Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Vaderis Gets FDA Fast Track for VAD044 in Hereditary Hemorrhagic Telangiectasia
Details : VAD044, a daily, oral allosteric AKT inhibitor, which has been investigated in a clinical proof of concept study in HHT patients.
Product Name : VAD044
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 18, 2024
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sapropterin Hydrochloride
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Relief Completes Phase in RLF-OD032 Study; Topline Results Expected October 2024
Details : RLF-OD032, an innovative and highly concentrated liquid formulation of sapropterin dihydrochloride, is designed to lower blood phenylalanine in adult and pediatric PKU patients.
Product Name : RLF-OD032
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 18, 2024
Lead Product(s) : Sapropterin Hydrochloride
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Vaderis Announces Positive Clinical Proof-of-Concept Trial in HHT
Details : VAD044, a daily, oral allosteric AKT inhibitor, which has been investigated in a clinical proof of concept study in HHT patients.
Product Name : VAD044
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
August 27, 2024
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Sapropterin Hydrochloride
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Relief Therapeutics Reports Positive Results from RLF-OD032 Study For Phenylketonuria
Details : RLF-OD032 (sapropterin dihydrochloride) is a PAH agonist small molecule drug candidate which is being evaluated for the treatment of phenylketonuria.
Product Name : RLF-OD032
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
April 10, 2024
Lead Product(s) : Sapropterin Hydrochloride
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Medicxi
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : VAD044 is a once daily, orally administered, allosteric AKT inhibitor which has the potential to treat the underlying cause of Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu Syndrome.
Product Name : VAD044
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
August 22, 2022
Lead Product(s) : VAD044
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Medicxi
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Viking Global Investors
Deal Size : $80.0 million
Deal Type : Series C Financing
Details : The proceeds from the Series C financing will fund the clinical advancement of Pharvaris’ pipeline of novel oral bradykinin-B2-receptor antagonists for the treatment of HAE, including both on-demand treatment and prophylactic prevention.
Product Name : PHVS416
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
November 18, 2020
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Viking Global Investors
Deal Size : $80.0 million
Deal Type : Series C Financing
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : The PK/PD profile suggests that rapid onset of action and prolonged efficacy with a single dose of PHA121 in the treatment of acute HAE attacks. Pharvaris is preparing to start an on-demand study of PHVS416, a soft capsule formulation of PHA121, for HAE ...
Product Name : PHVS416
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 13, 2020
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Onasemnogene Abeparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program
Details : Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (IT) formulation in older patients with SMA to further support registration.
Product Name : Zolgensma
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
September 23, 2020
Lead Product(s) : Onasemnogene Abeparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
PHA121 Shows Clinically Relevant Activity in a Translational in Vivo Model
Details : The objective of the study was to investigate the ability of PHA121 to attenuate blood-pressure changes induced by bradykinin injection.
Product Name : PHVS416
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
September 04, 2020
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Preclinical Characterization of Novel B2-Receptor Antagonists Published in Frontiers in Pharmacology
Details : The data set found in the publication demonstrates the preclinical profile of the new class of molecules relative to icatibant, an injectable B2-receptor antagonist used as the leading therapy for on-demand treatment of HAE.
Product Name : PHVS416
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
July 30, 2020
Lead Product(s) : Deucrictibant
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable