Mission Nixes Preclinical Studies, Falls Back on Clinical Candidates

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Mission Therapeutics is down to its clinical assets MTX652 and MTX325, which work by disabling a key enzyme that interferes with the cell’s normal process of removing faulty or dysfunctional mitochondria.

Mission Therapeutics will no longer move forward with its preclinical operations and is shutting down a U.K. laboratory involved in this work, Endpoints News reported on Thursday, citing a company spokesperson.

BioSpace has reached out to Mission to independently confirm the news and learn whether the decision was motivated by cost considerations. But in an interview with Endpoints on Thursday, a company spokesperson said that Mission’s cash runway is enough to keep its operations afloat “well into 2026.”

While the spokesperson did not disclose the number of employees affected, some employees have recently posted on LinkedIn that the company is “winding down” its operations, according to Endpoints.

With its preclinical work discontinued, Mission is now down to its two main clinical assets, both of which target and inhibit the action of USP30, a deubiquitylating enzyme (DUB) that disrupts the natural process through which cells remove damaged or dysfunctional mitochondria. On its website, Mission calls DUBs “attractive therapeutic targets” because they are often involved in several disease pathways.

The more mature of Mission’s two clinical assets is MTX652, which in December 2023 secured the FDA’s go-ahead to enter into Phase II studies for acute kidney injury. In January of that year, Mission reported first-in-human findings for the asset, touting an “excellent” pharmacokinetic profile and encouraging safety. According to the biotech’s pipeline page, MTX652 is also being developed for heart failure.

Mission’s other clinical candidate, dubbed MTX325, is a USP30 blocker being tested for Parkinson’s disease, with the potential to expand into other neurodegenerative diseases like amyotrophic lateral sclerosis. The biotech in March 2024 kicked off a “landmark” first-in-human Phase I study of MTX325 in healthy volunteers, building toward testing the drug’s “disease-modifying” potential in Parkinson’s patients this year.

Aside from MTX652 and MTX325, Mission is also working on an AbbVie-partnered program for Alzheimer’s disease. The collaboration dates back to 2018, when AbbVie made an undisclosed upfront payment to leverage Mission’s DUB approach to target Alzheimer’s and Parkinson’s. Mission in 2021 reported hitting a milestone and received a $20 million payment as a result.

Details of the collaboration remain sparse, however. On its pipeline page, Mission revealed only that it is working on two DUB targets with AbbVie but indicated that the development stages of these programs are “confidential.”

Tristan is an independent science writer based in Metro Manila, with more than eight years of experience writing about medicine, biotech and science. He can be reached at tristan.manalac@biospace.com, tristan@tristanmanalac.com or on LinkedIn.
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