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Bioasis has the best platform technology for shuttling drugs across the blood-brain barrier

Last updated: 07:30 18 Jun 2021 EDT, First published: 07:30 18 Jun 2020 EDT

Snapshot

The company’s patented xB3 platform has shown a superior delivery of active payloads into the brain, significantly higher than other methods

Bioasis Technologies Inc. - Bioasis has the best platform technology for shuttling drugs across the blood-brain barrier
  • Platform licensed to Chiesi for developing drugs  
  • Will receive $3 million upfront, and as much as $138 million in additional milestones and undisclosed royalties
  • Has inhouse development programs targeting neurological diseases

What Bioasis Technologies does:

Bioasis Technologies Inc (CVE:BTI) (OTCQB:BIOAF) is a biopharma company developing the xB3 platform to aid in the delivery of molecules to the brain, and the treatment of brain cancers and neurodegenerative diseases.

The blood-brain barrier plays an indispensable role in protecting the brain from blood-borne disease. However, it also blocks crucial medicines from reaching the brain posing an epic challenge to treating neurological diseases.

Scientists at Bioasis have worked for over a decade to develop the patented xB3 platform, which helps small molecules shuttle across the blood-brain barrier (BBB) safely. Like a key designed to open a lock, the xB3 platform unlocks the door to the blood-brain barrier, allowing compounds into the brain. In a nutshell, the xB3 platform uses a small peptide to ferry molecules across the BBB in a process called receptor-mediated transcytosis.

The Guilford, Connecticut-based company’s in-house drug programs are designed to develop treatments for neurological diseases. The lead program, xB3-001, is in preclinical development for brain metastases in HER2+ metastatic breast cancer. Bioasis is also working on xB3-002 aimed at treating glioblastoma, one of the most aggressive cancers originating in the brain, with 80% of diagnosed malignant brain tumors being gliomas. 

The company has additional preclinical programs in pain, neurodegenerative diseases and rare diseases.

How is it doing:

On June 3, Bioasis announced the publication of research validating the ability of its xB3 platform to cross the intact blood-brain barrier (BBB) and its localization in a variety of brain cell types. The company said Professor Wilfred Jefferies together with scientists from both the Jefferies Laboratory and Bioasis used multiple approaches to help pinpoint and then evaluate Bioasis’ xB3 platform technology by investigating its ability to traverse the BBB. 

The company said this research shows that the xB3 platform can cross intact BBB and enter intracellular organelles within neurons, glia, and microglia in the brain. It said the data from a study published on June 2 in the Frontiers in Neuroscience provide evidence for the utility of xB3 peptide (previously known as MTfpep) as a platform technology for delivery of recombinant and chemically conjugated drug across the BBB. 

Earlier, in March, a study published in the “Frontiers in Molecular Biosciences” showed that the xB3 platform can act as a delivery vector to facilitate blood-brain barrier translocation of siRNA, reducing stroke damage in the brain and improved neurological function. The data presented in the publication provided evidence for the utility of xB3 peptide (previously known as MTfpep) as a platform technology for delivery of recombinant and chemically conjugated drugs across the blood-brain barrier.

In the same month, Bioasis also revealed that it had formed a research collaboration with Aposense Limited, an Israeli bio-pharmaceutical company specializing in development of novel drugs utilizing membrane electrical forces. Bioasis said the research partnership will focus on the delivery of small interfering RNA (siRNA) into the brain using the company’s xB3 platform technology. 

Meanwhile, Bioasis recently struck a royalty purchase pact with XOMA Corp. to sell certain rights to milestone and royalty revenue from the advancement of four enzymes that the firm is investigating as a treatment for lysosomal storage disorders (LSDs), which are inherited diseases caused by a build-up of toxic material in the body's cells due to enzyme deficiencies. People with lysosomal storage disorders lack enzymes that break down certain lipids (fats) or carbohydrates (sugars) in the body.

