1) The FDA has scheduled a VRBPAC panel review of Heplisav for November 14-15. A positive vote could lead to approval in Q1 2013.
2) Key issues to watch include unequivocal efficacy data, safety profile comparisons to current standard of care, and discussions of a rare autoimmune event seen in clinical trials.
3) The analyst maintains an Outperform rating and $9 price target, seeing Heplisav becoming the standard of care for hepatitis B vaccination if approved.
AMBS is a regenerative medicine company developing new treatments for CNS disorders and regenerative medicine. It has several programs and subsidiaries developing treatments for conditions like Parkinson's disease, burns, cancer, and retinal diseases. Its lead programs include Eltoprazine for Parkinson's disease levodopa-induced dyskinesia through its subsidiary Elto Pharma, and recombinant MANF protein for retinal conditions like retinitis pigmentosa through its subsidiary MANF Therapeutics. AMBS is currently trading at very low prices but has achieved much higher values in the past, and it believes further progress in its clinical programs could increase its valuation.
Transforming time for prostate cancer therapiesJames Hilbert
This document provides an overview of prostate cancer treatments and competitive landscape. Prostate cancer is the most commonly diagnosed cancer in men. Current standard of care includes local therapy for early stage, androgen deprivation therapy for advanced or recurrent disease, chemotherapy (e.g. Taxotere) for metastatic castration-resistant prostate cancer (mCRPC), and newer agents like Provenge, Zytiga, and Jevtana for mCRPC. The pipeline includes promising agents from Dendreon, Medivation, and OncoGenex that could change treatment paradigms and improve outcomes for mCRPC patients.
The development & approval of Novoeight, a case studyAllen Che
The FDA approved Novoeight, a recombinant Factor VIII treatment for hemophilia A, in October 2013 based on positive results from clinical trials. Novoeight was found to effectively control and prevent bleeding episodes in adults and children with hemophilia A. The approval process involved reviewing manufacturing, clinical data, and inspections to ensure safety and efficacy. This approval expands treatment options for hemophilia A patients and represents continued innovation in the development of safer recombinant clotting factor therapies.
The purpose of this review is to provide veterinarians with key facts and information relevant to serological testing of individual dogs and cats in the clinical setting. Specifically, this paper addresses the role of antibody testing for the core, vaccine-preventable diseases canine distemper virus, canine and feline parvovirus, and canine adenovirus.
Clinical and Pharmacoeconomic analysis was extensivley done on Eylea for its placement on a hypothetical health plans formulary for Macular Degeneration. Information was presented to a panel of pharmacy professionals in managed care and industry
Nemus Bioscience is developing novel cannabinoid-based therapeutics for global medical challenges. It has an exclusive relationship with the University of Mississippi to develop cannabinoid compounds and targets. Its lead product NB1111 is a prodrug of THC being developed for glaucoma, a large global market with high unmet need. Preclinical studies show NB1111 significantly reduces eye pressure and may protect optic nerve cells, outperforming THC. Nemus aims to become a leading developer of second and third generation cannabinoid medicines.
DiagnoCure develops and commercializes more reliable cancer diagnostic tests to better support clinicians and patients in making decisions about clinical interventions. The global cancer diagnostics market is projected to double to $25.7 billion in 5 years, driven by trends like an aging population and increased adoption of molecular testing. DiagnoCure has two revenue-generating products and is developing additional clinically relevant oncology tests in its pipeline, including tests for prostate, colorectal, and lung cancers.
New Oncology Trends ADCs, Bispecific Antibodies & CAR-T Cell.pdfDoriaFang
Scientists are turning their attention to more innovative therapeutic strategies, such as next-generation ADCs, bispecific antibodies and CAR-T cell therapies, etc. as cancer therapy.
AMBS is a regenerative medicine company developing new treatments for CNS disorders and regenerative medicine. It has several programs and subsidiaries developing treatments for conditions like Parkinson's disease, burns, cancer, and retinal diseases. Its lead programs include Eltoprazine for Parkinson's disease levodopa-induced dyskinesia through its subsidiary Elto Pharma, and recombinant MANF protein for retinal conditions like retinitis pigmentosa through its subsidiary MANF Therapeutics. AMBS is currently trading at very low prices but has achieved much higher values in the past, and it believes further progress in its clinical programs could increase its valuation.
