FDA grants Fast Track Designation to X4’s Mavorixafor in WHIM Syndrome

Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4, currently being investigated in a global pivotal Phase 3 clinical trial, 4WHIM, for the treatment of WHIM syndrome.

In addition to Fast Track Designation, mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome.

WHIM syndrome is a rare, inherited, primary immunodeficiency disease caused by gain-of-function mutations in the chemokine receptor CXCR4, resulting in a reduced mobilization and trafficking of white blood cells from the bone marrow. The company estimates there to be more than 3,500 diagnosed and undiagnosed WHIM patients in the U.S. As a first-in-class, small-molecule antagonist of chemokine receptor CXCR4, mavorixafor is designed to address the underlying cause of WHIM directly. The candidate is currently being developed as a once-daily oral therapy in the Phase 3 4WHIM trial, a 52-week, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in genetically confirmed WHIM patients. The trial is anticipated to enroll up to 28 subjects in approximately 20 countries, followed by an open-label extension trial. Phase 3 results are expected in 2022.

FDA accepts Eton NDA for dehydrated alcohol injection

Eton’s application previously received orphan drug designation for the treatment of methanol poisoning and is expected to receive seven years of market exclusivity upon its approval.

The company currently owns or receives royalties from three FDA-approved approved products, including ALKINDI SPRINKLE, Biorphen, and Alaway Preservative Free, and has six additional products in its late-stage pipeline, including five that have been submitted to the FDA.

The U.S. market for dehydrated alcohol injection is estimated to be greater than $100 million annually and the drug is currently only supplied by a single manufacturer.

Moderna regains all rights to RSV vaccine from Merck

Separately, Moderna also announced the initiation of dosing in the Phase 1 study of its solely owned RSV vaccine candidate (mRNA-1345). This Phase 1 study includes initial dosing in adults, followed by age de-escalation into children. The company previously announced its intent to advance mRNA-1345 in children in combination with mRNA-1653, a vaccine against two other pediatric respiratory viruses (hMPV, PIV3) which is currently in its own age de-escalation study. With today’s announcement, Moderna will have the right to also advance RSV vaccines in adults, either alone or in combination with other respiratory virus vaccines.

Moderna and Merck will continue their ongoing collaboration in cancer vaccines. In 2016, Moderna and Merck entered into a collaboration for mRNA-4157, a personalized cancer vaccine candidate, which is currently being evaluated in a Phase 2 study. In 2018, the companies expanded the collaboration to include the development and commercialization of mRNA-5671 a mutant KRAS vaccine candidate currently in a Phase 1 study.

Moderna’s mRNA-1345 vaccine uses the Company’s proprietary lipid nanoparticle delivery technology also used in the Company’s *redacted* vaccine (mRNA-1273) and CMV vaccine (mRNA-1647).