Toa55/iStock via Getty Images
Mirum Pharmaceuticals (NASDAQ:MIRM) is a good speculative biotech play to look into. The reason why I state that is because it has already received FDA approval for a drug from its pipeline. It received regulatory approval for a drug known as LIMVARLI [Maralixibat], which was approved in 2021 for infants aged 1 year and older. In March 2023, the label was expanded to include the treatment of infants with pruritus aged 3 months and older. The biotech has also received European Authorization of LIMVARLI for cholestatic pruritus in patients with ALGS two months of age and older. There is an ability to expand sales of LIMVARLI with the treatment of patients with Progressive Familial Intrahepatic Cholestasis [PFIC]. That’s because a supplemental New Drug Application [sNDA] of LIMVARLI for PFIC was submitted to the FDA and assigned a Prescription Drug User Fee Act [PDUFA] date of December 13, 2023.
In addition, a label expansion of LIMVARLI for this patient population was submitted to the European agency as well. There are also several data readouts to expect from this company as well, which is the use of its drug Volixibat for the treatment of patients with primary sclerosing cholangitis and primary biliary cholangitis. Results from both of these ongoing studies are expected to be released in the 2nd half of 2023. The main reason why I wanted to go over this biotech is because of its use of LIMVARLI for the treatment of patients with Biliary Atresia. That’s because topline results from a study using this drug to treat this patient population, are expected to be released in the 2nd half of 2023. Why I believe this is a very important part of Mirum Pharmaceutical’s pipeline is because of the multi-billion dollar market opportunity for it, which I will be going over below. With an already regulatory approved product known as LIMVARLI, plus several data readouts expected in the 2nd half of 2023, these are the reason why I believe that Mirum Pharmaceuticals is a good speculative biotech play to look into.
EMBARK Study Results Are A Must Watch
While the other results expected in the 2nd half of 2023 are important to watch as well, I believe that the results to be released from the phase 2b EMBARK study are the most important to watch. This trial is using LIMVARLI to treat patients with Biliary Atresia. Why is this the most important study from Mirum’s pipeline that needs to be watched? That’s because of the possible multi-billion dollar market opportunity that could achieved in targeting this patient population. It is expected that the global Biliary Atresia market could grow to $1.69 billion by 2029. This is a large market opportunity, and it would definitely help the company to boost sales of its drug LIMVARLI, if it ultimately receives regulatory approvals for it and for this indication as well. Biliary Atresia is a disorder in infants whereby the bile ducts outside and inside the liver are scarred and blocked. With such ducts being blocked, the bile builds up inside the liver and damages it. Damaging of the liver leads to a multitude of issues such as: Loss of liver function, cirrhosis, and scarring.
How does Mirum Pharmaceuticals treat Biliary Atresia and other cholestatic liver diseases? It does so with ileal bile acid transporter [IBAT] inhibition in three ways, which are as follows:
By LIMVARLI performing these functions, it might be able to help these infants with Biliary Atresia. The phase 2B EMBARK study is using LIMVARLI to treat patients with biliary atresia who have already undergone a surgery known as hepatoportoenterostomy [Kasai procedure]. Such a surgery must be done early to prevent biliary cirrhosis and liver failure. The life expectancy of Biliary atresia patients after Kasai procedure in 5 years is only 65%. As you can see, this is why a treatment option like LIMVARLI might be important to be given after such a surgery. The primary endpoint of this study is the mean change in total serum bilirubin levels from baseline to week 26. The use of LIMVARLI is being compared to placebo with respect to this primary endpoint over the 26-week period. Another important endpoint would be to see a change in serum bile acids from baseline to week 26. A huge development to consider is that enrollment completion of this phase 2b study was announced back on May 11, 2023. As such, this sets up an important catalyst for investors to look forward. Final results from this phase 2b EMBARK study, using LIMVARLI for the treatment of infants with biliary atresia, are expected to be released in the 2nd half of 2023.
According to the 10-Q SEC Filing, Mirum Pharmaceuticals had cash, cash equivalents, restricted cash equivalents, and short-term investments of $232.1 million as of March 31, 2023. This pharmaceutical is in great shape compared to others, because it is already producing revenues for LIMVARLI. Q1 of 2023 net sales of grew by 167% as compared to Q1 of 2022. That is, product revenues in Q1 of 2023 were $29.1 million, compared to $10.9 million in Q1 of 2022. It should be well capitalized for a while, because it closed an upsized offering of $316.3 million aggregate principal amount of 4.00% convertible senior notes due 2029, including exercise of the initial purchasers’ option in full in April 2023.
Risks To Business
There are several risk that investors should be aware of before investing in Mirum Pharmaceuticals. The first risk to consider would be with respect to the release of results from the phase 2b EMBARK study, which is using LIMVARLI for the treatment of patients with biliary atresia. These results are expected to be released in the 2nd half of 2023 and there is no guarantee that they will be positive. Should this trial not meet upon the primary endpoint, then this biotech will not be able to target the multi-billion dollar market opportunity for BA. A second risk to consider would be with respect to the other two data readouts that are going to be released in the 2nd half of 2023. This would be with respect to the release of interim analysis results from the phase 2 studies using Volixibat for the treatment of patients with primary sclerosing cholangitis and primary biliary cholangitis.
A third risk to consider would be with possible competition in place. Mirum Pharmaceuticals isn’t the only company working on rare liver diseases. Ipsen has its own ileal bile acid transport inhibitor drug that has also already been approved by regulators. That is, it has received U.S. approval in 2021 for the treatment of pruritus in patients three months of age and older with progressive familial intrahepatic cholestasis [PFIC] and in the European Union for the treatment of PFIC in patients aged six months and older. Ipsen even completed its acquisition of Albireo back on March 2, 2023. This means that Mirum Pharmaceuticals will have competition with Ipsen relating to several cholestatic diseases.
The final conclusion is that Mirum Pharmaceuticals is a good speculative biotech play to look into. That’s because it has already been able to receive regulatory approvals of LIMVARLI for the treatment of patients with Alagille Syndrome. There is potential to expand the use of LIMVARLI with an sNDA for PFIC, which had already been submitted to the FDA. A PDUFA date of December 13, 2023 has already been established for review of this sNDA. Not only that, but I believe there are several catalysts which are expected in the 2nd half of 2023, which could possibly boost the stock price. In particular, I believe that the phase 2b EMBARK study is most important. Results from this study are also expected in the 2nd half of 2023 and if successful it would allow the biotech to target a very large multi-billion dollar market. It is expected that the global biliary atresia market could grow to $1.69 billion by 2029. With an already regulatory approved product known as LIMVARLI, plus several data readouts expected in the 2nd half of 2023, these are the reason why I believe that Mirum Pharmaceuticals is a good speculative biotech play to look into.
This content was originally published here.
