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Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 25, 2024
Details:
The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Undisclosed
Recipient: JCR Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration March 19, 2024
Details:
AG-10 (acoramidis) is an investigational, next-generation, orally-administered, highly potent, small molecule stabiliser of transthyretin (TTR). It is being evaluated in phase 3 clinical trials for the treatment of Transthyretin Amyloid Cardiomyopathy.
Lead Product(s): Acoramidis Hydrochloride
Therapeutic Area: Genetic Disease Product Name: AG10
Highest Development Status: Phase IIIProduct Type: Small molecule
Recipient: BridgeBio Pharma
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 02, 2024
Details:
Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Lead Product(s): Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Recipient: Pfizer Inc
Deal Size: $1,000.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement September 20, 2023
Details:
The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Undisclosed
Recipient: Verge Genomics
Deal Size: $882.0 million Upfront Cash: $42.0 million
Deal Type: Collaboration September 08, 2023
Details:
Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
Lead Product(s): Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy
Recipient: Pfizer Inc
Deal Size: $1,000.0 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement July 28, 2023
Details:
AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
Lead Product(s): Acoramidis Hydrochloride
Therapeutic Area: Rare Diseases and Disorders Product Name: AG10
Highest Development Status: Phase IIIProduct Type: Small molecule
Recipient: BridgeBio Pharma
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 17, 2023
Details:
ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.
Lead Product(s): Danicopan,Eculizumab,Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: ALXN2040
Highest Development Status: Phase IIIProduct Type: Small molecule
Recipient: AstraZeneca
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 09, 2023
Details:
Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a neurodegenerative disease.
Lead Product(s): Undisclosed
Therapeutic Area: Neurology Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Undisclosed
Recipient: JCR Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration April 03, 2023
Details:
The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.
Lead Product(s): AAV-based Gene Therapy
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Cell and Gene therapy
Recipient: LogicBio Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Acquisition November 16, 2022
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