(KDDF-201408-14) Preclinical study and IND approval of anti-scarring therapeutics BMT101, self-delivering RNAi molecule.
Others, Genetics
Our objective is to obtain preclinical safety data and complete an IND application/approval for BMT101, a new preventive medicine for intractable hypertrophic scarring, developed by using OliX’s self-delivering RNAi technology.
- In general, hypertrophic scarring has an incidence which is 3 times higher in Asians and other ethnicities, compared to the Caucasian population. In the US, it has been reported that approximately 45% of patients who suffer from illnesses, surgical operations and burns end up with scarring, and in Asians and other ethnicities, approximately 44.6% will develop a hypertrophic scar.
- Surgical interventions, laser treatment, ointments and patch-type treatments continue to remain the most common treatments. However, these treatments typically have limited effects on reducing the hypertrophic scar. For more effective treatment and prevention, a treatment that eradicates the formation of the hypertrophic scar is needed. Currently, no FDA approved drug exists for this indication.
- After asymmetric siRNA library sequence screening for CTGF, a key gene for the information of hypertrophic scars, our self-delivering RNAi technology was introduced to the corresponding sequence for the development of a new candidate substance, BMT101, for an anti-scarring therapeuticBMT101’s anti-scar efficacy was verified in-vitro through the confirmation of its selective silencing effects for CTGF and other fibrosis factors
- Through PK/PD testing, BMT101 has been determined as only topically active, and it has been confirmed that there are virtually no side effects with systemic exposure.
- Using animal models of scarring, we have demonstrated the efficacy of BMT101 as an anti-scarring therapeutic and are currently undergoing IND-enabling preclinical studies.
- A patent is held for 'Novel siRNA Structure for Minimizing Off-target Effects and Relaxing Saturation of RNAi Machinery and the Use Thereof’ (issue no. 10-0949791, issue date March 19th, 2010(Korea), PCT issued. Patent issued or pending in Europe, Japan, China, and Australia)
- 'Nucleic Acid Molecules Inducing RNA interference with Cell-penetrating Ability and the Use Thereof (issue no. 10-15811655, issue date November 3rd, 2015(Korea). PCT issued. Patent pending in several key territories)
Our cp-lasiRNA technology enables self-delivery into cells without the need for delivery vehicles, such as cationic lipids or polymers,, so there are no delivery vehicle-related side effects. Additionally, via pharmacodynamics test and preliminary toxicity test of BMT101, we have confirmed the low possibility for side effects due to systemic exposure.
- Unlike the conventional siRNA, when treated with BMT101, the quantity of IFN-α was almost identical to the negative control group, and this suggests that BMT101 does not elicit non-specific immune effects.
- Through the development of a new intractable anti-hypertrophic scarring therapeutic, BMT101, it may be possible to expand its indication to other diseases that are related to fibrosis from overexpression of CTGF.
BMT101 is 10 times more potent than other competing candidates and has outstanding competitiveness in terms of side effects and cost of production.
Hypertrophic Scar
Nov, 2014 ~ Oct, 2016
Olix Pharmaceuticals
Preclinical