Transforming time for prostate cancer therapiesJames Hilbert
This document provides an overview of prostate cancer treatments and competitive landscape. Prostate cancer is the most commonly diagnosed cancer in men. Current standard of care includes local therapy for early stage, androgen deprivation therapy for advanced or recurrent disease, chemotherapy (e.g. Taxotere) for metastatic castration-resistant prostate cancer (mCRPC), and newer agents like Provenge, Zytiga, and Jevtana for mCRPC. The pipeline includes promising agents from Dendreon, Medivation, and OncoGenex that could change treatment paradigms and improve outcomes for mCRPC patients.
The development & approval of Novoeight, a case studyAllen Che
The FDA approved Novoeight, a recombinant Factor VIII treatment for hemophilia A, in October 2013 based on positive results from clinical trials. Novoeight was found to effectively control and prevent bleeding episodes in adults and children with hemophilia A. The approval process involved reviewing manufacturing, clinical data, and inspections to ensure safety and efficacy. This approval expands treatment options for hemophilia A patients and represents continued innovation in the development of safer recombinant clotting factor therapies.
The purpose of this review is to provide veterinarians with key facts and information relevant to serological testing of individual dogs and cats in the clinical setting. Specifically, this paper addresses the role of antibody testing for the core, vaccine-preventable diseases canine distemper virus, canine and feline parvovirus, and canine adenovirus.
Clinical and Pharmacoeconomic analysis was extensivley done on Eylea for its placement on a hypothetical health plans formulary for Macular Degeneration. Information was presented to a panel of pharmacy professionals in managed care and industry
Nemus Bioscience is developing novel cannabinoid-based therapeutics for global medical challenges. It has an exclusive relationship with the University of Mississippi to develop cannabinoid compounds and targets. Its lead product NB1111 is a prodrug of THC being developed for glaucoma, a large global market with high unmet need. Preclinical studies show NB1111 significantly reduces eye pressure and may protect optic nerve cells, outperforming THC. Nemus aims to become a leading developer of second and third generation cannabinoid medicines.
DiagnoCure develops and commercializes more reliable cancer diagnostic tests to better support clinicians and patients in making decisions about clinical interventions. The global cancer diagnostics market is projected to double to $25.7 billion in 5 years, driven by trends like an aging population and increased adoption of molecular testing. DiagnoCure has two revenue-generating products and is developing additional clinically relevant oncology tests in its pipeline, including tests for prostate, colorectal, and lung cancers.
New Oncology Trends ADCs, Bispecific Antibodies & CAR-T Cell.pdfDoriaFang
Scientists are turning their attention to more innovative therapeutic strategies, such as next-generation ADCs, bispecific antibodies and CAR-T cell therapies, etc. as cancer therapy.
Targovax is developing two complementary and highly targeted approaches to cancer immunotherapy: a peptide-based targeted immunotherapy platform for patients with RAS-mutated cancers and a virus-based oncolytic immunotherapy platform based on engineered oncolytic viruses armed with potent immune-stimulating transgenes for patients with solid tumors.
Pre-ASCO Seminar: (Re)Defining Value in Cancer Care: Priorities for Patients, Providers, and Health Systems
Panel: International Experience with Health Technology Assessment (HTA) & Lessons for the United States,
Pharmacovigilance and Materiovigilance, Drugs and Cosmetics Actshashi sinha
Due to side effects of Medicines and Medical Devices increasing day by day it is important to monitor the Adverse Events arising out of use of Medicines and Medical Devices. The Pharmacovigilance and Materiovigilance monitors adverse events arising our of usage of Drugs and Medical Devices respectively. This chapter also deals with Drugs and Cosmetics Act 1940 and their important provisions.
Pharmacovigilance and Materiovigilance, Drugs and Cosmetics Act 1940shashi sinha
Pharmacovigilance and Materiovigilance monitors the adverse events arising out of use of Medicines and Medical Devices. This chapter also takes an overview of drugs and control act 1940. It is important to know about the side effects of medical devices and drugs and how to report the adverse events arising out of the usage of Drugs and Medical Devices
NanoViricides is developing nanomedicine-based drugs to treat various viral diseases like influenza, HIV, hepatitis C, and Ebola. The company has 9 drug candidates in development that have shown safety and efficacy in animal studies. NanoViricides' first drug to enter human trials will be FluCide for influenza, which completely protected animals against lethal viral exposure without toxicity.
- Positive phase 3 trial results for ARIAD Pharma's drug ridaforolimus in sarcoma, meeting the primary endpoint of increased progression-free survival.
