Several drugs failed in clinical trials in 2017, including:
- Brontictuzumab and tarextuzumab, anticancer stem cell antibodies from OncoMed Pharmaceuticals, failed trials for colorectal and small-cell lung cancer respectively.
- CA4P from Mateon Therapeutics failed a phase II/III trial for ovarian cancer.
- Fovista from Opthotech and Novartis failed two phase III trials for wet age-related macular degeneration.
- JCAR015, a CAR-T cancer immunotherapy from Juno Therapeutics, was halted after patient deaths in a phase II trial for leukemia.
- Several other drugs like levosimendan
Preparing Project Managers for Biotechnology (Arthur Miodozeniec PhD) April 2004
Dr Mlodozeniec, passed away in 2006, he was wonderful professor, rigorously taught us many critical skills, on risk management. Here reverse and forward engineering XL 119. Google his obituary.
Fda approves besremi (ropeginterferon alfa 2b-njft) for treating polycythemia...DoriaFang
On 12 November 2021, the US Food and Drug Administration (FDA) has approved Besremi (ropeginterferon alfa-2b-njft), developed by Pharma Essentia, for the treatment of polycythemia vera, a blood disorder that causes overproduction of red blood cells. Besremi is the first FDA-approved drug for the treatment of polycythemia vera that can be taken by patients regardless of treatment history.
To compare filing process of NDA of different countries of India, US and Euro...Aakashdeep Raval
To compare filing process of NDA of different countries of India, US and Europe.
B) Preparation of global list documents of registration of IND and NDA as per USFDA and Europe.
Preparing Project Managers for Biotechnology (Arthur Miodozeniec PhD) April 2004
Dr Mlodozeniec, passed away in 2006, he was wonderful professor, rigorously taught us many critical skills, on risk management. Here reverse and forward engineering XL 119. Google his obituary.
Fda approves besremi (ropeginterferon alfa 2b-njft) for treating polycythemia...DoriaFang
On 12 November 2021, the US Food and Drug Administration (FDA) has approved Besremi (ropeginterferon alfa-2b-njft), developed by Pharma Essentia, for the treatment of polycythemia vera, a blood disorder that causes overproduction of red blood cells. Besremi is the first FDA-approved drug for the treatment of polycythemia vera that can be taken by patients regardless of treatment history.
To compare filing process of NDA of different countries of India, US and Euro...Aakashdeep Raval
To compare filing process of NDA of different countries of India, US and Europe.
B) Preparation of global list documents of registration of IND and NDA as per USFDA and Europe.
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.
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This seminar provided an understanding of modern pharmaceutical drug development – the different phases of drug development and insight into different jobs.
Peptide drug conjugates (pd cs) new generation of targeted cancer treatmentDoriaFang
As a new generation of targeted cancer treatment, the well-designed PDC not only retains the advantages of traditional drug delivery, but also increases the penetration of tumor drugs and reduces the toxicity to liver and kidney.
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.
2016-11-28 Mentlife seminar: Pharmaceutical Drug Development; An Overall Pers...MentLife
This seminar provided an understanding of modern pharmaceutical drug development – the different phases of drug development and insight into different jobs.
Peptide drug conjugates (pd cs) new generation of targeted cancer treatmentDoriaFang
As a new generation of targeted cancer treatment, the well-designed PDC not only retains the advantages of traditional drug delivery, but also increases the penetration of tumor drugs and reduces the toxicity to liver and kidney.
OneMedForum New York 2010 - Company Presentation. Access Pharmaceuticals, Inc., a emerging biopharmaceutical company that focuses on adding value to exciting product concepts in research by advancing those products through clinical development.
OxfordSM's pharma case studies - providing a call to actionOxfordSM
Brand teams have to be increasingly innovative when finding ways to prompt patients and physicians to intervene at the right time.
Campaigns such as GSK’s Greatest Season Ever for FLONASE®, implemented last year in the United States which made the decision to prepare for the allergy season easier for patients by linking the proactive purchase of the brand to the start of the baseball season.
Providing A Call To Action:
We find that examples from within and outside of healthcare can often prompt this innovation. They act as a way of bringing in new perspectives and allowing teams to explore new avenues and new ideas.
So, in the spirit of hoping this will prompt some new ideas in your brand team, here are our favourite case studies that speak to the need to provide a call to action.
