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SGMO / PFE: Presented data today on their Hemophilia A Gene Therapy
The slides are part of an 8-k
https://www.sec.gov/Archives/edgar/data/1001233/000119312520171977/0001193125-20-171977-index.htm
Pfizer had a call (not just on Hem A but other recent data) and slides:
https://investors.pfizer.com/events-and-presentations/event-details/2020/Pfizer-Analyst-and-Investor-Call-to-Review-Data-Presentations-at-Scientific-Conferences-during-the-Week-of-June-15th/default.aspx
There is definite signs of decline I had some thoughts
https://twitter.com/MauriceOnTW/status/1273612205863436288
and here along with JQ1234 who has always been skeptical
https://twitter.com/jq1234t/status/1273617346733780999
FWIW I agree with JQ that the two programs at this point should be considered +/- comparable till proven otherwise. So I think the program is still viable but it'll be a harder sell for SGMO/PFE coming second to market and (not yet anyway) showing substantial differentiation.
BMRN:
Just to follow up they had their Zoom call with two docs and the full slide deck is posted here
https://investors.biomarin.com/download/WFH+IR+Deck_FINAL_061620.pdf
I tweeted out most of the Q&A
https://twitter.com/MauriceOnTW/status/1273361742995824640
BMRN:
There are a couple of observations I had about the release that I posted on twitter.
First to put in context of SOC I'm in the camp that believes its a tremendous advance and even with durability questions they will get meaningful commercial sales.
1-The PR was quite cheezy in what they did with the graphics!
https://twitter.com/MauriceOnTW/status/1273269482065203203?s=20
Fortunately you can get higher res images from here
https://twitter.com/MauriceOnTW/status/1273330596593504257?s=20
2-The four year high dose data mean 24.2/median 16.4 is a bit misleading (perhaps the reason for the power slide resolution?) as it includes just 6/7. If one goes by the 3 year 9 month reading looks to be about mean 20/median 12
https://twitter.com/MauriceOnTW/status/1273291002074345472?s=20
3-The data is not going to be reflective of commercial dose as Phase 3 interim data (what BMRN used to file on) produced very notably lower expression levels.
Roche’s Port Delivery System with ranibizumab shows positive phase III results in neovascular age-related macular degeneration
http://www.globenewswire.com/news-release/2020/05/27/2039063/0/en/Roche-s-Port-Delivery-System-with-ranibizumab-shows-positive-phase-III-results-in-neovascular-age-related-macular-degeneration.html
We don't know all the data so can't conclusively say but if this holds up and is reasonably safe/well tolerated why would anyone consider one of the current Wet AMD gene therapy options (ADVM and RGNX are the leading ones I'm aware of)? Both leading ones appear to need rescue injections unless you want to have significant vision loss like ADVM did with one of their cohorts to I guess make the claim no rescue injections.
The KOL who is also an investigator said it in passing when talking about the use of steroids and cataracts. There was no remark made by anyone at the company but the way the KOL said it ALL patients were pseudophakic!
XLRN:
I thought it a bit weird they didn't give out any hard numbers but JQ pointed out this isn't the first time (and not necessarily indicative of something negative). It still weird we don't even know which dose(s) and which patients were not in analysis. Martin Auster specifically asked about which dose(s) met primary endpoint and they didn't say.
Here is the twitter thread
https://twitter.com/MauriceOnTW/status/1221916481740472321
Does HEMA qualify for the list?
There was a report a couple months ago they hired bankers and were seeking strategic alternatives
https://www.hemacare.com/news/charles-river-laboratories-to-acquire-hemacare-corporation/
I was a bit disappointed when I first saw the clinical trials entry some time ago thinking it would taint Gene Therapy development and then seeing the price tag now and having consent done in Colombia doesn't exactly help matters....
I have no ideas if the treatment will work but I've seen several journal articles implicating short Telomerase with certain diseases. Since I follow IPF that is one I've often come across though I don't believe it is known why there is this association.
