026 – Global Blood Therapeutics’ Meager Approval Attempt

Sickle cell disease (SCD) is a genetic condition whereby mutated hemoglobin has a tendency to form polymers after unloading oxygen. These polymers cause red blood cells to form a ‘sickle’ shape and increases their likelihood of sticking to the blood vessel wall, causing vaso-occlusive crises (VOCs), stroke, or other serious problems. VOCs are painful episodes […]

Does BAN2401 Actually Work? Upcoming Subgroup Analyses

Biogen/Eisai to present updated subgroup analyses on their Phase 2 study looking at their Alzheimer’s medicine, BAN2401. This antibody binds to amyloid beta protofibrils, reducing plaques in the brain. In this video, I go through Biogen’s original data and talk about the problems parsing the Apoe E4+ patients. The options market is pricing in a […]

Trevena lied to investors? Fails advisory committee

-Trevena’s morphine competitor, Oliceridine, just received negative news from the FDA Advisory Committee. The FDA PDUFA date is November 2nd, but they are likely to follow the adcom’s decision -It also look like Trevena mislead investors by announcing they had successful meetings with the FDA, while the FDA claims they had many problems with the […]

Sarepta Declines on Good Micro-Dystrophin Data

Sarepta took a hit from their latest Micro-Dystrophin gene therapy trial update despite showing robust expression in all 4 patients, confirmed by protein analyses and behavioral improvements. In this video, I go through the details of muscular dystrophy, the current treatment options and how this new therapy could significantly improve patients outcomes. I have not […]

Amarin’s Vascepa Success

-Amarin’s EPA medicine called Vascepa recently showed a 25% relative risk reduction in newly announced data from a CardioVascular Outcomes Trial. -Today I breakdown the results and talk about how it compares to other CVOT data and whether Amarin is likely to rival companies with a LDL-c lowering drug pipelines. Relevant links: http://investor.amarincorp.com/node/15741/pdf http://investor.amarincorp.com/system/files-encrypted/nasdaq_kms/assets/2018/09/27/10-23-58/AMRN%20Investor%20Deck%20Sept%20Post%20R-IT%20v3.pdf https://www.ncbi.nlm.nih.gov/pubmed/29935936 […]

Sangamo Therapeutics drops after SB-913 update

-Sangamo Therapeutics recently announced their Phase 1/2 data for the treatment of MPS II/Hunter Syndrome -The market reacted negatively to the news because of the uncertainty associated with unclear IDS activity and unclear metrics for successful approval by the FDA I misspoke a couple times: -Around 2:00 I say the breakdown products can be detected […]

I was wrong about Zogenix! FDA updates guidelines for gene therapy.

Note: I meant to say that sub-1% factor activity is SEVERE hemophilia. Normal is from 50% to 150%. Today I talk again about Zogenix because I was wrong about Study 2 being priced in! Congrats to the ZGNX holders. I also talk about the new FDA guidelines on gene therapy for hemophilia and its implications […]