HBI-8000 granted orphan drug designation for peripheral T-cell lymphoma in Japan
San Diego, CA, USA – December 22, 2015 –HUYA Bioscience International (HUYA), Founder, CEO & Executive Chair, Dr. Mireille Gillings today announced that the Ministry of Health, Labour and Welfare (MHLW) granted HBI-8000 orphan drug designation in Japan for peripheral T-cell lymphoma (PTCL).
HBI-8000 is a novel class I-selective oral histone deacetylase (HDAC) inhibitor with immunomodulatory effects regulating antitumor activity, as well as repression of genes associated with drug resistance. The product is currently completing a Phase 1 open-label, dose escalation trial in Japan to evaluate the safety and pharmacokinetics of HBI-8000 in Japanese patients with non‑Hodgkin’s lymphoma (NHL). The orphan drug designation was based on the estimated size of the Japanese PTCL patient population, the non-clinical and clinical studies as well as the clinical development plan in Japan.
“The breadth of activity of HBI-8000 is now emerging strongly as more studies are conducted. The orphan drug designation for PTCL is an important demonstration of efficacy to combat a devastating unmet need.” said Dr. Mireille Gillings, Founder, CEO & Executive Chair of HUYA. “With the orphan drug designation, HBI-8000 should benefit from a priority review for marketing authorization and a 10-year market exclusivity as well as financial incentives.”
Under the Tripartite Cooperation Treaty between China, South Korea and Japan and given the recent approval of the drug for the treatment of PTCL in China, HUYA launched development of HBI-8000 in Japan for NHL. The Company plans to begin a Phase 2 registration trial in 2016 based on the Pharmaceutical and Medical Devices Agency’s (PMDA) acceptance of HUYA’s accelerated development strategy for Japan.
About Peripheral T-cell Lymphoma
Peripheral T-cell lymphoma (PTCL) represents an aggressive subset of non-Hodgkin’s lymphomas (NHLs) that encompasses a broad range of diverse but rare mature lymphomas of T-cell or NK-cell origin. Regardless of subtype, cases of PTCL share aggressive clinical behavior, refractoriness to conventional chemotherapy, and poor overall prognoses. In Japan, the incidence of PTCL is approximately 25% of all malignant lymphomas. According to the most recent patient statistical survey conducted by Japan’s Ministry of Health, Labour and Welfare (MHLW), the number of patients with malignant lymphoma is estimated at approximately 55,000, with an estimated PTCL number of about 14,000 patients. Treatment advances in PTCL have been slow compared to other lymphomas and relapse occurs after treatment with most of the current therapies, with few effective options for salvage therapies.
HBI-8000 is a member of the benzamide class of histone deacetylase (HDAC) inhibitors designed to block the catalytic pocket of Class I HDACs. HBI-8000 is an orally bioavailable, low-nanomolar inhibitor of cancer-associated HDAC enzymes with favorable pharmacology and safety profiles. HBI-8000 inhibits cancer-associated Class I HDAC1, HDAC2, HDAC3, as well as Class IIb HDAC10 at nanomolar concentrations and stimulates accumulation of acetylated histones H3 and H4 in tumor cells. Studies with human-derived tumor cell lines have demonstrated that HBI-8000 inhibits the growth of many tumor cell lines via multiple mechanisms of action, including epigenetic regulation of tumor cell growth and apoptosis, immunomodulatory effects regulating antitumor activity, as well as repression of genes associated with drug resistance. To date, HBI-8000 has been dosed in various types of hematological and solid tumors in several clinical trials, including a Phase 1 trial completed in the United States.
About HUYA Bioscience International
HUYA is the leader in enabling and accelerating the global development of novel biopharmaceutical product opportunities originating in China. Extensive collaborations are established with Chinese biopharmaceutical, academic and commercial organizations to speed development and value creation in worldwide markets for China-sourced product candidates. With the largest Chinese compound portfolio covering all therapeutic areas, HUYA has emerged as the partner-of-choice for maximizing the value of biopharmaceutical innovation in China. HUYA has offices in the U.S., Japan, South Korea and eight strategic locations across China. With the largest team of scientists working with Chinese innovators, HUYA identifies and advances promising drug candidates globally. HUYA was awarded the Asia-Pacific Stevie® Award in the Health Products and Services & Pharmaceuticals category and a Gold Stevie Award in the Woman of the Year 2015 American Business Award category. For more information, please visit www.huyabio.com