Netherton syndrome

Azitra, Inc. Announces Presentation at the 2024 BIO International Convention

Retrieved on: 
Freitag, Mai 31, 2024

Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced that management will present at the 2024 BIO International Convention being held June 3-6, 2024 in San Diego, California.

Key Points: 
  • Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced that management will present at the 2024 BIO International Convention being held June 3-6, 2024 in San Diego, California.
  • The presentation will take place on Wednesday, June 5, 2024 at 12:00 PM PDT in Theater 3 at the San Diego Convention Center.
  • The corporate update will highlight Azitra’s updates and progress on its pipeline, including ATR-12 in development for Netherton syndrome and ATR-04 in development for epidermal growth factor receptor (EGFR) inhibitor-induced dermal toxicity.
  • Azitra’s presentation details are as follows:
    To schedule a meeting with the Company’s management at the convention, please submit a meeting request through the BIO One-on-One Partnering™ system or contact [email protected]

Azitra, Inc. Announces Positive Preclinical Data of ATR-12 and Clinical Design in Netherton Syndrome Presented at the ASGCT Annual Meeting

Retrieved on: 
Freitag, Mai 10, 2024

The data in the two oral presentations today showcase the preclinical development of ATR-12 and the clinical study design of a Phase 1b study in Netherton syndrome patients.

Key Points: 
  • The data in the two oral presentations today showcase the preclinical development of ATR-12 and the clinical study design of a Phase 1b study in Netherton syndrome patients.
  • Additionally, in human ex vivo Netherton syndrome models, ATR-12 supernatant reduces protease activity nearly 7-fold to levels comparable to healthy skin.
  • The oral presentation entitled “Staphylococcus epidermidis Strain Expressing LEKTI-D6 (ATR-12) for Netherton Syndrome” also provides the study design for an active clinical trial of ATR-12 in Netherton syndrome patients.
  • “These data show the robust preclinical activity of ATR-12 in Netherton syndrome models and further supports the rationale behind our Phase 1b clinical trial in Netherton syndrome patients.”

Azitra, Inc. Announces Q1 2024 Financial Results and Provides Business Updates

Retrieved on: 
Donnerstag, Mai 9, 2024

Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the three months ended March 31, 2024, and provided a business update.

Key Points: 
  • Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the three months ended March 31, 2024, and provided a business update.
  • Moreover, preclinical evidence suggests that ATR-12 can markedly reduce IL-36g, a pro-inflammatory cytokine implicated in Netherton syndrome, further validating the potential of our approach.
  • Net Loss was $2.9 million for the quarter ended March 31, 2024, compared to $2.5 million for the comparable period in 2023.
  • Cash and cash equivalents: As of March 31, 2024, the Company had cash and cash equivalents of $3.0 million.

Quoin Pharmaceuticals Provides Corporate Update and Announces First Quarter 2024 Financial Results

Retrieved on: 
Donnerstag, Mai 9, 2024

ASHBURN, Va., May 09, 2024 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical stage, specialty pharmaceutical company focused on developing and commercializing novel treatments for rare and orphan diseases, today provides a business update and announces financial results for the first quarter ended March 31, 2024.

Key Points: 
  • ASHBURN, Va., May 09, 2024 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical stage, specialty pharmaceutical company focused on developing and commercializing novel treatments for rare and orphan diseases, today provides a business update and announces financial results for the first quarter ended March 31, 2024.
  • Quoin CEO, Dr. Michael Myers, said, “We are pleased to announce this encouraging start to 2024 as throughout the past quarter we successfully built upon the positive momentum from last year.
  • Quoin had approximately $14.7 million in cash, cash equivalents and marketable securities as of March 31, 2024.
  • Net loss for the quarter ended March 31, 2024 was approximately $2.3 million compared to approximately $2.6 million for the quarter ended March 31, 2023.

BioCryst Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Montag, Mai 6, 2024

RESEARCH TRIANGLE PARK, N.C., May 06, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today reported financial results for the first quarter ended March 31, 2024, and provided a corporate update.

