Primary sclerosing cholangitis

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

Retrieved on: 
Monday, January 9, 2023

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

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

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

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

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

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

“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.

Invea Therapeutics Appoints Mr. Jason Fenton to Its Board of Directors

Retrieved on: 
Tuesday, December 6, 2022

GUILFORD, Conn., Dec. 06, 2022 (GLOBE NEWSWIRE) -- Invea Therapeutics, Inc. (Invea), a biotechnology company developing an artificial intelligence-powered pipeline of precision therapeutics for highly debilitating gastrointestinal (GI) and hepatobiliary disorders, today announced the appointment of Mr. Jason Fenton to its Board of Directors. Mr. Fenton is a widely recognized healthcare-focused capital markets executive who brings over 20 years of experience.

Key Points: 
  • - Company also primed to advance additional programs, including a pan-inflammasome inhibitor
    GUILFORD, Conn., Dec. 06, 2022 (GLOBE NEWSWIRE) -- Invea Therapeutics, Inc. (Invea), a biotechnology company developing an artificial intelligence-powered pipeline of precision therapeutics for highly debilitating gastrointestinal (GI) and hepatobiliary disorders, today announced the appointment of Mr. Jason Fenton to its Board of Directors.
  • “We are honored to have a recognized advisor join our board of directors.
  • Jason has an outstanding depth and breadth of knowledge in the biotech and life sciences sectors and an unparalleled perspective on company building and capital formation,” said Krishnan Nandabalan, Ph.D., Chairman, President, and CEO, Invea Therapeutics.
  • Invea Therapeutics, Inc., or Invea, is a clinical-stage biotechnology company developing an artificial intelligence (AI) powered pipeline of precision therapeutics for highly debilitating gastrointestinal (GI) and hepatobiliary disorders.

Albireo Appoints Paul Streck, M.D., as Chief Medical Officer & Craig Hopkinson, M.D., to Board of Directors

Retrieved on: 
Tuesday, December 6, 2022

BOSTON, Dec. 06, 2022 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators to treat pediatric and adult liver diseases, today announced the appointments of two scientific leaders, Drs. Paul Streck and Craig Hopkinson, to the Enterprise Leadership Team and Board of Directors, respectively. These appointments coincide with an important time for Albireo as the Company continues to deliver on clinical, regulatory, and commercialization milestones for Bylvay® (odevixibat) for indications potentially across three pediatric cholestatic liver diseases while advancing investigational compounds for adult liver and viral diseases.

Key Points: 
  • Paul Streck and Craig Hopkinson, to the Enterprise Leadership Team and Board of Directors, respectively.
  • Paul and Craig bring a depth and breadth of industry, academic and corporate experience to help take our Company to a new level,” said Ron Cooper, President and Chief Executive Officer of Albireo.
  • Most recently, Dr. Streck served as Chief Medical Officer at Arena Pharmaceuticals, a publicly traded biotechnology company acquired by Pfizer in March 2022.
  • Before that, Dr. Streck served as Chief Medical Officer at Alder Biopharmaceuticals, acquired by Lundbeck Pharmaceuticals in October 2020, and as the Chief Medical Officer of Insmed, advancing the company from clinical to commercial stage.

Albireo Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Tuesday, December 6, 2022

The restricted stock units were granted as an inducement material to the employee’s acceptance of employment with Albireo in accordance with Nasdaq Listing Rule 5635(c)(4).

Key Points: 
  • The restricted stock units were granted as an inducement material to the employee’s acceptance of employment with Albireo in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The restricted stock units vest over a four-year period, subject to the employee’s continued service with Albireo through the applicable vesting dates.
  • The restricted stock units are subject to the terms and conditions of Albireo’s 2020 Inducement Equity Incentive Plan.
  • Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat pediatric and adult liver diseases.