- The drug is expected to be filed for regulatory approval by partner Merck by end of 2011 and could gain approval in 2012, providing milestones and royalty revenues.
- ARIAD is also conducting a pivotal phase 2 trial of its drug ponatinib in leukemia, with data expected in mid-2012 that could support approval in early 2013.
Bridging the Gap between Clinical Development and Patient Access with Real-Wo...Covance
Real World Evidence (RWE) is defined by the FDA as 'clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD).' RWD is the data collected from sources other than traditional clinical trials. **Disclaimer: This article was previously published. Sciformix is now a Covance company.
There are different models for determining the price of medicines, but value-based pricing that considers the outcomes a treatment provides relative to its cost is likely the most appropriate approach. However, current pricing and reimbursement systems face challenges in applying value-based pricing due to uncertainties in data, combination therapies, multi-indication drugs, and ensuring treatments are both cost-effective and affordable for healthcare systems. Fair pricing requires balancing incentives for pharmaceutical innovation with improving access and affordability for patients.
The analytical studies that demonstrate biosimilarity to the reference product form the foundation of a biosimilar's development and approval pathway. A biosimilar may directly use information from the reference product's package insert, but will not include clinical studies as these are not designed to independently establish safety and efficacy. The biosimilar label incorporates data from the reference product label but identifies information specific to the biosimilar such as its proprietary name and unique suffix.
Real World Evidence in the MedTech IndustryEMMAIntl
Real World Evidence (RWE) is playing an increasingly significant role in the medical device industry. The FDA has recently pushed for an increase in using RWE to support regulatory submissions, in an effort to promote public health. To define what constitutes RWE, we must first start with defining Real World Data (RWD)...
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Visit CANFinfo.com to learn more.
- The FDA's Vaccines and Related Biological Products Advisory Committee (AdCom) will meet on November 14-15, 2012 to review data for HEPLISAV, Dynavax's two-dose hepatitis B vaccine.
- The analyst expects a positive recommendation from the AdCom for HEPLISAV and ultimately FDA approval by the February 24, 2013 PDUFA date.
- The analyst maintains a "Buy" rating and $10 price target for Dynavax based on projected profitability in 2015 following regulatory approval and launch of HEPLISAV in early 2013.
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
Invitae is a genetic testing company that aims to bring genetic information into mainstream medical practice by making genetic testing affordable and accessible. The document discusses Invitae's mission, growth in 2015, and goals for 2016. Key points include:
- Invitae doubled its genetic test menu in 2015 to over 600 genes and aims to have over 1,000 genes by the end of 2016.
- Volume grew significantly in 2015 from 229 tests in Q1 to over 7,000 tests in Q4 as prices decreased and content expanded.
- Reimbursement contracts increased to include major insurers which will help drive further volume growth.
- The goal for 2016 is to continue expanding content, driving
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.
The document discusses efforts by the National Institutes of Health (NIH) and Food and Drug Administration (FDA) to advance personalized medicine through several initiatives:
1. Developing a more integrated pathway to connect target identification by researchers to drug approval to help fill the void of insufficient private sector interest in most new targets.
2. The TRND program will help accelerate development of drugs for rare and neglected diseases by funding preclinical development.
3. The FDA is developing standards to incorporate genetic information into drug and device development and using biomarkers to evaluate therapies through its Critical Path Initiative.
Novavax was recognized at the Clinton Global Initiative annual meeting for developing affordable methods of producing vaccines for avian and seasonal influenza using virus-like particles. Novavax's manufacturing process uses recombinant technology in insect cells and portable disposable equipment, allowing vaccines to be produced more quickly and at lower costs than traditional egg- or cell-based methods. Novavax has developed an H5N1 avian flu vaccine that has completed Phase II trials and shown to be safe, effective, and rapidly producible, and is also testing a seasonal flu vaccine in Phase II trials.
1706 ir deck full w_appendix v1_cmd_v12_netttargovax2017
The document discusses Targovax's TG01 peptide vaccine platform. TG01 primes the immune system to recognize and destroy cancer cells with RAS mutations through a cocktail of 7 peptides covering common RAS mutations. Earlier trials in pancreatic cancer showed encouraging median and 1-year survival rates compared to historical controls when TG01 was administered with GM-CSF adjuvant. Long-term survival data also correlated with immune responses detected following vaccination.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical-stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date.
