US biotech Regenxbio (Nasdaq: RGNX) has successfully completed the BLA submission for its mucopolysaccharidosis II (MPS II) RGX-111 and is on track for potential US Food and Drug Administration (FDA) approval via accelerated approval in second-half 2025.
The firm’s shares edged up 2.6% to $6.72 on the news, which came alongside its 2024 financial results. It reported a net loss of $51.2 million, or $1.01 basic and diluted net loss per share, for the three months ended December 31, 2024, compared to a net loss of $62.9 million, or $1.43 basic and diluted net loss per share a year earlier.
The company says RGX-121 remains on track to be the potential first gene therapy and one-time treatment approved for MPS II, also known as Hurler syndrome, adding that Regenxbio also continues to advance RGX-111 for MPS I through the recent strategic partnership with Japan’s Nippon Shinyaku (TYO: 4516).
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2024 | Headless Content Management with Blaze