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The commitment of a team

TREAT U is a Portuguese biotech company, created as a spin-off from the University of Coimbra.

The genesis of TREAT U is deeply rooted in the founders’ commitment to bringing to the clinic a new medicine for treating cancer patients more effectively and with the minimum incidence of adverse side effects.

My name is Vera Moura, I am currently the CEO of TREAT U, but I was also the PhD student behind the first product of our pipeline. The project was set up by João Nuno Moreira and co-supervised by Sérgio Simões. The three of us started this endeavor in 2006, and four years later, TREAT U was founded to translate the project from bench to bedside.

I am the narrator of this story, but it took a great team to put a project like this in motion.

My mother died from cancer when I was just 18 years old, so this project is dear to my heart. As a pharmacist, it is my mission and duty to ensure that patients receive the best available treatments. Unfortunately, often what is available falls short of what is needed. In such cases, it is my obligation to make full use of hard earned skills and knowledge to come up with a solution. And this is what we do at TREAT U. Together, our multidisciplinary team accumulates years of expertise in pharmacy, chemistry, clinical development, medicine, regulatory affairs, business development and finance, all tied to the mission that we are doing this for people in need.

Why and for whom we do our jobs is what guides us in how and what we do at TREAT U.

The starting point

We were aware that patenting before publishing was the right thing to do, to convert our project into a medicine. When we collected the first in vitro data, our technology was so promising that we decided to file a provisional patent application with the USPTO in 2008, supported by the Tech Transfer Office of the University of Coimbra. In January 2010, TREAT U was founded by 3 “friends and fools”. João Nuno Moreira named the company after the specificity of treatments we create. Our pipeline is intended for specific features of the tumor microenvironment. Should you ever need an oncology medicine, we are here to treat you.

Building the Business Plan

2010 was dedicated entirely to building the Business Plan and starting seed fundraising to progress the preclinical development of our first product. We codenamed the first platform PEGASEMP™, because it recognizes and sticks (‘pega’) to the tumor every time (‘sempre’).

We invested our efforts in building a solid Intellectual Property (IP), by searching for the best patent attorneys. After a few stumbles, we found the right partner in a Boston-based firm. Currently, we have a family of 3 patents already granted in the US and EU for the first product, and 1 patent application pending for the second technology.

In 2010, we competed in several contests. We participated in the Idea to Product (I2P) contest, where we were highly driven as a team to evolve our laboratory prototype into a product. We won the second prize with extremely positive notes on the business proposition.

Later that year, ISCTE-IUL - MIT Portugal Ventures selected TREAT U as a semi-finalist for its pilot program, today known as Building Global Innovators (BGI). Through intensive training, carried out by highly-experienced experts, we developed the company’s business plan and other core documents and practices. This process was later refined at Carnegie Mellon University (CMU) – Portugal program, Entrepreneurship in Residence, which flew us to Pittsburgh to get feedback on our early-developed technology from Pharma companies and Hospitals.

Fundraising – crucial and painful

Once we had the first business plan drafted, we decided it was time to apply for EU-funded competitive grants, managed by Portugal’s Ministry of Economy. Our first proposal to the National Strategic Reference Framework (QREN) was, not surprisingly, rejected. The team was incomplete, the investors were not sound enough and we lacked focus.

Nevertheless, I still remember how we celebrated the submission of the application and how happy we were in taking that big step. Those memories build up a team and create trust and resilience amidst setbacks.

I believe the success that followed both our Scientific and Business roadmaps was the result of intelligent choices and by not being afraid to ask the right questions ahead of time.

We took a moment to identify the missing internal competences, and we recruited Luís Almeida to join our team, a medical doctor with expertise in clinical development and regulatory affairs, and Amílcar Falcão, an expert in pharmacology and medicine development. At the same time, Bluepharma joined the project as an investor and a partner supporting TREAT U in everyday tasks. With this solid team, we succeeded in getting the approval of our first project from the QREN-Compete program. TREAT U was operating at full speed by 2012.

