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 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 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 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 (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 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 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 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
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.
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 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 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 (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 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 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 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
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. (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.
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.
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.
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.
This document discusses a pharmaceutical company focused on developing drugs for gastrointestinal disorders. It provides estimates and forward-looking statements about 3 late-stage clinical trials investigating treatments for hemorrhoids, anal fissures, and fecal incontinence. It notes risks and uncertainties that could influence projections, and that actual results may differ from estimates. Key company leadership and development team members are also summarized.
This document discusses estimates and forward-looking statements regarding a pharmaceutical company focused on developing products for gastrointestinal disorders. It contains 3 late-stage clinical trials, including Phase III trials for hemorrhoids and anal fissures. The document outlines risks and uncertainties that could impact projections, including regulatory approval, clinical trial results, funding, and competition. It also provides details on management experience and the development plans and milestones for the company's lead product candidates.
Chair, Charles Vega, MD, FAAFP, prepared useful Practice Aids pertaining to influenza for this CME/MOC/NCPD activity titled “Examining Novel Approaches to Improve the Management of Influenza: An Animated Exploration.” For the full presentation, downloadable Practice Aids, and complete CME/MOC/NCPD information, and to apply for credit, please visit us at https://bit.ly/31FYp8J. CME/MOC/NCPD credit will be available until July 20, 2022.
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.
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.
- 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.
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 “flair-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.
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
The MODDERN Cures Solution updates the regulatory system by removing barriers to invention and providing greater predictability in the search for answers to our nation’s unmet medical needs. It’s in everyone’s interest to push for a MODDERN regulatory system that encourages scientific inquiry, rewards ingenuity, and safely speeds modern miracles into everyday diagnostics and treatments that improve patient outcomes.
This document summarizes regulatory issues related to reporting and investigating adverse events following immunization (AEFI) in India. It provides definitions of key terms like drugs, adverse drug reactions, and serious adverse reactions. It describes the roles and responsibilities of different organizations in the regulatory framework, including the Central Drugs Standard Control Organization (CDSCO), National Regulatory Authority (NRA), and Central Drug Laboratory (CDL). It discusses some past AEFI cases reported to the Drug Controller General of India (DCGI) and the modus operandi adopted in investigations. Limitations and suggestions to strengthen the AEFI monitoring and response system are also highlighted.
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
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.
This white paper focuses on the 505(b)(2) New Drug Approval (NDA) regulatory pathway, which relies on the public literature of clinical studies and/or the FDA's filing of safety and efficacy data for a previously approved drug.
This document outlines the regulations for investigational new drugs as described in Part 312 of Title 21 of the Code of Federal Regulations. It discusses the requirements for investigational new drug applications (INDs), including the content that must be submitted in an IND. Key points covered include the phases of clinical investigation for a new drug, safety reporting requirements for INDs, protocols for amending an IND, and the conditions under which FDA can place a clinical hold, terminate an IND, or change a drug's status. The purpose is to ensure new drugs are properly evaluated for safety and effectiveness before being approved for marketing.
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 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.
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.
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.
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.
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.
This document discusses a pharmaceutical company focused on developing drugs for gastrointestinal disorders. It provides estimates and forward-looking statements about 3 late-stage clinical trials investigating treatments for hemorrhoids, anal fissures, and fecal incontinence. It notes risks and uncertainties that could influence projections, and that actual results may differ from estimates. Key company leadership and development team members are also summarized.
This document discusses estimates and forward-looking statements regarding a pharmaceutical company focused on developing products for gastrointestinal disorders. It contains 3 late-stage clinical trials, including Phase III trials for hemorrhoids and anal fissures. The document outlines risks and uncertainties that could impact projections, including regulatory approval, clinical trial results, funding, and competition. It also provides details on management experience and the development plans and milestones for the company's lead product candidates.
Chair, Charles Vega, MD, FAAFP, prepared useful Practice Aids pertaining to influenza for this CME/MOC/NCPD activity titled “Examining Novel Approaches to Improve the Management of Influenza: An Animated Exploration.” For the full presentation, downloadable Practice Aids, and complete CME/MOC/NCPD information, and to apply for credit, please visit us at https://bit.ly/31FYp8J. CME/MOC/NCPD credit will be available until July 20, 2022.
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.
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.
- 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.
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 “flair-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.
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
The MODDERN Cures Solution updates the regulatory system by removing barriers to invention and providing greater predictability in the search for answers to our nation’s unmet medical needs. It’s in everyone’s interest to push for a MODDERN regulatory system that encourages scientific inquiry, rewards ingenuity, and safely speeds modern miracles into everyday diagnostics and treatments that improve patient outcomes.
This document summarizes regulatory issues related to reporting and investigating adverse events following immunization (AEFI) in India. It provides definitions of key terms like drugs, adverse drug reactions, and serious adverse reactions. It describes the roles and responsibilities of different organizations in the regulatory framework, including the Central Drugs Standard Control Organization (CDSCO), National Regulatory Authority (NRA), and Central Drug Laboratory (CDL). It discusses some past AEFI cases reported to the Drug Controller General of India (DCGI) and the modus operandi adopted in investigations. Limitations and suggestions to strengthen the AEFI monitoring and response system are also highlighted.
