Kolon Ramps Up For Novel Osteoarthritis Cell Therapy
This article was originally published in PharmAsia News
Executive Summary
South Korea's Kolon Life Science is gearing up for mass production of Invossa, the world's first cell-mediated gene therapy for osteoarthritis, as it prepares to file for a domestic approval and a US Phase III study. The venture also plans to raise around $114m to hike its biologics production capacity.
SEOUL - Kolon Life Science Inc., which focuses on gene and cell therapies, has unveiled a financing plan to raise production capacity to prepare for the commercialization of Invossa, also known as TissueGene-C, as it aims to file for an approval in South Korea in the first half of this year.
The company is also set to begin a Phase III study in the US this year and aims to file a US BLA in 2019.
Invossa is an allogeneic (donor) cell therapy involving human chondrocytes (cartilage cells) that have been genetically modified to produce the therapeutic growth factor TGF-beta1, which induces cartilage regeneration in patients with osteoarthritis.
It is administered via intra-articular injection, without the need of anesthesia or surgical intervention. The therapy is said to control pain for a minimum of one to two years per injection and effectively delay disease progression.
The company plans to raise KRW130bn ($114m) in a rights offering, as part of a long-term investment plan to expand its novel biologics business. The funds will be invested over the next three years in building facilities for mass production of Invossa as well as in its R&D pipeline.
"We decided it was time to come up with detailed plans for investment in production facilities as we are aiming to file for approval of Invossa in the first half," said the company's CEO Woo-Sok Lee. "Through the latest rights offering, we will have an additional 50,000 doses of annual production capacity to pre-emptively deal with market demand and develop a new process that can sharply cut production cost."
At present, it has an annual production capacity of 10,000 doses and expects domestic demand for Invossa to exceed its current capacity by 2019.
"The increased production capacity will be for the domestic market. We are now searching for the new plant site," said an investor relations official at Kolon Life. She added that the existing plant capacity will be sufficient to meet the initial domestic demand.
Of the total funding, KRW95bn will be used for construction of a new plant and integration of its research center. The remainder will be used for its other novel biologics pipeline projects - which include a gene therapy for neuropathic pain and an anticancer gene therapy (all preclinical) - and in expansion of indications and additional clinical trials for Invossa after commercialization.
Invossa Plans
In a primary analysis of the Phase II results, Invossa showed a statistically significant improvement over placebo in the change from baseline in both pain (VAS) and function (IKDC) scores. MRI analysis showed cartilage regeneration evidence in selected Invossa-treated patients.
According to Kolon, the Maryland-based biopharma firm TissueGene Inc., its US unit, reached agreement with the FDA in May 2015 for a Special Protocol Assessment (SPA) on the design, endpoints and statistical analysis plan for the Phase III study.
Dongbu Securities has said Invossa has a competitive edge against existing cell therapies as it is administered via a single injection. The company can be more flexible in pricing as its unit production cost is relatively low. If it is covered by the national health insurance scheme in South Korea and if it gets approval from the US FDA, the therapy can grow into a blockbuster in the domestic market.
The brokerage firm expects the company to launch Invossa in Japan in 2021 after it is launched in the US in 2020.
Policy Risks?
There have been worries that Kolon Life may not be able to commercialize Invossa domestically due to the Bioethics and Safety Act. As Kolon's osteoarthritis therapy doesn't fall into the category of diseases that threaten life or bring about severe disabilities, concerns have been raised that this could effectively block the company from launching the drug in South Korea (Also see "Law Change Would Help Kolon As It Progresses Pipeline" - Scrip, 30 Sep, 2015.).
But South Korea's revision of the Act in December last year, which broadened the scope of allowable gene therapy research, has enabled Kolon Life Science to proceed with the development without a glitch (Also see "Korea Bolsters Stem Cell Rules, Allows Broader Gene Therapy" - Scrip, 5 Jan, 2016.).
Previously, the scope of gene therapy research under the Act was limited to work that satisfies all of the following conditions: to treat genetic disorders, cancer, AIDS or other diseases that threaten life or bring about severe disabilities; and for diseases that don't have available therapies or the effects of gene therapies are predicted to be sharply superior to other therapies.
The revised Act now allows gene therapy research that satisfy one of the two above conditions, given that the therapies simply involve delivering genetic materials or cells that introduce genetic materials into the body.