Primary sclerosing cholangitis

Ipsen completes acquisition of Albireo, expanding the scope of its Rare Disease portfolio

Retrieved on: 
Thursday, March 2, 2023
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The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.

Key Points: 
  • The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.
  • “The acquisition of Albireo will greatly strengthen our portfolio in rare diseases,” said David Loew, Chief Executive Officer of Ipsen.
  • “I am excited to welcome new colleagues to Ipsen, who led the innovation on the development of novel bile acid modulators, like Bylvay, to treat rare liver diseases in children and adults.
  • As part of the transaction, Ipsen has also acquired A3907 and A2342, two clinical-stage assets in Albireo’s pipeline.

Chemomab Announces FDA Clearance of Investigational New Drug Application for Phase 2 Clinical Trial of CM-101 in Patients with Systemic Sclerosis

Retrieved on: 
Tuesday, February 21, 2023
NASH, Biomarker, TEL, NAFLD, Scleroderma, Physician, Systemic scleroderma, Blood, Therapy, University, Food and Drug Administration, Non-alcoholic fatty liver disease, Tissue, Lung, Patient, CMMB, Clinical trial, Skin, Rheumatology, Food, Primary sclerosing cholangitis, Investigational New Drug, Pathology, Liver disease, Physiology, CCL24, Fatty liver disease, Disease, FDA, Orphan drug, PSC, Principal, Medical research, Doctor of Philosophy, IND, MD, Pharmaceutical industry, COVID

TEL AVIV, Israel, Feb. 21, 2023 /PRNewswire/ -- Chemomab Therapeutics, Ltd. (Nasdaq: CMMB) (Chemomab), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibro-inflammatory diseases with high unmet need, today announced U.S. Food and Drug Administration (FDA) clearance of the company's Investigational New Drug (IND) Application to evaluate CM-101 in a Phase 2 trial in adults with systemic sclerosis (SSc).

Key Points: 
  • In preclinical studies, CM-101 reduced inflammatory and fibrotic injury to the lung, skin and vasculature—organ systems often affected in SSc patients.
  • CM-101 has been granted Orphan Drug designation by the FDA for SSc and for primary sclerosing cholangitis (PSC), a rare liver disease.
  • Matt Frankel, MD, Chief Medical Officer of Chemomab, said, "Achieving FDA clearance to initiate our Phase 2 systemic sclerosis trial is an important milestone for Chemomab.
  • I welcome the opportunity to help launch and coordinate this innovative clinical trial for patients with SSc in the U.S., Europe and Israel, scheduled to open later this year."

WILLOW ANNOUNCES FOLLOW-ON ENGAGEMENT FOR NEXT PHASE OF PARTNERED PHARMACEUTICAL PROJECT

Retrieved on: 
Tuesday, January 17, 2023
API, WLLW, Sandhill, TUDCA, Active ingredient, Amyotrophic lateral sclerosis, Cannabinoid, TSX, Tauroursodeoxycholic acid, Organic acid, UDCA, Primary sclerosing cholangitis, Willow, Growth, Research, Ursodeoxycholic acid, ALS, Fine chemical

This represents the continuation of the project, the first phase of which the Company announced May 31, 2022.

Key Points: 
  • This represents the continuation of the project, the first phase of which the Company announced May 31, 2022.
  • Sandhill is partnered with a large-cap, multi-national healthcare company and the targeted product is manufacture of the large volume API, ursodeoxycholic acid (UDCA).
  • In this next phase of development, the Company anticipates several hundred thousand U.S. dollars in research fees, along with a milestone payment later this year for initial production batches.
  • Upon successful completion, it is expected that Willow's proprietary, optimized enzyme will be taken toward commercialization for the end market active pharmaceutical ingredients ("APIs"), where Willow will receive additional milestone and production-based royalty payments.

Mirum Pharmaceuticals Announces Preliminary Unaudited LIVMARLI 2022 Net Revenue and Provides Corporate Updates

Retrieved on: 
Monday, January 9, 2023
Biotechnology, Other Health, Health, Pharmaceutical, Clinical Trials, Supplement, CFO, FDA, Medication, Cholestasis, Conference, Alagille syndrome, Growth, Biliary atresia, Patient, Primary biliary cholangitis, IBAT, Spiroplasma mirum, Cholestatic pruritus, Investment, Disclosure, Primary sclerosing cholangitis, Pharmaceutical industry, Food delivery, PFIC

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided a preview of the company’s plans, estimated revenue and net product sales for Q4 and full year 2022 ahead of its presentation at the 41st Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided a preview of the company’s plans, estimated revenue and net product sales for Q4 and full year 2022 ahead of its presentation at the 41st Annual J.P. Morgan Healthcare Conference.
  • “2022 was a year of strong commercial performance and continued pipeline execution.
  • The foregoing amounts relating to 2022 financial data are unaudited and preliminary and are subject to completion of financial closing procedures.
  • View the Investors and Media section of Mirum’s corporate website for webcast links and additional information.

