India’s first siRNA drug to treat rare eye disease soon

The elderly patients afflicted by a rare eye disease, NAION (non-arteritic ischemic optic neuropathy) that leads to rapid loss of vision can now bank on a promising new drug to be tested for efficacy by Biocon for the first time in the country


Bangalore: The rare eye disease, NAION (non-arteritic ischemic optic neuropathy) affects thousands of people in the 50-65 age group and there is no current therapy to cure it. The optical nerves once affected leads to loss of vision very rapidly. However, the reversal of the condition may be possible if treated quickly.

Now in a positive development, the Drug Controller General of India (DCGI) has given the approval to do phase II/III study of a novel small interfering RNA(siRNA) drug, QPI-1007 in the country by Biocon. The drug has been developed by US-based Quark Pharmaceuticals. In 2013, Biocon and Quark Pharma signed an agreement to co-develop, manufacture and commercialize the drug in India and other key markets.

This is the first clinical trial of a drug developed using the siRNA technology in India. “Biocon is committed to develop innovative therapies that address unmet medical needs and the initiation of phase II/III studies investigating QPI-1007 in NAION in India is an important step towards this goal,” said Dr Kiran Mazumdar-Shaw, CMD of Biocon and Honorary chairperson of ABLE.

She added:” India has a significant NAION patient population and we are pleased to be the first BioPharma company in the country to provide an siRNA-based therapy that is likely to benefit thousands of patients who either have no access to treatment or cannot afford it.”

QPI-1007 inhibits the expression of caspase 2 to block the swelling at the tip of optical nerve that leads to the loss of vision due to the death of key retinal ganglion cells.  A global trial of this injectable drug that uses the principle of inactivating the genes that lead to the condition has started in six other countries.

Dr Daniel Zurr, chairman and CEO of Quark Pharma said:” siRNA is a new class of breakthrough medicines, with the potential to revolutionize drug development and offer therapeutic precision and widespread applicability and we are delighted to initiate QRK207 in India in this multi-national study to seek a treatment for NAION, a debilitating condition.”

“It takes strong partnerships to translate novel science into commercial therapies to help people and we are extremely happy to have partnered with Biocon, an innovative company best positioned to lead the siRNA based drug class development in India, Dr Zurr added.

The global trials are being conducted in 95 hospitals  across India, US, Israel, Germany, Australia, Italy and China. The trial process involves treating the patients over 12 months. A total of 465 patients will participate in the global trial. The drug may also help in the treatment of common eye disease glaucoma and has been tested for this in USA, Vietnam and Israel.