Thanks everyone for the replies!
MEDX seems like the only winner even though they probably sold the company way too cheap.
Thanks!
Reason I mention is krys is another and remembered they were the first.
Does anyone remember the Bio who had husband and wife in top positions think one was CEO? My memory isn't that great so can't recall for certain but think they were payed well not sure on the ending (guessing bad).
Thanks, appreciate your/her thoughts.
ABEO:
Selling the company would be in the best interest (programs and shareholders). I tend to think its a legit attempt and not a hype promotion but I certainly could be wrong. I tweeted a bunch of stuff on how I think the assets could be worth substantially more than current stock price. Some caveats though mainly we don't know all the data which for something like CF could mean the difference between a $5m valuation or a $500m valuation.
Here is a link to the thread if anyone is interested in my 2 cents:
https://twitter.com/MauriceOnTW/status/1168873206272405511
ARDX:
What do you (or better your wife ) make of the Diarrhea? People on twitter seem to be playing that up but there were only 3 discontinuations because of it and the PR seems to indicate its transient (few days). The company claims in part its how its reported (more bowel movements) and a high percentage of patients have constipation complaints so a couple more bowel movements a week is welcome. Is that a buyable argument?
ABEO:
Yes on following them and them needing cash soon.
Management (3rd rendition) has not done well thus far (telling story, executing, creating value, strengthening balance sheet...)
Assets/Valuation: I think they have a good mix of assets priced as cheaply as any gene therapy company one could find probably because of a management/financing discount. Their MPS IIIA + EB get all the publicity but the jewel could turn out to be their CF program and perhaps some differentiated vectors from their library.
They could announce a deal to relieve some of the financings overhang. The chairman owns a ton of shares or I'd be more worried about one of those 100% dilutive toxic financings though I guesss that can't be totally ruled out.
Any specific questions? I'm not on I-hub too much these days you could try ask on Twitter for a quicker reply (@MauriceOnTW).
Just to translate the (as usual) misleading PR the company gave. The PDUFA was almost certainly extended because additional materials were submitted during the review and FDA labeled them as a major amendment.
Don't think one can read too much into it beyond that as to a positive or negative outcome for the final decision.
$RGNX / $NVS Zolgensma royalty
Some important (especially to RGNX) disclosure on the call!
https://twitter.com/MauriceOnTW/status/1133034372653879296
NVS / AVXS / RGNX: Zolgensma links
Here is the FDA PR:
https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease
NVS PRs:
https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma
https://www.novartis.com/news/media-releases/avexis-announces-innovative-zolgensma-gene-therapy-access-programs-us-payers-and-families
RGNX PR:
https://regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-announces-first-fda-approval-gene-therapy-based-its
Zolgensma Label:
https://www.avexis.com/content/pdf/prescribing_information.pdf
NVS will have a webcast on 5/27 10:30am ET:
https://www.novartis.com/investors/event-calendar#ui-id-6=0&ui-id-2=2&ui-id-1=0
Thanks for the correction Dew!
ADVM:
I thought the wording on the PR was a bit unusual a few other skeptics too:
https://twitter.com/MauriceOnTW/status/1129128336259715076
$ADVM Strange...."Given the encouraging robust anatomical response from patients in the first cohort, we will begin dosing patients in the second cohort at a lower dose of 2 x 10^11 vg/eye, instead of dose escalating as previously planned.”
— Miljenko Zuanic (@Miljenkoz) May 16, 2019
This has to be the most clever way to continue a clinical trial without escaping clinical hold, while disguising it as a release off of clinical hold, that I've ever seen...
— BiotechObserver (@BiotechObserver) May 16, 2019
ADVM:
Dose escalation is always planned but if the efficacy is good (especially in Gene Therapy) they don't go up. I don't know enough about the eye and antibodies (to vector) but if there is a risk of not being able to be retreated FDA has guided to the first dose being one that preclinical models suggest provides some efficacy which is a bit different then normal dose escalation studies.
