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ExonHit Therapeutics Discusses Its Budding Relationship with Pharma

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Last week, ExonHit Therapeutics, a French drug-discovery company that specializes in analyzing splice variants in the human genome to announced that it had extended a drug-discovery and development collaboration with Allergan, an Irvine, Calif.-based pharmaceutical company, for therapeutics for neurodegenerative diseases, pain, and ophthalmology.

According to a statement from both companies, terms of the expanded alliance call for Allergan to pay ExonHit $2 million, and ExonHit will receive financial support in the form of ongoing R&D payments as well as payments for drug candidates achieving certain development milestones. Profit-sharing on commercialized drugs will also be an option for the duo in the future. In total the extension of the agreement between the companies, which was originally inked in late 2002, was an example of how ExonHit is trying to connect with its target market.

To get a better idea of how an array-based service is dealing with the needs of a pharmaceutical company like Allergan, BioArray News spoke with Christina Hedburg, Richard Einstein, and Matthew Pando from ExonHit this week.

Hedburg is the director of business development for ExonHit's US office in Gaithersburg, Md., while Einstein serves as vice president of research in the American market, and Pando is the firm's assistant director of therapeutics.

So how long have you been working with Allergan?

CH: We formed this alliance at the end of 2002.

And what technology are you making available to them?

CH: Well, initially the alliance was based on them using our Datus technology, which is a wet-lab method for identifying splice variants. And more recently, since we launched the splice array platform, that is also being utilized.

What areas of therapeutics are you assisting Allergan with?

CH: They are involved in opthomology, pain, and neurodegenerative disease, and we are working with them in those areas.

To what extent are you working with them? Are you just providing the service, or is your R&D team working with them?

CH: Well we have a very expansive R&D collaboration with them that's been going on for a number of years to which we are jointly discovering targets and compounds.

Your company also calls itself a drug-discovery company. How are you essentially different from Allergan?

CH: The company is a drug-discovery and -development company. We are about seven years old, and the company was formed based on an expertise that the funding scientists shared in the science of alternative splicing. Our mission has been to use information and knowledge about alternative splicing tools to identify targets in drugs.

RE: The major difference between what we do internally and Allergan is the fact that Allergan has focused models that they use in their research and now they are applying those models with our technology to find those novel targets and assess the compounds that come out of the program.

CH: Allergan's history is that it's a company that has been involved in medications for ophthalmology, and recently they diversified a little bit. And our mission has been cancer and neurodegenerative disease historically, so we have a little bit of an overlap.

What is going to come out of those places where you overlap? Can we expect something in the near future or is it a longer-term priority for your collaboration to produce therapeutics for the areas that you are both interested in?

RE: We certainly hope so. We are working diligently to produce efficacious compounds, so our hope, and I am sure Allergan's hope, is that the programs succeed very well, and that products will come out from this collaboration quickly.

In the press release Allergan said that it had one compound in pre-clinical trials based on your technology…

CH: We can't really comment on the timelines of how things are going forward, but the collaboration has originated a compound that's moving forward very well.

Is the new agreement you just signed substantially different from the one that you signed with Allergan in 2002?

CH: It's really just an extension. It's the same arrangement, we just have agreed to run it longer.

How has the financial agreement changed between you and Allergan between the 2002 agreement and this agreement?

CH: I can't comment on that; other than what we put in the press release.

You started offering SpliceArray in February, and you will have the final three gene families that you are offering arrays for by the end of this summer. You'll have the gene families finished by September, I gather.

RE: Well, finished is a relative term. We have four gene families currently available. And we have three others scheduled at this point. And we continue to talk to customers and researchers to see what they're interested in and provide those products to them.

Do you think that your clients will increase as you roll out the remaining three gene families? Are there certain gene families that may be more attractive to researchers from different companies that you haven't made available yet?

CH: I think that our business will continue to grow as we roll out different product lines, and as people get to know us better. I think, in terms of diversifying the product line, one of the things that we are considering doing is developing a line of mouse arrays because we have gotten a number of requests for that. We are working with some collaborators and we should have some papers out by the end of this year, so that should help build some momentum.

Has Allergan shown an interest in using your SpliceArray service in its research?

RE: Yes, very much so.

MP: We currently have ongoing projects and experiments as we speak using a variety of the arrays for various projects that we're doing with Allergan. So they are very much interested in it and they are taking advantage of the technology.

How does it complement what they've been doing with your technology so far?

MP: It seems that it is complementing it very well, and it is allowing them to do some secondary validation maybe a little more rapidly than previously and look at, within a family, look at a lot of genes at once, to really get a feel for which isoforms are being expressed.

RE: The main difference between the two technologies that we offer is that SpliceArrays themselves offer the ability to quickly scan through potential splice events with some experimental data on them through a microarray platform. The Datus platform allows you to generate novel information, information that has not prebiously been identified, but it's a lot lower through-put kind of technology. So it takes longer to get through a Datus analysis than it does through a SpliceArray analysis.

I know that Agilent Technologies provides the arrays for your service, but are you running the actual experiments or are they?

CH: The relationship with Agilent is that these SpliceArrays are of our own proprietary design. We use alternative splicing to design the content. We provide those designs to Agilent and they produce the arrays, and we run all the services here in Gaithersburg, Md. You may be interested to know, it's kind of related. The way we run these SpliceArrays is that we don't take any reach- through rights on the results when we have a SpliceArray client.

So anything Allergan creates using the SpliceArray service would be theirs to market and sell, is that correct?

CH: Well that's a little bit more complicated because we already have an overriding agreement with Allergan that would govern that. But in general, with any other client, when we do a SpliceArray service, the data are all theirs, we don't take any reach-through rights in the results from SpliceArray analysis.

Have other companies like Allergan contacted you for using the service?

CH: Yes. Other pharma companies? Definitely.

Can you disclose their names?

CH: No we cannot. Actually we haven't gotten permission from our clients to disclose who they are. So that is something we should be doing in the near future. But we do have a number of clients both in academia and in pharma.

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