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Welcome to Connecting ALS. This week, we discuss the approval of AMX0035.
Connecting ALS is a weekly podcast produced by The ALS Association in partnership with CitizenRacecar. We aim to discuss research and technology developments, highlight advocacy efforts, and share the personal stories woven through the community.
This transcript was exported on Oct 04, 2022 - view latest version here.
Calaneet Balas:
It won't be just for the ALS community, it'll be across the entire rare disease space. So this, to me, is a big step for not just our community, but many communities across our sector.
Jeremy Holden:
Hello everyone and welcome to Connecting ALS. I am your host, Jeremy Holden. In the summer of 2014, millions of us took part in the ALS Ice Bucket Challenge. Celebrities, friends, and family members all dumped ice on their heads to help raise awareness and funds for ALS.
Jeremy Holden:
Thanks to everyone who donated new ALS genes were discovered, new global research projects were funded, and care services were expanded to help more people with ALS. One of the potential treatments that The ALS Association funded was AMX0035, which was first imagined by two Brown University students in a dorm room the year before the Ice Bucket Challenge. The ALS Association spent over $2 million helping fund the development and clinical trial of AMX0035.
Jeremy Holden:
When the results of that trial showed that it was safe and effective in treating ALS, The ALS Association led an advocacy campaign to push the FDA to approve the drug. After two years of advocacy, the FDA finally approved AMX0035.
Jeremy Holden:
Joining me now to talk about the fight for access and approval of AMX0035 is The ALS Association President and CEO, Calaneet Balas.
Jeremy Holden:
Well, Calaneet, thank you as always for gracing us with your presence and giving us some time here on Connecting ALS.
Calaneet Balas:
Well, thanks for having me. I don't know about gracing you with my presence. It's lovely to talk to you as always.
Jeremy Holden:
Oh, come on. Yeah, of course.
Jeremy Holden:
Well look, the FDA has approved AM0035 for the treatment of ALS. Just for starters, how significantly does this change the landscape in terms of making ALS livable?
Calaneet Balas:
Oh, I mean, first of all, what a big moment for the ALS community, not just here in the United States, but across the globe. Just huge, huge progress and movement. I think it's going to change the landscape in so many different ways. We will be talking a lot about access and making sure people actually can get not only this therapy, but other therapies that I'm hoping and praying will come across the line. And when we think about making ALS a livable disease, I mean now we have a drug that has shown that it can extend life significantly and hopefully get people to that next therapy.
Calaneet Balas:
I see great things for the future for making ALS a livable disease. And I really believe that the community did this together and we will all continue to look toward how can we actually really come together to make some massive progress to reach our goal. I'm excited.
Jeremy Holden:
We shared some of the testimony from the advisory committee hearing from a couple weeks ago, we shared that with listeners, and it was so moving to hear, from the community, about their just desire and just really please make this available. We want to talk to our physicians. We shared that letter that dozens of ALS clinicians signed saying, "We want to talk about this. We want this as a potential treatment, for the patients that we serve."
Jeremy Holden:
The path to get here has been, at times, arduous. It's been a bit of time. But how do you reflect on the amount of work that it took to educate the FDA along the way?
Calaneet Balas:
I think that is time incredibly well spent. We educated not only ourselves, the community, and the FDA really all together. And I'm not sure it really would've happened any other way. We spent a lot of time, effort, blood, sweat, and tears to make sure that everyone was involved. We were all at the table in different ways. There were a lot of frontend conversations, whether it was the We Can't Wait conversation that we hosted. There were also a lot of backend conversations and emails and conversations with the FDA. And I think only through that process would we have landed here. That takes a lot of tenacity, but I think it's tenacity very well spent, again.
Calaneet Balas:
I really think what it will do is pave a way of how do we continue to have that conversation, not just with the FDA, but with other regulatory bodies that will have great impact as to: How accessible are drugs, and how quickly can we get them to people who need them. And it won't be just for the ALS community, it will be across the entire rare disease space. So this, to me, is a big step for not just our community, but many communities across our sector. Again, time absolutely well spent. I would do it again in a minute.
Jeremy Holden:
And you may have to. We may have to as a community. Dr, Kuldip Dave has been on this program frequently, and he's talked often about the robustness of the drug development pipeline today, and that's exciting. Any learnings from the process of getting AMX approved that can inform future drug approval decisions?
Calaneet Balas:
Well, we're going to take a lot of learnings, but I think to take a step back and think about that drug development pipeline that you just talked about, one of the big wins out of this is we wanted to make sure that our drug development colleagues and partners knew that we can get this over the line if the science is there. And that's really, really critical for people wanting to invest in research, in drug development in ALS. So, that's a massive win outside of just this particular drug and I think a big learning lesson of where we might go and what we might do going forward.
