TRACON Pharmaceuticals driving the development of targeted therapies for cancer via collaborations

Clinical-stage pipeline includes envafolimab to treat soft-tissue sarcoma Awarded Orphan Drug designation for TRC102 to treat brain and spinal cord cancers Looking for partnerships to help with regulatory and clinical development of drugs in the US What TRACON does: TRACON Pharmaceuticals Inc (NASDAQ:TCON) has positioned itself as a collaboration partner that leads the regulatory filings, clinical trials, as well as US commercialization of best-in-class drug candidates, as an alternative to expensive contract research organization (CRO) based development. By sharing in the cost and risk of clinical development and leading US commercialization, San Diego, California-based TRACON serves as a solution for companies without clinical and commercial capabilities in the US. In its early years, TRACON adopted the standard way most biotech companies develop drugs, which is to outsource the work to a CRO. In 2011, after disappointment with the cost, quality and speed of CRO-based drug development, the company internalized all of the clinical development operations that most companies outsource to a CRO. TRACON boss Charles Theuer has incredible experience and knowledge in the oncology space. He was director of clinical oncology at Pfizer Inc (NYSE:PFE), and led the clinical development of kidney cancer therapy Sutent, which was approved by the US Food and Drug Administration (FDA). In 2016, Dr Theuer steered TRACON to its first partnership, a collaboration with Johnson & Johnson’s (NYSE:JNJ) Janssen Pharmaceutica NV. As part of the deal, TRACON secured the rights to develop a pair of Janssen's oncology drugs. The first is TRC253, a small molecule drug being developed for treating prostate cancer, and the second is TRC694 which addresses blood cancers such as myeloma. Johnson & Johnson Innovation made a $5 million equity investment in TRACON via the purchase of stock at $59.50 per share. The company’s clinical-stage pipeline also includes envafolimab, which is being developed for the treatment of sarcoma and TRC102, a small molecule drug being developed for the treatment of lung cancer. How is it doing: TRACON reported fourth-quarter and fiscal year-end 2020 financial results on February 19, 2021, showing the company had cash and equivalents of $36.1 million as of December 31, 2020, while also highlighting some key upcoming company milestones. The company said it expects that its current cash, equivalents, and short-term investments will fund operations into the second half of 2022, after completing financings of about $13.8 million with multiple new and existing healthcare-focused institutional investors in December. The company recorded a 4Q net loss of $4.3 million, compared to a loss of $4 million during the same period last year, following a slight increase in research and general and administrative expenses. TRACON also outlined some expected key upcoming milestones for the company, including: Independent Data Monitoring Committee review of ENVASARC safety data in 1H 2021 Submit envafolimab response data to the FDA for orphan drug designation in sarcoma in 1H 2021 Data presentation on TJ004309 Phase 1 results in 1H 2021 Interim ENVASARC efficacy and safety data in 2H 2021 Data presentation on TRC102 Phase 2 results in 2H 2021 Request FDA breakthrough therapy designation for envafolimab in 2H 2021 Decision on envafolimab NDA in China for MSI-H/dMMR cancer The company also noted that, as of February 5, 2021, it had initiated 16 clinical sites and enrolled multiple patients at multiple sites in the pivotal ENVASARC trial of single-agent envafolimab and of envafolimab combined with Yervoy. TRACON is conducting the ENVASARC trial in the US in sarcoma, a rare group of cancers that arise in the bones, and connective tissue in the US in multiple sites. The ENVASARC registration trial is a multi-center, open label, randomized, non-comparative, parallel cohort study at 25 top cancer centers in the US. The company said there’s a planned enrollment for 160 patients, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy. At the end of 2020, TRACON also revealed that its corporate partners, Alphamab Oncology and 3D Medicines had received notification that the Chinese National Medical Products Administration (NMPA) had accepted for review the new drug application (NDA) for envafolimab (KN035) in the indication of MSI-H/dMMR cancer. In addition to the registration trial in MSI-H/dMMR advanced solid tumours in China, envafolimab is being studied in two other registration trials. One of them is a randomized Phase 3 trial in biliary tract cancer in China being conducted by 3D Medicines and Alphamab. Then on January 19, 2021, TRACON reported that the New Drug Application (NDA) for envafolimab was granted priority review by the Center for Drug Evaluation of the National Medical Products Administration (NMPA) in China, according to its corporate partners. Priority review is a procedure established to encourage the research and development of new drugs and accelerate the review and approval of new drugs with obvious clinical value and urgent clinical needs, the company said. In the boardroom, on March 8, 2021, TRACON announced the appointment of Dr Lisa Johnson-Pratt to its board of directors. The company noted that Dr Johnson-Pratt brings more than two decades of broad business and commercialization leadership experience to the company and she currently serves as the senior vice president, New Product Planning at Ionis Pharmaceuticals, Inc. Before Ionis, she was head of Global Pharma Commercial Operations at GlaxoSmithKline. During her time there, she also served as head of Early Pipeline Commercial Strategy supporting assets in early-stage development across multiple therapeutic areas, including oncology. Inflection points: Independent Data Monitoring Committee review of ENVASARC safety data in 1H 2021 Submit envafolimab response data to the FDA for orphan drug designation in sarcoma in 1H 2021 Data presentation on TJ004309 Phase 1 results in 1H 2021 What the boss says: In the company's latest results statement, TRACON CEO Charles Theuer said: “We ended 2020 on a high note by enrolling the first patient in the ENVASARC pivotal trial within one year of licensing envafolimab and raising additional capital at market price that further extends our cash runway into the second half of 2022,” He added: “We are focused on enrolling the ENVASARC trial expeditiously and expect the availability of interim data later this year, final data in 2022, and assuming positive clinical data and regulatory approval, to potentially commercialize envafolimab in 2023, in order to address a high unmet need for patients with myxofibrosarcoma (MFS) or undifferentiated pleomorphic sarcoma (UPS).” Contact the author at jon.hopkins@proactiveinvestors.com