Turning Point Therapeutics Presents TRIDENT-1 Study Clinical Data for Repotrectinib in NTRK+ Advanced Solid Tumors in Plenary Session at 2021 AACR-NCI-EORTC Conference

On October 8, 2021 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported the presentation of early clinical data from the NTRK-positive TKI-naïve and TKI-pretreated advanced solid tumor cohorts (EXP-5 and EXP-6) of the ongoing TRIDENT-1 Phase 1/2 study of its lead drug candidate repotrectinib (Press release, Turning Point Therapeutics, OCT 8, 2021, View Source [SID1234591020]).

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These data are being presented today at a plenary session at the Virtual 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) hosted by the American Association for Cancer Research (AACR) (Free AACR Whitepaper), the National Cancer Institute (NCI), and the European Organisation for Research and Treatment of Cancer (EORTC) being held October 7-10.

"These data in patients with NTRK-positive advanced solid tumors are encouraging and support further investigation of repotrectinib in this patient population, especially in the TKI-pretreated setting where there are no currently approved targeted therapies," said Mohammad Hirmand, M.D., chief medical officer. "Based on these findings and repotrectinib having recently been granted Breakthrough Therapy designation for NTRK-positive TKI-pretreated advanced solid tumors, we look forward to discussing next steps toward potential registration of repotrectinib in this patient population at a Type B meeting with the FDA anticipated in the first half of 2022."

Repotrectinib Early Data from Phase 1/2 TRIDENT-1 Study from NTRK-Positive Advanced Solid Tumor Cohorts (EXP-5, EXP-6)
The early Phase 2 TRIDENT-1 dataset utilizes an August 26, 2021 data cutoff. The safety analysis includes 301 treated patients from the pooled Phase 1 and Phase 2 portions of TRIDENT-1 across all cohorts, and the preliminary efficacy analysis includes 40 evaluable patients from the pooled Phase 1 and Phase 2 portions of TRIDENT-1 in the NTRK-positive advanced solid tumor cohorts (EXP-5, EXP-6). Of the 40 patients, 17 were TKI-naïve and 23 were TKI-pretreated. Phase 2 patients included in the efficacy analysis had baseline measurable disease and at least one post-baseline evaluable scan or were off treatment prior to first post-baseline scan. Responses were confirmed with a subsequent scan at least 28 days later per RECIST 1.1 and were determined by physician assessment for Phase 2 patients. Phase 1 patients included in the efficacy analysis were treated at or above the Phase 2 dose, with responses assessed by blinded independent central review (BICR). The Phase 1 data cutoff date was July 22, 2019 for responses and August 26, 2021 for duration of treatment.

The findings were reported in a pre-recorded oral plenary presentation by Benjamin Besse, M.D., Ph.D., professor of Medical Oncology at Paris-Saclay University and full-time cancer specialist at Gustave Roussy Cancer Center available on October 8 at 10:05 a.m. ET on the meeting website.

Pooled Phase 1 and Phase 2 Preliminary Efficacy Analysis (n=40)

In the NTRK-positive TKI-naïve advanced solid tumor population (EXP-5: n=17), the confirmed Objective Response Rate (cORR) was 41% (95% CI: 18-67). At the time of the data cutoff, three patients with limited time on treatment achieved stable disease with tumor regression of -21%, -23%, and -27% on their first post-baseline scans, and were awaiting their next scans. Duration of response ranged from 1.9+ to 7.4+ months, and the duration of treatment in the 17 patients ranged from 0.9 to 30.7+ months.

In the NTRK-positive TKI-pretreated advanced solid tumor population (EXP-6: n=23), the cORR was 48% (95% CI: 27-69). As of the cutoff date, three patients had unconfirmed partial responses (uPRs). Two uPRs have been confirmed since the cutoff date and are included in the cORR; the third patient with a uPR was on treatment awaiting a confirmatory scan and is not considered a responder in the cORR. The 48% cORR is an update since the pre-recorded presentation. Duration of response ranged from 0.9+ to 15.1 months, and the duration of treatment in the 23 patients ranged from 0.6 to 20.8 months.

