1) ConjuChem Inc., a Canadian biotech firm, saw its fortunes boosted by a new interim leader and promising new
technology.
2) The firm's shares increased dramatically in value after Jacques Lapointe took over as interim leader and focused
the company's strategy on developing its Drug Affinity Complex (DAC) technology more deeply with one drug
candidate in particular, DAC:GLP-1, for diabetes.
3) Positive trial results for DAC:GLP-1 contributed to a surge in the company's share price and market value,
positioning it to capitalize on its new opportunity and potentially sign a major partnership deal.
This document provides an overview of the challenges facing life science startups in therapeutics, diagnostics, medical devices, and digital health. It discusses the increasing costs and regulatory hurdles of drug and device development, including higher clinical trial standards and FDA scrutiny. This has led to declining venture capital investment in life sciences as startups face greater challenges in achieving approval and profitability. The author proposes testing a Lean LaunchPad approach to help life science startups commercialize research more efficiently and reverse negative industry trends.
GSK’S Andrew Witty: Addressing Neglected Tropical Diseases and global health ...Nejmeddine Jemaa
Every day, Non Governmental Organization NGOs is confronted with the lack of access to adequate or affordable medical tools in the field. They face two major challenges the high cost of existing medicines on the one hand, and the absence of appropriate or effective treatments for many of the diseases affecting our patients on the other, we are talking about Neglected Tropical Disease NTD in the Least developed Countries LDCs.
Andrew Witty, Chief Executive Officer of Glaxo Smith Klein (GSK) delivered a speech at the Harvard Business School in Boston on February 2009 entitled “Big pharma a catalyst for Change” focused on two issues: a) promoting innovation to prevent or treat NTDs in the world’s Least Developed Countries by creating a “pharmaceutical patent pool”; b) improving the access to medicine in the poorer countries by lowering the prices of GSK’s medicines.
In deed, we are assisting a radical change in pharma Business model, we are moving from conflict to collaboration through the Medicines Patent Pool in the hope that it speed up access to newer medicines, and boost initiatives that make use of alternative financing mechanisms in order to develop new, more appropriate treatments that respond to medical needs.
On the other hand the pricing strategy dilemma facing the generic manufacturers and the non inclusion of HIV which is a major neglected disease in LDCs in the patent pool may compromise the success of such business model.
In order to deal with that two issues, GSK should include HIV drugs in their patent pool as other manufacturers and NGO are doing, and concerning the pricing strategy they should emphasize on the high quality of the original drug mandatory to eradicate this NTDs and communicate more on the fact that GSK will invest 20% of these drugs profit to improve the infrastructure of these LDCs.
The document provides an overview of life science trends in 2016, focusing on regenerative medicine. It includes interviews with thought leaders in regenerative medicine on the past, present and future of the field. The document also covers research and innovation in areas like cancer immunotherapy and personalized medicine. It discusses fundamental trends in the industry including biosimilars, biopharma blockbusters, gene editing treatments and digital medicine. The document summarizes investment and deal making activity as well as regulatory issues and developments in healthcare related to areas like antibiotics and whole genome sequencing.
The document discusses trends in medical device manufacturing and regulation. It provides an overview of Georgia's Centers of Innovation which support industry collaborations. It also summarizes challenges with the FDA approval process, including lengthy times and high costs of clinical trials that have stalled innovation. Stakeholders are advocating for reforms that balance appropriate oversight with supporting new technologies to benefit patients.
The document summarizes recent developments in the pharmaceutical industry:
- Returns on R&D investment have declined for the fourth consecutive year to 4.8% according to a Deloitte/Thomson Reuters report, despite costs to launch a new drug rising to $1.3 billion. Late stage trial failures have removed $243 billion in value from top company pipelines.
- Roche signed a potential billion dollar deal with Molecular Partners to develop a new class of cancer drug conjugates using Molecular Partner's DARPin proteins, smaller than antibodies.
- An article in the document discusses mixed results for industry research and development, with phase 3 trial failures at Lilly and GSK, but also new partnerships by Roche and
The document discusses the process and costs associated with drug development. It notes that the average cost to develop a new drug is $350 million to $5.5 billion and the process takes 6.5-7 years from discovery to approval. Key barriers to drug development include high financial costs, lengthy timelines for clinical trials, and regulatory hurdles. Approaches to reduce costs and timelines include greater use of electronic health records, simplifying clinical trial protocols, and utilizing decentralized clinical trial models.
The document summarizes news from the medical device industry. Specifically, it discusses:
1) The FDA clearance of Lumendi's Dilumen endoscopic accessory, which uses balloons to help position an endoscope and treat colonic lesions. This represents a minimally invasive alternative to surgery.
2) Varian Medical Systems' plan to spin off its imaging components business to boost its valuation after a difficult year.
3) Continued growth in patent filings from China, including in medical devices, cementing its status as an innovation leader according to the World Intellectual Property Organization.
