Maralixibat

Mirum Pharmaceuticals and CANbridge Pharmaceuticals Enter into Exclusive Licensing Agreement to Develop and Commercialize Maralixibat in Greater China for Rare Liver Diseases

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星期四, 四月 29, 2021
Biotechnology, FDA, Health, Pharmaceutical, Clinical trials, Mirum Pharmaceuticals, Inc., CANbridge Pharmaceuticals Inc., Maralixibat, MIRUM PHARMACEUTICALS, INC., CANBRIDGE PHARMACEUTICALS INC., MARALIXIBAT

b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan).

Key Points: 
  • b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and CANbridge Pharmaceuticals, Inc., announced today that they have entered into a licensing agreement, pursuant to which CANbridge has agreed to develop and commercialize maralixibat in Greater China (China, Hong Kong, Macau and Taiwan).
  • Mirum has commenced a global Phase 2b maralixibat study (EMBARK) for the treatment of BA.\nUnder the terms of the licensing agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions for ALGS, PFIC, and BA.
  • Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS.
  • CANbridge also has entered into a strategic collaboration and licensing agreement with LogicBio Therapeutics to develop, manufacture and commercialize gene therapy candidates for treatments for Fabry and Pompe diseases.

Mirum Pharmaceuticals Receives Notice of Allowance for Methods of Use Patent for Maralixibat in Alagille Syndrome

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星期一, 四月 26, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Digestive diseases, Hepatology, Clinical medicine, Alagille syndrome, Progressive familial intrahepatic cholestasis, PFIC, Primary biliary cholangitis, Cholestasis, Liver, Patent and Trademark Office, Cholestasis facies, Maralixibat, Mirum Pharmaceuticals, Inc., MARALIXIBAT, MIRUM PHARMACEUTICALS, INC.

b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No.

Key Points: 
  • b'Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application No.
  • The allowed patent application covers methods of treating Alagille syndrome using maralixibat, an investigational treatment being evaluated for patients with Alagille syndrome, a rare and life-threatening cholestatic liver disease.
  • The majority of patent applications that receive a notice of allowance will proceed to issue as a U.S. patent.
  • More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Mirum Pharmaceuticals Announces FDA Acceptance of New Drug Application and Priority Review for Maralixibat in Alagille Syndrome

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星期一, 三月 29, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Digestive diseases, Hepatology, Clinical medicine, Alagille syndrome, Progressive familial intrahepatic cholestasis, Prescription Drug User Fee Act, PFIC, Biliary atresia, Priority review, Cholestasis, Food and Drug Administration, Alagille syndrome, Maralixibat, Mirum Pharmaceuticals, Inc.

Mirum completed the rolling NDA submission in January 2021 and the Prescription Drug User Fee Act (PDUFA) date, or FDA decision date, is September 29, 2021.

Key Points: 
  • Mirum completed the rolling NDA submission in January 2021 and the Prescription Drug User Fee Act (PDUFA) date, or FDA decision date, is September 29, 2021.
  • We are extremely pleased that our NDA will move forward in the regulatory review process, bringing maralixibat one step closer to being available for patients with Alagille syndrome, said Chris Peetz, president and chief executive officer at Mirum.
  • Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia.
  • The NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021.

Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2020 Results and Provides Business Updates

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星期二, 三月 9, 2021
Oncology, Health, Other Health, General Health, Pharmaceutical, Biotechnology, Maralixibat, Mirum Pharmaceuticals

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced financial results for the fourth quarter and year-end 2020 and provided business updates.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced financial results for the fourth quarter and year-end 2020 and provided business updates.
  • Total operating expenses for the quarter ended December 31, 2020 were $37.0 million, compared to $18.7 million for the fourth quarter of 2019.
  • Research and development expenses for the fourth quarter were $29.7 million, compared to $14.4 million for the comparable prior-year period.
  • General and administrative expenses for the fourth quarter of 2020 were $7.2 million, compared to $4.3 million for the comparable prior-year period.

Mirum Pharmaceuticals Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome

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星期一, 二月 1, 2021
Biotechnology, Pharmaceutical, Health, Medical specialties, Digestive diseases, Hepatology, Clinical medicine, Alagille syndrome, Progressive familial intrahepatic cholestasis, Cholestatic pruritus, Cholestasis, PFIC, Biliary atresia, Liver, Alagille syndrome, Maralixibat, Mirum

The NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year follow-up.

Key Points: 
  • The NDA submission included the results of the ICONIC study and additional supportive studies totaling 86 patients with ALGS with up to six-year follow-up.
  • Until maralixibat is approved by the FDA for prescription, maralixibat is available to eligible patients with ALGS through Mirums Expanded Access Program (EAP).
  • Mirums lead product candidate, maralixibat, is an investigational oral drug in development for Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia.
  • Mirum has submitted an NDA for maralixibat in the treatment of cholestatic pruritus in patients with ALGS.

