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 (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 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 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.
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
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 bbhic presentation may 3 st 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 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 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
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 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 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.
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
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 bbhic presentation may 3 st 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 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 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
Bellus Corporate Presentation July 2016BellusHealth
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.
2) The company also has programs in preclinical/Phase 2 development including Shigamab for sHUS and KIACTA for sarcoidosis.
3) Additional assets include royalty stakes in drugs being developed by partners for Alzheimer's disease, Fragile X syndrome, and an equity stake in an
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 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
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.
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.
Opexa therapeutics corporate presentation june 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa has completed enrollment in its Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) patients and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and commercial milestones are achieved.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTC) that cause damage in MS. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and sales milestones are achieved.
Opexa Therapeutics Corporate Presentation July 2014OpexaTherapeutics
Opexa Therapeutics is developing precision immunotherapy treatments, with its lead candidate Tcelna in Phase IIb clinical trials for secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are believed to trigger an immune response targeting pathogenic T-cells that destroy myelin. The Phase IIb trial in SPMS has fully enrolled 180 patients and top-line data is expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Revised advisory on the use of hydroxychloroquine as prophylaxis for sarscovi...sabrangsabrang
The document provides revised guidelines on the use of hydroxychloroquine (HCQ) as prophylaxis for COVID-19. It summarizes studies showing HCQ's antiviral effects and safety as prophylaxis for healthcare workers. It recommends HCQ prophylaxis for asymptomatic healthcare and frontline workers, and asymptomatic household contacts of COVID-19 patients. Precautions include avoiding contraindications, medical supervision, informed consent, and monitoring for adverse effects. Prophylaxis should continue with clinical and ECG monitoring beyond 8 weeks if initially beneficial.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET). Phase 1 and 2 trials show it has a favorable safety profile compared to immediate release anagrelide with fewer adverse events. A pivotal Phase 3 trial is planned for Q2 to initiate comparing GALE-401 to best available therapies in patients who failed or were intolerant to hydroxyurea.
NeuVax is an investigational immunotherapy targeting HER2-positive breast cancer. It contains an immunodominant HER2 peptide that stimulates CD8+ T-cells to destroy tumor cells. Phase 2 trials are ongoing in various breast cancer populations in combination with Hercept
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.
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.
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.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET) as a potential third line therapy. Phase 1 and 2 clinical trials showed that GALE-401 maintains platelet lowering effects while reducing peak plasma concentrations and adverse events compared to immediate release anagrelide. The company plans to initiate a Phase 3 trial in Q2 2017 to further evaluate GALE-401 in ET patients who are intolerant or refractory to first and second line therapies such as hydroxyurea and anagrelide.
GALE-401 is a controlled release formulation of anagrelide targeting patients who are intolerant to immediate release anagrelide, the current standard of care for third line treatment of essential thrombocythemia. Phase 1 and 2 clinical trials demonstrated GALE-401's improved pharmacokinetic profile with lower Cmax and longer half-life, maintaining platelet lowering effects while showing better tolerability. A Phase 3 trial is planned to initiate in Q2 2017. NeuVax is an immunotherapy targeting HER2-expressing cancers by eliciting a CD8+ T-cell immune response. Current clinical trials are investigating NeuVax in combination with trastuzumab for the treatment of HER2 1+/2+ breast cancer
This document provides a summary of Galena Biopharma's Q3 2016 financial results and corporate update. It discusses interim results from the Phase 3 PRESENT trial of NeuVax showing a potential delay in disease-free survival and highlights the risk of pseudoprogression in cancer immunotherapy trials. It also reviews the company's immunotherapy development pipeline, including NeuVax programs in breast and gastric cancer and GALE-301/302 programs in ovarian and breast cancer. Finally, it discusses the company's cash position and expected milestones for the remainder of 2016.
Clinical trials involve several phases of testing new medical interventions in human subjects to generate safety and efficacy data. Phase 0 trials involve microdosing to determine pharmacokinetic and pharmacodynamic properties. Phase I trials use small patient groups to determine maximum tolerated dose. Phase II trials screen for activity, safety and feasibility in larger patient groups. Phase III trials are large randomized controlled trials that compare new treatments to standard treatments. Phase IV trials monitor long-term safety and effectiveness if the treatment is approved. The goal is to progress from exploratory to definitive assessment through each phase to provide evidence for approval or non-approval of the new medical intervention.
NEWS: RespireRx Pharmaceuticals Inc. Announces Entry into Option Agreement to...RespireRX
RespireRx Pharmaceuticals Inc. Announces Entry into Option Agreement to License GABA(A) Receptor Allosteric Neuromodulator Intellectual Property from the UWM Research Foundation, Inc.
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.
3) The transaction provides BELLUS an attractive financial arrangement while leveraging NEO
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.
