An Emerging Medical Market Play with Great Opportunity

(Image via Bioasis Technologies Inc.)

Biopharmaceutical Company Bioasis Technologies Inc. (TSX-V: BTI, OTCQB: BIOAF, Forum) is developing its proprietary xB3TM platform technology to deliver therapeutics across the blood brain barrier (BBB) and into the brain. The Company was created through the acquisition of the forerunner of its core xB3 technology from the University of British Columbia (UBC).

Using its xB3 best-in-class technology platform, Bioasis has built a pipeline of de-risked drug candidates based on approved, marketed products targeting brain cancers, lysosomal storage disorders, neurodegenerative diseases such as Parkinson’s disease and Lewy Body Dementia and non-opioid treatment of pain. A number of the Company’s pipeline programs may have the potential to qualify for expedited marketing approval from the US Food and Drug Administration (FDA). The xB3 platform offers a versatility that allows Bioasis to partner with companies who are looking to overcome brain drug delivery problems posed by the BBB and to successfully deliver therapies into the brain.

The Company recently signed a licensing deal with the international research-focused specialty pharmaceutical company Chiesi Group working with one of its business units Chiesi Global Rare Diseases. In late June 2020, they announced that they had entered into a worldwide, exclusive licensing agreement for the xB3 BBB platform technology in treatment of rare diseases with a focus on four undisclosed lysosomal storage disorders. Under the deal, Chiesi Group will be responsible for all costs associated with research, development, and commercialization of the four undisclosed lysosomal storage disorder (LSD) programs.

It is through strategic partnerships such as this that Bioasis can retain commercial upside, from a broad range of central nervous system (CNS) therapeutics, while also providing non-dilutive financing and ensuring the broadest possible uptake of the technology. BTI boasts a very solid intellectual property (IP) portfolio and has been granted key patents in important markets, such as the US, Europe, China and Japan.

Stockhouse Editorial had the chance to catch up with Bioasis’ Chair & Chief Executive Officer Deborah Rathjen, Ph.D., to find out more ….

1. Thank you for taking the time to speak with us. To start, can you break down for us the unmet need this platform is designed to treat?

The xB3 platform is a drug delivery system for enabling drug treatments which would not normally be able to do so, to cross the BBB and move into the brain. There are many conditions affecting the brain for which there are currently no effective treatments, in many cases simply because potentially effective medicines cannot penetrate the BBB. This may because they are large molecules such as antibodies or enzymes or because other intrinsic physicochemical properties of the molecules prevent them from crossing the BBB unaided. The xB3 platform is a very versatile, high capacity delivery system able to deliver antibodies, enzymes, siRNA as well as small molecules across the BBB. We believe that xB3 has the potential to deliver effective treatments into the brain for a very large number of conditions such as Alzheimer’s disease and other forms of dementia, Parkinson’s disease as well as for the treatment of bran cancers.

2. It has been estimated that the overall market for drugs to treat brain disorders is estimated to reach a value of nearly $103.23 billion worldwide this year. How does the emergence of cancer as the fastest growing segment of the CNS therapeutics market play into the strengths of Bioasis’ technology and strategy?

With a number of programs in our pipeline targeting brain cancer, it is fair to say that the emergence of cancer as the fastest growing segment of the CNS therapeutics market plays to our strengths. For example, Bioasis’ lead program is xB3-001 a fusion protein containing both the xB3 peptide and Herceptin. Roche/Genentech’s Herceptin™ is used widely to treat HER2+ breast cancer across multiple lines of therapy. With annual sales of about USD $7 billion prior to the market entry of biosimilars, it is a well-known and very successful monoclonal antibody (biologic) that has been available since 1998 however because it cannot cross the BBB, HER2+ brain metastases, which occur in up to 50% of HER2+ breast cancer patients, remain essentially untreatable. We have a very strong preclinical data package that supports the move of xB3-001 into clinical development.

3. Can you tell us more about Bioasis Technologies’ proprietary platform, xB3, for enabling drugs to cross the BBB and how it works?

The xB3 platform exploits one of the body’s natural mechanisms to deliver its payloads across the BBB. The cells that form the capillary walls of the BBB have receptors on their surfaces to which proteins can attach themselves. Once attached to a receptor, some receptor/protein combinations are caused to enter the BBB cell and to then exit the opposite side of the cell into the extracellular fluid of the brain.

This combined process of transport across a BBB cell and into the brain is called receptor-mediated transcytosis. One of the receptors responsible for this process is LRP1, the Low-Density Lipoprotein Receptor-related protein receptor.

The main element of the xB3 platform is the xB3 peptide, derived from an iron-binding protein, melanotransferrin. The xB3 peptide has been optimized by Bioasis’ scientists to its key constituents of 12 amino acids, less than 2% of the size of its underlying protein. Using specially designed linkers, the xB3 peptide can be attached to virtually any biologic molecule. xB3 and its payload is moved across the BBB by the binding of xB3 to LRP1.

4. The Company’s lead product is xB3 – 001, based on the approved drug Herceptin, is in development for the treatment of HER2+ breast cancer and brain metastases, can you tell us more?

