Breaking Biotech

Today, I breakdown Sickle Cell Disease and Global Blood Therapeutics' Voxelotor. I write a more detailed article here: http://breakingbiotech.com/2018/11/13/026-global-blood-therapeutics-meager-approval-attempt/ Follow me on twitter @matthewlepoire Relevant Press Releases: http://ir.gbt.com/phoenix.zhtml?c=254105&p=irol-newsArticle&ID=2215253 http://ir.gbt.com/phoenix.zhtml?c=254105&p=irol-newsArticle&ID=2227616 http://ir.globalbloodtx.com/phoenix.zhtml?c=254105&p=irol-newsArticle&ID=2356168 Relevant Links: https://www.ncbi.nlm.nih.gov/pubmed/26758919 https://www.statnews.com/2018/09/13/global-blood-therapeutics-sickle-cell-fda/ gbt #sicklecelldisease

Today I go through the subgroup analyses of BAN2401 from Biogen/Eisai, the latest Esperion p3 data and the disappointing results from Adamas' Q3 results. This is not investment advice, but only for information purposes. Follow me @matthewlepoire www.breakingbiotech.com biib #espr #adms

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 22 pt move in the stock by Oct 26th. The CTAD 2018 presentation schedule can be found here: https://www.eisai.com/news/2018/news201886.html (Note: I said 24th in the video but it's really the 25th) Other relevant links: https://www.eisai.com/ir/library/presentations/pdf/4523_180726.pdf https://alzres.biomedcentral.com/articles/10.1186/s13195-016-0181-2 https://www.ncbi.nlm.nih.gov/pubmed/29317609 https://www.ncbi.nlm.nih.gov/pubmed/27582220 https://www.ncbi.nlm.nih.gov/pubmed/23828104 This is not investment advice Follow me @matthewlepoire www.breakingbiotech.com

-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 study-design proposed by TRVN including endpoints and statistical analyses. -Press releases from TRVN in the past say different than the adcom documents, which many firms are taking up as class action lawsuits...especially given TRVN's latest 8-K filing I do not have any position in the company and this is not investment advice. Follow me on twitter @matthewlepoire www.breakingbiotech.com Relevant links: http://www.trevena.com/news-details.php?id=211 https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AnestheticAndAnalgesicDrugProductsAdvisoryCommittee/UCM622730.pdf http://www.trevena.com/news-details.php?id=212

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 taken a position and this is not investment advice. Follow me @matthewlepoire www.breakingbiotech.com Relevant links: https://www.sarepta.com/our-pipeline https://www.sec.gov/Archives/edgar/data/873303/000119312518292068/d616670dex992.htm

-Amarin's EPA medicine, Vascepa, recently showed a 25% relative risk reduction in newly announced data from the cardiovascular outcomes trial, REDUCE-IT. -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. this is not investment/trading advice Follow me @matthewlepoire www.breakingbiotech.com 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

Viking Therapeutics reports their latest phase 2 data with their candidate thyroid hormone receptor β agonist. I compare both the madrigal and viking drug and discuss their potential in the NASH/NAFLD space. This is not investment advice Relevant links: http://ir.vikingtherapeutics.com/2018-09-18-Viking-Therapeutics-Announces-Positive-Top-Line-Results-from-Phase-2-Study-of-VK2809-in-Patients-with-Non-Alcoholic-Fatty-Liver-Disease-NAFLD-and-Elevated-LDL-Cholesterol https://clinicaltrials.gov/ct2/show/NCT02912260?term=madrigal&rank=4 https://clinicaltrials.gov/ct2/show/NCT02927184?term=viking+therapeutics&rank=1 https://www.madrigalpharma.com/wp-content/uploads/2018/09/Madrigal-Company-Overview-September-2018.FINAL_.20180903.pdf

-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 in the blood/urine. The uGAG measurements are not the breakdown products but the actual targets of IDS, which are what is measured in the blood/urine. -Around 3:30 I talk about the expression of the transgene. The AAV will circulate and infect a variety of cells. The gene editing will take place after albumin promoter of the genome, which is only active in hepatocytes. Hepatocytes will be the only cell type to express the transgene, and the product will circulate in the blood to other tissues. This is not investment advice Relevant links: https://www.sangamo.com/ https://seekingalpha.com/article/4205472-depth-analysis-sbminus-913-early-clinical-readout-mps-ii https://seekingalpha.com/article/4205037-sangamo-work https://doi.org/10.1016/j.ymthe.2018.03.002 https://twitter.com/gcbioinv/status/1038424280047013888 https://www.ncbi.nlm.nih.gov/pubmed/23837440 Follow me @matthewlepoire See the blogpost at www.breakingbiotech.com

Ionis Pharma has brough antisense therapy to the clinic. Unfortunately, they recently received a complete response letter from the FDA regarding their latest medicine, Waylivra for the treatment of familial chylomicronemia syndrome. In this video, I talk about antisense therapy and the potential way forward for the company. For a complete writeup, check out the post here - Follow me on twitter @matthewlepoire this is not investment advice Links: https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM606857.pdf http://ir.ionispharma.com/news-releases/news-release-details/akcea-and-ionis-receive-complete-response-letter-waylivra-fda http://ir.ionispharma.com/static-files/7e19a292-b451-41ef-9e14-4449962c6464

-Esperion Therapeutics hopes to bring their LDL-c lowering drug, Bempedoic Acid, to the market -Their latest data shows a slight increase in patient deaths, which has pushed the stock lower, but I don't believe this to be meaningful -PCSK9 inhibitors are very expensive, and ESPR believes they can charge a lower price and attract a large number of patients -The FDA may require a large-scale cardiovascular outcomes trial that will delay full approval of BA -Follow me @matthewlepoire Relevant links: http://investor.esperion.com/static-files/88da95da-bb52-4e7e-a9dd-1d52e70f3a6b https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4719137/pdf/ahdb-08-483.pdf https://www.nature.com/news/the-fda-chief-must-not-be-a-proxy-for-industry-1.21661 This is not investment/trading advice