In exchange for a $1.2 million upfront payment, XOMA will receive a low single-digit royalty on future net sales of each of the four enzymes and an undisclosed share of the up to $138 million in potential milestones made possible by the Bioasis-Chiesi Group alliance. The arrangement with XOMA, will allow Bioasis to deploy more capital to advance its unpartnered pipeline programs targeting Parkinson’s disease, Lewy Body Dementia and Fronto Temporal Lobe Dementia and also secure technology validating partnerships.

The enzymes are being developed under Bioasis’ strategic alliance with the Chiesi Group. At the end of June last year, Bioasis struck a deal with Chiesi to license its xB3 platform technology to develop drugs for four LSDs. In return, Bioasis will receive $3 million upfront and up to $138 million in additional milestones and undisclosed royalties. Chiesi will be responsible for all costs related to research, development and commercialization of the four undisclosed LSD programs.

Significantly, the US has issued a patent for xB3 technology, which represents a major milestone for the firm’s intellectual property protection around its core technology and its xB3 programs

Meanwhile, Bioasis has commissioned Bluestar BioAdvisors to provide a commercial assessment of its lead drug xB3-001, developed for the treatment of HER2+ cancers and brain metastases. Bluestar reported that when used strictly to treat HER2+ breast cancer and brain metastases, xB3-001 could generate nearly $440 million in annual worldwide revenue.

However, the company said preclinical data suggests that xB3-001 is likely to perform outside the brain as well as intravenous drug Herceptin that is part of a chemotherapy regimen. Therefore, xB3-001 may have the potential to become the standard of care for all patients with HER2+ breast cancer for which Herceptin is prescribed.

Bluestar BioAdvisors pegs this larger market to be as high as $12.4 billion annually for all treatments of HER2+ breast cancer worldwide. With an estimated 30% share of this market, xB3-001 could produce annual revenue of $3.7 billion, so has blockbuster potential.

For the time being, xB3-001 has investigational new drug (IND) status enabling studies. As additional funding is secured, it is likely to bring forward milestones for xB3-001, in particular the IND filing with the US Food and Drug Administration (FDA) which is currently anticipated towards the end of the calendar year 2021.

Inflection points:

  • Expect partnerships for the xB3 drug delivery platform and licensing deals
  • Several pipeline programs expected to reach key milestones
  • New data to drive fresh partnership interest

What the broker says:

Analysts at Edison Investment Research modeled a higher valuation for Bioasis driven by its strategic alliance with Chiesi Global Rare Diseases.

“We see this as a major validation of the platform and for additional future licensing potential,” said Edison analyst Nathaniel Calloway. “We increased our valuation to C$60.1 million or C$0.89 per basic share, from C$47.8 million or C$0.71 per basic share, driven by the addition of the Chiesi partnership to our model.”

Edison Research noted that the xB3 platform may offer an “attractive solution” to improving central nervous system (CNS) drug activity because it is entirely modular.

“Enzyme replacement therapies have already been developed for a range of lysosomal storage disorders, and the development of a brain penetrant derivative of these drugs could be as simple as producing a conjugate to the xB3 peptide developed by the company,” said Calloway.

Edison also emphasized that the value of the xB3 platform is both in the “wide array of cargo that can be delivered” as well as the “rate of transfer,” which is “significantly higher than other methods that have been tried, at least in model systems.”

In October 2020, Zacks Small-Cap Research gave Bioasis a US$0.60 price target. In the long term, Zacks estimates a 2030 approval and launch of the two candidates, known as xB3-001 and xB3-007, respectively. 

What the boss says:

In June's statement regarding the study published in the Frontiers in Neuroscience, Dr Deborah Rathjen, executive chair of Bioasis’ board commented: “These data further validate the utility of our xB3 platform technology to achieve delivery of therapeutic compounds across the BBB at levels that may help treat a variety of CNS diseases.

"This data is consistent with the previous studies where our technology successfully delivered enzymes, antibodies and siRNA across the BBB with demonstrated efficacy in both the brain and the periphery in settings as diverse as brain cancers, neuropathic pain and lysosomal storage disorders.”

Contact the author at jon.hopkins@proactiveinvestors.com

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