Sarepta Therapeutics Inc New (SRPT) 銘柄情報

SRPT 10Q due AUGUST5

Honeywell Acquires CAES Systems for $1.9 Billion, Sarepta Therapeutics Surges 34%, Gilead Continues Gains

SRPT obtains an expanded approval by FDA for treatment of DMD. Share price is $174, +~ 56% on news.

$SRPT Total Debt (mrq) $1.38B back to $.50

Readers might also check CPRX news release AH last Thursday, re FDA approval of a DMD drug for which CPRX has an exclusive license to market/sell in North America. CPRX plans introduction in early 2024.

Thanks. Like Barron's here, I speculated a while ago that if this trial succeeds as the company has long said it would - and we finally reach that happy $200 shore - I do think it may energize the XBI Biotech index ... which it looks like hasn't made any progress since 2015!!

It's like people think the science here is bogus, dead in the water. Well, it's not. And hopefully Sarepta is on the verge of proving that in the next few weeks.

Hi Joseph...and thanks for posting all of this information.

My curiosity of gene therapy doesn't really have anything to do with Sarepta other than to inquire if it breaks the double-strand in the DNA when it treats its patients?

The reason I ask that question is based on another company called BEAM. I don't want to go into detail here because it wouldn't be fair to the investors on this board. Please go to the BEAM board for more details.

Thanks

abe

abew4me (and unknown ghosts of the iHub Sarepta message board): Insurance funding for Elevidys has been a stock market issue for Sarepta. My (limited, but I think effective) due diligence in this has been in finding and keeping up with the Facebook page of Little Hercules Foundation. It has, I think, been assiduously following the issue, telling caretakers about both the insurers who have agreed to provide coverage (and any conditions thereon) and those denying coverage (with regard to which it tries to organize pressure campaigns). You'll have to review the entries yourself, but my (neither scientific nor well-organized) survey of them I felt showed that the considerable majority of insurers were providing coverage (per the rather restrictive current FDA label). If you look through those entries and conclude otherwise, please post your thoughts here. Once I did that bit of DD, I upped my investment.

You (abew) also asked about my DD on genomics. I don't remember what I posted. I do believe gene therapy will make many other cures obsolete. (I listen to a lot of science podcasts and read science books and articles some, too.) I think there was a Rett Syndrome genetic therapy in Phase 1, but all I recall doing was calculating how many years before it could possibly receive FDA approval.

Hi, Sarepta iHub ghost town. From Barron's:

The upside/downside trade-off for Sarepta Therapeutics [SPRT] is attractive going into the end of the year. We are coming up on results of what could be a confirmatory study of its gene-therapy drug for Duchenne muscular dystrophy. This could be a seminal moment for what has been a controversial stock, and provide a boost, albeit moderate, for the biotech space as a whole, especially
as the index is trending toward multiyear lows.
There appears to be two ways to win with Sarepta. One way is that the Phase 3 study shows an improvement for patients, even if very modest, but enough to illustrate a trend. This represents the base case or perhaps the most likely outcome, given the patient
population, but would also ensure that the gene therapy, which has already been conditionally approved by the Food and Drug
Administration (pending this trial), remains on the market. Best case, the trial demonstrates a statistically significant difference in the drug's favor versus the placebo, and Sarepta might address a larger market, including many patients who would otherwise not have access to the therapy. I believe that a positive trial would move the stock, now around $120, to or above $200 a share. If the drug fails to show even a modest benefit and investors remove gene therapy from models, a worst-case outcome could result in a stock price of around $60, depending on other assumptions. In the biotech sector, we are used to volatility.

Currently it's approved for only ages 4 and 5. CEO said there are 700 now in that age group in the US....The usual number of DMD kids born each year is 1 in 3,500. Check that with DMD organizations rather than what somebody plugged into some pretend "AI" toy. When approved for all ages you'd be talking 7,000 to 10,000. After they are all dosed (if that were to happen), then it reverts to maybe 350 a year. By that time it would be worldwide in the hands of Roche. I just saw a global number of 100,000. It is the most common rare disease.

Analysts and biotech journalists and scientists are the only ones who care about the drug and Sarepta and talk about it now that it's a success. its message boards are a barren desert where once it was an Amazonian rain forest.

I'm not sure how an individual investor can learn about the company's science at this point. Study the company's website for that. It's all there.

In a way, the fun (drama) is just about over. Just need the trial data in a few months to be good. The share price it seems to me will be under control of the big boys wanting to top off their positions before that data comes out. So it's a good time to do the same. After success in that trial is confirmed we'll be another Regeneron as far a stock goes. Possible much better. We should have as many diseases we can treat as it looks like Anavex will have someday.

I was pleased with yesterday's earnings and conference call. It also made me realize how much I know about blarcamesine! That is, I couldn't follow any of the discussion about Sarepta's research and trials in the call. But the expectation of broad insurance coverage and drug administration of the gene therapy before year's end has me optimistic enough to hold on to my now not-insubstantial Sarepta holdings through at least the first 2024 earnings report (assuming things appear to be proceeding as indicated yesterday).

