SPINK5

BioCryst R&D Day Highlights New Diversified Pipeline of First-in-Class/Best-in-Class Therapies with Five Programs Expected in Clinical Development in Next 24 Months

Retrieved on: 
Friday, November 3, 2023
GMG, BioCryst Pharmaceuticals, FDA, Research, Patient, Drug design, IgA nephropathy, Plasma, CAD, Lupus nephritis, KLK5, Genetic disorder, RESEARCH, C2, DME, Eculizumab, Infection, Injection, Skin, Diabetes, Autoantibody, Ryan Murphy, Edema, Netherton, R, Autoimmune hemolytic anemia, HAE, Kallikrein, Growth, Disease, Dehydration, Muscle weakness, Immune system, Hemolysis, Solubility, Netherton syndrome, Clinical trial, Paratriathlon, Eye, Ravulizumab, Cold agglutinin disease, PARK, IP, SPINK5, Fine chemical, Pharmaceutical industry, Vaccine

A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .

Key Points: 
  • A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .
  • The team is excited to share our approach, and our expanded pipeline of new programs, at our R&D Day,” said Jon Stonehouse, president and chief executive officer of BioCryst.
  • The disease is caused by the deficiency of a natural inhibitor (SPINK5) of KLK5, a serine protease responsible for regulating skin shedding.
  • Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections.

Quoin Pharmaceuticals Announces Dosing of First Patient in Clinical Trial in Netherton Syndrome

Retrieved on: 
Tuesday, December 13, 2022
Asthma, Scaly, Quality of life, Clinical trial, Itchy, Dermatitis, Food, FDA, Safety, Dehydration, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Epidermolysis bullosa, Skin, Hair, SPINK5, Infection, Risk, Netherton syndrome, Attention deficit hyperactivity disorder predominantly inattentive, Mutation, IND, Standard of care, Peeling skin syndrome, Patient, Pharmaceutical industry, Scleroderma, Netherton

ASHBURN, Va., Dec. 13, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company focused on rare and orphan diseases, announces that the first patient has been dosed in its ongoing clinical trial evaluating the safety and efficacy of QRX003 as a treatment for Netherton Syndrome.

Key Points: 
  • This second study, which has now been cleared by the FDA to initiate clinical testing, is an open-label, non-placebo controlled trial that will evaluate 10 Netherton patients over a twelve week period.
  • The dosing of our first patient and the clearance of our second study are big steps forward for Quoin and, hopefully, for the community in general.
  • Quoin Pharmaceuticals Ltd. is a clinical stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.
  • Netherton Syndrome is a rare and sometimes fatal skin disease for which there is no approved treatment, and no cure.

Quoin Pharmaceuticals Announces Plans to Initiate a Second Clinical Trial in Netherton Syndrome Patients

Retrieved on: 
Tuesday, October 18, 2022
Quality of life, LinkedIn, Peeling skin syndrome, Disease, SEC, IND, Infection, Epidermolysis bullosa, Attention deficit hyperactivity disorder predominantly inattentive, Scaly, Dehydration, NASDAQ, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, Standard of care, Asthma, Itchy, Private Securities Litigation Reform Act, Netherton syndrome, Food, Hair, Trial of the century, Company, Mutation, Application, Safety, Skin, GLOBE, Clinical trial, Dermatitis, Forward-looking statement, FDA, SPINK5, Risk, Pharmaceutical industry, Scleroderma, Netherton

ASHBURN, Va., Oct. 18, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, announces that it is initiating a second clinical trial in Netherton Syndrome patients.

Key Points: 
  • ASHBURN, Va., Oct. 18, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, announces that it is initiating a second clinical trial in Netherton Syndrome patients.
  • Quoin CEO, Dr. Michael Myers, said, We are very pleased to announce our plan to initiate this second clinical study in Netherton patients under our open IND application.
  • We firmly believe that, if approved, QRX003 has the potential to become the standard of care for Netherton patients.
  • Quoin Pharmaceuticals Ltd. is a clinical stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

Quoin Pharmaceuticals Establishes Patient Specific Website for Ongoing Netherton Syndrome Clinical Study

Retrieved on: 
Tuesday, September 13, 2022
Netherton syndrome, Risk, Trial of the century, SPINK5, FDA, LinkedIn, Company, Private Securities Litigation Reform Act, Itchy, Hair, Scaly, Forward-looking statement, Infection, Patient, Peeling skin syndrome, Dermatitis, NASDAQ, Epidermolysis bullosa, Dehydration, GLOBE, Marketing, SEC, Awareness, Skin, Asthma, Mutation, Pharmaceutical industry, Netherton, Scleroderma

ASHBURN, Va., Sept. 13, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, has established a patient specific website: https://www.nethertonsyndromeclinicaltrials.com/ , to facilitate patient awareness and participation in its ongoing clinical study evaluating QRX003 for the treatment of Netherton Syndrome.