The Importance of Biomarkers in Hematology/Oncology Drug Development - Presentation by Steven Fruchtman, Former Chief Medical Officer, Syndax Pharmaceuticals, at the marcus evans Evolution Summit 2014 held in Palm Beach, FL May 7-9
This presentation contains an overview of the scientific and business update provided by management during Critical Outcome Technologies' 2017 Annual General and Special Meeting of Shareholders on December 20, 2017.
SciTech Development pitch deck including company overview, proprietary technology, lead drug ST-001 nanoFenretinide, patents, addressable market sizes, competiton, key personnel, advisory board, drug product characteristics, fenretinide history, cancer indications and drug mechanism of action (MOA).
Summary of PROTAC Degraders in Clinical Trials.pdfDoriaFang
A major class of molecules that may enable such proteins to be modulated through TPD are known as proteolysis-targeting chimera (PROTAC) protein degraders. Here we talk about the summary of PROTAC degraders in clinical trials.
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Ethanol (CH3CH2OH), or beverage alcohol, is a two-carbon alcohol
that is rapidly distributed in the body and brain. Ethanol alters many
neurochemical systems and has rewarding and addictive properties. It
is the oldest recreational drug and likely contributes to more morbidity,
mortality, and public health costs than all illicit drugs combined. The
5th edition of the Diagnostic and Statistical Manual of Mental Disorders
(DSM-5) integrates alcohol abuse and alcohol dependence into a single
disorder called alcohol use disorder (AUD), with mild, moderate,
and severe subclassifications (American Psychiatric Association, 2013).
In the DSM-5, all types of substance abuse and dependence have been
combined into a single substance use disorder (SUD) on a continuum
from mild to severe. A diagnosis of AUD requires that at least two of
the 11 DSM-5 behaviors be present within a 12-month period (mild
AUD: 2–3 criteria; moderate AUD: 4–5 criteria; severe AUD: 6–11 criteria).
The four main behavioral effects of AUD are impaired control over
drinking, negative social consequences, risky use, and altered physiological
effects (tolerance, withdrawal). This chapter presents an overview
of the prevalence and harmful consequences of AUD in the U.S.,
the systemic nature of the disease, neurocircuitry and stages of AUD,
comorbidities, fetal alcohol spectrum disorders, genetic risk factors, and
pharmacotherapies for AUD.
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These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
Basavarajeeyam is an important text for ayurvedic physician belonging to andhra pradehs. It is a popular compendium in various parts of our country as well as in andhra pradesh. The content of the text was presented in sanskrit and telugu language (Bilingual). One of the most famous book in ayurvedic pharmaceutics and therapeutics. This book contains 25 chapters called as prakaranas. Many rasaoushadis were explained, pioneer of dhatu druti, nadi pareeksha, mutra pareeksha etc. Belongs to the period of 15-16 century. New diseases like upadamsha, phiranga rogas are explained.
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Title: Sense of Smell
Presenter: Dr. Faiza, Assistant Professor of Physiology
Qualifications:
MBBS (Best Graduate, AIMC Lahore)
FCPS Physiology
ICMT, CHPE, DHPE (STMU)
MPH (GC University, Faisalabad)
MBA (Virtual University of Pakistan)
Learning Objectives:
Describe the primary categories of smells and the concept of odor blindness.
Explain the structure and location of the olfactory membrane and mucosa, including the types and roles of cells involved in olfaction.
Describe the pathway and mechanisms of olfactory signal transmission from the olfactory receptors to the brain.
Illustrate the biochemical cascade triggered by odorant binding to olfactory receptors, including the role of G-proteins and second messengers in generating an action potential.
Identify different types of olfactory disorders such as anosmia, hyposmia, hyperosmia, and dysosmia, including their potential causes.
Key Topics:
Olfactory Genes:
3% of the human genome accounts for olfactory genes.
400 genes for odorant receptors.
Olfactory Membrane:
Located in the superior part of the nasal cavity.
Medially: Folds downward along the superior septum.
Laterally: Folds over the superior turbinate and upper surface of the middle turbinate.
Total surface area: 5-10 square centimeters.
Olfactory Mucosa:
Olfactory Cells: Bipolar nerve cells derived from the CNS (100 million), with 4-25 olfactory cilia per cell.
Sustentacular Cells: Produce mucus and maintain ionic and molecular environment.
Basal Cells: Replace worn-out olfactory cells with an average lifespan of 1-2 months.