Targovax is developing two complementary and highly targeted approaches to cancer immunotherapy: a peptide-based targeted immunotherapy platform for patients with RAS-mutated cancers and a virus-based oncolytic immunotherapy platform based on engineered oncolytic viruses armed with potent immune-stimulating transgenes for patients with solid tumors.
Pre-ASCO Seminar: (Re)Defining Value in Cancer Care: Priorities for Patients, Providers, and Health Systems
Panel: International Experience with Health Technology Assessment (HTA) & Lessons for the United States,
Pharmacovigilance and Materiovigilance, Drugs and Cosmetics Actshashi sinha
Due to side effects of Medicines and Medical Devices increasing day by day it is important to monitor the Adverse Events arising out of use of Medicines and Medical Devices. The Pharmacovigilance and Materiovigilance monitors adverse events arising our of usage of Drugs and Medical Devices respectively. This chapter also deals with Drugs and Cosmetics Act 1940 and their important provisions.
Pharmacovigilance and Materiovigilance, Drugs and Cosmetics Act 1940shashi sinha
Pharmacovigilance and Materiovigilance monitors the adverse events arising out of use of Medicines and Medical Devices. This chapter also takes an overview of drugs and control act 1940. It is important to know about the side effects of medical devices and drugs and how to report the adverse events arising out of the usage of Drugs and Medical Devices
NanoViricides is developing nanomedicine-based drugs to treat various viral diseases like influenza, HIV, hepatitis C, and Ebola. The company has 9 drug candidates in development that have shown safety and efficacy in animal studies. NanoViricides' first drug to enter human trials will be FluCide for influenza, which completely protected animals against lethal viral exposure without toxicity.
- Positive phase 3 trial results for ARIAD Pharma's drug ridaforolimus in sarcoma, meeting the primary endpoint of increased progression-free survival.
- The drug is expected to be filed for regulatory approval by partner Merck by end of 2011 and could gain approval in 2012, providing milestones and royalty revenues.
- ARIAD is also conducting a pivotal phase 2 trial of its drug ponatinib in leukemia, with data expected in mid-2012 that could support approval in early 2013.
Bridging the Gap between Clinical Development and Patient Access with Real-Wo...Covance
Real World Evidence (RWE) is defined by the FDA as 'clinical evidence regarding the usage and potential benefits or risks of medical products derived from analysis of Real World Data (RWD).' RWD is the data collected from sources other than traditional clinical trials. **Disclaimer: This article was previously published. Sciformix is now a Covance company.
There are different models for determining the price of medicines, but value-based pricing that considers the outcomes a treatment provides relative to its cost is likely the most appropriate approach. However, current pricing and reimbursement systems face challenges in applying value-based pricing due to uncertainties in data, combination therapies, multi-indication drugs, and ensuring treatments are both cost-effective and affordable for healthcare systems. Fair pricing requires balancing incentives for pharmaceutical innovation with improving access and affordability for patients.
The analytical studies that demonstrate biosimilarity to the reference product form the foundation of a biosimilar's development and approval pathway. A biosimilar may directly use information from the reference product's package insert, but will not include clinical studies as these are not designed to independently establish safety and efficacy. The biosimilar label incorporates data from the reference product label but identifies information specific to the biosimilar such as its proprietary name and unique suffix.
Real World Evidence in the MedTech IndustryEMMAIntl
Real World Evidence (RWE) is playing an increasingly significant role in the medical device industry. The FDA has recently pushed for an increase in using RWE to support regulatory submissions, in an effort to promote public health. To define what constitutes RWE, we must first start with defining Real World Data (RWD)...
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Visit CANFinfo.com to learn more.
- The FDA's Vaccines and Related Biological Products Advisory Committee (AdCom) will meet on November 14-15, 2012 to review data for HEPLISAV, Dynavax's two-dose hepatitis B vaccine.
- The analyst expects a positive recommendation from the AdCom for HEPLISAV and ultimately FDA approval by the February 24, 2013 PDUFA date.
- The analyst maintains a "Buy" rating and $10 price target for Dynavax based on projected profitability in 2015 following regulatory approval and launch of HEPLISAV in early 2013.
In a research report by Berdud, M., Drummond, M. and Towse, A. (2018), a reasonable price for an orphan drug was established based on the proposition that rates of returns from investments in developing orphan drugs should be no greater than the industry average (for all drugs). At the 2018 EuHEA conference held in Maastricht, The Netherlands, 11-14 July, Mikel showed (i) how the reasonable price should be established and (ii) how NICE's cost-effectiveness threshold should be adjusted to ensure a reasonable price for an orphan drug. In slides results are discussed and conclusions showed too.
