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Sarepta Soars As Pfizer's DMD Prospects Languish

Trent Welsh profile picture
Trent Welsh


  • Pfizer's recent PPMD conference revealed very concerning safety issues with its gene therapy candidate for DMD.
  • Pfizer's candidate might be headed down Solid Biosciences' path to disaster.
  • The only "sure" bet in the DMD space is Sarepta, who currently owns the space while continuing to build a moat.
  • Sarepta is a strong buy, especially after the pullback from recent highs and a possible golodirsen approval happening any time now through August 19th.

Pfizer Inc.'s (PFE) latest Duchenne Muscular Dystrophy data reveal at June's Parent Project Muscular Dystrophy Conference resulted in some safety issues for its lead DMD candidate PF-06939926. Pfizer's candidate shares some similar traits to Solid Biosciences Inc.'s (SLDB) lead DMD candidate, which might be bad news for Pfizer's long-term DMD prospects. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is the only "sure bet" currently in the DMD space with gene therapy data that is on par or better than the competition, has more transparent results, has no significant safety concerns associated with it, and which is already year(s) ahead of most of the competition. Sarepta continues to be a strong buy especially after the most recent pullback from recent highs.

Pfizer currently views DMD as a potential area of breakthrough treatment for the company as a possible sustaining growth initiative beyond 2022 in the treatment of rare diseases.

Slide from Pfizer's Q1/2019 Earnings Slides

Pfizer's most recent data release for Duchenne Muscular Dystrophy came from 6 DMD boys who each received one IV dose of PF-06939926. Each subject had an immune response with one child getting admitted to a pediatric ICU for 11 days for hemodialysis. This safety concern means that Pfizer should have to incorporate some additional FDA approved safety features into any future trials for PF-06939926, which could easily set back the candidate's advancement another 3-6 months or so even after it is already a year behind Sarepta's lead gene therapy candidate SRP-9001 whose data was reported back in June of 2018.

At the heart of the safety concern is the vector that companies are using to deliver their transgene to target cells with minimal immune response. Sarepta used AAVrh74 for its vector, which resulted in no serious adverse events in the company's studies to date. Solid Biosciences used vector AAV9 in its

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Trent Welsh profile picture
B.S. Psychology University of Missouri-Columbia MBA University of Missouri-Columbia Full time investor looking to capitalize on market overreactions and looking for value where others see nothing but wreckage. Long term buys and short term trades to build wealth.Investing Better Than A Money Manager: The Rise Of Retail Investing - By Trent WelshI have an investing book with the title above on Amazon written for beginning retail investors looking to set up a self-directed portfolio with their IRA's, 401k's, or other retirement or trading accounts. It details how to pick and choose stocks amidst the different sectors and how to figure out how much in each sector an investor should have to help achieve diversification.Please take a look at it and let me know your thoughts. Thanks and Best of luck to all :).

Analyst’s Disclosure: I am/we are long SRPT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Comments (18)

NDHT profile picture
Guess the stock price will be impacted on and after July 25th.

ICER report cites continued lack of evidence supporting efficacy of exon-skipping therapies for DMD

Jul. 12, 2019 10:02 AM ET|About: Sarepta Therapeutics, ... (SRPT)|By: Douglas W. House, SA News Editor

The Institute for Clinical and Economic Review (ICER), a Boston-based non-profit that evaluates the cost of medical treatments, has just published an Evidence Report assessing the comparative clinical benefit and value of Sarepta Therapeutics' (SRPT -0.8%) Exondys 51 (eteplirsen) and golodirsen and PTC Therapeutics' (PTCT -1.9%) corticosteroid Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD). The report will be reviewed at a public meeting of the New England Comparative Effectiveness Public Advisory Council in Cambridge, MA on Thursday, July 25.

The report cites the absence of "persuasive evidence" that exon-skipping therapies improve outcomes that matter to patients, including functional status, quality of life or length of life. The limited data that exists show that both drugs modestly increase dystrophin levels but the clinical significance of this is "uncertain."
Trent Welsh profile picture
ICER report is old news and shouldn't move anything.
Wall Street Titan profile picture
CAPR had an 11 million market cap going into trading today. A $100 million dollar market cap brings it to $28 per share. Capricor Therapeutics Announces Positive Results from its Interim Analysis in the HOPE-2 Trial to Treat Patients with Duchenne Muscular Dystrophy seekingalpha.com/... $CAPR
Wall Street Titan profile picture
In comparison, SPTA has a market cap of $11,700,000,000.
Good article, a mention of the PPMO may have been in order considering it may ultimately be Sarepta best tech. Read out EOY on P1/ P2
Trent Welsh profile picture
Thanks for reading. So much going on for this company its ridiculous.
Nice summary, especially of the competition. Thanks.
I randomly bought this company in the 40's based on a half baked idea of "Well they have drug that is approved so it couldn't be bad" - I will be honest, I really didn't know or understand what I was buying - but I bought well over 10K worth . I still don't know what I exactly own - but there is no style points in making money- I am stupid, lucky and ...I am sitting on a Boatload of profit.
Trent Welsh profile picture
marriottmare profile picture
Agree, buy and hold
I agree 100%. I am a long time SRPT holder. Anyone with a degree in money management would advise me to sell a sizable portion of my shares and lock in my gains. I am not going to do that because I believe the gains will be much larger. That is based on rational thought and not emotion. I can wait a little longer for the real payoff. GLTAL.
Really? not taking some off the table? It has been a long haul- so I am looking to pull 50% off- what level is Sarpeta at II?
Trent Welsh profile picture
I've traded SRPT in the past and made a lot of money ($10,000's worth, which is a lot for me means I own like 50 individual stocks along with over half a dozen mutual funds) since I first bought in at the $30 range. It is my biggest individual stock holding now and the only way I would feel like selling is if it did a giant leap to like $200 a share or something over night. How many company's do you know who have a highly profitable drug, more drugs soon to be approved, and are on the cutting edge of some of the leading medical science out there with spectacular results all while the competition around it continues to be unimpressive according to almost all the expert analysts out there. The only ones who looked at Pfizer's data and were impressed were Pfizer scientists working on that drug. I'm done selling until something changes that narrative.
There are a lot of companies like what you describe. Being over confident will cause you to lose a ton of money as well
Trent Welsh profile picture
@Bike for life Can you name some company's that fit that description at all? I'm always up for new investment ideas so I'm all ears :). And in my experience it is those who don't take risks at all that end up unhappy in the end and is why like 80% of money managers underperform simple indexes. Sarepta could go to 0 tomorrow and i'd still be fine with my other 49 stocks and mutual funds and what I have already made from it which has been redistributed into other stocks. I make big bets, but I never come even close to betting the farm. Best of luck.
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