september 03, 2020 - alphamab oncology (stock code: 9966 hk) announced that the u.s. food and drug administration (fda) has granted orphan drug designation (odd) to kn046, a recombinant humanized pd-l1/ctla-4 bispecific antibody developed by jiangsu alphamab biopharmaceuticals co., ltd. (“jiangsu alphamab”), a wholly-owned subsidiary of the company, for the treatment of thymic epithelial tumors (tet). this is the second odd awarded to alphamab oncology by fda after the first odd awarded to the subcutaneous pd-l1 antibody kn035 by fda for the treatment of biliary track cancer early this year.
the orphan drug designation from us fda originates from the orphan drug act which was enacted to encourage the development of innovative drugs to treat orphan diseases with target patient population less than 200,000 in the us. drug candidates with orphan drug designation qualify for seven-year market orphan drug exclusivity (ode). in addition, us fda also rewards odd drugs with comprehensive incentives including tax credit of 50% clinical trial cost, waiver of bla user fee, subsidies for r&d costs, protocol assistance and expedited regulatory approval pathway. in 2019, 21 (44%) out of the 48 new drugs approved by us fda are orphan drugs; and 8 out of the top 10 best-selling cancer drugs in us, have received orphan drug designation for certain indications.
thymic epithelial tumors (tet) is a group of thoracic tumors, predominantly thymomas and thymic carcinoma. the estimated number of patients with tet is about 7,000 to 10,000 cases in the united states in 2020. tet is inoperable and the metastatic tet has a very poor prognosis. there is currently no approved standard treatment for patients who have failed platinum-based chemotherapy; objective response rate of late-line chemotherapy or targeted therapy is less than 20%, and the median survival time is less than 12 months. there is an urgent need for effective drugs to improve the efficacy for patients. research have demonstrated that thymus is an important organ for the development of t cells. thymic epithelial tumor cells highly express pd-l1, so it is possible to benefit from immune checkpoint inhibitor therapy. the australian phase i clinical trial of kn046 has shown a high response rate, long-lasting response time and good safety in patients with thymic epithelial tumors. the phase ii registration clinical trial to treat thymic carcinoma started recently . it is planned to be carried out in more than 10 research centers in china and the united states.
about kn046
kn046 is the world's first recombinant humanized pd-l1/ctla-4 bispecific invented by alphamab. its innovative designs include: a proprietary ctla-4 domain antibody with a significantly improved safety profile; a bispecific antibody fused with pd-l1 antibody; engineered to target the tumor microenvironment with high pd-l1 expression, and treg clearing function. the preclinical and clinical trial results of kn046 have shown promising efficacy and significantly reduced toxicity to human peripheral system, with the potential to become a breakthrough immuno-oncology therapy.
there are about 20 clinical trials of kn046 in different stages covering more than 10 types of tumors including nsclc, tnbc, escc and pancreatic cancer in australia and china. the results of these clinical trials have shown a preliminary profile of good safety and promising efficacy. us fda has approved kn046 to enter phase ii trial based on the clinical results in china and australia. the phase iii clinical trials to evaluate efficacy and safety of combination therapy of kn046 and platinum-based chemotherapy in patients with stage iv squamous non-small cell lung cancer have started in china.
about alphamab oncology
alphamab oncology is a biopharmaceutical company focusing on innovative biologics medicine in oncology. on december 12, 2019, the company was listed in the mainboard of hong kong stock exchange with stock code 9966.
alphamab has fully integrated proprietary biologics platforms in bi-specifics and protein engineering. its highly differentiated in-house pipeline consists of eight anti-cancer drug candidates, four of which have advanced into phase i – iii clinical development phases in china, us and japan.
the company also has state-of-the-art manufacturing capability designed and built to meet nmpa and eu/fda’s cgmp standards. alphamab oncology is committed to further develop its robust pipeline in oncology/immunology to benefit patients around the world.
visit http://www.alphamabonc.com for more information.
alphamab oncology forward-looking statements
this press release contains statements related to our future business and financial performance and future events or developments involving alphamab oncology that may constitute forward-looking statements. these statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. these statements may be identified by words such as "expect," "look forward to," "anticipate" "intend," "plan," "believe," "seek," "estimate," "will," "project" or words of similar meaning. we may also make forward-looking statements in other reports, in presentations, in material delivered to shareholders and in press releases. in addition, our representatives may from time to time make oral forward-looking statements. such statements are based on the current expectations and certain assumptions of alphamab oncology’s management and business operation, many of which are difficult to predict and generally beyond alphamab oncology’s control. these are subject to a number of risks and uncertainties, including but not limited to, uncertainties inherent in research and development, future clinical data and analysis, decisions by regulatory authorities regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates, the absence of guarantee that the product candidates if approved will be commercially successful, the future approval and commercial success of therapeutic alternatives, alphamab oncology's ability to benefit from external growth opportunities, trends in exchange rates and prevailing interest rates, the impact of cost containment policies and subsequent changes thereto, the average number of shares outstanding and alphamab oncology’s disclosures. should one or more of these risks or uncertainties materialize, or should underlying expectations not occur or assumptions prove incorrect, actual results, performance or achievements of alphamab oncology may (negatively or positively) vary materially from those described explicitly or implicitly in the relevant forward-looking statement. other than as required by applicable law, alphamab oncology undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.
