On September 12, 2018, the U.S. Food and Drug Administration (FDA) granted an orphan drug designation to Syntimmune Inc.’s SYNT001 for the treatment of pemphigus.
“This is an important milestone for the SYNT001 clinical development program and highlights the high unmet medical need for new therapies with the potential to improve the lives of pemphigus patients and their families,” said Jean-Paul Kress, MD, president and CEO of Syntimmune. “We believe there is significant potential for SYNT001 in pemphigus and other autoimmune diseases and look forward to providing additional clinical data.”
Positive preliminary results from a phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus served as the basis for the approval. In the first cohort, SYNT001 was observed to be well tolerated and induced a rapid reduction in IgG and circulating immune complex levels. In addition, as measured by Pemphigus Disease Area Index (PDAI) score, SYNT001 induced clinical improvement, with clinical effect persisting beyond the treatment period.
Syntimmune recently announced positive preliminary results from its phase 1b proof-of-concept trial of SYNT001 in pemphigus vulgaris and foliaceus patients. It’s exciting for the IPPF to share good news related to research and treatments. The full press release from Syntimmune can be found here. The following is an excerpt:
Syntimmune, Inc., a clinical-stage biotechnology company developing antibody therapeutics targeting FcRn, today announced positive preliminary results from its Phase 1b proof-of-concept trial of SYNT001 in patients with pemphigus vulgaris and pemphigus foliaceus. The data showed clinically meaningful benefit of SYNT001, with a favorable safety and tolerability profile similar to that observed in the Phase 1a study.
“There remains a clear unmet need for a safe and fast-acting treatment for patients with pemphigus, who face serious symptoms and complications associated with their disease,” said Donna Culton, M.D., Ph.D., an assistant professor at the University of North Carolina School of Medicine. Culton presented preliminary results of the Phase 1b study at the International Investigative Dermatology conference being held on May 16-19, 2018 in Orlando, FL. “These preliminary data demonstrate safety as well as a rapid reduction in PDAI scores and lowering of IgG levels with treatment of SYNT001, which support further studies of this drug as a potential new therapeutic option,” Culton said.