FCSC:
I tried asking a question online about if they would start with commercial grade material and unfortunately it was not addressed (not that I was expecting much). Its quite noteable the milestones and mentions of manufacturing in that PR!
The PR also revealed their ownership interest (with XON subsidiary) as 50%. So really they now own just a 15% gross profit share in addition to the milestones.
ADVM:
The way I would interpret it is the first cohort did not have significant enough efficacy (thus the dose escalation).
Also, the wording is they had this info early April and so presumably sat on it (granted for just a week or two). Considering this is their ONLY program that seems material doesn't the three day rule apply? At the least, it puts little confidence in management (not that I had much).
I am in the shock the stock was not down to below cash (where it should have been once they cut all their AAV10 programs).
FCSC:
I don't follow the company too closely but did listen to the call. I don't have a positive sense for the company. As far as the PR/call adding to my sense of mistrust for the company is their mention of submitting CMC information prior to starting the study. I am guessing their mfg is not up-to-par for COMMERCIAL use and so by FDA gene therapy standards what they are calling a Phase 3 study (which is fine) would not be enough for registration.
https://seekingalpha.com/article/4251297-fibrocell-science-inc-fcsc-ceo-john-maslowski-q4-2018-results-earnings-call-transcript?part=single
Disclosure long ABEO which also has an RDEB program and is spending quite some time to get an in-house commercial manufacturing facility ready (tech transfer from Standford product) for their Phase 3 program
No that is not what I mean. They have > 100 different serotypes. How good or viable they are is a different question. One is disclosed as being used in multiple preclinical programs.
AXGT:
They also had Parkinsons (n=2) data PR which I'm guessing accounted for most of today's reaction. This is reworked version of Oxford's PD program which produced less than stellar results.
http://investors.axovant.com/news-releases/news-release-details/axovant-reports-positive-interim-results-first-cohort-sunrise-pd
Here is their updated slide deck too:
http://investors.axovant.com/static-files/02c997e8-92d8-443f-9f7e-91e60e364470
And they have an R&D day schedule for the 29th
That's not so clear to me... Certainly, the IP matters more than Orphan exclusivity (so RGNX limited IP really hurts differentiating them) but are vectors commodities? ABEO has a library of > 100 vectors and the enterprise value of the whole company is not much more than the upfront of what Pfizer paid for Bamboo several years ago (though they did get their DMD gene therapy). 4D has a library too (not much public info on it) and a bunch of other companies develop their own proprietary vectors.
From the story, it actually sounds like it is making exclusivity LESS exclusive. Basically, if two companies are delivering the SAME gene but using different vectors then both get approved. This would theoretically result in more competition by not having to wait for the exclusivity (of the first approved) to end to enter the market.
So I am a big listener/follower of webcasts and this is a very odd week. It's rare to see a company hold webcasts for two events (not IR conferences their own sponsored events). This week there are three including SRPT having 2 on the same day!
SRPT 2/27 8:00am LGMD Data 2/27 4:30pm Q4 call
http://investorrelations.sarepta.com/events-and-presentations
CTMX 2/26 8:00am R&D Day 2/27 5:00pm Q4 call
http://ir.cytomx.com/events-and-presentations
NKTR 3/1 6:00pm REVEAL data 2/28 5:00pm Q4 call
https://ir.nektar.com/events-and-presentations/events
There is also the leerink confernece this week 2/27-3/1
http://wsw.com/webcast/leerink32/
I was surprised to hear NKTR evaluating one of their oncology drugs in virology with Gilead. I believe they said HIV but the slide seems to allude to other possibilities. It's not a partnership per se each provides drug and maintain ownership. Gilead has the right of first refusal (for Virology only) though if NKTR decides to partner.
