biOasis Delivers siRNA to Animal Brain with Therapeutic Effect
This is “actually the very first time that someone has been able to deliver a siRNA to a brain in an animal and seen that there was a therapeutic effect to it,” CEO Rob Hutchison said Monday.
biOasis Technologies (TSXV:BTI) released interesting news on Monday, but understanding what it means requires a little bit of background information about what the biopharmaceutical company is all about.
Put simply, biOasis is focused on the delivery of therapeutics across the blood-brain barrier (BBB) and into the brain tissue. The company describes the delivery of therapeutics across the BBB as “the single greatest challenge in the treatment of over a thousand common and rare diseases of the central nervous system,” and it’s using its Transcend Platform to try to surmount it.
The blood-brain barrier
The reason it’s so difficult to get therapeutics across the BBB is that while the endothelial cells it’s made up of allow the transport of “desirable molecules” between the blood and the brain, they also prevent foreign substances — both harmful and beneficial — from getting across. Unfortunately, therapeutics fall into the category of foreign substances, and as a result over 98 percent of small molecule drugs and nearly all larger biologic drugs can’t enter the brain at therapeutic concentrations.
That’s where biOasis’ Transcend Platform comes in. It’s based on melanotransferrin (also known as MTf, CD228 and p97), a naturally occurring transport protein that’s able to cross the BBB via a process called Receptor Mediated Transcytosis. Under that process, MTf molecules attach to receptors on the cells of the BBB, with the molecules then being pulled into the brain. The way the Transcend Platform works is similar — essentially, it allows MTf to be attached to therapeutics; the resulting structure can then attach itself to a BBB receptor and cross the BBB when infused or injected as a therapeutic.
Ischemic stroke model results
Moving on to Monday’s news, biOasis announced the results from an animal ischemic stroke model. It was performed by National Research Council Canada with siRNA and MTfp, a carrier peptide that’s part of the Transcend Platform of carrier technologies.
Summing up the news in a video, CEO Rob Hutchison explained that back in July 2014 his company was able to deliver siRNA to the brain. “We’d shown that we could actually deliver the siRNA, but we didn’t know if we could deliver it in a therapeutic level — in other words, did it have an effect?” he said.
Ultimately, the model yielded two key findings. The first, said Hutchison, “was that we actually reduced the … infarct area of damage in the brain quite significantly when we linked the siRNA with our … MTf pep, our peptide that we identified.” Secondly, a neurological score test has revealed that “in the case of the Transcend linked with the siRNA, there was a significant reduction immediately of the neurological score, and, more importantly, 24 hours after the treatment had been done, we actually saw that those animals that were treated with the Transcend siRNA together with the peptide actually were returning back to normal.”
According to Hutchison, those findings are important because they “are the first indication and actually the very first time that someone has been able to deliver a siRNA to a brain in an animal and seen that there was a therapeutic effect to it.” That said, he made sure to point out, “how this relates to humans we don’t know until it’s tested in a human.”
Hutchison concluded by noting, “I think it opens up another area of therapeutic regime potentially.” That’s because while siRNA has primarily been used thus far to treat liver disease, it is thought to be involved in diseases like multiple sclerosis and ALS — possibly “a whole host of other neurological diseases [whose] foundation is potentially in the area of overexpressed genes.”
He added, “being able to look at gene therapy and being able take those potential gene therapies and regulate them, or deliver them to specific areas where they can have an effect I think is one of the things that companies will need to have a look at. This is certainly an indication … that we can actually deliver siRNAs and they can have a therapeutic effect.”
No word yet on what the company’s next steps may be, but it will certainly be interesting to keep an eye out moving forward. At close of day Monday, biOasis’ share price was sitting at $1.31, down 5.07 percent, after hitting a high of $1.48 earlier in the day.
Securities Disclosure: I, Charlotte McLeod, hold no direct investment interest in any company mentioned in this article.