Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Regenxbio Inc (RGNX, Financial) reported positive data for RGX-202, demonstrating consistent and robust microdystrophin expression across treated patients, including older ambulatory boys.
- The company completed a successful end-of-Phase 2 meeting with the U.S. FDA for RGX-202, with plans to file a BLA using microdystrophin as the primary endpoint for accelerated approval.
- Regenxbio Inc (RGNX) is advancing RGX-314 for diabetic retinopathy and wet AMD in collaboration with AbbVie, with plans to initiate pivotal trials in the first half of 2025.
- The company reported a strong financial position with $327 million in cash, cash equivalents, and marketable securities as of June 30, 2024, providing a runway into 2026.
- Regenxbio Inc (RGNX) is expanding its RGX-314 program to include patients with diabetic macular edema, further broadening the potential market for this gene therapy.
Negative Points
- Regenxbio Inc (RGNX) faces uncertainties and risks associated with forward-looking statements, which may cause actual results to differ from forecasts.
- The company reported a decrease in R&D expenses to $49 million for Q2 2024 from $60 million in Q2 2023, primarily due to reduced manufacturing and clinical supply costs, which may indicate slower progress in some areas.
- Regenxbio Inc (RGNX) is still awaiting final agreement from the FDA on the proposed threshold for microdystrophin levels for RGX-202, which could impact the approval process.
- The company has not yet finalized the safety database requirements for RGX-202, which could affect the timeline for approval.
- Regenxbio Inc (RGNX) faces competition in the gene therapy space, particularly for wet AMD, where other companies are also advancing their programs.
Q & A Highlights
Regenxbio Inc (RGNX) Q2 2024 Earnings Call Highlights
Q: Congrats on the new data regarding RGX-202. Do you have a goal for the protein level you want to achieve, and do you expect the FDA to set some thresholds for accelerated approval?
A: We have proposed a threshold with the FDA and are awaiting final agreement. We believe a level above 10% is likely to result in functional benefit. All our published data to date shows patients above this threshold. (Curran Simpson, CEO)
Q: Could you speak more about the functional assessment data for RGX-202 expected later this year?
A: We measure microdystrophin at three months and functional assays every three months. In the fall, we expect to see significant functional data out to 12 months for dose level one patients and six to nine months for early dose level two patients. (Curran Simpson, CEO)
Q: How do you see the RGX-314 suprachoroidal program moving towards a pivotal program for wet AMD?
A: We will assess safety and efficacy using biomarkers like retinal thickness, BCVA, and reduction in treatment burden. We aim for good durability and anatomic control while reducing injection burden. (Stephen Pakola, CMO)
Q: Can you elaborate on interest levels in the patient community for RGX-202 in light of recent approvals?
A: There is strong interest in our program due to its differentiation and demonstrated safety. We are confident in our ability to recruit for the pivotal program. (Curran Simpson, CEO)
Q: What kind of safety database do you expect to be required for RGX-202 approval?
A: We are still finalizing specifics with the FDA, but our proposal is for a sample size in the 30 to 40 patient range. Our commercial-ready process and initial safety record should positively influence the FDA's assessment. (Curran Simpson, CEO)
Q: Are you targeting younger patients for RGX-202, given the expanded age range to one to three years?
A: Yes, we aim to build an adequate safety database for patients aged one to three while enrolling across a wide variety of ages. (Curran Simpson, CEO)
Q: Do you have any in-house data on microdystrophin levels beyond three months?
A: We measure microdystrophin at three months and do not have biopsies beyond that time point. However, our preclinical data suggests excellent durability. (Curran Simpson, CEO)
Q: Can you expand on your recent end-of-Phase 2 meeting with the FDA for RGX-202?
A: We aim for a broad approval, not just for patients ineligible for current therapy. Our strategy is based on the differentiation of our product and addressing unmet needs. (Curran Simpson, CEO)
Q: What are the primary questions you expect the FDA to need answers to for the Phase 3 diabetic retinopathy trial?
A: We feel confident due to the established roadmap for diabetic retinopathy. The primary focus will be on definitions and endpoints, which are well understood. (Stephen Pakola, CMO)
Q: When do you anticipate sharing data from the fellow eye study for wet AMD?
A: We expect to share data roughly six months after completing enrollment. We are confident in the safety profile of suprachoroidal delivery and plan to assess fellow eye dosing over time. (Stephen Pakola, CMO)
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
