Itch

GENFIT Updates 2024 Outlook Following Acceptance of Elafibranor Filings in Primary Biliary Cholangitis (PBC)

Retrieved on: 
Friday, December 8, 2023
Hepatic encephalopathy, Ipsen, AASLD, Cell death, Food, Itch, Priority review, Food and Drug Administration, PSC, IND, ACLF, Marketing, FDA, EMA, MAA, PDUFA, Permeability (electromagnetism), Liver, NDA, Inflammation, Primary sclerosing cholangitis, ALP, Capital market, Hyperammonemia, Patient, NTZ, Fibrosis, PBC, ASK1

Launches in the US and Europe will also make GENFIT eligible for royalty payments.

Key Points: 
  • Launches in the US and Europe will also make GENFIT eligible for royalty payments.
  • Revenues will fund the development of GENFIT’s pipeline, now mainly focused on Acute On-Chronic Liver Failure (ACLF) with 5 differentiated assets.
  • Acceptance of filings in the US and Europe have triggered the first milestone payment for GENFIT.
  • Ipsen also indicated that it is developing elafibranor in Primary Sclerosing Cholangitis (PSC) which could lead to further revenues for GENFIT under the licensing agreement.

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Thursday, December 7, 2023
Liver, Ascending cholangitis, Ipsen, Food, Itch, Health care, Woman, FDA, EMA, MAA, PDUFA, Primary biliary cholangitis, Bile, Fatigue, NDA, Elafibranor, MHRA, ADR, New Drug Application, European Medicines Agency, Common, IPN, PPAR, Liver failure, Patient, Medicine, EVP, PBC, CHMP, Pharmaceutical industry

New Drug Application granted priority review with PDUFA date set for June 10, 2024

Key Points: 
  • New Drug Application granted priority review with PDUFA date set for June 10, 2024
    Investigational elafibranor is the first novel second-line treatment for primary biliary cholangitis (PBC) to be filed in E.U.
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The target FDA PDUFA date under priority review is June 10, 2024.
  • “This is a condition where many patients are living with worsening disease and debilitating symptoms despite being on treatment.

Ipsen confirms U.S. FDA grants priority review for New Drug Application for elafibranor for the treatment of rare cholestatic liver disease, PBC

Retrieved on: 
Thursday, December 7, 2023
Liver, UDCA, Chapter Two, Woman, FDA, MAA, Fatigue, Nausea, Primary biliary cholangitis, Inflammation, ADR, Patent, Liver disease, Diagnosis, AMF, CHMP, Ascending cholangitis, UCD, Business, ACLF, PDUFA, Itch, Risk, New Drug Application, OA, Common, EASL, MASH, Liver failure, Safety, Ammonia, Elafibranor, Therapy, Epidemiology, History, Patient, Cholangiocarcinoma, Food, Health care, EMA, Ursodeoxycholic acid, MHRA, Cirrhosis, Medicine, NTZ, EVP, Cholestasis, NASH, Research, Diarrhea, Bile, NDA, PBC, Abdominal pain, Clinical trial, European Medicines Agency, IPN, PPAR, P10, CCA, Fibrosis, Pharmaceutical industry

An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.

Key Points: 
  • An oral, once-daily dual peroxisome activated receptor alpha/delta (PPAR α,δ) agonist, investigational elafibranor could potentially be the first novel second-line treatment for the rare, cholestatic liver disease, PBC, in nearly a decade.
  • The trial enrolled 161 patients who were randomized 2:1 to receive either elafibranor 80mg once daily or placebo.
  • ELATIVE also investigated the effect of treatment with elafibranor on pruritus (severe itch), a significant symptom burden amongst people living with PBC.
  • Findings from the secondary endpoint using the PBC Worst Itch NRS score, showed a reduction in pruritis for elafibranor, which was not statistically significant.