Bioasis has developed its own version of Herceptin called xB3-001, a genetic fusion protein consisting of the xB3 peptide attached to Herceptin. Animal studies have shown that xB3-001 can cross the BBB and effectively treat brain tumours. Approximately ten times more Herceptin enters the brain when it is attached to xB3. Importantly, delivery by xB3 does not impact the pharmacokinetic, binding or activity of the payload outside the brain.

5. What are some projected peak revenues for xB3-001 and HER2+ breast cancer?

Bioasis’ xB3 pipeline drugs have all been selected to meet certain criteria, including significant medical need and potentially rapid advancement through clinical trials. Bioasis’ pipeline drugs, if approved by regulators, also have the potential to generate hundreds of millions to several billions of dollars in annual revenues. For a robust assessment of its lead drug, xB3-001, in September 2019 Bioasis commissioned Bluestar BioAdvisors to provide a report of the drug’s potential revenue streams.

Bluestar BioAdvisors reported that when used strictly to treat brain metastases, xB3-001 could produce approximately US$440 million in annual revenues, worldwide. However, xB3-001 is likely to perform outside the brain as well as Herceptin does. xB3-001, therefore, may have the potential to become Standard of Care for all patients with HER2+ breast cancer for which Herceptin is prescribed. Bluestar BioAdvisors report this larger market to be as high as US$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 revenues of US$3.7 billion.

6. Can you give us a timeline on when xB3-001, is expected to enter the clinical development phase?

We have made considerable progress towards the clinic in terms of manufacturing, stability testing and assay validation. IND enabling activities still to be initiated include non-human primate toxicology and GMP manufacture. These activities await additional funding and, in this regard, we are working on additional potential licensing deals to generate the required funding. We currently anticipating being in a position to complete these activities and file an IND in 2021.

7. Let’s talk about your recently announced partnership with Chiesi Group, what does this mean for your combined ability to bring innovative therapies to rare disease patients?

Bioasis pursues purposeful and strategic business development outreach that aims for synergies with our platform whilst retaining upside for Bioasis. We enter into alliances that drive long-term value creation and that broaden the utility and use of technology across multiple indications and treatment modalities. This collaboration with Chiesi Group is an exciting demonstration of the strength of our technology and the robustness of our platform along with attractive deal metrics.
Chiesi is an ideal partner for us, where we can combine our leading BBB technology with their expertise in rare disease. Chiesi Group is focused on advancing research and new product development for rare and ultra-rare diseases. In February Ghiesi Group opened a new business unit in Boston to concentrate on lysosomal storage diseases, rare hematology and ophthalmology disorders.

8. With an upfront payment of $3 million (USD), the eligibility of additional milestone payments of up to $138 million, as well as additional royalties on net sales of licensed products, there appears to be a lot of upside for Bioasis under this partnership, is it safe to say that together you will offer each other a lot of opportunity?

This deal provides Chiesi Group with worldwide, exclusive license to use Bioasis xB3 TM platform for delivery of undisclosed enzymes in treatment of four lysosomal storage disorders (LSD). Chiesi Group are responsible for all costs associated with research, development and commercialization of the four undisclosed LSD programs.

The alliance provides us with near-term capital to advance our preclinical programs. This alliance represents a further validation for Bioasis’ technology and endorses our current strategic priorities and goals. It also represents a further validation for Bioasis’ technology and endorses our current strategic priorities and goals.

9. Bioasis also has a strategic partnership with Prothena, where the Company is eligible to receive up to $33 million in milestone payments and royalties on product sales, can you expand on this partnership for our readers?

Our agreement with Prothena, for which Bioasis received US$1 million in upfront payments, granted Prothena a worldwide, exclusive license to use the xB3 platform in connection with one undisclosed neurodegenerative disease target as well as an option for three additional neuroscience targets. Under the terms of the agreement, Bioasis may receive up to an additional US$33 million in options exercise, regulatory and commercial milestone payments. Prothena will also pay additional royalties on net sales from the licensed products. In addition to the agreement with Prothena, Bioasis has a research agreement with an undisclosed global Pharmaceutical company.

10. Looking out to the next six months to a year, is there anything on the horizon with the Company that investors should keep an eye out for?

Several of our pipeline programs are expected to reach important milestones, with new data becoming available in coming months. This new data is anticipated to drive partnership interest, bringing those programs more into the spotlight for investors. These programs include xB3-004 our version of an IL-1 receptor antagonist in development for the treatment of CNS inflammatory pain, certain childhood epilepsy conditions and other new indications, xB3-007 in development for the treatment of Gaucher disease and other GBA1 gene associated conditions and a new program, as yet undisclosed, that is targeting a form on dementia. Data on each of the programs is anticipated by the end of 2020 and in Q1 of 2021. As additional funding is secured, we will seek to bring forward milestones for xB3-001, in particular, the IND filing which is currently anticipated towards the end of calendar year 2021.

Bioasis is focused on concluding additional strategic partnerships for its xB3 drug delivery platform. We are making significant progress in our discussions with potential partners, with additional licensing agreements anticipated.



FULL DISCLOSURE: This is a paid article produced by Stockhouse Publishing.