Albireo Pharma announced that results in Alagille syndrome and biliary atresia patients from its completed Phase 2 clinical trial evaluating A4250 in pediatric cholestasis were presented at the European Association for the Study of the Liver The International Liver Congress 2019 in Vienna, Austria. A4250 is a highly potent and selective inhibitor of the ileal bile acid transporter currently being studied in a Phase 3 clinical trial in children with progressive familial intrahepatic cholestasis, a life-threatening, rare cholestatic liver disease. Albireo plans to initiate a second A4250 pivotal trial, in biliary atresia, in the second half of 2019. The open-label, multicenter, dose-finding Phase 2 clinical trial assessed the safety and tolerability of A4250 in 20 children with cholestatic liver diseases, including PFIC, Alagille syndrome and biliary atresia. Efficacy endpoints included change in serum bile acid levels and pruritus. A4250 was administered orally in doses ranging from 10 microg/kg to 200 microg/kg once daily for 4 weeks. A4250 demonstrated marked sBA reductions of up to 92% in the majority of Alagille patients. The majority of Alagille patients also showed improvement in pruritus as measured by three different scales. One patient had an elevation in bile acids versus baseline. No effect was observed in a third patient with a low baseline sBA. These data, combined with the total dataset of n =24, form the basis for a pivotal development program in a second indication, biliary atresia. In patients with Alagille and biliary atresia, A4250 was generally well-tolerated. Adverse events, including some increased transaminases, were mild and transient. Two Alagille patients with high baseline transaminase levels experienced further increases, which informed the decision to not dose escalate.
Sunday, April 14, 2019
Summit Therapeutics presents in vivo PoC data for new mechanism antibiotics
Summit Therapeutics presented in vivo proof of concept data for the DDS-04 series of new mechanism antibiotics targeting Enterobacteriaceae in an oral session at the 29th European Congress of Clinical Microbiology & Infectious Diseases in Amsterdam. Enterobacteriaceae are a family of Gram-negative bacteria responsible for a large number of severe and often deadly infections. In the data presented, a DDS-04 series compound cured infection in a translationally-relevant animal model of urinary tract infection, one of the major sites for Enterobacteriaceae infection. Therapeutic concentrations of the DDS-04 compound were also observed in other major sites in the animal model where life-threatening Enterobacteriaceae infections occur, including the lungs and the bloodstream. With its new mechanism of action, the DDS-04 series was rapidly bactericidal and highly potent across globally diverse Enterobacteriaceae strains in research studies, which included multi-drug resistant isolates. Importantly, the DDS-04 series also showed low propensity for resistance development and did not show cross-resistance with existing classes of antibiotics, suggesting that DDS-04 compounds have the potential to overcome known resistance mechanisms. The company believes this profile makes the DDS-04 series “attractive” for further development for the treatment of Enterobacteriaceae infections.
Clovis price target lowered to $21 on ATLAS trial at Piper Jaffray
Piper Jaffray analyst Joseph Catanzaro lowered his price target for Clovis to $21 from $23 after the company announced that it was discontinuing the Phase II ATLAS trial of rucaparib in urothelial bladder cancer due to lack of efficacy. The analyst notes that this outcome is “not totally surprising” to him given the limited efficacy observed thus far for PARP inhibitors in the therapeutic setting outside of BRCA+. Catanzaro reiterates a Neutral rating on the shares.
Catalent to acquire Paragon Bioservices for $1.2B
Catalent has agreed to buy closely held Paragon Bioservices for $1.2B, The Wall Street Journal’s Jared Hopkins and Cara Lombardo report, citing people familiar with the matter. The all-cash deal, which is expected to be announced Monday morning, would help Catalent expand its gene-therapy manufacturing capabilities, sources said. The transaction is expected to close in the second quarter, the publication adds
Assembly Biosciences Presents Interim Data from Two Phase 2a Hep B Studies
Favorable safety and tolerability profile– Superior antiviral activity with ABI-H0731 in combination with nucleos(t)ide therapies– Selected for inclusion in “Best of ILC” presentation– Company to host conference call Monday, April 15, 2019 at 8am EDT
Assembly Biosciences, Inc. (NASDAQ: ASMB), a clinical-stage biotechnology company developing innovative therapeutics targeting hepatitis B virus (HBV) and diseases associated with the microbiome, presented interim results from two Phase 2a clinical trials of ABI-H0731 (731), a novel antiviral in development for the treatment of chronic HBV infection. The data were presented during a late-breaker oral session on Saturday, April 13, 2019, at The International Liver Congress™(ILC), the Annual Meeting of the European Association for the Study of the Liver (EASL) in Vienna, Austria. The late-breaker abstract was also selected for inclusion in the ‘Best of ILC’ presentation as well as for press release coverage at ILC 2019. A copy of the presentation will be posted to the Events & Presentations page in the Investors section of the company’s website at assemblybio.com.
“This interim analysis of two Phase 2a studies supports that 731 in combination with nucleos(t)ide therapy (Nucs) demonstrates rapid and enhanced anti-HBV activity,” said Dr. Jacob Lalezari, of Quest Clinical Research in San Francisco. “The data we have seen thus far are directionally correct and decreases in HBeAg and HBsAg have been observed in some individuals in both studies. The accelerated decline and significant loss of baseline RNA and DNA viremia suggest that combination therapy with a core inhibitor + Nuc has the potential to significantly advance treatments for patients with HBV.”
Saturday, April 13, 2019
Diabetic cardiomyopathy biotech Applied Therapeutics files for $86 million IPO
Applied Therapeutics, a Phase 2 biotech developing therapies for diabetic cardiomyopathy, filed on Friday with the SEC to raise up to $86 million in an initial public offering.
The New York, NY-based company was founded in 2016 and plans to list on the Nasdaq under the symbol APLT. Citi, Cowen and UBS Investment Bank are the joint bookrunners on the deal. No pricing terms were disclosed.
Heart rate condition biotech Milestone Pharmaceuticals files for $86M IPO
Milestone Pharmaceuticals, a Phase 3 biotech developing therapies for heart rate conditions, filed on Friday with the SEC to raise up to $86 million in an initial public offering.
The Montreal, Canada-based company was founded in 2003 and plans to list on the Nasdaq under the symbol MIST. Milestone Pharmaceuticals filed confidentially on December 21, 2018. Jefferies, Cowen and Piper Jaffray are the joint bookrunners on the deal. No pricing terms were disclosed.
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