Sarepta Therapeutics: BLA Filing Of SRP-9003 For LGMD Is The Next Step

seekingalpha11-09

Sarepta Therapeutics, Inc. reported Q3 2024 net product revenues of $429.8 million, a 39% year-over-year increase, driven by strong sales of ELEVIDYS and other DMD treatments.The company is advancing ...

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(NASDAQ:SRPT) recently reported its Q3 2024 earnings report just a few days ago. The significance of this is that it was able to report net product revenues for this period of $429.8 million, which was a 39% year-over-year increase. One part of the growth driver for this report was sales of ELEVIDYS, which reached $181 million. However, it is also important to note ex-US sales by Roche Holding AG (OTCQX:RHHBY), which came in at $9.5 million during the same period. The three other PMO drugs approved by the company for the treatment of patients with Duchenne Muscular Dystrophy [DMD] also did well, coming in at $248.8 million during the very same quarter. These are the regulatory-approved drugs of EXONDYS 51, VYONDYS 53 and AMONDYS 45. It continues to deliver on the front of having developed PMOs and a gene therapy to target these DMD patients. Such growth should continue forward, but why investors should care about this company now is because there might be another indication in the pipeline which might fuel additional growth. This would be regarding the development of gene therapy SRP-9003 [bidridistrogene xeboparvovec] for the treatment of patients with limb-girdle muscular dystrophy [LGMD]. As I will explain below, the goal is for it to target several subtypes that have different expressions of disease. There is one ongoing phase 3 study, known as SRP-9003-301, which is going after LGMD 2E/R4 type patients. This is an open-label trial, with incorporation of a natural history study at first to truly determine if the beta-sarcoglycan protein is restored, thus bringing about normal function for these LGMD patients. This program is important to note because it brings two catalysts, which are data from this phase 3 SRP-9003-301 study in early 2025 and then possibly a Biologics License Application [BLA] of SRP-9003 to treat these patients to the FDA by mid-2025. There will also be an expansion opportunity towards other LGMD expression types with new phase 3 studies as well. SRP-9003 For The Treatment Of Patients With Limb-Girdle Dystrophy Gets Ball Rolling As I stated above, a key thing about Sarepta Therapeutics is that it is not just looking at only pressing on with its three PMOs and one gene therapy [ELEVIDYS] for the treatment of patients with Duchenne Muscular Dystrophy [DMD]. Matter of fact, it has been able to make great progress in the development of a gene therapy program for the treatment of patients with Limb-Girdle Muscular Dystrophy [LGMD] with the use of SRP-9003 in the ongoing phase 3 SRP-9003-301 study. Before going over this specific trial for the treatment of these patients, plus any catalysts that are expected to come out of this program, it is first important to understand what this disorder is. Limb-Girdle Muscular Dystrophy [LGMD] is a type of muscle dystrophy disorder that affects the functioning of muscles. It is not in any way similar to that of DMD, though, because there are many differences to note. For instance, DMD impacts other portions of the body for starters, like the patient starting off not being able to walk or stand. Along with the fact that it progressed quicker than LGMD. This doesn't mean that LGMD is not to be concerned about. The main thing about this muscular dystrophy is that it affects the proximal muscles of portions of the body, like closer to the center of the hips and/or shoulders. Some symptoms that these patients can experience are as follows:  Wadding walk. Fixed joints. Large calves. Weakness of facial muscles.  The whole point of this gene therapy is to restore the functional beta-sarcoglycan depending on type. In the case of SRP-9003, the goal is to specifically go after type 2E/R4 LGMD patients. What I'm alluding to here is that this is the first subpopulation that it is going to go after. However, it is expected to start several other late-stage studies over time to go after other types. For instance, you may have the advancement of the following:  SRP-9004 : type 2D/R3. SRP-9005 : type 2C/R5. SRP-6004 : type 2B/R2. SRP-9006 : type 2L/R12. SRP-9010 : type 2A/R1.  Another item to point out is that SRP-9003, SRP-9004 & SRP-9005 are sarcoglycanopathies. On the flip side, LGMD can be broken into another category known as non-sarcoglycanopathies and this would include the company developing SRP-6004, SRP-9006 & SRP-9010. The end game goal is that Sarepta believes it could end up ultimately helping to treat >70% of the known LGMD patient population. To kick off this program and see if it can successfully develop SRP-9003 for the treatment of patients with Limb-Girdle Muscular Dystrophy [LGMD], it has been running the ongoing phase 3 SRP-9003-301 study [EMERGENE]. This specific open-label late-stage trial is expected to recruit up to a total of 15 patients ages 4 and up with LGMD who have the 2E/R4 genetic marker of disease. Another item worth mentioning is that it is going to recruit both ambulatory and non-ambulatory patients as well. The primary endpoint of this first phase 3 open-label study will be a change from baseline in beta-sarcoglycan expression from