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Details:
The data demonstrate that PHA121 (aka PHVS416), is 25-fold more potent than icatibant at inhibiting bradykinin interaction with the endogenous human B2 receptor.PHA121 is a highly potent, specific, and orally bioavailable competitive antagonist of the bradykinin B2 receptor.
Lead Product(s):
PHVS416
Therapeutic Area: Genetic Disease
Product Name: PHA121
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
September 16, 2022
Details:
VBP15 (vamorolone) met its primary endpoint by demonstrating statistically significant and clinically relevant improvement in time to stand from floor compared to placebo, the first functional milestone to deteriorate in young children with DMD.
Lead Product(s):
Vamorolone
Therapeutic Area: Genetic Disease
Product Name: VBP15
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
September 01, 2022
Details:
Branaplam (LMI070), is being developed as a potential first in class orally administered disease modifying therapy for HD. Branaplam has the potential to address this unmet medical need.
Lead Product(s):
Branaplam
Therapeutic Area: Genetic Disease
Product Name: LMI070
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
August 25, 2022
Details:
VBP15 (vamorolone) is a drug candidate with a mode of action that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is considered a dissociative anti-inflammatory drug.
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
August 22, 2022
Details:
PHVS416/PHA121 (PHA-022121) is a highly potent, specific, and orally bioavailable competitive antagonist of the bradykinin B2 receptor that has completed Phase 1 clinical development for the treatment of HAE.
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
August 22, 2022
Details:
Trial met primary endpoint demonstrating statistically significant and clinically meaningful reductions across all doses (63% at the 27mg QW dose; p<0.001) in triglycerides (TG) from baseline; results were consistent in patients on or not on background therapy.
Lead Product(s):
Vamorolone
Therapeutic Area: Genetic Disease
Product Name: VBP15
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 29, 2022
Details:
VBP15 (Vamorolone) is a drug candidate with a novel mode of action that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative agonist.
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
June 09, 2022
Details:
The trial met its primary endpoint of superiority in change of time to stand from supine position (TTSTAND) velocity with vamorolone, a dissociative agonist 6 mg/kg/day versus placebo (p=0.002) at 24 weeks (period 1).
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
March 29, 2022
Details:
The preclinical data demonstrate PHA121 specificity and potency for both recombinant and endogenous B2 receptor, including 20-fold higher potency than icatibant, an approved injectable B2-receptor antagonist.
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
March 22, 2022
Details:
Evidence of clinical efficacy and safety of vamorolone in patients with DMD is based on the positive outcome of the pivotal Phase 2b VISION-DMD study and additional Phase 2a data covering a vamorolone treatment period of up to 30 months.
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
March 15, 2022
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