Ionis Pharma’s drug for rare diseases without approved therapy meets the objectives of the Pivot study

Patients who have a particular genetic disease whose symptoms include a progressive worsening of muscle control were able to walk faster after treatment with an experimental genetic medicine of Pharmaceutical Ionis, achieving the main objective of a pivotal study. Based on these results, the drug manufacturer said that he provided regulatory submission to what could become the first therapy approved by the FDA for this ultra-rare condition.

The Ionis, Zilganersen medication has been developed to treat Alexander’s disease, a hereditary disorder that leads to neurological deterioration and symptoms that include loss of functional mobility, the inability to control muscles and the difficulty of breathing and swallowing. As these symptoms aggravate, they finally become fatal. The current treatment of this ultra-rare disease, which occurs in an estimated birth of 1 million, is a favorable care for the management of symptoms.

Alexander’s disease affects astrocytes, a type of cell spread in the central nervous system and the key to its function. The disease stems from a genetic mutation which leads to an abnormal accumulation of the glial fibrillar acid (GFAP) in astrocytes. Zilganersen is an antisen oligonucleotide (ASO) designed to stop the production of an excess of GFAP caused by mutations in the GFAP gene.

Ionis evaluated Zilganersen in a phase 1 to 3-3 multiple dose study, controlled by placebo, which recruited patients with Alexander’s disease of 18 months and 53 years. Participants, mainly children, were randomly assigned to receive a low or high dose of the study medication or a placebo administered as an intrathecal injection every 12 weeks. The 50 mg high -dose group was considered the central dose cohort.

The main objective of the study is to measure, from the basic line to 60 weeks, the change of walking speed according to a 10 -meter walking test. Ionis said on Monday that the high dose of Zilganersen had shown a 33% increase in the goal of walking speed, which was both statistically significant and clinically significant. The secondary objectives include the measurement of the scores reported by patients and doctors as a function of various scales to assess the symptoms and severity of the disease. On these objectives, Ionis only said that the results showed “coherent favorable trends”.

Ionis said that the study medication has shown favorable security and tolerability, adding that most of the undesirable or moderate events. The incidence of serious undesirable events was digitally lower in the Zilganersen arm compared to the control arm. Detailed data will be presented at a next medical conference. Ionis plans to submit a new request for medication to the FDA for Zilganersen in the first quarter of 2026.

Ionis specializes in Asos, a type of genetic medicine that uses small RNA pieces to bind to messenger RNA in order to reduce the expression of a pathogenic protein of the disease. ASO research of the company has led to the drug drugs approved by the FDA Spinraza for spinal muscular atrophy and the quality for a rare genetic form of amyotrophic lateral sclerosis. The two were developed and marketed in partnership with Biogen.

Ionis always has partnerships, but its strategy now includes the development and marketing of fully -fledged drugs. Last year, the approval of the Tryngolza FDA made this family chylomicronemia syndrome. The Hochement of the FDA’s head last month for the hereditary medication of the Angio-œnda Dawnzera edema gave Ionis its second.

In a note on Monday sent to investors, William Blair Myles Minter analyst said that the details on Zilganersen’s results according to the secondary objectives would have read the price of the drug, if this ASO is approved. The company models $ 295 million in advanced medication; About $ 120 million in the United States

“Alexander’s disease is an ultra-orphing condition with an estimated population of less than 500 patients worldwide,” Mininter said. “However, there is currently no approved therapy for Alexander’s disease, and taking into account the need not satisfied here, we consider the results of phase 3 of today as another victory for ionis within its entirely detained neurology franchise, which continues to generate positive clinical results.”

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