On January 18, 2024 Tvardi Therapeutics, Inc., a privately held, clinical-stage biopharmaceutical company focused on the development of STAT3 inhibitors, reported that it will present a trials in progress poster at the upcoming 2024 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium on Friday, January 19, 2024, in San Francisco, CA (Press release, Tvardi Therapeutics, JAN 18, 2024, View Source [SID1234639335]). The poster will highlight the design of the ongoing REVERTLIVER CANCER study, a Phase 1b/2 trial evaluating safety and clinical activity of TTI-101, a novel STAT3 inhibitor, as monotherapy and in combination with standard of care in patients with locally advanced or metastatic, and unresectable hepatocellular carcinoma (HCC).

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Presentation Details:

Date: Friday, January 19, 2024
Time: 12:30 p.m. PST

Location: Moscone West, San Francisco, CA
Session Type: Trials in Progress Poster Session
Abstract Title: A Phase 1b/2 Study to Evaluate the Safety and Efficacy of TTI-101 as Monotherapy and in Combination in Advanced Hepatocellular Carcinoma. LEARN MORE
Abstract #: TPS577
Poster Bd #: P6

The REVERTLIVER CANCER trial is currently enrolling HCC patients at top US cancer research institutes and is designed to evaluate TTI-101 across multiple lines of therapy:

TTI-101 alone for patients previously treated with up to three prior lines of systemic therapy
TTI-101 in combination with pembrolizumab (Keytruda) for patients previously treated with immunotherapy
TTI-101 in combination with atezolizumab (Tecentriq) and bevacizumab (Avastin) for treatment-naïve patients
HCC is the most common form of liver cancer; however, treatment options for the disease are limited and overall prognosis is poor, with a 5-year survival rate of 18%. TTI-101’s target, STAT3, is a key regulatory protein which plays a critical role in the pathogenesis of HCC by initiating tumorigenesis as well as promoting an immunosuppressive tumor microenvironment. TTI-101 is an orally delivered, small molecule, direct inhibitor of STAT3.

"We are excited to present this unique trial design that allows us to evaluate the potential of TTI-101 to enhance outcomes across 1st, 2nd, and last line HCC patients, where the current standard of care is insufficient," said Imran Alibhai, Ph.D., CEO of Tvardi Therapeutics.

For more information about the REVERTLIVER CANCER trial, please visit ClinicalTrials.gov (NCT05440708).

On January 18, 2024 Puma Biotechnology presented its corporate presentation (Presentation, Puma Biotechnology, JAN 18, 2024, View Source [SID1234639334]).

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On January 18, 2024 Portage Biotech presented its corporate presentation (Presentation, Portage Biotech, JAN 18, 2024, View Source [SID1234639333]).

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On January 18, 2024 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs reported the publication of clinical data from studies of CPP-1X (also known as α-Difluoromethylornithine (DFMO) or Eflornithine) in neuroblastoma (Press release, Panbela Therapeutics, JAN 18, 2024, View Source [SID1234639332]). According to Hogarty et al, children with relapsed refractory neuroblastoma have dismal outcomes and new therapeutic options are needed. Data published in the British Journal of Cancer investigated the tolerability and activity of depleting polyamines by high dose CPP-1X and celecoxib in combination with standard of care chemotherapy in heavily pretreated neuroblastoma patients. Results showed that DFMO treatment was well tolerated, and the median time-to-progression was 19.8 months. The work reflects the Company’s previous collaboration with New Advances in Neuroblastoma Therapy Consortium (NANT) (View Source). A link to the publication can be found here: View Source

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From the Phase 1 dose range finding study of CPP-1X in heavily pretreated neuroblastoma patients, CPP-1X was well tolerated. The best overall response included 2 partial responses (PR), 4 minor responses (MR), 10 Stable disease (SD), 7 progressive disease (PD) and 1 unevaluable. All patients with an overall response of PR or MR sustained this response until stopping or completing protocol therapy. The overall objective response rate (CR+PR) was 9% and rate of any response (CR+PR+MR) was 26%. At 2 years, PFS (progression free survival) for the entire cohort was 29.5%. Notably, three patients completed protocol therapy and remain without disease progression or event at >4 years from treatment end in the absence of additional therapy.

