On December 18, 2023 Blueprint Medicines Corporation (Nasdaq: BPMC) reported that Kate Haviland, Chief Executive Officer, will present a corporate overview and 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024, at 10:30 a.m. PT (1:30 p.m. ET) (Press release, Blueprint Medicines, DEC 18, 2023, View Source [SID1234638664]).

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A live webcast of the presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

On December 18, 2023 Nectin Therapeutics Ltd., (Nectin), a biotechnology company developing novel targeted immunotherapies that address resistance to approved immune oncology treatments, reported that it has progressed its Phase 1 clinical trial of NTX1088 to include a combination therapy arm with the immune-oncology drug KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, and expanded the clinical trial to five global sites (Press release, Nectin Therapeutics, DEC 18, 2023, View Source [SID1234638663]).

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The clinical trial sites include City of Hope in California, Sheba Medical Center and Hadassah Medical Center in Israel, along with MD Anderson Cancer Center in Houston, Texas and Ochsner MD Anderson Cancer Center in Louisiana. Notably, the NTX1088 clinical trial is the first to enroll patients at the new Ochsner MD Anderson Cancer Center partnership that was established to ensure that Ochsner cancer patients have access to the most advanced care, including to clinical trials of innovative therapies.

"As a leader in cancer care, Ochsner MD Anderson Cancer Center is committed to providing world-class care to the southeastern Louisiana community," said Dr. Marc Matrana, Director, Precision Cancer Therapies and Research Program at Ochsner. "Part of that commitment is ensuring that our patients have access to clinical trials of innovative new cancer therapies. We are impressed by the promising preclinical data and the novel mechanism of action of PVR blockade, and we are delighted to join with Nectin Therapeutics to support the Phase 1 trial of NTX1088."

NTX1088 is Nectin’s first-in-class lead candidate – a highly potent monoclonal antibody directed against PVR (CD155), a transmembrane protein expressed on cancer cells and associated with resistance to PD1 and PDL1 immune checkpoint inhibitors. PVR blockade by NTX1088 is the first and only therapeutic approach aimed at restoring the antitumor immune activity of DNAM1 (CD226). DNAM1 is a cell surface glycoprotein, central to the function of T-cells and NK cells, which are degraded by PVR on tumor cells. Restoring the expression and activation of DNAM1 by blocking PVR results in increased antitumor activity from T-cells and NK cells. Additionally, PVR blockade by NTX1088 further stimulates an antitumor immune response by preventing the suppressing signaling of several immune checkpoint receptors, including TIGIT and CD96. NTX1088 is currently being clinically evaluated as a monotherapy and in combination with KEYTRUDA in patients with advanced solid tumors.

"We are encouraged by the rapid progress of our NTX1088 clinical program," said Dr. Keren Paz, Chief Development Officer of Nectin Therapeutics. "Our collaboration with Merck, and the rapid expansion of our clinical trial sites significantly increase cancer patients’ access to the trial. We value the commitment of our new partners to the mission of developing life-saving therapies and look forward to advancing innovative medicines to improve the lives of cancer patients."

The NTX1088 Phase 1 trial is being conducted with support from The Cancer Focus Fund, a unique investment fund established in collaboration with The University of Texas MD Anderson Cancer Center. The fund provides investment support to advance promising cancer therapies along with the clinical trial expertise and infrastructure of MD Anderson Cancer Center sites.

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

About NTX1088
NTX1088 is a first-in-class monoclonal antibody directed against a key immune checkpoint, PVR (CD155), currently in a Phase I clinical trial. NTX1088 blocks the interaction between PVR and DNAM1 (CD226), a transmembrane molecule involved in the activation of anti-cancer T-cells and NK cells. By preventing internalization and degradation of DNAM1, NTX1088 leads to restoration of DNAM1 expression on the surface of immune cells, resulting in robust antitumor activity. NTX1088 also blocks PVR interactions with its other ligands, such as TIGIT and CD96, preventing their immune inhibitory signaling. NTX1088 demonstrated superior antitumor activity compared to approved and investigational immune checkpoint inhibitors in preclinical models and had a favorable safety profile in non-human primates. NTX1088 is currently being clinically evaluated as a monotherapy and in combination with KEYTRUDA (pembrolizumab) in patients with advanced solid tumors.

