Sanofi to present data on Pompe enzyme replacement therapy June 16

Sanofi (NASDAQ:SNY) will host a virtual scientific session on Tuesday, June 16, at 8:00 am ET to present Phase 3 data on enzyme replacement therapy avalglucosidase alfa in patients with late-onset Pompe disease.

Pompe is an inherited disorder caused by the buildup of glycogen in the body’s cells due to mutations in the gene that codes for an enzyme called acid alpha-glucosidase.

https://seekingalpha.com/news/3580997-sanofi-to-present-data-on-pompe-enzyme-replacement-therapy-june-16

Astellas’ roxadustat successful in late-stage anemia study

Astellas Pharma (OTCPK:ALPMF) announces positive results from a Phase 3 clinical trial, DOLOMITES, evaluating roxadustat for the treatment of anemia in non-dialysis-dependent adults with stage 3-5 chronic kidney disease. The data were virtually presented at the European Renal Association-European Dialysis and Transplant Association Congress.

The study demonstrated the non-inferiority (no worse than) of roxadustat to Amgen’s (NASDAQ:AMGN) Aranesp (darbepoetin alfa) as measured by hemoglobin response up to week 24. Specifically, the change in hemoglobin in the roxadustat cohort was 89.5%, compared to 78.0% in the darbepoetin alfa cohort.

Roxadustat demonstrated superiority over darbepoetin alfa in reducing low-density lipoprotein cholesterol (“bad” cholesterol), a secondary endpoint.

The safety profiles were similar.

The company’s marketing application in Europe is under EMA review.

Roxadustat is an orally administered small molecule inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase. HIF is a protein transcription factor that “turns on” the production of red blood cells (erythropoiesis). Its value proposition is the ability to maintain hemoglobin levels without affecting inflammation and potentially avoiding the need for ongoing intravenous iron repletion therapy as needed with bone marrow stimulating agents like Aranesp and Johnson & Johnson’s (NYSE:JNJ) Procrit (epoetin alfa).

Related tickers: FibroGen (NASDAQ:FGEN), AstraZeneca (NYSE:AZN)

https://seekingalpha.com/news/3581007-astellas-roxadustat-successful-in-late-stage-anemia-study

Boston Scientific nabs Medicare reimbursement for Exalt duodenoscope

The U.S. Centers for Medicare and Medicaid Services (CMS) has signed off on a transitional pass-through (TPT) payment category for single-use endoscopes, including Boston Scientific’s (NYSE:BSX) EXALT Model D Single-Use Duodenoscope (a lighted tube that allows a doctor to visually inspect the small intestine). The code will be effective July 1.

The intent of TPT payment allows for reimbursement, while cost data are collected for the inclusion of the device in the procedure Ambulatory Payment Classification (APC) rate.

https://seekingalpha.com/news/3581014-boston-scientific-nabs-medicare-reimbursement-for-exalt-duodenoscope

Bavarian Nordic encephalitis vaccine shows positive effect in early-stage study

Bavarian Nordic A/S (OTCPK:BVNKF) announces topline results from a Phase 1 study evaluating MVA-BN WEV, a prophylactic vaccine candidate against three equine encephalitis viruses, in 45 healthy adult volunteers.

The vaccine was well-tolerated with neutralizing antibody responses observed across all dose groups. Peak levels were reached after the second vaccination.

The most common adverse event was injection site pain.

The company is in the process of securing additional funding to advance development.

Last year there were 38 cases in the U.S. that resulted in 15 fatalities. There are usually ~seven cases each year in America, most in the Northeast where mosquitoes have been found to be the vector.

https://seekingalpha.com/news/3581042-bavarian-nordic-encephalitis-vaccine-shows-positve-effect-in-early-stage-study

AstraZeneca’s Calquence shows positive effect in hospitalized COVID patients

AstraZeneca (NYSE:AZN) announces positive preliminary data from a Phase 1 clinical trial evaluating BTK inhibitor Calquence (acalabrutinib) in 19 hospitalized severely ill COVID-19 patients. The results were recently published in Science Immunology.

11 patients were receiving supplemental oxygen and eight were on mechanical ventilation. All had increasing oxygen requirements at baseline.

Over a 10-14-day treatment period, oxygenation improved in the majority of patients with no discernable toxicity. Measures of inflammation normalized rapidly, including lymphopenia (low levels of a type of white blood cell called lymphocytes). 72.7% (n=8/11) of patients in the supplemental oxygen cohort were discharged on room air. 50% (n=4/8) of patients in the mechanical ventilation group were successfully extubated (breathing tube removed) and 25% (n=2/8) were discharged on room air.

Phase 2 studies are in process.

https://seekingalpha.com/news/3581055-astrazenecas-calquence-shows-positive-effect-in-hospitalized-covid-patients

Veeva gets Street-high price target

Noting Veeva’s (NYSE:VEEV) “enviable financial model,” Citi analyst Tyler Radke starts the company with a Buy rating and a $265 price target, a 31% upside.

Radke notes the 20-30% Y/Y revenue growth and strong profitability, which should help drive strong returns for Veeva.

The analyst mentions that Veeva’s SaaS suite targets the “lucrative” life sciences market.

Radke compares Veeva to Salesforce’s growth story, but he sees stronger profits for Veeva.

https://seekingalpha.com/news/3581061-veeva-gets-street-high-price-target

Enochian Biosciences up big on advancement of potential HIV cure

Thinly traded micro cap Enochian Biosciences (NASDAQ:ENOB) rockets 292% premarket on robust volume in reaction to its announcement that it has completed a meeting with the FDA during which there was “strong alignment” between its planned development strategy for ENOB-HV-01 and comments from agency reviewers.

It plans to file an IND next year.

The company says ENOB-HV-01 is a novel approach to autologous stem cell transplantation that may be able to cure HIV via increasing engraftment of gene-modified cells that are resistant to HIV infection.

https://seekingalpha.com/news/3581065-enochian-biosciences-up-big-on-advancement-of-potential-hiv-cure