Mirum Pharmaceuticals and Oberland Capital Announce Up to $210 Million Funding Arrangement

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星期三, 十二月 9, 2020
Biotechnology, Finance, Health, Professional services, Pharmaceutical, Oberland, Maralixibat, Mirum, Oberland Capital

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Oberland Capital Management LLC (Oberland Capital) today announced a $200 million capped, tiered, revenue-based funding agreement based on net revenues of maralixibat, as well as a $10 million equity investment agreement.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Oberland Capital Management LLC (Oberland Capital) today announced a $200 million capped, tiered, revenue-based funding agreement based on net revenues of maralixibat, as well as a $10 million equity investment agreement.
  • The funding from Oberland Capital will enable us to invest in the launch and commercialization of maralixibat in the United States and Europe, said Ian Clements, PhD, chief financial officer at Mirum.
  • The agreement with Oberland Capital provides us the support and flexibility to advance our pipeline focused on rare liver diseases for children and adults, an area where there is tremendous need.
  • We are pleased to support Mirum as they seek to commercialize new medicines for diseases with high unmet need, said Andrew Rubinstein, managing partner of Oberland Capital.

Mirum Pharmaceuticals Announces European Medicines Agency Validation of the Marketing Authorization Application for Maralixibat in Patients with PFIC2

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星期一, 十一月 30, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Hepatology, Maralixibat, Mirum Pharmaceuticals

We are excited about the opportunity to make maralixibat available to patients with PFIC2 in Europe.

Key Points: 
  • We are excited about the opportunity to make maralixibat available to patients with PFIC2 in Europe.
  • Data from the Phase 2 INDIGO study evaluating maralixibat for pediatric patients with PFIC2 served as the basis of the MAA submission.
  • Mirum recently announced data showing five-year transplant-free survival for patients who achieved serum bile acid control.
  • Additionally, Mirums marketing authorization application for the treatment of pediatric patients with PFIC2 has been accepted for review (validated) by the European Medicines Agency.

Mirum Pharmaceuticals Announces Data Presented During AASLD Highlighting Durable Improvements in Pruritus and Quality of Life in Children with Alagille Syndrome Treated with Maralixibat

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星期日, 十一月 15, 2020
Health, Consumer, Infectious diseases, Children, Pharmaceutical, Biotechnology, Medical specialties, Hepatology, Branches of biology, Clinical medicine, Digestive system, Bile acid, Enterohepatic circulation, Cholesterol, Bile, Ileal sodium/bile acid cotransporter, Bile acid sequestrant, the ITCH and IMAGINE II study, Alagille Syndrome, Maralixibat, Mirum Pharmaceuticals

Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus .

Key Points: 
  • Maralixibat, an apical sodium bile acid transporter (ASBT) inhibitor, has previously been shown to interrupt the enterohepatic circulation of bile acids, reducing pruritus .
  • Maralixibat treatment improved quality of life and led to improved growth parameters.
  • To view the presentation and the complete data, please visit the AASLD section within the Events page on Mirums website.
  • Children receiving maralixibat in the studies also demonstrated improvements in biomarkers of disease, including reductions in cholesterol and bile acid levels.

Mirum Pharmaceuticals Presents New Data From Its Maralixibat and Volixibat Clinical Studies at AASLD Annual Meeting

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星期五, 十一月 13, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Medical specialties, Hepatology, Bile acid, Primary biliary cholangitis, Liver disease, American Association for the Study of Liver Diseases, Cholestasis, Liver, Maralixibat, Volixibat, Mirum Pharmaceuticals

To view the data in full, please visit the AASLD section within the Events page on our corporate website.

Key Points: 
  • To view the data in full, please visit the AASLD section within the Events page on our corporate website.
  • Overall, the positive treatment effects of maralixibat in patients with ALGS demonstrate important correlations with multiple clinically relevant parameters.
  • The effects on bile acid trafficking and synthesis support the further study of volixibat in patients with cholestatic liver disease.
  • Such forward-looking statements include statements regarding, among other things, the results, conduct and progress of Mirums ongoing and planned studies for maralixibat and volixibat, and the regulatory approval path for maralixibat and volixibat.

Mirum Pharmaceuticals Provides Third Quarter 2020 Financial Results and Business Update, and Announces Virtual Investor Day

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星期四, 十一月 12, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Asset, Cash, Cash and cash equivalents, Food and Drug Administration, Articles, Maralixibat, Mirum Pharmaceuticals

Received Orphan Drug Designation from the U.S. FDA for maralixibat in biliary atresia; Phase 2 study initiation planned for the first quarter of 2021.

Key Points: 
  • Received Orphan Drug Designation from the U.S. FDA for maralixibat in biliary atresia; Phase 2 study initiation planned for the first quarter of 2021.
  • Total operating expenses for the quarter ended September 30, 2020 were $21.7 million, compared to $15.9 million for the third quarter of 2019.
  • This increase was primarily due to increased personnel related expenses, manufacturing activities to support Mirums NDA, and higher consulting expenses.
  • As of September 30, 2020, Mirum had cash, cash equivalents and investments of $133.7 million.