Small interfering RNA

I want to ask Yaron to talk to us a little bit about the Ionis fusion trial success.

I mean, company has had some tough times these days with the continued conversation about antisense ASOs versus siRNA and the Lp little a and the, um, situation, uh, the story with AstraZeneca.

Talk us through what's going on here, and maybe, maybe Yaron, um, just bracket the Ionis story as it stands today.

Yeah, yeah.

So it's a great point.

So I think one of the challenges for Ionis is the, the conversation, and I'm not faulting it, um, I'm just pointing it out, is still siRNA versus ASOs.

And, um, I, I don't know that we agree with that necessarily, uh, that that should be the conversation because each one of these, uh, studies did well or didn't do well by virtue of its own design, where we are in the treatment paradigm in this area, and comparing data necessarily to historical data, um, across different populations is challenging.

And now we actually can compare the ASO to the siRNA and SHTG, and they actually look very similar.

And that was critical for treatment of transthyrethin.

It's an amyloid disease when there's a mutation there and And you deliver these siRNAs, that gene was expressed in the liver, and you could silence it.

And then we've developed the technology for other liver-based diseases, Nylum has, and then many others.

I think it's got six approved products now.

They're a California company, and they're hoping to raise around $318 million at around $16 per share.

So that's a pretty sizable IPO, and that money will go towards fueling the biotech's pipeline of siRNA candidates.

I'm excited to see, you know, when they actually hit the market, if they kind of fit into that upsized financing trend that we've been seeing so far for a lot of other biotechs this year.

Yeah, I was gonna say that is a big number, but we've seen it this year before.

They did, yes, uh, early this morning, pointing to another offering in roughly the same range, three hundred and fifty million.

That company is an siRNA focused organization with three clinical candidates.

I don't see a timeline yet on Nasdaq's website, but usually if you have terms by Monday morning, it's likely to get out later in the week.

So, you know, we'll watch.

Welcome back to the Business of Biotech.

I'm your host, Ben Comer, Chief Editor at Life Science Leader, and today I'm speaking with Alex Arivanis, MD, PhD, CEO at Moonwalk Biosciences, a company developing new therapies targeting adipose tissue or body fat that aim to improve upon currently available treatments for obesity, namely GLP-1s, using siRNA therapies.

Alex was previously a co-founder and the head of R&D at Grail.

He was chief technology officer at Illumina and also worked as VP of development and chief science officer at Sapphire, a company that made crude oil out of algae.

I mentioned that in the intro as well.

You're working in a red-hot therapeutic area where you have some formidable competitors in Eli Lilly and Novo Nordisk.

What made you, Alex, want to tackle obesity with an siRNA therapy?

Yeah.

The, uh, livodeserin is either gonna be once or twice a year, so that's kind of interesting that, again, if their trials are successful and it comes out.

And there's another, uh, S-I-R-N-A, but they're looking for funding, so phase three hasn't been entered yet.

Zero deserin, I think it's called.

And then we have a very intriguing oral drug.

It was a great company.

SIRNA is a Nobel-winning technology that is transformational.

Alnylam had, at that point, a couple of million dollars in sales on products at the time.

It was a commercial company, but most people looked at it as a R&D organization with a very large R&D budget, a lot of promise, but nevertheless, they were very anxious to see, many investors were anxious to see Alnylam become a profitable company, a real pharmaceutical company.