Primary biliary cholangitis

GENFIT Reports Full-Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, April 4, 2024
MAA, ACLF, Primary biliary cholangitis, Patient, Research, Ipsen, Placebo, EMA, Bank statement, Marketing, PBC, FDA, EF, Pharmaceutical industry

“A remarkable milestone was reached by GENFIT in 2023 with the announcement of positive topline data for our Phase 3 ELATIVE® trial evaluating elafibranor in Primary Biliary Cholangitis.

Key Points: 
  • “A remarkable milestone was reached by GENFIT in 2023 with the announcement of positive topline data for our Phase 3 ELATIVE® trial evaluating elafibranor in Primary Biliary Cholangitis.
  • This was followed by another asset acquisition in July 2023, where GENFIT licensed the exclusive worldwide rights of CLM-022, a potential first-in-class inflammasome inhibitor, from Celloram.
  • IND was in effect as of April 17, 2023 and the first patient was randomized in the UNVEIL-IT® Phase 2 trial in July 2023.
  • GENFIT equally obtained a “Prime status” label by ISS ESG, upgrading its corporate rating from C to C+.

Gilead Sciences Announces Completion of Acquisition of CymaBay

Retrieved on: 
Friday, March 22, 2024
Health, Hospitals, General Health, Research, Science, Pharmaceutical, Biotechnology, EPS, Patient, Gilead Sciences, Acquisition, Primary biliary cholangitis, PBC, Therapy, Safety, Itch, Pharmaceutical industry

Gilead Sciences, Inc. (Nasdaq: GILD) today announced the completion of the previously announced transaction to acquire CymaBay Therapeutics, Inc. (Nasdaq: CBAY) for approximately $4.3 billion in total equity value.

Key Points: 
  • Gilead Sciences, Inc. (Nasdaq: GILD) today announced the completion of the previously announced transaction to acquire CymaBay Therapeutics, Inc. (Nasdaq: CBAY) for approximately $4.3 billion in total equity value.
  • “The acquisition of CymaBay brings us a potential best in disease therapy that could transform the treatment landscape for people with primary biliary cholangitis,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences.
  • “I want to thank the CymaBay team for their efforts and commitment to addressing this high unmet need.
  • Pursuant to the merger agreement, Gilead and Purchaser commenced a tender offer on February 23, 2024, to acquire all outstanding shares of CymaBay at a price of $32.50 per share.

CymaBay Announces European Medicines Agency Accepts for Review the Marketing Authorization Application for Seladelpar for the Treatment of Primary Biliary Cholangitis

Retrieved on: 
Monday, March 4, 2024
Liver, Child, Marketing, EMA, Ursodeoxycholic acid, MAA, Chemistry, Cirrhosis, Inflammation, FDA, Quality of life, Food, Chief, Conditional sentence, PBC, Risk, European, Health care, Therapy, Fatigue, CHMP, European Medicines Agency, Primary biliary cholangitis, Patient, Itch

Seladelpar is an investigational, potent, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist or delpar, in development for PBC treatment.

Key Points: 
  • Seladelpar is an investigational, potent, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist or delpar, in development for PBC treatment.
  • The study’s primary endpoint measure reflects registrational studies of the current second line PBC treatment authorized by the EMA.
  • In this study seladelpar demonstrated statistically significant improvements in biochemical markers of disease progression and PBC-related pruritus (itch) pre-specified endpoints.
  • “Validation of the European Marketing Authorization Application for seladelpar and the start of the agency’s formal review is an important step forward in seladelpar’s journey to potential approval in Europe.

Intercept Announces FDA Acceptance of Supplemental New Drug Application for Ocaliva® (obeticholic acid) for the Treatment of PBC

Retrieved on: 
Thursday, February 29, 2024
Therapy, Senior, Ursodeoxycholic acid, Pamlab, New Drug Application, FDA, Food, Shanda, PDUFA, Regulatory affairs, Database, PBC, Communication, Hepatology, Cirrhosis, Intercept, Primary biliary cholangitis, Patient, UDCA, Obeticholic acid, Intercept Pharmaceuticals, Advisory Committee, Pharmaceutical industry

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024.

Key Points: 
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 15, 2024.
  • In this communication, the agency informed Intercept that they are planning to hold an Advisory Committee meeting to discuss the application.
  • The precedent-setting sNDA for Ocaliva is intended to satisfy the post-marketing requirements to confirm a clinical benefit in patients with PBC.
  • Intercept and Alfasigma remain committed to supporting people living with PBC and look forward to advancing discussions with the agency.

GENFIT Announces Revenues and Cash Position as of December 31, 2023

Retrieved on: 
Thursday, February 29, 2024
Marketing, EMA, New Drug Application, MAA, Annual report, FDA, Food, Part, PDUFA, NTZ, PBC, Primary biliary cholangitis, Collaboration, European Medicines Agency, Patient, Elafibranor, NDA, Research, Priority review, Pharmaceutical industry

As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.

Key Points: 
  • As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.
  • As of September 30, 2023, cash and cash equivalents totaled €93.9 million.
  • Revenues for 2023 amounted to €28.6 million compared to €20.2 million for the same period in 2022.
  • The 2023 Universal Registration Document, the 2023 Annual Financial Report (included in the 2023 Universal Registration Document), and the Annual Report on Form 20-F will be published by the end of April 2024.