Of the 23 NTRK-positive TKI-pretreated advanced solid tumor patients, 13 (57%) had NTRK solvent front mutations. In these 13 patients, the cORR was 62% (95% CI: 32-86) including one patient who had a complete response. As of the cutoff date, three patients had unconfirmed partial responses (uPRs). Two uPRs have been confirmed since the cutoff date and are included in the cORR; the third patient with a uPR was on treatment awaiting a confirmatory scan and is not considered a responder in the cORR. The 62% cORR is an update since the pre-recorded presentation. Duration of response ranged from 0.9+ to 13.7 months.
Preliminary Safety Analysis (n=301)

Repotrectinib was generally well tolerated.

The most frequently reported treatment-emergent adverse event (TEAE) was low-grade dizziness (60%) of which 76% of reported cases were grade 1. Eleven patients (4%) reported ataxia in the absence of dizziness. No events of dizziness or ataxia led to treatment discontinuation.

Dose modifications due to TEAEs included 27% of patients who had dose reduction and 11% who had drug discontinuation.
Turning Point also announced the publication of preclinical data of repotrectinib in the American Association of Cancer Research’s peer reviewed journal, Molecular Cancer Therapeutics. Preclinical studies described in the publication titled "Molecular Characteristics of Repotrectinib That Enable Potent Inhibition of TRK Fusion Proteins and Resistant Mutations" show that repotrectinib potently inhibited TRK fusion proteins and resistance mutations. Repotrectinib was the most potent inhibitor of wild-type TRKA/B/C fusions and was more potent than selitrectinib against all tested resistance mutations.

Repotrectinib was recently granted Breakthrough Therapy designation for the treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatments. The company is planning to discuss next steps towards potential registration of repotrectinib in this patient population at a Type B meeting with the U.S. Food and Drug Administration (FDA) anticipated in the first half of 2022.

OncoNano Medicine Announces Positive Preclinical Data for ONM-501 at AACR Virtual Conference on Tumor Immunology and Immunotherapy

On October , 2021 OncoNano Medicine, Inc. reported that positive results from its preclinical study of ONM-501, a novel dual-activating polyvalent STING agonist for immuno-oncology applications (Press release, OncoNano Medicine, OCT 8, 2021, View Source [SID1234591022]). The data, presented at The American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Conference on Tumor Immunology and Immunotherapy, demonstrate strong efficacy in multiple tumor models.

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"We are excited by the positive preclinical results for ONM-501 recently presented at AACR (Free AACR Whitepaper). STING plays a crucial role in mediating our innate immune systems but has consistently been a challenging pathway to target," said Martin Driscoll, Chief Executive Officer of OncoNano Medicine, Inc. "We are encouraged by the constellation of preclinical data that demonstrates ONM-501 could have a clinical profile differentiated from earlier generation cyclic dinucleotide STING agonist compounds. The novel ONM-501 formulation consisting of our STING activating pH-sensitive micelle loaded with an endogenous agonist has demonstrated a capability to produce a dual and prolonged activation of STING while recruiting a robust adaptive immune response to the tumor microenvironment. We look forward to continuing our IND-enabling activities as we advance ONM-501 to first in human trials."

Presentation Overview

TITLE: ONM-501 ― A synthetic polyvalent STING agonist for cancer immunotherapy

PRESENTER: Qingtai Su, Ph.D., Senior Scientist, OncoNano Medicine, Inc.

