ASLAN gets U.S. FDA orphan designation on ASLAN001

Singapore-based ASLAN Pharmaceuticals has received a U.S. FDA orphan designation for candidate ASLAN001 (varlitinib), a pan-HER inhibitor, as a treatment for cholangiocarcinoma, a rare and aggressive form of bile duct cancer which has no cure, the company said in a press release.

ASLAN CEO Carl Firth

The company, which conducts work in Taiwan and China, said that the incidence of the cancer in China "is 10 times that of the West."

The candidate made waves in July when ASLAN announced plans to work with Taiwan's ACT Genomics on detailed genomic studies of better-than-expected therapeutic responses seen in which one bile duct cancer patient showed 87% tumor shrinkage over a prolonged period of time.

It followed that up by signing an understanding with the National Cancer Centre of Singapore (NCCS) to study novel combination therapies to treat gastric cancer, hepatocellular carcinoma and cholangiocarcinoma to explore wider avenues for its existing clinical trial candidates.

"The Orphan Drug Designation is an important step in the development of ASLAN001," said Carl Firth, CEO of ASLAN, in the press release.

"With no approved targeted therapies for cholangiocarcinoma, the prognosis for patients in both Asia and the west is poor. In addition to our work in cholangiocarcinoma, we are progressing our portfolio of immunotherapies and targeted agents in other Asia prevalent tumor types, including gastric cancer and hepatocellular carcinoma."

In March, ASLAN said positive Phase I results with gastric cancer candidate ASLAN002, licensed from Bristol-Myers Squibb ($BMY) (BMS-777607), will lead to Phase II trials in Asia-Pacific with the small-molecule dual inhibitor of the cMET receptor tyrosine kinase and RON immune checkpoint.

ASLAN002 was dose-escalated in 39 solid tumor patients for a minimum of 28 days as both once daily and twice daily dose regimens, the company said in a press release.

The company also has ASLAN003, a DHODH inhibitor, licensed from Almirall, currently in Phase I trials for rheumatoid arthritis, and ASLAN004, a fully human monoclonal antibody against interleukin-13 receptor α1 that has been shown to block binding and signal transduction of both IL4 and IL13 and licensed from CSL (CSL334). It is in preclinical development.

Last year, ASLAN secured $22 million in Series B financing led by Shanghai-based Cenova Ventures, which joined Morningside Group and XinChen Ventures with BioVeda Capital and Sagamore Bioventures, to fund ongoing clinical development of ASLAN's portfolio.

Sources said the company is eyeing raising more capital and eventually listing in either Asia or the U.S.

- here's the release (PDF)