This document provides an overview of phase 3 clinical trials. Phase 3 trials involve large randomized controlled trials of up to 3000 patients to generate statistically significant data on a drug's safety and efficacy in different patient populations. The objectives are to demonstrate therapeutic efficacy and safety/tolerability in a representative sample. Results are submitted to regulatory agencies for marketing approval. Challenges include long duration, large sample sizes, high costs, and coordinating multiple study sites. If approved, the new drug application process requires submission of all safety, efficacy and manufacturing data to the regulatory agency for review and potential approval.
Bellus Corporate Presentation July 2016BellusHealth
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.
2) The company also has programs in preclinical/Phase 2 development including Shigamab for sHUS and KIACTA for sarcoidosis.
3) Additional assets include royalty stakes in drugs being developed by partners for Alzheimer's disease, Fragile X syndrome, and an equity stake in an
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 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
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.
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.
Opexa therapeutics corporate presentation june 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTCs) that cause damage in MS. Opexa has completed enrollment in its Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) patients and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and commercial milestones are achieved.
Opexa Therapeutics is developing Tcelna, a precision immunotherapy for the treatment of multiple sclerosis (MS). Tcelna consists of attenuated antigen-specific T-cell clones that are designed to program the immune system to target pathogenic myelin reactive T-cells (MRTC) that cause damage in MS. Opexa has completed enrollment in a Phase IIb clinical trial of Tcelna in secondary progressive MS (SPMS) and expects top-line data in mid-2016. The company has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments if certain development and sales milestones are achieved.
Opexa Therapeutics Corporate Presentation July 2014OpexaTherapeutics
Opexa Therapeutics is developing precision immunotherapy treatments, with its lead candidate Tcelna in Phase IIb clinical trials for secondary progressive multiple sclerosis (SPMS). Tcelna consists of attenuated myelin-reactive T-cell clones that are believed to trigger an immune response targeting pathogenic T-cells that destroy myelin. The Phase IIb trial in SPMS has fully enrolled 180 patients and top-line data is expected in mid-2016. Opexa has an option agreement with Merck for the development and commercialization of Tcelna in MS that could provide up to $220 million in payments.
Revised advisory on the use of hydroxychloroquine as prophylaxis for sarscovi...sabrangsabrang
The document provides revised guidelines on the use of hydroxychloroquine (HCQ) as prophylaxis for COVID-19. It summarizes studies showing HCQ's antiviral effects and safety as prophylaxis for healthcare workers. It recommends HCQ prophylaxis for asymptomatic healthcare and frontline workers, and asymptomatic household contacts of COVID-19 patients. Precautions include avoiding contraindications, medical supervision, informed consent, and monitoring for adverse effects. Prophylaxis should continue with clinical and ECG monitoring beyond 8 weeks if initially beneficial.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET). Phase 1 and 2 trials show it has a favorable safety profile compared to immediate release anagrelide with fewer adverse events. A pivotal Phase 3 trial is planned for Q2 to initiate comparing GALE-401 to best available therapies in patients who failed or were intolerant to hydroxyurea.
NeuVax is an investigational immunotherapy targeting HER2-positive breast cancer. It contains an immunodominant HER2 peptide that stimulates CD8+ T-cells to destroy tumor cells. Phase 2 trials are ongoing in various breast cancer populations in combination with Hercept
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.
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.
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.
GALE-401 is a proprietary controlled release formulation of anagrelide being developed for the treatment of essential thrombocythemia (ET) as a potential third line therapy. Phase 1 and 2 clinical trials showed that GALE-401 maintains platelet lowering effects while reducing peak plasma concentrations and adverse events compared to immediate release anagrelide. The company plans to initiate a Phase 3 trial in Q2 2017 to further evaluate GALE-401 in ET patients who are intolerant or refractory to first and second line therapies such as hydroxyurea and anagrelide.
GALE-401 is a controlled release formulation of anagrelide targeting patients who are intolerant to immediate release anagrelide, the current standard of care for third line treatment of essential thrombocythemia. Phase 1 and 2 clinical trials demonstrated GALE-401's improved pharmacokinetic profile with lower Cmax and longer half-life, maintaining platelet lowering effects while showing better tolerability. A Phase 3 trial is planned to initiate in Q2 2017. NeuVax is an immunotherapy targeting HER2-expressing cancers by eliciting a CD8+ T-cell immune response. Current clinical trials are investigating NeuVax in combination with trastuzumab for the treatment of HER2 1+/2+ breast cancer
This document provides a summary of Galena Biopharma's Q3 2016 financial results and corporate update. It discusses interim results from the Phase 3 PRESENT trial of NeuVax showing a potential delay in disease-free survival and highlights the risk of pseudoprogression in cancer immunotherapy trials. It also reviews the company's immunotherapy development pipeline, including NeuVax programs in breast and gastric cancer and GALE-301/302 programs in ovarian and breast cancer. Finally, it discusses the company's cash position and expected milestones for the remainder of 2016.
