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042 | Bellus Health back in the game, BC Cancer Agency makes two breakthrough discoveries, and RepliCel Life Sciences gets a little help from its friends.
Ahead on Biotechnology Focus Radio : Bellus Health back in the game with a new therapeutic asset, BC Cancer Agency scientists make two breakthrough discoveries, and RepliCel Life Sciences gets by with a little help from its friends.
We have this and more in store for you on this week’s show.
Welcome to another episode of Biotechnology Focus Radio. I’m your host Shawn Lawrence, here to give you a rundown of this week’s top stories on the Canadian biotech scene.
Our first story this week takes us to CALGARY, AB where a team of Canadian physicians and researchers are believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten lifespan. Specifically, people with the disease have a gene called GLA that doesn’t function as it should; as a result their bodies are unable to make the correct version of a particular enzyme that breaks down a fat called Gb3. A buildup of Gb3 can lead to problems in the kidneys, heart and brain. In their experimental trial, the researchers led by Dr. Aneal Khan, a Alberta Health Services medical geneticist and member of the Alberta Children’s Hospital Research Institute, Cumming School of Medicine, University of Calgary collected a quantity of a Fabry patient’s own blood stem cells then used a specially engineered virus to augment those cells with copies of the fully functional gene that is responsible for the enzyme. The altered stem cells were then transplanted back into the patient on Jan. 11, 2017. While Dr. Khan says it’s too soon to say whether this therapy will ultimately be a long-term treatment for the disease, based on the team’s success in animal trials, he is hopeful it will be a beneficial to patients Dr. Jeffrey Medin, a researcher with the Medical College of Wisconsin and the project’s principal investigator, adds that the trial is a major step forward in treating inherited genetic diseases in adults. The treatment, which has been approved by Health Canada for experimental purposes, is also believed to be the first trial in Canada to use a lentivirus in gene therapy. In this case, the specially modified virus was stripped of its disease-causing capability and augmented with a working copy of the gene that’s responsible for the missing enzyme. The project is being funded by the Canadian Institutes of Health Research and the Kidney Foundation of Canada. The Queen Elizabeth II Health Sciences Centre in Halifax and the University Health Network in Toronto are also recruiting people with Fabry disease for the trial.
In R&D news, a team of BC Cancer Agency scientists has made two exciting drug discoveries that could potentially allow for new approaches to target various cancers more specifically, through the exploitation of mutations found only in cancer cells and not normal cells. The first discovery, which has already led to a clinical trial, exploits the inability of BRCA1/2 deficient cancers to repair their own DNA. The second discovery is of a drug-like molecule that can alter the way cells translate genetic information into proteins. Both discoveries were made by Dr. Sam Aparicio, head of the Department of Breast and Molecular Oncology, and his research team at the BC Cancer Agency.The first success in this area is a discovery published in Nature Communications, where Dr. Aparicio’s team has discovered that the drug, CX-5461, originally developed for cancers of the blood and lymph system, can be repurposed as a drug treatment for breast cancer. Still early in its clinical development life cycle, CX-5461 has been shown through Dr. Aparicio’s latest work, to bind to the DNA of certain regions of the genome causing it to fold up and interrupt the DNA copying process. Thus, the compound is selectively active in tumours from patients with mutations in the BRCA1/2 gene, known to cause a strong familial predisposition to breast cancer, and account for approximately 15 per cent of the population with the disease. The study is currently in Phase 1 of a multi-centre clinical trial coordinated by Canadian Cancer Trials Group, which began in June of 2016. Phase two will accept even more patients to determine whether the activity found through preclinical studies is reflected in responses in patients. Both Dr. Aparicio and Dr. Karen Gelmon, senior scientist, medical oncologist and the clinical trial lead for the study add that if the trial is successful, they then hope to expand testing to other types of cancer in the near future. In addition to the CX-5461 findings, a second paper published in Nature Communications communicates the discovery of a different prototype drug, a compound called ‘T3’, engineered to alter the way that cells translate DNA, through splicing of RNA, into proteins. According to the researchers, this small, yet highly-potent drug-like molecule, currently in lab-testing, is being used to understand how different breast cancer cells might be susceptible to having RNA splicing interrupted. The drug molecule interferes with the molecular machinery that stiches gene sequences together to make fully functional proteins. Mutations in RNA splicing genes and defects in splicing have been found in diverse cancers, including breast cancer. The prototype drug molecules are allowing Dr. Aparicio and his team to seek out situations where cancer cells are uniquely susceptible to interference with RNA splicing. As alluded to, both drug development studies are currently centered in breast cancer treatment, but hope to expand to other types, including prostate and ovarian. Dr. Aparicio and his team’s work was supported with strategic funding from the BC Cancer Foundation.
