NEW YORK – Fujita Health University of Japan and BostonGene said this week that the Fujita Health University Haneda Clinic will start offering genomic profiling tests for cancer patients using the BostonGene Tumor Portrait test. The test integrates DNA and RNA sequencing and detects actionable mutations, properties of the tumor microenvironment, and treatment response biomarkers.
Pillar Biosciences this week said that the American Medical Association has granted proprietary laboratory analyses code 0523U to the firm's OncoReveal CDx pan-cancer in vitro diagnostic NGS kit. The kit, which was approved by the US Food and Drug Administration in April, leverages Pillar's Stem-Loop Inhibition-Mediated amplification (SLIMamp) technology to detect single nucleotide variants, insertions, and deletions in 22 genes using DNA isolated from formalin-fixed, paraffin-embedded tumor tissue specimens. OncoReveal CDx was cleared for use on the Illumina MiSeq Dx instrument and approved as a companion diagnostic test to identify patients who may benefit from EGFR TKI therapy in NSCLC, and from Eli Lilly's Erbitux (cetuximab) or Amgen's Vectibix (panitumumab) for KRAS in colorectal cancer, Pillar noted.
The Muscular Dystrophy Association this week said that a gene therapy it is developing for congenital myasthenic syndrome caused by CHAT (choline acetyltransferase) gene mutations has received the rare pediatric disease and orphan drug designations from the US Food and Drug Administration. The MDA began the Kickstart for Ultra-Rare Neuromuscular Disease program in 2023 and announced in September that the first project would focus on developing a gene therapy for CHAT-mutated congenital myasthenic syndrome and work with Forge Biologics as a manufacturing partner. Sponsors that get orphan disease status for certain drugs receive tax credits for qualifying clinical trials, exemption from user fees, and seven years of market exclusivity if their orphan drug gains regulatory approval. With rare pediatric disease designation comes the chance to receive a priority review voucher, an important incentive for developing rare disease drugs.
The US Food and Drug Administration has granted rare pediatric disease designation to San Diego-based Papillon Therapeutics' PPL-002, an investigational treatment that the company is developing for Danon disease, the biotech said this week. PPL-002 is a gene-modified CD34-positive hematopoietic stem and progenitor cell therapy that's designed to express functional Lamp-2, the protein encoded by the LAMP2 gene and that's deficient in patients with Danon disease. The FDA previously granted orphan drug designation to PPL-002, as well.
Artera, a developer of artificial intelligence-based cancer tests in San Francisco, this week said it has received a lab license from the California Department of Public Health, through which it has begun offering the ArteraAI Prostate Test to clinicians and patients in the state. ArteraAI Prostate analyzes digital images from patients' biopsies and their clinical data using an algorithm. According to Artera, the test has been validated using data from multiple Phase III randomized trials and has shown it can prognosticate long-term outcomes for patients with localized prostate cancer and predict whether they'll benefit from therapy. Artera's lab also has accreditation from the College of American Pathologists.
Erasca this week said its lead candidate, pan-RAF inhibitor naporafenib, demonstrated favorable outcomes in combination with Novartis' MEK inhibitor Mekinist (trametinib) in pooled data from a Phase Ib/II trial in NRAS-mutant melanoma. The pooled data further supports Erasca's ongoing Phase III trial of the combination in NRAS-mutant melanoma and confirms the company's regulatory pathway in melanoma. Erasca also said it remains on track to file investigational new drug (IND) applications in the US for its two in-licensed RAS-targeting programs, ERAS-0015 and ERAS-4001, after completing certain preclinical studies. The firm plans to file an IND for ERAS-4001 in Q1 2025 and for ERAS-0015 in the first half of 2025.
Malvern, Pennsylvania-based Ocugen this week said that a data and safety monitoring board has approved it to begin enrolling patients in the second phase of the Phase I/II GARDian trial, in which investigators are assessing OCU410ST as an investigational gene therapy treatment for Stargardt disease. In the Phase I portion of the study, patients received one of three doses of OCU410ST. Ocugen will proceed into the Phase II portion of the clinical trial and test the high and medium doses of OCU410ST that it evaluated in the initial phase.
The Qatar Precision Health Institute (QPHI) of the Qatar Foundation said this week that it has released data for 25,000 human genomes to the research community, half of which have electronic medical data associated with them. The dataset is aimed at helping with research into cancer genetics, cardiovascular diseases, reproductive health, and mental health. Members of the second round of the Qatar Precision Health Research Consortium (QPHRC) plan to focus on these and other areas.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.