Up until then, we were building the company in our spare time, after business hours. I worked at the Tech Transfer Office of the University of Coimbra, a job I had to leave after 18 months, to dedicate myself 100% to TREAT U.

In 2013, we submitted another application to QREN-Mais Centro, now with Portugal Ventures on our side as a Venture Capital (VC) investor. Being a VC investor with an active role on the company’s Board, Portugal Ventures brought a new perspective to the business model and facilitated networking with potential investors and experts. More than once, our project was presented to international panels in The International Investors Forum organized by PV.

We recruited Nuno Fonseca as our Laboratory Director and Isabel Pinto as a Project Assistant. The team grew with highly-qualified collaborators who embraced the project with the same passion as its founders.

In 2016, Portugal2020 approved our third project for the development of our lead product in the treatment of a rare tumor. These funds enabled us to apply to the European Medicines Agency (EMA) for an Orphan Drug Designation (ODD) for PEGASEMP™ in the treatment of malignant mesothelioma.

We have already successfully raised 2.3 million euros and we are currently looking for a Lead investor to progress to Series A and start clinical trials.

The medicine value chain development

More than 2 million euros were spent over four years to establish what we call the preclinical proof-of-concept. This is intended to show, in animal models, that our technology works better than other medicines in the treatment of cancer.

Before detailing our roadmap, I would like to emphasize that, at TREAT U, we value the time we spend preparing our strategy more than the urgency of generating data. Therefore, we took a particular interest in the interactions with the Regulatory Authorities prior to setting the preclinical plan in motion. Our team carefully elaborated the plan for efficacy studies and toxicology studies according to Good Laboratory Practices (GLP), and submitted it for analysis to the Portuguese Regulatory Authority (INFARMED, IP). Their remarks were included in the final plan, and the GLP part was contracted to a CRO (Contracted Research Organization) with an excellent track record.

We followed the criteria once established for our IP attorneys to choose our next partners. Those were hard lessons to learn, but having the right partners makes all the difference. And it pays off in the end!

Currently, we have the toxicology profile of PEGASEMP™ defined in two different animal species (rats and dogs), and efficacy studies in mice presenting different tumors.

Simultaneously, we took care of a major risk associated to technologies such as ours: the scale-up from laboratory to an industrial setting. We worked alongside a North American Contracted Manufacturing Organization (CMO) to conduct feasibility studies with our existing protocol, and manufactured the pilot batch of PEGASEMP™ under GLP conditions. The product was successfully delivered and has been stable for at least 24 months at defined storage conditions.

The protocol for the efficacy studies in a rare tumor was also discussed previously with the EMA, aiming at filing a proper request for the designation of Orphan Drug for PEGASEMP™ in the treatment of mesothelioma. The EMA granted the requested Orphan Drug designation in December 2016 (EU/3/16/1814), after results showing a 183-fold increase in tumor growth inhibition by PEGASEMP™, compared to the current standard of care.

Fortunately, I am glad to witness the Portuguese clinical trials industry building a solid reputation. We are working with Blueclinical to put together the regulatory-mandated core documents for the first-in-human trials. Together with the European Clinical Research Infrastructure Network, we have a strong contribution that facilitates our multinational clinical research.

Growing pains

The life of a startup is extremely exciting. There are no dull moments. We are either up and running, preparing the next step or trying to avoid the valley of death.

In our experimental design, quality always comes first. A bad trial means a do-over, a loss of time and money. Therefore, it is our policy to ask first and act second. Our data must provide answers to the questions raised by our investors, customers (pharma companies) and end users (medical doctors and patients). Hence, we keep a constant dialogue with every representative of each one of these groups, to ensure a standard development that will meet their endpoints, with cost and time efficiency.

