Verastem Oncology Granted Fast Track Designation for Combination of Avutometinib and Sotorasib for the Treatment of KRAS G12C-Mutant Non-Small Cell Lung Cancer (NSCLC)

On January 18, 2024 Verastem Oncology (Nasdaq:VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Verastem Oncology’s investigational RAF/MEK clamp, avutometinib, in combination with Amgen’s KRAS G12C inhibitor, LUMAKRASTM (sotorasib), for the treatment of patients with KRAS G12C-mutant metastatic non-small cell lung cancer (NSCLC) who have received at least one prior systemic therapy and have not been previously treated with a KRAS G12C inhibitor (Press release, Verastem, JAN 18, 2024, View Source [SID1234639336]). Fast Track is a process designed to facilitate the development and expedite the review of new drugs intended to treat or prevent serious conditions and address unmet medical need.

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"Receiving Fast Track Designation for the combination of avutometinib and sotorasib reinforces the importance of improving the depth of MAPK pathway inhibition to enhance tumor regression relative to KRAS G12C inhibition alone and the potential of the combination of avutometinib and sotorasib in KRAS G12C mutant locally advanced or metastatic NSCLC," said Dan Paterson, President and CEO, Verastem Oncology. "Given that KRAS G12C is the most common KRAS mutation in NSCLC, the advancement of the combination is important in understanding potential new treatment approaches. We look forward to continued interaction with the FDA as we advance the development of this promising treatment regimen."

While newer targeted therapies specific to KRAS G12C-mutant NSCLC have shown significant promise, avutometinib targets the RAS pathway, which is a common source of acquired mutation. Preclinical proof-of-concept studies demonstrated improvements with the combination of avutometinib and sotorasib vs sotorasib alone, including deeper tumor regression through enhanced blockade of ERK activation and a decrease in the frequency of relapse of tumors. The RAMP 203 clinical development program will determine whether the promising preclinical results observed with the combination of avutometinib with sotorasib translate into improved clinical outcomes for patients with KRAS G12C-mutant locally advanced or metastatic NSCLC who have received prior therapy for metastatic disease and have not been previously treated with a KRAS G12C inhibitor.

Initial results of the RAMP 203 (NCT05074810) Phase 1/2, multicenter, open label, dose evaluation/expansion study evaluating the efficacy and safety of avutometinib + sotorasib in patients with KRAS G12C-mutant NSCLC who have not been previously treated with a KRAS G12C inhibitor as well as in patients who have been previously treated with a KRAS G12C inhibitor demonstrated confirmed responses in both KRAS G12C inhibitor resistant and naïve patients. The pharmacokinetic profile of avutometinib in combination with sotorasib in the RAMP 203 trial was similar to results in monotherapy studies. No drug-drug interactions were observed between avutometinib and sotorasib. Avutometinib 4.0 mg PO BIW 21/28 days + sotorasib 960 mg PO QD 28/28 days was selected as RP2D based on dose limiting toxicity (DLT) assessment. These initial RAMP 203 results were presented at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October, 2023. Enrollment of patients with KRAS G12C-mutant NSCLC who are either naïve to or previously treated with a KRAS G12C inhibitor is ongoing in the expansion phase of RAMP 203 with updated results expected in the first half of 2024.

About Avutometinib

Avutometinib is a RAF/MEK clamp that induces inactive complexes of MEK with ARAF, BRAF and CRAF potentially creating a more complete and durable anti-tumor response through maximal RAS pathway inhibition. Avutometinib is currently in late-stage development.

In contrast to currently available MEK inhibitors, avutometinib blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows avutometinib to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors. The U.S. Food and Drug Administration granted Breakthrough Therapy designation for the combination of Verastem Oncology’s investigational RAF/MEK clamp avutometinib, with defactinib, its FAK inhibitor, for the treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy.

Verastem Oncology is currently conducting clinical trials with its RAF/MEK clamp avutometinib in RAS pathway-driven tumors as part of its RAMP (Raf And Mek Program) trials. RAMP 201 is a Phase 2 registration-directed trial of avutometinib in combination with defactinib in patients with recurrent LGSOC and has completed enrollment in the dose optimization and expansion phases and is enrolling for low-dose evaluation. Verastem Oncology has established clinical collaborations with Amgen and Mirati to evaluate LUMAKRAS (sotorasib) and KRAZATI (adagrasib) in combination with avutometinib in KRAS G12C mutant NSCLC as part of the RAMP 203 and RAMP 204 trials, respectively. Supported by the "Therapeutic Accelerator Award" Verastem Oncology received from PanCAN, the Company is conducting RAMP 205, a Phase 1b/2 clinical trial evaluating avutometinib and defactinib with gemcitabine/nab-paclitaxel in patients with front-line metastatic pancreatic cancer.

Tvardi Therapeutics Announces Presentation of REVERTLIVER CANCER Phase 2 Clinical Trial Using TTI-101, A Novel STAT3 Inhibitor, at 2024 ASCO Gastrointestinal Cancers Symposium

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).

B. Riley Securities 4th Annual Oncology conference

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

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Corporate presentation

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

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Panbela Announces Publication of Clinical Data: A New Approaches to Neuroblastoma Therapy Trial

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."