KangStem Biotech changed its strategy for Furestem-AD, an investigational stem cell therapy for atopic dermatitis, to use a repeated dosage after failing to prove the drug’s efficacy with a single dosage in a phase-3 trial. The change is likely to affect the company’s licensing-out deal currently under discussion.
|KangStem Biotech CEO Lee Tae-wha speaks during an investor relations briefing in Seoul, Friday.|
KangStem Biotech CEO Lee Tae-wha held an investor relations briefing at the Kosdaq Listed Companies Association in Yeoui-do, Seoul, on Friday, and explained about the failure to achieve the primary endpoint of the local phase-3 study.
“We were certain about success but had an unexpected result. I apologize,” Lee said.
The phase-3 trial on Furestem-AD took place at 11 domestic medical institutions for 197 adult patients with moderate to severe chronic atopic dermatitis. The study divided patients into the placebo group and the treatment group half and half and observed for 12 weeks after one administration. The treatment group drew much attention because they received a high dose of Furestem-AD that was highly effective in the phase-2 study.
The outcome was unexpectedly disappointing, however. The company could not secure statistical significance in the evaluation of the Eczema Area and Severity Index-50 (EASI-50), the primary endpoint result, in the treatment group. In the 12th week of treatment, only 31.82 percent of the treatment group achieved EASI-50, not statistically significant compared to 27.16 percent of the placebo group.
“We had a higher-than-expected percentage in the placebo group and a lower-than-expected one in the treatment group,” Lee said. “There was almost no difference in the protocol, compared to the previous phase-2a trial. We are analyzing the results from multiple angles.”
As other indicators showed that Furestem-AD still had some possibilities, however, the company would revise its strategy and continue trials on the agent, he noted.
“In the fourth week, the agent showed significant improvement. TARC proteins, which go up with the severity of atopic dermatitis, were reduced by nearly 10 times in the treatment group, compared to the placebo group,” he said.
The company plans to conduct an additional phase1/2a trial to administer Furestem-AD three times with a four-week interval. The company won approval for the trial from the Ministry of Food and Drug Safety in July. The test will compare three groups – low-dose repeated administration group, high-dose repeated administration group, and the placebo group. The company aims to confirm the optimal dosage of the drug to maximize its efficacy.
The trial was selected as one of the “Smart Bio” national projects five years ago.
KangStem Biotech said it would discuss a licensing-out deal further, after securing data to confirm Furestem-AD’s efficacy in a new study.
“We plan to secure effective data of about 30 patients in the upcoming phase-1/2a trial and decided on an additional phase-3 study and a licensing-out agreement,” Lee said.
He added that the company would also test a combination therapy to raise marketability because it could maximize the treatment effect by mixing Furestem-AD with existing medicines such as steroids and antihistamines. The company is working on a nonclinical trial to select drugs that could create the most synergy with Furestem-AD.
Although KangStem Biotech vowed to continue developing Furestem-AD with repeated dosage and combination therapy, the changed strategy could have a significant impact on Furestem-AD’s licensing-out deal.
In the atopic dermatitis treatment area, Dupixent is the strongest player which secured the safety and efficacy even in the long-term use. In a trial, 62 percent of patients treated with Dupixent achieved EASI-75 (the percentage of patients whose eczema area shrank by more than 75 percent compared to the baseline) at the 52nd week, and 63.7 percent at the 72nd week. The high cost, which was the weakest point, will come down with local reimbursement in 2020. Japan, the U.K., and some European countries have already granted insurance coverage to Dupixent.
It is questionable if Furestem-AD, which could arrive in the market four years later at the earliest, could compete against Dupixent. If Furestem-AD is administered several times, the price could go up further. A company that seeks to introduce the technology of Furestem-AD may consider these changes when negotiating a deal with KangStem Biotech.
As for the matter, Lee said, “The company is gradually reducing the production cost of Furestem-AD by mass production.”
The company is analyzing market changes and planning to compare the agent directly with a rival drug in a global trial, he added.