Primary sclerosing cholangitis

ProQR Announces First Quarter 2023 Operating and Financial Results

Retrieved on: 
Tuesday, May 16, 2023
ProQR, OPT, Partnership, NTCP, ADAR, University of Massachusetts, Primary sclerosing cholangitis, GRC, Genetics, Bank statement, Cardiovascular disease, European Patent Office, Medication, Virtual, Biliary atresia, Research, Biomarker, RNA, LOF, EPO, Congress, Publishing, Lilly, Patent, Cholestasis, RNA therapeutics, Gene, European Patent Convention, Liver, NHP, R, Therapy, PSC, Conference, B4GALT1, Disease, Oligonucleotide, Mobile phone, Pharmaceutical industry, Dietary supplement, Airline

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the first quarter ended March 31, 2023, and provided a business update.
  • Research and development (R&D) costs were €6.1 million for the quarter ended March 31, 2023 compared to €13.4 million for the same period last year.
  • General and administrative costs were €4.0 million for the quarter ended March 31, 2023 compared to €4.9 million for the same period last year.
  • For further financial information for the period ended March 31, 2023, please refer to the financial statements appearing at the end of this release, or the Q1 financial report filing.

Pliant Therapeutics Provides Corporate Update and Reports First Quarter 2023 Financial Results

Retrieved on: 
Tuesday, May 9, 2023
Exercise, Initiation, Therapy, Duchenne muscular dystrophy, TGF, ALP, Pharmacokinetics, FVC, Biomarker, Duchenne, QLF, ELF, Alkaline phosphatase, Fibrosis, Plasma, Vital capacity, DMD, Novartis, Investment, Patient, Integrin, Research, IPF, PSC, IND, Tumor microenvironment, Idiopathic pulmonary fibrosis, Primary sclerosing cholangitis, Cirrhosis, Safety, Security (finance), Pharmaceutical industry, Medical imaging

SOUTH SAN FRANCISCO, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, today provided a corporate update and reported first quarter 2023 financial results.

Key Points: 
  • $577 million in cash, cash equivalents and short-term investments at end of first quarter 2023,
    SOUTH SAN FRANCISCO, Calif., May 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, today provided a corporate update and reported first quarter 2023 financial results.
  • INTEGRIS-PSC Phase 2a 12-week clinical data expected in the third quarter of 2023.
  • Initiation of a Phase 1 first-in-human trial of PLN-101095 in oncology expected in the second quarter of 2023.
  • Initiation of a Phase 1 trial in patients with solid tumors that are resistant to immune checkpoint inhibitors is expected in the second quarter of 2023.

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

Retrieved on: 
Thursday, May 4, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Exercise, Spiroplasma mirum, European Medicines Agency, Food and Drug Administration, Prescription Drug User Fee Act, Primary sclerosing cholangitis, Biliary atresia, Medication, Primary biliary cholangitis, Shanda, Patient, Investment, PDUFA, Liver, Alagille syndrome, PFIC, Food, Volixibat, Pharmaceutical industry, Video game, Security (finance)

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

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for first quarter 2023 and provided a business update.
  • Total net product sales of LIVMARLI for the quarter ended March 31, 2023 was $29.1 million compared to $10.9 million for the quarter ended March 31, 2022.
  • Total operating expenses were $58.7 million for the quarter ended March 31, 2023 in comparison to $45.6 million for the quarter ended March 31, 2022.
  • As of March 31, 2023, Mirum had cash, cash equivalents, restricted cash equivalents, and short-term investments of $232.1 million.

Invea Therapeutics Research to be Showcased at the Prestigious 2023 DDW Meeting

Retrieved on: 
Wednesday, April 26, 2023
Translation, Abstract, Phenotype, Probability, Primary sclerosing cholangitis, Gastroenterology, Doctor of Philosophy, Hepatology, Indiana University School of Medicine, Novel, Department of Medicine – University of Pamplona, ML, Research, Inflammation, Endoscopy, Immunotherapy, DDW, Fibrosis, MD, Translational research, Physician, Machine learning, Division, Immune-mediated inflammatory diseases, Annual general meeting, GBLA, Artificial intelligence, Liver, AI, Ecosystem, Therapy, GI, PSC, Skin, Safety, Medical device, Vaccine

GUILFORD, Conn., April 26, 2023 (GLOBE NEWSWIRE) -- Invea Therapeutics (Invea), a biotechnology company developing an artificial intelligence (AI) and machine learning (ML) powered pipeline of potentially transformational medicines for highly debilitating immune-mediated inflammatory diseases (IMIDs), today announced the acceptance of two abstracts for oral presentations at the upcoming Digestive Disease Week (DDW) Annual Meeting.

