The Road to the Clinic for Small Molecule

Oct 4 2016

Johnson & Johnson Innovation, JLABS, Janssen Discovery Sciences and Karolinska Institutet Science Park invite you to an in-depth look into the process of filing a CTA.

Whether your goal is to develop a pipeline through commercial launch or partner as early as possible, submitting a CTA is a critical early milestone for every biotech company. With big Pharma and VC firms competing for the most promising compounds, startups are expected to demonstrate a clear blueprint for CTA approval, and we want you to be ready.

Join us for a series of short presentations exploring the basic requirements and considerations necessary to achieve a successful small molecule CTA submission. The presentations will highlight the following:

  • Janssen Research & Development - a brief overview
  • Preclinical Development strategies for pharmacokinetics, drug metabolism and toxicology
  • Key CMC strategies to balance cost, time and quality risks
  • Designing First-in-Human trials to highlight the qualities of your molecule
  • Regulatory strategy, CTA requirements, and Pre-CTA meetings

Event Partners:


9:00 | Registration Opens, Networking and Coffee
9:30 | Introduction by Johnson & Johnson Innovation and KI Science Park
9:45 | Introduction to Janssen Research & Development
10:00 | Preclinical Development Strategies
  - Designing drugable molecules with appropriate potency, selectivity, efficacy, solubility, kinetics and metabolism
- The importance of estimated low Dose to Man and high bioavailability
- Mitigate in vivo toxicology by in vitro toxicology screening
- Preclinical toxicology incl CV tox and mutagenicity evaluations before and during GLP toxicology
- Considerations for typical First-in-Human (FIH) Program
- Validation and translational aspects for positive human PoC (cellular - in vivo PKPD/Disease models)
- Predictive biomarker strategy and validation
- Budgetary aspects for this phase
11:00 | Break
11:15 | Chemistry, Manufacturing and Controls Strategies
  - Optimizing manufacture of your Active Pharmaceutical Ingredient (API)
- The challenge of chiral APIs
- Drug product formulations for FIH
- Budgetary aspects for this phase
12:00 | Lunch
13:00 | Clinical Pharmacology
  - Designing FIH trials
- Understanding PK/PD
- Biomarkers of safety and efficacy
- Budgetary aspects for this phase
14:00 | Regulatory Overview
  - Data requirements: CMC, safety, clinical plan
- eCTD
- Pre-CTA meetings and regulatory authority (EMA,FDA, MPA) support
- Regulatory Strategy
- Budgetary aspects for this phase
14:45 | Break
15:00 | Case Study
15:45 | Designing a Lean R&D Plan
16:30 | Discussion and Q&A
17:00 | Networking and Drinks
18:30 | Programme Close

Kristof Van Emelen | Director, Emerging Sciences and Innovation, Janssen Discovery Sciences, Janssen R&D read bio»
Ann Lampo | Head Preclinical Project Development EU, Janssen R&D read bio»
Peter D'hoore | Senior Scientific Director, Janssen R&D read bio»
Erik Mannaert | Senior Scientific Director, Janssen R&D read bio»
Birge Berns | Senior Director, Global Regulatory Affairs Immunology, Janssen R&D read bio»
Sanjay Mistry | Head of External Value Creation, Janssen R&D read bio»

FREE | Karolinska Institutet affiliate
30 € | General Public
15 € | Other Students/Academic
40 € | At the door

Karolinska Institutet Science Park
Tomtebodavägen 23A
171 65 Solna

Speakers' Biographies:

Kristof Van Emelen, Ph.D. | Director, Emerging Sciences and Innovation, Janssen Discovery Sciences, Janssen R&D
Kristof Van Emelen obtained his Ph.D. degree in Organic Chemistry and worked from 1997 to 2006 at the Medicinal Chemistry Department of Johnson and Johnson Pharmaceutical R&D in Belgium. In 2004, Kristof was appointed Program Leader and assumed responsibility for the development and implementation of an integrated kinase-platform that served several applications across various therapeutic areas. Between 2007 and 2011, he held the position of Director of Medicinal Chemistry at Tibotec, a daughter company of the J&J Family of Companies, overseeing Medicinal Chemistry activities in the area of Infectious Disease. From April 2011 onwards, he served as Scientific Director Advanced Chemical Sciences, applying chemical expertise in a cross-disciplinary fashion at the interface of Discovery and Development. Since 2013, he is serving as Director Emerging Sciences and Innovation, liaising the Janssen small molecule drug discovery organization with the J&J Innovation Centers. In this role, he is responsible for identifying, forming and enabling productive collaborations with leading external innovators and developing new partnering models.
Connect with Kristof: Connect on LinkedIn