These results build upon the recent FDA approval of CPP-1X or DFMO to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Results from these studies suggest that CPP-1X is a safe, oral treatment option that may improve response rates in heavily pretreated relapsed refractory neuroblastoma patients and are the basis for the ongoing ANBL-1821 Phase 2 trial.

"We are excited about the publication of these Phase 1 trial results in light of the recent DFMO FDA approval for patients in maintenance therapy. From this dose escalation study, our collaborators were able to demonstrate high dose DFMO is well tolerated and demonstrated activity in patients with heavily pretreated neuroblastoma," said Elizabeth Bruckheimer, PhD, Vice President & Chief Scientific Officer of Panbela. "Moreover, three patients remain alive over four years from treatment end without any additional therapy which suggests that high dose DFMO treatment in combination with chemotherapy may be a potential treatment option for this high unmet need population."

"Overall, these results in addition to the recent approval of DFMO as a maintenance therapy, suggests a role for polyamine inhibition therapy for neuroblastoma that may impact other cancer types such as prostate cancer. We are excited by these results and the potential role for CPP-1X in the clinical management of neuroblastoma and cancer as a whole." said Dr. Bruckheimer. "These studies were the basis for the ongoing Children’s Oncology Group Phase II trial in relapsed refractory neuroblastoma to support the goal of developing effective novel therapies for patients with unmet medical needs."

First author Michael Hogarty, MD, Professor of Pediatrics at the University of Pennsylvania, and Children’s Hospital of Philadelphia said, "The results from the Phase 1 study have built upon the preclinical work performed in my laboratory demonstrating a role of deep polyamine depletion as a potential therapeutic target for relapsed or refractory neuroblastoma. By understanding the underlying biology and role of MYC signaling and the polyamine pathway in neuroblastoma, we are able to show the potential impact of high-dose DFMO in neuroblastoma."

On January 18, 2024 Ikena Oncology, Inc. (Nasdaq: IKNA, "Ikena," "Company"), a targeted oncology company forging new territory in patient-directed cancer treatment, reported an organizational update outlining key objectives toward advancing the development of its lead targeted oncology assets, IK-930 and IK-595 (Press release, Ikena Oncology, JAN 18, 2024, View Source [SID1234639330]). The Company also announced an organizational streamlining that allows for the reallocation of resources from exploratory research and discovery towards the ongoing targeted oncology clinical programs. These efforts reinforce the Company’s dedication to maximize impact and drive advancements in patient-directed treatments for cancer.

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"We are laser focused on driving IK-930 and IK-595 forward in the next year to interpretable and clear data reads as we continue to build value for investors," commented Mark Manfredi, Ph.D., Chief Executive Officer of Ikena. "Our extended team has achieved many significant milestones together, and IK-930 and IK-595’s clinical progress is evidence of these collective efforts. Ikena will be forever grateful for the impact that each of the members of our discovery and research team has had. The renewed focus on our lead assets, IK-930 and IK-595, underscores our dedication to delivering the full therapeutic potential of our clinical candidates that we believe could transform the lives of cancer patients."

IK-930: TEAD1-Selective Hippo Pathway Inhibitor

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An optimized formulation is now being dosed in the clinic concurrently with the original formulation

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The Company has expanded and accelerated targeted recruitment of mesothelioma patients and additional epithelioid hemangioendothelioma (EHE) patients

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A clinical data update is planned for the second half of 2024

IK-595: MEK-RAF Molecular Glue

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The initial cohort was dosed with IK-595 in December 2023 and has subsequently cleared the safety evaluation window

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Enrollment of targeted RAS and RAF mutant cancer patients in dose escalation continues, where multiple dosing schedules are being explored

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Backfill and expansion cohorts are planned in multiple indications where IK-595 may have differentiated advantages

Strategic and Corporate Updates

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With the advancement of IK-930 and IK-595, the Company has made the strategic decision to reallocate resources from the discovery organization to the clinical programs

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This includes a workforce reduction of approximately 35%, to be implemented over the course of the first quarter of 2024

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With approximately $175 million in cash, cash equivalents and marketable securities as of December 31, 2023 (unaudited), and as a result of the organization changes, the Company’s runway is extended into the second half of 2026