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Snapshot: aTyr Pharma

aTyr Pharma
Name: Melissa Ashlock, M.D.
Title: Vice President, External Scientific Alliances and Human Genetics
Company: aTyr Pharma
Location: San Diego, CA
aTyr Pharma
Social Media: @aTyrNews
Description: aTyr Pharma is developing protein therapeutics based on Physiocrine biology. Physiocrines are naturally occurring proteins that modulate extracellular signaling pathways in a variety of physiological processes. These endogenous human proteins act through a variety of receptor classes via mechanisms distinct from current pharmaceuticals and have potential applications in a number of therapeutic areas.
Products or Pipeline, Phase: aTyr’s lead programs are focused on immunomodulation disorders in the areas of inflammation and immunity.

Can you tell me a little bit about the collaboration between CFF/CFFT, Aurora and Vertex?

The collaboration is exemplary because it started at the concept stage. CFF/CFFT invested in an idea. The company had the technology but acquired the skills and expertise to transform the disease, through the CFFT investment and the collaboration. Right now, when many foundations are looking to make a big investment, just like investors are, they are not looking for a concept, they want something in hand (molecule, clinical data). It was a risk that was uniquely able to be taken at that moment. The opportunity for a collaboration like this to happen again will depend on someone taking a risk on concept.

How did the collaboration evolve?

For me being the leader from the CFFT point of view, I saw a dynamic process over 10 years. First, we helped with skills and expertise in cystic fibrosis research, meeting the CF community, and bringing expertise internally to decrease their dependence on CFFT and others for basic research. Then we moved to the next stage to help translate the work. Thus, the Company and foundation grew together.

Can you describe your role?

The general steps for developing a drug are not so different across the board. The specific development requires assays to demonstrate how well a drug candidate works in the lab and in the clinic. Without those assays, the effect of the drug cannot be measured. I had led this aspect of development for the first gene therapy efforts at the NIH for the same disease. Assays we developed for the non-clinical studies were similar to what we needed to test drugs in the clinic. It wasn’t called translation back then. It was just what we did to move potential new therapies forward.

What were the highlights / timeline from discovery to approval?

Two important points: 1) Based on data, a few years into the collaboration, we as a group, CFF/CFFT, Aurora, and Vertex, decided to focus on a small subset of patients. It was a big leap at the time to say we are going to focus a lot of time and resources on a small group of patients to see a proof of concept for CFTR modulation. 2) We decided as a group to accelerate the program, by speeding up some aspects of the program without having a 100% knowledge that we are going to need it. In the end, the data being generated supported this decision and it saved months. Bob was really good a calculated risks – we all learned from this and practiced it. Not all are willing to take big risks like this.

What can the industry learn from this collaboration?

The focus on the small population was a data driven genetic-based focus that resulted in one of the first personalized medicine approaches for a rare genetic disease.

Another lesson is that the venture philanthropy approach to collaborative drug discovery is good if you can find the right organization. Groups need to be willing to be in it for the long haul and even develop the management team. It doesn’t help to fund one part, if you can’t fund it all.

You have to drive the collaboration on the science not the money. At first, milestones are important to guide activities, but they only work if the motivations are right. With a true collaborator, you have the same or complementary motivations, and then everyone wins – getting the drug to the patient. Thus, the contract has to be structured to motivate for the right reasons.

Finally seeing and mitigate the risk was really important. CFF was able to lower risk at the beginning of the collaboration, through the translation and in the clinic. But CFFT could place a value on this risk lowering at the outset. CFF had already taken millions of dollars to fund clinicians and set up clinical trial network. This gave confidence to companies and made many aspects of development easier.

There are many obstacles to drug development, and thus investors have lots of reasons to avoid making big investments or taking big risks. To get around this, organizations or collaborations that develop paths for clinical evaluation of new drugs or lower risk somehow, are needed.

Why is new biology essential for the life science industry? For patients?

We need a lot of new medicines because there are many diseases that have no treatments. We need new ways to think about them from new science (biology, chemistry, physics).

We need new biology to solve difficulty problems not solvable in a timely fashion by existing tools alone. New science can help you understand a way of looking at diseases, medicines, drug discovery. New biology is a bonus because it can give you a new approach to a certain single disease and open your eyes to understanding at other diseases as well.

For example, cancer has historically been characterized by which tissue it originates. With new molecular characterization, we are understanding that cancers from different tissues may have similar mutations, so a drug developed to treat HER2-postiive breast cancer may be applicable in other cancers. These insights shine new light on unifying pathogenic concepts and help us understand the breadth of indications we can pursue.

What are the challenges in translating new biology to medical use?

  1. Convincing people that your novel, sometimes out there discovery is relevant to treating human disease.
  2. Contending with the absence of a path to develop the drug (versus me-too, biology old and path is clear)
  3. New biology is that you can go into new diseases. The disease may not have a path as well as the biology. Path for biology and path for patient population that may have never been part of a clinical trial
  4. Take a lot longer to understand what you have in your hand and what your potential is and people don’t want to take bets on it. Take risks.
  5. No reimbursement path…will it be paid for – which is a big factor if people will invest in it.

What can be done to mitigate or overcome these challenges?

People need to be willing to take risk, so we need to overcome what is preventing them from taking risks. We need to find better ways to generate meaningful clinical data and assess market opportunity. To do that, we need more resources to envision clinical trial and how to do it and see the potential for reimbursement after launch. Government incentives may need to be more targeted to extend upon the incentives for orphan indications.

What role does government funding play in translating new biology?

When NIH is funding basic research, they are funding new biology. New translational programs could really make a difference. Funding just this year, the National Center for Accelerating Translational Science (NCATS) will be tested on the ability to effectively advance new biology. SBIR programs have track record of encouraging and advancing new ideas. We just need more money to be available.

Once the government has made the investments, some companies may need help finding the right team and partners to take the program to the next step. People who understand the whole process of drug development are key to the success of making government funding programs for translation. Capturing the knowledge and skills of funders, such as venture capitalists, would be helpful to understanding how to build a good team and partnerships for a small start-up company.

What helps you continue to innovate?

Lots of flexibility to evolve new biology with through opportunities to collaborate with various science and technology companies.

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