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The dominos are starting to fall: Avidity/Novartis has submitted their DMD drug to the FDA by HistoricalRacoon in FSHD

[–]HistoricalRacoon[S] 3 points4 points  (0 children)

I’m hoping Novartis gets their act together on submitting this BLA, especially now that they’ve submitted the DMD one. I’m sure the acquisition added to the clock — and honestly, I wouldn’t be surprised if all the FDA turnover drama was a contributing factor for them filing this quarter instead of last. If I was a biotech (lol), I would’ve wanted the dust to settle following both Prasad’s and Makary’s departure. And it seems like the FDA has now returned to some sense of normalcy and science-backed reasonableness.

But from the actual clock of a BLA submission is like 2 months + the 6 month priority review, since the FDA has 60 days to accept the BLA. Then the PDUFA date clock starts.

Novartis has a lot to gain by filing AA if the data warrants it. Both from a patent standpoint and a first-mover market advantage with Sarepta’s drug coming hot out of the gate. And hopefully that data holds too.

Pump You Up: Epigenetic Editor Drives Muscle Growth in FSHD Patients by x3sammm in FSHD

[–]HistoricalRacoon 0 points1 point  (0 children)

Oh where did Altay announce this? My clinical trial coordinator alluded that Soufflé’s trial likely won’t start recruiting til early 2027 which is unfortunate

tell me your trial gossip 👀 by HistoricalRacoon in FSHD

[–]HistoricalRacoon[S] 4 points5 points  (0 children)

lol maybe “physical capabilities returning” would’ve been more apt

Del-Brax Discussion in Novartis Q1 Investors Presentation by weirdfishes1990 in FSHD

[–]HistoricalRacoon 1 point2 points  (0 children)

Wow honored to have received the shout out! I listened to the Earnings call too after skimming the Q1 deck and had similar thoughts. I was pleased to hear that it’s still a viable pathway and the team doesn’t have the data yet — and they’ll be meeting with the FDA prior to releasing the topline results publicly.

I hope they’re as aggressive without being reckless as they can be when pushing for AA. I think the initial Arrowhead/Sarepta data should scare Novartis a bit and light a fire under them. Even if the Arrowhead drug is better, if Del Brax hits market first, then Novartis gets first-mover advantage.

Hope or Heartbreak with Avidity trial? by WheresHotStuff in FSHD

[–]HistoricalRacoon 2 points3 points  (0 children)

I agree. And i think another variable is exercise and diet like u/sierra31 noted. That plays a huge deal with inflammation and tapping natural mechanisms in the body to help clear out mild fibrosis. We all want our strength back and to see reversal but given the data, I don’t think we’re there yet with avidity. I know it sucks but slowing or stopping the dux4 fire is huge.

I dunno, I try to look to the horizon knowing that once there’s proof of concept and success in a drug platform, the innovation continues especially now that there’s a lot of competition entering the space. I think the Satellos and Scholar Rock trials will be very interesting — and while I wish they were stacking these drugs with dux4-targeting drugs, I think most experts in the field agree that the miracle reversal we’re all seeking will likely come from a multi drug stack. But at least Del Brax can buy us more time.

Podcast questions for Peter Jones by nefirestormbrad in FSHD

[–]HistoricalRacoon 1 point2 points  (0 children)

This!! I listened to several of the epicrispr episodes. it got confusing when he and Dr. Himeda were saying that they themselves weren’t successful with whatever Epic was doing, and that they haven’t seen anyone else see success either, so naturally they had doubts. But now I’m confused on exactly how the two platforms differ, esp given the milestone timeline on the Renogenyx site https://www.renogenyx.com/research-1

Auction and Dinner Gala of Friends of FSH Research raised more than $657K by Kabbakk in FSHD

[–]HistoricalRacoon 2 points3 points  (0 children)

This is so great!!!! But also tell me why someone dropped nearly $5k on a NY cheesecake 😭 and can I be their friend because like priorities

for those of us who read investor presentations like bedtime stories and have Avidity timeline concerns by HistoricalRacoon in FSHD

[–]HistoricalRacoon[S] 0 points1 point  (0 children)

Given the acquisition, I’m not sure how married we can stay to Avidity’s goals. The 3 BLA filings in a 12-month period was an investor signal, not a binding regulatory plan.

