$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

The Multiple Myeloma Landscape

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

BLOOD CANCER: MULTIPLE MYELOMA Plasma cells are the antibody-producing cells of our immune system which happen to play a critical role in our defense against infections. In multiple myeloma, plasma cells begin to grow and divide in an uncontrolled manner, forming a cancerous mass known as a plasmacytoma. Marrow — which produces plasma — no longer functions in our defense, it simply takes … Read More

Putting The CAR-T Before The Horse

Emily BurkeThe WEEKLY

THE STORY BEHIND CAR-T The hottest cancer therapy in the pipeline — chimeric antigen receptor therapy (CAR-T) — got a big boost last month when an FDA advisory panel unanimously recommended approval of the treatment for children and young adults with a severe form of leukemia who have run out of other options. Developed by Novartis (Basel, Switzerland), this elegant … Read More

Breast Cancer Subtypes

Emily BurkeBiotech Basics, Cancer, Genetics, Mechanism of Action

FOUR MOLECULAR VARIANTS EXPLAINED Hearing your doctor utter the words HER2-positive, HR-positive, triple-negative, or BRCA mutation can be devastating — even for the most resilient person. Simply put, breast cancer is a complex disease. A diagnosis can be derived from any combination of the factors listed above — or, none at all. The National Cancer Institute (Bethesda, MD) has outlined four molecular subtypes of the disease. … Read More

Epigenome: Writing, Reading & Erasing

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Small Molecule Drugs

FOUNDATIONS OF EPIGENETICS Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell growth and division, scientists better understand the molecular range of different cancers and consequently develop … 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 Mechanics Of Melanoma

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

Spectrum Of Therapies Melanoma accounts for less than 1% of skin cancer cases yet causes the vast majority of skin cancer deaths. If detected early enough, melanoma is almost always curable. If not, its ability to metastasize makes it difficult to treat. Melanoma is more common in young adults than many other types of cancer, with 25% of new cases occurring in people under age … Read More

Therapeutic Antibody Primer

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

Basics & Innovations Monoclonal antibody (mAb) therapeutics burst onto the healthcare scene 20 years ago, and today they remain one of the most versatile and effective therapeutics available. The tried and true mAbs are still in high demand, and we suspect this first wave of derivative products clamoring their way through the pipeline will be equally as successful. In this WEEKLY, we’ll … Read More

New Hope For Spinal Muscular Atrophy

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA). SMA robs people of their ability to walk, eat, and ultimately, breathe. In addition to Spinraza, there are 13 … Read More

Swallowing A Biologic Drug?

Emily BurkeBiologics, Biotech Basics, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

Surviving The Stomach Is Key Over the past two decades, biologic drugs — drugs composed of proteins produced by living cells — have become the safest, most effective top sellers within the pharmaceutical industry. Approved to treat a variety of diseases including rheumatoid arthritis, diabetes, multiple sclerosis, Crohn’s disease, and a whole range of cancers, these drugs include monoclonal antibody … Read More

The Top Tech Of 2016

Emily BurkeBiotech Basics, Cancer, Clinical Trials, Drug Targets, FDA, Mechanism of Action

CAR-T Primer The hottest cancer therapy in the pipeline — chimeric antigen receptor therapy (CAR-T) — continued to mature in 2016 with its first FDA approval for blood cancer patients set to arrive this year. What’s next in the world of CAR-T? A whole lot: Additional cancer and autoimmune disease indications in preclinical development. Added safety features. More affordable “off … Read More

The Molecular Cause Of Obesity

Emily BurkeClinical Trials, Diabetes, Drug Development, Drug Targets, Mechanism of Action, Term of the Week

Targeting Fat Obesity is considered one of the most pressing public health issues of the day. According to the Center for Disease Control, 37% of adults and 17% of children in the U.S. are obese. The latest drug interventions work by attempting to suppress food intake, which has proven beneficial for some. However, there is still a large unmet need … Read More

The Race To Beat SMA

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Zeroing In On The SMA Pipeline A decade ago, there was only one drug in development for a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA) — robbing people of their ability to walk, eat, and ultimately, breathe. Today, there are 14 therapies making their way through the clinic according to the patient advocacy group Cure SMA. The increase is … 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

Biotech In Space!

Emily BurkeBiologics, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, The WEEKLY

Experimenting In Lower Earth’s Orbit Drug discovery in space? If this sounds like a page from science fiction, think again. Leading pharmaceutical companies are now collaborating with NASA to run experiments on the International Space Station (ISS). The focus on bone density, protein structure, vaccine development, immune function, and aging brings a swath of potential to this fascinating endeavor. In … Read More

On A Tumor’s Turf

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs, The WEEKLY

TACKLING THE SPACE AROUND SOLID TUMORS Covering the science behind T-cell-based immunotherapies has been the name of the game for our past couple of issues. CAR-T and TCR therapies show significant promise in early phase blood cancer clinical trials, but what about solid tumors? Previously mentioned Juno Therapeutics’ (Seattle, WA) Armored CAR technology has declared war on tumors, and as you will read … Read More