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[sev22] 19/10/2021 11:39

SwanBio Therapeutics Announces New Preclinical Data Demonstrating Proof of Principle for SBT101 Gene therapy as a Potential Treatment of Adrenomyeloneuropathy (19th October 21).

- SBT101 Demonstrated Dose-Dependent Improvement of AMN Disease Markers in Mouse Models

- SBT101 Well-Tolerated in Non-Human Primates at 3-Months Post-Treatment

- Data Support Continued Advancement of SBT101 in IND-Enabling Studies

SwanBio Therapeutics, a gene therapy company focused on developing treatments for patients with genetically defined neurological conditions, today announced new preclinical efficacy and safety data supporting the potential of the company’s lead program, SBT101, as a disease-modifying treatment for adrenomyeloneuropathy (AMN). The data will be presented in two posters, “Intrathecal delivery of an AAV encoding human ABCD1 shows dose-responsive expression and activity in a mouse model of Adrenomyeloneuropathy,” and, “Three-month preclinical safety data of AAV9-hABCD1 following intrathecal delivery in non-human primates,” during the European Society for Gene and Cell Therapy (ESGCT) Virtual Congress. The Congress is being held October 19-22, 2021.

AMN, the adult-onset degenerative spinal cord disease occurring in patients with adrenoleukodystrophy (ALD), is characterized by progressive loss of mobility, incontinence and debilitating pain. ALD is caused by mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Dysfunction of this protein leads to VLCFA accumulation in tissue and the progressive axonopathy of AMN. SBT101, a novel AAV-based gene therapy, is designed to compensate for the disease-causing ABCD1 mutation, increasing ABCD1 expression and reducing VLCFA levels.

“We are excited to share the first data on SBT101, which demonstrate its potential to directly address the underlying cause of AMN by establishing ABCD1 expression in the spinal cord,” said Karen Kozarsky, Ph.D., Swan’s chief scientific officer. “AMN is a devastating disease with no approved treatments, and we are pleased to have observed improvements in markers of AMN disease as well as good tolerability in both mice and NHPs. Taken together, these findings support the continued evaluation of SBT101 as a potential disease-modifying treatment for AMN as we advance towards the clinic.”

Summary of Findings

The safety and efficacy of SBT101 were evaluated in mouse models of AMN and non-human primates (NHPs). Key findings include:

Dose-dependent expression of hABCD1 and reduction of VLCFA levels after transduction of SBT101 in glial cell cultures from ABCD1 knockout mice.

Durable increase in hABCD1 protein expression and significant reductions in VLCFA levels at 13 weeks post-treatment with intrathecal delivery of SBT101, which were maintained at 24 weeks post-treatment, in an ABCD1 knockout mouse model.

Well-tolerated in NHPs at 3 months with expected transient dose-dependent increases in ALT and AST observed that resolved within 2 months post-treatment. Further, no advanced grade histopathological findings were observed in tissues, including the dorsal root ganglion.

Based on these findings, SwanBio plans to continue its ongoing investigational new drug (IND) enabling studies for SBT101 to support a planned IND submission.

About Adrenomyeloneuropathy

Adrenomyeloneuropathy (AMN), the adult-onset spinal cord disease of adrenoleukodystrophy (ALD), is a progressive neurodegenerative disorder. It is caused by mutations in a single X-linked gene, ABCD1. The ABCD1 gene encodes a protein that resides in the cells' peroxisomal membrane. Dysfunction of this protein results in accumulation of very long chain fatty acids (VLCFA) in the blood, tissue and cerebrospinal fluid and progressive neurodegeneration in the spine. Symptoms of AMN typically begin in men between the ages of 20 and 30. Women can experience symptoms later in life. Early symptoms for people living with AMN include leg weakness, such as difficulty walking and balance issues. Later in the progression of the disorder, patients lose mobility and bladder and bowel dysfunction have a significant impact on quality of life. Beyond physical symptoms, people living with AMN report economic hardship and loss of dignity and self-esteem as the disease progresses over time.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTx.com.

[sev22] 30/9/2021 16:03

Gyroscope announces additional positive interim data from Phase I/II FOCUS trial.

[chc15] 30/9/2021 08:07

Yeh these biotechs are just a gamble. Only have a small bit in here because of that.