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
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.
This white paper focuses on the 505(b)(2) New Drug Approval (NDA) regulatory pathway, which relies on the public literature of clinical studies and/or the FDA's filing of safety and efficacy data for a previously approved drug.
This document outlines the regulations for investigational new drugs as described in Part 312 of Title 21 of the Code of Federal Regulations. It discusses the requirements for investigational new drug applications (INDs), including the content that must be submitted in an IND. Key points covered include the phases of clinical investigation for a new drug, safety reporting requirements for INDs, protocols for amending an IND, and the conditions under which FDA can place a clinical hold, terminate an IND, or change a drug's status. The purpose is to ensure new drugs are properly evaluated for safety and effectiveness before being approved for marketing.
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 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 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) 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 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
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.
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- 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.
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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.
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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.
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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:
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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
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Blu 5937 kol event sept 20 v final
1. BLU-5937 Update and Chronic
Cough Key Opinion Leader Event
September 20, 2017
2. Forward Looking Statements
Certain statements contained in this news release, 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 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. The Company
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 news release. 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 InformationForm, for further risk factorsthat might affect BELLUS HealthInc. and itsbusiness.
2
3. Differentiatedprofile targetinglarge unmet need in chronic cough
BLU-5937 Overview
3
P2X3:
validatedtarget
for chronic
cough
Developed atAstraZeneca in P2X3 antagonist discovery
program, then NEOMED Institute
Global rights licensedby BELLUS in February 2017
BLU-5937
Background
BLU-5937:
Potentially
best-in-class P2X3
antagonist
Potential for differentiated product profile with improved
efficacyand reduced/no taste disturbance
Clear, efficient path to demonstratesuperiority
Merck acquiredAfferent Pharma’s P2X3 antagonist program in
2016 for US$500M based on positive Phase 2 data
Problematic side effect profile: 80% of patients experienced taste
disturbance
4. A Review of Chronic Cough
Prof Jacky A. Smith MB, ChB, FRCP, PhD
University of Manchester
r
5. Chronic Cough
5
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)
5
6. UK Postal Questionnaire Survey Danish PopulationBased Cohort
Prevalence of Chronic Cough
6
Ford et al Thorax2006;61;975-979 Colak et al Chest 2017;152:563-573
3500
3000
2500
2000
1500
1000
500
Never Once a
month
Once a
month to
once a
week
Once
a
week
to
once
a day
Once a
day or
more
Numberofindividuals
Frequency in last 8
weeks
Interfered with Daily
Activities in last 8 weeks
7. Major Impact on Patients with Refractory /
Unexplained Chronic Cough
7
Social
complications
Physical
complications
Significant disruptionin day to day life for chronic cough patients
7
Fatigue
Sleep deprivation
Vomiting
Incontinence
Headache
Chest pain
Rib fracture
Psychosocial
complications
Interference with
lifestyle, work &
leisure
Difficulty
conversing
Embarrassment
of coughing in
public
Anxiety
Anger
Depression
Distress
8. Few Treatment Options for Chronic
Refractory/Unexplained Cough
Can be efficacious
Limited use due to side
effects and potential for
addiction
Anesthetize the stretch
receptors in the lungs
Temporary relief
Potential serious side effects
if capsule broken
BenzonatateOpioids Dextromethorphan
Key ingredient in OTC
cough suppressants
Limited efficacy
Significant need for efficacious chronic cough therapy that is non-narcotic and non-
sedating
Gabapentin/pregabalin Speech Therapy
Has shown some efficacy
especially in combination
with pharmacotherapy
Neuromodulators with
variable efficacy and
significant CNS side effects
8
10. P2X3 Receptor: Clinically Validated Target
Targeting P2X3 is an efficacious strategyfor treatingchronic cough
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 - P2X3 Antagonist
Reduction in Awake Cough Frequency
(from Baseline Compared to Placebo)
* p<0.05 vs. placebo
0%
20%
40%
60%
80%
Placebo 7.5 mg 20 mg 50 mg
*
Phase IIb (253 patients; 12
week study) showed
reduction in awake cough
frequency of
84% vs baseline
37% vs placebo
at 50mg dose
10
11. MK-7264: Significant Adverse Taste Effect
