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Details:
ELX-02 (exaluren sulfate) is a small molecule drug candidate designed to restore production of full-length functional proteins. It is being developed for the treatment of alport syndrome.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 09, 2023
Details:
ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. It is being developed for the treatment of CF patients with nonsense mutations.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 24, 2023
Details:
ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. It is being developed for the treatment of CF patients with nonsense mutations.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 27, 2023
Details:
ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins (collagen IV and collagen VII). It has been granted fast track designation for the treatment of CF patients with nonsense mutations.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 25, 2023
Details:
ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins,for the treatment of CF patients with nonsense mutations.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 01, 2022
Details:
Evidence of activity for ELX-02 was observed, as patients with higher baseline sweat chloride levels demonstrated increased responses as indicated by SCC (p=0.00013 at Day 35).
Lead Product(s): ELX-02,Ivacaftor
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 14, 2022
Details:
In recently published preclinical studies, ELX-02 is a small molecule drug candidate designed to restore production of full-length functional proteins has demonstrated readthrough in COL4A5 mutations, which represent 85% of nonsense mutations in this population.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: PreclinicalProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 30, 2022
Details:
The new funding will enable the company to expand the studies to determine whether the combination of ELX-02 with a cystic fibrosis transmembrane conductance regulator (CFTR) modulator results in increased biological activity over ELX-02 alone.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: The Cystic Fibrosis Foundation
Deal Size: $19.5 million Upfront Cash: Undisclosed
Deal Type: Funding March 29, 2022
Details:
In preclinical studies, Class 1 CF patient organoids had a 2- to 3-fold higher swelling response with a combination of ELX-02 and Kalydeco than with ELX-02 as a monotherapy. Topline results are expected by the end of the first half of 2022.
Lead Product(s): ELX-02,Ivacaftor
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 17, 2021
Details:
This non-dilutive financing will provide additional capital to advance the product pipeline, including ELX-02, which is currently in Phase 2 clinical trials in cystic fibrosis patients affected by nonsense mutations, as well as preclinical programs.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Hercules Capital
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Financing October 05, 2021
Details:
ELX-02 is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 06, 2021
Details:
The CF Foundation’s partial funding of the U.S. trial has been extended to partial funding of Phase 2 proof of concept global clinical trial program for ELX-02 which includes Europe and Israel.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: CF Foundation
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Funding January 13, 2021
Details:
ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 27, 2020
Details:
ELX-02 produces significant read-through of premature stop codons leading to full length proteins, demonstrated using DMS-114 cells with the R213X nonsense mutation in the TP53 gene.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 22, 2020
Details:
The presentation shows that ELX-02’s selectivity for read-through of premature stop codons versus native stop codons and its ability to restore production of functional CFTR in patient-derived organoids.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 26, 2020
Details:
Eloxx Pharmaceuticals has initiated enrolment in its Phase 2 clinical trial for ELX-02 in cystic fibrosis has resumed at certain clinical sites in the U.S. after being temporarily paused in response to the COVID-19 pandemic.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 12, 2020
Details:
Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: ELX-02
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 04, 2020
Details:
Enrollment in Phase 2 clinical trials for ELX-02 in cystic fibrosis have been temporarily paused in response to the COVID-19 pandemic.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2020
Details:
Results from clinical and preclinical research to date support Company’s prioritization of Cystic Fibrosis programs.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 26, 2020
Details:
Phase 2 cystic fibrosis program for ELX-02 expected to be fully enrolled in the first quarter of 2020 and topline results expected during the first half of 2020.
Lead Product(s): ELX-02
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 14, 2020
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