4) Blackfynn's neurology data platform that aims to advance research by aggregating data, with
2014 Overview of significant trends in the life sciences (Biotechnology, Pharmaceutical, Device and Diagnostics) industry with Big Data in the Life Sciences featured articles.
This document provides an overview of the challenges facing life science startups in therapeutics, diagnostics, medical devices, and digital health. It discusses the increasing costs and regulatory hurdles of drug and device development, including higher clinical trial standards and FDA scrutiny. This has led to declining venture capital investment in life sciences as startups face greater challenges in achieving approval and profitability. The author proposes testing a Lean LaunchPad approach to help life science startups commercialize research more efficiently and reverse negative industry trends.
GSK’S Andrew Witty: Addressing Neglected Tropical Diseases and global health ...Nejmeddine Jemaa
Every day, Non Governmental Organization NGOs is confronted with the lack of access to adequate or affordable medical tools in the field. They face two major challenges the high cost of existing medicines on the one hand, and the absence of appropriate or effective treatments for many of the diseases affecting our patients on the other, we are talking about Neglected Tropical Disease NTD in the Least developed Countries LDCs.
Andrew Witty, Chief Executive Officer of Glaxo Smith Klein (GSK) delivered a speech at the Harvard Business School in Boston on February 2009 entitled “Big pharma a catalyst for Change” focused on two issues: a) promoting innovation to prevent or treat NTDs in the world’s Least Developed Countries by creating a “pharmaceutical patent pool”; b) improving the access to medicine in the poorer countries by lowering the prices of GSK’s medicines.
In deed, we are assisting a radical change in pharma Business model, we are moving from conflict to collaboration through the Medicines Patent Pool in the hope that it speed up access to newer medicines, and boost initiatives that make use of alternative financing mechanisms in order to develop new, more appropriate treatments that respond to medical needs.
On the other hand the pricing strategy dilemma facing the generic manufacturers and the non inclusion of HIV which is a major neglected disease in LDCs in the patent pool may compromise the success of such business model.
In order to deal with that two issues, GSK should include HIV drugs in their patent pool as other manufacturers and NGO are doing, and concerning the pricing strategy they should emphasize on the high quality of the original drug mandatory to eradicate this NTDs and communicate more on the fact that GSK will invest 20% of these drugs profit to improve the infrastructure of these LDCs.
The document provides an overview of life science trends in 2016, focusing on regenerative medicine. It includes interviews with thought leaders in regenerative medicine on the past, present and future of the field. The document also covers research and innovation in areas like cancer immunotherapy and personalized medicine. It discusses fundamental trends in the industry including biosimilars, biopharma blockbusters, gene editing treatments and digital medicine. The document summarizes investment and deal making activity as well as regulatory issues and developments in healthcare related to areas like antibiotics and whole genome sequencing.
The document discusses trends in medical device manufacturing and regulation. It provides an overview of Georgia's Centers of Innovation which support industry collaborations. It also summarizes challenges with the FDA approval process, including lengthy times and high costs of clinical trials that have stalled innovation. Stakeholders are advocating for reforms that balance appropriate oversight with supporting new technologies to benefit patients.
The document summarizes recent developments in the pharmaceutical industry:
- Returns on R&D investment have declined for the fourth consecutive year to 4.8% according to a Deloitte/Thomson Reuters report, despite costs to launch a new drug rising to $1.3 billion. Late stage trial failures have removed $243 billion in value from top company pipelines.
- Roche signed a potential billion dollar deal with Molecular Partners to develop a new class of cancer drug conjugates using Molecular Partner's DARPin proteins, smaller than antibodies.
- An article in the document discusses mixed results for industry research and development, with phase 3 trial failures at Lilly and GSK, but also new partnerships by Roche and
The document discusses the process and costs associated with drug development. It notes that the average cost to develop a new drug is $350 million to $5.5 billion and the process takes 6.5-7 years from discovery to approval. Key barriers to drug development include high financial costs, lengthy timelines for clinical trials, and regulatory hurdles. Approaches to reduce costs and timelines include greater use of electronic health records, simplifying clinical trial protocols, and utilizing decentralized clinical trial models.
The document summarizes news from the medical device industry. Specifically, it discusses:
1) The FDA clearance of Lumendi's Dilumen endoscopic accessory, which uses balloons to help position an endoscope and treat colonic lesions. This represents a minimally invasive alternative to surgery.
2) Varian Medical Systems' plan to spin off its imaging components business to boost its valuation after a difficult year.
3) Continued growth in patent filings from China, including in medical devices, cementing its status as an innovation leader according to the World Intellectual Property Organization.
4) Blackfynn's neurology data platform that aims to advance research by aggregating data, with
2014 Overview of significant trends in the life sciences (Biotechnology, Pharmaceutical, Device and Diagnostics) industry with Big Data in the Life Sciences featured articles.