Calaneet Balas:
The other piece is that we've learned is how important it is to bring the community along. I think everyone knows that in their gut, but we really saw the fruits of that labor. I was so impressed by the amount of physicians that also stepped up later in the process to say, "No, this is what we really think." And this is how they can come together and be a cohort. So I think those learning lessons are: keep pressing; maybe do that in a very time sensitive manner, which is what we have been trying to do.
Calaneet Balas:
I think one of the big wins here was, the company was very diligent about making sure that we had the information we needed to try to make very informed decisions. Sometimes when those are delayed or that information is delayed, what we can do or how we can step forward is also delayed because we want to make sure we have the best information in hand.
Jeremy Holden:
Always need to be grounded in the science and driven by the science and what the data actually says. It's not a situation where a drug is approved and someone can go to CVS tomorrow and get a prescription filled. What needs to be done in the coming weeks to make sure that people have access, that the people that we serve have access to AM0035?
Calaneet Balas:
Yeah, now's the hard part. Now's the hard part.
Jeremy Holden:
Now's the hard part.
Calaneet Balas:
It's all kind of the hard part. Now we need to get focused and understand. I know the company's getting in front of payers and providers and trying to understand what that pricing mechanism will be and really ramping up. I think it will be really critical, as a community, to lean into CMS, Medicare, Medicaid, and those areas to make sure that not only is it priced in a way that can it actually get to people living with ALS, but that we have accessible channels.
Calaneet Balas:
This is going to be a big shift for our community. We saw it happen when Radicava hit and started to come out. Obviously a very different drug. When it originally came out, now there's an oral form, but when it originally came out, it was an infused drug, so much more complicated for a person to take that type of drug.
Calaneet Balas:
But there's a lot of lifting that goes on, not just with the payers, but with the providers, meaning the clinics, the doctors, all that backend stuff. If anyone's ever gotten a prescription drug that's a little bit, not just a Z-Pak for your cold or flu, it can be a lot of back and forth, so there's a lot of education that has to happen through our entire health care network so people feel comfortable and are resourced to maybe have to battle a little bit with some of our payers. I think those will be the next steps that we are already really focused on.
Jeremy Holden:
A fight that never ends. Hopefully, you mentioned the approval of Radicava, hopefully with that drug development pipeline being as robust as it is, this process becomes smoother as more drugs get through the process of being approved and being made available.
Jeremy Holden:
It's hard to believe that it was eight years ago that the Ice Bucket Challenge kind of swept the nation. AMX0035 was one of the first research projects funded by the proceeds and the donations that came from the Ice Bucket Challenge.
Jeremy Holden:
What message do you have for the millions of people who took part in the Challenge and the role they played in shifting the landscape of ALS?
Calaneet Balas:
Yeah, I remember that funding stream very well, partly because of just the unusual nature of how it came about. These two young men sitting in their dorm room at Brown and decided, "Oh, let's try to do this very miracle cure of some sort for ALS."
Calaneet Balas:
When you talk about ALS and the Ice Bucket Challenge, the message I have is thank you. Thank you for being a part of it. See, this works.
Jeremy Holden:
Yeah.
Calaneet Balas:
I've said for years that when you have money, money invested in research and we find answers, we learn things, good things, bad things, how to move forward. And when we don't have those resources to invest in understanding, it doesn't move. And this is a perfect example of how something can move, and actually at a pretty quick pace when it comes to drug development. I mean, this is not a long time to go from conception to market and really get out to the patient.
Calaneet Balas:
We have to keep going. That's the other message is we know this isn't a miracle cure, this isn't a complete home run, but it's hopefully a good base hit, and we really need to keep moving forward. Thank you. It works when you put dollars into the pipeline and you start to understand the disease more. And we're starting to see the fruits of that.
Jeremy Holden:
Well, as a fan of the game, I know a lot of the best ones are won with a series of base hits. It doesn't always take a home run-
Calaneet Balas:
That's right.
Jeremy Holden:
... to win the ball game. Calaneet, thanks, as always, for dropping by and catching us up on a huge development in the ALS world.
Calaneet Balas:
Thank you. It's always a pleasure to spend some time, especially in a moment like this, so thank you so much and thank you to the entire community and all of our listeners.
Jeremy Holden:
I want to thank my guest this week, Calaneet Balas. If you liked this episode, please share with a friend. And while you're at it, rate and review Connecting ALS wherever you listen to podcasts. It is a great way for us to connect with more listeners.
Jeremy Holden:
Our production partner for this series is CitizenRaceCar, post production by Alex Brouwer, Production Management by Gabriela Montequin, supervised by David Hoffman.
Jeremy Holden:
That's going to do it for this week. We'll connect with you again soon.
ConnectingALS_100622_Ready1 (Completed 10/04/22)
Transcript by Rev.com
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