TAIPEI, Taiwan – Calling its recent FDA orphan designation for small-molecule drug CX-4945 in cholangiocarcinoma an important milestone in its development and regulatory strategy, 2012 startup Senhwa Biosciences Inc. enters 2017 with plans for an uplisting on the Taiwan stock exchange and hopes for restoring investor enthusiasm for Taiwan’s biopharma sector.
Effective therapies are few for cholangiocarcinoma – a cancer which poses challenges in terms of early detection and has a survival rate at five years of only 20 percent. Bile duct cancers are increasing in prevalence globally and are of particular significance and concern in East Asian markets, including South Korea, China, Japan and Taiwan. CX-4945, which is designed to inhibit protein kinase CK2, which serves a significant function in the DNA damage repair mechanisms of cancer cells, has so far demonstrated favorable safety, pharmacokinetic and pharmacodynamics characteristics in treating cholangiocarcinoma.
Senhwa recently published phase I results, showing that CX-4945 can be administered safely in combination with the two chemotherapy agents, gemcitabine plus cisplatin, and demonstrated observable clinical activity suggesting an advantage in the combination. The firm is conducting phase I and phase II trials of CX-4945 for the CK2 platform in the U.S., Taiwan and South Korea.
“This proof-of-concept study is designed to evaluate the combination of CX-4945 with DNA damaging chemotherapy agents,” said Tai-Sen Soong, CEO of Senhwa. “Positive results in this orphan indication may propel the development of CX-4945 toward similar combination chemotherapies in additional solid tumor indications, such as breast, lung and bladder cancers, with greater incidence and substantial markets.” In North America, cholangiocarcinoma has a low incidence rate of only about 4,000 cases a year, Soong said.
“The importance of orphan drug designation is that it will allow us to get a better market value for the drug, once it’s been approved,” he told BioWorld Today. In the U.S. market, insurance companies might pay an average of $100,000 per patient per year, he noted.
Ted Lynch, an equity analyst at Pro Standard Global Capital in Taipei, said CX-4945’s orphan drug designation was a positive step for the company and Taiwan’s biotech sector. “For the Taiwan investment community it is important for local companies to demonstrate bringing drugs to market and profiting from their sale,” Lynch said.
CX-4945’s orphan drug designation also “breaks up a string of high-profile disasters – OBI Pharma Inc. last year and Medigen [Biotechnology Corp.] in 2014 – that materially curbed the enthusiasm that a lot of institutional investors felt for the sector,” Lynch added.
OBI Pharma was involved in a high-profile conflict of interest scandal and faced allegations of insider trading after claiming to have developed a breast cancer drug that did not materialize. In 2014, Medigen went through a 20-day sell-off after a drug trial fell short. (See BioWorld Today, Aug. 1, 2014.)
Senhwa (6492:TT), which is listed on the emerging stock board (ESB) of the Taipei stock exchange, is also preparing for a listing on Taiwan’s OTC market. Soong said he hopes the offering will take place in April. Senhwa’s stock closed Friday at NTD195 (US$6.31).
“In Taiwan these days, it’s very tough for biopharma companies to get listed,” Soong said. Senhwa passed final regulatory review for a listing on Taiwan’s OTC exchange last month, but approval from the stock exchange and several government agencies for fundraising must be attained before the issue can take place.
In addition to CX-4549, Senhwa is developing another drug, CX-5461, a p53 activating Pol I inhibitor, for the treatment of several types of cancers, especially those that have difficulty repairing damages to their cells. In animals, CX-5461 has demonstrated effectiveness in shrinking tumors, but it has not been thoroughly studied in people.
A phase I/II study of CX-5461 for the G-quadruplex platform for breast cancer, sponsored by the Canadian Cancer Trials Group, is currently recruiting, with Senhwa and Stand Up To Cancer as collaborators. Primary outcome measures will be to confirm the recommended phase II dose and schedule of CX-5461 in patients with solid tumors (phase I), and to measure antitumor activity assessed by the response rate using RECIST in each cohort (phase II). Phases I and II have been given time frames of 12 months and 24 months, respectively.
Phase I trials of CX-5461 have also been conducted for the Pol I platform and the indication of hematological malignancies at the Peter MacCallum Cancer Centre in Melbourne,Australia.
Phase I trials of CX-5461 have also been conducted for the Pol I platform and the indication of hematological malignancies at the Peter MacCallum Cancer Centre in Melbourne,Australia.
With its headquarters in Taiwan and an operational base in San Diego, Senhwa is a value-added development company whose primary focus is to uncover validated targets or therapies that could significantly improve treatments. Senhwa secured $17 million in series B financing for clinical trials in 2013 from investors, including H&Q Asia Pacific, Morningside Ventures and China Investment & Development Co. Ltd.
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