On November 17, 2023 Avistone Biotechnology Co. Ltd. ("Avistone" or "the Company"), an innovative biotechnology company focused on precision oncology therapeutics, reported that they received conditional approval from the National Medical Products Administration (NMPA) to commercialize Vebreltinib (also referred to as PLB1001, APL-101, Bozitinib, CBT-101) to treat patients with MET exon 14 skipping non-small cell lung cancer (NSCLC) in China (Press release, Avistone Pharmaceuticals, NOV 17, 2023, View Source [SID1234637789]).

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"The development of drugs for indications related to MET targets has always been a difficult one. With the NMPA approval of Vebreltinib, we look forward to providing this new treatment option to patients with MET exon 14 skipping NSCLC in China"

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The conditional approval was based on results from a pivotal Phase II KUNPENG study with an objective response rate (ORR) of 75.0% and median progression-free survival (PFS) in patients with advanced METex14 mutations to 14.1 months with a good safety profile as was recently presented at the 2023 European Society for Clinical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Madrid, Spain (Poster# 1379P).

MET signaling is implicated in cell proliferation, migration, invasion, and survival. Genomic alterations in MET can manifest in driving oncogenesis in the form of MET exon 14 skipping (METex14) or other activating mutations, MET gene amplification, gene fusion and MET protein overexpression. METex14 is an independent prognostic factor associated with poorer survival rates in patients with NSCLC.

"The development of drugs for indications related to MET targets has always been a difficult one. With the NMPA approval of Vebreltinib, we look forward to providing this new treatment option to patients with MET exon 14 skipping NSCLC in China" said Dr. Hepeng Shi, Chairman, CEO, and Founder of Avistone.

Avistone holds the exclusive rights to Vebreltinib in China, Hong Kong and Macau.

On November 17, 2023 Phase Genomics, Inc., a global innovator for industry-leading genomic solutions, reported the presentation of new data from the proximity ligation sequencing-based OncoTerra cytogenomics platform on the leading-edge AVITI next-generation sequencing system (Press release, Phase Genomics, NOV 17, 2023, View Source [SID1234637788]). The platform offers a revolutionary leap toward rapid risk stratification for acute myeloid leukemia (AML).

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Traditional cytogenetics has relied on a battery of assays for comprehensive evaluation, including karyotyping, FISH, and CMA, identifying large-scale structural alterations in chromosomes. Not only do these tests often fail to detect clinically relevant sub-microscopic variations, the cascade approach prolongs the time from sample collection to actionable insight.

Phase Genomics addresses these legacy challenges with the combination of its OncoTerra technology and Element’s AVITI system for next-generation sequencing, providing a high-resolution alternative for comprehensive, streamlined risk stratification in the research setting. OncoTerra on AVITI has demonstrated detection of the full range of chromosomal abnormalities observed in cancer from sample to cytogenomic insights.

"Our results speak to the real potential of OncoTerra on the AVITI sequencer. The next-generation cytogenomics all-in-one approach is primed to replace fragmented legacy tests with a single, rapid assay," said Ivan Liachko, founder and CEO of Phase Genomics. "The ability to uncover previously undetected structural alterations in AML opens the exploration for new avenues for targeted therapies and personalized medicine. OncoTerra on AVITI represents a major stride toward accelerating cancer diagnoses and improving patient outcomes."

The research leveraged OncoTerra for de novo risk assessment of 32 AML patient samples in the retrospective setting. Utilizing proximity ligation sequencing, OncoTerra delivered exceptional proficiency in detecting structural aberrations directly within AML samples, as well as seven variants of known clinical significance not detected by standard-of-care cytogenetic testing. These alterations included sub-microscopic inversions, terminal translocations, insertions, and copy-neutral loss of heterozygosity. The increased resolution and integrated analysis of the genome using OncoTerra yielded additional findings in eleven cases without previously detected chromosome abnormalities.

The study also highlighted the improved efficiency and read quality of the AVITI sequencer compared to other platforms. Benchmarking AVITI against Illumina NovaSeq platforms revealed a 20% enhancement in the fraction of effective read pairs, indicating greater sequencing efficiency and coverage.

Phase Genomics, the recent recipient of the Biotech Breakthrough Award for Metagenomics Innovation Of The Year, will highlight the power of OncoTerra for AML in poster G025, "Next-generation cytogenomics using proximity ligation technology on the AVITI genome sequencing platform" on November 18. Discover more about next-generation cytogenomics powered by proximity ligation technology in four presentations at AMP23, or connect with the Phase Genomics team at Booth 810.

OncoTerra is available for research use only and is not for use as a clinical diagnostic.

