Alagille syndrome

Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI

Retrieved on: 
Thursday, February 29, 2024

The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.

Key Points: 
  • The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.
  • The GALA control group was identified based on a pre-specified and blinded selection process to align with eligibility criteria from the LIVMARLI clinical studies.
  • Data from the analysis demonstrated a significant improvement in six-year event-free survival with a p-value of
  • The full publication including additional data from the analysis is available on the Hepatology website.

Building on solid FY 2023 results, Ipsen anticipates four launches in 2024

Retrieved on: 
Thursday, February 8, 2024

Ipsen ended the year with net cash of €65.1m, driven by solid free cash-flow generation of €710.9m.

Key Points: 
  • Ipsen ended the year with net cash of €65.1m, driven by solid free cash-flow generation of €710.9m.
  • Ipsen anticipates four commercial launches in 2024, following regulatory decisions for Onivyde in 1L PDAC in the U.S (H1), elafibranor in second-line PBC in the U.S. (H1) and in the E.U.
  • Ipsen has set the following financial guidance for FY 2024, which excludes any impact from potential late-stage external-innovation transactions:
    Total-sales growth greater than 6.0%, at constant currency.
  • The performance shown in this announcement covers the twelve-month period to 31 December 2023 (FY 2023) and the three-month period to 31 December 2023 (Q4 2023), compared to the twelve-month period to 31 December 2022 (FY 2022) and the three-month period to 31 December 2022 (Q4 2022), respectively, unless stated otherwise.

Mirum Pharmaceuticals Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 2, 2023

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for third quarter 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for third quarter 2023 and provided a business update.
  • Third quarter 2023 net sales of $9 million recorded since acquisition close August 31st, 2023.
  • Total net revenue for the quarter ended September 30, 2023, was $47.7 million compared to $18.8 million for the quarter ended September 30, 2022.
  • Total operating expenses were $72.9 million for the quarter ended September 30, 2023, compared to $51.7 million for the quarter ended September 30, 2022.

Ipsen delivers solid sales growth in the first nine months of 2023 and confirms its full-year guidance

Retrieved on: 
Thursday, October 26, 2023

Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.

Key Points: 
  • Our portfolio has performed well across the three therapy areas, driven by strengthened commercial execution and the results of our external-innovation strategy.
  • Based on the solid sales momentum, today we are confirming our guidance for the full year.
  • This was despite a positive opinion from the Committee for Medicinal Products for Human Use in July 2023.
  • The Company is planning to host a capital-markets event, starting at 12.30pm GMT on 7 December 2023 in London.

Ipsen updates on E.U. Marketing Authorisation Application for odevixibat in Alagille syndrome

Retrieved on: 
Monday, October 23, 2023

This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.

Key Points: 
  • This is despite a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in July 2023.
  • Orphan designation has a strong influence on the reimbursement mechanisms and access for patients to medicines in some countries in the E.U.
  • “We are disappointed with the opinion of the COMP as the Orphan Medicinal Product Regulation aims to stimulate research and development for rare diseases.
  • In the trial, there were no patient discontinuations and 96% of patients rolled over into the open-label extension trial.

Mirum Pharmaceuticals Presents LIVMARLI Clinical Data and Real-World Evidence at NASPGHAN

Retrieved on: 
Saturday, October 7, 2023

Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS.

Key Points: 
  • Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS.
  • Presentations included:
    Data from the MARCH PFIC Phase 3 Study was awarded the prestigious Gerard Odell prize for Excellence in Pediatric Hepatology Research.
  • The MARCH PFIC clinical trial was conducted across the broadest range of PFIC types ever studied.
  • Real-world data in patients with ALGS was presented on adherence to LIVMARLI as well as reduction of other medications and vitamin supplementation while on LIVMARLI.

CANbridge Announces Marketing Approval of CAN108 (/LIVMARLI®) in Taiwan for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome (ALGS)

Retrieved on: 
Monday, October 9, 2023

LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older.

Key Points: 
  • LIVMARLI is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, and the first and only treatment approved medication in Taiwan for the treatment of cholestatic pruritus (itching caused by slowed or stalled bile flow) in patients with Alagille syndrome (ALGS) aged one year or older.
  • ALGS is a rare genetic disorder that can lead to end-stage liver disease and death.
  • Cholestatic pruritus is the most burdensome symptom in ALGS, greatly reducing quality of life.
  • The availability of LIVMARLI in Taiwan represents a significant turning point in addressing the unique challenges faced by individuals affected by ALGS.

Mirum Pharmaceuticals to Highlight LIVMARLI Data from its ALGS and PFIC Programs at the NASPGHAN Annual Meeting

Retrieved on: 
Tuesday, September 26, 2023

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI® (maralixibat) oral solution studies as well as data from real- world experience will be presented at the upcoming North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting taking place October 4-7, 2023, in San Diego, California.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI® (maralixibat) oral solution studies as well as data from real- world experience will be presented at the upcoming North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting taking place October 4-7, 2023, in San Diego, California.
  • Highlighted below are the titles that have been accepted for presentation during the meeting.
  • The abstracts are available via the NASPGHAN website .
  • Full analyses will be available following their presentation within the Publications & Presentations section on Mirum’s website .

Mirum Pharmaceuticals Reports Second Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 3, 2023

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.
  • Mirum to pay $210 million upfront and up to $235 million in potential tiered annual net sales-based milestones.
  • Second quarter 2023 global net sales grew 86% as compared to second quarter 2022, on track for 50% US net product sales growth in 2023 as compared to 2022.
  • Total operating expenses were $61.8 million for the quarter ended June 30, 2023 in comparison to $48.9 million for the quarter ended June 30, 2022.

Ipsen delivers solid H1 2023 results and upgrades its full-year guidance

Retrieved on: 
Thursday, July 27, 2023

The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.

Key Points: 
  • The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.
  • The Company’s auditors performed a limited review of the H1 2023 condensed consolidated financial statements.
  • Ipsen intends to publish its year-to-date and third-quarter sales update on 26 October 2023.
  • The performance shown in this announcement covers the six-month period to 30 June 2023 (H1 2023) and the three-month period to 30 June 2023 (Q2 2023), compared to six-month period to 30 June 2022 (H1 2022) and the three-month period to 30 June 2022 (Q2 2022), respectively, unless stated otherwise.