Yesterday 10/04, at 7:00 am ET, Sarepta Therapeutics (SRPT) with Dr. Mendell released the biopsy data from the fourth patient including full 4 patients “Functional Data” from Argentina at #WMS2018 the 23rd International Annual World Muscle Society meeting Link to the full slide deck: https://www.sec.gov/Archives/edgar/data/873303/000119312518292068/d616670dex992.htm The after-hours share price reaction was wild swing, spiking up […]
SRPT – Preparing for WMS-2018 Presentation
Tomorrow 10/04, at 7:00 am ET, Sarepta Therapeutics (SRPT) with Dr. Mendell are hosting a conference call from Argentina at #WMS2018 the 23rd International Annual World Muscle Society meeting, to present and discuss new data from the micro-dystrophin gene-therapy study Ph1/2. Link to SRPT webcast page: http://investorrelations.sarepta.com/events-and-presentations Back in June 19th at the R&D day SRPT […]
XENE Pipeline Keep Growing – Is XEN007 The Next Surprise?
Last week, Xenon pharmaceuticals (XENE) passed one of the most important tests in the stock market, where the share price closed on Friday at $14.05, above the $14.03 resistance line, despite the news of the $50 million public offering, followed by the news of upsizing the offering to $63 million at $14.00 per share, only […]
SRPT’s CEO Fireside Chat at Morgan Stanley Conference
Sarepta (SRPT) had a great run last week, a kick off following MS fireside chat, closing at $152.76 the highest “close” since June 21st (highest 52W close 06/20 was $153.69) on nice volume, but the highest “Weekly-Close” in the weekly chart (Close & not highest price point). Looking for a continuation next week, while the target […]
XENE – Quick note, Positive TMS Data Presented at The European Congress on Epileptology
Today morning, Xenon Pharmaceutical (XENE) reported positive data from its XEN1101 Phase 1b transcranial magnetic stimulation (TMS) study. (LINK) XEN1101 Demonstrates Statistically Significant Reduction in Corticospinal and Cortical Excitability as Measured by TMS-EMG and TMS-EEG XEN1101 Demonstrates Greater Effect on TMS-EMG Resting Motor Threshold at Significantly Lower Dose When Compared to Historical Ezogabine Data XEN1101 […]
Bellus – David vs. Goliath Can BLU Deliver A Better Chronic Cough Drug Than Merck?
BELLUS Health (BLU.TO) (BLUSF) is a biopharmaceutical development company based in Quebec, Canada, its pipeline includes several partnered clinical-stage drug development programs, but the company’s lead drug candidate is BLU-5937 which is a highly selective P2X3 antagonist for chronic cough and has the potential to be a best-in-class therapeutic for chronic cough patients who do […]
XENE – Quick note, Q2 Earnings and Epilepsy Long Term Value Creation
Follow up to my previous report “XENE – Epilepsy and Advocacy Under the New FDA” (Link) XENE released their Q2 earning report and hosted a conference call and webcast to discuss the 2Q 2018 financial results and provide corporate update. Xenon pharmaceutical (XENE) story keep evolving, with plenty of cash runway through at least mid-2020, as of […]
XENE – Epilepsy and Advocacy Under the New FDA
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 […]
