Disc Medicine (NASDAQ:IRON) has made great progress in advancing its lead candidate for its pipeline known as bitopertin for the treatment of patients with erythropoietic protoporphyria [EPP] and X-linked protoporphyria [XLP]. The use of this drug is being advanced for the treatment of this specific patient population in the ongoing phase 2 randomized placebo-controlled clinical study known as AURORA. This is a key program for investors to look out for because it is said that this biotech is going to present results from this mid-stage study in early 2024. The thing is that this drug is being developed to target disorders caused by dysregulated heme synthesis. Having said that, it has already initiated and enrolled the first patient in an NIH-sponsored phase 1/2 study of bitopertin for the treatment of patients with Diamond-Blackfan Anemia.
Not only does this company have this catalyst for investors to look forward to, but it also has two other data readouts which are expected to be released by the end of 2023. This would be with respect to DISC-0974, which is being used to help patients with disorders suffering from anemia of inflammation. One data readout expected by the end of this year would be a phase 1b/2 study in patients with anemia due to chronic kidney disease [CKD] who are not receiving dialysis. A second data readout that is expected by the end of this year would be a phase 1b/2 study in patients with myelofibrosis [MF] with severe anemia on stable background therapy.
Bitopertin For The Treatment Of Patients With EPP And X-linked Protoporphyria
The main clinical program in Disc Medicines’ pipeline would be the use of its drug bitopertin, which is being developed for the treatment of patients with EPP and x-linked protoporphyria [XLP]. Erythropoietic protoporphyria is an inherited disorder, which results in the accumulation of protoporphyrins in red blood cells that causes acute, painful photosensitivity and potential liver disease. It is observed in early childhood and pain occurs when a person is exposed to bright sunlight. What occurs in this disorder is that the heme synthesis pathway is greatly affected. Having said that, this results in a toxic accumulation of porphyrins, which are activated when a patient is exposed to sunlight. This results in a lot of pain on the skin, along with other problems like skin damage as well. Thus, sun exposure for a person with this disorder is not ideal. Another issue that could occur as a result of EPP would be gallstone formation and chronic liver disease as well. The use of bitopertin is being explored in the ongoing phase 2 randomized, placebo-controlled AURORA trial treating adults with EPP. It is expected that results from this phase 2 study are going to be presented at a medical conference in early 2024.
Whether this additional clinical data to be released at this medical conference ends up being positive remains to be seen, but Disc Medicine has already reported positive results from another study. Such a study is an open-label trial known as BEACON, which used bitopertin for the treatment of patients with EPP and XLP. It was noted that there was a dose-dependent reduction of protoporphyrin IX [PPIX] that was observed for patients who took this drug. There was a mean >40% dose dependent reduction of metal-free PPIX levels when compared to baseline. Why is this an important finding? Well, that’s because this PPIX that has free radicals roaming the body causes these patients to experience issues/pain when exposed directly to sunlight. This is also stated in scientific literature whereby reductions in PPIX levels >30% has a major impact on photosensitivity in these EPP and XLP patients. Besides achieving the necessary reduction of PPIX levels which was observed in these patients, it also translated to an improvement in quality of life for many. That is, there were several scales reported whereby the quality of life for these patients had improved greatly. This was with respect to scales such as Patient Global Impression of Change [PGIC], Patient Global Impression of Severity and EPP Impact Questionnaire [EPIQ] as well.
It was good to see that treatment with bitopertin resulted in patients being able to improve their quality of life. Hopefully, the next set of results to be released in early 2024, reconfirms all the positive data observed to date. Specifically, sunlight tolerance greatly improves for these patients. One thing to note is that this biotech has another shot on goal to consider as well. That’s because the use of bitopertin can be applied toward other genetic disorders caused by dysregulated heme synthesis. For instance, another target indication being explored by the company would be Diamond-Blackfan Anemia. It has already initiated and enrolled the first patient in an NIH-sponsored phase 1/2 study of bitopertin for the treatment of patients with Diamond-Blackfan Anemia in July of 2023. This is another shot on goal for this biotech and may prove that heme synthesis modulation with bitopertin is feasible.
Financials
According to the 10-Q SEC Filing, Disc Medicine had cash and cash equivalents of $377.6 million as of June 30, 2023. The reason for the large amount of cash on hand is because back on June 22, 2023, it raised aggregate gross proceeds of $158 million through a public offering of common stock and pre-funded warrants. It believes that the current cash on hand should be enough to fund its operations well into 2026. This means that there should be no near-term risk of dilution and the company is now well capitalized to fund itself for a few years. The only way a cash raise might be done is if the results to be released from the phase 2 AURORA study in treating patients with EPP are positive. If such results cause the stock price to climb significantly, then it is quite possible that a cash raise could be done to further strengthen its balance sheet if nothing else.
Risks To Business
There are several risks that investors should be aware of before investing in Disc Medicine. The first risk to consider would be with respect to the ongoing phase 2 randomized, placebo-controlled AURORA study using bitopertin for the treatment of patients with EPP and XLP. Even though the company was able to obtain dose-dependent results showing a reduction of PPIX and other quality-of-life measures in the BEACON study, there is no assurance that positive results will be released from the AURORA study. A second risk to consider would then be with respect to the use of bitopertin for the treatment of patients with Diamond-Blackfan Anemia.
This is another shot on goal for this biotech, but just because the drug was able to achieve preliminary positive clinical data in EPP/XLP, there is no guarantee that it will be able to do well for this other patient population with dysregulated heme synthesis. A third and final risk to consider would be the advancement of another clinical candidate in the pipeline known as DISC-0974, which is being used to help patients suffering from anemia or inflammation. That’s because there are two ongoing studies using this drug to target patients with anemia due to chronic kidney disease [CKD] who are not receiving dialysis and Myelofibrosis [MF] patients with severe anemia on stable background therapy. Results from both of these studies are expected to be released by the end of 2023. There is no guarantee that one or both of these studies will be successful in using DISC-0974 effectively against them.
Conclusion
Disc Medicine has made great progress in advancing the use of its drug bitopertin for the treatment of patients with EPP and XLP. Thus far, it has been able to establish proof-of-concept in being able to use this drug to help boost sunlight tolerance and to also improve their quality of life as well. What investors should keep an eye on is data to be released from the phase 2 AURORA study. This is going to be an important inflection point for the company and will reaffirm positive findings already observed from the BEACON study. There is another program to consider as part of its pipeline as well, which is MWTX-003 that is being advanced for the treatment of patients with polycythemia vera as well as other indications. However, the goal is to first initiate a phase 1 study in healthy volunteers in the 2nd half of 2023 to establish initial safety first. With positive preliminary data established in using bitopertin for the treatment of patients with EPP and XLP, plus several catalysts on the way, these are the reasons why I believe that investors might be able to capitalize on potential gains made.
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