Clinical trials involve several phases of testing new medical interventions in human subjects to generate safety and efficacy data. Phase 0 trials involve microdosing to determine pharmacokinetic and pharmacodynamic properties. Phase I trials use small patient groups to determine maximum tolerated dose. Phase II trials screen for activity, safety and feasibility in larger patient groups. Phase III trials are large randomized controlled trials that compare new treatments to standard treatments. Phase IV trials monitor long-term safety and effectiveness if the treatment is approved. The goal is to progress from exploratory to definitive assessment through each phase to provide evidence for approval or non-approval of the new medical intervention.
NEWS: RespireRx Pharmaceuticals Inc. Announces Entry into Option Agreement to...RespireRX
RespireRx Pharmaceuticals Inc. Announces Entry into Option Agreement to License GABA(A) Receptor Allosteric Neuromodulator Intellectual Property from the UWM Research Foundation, Inc.
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.
3) The transaction provides BELLUS an attractive financial arrangement while leveraging NEO
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.
This document provides an overview of phase 3 clinical trials. Phase 3 trials involve large randomized controlled trials of up to 3000 patients to generate statistically significant data on a drug's safety and efficacy in different patient populations. The objectives are to demonstrate therapeutic efficacy and safety/tolerability in a representative sample. Results are submitted to regulatory agencies for marketing approval. Challenges include long duration, large sample sizes, high costs, and coordinating multiple study sites. If approved, the new drug application process requires submission of all safety, efficacy and manufacturing data to the regulatory agency for review and potential approval.
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
Opexa therapeutics corporate presentation september 2016OpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their lead asset, Tcelna, is in Phase 2b development for secondary progressive multiple sclerosis and top-line data is expected in the next 1-2 months. They are also developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies. Opexa has secured an option agreement with Merck Serono for Tcelna in MS and could receive up to $25 million in milestones if Merck exercises their option after seeing Phase 2b data.
Opexa therapeutics corporate presentation october 2014OpexaTherapeutics
Opexa Therapeutics is developing Tcelna, an autologous T cell immunotherapy, for the treatment of multiple sclerosis (MS). A Phase 2b clinical trial of Tcelna in secondary progressive MS (SPMS) is ongoing, with top-line results expected in late 2016. Previous clinical trials of Tcelna in relapsing-remitting MS showed a 37% reduction in relapse rate compared to placebo and reversal of disability progression in more active patients. Opexa signed an agreement with Merck Serono for development and commercialization of Tcelna in MS worldwide, excluding Japan, which could provide over $220 million in milestones to Opexa.
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.
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.
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Opexa therapeutics corporate presentation february 2016 webOpexaTherapeutics
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead program, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has received Fast Track designation from the FDA. Opexa is also developing OPX-212 for neuromyelitis optica, an orphan disease with no approved therapies. Preclinical studies of OPX-212 showed a statistically significant reduction in pathogenic T-cells. Topline results for Tcelna in SPMS are expected in the second half of 2016, which would be a key inflection point if positive.
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- Pipeline expansion includes OPX-212 for neuromyelitis optica, with IND submission planned for mid-2015.
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Denis garceau blu 5937 in chronic cough - cough conferenceBellusHealth
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
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- 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.