The synthetic cell created by J. Craig Venter Institute researchers represents an important milestone, though the cell is not truly synthetic from scratch. While synthesizing the Mycoplasma mycoides genome was successful, the researchers encountered challenges in booting up the synthetic genome in a host cell. The creation of synthetic life raises philosophical and ethical issues and shows that life can be explained mechanistically. Additionally, Santhera Pharmaceutical's drug idebenone failed a second pivotal trial for Friedreich's ataxia, causing its stock price to plunge.
This document provides a summary of recent news in the biotech industry. It discusses several studies and clinical trial results, including the successful creation of a bacterial cell controlled by a synthetic genome and Santhera Pharmaceutical's failure in a Phase III trial for idebenone to treat Friedreich's ataxia. It also reports on financing news, such as NeuroTherapeutics Pharma raising $43 million for epilepsy and pain drug development. In addition, it notes commentary from NIH Director Francis Collins on the importance of continued funding for NIH and organizations like the Cystic Fibrosis Foundation to derisk drug development.
What's In An Idea-Chanda-UofT Life Science CoachDebra A. Chanda
The document discusses challenges and opportunities in the medical innovation process. It notes that while regulatory approval and reimbursement processes can be slow, improvements are being made. Medical device innovation requires collaboration between physicians, engineers, and industry. The future includes more implanted diagnostics to better manage diseases and promote wellness. Overall, the field remains promising for new technologies despite barriers that can be further addressed.
This document provides an overview of the science and business of drug discovery and development. It discusses the histories of large pharmaceutical companies and biotech startups. The current business landscape is defined by mergers and acquisitions as companies strive for efficiency. The scientific landscape is constrained by a limited number of known drug targets. Future trends may include increased collaboration between industry and academia to improve productivity and develop pre-competitive standards.
Crossroads: U.S. Medical Device Regulation vs. Innovation
The U.S. medical device industry is at a regulatory and potentially economic crossroad as the FDA continues to refine its 510(k) regulatory submission requirements and guidelines. Medical device manufacturers have been urging the FDA and Congress to expedite new product review processes to spur innovation and bring new medical technologies to market faster. However, supporters of stricter FDA regulations claim that a faster regulatory review process causes unsafe devices to enter the market.
As a result of numerous exchanges between both sides of the issue, CDRH (FDA) recently issued multiple updates to its initiatives for the 510(k) approval process. To shed light on key changes, we obtained the support of the office of Dr. Jeffrey Shuren MD JD, Director of CDRH and Dr. John Smith MD JD, of Hogan Lovells, a prominent international law firm with a medical regulatory specialty, on their interpretations of the 510(k) regulatory guidelines and the impact these guidelines will have on medical device manufacturers.
Listen to the Dr. John Smith podcast interview here:
http://youtu.be/iHVpwwXi7dY
The MarkeTech Group
502 Mace Blvd.
Davis, CA 95618
http://www.themarketechgroup.com
John Ludlow presented on regenerative medicine past, present, and future. He discussed early work in organ transplantation and tissue engineering. Currently, there are over 50 companies developing regenerative medicine products and the field is starting to provide commercialized solutions. However, the path has not been straightforward. Moving forward, success may come from focusing on clinical and commercial viability, strategic intellectual property management, and iterative technology evolution driven by clinical needs. The future of regenerative medicine may include more personalized and rapid regenerative options.
1) Maintaining US leadership in medical innovation requires consistent application of policies that incentivize innovation, such as tax breaks for research and strong intellectual property protections.
2) Collaboration between government, private industry, and academia can speed up the discovery process and maximize potential by bringing together diverse expertise and resources.
3) The pharmaceutical industry has a responsibility to safely develop and validate new treatments while also taking risks to pursue new cures, but government policies must continue fostering an environment where innovation can thrive.
Introduction to Regulatory Affairs - Pauwels Consulting AcademyPauwels Consulting
On Tuesday, June 14, our colleagues Fiorenzo Savoretti, Senior Regulatory and Quality Consultant at Pfizer and Nick Deschacht, Senior RA Consultant at GSK, gave an interesting “Introduction to Regulatory Affairs”.
Fiorenzo and Nick talked about RA and their projects, each from their unique angle. They delivered their presentations for ## attendees at our Brussels office at the Lambroekstraat 5a in Diegem.
This is part of the MaRS BioEntrepreneurship series.
Speaker: Lynne Zydowsky, Ph.D., Managing Principal Zydowsky Consultants
* Explore the development of regulated drugs and devices
* Understand where and how value is generated in the pharmaceuticals industry
* Appreciate the interplay between science and business in a biotech company
To download a copy of the audio for this presentation, please go to:
http://www.marsdd.com/bioent/oct16
For the event blog and Q+A, please see:
http://blog.marsdd.com/2006/10/17/bringing-together-art-and-science/
Celgene launched its drug Revlimid in China in June 2013, becoming one of the first Western biotechs to enter China independently without a local partner. While unusual, analysts believe Celgene's move could succeed due to preparations made by other companies like Roche. Roche worked with Chinese insurers to increase reimbursement for cancer drugs, which may help Revlimid. However, Revlimid's high cost could limit initial usage to private pay patients until broader reimbursement is established. Celgene aims to expand Revlimid's global sales, including in China where the multiple myeloma market is growing rapidly.
