Roger L. Whiting PhD
Co-Founder and CEO of Altos Therapeutics, California, USA
Roger Whiting is Co-founder and CEO of Altos Therapeutics. Altos is a virtual company that is developing ATC-1906M for the treatment of gastroparesis. He was previously Co-founder and CSO of ROXRO Pharma, who developed SPRIX® utilizing a semi-virtual business model. Prior to ROXRO, he was the Head of the Neurobiology Business Unit of Roche Bioscience, a unique industry concept for developing drugs. Prior to the acquisition by Roche he was Syntex’s SVP and Head of Basic Research, North America. Dr Whiting received his B. Pharm. (Spec. Hons.) from Bradford University and his PhD from the University of Aston.


Virtual Bio-Pharmaceutical Companies and Clinical Development

Virtual bio-pharmaceutical companies (sometimes referred to as Micro Pharma) have become very popular in recent years as a way to address the depletion in Big Pharma’s pipelines.  The essential concept of these organizations is to utilize the expertise of external consultants and CROs while keeping in-house personnel to a minimum.  In addition to leading the company and securing funding the in-house personnel are responsible for choosing the consultants and coordinating their efforts.  The consultants are chosen with a view to not only their expertise but also to their interactive skills since they are responsible for directing the activities of, and working directly with the respective CROs.  

I will utilize examples from two virtual bio-pharmaceutical companies that I co-founded, ROXRO Pharma and Altos Therapeutics to demonstrate the clinical development of the medicinal agents, SPRIX and ATC-1906.  ROXRO Pharma shepherded SPRIX from Phase 1 through to NDA approval using various consultants and CROs and the lessons learned from that process influenced the plan for ATC-1906.  Altos Therapeutics plans to advance ATC-1906 through Phase 1 and possibly Proof of Concept before transferring to Big Pharma for later stage clinical studies.  The clinical development of both compounds will be summarized and the salient teachings, both positive and negative will be described.  

Big Pharma have publically stated that they will acquire up to half of the compounds that they develop from smaller companies so the virtual bio-pharmaceutical model is likely to continue for the foreseeable future.


ECG Assessment in Early Stage Clinical Trials can Replace the Thorough QT Study

All drugs with systemic exposure are expected to undergo a careful assessment of the potential to cause ECG changes. In December 2015, the revised ICH E14 guidance was endorsed in all regions and now allows ECG assessment using exposure response (ER) analysis applied to early stage clinical pharmacology studies, to replace the thorough QT (TQT) study. In the conventional TQT study, the effect of the drug is tested at each post-dosing timepoint and the analysis is therefore from a power perspective inefficient and often requires up to 80 subjects per dose group/arm. In contrast, ER analysis uses all data from all dose groups and timepoints and has therefore substantially better power to exclude small QT effects, i.e. an effect exceeding the threshold of concern, 10 ms.  

In First-in-Human studies, plasma concentrations of the drug often substantially exceed those of clinical relevance and these studies are therefore often the best opportunity to confidently exclude small QT effects with criteria tailored to ER analysis. A positive control is not required if a QT effect can be excluded at supratherapeutic plasma levels. This presentation will discuss parameters of importance for this new approach with supporting case studies.