Regenxbio Misleading Selloff
A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.
Why?
Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true.
The partial hold was put only on the product administered intrathecally in a clinical trial for the treatment of spinal muscular atrophy Type 2 (SMA-2). The agency has not put a hold on the same gene therapy Zolgensma that is already approved and administered intravenously for the treatment of spinal muscular atrophy type 1 (SMA-1).
The Misleading Selloff
Zolgensma, which was called AVXS-101, is an adeno-associated virus vector-based gene therapy belonging to Novartis (NVS) with the vector sublicensed from Regenxbio. The stock selloff has given the impression that Regenxbio’s AAV vector has failed and that its royalty on the sales of Zolgensma could be eroded or erased; this is far from being true.
It is obvious that the safety and efficacy of Zolgensma are intact and that the FDA’s partial hold does not include the gene therapy drug that is administered intravenously for the treatment of SMA-1. What is also obvious is that Regenxbio’s royalty payments will not be eroded nor erased, but are expected to increase as Novartis’ sales of Zolgensma are growing.
What the FDA and AveXis were aiming at by calling for the partial hold was to make sure that AVXS-101 will be safe to administer intrathecally for SMA-2. The partial hold impacts only the enrollment in the high dose cohort of the ongoing STRONG trial designed to evaluate the efficacy, safety and tolerability of the one-time intrathecal administration of AVXS-101. The low and mid-dose cohort enrollment has previously been completed and interim results have been presented.
Regarding the DRG inflammation observed, in this pre-clinical animal study which led to the partial hold, the firm stated that the clinical significance of this inflammation is not known and was not seen in prior animal studies with AVXS-101. DRG inflammation can be associated with sensory effects. The firm completed a review of human safety data from all available sources to date and no adverse effects related to sensory changes have been seen in the intrathecal and the intravenous administration of Zolgensma.
Regenxbio’s Pipeline Comprises:
- RGX-214 for Wet AMD and diabetic retinopathy, and anti-VEGF treated conditions
- RGX-121 for mucopolysaccharidosis II (MPS II)
- RGX-111 for mucopolysaccharidosis I (MPS I)
- RGX-181 for CLN2 Disease, which affects the nervous system
- RGX-501 for homozygous familial hypercholesterolemia (HoFH).
And more
Prohost Observations
Selling RGNX offers an opportunity for accumulating the stock at a bargain price. Regenxbio has improved on the safety and efficacy of AAV vectors from its proprietary gene delivery platform which the firm refers to as its NAV Technology Platform. The firm is selectively licensing the vectors to other biotech and biopharmaceutical firms.
Regenxbio is a firm with solid science. This firm is one of a few contributors to the success of gene therapy that was once just a dream but has now become a great reality.
To read more about these firms please run a search from our website by clicking here.
News & Comments
October 31, 2019
When Negative Reactions to Biotechnology News Mislead. The Case of Regenxbio.
Regenxbio Misleading Selloff
A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.
Why?
Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true.
The partial hold was put only on the product administered intrathecally in a clinical trial for the treatment of spinal muscular atrophy Type 2 (SMA-2). The agency has not put a hold on the same gene therapy Zolgensma that is already approved and administered intravenously for the treatment of spinal muscular atrophy type 1 (SMA-1).
The Misleading Selloff
Zolgensma, which was called AVXS-101, is an adeno-associated virus vector-based gene therapy belonging to Novartis (NVS) with the vector sublicensed from Regenxbio. The stock selloff has given the impression that Regenxbio’s AAV vector has failed and that its royalty on the sales of Zolgensma could be eroded or erased; this is far from being true.
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