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ALXN1840 (WTX101) is a potential new once-daily, oral medicine in development for the treatment of Wilson disease. This investigational, novel molecule is designed to selectively and tightly bind to and remove copper from the body’s tissues and blood.
Lead Product(s): WTX101
Therapeutic Area: Genetic Disease Product Name: ALXN1840
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Alexion Pharmaceuticals
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 27, 2022
Details:
Results from positive FoCus trial in Wilson disease showed that WTX101 (ALXN1840), met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to SoC including in patients who had been treated previously for an average of 10 years.
Lead Product(s): Bis(choline)tetrathiomolybdate
Therapeutic Area: Genetic Disease Product Name: WTX101
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 23, 2022
Details:
Eplontersen (IONIS-TTR-LRX) is a LICA investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.
Lead Product(s): Eplontersen
Therapeutic Area: Genetic Disease Product Name: IONIS-TTR-LRX
Highest Development Status: Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 21, 2022
Details:
ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments.
Lead Product(s): Bis(choline)tetrathiomolybdate
Therapeutic Area: Genetic Disease Product Name: WTX101
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 26, 2021
Details:
The Phase 3 study results published by the JCEM found that although the standard-deviation-score-focused primary endpoint of the study was missed, Chronocort® improved morning and early afternoon biochemical control for adults with CAH over standard glucocorticoid therapy.
Lead Product(s): Hydrocortisone
Therapeutic Area: Genetic Disease Product Name: Chronocort
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 02, 2021
Details:
The submission for the MHRA is based on the data from the Company’s Phase 3 study, an open-label safety extension study of Chronocort® and written formal Scientific Advice received in April 2019 confirming the clinical and regulatory pathway for Chronocort® for CAH treatment.
Lead Product(s): Hydrocortisone
Therapeutic Area: Genetic Disease Product Name: Chronocort
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 12, 2021
Details:
FDA has cleared the company’s Investigational New Drug application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy.
Lead Product(s): OTL-200
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 19, 2020
Details:
Libmeldy TM receives positive CHMP opinion for the treatment of Early-onset metachromatic leukodystrophy (MLD); U.S. IND application on Track for Year End 2020.
Lead Product(s): Libmeldy
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 03, 2020
Details:
The CHMP’s positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant marketing authorization for Libmeldy in the European Union (EU).
Lead Product(s): Libmeldy
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 16, 2020