Author(s) and affiliation(s): Mikel Berdud, PhD (OHE); Prof. Mike Drummond (University of York); Prof. Adrian Towse (OHE)
Conference/meeting: EuHEA 2018
Location: Maastricht, The Netherlands
Date: 12/07/2018
Invitae is a genetic testing company that aims to bring genetic information into mainstream medical practice by making genetic testing affordable and accessible. The document discusses Invitae's mission, growth in 2015, and goals for 2016. Key points include:
- Invitae doubled its genetic test menu in 2015 to over 600 genes and aims to have over 1,000 genes by the end of 2016.
- Volume grew significantly in 2015 from 229 tests in Q1 to over 7,000 tests in Q4 as prices decreased and content expanded.
- Reimbursement contracts increased to include major insurers which will help drive further volume growth.
- The goal for 2016 is to continue expanding content, driving
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.
The document discusses efforts by the National Institutes of Health (NIH) and Food and Drug Administration (FDA) to advance personalized medicine through several initiatives:
1. Developing a more integrated pathway to connect target identification by researchers to drug approval to help fill the void of insufficient private sector interest in most new targets.
2. The TRND program will help accelerate development of drugs for rare and neglected diseases by funding preclinical development.
3. The FDA is developing standards to incorporate genetic information into drug and device development and using biomarkers to evaluate therapies through its Critical Path Initiative.
Novavax was recognized at the Clinton Global Initiative annual meeting for developing affordable methods of producing vaccines for avian and seasonal influenza using virus-like particles. Novavax's manufacturing process uses recombinant technology in insect cells and portable disposable equipment, allowing vaccines to be produced more quickly and at lower costs than traditional egg- or cell-based methods. Novavax has developed an H5N1 avian flu vaccine that has completed Phase II trials and shown to be safe, effective, and rapidly producible, and is also testing a seasonal flu vaccine in Phase II trials.
1706 ir deck full w_appendix v1_cmd_v12_netttargovax2017
The document discusses Targovax's TG01 peptide vaccine platform. TG01 primes the immune system to recognize and destroy cancer cells with RAS mutations through a cocktail of 7 peptides covering common RAS mutations. Earlier trials in pancreatic cancer showed encouraging median and 1-year survival rates compared to historical controls when TG01 was administered with GM-CSF adjuvant. Long-term survival data also correlated with immune responses detected following vaccination.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical-stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Rheumatoid Arthritis and Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date.
Methanex is the world's largest producer and supplier of methanol. We create value through our leadership in the global production, marketing and delivery of methanol to customers. View our latest Investor Presentation for more details.
ZKsync airdrop of 3.6 billion ZK tokens is scheduled by ZKsync for next week.pdfSOFTTECHHUB
The world of blockchain and decentralized technologies is about to witness a groundbreaking event. ZKsync, the pioneering Ethereum Layer 2 network, has announced the highly anticipated airdrop of its native token, ZK. This move marks a significant milestone in the protocol's journey, empowering the community to take the reins and shape the future of this revolutionary ecosystem.
UnityNet World Environment Day Abraham Project 2024 Press ReleaseLHelferty
June 12, 2024 UnityNet International (#UNI) World Environment Day Abraham Project 2024 Press Release from Markham / Mississauga, Ontario in the, Greater Tkaronto Bioregion, Canada in the North American Great Lakes Watersheds of North America (Turtle Island).
Cleades Robinson, a respected leader in Philadelphia's police force, is known for his diplomatic and tactful approach, fostering a strong community rapport.
