Tofersen

But on Friday, I joined the MND community at their annual conference in Adelaide to announce a step change in treatment.

From the first of October, a new drug will be listed on the PBS known as Qalsody.

It's only available for a relatively small cohort of patients that have a particular mutation of the SOD1 protein.

But the results, Mr.

Speaker, are quite extraordinary.

I was joined by Luke, who's only thirty-four, uh, and his two beautiful young children who are running around the media conference.

And Luke has been on Qalsody on a compassionate access basis for two and a half years, and he reported that in that extended period of time for an, uh, for an MND patient, he has experienced no progression of the disease.

Indeed, there are some reports of patients on Qalsody actually, uh, experiencing an improvement in function.

I'm not really impressed by the data, to be honest.

Then there's one called Tofersen, which is all the rage.

Um, the news, news arti- uh, media comes out and touts, uh, drugs like this once in a while.

It, uh, suppresses mutant SOD1 gene, the, the messenger RNA that's produced by that gene specifically.

But honestly, the real excitement right now is in emerging gene therapies.

Oh, like Tofersen.

Yes.

Tofersen was recently approved for patients with a very specific genetic mutation, the, uh, SOD1 mutation.

It literally binds to the faulty genetic blueprint to stop the production of a toxic protein.

It's just wild what they can do now.

And what she allowed me to do, just like we're saying, is that although I don't have the same form of ALS that she has, Developments are happening.

And by seeing that happening, again, in my lifetime, although Tofersen may not be the thing that works for me in any capacity, the next Tofersen hopefully is on some scientist's table right now being developed.

By being such a great advocate for what she's going through, giving hope to not only those who have the same SOD1 variant, it's also very empowering to people like myself thinking the next development is just around the corner.

Yeah.

Okay.

Jason participated in the Phase 1 clinical trial for Lumina, a trial investigating the drug AMX-0114.

Like Kalsodi, also known as Tofersen, a medication approved by Health Canada for the treatment of SOD1 ALS in 2025, AMX-0114 utilizes antisense oligonucleotide technology and may provide a viable treatment option for PALS without the SOD1 variant.

Jason and his neurologist have an understanding that nothing is off the table for Jason.

The trial is blinded, so nobody at the company has seen the data, um, just to say that.

And we're very, very excited about that because if you look at, you know, current treatment for ALS in the sporadic population of what we're going after, unlike what, like, Biogen is going after, um, with Tofersen, which is the SOD1 genetic patient population.

Um, you know, the, the drugs are frankly modest in efficacy.

If you look at Riluzole-

It's never been approved in, in Europe.

So not a great option.

And then you have Tofersen approved for SOD1 patients.

That's, like, 3% or 4% of ALS, uh, you know, patient population.