From Drug Development To Approval: Phase IV

Emily BurkeClinical Trials, Cocktail Fodder, Drug Approvals, Drug Development, FDA, Orphan Drugs

Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of truth: does the study data support claims that the new drug is both safe … Read More

From Drug Development to Approval: Phase I/II

Emily BurkeClinical Trials, Cocktail Fodder, FDA, The WEEKLY

Phase I and II Clinical Trials Every drug in clinical use today, from the latest CAR-T treatment to older cholesterol-lowering statins, share one thing in common: they have all successfully navigated the rigorous clinical trials process. This is no small feat, as only ~10% of the drugs that enter Phase I testing successfully emerge as marketed products. Those few drugs … Read More

Drug Discovery 301

Emily BurkeBiologics, Biomanufacturing, Cocktail Fodder

DRUG DISCOVERY 301 Biotech Primer Weekly wrapped up last year by exploring the first two stages of drug discovery. We looked at how pharmaceutical companies identify drug targets, or the molecules (usually proteins) involved in an illness that an ‘as yet undeveloped drug’ will hopefully act on. Next, we examined how researchers develop those pharmaceutical candidates. Now we turn to … Read More

The Science Behind Opioid Addiction

Emily BurkeBusiness of Biotech, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, The WEEKLY

THE SCIENCE BEHIND OPIOIDS Concerns over the opioid epidemic continue to grow, with deaths from narcotic overdoses the leading cause of death in people under 50 last year. Nearly half of those deaths were attributable to prescription opioids. The directors of both the Center for Disease Control (Atlanta, GA) and the Food and Drug Administration (Silver Spring, MD) have called … Read More

DNA Vaccines Explained

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, The WEEKLY, Vaccine

MORE ON THE POWERFUL, ELEGANT SIMPLICITY OF VACCINES Last week, we overviewed vaccine development and manufacture, focusing on those that use whole pathogens to protect us from a disease. This week, we examine subunit and polysaccharide vaccines, which use different strategies to fight infection. We also take a brief look at the US Food and Drug Administration (FDA)’s vaccine approval … Read More

Vaccines: Powerful Simplicity

Emily BurkeBiologics, Biomanufacturing, Cocktail Fodder, The WEEKLY, Vaccine

Vaccines: Elegant, Powerful Simplicity Anyone who’s suffered the aches and fever of influenza has good reason to value the simple flu shot. In fact, millions roll up their sleeves and literally take their medicine. The US Centers for Disease Control (CDC) (Atlanta, GA) estimates that about 146 million doses of influenza vaccine went to doctors’ offices, health departments, and the … Read More

From Fantasy To Reality: Xenotransplantation

Emily BurkeCocktail Fodder, Deoxyribonucleic Acid (DNA), Genomics

TRANSPLANTING ORGANS FROM ANIMALS INTO HUMANS Every ten minutes, a new person is added to the national transplant waiting list. A little more than 75,000 people are active waiting list candidates — meaning they are medically eligible for transplantation according to the Organ Procurement and Transplantation Network. Over the past decade, the gap between organ supply and demand has continued to grow; … Read More

Stopping A Big Problem: Blood Clots

Emily BurkeCocktail Fodder, Drug Approvals, Drug Targets, Mechanism of Action, Small Molecule Drugs

MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW? The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart: “venous” means relating … Read More

The Intrigue Of HIF

Emily BurkeCancer, Clinical Trials, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, Term of the Week, The WEEKLY

The Versatility Of HIFs Quite a few headlines touting a term called “HIF compound” or “hypoxia-inducible factor compound” have intrigued us here at WEEKLY headquarters. GlaxoSmithKline (London, England), Akebia Therapeutics (Cambridge, MA) and more have HIF-inducing drugs in the pipeline which may prove to be attractive alternatives to Amgen’s (Thousand Oaks, CA) injectable Epogen currently on the market. In earlier stages of research, HIFs are being studied for their connection to tumor … Read More

The Science Behind The Opioid Epidemic

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

A Big Pain The opioid addiction crisis gained attention at the highest levels of U.S. policy circles this past year. Presidential candidates that disagreed on nearly everything else vowed to make fighting the epidemic a priority if elected. In July, the U.S. Senate overwhelmingly approved a bill to strengthen prevention, treatment, and recovery efforts. And no wonder – according to … Read More

The First Three-Parent Baby

Emily BurkeCocktail Fodder, Deoxyribonucleic Acid (DNA), FDA

BREAKING DOWN THE SCIENCE & SCRUTINY “World’s First Three-Parent Baby” made headlines in publications ranging from Nature to CNN last week. This human interest story is of a baby boy born in Mexico with genetic material from three different parents, achieved by a technique known as three-parent in vitro fertilization (TPIVF). In this issue, we will explain why TPIVF is used … Read More

DMD Makes The Cut

Emily BurkeAntisense, Biologics, Clinical Trials, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, FDA, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

The Science Behind Sarepta’s Hotly Debated Antisense Drug Sarepta’s (Cambridge, MA) Duchenne muscular dystrophy drug Exondys 51 crossed the finish line earlier this week, with a conditional stamp of approval by the FDA. This hotly debated regulatory result offers new hope for patients and families whose previous treatment options only managed the descent of the disease. Affecting one in approximately 3,500 … Read More

Breaking Bad With NSCLC & SCLC

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Cocktail Fodder, Drug Approvals, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

BREAKING BAD The hit TV series Breaking Bad features anti-hero Walter White, who starts out as a sympathetic character: a mild-mannered high school chemistry teacher with a nagging cough that turns out to be lung cancer. Money problems precipitated by costly treatments, poor insurance, and a modest salary push him to start cooking up meth to ensure the financial security … Read More

Attack Of The Migraine

Emily BurkeClinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

New Avenues For Migraine Therapy Migraines. One of the most convenient excuses to get out of work or dinner with your mother-in-law. More than just a headache, migraines often include symptoms like intense pain, nausea, dizziness, and extreme sensitivity to light or noise. They can last anywhere from a few hours to days on end. Episodes may be as frequent as several times a … Read More

An Inborn Error Of Metabolism

Emily BurkeBiologics, Clinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Orphan Disease, Orphan Drugs, Small Molecule Drugs

Diet Soda’s Ominous Warning “Phenylketonurics—contains phenylalanine” is listed on many diet sodas, including Diet Coke and Diet Pepsi. There is no elaboration, just an ominous warning in bold typeface. Since a few of us at BioTech Primer are big fans of diet soda, we wondered: Should we be worried? And what exactly is a phenylketonuric? A quick Google search reveals … Read More

The Medicine Machine

Emily BurkeCocktail Fodder, Drug Development, Drug Targets, Small Molecule Drugs, The WEEKLY

Modern Technology & Mother Nature For thousands of years, nature has been the best medicine cabinet around. Natural products are drugs derived from nature, typically plants or microbes, and have been especially useful in fighting cancer and infectious disease. Drugs from nature can be highly effective. Think Bristol-Myers Squibb’s (New York, NY) Taxol, the breast cancer drug derived from the sap … Read More