ONM-501 demonstrated antitumor efficacy in six different syngeneic mouse models from different tissues of origin (MC38, 4T1, TC-1, B16-F10, CT26 and A20). The animals were treated intratumorally with ONM-501 as a monotherapy or in combination with PD-1 blockade. The findings indicate that ONM-501 demonstrated:

Strong antitumor efficacy across all tumor models tested as a mono or combo therapy
Significantly improved efficacy with increased complete response in several models when combined with PD-1 blockade
Successful combination of a novel, proprietary STING activating micelle with the endogenous cGAMP potentially offers a synergistic immunotherapy strategy against cancer

Advocacy in Action: Updates on the Childhood Cancer STAR Act and Childhood Cancer Data Initiative

On October 8, 2021 The Childhood Cancer STAR Act and Childhood Cancer Data Initiative (CCDI) reported that are major federal government-led initiatives that exist in part because of years of advocacy work by members of the brain tumor and childhood cancer communities (Press release, National Brain Tumor Society, OCT 8, 2021, View Source [SID1234591041]). As we reflect back on Childhood Cancer Awareness Month (CCAM) this past September, we want to share updates on these initiatives as a reminder of the ongoing impact that advocacy makes possible.

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"The progress of the STAR Act and CCDI illustrate the commitment and increase in focus on research in childhood cancer," says Danielle Leach, chief of government and community relations at NBTS. "Cancer is the number one cause of death by disease among children, and pediatric brain tumors are the leading cause of cancer-related death in this population. Therefore, seeing this increased collaboration and commitment is promising. Now is the time for advocates to continue to work together to support increases in federal research funding so this critical work can create the change we so desperately need to find cures and quality of life."

Childhood Cancer Data Initiative

In late 2020, following extensive input from NBTS and advocates, Congress approved funding for CCDI at $50 million per year for 10 years. Once that funding was secured, the CCDI steering committee, which includes NBTS CEO David Arons, delivered a report to the National Cancer Institute on the goals of the initiative:

Gather data from every child, adolescent, and young adult diagnosed with pediatric cancer, regardless of where they receive their care.
Create a national strategy of appropriate clinical and molecular characterization to speed diagnosis and inform treatment for all types of pediatric cancers.
Develop a platform and tools to bring together clinical care and research data that will improve preventive measures, treatment, quality of life, and survivorship for pediatric cancers.
Based on these goals, the National Cancer Institute (NCI) is currently building the National Childhood Cancer Registry, which will centralize all existing data on cancer patients from birth to 39 years old and include information on their treatments, tumor types, outcomes, and post-care experiences. This registry will make it easier for researchers to collaborate and build on each others’ work, and hopefully, increase the pace of progress.

Danielle Leach

Also leveraging funding from the STAR Act, the NCI is working with Children’s Oncology Group (COG) to build a "biobank" of tumor tissue samples— a national "Molecular Characterization Protocol"— that will make it easier for researchers leading studies to acquire samples of tissue for their work. According to the NCI, this biobank will focus on cancers for which existing treatments are limited or ineffective and tissue for research is lacking and therefore will help fill a gap in the current research landscape.

As NCI Director Dr. Ned Sharpless wrote in a recent blog, these initiatives will "provide unprecedented insights into the drivers of childhood cancers and how they become resistant to treatment, as well as factors that influence the risk of treatment-related side effects…Collecting comprehensive data from as many children as possible will give researchers a more thorough understanding of how well treatments work…[and] make it easier for researchers to monitor the health of survivors of childhood cancer throughout their lives, providing further insights into the impact of cancer and its treatments."

STAR ACT

The STAR Act is the most comprehensive childhood cancer bill in history. It expands opportunities for childhood cancer research, improves efforts to identify and track childhood cancer incidences, and enhances the quality of life for childhood cancer survivors.

Since the bill was signed into law in 2018, NBTS advocates have played an active role in ensuring that the STAR Act has been fully funded at $30 million every year by asking for funding through activities like Head to the Hill and our online action alerts. Additionally, Danielle Leach sits on the Centers for Disease Control and Prevention Childhood Cancer STAR Act advisory group.

To date, funding from the STAR Act has led to:

Seven new research grants for "Improving Outcomes for Pediatric, Adolescent, and Young Adult Cancer Survivors."
Ten new research grants for "Research to Reduce Morbidity and Improve Care for Pediatric, and Adolescent and Young Adult (AYA) Cancer Survivors."
Additional funding for the CDC to expand the capacity of state cancer registries and their abilities to collect data on childhood cancer cases and trends.
More details related to these and other updates were included in a comprehensive overview put together by the Alliance for Childhood Cancer, for which NBTS serves as the co-chair of the policy committee.

