On August 15, 2024 Pathos AI, Inc. (www.pathos.com), a biotechnology company focused on revolutionizing precision medicine in cancer by harnessing the power of machine learning to transform drug development, reported the world-wide license of PRT811, a potent, selective, and orally bioavailable brain penetrant SAM-competitive PRMT5 inhibitor from Prelude Therapeutics (Press release, Pathos AI, AUG 15, 2024, View Source [SID1234653830]).

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PRT811 (renamed P-500) was developed by Prelude Therapeutics and completed a Phase 1 trial in March 2023. The trial enrolled patients with solid tumors including high-grade glioma and uveal melanoma and has potential application in other indications with high unmet need. Out of 16 patients with high-grade glioma with isocitrate dehydrogenase mutations (IDH+) in the Phase 1 trial, two confirmed complete responses (CR) were observed. At last follow-up, 1 response is ongoing and has lasted 31.0 months while the duration of response for the second CR patient was 7.5 months. Additionally, 1 patient achieved an unconfirmed partial response (PR).

In addition, out of 23 uveal melanoma patients (10 patients with splicing factor 3B subunit 1 (SF3B1) splicing mutations and 13 without an SF3B1 mutation), one confirmed PR (duration of response of 10 months) and a second unconfirmed PR were observed, both in patients SF3B1 mutations.

In the entire safety population (N=86), the most common adverse events of any grade, with an incidence of >20% were nausea (60.5%), vomiting (46.5%) fatigue (36.0%), constipation (29.1%), and thrombocytopenia (24.4%), and were predominantly grade 1-2. The most common adverse events (grade ≥3), occurring >5% were thrombocytopenia (9.3%), anemia (9.3%), and fatigue (5.8%).

"These results from Prelude’s Phase 1 study are promising news for high-grade glioma patients and clinicians, who still have limited treatment options with chemotherapy and radiation that hasn’t changed in decades. With our AI Platform, we aim to increase the already encouraging response rate of P-500 through a novel biomarker-driven strategy, ultimately bringing this medicine to patients as efficiently as possible," said Ryan Fukushima, Pathos CEO.

"Prelude’s discovery engine has delivered a number of first- or best-in-class precision medicines including PRT811, a molecule that has shown early promise in the treatment of high-grade glioma. We are confident that Pathos AI, sharing our passion for precision medicine and commitment to serving cancer patients with high unmet need, is an ideal company to drive the development of this molecule forward for patients." said Sean Brusky, Prelude CBO. "The resources from this transaction will support advancing Prelude’s pipeline."

About P-500
P-500 (previously PRT811) is a selective, brain-penetrant small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5) that has the potential to provide clinical benefit for patients with advanced solid tumors, including high-grade glioma and uveal melanoma.

PRMT5 is an enzyme that adds methyl groups to proteins in cells using a molecule called S-adenosylmethionine (SAM) which regulates protein function and interactions. Several processes that support cancer cell growth and spread depend on PRMT5, making P-500 relevant not only to advanced high-grade glioma and uveal melanoma (in which objective responses to P-500 were observed in the Phase 1 clinical trial) but also to a number of other cancer indications. Preclinical studies have demonstrated PRMT5 inhibition can sensitize cancer cells to other treatments, expanding the application of P-500 to combination therapy in additional indications.

On August 15, 2024 Stada reported Interim Report on the First Half of 2024 (Presentation, Stada, AUG 15, 2024, View Source [SID1234647165]).

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On August 15, 2024 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical stage pharmaceutical company, reported it will be presenting at two investor conferences (Press release, Lixte Biotechnology, AUG 15, 2024, View Source [SID1234645984]):

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■ The Investor Summit Summer 2024 Virtual Conference Tuesday, August 20, 2024, at 2 p.m. EDT/11 a.m. PDT. To access the presentation, visit LIXTE’s investor relations website at View Source

■ The H.C. Wainwright 26th Annual Global Investor Conference, on demand starting at 7 a.m. EDT/4 a.m. PDT, September 9-11, 2024. To access the virtual presentation, register for the conference at www.hcwevents.com/annualconference.

Bas van der Baan, CEO of LIXTE, will present an overview of the Company’s clinical trials with LB-100, its proprietary compound focused on enhancing chemotherapy and immunotherapy treatments as cancer therapies. Proof-of-concept clinical trials are currently in progress for colorectal, ovarian and sarcoma cancers, including trials funded by GSK and Roche.

On August 15, 2024 Nuntius Therapeutics ("Nuntius"), a biotech company developing transformative mRNA therapies through their advanced delivery technology, reported that they have entered into a collaboration agreement with Taiho Pharmaceutical Co., Ltd. ("Taiho"), a leading company in Japan for developing innovative medicines for the treatment of cancer (Press release, Nuntius Therapeutics, AUG 15, 2024, View Source [SID1234645957]). Taiho will use Nuntius’ cell-specific peptide dendrimer- and lipid-based nanocarriers to develop novel mRNA cancer immunotherapies.