Key Points: 
  • These two successes helped us exceed our revenue projections for the quarter and, as a result, we are increasing our revenue guidance for 2024,” said Charlie Gayer, chief commercial officer of BioCryst.
  • Interest expense was $24.5 million in the first quarter of 2024, compared to $27.4 million in the first quarter of 2023 (-10.6 percent y-o-y).
  • Our references to our first quarter 2024 “non-GAAP pro forma” financial measures of adjusted net loss and adjusted earnings per share constitute non-GAAP financial measures.
  • ET today to discuss the financial results and provide a corporate update.

Azitra, Inc. Announces New Preclinical Data to be Presented at the American Society of Gene and Cell Therapy Meeting

Retrieved on: 
Montag, April 22, 2024

Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced preclinical data from the Company’s platform and pipeline.

Key Points: 
  • Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today announced preclinical data from the Company’s platform and pipeline.
  • ATR-12 has been engineered to deliver missing LEKTI protein when applied topically to Netherton syndrome patients.
  • The data in the abstracts released online today show that topical application of ATR-12 in preclinical models reduced produced reduced IL-36γ by 93% compared to skin extracts induced to overexpress IL-36γ.
  • Additionally, topical application of ATR-12 significantly reduced protease activity in skin samples compared to a Netherton syndrome model skin (p

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1[...]

Retrieved on: 
Dienstag, April 9, 2024

Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

Key Points: 


Orphan designation: (S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide} Treatment of Netherton syndrome, 17/10/2019 Positive

Quoin Pharmaceuticals Provides Corporate Update and Announces Fourth Quarter and 2023 Financial Results

Retrieved on: 
Mittwoch, März 13, 2024

Quoin CEO, Dr. Michael Myers, said, “2023 marked another year of significant progress for Quoin and for the development of QRX003 for the treatment of Netherton Syndrome.

Key Points: 
  • Quoin CEO, Dr. Michael Myers, said, “2023 marked another year of significant progress for Quoin and for the development of QRX003 for the treatment of Netherton Syndrome.
  • Quoin had approximately $10.7 million in cash, cash equivalents and marketable securities as of December 31, 2023.
  • Net loss for the quarter ended December 31, 2023 was approximately $2.0 million compared to approximately $2.0 million for the quarter ended December 31, 2022.
  • The call will include a discussion of fourth quarter and full year 2023 financial results and a corporate update.

Azitra, Inc. Announces Full Year 2023 Financial Results and Provides Business Updates

Retrieved on: 
Freitag, März 15, 2024

Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the full year ended December 31, 2023, and provided a business update.

Key Points: 
  • Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, today reported financial results for the full year ended December 31, 2023, and provided a business update.
  • Completed and filed post-IND, FDA commitments for characterization of drug substance and drug product for ATR-12.
  • Selected and hired clinical research organization for ATR-12 clinical trial and selected initial clinical sites for activation.
  • Net Loss was $11.3 million for the year ended December 31, 2023, compared to $10.7 million for the comparable period in 2022.

Quoin Pharmaceuticals Files U.S. and International Patent Applications for Novel Netherton Syndrome Combination Product

Retrieved on: 
Donnerstag, Februar 8, 2024

ASHBURN, Va., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company exclusively focused on the development and commercialization of innovative therapies that treat rare and orphan diseases, today announced it has filed U.S. and International patent applications for a novel combination product as a potential treatment of Netherton Syndrome.

Key Points: 
  • ASHBURN, Va., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a specialty pharmaceutical company exclusively focused on the development and commercialization of innovative therapies that treat rare and orphan diseases, today announced it has filed U.S. and International patent applications for a novel combination product as a potential treatment of Netherton Syndrome.
  • This new product combines a broad spectrum serine protease inhibitor with an anti-inflammatory agent in a proprietary topical formulation.
  • Quoin anticipates that patents arising from these applications will extend to at least 2044.
  • Quoin is currently enrolling patients in two clinical trials being conducted under its open Investigational New Drug (IND) application, evaluating its QRX003 topical lotion as a potential treatment of Netherton Syndrome.