1. Equity Research
Healthcare | Biotechnology
Dynavax Technologies Corporation
August 28, 2012
Heplisav VRBPAC Panel Set for November 14-15; Key Issues to Watch; Stock Rating: Outperform
Reiterate Outperform Company Profile: Aggressive Growth
Price Target: $9.00
On Tuesday, August 28, before the markets opened, Dynavax announced that the
FDA has set November 14-15 as the date for a Vaccines and Related Biological Symbol: DVAX (NASDAQ)
Products Advisory Committee (VRBPAC) panel to discuss Heplisav’s approvability. Price: $3.89 (52-Wk.: $2-$5)
A favorable vote could lead to potential approval and launch in the United States in Market Value (mil.): $660
first quarter 2013. We continue to assign 90% probability to eventual approval and Fiscal Year End: December
a full adult label (18-70 years as opposed to 40-70 years). We note that Dynavax Long-Term EPS Growth Rate:
submitted Heplisav’s biological license application (BLA) in April, pursuing an indication Dividend/Yield: None
for immunization against infection caused by all known subtypes of hepatitis B virus in 2011A 2012E 2013E
adults aged 18 through 70. On June 26, Dynavax announced that the U.S. Food and Drug Estimates
Administration (FDA) accepted the BLA and assigned the BLA a Standard Review, EPS FY $-0.39 $-0.40 $-0.18
establishing a PDUFA (Prescription Drug User Fee Act) date of February 24, 2013. CY $-0.40 $-0.18
Sales (mil.) 22 9 74
We summarize the key issues to watch for in the VRBPAC panel review as follows. Valuation
FY P/E NM NM NM
• Efficacy should be unequivocal. Heplisav has been shown to be superior to the
CY P/E NM NM
current standard of care Engerix-B in all populations tested to date, so the
discussions on efficacy should be unequivocal.
Trading Data (FactSet)
• Safety will be the focus of discussion, in our opinion. To date, the safety profile of Shares Outstanding (mil.) 125
Heplisav has been shown to be comparable to Engerix-B. Integrated safety analysis Float (mil.) 112
demonstrated that serious adverse event (SAE) rates for Heplisav and Engerix-B are Average Daily Volume 1,805,454
2.7% and 3.7%, respectively. Integrated autoimmune event rates are 0.23% for
Financial Data (FactSet)
Heplisav and 0.35% for Engerix-B. To our knowledge, no single category of adverse
Long-Term Debt/Total Capital (MRQ) 0.0
events stands out at a higher incidence than others in Heplisav-vaccinated subjects.
Book Value Per Share (MRQ) 0.8
• Discussions might also focus on the one case of Wegener’s granulomatosis that put Enterprise Value (mil.) 475.9
EBITDA (TTM) -49.5
Heplisav on clinical hold during 2008-2009. In March 2008, the FDA placed a clinical
Enterprise Value/EBITDA (TTM) -9.6x
hold on Heplisav after occurrence of a single SAE of Wegener’s granulomatosis, a rare Return on Equity (TTM) -63.9
form of autoimmune vasculitis that stains positive for c-ANCA, in a Heplisav-vaccinated
subject. A few months later, a vasculitis SAE was confirmed in a subject in the Engerix- Two-Year Price Performance Chart
B arm as well, stained positive for p-ANCA, demonstrating a more-balanced incidence
of systemic vasculitis between Heplisav and Engerix-B (3:1 randomization favoring $5
Heplisav). During the clinical hold, Dynavax re-tested more than 9,000 sera from $4
subjects vaccinated with Heplisav, and none of the sera, except for the one Wegener’s $3
case, was positive for c- or p-ANCA. Further, no presence of ds-DNA antibodies
(another indicator of potential autoimmune events) was detected either. After removal
$2
of the clinical hold in September 2009, Dynavax launched two Phase III studies $1
involving over 3,000 subjects and a data safety monitoring board (DSMB) was put in $0
12/31/10 12/30/11
place to detect and adjudicate SAEs and autoimmune-related SAEs. Heplisav passed
Sources: FactSet, William Blair & Company
every review by the DSMB, and the final integrated safety data, as discussed above, estimates
shows numerically lower adverse event rates than Engerix-B. Thus, we believe the
VRBPC panel might come to the conclusion that Heplisav is not likely associated with
Wegener’s (statistically, the p value for this event is 1.00, meaning a random event).
Dynavax Technologies (Berkeley, California) is a biotechnology company that has developed a
diverse pipeline of toll-like receptor (TLR) drug candidates for the treatment of infectious,
respiratory, autoimmune, and inflammatory diseases.
Y. Katherine Xu, Ph.D. Filippo Petti
+1 212 237 2758 +1 212 237 2741
kxu@williamblair.com fpetti@williamblair.com
Please consult the last page of this report for all disclosures.
William Blair & Company, L.L.C. receives or seeks to receive compensation for investment banking services from Dynavax
Technologies Corporation. Investors should consider this report as a single factor in making an investment decision.