Key Points: 
  • ASHBURN, Va., Sept. 13, 2022 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the Company or Quoin), a specialty pharmaceutical company focused on rare and orphan diseases, has established a patient specific website: https://www.nethertonsyndromeclinicaltrials.com/ , to facilitate patient awareness and participation in its ongoing clinical study evaluating QRX003 for the treatment of Netherton Syndrome.
  • Based on discussions with the FDA, a number of different clinical endpoints will be assessed in the study.
  • We believe the establishment of this patient specific website could be an effective tool to provide information about our clinical study and facilitate patient recruitment.
  • Quoin Pharmaceuticals Ltd. is a clinical stage specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

ResVita Bio receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome

Retrieved on: 
Thursday, June 9, 2022
LEKTI, Metabolic engineering, Proteolysis, Skin, Dehydration, SPINK5, FDA, Infection, Patient, Mortality, Atopic dermatitis, Psoriasis, Itch, Synthetic biology, Netherton syndrome, Pharmaceutical industry

BERKELEY, Calif., June 9, 2022 /PRNewswire/ --ResVita Bio, a cellular therapies startup, announces that the FDA has granted Rare Pediatric Disease Designation to RVB-001 to treat Netherton Syndrome, a chronic and life-threatening genetic skin disease.

Key Points: 
  • BERKELEY, Calif., June 9, 2022 /PRNewswire/ --ResVita Bio, a cellular therapies startup, announces that the FDA has granted Rare Pediatric Disease Designation to RVB-001 to treat Netherton Syndrome, a chronic and life-threatening genetic skin disease.
  • "The physical and emotional challenges of chronic skin diseases are devastating to patients, particularly children" said Amin Zargar, Chief Executive Officer of ResVita Bio.
  • "Newborns with Netherton Syndrome face a severe lifelong disease with few treatment options that offer limited efficacy.
  • With the Rare Pediatric Disease Designation, if a new drug application for RVB-001 is approved, ResVita Bio may be eligible to receive a priority review voucher, which can be redeemed for priority review of any subsequent drug marketing application.

Quoin Pharmaceuticals Announces FDA Clearance of IND for its Lead Asset, QRX003, for Netherton Syndrome

Retrieved on: 
Monday, April 25, 2022
Patient, Netherton syndrome, Lists of diseases, Dermatitis, IND, NASDAQ, Dehydration, LEKTI, Food, Infection, Private Securities Litigation Reform Act, Peeling skin syndrome, GLOBE, Risk, Growth, Company, Food and Drug Administration, SEC, Forward-looking statement, NS, Epidermolysis bullosa, SPINK5, FDA, Mutation, Allergy, Asthma, Pharmaceutical industry, Netherton, Ichthyosis

The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier.

Key Points: 
  • The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier.
  • Daily application of QRX003 is designed to lead to a more normalized skin shedding process and the formation of a stronger and more effective skin barrier.
  • This is an important milestone both for Quoin and potentially for this very underserved patient population, said Dr. Michael Myers, Chief Executive Officer of Quoin.
  • Quoin Pharmaceuticals Ltd. is an emerging specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

Quoin Pharmaceuticals Announces Submission of IND for its Lead Asset, QRX003, for Netherton Syndrome

Retrieved on: 
Tuesday, March 15, 2022
Epidermolysis bullosa, Mutation, SEC, Quality of life, GLOBE, Patient, Private Securities Litigation Reform Act, FDA, SPINK5, Risk, Infection, Allergy, Forward-looking statement, NASDAQ, Asthma, IND, Dermatitis, Dehydration, Investigational New Drug, Food, Netherton syndrome, Company, Growth, Lists of diseases, Regulation of tobacco by the U.S. Food and Drug Administration, Peeling skin syndrome, LEKTI, Pharmaceutical industry, Netherton, Ichthyosis

The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier.