On November 17, 2023 BioMimetix JV, LLC, a clinical-stage biotechnology company developing metalloporphyrins, a novel drug class for the treatment of cancer patients, reported the presentation of encouraging data from its Phase 2 study of lead candidate, BMX-001, for the treatment of high-grade glioma (HGG) at the 2023 Society for Neuro-Oncology (SNO) Annual Meeting (Press release, BioMimetix Pharmaceutical, NOV 17, 2023, View Source [SID1234637787]). In the Phase 2 study, BMX-001 in combination with radiotherapy (RT) and temozolomide (TMZ) demonstrated a 6.6 month increase in median survival, mitigation of radiation-induced cognitive issues, and improved white matter integrity with a tolerable safety profile, compared to current standard of care. The abstract will be published in SNO’s official journal, Neuro-Oncology.

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"Patients with high grade glioma are challenging to treat because the disease grows quickly, ultimately outcomes are very poor for these patients and often carries an unfavorable prognosis. Current treatments aim to remove the tumor through surgery or slow its growth using chemotherapy and radiation therapy, but they are not curative and are associated with significant toxicities, including radiation-induced cognitive dysfunction that is responsible for negatively impacting the quality of life for our patients. The diversity of cases makes finding a cure difficult, and seeing durable responses in these patients is encouraging," said Katy Peters, MD PhD FAAN (Lead Principal Investigator). "BMX-001 is a promising first-in-class molecule with clinically meaningful activity and the potential to become a new treatment option for patients diagnosed with high grade glioma."

"The exciting data presented today at SNO underscore what I have sought to achieve over the past several decades in our scientific research. The primary mechanism of action of a redox active metalloporphyrin is modulation of key pathways of cellular signaling in a pattern that augments radiation therapy in killing tumor cells while protecting normal tissues. This is demonstrated by the promising effects seen in this trial with BMX-001 on patients with high grade glioma receiving concurrent radiation and chemotherapies," said James Crapo, M.D., Chief Executive Officer at BioMimetix. "High grade glioma is an aggressive brain cancer with a low survival rate that affects 10,000-15,000 people in the US per year. A substantial increase in median survival over the standard of care coupled with improved quality of life represents a significant stride towards bringing patients a transformative treatment. We are motivated to continue developing BMX-001 for this area of high unmet need, while also continuing to explore the utility of BMX-001 in other cancer types."

Presentation Highlights:

The Phase 2 clinical trial included 160 subjects with newly diagnosed high grade glioma of which 145 completed protocol treatment.
Patients on concurrent RT/TMZ who also received BMX-001 were found to have an increased median survival of 6.6 months.
BMX-001 also mitigated radiation-induced cognitive issues – demonstrated by an increase in Hopkins Verbal Learning Test scores at 6-months post-baseline versus the control group, and an improvement in Brief Assessment of Cognition (BAC) scores.
BMX-001 was found to have an excellent safety profile.
Presentation Details:
Title: Results of BMX-HGG study: a multi-institutional, randomized phase 2 clinical trial of concurrent chemoradiation with or without BMX-001 in patients with newly diagnosed high-grade glioma
Abstract Number: LTBK-09
Date and Time: Friday, November 17, 2023, from 9:05 – 9:15 a.m. PT
Session: Plenary I, Exhibit Hall C
Presenter: Katherine Peters, M.D., Duke University Medical Center

About BMX-001

BMX-001 is a metalloporphyrin, a novel class of redox-active, small molecule. The active center is designed to mimic the center of superoxide dismutase. The primary mechanism of action is modulation of cellular signaling pathways. BMX-001 inhibits both NFkB and HIF-1a. By inhibiting these pro-survival and pro-angiogenic transcription factors, BMX-001 augments tumor killing by radiation therapy and inhibits tumor regrowth. The inhibition of NFkB blocks major components of the inflammatory cascade which simultaneously results in protection of normal tissue from radiation induced injury. BMX-001 is also being developed in head and neck cancer, anal cancer, and rectal cancer and has been previously granted Orphan, Fast Track and Breakthrough designations by the FDA.

On November 17, 2023 March Biosciences (March Bio), a clinical stage biotechnology company committed to combating challenging cancers unresponsive to existing immunotherapies, reported that it has received a notice of award for a major competitive grant from the Cancer Prevention and Research Institute of Texas (CPRIT) to help support continued clinical development of its innovative chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of relapsed and refractory CD5 positive T-cell cancers (Press release, March Biosciences, NOV 17, 2023, View Source [SID1234637786]). The approximately $13.4 million product development award is intended to support March Bio’s upcoming Phase 2 clinical trial of MB-105 for the treatment of relapsed and refractory T-cell lymphomas.

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MB-105 is currently being assessed in a Phase 1 clinical trial in patients with relapsed and refractory T-cell malignancies at Baylor College of Medicine. This grant follows a $4.8 million investment from the Cancer Focus Fund announced earlier this month also to support the MB-105 Phase 2 trial.