Budesonide in Primary Biliary Cholangitis Research Report 2024: Comprehensive Market Insights to 2032 with Focus on 7 Major Markets - ResearchAndMarkets.com

Retrieved on: 
Tuesday, March 5, 2024
Biotechnology, Pharmaceutical, Health, Outline, Ursodeoxycholic acid, Chronic liver disease, Budesonide, Strategic planning, Drug development, SWOT, Primary, PBC, Primary biliary cholangitis, Patient, UDCA, Research, Pharmaceutical industry

The healthcare industry witnesses a significant milestone as the Budesonide Market Size, Forecast, and Drug Insight - 2032 report is introduced to the research inventory.

Key Points: 
  • The healthcare industry witnesses a significant milestone as the Budesonide Market Size, Forecast, and Drug Insight - 2032 report is introduced to the research inventory.
  • This latest analytical document delineates the progressive outlook of budesonide in treating primary biliary cholangitis (PBC), providing a detailed market forecast and insights into drug development and innovation within the pharmaceutical arena through to 2032.
  • Primary biliary cholangitis, a chronic liver disease, presents a therapeutic challenge, compelling industry stakeholders to eagerly explore new treatment avenues.
  • With a thorough research methodology encompassing secondary and primary data, industry expertise, and comprehensive market analysis, the report equips stakeholders with pivotal insights.

Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, February 28, 2024
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, International, New Drug Application, FDA, Food, CTX, Growth, Primary sclerosing cholangitis, PDUFA, PFIC, Event, Food and Drug Administration, Primary biliary cholangitis, Webcast, Patient, Medication, NDA, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2023 and provided a business update.
  • 2023 LIVMARLI net product sales totaled $141.8 million, representing approximately 89% growth over 2022 net product sales.
  • As of December 31, 2023, Mirum had cash and cash equivalents of $286.3 million.
  • The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing.

Ironwood Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Results; Achieves 2023 Financial Guidance

Retrieved on: 
Thursday, February 15, 2024
Managed Care, Biotechnology, Pharmaceutical, Health, Acquisition, Liver, Autoimmune disease, Income tax, Hepatology, FDA, Food, Endometriosis, PBC, Interest expense, Constipation, Annual general meeting, Short bowel syndrome, Safety, Society, GAAP, The Lancet, UEG, Pharmacokinetics, Food and Drug Administration, Primary biliary cholangitis, Interest, Patient, Disease, STARS, Phase, Interstitial cystitis, Trial of the century, Ageing, United European Gastroenterology, CIC, Research, SBS, Depreciation, Video game

Total revenues in the fourth quarter of 2023 were $117.6 million, compared to $107.2 million in the fourth quarter of 2022.

Key Points: 
  • Total revenues in the fourth quarter of 2023 were $117.6 million, compared to $107.2 million in the fourth quarter of 2022.
  • Operating expenses in the fourth quarter of 2023 were $80.0 million, compared to $38.8 million in the fourth quarter of 2022.
  • Adjusted EBITDA was $39.9 million in the fourth quarter of 2023, compared to $68.7 million in the fourth quarter of 2022.
  • Ironwood generated $35.8 million in cash from operations in the fourth quarter of 2023, compared to $79.2 million in cash from operations in the fourth quarter of 2022.

Gilead Sciences Expands Liver Portfolio With Acquisition of CymaBay Therapeutics

Retrieved on: 
Monday, February 12, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Hospitals, Clinical Trials, Alkaline phosphatase, EMA, Woman, Gilead Sciences, Multimedia, Liver disease, Quality of life, Ascending cholangitis, PBC, Risk, Hepatology, Cirrhosis, Seladelpar, Therapy, Fatigue, PRIME, Primary biliary cholangitis, Patient, BofA Securities, Guggenheim Partners, Itch, Centerview Partners, Lazard, Pharmaceutical industry

Gilead Sciences, Inc. (Nasdaq: GILD) and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion.

Key Points: 
  • Gilead Sciences, Inc. (Nasdaq: GILD) and CymaBay Therapeutics, Inc. (Nasdaq: CBAY) announced today a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3 billion.
  • View the full release here: https://www.businesswire.com/news/home/20240211034242/en/
    “We are looking forward to advancing seladelpar by leveraging Gilead’s long-standing expertise in treating and curing liver diseases,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences.
  • Seladelpar is an investigational, oral, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, shown to regulate critical metabolic and liver disease pathways.
  • “Today’s agreement with Gilead is the culmination of years of focus and determination at CymaBay to advance seladelpar and bring new hope to people living with PBC and their families,” said Sujal Shah, President, and CEO at CymaBay Therapeutics.

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

Retrieved on: 
Thursday, February 8, 2024
GLT, Growth, Certification, Prostate cancer, Palovarotene, Statute, Alagille syndrome, Primary biliary cholangitis, PDAC, Patient, FDA, FOP, Cabozantinib, Food, PBC, Botulinum toxin, Irinotecan, Ipsen, Phase, Acquisition, Environment, Albireo, Bank statement, Webcast, Carbon accounting, Core, Woman, Pharmaceutical industry

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.