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REGENXBIO's (RGNX) Hunter Syndrome Study Meets Goal, Stock Up
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REGENXBIO Inc. (RGNX - Free Report) announced positive top-line results from the phase I/II/III CAMPSIITE study of its investigational one-time gene therapy candidate, RGX-121, for the treatment of patients (up to five years old) with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome.
The pivotal phase of the study met its primary endpoint of patients achieving a reduction in cerebrospinal fluid (CSF) biomarker of MPS II disease with statistical significance.
MPS II is a rare, debilitating disease that leads to cell, tissue and organ dysfunction, including in the central nervous system (CNS). The disease is estimated to occur in one in 100,000 to 170,000 births. Currently, there are no treatments available to address fatal neuronopathic CNS disease in MPS II.
Per REGENXBIO, data from the pivotal study highlights RGX-121’s capability to change the course of the disease by restoring the gene missing in MPS II patients. The candidate also demonstrated its potential to significantly improve vital brain function for patients with this disease.
REGENXBIO has met with the FDA to discuss the positive results of the MPS II study. The FDA has confirmed that the top-line results support a biologics license application (BLA) submission for RGX-121 to treat MPS II in 2024, under the accelerated approval pathway, subject to review of the full data.
The company’s stock gained 11.7% on Feb 8, in response to the encouraging results from its CAMPSIITE study of RGX-121 for MPS II and continued to rise another 1.7% in the after-market hours. In the past year, shares of REGENXBIO plunged 36.7% compared with the industry’s 11.7% decline.
Image Source: Zacks Investment Research
Per the data readout from the study, it was observed that MPS II patients treated with RGX-121 achieved decreased CSF levels of D2S6, a key biomarker of brain disease activity, below maximum attenuated disease levels at 16 weeks. A median reduction in D2S6 of 86% was observed in patients receiving RGX-121 therapy, approaching normal levels.
REGENXBIO further stated that the pivotal results were consistent with those observed in the dose-finding phase of the CAMPSIITE study. Most MPS II patients treated with RGX-121 in the dose-finding phase exceeded expectations in neurodevelopmental function compared with natural history data up to four years.
The current standard-of-care treatment for MPS II is intravenous enzyme replacement therapy (ERT). However, new long-term follow-up data from the dose-finding phase of the study observed a high rate of patients for whom study investigators chose to discontinue ERT or were allowed to remain ERT-naïve, representing a meaningful breakthrough.
Furthermore, the gene therapy candidate demonstrated a favorable safety profile and continues to be overall well tolerated in MPS II patients.
REGENXBIO is currently gearing up to use CSF levels of D2S6 as a surrogate endpoint for accelerated approval of RGX-121 to treat the CNS indication. The company is currently completing the remaining activities and expects to file the BLA in the second half of 2024.
Subject to potential approval of the planned BLA under the FDA’s priority review status, REGENXBIO anticipates the reception of a Rare Pediatric Disease Priority Review Voucher in 2025.
In a separate press release, REGENXBIO announced that enrollment has been completed in cohort 2 (at dose level 2) of the phase I/II AFFINITY DUCHENNE study of another pipeline candidate, RGX-202, a potential one-time AAV therapeutic, which is being developed to treat Duchenne muscular dystrophy (DMD). RGX-202 therapy includes an optimized transgene for a novel microdystrophin.
The company also reported additional interim safety and efficacy data from the AFFINITY DUCHENNE study of RGX-202 in patients with DMD (aged four to 11 years old).
Per the updated safety data, RGX-202 has been well tolerated with no drug-related serious adverse events in five patients aged 4.4 to 12.1 at dose levels 1 and 2.
Furthermore, RGNX reported new three-month assessment data of the third patient receiving RGX-202 dose level 1 that observed the largest increase in microdystrophin expression (83.4%) compared with the control cohort. The patient also experienced a 93% reduction in serum creatinine kinase levels from baseline at week 10.
The company expects to select a pivotal dose of RGX-202 in mid-2024, followed by the initiation of a pivotal DMD study in the second half of 2024. REGENXBIO also plans to share initial strength and functional assessment data for both dose levels in the second half of 2024.
In the pivotal study, RGNX plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a BLA submission under the FDA’s accelerated approval pathway.
REGENXBIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the drug/biotech industry are Puma Biotechnology, Inc. (PBYI - Free Report) , Immunocore (IMCR - Free Report) and ImmunoGen . While both PBYI and IMGN currently sport a Zacks Rank #1 (Strong Buy), IMCR carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, the Zacks Consensus Estimate for Puma Biotech’s 2023 earnings per share (EPS) has remained constant at 73 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has remained constant at 69 cents. Over the past year, shares of PBYI have gained 10.3%.
PBYI beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.
In the past 30 days, the Zacks Consensus Estimate for Immunocore’s 2023 loss per share has remained constant at 95 cents. During the same period, the consensus estimate for Immunocore’s 2024 loss per share has remained constant at $1.42. Over the past year, shares of IMCR have jumped 22.3%.
IMCR beat estimates in two of the trailing four quarters and missed on the other two occasions. In the last reported quarter, IMCR reported an earnings surprise of 112.5%.
In the past 30 days, the Zacks Consensus Estimate for ImmunoGen’s 2023 EPS has remained constant at 7 cents. During the same period, the consensus estimate for ImmunoGen’s 2024 EPS has remained constant at 37 cents. Over the past year, shares of IMGN have skyrocketed 601.9%.
IMGN beat estimates in each of the trailing four quarters, delivering an average earnings surprise of 136.05%.
