On December 16, 2024 MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, reported that the FDA has designated THIO for the treatment of pediatric-type diffuse high-grade gliomas (PDHGG) as a drug for a "rare pediatric disease (Press release, MAIA Biotechnology, DEC 16, 2024, View Source [SID1234649147])."

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"THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children. THIO is shown to activate the immune system while evading tumor immunosuppression, a novel therapeutic approach for this devastating childhood disease," said MAIA Chairman and Chief Executive Officer Vlad Vitoc, M.D. "We are proud to receive the FDA’s Rare Pediatric Disease designation for THIO, which significantly bolsters our plans for continuing research in the PDHGG indication."

MAIA’s Vice President and Head of Regulatory and Quality K. Robinson Lewis added, "Rare pediatric disease designation also offers a highly valuable incentive for MAIA. Upon FDA approval of a future new drug application in PDHGG, MAIA would be eligible to receive a priority review voucher that can be redeemed or sold as an asset at a very high valuation."

Rare pediatric disease priority review vouchers (PRVs) can be redeemed by drug developers for FDA priority review of a different product or transferred or sold to another sponsor. Since 2015, FDA priority review vouchers have sold as assets at an average amount of $100 million.1

Previous research showcased THIO’s potency as a treatment for a PDHGG subtype known as diffuse intrinsic pontine glioma (DIPG). A research collaboration between MAIA and Nationwide Children’s Hospital found that THIO combined with ionizing radiation (IR) resulted in significantly decreased cell proliferation and produced potent anticancer effects in highly aggressive DIPG. The data was presented in April 2024 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

MAIA collaborated with Only Orphans Cote for THIO’s designation request. Only Orphans Cote is a foremost provider of regulatory services and strategies for FDA orphan drug designations and marketing authorization.

In addition to its rare pediatric disease designation in PDHGG, THIO holds orphan drug designations (ODD) in three cancer types: hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma. MAIA believes that THIO is the only direct telomere-targeting agent currently in clinical development.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

On December 16, 2024 The Children’s Brain Tumor Network (CBTN), a consortium of leading pediatric brain tumor research institutions, reported that it has formed a groundbreaking partnership with Day One Biopharmaceuticals (Day One), a company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases (Press release, Day One, DEC 16, 2024, View Source [SID1234649146]). This new partnership between CBTN and Day One aims to accelerate the development of new treatments for children with brain tumors. It leverages the robust Pediatric Brain Tumor Atlas (PBTA), a comprehensive resource containing genomic, clinical, and imaging data from over 7,600 pediatric brain tumor patients. Day One will utilize this data set to design clinical trials.

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"We are deeply grateful for this partnership, which reflects a shared commitment to advancing therapies for children with brain tumors," said Dr. Angela Waanders, LLG Study Chair for the Children’s Brain Tumor Network. "Uniting CBTN resources with Day One’s innovative approach accelerates the delivery of clinical trials to children worldwide."

Together, CBTN and Day One aim to accelerate the development of new treatments for children with brain tumors.

CBTN’s innovative approach ensures data remains open to the global research community while facilitating collaboration with commercial entities like Day One.

"CBTN’s commitment to open access data, coupled with their world-class expertise in pediatric brain tumors, makes them an ideal partner for Day One," said Elly Barry, M.D., chief medical officer, Day One Biopharmaceuticals. "This collaboration should allow us to better design clinical trials to potentially bring new therapies to children in need."

The partnership between CBTN and Day One also paves the way for developing trial-ready external control arms in pediatric clinical studies.

On December 16, 2024 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported the development candidate nomination of IDE251, a potential first-in-class KAT6/7 dual inhibitor (Press release, Ideaya Biosciences, DEC 16, 2024, View Source [SID1234649145]).

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"We are pleased to announce the nomination of IDE251 as our third development candidate this quarter and 8th development candidate in our precision medicine oncology pipeline. IDE251 has a potential first-in-class product profile and selectively inhibits two epigenetic modulators, KAT6 and KAT7, and based on the preclinical profile we believe there is an opportunity for an enriched response in 8p11 amplified cancers, which occur in 15% of breast cancers patients and in up to 17.5% in squamous NSCLC," said Yujiro S. Hata, President and Chief Executive Officer of IDEAYA Biosciences.

"IDE251 is a promising potential first-in-class molecule designed to selectively target both KAT6 and KAT7 while sparing other structurally similar KAT family members. KAT6 and KAT7 are mechanistically intertwined epigenetic modulators of cell identity and lineage commitment programs corrupted by oncogenic transformation. Dual KAT6/7 inhibition with IDE251 delivers robust and durable anti-tumor activity, superior to KAT6 inhibition alone, in preclinical tumor models with 8p11 amplifications as well as in biomarker selected indications dependent upon lineage-specific transcription factor activity. IND-enabling studies are progressing as planned and we are targeting to bring this program to the clinic next year," commented Michael White, Ph.D., Chief Scientific Officer, IDEAYA Biosciences.