On December 18, 2023 Avstera Therapeutics Corp, an oncology-focused biotech dedicated to addressing the large disease burden and significant unmet need of cancer patients, reported the U.S. FDA clearance of its Investigational New Drug application (IND) for AVS100, a novel highly selective oral HDAC6 inhibitor intended to treat locally advanced or metastatic solid tumors in a Phase I clinical study (Press release, Avstera Therapeutics, DEC 18, 2023, View Source [SID1234638662]).

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"The FDA clearance of our IND for AVS100 marks an amazing step for our company towards the fulfillment of our mission. We are excited to advance this promising new therapy into the clinic to address the disease burden for the benefit of patients, and our transition to a viable clinical stage biotech. AVS100 has demonstrated significant preclinical efficacy, remarkable safety, and durability in numerous in-vivo studies." said Karthik Musunuri, CEO & Co-Founder, Director of Avstera Therapeutics.

AVS100 is a highly selective, orally bioavailable, isoxazole-3-hydroxamate based HDAC6 inhibitor. AVS100 demonstrated unique ability in its class preclinically in suppressing the polarization of macrophages toward pro-tumoral phenotypic pathways. This immunomodulatory function is both pivotal and of keen interest as tumor associated macrophages can constitute up to 50% or more of solid tumor cell mass. AVS100 is AMES negative and demonstrated a strong safety profile in animals, including GLP toxicology studies in dogs and rats with no major adverse events.

"FDA clearance of our IND for AVS100 represents a significant milestone for Avstera’s mission in providing state of the art oncological agents to tackle solid tumors. I am proud of our team’s efficiency as drug developers in advancing to FIH trials in a just over a year since our seed round." said Ajay Raju, Co-Founder & Director of Avstera Therapeutics.

"New findings demonstrating a critical role of tumor-associated macrophages in resistance to immunotherapy suggest that targeting this cell population could both effectively reverse and/or avoid resistance to existing ICB. Therefore, an emerging interest is in developing new immunotherapies targeting this immune population, including selective HDAC6 inhibitors, which have been proven effective modulators of immunosuppressive macrophages. Undoubtedly, the IND approval of AVS100 selective HDAC6 inhibitor will open new opportunities to potentially enhance response rates for patients." said Alejandro Villagra, PhD, Georgetown University Associate Professor and Co-Inventor of AVS100 who also serves on Avstera’s Scientific Advisory Board (SAB).

More About the Phase I Clinical Study

The Phase Ia/b clinical trial for AVS100 is targeting locally advanced or metastatic solid tumors. This trial is an open label, dose-escalation and confirmation study to characterize the safety, tolerability, pharmacokinetics, and MTD of AVS100 when administered as a monotherapy and in combination with pembrolizumab. The primary endpoints are to evaluate the incidence of adverse events including DLTs in monotherapy and in combination, while secondarily assessing the PK, Objective Response Rate (ORR) using RECIST v1.1, and Progression Free Survival (PFS). Avstera will also assess multiple exploratory biomarkers, including conducting multi-omics analyses on patient samples.

Avstera intends to initiate the Phase Ia/b clinical trial in the first half of 2024. The principal investigator of the study is Apostolia M Tsimberidou, MD, PhD, FASCO, FAAAS, Professor of Medicine at MD Anderson Cancer Center, Houston, TX.

On December 18, 2023 Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) reported that it has submitted its Biologics License Application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for its investigational positron emission tomography (PET) imaging agent TLX250-CDx (Zircaix,[1] 89Zr-DFO-girentuximab) in clear cell renal cell carcinoma (ccRCC) (Press release, Telix Pharmaceuticals, DEC 18, 2023, View Source [SID1234638661]).

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Under the Breakthrough Therapy designation, TLX250-CDx has been granted a rolling review process, which enables a progressive submission and review of required modules in a timetable pre-agreed with the FDA. With the BLA submission, Telix has also requested Priority Review, which if granted would support an expedited review time.

Dr. Christian Behrenbruch, Managing Director and Telix Group CEO said, "This is a major milestone and achievement for Telix, which paves the way for a commercial availability for patients in the U.S. in 2024, subject to regulatory review and approval."

James Stonecypher, Chief Development Officer at Telix added, "If approved by the FDA, TLX250-CDx will be the first targeted radiopharmaceutical imaging agent for kidney cancer to be commercially available to patients in the U.S.. The collaborative approach shown by the FDA under the Breakthrough Therapy designation has been highly valuable as we work to bring this novel, non-invasive, first-in-class 89-zirconium-labeled monoclonal antibody (mAb) based imaging agent to market."