The document discusses the need for a new paradigm for funding and conducting biotech research and development (R&D) given constraints in the current financing environment. It proposes a model called Holistic Open Learning Networks (HOLNets) that would bring together diverse participants like healthcare providers, patient groups, data analytics firms, and social media networks. HOLNets could fundamentally change R&D by encouraging data sharing, allowing researchers to learn from each other in real time, and taking advantage of the shift in healthcare to outcomes-focused and data-driven models.
The document discusses a proposed non-profit approach to developing ibogaine into an FDA-approved medication for treating drug abuse. Key points include: (1) It would cost an estimated $5 million over 5 years to fund the necessary clinical research; (2) A non-profit is the best strategy given ibogaine's status as a Schedule I drug; (3) The FDA can be trusted to evaluate research based on science rather than politics and has approved studies of other Schedule I drugs.
Johnson & Johnson is a large healthcare company with a history of 10% annual earnings growth. To maintain growth, the company must create $4 billion in new business each year. William Weldon leads efforts to foster collaboration between business units and drive innovation through combining drugs, devices, and diagnostics. However, maintaining growth is challenging as some major drugs face competition and acquisitions are difficult to integrate. Weldon works to improve cooperation and create breakthrough innovations to sustain the company's success.
J & J Solutions has developed a closed system transfer device for safer handling of chemotherapy drugs. Their device eliminates risks of contamination, standardizes compounding to reduce errors, improves workflow efficiency, and reduces costs. They are seeking funding to complete product development including alpha and beta testing, obtain FDA approval, and launch the product to address a $1 billion market that is growing due to increasing cancer rates.
The pharmaceutical industry faced a challenging period from 2008-2012 due to numerous major drug patents expiring, resulting in $30 billion in lost annual brand drug revenue. Another peak of patent expirations is projected for 2014-2015, equal to the prior period. Growth in overall drug spending declined 1% in 2012 but is predicted to rebound to 3-4% growth in 2014, driven by new drug approvals and healthcare reform, before slowing again as generics continue to capture a larger share of prescriptions. Demonstrating the value of new drugs through real-world evidence will be important for companies to sustain pricing levels in this challenging environment.
This document provides an overview of the business model for therapeutic product discovery and development companies. It discusses key aspects like securing intellectual property and identifying a niche market. It also examines case studies of MedImmune and Panacea, highlighting strengths like management teams and weaknesses such as inefficient leadership. The document notes the high costs and risks involved but also the potential rewards, making it an attractive field for investors.
The process of drug discovery and development is a multifaceted journey, marked by innovation, scientific rigor, and regulatory scrutiny. This article provides a comprehensive overview of the intricate stages involved in bringing a new therapeutic entity from conception to market. Beginning with target identification and validation, the review navigates through lead discovery, optimization, and preclinical studies, emphasizing the pivotal role of molecular biology, high-throughput screening, and computational methods in shaping the drug development landscape. The integration of pharmacokinetics, pharmacodynamics, and toxicology assessments during preclinical phases ensures a robust foundation for advancing candidates into clinical trials. The complexities of Phase I-III clinical trials, including patient recruitment, safety monitoring, and efficacy evaluation, are dissected to underscore the meticulous processes that define the clinical development stage. Regulatory interactions, submission of New Drug Applications (NDAs), and post-marketing surveillance constitute the final regulatory phases, ensuring both efficacy and safety in real-world settings. The article also explores contemporary trends, such as precision medicine and the incorporation of artificial intelligence, that are reshaping traditional drug development paradigms. Challenges, including high attrition rates and escalating costs, are addressed alongside innovative strategies aimed at mitigating these hurdles. As the pharmaceutical landscape evolves, a nuanced understanding of the drug discovery and development process is imperative for fostering innovation and delivering transformative therapies to patients.
The document discusses Signals' approach to drug repositioning using big data. It introduces Signals and their product intelligence expertise. Their solution automatically produces and delivers business analytics by collecting, integrating and analyzing big data from open web sources. The presentation discusses the challenges in drug development, need for repositioning, and Signals' evidence-based data model and methodology for characterizing a drug and generating queries to identify novel opportunities for increasing its ROI by detecting similar drugs, modifications, conditions and genomic data.
Advanced Medical Isotope Corporation is developing a brachytherapy device called RadioGel for cancer treatment in humans and animals. It plans to generate near-term revenue from veterinary clinics and international licensing while pursuing FDA approval. RadioGel consists of yttrium-90 phosphate particles delivered via a hydrogel vehicle that solidifies in the body. It has the potential to treat multiple cancer types more effectively than existing therapies.
