Yesterday July 11, 2018 the FDA FDA issues 6 new draft guidance documents for gene therapy, clinical development and manufacturing guidance. Human Gene Therapy for Rare Diseases – FDA Draft Guidance for Industry https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf The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 […]
Dave Trading – May 2018
*** David Sobek is a disciplined value investor. Using models, research, and fundamental data, David devises a value for biotech stocks, buys when significantly undervalued, and sells when significantly overvalued.*** As a reminder, this is my personal guide. You can alter it in accordance with your risk tolerance. >3 Standard deviations – No position 2-3 […]
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 […]
Celldex: Quick Take
Celldex has struggled for a couple years now since its brain cancer drug Rintega failed in 2016. In Q2 this year, results from a pivotal study of the gpNMB antibody drug conjugate, Glembatumumab Vedotin, may change the company’s fate. Glembatumumab is being tested in women with triple negative breast cancer with >25% gpNMB expression. Patients […]
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. […]
Achaogen: Is It A Buy?
South San Francisco based Achaogen is on the verge of gaining FDA approval for its bacteria fighting drug Plazomicin. The decision date has been set for June 25 with an Advisory Committee (AdCom) meeting likely to occur in the April-May timeframe. If the rulings are positive, the drug will be launched this year. Plazomicin is […]
The Kodak Hail Mary
Kodak is just the latest in a series of companies that rescued a failing stock price by announcing a crypto-currency scheme. While there is a Kodak crypto-currency that will be launched, the more interesting part is the selling of ASIC bitcoin mining machines. The Kodak pitch is that you front $3400 for the machine and […]
What is the Difference (Crypto-Currencies)
What’s the Difference? There are hundreds and soon to be thousands of crypto-currencies. It is impossible to know them all but I want to highlight some of the important aspects of crypto-currencies in general and how these differ among the larger currencies. While interesting in terms of organizing the information, it also helps highlight important […]
What Drives the Bitcoin Market?
What is Bitcoin? To answer that question we need a good analogy as to what bitcoin actually is or what it is supposed to do. Bitcoin as a Digital Currency This is actually a more difficult question to answer that you think and the answer has implications as to what the appreciation means and how […]
The Bitcoin Bubble
Bitcoin is a bubble. Bitcoin is overvalued. There is a lot of hysteria about bitcoin but that is from both those drinking the koolaid as well as those screaming bubble all the time. It is easy to look smart and snarky and call it a bubble but I refuse to believe the world is that […]
Dave-Trading – December 2017
*** David Sobek is a disciplined value investor. Using models, research, and fundamental data, David devises a value for biotech stocks, buys when significantly undervalued, and sells when significantly overvalued.*** As a reminder, this is my personal guide. You can alter it in accordance with your risk tolerance. >3 Standard deviations – No position 2-3 […]
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 […]
Dave-Trading October 2017
*** David Sobek is a disciplined value investor. Using models, research, and fundamental data, David devises a value for biotech stocks, buys when significantly undervalued, and sells when significantly overvalued.*** As a reminder, this is my personal guide. You can alter it in accordance with your risk tolerance. >3 Standard deviations – No position 2-3 […]
Dave-Trading – September 2017
*** David Sobek is a disciplined value investor. Using models, research, and fundamental data, David devises a value for biotech stocks, buys when significantly undervalued, and sells when significantly overvalued.*** As a reminder, this is my personal guide. You can alter it in accordance with your risk tolerance. >3 Standard deviations – No position 2-3 […]