Of course, this is not a straightforward process. There are times when we need to stop and analyze our flaws and gaps, pose the questions for which we must find the answers, and search for partners and collaborators that might enlighten us. Sometimes, we look at what we would like to do, and we realize that our budget is not enough. Those are the days of reassessment, diligence and compromise. I am extremely grateful for my team, the way we are able to communicate openly, call up brainstorm meetings and keep the project running. I often find it hard to realize that this struggle has been taking place for more than 10 years. The stress does not build up, because they are really an amazing team. 

I will try to summarize our list of ‘hiccups’. I call them this because they are not real problems, in comparison to the diseases we are dealing with.

From the filing of the first patent to the hurdles of approval

The first and second patent attorneys that handled our provisional patent application did not do their job properly. Consequently, we almost had our patent refused. Nevertheless, we kept on looking for the perfect partner. And we found the firm that accompanies us until this very day. In our meetings, we feel that we speak the same language. They understand us easily and build on what we develop that is innovative, inventive and has industrial application. So far, we have assembled a solid IP with 2 more patent divisional applications granted and another patent application that is pending. I am confident that there will be many more in the future.

The first refusal for a grant

We invested time and money (which were especially scarce in the beginning) to get our first grant, and the end result was negative. Instead of giving up, we took the criticisms made about the project as advice, called in the right team and a set of investors who believed in us and in the project, and got back up. The second grant application came in with good results and the third was even better. Those funds allowed us to complete the preclinical proof-of-concept with PEGASEMP™.

The choice of the clinical indication

Fortunately, we came across with a versatile medicine that can be recommended for more than one clinical indication. However, a wide variety of options brings to the table multiple factors to consider, besides scientific ones, namely market parameters, medical needs, economic considerations, including economic viability and profitability, of each medical treatment. Our first step was to assess how attractive some clinical indications were to investors and to big pharma. Sitting there and looking at their questions, analyzing an ever-growing competitive market and the big players already in motion brought us to dismay. We then decided to look for validation of our data in meetings with consultants and patent attorneys; to present our data at international meetings of Oncology (such as the American Association for Cancer Research, with more than 21,000 participants from 80+ countries) and our business plan in Business Fora (such as the Bio International, with 16,000+ of the industry’s brightest); to engage with National and European Regulatory Authorities and have their assessment of our plans and data; and also having our studies conducted by certified CROs or collaborating with experts in the specific oncology disease we have selected for PEGASEMP™. These activities defined a clear path of what is both attractive and feasible among the wide variety of clinical indications for PEGASEMP™. 

Finding a Lead investor – the Series A round of financing

It is true that moving on from a seed investment to a Series A seems like entering another dimension to a startup company. The stakes are high, but there is only so much we can do with animal testing. The data we have collected built up to a point where a huge question mark appeared: “Now what? We know this works in animals, but how will it behave in humans?”. This is when we realized that we need supplementary funds to conduct a clinical trial. The roadmap had progressed into a series A round of financing.

This moment meant we needed to gather the entire team to search and find the gaps in our development plan. We compiled all existing data and associated it again with the previous validation from Pharma companies, scientific experts, investors, consultants, medical doctors, fellow scientists, regulatory authorities, and, of course, with our vision for the translation of this medicine from bench to bedside. Altogether, the previous data supported the decision to take the next step and expand our team by including a regulatory expert to work alongside a CRO to plan the clinical trial.

Currently, we are engaging in several meetings and exchanging information with potential lead investors, but we do not exclude the possibility of licensing-out the technology at a preclinical stage. Our success is not only a victory for TREAT U, but also for Portugal, our country. We must not forget that, as a nation, Portugal is taking its first steps in developing innovative oncology medicines that can be a breakthrough for cancer patients worldwide. Attracting investment to our companies means gaining a solid reputation, which will help Portuguese startups and open further investments in the field of Life Sciences.

Personally, I am extremely confident that we will complete our development plan. I am expecting to see a significant increase in patient overall survival rates and quality of life during and after treatment with PEGASEMP™. This is what my team and I are determined to accomplish!