JEB

Chiesi Global Rare Diseases Receives FDA Approval for FILSUVEZ® (birch triterpenes) topical gel for the Treatment of Epidermolysis Bullosa

Retrieved on: 
Tuesday, December 19, 2023
European Commission, Food, Risk, Infection, Caregiver, System, FDA, Acquisition, JEB, Disease, Civil service commission, DEB, Dermatitis, Hypersensitivity, Blister, Patient, EB, Skin, Pharmaceutical industry

BOSTON, Dec. 19, 2023 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, announced today that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes) topical gel for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB). FILSUVEZ is the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of EB with blisters beginning in infancy. FILSUVEZ joined the Chiesi portfolio as part of the agreement reached during the acquisition of Amryt Pharma in January of this year.

Key Points: 
  • FILSUVEZ is the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of EB with blisters beginning in infancy.
  • FILSUVEZ joined the Chiesi portfolio as part of the agreement reached during the acquisition of Amryt Pharma in January of this year.
  • "At Chiesi Global Rare Diseases we are driven by a need to alleviate the burdens faced in the rare disease community by providing innovative therapies and solutions that address debilitating unmet needs.” Giacomo Chiesi, Head of Chiesi Global Rare Diseases, said.
  • FILSUVEZ topical gel is indicated for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in adult and pediatric patients 6 months of age and older.

Eloxx Pharmaceuticals Reports Third Quarter 2023 Financial and Operating Results and Provides Business Update

Retrieved on: 
Monday, November 13, 2023
Sale, FSD, RDEB, ASN, Kidney, GBM, American Society of Nephrology, Patient, CFF, FDA, Glomerular basement membrane, G&A, Health, R, JEB, ATM, Society, TEM, OTC, RNA, Proteinuria, Biopsy, Pharmaceutical industry, Phosphorus, Microscope, Cryptocurrency, Alport syndrome

WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.

Key Points: 
  • WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.
  • Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the American Society of Nephrology (ASN) Kidney Week 2023.
  • R&D expenses were $1.3 million for the three months ended September 30, 2023, which included $0.1 million in stock-based compensation.
  • Additionally, we sold shares of common stock through our ATM program during the quarter for gross proceeds of $1.8 million.

Gilbane Building Company Breaks Ground on Washington Yu Ying Public Charter School in Washington, DC

Retrieved on: 
Thursday, October 5, 2023
Partnership, Certification, Gilbane Building Company, Washington Latin Public Charter School, Page, Communication, Immersion, Education, SF, DMV, Gold, JEB, Airline

WASHINGTON, Oct. 5, 2023 /PRNewswire-PRWeb/ -- Gilbane Building Company is proud to announce the groundbreaking of the Washington Yu Ying Public Charter School (Yu Ying) in Washington, DC. The ceremony included key stakeholders from Level Field Partners, Yu Ying, JEB Consulting Group, Page Southerland Page, and Gilbane to celebrate the start of construction for the Yu Ying Charter School.

Key Points: 
  • WASHINGTON, Oct. 5, 2023 /PRNewswire-PRWeb/ -- Gilbane Building Company is proud to announce the groundbreaking of the Washington Yu Ying Public Charter School (Yu Ying) in Washington, DC.
  • The ceremony included key stakeholders from Level Field Partners, Yu Ying, JEB Consulting Group, Page Southerland Page, and Gilbane to celebrate the start of construction for the Yu Ying Charter School.
  • The Yu Ying Charter School, a Chinese immersion charter school, fosters a multicultural and bilingual educational environment for kindergarten through eighth-grade students.
  • "We are thrilled to break ground on Yu Ying Charter School's new campus in Washington, DC," said Tyler Swartzwelder, vice president and business leader for Gilbane Building Company in Washington, DC.

