Mirum: Potential For European Approval To Move It Forward (MIRM)

Mirum Pharmaceuticals (NASDAQ:MIRM) is a great speculative biotech play to look into. The reason why I say that is because it has already received FDA approval of its drug, known as LIVMARLI, for cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. Not only that, but it has also already received a positive CHMP opinion, which puts it in a good position to possibly obtain European approval of LIVMARLI for cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. Mirum has given guidance that full-year 2022 sales will reach $70 million. Not only does it have the potential for European approval of LIVMARLI in Alagille syndrome, but it received regulatory approval of this drug for ALGS in Israel. In addition, the drug has also established proof of concept in treating patients with familial intrahepatic cholestasis (PFIC). This was proven in the ongoing phase 3 MARCH study, which used LIVMARLI to treat this specific patient population. With a possible European approval of LIVMARLI on the way, plus proof of concept established using this drug in PFIC as well, these are the reasons why I believe that Mirum Pharmaceuticals is a great speculative biotech play to look into.

Possible European Expansion For LIVMARLI Could Bring In Added Revenue

As I stated in the intro above, Mirum Pharmaceuticals had already received FDA approval of LIVMARLI for the treatment of patients with cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. The ALGS population in the United States is not large, as only 2,000 to 2,500 children are affected by it in this territory. However, there are two positive items that this company can hang its hat on. The first item is that LIVMARLI is the only FDA approved drug to treat this rare liver disease population. It hasn’t done too bad thus far in terms of revenue either as it was able to generate roughly $18.8 million in revenue in Q3 of 2022 and it expects to generate about $70 million in 2022 full year net product sales. Despite the small revenue generated to date, this is where a potential expansion opportunity comes into play as I highlighted above. That is, Mirum has potential to also receive European Medicines Agency (EMA) approval of LIVMARLI for the treatment of patients with LIVMARLI for cholestatic pruritus in patients with ALGS two months of age and older. An expansion of LIVMARLI into the European territory, for the treatment of patients with ALGS, would only serve to help it see an increase in sales. As far as a catalyst goes for traders/investors to look forward to, it would be the European Union decision on whether or not to approve LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older, before the end of 2022.

Proof Of Concept Established For LIVMARLI In Other Rare Liver Disease Indication

LIVMARLI seems to be a strong drug as it has already done well in treating cholestatic patients with ALGS. However, it is not the type of drug that has only achieved proof of concept in just one indication and that’s it. Nope, it has also been able to do well in the phase 3 MARCH study, using LIVMARLI to treat patients with progressive familial intrahepatic cholestasis (PFIC). This late-stage study was a huge success, as the primary endpoint was met. This trial recruited a total of 93 patients with PFIC, who were between the ages of 1 and 17 years. The primary endpoint was an improvement in pruritus severity in PFIC2. Before stating the results, it’s important to understand two key items. First, that the primary analysis was only done in 31 PFIC2 patients. Secondly, PFIC2 means a specific subtype of PFIC. The primary endpoint of improvement in pruritus (itch) severity in PFIC2 patients was met with statistical significance with a p-value of p=0.0098. PFIC is also a rare disease, which is said to affect one in every 50,000 to 100,000 births in the United States and Europe. Despite this, it is important to put this population into context. There is a total of six types of PFIC. However, it is said that the PFIC2 patient population accounts for about 60% of the PFIC patient population. With Mirum Pharmaceuticals achieving the primary endpoint in this primary PFIC2 cohort population, it can discuss the potential to file for regulatory approval of LIVMARLI for these patients.

Financials

According to the 10-Q SEC Filing, Mirum Pharmaceuticals had cash, cash equivalents, restricted cash equivalents and investments of $285.3 million as of September 30, 2022. The reason for the cash on hand is because of a cash raise that was done back in August of 2022. This is where it sold a total of 3,478,261 shares of common stock at a price to the public of $23 per share. In addition, the underwriters were given an option to purchase up to 521,739 additional shares of its common stock at the very same public offering price. In total, it raised approximately $86.1 million after deducting expenses. This biotech is still starting to generate revenue, but it seems that its launch of LIVMARLI in the United States is doing pretty good. Especially, considering that it has guided full-year 2022 sales of LIVMARLI to be about $70 million. I believe that, should it get a nod to market this drug for ALGS patients in Europe, that its sales should start to grow at a more rapid pace. It believes that its cash on hand is enough to fund its operations for at least the next 12 months from the date of this 10-Q SEC filing, which was filed on November 9, 2022. I don’t believe it will raise cash before the end of 2022, but I believe it might do so in early 2023. The reason why I state that is because European approval of LIVMARLI for the treatment of cholestatic pruritus in patients with ALGS of two months of age and older, is expected before the end of 2022. I expect the stock price to rise on the back of approval of LIVMARLI for ALGS in this territory. Not only because it would be another drug approval for Mirum Pharmaceuticals, but also since it will be the first medication in Europe to treat cholestatic pruritus for patients with ALGS.

Risks To Business

There are several risks that investors/traders should be aware of before investing in Mirum pharmaceuticals. The first risk to consider involves the decision by the European Union on whether or not they should approve LIVMARLI for the treatment of cholestatic pruritus in patients with ALGS two months of age and older. This decision is expected before the end of 2022 and there is no guarantee that it will be approved for marketing in the European Union. A second risk to consider would be with respect to the positive results obtained in the phase 3 MARCH study, which used LIVMARLI for the treatment of patients with PFIC2. The primary endpoint was met, all that is left now is to see if the biotech is able to move forward with a regulatory filing for this indication in several territories. There is no assurance that LIVMARLI will also be approved for this other indication. A third risk would be with respect to the potential revenue it could generate with LIVMARLI in the United States and Israel territories. It estimates that full-year 2022 sales will end up being $70 million for LIVMARLI, but there is no assurance of this happening. The final risk to consider would be with respect to the ongoing financial position, which has been laid out by Mirum. That is, it believes it only has enough cash to fund its operations for at least 12 months from the date of the 10-Q SEC filing, which was filed November 9, 2022. Again, it is my belief that it will likely have to raise cash again in 2023. It’s possible it could choose to do so if it receives European approval of LIVMARLI for the treatment of cholestatic pruritus in patients with ALGS two months of age and older.

Conclusion

The final conclusion is that Mirum Pharmaceuticals is a great speculative biotech play to look into. The reason why I state that is because there is a major catalyst approaching before the end of 2022. This involves the possibility to obtain marketing approval of LIVMARLI, for the treatment of cholestatic pruritus in patients with ALGS two months of age and older. It already has been able to obtain regulatory approvals of LIVMARLI for this indication in the United States and Israel thus far. Marketing approval being obtained for the European Union would only sever to help the possible revenue that could be generated. Sales of LIVMARLI seem to be doing fairly well. It has given guidance that full-year 2022 sales of this drug will reach $70 million. The drug being able to do well in the other indication, which is PFIC2, is another positive. That’s not all either, because LIVMARLI is also being used to target another rare liver disease in the ongoing phase 2b/3 EMBARK study, which is Biliary Artesia. It is expected that results from this late-stage study will be released in the 2nd half of 2023. Should the company be successful in using LIVMARLI to treat this indication as well, then it will provide another avenue possibly for multiple regulatory approvals in the coming years.