baseline to day 60. The first patient of this trial was screened back on January 16th of 2024. Considering that the way this study is designed for patients not be given a placebo counterpart, there is one way of knowing whether treatment works. How so? Well, the study itself is going to incorporate a natural history study portion. That is, there will be a 6-month Natural History study lead in. This portion of the study will be compared to that of the other portion of the study where patients are given SRP-9003. The promise of this program is that investors won't have to wait long to see any catalysts from it, either. That's because results from this phase 3 EMERGENE study should be released by early 2025. If the primary endpoint of beta-sarcoglycan is met with statistical significance, then it will be in a position to file a BLA of SRP-9003 to the FDA in mid-2025. Sarepta is already expected to start several other phase 3 studies for this program to get the ball rolling towards the expansion of targeting other LGMD patient subtypes, such as the ones I highlighted above. Financials According to the 10-Q SEC Filing, Sarepta Therapeutics had cash, cash equivalents and investments of $1.37 billion as of September 30th of 2024. Despite all of this cash on hand, though, the truth is that this company might have to raise cash in the coming months. Why is that? The reason, it notes in its 10-Q SEC Filing about its cash runway, is that it believes it will only have enough cash on hand to fund its operations for at least the next 12 months. This company is already producing a large amount of revenues already from the approved clinical products from its pipeline. Having said that, it was able to produce net product revenues of $429.8 million for Q3 of 2024, which was an increase of 39% from the same period the prior year. Again, ELEVIDYS revenues during this most recently reported quarter, including Roche ex-U.S. sales, were $190.5 million. The company cash burn is roughly $350.6 million of cash per quarter. This is broken down into $222.4 million in R&D expenses and then $128.2 million in G&A expenses. Risks To Business There are several risks that investors should be aware of before investing in Sarepta Therapeutics. The first risk to consider would be in terms of the development of SRP-9003 being explored in the ongoing phase 3 EMERGENE study targeting patients with LGMD type 2E/R4. The risk here is that there is no assurance that the primary endpoint of this study of change from baseline in beta-sarcoglycan from baseline to Day-60 will be achieved. The second risk to consider would then be the BLA filing of SRP-9003 for the treatment of patients with LGMD type 2E/R4 to the FDA for review. There is no guarantee that the FDA will accept the regulatory application in its present form, nor that it will ultimately approve this gene therapy for marketing in the United States. Nor, that this gene therapy will eventually be approved for these patients in any other global territory. A third risk to consider would be in terms of expanding this LGMD treatment program towards other patient population subtypes. That's because the goal is to attempt to initiate several other phase 3 studies using other candidates such as SRP-9004 and SRP-9005 to go after 2D/R3 and 2C/R5 respectively. Even though SRP-9003 has shown to be effective in being able to target 2E/R4 LGMD type patients, there is no assurance that a similar or superior outcome will be achieved for these others to be targeted. A fourth risk to consider would be in terms of the financial position that this company is in. Despite the amount of cash on hand, it has to be able to fund the development of its LGMD treatment program plus the commercialization of its three PMOs and ELEVIDYS for the treatment of patients with DMD. Having said that, it believes that its cash on hand will only be enough to fund its operations for at least the next 12 months. Thus, this means that it is likely going to have to raise cash in the coming months. Conclusion Sarepta Therapeutics has done well in continuing with the commercialization of its products, which have been approved to treat patients with DMD. I state this because of the sales it has been able to produce for ELEVIDYS alone as a gene therapy for this disorder. It was able to produce $190.5 million sales during Q3 of 2024 with it. It continues to also grow its overall net product revenues as well, including the three PMOs approved for DMD patients. This would be in terms of being able to grow net produce revenues year-over-year by 39% to $429.8 million. I believe that considerable value remains here, first and foremost because of the continued growth of its revenues being generated from the DMD portion of its pipeline. Where further shareholder value creation could be achieved would be in terms of the development of SRP-9003 and beyond targeting patients with LGMD. With the first gene therapy candidate looking to specifically go after type 2E/R4 patients. If success continues, then it could ultimately target >70% of the known LGMD cases. With the release of results from the phase 3 EMERGENE study using SRP-9003 in early 2025, plus the potential BLA fling of it in mid-2025 for LGMD patients, I believe that investors could benefit with any potential gains made here.  Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. 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