MC2 Therapeutics Announces Completion of Enrollment in MC2-25 Ph2 Chronic Kidney Disease-associated Pruritus Trial

Retrieved on: 
Thursday, December 7, 2023
Urea, Quality of life, Protein, Senior lecturer, Hope, Itch, Barts Health NHS Trust, Dry, MD, Therapy, Skin, Dicarboxylic acid, MC2, European, Patient, Safety, Dialysis, CKD, Pharmaceutical industry

CKD-aP Stage 5 (dialysis) and stages 3 and 4 (pre-dialysis), the latter segment having no approved treatment options.

Key Points: 
  • CKD-aP Stage 5 (dialysis) and stages 3 and 4 (pre-dialysis), the latter segment having no approved treatment options.
  • MC2-25 CKD presents a potential breakthrough in the understanding and treatment of urea associated skin diseases that has puzzled scientists for decades.
  • Jesper L. Lange, CEO of MC2 Therapeutics said: “There is increasing evidence that carbamylation plays a central role in various urea associated skin diseases.
  • This new understanding of the role played by carbamylation in CKD-aP is very exciting and offers hope to millions of patients.”

MC2 Therapeutics Announces Completion of Enrollment in MC2-25 Ph2 Chronic Kidney Disease-associated Pruritus Trial

Retrieved on: 
Thursday, December 7, 2023
Urea, Quality of life, Protein, Senior lecturer, Hope, Itch, Barts Health NHS Trust, Dry, MD, Therapy, Skin, Dicarboxylic acid, MC2, European, Patient, Safety, Dialysis, CKD, Pharmaceutical industry

CKD-aP Stage 5 (dialysis) and stages 3 and 4 (pre-dialysis), the latter segment having no approved treatment options.

Key Points: 
  • CKD-aP Stage 5 (dialysis) and stages 3 and 4 (pre-dialysis), the latter segment having no approved treatment options.
  • MC2-25 CKD presents a potential breakthrough in the understanding and treatment of urea associated skin diseases that has puzzled scientists for decades.
  • Jesper L. Lange, CEO of MC2 Therapeutics said: “There is increasing evidence that carbamylation plays a central role in various urea associated skin diseases.
  • This new understanding of the role played by carbamylation in CKD-aP is very exciting and offers hope to millions of patients.”

Beardoholic Announce Expansion to Offices in the US and Europe

Retrieved on: 
Wednesday, December 6, 2023
Human, Nutrient, Itch, Collection, Life

“Beardoholic is a one-stop destination for everything related to beards and hairstyles,” said a spokesperson for Beardoholic .

Key Points: 
  • “Beardoholic is a one-stop destination for everything related to beards and hairstyles,” said a spokesperson for Beardoholic .
  • “Our artists provide personalized grooming services that cater to each individual’s unique preferences and facial features.
  • Investing in the right beard products, especially beard oil, can make all the difference in a beard care routine.
  • So, don’t hold back; give a beard the royal treatment it deserves with Beardoholic ’s barbers’ choice of beard oils.

Replimune Shares Initial Primary Analysis Results from CERPASS Clinical Trial in Advanced Cutaneous Squamous Cell Carcinoma and Presents New Data from IGNYTE Clinical Trial of RP1 in Anti-PD1 Failed Melanoma and Non-Melanoma Skin Cancers

Retrieved on: 
Tuesday, December 5, 2023
Chills, CRS, Melanoma, RP1, CRC, Food, Melanoma Research, RECIST, NMSC, Transplant, Diarrhea, FDA, Cytokine release syndrome, Colorectal cancer, SCCHN, Type, RP3, CRR, Fatigue, Headache, Nausea, Itch, Myocarditis, Incidence, Nivolumab, Influenza-like illness, Research, Clinical trial, Injection site reaction, Myalgia, CSCC, Skin cancer, HCC, Weakness, Society, Head, Appetite, RP2, Regeneron Pharmaceuticals, Carcinoma, Investment, Webcast, Patient, Lipase, Angiosarcoma, MCC, Vomiting, Neck, Uveal melanoma, Rash, BLA, Pharmaceutical industry

The CERPASS study was conducted under a Master Clinical Trial Collaboration and Supply Agreement with Regeneron Pharmaceuticals.

