On September 11, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS) (Press release, Agios Pharmaceuticals, SEP 11, 2024, View Source [SID1234646507]).

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"Receiving orphan drug designation for tebapivat in MDS underscores the importance of bringing new oral treatment options to patients suffering from this rare disease," said Sarah Gheuens, M.D., Ph.D., chief medical officer and head R&D at Agios. "We aim to deliver the first oral therapy that addresses anemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000-80,000 patients in the U.S. and EU5 and accounts for approximately 70% of MDS cases."

Agios completed a Phase 2a study of tebapivat in lower-risk MDS late last year and is currently initiating a Phase 2b study of tebapivat in lower-risk MDS.

The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, orphan drug designation qualifies a company for incentives, including tax credits, exemptions from certain FDA fees for clinical trials, and the potential for seven years of market exclusivity following drug approval.

Mitapivat, the company’s lead PK activator, was previously granted FDA orphan drug designation for the treatment of PK deficiency, thalassemia, and sickle cell disease.

Tebapivat is not approved for use by any regulatory authority.

On September 10, 2024 PDX Pharma reported that it has successfully met all the milestones of our Phase I SBIR and has received approval from the National Cancer Institute (NCI) to advance to the Phase II project, valued at $2 million (Press release, PDX Pharmaceuticals, SEP 10, 2024, View Source [SID1234646513]). This project (R44CA285233) focuses on the development of our nanotherapeutic, PETTRA (PLK1 and EGFR Targeted Therapy and Radiation Sensitizer). PETTRA is built upon our patented nanoparticle delivery platform, Pdx-NP, which enables the co-delivery of an anti-EGFR antibody (serving as both a cancer-homing agent and an EGFR inhibitor) and PLK1 siRNA (killing cancer cells and sensitizing them to radiation). We have overcome the limitations of traditional nanoparticle delivery systems, achieving a long circulation half-life (e.g., 25 hours in monkeys), a tenfold increase in siRNA accumulation in tumors, and specific delivery to target cells (by 5 to 8-fold over normal cells). Additionally, we have demonstrated excellent PLK1 gene knockdown (e.g., 84%) and significant tumor inhibition (e.g., 91%) in mouse models. We would like to extend our gratitude to the NCI for their continued support and congratulate our team on this achievement!

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On September 10, 2024 Xcell Biosciences Inc. (Xcellbio), an instrumentation company focused on cell and gene therapy applications, reported it has advanced its collaboration with Labcorp with the installation of a new AVATAR Foundry system at the healthcare company’s Ann Arbor, Michigan site (Press release, Xcell Biosciences, SEP 10, 2024, View Source [SID1234646497]). This installation, part of the beta access program for Xcellbio’s latest instrument, allows scientists at Labcorp to extend the potency benefits observed in small-scale workflows on the previously installed AVATAR Odyssey platform up to large-scale automated cell therapy manufacturing workflows.

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Xcellbio has developed the AVATAR incubator system for cell and gene therapy research and development with the AVATAR Odyssey system designed for small-scale research and process development needs. Its latest platform, the AVATAR Foundry system, is a current good manufacturing platform (cGMP) that delivers novel capabilities for improving the potency of cell and gene therapies. These capabilities are especially important for cell therapies targeting solid tumors, which often face overwhelming challenges in the hostile tumor microenvironment (TME).

"After using the AVATAR Odyssey system for more than two years, we have seen results demonstrating benefits for both cell therapy expansion rates and anti-tumor potency. We are eager to scale our workflows with the new AVATAR Foundry instrument, which could allow us to support customers early in their development of novel manufacturing techniques for cell and gene therapies," said Maryland Franklin, Ph.D., vice president and enterprise head of cell and gene therapy at Labcorp. "This technology complements our strong capabilities in analytical testing and our commitment to using potency assays and downstream analytics for monitoring and release of candidate therapies to provide delivery of only the most effective and reliable products."

Labcorp scientists have used AVATAR incubation technology to study the effectiveness and performance of cell therapy candidates grown in standard culture conditions versus those grown in customized levels of oxygen and pressure. The use of these custom conditions during cell expansion metabolically rewires therapeutic cells to improve their survival and persistent tumor killing activity in the harsh TME. Labcorp scientists have been going beyond in vitro studies with ongoing in vivo experiments to further evaluate this approach.

