At the Forefront of Drug Discovery and Development: Small to Midsize Biotech/Biopharma Companies are the Leaders of Innovation

By Peter Benton, COO and President of Worldwide,

small to midsize companies are leaders of innovation

Today, the innovators of drug discovery and development often are found in the smaller, biotech and biopharma companies around the world. In the past several years, more than 60% of new drugs approved originated at small and midsize companies – those outside the 30 largest pharmaceutical firms.

After having spent more than 16 years working in this industry – at both a large pharma company and in clinical research serving small to midsize biotech and biopharma companies – I continually think about innovation and why the smaller companies are driving innovation. Larger companies want and desire to be innovative, but in reality most (though not all) can be more risk-averse, and therefore less willing to take chances on an unlikely new drug. Everyone loves new molecular entities and new treatment areas, but the financial rewards are greater when risk is adjusted by expanding upon an existing therapy. There also is a lot more complexity in a larger organization – more people to convince, more hurdles to jump, and more competition for who has the next big idea. And there are more distractions – more bureaucracy to wade through, changing processes to manage, and larger teams to navigate. Generally, speed and innovation are inversely related to the size of the company; not by design or desire, but by the shear reality of organizational size and complexity.

Worldwide Clinical Trials: Because We Care

These are the same reasons that innovator companies choose Worldwide Clinical Trials as their clinical research organization (CRO). We have unmatched medical and scientific expertise, rigorous operational processes and commitment to quality that any size pharmaceutical company would expect for their most complex clinical trial, yet we are small enough to deliver the personalized service that every small company needs.

Our clients – big and small – have access to their entire Worldwide team, from the front-line clinical research associate (CRA), to the project manager, to the medical expert and all the way to me or any member of our senior management team. We are truly here for our customers when things go wrong and when they go right. As we like to say, and – more importantly – we mean from the bottom of our hearts: “We’re big enough to matter, and small enough to care.”

Our Clients Are Making News

Several of our small, innovator clients have been in the news lately, and we’re extremely proud to be working with them to advance their clinical research. Here are some news highlights from just the past six weeks:

  • A Worldwide customer since 2010, Bellerophon Therapeutics is currently investigating the INOpulse product candidate, a portable device that delivers inhaled nitric oxide to patients. This unique solution, combining a drug and device, may reduce blood pressure in the lungs and therefore reduce the strain on the right ventricle of the patient’s heart and allow blood circulation to become more normal. Bellerophon recently announced that it received acceptance from the U.S. Food and Drug Administration (FDA) for modifications proposed to its Phase 3 program for INOpulse in PAH. These protocol changes have the potential to make INOpulse available to patients approximately two years earlier than previously predicted, therefore reducing time to market and resulting in substantial clinical development cost savings for the company. We’re supporting Bellerophon Therapeutics with a two-year clinical development program for INOpulse, providing a broad range of services, including regulatory affairs, site monitoring, project management, safety and medical monitoring support, and trial master file services.
  • Renova Therapeutics is an exciting biotechnology company developing gene therapy treatments for congestive heart failure and type 2 diabetes. Following a successful End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for RT-100 last year, Renova selected Worldwide as its contract research organization for a Phase 3 study of RT-100 AC6 gene transfer for the treatment of patients with reduced ejection fraction heart failure. With our support, Renova Therapeutics will conduct a randomized, placebo-controlled, double-blind multicenter Phase 3 trial. Gene therapy holds tremendous promise for the treatment and management of a wide range of diseases, and we’re thrilled to be working with Renova on this ground-breaking, new heart failure treatment.
  • A clinical stage biopharmaceutical company focused on the global immunology market, Aurinia Pharmaceuticals, Inc. recently chose Worldwide as its CRO for the AURORA Phase 3 study of volcosporin for the treatment of active lupus nephritis (LN). The Worldwide team is eager to help Aurinia with the development of voclosporin, which has the potential to become the first FDA-approved treatment for LN. With Worldwide’s support, Aurinia will conduct a randomized, placebo-controlled, double-blind global 52-week trial in approximately 320 patients.

Worldwide: The Contract Research Organization for Innovators

At Worldwide, we work hard to stand out from the crowd of more than 1,300 CROs around the world. We are big enough to be in the top 15 contract research organizations, but behave as if each customer is our only customer. We focus on what we can do well and will never try to support a study if we aren’t best suited for the project. For our small, innovator clients in particular, Worldwide has demonstrated time and again that we can deliver both the personalized attention they crave and the broad range of clinical research expertise they need to advance their discoveries from clinical development to commercialization.