Aridis Pharma’s AR-105 flunks mid-stage study

Ultra-thinly traded micro cap Aridis Pharmaceuticals (NASDAQ:ARDS) slumps 27% premarket on light volume in reaction an unsuccessful Phase 2 clinical trial evaluating candidate AR-105, a fully human IgG1 monoclonal antibody, for the treatment of ventilator-associated pneumonia caused by Gram-negative Pseudomonas aeruginosa.

AR-105 failed to sufficiently separate from placebo in terms of clinical cure rate at day 21. There were also statistically significant imbalances in all-cause mortality and serious adverse events (SAEs) between the treatment and placebo arms, although no SAE or death was determined to be related to AR-105.

The company will not allocate any further resources to development.

https://seekingalpha.com/news/3496240-aridis-pharmas-arminus-105-flunks-mid-stage-study-shares-27-percent-premarket

IGM Bio sets IPO terms

IGM Biosciences (IGMS) plans to offer 7,812,500 common shares at $15 – 17 in its IPO. Gross proceeds should be $125M.

https://seekingalpha.com/news/3496252-igm-bio-sets-ipo-terms

SmileDirectClub sets terms of upsized IPO

SmileDirectClub (SDC) plans to offer 58,537,000 Class A common shares at $19 – 22 in its IPO. Gross proceeds should be ~$1.2B.

Related ticker: Align Technology (NASDAQ:ALGN)

https://seekingalpha.com/news/3496253-smiledirectclub-sets-terms-upsized-ipo

Vertex to acquire Semma Therapeutics for $950M

Vertex Pharmaceuticals (NASDAQ:VRTX) has agreed to acquire privately held Semma Therapeutics, a developer of stem cell-derived human islets for the potential cure of type 1 diabetes, for $950M in cash.

The transaction should close next quarter.

https://seekingalpha.com/news/3496272-vertex-acquire-semma-therapeutics-950m

Aeglea Bio reports positive data for pegzilarginase in deficiency patients

Aeglea BioTherapeutics (NASDAQ:AGLE) has presented positive 20-dose data on 14 patients from its completed Phase 1/2 trial and ongoing Phase 2 open-label extension (OLE) trial for pegzilarginase in patients with Arginase 1 Deficiency (ARG1-D) at the 2019 Symposium of the Society for the Study of Inborn Errors of Metabolism.

Data from all patients continued to demonstrate marked and sustained reductions in plasma arginine.

79% (11 of 14) of patients were clinical responders, using mobility assessment components that correspond with the pivotal PEACE trial secondary endpoint.

Pegzilarginase was well tolerated and the rates of treatment-related adverse events decreased over time.

The Company expects to continue discussions with the FDA on the pegzilarginase program and next steps in Q4 2019 or Q1 2020.

Topline data from the PEACE trial is expected in Q1 2021.

https://seekingalpha.com/news/3496275-aeglea-bio-reports-positive-data-pegzilarginase-patients-argi-d

UniQure achieves planned enrollment in HOPE-B trial of AMT-061

UniQure N.V. (NASDAQ:QURE) announces that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.

Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September.

The study’s primary endpoint is the assessment of Factor IX activity 26 weeks after dosing.

Patients enrolled in the HOPE-B trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers.

https://seekingalpha.com/news/3496320-uniqure-achieves-planned-enrollment-hope-b-trial-amtminus-061

Emergent Bio awarded 10-year HHS contract to deliver ACAM2000

Emergent BioSolutions (NYSE:EBS) has been awarded a contract by the Office of the Assistant Secretary for Preparedness and Response (ASPR) in the U.S. Department of Health and Human Services (HHS) valued at ~$2B over 10 years for the continued supply of ACAM2000, (Smallpox Vaccine, Live) into the U.S. Strategic National Stockpile (SNS).

ACAM2000 is the only vaccine licensed by the FDA for active immunization against smallpox disease.

This contract for the procurement of ACAM2000 vaccine consists of a one-year base period of performance, valued at ~$170M, and nine option years.

The company expects to deliver a majority of the doses under the base year by end of 2019. This multiple year contract is intended to support the replacement of the smallpox vaccine stockpile and follows the conclusion of a prior 10-year, $425M contract to establish domestic warm-base manufacturing capabilities and procure smallpox vaccines issued by the U.S. government April 1, 2008.

https://seekingalpha.com/news/3496322-emergent-bio-awarded-10-year-hhs-contract-deliver-acam2000