Blue Water Vaccines, Inc. Files Registration Statement for Initial Public Offering of Common Stock

On October 8, 2021 Blue Water Vaccines, Inc. ("BWV" or "Blue Water Vaccines" or "the Company") a biopharmaceutical company developing vaccines, reported that it has filed a registration statement (Registration No.: 333-260137) on Form S-1 with the U.S. Securities and Exchange Commission relating to the proposed initial public offering of its common stock (Press release, Onconetix, OCT 8, 2021, View Source [SID1234641101]). The shares of common stock to be sold in the offering are expected to be offered by the Company. The number of shares to be offered and the price range for the proposed offering have not yet been determined.

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Maxim Group LLC will serve as sole book-running manager for the proposed offering. The Company plans to use the proceeds from this offering to fund its research and development activities, for working capital and other general corporate purposes. The proposed offering will be made only by means of a prospectus, which forms a part of the registration statement. Copies of the registration statement and the preliminary prospectus included therein relating to the proposed offering, when available, may be obtained for free by visiting EDGAR on the SEC’s website at www.sec.gov.

A registration statement on Form S-1 (Registration No.: 333-260137)relating to the proposed sale of these securities has been filed with the SEC but has not yet become effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration statement becomes effective.

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

Tachyon Presents TACH101 Data at the 2021 AACR-NCI-EORTC Conference

On October 8, 2021 Tachyon Therapeutics, Inc. ("Tachyon" or "the Company"), a research and development biotechnology company, reported the presentation of data from its TACH101 program in a virtual poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference On Molecular Targets and Cancer Therapeutics being held virtually from October 7-10, 2021 (Press release, Tachyon Therapeutics, OCT 8, 2021, View Source [SID1234591023]).

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Data presented in this abstract and poster are from preclinical studies demonstrating TACH101 target engagement and potency screening to identify molecular biomarkers of response and/or an indication associated with high TACH101 sensitivity. The data showed potent inhibition of gene expression by TACH101 and an increased sensitivity of TACH101 in colorectal cancer, especially those harboring microsatellite instability high (MSI-H) signature.

"Patient selection is critical for the successful development of any drug candidate," stated Frank Perabo, MD, PhD, CEO of Tachyon Therapeutics. "One of the key objectives for our program is to prioritize target indications in which TACH101 is likely to exert most therapeutic benefit. Being able to understand target populations early on generates knowledge that guides clinical trial design, thereby increasing the likelihood of successful transition into clinic."

Highlights from the AACR (Free AACR Whitepaper)-NCI-EORTC abstract and poster presentation are summarized below:

Abstract #P086

RNA-seq evaluation in tumor tissue identified several genes that were up- or downregulated by TACH101, including Protein Phosphatase 1 Regulatory subunit 10 (PPP1R10 or PNUTS).
TACH101 treatment caused 86% repression of PNUTS mRNA, as well as a 51% increase in H3K9me3 (a mark of repressed transcription); a 78% decrease in H3K36me3 at the PNUTS gene was also observed.
Bioinformatics analyses conducted across a large panel of cancer cell lines (>300) showed that cell lines with MSI-H status tended to be more sensitive to TACH101 in vitro. This association was found with other markers of MSI-H status such as MMR gene mutations, MLH1 methylation status, and overall mutation frequency.
To further test this association, TACH101 was evaluated in a panel of patient-derived xenograft (PDX) and organoid models. The results showed a strong correlation of TACH101 sensitivity with MSI-H status (IC50 ranges 1~150 nM).
The virtual poster presentation titled, "Identification of pharmacodynamic and sensitivity biomarkers for TACH101, a pan-inhibitor of KDM4 histone lysine demethylase" is available for on-demand viewing by conference attendees on the AACR (Free AACR Whitepaper)-NCI-EORTC conference website at View Source