Associate Professor Brian Shuch, MD, Director of the Kidney Cancer Program and the Alvin & Carrie Meinhardt Endowed Chair in Kidney Cancer Research at UCLA Institute of Urologic Oncology said, "The ZIRCON study demonstrated the superior sensitivity and specificity of this advanced diagnostic imaging agent, which, if approved, will be the first and only agent available to target carbonic anhydrase IX, a highly relevant target in kidney cancer. This delivers on a major unmet need to provide confidence in the diagnosis of ccRCC, the most aggressive and common form of kidney cancer."

This submission is based on Telix’s successful global Phase III ZIRCON study (Zirconium in Renal Cancer Oncology, ClinicalTrials.gov ID: NCT03849118), which reported positive results in November 2022, meeting all co-primary and secondary endpoints.[2]

Telix has opened an expanded access program in the U.S. and a named patient program in Europe to allow access to TLX250-CDx outside of a clinical trial to patients for whom there are no comparable or satisfactory alternate options.

On December 18, 2023 Innovent Biologics, Inc. ("Innovent") (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune, ophthalmology and other major diseases, and AnHeart Therapeutics ("AnHeart"), a global clinical-stage biopharmaceutical company developing novel precision therapies for people with cancer, reported the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has granted Priority Review Designation[1] for the New Drug Application (NDA) of taletrectinib, a next-generation ROS1 tyrosine kinase inhibitor (TKI), for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who have been previously treated with ROS1 TKIs (Press release, AnHeart Therapeutics, DEC 18, 2023, View Source [SID1234638660]). The CDE accepted the NDA in November 2023.

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Dr. Hui Zhou, Senior Vice President of Innovent, stated: "Taletrectinib’s potential as a best-in-class precision therapy is further underscored by the Priority Review Designation. We will continue to work with the regulatory authorities to support the review of taletrectinib with the goal of bringing this next-generation option to address unmet needs for ROS1-positive NSCLC patients in China."

Bing Yan, MD, Chief Medical Officer of AnHeart, stated: "Taletrectinib’s Priority Review Designation reinforces the urgent need for patients with ROS1-positive NSCLC who were previously treated with ROS1 TKIs in China and the significant clinical advance that taletrectinib may represent for these patients. We look forward to working with the regulatory authorities as they conduct their review of taletrectinib to hopefully bring this breakthrough investigational therapy to patients as soon as possible."

The NDA and Priority Review Designation in China are based on data from the Phase 2 TRUST-I trial (NCT04395677). Data from an interim analysis of TRUST-I were presented at the European Lung Cancer Congress (ELCC) 2023.[Link]

About ROS1-positive Non-small Cell Lung Cancer

More than one million people globally are diagnosed with NSCLC annually, the most common form of lung cancer. It is estimated that approximately 3% of people with NSCLC in China are ROS1-positive. There are two approved first-generation TKIs for people with newly diagnosed advanced or metastatic ROS1-positive NSCLC and no approved therapies for people whose ROS1-positive NSCLC has progressed following treatment with these medicines. Up to 35% of people newly diagnosed with metastatic ROS1-positive NSCLC have tumors that have spread to their brain (brain metastases), increasing up to 55% for those whose cancer has progressed following initial treatment.

About Taletrectinib

Taletrectinib is an oral, potent, brain penetrant, selective, next-generation potential best-in-class ROS1 inhibitor.

Taletrectinib is being evaluated in ROS1-positive NSCLC patients in two Phase 2 trials, TRUST-I (NCT04395677) in China, and TRUST-II (NCT04919811), a global pivotal trial.

In March 2022, taletrectinib was granted Breakthrough Therapy Designation by the CDE of China’s NMPA for the treatment of adult patients with advanced or metastatic ROS1-positive NSCLC who have previously been treated with ROS1 TKIs as well as those who have not previously been treated by ROS1 TKIs (TKI-naïve).

Taletrectinib has also been granted Breakthrough Therapy Designation in the United States for the treatment of ROS1-positive NSCLC by the U.S. Food and Drug Administration (FDA).

In June 2021, Innovent and AnHeart entered into an exclusive license agreement for the co-development and commercialization of taletrectinib in Greater China, including mainland China, Hong Kong, Macau and Taiwan.