In Business new, two European firms are teaming up with Vancouver based regenerative medicine company, RepliCel Life Sciences Inc.to assist RepliCel in getting its commercial-grade RCI-02 dermal injector prototypes manufactured and tested. One of the partnering firms, AMI, is an Austrian manufacturer of medical technology based near the shores of Lake Constance, within easy reach of Germany and Switzerland. AMI develops, manufactures and distributes their medical products throughout the world. All of them are made according to the highest quality standards and enable doctors to take even better care of their patients. The second partner, Art of Technology (AoT), is based in Zurich Switzerland and is an independent contract developer specializing in the design, development and miniaturization of complex customer specific electronic devices and embedded systems for use in industrial, medical and space applications. The RCI-02 injector itself was designed with input from dermatologists, industrial designers, and electronic and medical device engineers to improve the delivery of a variety of injectables in a controlled, precise manner, removing the risks and uncertainties of injection outcomes currently resulting from manually operated, single-needle syringes. According to RepliCel president and CEO, Lee Buckler, it is the world’s first motorized injection device with programmable depth and volume, a built-in Peltier element for pre-injection anaesthetising, and interchangeable needle head configurations. It is designed to deliver a variety of injectable substances including cells, dermal fillers, drugs or biologics intradermally (dermis), subcutaneously (fat) or intramuscularly (muscle) via an array of needle configurations ranging from a single needle to a 16 needle configuration (4×4) on one head. Buckler adds that the execution of these agreements covers what RepliCel believes to be the final stages needed to prepare RCI-02 for a market authorization application in the form of a CE mark in Europe. The company hopes to have the device ready for a CE mark application and in the hands of a licensing and commercial partner next year.
Our next story takes us to the Maritime provinces where the Terry Fox Research Institute is investing $5-million in support of New Brunswick researchers and their colleagues at other cancer centres in Canada to study how new precision medicine tools could improve, and potentially save the lives of patients diagnosed with the incurable cancer of the blood and bone marrow, known as multiple myeloma. The initiative is known as the Multiple Myeloma Molecular Monitoring (M4) Study, and Dr. Tony Reiman, a medical oncologist and professor at the University of New Brunswick, will lead the team, which comprises researchers and clinicians at multiple sites including Vancouver, Calgary, Toronto and Montreal. Dr. Reiman says that he hopes the five-year study will result in game-changing new approaches to identifying, treating and monitoring the disease in patients, including those who are at high risk of relapse. His team in Saint John will organize all the participating centres as well as conduct its own research and receive and bank specimens (blood and marrow) from the 250 myeloma patients that will participate in the project. Additionally, M4 study team members will use tests based on advanced techniques like immunoglobulin gene sequencing, multiparameter flow cytometry, PET scans, circulating tumour DNA analysis, and novel drug resistance assays to evaluate the patient specimens and other biosamples. Principal investigators at the partner sites are: Drs. Donna Reece and Suzanne Trudel, Princess Margaret Cancer Centre; Dr. Nizar Bahlis, University of Calgary; and Dr. François Bénard, BC Cancer Agency. Patients will be recruited by the study investigators at their own sites. Principal investigators Drs. Reece and Trudel (PM) explain their role in M4 study in the following audio.
