By Kunal Das and Kamal Choudhury
April 27 (Reuters) – Intellia Therapeutics said on Monday its experimental gene-editing therapy reduced the frequency of swelling attacks in patients with a rare genetic disorder in a late-stage trial, sending the company’s shares 3% higher.
The results prompted Intellia to start a rolling submission of its data for the gene therapy as the company aims for the U.S. Food and Drug Administration’s approval next year, the company said.
Intellia, co-founded by CRISPR pioneer and 2020 Nobel Prize winner Jennifer Doudna, was testing the treatment, lonvoguran ziclumeran, for hereditary angioedema (HAE), an inherited disorder that causes recurring swelling attacks.
The therapy itself is based on CRISPR gene-editing technology, which uses molecular “scissors” to trim faulty parts of genes that can then be disabled or replaced with new strands of normal DNA. In the case of lonvo-z, it works by disabling a gene involved in producing kallikrein, a protein that triggers swelling attacks.
In the trial involving 80 patients, a one-time infusion of the therapy reduced swelling episodes by 87%, compared with placebo over six months, meeting the main goal.
It also helped 62% of patients remain attack-free, compared with 11% in the placebo group during the period.
On pricing strategy, Intellia finance chief Edward Dulac said on an analyst call that lonvo-z would likely be priced at a premium to existing HAE treatments, while cautioning that overly aggressive pricing could provoke payer resistance.
Current treatments for the condition include Ionis Pharma’s Dawnzera and Takeda Pharmaceutical’s Takhzyro.
HAE affects about one in 50,000 people, according to Intellia.
Cantor analyst Steve Seedhouse said lonvo-z “will probably be a niche product,” and warned about a potential ‘black box’ warning for a fatal liver toxicity event seen in a separate Intellia CRISPR program.
Side effects were mostly infusion‑related reactions such as headache and fatigue, and were all mild or moderate, the company said.
Intellia said it expects to complete its U.S. regulatory filing in the second half of the year.
(Reporting by Kunal Das and Kamal Choudhury in Bengaluru; Editing by Vijay Kishore and Leroy Leo)
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