Reneo Readies for Global Test of Drug for Rare Mitochondrial Disease

Xconomy San Diego — 

Reneo Pharmaceuticals has encouraging results from an early trial of a compound it is advancing for rare diseases characterized by impaired mitochondria—our cells’ energy generators—and the company says it is now designing a large global study of its drug candidate.

Headed by president and CEO Niall O’Donnell (pictured), the San Diego-based company launched last year with $50 million and an investigational small molecule drug it licensed from High Point, NC-based vTv Therapeutics (NASDAQ: VTVT).

The molecule, REN001, is being developed for patients with primary mitochondrial myopathies (PMM). Muscle cells use the energy generated by mitochondria to function, and when that process doesn’t work correctly people experience muscle fatigue, weakness, and exhaustion following exercise.

No FDA-approved therapies exist for PMM, which are caused by genetic mutations. Some patients take combinations of vitamins and supplements in an effort to ameliorate the symptoms. The reduced muscle function that characterizes the diseases can make it harder to go about daily functions and reduce life expectancy.

“Skeletal muscle, the muscles used to walk, are energy-loving tissues in the body, so any improvements in [patients’] ability to walk will have a good impact from a clinical endpoint perspective, but [also] probably more importantly … the ability to go about the normal course of activities, the ability to walk to the shop, to go for a walk to the park,” O’Donnell said. “A lot of these patients experience, upon walking, extreme fatigue, muscle pain, and a lack of endurance, and our aim in this study, over and above safety and tolerability, was to see if we could improve their ability to walk further, walk longer, and we were able to show that from the variety of endpoints we explored.”

On Tuesday the company announced it is using the preliminary results from a 12-week trial that enrolled 23 PMM patients to begin sketching out a global study that will kick off next year with about 200 patients—the only evaluation of its size and scope of a potential PMM drug likely to be conducted by a biotech that year, according to O’Donnell.

The Reneo drug is intended to improve how these patients’ mitochondria work in an effort to preserve muscle function and prevent muscle injury, weakness, and wasting. The company is also testing REN001 as a potential treatment for fatty acid oxidation disorders, a group of conditions that makes it difficult for patients to use fat for energy, and glycogen storage disease type V, also called McArdle disease.

Reneo is a product of a biotech-creating cottage industry of sorts developed by O’Donnell and Reneo executive chairman Mike Grey, who previously worked together at San Diego’s Lumena Pharmaceuticals. That rare disease company was acquired in 2014 for $260 million up front by Shire, which is now part of Takeda Pharmaceutical (NYSE: TAK). Reneo’s Alejandro Dorenbaum, its chief medical officer, was also a key part of the Lumena team.

Grey, a venture partner at Pappas Capital, and O’Donnell, a managing director at RiverVest Venture Partners, have also worked together to launch Mirum Pharmaceuticals (NASDAQ: MIRM), Amplyx, and Spruce Biosciences.

Reneo’s team of about 14 people is split between the UK and San Diego. The group in the UK, who formerly worked together at Pfizer (NYSE: PFE), handles its clinical operations. Renoe plans to add to its headcount to handle the responsibilities of a large global trial, and will need to raise additional funds to do so, O’Donnell said.