"We are committed to advancing MB-105 and creating a best and first-in-class therapy for patients with these difficult cancers," commented Sarah Hein, cofounder and CEO of March Biosciences. "We expect that receipt of this substantial non-dilutive award from CPRIT will serve as a catalyst to our current financing activities and allow us to continue to move quickly to advance MB-105 as a novel treatment for patients seeking better options."

Patients with T-cell malignancies face a dismal prognosis once their disease becomes relapsed or refractory to first-line therapies and have extremely limited continuing treatment options, resulting in just 15% survival over about 3 years. The development of specific therapies for these indications is difficult due to the fact that many potential targets, including the CD5 receptor targeted by MB-105, are present on both normal and malignant T-cells, which means that therapies targeting malignant T-cells risk damaging the healthy T-cells needed for normal immunity.

Since CD5 is widely expressed by both normal and malignant T-cells, the MB-105 CD5 CAR-T cell therapy has been specifically engineered to preserve many healthy T-cells while maintaining potency against CD5 positive tumor T-cells. In an ongoing Phase 1 trial conducted at the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital, and Texas Chidren’s Hospital, MB-105 has demonstrated a favorable safety profile and encouraging early efficacy results in patients with both relapsed and refractory T-cell lymphoma and T-cell acute lymphoblastic leukemia.

March Biosciences was cofounded with inventors Drs. Max Mamonkin and Malcolm Brenner, leading scientists from the Center for Cell and Gene Therapy who are both recipients of CPRIT research awards. Dr. Mamonkin is an Associate Professor at Baylor and serves as Chief Scientific Officer at March Biosciences. He was recently awarded the Outstanding New Innovator Award by the American Society for Cell and Gene Therapy. Dr. Brenner is a renowned leader in the field, with an extensive history of technology invention contributing to the launch of both private and public biotechnology companies. He serves as an advisor to cell therapy companies across the industry.

Dr. Mamonkin commented, "The treatment of T-cell lymphomas and leukemias has been particularly challenging, which led us to focus on the development of this new approach early on. We have been highly encouraged by the clinical results we have already seen, and I am pleased that CPRIT has recognized the potential importance of these efforts to patients and will support the continued development of the CD5 CAR-T approach."

March Bio is currently preparing for its Phase 2 trials of MB-105 with a development collaboration with recent CPRIT awardee, CTMC, a joint venture between MD Anderson Cancer Center and National Resilience.

On November 17, 2023 CytoMed Therapeutics Limited (NASDAQ: GDTC) ("CytoMed" or "Company"), a Singapore-based biopharmaceutical company focused on harnessing its proprietary technologies to develop novel donor-derived cell-based allogeneic immunotherapies for the treatment of various cancers, reported its first half of 2023 financial results and provided clinical and corporate updates (Press release, Cytomed Therapeutics, NOV 17, 2023, View Source [SID1234637784]).

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"We are committed to advancing our "off-the-shelf" allogeneic cellular immunotherapies for a broad spectrum of cancer," said Peter Choo, Chairman of CytoMed. "By committing ourselves to our stem cell research and cell therapy, we have made extraordinary progress thus far and look towards the future with overseas ambition especially China. We benefit from the low cost infrastructure in Southeast Asia."

Clinical Updates

In January 2023, the Company received formal approval from the Health Sciences Authority (HSA) in Singapore to conduct a Phase I clinical trial and has begun to recruit blood donors in July 2023. The clinical trial, in partnership with the National University Hospital (NUH), Singapore, will use the donor blood to manufacture allogeneic CAR-γδ T cells for the trial. The cells will be processed in CytoMed’s Good Manufacturing Practice (GMP) facility in Malaysia.

As of November 2023, the Company are translating two exclusively licensed technologies, namely donor blood cell-based CAR-γδ T cell technology and induced pluripotent stem cell-based γδ NKT cell technology. The former has been granted patents in the US and China, the latter in Japan and China.

Financial Results for the Six Months Ended June 30, 2023

Net Loss: For the six months ended June 30, 2023, the Company’s unaudited net loss amounted to S$1.16M ($860,695) excluding expenses related to its NASDAQ Initial Public Offering (IPO) in April 2023 and the costs associated with being a public listed company, compared to S$936,377 for the six months ended 2022.

Cash and Cash Equivalents: As of June 30, 2023, the Company had cash and cash equivalents of S$10.44M ($7.72M). Over the course of the last six months, the Company raised S$12.94M ($9.57M) gross proceeds from the IPO.

R&D Expenses: The Company’s research and development expenses were S$811,319 ($599,955) and S$604,043 for the six months ended June 30, 2023, and 2022, respectively. This was primarily due to the clinical progress achieved over the last six months.

G&A Expenses: The Company’s general and administrative expenses were S$1.50M ($1.11M) and S$306,457 for the six months ended June 30, 2023, and 2022, respectively. The increase was primarily driven by non-recurring IPO expenses and the costs associated with being a public listed company.

Conference Call Information

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