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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. Core capability: generatingvalue by advancing drug candidates
Identifying and Developing Innovative Drugs
3
• BLU-5937: potentiallybest-in-class drug for multi billiondollar 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
• Balanced portfolio: partner in three mid-stage programs
• Financed for 2+ years through multiple clinical milestones
• Management with strong track record of execution
5. Low risk and superior profile targetingpotential multi billiondollar drug class
BLU-5937: Best-in-Class Potential
5
P2X3:
Validatedtarget
for chronic
cough
Developed at Astra Zeneca and then NEOMED Institute
Global rights licensed by BELLUS in February 2017
BLU-5937
History
BLU-5937:
Potentially
best-in-class
P2X3 antagonist
High P2X3 selectivity driver for potential differentiated
product profile with improved efficacy and reduced/no
taste disturbance
Clear, efficient path to demonstrate superiority
Merck acquired a P2X3 antagonist program for
US$500M based on positive Phase 2 data
Problematic side effect profile with 80% patients
experiencing taste disturbance
6. “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
6
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)
7. “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
7
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
8. Limited and Inadequate Treatment Options for Chronic
Refractory/Unexplained Cough
8
• 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 8
9. Cause: Hypersensitive Cough Reflex
Coughing trigger
(ex. asthma attack)
Cellular damage activates
P2X3 receptors in airway
Airway hyper-excitability
Chronic cough
Inflammation/C
ell injury
P2X3 receptors found in peripheral nervous system,involved in cough as well as
pain and taste 9
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)
10. P2X3 Receptor: Clinically Validated Target
Targeting P2X3 is an efficacious strategyfor treatingchronic cough 10
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
(from baseline compared to placebo)
*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
84%*
11. MK-7264: Significant Adverse Taste Effect
Taste effect likelydue to low selectivityfor P2X3: opportunityfor highly selective
P2X3 antagonist with better efficacy/safetyprofile 11
0%
10%
20%
30%
40%
50%
60%
70%
80%
90%
Placebo 7.5mg 20mg 50mg
Taste Disturbance Complete Loss of Taste
At therapeutic dose (50 mg BID):
~80%
of patients
reported taste
alteration
~40%
of patients reported
very/extremely
bothersome taste
effect
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
12. P2X3 and P2X2/3 Roles in Cough and Taste
Inhibit P2X3 to reduce cough; do not inhibit P2X2/3 to maintaintaste 12
P2X2/3 heterotrimers
have major role in
taste
P2X3 homotrimers
have primary role in
cough reflex
ATP-gated ion channels that transmit sensory signals, function in two predominant
trimer structures:
P2X3
P2X2/3
13. Strong drug candidate profile with potential to be best in P2X3 class
BLU-5937: Best-in-Class Profile
13
Targeting
~2.6M
US Patients
Broad and
comprehensive IP to
2034
High
Potency and Selectivity
for P2X3
Twice Daily
Oral Dosing
Expected
No
safety findings of
concern
14. 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 expressingP2X3 and
P2X2/3; 12 concentrations of each compound tested.
BLU-5937: potential to inhibit cough with little/notaste disturbance 14
BLU-5937 is
than MK-7264 for the human P2X3 receptor
10x
more potent
>1000x
more selective (vs P2X2/3)
15. 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 15
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
Coughcounts
Cough Response Study
16. Taste Effectin Rat Taste Model
0
10
20
30
40
Control 10 mg/kg 20 mg/kg
BLU-5937 MK-7264
MK-7264 alters taste; BLU-5937 does not 16
%Quinine/waterconsumed
Two Bottle Rat Taste Study
Treatments (control,BLU-5937, MK-7264)were administered ip: animals were water-fasted overnight andpresented with one bottle
water and quinine (0.3mM) at Tmax ; volume of liquid consumedmeasured for15 minutes; n=10 animals per group
*
*
* p < 0.05 vs control
18. Payers discussions and product analogs support pricingof $300-600 per month
Comparables Analysis and Pricing
18
Indication
Addressable US
Patient Population Market Dynamics
2016
WACC/mo
Chronic idiopathic
constipation
35M Genericized $319
IBS with
constipation
4M Genericized $330
Adult asthma and
Adult COPD
18.4M
12M
Highly competitive,
several generics
$289
Partial onset
seizures
1M Highly competitive $570
BLU-5937 Price Analogs
Torreya Insights Report 2017
19. Key Development Milestones
Safety margins
Starting dose for Phase 1
Effect on taste
Safety/tolerability
Dose selection for Phase 2
Start Phase 1File Clinical Trial Application Start Phase 2
Effect on cough and taste
Dose selection for Phase
3
Q3 2018Q2 2018 2019
Value creatingmilestones throughout development path 19
20. 2020
Board of Directors
Dr. Francesco Bellini (Chair) Franklin Berger Pierre Larochelle
Dr. Youssef Bennani Joseph Rus Dr. Clarissa Desjardins Roberto Bellini
Management
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
Governance
21. Stock and Financial Information
2121
Current cash runway provides 2+ years of capital through multiple milestones
Stock Information
Shares (basic) 119.5M
Shares (fully diluted) 128.6M
Market Capitalization ~$45M
Key Financials1
Cash $24.0M
Ownership
Insiders ~30%
Institutional ~35%
1
as at December 12, 2017
$20M
financingcompleted on
December 12th with
significant institutional
healthcare investor
participation
22. Important operational milestones and value inflectionpoints in 2018
Multiple Upcoming Milestones to Drive Value
22
Management Past Execution Milestones
ü Attracted >$100M to funding
projects
ü Executed multiple global clinical
studies including Phase 3
ü Completed multiple transactions
(in-licensing, partnering, out-
licensing)
• 28 Day Toxicity Studies (Q2 2018)
• File regulatory dossier for BLU-
5937 Phase 1 (Q2 2018)
• Start BLU-5937 Phase 1 study (Q3
2018)
• Topline Phase 1 data including taste
(Q4 2018)
• Progress on other pipeline projects