Advanced Medical Isotope Corporation is developing a brachytherapy device called RadioGel for cancer treatment in humans and animals. The company plans to generate near-term revenue from veterinary clinics and international licensing while pursuing FDA approval for human use. RadioGel consists of yttrium-90 phosphate particles delivered via a hydrogel that solidifies inside the body. The company expects to begin sales to veterinary clinics in early 2018 and obtain international licensing deals. It is working with national labs and universities to optimize the device and treatment techniques.
The synthetic cell created by J. Craig Venter Institute researchers represents an important milestone, though the cell is not truly synthetic from scratch. While synthesizing the Mycoplasma mycoides genome was successful, the researchers encountered challenges in booting up the synthetic genome in a host cell. The creation of synthetic life raises philosophical and ethical issues and shows that life can be explained mechanistically. Additionally, Santhera Pharmaceutical's drug idebenone failed a second pivotal trial for Friedreich's ataxia, causing its stock price to plunge.
This document provides a summary of recent news in the biotech industry. It discusses several studies and clinical trial results, including the successful creation of a bacterial cell controlled by a synthetic genome and Santhera Pharmaceutical's failure in a Phase III trial for idebenone to treat Friedreich's ataxia. It also reports on financing news, such as NeuroTherapeutics Pharma raising $43 million for epilepsy and pain drug development. In addition, it notes commentary from NIH Director Francis Collins on the importance of continued funding for NIH and organizations like the Cystic Fibrosis Foundation to derisk drug development.
What's In An Idea-Chanda-UofT Life Science CoachDebra A. Chanda
The document discusses challenges and opportunities in the medical innovation process. It notes that while regulatory approval and reimbursement processes can be slow, improvements are being made. Medical device innovation requires collaboration between physicians, engineers, and industry. The future includes more implanted diagnostics to better manage diseases and promote wellness. Overall, the field remains promising for new technologies despite barriers that can be further addressed.
This document provides an overview of the science and business of drug discovery and development. It discusses the histories of large pharmaceutical companies and biotech startups. The current business landscape is defined by mergers and acquisitions as companies strive for efficiency. The scientific landscape is constrained by a limited number of known drug targets. Future trends may include increased collaboration between industry and academia to improve productivity and develop pre-competitive standards.
Crossroads: U.S. Medical Device Regulation vs. Innovation
The U.S. medical device industry is at a regulatory and potentially economic crossroad as the FDA continues to refine its 510(k) regulatory submission requirements and guidelines. Medical device manufacturers have been urging the FDA and Congress to expedite new product review processes to spur innovation and bring new medical technologies to market faster. However, supporters of stricter FDA regulations claim that a faster regulatory review process causes unsafe devices to enter the market.
As a result of numerous exchanges between both sides of the issue, CDRH (FDA) recently issued multiple updates to its initiatives for the 510(k) approval process. To shed light on key changes, we obtained the support of the office of Dr. Jeffrey Shuren MD JD, Director of CDRH and Dr. John Smith MD JD, of Hogan Lovells, a prominent international law firm with a medical regulatory specialty, on their interpretations of the 510(k) regulatory guidelines and the impact these guidelines will have on medical device manufacturers.
Listen to the Dr. John Smith podcast interview here:
http://youtu.be/iHVpwwXi7dY
The MarkeTech Group
502 Mace Blvd.
Davis, CA 95618
http://www.themarketechgroup.com
John Ludlow presented on regenerative medicine past, present, and future. He discussed early work in organ transplantation and tissue engineering. Currently, there are over 50 companies developing regenerative medicine products and the field is starting to provide commercialized solutions. However, the path has not been straightforward. Moving forward, success may come from focusing on clinical and commercial viability, strategic intellectual property management, and iterative technology evolution driven by clinical needs. The future of regenerative medicine may include more personalized and rapid regenerative options.
1) Maintaining US leadership in medical innovation requires consistent application of policies that incentivize innovation, such as tax breaks for research and strong intellectual property protections.
2) Collaboration between government, private industry, and academia can speed up the discovery process and maximize potential by bringing together diverse expertise and resources.
3) The pharmaceutical industry has a responsibility to safely develop and validate new treatments while also taking risks to pursue new cures, but government policies must continue fostering an environment where innovation can thrive.
Introduction to Regulatory Affairs - Pauwels Consulting AcademyPauwels Consulting
On Tuesday, June 14, our colleagues Fiorenzo Savoretti, Senior Regulatory and Quality Consultant at Pfizer and Nick Deschacht, Senior RA Consultant at GSK, gave an interesting “Introduction to Regulatory Affairs”.
Fiorenzo and Nick talked about RA and their projects, each from their unique angle. They delivered their presentations for ## attendees at our Brussels office at the Lambroekstraat 5a in Diegem.