Key Points: 
  • GUILFORD, Conn., April 26, 2023 (GLOBE NEWSWIRE) -- Invea Therapeutics (Invea), a biotechnology company developing an artificial intelligence (AI) and machine learning (ML) powered pipeline of potentially transformational medicines for highly debilitating immune-mediated inflammatory diseases (IMIDs), today announced the acceptance of two abstracts for oral presentations at the upcoming Digestive Disease Week (DDW) Annual Meeting.
  • DDW is the world’s premier meeting for physicians, researchers, and industry in the fields of gastroenterology, hepatology, endoscopy, and gastrointestinal surgery, which will take place in Chicago, IL, on May 6-9, 2023.
  • Chymase, a mast cell activation mediator, plays a crucial role in various pathophysiological processes, including epithelial barrier damage, inflammatory response, and fibrosis.
  • As such, INVA8001 has the potential to counteract chronic inflammation, its associated symptoms and complications in a variety of GI and extra-GI disorders.

Mirum Pharmaceuticals Announces Preliminary Unaudited First Quarter 2023 Financial Results

Retrieved on: 
Wednesday, April 12, 2023
Health, Hospitals, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Medication, VISTAS, Disclosure, Primary biliary cholangitis, Patient, Investment, Biliary atresia, Primary sclerosing cholangitis, Pharmaceutical industry

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced estimated revenue, including net product sales, and cash balance for the first quarter 2023.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced estimated revenue, including net product sales, and cash balance for the first quarter 2023.
  • The company has not completed quarter‐end financial close processes for the quarter ended March 31, 2023.
  • These estimates are preliminary, have not been audited, and are subject to change upon completion of company’s financial statement closing procedures.
  • Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of March 31, 2023.

ProQR Announces Initial Pipeline Targets and Highlights Axiomer® RNA Editing Platform Technology at R&D Event

Retrieved on: 
Wednesday, March 29, 2023
Heart, CVD, ProQR, Disease, Partnership, NTCP, University, Heart failure, Q&A, Genetics, Davis, Stroke, Cardiovascular disease, Splenic infarction, Eon, Research, Biliary atresia, Biomarker, IP, RNA, Doctor of Philosophy, Atherosclerosis, EDT, LOF, Eli Lilly, Blood, Free File Alliance, Protein, N-Acetylgalactosamine, Cholestasis, ADAR, Patient, Patent, Primate, Ophthalmology, Nervous system, Annual report, Liver, NHP, R, Liver failure, PSC, RNA editing, B4GALT1, Primary sclerosing cholangitis, Dietary supplement, Vaccine, Pharmaceutical industry, Cryptocurrency, Lilly, Axiom Space

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today announced initial pipeline programs focused on diseases that originate in the liver.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today announced initial pipeline programs focused on diseases that originate in the liver.
  • ProQR will host a virtual R&D event today, during which the Company will showcase its proprietary Axiomer RNA-editing technology platform, detail the pipeline, and provide guidance on the advancement of programs toward the clinic.
  • ProQR today announced AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

Pliant Therapeutics Announces Positive DSMB Safety Review and Initiation of Enrollment of Phase 2a Trial of Bexotegrast (PLN-74809) at 320 mg Dose in Primary Sclerosing Cholangitis

Retrieved on: 
Monday, March 13, 2023
Primary sclerosing cholangitis, IPF, Integrin, Data monitoring committee, Fibrosis, Patient, Idiopathic pulmonary fibrosis, Therapy, Safety, PSC, FDA, SAN, DSMB, Lefebvre, Pharmaceutical industry

Bexotegrast, is an oral, small molecule, dual-selective inhibitor of αvβ6 and αvβ1 integrins being developed for the treatment of PSC and idiopathic pulmonary fibrosis (IPF).

