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Financing ATMPs – Bedside to Reimbursement


Part 2 of our September 2019 ATMP financing events in Skåne.

The 11th of September CAMP/Swelife hosted a panel at the NLSdays. The panel was a continuation of the Medicon Village event September 10th that began to introduce ATMP financing options. While the Medicon Village focused on financing options in early phases of development the NLSdays panel extended this to later phases of development. The panel was moderated by Swelife/Novo Nordisk representative Christina Östberg Lloyd.

Professor Malin Parmar from Lund University represented an academic collaboration with big pharma. Novo Nordisk is very clear about their desire to work with academics who want to be academics. The relationship with Malin’s pluripotent cell derived cellular therapy for Parkinson’s disease is a great example of this. Malin is free to continue research on improving the cells and Novo Nordisk translates this research to products. While Malin’s lab has largely driven development of this ATMP to clinical trial Malin says Phase I is not enough, not the endpoint. The regulatory requirements of ATMP development require commercialization to be able to take the product through the clinical trials process to market approval and finally reimbursement. Bench to bedside is not the goal. Bedside to reimbursement is the contribution of this type of Novo Nordisk collaboration. Malin says the commercial interest came almost overnight, Novo Nordisk planned an expansion of their stem cell activities and Malin’s therapy, validated by EU funding, had reached the critical stages to spark their interest. The experience of Novo Nordisk in diabetes cellular therapy derived from pluripotent cells meant a lot of the competence could be applied to other pluripotent cell differentiation therapies.  Malin says the culture of interacting with pharma needs to change in regard to ATMP technologies. Knowing people, building networks, catalyzing interactions is important to ensure opportunities are not lost to academic endeavor.

The ATMP field is commonly looked upon as being high risk. The biology is complex, the development costs can be high, the manufacture is complicated compared to small molecule pharmaceuticals or biologics. Björn Odlander from the Venture Capital firm HealthCap first supported this saying that development of effective ATMP treatments requires a deeper understanding of the cause of the disease and the disease mechanism. But then he continued to flip this on its head saying there are great opportunities right now in ATMP for ‘low hanging fruit’. The understanding of disease mechanisms and of tailored interventions provide an investment universe where risk can be mitigated. Linking solid disease research with technology developments to create novel products/treatments/companies enables low risk venture into novel territory.

NextCell Pharma AB is an example of a public company with strong roots in Swedish academia and achieved great successes in a short time frame by finding the external expertise needed to swiftly movre through the different phases of product development. Very early the company partnered with the contract manufacture organization (CMO) PBKM who isolate and manufacture the umbilical cord derived mesenchymal stem cell product called ProTrans. This product was then driven into clinical trials for treatment of type-1 diabetes through the capabilities of the Karolinska Trial Alliance. In less than 5 years of business, and fewer then 3 years of activity in drug development this company now has 2 ongoing clinical trials. Leo Groenewegen from NextCell said, “It was essential that as a small company they did not attempt to do everything themselves but instead search for suitable partnerships with relevant expertise”.

Immunicum is another example of public company derived from Swedish academic research. Like NextCell, part of Immunicum’s success was an early recognition of the need to utilise CMO services to manufacture their product with the required consistency and in quantities needed to maximise clinical success.

When it comes to going public the venture capital funding ends and shares become important. This is where mutual funds may start investing. Daniel Bolanowski from Arctic Aurora LifeScience said a robust phase II trial with randomized control data was needed to ensure the clinical efficacy that would predict commercial success. The technology must be sound, innovative and differentiative compared to other treatments, only then would a mutual fund be interested in investing. However, Daniel said the potential for revolutionizing disease treatment with ATMPs could mean that sound technology and clinical efficacy can be shown already with very few patients, for example a gene therapy for an untreatable disease that is able to completely restore normal functions. In such circumstance even data from 3 patients may be enough to attract mutual funding. Like Björn, Daniel said there is great potential for exciting results to lead to ‘quick’ medical treatments in ATMP. Gene and cell therapies offer completely new ways of treating disease, particularly exciting for those diseases where conventional therapies were not effective. New technologies may quickly turn an untreatable disease into a cured disease. Daniel cautions that even from academia developments may happen in very short time frames and will require varied competencies for success.

Next up was Pfizer who are focusing on gene therapies utilizing recombinant adeno-associated virus (AAV). They are aiming for breakthroughs for 80% of rare disease patients afflicted by genetic disease, the majority of which are monogenic mutations. Christoph Varenhorst from Pfizer says the stakeholders in ATMP development need to understand each others perspectives, needs, limitations, in order to reach the joint goal of treatments coming to patient hands. There have been ATMP failures, not due to the technology, due to market failure. Academics need to ensure commercial foundations are established. In order to implement this early pharma relationships become important. However, Christoph warns that current standards for disease treatment are set for yesterdays therapies. The cost of inefficient implementation in healthcare cannot be underestimated in affecting market success.

The Pfizer message was extended by Cecilia Ahlin from Novartis who says reimaging medicine is needed for implementation of new technologies and innovations like Kymriah. From a healthcare perspective, new mindsets will be needed to understand new side effects, where it may be ‘normal’ for a patient to require intensive care treatment. Further, Sweden should consider how equal access for patients around Sweden will be handled, where infrastructure and special facilities may be needed. Sweden needs to consider their abilities in clinical trials and patient recruitment. It is essential to have the ability to quickly recruit sufficient numbers of patients for companies to choose Sweden as a place to test their therapies. In terms of approved therapies, we need to ensure development of suitable payment models for current and coming therapies.

Article written by: Heather Main