Regenxbio seeks to improve lives through the curative potential of gene therapy.
The Phase I/II Trial of RGX-111:
A multi-center, open-label, dose-escalation trial that will evaluate the safety, tolerability and pharmacodynamics of RGX-111 delivered to patients with MPS I via injection directly into the cerebrospinal fluid (CSF). Up to five patients will be enrolled at two dose levels: 1.0×1010 GC/g of brain mass and 5.0×1010 GC/g of brain mass.
The Primary Endpoint of the Study:
Safety and tolerability of RGX-111. Other endpoints include the effect of RGX-111 on biomarkers of IDUA enzyme activity in the CSF, serum and urine, neurocognitive development and other outcome measures.
Mucopolysaccharidosis Type I (MPS I)
MPS I is an autosomal recessive genetic disease caused by deficiency of IDUA enzyme required for the breakdown of the polysaccharides in lysosomes. These polysaccharides, called glycosaminoglycans (GAGs), accumulate in the tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the CNS, causing excessive accumulation of fluid in the brain, spinal cord compression and cognitive impairment. MPS I is estimated to occur in 1 in 100,000 births. Current disease-modifying therapies for MPS I include hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously. However, intravenous enzyme therapy does not treat the CNS manifestations of MPS I, and HSCT can be associated with clinically significant morbidity and mortality.
Steve Pakola, M.D., Chief Medical Officer of Regenxbio, said, “We are leaders in the development of one-time treatments using our NAV Technology Platform and proprietary delivery procedure which is designed to administer our gene therapy candidates directly to the central nervous system (CNS). RGX-111 is our second product candidate for the treatment of a rare, neurodegenerative disease to be dosed in patients, following RGX-121, which is in clinical development for MPS II. We believe one-time treatment with RGX-111 can provide sustainable IDUA enzyme for patients, potentially preventing the progression of the disease. The dosing of the first patient in the clinical trial for RGX-111 marks an important milestone in our neurodegenerative disease program and our commitment to the MPS community.”
Regenxbio’s NAV Technology Platform
A proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. Regenxbio and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
The dosing of the first patient in the Phase I/II trial of RGX-111 for the treatment of MPS I is useful news that fulfills one of the firm’s important promises. We also became aware that the firm’s subretinal pivotal wet AMD trial of the firm’s product RGX-314 is expected to be initiated in the first quarter of 2021, and that initial safety data for RGX-314 suprachoroidal Phase II AAVIATE study are also expected in early 2021. We know that additional patients were enrolled in the expanded Phase I/II study of the firm’s product RGX-121 for MPS II.
We remind that Regenxbio has received an $80 million milestone payment from Novartis in October 2020 as a royalty payment on the sales of the gene therapy Zolgensma for the treatment of spinal muscular atrophy.
Regenxbio has sufficient cash and we believe its stock is undervalued.
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Regenxbio Announced the First Patient has been Dosed in the Phase I/II trial of RGX-111 for MPS I
Regenxbio Dosed First Patient with RGX-111 for MPS I
Regenxbio (RGNX) has dosed the first patient with RGX-111 in the Phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I).
About Regenxbio Product RGX-111
RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector.
RGX-111 is a product candidate for the treatment of MPS I also known as Hurler syndrome. RGX-111 uses the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene. Delivery of the enzyme that is deficient within cells in the CNS is expected to provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients. RGX-111 has received orphan drug products, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration (FDA).