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Lead Product(s): PHA121
Therapeutic Area: Genetic Disease Product Name: PHVS416
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 26, 2021
Details:
PHVS416 is a softgel capsule formulation containing PHA121. RAPIDe-1 is a Phase 2 study evaluating the efficacy and safety of orally administered PHVS416 for the acute treatment of attacks in patients with HAE type 1 or 2.
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Lead Product(s): Allogeneic cardiosphere-derived cells
Therapeutic Area: Genetic Disease Product Name: CAP-1002
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Capricor Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration January 12, 2021
Details:
Capricor to leverage Lonza’s expertise in technology transfer and development of cellular therapies to take CAP-1002 through potential product launch and commercial supply.
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Lead Product(s): Vamorolone
Therapeutic Area: Genetic Disease Product Name: VBP15
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Highbridge Capital Management
Deal Size: $16.5 million Upfront Cash: Undisclosed
Deal Type: Financing November 02, 2020
Details:
Vamorolone, the only dissociative steroid in development, is currently in a pivotal Phase 2b study in DMD patients with a 6-month readout expected in the second quarter of 2021.
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Lead Product(s): Vamorolone
Therapeutic Area: Genetic Disease Product Name: VBP15
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: ReveraGen BioPharma
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 14, 2020
Details:
Data further show that vamorolone uniformly weakens co-activator associations, which leads to loss of gene transcriptional activities associated with safety concerns of corticosteroids.
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Lead Product(s): Vamorolone
Therapeutic Area: Genetic Disease Product Name: VBP15
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Santhera Pharmaceuticals
Deal Size: $11.4 million Upfront Cash: Undisclosed
Deal Type: Licensing Agreement September 02, 2020
Details:
Santhera has obtained an exclusive license from ReveraGen, the originator of vamorolone, for all indications worldwide. Vamorolone is currently being investigated in the pivotal Phase 2b VISION-DMD study in patients with Duchenne muscular dystrophy (DMD).
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Lead Product(s): ER-004
Therapeutic Area: Genetic Disease Product Name: ER-004
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 06, 2020
Details:
The Breakthrough Therapy Designation was granted following the promising results from three XLHED subjects who were treated by Professor Holm Schneider with a course of ER-004 intra-amniotic injections during the third trimester of pregnancy.
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Lead Product(s): Vamorolone
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 02, 2020
Details:
Data from this VBP15-003 study in comparison to natural history study data demonstrated dose-dependent improvement in timed function tests. Vamorolone was reported to be safe and well tolerated up to the highest dose tested (6.0 mg/kg/day).
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