2. William Blair & Company, L.L.C.
• The mechanism of the novel adjuvant in Heplisav, a TLR9 agonist, might be discussed as well. Heplisav is the first
product containing the novel adjuvant TLR9 agonist that is up for approval. We note that GlaxoSmithKline’s (GSK
$45.79) Cervarix was the first vaccine approved in the United States that contained a novel adjuvant (a TLR4 agonist)
other than alum. The FDA approved Cervarix for preventing human papillomavirus (HPV) infection in October 2009,
two and a half years after the first submission of the BLA and one month after removing the clinical hold on Heplisav.
We understand the traditional cautious stance from the FDA on the safety profile of novel vaccine adjuvants, but
believe that should the Heplisav package demonstrate a satisfactory safety profile, it is highly likely that the FDA will
approve the vaccine, as it did Cervarix. We believe the FDA recognizes the high unmet need in the hyporesponsive
populations and desires to have these subjects adequately protected against potential HBV infection.
• The extent of the label: for adults over 40 or over 18. After the removal of the clinical hold, the FDA instructed that
further studies should be conducted in older adults who are less responsive to the currently licensed vaccines,
including adults over 40 years of age, and individuals with chronic kidney disease. In February 2012, the FDA agreed
in the pre-BLA meeting that Heplisav’s label could be expanded to include healthy adults between the age of 18 and
70, which is the full adult label. We believe the VRBPAC panel could discuss this topic as well and vote on it.
We maintain our Outperform rating and $9 price target. We continue to believe that if approved, Heplisav will become the
standard-of-care hepatitis B vaccine in six years after launch; we continue to assume 90% probability for Heplisav to reach the
market. We forecast peak sales of $330 million for Heplisav in 2018 for the CKD population and the HIV/HCV/liver disease
population, and Dynavax has a commercial organization of 150 people in total for the U.S. market. For the U.S. diabetic
opportunity, although Dynavax could cover this market itself, we continue to model a bell-shaped curve peaking at about $475
million in revenue, with 35% royalties (net of COGS) to Dynavax on total U.S. sales from a partner with an established diabetes
franchise, to be conservative. With such assumptions, we derive the fair value of the stock at $9 per share. We believe Dynavax
shares continue to represent a compelling risk/reward opportunity.
Risks to our Outperform thesis include regulatory risks and intellectual property risks associated with Dynavax’s lead
candidate Heplisav, as well as risks related to business development and clinical development activities.
2 | Y. Katherine Xu, Ph.D. +1 212 237 2758
3. William Blair & Company, L.L.C.
William Blair & Company, L.L.C. was a manager or co-manager of a public offering of equity securities for Dynavax Technologies Corporation
within the prior 12 months.
William Blair & Company, L.L.C. and its affiliates beneficially own or control (either directly or through its managed accounts) 1% or more of the
equity securities of Dynavax Technologies Corporation as of the end of the month ending not more than 40 days from the date herein.
William Blair & Company, L.L.C. is a market maker in the security of Dynavax Technologies Corporation and may have a long or short position.
Additional information is available upon request.
Dynavax Technologies Corp. (DVAX)
Current Rating: Outperform
Aug 27, 2009 - Aug 27, 2012 Previous Close: $3.71
6/2/11 - I-O
$5
11/14/11 - O - PT:$8
$4
11/18/11 - PT:$10
$3
5/9/12 - PT:$9
$2
6/15/11 - PT:$8
$1
11/3/11 - R - PT:$8
$0
12/31/09 12/31/10 12/30/11
Source: William Blair & Company, L.L.C. and FactSet Initiation, RI = Reinitiated, @ = Analyst Change PT = Price Target
Legend: I =
Current Rating Distribution (as of 07/31/12)
Coverage Universe Percent Inv. Banking Relationships* Percent
Outperform (Buy) 61 Outperform (Buy) 7
Market Perform (Hold) 34 Market Perform (Hold) 2
Underperform (Sell) 1 Underperform (Sell) 0
*Percentage of companies in each rating category that are investment banking clients, defined as companies for which William Blair has
received compensation for investment banking services within the past 12 months.
Y. Katherine Xu attests that 1) all of the views expressed in this research report accurately reflect his/her personal views about any and all of the
securities and companies covered by this report, and 2) no part of his/her compensation was, is, or will be related, directly or indirectly, to the
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3 | Y. Katherine Xu, Ph.D. +1 212 237 2758
4. William Blair & Company, L.L.C.
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4 | Y. Katherine Xu, Ph.D. +1 212 237 2758