BrainStorm Cell Therapeutics Inc., a HACKENSACK, N.J.- based company developing adult stem cell technologies for neurodegenerative diseases, has signed an agreement with CCRM in the hopes of furthering its market authorization request for NurOwn®. For our new listeners, CCRM is a Toronto-based company focused on developing and commercializing regenerative medicine technologies, specifically cell and gene therapies. Through the agreement, CCRM will help Brainstorm explore opportunities to access Health Canada’s early access pathway for treatment of patients with Amyotrophic Lateral Sclerosis (ALS). If NurOwn® qualifies for Health Canada’s “Notice of Compliance with Conditions” pathway, it could be authorized in Canada for distribution in early 2018. Through the agreement, the company will work Patrick Bedford, manager of clinical translation and regulatory affairs at CCRM. Stacey Johnson (@msstaceyerin) , director, communications and marketing at CCRM and editor of Signals Blog discussed this deal recently via her regular Right Turn column Be sure to check it out at http://www.signalsblog.ca/right-turn-new-stem-cell-product-for-als-seeking-approval-in-canada/.
For our final story, nearly a year since it’s failed KIACTA™ Phase 3 trial and subsequently being forced into pulling the plug on its KIACTA program, BELLUS Health is back from the brink as the Montreal-based company announced a new partnership and licensing deal with The NEOMED Institute to take over the development and commercialization activities for a potential new treatment for chronic cough. According to Bellus stakeholders,, this is a transformative transaction as this exclusive worldwide license agreement adds to the company’s pipeline a potentially best-in-class drug candidate, BLU-5937, an asset which was formerly known as NEO5937. Its development through the P2X3 antagonist program was initiated by AstraZeneca scientists in Montreal, and assigned to NEOMED in October 2012 when the NEOMED Institute was first launched. It was selected as a drug candidate to advance towards the clinic based on development efforts and extensive pre-clinical work in chronic cough done at NEOMED. According to Roberto Bellini, president and CEO of the company, the drug now going by its new name BLU-5937 will be a core focus of BELLUS’s drug development efforts. About the condition, Chronic cough is a cough that lasts eight weeks or longer and significantly impacts quality of life, with significant social (exclusion, embarrassment, difficulty speaking), physical (sleep deprivation, rib fracture, vomiting) and psychosocial (anxiety, depression) repercussions. It is estimated that in the U.S. alone, more than 2.7 million patients suffer from chronic cough that is not controlled by currently available medications. How BLU-5937 works to treat it, is it acts on a clinically validated target in the chronic cough pathway, the P2X3 receptor. Both the company and NEOMED believe BLU-5937 has the potential to become a best-in-class treatment option because of its superior potency and selectivity for the P2X3 receptor. These properties suggest BLU-5937 will be effective and less likely to cause a problematic side effect seen with less-selective drugs: taste disturbances that are significant enough to affect drug compliance. Under the terms of the agreement, BELLUS Health will pay NEOMED an upfront fee of $3.2 million, consisting of $1.7 million in cash with $1.5 million worth of BELLUS Health common shares (Approximately 5,802,177 shares). NEOMED will also be entitled to receive a royalty on net sales-based revenues. Additionally, in lieu of milestone payments, a certain portion of all other revenues received by BELLUS Health from BLU-5937 will be shared with NEOMED according to a pre-established schedule whereby the shared revenue portion decreases as the program progresses in development.
Well that wraps up another episode of the Biotechnology Focus Podcast. We hope you enjoyed it. Be sure to let us know what you think, and we’re also always looking for story ideas and suggestions for future shows, and of course we’d love to hear from you as well, simply reach out to us via twitter @biotechfocus, or by email at the following email address [email protected]. And remember, you can also listen to past episodes online via our podcast portal at www.biotechnologyfocus.ca .
