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

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

Natural Born Cancer Killers

Emily BurkeCancer, CAR-T, FDA, Immunotherapy, The WEEKLY

Further Down the Cancer Treatment Road with CARs This past August, to much fanfare, the FDA approved the first chimeric antigen receptor (CAR) T-cell therapy for blood cancer. Called Kymriah (Novartis), it promises to revolutionize treatment by genetically altering a patient’s own cells to fight cancer. Less than eight weeks later, Kite Pharma, now a part of Gilead Sciences (Foster … Read More

Unpacking Digital Medicine

Emily BurkeCardiovascular Disease, Diabetes, FDA, Medical Device

CAN APPS PROGRAM BETTER HEALTH? Digital medicine is defined by the field’s pioneer Dr. Eric Tool of the Scripps Translational Science Institute (La Jolla, CA) as “the ability to digitize human beings, by a variety of means (sequencing, sensors, imaging, etc.), fully exploiting our digital infrastructure of ever-increasing bandwidth, connectivity, social networking, the Internet of all things, and health information systems.” This new field is changing the way … Read More

Decoding Your Genes

Emily BurkeBiotech Basics, Deoxyribonucleic Acid (DNA), Diagnostics, Drug Approvals, Easily Confused, FDA, Genetics, Genomics, Orphan Disease, Term of the Week, The WEEKLY

The Skinny On DNA Testing 23andMe (Mountain View, CA) recently found itself back in the limelight after the disease risk section of its mail-in DNA kit received an OK from the FDA. The Silicon Valley biotech had to halt sales of its direct-to-consumer genetics testing back in 2013 after regulatory officials grew concerned over marketing claims and the possibility of consumers misinterpreting the test results. 23andMe rebooted a limited part of … Read More

The PARP Race Is On

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, Easily Confused, FDA, Mechanism of Action, The WEEKLY

PARP1 INHIBITOR LINEUP PARP1 inhibitors are making a strong statement! Tesaro’s (Waltham, MA) just-approved Zejula has garnered predictions of blockbuster status. AstraZeneca’s (Cambridge, UK) Lynparza was the first PARP1 inhibitor to make it to market back in 2014, and their recent clinical trial results showed significant survival benefit in ovarian cancer. Clovis Oncology (Boulder, CO) achieved the second FDA approval of a PARP1 inhibitor with Rubraca in … 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

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 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

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

Pushing The Self-Destruct Button

Emily BurkeCancer, Drug Approvals, FDA, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

 Pushing the Self-Destruct Button Breakthrough drug Venclexta charged onto the marketplace three months early to battle chronic lymphocytic leukemia. AbbVie’s (North Chicago, IL) and Roche’s (Basel, Switzerland) new therapy gained a quick approval after 80% of patients in the 106-person clinical trial responded to the small molecule inhibitor. Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults, with approximately … Read More

Orphan Drugs Lead 2015 Approvals

Emily BurkeBiologics, Cardiovascular Disease, Drug Approvals, FDA, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel syndrome. According to FDA numbers: 19 were for orphan diseases that affect fewer than 200,000 patients per year in the US. … Read More

A Foodie’s First-In-Class FDA Approval

Emily BurkeFDA, GMO, The WEEKLY

GMOs Finally Swim Upstream AquaBounty’s genetically modified salmon will make it to your dinner table in less time than it took to toil through the FDA pipeline. Coined AquAdvantage Salmon, it is the first genetically modified animal available for human consumption thanks to an approval last month. The two year wait time to grow the first batch pales in comparison … Read More