Slides 28 & 29 from the webcast here is a link for anyone interested
https://www.nektar.com/application/files/2315/4696/3638/JPM_2019_Nektar_Therapeutics.pdf
ARDX:
I've been trying to figure out the holes in their IBS/C IND it seems like a very good chance for approval. There is hyperphosphatemia Phase 3 data (though it seems likely they push back the data) at under cash (around net cash if factor in the debt) seems like a good speculative play.
TRIL:
At these prices, an ATM is pretty worthless with the kind of money they need to advance one path forward much less multiple!
I've been fearing a rights offering which seems more popular in Canada but even that now may be hard to raise enough at these levels! So hopefully it finally pushes management to partner or sell out! Would like to see a 13D with some comments about pushing a vote on strategic alternatives.
Thanks for the heads up!
I didn't catch it yet but saw they have an IPF program. Do you know anything about it? Is it credible?
I haven't followed them closely didn't they split the company a while back?
QURE:
I thought the data points quite good... The caveat is why no curves and why present it today and not at R&D Day Monday? They also presented the top line data a little weird. The PR said average at 6 weeks but they give us values at week 6, week 10 and week 8 (which avg to 30 could be 31 with rounding). So its a bit ambiguous were their values the same a few weeks earlier (or one patient up one down)? I'd expect the values to be rising for several more weeks (based on what others have shown).
Graphs should clarify any doubt Monday my concern would be those two who screen-failed another GT (Sparks or Dimension most likely?). Qure may have been too greedy doing an all comers. While its true they did "accidentally" dose I believe a couple patients with low antibody titers in their prior study why risk it here? Developing antibodies to one serotype is known to make a patient develop antibodies against others. BMRN which is also using AAV5 is screening out those with pre-existing antibodies. Of course, they have the dollars so they are doing a separate study for those with low levels.
Here is a nice graph From @AndyBiotech (hope he doesn't mind me stealing it)
and a link to his tweet (note the third company has Nathwani who did the original Hemophilia B studies)
$QURE vs $ONCE vs Freeline#Hemophilia B Gene therapy data competitive landscape in one chart
— Andy Biotech (@AndyBiotech) November 15, 2018
Freeline is one serious competitor to watch out for $QURE and $ONCE.
Impressive initial data at low dose, more data coming at #ASH18:https://t.co/7lMQ42J1Y6 pic.twitter.com/Wyv18n9Gg7
ETON is a spin-out from IMMY. I sold out (at a loss) a while ago. The company is spinning out companies like crazy though!
http://globenewswire.com/news-release/2018/11/13/1650904/0/en/Imprimis-Pharmaceuticals-Announces-Third-Quarter-2018-Results.html
Nktr:
Thanks for the added color. Did you make anything of Dr. Diab's comments about academic centers managing AE's better and getting higher responses or is that a generalization that can be applied to most new immuno-oncology therapies?
VRTX:
U.K. PARLIAMENT ISSUES ULTIMATUM TO VERTEX, NHS, NICE
https://www.biocentury.com/bc-extra/company-news/2018-11-09/uk-parliament-issues-ultimatum-vertex-nhs-nice
I thought this also an interesting Biocentury article. Don't if its a bluff but would make for some interesting reading!
The Biocentury article adds some interesting color quoting Dr. Diab
https://www.biocentury.com/bc-extra/clinical-news/2018-11-09/nktr-214-cr-data-sitc-suggest-advantage-over-soc
SGMO:
Not a close follower of the stock to know for sure but they pulled their guidance of showing updated Hemophilia A data (I think for ASH) expect that may be the reason. Sandy sounded upset when taking the question too :).
I've been on the fence for years with SGMO each time I look a little closer something doesn't quite seem right so I keep deciding to wait for more progress (direction at first, now data).
FWIW I think Sandy is quite the enigma. I'll give him credit for doing some good deals and directing them into rare diseases but he really comes across to me quite badly (don't trust, seems to not understand his disease areas/market) each time I hear him talk!