NEW YORK – Sernova this week said the US Food and Drug Administration has granted orphan drug designation and rare pediatric disease designation to its hemophilia A program. The company, headquartered in London, Ontario, is hoping to treat the hereditary hemorrhagic disorder by correcting a patient's own blood outgrowth endothelial cells and returning the edited cells back into the patient through its Cell Pouch system. The modified cells are designed to release factor VIII, the blood-clotting protein lacking in hemophilia A patients, into their bloodstream and, hopefully, restore their blood-clotting ability during bleeding episodes. Developers of orphan drugs for diseases affecting fewer than 200,000 people in the US are eligible for tax credits, waivers for FDA application fees, and if approved by the agency, seven years of market exclusivity. Furthermore, products for rare pediatric diseases, once approved, also receive a priority review voucher from the agency.
Akeso said this week it began construction of a global R&D center in Shanghai's Zhangjiang headquarters park. The center will carry out R&D activities in areas such as gene therapy, cell therapy, neurological diseases, and anti-aging with platforms dedicated to tumor microenvironment data analysis and translational research. Akeso is conducting Phase III studies of its anti-PD-1/VEGF antibody ivonescimab with Merck's Keytruda (pembrolizumab) in patients with PD-L1-positive non-small cell lung cancer and as a monotherapy in patients with EGFR-mutated advanced non-squamous NSCLC that have failed prior EGFR-tyrosine kinase inhibitor therapy.
Edinburgh, Scotland-based RoslinCT, a cell and gene therapy contract development and manufacturing organization, this week said it will manufacture Vertex Pharmaceuticals' sickle cell disease and transfusion-dependent beta thalassemia therapy Casgevy (exagamglogene autotemcel), which became the first approved CRISPR-based treatment in the world after receiving conditional marketing authorization from the UK Medicines and Healthcare Products Regulatory Agency earlier this month. Under the terms of the deal, RoslinCT will be tasked with manufacturing Casgevy worldwide.
GeNeuro this week said it has completed recruitment for its Phase II trial evaluating temelimab, an investigational disease-modifying therapy and monoclonal antibody it's testing in a biomarker-defined subset of patients with long COVID. More than 200 patients in Switzerland, Spain, and Italy have been enrolled in the randomized, placebo-controlled study, in which investigators will assess the efficacy and safety of temelimab for long COVID patients with neuropsychiatric symptoms and the presence of the pathogenic human endogenous retrovirus-W envelope (W-ENV) protein in their blood. The Geneva, Switzerland-based firm expects to publish top-line results from the trial in June 2024.
Effector Therapeutics this week said the US Food and Drug Administration granted fast-track designation for its eIF4A inhibitor zotatifin in combination with fulvestrant and Eli Lilly's Verzenio (abemaciclib) as second- or third-line therapy treatment for estrogen receptor (ER)-positive, HER2-negative advanced or metastatic breast cancer patients who had disease progression after treatment with endocrine therapy and a CDK4/6 inhibitor. Effector is studying the zotatifin-fulvestrant-Verzenio combination in a Phase I/II trial in this patient population. With this designation, a sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
ARUP Laboratories issued a public comment this week on the US Food and Drug Administration’s proposed rule for regulation of laboratory-developed tests, urging the agency to withdraw the rule. In its comment, the lab said it believes the rule uses flawed estimates regarding the number of LDTs ordered and their performance and also challenged the agency’s authority to regulate these tests. It said the rule would negatively impact patient care and reduce access to testing.
Orchard Therapeutics this week said it received fast-track designation from the US Food and Drug Administration for OTL-203, its investigational hematopoietic stem cell gene therapy being developed as a treatment for Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The rare, inherited neurometabolic disease is caused by a lack of IDUA lysosomal enzyme, which the firm seeks to address by administering a functional copy of the IDUA gene into patients' cells. The gene therapy previously received rare pediatric disease designation from the FDA and priority medicines designation from the European Medicines Agency.
Merck said this week that its board of directors has declared a quarterly dividend of $.77 per share payable on Jan. 8 to Merck shareholders of record as of the close of business on Dec. 15.
Oxford BioTherapeutics this week said the US Food and Drug Administration granted fast-track designation to its bispecific T-cell engager BI764532 as a second-line therapy for DLL3-expressing advanced or metastatic large-cell neuroendocrine carcinoma of the lung.
Twinsburg, Ohio-based pharmacogenomic testing firm Xact Laboratories this week said it is partnering with an unnamed national prescription claims clearinghouse to offer a new PGx service to health insurers. With the new offering, PGx test results are automatically integrated into the prescription fulfillment workflow, so that clinicians and pharmacists treating members of participating health plans can be notified if a patient has actionable PGx results that might impact their prescription.
Health Canada has authorized a protocol amendment to Taysha Gene Therapies' ongoing open-label, randomized, dose-escalation Phase I/II trial evaluating TSHA-102 as an investigational treatment for Rett syndrome, the Dallas-based biotech firm said this week. Previously, only adults with stage 4 Rett syndrome due to MECP2 loss-of-function mutations were eligible for the REVEAL trial, but now the company will enroll adolescent patients ages 12 years and older. Taysha said it expects to complete the first low-dose cohort of the trial by Q1 2024.
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.