ABBV - Regenxbio: DMD Program Lends Additional Credibility To NAV Technology Platform

2024-03-11 11:53:40 ET

Summary

• Positive interim results released from phase 1/2 AFFINITY study, using RGX-202 for the treatment of patients with Duchenne Muscular Dystrophy.
• Functional and strength data from the phase 1/2 AFFINITY study, using RGX-202 for the treatment of patients with Duchenne Muscular Dystrophy, expected in the 2nd half of 2024.
• The Duchenne Muscular Dystrophy Treatment Market size is estimated to reach $4.32 billion by 2029.
• Primary endpoint met for the CAMPSIITE phase 2/3 study, using RGX-121 for the treatment of patients with MPS II; BLA filing to the FDA of RGX-121 for this patient population expected in 2024.

REGENXBIO Inc. ( RGNX ) has made remarkable progress in being able to advance its pipeline with various types of gene therapies. The last time I wrote about this article it was in a Seeking Alpha article entitled " Regenxbio: Biotech To Watch With 2 Possible Accelerated Approval Pathways ". Since then, I'm happy to report that it was able to achieve two important milestones. The first of which is that it reported 3-month data from the phase 1/2 study using RGX-202 for the treatment of patients with Duchenne Muscular Dystrophy [DMD]. That is, the higher dose level 2 of this treatment was able to help patients achieve robust microdystrophin expression. In addition, there was the release of the primary endpoint being met in the ongoing phase 1/2/3 CAMPSITE study, using RGX-121 to treat patients with MPS II. It was noted that the primary endpoint in reduction of CSF biomarker of MPS II disease was met with statistical significance. Such statistical significance was achieved with a p-value of 0.00016. Both of these reasons have led me to increase the rating from buy to strong buy. That is because the odds of reaching the finish line, with respect to regulatory approvals, have increased significantly. The whole basis for the company being able to achieve such results is because of its NAV Technology Platform, which is building upon next-generation AAV vectors to enhance efficacy, compared to prior generation types. The thing is that dose escalation presses on and leads to one minor catalyst, which is a pivotal dose determination assessment in mid-2024....

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