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REGENXBIO's (RGNX) Hunter Syndrome Study Meets Goal, Stock Up
REGENXBIO Inc. (RGNX - Free Report) announced positive top-line results from the phase I/II/III CAMPSIITE study of its investigational one-time gene therapy candidate, RGX-121, for the treatment of patients (up to five years old) with Mucopolysaccharidosis Type II (MPS II), which is also known as Hunter syndrome.
The pivotal phase of the study met its primary endpoint of patients achieving a reduction in cerebrospinal fluid (CSF) biomarker of MPS II disease with statistical significance.
MPS II is a rare, debilitating disease that leads to cell, tissue and organ dysfunction, including in the central nervous system (CNS). The disease is estimated to occur in one in 100,000 to 170,000 births. Currently, there are no treatments available to address fatal neuronopathic CNS disease in MPS II.
Per REGENXBIO, data from the pivotal study highlights RGX-121’s capability to change the course of the disease by restoring the gene missing in MPS II patients. The candidate also demonstrated its potential to significantly improve vital brain function for patients with this disease.
REGENXBIO has met with the FDA to discuss the positive results of the MPS II study. The FDA has confirmed that the top-line results support a biologics license application (BLA) submission for RGX-121 to treat MPS II in 2024, under the accelerated approval pathway, subject to review of the full data.
The company’s stock gained 11.7% on Feb 8, in response to the encouraging results from its CAMPSIITE study of RGX-121 for MPS II and continued to rise another 1.7% in the after-market hours. In the past year, shares of REGENXBIO plunged 36.7% compared with the industry’s 11.7% decline.
Image Source: Zacks Investment Research
Per the data readout from the study, it was observed that MPS II patients treated with RGX-121 achieved decreased CSF levels of D2S6, a key biomarker of brain disease activity, below maximum attenuated disease levels at 16 weeks. A median reduction in D2S6 of 86% was observed in patients receiving RGX-121 therapy, approaching normal levels.
REGENXBIO further stated that the pivotal results were consistent with those observed in the dose-finding phase of the CAMPSIITE study. Most MPS II patients treated with RGX-121 in the dose-finding phase exceeded expectations in neurodevelopmental function compared with natural history data up to four years.
The current standard-of-care treatment for MPS II is intravenous enzyme replacement therapy (ERT). However, new long-term follow-up data from the dose-finding phase of the study observed a high rate of patients for whom study investigators chose to discontinue ERT or were allowed to remain ERT-naïve, representing a meaningful breakthrough.
Furthermore, the gene therapy candidate demonstrated a favorable safety profile and continues to be overall well tolerated in MPS II patients.
REGENXBIO is currently gearing up to use CSF levels of D2S6 as a surrogate endpoint for accelerated approval of RGX-121 to treat the CNS indication. The company is currently completing the remaining activities and expects to file the BLA in the second half of 2024.
Subject to potential approval of the planned BLA under the FDA’s priority review status, REGENXBIO anticipates the reception of a Rare Pediatric Disease Priority Review Voucher in 2025.
In a separate press release, REGENXBIO announced that enrollment has been completed in cohort 2 (at dose level 2) of the phase I/II AFFINITY DUCHENNE study of another pipeline candidate, RGX-202, a potential one-time AAV therapeutic, which is being developed to treat Duchenne muscular dystrophy (DMD). RGX-202 therapy includes an optimized transgene for a novel microdystrophin.
The company also reported additional interim safety and efficacy data from the AFFINITY DUCHENNE study of RGX-202 in patients with DMD (aged four to 11 years old).
Per the updated safety data, RGX-202 has been well tolerated with no drug-related serious adverse events in five patients aged 4.4 to 12.1 at dose levels 1 and 2.
Furthermore, RGNX reported new three-month assessment data of the third patient receiving RGX-202 dose level 1 that observed the largest increase in microdystrophin expression (83.4%) compared with the control cohort. The patient also experienced a 93% reduction in serum creatinine kinase levels from baseline at week 10.
The company expects to select a pivotal dose of RGX-202 in mid-2024, followed by the initiation of a pivotal DMD study in the second half of 2024. REGENXBIO also plans to share initial strength and functional assessment data for both dose levels in the second half of 2024.
In the pivotal study, RGNX plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a BLA submission under the FDA’s accelerated approval pathway.
REGENXBIO Inc. Price and Consensus
REGENXBIO Inc. price-consensus-chart | REGENXBIO Inc. Quote
Zacks Rank and Stocks to Consider
REGENXBIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the drug/biotech industry are Puma Biotechnology, Inc. (PBYI - Free Report) , Immunocore (IMCR - Free Report) and ImmunoGen . While both PBYI and IMGN currently sport a Zacks Rank #1 (Strong Buy), IMCR carries a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, the Zacks Consensus Estimate for Puma Biotech’s 2023 earnings per share (EPS) has remained constant at 73 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has remained constant at 69 cents. Over the past year, shares of PBYI have gained 10.3%.
PBYI beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.
In the past 30 days, the Zacks Consensus Estimate for Immunocore’s 2023 loss per share has remained constant at 95 cents. During the same period, the consensus estimate for Immunocore’s 2024 loss per share has remained constant at $1.42. Over the past year, shares of IMCR have jumped 22.3%.
IMCR beat estimates in two of the trailing four quarters and missed on the other two occasions. In the last reported quarter, IMCR reported an earnings surprise of 112.5%.
In the past 30 days, the Zacks Consensus Estimate for ImmunoGen’s 2023 EPS has remained constant at 7 cents. During the same period, the consensus estimate for ImmunoGen’s 2024 EPS has remained constant at 37 cents. Over the past year, shares of IMGN have skyrocketed 601.9%.
IMGN beat estimates in each of the trailing four quarters, delivering an average earnings surprise of 136.05%.