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FDA GT Draft Guidance is Bullish for Sarepta's DMD Pivotal Trial
Yesterday July 11, 2018 the FDA FDA issues 6 new draft guidance documents for gene therapy, clinical development and manufacturing guidance. Human Gene Therapy for Rare Diseases – FDA Draft Guidance for Industry https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf The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. […]
June 21 Biotech Update
Sector strength continued and is clearly outperforming the broader market. While we have seen this happen for a couple days before, this feels more like a real breakout. Ideally, we would need to pullback and test the previous resistance and have it act as support to confirm the move but I suspect it would hold. […]
June 20 Biotech Update
You would think replacing a computer would be an easy job, it apparently requires different layers of people and so not everyone was on campus yesterday. So at some point in the future I have to look forward to the computer replacement process to eat half of my day. In any case, at least the […]
June 19 Biotech Update
The trade war is taking a lot of the headlines and I am running out of time to write as they are replacing my computer soon, which will likely take a lot more time that it reasonably should but I wanted to write a quick note. 1. SRPT hit a grand slam. Not only did […]
June 18 Biotech Update
Nothing new to start the week outside of a pretty weak market. I am not sure what the catalyst outside of an extended market but it looks again like the sector is hitting resistance and unable to break out. Perhaps there is continued disappoint in term of the lack of deals but regardless of the […]
June 13 Biotech Update
The sector had a decent day, although participation was not great. We seem to be on the verge of breaking higher (at least in the XBI) but it will be difficult to do unless the rally is broader in its scope. Of course, one cannot complain too much given that we are generally moving higher […]
June 6 Biotech Update
The sector did a little better yesterday but given the strength of the market, it is not a great performance. That being said another couple days in the green and perhaps this year will be different and we will not see the post-ASCO selloff. Obviously it is early but something to keep an eye on […]
June 1 Biotech Update
Happy ASCO days. The first day tends to be slow as news as it is information sessions, although later in the day we get some important CART data. In any case, by Monday we should have some interesting data to dissect. 1. CELG and BLUE have their BCMA CART data this evening. Expectations are high […]
Star Wars Day Biotech Update
We end the week on an OK set of news. It could certainly have been worse but nothing that is likely to fundamentally revalue the sector or reverse the chop. 1. SRPT finally reported the negative trend vote that most expected. As I also expected, there was a lot of notes talking about the expected […]
May 3 Biotech Update
There was a relative lull in the news yesterday and today but I would argue that the sector held up pretty well despite the GILD miss. That either means that GILD is less meaningful for the sector or that the drumbeat of disappointing earnings has priced in pretty negative views of the sector. In any […]
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 […]
April 2 Biotech Update
Thursday was not a bad follow through for the sector. I am still not convinced that we are out of the forest quite yet but we are at least further away from the abyss that was staring at the sector earlier last week. This week looks to be quiet to start as there is not […]
March 21 Biotech Update
You would think the sector would have given us some more clarity by now but it still seems on the verge of breaking out or breaking down. The XBI could have put in a double top or is setting up a cup and handle pattern. Sentiment is not great as the reaction to Trump’s talk […]
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 […]
March 12 Biotech Update
An interesting start to the week thanks to the weekend news. I thought there would be a little more price action after the news but it is still early and perhaps this just signals how far PCSK9 has moved from investor interest. In any case, there are some interesting pin actions from the news. 1. […]
February 9 Biotech Update
The broader market looks heavy and it appears that if we end the day red that the weekly losses will be accelerating. Markets do not bottom on a Friday, so if we cannot have a real move higher today, then we will likely be lower again to start next week. We are obviously higher to […]
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. […]