For all of us here at Biotechnology Focus, thanks for listening
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By Biotechnology FocusAhead on Biotechnology Focus Radio : Bellus Health back in the game with a new therapeutic asset, BC Cancer Agency scientists make two breakthrough discoveries, and RepliCel Life Sciences gets by with a little help from its friends.
We have this and more in store for you on this week’s show.
Welcome to another episode of Biotechnology Focus Radio. I’m your host Shawn Lawrence, here to give you a rundown of this week’s top stories on the Canadian biotech scene.
Our first story this week takes us to CALGARY, AB where a team of Canadian physicians and researchers are believed to be the first in the world to have used gene therapy to treat a patient with Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten lifespan. Specifically, people with the disease have a gene called GLA that doesn’t function as it should; as a result their bodies are unable to make the correct version of a particular enzyme that breaks down a fat called Gb3. A buildup of Gb3 can lead to problems in the kidneys, heart and brain. In their experimental trial, the researchers led by Dr. Aneal Khan, a Alberta Health Services medical geneticist and member of the Alberta Children’s Hospital Research Institute, Cumming School of Medicine, University of Calgary collected a quantity of a Fabry patient’s own blood stem cells then used a specially engineered virus to augment those cells with copies of the fully functional gene that is responsible for the enzyme. The altered stem cells were then transplanted back into the patient on Jan. 11, 2017. While Dr. Khan says it’s too soon to say whether this therapy will ultimately be a long-term treatment for the disease, based on the team’s success in animal trials, he is hopeful it will be a beneficial to patients Dr. Jeffrey Medin, a researcher with the Medical College of Wisconsin and the project’s principal investigator, adds that the trial is a major step forward in treating inherited genetic diseases in adults. The treatment, which has been approved by Health Canada for experimental purposes, is also believed to be the first trial in Canada to use a lentivirus in gene therapy. In this case, the specially modified virus was stripped of its disease-causing capability and augmented with a working copy of the gene that’s responsible for the missing enzyme. The project is being funded by the Canadian Institutes of Health Research and the Kidney Foundation of Canada. The Queen Elizabeth II Health Sciences Centre in Halifax and the University Health Network in Toronto are also recruiting people with Fabry disease for the trial.
In R&D news, a team of BC Cancer Agency scientists has made two exciting drug discoveries that could potentially allow for new approaches to target various cancers more specifically, through the exploitation of mutations found only in cancer cells and not normal cells. The first discovery, which has already led to a clinical trial, exploits the inability of BRCA1/2 deficient cancers to repair their own DNA. The second discovery is of a drug-like molecule that can alter the way cells translate genetic information into proteins. Both discoveries were made by Dr. Sam Aparicio, head of the Department of Breast and Molecular Oncology, and his research team at the BC Cancer Agency.The first success in this area is a discovery published in Nature Communications, where Dr. Aparicio’s team has discovered that the drug, CX-5461, originally developed for cancers of the blood and lymph system, can be repurposed as a drug treatment for breast cancer. Still early in its clinical development life cycle, CX-5461 has been shown through Dr. Aparicio’s latest work, to bind to the DNA of certain regions of the genome causing it to fold up and interrupt the DNA copying process. Thus, the compound is selectively active in tumours from patients with mutations in the BRCA1/2 gene, known to cause a strong familial predisposition to breast cancer, and account for approximately 15 per cent of the population with the disease. The study is currently in Phase 1 of a multi-centre clinical trial coordinated by Canadian Cancer Trials Group, which began in June of 2016. Phase two will accept even more patients to determine whether the activity found through preclinical studies is reflected in responses in patients. Both Dr. Aparicio and Dr. Karen Gelmon, senior scientist, medical oncologist and the clinical trial lead for the study add that if the trial is successful, they then hope to expand testing to other types of cancer in the near future. In addition to the CX-5461 findings, a second paper published in Nature Communications communicates the discovery of a different prototype drug, a compound called ‘T3’, engineered to alter the way that cells translate DNA, through splicing of RNA, into proteins. According to the researchers, this small, yet highly-potent drug-like molecule, currently in lab-testing, is being used to understand how different breast cancer cells might be susceptible to having RNA splicing interrupted. The drug molecule interferes with the molecular machinery that stiches gene sequences together to make fully functional proteins. Mutations in RNA splicing genes and defects in splicing have been found in diverse cancers, including breast cancer. The prototype drug molecules are allowing Dr. Aparicio and his team to seek out situations where cancer cells are uniquely susceptible to interference with RNA splicing. As alluded to, both drug development studies are currently centered in breast cancer treatment, but hope to expand to other types, including prostate and ovarian. Dr. Aparicio and his team’s work was supported with strategic funding from the BC Cancer Foundation.
In Business new, two European firms are teaming up with Vancouver based regenerative medicine company, RepliCel Life Sciences Inc.to assist RepliCel in getting its commercial-grade RCI-02 dermal injector prototypes manufactured and tested. One of the partnering firms, AMI, is an Austrian manufacturer of medical technology based near the shores of Lake Constance, within easy reach of Germany and Switzerland. AMI develops, manufactures and distributes their medical products throughout the world. All of them are made according to the highest quality standards and enable doctors to take even better care of their patients. The second partner, Art of Technology (AoT), is based in Zurich Switzerland and is an independent contract developer specializing in the design, development and miniaturization of complex customer specific electronic devices and embedded systems for use in industrial, medical and space applications. The RCI-02 injector itself was designed with input from dermatologists, industrial designers, and electronic and medical device engineers to improve the delivery of a variety of injectables in a controlled, precise manner, removing the risks and uncertainties of injection outcomes currently resulting from manually operated, single-needle syringes. According to RepliCel president and CEO, Lee Buckler, it is the world’s first motorized injection device with programmable depth and volume, a built-in Peltier element for pre-injection anaesthetising, and interchangeable needle head configurations. It is designed to deliver a variety of injectable substances including cells, dermal fillers, drugs or biologics intradermally (dermis), subcutaneously (fat) or intramuscularly (muscle) via an array of needle configurations ranging from a single needle to a 16 needle configuration (4×4) on one head. Buckler adds that the execution of these agreements covers what RepliCel believes to be the final stages needed to prepare RCI-02 for a market authorization application in the form of a CE mark in Europe. The company hopes to have the device ready for a CE mark application and in the hands of a licensing and commercial partner next year.
Our next story takes us to the Maritime provinces where the Terry Fox Research Institute is investing $5-million in support of New Brunswick researchers and their colleagues at other cancer centres in Canada to study how new precision medicine tools could improve, and potentially save the lives of patients diagnosed with the incurable cancer of the blood and bone marrow, known as multiple myeloma. The initiative is known as the Multiple Myeloma Molecular Monitoring (M4) Study, and Dr. Tony Reiman, a medical oncologist and professor at the University of New Brunswick, will lead the team, which comprises researchers and clinicians at multiple sites including Vancouver, Calgary, Toronto and Montreal. Dr. Reiman says that he hopes the five-year study will result in game-changing new approaches to identifying, treating and monitoring the disease in patients, including those who are at high risk of relapse. His team in Saint John will organize all the participating centres as well as conduct its own research and receive and bank specimens (blood and marrow) from the 250 myeloma patients that will participate in the project. Additionally, M4 study team members will use tests based on advanced techniques like immunoglobulin gene sequencing, multiparameter flow cytometry, PET scans, circulating tumour DNA analysis, and novel drug resistance assays to evaluate the patient specimens and other biosamples. Principal investigators at the partner sites are: Drs. Donna Reece and Suzanne Trudel, Princess Margaret Cancer Centre; Dr. Nizar Bahlis, University of Calgary; and Dr. François Bénard, BC Cancer Agency. Patients will be recruited by the study investigators at their own sites. Principal investigators Drs. Reece and Trudel (PM) explain their role in M4 study in the following audio.
BrainStorm Cell Therapeutics Inc., a HACKENSACK, N.J.- based company developing adult stem cell technologies for neurodegenerative diseases, has signed an agreement with CCRM in the hopes of furthering its market authorization request for NurOwn®. For our new listeners, CCRM is a Toronto-based company focused on developing and commercializing regenerative medicine technologies, specifically cell and gene therapies. Through the agreement, CCRM will help Brainstorm explore opportunities to access Health Canada’s early access pathway for treatment of patients with Amyotrophic Lateral Sclerosis (ALS). If NurOwn® qualifies for Health Canada’s “Notice of Compliance with Conditions” pathway, it could be authorized in Canada for distribution in early 2018. Through the agreement, the company will work Patrick Bedford, manager of clinical translation and regulatory affairs at CCRM. Stacey Johnson (@msstaceyerin) , director, communications and marketing at CCRM and editor of Signals Blog discussed this deal recently via her regular Right Turn column Be sure to check it out at http://www.signalsblog.ca/right-turn-new-stem-cell-product-for-als-seeking-approval-in-canada/.
For our final story, nearly a year since it’s failed KIACTA™ Phase 3 trial and subsequently being forced into pulling the plug on its KIACTA program, BELLUS Health is back from the brink as the Montreal-based company announced a new partnership and licensing deal with The NEOMED Institute to take over the development and commercialization activities for a potential new treatment for chronic cough. According to Bellus stakeholders,, this is a transformative transaction as this exclusive worldwide license agreement adds to the company’s pipeline a potentially best-in-class drug candidate, BLU-5937, an asset which was formerly known as NEO5937. Its development through the P2X3 antagonist program was initiated by AstraZeneca scientists in Montreal, and assigned to NEOMED in October 2012 when the NEOMED Institute was first launched. It was selected as a drug candidate to advance towards the clinic based on development efforts and extensive pre-clinical work in chronic cough done at NEOMED. According to Roberto Bellini, president and CEO of the company, the drug now going by its new name BLU-5937 will be a core focus of BELLUS’s drug development efforts. About the condition, Chronic cough is a cough that lasts eight weeks or longer and significantly impacts quality of life, with significant social (exclusion, embarrassment, difficulty speaking), physical (sleep deprivation, rib fracture, vomiting) and psychosocial (anxiety, depression) repercussions. It is estimated that in the U.S. alone, more than 2.7 million patients suffer from chronic cough that is not controlled by currently available medications. How BLU-5937 works to treat it, is it acts on a clinically validated target in the chronic cough pathway, the P2X3 receptor. Both the company and NEOMED believe BLU-5937 has the potential to become a best-in-class treatment option because of its superior potency and selectivity for the P2X3 receptor. These properties suggest BLU-5937 will be effective and less likely to cause a problematic side effect seen with less-selective drugs: taste disturbances that are significant enough to affect drug compliance. Under the terms of the agreement, BELLUS Health will pay NEOMED an upfront fee of $3.2 million, consisting of $1.7 million in cash with $1.5 million worth of BELLUS Health common shares (Approximately 5,802,177 shares). NEOMED will also be entitled to receive a royalty on net sales-based revenues. Additionally, in lieu of milestone payments, a certain portion of all other revenues received by BELLUS Health from BLU-5937 will be shared with NEOMED according to a pre-established schedule whereby the shared revenue portion decreases as the program progresses in development.
Well that wraps up another episode of the Biotechnology Focus Podcast. We hope you enjoyed it. Be sure to let us know what you think, and we’re also always looking for story ideas and suggestions for future shows, and of course we’d love to hear from you as well, simply reach out to us via twitter @biotechfocus, or by email at the following email address [email protected]. And remember, you can also listen to past episodes online via our podcast portal at www.biotechnologyfocus.ca .
For all of us here at Biotechnology Focus, thanks for listening