This is part of the MaRS BioEntrepreneurship series.
Speaker: Lynne Zydowsky, Ph.D., Managing Principal Zydowsky Consultants
* Explore the development of regulated drugs and devices
* Understand where and how value is generated in the pharmaceuticals industry
* Appreciate the interplay between science and business in a biotech company
To download a copy of the audio for this presentation, please go to:
http://www.marsdd.com/bioent/oct16
For the event blog and Q+A, please see:
http://blog.marsdd.com/2006/10/17/bringing-together-art-and-science/
Celgene launched its drug Revlimid in China in June 2013, becoming one of the first Western biotechs to enter China independently without a local partner. While unusual, analysts believe Celgene's move could succeed due to preparations made by other companies like Roche. Roche worked with Chinese insurers to increase reimbursement for cancer drugs, which may help Revlimid. However, Revlimid's high cost could limit initial usage to private pay patients until broader reimbursement is established. Celgene aims to expand Revlimid's global sales, including in China where the multiple myeloma market is growing rapidly.
The document discusses the need for a new paradigm for funding and conducting biotech research and development (R&D) given constraints in the current financing environment. It proposes a model called Holistic Open Learning Networks (HOLNets) that would bring together diverse participants like healthcare providers, patient groups, data analytics firms, and social media networks. HOLNets could fundamentally change R&D by encouraging data sharing, allowing researchers to learn from each other in real time, and taking advantage of the shift in healthcare to outcomes-focused and data-driven models.
The document discusses a proposed non-profit approach to developing ibogaine into an FDA-approved medication for treating drug abuse. Key points include: (1) It would cost an estimated $5 million over 5 years to fund the necessary clinical research; (2) A non-profit is the best strategy given ibogaine's status as a Schedule I drug; (3) The FDA can be trusted to evaluate research based on science rather than politics and has approved studies of other Schedule I drugs.
Johnson & Johnson is a large healthcare company with a history of 10% annual earnings growth. To maintain growth, the company must create $4 billion in new business each year. William Weldon leads efforts to foster collaboration between business units and drive innovation through combining drugs, devices, and diagnostics. However, maintaining growth is challenging as some major drugs face competition and acquisitions are difficult to integrate. Weldon works to improve cooperation and create breakthrough innovations to sustain the company's success.
J & J Solutions has developed a closed system transfer device for safer handling of chemotherapy drugs. Their device eliminates risks of contamination, standardizes compounding to reduce errors, improves workflow efficiency, and reduces costs. They are seeking funding to complete product development including alpha and beta testing, obtain FDA approval, and launch the product to address a $1 billion market that is growing due to increasing cancer rates.
The pharmaceutical industry faced a challenging period from 2008-2012 due to numerous major drug patents expiring, resulting in $30 billion in lost annual brand drug revenue. Another peak of patent expirations is projected for 2014-2015, equal to the prior period. Growth in overall drug spending declined 1% in 2012 but is predicted to rebound to 3-4% growth in 2014, driven by new drug approvals and healthcare reform, before slowing again as generics continue to capture a larger share of prescriptions. Demonstrating the value of new drugs through real-world evidence will be important for companies to sustain pricing levels in this challenging environment.
This document provides an overview of the business model for therapeutic product discovery and development companies. It discusses key aspects like securing intellectual property and identifying a niche market. It also examines case studies of MedImmune and Panacea, highlighting strengths like management teams and weaknesses such as inefficient leadership. The document notes the high costs and risks involved but also the potential rewards, making it an attractive field for investors.
The process of drug discovery and development is a multifaceted journey, marked by innovation, scientific rigor, and regulatory scrutiny. This article provides a comprehensive overview of the intricate stages involved in bringing a new therapeutic entity from conception to market. Beginning with target identification and validation, the review navigates through lead discovery, optimization, and preclinical studies, emphasizing the pivotal role of molecular biology, high-throughput screening, and computational methods in shaping the drug development landscape. The integration of pharmacokinetics, pharmacodynamics, and toxicology assessments during preclinical phases ensures a robust foundation for advancing candidates into clinical trials. The complexities of Phase I-III clinical trials, including patient recruitment, safety monitoring, and efficacy evaluation, are dissected to underscore the meticulous processes that define the clinical development stage. Regulatory interactions, submission of New Drug Applications (NDAs), and post-marketing surveillance constitute the final regulatory phases, ensuring both efficacy and safety in real-world settings. The article also explores contemporary trends, such as precision medicine and the incorporation of artificial intelligence, that are reshaping traditional drug development paradigms. Challenges, including high attrition rates and escalating costs, are addressed alongside innovative strategies aimed at mitigating these hurdles. As the pharmaceutical landscape evolves, a nuanced understanding of the drug discovery and development process is imperative for fostering innovation and delivering transformative therapies to patients.
