TLC Submits IND to US FDA Application for TLC178, Liposomal Vinorelbine Candidate for Pediatric Rhabdomyosarcoma

TAIPEI, Taiwan, June 03, 2018 (GLOBE NEWSWIRE) — Taiwan Liposome Company, Ltd. (4152.TWO) (TLC), a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of novel nanomedicines designed to target areas of unmet medical need in pain management, ophthalmology, and oncology, today announced the submission of an Investigational New Drug (IND) application with the U. S. Food and Drug Administration (FDA) to initiate a Phase I/II, open label, dose-escalation clinical trial of TLC178, a NanoX™ liposomal formulation of the anticancer drug vinorelbine, in pediatric rhabdomyosarcoma patients.

TLC178 received a Rare Pediatric Disease Designation last year from the FDA for the treatment of rhabdomyosarcoma (RMS), a very rare form of cancer that most frequently occurs in children, with only about 350 cases per year in the United States. Due to the rarity of RMS, TLC plans to first enroll all types of pediatric refractory or relapsed sarcoma patients to evaluate the safety and tolerability of TLC178 in combination with low-dose oral cyclophosphamide in Part 1 of the trial; this approach will also explore the potential activity of TLC178 in patients with various pediatric sarcomas. Part 2 of the trial will then evaluate the antitumor activity of TLC178 in combination with low-dose oral cyclophosphamide in pediatric patients with relapsed or refractory RMS.

“We believe TLC178 has the potential to modulate the pharmacokinetic profile and enhance tumor localization of vinorelbine, which may reduce drug-related toxicities and reduce dosing frequency, potentially bringing tremendous advancements in the treatment of RMS, which has had little improvement over the last 30 years,” commented George Yeh, President of TLC. “We are excited about the prospect of TLC178, if approved, in providing a better quality of life for children with cancer as well as their families.”

About TLC178
TLC178 is TLC’s proprietary NanoX liposomal formulation of vinorelbine, an anticancer drug frequently used off-label to treat rhabdomyosarcoma, a type of soft tissue sarcoma, and other sarcomas. Compared to vinorelbine, TLC178 is designed to have better pharmacokinetics, lower toxicities, reduced myelosuppressive side effects, longer dosing intervals, higher drug concentration at neovascular-rich and subcutaneous tumor sites and capability to broaden indications. In April 2017, the FDA granted TLC178 a Rare Pediatric Disease Designation in RMS, which will qualify TLC178 for submission of a new drug application in this indication for priority review, and may result in the granting of a transferable Priority Review Voucher that can reduce the standard ten-month FDA review time to six months. In July 2017, the FDA also granted TLC178 an Orphan Drug Designation for the treatment of soft tissue sarcomas. If TLC178 is approved for the treatment of soft tissue sarcomas, the Orphan Drug Designation will provide marketing exclusivity for up to seven years. A preclinical study showed TLC178 demonstrating more effective control of tumor growth than free vinorelbine in a cyclophosphamide combo. An ongoing Phase I/II dose escalation trial in adult patients with advanced malignancies has enrolled 12 patients so far with no hematological toxicity observed; findings from this trial can be used to model the dose-toxicity relationship and calculate the initial dose level in pediatric patients.

About TLC
TLC (4152.TWO) is a clinical-stage specialty pharmaceutical company dedicated to the research and development of best-in-class novel nanomedicines that maximize the potential of its proprietary lipid-assembled drug delivery platform (LipAD™). TLC believes that its deep experience with liposome science allows TLC to combine onset speed and benefit duration, while establishing drug levels at target tissues without incurring large systemic exposures. TLC’s BioSeizer™ technology enables local sustained release of therapeutic agents at the site of disease or injury; its NanoX™ targeted delivery technology enables prolonged pharmacokinetic profiles and enhanced distribution of liposome-encapsulated active agents to the desired site. These technologies are versatile in the choice of active pharmaceutical ingredients and scalable with respect to manufacturing. TLC has a diverse, wholly owned portfolio of therapeutics that target areas of unmet medical need in pain management, ophthalmology, and oncology. TLC is consistently ranked in the top 5% among all listed companies in Taiwan’s Corporate Governance Evaluations. 

Cautionary Note on Forward-Looking Statements
This press release may contain forward-looking statements. Forward-looking statements contained in this press release include, without limitation, statements regarding TLC’s expectations regarding the clinical development of TLC178 for the treatment of rhabdomyosarcoma, the potential clinical benefits of TLC178, the expected enrollment in and the timing of clinical trials. Words such as “may,” “believe,” “will,” “plan,” “potential” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are not guarantees of future performance and involve a number of unknown risks, assumptions, uncertainties and factors that are beyond TLC’s control. All forward-looking statements are based on TLC’s expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, TLC expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

CONTACT: Contacts:

Taiwan Liposome Company (TLC)
Dawn Chi
Corporate Communications
+886 2 2655 7377 ext. 136

Solebury Trout
Julia Balanova
Vice President
+1 646 378 2936