Ann Lampo | Head Preclinical Project Development EU, Janssen R&D
Ann Lampo, DVM, EU Head of Preclinical Project Development and Single Point of Contact Infectious Diseases & Vaccines, Neuroscience and Global Public Health, obtained the degree of Doctor in Veterinary Medicine at the University of Ghent Belgium in 1987. She was certified in European Regulatory Affairs at the Institute for Pharmaceutical Business Administration in the Netherlands (1996) and is a Eurotox Registered Toxicologist since 1998. Ann joined Janssen in 1988 as a Scientific writer and has taken several leadership roles since that time. In 2000, she was appointed Director in Toxicology and became more broadly involved in Preclinical Development (PCD) projects, which led to the approval of REMINYL and the submission of the registration file of Risperdal ConstaTM. From 2001 to 2007, she headed up a group of scientific experts responsible for Genetic and In Vitro Toxicology, GLP safety pharmacology and General Toxicology in In the Drug Evaluation organization. As a member of several early development teams, she was responsible for the toxicological contribution of more than 50 First-in Human packages. In 2007, Ann became the Pre-Clinical Single Point of Contact for the Research & Early Development (RED) EU unit. Since 2010, she is the Therapeutic Area Point of Contact for Neuroscience, Oncology (until 2013), Infectious Diseases including Vaccines, and, since 2013, Global Public Health. In this function, she heads up the Preclinical Project Leaders group based in Beerse. This team also provides Preclinical Submission support for projects from NS, IDV and GPH. Within PD&S, she is also the Single Point of Contact for the London Innovation Center.
Connect with Ann: Connect on LinkedIn

Peter D'hoore | Senior Scientific Director, Janssen R&D
Peter D'hoore has over 25 years of experience in the pharmaceutical industry. In 1987 he graduated as a Pharmacist at the University of Leuven and was certified as an Industrial Pharmacist two years later. Shortly thereafter, he joined the International Quality Department at Janssen Pharmaceutica, Belgium. In 1996 Peter moved to the Operations side of the business where he was responsible for scale-up, process validation and launch readiness of several new products as part of the New Product Introductions group. Three years later,he returned to R&D as CMC leader where he was responsible for the CMC development and launch of several new products such as the long-acting injectable antipsychotics Risperdal® Consta®, Xeplion® and Trevicta®, as well as the oral solid formulations Invega® and Risperdal Quicklet®. He further manages an international group of CMC Leaders and acts as the CMC Single Point of Contact with the Janssen Neuroscience Therapeutic Area and the London Innovation Center.
Connect with Peter: Connect on LinkedIn

Erik Mannaert, Ph.D. | Senior Scientific Director, Janssen R&D
Erik Mannaert graduated from the K.U.Leuven, Belgium, as a Pharm D and obtained a Ph.D. in Pharmaceutical Sciences at the same university, after a residency in the Department of Toxicology. He joined Janssen R&D in 1996, and held positions of increasing responsibility in the area of clinical pharmacology. Most of his experience is as a Clinical Pharmacology Leader overseeing the clinical pharmacology development aspects of compounds in internal medicine, CNS and oncology, both in early and late stages of clinical development. His most recent assignment is as Biopharmaceutics Leader, Global Clinical Pharmacology, at Janssen R&D. In addition, he is an invited professor at the University of Antwerp, Belgium.
Connect with Erik: Connect on LinkedIn

Birge Berns, M.D., MRCP, FFPM | Senior Director, Global Regulatory Affairs Immunology, Janssen R&D
Dr Berns is a physician with > 20 years experience in early and late stage drug development including strategic, regulatory and operational aspects, therapeutic area focus on rheumatology, oncology and anti-infectives within Janssen/Centocor, Eisai and Aventis. Currently, she is leading multiple global regulatory biologic teams within the Janssen Immunology group, integrating inputs from regional and functional Regulatory Team members to create a coherent and unified global regulatory strategy across our Immunology portfolio. In her role, she is responsible for negotiations with Health Authorities around the world. She received her medical qualification from the University of Erlangen Nuernberg, Germany where she also received her MD in Forensic Immunology. She gained her clinical experience in the UK NHS. She obtained her MRCP(UK) and Fellowship of the Faculty of Pharmaceutical Medicine in the UK.
Connect with Birge: Connect on LinkedIn

Sanjay Mistry, Ph.D. | Head of External Value Creation, Janssen R&D
Sanjay rejoined the Janssen Pharmaceutical Companies of Johnson & Johnson (JNJ) in March 2013. He is Head of External Value Creation, a new group that is focused on the accelerated development of select early-stage pre-clinical and clinical portfolio candidates via the attainment of external investment or via direct out-licenses to Biotech, Pharmaceutical or VC backed startups. Prior to this role he was Head of Business Operations for the Janssen Incubator and Venture Leader for an internal natural product drug discovery start-up incubator that was absorbed by Janssen R&D in 2015. Prior to re-joining JNJ, Sanjay was a Vice President with Quaker Partners, a Philadelphia based venture capital firm where he was responsible for the assessment of therapeutic, diagnostic and tools investment opportunities at early, mid-late and commercial stages of development and had board responsibilities on a number of portfolio companies. Prior to Quaker, Sanjay was a founding member of an Internal Venture within Centocor (JNJ) where he led the preclinical retinal degeneration development team for a cell therapy program (CNTO 2476) which is now in Phase IIb clinical testing for dry AMD. Prior to JNJ, Sanjay was a development scientist at Morphogen Pharmaceuticals. He completed Post-Doctoral fellowships at UPMC and the Scripps Research Institute and received his Ph.D. in Pharmacology and MSc in Clinical Pharmacology from the University of Aberdeen. Sanjay is also a graduate of Kauffman Fellows Class 14. The Kauffman Fellowship is a highly sought-after two-year program dedicated exclusively to the world of venture capital and the cultivation of new high-technology, high-growth, high-impact companies. He is the holder of 12 issued patents with 23 pending applications and 20 peer-reviewed publications.
Connect with Sanjay: Connect on LinkedIn