Taste effect likelydue to low selectivityfor P2X3; MK-7264 also inhibiting
P2X2/3, particularlyat 50mg dose
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
Phase IIb: Percent of Patients Reporting Taste
Side Effect
11
12. Clinical Studies in Chronic Cough
Crossover design is very
efficient for Phase 2 proof of
concept
Crossover design requires
limited number of patients
and short duration with
objective cough monitoring
Crossover design results have
been confirmed in longer
term study
Reduction in awake cough
frequency as measured by
cough monitor
Good correlation between
cough frequency and patient
reported measures
Potential for important
placebo effect in parallel
group studies
EndpointsDesign Regulatory
At least 2 large Phase 3
studies required for
approval
Primary endpoint likely to
be cough frequency
reduction using validated
cough recorder
Recent learnings in clinical studies have provided clear path for development of
chronic cough drugs 12
13. 13
TRP modulators
• Main target (TRPV1) has shown
serious toxicity issues with first
compounds
• Two recent Phase 2 trials in chronic
cough patients showed effect on cough
challenge but not cough frequency
NK1 antagonists
• Repurposed class initially developed for
depression
• Also target afferent nerve signaling
especially at first synapse
• Limited clinical validation in chronic
cough
P2X3 antagonists
• Drug class inhibiting afferent pathway signals
from respiratory tract
• Most promising and competitive novel class of
antitussive medicine
nAChR modulation
• CNS acting modulators, could inhibit
cough signal processing in the brain
• Limited mechanistic characterization in
humans
Drug Classes in Development
13
15. Strong drug candidate profile with potential to be best in P2X3 class
BLU-5937: Best-in-Class Profile
15
Targeting
~2.7M
US Patients
Broad and
comprehensive IP to
2034
High
Potency and Selectivity
for P2X3
Twice Daily
Oral Dosing
Expected
No
safety findings of
concern
16. P2X3 and P2X2/3 Roles in Cough and Taste
Target P2X3 to reduce cough; avoid P2X2/3 to maintaintaste 16
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:
P2X2/3
P2X3
17. Potency, Selectivity for Human hP2X3 vs. hP2X2/3
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 17
BLU-5937 is
than MK-7264 for the human P2X3 receptor
10x
more potent
>1000x
more selective (vs P2X2/3)
18. 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
Preclinical Efficacy:Cough Response in Guinea Pig
BLU-5937 inhibitscough dosedependentlyand comparablyto MK-7264 18
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
19. 0
5
10
15
20
25
30
Time Course Study (Guinea Pig Cough Model)
BLU-5937 inhibitscough comparably to MK-7264 and fora similar duration
4h 6h 8h 12h2h
BLU-5937 MK-7264
*
*
* p < 0.05
*
*
Coughcount
19
Control +
histamine
2h 8h
Treatments (control,BLU-5937, MK-7264; 30 mg/kg) were administeredorally (p.o.) 2 hours priorto tussive agent exposure:
citric acid (0.1 M, aerosol) and histamine (0.6 mM, aerosol); tussive agent exposure at 2, 4, 6, 8 and 12 hours forBLU-5937; at
2 and 8 hours for MK-7264; n=6 animals per group
Time Course Study
20. Effectof BLU-5937, MK-7264 on Taste in Rats
0
10
20
30
40
Control 10 mg/kg 20 mg/kg
BLU-5937 MK-7264
MK-7264 alters taste; BLU-5937 does not 20
%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
21. Safety & ADME Profile Overview
No safetyfindings of concern, expected BID dosing 21
No safety findings of concern
• Good safety margin in 7 day toxicity studies
(rat & dog)
• No genotoxicity
• Highly selective without off-target effect
Projected BID dosing
• Good oral bioavailability
• Elimination through metabolism
• No drug interaction anticipated
• Very low brain permeability
Safety Profile ADME Profile
22. Phase 1 Study Design
Traditional design; assess safety, tolerability(includingtaste),drug levels
Measure Drug
Plasma Levels for
Phase 2 Dosing
Assess Tolerability
IncludingTaste
Effect
Assess Safety
Key Objectives
22
Single Ascending Dose
n=48 healthy adult subjects
5 cohorts of 8 subjects
administered single dose
1 cohort of 8 subjects to
assess taste effect
Multiple Ascending Dose
n=24 healthy adult subjects
3 cohorts of 8 subjects
administered multiple dose
BID for 7 days
23. Phase 2 Proof of Concept Study Design
BLU-5937 dose escalation (n =18)
Placebo (n=18)
BLU-5937 dose escalation (n=18)
Placebo (n=18)
• N=36 unexplained/refractory chronic cough patients; >1 year coughing
• 6 sites in UK and 2 sites in US
• 4 dose levels escalated at 4-day intervals
• Endpoint: reduction in frequency of cough (cough recorder)
• Safety and tolerability assessment, including taste effect
16-day treatment 16-day treatment
4-day
washout
Similar design to Afferent/MerckPhase 2 proof of concept
23
24. Development Milestones
Safety margins
Starting dose for Phase 1
24
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
24
Q3 2018Q2 2018 2019
25. Summary – BLU-5937
Robust and efficient design
More potent and selective P2X3
inhibitor versus leading P2X3
antagonist
In vitro
Phase 2 (2019)Phase 1 (2018)
Animal
Strong and differentiated P2X3 drug candidate profile with efficient path to data
Taste effect data in humans
Cough: comparable efficacy and
duration vs. leading P2X3 antagonist
Taste: no taste effect observed
25