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The collaboration agreement follows a successful feasibility study which evaluated Nuntius’ delivery capabilities.

"We are thrilled to embark on this collaboration with Taiho to improve treatments for cancer patients. Taiho is an excellent partner for us given their strong oncology focused research and commercial capabilities," said Benita Nagel, CEO & Co-Founder of Nuntius.

"Delivery remains a major challenge for mRNA therapies. This agreement validates our nanocarriers as an exceptionally effective cell-specific delivery technology for genetic medicines," added Albert Kwok, PhD, CSO & Co-Founder of Nuntius.

The financial terms of the collaboration agreement were not disclosed.

Nuntius’ programmable and scalable nanocarriers can effectively and safely target cell types beyond the liver and outperform leading delivery technologies. The company recently published its machine learning approach to discovering high-performing mRNA nanocarriers in the journal Advanced Science. Nuntius’ high-throughput, in silico screening of delivery vehicle candidates significantly reduces the time and cost required to bring mRNA therapies to the clinic.

On August 15, 2024 EnPlusOne Biosciences, Inc., a biotechnology company harnessing the power of enzymes to deliver better RNA at scale, reported that it is part of a collaboration led by the Wyss Institute for Biologically Inspired Engineering at Harvard University that has been awarded an agreement for up to $27 million by the Advanced Research Projects Agency for Health (ARPA-H) (Press release, EnPlusOne Biosciences, AUG 15, 2024, View Source [SID1234645955]). The agreement covers multi-disciplinary efforts to develop a disease-agnostic novel RNA therapeutic with the potential to treat diverse diseases, including types of cancer and infectious diseases, and to be effectively and rapidly deployable. Initially, up to $3.5 million of the agreement is focused on the EnPlusOne enzymatic platform.

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ARPA-H is a federal funding agency which funds transformative biomedical and health research breakthroughs, translating prioritized research from the lab to applications in the marketplace. The ARPA-H award will allow the Wyss Institute team to significantly accelerate and expand efforts to advance the therapy towards an Investigational New Drug (IND) submission to the Federal Food and Drug Administration (FDA).

The team is initially focusing on cancer and will explore the platform’s potential for difficult-to-treat infectious diseases. The ARPA-H project will build on a newly developed Duplex RNA technology and leverage the powerful RNA delivery capabilities developed by groups at the Wyss Institute, along with the groundbreaking enzymatic RNA synthesis capabilities of EnPlusOne to optimize the RNA’s design and scalability.

Dan Ahlstedt, co-founder and Chief Operating Officer said, "We come full circle rejoining our Wyss colleagues for this exciting collaboration. This comprehensive program will give us the opportunity to demonstrate how enabling modifications and limitless scale can be unlocked by our ezRNA platform. We are grateful to the Wyss Institute and ARPA-H for recognizing the current problems facing RNA manufacturing and sharing our vision that an enzymatic approach is the future. We look forward to progressing our platform alongside this team as we collectively work to address critical human health challenges."

Wyss Institute Core Faculty member Natalie Artzi, Ph.D. is the lead-investigator on the project with co-principal investigator and Wyss Institute Founding Director Don Ingber, M.D., Ph.D. Artzi also is Associate Professor of Medicine at Harvard Medical School (HMS) and Brigham and Women’s Hospital and a Principal Research Scientist at MIT. Additional key investigators include Wyss Institute Director of Translational R&D Kenneth Carlson, Ph.D., a drug discovery and development specialist with extensive industry experience, and Wyss Institute Core Faculty member William Shih, Ph.D., who developed a DNA origami platform that allows the precise and highly effective presentation of RNA drugs, cancer and pathogen-derived antigens, and immune activating adjuvants to the immune system. Shih’s team will provide their DNA nanotechnology approach as an additional drug delivery option.

The collaboration is another validation of the potential for enzymatic synthesis to address the needs of a rapidly expanding RNA therapeutic market as the industry faces demand for RNA production that cannot be met by chemical synthesis alone. It builds upon recent important progress shared by EnPlusOne, including the breakthrough synthesis of the antisense strand of the commercially approved siRNA drug, Leqvio (inclisiran), a treatment for hypercholesteremia (high cholesterol) licensed from Alnylam Pharmaceuticals, Inc. by Novartis, that currently addresses a multi-million patient population.

EnPlusOne was launched by the Wyss Institute in 2022 to commercialize its enzymatic RNA oligonucleotide synthesis technology developed in the laboratory of co-founder George Church, PhD. Seed financing was led by Northpond Ventures, with participation from Breakout Ventures, Coatue, and individual investors.