advanced disease, third-line setting where patients are unresponsive to checkpoint inhibitors and have almost no treatment options. One patient had a complete response ("CR") and has been progression-free for over one year which is notable in this patient population, while another patient had stable disease ("SD") for 6 months. As a point of comparison, the current standard of care treatment for this patient population has resulted in an ORR of 1-6% and a median progression-free survival ("mPFS") of 2-5.6 months. There were also no new safety observations specific to Turnstone’s Selected TILs.

Completed strategic restructuring and portfolio review to extend cash runway. In October, Turnstone announced the streamlining of its operations to optimize its portfolio and strengthen its financial position to focus on achieving clinical milestones. Key initiatives include:

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Pipeline strategy and prioritization: Following a comprehensive evaluation of the business, Turnstone has decided to sharpen its focus on development of the TIDAL-01 program.
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Organizational restructuring: The Company executed a 60% workforce reduction to prioritize the development of its core Selected TIL program and extend its cash runway.

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Financial strategy: Turnstone expects its cash runway to support operations and clinical development through the second quarter of 2026.
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Talent strategy: As part of Turnstone’s updated corporate strategy, Ines Verdon, M.D., Senior Vice President of Clinical Development, is assuming leadership of all clinical activities. Michael Fitch, Ph.D., has been promoted to Senior Vice President of Manufacturing and will oversee all manufacturing and technical operations activities. Wendy Worcester, CPA, is assuming the responsibility of the Finance function as the Principal Financial and Accounting Officer. Saryah Azmat has been promoted to Chief Operating Officer.

Third Quarter 2024 Financial Results

Cash, cash equivalents and short-term investments: As of September 30, 2024, cash, cash equivalents and short-term investments were $45.3 million. The Company expects that the combined cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second quarter of 2026.

Research and development (R&D) expenses: R&D expenses for the three months ended September 30, 2024, were $14.4 million, compared to $14.2 million for the same period in 2023. The increase was due primarily to an increase in manufacturing, clinical, and regulatory costs related to TIDAL-01 clinical trials.

General and administrative (G&A) expenses: G&A expenses for the three months ended September 30, 2024, were $3.9 million, compared to $4.8 million for the same period in 2023. The decrease was due primarily to reductions in personnel costs, professional service costs, and other general and administrative costs.

Net loss: Net loss for the three months ended September 30, 2024, was $17.0 million, compared to net loss of $17.3 million for the same period in 2023.