GSK vets theorize that a mitochondrial target may unlock Parkinson's and ALS treatments in new startup

2022-11-09

小分子药物引进/卖出

Can targeting a mitochondrial pathway previously thought to be undruggable crack the code in devastating neurodegenerative diseases, such as Parkinson’s and ALS? For a new British startup, that’s the £16 million question.

NRG Therapeutics unveiled its Series A early Wednesday morning on the backs of two GSK vets and their research that, they claim, has pinpointed a way to protect mitochondria from the hallmark neuronal damage seen in neurodegeneration. If everything goes according to plan, the biotech could develop “disease-modifying” small molecule treatments for both Parkinson’s and ALS, CEO Neil Miller told Endpoints News.

Funds will be used to get NRG through IND-enabling studies, but Miller said the biotech could partner out the lead asset rather than raise a Series B to take it into human testing itself. Execs are targeting 2024 or early 2025 for their IND submission, after which several options remain on the table.

NRG could license out all the rights, choose to go 50/50 with a partner or raise more money on its own, Miller said. It’s still too early to say which way NRG might go, he cautioned, but the deciding factors involve how much neuron rescue researchers see in preclinical data — possibly predictive of a survival benefit — and the market conditions.

“We feel there’s a strong rationale” for a partnership, Miller said, noting the big disease population in Parkinson’s and the rare disease nature of ALS. If it includes positive preclinical data, he added, “From the feedback we’ve had talking to pharma that that’s an attractive package. And so, you know, that’s what we’re seeking to build.”

The science behind the company involves what’s known as the mitochondrial permeability transition pore, which for years was considered undruggable, Miller said. Recent studies changed that perception, however, linking the pore to both alpha synuclein — a key protein in Parkinson’s — and TDP-43, a protein implicated in both sporadic and familial ALS.

In the normal course of these diseases, the pore is ajar, allowing the associated proteins to enter the mitochondria and release mitochondrial DNA into the cytoplasm. The DNA is then recognized by the immune system as foreign, resulting in a lot of inflammation and downstream neuronal death.

But Miller says his team is researching ways to prevent the pore from opening by using a kind of biological “sticking plaster,” ultimately preventing mitochondrial damage and hopefully slowing neurodegeneration.

“We are unique in that we are targeting very specific effects on mitochondria,” Miller said. “A common feature of neurodegenerative diseases is the dysfunction of mitochondria, and we’re looking to rectify that, in effect, put a sticking plaster on the mitochondria to keep them alive, keep the cell alive and allow the body’s natural processes for regenerating mitochondria to kick in.”

NRG is still considering whether it will develop one drug candidate to treat a range of diseases, or separate programs for each disease — “We’re going into this open-minded,” Miller said. Beyond Parkinson’s and ALS, NRG may also try to go after other neurodegenerative conditions like Friedreich’s Ataxia and, down the line, could venture into multiple sclerosis and Alzheimer’s.

Omega Funds led Wednesday’s Series A and was joined by an additional new investor, Brandon Capital.

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