The document discusses Signals' approach to drug repositioning using big data. It introduces Signals and their product intelligence expertise. Their solution automatically produces and delivers business analytics by collecting, integrating and analyzing big data from open web sources. The presentation discusses the challenges in drug development, need for repositioning, and Signals' evidence-based data model and methodology for characterizing a drug and generating queries to identify novel opportunities for increasing its ROI by detecting similar drugs, modifications, conditions and genomic data.
Advanced Medical Isotope Corporation is developing a brachytherapy device called RadioGel for cancer treatment in humans and animals. It plans to generate near-term revenue from veterinary clinics and international licensing while pursuing FDA approval. RadioGel consists of yttrium-90 phosphate particles delivered via a hydrogel vehicle that solidifies in the body. It has the potential to treat multiple cancer types more effectively than existing therapies.
Advanced Medical Isotope Corporation is developing a brachytherapy device called RadioGel for cancer treatment in humans and animals. The company plans to generate near-term revenue from veterinary clinics and international licensing while pursuing FDA approval for human use. RadioGel consists of yttrium-90 phosphate particles delivered via a hydrogel that solidifies inside the body. The company expects to begin sales to veterinary clinics in early 2018 and obtain international licensing deals. It is working with national labs and universities to optimize the device and treatment techniques.
1. Second chance; New technology, and a new leader,give a boost to ConjuChem Inc.'s fortunes
Canadian Business
Monday, September 29, 2003
Section: Technology
Byline: Sarah Staples
Oh, to have plunked a chunk of change down on ConjuChem Inc. lastwinter. Back then, the Montreal biotech
firm's future > was uncertain to say the least; its shares (TSX: CJC) were worth a measly 31ó, less than a quarter of
the company's cash on hand. Even two months ago, exhortations to invest in ConjuChem di dn'texactly endear
David Dean, a biotech analystwith Sprott Securities in Toronto, to buy-sidecustomers. "I had people callingme
retarded, phoningmy sales guys to tell them that," says Dean,who recently upgraded his pick froma "speculative
buy" to justplain "buy." Adds a vindicated Dean: "It was hell then, but I'm sittingpretty now."
And how. An investment of, say,$10,000 in ConjuChem shares in March was worth nearly $130,000 in early
September. Analysts arefrantically initiating--or reinitiating--coverageof the now-hot stock,which nearly tripled in
valueto about $4 duringthe pastmonth alone.And in this fall'ssizzlingfollow-on market, ConjuChem is rumored
to be closeto pullingthe trigger on yet another round of equity financing,its second in less than six months.
Dean, whose firmhas an investment-banking relationship with ConjuChem that includes shareownership,credits
the firm's spectacular reversal of fortune to its new interimleader, biotech industry veteran Jacques Lapointe
(above). "He would have had multipleopportunities to do whatever he wanted, and ConjuChem is what he's
chosen," says Dean. "I think it brings a ton of credibility to the name."
ConjuChem was founded in 1997 with the purchaseof blood-related intellectual property from San Francisco-
based RedCell Inc.The assets were improved and refined, and eventually yielded a new technology for designing
less toxic,longer-lastingversions of proven drugs. The technology--Drug Affinity Complex, or DAC--is deceptively
simple:scientists join drugmolecules to a "linker" molecule, and this to a chemical bond called a "reactive
chemistry." Insidethe human body, the reactive portion seeks out and attaches itself to albumin,a protein that
makes up as much as 75% of blood plasma and is found abundantly throughout tissueand fluids.The resultis a
triple-combination drugthat features almostthe same potency and desired pharmaceutical effects as the original,
plus the advantages of albumin:it is distributed more evenly and predictably throughout the body, and remains
there for days instead of hours.
That led to a nifty business proposition:ConjuChemwould rescue experimental -stage drugs written off as unsafe
or too costly to develop, redesign them and give them a second chanceat commercial success.Investors loved the
plan.They poured about $65 million into thecompany, including$25 million in grossproceeds from an IPO on the
Toronto Stock Exchange in November 2000.And they licked their lips when then-CEO Robert (Duffy) DuFresne
signed up tier-one research partners that included British pharmaceutical giantGlaxoSmithKline."Of the
companies that went public in the 2000 field,itwas the darlingof all of them," recalls Dean."Everybody had a
'buy' on ConjuChem back in those days."
But enthusiasmfizzled after the firsttwo "rescued" drugs failed dismally in clinical trials.DAC:TI, designed to treat
blood clots associated with kidney dialysis,was modeled on a Glaxo anticoagulantknown as argatroban (Novastan)
that in the original formlasted only 30 minutes in the body and had been blamed for provokinglife-threatening
hemorrhages. ConjuChem's DAC form of argatroban sailed through initial safety tests, but didn't work as itwas
designed to duringa later PhaseII/III clinical trial.Thesame was true of DAC:Opioid, a painkiller derived from a
natural opioid peptide,or protein fragment, dubbed Dynorphin A. Initial testingon humans proved that the drug,
for patients with shingles or post-surgical pain fromhysterectomy and knee replacement operations,remained in
the body 10,000 times longer than the natural protein.Ultimately, though, it proved ineffective in dullingpain.
