StemCell Therapy

Dive Brief:

After a disappointing initial public offering and a tough debt load looming for 2018 and 2019, Kadmon spent the latter half of 2016 putting its house in order, with a credit agreement renegotiation, staff cuts and optimization of its sales strategies. While these changes work in the background, in the foreground the company spent its R&D day yesterday celebrating some good news for KD025, its Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, which has shown similar efficacy to other drugs in development such as Imbruvica (ibrutinib) and Jakafi (ruxolitinib).

"These positive interim findings indicate activity and a favorable safety profile of KD025 in cGVHD, a fatal disease with no approved therapies,"said John Ryan,CMO of Kadmon. Steroids are the current standard therapy for cGVHD and have severe side effects associated with long-term use. We are pleased to see that the majority of patients in the first cohort have been able to reduce their steroid doses, indicating that KD025 potentially offers a well-tolerated treatment option for cGVHD patients."

The company will roll out further data for the other two cohorts, with more results on twice-daily dosing at 200 mg, and data on once daily dosing at 400 mg, during 2018. According to Jefferies analysts, Kadmon will seek breakthrough designation for KD025 by the end of 2017, and begin a clinical trial in newly diagnosed cGVHD in early 2018.

KD025 is also being assessed in a placebo-controlled Phase 2 clinical trial in moderate to severe psoriasis, and an open-label Phase 2 clinical trial in idiopathic pulmonary fibrosis. The company's pipelineincludes tesevatinib, in Phase 2 for non-small cell lung cancer and glioblastoma, and in Phase 2 and 3 for polycystic kidney disease.

Chronic graft-versus-host disease (cGVHD) is a complication of allogeneic bone marrow transplantation and hematopoietic stem cell transplantation, often used to treat myeloma and leukemia. Symptoms affect skin, mouth, liver, eyes, gastrointestinal tract, vagina, esophagus, musculoskeletal tissue and lungs. While the global GVHD market is still a relatively small one, worth around $360 million in 2016, rising to an estimated $640 million by 2026, according to Visiongain.cGVHD affects up to 50% of patients who survive longer than three months post-transplant and has a major impact on quality of life in otherwise successfully treated patients.

More:
Kadmon ROCKing some positive results - BioPharma Dive

This entry was posted on July 12, 2017 at 4:49 pm and is filed under Stem Cell kidney Failure. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |