Roberto Bellini, President and CEO of BELLUS Health, presented an overview of the company and its lead drug candidate, BLU-5937, for the treatment of chronic cough. BLU-5937 is a highly selective P2X3 antagonist that has shown in animal studies to reduce cough frequency comparably to MK-7264 without impacting taste, unlike MK-7264 which causes taste alteration or loss in many patients. BELLUS Health plans to initiate a Phase 1 clinical trial of BLU-5937 in Q3 2018 to evaluate its safety, tolerability, and effects on cough and taste. The company believes BLU-5937 has potential to be best-in-class for
Bellus health agm presentation en finalBellusHealth
- The document summarizes an annual general meeting for shareholders of BELLUS Health Inc. held on May 15, 2018.
- BELLUS is developing BLU-5937, a potentially best-in-class drug for chronic cough that selectively targets P2X3 receptors. Chronic cough affects millions of people and current treatments have significant side effects.
- BLU-5937 has shown potential to be as effective as the leading P2X3 drug in development (MK-7264) at reducing cough frequency without impacting taste, which MK-7264 does. BLU-5937 is expected to have twice daily oral dosing.
Bellus health corporate presentation baird conference september 6BellusHealth
- Roberto Bellini, President and CEO of BELLUS Health, presented at the Baird Global Healthcare Conference on their lead drug candidate BLU-5937 for chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown in animal studies to reduce cough frequency equivalently to MK-7264 while avoiding its side effect of taste alteration.
- BELLUS is currently conducting a Phase 1 trial with data expected in Q4 2018 to select doses for future trials, with the goal of BLU-5937 becoming the best-in-class P2X3 antagonist for chronic cough.
Bellus health corporate presentation (biotech showcase) jan 9 2018 v final PDFBellusHealth
- BELLUS Health Inc. is presenting on its drug candidate BLU-5937 for the treatment of chronic cough. BLU-5937 is a highly selective P2X3 antagonist that has the potential to be best-in-class by inhibiting cough with little to no taste disturbance.
- Chronic cough affects approximately 10% of adults in the US, representing a large unmet medical need. Current treatment options are limited and inadequate.
- BLU-5937 has shown promise in preclinical studies at inhibiting cough similarly to the leading P2X3 antagonist MK-7264 while not altering taste like MK-7264 did. This differential effect is due to BLU-5937's greater
Bellus health corporate presentation (biotech showcase) jan 9 2018 v finalBellusHealth
Roberto Bellini, President and CEO of BELLUS Health, presented an overview of the company and its lead drug candidate BLU-5937 for the treatment of chronic cough. BLU-5937 works by selectively targeting the P2X3 receptor and has the potential to be best-in-class by inhibiting cough with little to no taste side effects. Key upcoming milestones include filing the Phase 1 regulatory dossier in Q2 2018 and initiating the Phase 1 study in Q3 2018. BELLUS Health has cash runway into 2020 to progress the development of BLU-5937 and its other pipeline assets.
Bellus health corporate presentation july 9 v finalBellusHealth
- Roberto Bellini, President and CEO of BELLUS Health Inc., presented an overview of the company and its lead drug candidate, BLU-5937, for the treatment of chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown in animal studies to reduce cough frequency equivalently to MK-7264 while avoiding its side effect of taste alteration.
- BELLUS is currently conducting a Phase 1 trial of BLU-5937 to assess safety, tolerability, and pharmacokinetics with data expected in Q4 2018. Pending these results, Phase 2 trials are planned to further evaluate BLU-5937's effects on cough and taste.
Bellus health presentation (g) october 10 v final BellusHealth
Bellini presented on BLU-5937, a potentially best-in-class P2X3 antagonist for chronic cough that could address a multi-billion dollar market. If successful, BLU-5937 may have improved efficacy over the first-in-class drug MK-7264 while avoiding its problematic taste side effects, due to its greater selectivity for the P2X3 receptor. Upcoming milestones include filing the regulatory dossier for a Phase 1 trial in Q2 2018 and initiating the trial in Q3 2018. Bellini believes targeting the validated P2X3 receptor has the potential to deliver the first safe and effective non-narcotic treatment for chronic cough sufferers.
Bellus health presentation september 14, 2017BellusHealth
Roberto Bellini, President and CEO of BELLUS Health, provided an overview of the company's chronic cough drug candidate BLU-5937 and development plans. BLU-5937 is a potentially best-in-class P2X3 antagonist that could treat the large unmet need in chronic cough with improved efficacy and no taste side effects. If successful, BLU-5937 has the potential to become a blockbuster drug, addressing a multi-billion dollar market. BELLUS Health expects to complete IND-enabling studies for BLU-5937 and advance it into Phase 1 clinical trials to assess safety, tolerability, and effects on taste.
Bellus health corporate presentation mar 16 v finalBellusHealth
- Roberto Bellini, President and CEO of BELLUS Health Inc., presented an overview of the company which is developing BLU-5937 as a potentially best-in-class treatment for chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown potential to significantly reduce chronic cough in preclinical studies with little to no effect on taste perception, unlike competitor drugs.
- Chronic cough represents a large unmet medical need as it affects 10% of adults and currently available treatments are inadequate with significant side effects. The P2X3 receptor is a validated target for chronic cough treatments.
Bellus health agm presentation en finalBellusHealth
- The document summarizes an annual general meeting for shareholders of BELLUS Health Inc. held on May 15, 2018.
- BELLUS is developing BLU-5937, a potentially best-in-class drug for chronic cough that selectively targets P2X3 receptors. Chronic cough affects millions of people and current treatments have significant side effects.
- BLU-5937 has shown potential to be as effective as the leading P2X3 drug in development (MK-7264) at reducing cough frequency without impacting taste, which MK-7264 does. BLU-5937 is expected to have twice daily oral dosing.
Bellus health corporate presentation baird conference september 6BellusHealth
- Roberto Bellini, President and CEO of BELLUS Health, presented at the Baird Global Healthcare Conference on their lead drug candidate BLU-5937 for chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown in animal studies to reduce cough frequency equivalently to MK-7264 while avoiding its side effect of taste alteration.
- BELLUS is currently conducting a Phase 1 trial with data expected in Q4 2018 to select doses for future trials, with the goal of BLU-5937 becoming the best-in-class P2X3 antagonist for chronic cough.
Bellus health corporate presentation (biotech showcase) jan 9 2018 v final PDFBellusHealth
- BELLUS Health Inc. is presenting on its drug candidate BLU-5937 for the treatment of chronic cough. BLU-5937 is a highly selective P2X3 antagonist that has the potential to be best-in-class by inhibiting cough with little to no taste disturbance.
- Chronic cough affects approximately 10% of adults in the US, representing a large unmet medical need. Current treatment options are limited and inadequate.
- BLU-5937 has shown promise in preclinical studies at inhibiting cough similarly to the leading P2X3 antagonist MK-7264 while not altering taste like MK-7264 did. This differential effect is due to BLU-5937's greater
Bellus health corporate presentation (biotech showcase) jan 9 2018 v finalBellusHealth
Roberto Bellini, President and CEO of BELLUS Health, presented an overview of the company and its lead drug candidate BLU-5937 for the treatment of chronic cough. BLU-5937 works by selectively targeting the P2X3 receptor and has the potential to be best-in-class by inhibiting cough with little to no taste side effects. Key upcoming milestones include filing the Phase 1 regulatory dossier in Q2 2018 and initiating the Phase 1 study in Q3 2018. BELLUS Health has cash runway into 2020 to progress the development of BLU-5937 and its other pipeline assets.
Bellus health corporate presentation july 9 v finalBellusHealth
- Roberto Bellini, President and CEO of BELLUS Health Inc., presented an overview of the company and its lead drug candidate, BLU-5937, for the treatment of chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown in animal studies to reduce cough frequency equivalently to MK-7264 while avoiding its side effect of taste alteration.
- BELLUS is currently conducting a Phase 1 trial of BLU-5937 to assess safety, tolerability, and pharmacokinetics with data expected in Q4 2018. Pending these results, Phase 2 trials are planned to further evaluate BLU-5937's effects on cough and taste.
Bellus health presentation (g) october 10 v final BellusHealth
Bellini presented on BLU-5937, a potentially best-in-class P2X3 antagonist for chronic cough that could address a multi-billion dollar market. If successful, BLU-5937 may have improved efficacy over the first-in-class drug MK-7264 while avoiding its problematic taste side effects, due to its greater selectivity for the P2X3 receptor. Upcoming milestones include filing the regulatory dossier for a Phase 1 trial in Q2 2018 and initiating the trial in Q3 2018. Bellini believes targeting the validated P2X3 receptor has the potential to deliver the first safe and effective non-narcotic treatment for chronic cough sufferers.
Bellus health presentation september 14, 2017BellusHealth
Roberto Bellini, President and CEO of BELLUS Health, provided an overview of the company's chronic cough drug candidate BLU-5937 and development plans. BLU-5937 is a potentially best-in-class P2X3 antagonist that could treat the large unmet need in chronic cough with improved efficacy and no taste side effects. If successful, BLU-5937 has the potential to become a blockbuster drug, addressing a multi-billion dollar market. BELLUS Health expects to complete IND-enabling studies for BLU-5937 and advance it into Phase 1 clinical trials to assess safety, tolerability, and effects on taste.
Bellus health corporate presentation mar 16 v finalBellusHealth
- Roberto Bellini, President and CEO of BELLUS Health Inc., presented an overview of the company which is developing BLU-5937 as a potentially best-in-class treatment for chronic cough.
- BLU-5937 is a highly selective P2X3 antagonist that has shown potential to significantly reduce chronic cough in preclinical studies with little to no effect on taste perception, unlike competitor drugs.
- Chronic cough represents a large unmet medical need as it affects 10% of adults and currently available treatments are inadequate with significant side effects. The P2X3 receptor is a validated target for chronic cough treatments.
BioVie Inc. (OTCQB: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Visit BIVIinfo.com to learn more.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
Bellus health presentation (g) april 21 2017 v finalBellusHealth
Bellini provides a corporate presentation on BELLUS Health Inc. highlighting their lead drug candidate BLU-5937, which is a potentially best-in-class P2X3 antagonist for chronic cough. BLU-5937 has shown dose-dependent reduction of cough in preclinical models. Its high selectivity for P2X3 over P2X2/3 may allow it to avoid taste side effects seen with less selective competitors. The chronic cough market is large, with about 10% of adults affected globally. BLU-5937 has the potential to be superior to the only other P2X3 drug in development due to its differentiated profile. Bellini outlines the development plan for BLU-5937 and
NE3107 is a small molecule in Phase 3 clinical trials for Alzheimer's disease and Parkinson's disease. It works by inhibiting neuroinflammation and insulin resistance, two key drivers of cognitive decline. A Phase 3 trial for Alzheimer's is underway testing NE3107's ability to slow cognitive decline compared to placebo. Preclinical studies show NE3107 reduces inflammation, enhances insulin sensitivity, and has neuroprotective effects, supporting its potential in neurodegenerative diseases. A Phase 2 trial will assess NE3107's activity and safety when combined with L-dopa for Parkinson's disease.
Reviva Pharmaceuticals Holdings Inc is a clinical development pharmaceutical company. It is developing a portfolio of internally discovered therapies that address unmet medical needs in the areas of central nervous system, cardiovascular, metabolic and inflammatory diseases.
Virios Therapeutics is a clinical-stage biotechnology company focused on advancing novel, dual mechanism antiviral therapies to treat conditions associated with virally triggered or maintained immune responses, such as Fibromyalgia (“FM”). Immune responses related to the activation of tissue resident Herpes Simplex Virus-1 (“HSV-1”) have been postulated as a potential root cause triggering and/or sustaining chronic illnesses such as FM, irritable bowel disease (“IBS”), and chronic fatigue syndrome, all of which can be characterized by waxing and waning symptom flare-ups with no obvious etiology. Virios’ lead development candidate (“IMC-1”) is a novel, proprietary, fixed dose combination of famciclovir and celecoxib designed to synergistically suppress HSV-1 replication, with the end goal of reducing virally promoted disease symptoms.
Phase 1 data presentation final with appendixBellusHealth
BLU-5937 is a P2X3 antagonist being developed for chronic cough. Phase 1 data showed it had an excellent pharmacokinetic profile and was safe and well tolerated, with minimal taste effects seen at projected therapeutic doses of 50-100mg twice daily. This taste effect profile was significantly better than the first-in-class gefapixant. Based on the positive Phase 1 results, a Phase 2 study in patients with unexplained chronic cough is expected to start in mid-2019.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met all endpoints for its phase 2 clinical trial and the Company is now preparing for a phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody approach is a proven Mechanism of Action and has shown efficacy as a combination therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. It also recently began enrolling for a phase 2 clinical trial for the treatment of Cystic Fibrosis patients with acute pneumonia, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with its novel inhalation antibody technology for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
- Palisade Bio was formed through the merger of Leading BioSciences and Seneca to focus on developing drugs to restore intestinal barrier health.
- Their lead product, LB1148, is an oral protease inhibitor that has shown in clinical trials to accelerate the return of normal bowel function after surgery by 30%.
- They plan to initiate a Phase 3 trial in 2021 to evaluate LB1148 in improving GI recovery in neonates undergoing cardiac surgery, which could potentially qualify for priority review.
- If successful, LB1148 has the potential to reduce postoperative complications and lower healthcare costs through shorter hospital stays.
CLBS Corporate Slide Presentation March 2018Steve Sizer
This corporate presentation discusses Caladrius Biosciences' business model evolution and future plans. It summarizes that Caladrius has transitioned to focus solely on clinical-stage therapeutics development using two technology platforms: autologous T-regulatory cells for immune modulation and CD34 cells for ischemic repair. Key programs outlined include an ongoing Phase 2 trial of T-regulatory cells for recent-onset type 1 diabetes and planned Phase 2 trials of CD34 cells for critical limb ischemia and coronary microvascular dysfunction. The presentation positions Caladrius for continued growth with a well-funded pipeline and strategic partnerships.
Commercial considerations in early drug developmentSunil Ramkali
It is important in the drug development process that marketers and researchers collaborate early to ensure that products being developed are truly innovative and deliver brand value to the different end users in a way that the product and the subsequent brand messaging is relevant, compelling and differentiating compared to the competition. T
In the market place that is heavily cost constraint, innovation is no longer about a unique mode of action or a new formulation, but more about the incremental brand value offered by new pharmaceutical products over existing treatments (standard of care) and how much healthcare systems are prepared to pay for these incremental benefits. My lecture at the Department of Innovation, Lund University, Sweden explored the importance of R&D functions getter closer to external stakeholders to really understand their needs, how they define brand value and the importance of considering this early in the drug development process.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2021, the company plans to bring two candidates to
clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of three
drug candidates for orphan diseases to clinical trials every 12-18 months. The company is
now preparing to launch a dietary supplement for women undergoing menopause and
experiencing related symptoms, including osteoporosis. Targeting a global woman’s health
supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to
generate near-term revenue with significant long-term growth potential.
NPS Pharmaceuticals is a global biopharmaceutical company focused on developing therapies for rare diseases. It has recently launched Gattex/Revestive (teduglutide) for the treatment of adult Short Bowel Syndrome in the US and EU. It is also developing Natpara (recombinant human parathyroid hormone 1-84) as a potential first approved replacement therapy for hypoparathyroidism, with a BLA submission planned for the US in the second half of 2013. The company has a proven track record of successfully developing and launching orphan drugs and is focused on executing its Gattex launch and advancing its pipeline to become a leading global orphan specialty drug company.
Presentation Part 2 – Leading with innovationSanofi
Sanofi is building an innovative and diversified vaccine pipeline by expanding into new disease areas and technologies. They have added mRNA and LNP platforms and have 10 new development candidates by 2025, including 6 mRNA vaccines. Sanofi is focusing on first-in-class or best-in-class vaccines and leveraging immunology, antigen design, and the best technology for each target. They are broadening their pipeline to address additional chronic diseases and expanding populations.
Immuron Limited is a clinical-stage biopharmaceutical company targeting inflammatory and infectious diseases with oral immunotherapies. It has two lead clinical assets in Phase 2 development for fatty liver disease and C. difficile infection. It also has a registered and revenue-generating Travelan product targeting traveler's diarrhea. The company is listed on the NASDAQ and ASX exchanges and has an experienced management team supported by key opinion leaders. It is well positioned to address significant unmet needs in liver disease and infectious disease markets with blockbuster potential. Near-term milestones include interim data readouts from multiple Phase 2 NASH and pediatric NAFLD trials in 2017-2018.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Regulation, ethics and reimbursement of novel biological therapies in Austral...TGA Australia
The document provides an overview of novel biological therapies such as gene therapy, cell therapy, and tissue engineering in Australia. It discusses the regulatory pathways through the Therapeutic Goods Administration (TGA) for approval of these therapies. Clinical trials of novel biological therapies must be submitted through the CTX scheme for approval rather than just notification. Guidelines from the European Medicines Agency are a good resource for requirements for registration and approval of these therapies in Australia. Risks associated with gene therapy include potential for delayed adverse effects, off-target effects, insertional mutagenesis, and immune responses.
This document provides Galena's Q2 2016 financial results and corporate update. It discusses Galena's product pipeline including GALE-401 for essential thrombocythemia, NeuVax for breast cancer and gastric cancer, and GALE-301/302 for ovarian and breast cancer. For GALE-401, it outlines positive preliminary results from a Phase 2 trial and plans for a Phase 2/3 trial. For NeuVax, it describes ongoing Phase 2 trials in breast cancer. For GALE-301/302, it discusses a Phase 1/2a trial showing preliminary efficacy for GALE-301. The document also provides Galena's Q2 2016 financial results and cash position.
Bellus health corporate presentation march 17 2017 v final (002) [read only]BellusHealth
This corporate presentation summarizes Bellus Health Inc., a biopharmaceutical company developing innovative drug candidates. Their lead product is BLU-5937, a P2X3 receptor antagonist being developed for chronic cough. P2X3 antagonists have shown efficacy in clinical trials but with problematic taste side effects. BLU-5937 has the potential to be best-in-class due to its high potency, selectivity for P2X3, and preclinical efficacy in reducing cough. Near term milestones include IND-enabling studies for BLU-5937 and progressing partnered programs in sarcoidosis, fragile X syndrome, and Alzheimer's disease. The company has cash runway into Q4 2018
BLU-5937 is a P2X3 receptor antagonist being developed by BELLUS Health for the treatment of chronic cough. P2X3 receptors are involved in cough signaling and are a validated target for chronic cough therapies. In preclinical studies, BLU-5937 showed potent and selective inhibition of the P2X3 receptor with no effect on taste, unlike leading P2X3 antagonist MK-7264 which caused taste disturbances. BLU-5937 inhibited cough in guinea pig models comparably to MK-7264 but with a wider safety margin. BELLUS Health is conducting a Phase 1 trial in 2018 to assess the safety, tolerability and pharmacokinetics of BLU-59
BioVie Inc. (OTCQB: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Visit BIVIinfo.com to learn more.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing what it believes will be transformative therapies to overcome unmet medical needs in neurodegeneration and liver disease. The Company is developing NE3107 for Alzheimer’s (AD) and Parkinson’s (PD) and BIV201 for refractory ascites and HRS-AKI.
Bellus health presentation (g) april 21 2017 v finalBellusHealth
Bellini provides a corporate presentation on BELLUS Health Inc. highlighting their lead drug candidate BLU-5937, which is a potentially best-in-class P2X3 antagonist for chronic cough. BLU-5937 has shown dose-dependent reduction of cough in preclinical models. Its high selectivity for P2X3 over P2X2/3 may allow it to avoid taste side effects seen with less selective competitors. The chronic cough market is large, with about 10% of adults affected globally. BLU-5937 has the potential to be superior to the only other P2X3 drug in development due to its differentiated profile. Bellini outlines the development plan for BLU-5937 and
NE3107 is a small molecule in Phase 3 clinical trials for Alzheimer's disease and Parkinson's disease. It works by inhibiting neuroinflammation and insulin resistance, two key drivers of cognitive decline. A Phase 3 trial for Alzheimer's is underway testing NE3107's ability to slow cognitive decline compared to placebo. Preclinical studies show NE3107 reduces inflammation, enhances insulin sensitivity, and has neuroprotective effects, supporting its potential in neurodegenerative diseases. A Phase 2 trial will assess NE3107's activity and safety when combined with L-dopa for Parkinson's disease.
Reviva Pharmaceuticals Holdings Inc is a clinical development pharmaceutical company. It is developing a portfolio of internally discovered therapies that address unmet medical needs in the areas of central nervous system, cardiovascular, metabolic and inflammatory diseases.
Virios Therapeutics is a clinical-stage biotechnology company focused on advancing novel, dual mechanism antiviral therapies to treat conditions associated with virally triggered or maintained immune responses, such as Fibromyalgia (“FM”). Immune responses related to the activation of tissue resident Herpes Simplex Virus-1 (“HSV-1”) have been postulated as a potential root cause triggering and/or sustaining chronic illnesses such as FM, irritable bowel disease (“IBS”), and chronic fatigue syndrome, all of which can be characterized by waxing and waning symptom flare-ups with no obvious etiology. Virios’ lead development candidate (“IMC-1”) is a novel, proprietary, fixed dose combination of famciclovir and celecoxib designed to synergistically suppress HSV-1 replication, with the end goal of reducing virally promoted disease symptoms.
Phase 1 data presentation final with appendixBellusHealth
BLU-5937 is a P2X3 antagonist being developed for chronic cough. Phase 1 data showed it had an excellent pharmacokinetic profile and was safe and well tolerated, with minimal taste effects seen at projected therapeutic doses of 50-100mg twice daily. This taste effect profile was significantly better than the first-in-class gefapixant. Based on the positive Phase 1 results, a Phase 2 study in patients with unexplained chronic cough is expected to start in mid-2019.
BioVie Inc. (NASDAQ: BIVI) is a clinical-stage company developing innovative drug therapies for liver disease. The Company’s drug candidate, BIV201 (continuous infusion terlipressin), has an Orphan Drug designation for the treatment of refractory ascites, FDA Fast Track status, and US patent pending. BIV201 also has an Orphan Drug designation for the treatment of hepatorenal syndrome (HRS). The active agent in BIV201, terlipressin, is approved for use in about 40 countries for the treatment of related complications of advanced liver cirrhosis but is not available in the US or Japan. The FDA has never approved terlipressin. BioVie is targeting this landmark achievement.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met all endpoints for its phase 2 clinical trial and the Company is now preparing for a phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody approach is a proven Mechanism of Action and has shown efficacy as a combination therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. It also recently began enrolling for a phase 2 clinical trial for the treatment of Cystic Fibrosis patients with acute pneumonia, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with its novel inhalation antibody technology for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
- Palisade Bio was formed through the merger of Leading BioSciences and Seneca to focus on developing drugs to restore intestinal barrier health.
- Their lead product, LB1148, is an oral protease inhibitor that has shown in clinical trials to accelerate the return of normal bowel function after surgery by 30%.
- They plan to initiate a Phase 3 trial in 2021 to evaluate LB1148 in improving GI recovery in neonates undergoing cardiac surgery, which could potentially qualify for priority review.
- If successful, LB1148 has the potential to reduce postoperative complications and lower healthcare costs through shorter hospital stays.
CLBS Corporate Slide Presentation March 2018Steve Sizer
This corporate presentation discusses Caladrius Biosciences' business model evolution and future plans. It summarizes that Caladrius has transitioned to focus solely on clinical-stage therapeutics development using two technology platforms: autologous T-regulatory cells for immune modulation and CD34 cells for ischemic repair. Key programs outlined include an ongoing Phase 2 trial of T-regulatory cells for recent-onset type 1 diabetes and planned Phase 2 trials of CD34 cells for critical limb ischemia and coronary microvascular dysfunction. The presentation positions Caladrius for continued growth with a well-funded pipeline and strategic partnerships.
Commercial considerations in early drug developmentSunil Ramkali
It is important in the drug development process that marketers and researchers collaborate early to ensure that products being developed are truly innovative and deliver brand value to the different end users in a way that the product and the subsequent brand messaging is relevant, compelling and differentiating compared to the competition. T
In the market place that is heavily cost constraint, innovation is no longer about a unique mode of action or a new formulation, but more about the incremental brand value offered by new pharmaceutical products over existing treatments (standard of care) and how much healthcare systems are prepared to pay for these incremental benefits. My lecture at the Department of Innovation, Lund University, Sweden explored the importance of R&D functions getter closer to external stakeholders to really understand their needs, how they define brand value and the importance of considering this early in the drug development process.
Aptorum Group Limited (NASDAQ: APM) is a pharmaceutical company dedicated to
developing and commercializing novel therapeutics to tackle unmet medical needs.
Aptorum's current drug pipeline includes indications in orphan diseases, infectious
diseases, and metabolic diseases. In 2021, the company plans to bring two candidates to
clinical trials. Aptorum’s Smart-ACT™ platform is designed to bring an average of three
drug candidates for orphan diseases to clinical trials every 12-18 months. The company is
now preparing to launch a dietary supplement for women undergoing menopause and
experiencing related symptoms, including osteoporosis. Targeting a global woman’s health
supplement market that is expected to reach $17 billion in 2025, Aptorum is expected to
generate near-term revenue with significant long-term growth potential.
NPS Pharmaceuticals is a global biopharmaceutical company focused on developing therapies for rare diseases. It has recently launched Gattex/Revestive (teduglutide) for the treatment of adult Short Bowel Syndrome in the US and EU. It is also developing Natpara (recombinant human parathyroid hormone 1-84) as a potential first approved replacement therapy for hypoparathyroidism, with a BLA submission planned for the US in the second half of 2013. The company has a proven track record of successfully developing and launching orphan drugs and is focused on executing its Gattex launch and advancing its pipeline to become a leading global orphan specialty drug company.
Presentation Part 2 – Leading with innovationSanofi
Sanofi is building an innovative and diversified vaccine pipeline by expanding into new disease areas and technologies. They have added mRNA and LNP platforms and have 10 new development candidates by 2025, including 6 mRNA vaccines. Sanofi is focusing on first-in-class or best-in-class vaccines and leveraging immunology, antigen design, and the best technology for each target. They are broadening their pipeline to address additional chronic diseases and expanding populations.
Immuron Limited is a clinical-stage biopharmaceutical company targeting inflammatory and infectious diseases with oral immunotherapies. It has two lead clinical assets in Phase 2 development for fatty liver disease and C. difficile infection. It also has a registered and revenue-generating Travelan product targeting traveler's diarrhea. The company is listed on the NASDAQ and ASX exchanges and has an experienced management team supported by key opinion leaders. It is well positioned to address significant unmet needs in liver disease and infectious disease markets with blockbuster potential. Near-term milestones include interim data readouts from multiple Phase 2 NASH and pediatric NAFLD trials in 2017-2018.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Regulation, ethics and reimbursement of novel biological therapies in Austral...TGA Australia
The document provides an overview of novel biological therapies such as gene therapy, cell therapy, and tissue engineering in Australia. It discusses the regulatory pathways through the Therapeutic Goods Administration (TGA) for approval of these therapies. Clinical trials of novel biological therapies must be submitted through the CTX scheme for approval rather than just notification. Guidelines from the European Medicines Agency are a good resource for requirements for registration and approval of these therapies in Australia. Risks associated with gene therapy include potential for delayed adverse effects, off-target effects, insertional mutagenesis, and immune responses.
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Bellus health corporate presentation march 17 2017 v final (002) [read only]BellusHealth
This corporate presentation summarizes Bellus Health Inc., a biopharmaceutical company developing innovative drug candidates. Their lead product is BLU-5937, a P2X3 receptor antagonist being developed for chronic cough. P2X3 antagonists have shown efficacy in clinical trials but with problematic taste side effects. BLU-5937 has the potential to be best-in-class due to its high potency, selectivity for P2X3, and preclinical efficacy in reducing cough. Near term milestones include IND-enabling studies for BLU-5937 and progressing partnered programs in sarcoidosis, fragile X syndrome, and Alzheimer's disease. The company has cash runway into Q4 2018
BLU-5937 is a P2X3 receptor antagonist being developed by BELLUS Health for the treatment of chronic cough. P2X3 receptors are involved in cough signaling and are a validated target for chronic cough therapies. In preclinical studies, BLU-5937 showed potent and selective inhibition of the P2X3 receptor with no effect on taste, unlike leading P2X3 antagonist MK-7264 which caused taste disturbances. BLU-5937 inhibited cough in guinea pig models comparably to MK-7264 but with a wider safety margin. BELLUS Health is conducting a Phase 1 trial in 2018 to assess the safety, tolerability and pharmacokinetics of BLU-59
The document summarizes Bellus Health's annual meeting presentation from May 15, 2013. It discusses Bellus' product pipeline, including KIACTA for AA amyloidosis, which has completed a positive Phase II/III trial. Bellus' partner Auven Therapeutics is funding the Phase III confirmatory study of KIACTA. Bellus also discusses its memory supplement VIVIMIND and its Alzheimer's drug BLU8499. The presentation outlines Bellus' business model, partnerships, financial position, and future milestones including completing enrollment in the KIACTA Phase III study and initiating BLU8499's Phase IIa trial.
The document summarizes an annual general meeting presentation for shareholders of BELLUS Health Inc. It discusses the company's focus on developing drugs for rare diseases starting with conditions affecting the kidneys. Key points include BELLUS having a late-stage pipeline with 4 rare disease projects, their lead drug candidate KIACTA completing a positive Phase 2/3 study for AA amyloidosis and now being in a confirmatory Phase 3 study fully funded by their partner. The presentation provides details on KIACTA's clinical results, the Phase 3 study and potential commercialization plans upon results. It also outlines their second rare disease candidate Shigamab in preclinical development for sHUS and upcoming milestones.
Bellus Health Corporate Presentation June 2016BellusHealth
- The document is a corporate presentation for Bellus Health Inc. discussing their rare disease drug pipeline and lead product candidate KIACTA for AA amyloidosis.
- KIACTA showed positive results in a Phase 2/3 trial, reducing risk of worsening kidney events. A Phase 3 confirmatory study has completed enrollment with results expected in Q2 2016.
- If successful, KIACTA has the potential for regulatory approval and commercialization through a partnership, as Bellus' partner Auven Therapeutics is funding the entire development program.
Bellus Health Corporate Presentation May 2016BellusHealth
- The document is a corporate presentation for Bellus Health Inc. discussing their rare disease drug pipeline and lead product candidate KIACTA for AA amyloidosis.
- KIACTA has shown positive results in a Phase 2/3 trial, significantly reducing kidney function decline. A Phase 3 confirmatory study has completed enrollment with results expected in Q2 2016.
- If successful, KIACTA has the potential for regulatory approval and commercialization through a partnership, providing funding to advance Bellus' pipeline of rare disease programs.
Bellus corporate presentation january 2014 v finalBellusHealth
BELLUS Health is developing therapies for rare diseases starting with conditions affecting the kidneys. Its lead product KIACTA is in Phase III trials for AA amyloidosis, a rare kidney disease with no approved treatments. It has executed a partnership for KIACTA's development and aims to complete enrollment in 2014. BELLUS' pipeline also includes Shigamab for STEC-HUS and a research program in AL amyloidosis. The company expects key milestones in 2014 to progress these programs and generate long-term value.
Bellus corporate presentation feb 2015 (r)BellusHealth
This corporate presentation by Roberto Bellini of BELLUS Health Inc. discusses the company's focus on developing drugs for rare diseases. The lead product candidate is KIACTA for AA amyloidosis, a rare kidney disease with no existing treatment. Key highlights include:
- KIACTA showed positive results in a Phase III trial, significantly reducing kidney function decline. A Phase III confirmatory study is ongoing.
- If approved, KIACTA could achieve premium pricing like other orphan drugs, with over $70M in funding from partner Auven Therapeutics.
- The company also has a preclinical antibody, Shigamab, targeting STEC-related HUS, a rare kidney disease
Bellus corporate presentation fall finalBellusHealth
- Bellini Health is focused on developing drugs for rare diseases, starting with conditions affecting the kidneys. Its lead product, KIACTA, is in Phase III trials for AA amyloidosis, a rare and deadly kidney disease.
- The company has partnered with Auven Therapeutics to fund the Phase III confirmatory study of KIACTA in exchange for future profits.
- Bellini Health is also developing Shigamabs for hemolytic uremic syndrome, a rare kidney disease primarily affecting children. It aims to progress this program towards a partnership within 18-24 months.
Senesco Technologies is developing a gene regulation technology to treat cancer. They are running a Phase 1b/2a clinical trial of their lead product, SNS01-T, to treat B-cell cancers like multiple myeloma and lymphoma. Preclinical studies show SNS01-T significantly inhibits tumor growth and improves survival in mouse models of these cancers. The presentation provides an overview of Senesco's technology, clinical trial status, financial information, and development plans to advance SNS01-T and expand to additional cancer indications.
This document provides an overview and summary of Bellus Health Inc., a biopharmaceutical company focused on developing treatments for rare kidney diseases. Their lead drug candidate, KIACTA, recently completed a Phase III confirmatory study in patients with AA amyloidosis, a rare and fatal kidney disease with no approved treatments. Topline results from the study are expected in Q2 2016 and, if positive, could support KIACTA's approval. The company has funding to progress KIACTA's development and pursue additional rare disease programs, with potential value-driving milestones in 2016.
The document provides an overview of Bellus Health Inc., a biopharmaceutical company focused on developing treatments for rare diseases. It discusses Bellus' lead drug candidate KIACTA, currently in a Phase III confirmatory study for AA amyloidosis, a rare and deadly kidney disease with no approved treatments. The Phase II/III study showed positive efficacy and safety results. The document outlines the Phase III study design and expected milestones, including completion of patient enrollment and top-line data readout. It also summarizes Bellus' pipeline, partnership, financial position, and potential exit strategy following Phase III results for KIACTA.
This corporate presentation discusses BELLUS Health's focus on developing drugs for rare diseases. Their lead product candidate, KIACTA, is in Phase 3 trials for AA amyloidosis, a rare and deadly kidney disease with no existing treatment. Positive results from a previous Phase 3 trial showed KIACTA slowed kidney function decline. If successful, KIACTA could achieve premium pricing compared to other orphan drugs. BELLUS is also developing treatments for other rare diseases including sHUS and AL amyloidosis through clinical trials and partnerships.
Bellus Corporate Presentation - February 2016BellusHealth
This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc., provides an overview of the company's rare disease pipeline and lead product candidate KIACTA. KIACTA is in Phase III development for AA amyloidosis, a rare and deadly kidney disease with no approved treatment. Positive results from an earlier Phase II/III study showed KIACTA slowed kidney function decline. A confirmatory Phase III study has completed enrollment with topline data expected in Q2 2016. If successful, KIACTA has potential peak sales of $600M-$1B annually. BELLUS also has a second rare disease candidate, Shigamab, in preclinical development for STEC related
IntelGenx is an innovative pharmaceutical company focused on oral thin film drug delivery technologies. They have developed a proprietary oral thin film technology platform called VersaFilm that can be used to improve existing drugs or develop new products. Their pipeline includes several product candidates targeting large markets like migraines, erectile dysfunction, and brain degenerative diseases. They have state-of-the-art manufacturing facilities and strategic partnerships to commercialize their products globally.
This management presentation discusses Bellus Health's focus on developing drugs for rare diseases, starting with conditions affecting the kidneys. It highlights the company's lead drug KIACTA for treating AA amyloidosis, which has shown positive Phase 2/3 results and is currently in a Phase 3 confirmatory study. The presentation also discusses KIACTA's potential in additional indications like sarcoidosis, as well as the company's partnership with Auven Therapeutics and its diversifying pipeline. Bellus Health aims to drive shareholder value through executing ongoing clinical trials, progressing its rare disease pipeline, and achieving near-term milestones.
- Sanofi's CEO Olivier Brandicourt presented at a healthcare conference on September 14, 2018.
- He discussed Sanofi's continued progress on its strategic transformation including acquiring new assets, driving simplification through restructuring, reshaping its portfolio, and executing new product launches.
- Brandicourt highlighted several potentially significant drug approvals and trial results for new drugs and indications expected over the next 12 months that could further advance Sanofi's growth.
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Bellus corporate presentation march 23BellusHealth
This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc (TSX: BLU), provides an overview of the company and its rare disease drug pipeline. The lead product candidate KIACTA has shown positive results in a Phase II/III trial for AA amyloidosis, a rare and deadly kidney disease with no approved treatments. BELLUS plans to complete the ongoing Phase III confirmatory trial in 2016 to support regulatory approval. The company is also developing KIACTA for sarcoidosis and has a second rare disease candidate, Shigamab, in preclinical testing for STEC related hemolytic uremic syndrome.
Roberto Bellini presented an overview of BELLUS Health Inc. Key points included:
- BELLUS is focused on developing products for amyloid-related diseases including its lead product KIACTA for AA amyloidosis currently in Phase III trials.
- KIACTA has shown statistically significant results in reducing kidney function decline in Phase II/III trials. A partnership with Auven Therapeutics is funding the Phase III confirmatory trial.
- BELLUS is acquiring Thallion which adds the antibody Shigamabs targeting sHUS to its pipeline. Shigamabs has completed Phase II trials and the strategy is to reposition it for treatment of sHUS.
This document discusses new drug applications (NDAs) and abbreviated new drug applications (ANDAs) submitted to the US Food and Drug Administration (FDA).
It begins with an overview of the drug development process, then describes the goal and contents of an NDA, including clinical data from trials and information on safety, efficacy, and manufacturing. It also classifies types of NDAs.
The document then discusses ANDAs for generic drugs, noting they do not require new clinical trials but must demonstrate bioequivalence to the brand drug. It covers patent certification requirements for ANDAs.
In conclusion, the key differences between NDAs and ANDAs are summarized, with ANDAs taking less time and money but
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BLU-5937 is a selective P2X3 antagonist being developed for the treatment of chronic cough. It has shown potent anti-tussive effects in guinea pig models of cough by blocking P2X3 homotrimeric receptors linked to cough hypersensitivity, without impacting taste function which is linked to P2X2/3 heterotrimeric receptors. Preclinical studies demonstrate BLU-5937 is highly selective for the P2X3 receptor over P2X2/3, reduces cough at doses blocking P2X3 without affecting taste, and has favorable drug-like properties. A Phase 1 and Phase 2 clinical trial program is planned to evaluate BLU-5937's tolerability
Physicians were enthusiastic about BLU-5937's potential to treat chronic cough due to its novel mechanism of action targeting P2X3 receptors. BLU-5937 may provide efficacy comparable to the first-in-class P2X3 antagonist with reduced taste disturbances based on preclinical data. Physicians saw potential for BLU-5937 to be used across multiple patient types and treatment settings. However, physicians expressed concern over taste disturbances reported with the competitor, particularly the 20% rate of complete taste loss and 40% reporting bothersome effects.
Bellus health agm presentation may 9 2017 v finalBellusHealth
Bellini provided an overview of BELLUS Health Inc., including its core capability of identifying and developing innovative drug candidates. Its lead drug candidate, BLU-5937, is a potentially best-in-class P2X3 antagonist for chronic cough that has shown positive preclinical efficacy and a strong safety profile. BELLUS also has a balanced pipeline including three partnered mid-stage programs. Upcoming milestones include completing IND-enabling studies for BLU-5937 and progressing partnered programs.
Bellus Health Bloom Burton Presentation 2017BellusHealth
- BELLUS Health Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for chronic cough and other hypersensitization disorders.
- Their lead drug candidate, BLU-5937, is a highly selective P2X3 antagonist being developed for chronic cough. It has the potential for best-in-class efficacy and tolerability based on preclinical and clinical data.
- BELLUS has a balanced pipeline that includes partnered programs in sarcoidosis, Alzheimer's disease, and Fragile X syndrome in addition to their wholly-owned chronic cough program.
This document discusses BELLUS Health licensing BLU-5937, an oral P2X3 antagonist for chronic cough, to NEOMED Institute. It summarizes that BLU-5937 has potential to be best-in-class for this large market with high unmet need, as P2X3 is a validated target. The drug candidate has favorable properties including potency, selectivity, oral bioavailability and a clear development path. Key terms of the transaction provide significant upside potential for BELLUS investors through milestone payments and low single digit royalties.
This document summarizes the licensing of BLU-5937, an oral P2X3 antagonist in development by BELLUS Health, to NEOMED Institute for chronic cough. Key points include:
1) BLU-5937 has potential to be best-in-class for treating chronic cough, a large market with high unmet need, by having superior selectivity, potency, efficacy and safety over other P2X3 antagonists.
2) Chronic cough significantly impacts patient quality of life and there are few existing treatment options. P2X3 is a validated molecular target involved in cough reflex hypersensitivity.
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This corporate presentation by Roberto Bellini, President and CEO of BELLUS Health Inc (TSX: BLU), provides an overview of the company's pipeline and assets. The key points are:
1) BELLUS has a late stage pipeline focused on developing drugs for rare kidney diseases, led by KIACTA for AA amyloidosis which has completed Phase 3 trials.
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The document summarizes results from a phase 3 clinical trial testing Kiacta (Eprodisate disodium) for the treatment of AA amyloidosis. The trial did not meet its primary endpoint of reducing renal function decline. Specifically, there was no significant difference between the Kiacta and placebo groups in time to first renal deterioration event. Secondary and safety analyses also found no significant differences between the groups. The phase 3 trial was larger than the previous phase 2/3 trial but had a similar study design and patient population.
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This corporate presentation by BELLUS Health provides an overview of the company and its lead drug candidate KIACTA for the rare kidney disease AA amyloidosis. Key points include:
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- AA amyloidosis has no approved treatments and represents a potential $600M-$1B market opportunity for KIACTA.
- The company has a late-stage pipeline of drugs for rare diseases and is fully funded through milestones. Positive Phase III data for KIACTA could lead to a commercial partnership.
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1. Bloom Burton Healthcare Investor
Conference Presentation
May 3, 2018
r
Roberto Bellini
President and Chief ExecutiveOfficer
Twitter: @rbellini
2. Forward-Looking Statements
Certain statements contained in this presentation, other than statements of fact that are independently verifiable at the
date hereof, may constitute “forward-looking statements” within the meaning of Canadian securities legislation and
regulations. Such statements, based as they are on the current expectations of management, inherently involve numerous
important risks, uncertainties and assumptions, known and unknown, many of which are beyond BELLUS Health Inc.'s
control. Such risks factors include but are not limited to: the ability to expand and develop its project pipeline, the ability
to obtain financing, the impact of general economic conditions, general conditions in the pharmaceutical industry,
changes in the regulatory environment in the jurisdictions in which BELLUS Health Inc. does business, stock market
volatility, fluctuations in costs, changes to the competitive environment due to consolidation, achievement of forecasted
burn rate, potential payments/outcomes in relation to indemnity agreements and contingent value rights, achievement of
forecasted pre-clinical and clinical trial milestones and that actual results may vary once the final and quality-controlled
verification of data and analyses has been completed. In addition, the length of BELLUS Health Inc.’s drug candidates’
development process, their market size and commercial value, as well as the sharing of proceeds between BELLUS
Health Inc. and its potential partners from potential future revenues, if any, are dependent upon a number of factors.
Consequently, actual future results and events may differ materially from the anticipated results and events expressed in
the forward-looking statements. BELLUS Health Inc. believes that expectations represented by forward-looking
statements are reasonable, yet there can be no assurance that such expectations will prove to be correct. The reader
should not place undue reliance, if any, on any forward-looking statements included in this presentation. These forward-
looking statements speak only as of the date made, and BELLUS Health Inc. is under no obligation and disavows any
intention to update publicly or revise such statements as a result of any new information, future event, circumstances or
otherwise, unless required by applicable legislation or regulation. Please see BELLUS Health Inc.’s public filings with
the Canadian securities regulatory authorities, including the Annual Information Form, for further risk factors that
might affect BELLUS HealthInc. and its business.
2
3. Generating value by advancing drug candidates through clinical studies
BELLUS Overview
3
• Listed on Toronto Stock Exchange (BLU.TO)
• BLU-5937: potentiallybest-in-class drug for multi $B market
− Chronic cough affects ~10% of adults in U.S., large unmet need
− Clinically validated target, clear and efficient development path
− Entering Phase 1: Q3 2018
• Pipeline with three mid-stage partneredprograms
• Financed for 2+ years through multiple clinical milestones
• Management with track record of execution
4. Team
Dr. Francesco Bellini
(Chair)
Franklin Berger
Pierre Larochelle
Roberto Bellini
Dr. Clarissa Desjardins
Joseph Rus
Dr. Youssef Bennani
Board of Directors
Roberto Bellini,
President and Chief
Executive Officer
Dr. Denis Garceau,
Senior Vice President,
Drug Development
François Desjardins,
Vice President,
Finance
Tony Matzouranis,
Vice President,
Business Development
Management
Management with a track record of execution 4
5. Problem: Refractory Chronic Cough
5
Cough lasting
>8 weeks,
0
therapies that are
safe and effective
2.6M
patients in U.S. with
longstanding refractory chronic
cough
Multi $B
market potential
“I see patients that have been
coughing 2 months to 30 years.
Within that group, there is a good
portion where I am the 8th or
10th doctor.”
– Chronic Cough KOL
Major
impact on
quality of life
6. Cause: Hypersensitive Cough Reflex
Coughing trigger
(ex. asthma attack)
Cellular damage activates
P2X3 receptors in airway
Airway hyper-excitability
Chronic cough
Inflammation
/Cell injury
P2X3 is a sensory receptor found in peripheral nervous system with central role in
triggeringcough reflex 6
ATP and
Cytokines
Primary afferent
(A𝛿 or C-fiber)
ATP
ATP
Receptors
P2X3
P2X3-containing
Primary afferent
Modified from Purinergic Signaling (2012) 8 (Suppl 1)
7. Treatment in Development is Suboptimal
Acquired in 2016 for $1.25B ($500M upfront) based on phase 2 data 7
Merck & Co., Inc. (2017). Merck Announces Presentation of Phase 2 Results for MK-7264, an Investigational, P2X3 Receptor Antagonist, Being Evaluated for the Treatment of Chronic Cough. [Press Release]. Retrieved from
http://www.mrknewsroom.com/news-release/research-and-development-news/merck-announces-presentation-phase-2-results-mk-7264-inve
Reduces awake cough
frequency by
86%
Effective
80%
of patients have taste
alteration or taste loss
Major Side Effect
MK-7264Merck
Mechanism: P2X3 antagonist
8. MK-7264 Effecton Taste Likely Caused by Inhibition of
P2X3 and P2X2/3
Project hypothesis: Opportunityfor highly selective P2X3 antagonist to reduce
cough, maintaintaste (no P2X2/3 inhibition) 8
P2X2/3 heterotrimers
have major role in
taste
P2X3 homotrimers
have primary role in
cough reflex
P2X3 and P2X2/3 are ATP-gated ion channels that transmit sensory signals:
P2X3
P2X2/3
9. Our Solution
vs. MK-7264 in animal studies
No
impact on taste
BLU-5937BELLU
S
Equivalent
reduction in cough
frequency
Project hypothesis validatedin animal studies 9
Highly selective P2X3 antagonist
10. 0
5
10
15
20
25
Control 0.3 mg/kg p.o. 3 mg/kg p.o. 30 mg/kg p.o.
BLU-5937 MK-7264
Cough Inhibition in Guinea Pig Model
BLU-5937 inhibitscough dosedependentlyand comparablyto MK-7264
10
Treatments (control,BLU-5937, MK-7264)were administered orally(p.o.) 2 hours prior to tussive agent exposure: citric acid
(0.1 M, aerosol) and histamine (0.6 mM,aerosol); n=6 animals per group
* **
* p<0.05
Coughcount
Cough Response Study
11. Taste Effectin Rat Taste Model
Control 10mg/kg 20 mg/kg Control 10 mg/kg 20 mg/kg
BLU-5937 MK-7264
VolumeofQuinineWaterConsumed(ml)
*
*
0
0.5
1
1.5
2
2.5
3
3.5
4
4.5
Two Bottle Rat Taste Study
Treatments (control,BLU-5937, MK-7264)were administered ip: animals were water-fasted overnight andpresented with one bottleof
water and one bottle of water plus quinine (0.3mM) at Tmax ; volume of liquid consumedmeasured for15 minutes; n=10 animals per
group; * p < 0.05 vs control
MK-7264 changes taste perception; BLU-5937 does not 11
12. Strong drug candidate profile with potential to be best in P2X3 class
BLU-5937: Potential Best-in-Class Profile
12
Targeting
~2.6M
US Patients
Broad and
comprehensive IP to
2034
High
Selectivityand Potency
for P2X3
Twice Daily
Oral Dosing
Expected
No
safety findings of
concern
13. Market
Payer discussions and comparable
product analysis support $300-600 per
month pricing
BLU-5937: addressing potential multi billiondollar refractorychronic cough market
Large addressable patient population
Torreya Insights Report 2017
Comparable products
2.6M
Primary addressable patients
(idiopathic, treatment refractory
> 1 yr)
10% or
26.3M
chronic cough
patients
263M U.S. adults
13
14. Key Development Milestones
Q2 2018
File clinical trial application
2019
Q3 2018
Q4 2018
Start Phase 1
Start Phase 2
Phase 1 data
Phase 1 enabling studies Complete
Complete
Phase 1 design
First subject dosed
Effect on cough and taste
Dose selection for Phase 3
Effect on taste
Safety/tolerability
Dose selection for Phase 2
Efficient development plan with short term value inflectionpoints
14
ISOC Presentation
June 27th, London
15. Proposed Phase 1 Study Design
N ≈ 60 healthy adult subjects
~6 cohorts of 10 subjects (8 active:
2 placebo) administeredsingle dose
N ≈ 30 healthy adult subjects
~3 cohorts of 10 subjects (8 active:
2 placebo) administeredmultiple
dose for 7 days
Dose selectedbased on single dose
portion of study
Phase 1 designed to assess safety, tolerability (including taste effect) and
drug levels
Single Ascending Dose Multiple Ascending Dose
Measure Drug
Plasma Levels for
Phase 2 dosing
Assess Tolerability
includingtaste effect
Assess Safety
Key Objectives
15
16. Three mid-stage partneredprojects with revenue share and/or royaltypotential
Additional Partnered Programs in Pipeline
16
Program Partner Indication Stage Next Steps
KIACTATM Sarcoidosis
Phase 2
ready
Auven evaluating partnering options
to finance/conduct Phase 2 study
AMO-01
Intellectual
disabilities
Phase 2
ready
AMO’s Phase 2 study initiation
planned for 2018
ALZ-801
Alzheimer’s
disease
Phase 3
ready
Alzheon preparing for Phase 3 in
APOe4 homozygous patients
17. Stock and Financial Information
Current cash runway provides 2+ years of capital through multiple milestones
Stock Information
Shares (basic) 119.5M
Shares (fully diluted) 132.7M
Market Capitalization ~$60M
Key Financials1
Cash $23.9M
Ownership
Insiders ~30%
Institutional ~35%
1
as at December 31, 2017
$20M
financingcompleted on
December 12th with
significant institutional
healthcare investor
participation
1717
18. r
Connect With Us
Follow our ticker: BLU.TO
Follow us on Twitter: @rbellini / @BELLUSHealth
Join our LinkedIn group
Check out our website and read our blog @
www.bellushealth.com
Join our mailing list
20. “I see patients that have been coughing 2 months to 30 years. Withinthat group,
there is a good portionwhere I am the 8th or 10th doctor.”
– Chronic Cough KOL
Chronic Cough
20
Characteristics
Cough lasting > 8 weeks, associated
with:
• Pulmonary diseases (asthma, COPD, IPF)
• Extra-pulmonary disorders (allergic
rhinitis, gastro-oesophageal reflux)
• Side effect of certain drugs
• No identifiable cause
Cough frequency can be high (10-100s
times per hour) with lengthy duration
(months or years)
21. “There are some patients who really can’t functionand have urinary incontinence
issues or can’t sleep in the same bed as their spouses”
-Allergist
Impact on Society
21
Time and resource intensive for the
healthcare system
• Chronic cough affects ~10% of the adult
population
• 38% of pulmonologist practice
• Resource intensive diagnosis
• Complex patient pathway with many
physicians involved (PCP, Allergist, ENT,
Pulmonologist, Cough Clinic)
Patients Healthcare System
Physical, social, psychosocial
complications
• Sleep deprivation
• Chest pain
• Urinary incontinence
• Interference with lifestyle, work & leisure
• Anxiety / Depression
22. Limited and Inadequate Treatment Options for Chronic
Refractory/Unexplained Cough
• Limited efficacy and/or duration of effect
• Addiction potential
• Significant side effects
• Inability to use on a chronic basis
Treatments Inadequate Results
• Opioids
• Benzonatate
• Gabapentin / Pregabalin
• Speech therapy
• Dextromethorphan (cough syrup)
“There is truly a need for a safe and effective non-narcotic,non-sedatingcough
suppressant for those that have gone through a thorough workup and treatment for
reversible causes” -Pulmonologist 22
23. P2X3 Receptor: Clinically Validated Target
Targeting P2X3 is a clinicallyvalidatedstrategyfor treatingchronic cough 23
Merck & Co., Inc. (2017). Merck Announces Presentation of Phase 2 Results for MK-7264, an Investigational, P2X3 Receptor Antagonist, Being Evaluated for the Treatment of Chronic Cough. [Press Release]. Retrieved from
http://www.mrknewsroom.com/news-release/research-and-development-news/merck-announces-presentation-phase-2-results-mk-7264-inve
Merck’s MK-7264 / AF-219 - P2X3 benchmark compound
Reduction in Awake Cough Frequency
*37% vs. placebo (p<0.05)
NB: Doses are BID
0%
20%
40%
60%
80%
100%
Placebo 7.5 mg 20 mg 50 mg
*
Phase IIb (253 patients; 12
week study) meets primary
endpoint by reducing awake
cough frequency by
86%*
24. MK-7264: Significant Adverse Taste Effect
Two Phase 3 studies for MK-7264 startedin March 2018 using 15mg and 45mg
BID doses 24
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
Placebo 7.5mg 20mg 50mg
Change in Taste Perception Complete Loss of Taste
At therapeutic dose (50 mg BID):
~80%
of patients
reported change
in taste
perception
~40%
of patients reported
very/extremely
bothersome change in
taste perception
Merck & Co., Inc. (2017). Merck Announces Presentation of Phase 2 Results for MK-7264, an Investigational, P2X3 Receptor Antagonist, Being Evaluated for the Treatment of Chronic Cough. [Press Release]. Retrieved from
http://www.mrknewsroom.com/news-release/research-and-development-news/merck-announces-presentation-phase-2-results-mk-7264-inve
Percent of Patients Reporting Taste Side Effect
NB: Doses are BID
Effect on taste perception likely
due to MK-7264 inhibiting
P2X3 and P2X2/3
25. Key Differentiating Factor: P2X3 vs P2X2/3 Selectivity
BLU-5937 MK-7264
hP2X3 (IC50) Low nM Mid nM
hP2X2/3 (IC50) Mid µM High nM
Fluorescent calcium flux assay, using Fluo-8 kit and 3 µM α,β Me AT, performed in
HEK293 cells stably expressing P2X3 and P2X2/3; 12 concentrations of each compound
tested.
BLU-5937: right characteristics toinhibit cough with little/noeffect on taste 25
BLU-5937 is
than MK-7264 for the human P2X3 receptor
10x
more potent
>1000x
more selective (vs P2X2/3)