Bowman’s Gland: Secretes mucus.
Stimulation of Olfactory Cells:
Odorant dissolves in mucus and attaches to receptors on olfactory cilia.
Involves a cascade effect through G-proteins and second messengers, leading to depolarization and action potential generation in the olfactory nerve.
Quality of a Good Odorant:
Small (3-20 Carbon atoms), volatile, water-soluble, and lipid-soluble.
Facilitated by odorant-binding proteins in mucus.
Membrane Potential and Action Potential:
Resting membrane potential: -55mV.
Action potential frequency in the olfactory nerve increases with odorant strength.
Adaptation Towards the Sense of Smell:
Rapid adaptation within the first second, with further slow adaptation.
Psychological adaptation greater than receptor adaptation, involving feedback inhibition from the central nervous system.
Primary Sensations of Smell:
Camphoraceous, Musky, Floral, Pepperminty, Ethereal, Pungent, Putrid.
Odor Detection Threshold:
Examples: Hydrogen sulfide (0.0005 ppm), Methyl-mercaptan (0.002 ppm).
Some toxic substances are odorless at lethal concentrations.
Characteristics of Smell:
Odor blindness for single substances due to lack of appropriate receptor protein.
Behavioral and emotional influences of smell.
Transmission of Olfactory Signals:
From olfactory cells to glomeruli in the olfactory bulb, involving lateral inhibition.
Primitive, less old, and new olfactory systems with different path
Flu Vaccine Alert in Bangalore Karnatakaaddon Scans
As flu season approaches, health officials in Bangalore, Karnataka, are urging residents to get their flu vaccinations. The seasonal flu, while common, can lead to severe health complications, particularly for vulnerable populations such as young children, the elderly, and those with underlying health conditions.
Dr. Vidisha Kumari, a leading epidemiologist in Bangalore, emphasizes the importance of getting vaccinated. "The flu vaccine is our best defense against the influenza virus. It not only protects individuals but also helps prevent the spread of the virus in our communities," he says.
This year, the flu season is expected to coincide with a potential increase in other respiratory illnesses. The Karnataka Health Department has launched an awareness campaign highlighting the significance of flu vaccinations. They have set up multiple vaccination centers across Bangalore, making it convenient for residents to receive their shots.
To encourage widespread vaccination, the government is also collaborating with local schools, workplaces, and community centers to facilitate vaccination drives. Special attention is being given to ensuring that the vaccine is accessible to all, including marginalized communities who may have limited access to healthcare.
Residents are reminded that the flu vaccine is safe and effective. Common side effects are mild and may include soreness at the injection site, mild fever, or muscle aches. These side effects are generally short-lived and far less severe than the flu itself.
Healthcare providers are also stressing the importance of continuing COVID-19 precautions. Wearing masks, practicing good hand hygiene, and maintaining social distancing are still crucial, especially in crowded places.
Protect yourself and your loved ones by getting vaccinated. Together, we can help keep Bangalore healthy and safe this flu season. For more information on vaccination centers and schedules, residents can visit the Karnataka Health Department’s official website or follow their social media pages.
Stay informed, stay safe, and get your flu shot today!
2. 1. BRONTICTUZUMAB (OMP-52M51)
• Sponsor: OncoMed Pharmaceuticals
• Indication: Colorectal cancer
• Type of drug: Anticancer stem cell antibody designed to bind the Notch1 receptor
• How drug failed: The company halted enrollment of patients in Phase Ib trial assessing brontictuzumab in
combination with trifluridine/tipiracil (Lonsurf®) in third-line colorectal cancer patients, saying that the
combination of brontictuzumab plus chemotherapy “was not tolerable in this patient population.”
Brontictuzumab was one of two drug candidates whose failures within two weeks prompted OncoMed to
eliminate half of its workforce.
3. 2. CA4P (COMBRETASTATINA4-PHOSPHATE,OR
FOSBRETABULIN)
• Sponsor: Mateon Therapeutics
• Indication: Platinum-resistant ovarian cancer
• Type of drug: Tubulin-binding vascular disrupting agent (VDA)
• How drug failed: Company eliminated 60% of its workforce and ended development of its lead
candidate after it missed its primary endpoint of progression-free survival (PFS) in the Phase II/III FOCUS
trial. FOCUS assessed CA4P in combination with Roche-owned Genentech’s Avastin®(bevacizumab) and
physician’s choice chemotherapy in patients with platinum-resistant ovarian cancer.