Eloxx Pharmaceuticals Granted Extension by Nasdaq to Regain Compliance with the Market Value of Listed Securities Continued Listing Requirement

Retrieved on: 
Tuesday, June 6, 2023
Familial adenomatous polyposis, Market value, DSM-IV codes, RDEB, Food, Calendar, FAP, Concha bullosa, RNA, Proteinuria, JEB, Patient, Hearing, Rare, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Disease, Pharmaceutical industry, Cryptocurrency, Alport syndrome

“We are pleased that Nasdaq has granted us this extension and intend to take the necessary steps to regain compliance with Nasdaq’s minimum market value of listed securities requirement,” said Sumit Aggarwal, President and CEO of Eloxx.

Key Points: 
  • “We are pleased that Nasdaq has granted us this extension and intend to take the necessary steps to regain compliance with Nasdaq’s minimum market value of listed securities requirement,” said Sumit Aggarwal, President and CEO of Eloxx.
  • On October 11, 2022, Nasdaq notified the Company that it no longer complied with the Market Value of Listed Securities requirement pursuant to Listing Rule 5550(b)(2).
  • In accordance with Listing Rule 5810(c)(3)(C), the Company was provided 180 calendar days, or until April 10, 2023, to regain compliance.
  • Based on the information presented, Nasdaq granted the Company’s request for an exception through July 30, 2023 to regain compliance with the continued listing requirements.

Eloxx Pharmaceuticals Announces FDA Clearance to Begin Single Ascending Dose Study of ZKN-013

Retrieved on: 
Tuesday, May 2, 2023
Polyp, Polyp (medicine), Skin, Survival, Clinical trial, Mutation, Lead, SAD, Cystic fibrosis, FAP, RNA, JEB, Patient, Cancer, Cystic Fibrosis Foundation, Adenoma, Mouse, MAD, Epidermolysis bullosa dystrophica, Rare, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Vaccine, Pharmaceutical industry, RDEB

WATERTOWN, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the “Company”), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

Key Points: 
  • RDEB is a rare skin disease characterized by mutations in Collagen7 gene.
  • “FDA clearance to begin our planned single ascending dose trial is an important milestone towards providing a potential treatment option for patients with RDEB and JEB,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA.
  • In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival.

Eloxx Pharmaceuticals Announces Submission of Investigational New Drug (IND) Application for ZKN-013

Retrieved on: 
Tuesday, March 28, 2023
COL7, Polyp, Polyp (medicine), Skin, Drug development, Familial adenomatous polyposis, Survival, Mutation, FAP, Fibroblast, RNA, Cancer, Investigational New Drug, JEB, Patient, Keratinocyte, DSM-IV codes, Adenoma, COL, Growth, Epidermolysis bullosa dystrophica, APC, Rare, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, IND, Food, Vaccine, Pharmaceutical industry, RDEB

WATERTOWN, Mass., March 28, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

Key Points: 
  • “This IND application is an important milestone towards providing a treatment option for patients with RDEB and JEB, as there are currently no approved disease-modifying treatments,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps.
  • Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.
  • In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP.

Purepoint Uranium to Present at the Red Cloud's 2023 Pre-PDAC Mining Showcase and at the PDAC Conference

Retrieved on: 
Wednesday, March 1, 2023
Cloud, TGI, Camp Red Cloud, Hook Lake, Metro Toronto Convention Centre, Cameco, JEB, Sheraton Centre Toronto Hotel, 3RRR, EM, PTU, Conference, Red Cloud, MUFG Union Bank, Eagle Point, IP, Red Willow, Oil, Uranium mining, Arrow, Athabasca Basin, Patterson Lake

Toronto, Ontario--(Newsfile Corp. - March 1, 2023) - Purepoint Uranium Group Inc. (TSXV: PTU) (OTCQB: PTUUF) ("Purepoint" or the "Company") will be presenting at Red Cloud's Pre-PDAC Mining Showcase that will be held on March 2 and 3, 2023 and at the PDAC Conference that will be held from March 5 to 8, 2023.

