Science Pool

Date: 4th-7th May 2021

Location: Virtual

Evotec will attend RDD online with a poster presentation on: Formulation and Characterization of Powders for Inhalation using an Antitussive API to Treat Cough in Idiopathic Pulmonary Fibrosis
Presenter will be Carlotta Giulieri - poster co-authors: Stefano Cagliero, Gianluca Trentin

Attending: Carlotta Giulieri

Learn more about RDD online

Date: 14th-16th April 2021

Location: Tokyo Big Sight, Japan

Attending: Masahiko Otani

Visit the CPhI Japan event site

Orphan drugs are defined by regulatory authorities as diseases that affect fewer than 200,000 patients in the US, or no more than five in 10,000 people in the EU. These diseases are often genetic and therefore lifelong conditions, typically affecting patients from very early age on.

While financial incentives and flexible regulatory framework present a wealth of opportunities for developers, the complexities around developing drugs for small patient populations can present significant additional challenges for sponsors. But what are the challenges facing developers of drugs for orphan diseases beyond financial and regulatory issues, and how can these be overcome?

A key challenge is understanding the regulatory and financial framework for orphan drugs in different regions. The following table provides an overview of the European, US and Japanese markets:

Another major challenge is the lack of information that is often known about individual conditions and their disease mechanism. This, in turn, makes it very difficult to assess the right endpoints for clinical trials. In addition, the success of clinical trials in orphan diseases is also hampered by the identification of countries with a sufficient number of study participants and suitable study centres with the capabilities to conduct the required type of trial and ensure proper patient retention throughout the trial. Therefore, although orphan drugs might seem a safe and attractive bet at first glance, the implementation of successful drug development programs can be tricky.

The support of an experienced orphan drug development partner can significantly increase the chances of success. Building on a wealth of long-term orphan drug development expertise, Evotec´s tightly integrated approach and culture of close collaboration ensures that development challenges can be solved, and innovative products can be delivered at demanding timelines. By implementing the right strategies and putting careful de-risking plans in place, it is possible to overcome the challenges associated with orphan drug development to put safe and effective medicines in the hands of the patients who need them. 

Evotec is the right partner to support orphan drugs product development because the Company has GMP facilities approved both for clinical and commercial batches manufacturing well suited for the small amounts of active ingredient and drug product required in this field.

Find out more about Evotec´s orphan drug development and manufacturing capabilities:

API Capabilities
Development Manufacturing
Read our Whitepaper

Stem cells are undifferentiated or partially differentiated cells that can proliferate indefinitely and give rise to various types of specialised cells. They are, therefore, very interesting for therapeutic purposes. In 2006, Shinya Yamanaka’s lab in Japan demonstrated that the introduction of four specific transcription factor genes, now known as Yamanaka factors, could convert differentiated, somatic cells into pluripotent stem cells (also known as iPS cells or iPSCs). For this discovery, Yamanaka was awarded the 2012 Nobel Prize along with Sir John Gurdon, honouring their findings that mature cells can be reprogrammed to become pluripotent.

The iPSC technology holds great promise in the field of regenerative medicine. iPSCs represent an invaluable source of cells, e.g. to replace lost, damaged or diseased cells. Specifically, iPSCs have significant potential in disease areas with high unmet medical need, e.g. neurodegenerative diseases such as Alzheimer’s, ALS or Huntington’s or conditions such as diabetes or age-related macular degeneration (AMD). In these indications, the application of iPSC-based models represents a paradigm shift in developing desperately needed new therapies.

Why iPSCs?

Compared to previous models, patient-derived iPSCs are more physiologically relevant and better suited for modelling disease pathophysiology and for understanding a drug’s mechanism of action. Therefore, iPSC-based high-throughput screening approaches provide unique opportunities as a tool for disease modelling and predicting drug efficacy. This is especially important in complex, age-related or genetic indications such as neuronal diseases. Moreover, patient-derived iPSCs may eventually be utilised to stratify patient populations for clinical trials - a key success factor for electing better and safer drugs for clinical development in disease areas with high unmet clinical need.

iPSCs at Evotec

Evotec has built one of the largest and most sophisticated iPSC platforms in the industry. The platform has been developed over recent years with the goal to industrialise iPSC-based drug screening in terms of throughput, reproducibility and robustness to reach the highest industrial standards, and to use iPSC-based cells in cell therapy approaches via the Company’s proprietary EVOcells platform.

While culturing and differentiating iPSC-derived cells in a reproducible manner at industry scale used to be a challenge in the past, Evotec has succeeded in establishing scalable and robust protocols that allow a stable production of specific disease-relevant cell types. This includes generation of a cell bank of fully validated iPSC lines, upscaling of iPSC culture and differentiation protocols to industry standards, as well as automation of iPSC-derived cultures - all meeting the highest quality control standards.

In addition to phenotypic screening, Evotec has developed more complex models, such as co-cultures of multiple cell types or microfluidic organs-on-a-chip approaches, which enable interaction between different cell types. These systems allow the modelling of human diseases under conditions that closely resemble the physiological environment.

Smart Partnerships

Evotec has entered into several long-term partnerships that leverage the Company’s iPSC platform to develop highly relevant disease models, e.g. with Bristol Myers Squibb (formerly Celgene) in neurodegeneration (2016), and with Centogene in rare diseases (2018).

In its TargetRD project in collaboration with Centre for Regenerative Therapies TU Dresden, Evotec is using the advances in iPSC technology to generate iPSC-derived Retinal Pigment Epithelium cells from patients with retinal degenerative diseases to accelerate drug discovery.

Read our DDup on iPSC-Based Drug Discovery

Read our DDup focused on iPSC-Based Drug Discovery and Retinal Disease

Learn more about biomanufacturing at Just-Evotec Biologics by downloading this presentation first presented at IFPAC in March 2021.

In this presentation, we touch on:

• Historic manufacturing practices
• Macro trends impacting affordability
• Speed and flexible capacity solutions
• New plan design principles

Date: 25 Oct - 19 Nov 2021 Online | 9 - 11 November 2021 Fiera Milano

Location: Fiera Milano, Milan, Italy

Attendees: Lynsey Haskayne, Giuseppe D'Angelo, Ciriaco Maraschiello, Elisabetta Verardo, Paolo Gatti, Emanuela del Vesco, Paola Tocchetti, Elena Bernabè, Marina Galvani, Alessio Rodari, Claudio Bismara, Nerina Coppini

Booth: 2D80 Hall 2, ICSE

Learn more about Evotec's presence and book a meeting!