4. 3. FOVISTA (PEGPLERANIB)
• Sponsors: Opthotech and Novartis
• Indication: Wet age-related macular degeneration (AMD)
• Type of drug: Anti PDGF-B pegylated aptamer
• How drug failed: Fovista missed its primary endpoints in two Phase III trials, OPH1002 and OPH1003.
Both evaluated the safety and efficacy of 1.5 mg of Fovista in combination with the Roche
(Genentech)/Novartis-marketed drug vascular endothelial growth factor (VEGF) therapy Lucentis®,
compared to Lucentis alone. Following the trial failures, Ophthotech slashed its workforce by
approximately 80%. In July, Opthotech and Novartis revised their 2014 co-development agreement in
anticipation of a third Phase III trial failure—a failure announced the following month.
5. 4. JCAR015
• Sponsor: Juno Therapeutics
• Indication: Adults with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL)
• Type of drug: CD19-directed chimeric antigen receptor technology (CAR-T) cancer immunotherapy
• How drug failed: Juno halted development of JCAR015, its lead CAR-T cancer immunotherapy, and
terminated its Phase II ROCKET trial, nearly four months after the study was placed on its second clinical
hold following two additional patient deaths. The deaths brought to five the number of patients who
died from cerebral edema in the ROCKET study, on which the FDA imposed a partial clinical hold in July
2016, a hold that was lifted days later
6. 5. LEVOSIMENDAN
• Sponsor: Tenax Therapeutics
• Indication: Low cardiac output syndrome (LCOS)
• Type of drug: Calcium sensitizer
• How drug failed: Levosimendan missed its co-primary endpoints in the Phase III LEVO-CTS trial of
significant reductions in the dual endpoint of death or use of a mechanical assist device at 30 days, as
well as in the quad endpoint of death, myocardial infarction, need for dialysis, or use of a mechanical
assist device at 30 days. The failure caused a one-day 76% plunge in Tenax’s share price, from $1.95 to
46 cents. CEO John Kelley resigned in April, and Tenax began evaluating strategic alternatives that
include a merger or sale. In July, both the FDA and Health Canada determined that another trial would
be needed to support a future NDA filing for levosimendan. “We are currently evaluating clinical,
regulatory, and financial options,” the company disclosed in its Form 10-Q for Q3 2017, filed in
November.
7. 6. PROPHAGE G-200 (HEAT SHOCK PROTEIN PEPTIDE
COMPLEX-96, OR HSPPC-96)
• Sponsor: Agenus
• Indication: Glioblastoma
• Type of drug: Vaccine
• How drug failed: Prophage G-200 failed a Phase II trial assessing the vaccine in combination with the
Roche/Genentech marketed cancer drug Avastin® (bevacizumab) in patients with surgically resectable,
recurrent glioblastoma, the company acknowledged in a February regulatory filing. A month later,
Agenus announced a reorganization designed to refocus the company on clinical development of
another vaccine program and two checkpoint inhibitor antibodies. The reorganization included
the elimination of 50 jobs and the shutdown of a facility in Basel, Switzerland
8. 7. SBC-103 (RHNAGLU ENZYME)
• Sponsor: Alexion Pharmaceuticals
• Indication: Mucopolysaccharidosis (MPS) IIIB
• Type of drug: Enzyme replacement therapy
• How drug failed: In its Form 10-Q report for the third quarter filed in October, Alexion disclosed that it
discontinued SBC-103 development in July based on results of a Phase I/II dose escalation study, and
recorded a $31 million impairment charge. The disclosure came a month after the company announced
it would eliminate approximately 20% of its workforce and move its headquarters from New Haven, CT,
to Boston, MA, by mid-2018 in a restructuring that followed Ludwig Hantson, Ph.D., becoming CEO in
March. Earlier that month, the company said it was eliminating 7% of its workforce. Alexion inherited
SBC-103 in 2015 when it acquired Synageva for $8.4 billion. But in February, the company signaled a
retreat from SBC-103 by reducing its investment in the candidate and recognizing an $85 million
impairment charge against Q4 2016 earnings.
