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| So, I feel like RGX-121 is a very strong clinical data set to support high unmet need across a lot of boys with Hunter |
| So I just think a few months removed from the updated strategic plan and the focus, we’re really enthusiastic about the momentum that we have with the science and with this plan right now |
| I think with the microdystrophin expression that we’ve been seeing to date with dose level 1, we continue to be really enthusiastic about the fact that there’s an approval endpoint that has been supported by the FDA |
| As a result of these recent updates and upcoming near-term milestones and milestones throughout 2024 announced today, we believe REGENXBIO is well positioned for success this year and in the long term |
| We are thrilled to see RGX-202 is demonstrating strong microdystrophin expression across a wide range of patients |
| And with feedback all along, I think we’re in an excellent position with our approach to file a BLA as quickly as possible here |
| But I think it really, for us, reflects the robustness of the treatment, and we’re going to be excited as we get into dose level 2 to be able to continue to reinforce that RGX-202 is highly expressive and also contributes meaningfully to, hopefully, improvement in these boys |
| We’re very excited about the opportunity in DR given the size of this market, which exceeds that of wet AMD, and because we believe this patient population could benefit the most from a potential one-time gene therapy |
| Duchenne is a market where there’s a large unmet need for new therapies and that is capable of supporting multiple gene therapies, and we believe RGX-202 has a unique, differentiating set of features that support its potential to be a best-in-class product |
| The anti-VEGF market opportunity is poised to grow significantly as the population ages |
| Our recent milestones demonstrate how our new plan is supporting the acceleration in the development of gene therapies and the expansion of value for our shareholders |
| But when it comes to things like using a biomarker to support accelerated approval, when it comes to using that biomarker to assess it as a surrogate endpoint, we’re on the same page with a lot of other people, and we’re really encouraged about where our program is going and hope that that also is, frankly, something that’s an observational change for other programs as well |
| Early on in 2024, we’ve experienced exciting progress for each of these programs |
| And for MPS II, we achieved what I think is a major breakthrough with statistically significant results for our pivotal trial on which we are filing a BLA |
| Feeling really confident about the BLA filing, really working as quickly as possible to get that on track |
| And so it will be two or one, and the preclinical evidence says it’s two, but the clinical evidence right now says one is really good |
| So that really puts us in the AbbVie in a good position as far as assessing the potential to move into later stage development |
| In November, we presented one-year data from dose levels one and two showing RGX-314 to be well tolerated, with patients demonstrating clinically meaningful improvements in disease severity with reductions in vision threatening events |
| This particular patient was 6.5-year old, and I think we were really enthusiastic about this being the largest increase in microdystrophin |
| I think the durability as you mentioned, and certainly the opportunity to have this big of a database of patients and patient exposures, combined with the durability that we can show, and when you throw in the VA stability and even improvement in the setting of a dramatic reduction in treatment burden, that’s really unparalleled compared to any of the competitors out there, when you think of the combination of durability, reduction in treatment burden with the VA findings |
| We’ve been seeing early on this year that we’ve been achieving those goals and continue to be on track to not only maintain that guidance, but continue to be confident in it |
| I think the capital is in a good place |
| I mean, they’re just – they’re obviously a great global partner with a global footprint and an unbelievable engine for really tuning into the patient and in this case the retinal specialist experience to reinforce what they need |
| And with that we’ve even seen improvement in vision in some patients at three years and beyond four years from a single injection and that is with more than overcoming |
| And we know that that’s at a one-year time point and we know, as Ken mentioned we fortunately late last year were able to present one-year data from dose levels 1 and 2, where they both were well tolerated |
| We reran some preclinical studies directly comparing dose level 1 and dose level 2 in MDX mice, and we see separation, we see better results with dose level 2 |
| We have differentiated biology |
| These events were exciting for us, and today we’re going to be talking about additional upcoming data and events that we’re giving guidance on |
| The data is great |
| 2024 our plans are intended to generate significant value for our shareholders as we ensure that resources are allocated to our most valuable product candidates and to accelerate the development of these candidates |
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| Maybe thinking beyond an approval, and I realize there’s a lot of uncertainty to get to that point |
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