Key Points: 
  • Toronto, Ontario--(Newsfile Corp. - March 1, 2023) - Purepoint Uranium Group Inc. (TSXV: PTU) (OTCQB: PTUUF) ("Purepoint" or the "Company") will be presenting at Red Cloud's Pre-PDAC Mining Showcase that will be held on March 2 and 3, 2023 and at the PDAC Conference that will be held from March 5 to 8, 2023.
  • Red Cloud Conference: Friday, March 3, 2023 at 1:50 pm ET at the Sheraton Centre Toronto Hotel.
  • PDAC 2023 Conference: Wednesday, March 8th at 10:26 am, Uranium Session at room 803 at the Metro Toronto Convention Centre.
  • The Purepoint Team will also be available on booth # 2636 at the Metro Toronto Convention Centre for the duration of the conference.

The British Journal of Dermatology Publishes Results from Amryt’s EASE Phase 3 Trial in Epidermolysis Bullosa

Retrieved on: 
Tuesday, October 25, 2022
Royal Melbourne Hospital, Wound healing, Journal of the American Academy of Dermatology, Acceleration, Epidermolysis bullosa, Patient, JEB, University of New South Wales, NSW, Degenerative disease, University, DEB, EB, Pain, Safety, St George's Hospital, Principal, Pharmaceutical industry, Dermatology, EU, EASE

EASE is the largest Phase 3, randomised, controlled study in Epidermolysis Bullosa (EB), that examined the efficacy and safety of Filsuvez (birch bark extract) gel/Oleogel-S10 versus vehicle control gel in EB.

Key Points: 
  • EASE is the largest Phase 3, randomised, controlled study in Epidermolysis Bullosa (EB), that examined the efficacy and safety of Filsuvez (birch bark extract) gel/Oleogel-S10 versus vehicle control gel in EB.
  • *
    Key outcomes from the EASE double-blind phase included:
    Professor Dedee Murrell, Head of Dept.
  • The EASE trial (NCT03068780) is the largest ever global Phase 3 trial conducted in patients with EB, performed across 58 sites in 28 countries.
  • It comprises a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase.

Amryt Announces New Patent for Mycapssa®

Retrieved on: 
Tuesday, September 13, 2022
Octreotide, COVID-19, Diet, FDA, Lists of diseases, Metreleptin, Lipodystrophy, Degenerative disease, Environment, Forward-looking statement, Patient, Investment, JEB, USPTO, Lomitapide, Orange Book, DEB, Birth, GL, Acromegaly, Patent, EMA, Pharmaceutical industry, Dietary supplement, EU

DUBLIN, Ireland, and Boston MA, September 13, 2022 Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, announces an update regarding the patents for its product Mycapssa (octreotide).

Key Points: 
  • DUBLIN, Ireland, and Boston MA, September 13, 2022 Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, announces an update regarding the patents for its product Mycapssa (octreotide).
  • The USPTO has issued to Amryt US Patent No.
  • Amryt has nine Orange Book-listed patents for Mycapssa with patent protection through December 2040.
  • Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases.

Amryt Receives Marketing Authorisation Approval and Orphan Drug Designation for Filsuvez® in Great Britain

Retrieved on: 
Thursday, September 8, 2022
Acromegaly, COVID-19, EMA, GB, Scar, Birth, CEO, Degenerative disease, Lomitapide, DEB, GL, Lists of diseases, JEB, MAA, Skin, Royal commission, EB, Forward-looking statement, Diet, Octreotide, Medicines and Healthcare products Regulatory Agency, Infection, Investment, Metreleptin, ODD, Ulcer, Lipodystrophy, Patient, FDA, Company, MHRA, Stenosis, Risk, European Commission, Environment, Death, Pharmaceutical industry, Dietary supplement, EU

Joe Wiley, CEO of Amryt Pharma, commented: The approval of Filsuvez by the MHRA is an important development for patients suffering from this debilitating condition and follows the approval of Filsuvez by the European Commission in June.

Key Points: 
  • Joe Wiley, CEO of Amryt Pharma, commented: The approval of Filsuvez by the MHRA is an important development for patients suffering from this debilitating condition and follows the approval of Filsuvez by the European Commission in June.
  • The global market opportunity for EB is estimated by the Company to be in excess of$1.0 billion.
  • Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases.
  • Filsuvez is approved in theEU and Great Britain for the treatment of partial thickness wounds associated with JEB and DEB in patients 6 months and older.