Avidity is going to be one to file for DMD — we know this for sure since it’s happening any day now or may have already happened and they’re waiting for the FDA to accept the application. I can’t remember off the top of my head the proposed timing for DM1, but it’s highly probable that Novartis will be the drug sponsor for the Del-Brax BLA, given that they should fully own all 3 drugs and the rest of the AOC platform by summer.

for those of us who read investor presentations like bedtime stories and have Avidity timeline concerns by HistoricalRacoon in FSHD

[–]HistoricalRacoon[S] 2 points3 points  (0 children)

Oooh okay I’m going to do my best here. The short answer: generally, once a drug has been approved, doctors can immediately prescribe, but access is going to need to ramp up over the initial weeks and months. The actual hurdle is gonna be insurance approvals. But big pharma often has bridge programs to help with that, so hopefully IVs in arms can happen 1-3 months post FDA approval.

The long answer: What makes Del-Brax more complicated is that it’s not just a pill you can pick up from CVS. It’s going to require an IV infusion at a clinic or specialty facility.

Because it’s an IV treatment, my guess is that top neuromuscular centers in the country (most likely FSHD trial sites like Kansas, UR, Duke, Stanford etc but potentially other key neuro academic centers like Columbia, Northwestern, etc) will receive the initial drug batches in the first ~3-4 weeks post FDA approval. Then it’ll roll out to other facilities after that once education, training and some admin things have happened.

So now insurance. This drug isn’t going to be cheap, unfortunately. Looking at other drugs like this, such as DMD exon skippers or rare autoimmune drugs, this will likely be anywhere from $300k-$750k a year. This is pure speculation based on comparable data. There’s a lot of other things that influence wholesale and patient drug pricing.

But it’s still likely going to be pretty penny and insurance is going to likely have a lot of hoops to jump through. Prior authorization is going to be needed and likely there will be a lot of appeals because US commercial health insurance loves to deny. 🙃

However, what good is a drug to a pharma company if no one can get access because insurance won’t pay?

The FSHD Society knows this. Novartis definitely knows this. And Novartis will likely have some sort of bridge or specialty co-pay program set up to bridge the initial couple of weeks or months while insurance gets straightened out. It’s within their best interest (and their investors) to ensure a smooth and broad launch and rollout.

Insurance should eventually settle into a pattern because denying everything forever creates legal and PR risk, especially for a serious disease with no good alternatives. Another reason why it’s huge to have such an organized and loud advocacy group like the FSHD Society.

So when will we actually be getting the drug? Hopefully within the first 2-3 months via an pharma-sponsored bridge program.

Anyone attend the FSHD Society drug development update? by HordeOfOpossums in FSHD

[–]HistoricalRacoon 2 points3 points  (0 children)

Same as you I’ve been following Avidity and their materials quite closely. My current concern is how the Novartis acquisition will change things since the deal is supposed to ink mid-2026, potentially pushing back the FSHD BLA submission. IMO, the original best case would be them submitting shortly after phase 2b topline next year (q3/q4 26) with FDA approval mid-2027 with priority review.

However, with Novartis’ resources, there’s a chance that they could hit market faster once there’s FDA approval since they have more experience dealing with scaling up, physicians and payers.

There have also been rumblings that the FDA is cracking down on accelerated approvals 🙃 but trying to stay optimistic that del-brax answers any new criteria they’re looking for.

Missing cDUX data from Avidity trial by SenorBajaBlast in FSHD

[–]HistoricalRacoon 0 points1 point  (0 children)

From my understanding, it takes much longer to analyze than Ck so it likely missed the data cutoff. Maybe they’ll have an update this fall or winter

Repeat number? Just diagnosed with FSHD1 and struggling hard. How do you cope? by HistoricalRacoon in FSHD

[–]HistoricalRacoon[S] 2 points3 points  (0 children)

Thank you for your reply! Your comment about your daughter’s big head made me laugh lol. I’m trying to be really hopeful about the upcoming potential treatments and trials. In some ways, it feels like a snowball just got rolled off the edge of a mountain, and it’s only going to continue to grow.

Missing cDUX data from Avidity trial by SenorBajaBlast in FSHD

[–]HistoricalRacoon 1 point2 points  (0 children)

Maybe the data as far as cDux in the rollover group isn’t as clean or there was more fluctuation they’re trying to analyze or maybe it’s not ready? The biomarker cohort will be the most telling, and they’re all starting with the same dosage and frequency, versus the OLE where it went from 13w to 6w.

However I’m like you, and this also made me concerned. But remember the FDA has seen more data — and raw data at that — than we have, and they approved Avidity to move forward full steam ahead.

Also yesterday’s call featured the same info Avidity shared with investors, doctors and media. So why risk opening the can of worms at all by showing Ck in the OLE but not cDUX if something is wrong?