$8.7 Billion SMA Drug Explained

Emily BurkeGene Therapy, Orphan Drugs, SMA

The Science Behind the Deal Earlier this week, news of the $8.7 billion acquisition of gene therapy company AveXis (Bannockburn, IL) by Novartis (Basel, Switzerland) made big biotech headlines. AveXis’ lead candidate, AVXS-101, is now in Phase III clinical studies for the treatment of spinal muscular atrophy (SMA). In this Weekly, we’ll take a look at the science behind these headlines by explaining exactly … Read More

Turning On Cellular Garbage Disposals

Emily BurkeDrug Discovery, Drug Targets, Proteasomes

Proteasomes to the Rescue Many drugs work by stopping overactive proteins that cause disease. The leukemia drug Gleevec, for example, is a small-molecule inhibitor (antagonist) of the protein Bcr-Abl, whose over-activity promotes excessive cell division. Humira treats a range of autoimmune diseases by stopping TNF-alpha, a protein that activates inflammation. Such antagonists can be powerful. However, it’s not always possible … Read More

Cancer Diagnostic In A Drop

Emily BurkeDiagnostics, Liquid biopsy

THE LATEST IN CANCER DIAGNOSTICS Hearing the words “it might be cancer” paired with your doctor’s perplexed look is enough to send shock waves through your body. Getting to the heart of a diagnosis usually requires a surgical biopsy—removal and examination of the suspected tissue for visible signs of cancer. Less invasive diagnostic tests—called liquid biopsies—might just bring more choices … Read More

Exploring Different Strategies to Fight Alzheimer’s

Emily BurkeAlzheimer's Disease, Drug Development, Drug Discovery, Drug Targets

TAKE THAT, ALZHEIMER’S Alzheimer’s pernicious amyloid-beta plaques and tau tangles, discussed last week, remain important targets for the biotech industry. In the past few years, however, companies have begun to search more broadly for new treatments. This Weekly looks at products in development that use different strategies to fight this heartbreaking illness. REVIVING THE BRAIN? Loss of neurons is Alzheimer’s … Read More

Alzheimer’s Disease: A Tough Nut To Crack

Emily BurkeAlzheimer's Disease, The WEEKLY

AFFECTING 5.1 MILLION Alzheimer’s disease (AD) ranks as one of the toughest nuts to crack within drug discovery and development. Current treatments merely manage symptoms, so finding a better solution becomes more and more urgent as the aging population grows. Approximately 70 percent of dementia cases are caused by AD. It is a neurodegenerative disorder— neurons progressively lose structure and … Read More

Meatless Meat: Biotech Burger Ain’t No Bean Patty

Emily BurkeGMO, Sustainability

MISSION IMPOSSIBLE: MEATLESS MEAT? Imagine biting into a juicy cheeseburger: the flavor, the texture, the smell. Now, imagine the cheeseburger meatless. Impossible? No. Impossible Foods, a Redwood City, CA-based company has used biotechnology to create a plant-based burger amazingly similar to the bovine original. In this edition of the Biotech Primer Weekly, we examine how they did it. UNCOVERING THE … Read More

Who’s Your Daddy? The Science of 23andMe

Emily BurkeDNA Ancestry

Hey—Check Out Those Genes! There’s an old saying, “If you don’t know where you’ve come from, you can’t know where you’re going.” We used to rely on paper birth certificates, marriage licenses and memory to help discover where we’ve come from; but paper gets damaged, people are fallible, and memories fade. Leave it to biotech to come up with a … Read More

From Drug Development to Approval: A Recap

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Discovery, Drug Targets, FDA

From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980’s, scientists took a new tack in developing drugs. They adopted an approach known as rational drug discovery. Using this methodology, researchers first … Read More

Market Access

Linda LanderDrug Approvals, Market Access, The WEEKLY

Market Access Primer  For the last few weeks we here at Biotech Primer have tracked the progression of a drug candidate from the lab to the marketplace, where only the fittest survive. Winning at clinical trials means earning an official regulatory approval. Congratulations! But as any seasoned drug developer will tell you, the game has only just begun. Ensuring newly-approved … Read More

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 III

Emily BurkeAuthor Emily Burke PhD, Drug Approvals, Drug Development, FDA, The WEEKLY

Phase III Is No Guarantee Our last Biotech Primer WEEKLY explored the riskiest part of the human clinical trials pathway: Phase II. About 70% of drugs that enter Phase II never make it out. Most often, it’s because they fail to demonstrate effectiveness. Even making it to Phase III is no guarantee of success – about 40% of drugs fizzle … 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

Gene Therapy Cures

Emily BurkeBiologics, Gene Therapy, Genetics, Term of the Week

The Promise of Gene Therapy Unfolds In many ways, 2017 was the year of gene therapy in the United States. Patients and pharmaceutical companies celebrated the approval of not one, but three treatments for otherwise untreatable health conditions. Researchers have been working on developing safe, effective gene therapies for three decades. Early trials were plagued with safety issues. Consequently, the … Read More

Drug Discovery 201

Emily BurkeDrug Development, Drug Discovery, Drug Targets

WE WANNA NEW DRUG “One that won’t make me sick/ One that won’t make me crash my car/ and make me feel three feet thick…” Huey Lewis is singing about love, but he voices very human concerns when it comes to the medicines that heal bodies and minds. Last time, the Weekly explored how researchers identify drug targets—the molecules in … Read More

Drug Discovery 101

Emily BurkeDrug Development, Drug Discovery, Drug Targets

On the Road to New Medicines For most of the 20th century, we discovered new drugs by trial and error. Scientists investigated countless unrelated compounds in animals to see which improved disease symptoms. For instance, in the 1950s and 60s, British scientists at Boots Laboratories tested hundreds of unrelated chemicals on guinea pigs searching for an alternative to aspirin for … Read More