Key Points: 
  • Bexotegrast, is an oral, small molecule, dual-selective inhibitor of αvβ6 and αvβ1 integrins being developed for the treatment of PSC and idiopathic pulmonary fibrosis (IPF).
  • Bexotegrast has been administered to over 600 study participants to date, including healthy volunteers and patients, with no drug-related severe or serious adverse events reported to date.
  • “We are pleased to see the continued favorable safety and tolerability profile exhibited by bexotegrast, allowing us to evaluate a higher dose at a longer treatment duration in patients with PSC,” said Éric Lefebvre M.D.
  • The DSMB recommended that the INTEGRIS-PSC trial continue without modification, enabling the initiation of enrollment of the 320 mg dose group.

Autoimmune Liver Disease Forum to Examine Solutions for Improving Health Outcomes

Retrieved on: 
Tuesday, March 14, 2023
Hepatology, PBC, Autoimmune hepatitis, Intercept, Caregiver, Organization, Ipsen, Primary biliary cholangitis, AIH, ALF, Primary sclerosing cholangitis, Quality of life, Inspiration, MD, Patient, Overalls, American Liver Foundation, Stanford University School of Medicine, Nature Reviews Gastroenterology & Hepatology, Immune system, Division, Icahn School of Medicine at Mount Sinai, Diagnosis, PSC, Mount Sinai, Physician, Liver, Human, Pharmaceutical industry, Medical imaging, Vaccine, Dietary supplement, Medicine

FAIRFIELD, N.J., March 14, 2023 /PRNewswire/ -- A two-day virtual event on March 15th and 16th will bring together more than 350 patients, caregivers, hepatologists, primary care providers, patient advocacy organizations and industry representatives all with one goal in mind—find solutions to improve health outcomes for all those suffering from rare autoimmune liver diseases. The group will address two main questions: how can people avoid misdiagnosis and achieve earlier diagnosis; and how can we reduce the patient burden to improve the overall patient experience.

Key Points: 
  • Two-day Autoimmune Liver Disease Forum brings together key stakeholders to improve health outcomes for patients.
  • "We were overwhelmed with the positive response from our community for our Autoimmune Liver Disease Forum .
  • An autoimmune liver disease develops when your own immune system mistakes normal, healthy tissue for a foreign body.
  • The Autoimmune Liver Disease Forum is made possible through generous support provided by lead sponsor Ipsen and partnering sponsor Intercept.

Pliant Therapeutics Provides Corporate Update and Reports Fourth Quarter 2022 Financial Results

Retrieved on: 
Thursday, March 9, 2023
Pharmacokinetics, Idiopathic pulmonary fibrosis, IND, ELF, Novartis, Primary sclerosing cholangitis, Cirrhosis, Therapy, QLF, SAN, HIV disease progression rates, FDA, Fibrosis, Patient, Investigational New Drug, Serum, Public expenditure, Research, Mortality, Orphan drug, PSC, FVC, NASH, Exercise, Investment, Alkaline phosphatase, Safety, Human resources, Plasma, Integrin, ALP, Ironwood Pharmaceuticals, Biomarker, IPF, Pharmaceutical industry, Life insurance, EU

SOUTH SAN FRANCISCO, Calif., March 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, today provided a corporate update and reported fourth quarter 2022 financial results.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., March 09, 2023 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, today provided a corporate update and reported fourth quarter 2022 financial results.
  • We are positioned to deliver multiple clinical catalysts in 2023," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics.
  • Exploratory efficacy endpoints demonstrated strong treatment effects on FVC, Quantitative Lung Fibrosis (QLF) imaging and biomarkers over 12 weeks.
  • INTEGRIS-IPF Phase 2a trial on track for final 24-week data readout from the bexotegrast 320 mg dose group in the second quarter of 2023.

Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2022 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 8, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, New Drug Application, Webcast, Biliary atresia, PFIC, The Journal of Pediatrics, Growth, Spiroplasma mirum, Volixibat, Event, Itch, Sale, AASLD, IBAT, Quality of life, Patient, Shanda, European Commission, Food, Investment, Pediatrics, Primary sclerosing cholangitis, Food and Drug Administration, Alagille syndrome, Primary biliary cholangitis, Sleep, Medication, Pharmaceutical industry, Satellite navigation device, Cryptocurrency

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2022 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the fourth quarter and year-end 2022 and provided a business update.
  • Published LIVMARLI data highlighting quality of life and sleep improvements in patients with Alagille syndrome in the Journal of Pediatrics.
  • As of December 31, 2022, Mirum had cash, cash equivalents, restricted cash equivalents, and short-term investments of $251.7 million.
  • Mirum will host a conference call today, March 8, 2022, at 1:30 p.m. PT/4:30 p.m.