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In Brief This Week: Lexeo, AnHeart, Innovent Biologics, Krystal Biotech, NeuExcell, CareDx

NEW YORK – Lexeo Therapeutics this week said that the US Food and Drug Administration has granted fast-track status and orphan drug designation to LX2020, its gene therapy candidate for PKP2 gene-mutated arrhythmogenic cardiomyopathy, a severe cardiac disorder that causes lethal arrhythmias and sudden cardiac death. Three-fourths of patients have PKP2-mutated arrhythmogenic cardiomyopathy, and LX2020 uses an adeno-associated virus rh10 vector to intravenously deliver a functional PKP2 gene into patients' cardiac muscle. In the Phase I/II HEROIC-PKP2 trial, New York-based Lexeo is tracking the treatment's preliminary efficacy, safety, and tolerability, as well as its long-term activity over four years. The FDA grants orphan designation to drugs for rare diseases, providing sponsors with tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity after regulatory approval. For fast-track-designated agents, a sponsor can meet with the FDA more frequently to get drug development advice, submit data on a rolling basis, and apply for accelerated approval or priority review. 

AnHeart Therapeutics and Innovent Biologics said this week that China's National Medical Products Administration (NMPA) granted priority review designation for their ROS1 inhibitor taletrectinib for the treatment of locally advanced or metastatic ROS1-positive non-small cell lung cancer patients who have been previously treated with ROS1 inhibitors. The designation was based on data from the Phase II TRUST-I trial, which showed taletrectinib had activity in both ROS1 inhibitor-naïve patients and those who had prior treatment with Pfizer's ROS1 inhibitor Xalkori (crizotinib). In China, priority review designation allows the NMPA's Center for Drug Evaluation (CDE) to prioritize the review process and evaluation resources for new drug applications. 

Krystal Biotech this week said that the Japan Ministry of Health, Labor, and Welfare has granted orphan drug designation to Vyjuvek (beremagene geperpavec) as a treatment for dystrophic epidermolysis bullosa. Vyjuvek uses a genetically modified herpes-simplex virus type 1 vector to deliver a normal version of the COL7A1 gene to patients. The US Food and Drug Administration approved Vyjuvek earlier this year for treating wounds in pediatric and adult patients with dystrophic epidermolysis bullosa due to mutations in the collagen type VII alpha 1 chain (COL7A1) gene. 

Regulators in Japan grant orphan drug status to treatments for diseases that affect fewer than 50,000 people in Japan and need better treatments. Sponsors are eligible for subsidies for R&D expenses, reduced consultation and application fees, and tax incentives, and they can consult regulators for advice on clinical development, seek priority review of applications, and have an extended registration validity period. 

The US Food and Drug Administration this week granted orphan drug designation to NeuExcell Therapeutics’ AAV gene therapy, NXL-004, for malignant glioma patients. The therapy, which involves regenerating neuronal tissues and restoring neurological function, is expected to enter clinical trials in early 2024. The FDA grants orphan designation to drugs for rare diseases, providing the drugs' sponsors with tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval. 

CareDx said this week that it is offering its Transplant Pharmacy services to patient members of the National Foundation for Transplants in an effort to help them better manage post-transplant medication adherence. The company's Transplant Pharmacy team helps patients coordinate benefits to reduce out-of-pocket costs and helps ensure that patients receive regular personalized care.  

Proteomics International Laboratories (PIL) this week said it has signed a five-year licensing agreement with longtime partner Omics Global Solutions to make PIL's PromarkerD predictive test for diabetic kidney disease available in Chile. The agreement also includes royalties based on sales of the test, which Omics manufactures under license. Omics will market the test initially to Chile's private payor market and eventually expand into other markets in Central and South America. PromarkerD measures three serum protein biomarkers, combined with routinely available conventional clinical variables (age, HDL cholesterol, and estimated glomerular filtration rate) to predict future kidney function decline in patients with type 2 diabetes and no existing diabetic kidney disease. 

Pangea Laboratory said this week that the US Food and Drug Administration has granted breakthrough device designation for the firm's molecular assay for detection of bladder cancer and upper tract urothelial carcinoma. The Tustin, California-based company's Bladder Care Assay is a urine-based qPCR test that is used in patients with hematuria for quantitative detection of DNA methylation biomarkers. The firm said its study results indicate the assay detects bladder cancer with 94 percent sensitivity and 93 percent specificity, upper tract urothelial carcinoma with 96 percent sensitivity and 88 percent specificity, and carcinoma in situ with 89 percent sensitivity. 

Thermo Fisher Scientific said this week that the Biomedical Advanced Research and Development Authority (BARDA) has selected the company's PPD clinical research business to implement the first BARDA-supported Phase II platform clinical trial to study therapeutic options for treating acute respiratory distress syndrome (ARDS). The goal of the study is to gather clinical and biomarker data on ARDS patients to inform future clinical studies and the development of targeted therapies. The three-year, randomized, double-blind, placebo-controlled, multicenter trial will evaluate the safety and efficacy of three therapeutics at up to 60 sites in the US, enrolling 600 hospitalized adult patients with ARDS. The drug candidates to be tested will be announced in early 2024. 

The US Food and Drug Administration this week granted breakthrough therapy designation to BioNTech and DualityBio's antibody-drug conjugate BNT323/DB-1303 as treatment for HER2-expressing endometrial cancer patients. The designation  which is meant to expedite development and review for particularly promising drugs in difficult-to-treat diseases  is based on encouraging early data from a Phase I/II basket trial evaluating BNT323/DB-1303 in HER2-expressing solid tumors.  

Gilead Sciences said this week that the US Food and Drug Administration has updated the label for Yescarta (axicabtagene ciloleucel) to include overall survival data from the primary analysis of the Phase III ZUMA-7 trial, in which second-line treatment with the autologous CAR T-cell therapy benefitted patients with diffuse large B-cell lymphoma versus standard of care treatment. In ZUMA-7, the estimated 39-month overall survival rate was 55.9 percent with Yescarta versus 46 percent in the standard of care arm.  

In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.