Fibrosarcoma

Following a special meeting of its board of directors, Tracon Pharmaceuticals has terminated employees and will wind down operations. Tracon Pharmaceuticals has decided to wind down operations weeks after an injectable immune checkpoint inhibitor that was licensed from China flunked a pivotal trial in a rare cancer.The biotech gave up on envafolimab after the subcutaneous PD-L1 inhibitor only triggered responses in four out of 82 patients who had already received therapies for their undifferentiated pleomorphic sarcoma or myxofibrosarcoma. At 5%, the response rate was below the 11% the company had been aiming for.The disappointing results ended Tracon’s plans to submit envafolimab to the FDA for approval as the first injectable immune checkpoint inhibitor, despite the drug having already secured the regulatory green light in China.At the time, CEO Charles Theuer, M.D., Ph.D., said the company was moving to “immediately reduce cash burn” while seeking out strategic alternatives.It looks like those options didn’t pan out, and, this morning, the San Diego-based biotech said that following a special meeting of its board of directors, the company has terminated employees and will wind down operations. As of the end of 2023, the small biotech had 17 full-time employees, according to its annual securities filing.It’s a dramatic fall for a company that only weeks ago was eyeing the chance to cement its position with the first subcutaneous checkpoint inhibitor approved anywhere in the world. Envafolimab claimed that title in 2021 with a Chinese approval in advanced microsatellite instability-high or mismatch repair-deficient solid tumors regardless of their location in the body. The tumor-agnostic nod was based on results from a pivotal phase 2 trial conducted in China.Tracon in-licensed the North America rights to envafolimab in December 2019 through an agreement with the drug’s Chinese developers, 3D Medicines and Alphamab Oncology.

Day One’s Ojemda received accelerated approval from the US FDA to treat low-grade glioma in paediatric patients aged six months and older. Image Credit: Lightspring / Shutterstock. Ipsen is looking to expand its paediatric medication portfolio and has signed a licensing agreement potentially worth $461m with Day One Biopharmaceuticals for its paediatric glioma therapy Ojemda (tovorafenib). Ipsen will make an upfront payment of $111m, of which $71m will be in cash and the rest will be an equity investment. Day One will also be in line to receive up to $350m in milestone-based milestones and ‘double-digit’ royalties on sales. As part of the agreement, the French pharmaceutical will gain global commercial rights, excluding the US, for Ojemda. Day One will retain global development and the US commercial rights for the therapy. Ojemda inhibits type II rapidly accelerated fibrosarcoma (RAF) kinases and is being developed to treat paediatric low-grade glioma with BRAF rearrangements. Low-grade gliomas are a group of slow-growing tumours that can occur in the brain and spinal cord. It is the most common central nervous system tumour type in children and adolescents, accounting for approximately a third of brain and spinal cord tumours in this population, as per the American Brain Tumor Association. See Also: EC approves measure to produce radioisotopes for cancer FDA approves Luye Pharma’s ERZOFRI for schizophrenia In April, Ojemda received accelerated approval by the US Food and Drug Administration (FDA) to treat patients six months of age and older with relapsed or refractory paediatric low-grade glioma harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation. The therapy is expected to generate approximately $797m in sales in 2030, as per GlobalData analysis. GlobalData is the parent company of Pharmaceutical Technology. Ojemda is being evaluated in patients with low-grade gliomas in a Phase III FIREFLY-2 trial (NCT05566795). The study will investigate the therapy against standard chemotherapy in approximately 400 patients. The trial’s primary endpoint is the objective response rate based on response assessment in neuro-oncology (RANO) criteria, as determined by an independent review. Another therapy area in which both Ipsen and Day One have individually invested is antibody-drug conjugates (ADCs). Last month, Day One signed a licensing agreement with MabCare Therapeutics for its ADC worth up to $1.2bn. Ipsen signed two ADC licensing agreements this year—with Sutro Biopharma in April and Foreseen Biotechnology this month. Phase I trials investigating both ADCs are expected to start in the coming months.

Tracon licensed the North American rights to envafolimab from China-based Jiangsu Alphamab Biopharmaceuticals and 3D Medicines in 2019. Image Credit: TarikVision / Shutterstock. TRACON Pharmaceuticals has terminated the Phase II ENVASARC trial for its sarcoma therapy, envafolimab , as the therapy is unlikely to show the level of efficacy required to support a biologics licence application (BLA). The decision to scrap the Phase II ENVASARC trial (NCT04480502) was informed by a blinded independent central review analysis that showed that the therapy’s objective response rate (ORR) is 5% in the 82 patients evaluated for the analysis. This is lower than the 11% ORR needed to meet the trial’s primary endpoint and support a BLA. Tracon has discontinued all its clinical development activities related to envafolimab and plans to reduce its cash burn. The company also plans to explore strategic alternatives, including a “merger, reverse merger, acquisition, other business combination, sales of assets, licensing or other strategic transactions”. The main asset that the company plans to leverage is its in-house product development platform. Tracon claims that the platform can offer cost and time savings while remaining independent of contract research organisations (CROs). Following the news, the company stock was down by 30.6% at market close on 1 July, compared to market close on 28 June. Tracon’s market cap now stands at $2.97m. Sarcomas are rare cancers that develop in the bones and soft tissues. Tracon investigated envafolimab as a treatment for two types of advanced or metastasised soft tissue sarcomas, undifferentiated pleomorphic sarcoma, or myxofibrosarcoma. See Also: CAN-3110 by Candel Therapeutics for High-Grade Glioma: Likelihood of Approval ICTCAR-028 by Innovative Cellular Therapeutics for Hematological Tumor: Likelihood of Approval Envafolimab is a programmed death ligand 1 (PD-L1) targeting single-domain antibody. Tracon licenced North American rights to the therapy from China-based Jiangsu Alphamab Biopharmaceuticals and 3D Medicines in 2019. In 2021, envafolimab was approved in China as a treatment for advanced microsatellite instability-high (MSI-H) or deficient mismatch repair (dMMR) advanced solid tumours, including advanced colorectal cancer. The therapy also received a breakthrough designation last year as a second-line treatment for non-MSI-H/non-dMMR advanced endometrial cancer, in China. Other therapies in Tracon’s pipeline include YH001, a cytotoxic T lymphocyte-associated antigen 4 (CTLA-4)-targeting antibody being developed in partnership with China-based Eucure Biopharma, and TRC102 (methoxyamine), a small molecule inhibitor of the DNA base excision repair pathway.