Reneo Pharmaceuticals, Inc. (NASDAQ:RPHM) has made great progress in being able to advance its pipeline. That’s because it is in the process of advancing its phase 2/3 study known as STRIDE, which is using its drug mavodelpar for the treatment of patients with primary mitochondrial myopathies [PMM].
Why investors should keep an eye on this biotech is because results from this pivotal phase 2/3 study are expected to be released in December of 2023. Should this data turn out to be positive, then the company will likely be able to file a regulatory application of mavodelpar for the treatment of this patient population in 2024.
Not only that, but this biotech has even gone on to initiate an open-label study for this program as well, known as STRIDE AHEAD. The reason why it will be important for investors to also track this program will be because of the addition of patients with PMM due to nuclear DNA defect. Interim results from this open-label study are expected to be released in 2024.
Phase 2/3 Pivotal Study Data Is Major Inflection Point
As I noted above, Reneo Pharmaceuticals is gearing up to report results from its phase 2/3 pivotal STRIDE study, which is using its drug mavodelpar to treat patients with primary mitochondrial myopathies [PMM]. Such results are expected to be released in December of 2023 and if it positive, it would allow for this biotech to file regulatory applications of this drug for the treatment of this patient population in 2024. These would be both a New Drug Application [NDA] and Marketing Authorization Application [MAA] of mavodelpar to the FDA and EMA respectively.
This STRIDE study is a global, randomized, double-blind study using mavodelpar for the treatment of adults with PMM due to mtDNA defects. Patients are to be receive either 100 mg of mavodelpar or placebo once-daily over a 24-week period. The primary endpoint of this study is going to be the change from baseline in the distance walked during the 12-minute walk test [12MWT] at week 24. What happens in Primary mitochondrial myopathies [PMM] is that there are changes that occur in genetic material, either found in the DNA of mitochondria [mtDNA] or genes outside of the mitochondria [nuclear DNA]. With such drastic changes in genetic material, the skeletal muscle is greatly affected.
Weakness muscle can occur in the face and neck, but can also happen in the arms and legs. What this pivotal study is looking at is patients who have genetic changes that occur in mtDNA only. An analyst believes that if mavodelpar is approved by the FDA, it could generate approximately $1.48 billion in peak sales in the United States alone. what was also stated is that with the mechanism of action in place, it would also be used to treat other mitochondrial disorders. Reneo Pharmaceuticals has already moved on towards possibly expanding the primary mitochondrial myopathies [PMM] population that it is going after. That’s because it initiated an open-label extension [OLE] trial which not only recruited mtDNA patients from the phase 1b study, but also treatment naive patients with PMM due to nDNA defects. As I stated above, nDNA stands for “Nuclear DNA” and this deals with genetic material being changed outside the mitochondria. The advancement of targeting nDNA in PMM patients is that the label could eventually be expanded to allow for treatment of this patient population as well.
Financials
According to the 10-Q SEC Filing, Reneo Pharmaceuticals had cash, cash equivalents and short-term investments of $125.6 million as of September 30th of 2023. It believed that it has enough to fund itself through the potential United States Application filing of mavodelpar that is anticipated in the 1st half of 2024.
Being that the company notes that it has the regulatory applications approaching, plus the increase in commercial development expenses it will need to start looking at ways to raise capital. Thus, it chose to file a form S-3 Registration Statement. This is where it intends to from time to time sell up to $300 million worth of any combination of securities including common stock, preferred stock, debt securities or warrants in order to raise cash as needed. With the use of this S-3 registration form, it should be able to raise the funds necessary to be able to file the regulatory applications of mavodelpar and adequately fund itself for quite some time.
Risks To Business
There are several risks that investors should be aware of before investing in Reneo Pharmaceuticals. The first risk to consider would be with respect to the release of the upcoming results from the phase 2/3 STRIDE study, which is using mavodelpar for the treatment of patients with primary mitochondrial myopathies [PMM] due to mitochondrial DNA [mtDNA]. There is no assurance that once that results are released that the primary endpoint of change in baseline in the distance walked during the 12-minute walk test [12MWT] at week 24 will be met with statistical significance.
The second risk would then come about if somehow Reneo does end up meeting the primary endpoint of this late-stage pivotal STRIDE study. That’s because the goal for this biotech is to be able to file a New Drug Application [NDA] and Marketing Authorization Application [MAA] to the FDA and EMA of mavodelpar for PMM respectively in 2024. There is no guarantee that this company will be able to receive regulatory approval of this drug for the treatment of this population in one or both of these territories.
The third and final risk to consider would then be with respect to the ability of mavodelpar to be able to be used towards the targeting of other mitochondrial disorders. Especially, when you consider that Reneo has already included the treatment of PMM due to nDNA defects. Even though this drug was shown to do well in PMM patients with mtDNA, there is no assurance that it will do well in nDNA patients.
Conclusion
Reneo Pharmaceuticals, Inc. has done well to advance the use of mavodelpar for the treatment of patients with primary mitochondrial myopathies [PMM]. It is expecting to report results from its phase 2/3 STRIDE study next month and if the primary endpoint is met, it would allow for it to be able to file regulatory applications of this drug for the treatment of this patient population thereafter.
Not only are there these items to look forward to for this biotech, but there is another catalyst to keep an eye on. This would be the release of results from the open-label extension STRIDE AHEAD study, which is looking at the long-term outlook of PMM patients [both mtDNA and nDNA] who are treated with mavodelpar. An interim-analysis from this open-label extension study is expected to be released in 2024. This is another opportunity for long-term Reneo Pharmaceuticals, Inc. investors to keep an eye on.
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