Key Points: 
  • The CERPASS study was conducted under a Master Clinical Trial Collaboration and Supply Agreement with Regeneron Pharmaceuticals.
  • The ORR was comparable between the two study groups (52.5% for RP1 plus cemiplimab vs. 51.4% for cemiplimab alone, p=0.692).
  • The registration directed anti-PD1 failed melanoma cohort from the IGNYTE clinical trial includes 140 patients and completed enrollment earlier this year.
  • RP1 monotherapy was well tolerated, and the safety profile was similar to that observed in non-immunocompromised patients with advanced skin cancers.

RAPT Therapeutics Announces Publication of Phase 1a/1b Clinical Trial of Zelnecirnon (RPT193) to Treat Atopic Dermatitis in Allergy

Retrieved on: 
Monday, November 27, 2023
Patient, Atopic dermatitis, Cytokine, Inflammation, Itch, Therapy, Asthma, NRS, Numerical, Safety, RAPT, Pharmaceutical industry

The Phase 1a portion of the trial was a standard single and multiple dose-escalation study in 72 healthy volunteers.

Key Points: 
  • The Phase 1a portion of the trial was a standard single and multiple dose-escalation study in 72 healthy volunteers.
  • The Phase 1b portion of the trial was a randomized, double-blind, placebo-controlled study examining zelnecirnon as monotherapy in 31 patients with moderate-to-severe atopic dermatitis (AD).
  • “We are pleased to have these exciting data published in the prestigious, peer-reviewed journal Allergy,” said Brian Wong, M.D., Ph.D., President and CEO of RAPT Therapeutics.
  • Based on the efficacy and safety data observed in the Phase 1b study, RAPT initiated a dose-ranging Phase 2b study in patients with moderate-to-severe AD and a Phase 2a study in asthma.

MC2 Therapeutics Announces First Patients Enrolled in Phase 2 Proof of Concept Clinical Trial in Vulvar Lichen Sclerosus

Retrieved on: 
Thursday, November 23, 2023
Urea, Quality of life, Protein, Woman, Chronic kidney disease, Pain, CMO, Lichen, Itch, Health, Therapy, POC, Inflammation, Skin, PAD, Dicarboxylic acid, Fissure, MC2, VLS, Patient, Safety, Pharmaceutical industry

MC2-25 VLS is a novel treatment paradigm based on a di-peptide that inhibits carbamylation of amino acids and proteins in skin.

Key Points: 
  • MC2-25 VLS is a novel treatment paradigm based on a di-peptide that inhibits carbamylation of amino acids and proteins in skin.
  • MC2 Therapeutics believes that carbamylation of amino acids and proteins in the vulvar region by iso-cyanate is the root cause of VLS.
  • The Phase 2 clinical trial is being conducted at multiple centres across Denmark and will evaluate the safety and efficacy of MC2-25 VLS in patients with VLS.
  • The data generated from this double-blinded, placebo-controlled PoC trial will inform the trial design for a larger Phase 2b trial.

MC2 Therapeutics Announces First Patients Enrolled in Phase 2 Proof of Concept Clinical Trial in Vulvar Lichen Sclerosus

Retrieved on: 
Thursday, November 23, 2023
Urea, Quality of life, Protein, Woman, Chronic kidney disease, Pain, CMO, Lichen, Itch, Health, Therapy, POC, Inflammation, Skin, PAD, Dicarboxylic acid, Fissure, MC2, VLS, Patient, Safety, Pharmaceutical industry

MC2-25 VLS is a novel treatment paradigm based on a di-peptide that inhibits carbamylation of amino acids and proteins in skin.

Key Points: 
  • MC2-25 VLS is a novel treatment paradigm based on a di-peptide that inhibits carbamylation of amino acids and proteins in skin.
  • MC2 Therapeutics believes that carbamylation of amino acids and proteins in the vulvar region by iso-cyanate is the root cause of VLS.
  • The Phase 2 clinical trial is being conducted at multiple centres across Denmark and will evaluate the safety and efficacy of MC2-25 VLS in patients with VLS.
  • The data generated from this double-blinded, placebo-controlled PoC trial will inform the trial design for a larger Phase 2b trial.