Perhaps the single best-performing biotech stock on Wednesday was a small company that’s remained relatively out of the financial limelight. Mirum Pharmaceuticals (NASDAQ: MIRM) shot up as much as 44% over the course of Wednesday. What’s even more impressive, however, is that this stock has gained an addition 190% over the past three days following an FDA meeting that could see its rare liver disease drug receive approval.
Since Monday, shares of Mirum have jumped from $7 to $22 after the company announced that the Food and Drug Administration (FDA) had arranged for a pre-NDA (New Drug Application) meeting, something which bodes very well for the company’s signature drug candidate.
“We are pleased that the FDA recommended that we change the classification of our requested Type C meeting to a pre-NDA meeting and that we have a path to a rolling NDA submission for maralixibat beginning in the third quarter of 2020,” said Chris Peetz, CEO and President of Mirum. “We look forward to working with the FDA to bring this much-needed treatment option to children living with Alagille syndrome as rapidly as possible. As part of our commitment to patients and their families we also plan to initiate an expanded access program in the second half of 2020.”
Specifically, Mirum is developing a drug called maralixibat, which targets patients with pruritus associated with Alagille syndrome. Pruritus is a type of itch that tends to emerge on the bodies of patients with various kidney and liver diseases, while Alagille syndrome is a very rare genetic disorder where the bile ducts in patients become far too narrow, which leads to a number of other problems as well.
While maralixabat is Mirum’s primary drug candidate, which is currently enrolled in a phase 3 as well as a phase 2b clinical study, the company also has a second treatment in the works called volixabat. However, this drug candidate is still at the preclinical planning stage, and as such, won’t really impact Mirum’s short-to-mid term prognosis.
Shares of Mirum shot up by 44% on Wednesday, following already strong gains on Tuesday and Monday. Although there are only five Wall Street analysts covering the stock, all of them are extremely bullish at the moment. Four of them hold a “buy” rating, while one has a slightly less bullish “overweight” rating. Time will tell how Mirum ends up performing, but the fact that its main drug candidate is in an area with little to no competition means it could easily become a success should it receive FDA approval. Biotech investors should keep their eyes out for future developments from the company in regards to potential drug approval.
Mirum Pharmaceuticals Company Profile
Mirum Pharmaceuticals Inc is a biopharmaceutical company focused on the development and commercialization of therapies for debilitating liver diseases. It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases. – Warrior Trading News
