076 – Krystal Biotech’s NOVEL Gene Therapy for Rare Diseases | Interview with WX Capital

Krystal Biotech (#KRYS) is using a novel gene therapy platform to treat various rare diseases. Their primary indication is Dystrophic Epidermolysis Bullosa (DEB) with an upcoming Phase 3 readout in 2021. Michael McGuire from WX Capital and I discuss the bull case for rare disease indications, the epidemiology of DEB and what the company might price the drug at. We also talk price targets and timing, which more often than not, is the most important factor when making a trade.

Check out more from Michael McGuire from WX Capital at wx.capital

If you want to help out the show (or join the discord), take a look at my patreon: https://www.patreon.com/breakingbiotech

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Send me an email matthewlepoire@gmail.com

http://www.breakingbiotech.com #breakingbiotech #wxcapital

Disclaimer: All opinions expressed by Matt in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt’s opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech


047 – Sarepta Receives CRL for Golodirsen! IOVA impresses in solid tumors.

In this video, I talk about Sarepta and their issues regarding the CRL received for Golodirsen. I also take a close look at Iovance Biotherapeutics and their tumor infiltrating lymphocyte cell therapy for cancer treatment.

this is not investment advice



043 – Sarepta keeps the gene therapy throne in DMD! ContraVir submits NASH IND!

Hey everybody, hope you’re all doing well! In this video, I talk about Pfizer’s DMD gene therapy update that indicates continued Sarepta hegemony in the space. I also talk about ContraVir and their NASH drug, CRV431.

I must have drank too much coffee before recording because I made more than a couple errors this time around. So, if you’re an audio-only listener and hear something that doesn’t sound right, double check the video version, which corrects the mistakes via captions or has it written properly even though I say it wrong (i.e. I say 3E16 when 3E13 is clearly written on the slide).

Thanks to everyone for listening and do your own due diligence if you are considering investing, this is not a substitute for investment advice.

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032 – GILD Fails Phase 3 in NASH!

On this episode, I talk about results from the Gilead Phase 3 NASH trial that announced no improvement in patients treated with Selonsertib (ASK1 inhibitor). I also talk about the DBVT BLA resubmission and a hot hot Q319 trading idea on CBIO.

Relevant links:

*this is not investment advice, only my opinion and ideas*


#biotech #breakingbiotech #nash


031 – Sangamo woes

Today, I talk about Sangamo’s updated data from their talks at the WORLDSymposium. We saw updates on their MPSI and MPSII programs that the market responded to with big selling.

I refer to their pre-clinical data and see how it compares to the updated human data. I finish up by giving my predictions and what I’m watching this week.

Relevant links:

*This is not investment advice*


Sarepta Declines on Good Micro-Dystrophin Data

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

Relevant links:


Sangamo Therapeutics drops after SB-913 update

-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:


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See the blogpost at http://www.breakingbiotech.com


I was wrong about Zogenix! FDA updates guidelines for gene therapy.

Note: I meant to say that sub-1% factor activity is SEVERE hemophilia. Normal is from 50% to 150%.

Today I talk again about Zogenix because I was wrong about Study 2 being priced in! Congrats to the ZGNX holders. I also talk about the new FDA guidelines on gene therapy for hemophilia and its implications for spark tx ($ONCE).

Follow me @matthewlepoire

*This is not investment or trading advice*

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