Mirum Pharmaceuticals - MIRM STOCK NEWS

Mirum Pharmaceuticals (MIRM) announced a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher for $110 million. This PRV was granted by the U.S. FDA in September 2021 for LIVMARLI™, a treatment for cholestatic pruritus in Alagille syndrome. The company expects to receive the payment upon closing the transaction, pending U.S. antitrust clearance. Jefferies LLC acted as the exclusive financial advisor for this transaction.

Mirum Pharmaceuticals announced the U.S. commercial launch of LIVMARLI™, the first approved treatment for cholestatic pruritus in Alagille syndrome (ALGS) patients aged one and older. The FDA approval marks a pivotal moment for the company, supported by a robust financial position with $205 million in cash. Significant clinical data was presented, showing improved six-year event-free survival rates (p<0.0001) and durable responses in pruritus from the pivotal ICONIC study. Despite a net loss of $47.1 million for Q3 2021, Mirum aims for sustained growth with an advancing clinical pipeline.

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) presented a new analysis of its drug LIVMARLI™ (maralixibat) at The Liver Meeting®, showing statistically significant improvements in six-year event-free survival and transplant-free survival for patients with Alagille syndrome (ALGS) (p<0.0001). The study, conducted by the Global Alagille Alliance (GALA), compared LIVMARLI's clinical trial data against a natural history cohort, indicating a 70% reduction in adverse clinical outcomes. These results support LIVMARLI's potential as a significant treatment for ALGS patients, especially in delaying liver transplants.

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) will report its financial results for Q3 2021 on November 15, 2021, at 1:30 p.m. PT. A conference call and audio webcast will follow to discuss the results and business updates. Mirum is focused on treating rare liver diseases with its approved drug LIVMARLI™ and investigational treatments. The company is advancing its late-stage pipeline, which includes pivotal studies for conditions like Alagille syndrome and biliary atresia.

Mirum Pharmaceuticals (MIRM) announced the presentation of new data for LIVMARLI (maralixibat) at the American Association for the Study of Liver Diseases annual congress from November 12-15, 2021. The highlights include a late-breaker oral presentation on a six-year event-free survival analysis in Alagille syndrome, selected for Best of the Liver Meeting. Additionally, a second late-breaker presentation will showcase predictors of long-term survival in patients treated with maralixibat. LIVMARLI is FDA-approved for cholestatic pruritus in Alagille syndrome.

Mirum Pharmaceuticals recently announced significant findings regarding its drug maralixibat, published in The Lancet. The four-year study shows that maralixibat offers durable improvements in cholestasis parameters, including pruritus, for children with Alagille syndrome. The drug demonstrated a robust clinical response with >80% of participants experiencing a reduction in pruritus. Approved by the FDA as LIVMARLI, maralixibat is pivotal for treating this rare liver condition. Mirum aims to further expand its market presence in Europe.

Mirum Pharmaceuticals has received FDA approval for LIVMARLI™ (maralixibat), the first medication for treating cholestatic pruritus in patients with Alagille syndrome aged one year and older. This rare liver disease affects approximately 2,000 to 2,500 children in the U.S. LIVMARLI is an oral solution that targets the underlying cause of debilitating itch associated with this condition. The approval is based on the pivotal ICONIC study showing significant symptom reduction. Mirum also received a rare pediatric disease priority review voucher.

Mirum Pharmaceuticals has entered into an exclusive licensing agreement with Takeda Pharmaceutical Company for the development and commercialization of maralixibat in Japan. This investigational drug targets rare liver diseases such as Alagille syndrome, progressive familial intrahepatic cholestasis, and biliary atresia. Takeda will oversee regulatory approval and conduct clinical studies. Mirum has also submitted a new drug application for maralixibat in the U.S., with a priority review by the FDA expected by September 29, 2021.

Mirum Pharmaceuticals has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for treating cholestatic liver disease in Alagille syndrome (ALGS). This marks a significant advancement as it could introduce the first pharmacologic treatment for this rare disease. The MAA includes positive results from the ICONIC study, indicating marked improvements in survival and liver health metrics. Furthermore, Mirum plans to resubmit its MAA for progressive familial intrahepatic cholestasis (PFIC) after the MARCH-PFIC Phase 3 study results are available.