[grim] 29/9/2021 20:24

I'm hoping mm's are accumulating in anticipation of major news/results from investee trials - but it's only a hope. These were flavour of the month with many Fund Mgrs & pundits a couple of years ago and nothing majorly negative has been announced but wtfdik

[chc15] 29/9/2021 19:55

Wow this is plummeting fast, any ideas or buyers?

[salpara111] 17/8/2021 14:01

Glad I took a small loss a couple of days ago as opposed to a bigger loss today!
NAV is falling at quite a rate.

[davemac3] 02/8/2021 22:08

is this the reason for today's rise?

[tomps2] 16/7/2021 11:56

John Rosier mentions Syncona (SYNC) in the latest PIWORLD interview at 12m14s

Watch the video here:

Or listen to the podcast here:

[1seanshare] 24/6/2021 19:09

SYNC sinking further. Why so negative?

[luxaeterna1] 21/5/2021 11:01

Depends how you want to look at it. CAR-T sales are probably growing 40-80% per year at the moment, with a substantial number of trials with people like Autolus.

Heck, Autolus are even on the lower end of the risk/safety profile, so they have some 1st mover advantages there. Although I am not clear how Autolus will scale long term. CAR-T are not simple processes.

[chc15] 13/5/2021 15:27

T Henderson, check director rns.

[1seanshare] 13/5/2021 10:16

Yes that's correct. My concern is about CAR-Ts in general. They aren't the be all they were promised to be, there is considerable toxicity for patients who are already heavily pretreated, and they are very complex and onerous to deliver. It could be that CAR-Ts in general are not being used as much as first envisaged

[luxaeterna1] 13/5/2021 09:31

Am I correct, that iNHL is basically slow growing lymphoma, so a 100% complete response rate (in phase 1), would be indicative that Autolus CARs being used at earlier stages?

[mayers] 12/5/2021 23:09

As you say 1seanshare "that's welcome", may be you should have said Welcome :-)a very aristocratic organisation which has had tremendous power but with whom I have mixed feelings in the modern competitive market. Maybe past its heyday.

[luxaeterna1] 12/5/2021 20:09

iNHL results go BOOM for Autolus.

[1seanshare] 12/5/2021 14:01

Which one, where do you see that?

Is the price drop not an over reaction to gyroscope delay?

[chc15] 12/5/2021 13:33

Director dumping loads.

[acuere] 11/5/2021 13:10

Sold out a while ago and been watching from the sidelines. Tempted to jump back in but I’d prefer to see a change in fortunes.
Even since the poor Q1 figs to 31st March, Autolus down 16%, Freeline down 25% and Gyroscope IPO announced and pulled.
Not good.

[luxaeterna1] 11/5/2021 09:08

It is odd. I suspect they meant it was simply due to covid-related uncertainty, which meant an unacceptable pricing offer. Although Gyroscope (long term eye health) may operate on a longer timeframe than your average IPO and that might discourage investors.

Interesting to see if they wait 6-9 months, or look at Syncona/3rd party private partnership.

Ha, oh wait, I just re-read the RNS, it does look quite silly having a 4-day delay between the RNS announcing and then postponing it doesn't it? I'm sure they cringed at that.

On the other hand, a good drop in sentiment like this, could actually be a solid entry position if you believe in the long-term prospects of SYNC, & in particular Autolus.

[salpara111] 07/5/2021 08:39

Beginning to think I made a mistake getting into this.
To announce an IPO and then decide only a couple of weeks later that you are pulling it "due to market conditions" makes management look incompetent!

[1seanshare] 06/5/2021 19:56

What on earth happened to Freeline today? At one point it was down 25%. SYNC being held back by Freeline and Autolus?

[luxaeterna1] 18/4/2021 11:05

What do you guys think about the recent Autolus decision to focus manufacture on the UK plant? I suppose cashflow has been tight, so they are trying to avoid initial capex, on a facility that I asusme would take significant staff qty, training, QA and approvals etc. In my head, it doesn't seem that it would grossly affect logistics by having to ship to UK instead (package deliveries can be overnight from major cities, so frankly it would take overnight to get domestic shipments done).

Overall, I see it as a positive development for the UK staff and getting things rolling. Perhaps when they have significant cash revenues they can look at expansion. Does anyone know what the ultimate Autolus target market is? Initial treatments in EU/US could show ~ 20k patients per year, but this could become a significantly higher figure if they get more approved treatments.

Between the various genetic treatments being presented by the wider Syncona group, you could argue that number starts to become millions in 10 years time.