Has anyone heard any updates on apitegromab?

[squarebob]

My neurologist believes it will be more like 3rd quarter of this year.

[alyssa-silva]

Are you referring to the filing or the drug getting FDA-approved? I’m sure it will take a while once the application is put through.

[mike-huddleston]

Hey Alyssa –

Scholar Rock and CureSMA just did a webinar on this on December 18, 2024. I attended it. They are still filing for FDA approval in 1Q25 and hope for an approval before the end of this year. They also received FDA approval for an expedited review after that webinar, which means it could be approved more quickly.

Of note, this requires an infusion every 4 weeks.

[alyssa-silva]

Interesting! Did they say anything else worth noting?

I’m curious how this drug will impact adults, if at all. My neurologist explained how myostatin decreases as you age, and this drug targets the myostatin on the muscle. So, there is some ambiguity there. I guess time will tell.

[mike-huddleston]

I think it’s worth noting that Spinraza clinical trials were not on adults and the FDA’s approval at the end of 2016 covered all ages. So, since it works in conjunction with Spinraza and Evrysdi, it seems to make sense that it would be approved for all age groups. Making sense may not be enough though.

The concern I have is for it to be effective, it would need functioning motor neurons. We are born with the number of motor neurons we lose them with SMA, in different locations and at different rates. In other words, I may lose motor neurons more quickly in my shoulders and another patient may lose them more quickly in their quads. So, it seems to follow that the myostatin inhibitor would be more effective in different ways for patients, in the areas with more functioning motor neurons, allowing for muscle growth to help compensate in areas of muscle loss and help strengthen areas less affected.

I had lunch with several folks in our community yesterday. One of them said they know of a 7 year old who was in the Scholar Rock clinical trials who has had significant benefit in his upper body strength and less so in the lower body. He’s a type 2, so not sure if that makes sense, but it’s certainly anecdotal.

Ultimately, I’m thankful for the ongoing efforts of those working to help us and improve our quality of life. There are studies underway and something called anthrobots, which generate new motor neurons, have been created in the lab. How that translates to animal and then human trials is TBD, but in combination with other treatments seems to point to significant progress. Plus, CRISPR and gene editing is another light at the end of the tunnel. There’s a lot of hope now and more on the horizon!

[alyssa-silva]

It is quite remarkable that we are witnessing and experiencing treatments in our lifetime. I was the first older adult in the world to receive Spinraza in 2016. I think they did trials for patients under 21 years of age. I was 26. My hospital offered an early access program before the FDA approved it, and I was eligible. Receiving that news was the best. feeling. ever. Just had my 32nd dose yesterday!

I’m excited to see where apitegromab goes. I met a young boy receiving it, and he was doing really well. I have the same concerns as you, but I’m so happy children are benefiting from it.

[mike-huddleston]

Could not agree more Alyssa! I was in the first clinical trial for adults almost 20 years ago for valproic acid at Ohio State University (add a “THE” in front of that if it matters to you!! 🤣). At least that’s what I was told at the time. My thoughts then and now were whatever it takes to help get us where we need to be. And, more importantly, as someone who is 61 years old, it has always been about the children first, the rest of us who benefit as a result of studies and clinical trials is secondary. I mean, the treatment options mean SMA type 1 is no longer the leading cause of death in the US for children under 2. How absolutely amazing is that????!!!!

I was also fortunate to have been selected as a member in the first group of 5 adult patients in the state of Maryland to be treated at Hopkins. This was May of 2018 and I still get emotional thinking about that. I did switch to Evrysdi this past September, but the fact that we have options and more treatments on the way is overwhelming.

[alyssa-silva]

So amazing!!! I’ve been reflecting a lot lately because my nonprofit (we raise money for SMA) celebrated its tenth birthday a few weeks ago. SO many breakthroughs have happened in the past decade that it’s almost hard to wrap my head around. And it’s only going to get better from here, which is the best part.

How did the valproic acid study go? I’ve heard a lot of mixed feedback on its efficacy.

Also, Spinraza vs. Evrysdi…there seems to be a lot of back and forth on which is better. Have you noticed a difference– good or bad? Only answer if you feel comfortable!

[mike-huddleston]

First. congrats on the 10th anniversary for your non-profit. That is wonderful!

As far as the valproic acid study, personally I didn’t notice any difference or benefit. That said, I was still ambulatory at the time and could do most things they asked for, but it was basically the Hammersmith, a neurological exam, a DEXA scan annually, and the 6-minute walk test. There were no improvements in any of these in the roughly 2-year study. I have been using a power wheelchair full time since mid 2015.

The switch to Evrysdi was due to a significant drop in my shoulder and upper arm strength, especially on the right side. In September 2021, I had the Spinraza injection site changed from lumber to C-spine in an effort to get more drug closer to the area of greatest concern. Unfortunately, it didn’t help. So, the switch to Evrysdi was in an effort to provide a “jump start” as I may have plateaued on Spinraza. So far, I like it, but admittedly haven’t seen many improvements. A few things are about the same, and a few seem to be slightly better. I do like not needing an injection and taking it daily orally is convenient. I’ll know after my next annual PT assessment, which will be this summer.

[alyssa-silva]

Hopefully, you start to see some improvement soon! I’m still getting Spinraza, but I agree that an oral medication is much easier.

[nikowill399]

From what I’ve seen recently, Scholar Rock is still planning to apply for FDA approval in early 2025, so it seems like things are on track. I agree—it does look really promising, especially for what it’s targeting. And you’re right, I don’t think they’ve done trials on adults yet, but hopefully, we’ll hear more about that soon.

[alyssa-silva]

I’m so eager to learn more! I hope we get more information soon. Do you receive any treatments currently?