Roche Trial of Ionis Huntington’s Disease Drug Halted After Side Effects

Xconomy San Diego — 

A 20-patient Phase 1 study of an RNA drug discovered by Ionis Pharmaceuticals that is being tested as a treatment for Huntington’s disease by Roche was suspended after two reports of infections related to a device used to take samples of cerebral spinal fluid during the trial.

The suspension doesn’t appear to be related to the drug, tominersen, and no other trials of the investigational treatment were impacted—including an ongoing global Phase 3 study due to read out in 2022.

The trial was likely paused to allow the pharma to reduce the amount of time the intrathecal catheters used for sampling are in patients or to rework its fluid sampling process, SVBLeerink analyst Mani Foroohar said in a research note.

In the pivotal trial of tominersen, the drug is administered via intrathecal injection, and there is no use of the catheter believed to have caused the infections in the Phase 1 trial. The device is being used in the early-stage trial to gather data on the drug’s pharmacokinetics following its administration, Foroohar noted.

After talking with Ionis (NASDAQ: IONS) management, Foroohar said the firm’s rating on Ionis stock would remain unchanged. Ionis’s stock price fell 3 percent as of market close Thursday under pressure Foroohar said he believes reflects “a concerning headline in a risk-off market environment more than fundamental impact on the pivotal program” or the other studies underway.

Tominersen is part of a class of medicines known as antisense drugs, which Ionis pioneered. The potential treatment for the inherited brain disorder was designed to reduce the production of the huntingtin protein, which is produced in abnormally high levels in people with a mutated form of the gene and progressively destroys neurons in the brain. As those neurons are damaged, people with the disease progressively lose their mental faculties and physical control.

Historically Ionis has partnered many of its programs, such as the potential Huntington’s disease drug. All of its clinical-stage neurological programs, for example, in addition to the Huntington’s program, are partnered: potential treatments for amyotrophic lateral sclerosis, Alzheimer’s disease, and frontotemporal degeneration are being advanced by Biogen (NASDAQ: BIIB), and an investigational centronuclear myopathy drug is under evalution by Dynacure.

But CEO Brett Monia on Wednesday, speaking at the annual Cowen and Co. healthcare conference in Boston, said he plans to focus on adding to the biotech’s lineup of proprietary programs. Monia, a company co-founder who has served in a number of positions since its 1989 launch, including as head of drug discovery and chief operating officer, became its chief executive in January after founding CEO Stanley Crooke transitioned from the role to that of executive chairman.

“A very important priority to me … is to create, build, and expand our Ionis-owned pipeline,” Monia said on Wednesday. “[Ionis’s] partnerships are doing very well … but today we’re growing the pipeline that Ionis owns, and it’s expanding rapidly, including in neurological diseases, which is an area we’re particularly excited about, and we’re building our commercial capabilities at Ionis as well to maximize the value of each of those Ionis-owned medicines in the future.”

Of the 40 drugs Ionis has under development, the ones it controls include investigational treatments for a handful of rare diseases, including agromegaly, hereditary angioedema, beta thalassemia, and cystic fibrosis; and an experimental drug for treatment-resistant hypertension. It also controls experimental antisense drugs designed to treat diabetes and prostate cancer in all geographies except China, where it has licensed the rights to Suzhou-Ribo.

Monia cited broad validation of the company’s RNA-targeting therapeutics technology and Ionis’s profitability as drivers of its newfound focus on proprietary programs.