Iterum Therapeutics Plc

Monday, October 28, 2024, at 8:30 a.m. EDT DUBLIN and CHICAGO, Oct. 28, 2024 /PRNewswire/ -- About Iterum Therapeutics plc Iterum Therapeutics plc is focused on delivering differentiated anti-infectives aimed at combatting the global crisis of multi-drug resistant pathogens to significantly improve the lives of people affected by serious and life-threatening diseases around the world. Iterum is advancing the development of its first compound, sulopenem, a novel penem anti-infective compound, with an oral formulation and IV formulation. Sulopenem has demonstrated potent in vitro activity against a wide variety of gram-negative, gram-positive and anaerobic bacteria resistant to other antibiotics. Iterum has received approval of its NDA for ORLYNVAH™ (oral sulopenem) for the treatment of uncomplicated urinary tract infections caused by the designated microorganisms Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women with limited or no alternative oral antibacterial treatment options by the U.S. Food and Drug Administration and has received Qualified Infectious Disease Product (QIDP) and Fast Track designations for its oral and IV formulations of sulopenem in seven indications. For more information, please visit . SOURCE Iterum Therapeutics plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

LAMA2-CMD is characterised by the absence of the LAMA2 protein, leading to severe, early-onset muscular dystrophy. Credit: Ines Nepo/Shutterstock. The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Modalis Therapeutics’ investigational therapy, MDL-101, aimed at treating congenital muscular dystrophy type 1A (LAMA2-CMD). An epigenetic editing therapy, MDL-101 includes a guide nucleotide targeting the LAMA1 gene — a close homolog of the disease-causing LAMA2 gene. It utilises an enzyme-null Cas9 (dCas9) fused with a trans-activating domain, driven by a muscle-specific promoter and delivered via a muscle-specific AAV [adeno-associated virus] vector. MDL-101 works by upregulating LAMA1 gene products in patients’ muscle tissue to compensate for loss of function resulting from LAMA2 mutations. This approach has the potential to offer a one-time, long-lasting treatment option for individuals with LAMA2-CMD. ODD status facilitates the development of treatments for conditions affecting fewer than 200,000 patients in the US. See Also: UAE’s clinical trials space matures with experience from Covid-19 FDA approves Iterum’s ORLYNVAH NDA for uUTI treatment Under the ODD status for MDL-101, Modalis will receive benefits including exemption from application fees on filing new drug applications, tax reductions for clinical development and preferential treatment for the development and promotion of the asset in the US. On receipt of regulatory approval, MDL-101 would also receive seven years of exclusive sales rights in the US market. The US regulator previously granted rare paediatric disease designation for gene therapy for patients with LAMA2-CMD. LAMA2-CMD is characterised by the absence of the LAMA2 protein, a critical component comprising more than 3,000 amino acids. This leads to severe, early-onset muscular dystrophy, and due to the protein’s size, conventional gene therapy approaches such as using an AAV vector are challenging. Modalis focuses on the development of precision genetic therapies leveraging its epigenome editing technology, CRISPR-GNDM. This technology enables modulation of gene expression without causing double-strand DNA breaks. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

The FDA has approved Iterum Therapeutics’ Orlynvah (sulopenem etzadroxil and probenecid), marking it as the first oral penem approved in the U.S. for the treatment of uncomplicated urinary tract infections (uUTIs). Designed for adult women with limited alternative oral antibacterial options, orlynvah targets infections caused by E. coli, K. pneumoniae, or P. mirabilis. The approval is Iterum’s first FDA-cleared product, expanding treatment possibilities in the uUTI field. The FDA nod was based on positive results from two phase 3 trials, SURE 1 and REASSURE, assessing orlynvah’s effectiveness against uUTI-causing bacteria. SURE 1 showed the drug’s superiority over ciprofloxacin in treating fluoroquinolone-resistant infections, while REASSURE demonstrated non-inferiority to augmentin in susceptible populations. orlynvah was generally well tolerated in these studies, highlighting its potential for addressing antibiotic-resistant uUTI strains. With over 40 million uUTI prescriptions issued annually in the U.S., Iterum’s new drug offers an essential alternative in a market grappling with rising antibiotic resistance.