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REGENXBIO (RGNX) Soars 15% on Interim Data From DMD Study
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Shares of REGENXBIO (RGNX - Free Report) rose 15.2% on Mar 5 after management reported positive interim data from the phase I/II AFFINITY DUCHENNE study evaluating its investigational one-shot gene therapy RGX-202 for Duchenne muscular dystrophy (DMD).
Data from the AFFINITY DUCHENNE study showed that patients who received RGX-202 demonstrated improved strength and motor function. As of Feb 28, the therapy was well tolerated with no serious adverse events in DMD patients aged between four and 12 years across both dose levels — dose level 1 and dose level 2.
All four patients who received RGX-202 showed encouraging increases in microdystrophin expression level and reduction from baseline in serum CK levels, following three months of gene therapy infusion.
Management also revealed data from the first patient, aged 12.1 years, who received RGX-202 at dose level 2. Caregivers noted that this patient demonstrated an increased RGX-202 microdystrophin expression level of 75.7% at three months compared with control. A reduction from baseline in serum creatinine kinase (CK) levels of 77% was also observed post 10 weeks of RGX-202 infusion.
REGENXBIO intends to share additional strength and functional assessment data for both dose levels being evaluated and initiate a pivotal study before year-end. Prior to initiating this study, it intends to determine a pivotal dose for the therapy by mid-2024.
Management also intends to use the RGX-202 microdystrophin expression as a surrogate endpoint to support a regulatory filing with the FDA seeking approval under the accelerated pathway.
Shares of REGENXBIO have risen 37.1% in the year-to-date period compared with the industry’s 2.8% rise.
Image Source: Zacks Investment Research
Currently, the only approved DMD gene therapy is marketed by Sarepta Therapeutics (SRPT - Free Report) . This therapy, marketed under the trade name Elevidys (SRP-9001), was granted accelerated approval by the FDA last June to treat ambulatory pediatric patients aged between four and five years with DMD.
Last month, Sarepta announced that the FDA accepted its regulatory filing, seeking to expand the treatment label for Elevidys. The purpose of this filing is two-fold. It aims to expand the labeled indication for Elevidys to treat all DMD patients, irrespective of age and ambulation status. Subject to approval, it will also convert the Elevidys accelerated approval to a traditional approval. The FDA has assigned the Priority Review status to this filing and a final decision is expected by Jun 21, 2024.
Another company that is also working on a gene therapy candidate for DMD is Solid Biosciences (SLDB - Free Report) . Last year in November, the FDA cleared Solid Biosciences’ investigational new drug (IND) application to begin clinical studies on its novel gene therapy candidate, SGT-003, to treat DMD.
The IND clearance was based on encouraging data from the Solid Biosciences’ pre-clinical study wherein treatment with SGT-003 demonstrated increased biodistribution to cardiac and skeletal muscle, including the diaphragm, compared with AAV9 in nonhuman primates. Earlier in January, the FDA granted orphan drug designation to SGT-003 in DMD indication.
SLDB is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical study sites to begin the planned phase I/II study of SGT-003 for DMD. Patient screening for the early to mid-stage DMD will commence shortly after the IRB approvals are secured.
In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share (EPS) have risen from 18 cents to 30 cents. Meanwhile, during the same period, EPS estimates for 2025 have improved from 32 cents to 50 cents. Year to date, shares of ADMA have risen 28.3%.
Earnings of ADMA Biologicsbeat estimates in three of the last four quarters while meeting the same on one occasion. ADMA delivered a four-quarter average earnings surprise of 85.00%.
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REGENXBIO (RGNX) Soars 15% on Interim Data From DMD Study
Shares of REGENXBIO (RGNX - Free Report) rose 15.2% on Mar 5 after management reported positive interim data from the phase I/II AFFINITY DUCHENNE study evaluating its investigational one-shot gene therapy RGX-202 for Duchenne muscular dystrophy (DMD).
Data from the AFFINITY DUCHENNE study showed that patients who received RGX-202 demonstrated improved strength and motor function. As of Feb 28, the therapy was well tolerated with no serious adverse events in DMD patients aged between four and 12 years across both dose levels — dose level 1 and dose level 2.
All four patients who received RGX-202 showed encouraging increases in microdystrophin expression level and reduction from baseline in serum CK levels, following three months of gene therapy infusion.
Management also revealed data from the first patient, aged 12.1 years, who received RGX-202 at dose level 2. Caregivers noted that this patient demonstrated an increased RGX-202 microdystrophin expression level of 75.7% at three months compared with control. A reduction from baseline in serum creatinine kinase (CK) levels of 77% was also observed post 10 weeks of RGX-202 infusion.
REGENXBIO intends to share additional strength and functional assessment data for both dose levels being evaluated and initiate a pivotal study before year-end. Prior to initiating this study, it intends to determine a pivotal dose for the therapy by mid-2024.
Management also intends to use the RGX-202 microdystrophin expression as a surrogate endpoint to support a regulatory filing with the FDA seeking approval under the accelerated pathway.
Shares of REGENXBIO have risen 37.1% in the year-to-date period compared with the industry’s 2.8% rise.
Image Source: Zacks Investment Research
Currently, the only approved DMD gene therapy is marketed by Sarepta Therapeutics (SRPT - Free Report) . This therapy, marketed under the trade name Elevidys (SRP-9001), was granted accelerated approval by the FDA last June to treat ambulatory pediatric patients aged between four and five years with DMD.
Last month, Sarepta announced that the FDA accepted its regulatory filing, seeking to expand the treatment label for Elevidys. The purpose of this filing is two-fold. It aims to expand the labeled indication for Elevidys to treat all DMD patients, irrespective of age and ambulation status. Subject to approval, it will also convert the Elevidys accelerated approval to a traditional approval. The FDA has assigned the Priority Review status to this filing and a final decision is expected by Jun 21, 2024.
Another company that is also working on a gene therapy candidate for DMD is Solid Biosciences (SLDB - Free Report) . Last year in November, the FDA cleared Solid Biosciences’ investigational new drug (IND) application to begin clinical studies on its novel gene therapy candidate, SGT-003, to treat DMD.
The IND clearance was based on encouraging data from the Solid Biosciences’ pre-clinical study wherein treatment with SGT-003 demonstrated increased biodistribution to cardiac and skeletal muscle, including the diaphragm, compared with AAV9 in nonhuman primates. Earlier in January, the FDA granted orphan drug designation to SGT-003 in DMD indication.
SLDB is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical study sites to begin the planned phase I/II study of SGT-003 for DMD. Patient screening for the early to mid-stage DMD will commence shortly after the IRB approvals are secured.
REGENXBIO Inc. Price
REGENXBIO Inc. price | REGENXBIO Inc. Quote
Zacks Rank & A Key Pick
REGENXBIO currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the overall healthcare sector is ADMA Biologics (ADMA - Free Report) , which sports a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ADMA Biologics’ 2024 earnings per share (EPS) have risen from 18 cents to 30 cents. Meanwhile, during the same period, EPS estimates for 2025 have improved from 32 cents to 50 cents. Year to date, shares of ADMA have risen 28.3%.
Earnings of ADMA Biologicsbeat estimates in three of the last four quarters while meeting the same on one occasion. ADMA delivered a four-quarter average earnings surprise of 85.00%.