Ipsen to Acquire Albireo Accelerating Growth in Rare Disease With Treatments for Several Pediatric Liver Diseases

Retrieved on: 
Monday, January 9, 2023
Banking, Research, General Health, Professional Services, Pharmaceutical, Oncology, Clinical Trials, Science, Biotechnology, FDA, Finance, Health, Other Science, BOLD, Itch, Ageing, Primary sclerosing cholangitis, Institute of Liver and Biliary Sciences, Cirrhosis, Food, PSC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hope, Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, Science, Liver failure, Acquisition, Partner, Patient, IPN, Alagille syndrome, Liver disease, Hart–Scott–Rodino Antitrust Improvements Act, Albireo, Liver transplantation, Biliary atresia, Bile, CVR, Paul, Weiss, Rifkind, Wharton & Garrison, FDA, Webcast, Genetic disorder, ADR, Goldman Sachs, IND, Apache iBATIS, Rare disease, Centerview Partners, P.C, Phase, NTCP, Liver, Medical imaging, Pharmaceutical industry, Ipsen, PFIC, Euronext

Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases.

Key Points: 
  • Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases.
  • “Our Rare Disease franchise is strengthened with Bylvay, which, in addition to being the first-approved treatment in PFIC, has two further indications being investigated in rare liver conditions that are underserved.
  • “Our talented team at Albireo have advanced the first Phase III studies in three different pediatric liver diseases while discovering two promising new clinical stage bile acid modulators.
  • Goldman Sachs is acting as exclusive financial advisor to Ipsen and Orrick Herrington & Sutcliffe LLP as legal counsel to Ipsen.

HighTide Therapeutics Raises $107 Million in Series C/C+ Financing to Advance Innovative Pipeline and Business Collaborations

Retrieved on: 
Thursday, January 5, 2023
T2DM, Food, Venture round, PSC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Primary sclerosing cholangitis, NASH, Trial of the century, Series, HighTide, Patient, Fatty liver disease, FDA, Pharmaceutical industry, MD

Proceeds of the financing will be used to advance multiple global development programs, including mid-to-late-stage clinical trials, and the commercialization and business development of the company's robust pipeline.

Key Points: 
  • Proceeds of the financing will be used to advance multiple global development programs, including mid-to-late-stage clinical trials, and the commercialization and business development of the company's robust pipeline.
  • "After completing a successful $60 million Series B+ round at the end of 2020, we are thrilled to have well-recognized investors participate in our C/C+ round.
  • "The Series C/C+ financing is a significant milestone for HighTide.
  • It will enable us to move aggressively to accelerate the clinical and commercial development of our innovative pipeline and external business collaborations."

CymaBay Therapeutics Announces Collaboration with Kaken Pharmaceutical Co., Ltd. to Develop and Commercialize Seladelpar in Japan for Primary Biliary Cholangitis

Retrieved on: 
Sunday, January 8, 2023
Primary sclerosing cholangitis, PBC, Primary biliary cholangitis, Therapy, Patient, Pharmaceutical industry, Cycloheximide

NEWARK, Calif. and TOKYO, Jan. 08, 2023 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY) (“CymaBay” or the “Company”) announced today that it has entered into a collaboration and license agreement with Kaken Pharmaceutical Co., Ltd. (“Kaken”) for the development and commercialization in Japan of CymaBay’s investigational drug seladelpar for the treatment of primary biliary cholangitis (“PBC”).

Key Points: 
  • Under the terms of the agreement, Kaken receives an exclusive license to develop, commercialize and market seladelpar in Japan for PBC.
  • Kaken will be responsible for development, regulatory approval and commercialization of seladelpar in Japan.
  • “Kaken is a well-regarded leader in development and commercialization in Japan with a proud history of scientific and medical innovation.
  • “With this collaboration, we hope we can help improve the lives of patients with cholestatic liver disease in Japan.”

Chemomab Reports Top-Line Results from CM-101 Phase 2a Liver Fibrosis Biomarker Trial in NASH Patients

Retrieved on: 
Tuesday, January 3, 2023
Medical school, F2, Fatty liver disease, Fibrosis, Patient, CCL24, PSC, Communication, ELF, Optimism, NAFLD, Primary sclerosing cholangitis, Systemic scleroderma, Institute of Liver and Biliary Sciences, Sheila Sherlock, Cirrhosis, SC, University College London, Marketing, Division, Nature, Faculty, Safety, Inflammation, Clinical trial, CMMB, UCL, Scleroderma, Icahn School of Medicine at Mount Sinai, Intracellular digestion, Therapy, Principal, TEL, Gastroenterology, F3, Proning, Doctor of Philosophy, Risk, Pharmacokinetics, Non-alcoholic fatty liver disease, Liver, University College London Hospitals NHS Foundation Trust, Research, NASH, Biomarker, Hepatology, Medical imaging, Pharmaceutical industry, Vaccine, Medicine, COVID, MD