After test results for the two drugs were made public in July 2002,DuFresne left the firm,and deals with Glaxo and
others were abandoned.ConjuChem's market cap quickly fell froma high of more than $215 million to about $10
million.Its shares,which had already been under pressuresincethe tech bubble burst,plummeted 85% in a
2. month, and stayed there. "The stock went 'no bid,'" says Dean."For months, nobody wanted to buy it." The board
responded by appointingan executive management committee consistingof Lapointe, chief financial officer
Lennie Ryer and scientific leader Dr.Jean-Paul Castaigne,but the arrangement foundered. With littleprospect of
attractinga decent CEO, Lapointe agreed to step in as an "active chairman"lastNovember.
A former president and chief operating officer of BioChem Pharma Inc.,Lapointe had planned to settle into
comfortable semiretirement consistingof charity work,angel investingand appointments on the boards of several
Canadian biotechs,amongthem ConjuChem--in which he had taken a "sizable"position in 2001,when the stock
was at $6.50. He had made a fortune on Shire PharmaceuticalsGroup's $5.6-billion acquisition of BioChem that
year, the largestsuch deal in Canadian biotech history.Before that, as CEO of Glaxo's Canadian and British
subsidiaries,hehad overseen a staff of 15,000.And before that, he'd spent nearly two decades at Johnson &
Johnson. "The intention was not to go back to full-time leadership of an organization,"he says.
Still,where the Street perceived only weakness and failure,Lapointe sawreasons for hope. Although ConjuChem's
drugs hadn't worked, he believed in the underlyingtechnology. And fortunately, the company had $30 million
worth of convertibledebentures, courtesy of a deal that had closed weeks before the clinical failures cameto light.
In a difficultmarket,"money in the bank was a luxury most biotech companies probably didn'thave at the time,"
says Lapointe.
The new leader immediately cut staff to conserve capital,trimmingoperatinglosses by one-third. But the main
problem, as he sawit, was a flawed corporate strategy: ConjuChem's business model called for itto boostthe
number of promisingexperimental drugs it could offer by findingpartners to help pay the enormous costof
research.Collaboratorsfunded early rounds of testing for a drug they wished to engineer, and could negotiate a
licenceto take the compound forward. It was a "breadth" approach that yielded many DAC drugs. Too many, in
fact.
"DAC was being combined with a lot of peptides in different diseaseareas,with the hope that the odds would play
in our favor," recallsLapointe."But this was at the expense of a 'depth' strategy, where you really understand the
technology and know how to manipulateit in order to achieve a certain result.Even in the lasteightor nine
months, we've considerably deepened our knowledge of what works and what doesn't with DAC."
Lapointe's team also continued developingone of the survivingcandidates,DAC:GLP-1, designed to lower aberrant
blood-sugar levels in patients with Type II diabetes. At first,batches had to be recalled after human testing proved
the drug's formulation was unstable.
But the formula was remixed and testing resumed a few months before Lapointe assumed full control as interim
president and CEO on July 15. He next revealed positivepreliminary results for the diabetes drug and landed $12
million in stopgap fundingthrough a privateplacement, followed by the releaseon Aug. 21 of more detailed da ta
from the PhaseI/II clinical trial.
The stock surged to $4.15 by month's end. And itcontinues to trade heavily in anticipation of the next major
milestone: a PhaseII clinical programfor up to 450 diabetic patients beginningin October. Indications arethat
second-round results,due in the firsthalf of 2004,will be positive,too. Whereas commercially availableforms of
insulin control diabetes by indiscriminately loweringtheamount of glucosein the blood, DAC:GLP-1, which is
based on a natural hormone, induces the pancreas to secrete insulin only when a patient's blood sugar is
abnormally high.The drug has so far proven to extend the hormone's positiveeffects from five minutes to as long
as a week. And if the results of DAC:GLP-1's animal testingcan be duplicated in humans,itwon't justcontrol
glucose;it may guide levels back to "normal," and possibly even reverse the courseof the disease.
Lapointe is betting a blockbuster partnership deal for the Canadian drugwith a major pharmaceutical c ompany
won't be far behind. But it's a critical timefor ConjuChem. Lapointe needs to take DAC:GLP-1 pastPhaseII if he's
going to continue buildingthe company's credibility.He'll stay on as CEO longenough to make a deal happen, he
says,without offering specifics.
3. Meantime, with a market cap of $140 million and shares roundingthe$4 mark, the company looks poised to
capitalizeon its second chance. If the Street is right,Lapointe himself has turned out to be justwhat the doctor
ordered.