Key Points: 
  • The absence of LEKTI in Netherton patients leads to excessive skin shedding resulting in a highly porous and compromised skin barrier.
  • Daily application of QRX003 could potentially lead to improvement in skin architecture and restoration of the skins barrier properties, resulting in substantially improved quality of life for patients with Netherton Syndrome.
  • This is an important milestone for Quoin, and wed like to thank everyone who participated in the preparation of the IND submission.
  • Quoin Pharmaceuticals Ltd. is an emerging specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

Quoin Pharmaceuticals Submits Scientific Advice Briefing Document to EMA for its Lead Asset, QRX003, for Netherton Syndrome

Retrieved on: 
Thursday, March 10, 2022
NASDAQ, Lists of diseases, SPINK5, Asthma, Science, Private Securities Litigation Reform Act, Patient, Company, Marketing, FDA, Mutation, EMA, Netherton syndrome, Risk, Dermatitis, GLOBE, European Medicines Agency, Dehydration, Allergy, Growth, Peeling skin syndrome, Infection, Epidermolysis bullosa, SEC, Forward-looking statement, Pharmaceutical industry, Medical device, EU, Ichthyosis

The submission of this briefing document is the first step in the formal development process of QRX003 as a potential treatment for Netherton Syndrome in the EU, said Dr. Michael Myers, Chief Executive Officer of Quoin.

Key Points: 
  • The submission of this briefing document is the first step in the formal development process of QRX003 as a potential treatment for Netherton Syndrome in the EU, said Dr. Michael Myers, Chief Executive Officer of Quoin.
  • We look forward to receiving guidance from the EMA on the clinical and regulatory requirements to advance the development of the product in the EU.
  • Quoin has previously received written guidance from the FDA on the clinical requirements for the US and key elements of that feedback have been incorporated into this submission to the EMA.
  • Quoin Pharmaceuticals Ltd. is an emerging specialty pharmaceutical company focused on developing and commercializing therapeutic products that treat rare and orphan diseases.

Quoin Pharmaceuticals Extends Exclusive Distribution Agreement with ER-Kim for its Lead Asset, QRX003, for Netherton Syndrome

Retrieved on: 
Tuesday, March 1, 2022
GLOBE, Partnership, Risk, Growth, NASDAQ, Asthma, Dehydration, Company, Dermatitis, Private Securities Litigation Reform Act, Mutation, Lists of diseases, Netherton, Peeling skin syndrome, Allergy, Intermediary, Biotechnology, CEE, Forward-looking statement, Patient, Netherton syndrome, Epidermolysis bullosa, Infection, SEC, SPINK5, Pharmaceutical industry, Mediterranean Sea, Ichthyosis

Under the terms of the revenue sharing agreement, which was initially announced earlier in February 2022, ER-Kim gains exclusive rights to commercialize QRX003 in six additional countries.

Key Points: 
  • Under the terms of the revenue sharing agreement, which was initially announced earlier in February 2022, ER-Kim gains exclusive rights to commercialize QRX003 in six additional countries.
  • Quoin will be the exclusive supplier of QRX003 to ER-Kim.
  • Quoin now has 60 countries under partnership distribution agreements for QRX003, which has the potential to become the first approved treatment for Netherton Syndrome.
  • Currently, there is no cure for Netherton Syndrome, nor are there any approved therapeutic treatments.

Quoin Pharmaceuticals Signs Exclusive Distribution Agreement with Neopharm for its Lead Asset, QRX003, for Netherton Syndrome

Retrieved on: 
Thursday, February 17, 2022
NASDAQ, Asthma, Patient, Intermediary, Netherton syndrome, Allergy, Health, Company, SPINK5, SEC, Forward-looking statement, Peeling skin syndrome, Infection, Private Securities Litigation Reform Act, Lists of diseases, Dehydration, Mutation, Partnership, Risk, Dermatitis, Growth, GLOBE, Epidermolysis bullosa, Pharmaceutical industry, Ichthyosis

Under the terms of the revenue sharing agreement, Neopharm gains exclusive rights to commercialize QRX003 in Israel.

Key Points: 
  • Under the terms of the revenue sharing agreement, Neopharm gains exclusive rights to commercialize QRX003 in Israel.
  • Quoin will be the exclusive supplier of QRX003 to Neopharm.
  • Dr. Michael Myers, Chief Executive Officer of Quoin, commented, This is Quoins sixth distribution agreement in the last four months for QRX003 and were excited to add Neopharm, who is a leading distributor in Israel.
  • Neopharm Medical Supplies was established in the early 1980s and is the exclusive Israeli distributor of leading medical and pharma manufacturers.