Xenon Pharmaceuticals (XENE) is a clinical stage biopharmaceutical company focusing on discovering and developing innovative therapeutics for neurological disorders. XENE has an extensive knowledge of human genetics and diseases caused by mutations in ion channels, known as channelopathies, focusing their efforts on novel product pipeline of CNS-central nervous system therapies, to address areas of high […]
FDA GT Draft Guidance is Bullish for Sarepta's DMD Pivotal Trial
Yesterday July 11, 2018 the FDA FDA issues 6 new draft guidance documents for gene therapy, clinical development and manufacturing guidance. Human Gene Therapy for Rare Diseases – FDA Draft Guidance for Industry https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf The FDA guidance noting the pre-existing antibody to GT, this is the strongest SRPT’s & Dr. Mendell point, choosing the AAVrh74 which […]
SRPT – Building a Precision Genetic Medicine Powerhouse
Sarepta Therapeutics (SRPT) is attempting to build the most meaningful precision genetic medicine company in the world, and has laid out its strategy to investors of exactly how it plans to get there. It starts with Duchenne Muscular Dystrophy (DMD) and the goal of dramatically changing patients’ lives. The company may have just done that. […]
PSTI – Placenta Stem-Cells Brings Hope to PAD-Peripheral Artery Disease Patients
Intermittent Claudication or as called IC is a peripheral artery disease (PAD) caused by fatty deposits that accumulate in the arteries of the leg and reduce blood flow to exercising muscle and is generally a reliable indicator of occlusive arterial disease, which lead to a discomfort, weakness and a leg pain after any effort like exercise, […]
PSTI – Ahead of IC Phase-2 Top-Line Results
PLURISTEM (PSTI) a cell therapy company, developer of placenta-based cell therapy product candidates for the treatment of multiple ischemic, inflammatory and hematologic conditions, company I’m following for years. Pluristem trading under the radar and getting even more interesting these days as they already enter late-stage trials in 3 indications, gained the FDA’s Fast Track Designation, EMA’s […]
What Do You Need To Know Ahead of Next Week – SRPT CHMP Oral-Explanation
The CHMP meeting will take place next week April 23-26, we have no idea what day exactly is SRPT meeting, (Update 04/23: today the CHMP published this week agenda and SRPT Oral-Explanation set on 04/24 at 8:00 EST) . Usually after the Oral-Explanation the CHMP will ask the applicant representatives to step out from the […]
SRPT – Preparing For The CHMP Oral-Explanation
It was an intense week for SRPT’s investors, where the share price bounced from $74.36 (close Friday 03/09) to reach the high of $84.37 this week after the press release from Sarepta getting the green light from the FDA for Golo accelerated approval pathway (AA), and preparing to start the rolling NDA submission. Followed with […]
SRPT Was and Still The DMD Gene Therapy Leader
Today after the market close, Solid Biosciences (SLDB) announced it has received notification from the U.S. Food and Drug Administration (FDA) that IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold. The news sent SLDB share price down to $11.90 or […]
Sarepta Official Statement Regarding The UK Study Halt
This morning, EP Vantage reported a temporary UK study halt in dosing after “one serious adverse event that could possibly be related to the investigational drug product” but the halt was for the UK only and did not include any other clinical trial which include Belgium, Czechia, France, Germany, Israel, Italy, Spain, Sweden, and the United States. […]
Dr. James Wilson Paper About Safety Concerns, Is Not Sarepta’s Concern!
Today morning, most of the gene-therapy stocks including SLDB, AVXS, ONCE, RGNX, BOLD, ABEO and even SRPT Stumbled following Dr. James Wilson paper published online “Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN” (link to the paper) highlighting the toxicity in NHP- non-human primate study. […]
A Quick Technical Look At SRPT Full Year Chart
Sarepta Therapeutics, Inc. (SRPT) Nasdaq A quick look at Sarepta’s yearly chart, from the spike to $63.73 after Exondys-51 FDA approval and the drop to $26.66 lowest point by Dec 30th, the last trading day of 2016. What is important to watch is the big picture, as we are right now above the Fibo-50% retracement […]
SRPT – Preparing For Blast Of Catalysts
Already a year has passed since Sarepta’s first drug Exondys51 was granted FDA approval on 09/19/2016. The landmark decision by Janet Woodcock, despite the review team’s opposition, gave hope to all the kids with DMD (Duchenne Muscular Dystrophy) Exondys51 is in the middle of a great launch, one of the most successful first-year launches of […]
A Deep Dive in SRPT Chart!
Sarepta Therapeutics, Inc. (SRPT) -Nasdaq During the past six weeks we had so many stories, with or against SRPT approval, where a 10-million shares short sellers are still traped in this name with a significant loss after the FDA approval, and they are trying nonstop any way or attack to find a way to make […]
TRIL – A quick update from BloomBurton 2016 conference
Trillium Therapeutics Inc. (TRIL) – Nasdaq Trillium Therapeutics (TRIL) presented at BloomBurton-2016 Healthcare Investor Conference in Toronto on May 3rd, the same as last year the speaker was TRIL’s Chief Scientific Officer (CSO) Dr. Bob Uger, he took the stand in Hall A (the largest room at the conference) that was full with investors, press […]
SRPT – Final thoughts ahead of the AdCom
Reaching the decision point… Today is the day we have to make decisions in SRPT position. As has been explained several times in the forum and CRG twitter private feed, the date of the panel is 04/25 and the FDA BD comes two days before the preliminary panel (2 business days), it should be […]
SRPT – YEAR 4 DATA ANALYSIS
YEAR 4 DATA ANALYSIS Review of Eteplirsen to treat DMD By: @boscaptn, et al. Compiled From SAREPTA Public Data For Week 216 (Year 4) of Study 201/202 for Exon 51 Disclaimer: NO FINANCIAL ADVICE – The Information in this report is provided for education and informational purposes only, without any express or […]
Biotech Inflection Point – Watch List-2
Following my last technical analysis “Biotech Inflection Point – Watch List-1” I will keep adding names to our watch list with some beaten down tickers, we can find many deep value biotech stocks in the recent pullback, some are trading at or very close to their cash level, at this point their assets are […]
Biotech Inflection Point – Watch List-1
Following my last technical analysis “XBI – A Look At The Near and Long Term In The Biotech Sector” I will start adding names to our watch list with some beaten down tickers, we can find many deep value biotech stocks in the recent pullback, some are trading at or very close to their cash […]
XBI – A Look At The Near and Long Term In The Biotech Sector
SPDR S&P Biotech ETF (XBI)–NYSE We will maintain our focus on the XBI as the biotech barometer, many are waiting for the XBI to outperform the IBB again and lead the way to a real recovery, to give us the sign that the technical correction is done and we are back to a […]
The XBI In The Take Or Break Zone!
SPDR S&P Biotech ETF (XBI)–NYSE We will maintain our focus on the XBI as the biotech barometer. After the double bottom around 59 points a month ago, the XBI start printing a higher high and a higher low, the first step in the new uptrend that we are looking for. Looking again at […]
