NodThera says inflammation drug has positive effect in Parkinson’s

2024-03-07

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A Boston-based biotechnology company on Thursday said its most advanced drug showed positive effects in a clinical trial of Parkinson’s disease patients, results that at least one analyst believes could help validate another, similar therapy.

The company, NodThera, said that over the course of four weeks, patients given its drug experienced reductions in “key” biological markers tied to the inflammation and degradation of the nervous system. The drug was also safe and well tolerated, according to NodThera, with no serious adverse events observed.

Specific data weren’t disclosed, though the trial’s findings will be presented Friday at a conference dedicated to Parkinson’s and Alzheimer’s disease research. NodThera now plans to push its drug into mid-stage studies.

“Given that existing Parkinson’s treatments primarily manage symptoms, our innovative, disease-modifying strategy presents a significant shift, aiming to stop the disease progression,” said Alan Watt, the company’s CEO, in a statement.

Taken orally, NodThera’s drug is designed to inhibit a protein complex known scientifically as an NLRP3 inflammasome. Research suggests Parkinson’s is caused, at least in part, by misfolded proteins that form toxic aggregates. These aggregates are thought to then activate inflammasomes, which can lead to nerve cell damage and death.

Drug hunters expect that inflammasome-blocking agents could be useful in treating a variety of diseases, from neurodegenerative ones to obesity to a common liver illness called MASH. NodThera itself is also testing its leading drug in people who are obese and at risk of cardiovascular disease.

The area of research has even attracted investment from some of the world’s largest developers. Swiss pharmaceutical giant Roche bought two biotechs focused on NLRP3 biology. And in 2022, Novo Nordisk spent $70 million to license a medicine targeting NLRP3 proteins.

Shares of Ventyx Biosciences, another small drugmaker developing a couple of these agents, rose nearly 15% following NodThera’s disclosure. Also on Thursday, Ventyx announced it was raising $100 million through a private placement of common stock.

Michael Yee, an analyst at Jefferies, wrote in a note to clients that the NodThera results are a positive for Ventyx and gives his team “confidence we will see promising signals” in an early-stage trial testing one of Ventyx’s NLRP3 drugs in healthy volunteers. The biotech plans to release high-level results from that trial on Monday.

NodThera’s study represents “another datapoint supporting NLRP3's role and importance ... for neuro indications and possibly obesity,” Yee added.

Founded in 2016 by Epidarex Capital, NodThera is now backed by a group of high-profile life sciences investors that includes 5AM Ventures, Cowen Healthcare Investments, F-Prime Capital, Novo Holdings, Sanofi Ventures and Sofinnova Partners. The company raised $40 million through a Series A round in 2018 and then $55 million two years later via a Series B financing.

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