TEL AVIV, Israel, Jan. 3, 2023 /PRNewswire/ -- Chemomab Therapeutics, Ltd. (Nasdaq: CMMB) (Chemomab), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, today reported top-line results from its Phase 2a trial assessing CM-101, its first-in-class CCL24-neutralizing monoclonal antibody, in non-alcoholic steatohepatitis (NASH) patients. The trial met its primary endpoint of safety and tolerability, and CM-101 achieved reductions in secondary endpoints that include a range of liver fibrosis biomarkers and physiologic assessments measured at baseline and at week 20.

Key Points: 
  • The randomized, placebo-controlled trial enrolled 23 NASH patients with stage F1c, F2 and F3 disease who were randomized to receive either CM-101 or placebo.
  • Dr. Pfost continued, "This is the third clinical trial in patients demonstrating the activity of CM-101 as measured by fibro-inflammatory biomarkers and physiological assessments.
  • A Phase 2 liver fibrosis biomarker study in NASH patients was recently completed and a Phase 2 trial in primary sclerosing cholangitis patients is ongoing.
  • Chemomab expects to begin enrolling patients in a Phase 2 trial in systemic sclerosis early in 2023.

Chemomab Reports Independent Drug Monitoring Committee Safety Review of CM-101 Phase 2 Trial in Primary Sclerosing Cholangitis

Retrieved on: 
Wednesday, December 21, 2022
CCL24, Fibrosis, Patient, PSC, Communication, Primary sclerosing cholangitis, Cirrhosis, Marketing, Nature, Safety, Inflammation, MD, CMMB, Therapy, Data monitoring committee, TEL, Risk, Research, NASH, Biomarker, DMC, Pharmaceutical industry

TEL AVIV, Israel, Dec. 21, 2022 /PRNewswire/ -- Chemomab Therapeutics, Ltd. (Nasdaq: CMMB) (Chemomab), a clinical-stage biotech company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, today announced that an independent Data Monitoring Committee (DMC) has completed a safety review of the company's ongoing Phase 2 trial of lead product CM-101 in primary sclerosing cholangitis (PSC) patients. The DMC had no safety concerns with proceeding with adding a planned 20 milligram per kilogram (mg/kg) dosing cohort to the PSC trial, as defined in a recent protocol amendment. The PSC trial currently has a single 10mg/kg dose, administered every three weeks by intravenous (IV) infusion.

Key Points: 
  • The DMC review was based on both blinded safety data from the PSC trial and from Chemomab's Phase 2a liver fibrosis biomarker trial in patients with non-alcoholic steatohepatitis (NASH).
  • Chemomab plans to report top-line results from this latter trial in the coming weeks.
  • The CM-101 Phase 2 SPRING trial is a randomized, placebo-controlled, multiple dose study enrolling PSC patients with large duct disease.
  • It is currently in Phase 2 trials for primary sclerosing cholangitis and liver fibrosis, with a Phase 2 trial in systemic sclerosis expected to begin enrolling patients in early 2023.

Pliant Therapeutics Receives Orphan Designation from the European Medicines Agency for Bexotegrast (PLN-74809) for the Treatment of Idiopathic Pulmonary Fibrosis

Retrieved on: 
Thursday, December 15, 2022
European Medicines Agency, PSC, Muscular dystrophy, EMA, Result, Patient, Annual report, SAN, Partnership, Cirrhosis, Food, FDA, Conversation, News, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NASH, IPF, Financial condition report, Union, Risk, Analysis, Primary sclerosing cholangitis, Orphan, Fibrosis, Walking, HIV disease progression rates, Therapy, Fatty liver disease, COVID-19, Idiopathic pulmonary fibrosis, Integrin, Pharmaceutical industry, EU, Novartis

“Following the positive interim results from our Phase 2a trial of bexotegrast, we are pleased to receive the EMA's orphan medicinal product designation,” said Éric Lefebvre, M.D., Chief Medical Officer at Pliant Therapeutics.

Key Points: 
  • “Following the positive interim results from our Phase 2a trial of bexotegrast, we are pleased to receive the EMA's orphan medicinal product designation,” said Éric Lefebvre, M.D., Chief Medical Officer at Pliant Therapeutics.
  • Bexotegrast received Orphan Drug Designation from the United States Food and Drug Administration (FDA) in 2018.
  • Pliant Therapeutics is a clinical stage biopharmaceutical company focused on discovering and developing novel therapies for the treatment of fibrosis.
  • Bexotegrast (PLN-74809) has received Fast Track Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in IPF and PSC and Orphan Drug Designation from the European Medicines Agency in IPF and PSC.