IDE251 is an equipotent, highly selective, small molecule dual inhibitor of the lysine acetyltransferase (KAT) 6 and 7, both of which have been shown to support cancer cell survival. IND-enabling studies to support the potential clinical evaluation of IDE251 monotherapy in patients with breast and lung cancers with 8p11 amplification are ongoing, as well as additional opportunities in the setting of lineage addiction. Based on IDEAYA’s biomarker evaluation, 8p11 amplification prevalence is projected to be approximately 15% in breast cancer and 17.5% in squamous NSCLC.

IDEAYA is targeting an Investigational New Drug (IND) submission to the U.S. Food and Drug Administration (FDA) in 2025 for IDE251, subject to satisfactory completion of ongoing preclinical and IND-enabling studies.

On December 16, 2024 Microbio Co., Ltd. (4128) reported encouraging results from an exploratory clinical trial of MS-20 combined with Keytruda (pembrolizumab) in stage IIIb/IV non-small cell lung cancer (NSCLC) (Press release, Microbio Co, DEC 16, 2024, View Source [SID1234649143]). The trial showed a threefold increase in the objective response rate (ORR) for the MS-20-Keytruda group (75%) compared to Keytruda alone (25%). Additionally, the median progression-free survival (PFS) for MS-20 patients improved from 4.5 months to over 12 months, with a complete response (CR) rate of 12.5%.

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Enhancing Immunotherapy with MS-20

MS-20, developed by Microbio, is a microbiome-based postbiotic designed to improve cancer immunotherapy by modulating the gut microbiome. "The results support MS-20’s potential to reshape the microbiome and enhance Keytruda’s effectiveness in treating advanced NSCLC," said Dr. Wan-Jiun Chen, Executive VP of Research & Development at Microbio. "Currently, hundreds of ongoing trials are aimed to find novel therapeutic agents for improving the efficacy of cancer immunotherapy, and this proof-of-concept trial supports the use of MS-20 in such application."

This randomized, double-blind, placebo-controlled trial involved 15 patients, with 12 analyzed in the modified intent-to-treat (mITT) population (ClinicalTrials.gov ID: NCT04909034). Of the 8 patients receiving MS-20 and Keytruda, 6 (75% ORR) responded positively, including 1 with an ongoing CR lasting over 22 months and 5 with partial responses (PR). In contrast, only 1 of 4 patients in the placebo and Keytruda group showed a PR (25% ORR). The median PFS for the MS-20-Keytruda group was over 12 months, compared to 4.5 months in the placebo group.

Scientific Insights and Next Steps

Preclinical research published in Gut Microbes earlier this year supported these findings, showing that MS-20 enhances immune responses by increasing effector CD8 T cells in the tumor microenvironment. Microbio plans to use these results to pursue global collaborations and further its microbiome-based cancer immunotherapy platform.

On December 16, 2024 SK Biopharmaceuticals, a biotech company focusing on the research, development and commercialization of treatments for disorders of the central nervous system and oncology worldwide, reported a research collaboration agreement with ProEn Therapeutics, a biotech company dedicated to advancing oncology treatments, to further extend its oncology research capability and expand its pipeline of radiopharmaceutical therapies (RPT) (Press release, SK biopharmaceuticals, DEC 16, 2024, View Source [SID1234649142]).

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Under this agreement, both sides seek to advance up to two preclinical candidates for the development of novel radiopharmaceutical drugs by 2027 – the year when SK Biopharmaceuticals aims to become a global leading RPT player, via strengthened internal and external resources.

This joint research builds on a series of SK Biopharmaceuticals’ global strategic partnerships, including the in-licensing of a radiopharmaceutical compound, and a supply agreement to secure actinium-225, an alpha-particle emitting radioisotope, since the company unveiled its "RPT Roadmap" to gain a competitive edge in the rapidly growing field of nuclear medicine.

SK Biopharmaceuticals will leverage ProEn Therapeutics’ ArtBody platform, a dual-target binding technology that incorporates small proteins[1] to identify and target specific tumor antigens – enhancing tumor selectivity – for the development of potential cancer treatments, while minimizing damage to healthy tissues. ArtBody, which has intrinsic advantages of high stability and structural robustness, can be mass-produced using bacteria, making it ideal for industrial applications.

Il-Han Lee, Chief Executive Officer of ProEn Therapeutics, said, "We are pleased to enter this joint research, and positive that the ArtBody platform will generate synergy with and complement SK Biopharmaceuticals’ radiopharmaceutical therapy business. ProEn Therapeutics will push to produce the best possible outcome that can meet not only the two companies’ expectations, but also patients’ needs."

Donghoon Lee, Chief Executive Officer of SK Biopharmaceuticals, said, "This collaboration with ProEn Therapeutics is significant as the platform technology will help overcome the limitations of existing therapies. We will aim to develop more effective, safer treatments, while leading global RPT research and development efforts."