Your calculation on Investor Village for administrations of the gene therapy will obviously have to slow down as, from what I gathered from the call, only 400 children per year are diagnosed with Duchenne. (Hmm, I just asked Google Bard, which replied, "According to the CDC, about 1 in 5,000 live male births in the United States are affected by Duchenne muscular dystrophy (DMD). This means that about 200 new cases of DMD are diagnosed in the US each year.") I guess, OTOH, it's a big world out there to expand into, though most of it will not be coughing up millions of dollars per child. (I hope there's some sort of charitable solution for them over time. What a horrible disease.)

Also, it was interesting, for a change, compared to Anavex, to hear a conference call with all these questions from analysts who work for companies I'd already heard of!

Hellooooo.....I hear an echo in here .....

Sarepta will be fine. I expect the strange people at the FDA to take another look at us in September/October and some are suggesting they will end the trial early (it will be finished in effect at that point anyway) due to unambiguous success seen ... and approve the drug for all ages - as they apparently told our CEO a few months ago would be "the label" upon any formal approval - which was expected to be early next year or late this year. But, as i say, some expect they take an October re-look at the trial and accept it's success and expand the label then and there. That will bring the stock to 200 more or less. That will unleash the dam and we can move on to other things, finally. Check out the Investor village board for more of my ranting and raving if you dare. Otherwise, maybe you can explore any other Sarepta boards - stocktwits? reddit? for me too .... and if you find one that's wiggling a bit more than this one (!) or IV, let me know...with links. (I'm pretty lame. I had a hard time finding this one again just now!) Thanks and all should be well now with Sarepta, finally.

Maybe there's someone here who can correct or expand on my info for newbie Mr. K's benefit. And to exercise your fingers a bit as well!

Hi, Schmiggins! Any thoughts on where Sarepta is heading? Your thoughts on staying invested or not? I bought in a week ago after the fall following the NDA approval, stupidly catching a falling knife, but so far retrospectively okay, at 111+ and 109+. I'm ahead for now, but ... (By the way, I have much less SRPT than AVXL. I've been in Anavex since mid-2021. I'm considerably underwater on Anavex, having doubled my holdings pre-market the morning after the CTAD conference in December 2022. AVXL shot up that morning then slid, slid, slid.)

Congrats to longs. Monday should be a good day. Anyone following Capricor - CAPR
CEO said CAPR drug helps cardiac election fraction and helps upper limb function in DMD. She says combination with Sarepta’s drug could be better than either alone. Maybe Sarepta should by Capricor?

Go SRPT , buyout AMLX

looking to get some $SRPT call options

Thankful I didn't sell all my shares.

Good stuff

Indeed. Wish I had more shares but this market is freaky!

Trend is UP…

Thank you!

https://musculardystrophynews.com/2022/03/21/mda-2022-dmd-boys-improved-walking-motor-function-3-years-after-srp-9001-gene-therapy/

Cheers!

I have a core position in SRPT in an IRA. Been fun trading the high 60’s to 80ish channel over past year or so.

Good Luck about to board a plane back to the States!

My large positions are this and $SOF.I @ 11.20

Yes it has. Especially considering biotech and therapeutics are out of favour for awhile.

It’s been a decent trade

I was early buying these 68's but....added some 61's and 63's so actually am ahead right now. Nice comeback for SRPT today with the markets!

Bought a few more shares just now in low 68's.

Good Luck!

You too

Have very few shares left….good luck to us.

Lightened the load but Monday is a big news update regarding 9001-102 with JP
Morgan conference at 10:30AM

This thing is just gonna chill at 80-85 until DI gets his hand outta his ass and updates 102 in January

Same with $M

I’m anticipating a great 2022 especially with all the insider buying and anticipated news in January

I have been holding a smallish core position in an IRA and trading 100 size lots when appropriate. I usually let some go on good up days.

Otherwise.....be careful out there.

M...did let go of my entire position.

I rebought my entire position and some with a now $79 average.

Back in!

SRPT....

POS...

I was waiting for 120.

And sold for a massive profit that was told to be “dead money”

It’s funny, cause atown is right again!

Srpt

I'm waiting for 120.00

the dilution news was GREAT !!

You’re right
Out at 94.52!

wow....down 11.85....


this is much worse than dead money....

lol....

so much bullshit...don't even know where to start...

I’m holding for $120 short term.
With Pfizer pretty much out of DMD sarepta is 90%+ of the pipeline

New trial announcement coming, it’s going to go up especially on a technical standpoint above 98-99.

what was good about it ?

please be precise, thanks !

there's no truth to any of that....

Sorry out at 94.52

Nope, but long term hopefully be good! I’ll be waiting to get back in, out at 92!

looks like the public doesn't like dilution...

https://www.sec.gov/Archives/edgar/data/873303/000119312521296879/d239735d424b5.htm

wow....tanking again....

this stock can't take any more " good " news !

blood red...