"Labcorp has long been one of Xcellbio’s closest collaborators, and we are pleased to include them as early members of our beta access program for the AVATAR Foundry," said Brian Feth, co-founder and CEO at Xcellbio. "We look forward to seeing the innovative ways they will use this platform to enhance their already impressive cell and gene therapy work."

On September 10, 2024 Incendia Therapeutics, a precision oncology company discovering and developing a novel class of therapies that reprogram the tumor microenvironment (TME), reported that the first patient has been enrolled in the Phase 1c study of PRTH-101 for the treatment of patients with advanced or metastatic solid tumors (Press release, Incendia Therapeutics, SEP 10, 2024, View Source [SID1234646496]).

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"In our Phase 1a and 1b studies, we were able to select an optimal dose and identify potential biomarkers to carry forward" said Irena Webster, SVP Development and Operations of Incendia. "The data from this study will inform the design of the Phase 2/3 program, for which planning and global feasibility are currently underway."

Dr. Joseph Paul Eder, CMO, added "Incendia, with its outstanding team of investigators and investigative sites, has determined a recommended Phase 2 dose of PRTH 101. PRTH 101 has shown consistent safety as a single agent and in combination with pembrolizumab. We will now focus on tumor types that have demonstrated signs of clinical activity and patient benefits."

The Phase 1c study will evaluate the safety, tolerability and anti-tumor activity of PRTH-101 as both a monotherapy and in combination with KEYTRUDA (pembrolizumab).

About PRTH-101
PRTH-101 is a therapeutic antibody that targets DDR1, a collagen binding protein and kinase present on epithelial cells. Some tumor cells are believed to co-opt DDR1 – collagen binding to build an impenetrable barrier around the tumor. By allosterically disabling DDR1, PRTH-101 disrupts tumor associated collagen alignment to permit immune cell access into the tumor core. In preclinical experiments, PRTH-101 mediated DDR1 blockade has demonstrated both single agent anti-tumor activity as well as marked augmentation of checkpoint inhibitor function. Tumor types that express high levels of DDR1-associated collagen barriers include thymic, colorectal, pancreatic, ovarian, glioma, and non-small cell lung cancer. Currently, there are no approved drugs that target DDR1.

About the Phase 1 Trial
The Phase 1 trial (NCT05753722) is a multi-center, open-label, dose escalation and dose expansion study that is expected to enroll up to 270 patients in the US with advanced or metastatic solid tumors. The goals of the study are to assess safety and tolerability of PRTH-101, evaluate anti-tumor activity in select indications alone and in combination with anti-PD-1 inhibitors, and determine dosing regimens for the Phase 2 clinical program. In addition to examining the clinical profile of PRTH-101, the trial will evaluate DDR1 and pathway-related proteins as predictive biomarkers for patients whose tumors respond to treatment.

On September 10, 2024 CEL-SCI Corporation (NYSE American: CVM) reported that it will report new data from its Phase 3 study of Multikine (Leukocyte Interleukin, Injection)* at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress which takes place from September 13 – 17, 2024 in Barcelona, Spain (Press release, Cel-Sci, SEP 10, 2024, View Source [SID1234646495]). A poster titled "Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy" will be presented by the study’s co-author József Tímár MD, PhD, DSc, a prominent and highly respected pathologist.

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"Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy"

Post this
CEL-SCI has received the go-ahead from the U.S. Food and Drug Administration (FDA) to commence a confirmatory Registration Study of Multikine in the treatment of head and neck cancer based on strong safety and efficacy data from its IT-MATTERS completed Phase 3 study.

Dr. Timar is Professor Department of Pathology, Forensic and Insurance Medicine at Semmelweis University in Budapest, Hungary, and served as the Director of the Central Pathology Laboratory for the IT-MATTERS study. With 174 peer reviewed studies published, Dr. Timar is a founding editor, editor in chief, or a member of the editorial board of four oncology journals. He is the recipient of a dozen honors and awards for excellence in cancer research and teaching.