9. 8. SERELAXIN (RLX030)
• Sponsor: Novartis
• Indication: Acute heart failure
• Type of drug: Relaxin receptor agonist, a recombinant form of the human relaxin-2 hormone
• How drug failed: Serelaxin missed its primary endpoints in the Phase III RELAX-AHF-2 trial of a reduction in
cardiovascular death through day 180, or a reduction in worsening heart failure through day 5, Novartis
acknowledged in March. The following month, Novartis took a $200 million charge against first-quarter
earnings related to serelaxin, which the company cited as one of several factors in a 17% year-over-year
decline in Q1 net income.
10. • Sponsors: OncoMed Pharmaceuticals and GlaxoSmithKline (GSK)
• Indication: Small-cell lung cancer (SCLC).
• Type of drug: Anticancer stem cell antibody designed to prevent signaling through both the Notch2 and
Notch3 receptors.
• How drug failed: Tarextumab plus chemotherapy failed to meet both its primary endpoint of
progression-free survival (PFS) and secondary endpoints in the Phase II PINNACLE study. The placebo-
controlled trial combined tarextumab with etoposide plus either cisplatin or carboplatin in patients with
previously untreated extensive-stage SCLC. Tarextumab was one of two drug candidates whose failures
within two weeks prompted OncoMed to eliminate half of its workforce.
9.TAREXTUMAB (OMP-59R5)
11. 10.TIRASEMTIV
• Sponsor: Cytokinetics
• Indication: Amyotrophic lateral sclerosis (ALS)
• Type of drug: Fast skeletal muscle troponin activator
• How drug failed: Cytokinetics halted development of its lead candidate tirasemtiv in November after it
missed its primary endpoint in the Phase III VITALITY-ALS trial. The company said that tirasemtiv failed
to show change from baseline in slow vital capacity (SVC) following 24 weeks of double-blind, placebo-
controlled treatment, then followed up in December with details. “We will seek advice from the
academic leadership of VITALITY-ALS and its clinical investigators, regulatory authorities and other
consultants before making decisions about continuing treatment with tirasemtiv in VIGOR-ALS,” an
open-label extension study, Cytokinetics states on its website.
12. 11.TOZADENANT
• Sponsor: Acorda Therapeutics
• Indication: Parkinson’s disease
• Type of drug: Oral adenosine A2a receptor antagonist developed as an adjunctive treatment to
levodopa in Parkinson’s disease patients to reduce OFF time
• How drug failed: Seven patients treated with tozadenant developed sepsis—five of whom died. As a
result, Acorda on November 15 paused enrollment of new patients in its Phase III studies. Five days
later, Acorda terminated development of tozadenant. Of the seven sepsis cases, four were associated
with agranulocytosis, two had no white blood cell counts available at the time of the event, and one
had a high white blood cell count.
13. 12.TRABODENOSON
• Sponsor: Inotek Pharmaceuticals
• Indication: Glaucoma
• Type of drug: First-in-class, highly selective adenosine mimetic targeting the A1 subreceptor
• How drug failed: Inotek acknowledged in January that its lead candidate trabodenoson missed its
primary endpoint in the Phase III MATrX-1 trial of superiority to placebo in reduction of intraocular
pressure (IOP) at all 12 of the study’s time points. The failure sparked a selloff that sent the share price
plunging 64% that morning, to $2.20 in premarket trading. Six months later in July, Inotek halted
development of trabodenoson after it failed a Phase II fixed-dose combination trial in combination with
latanoprost, also in glaucoma, by showing no meaningful clinical advantage in IOP reduction at Day 56,
after 4 additional weeks of treatment and night-time dosing. Inotek responded by exploring strategic
alternatives, with relatively quick success. In September, the company announced a merger agreement
with gene therapy developer Rocket Pharmaceuticals, a deal that is expected to close in the first quarter
of 2018.
14. 13.VADASTUXIMABTALIRINE (SGN-CD33A)
• Sponsor: Seattle Genetics
• Indications: Frontline older acute myeloid leukemia (AML); frontline high-risk myelodysplastic syndrome
(MDS)
• Type of drug: Antibody-drug conjugate (ADC) targeting CD33 protein
• How drug failed: Company terminated its Phase III CASCADE trial in AML after reviewing unblinded data
indicating a higher rate of deaths, including fatal infections, in the vadastuximab talirine arm compared
with the control arm. Seattle Genetics also halted enrollment in all other clinical trials assessing the
ADC. Two days later, the FDA placed the company’s IND on hold, barring clinical trials from resuming
until the agency lifts the clinical hold.