PRECISION DEVELOPMENT CONSULTING

Pharmaceutical Consultant Specializing in Novel Technologies

Unlocking Drug Development

Moving compounds from discovery into development and human clinical trials is a heavy lift. It requires carefully planning and execution of decisive preclinical proof-of-concept (POC) studies, understanding the regulatory requirements around preclinical toxicology testing, Chemistry, Manufacturing and Controls (CMC), thoughtful clinical trial design, and creating a complete regulatory application that can satisfy the regulatory agencies. These are all critical for the success of a development program. Furthermore, the selection and management of contract manufacturing organizations (CMOs) to source raw materials and provide drug substance and drug product manufacturing/testing activities and contract research organizations (CROs) to execute clinical and nonclinical studies under appropriate regulatory compliance are also complex, and often where virtual companies have the most difficulties.

Given the complexities and criticality in the success of a development program, executing on these components can frequently be barriers to the progress of a program. At Precision Development Consulting (PDC), our goal is to serve as a key resource for our clients to unlock the potential of exciting pharmaceutical development programs and progress them to human clinical trials as quickly as possible so they can serve patients in need.

Chemistry, Manufacturing and Controls

Regulatory requirements for CMC are complex, even for early human trials. Materials must be manufactured, tested, and stored according to current Good Manufacturing Practices (cGMP), and this means extensive development work well before regulatory submission. Drug substance and drug product characterization, formulation development, analytical methods development and validation, stability testing, and packaging selection are all necessary steps to a successful submission.

Preclinical Proof-of-Concept and Toxicology

Preclinical POC and toxicology studies are essential parts of drug development as they help to evaluate the potential therapeutic range, safety, and toxicity of a drug candidate before it is tested in human clinical trials. Regulators require extensive toxicology evaluations in at least two preclinical species, one rodent and one non-rodent, prior to clinical evaluation. Designing appropriate POC and toxicology studies, developing and properly validating bioanalytical assays, and performing adequate pharmacokinetic and pharmacodynamic evaluations can be challenging and resource intensive. Successful studies demand the contributions of skilled study directors, toxicologists, pathologists, veterinarians, surgeons, and regulatory specialists. It is critical to choose and effectively manage reputable Good Laboratory Practices (GLP) compliant CROs and understand applicable regulatory guidance to balance what is necessary to support your clinical trial designs, program milestones, and corporate value inflection points.

Clinical Trials

The complexities of clinical trial design and the associated costs of implementation can significantly hinder the progress of a development program. Trials must be safe, appropriately controlled to ensure the drug works as well as possible for its intended purpose, generate meaningful data for the program, and be completed under a finite budget. To support the trial, appropriate bioanalytical assays for pharmacokinetic and pharmacodynamic assessments must be established and validated. Appropriate biomarkers need to be identified and assays developed/validated. Critically, reputable and Good Clinical Practice (GCP) compliant CROs must be selected and effectively managed to ensure successful execution within the established budget and timeline.

Regulatory Affairs

Understanding the regulatory landscape is equally as important for a development program as any other component. All CMC, preclinical, and clinical trial information must be formally drafted into a specific format for submission to the appropriate regulatory agency. The mechanics under which regulatory interactions occur and applications of specialty programs (i.e., Orphan, Fast Track designations) are important to thoughtfully navigate to avoid fatal barriers of a program's progress.

Precision, Quality, and Accuracy: The Core of What We Do

PDC is a scientific and regulatory consulting firm dedicated to assisting emerging and established pharmaceutical companies with preclinical POC and toxicology, translational research, CMC, clinical, and regulatory challenges for novel technologies. We also provide assistance in the writing and defense of relevant sections of regulatory submissions. We have unique expertise in the development of oligonucleotide-based therapeutics.

We are intimately familiar with the challenges faced by start-up companies with respect to meeting development milestones, partnering issues, regulatory interactions and cash flow management. We work with companies on topics beyond the science, helping to manage development programs in a phase appropriate manner to better utilize limited resources while minimizing overall risk to the program. We are focused on maximizing high-quality output while reducing expense.

At PDC, we provide guidance and hands-on assistance with evaluating, selecting, and managing CMOs and CROs for nonclinical, clinical, drug substance, and drug product activities. We have experience working with most major and niche CMOs/CROs in North America, Europe, and Asia. Partnering with CMOs/CROs is not a turnkey activity – keeping CMOs/CROs on track technically and timewise requires active management and input in response to unexpected technical hurdles.

Experienced in the development of: Antisense oligonucleotides, Immunostimulatory oligonucleotides, siRNA (RNA duplexes), Oligonucleotide conjugates, and Oligonucleotides in complex dosage forms (intrathecal, topical, liposomes, nanoparticles).

Therapeutic area experience: Oncology, Dermatology, CNS, Ophthalmology, Intravenous, Subcutaneous, Intratumoral, Intravitreal, Intrathecal, and Topical administration.

Regulatory experience: US (FDA), UK (MHRA), and Europe (BfArM).

Report abuse