BOSTON--(BUSINESS WIRE)--Syntimmune, Inc., a clinical-stage biotechnology company developing antibody therapeutics targeting FcRn, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SYNT001, Syntimmune’s lead investigational candidate, for the treatment of pemphigus.
Positive preliminary results from a Phase 1b proof-of-concept trial of SYNT001 in pemphigus, a rare and severe autoimmune disease that leads to painful blistering, were reported at the International Investigative Dermatology meeting earlier this year. The preliminary results showed that SYNT001 appeared to be well-tolerated and induced a rapid reduction in IgG and circulating immune complex levels, as well as clinical improvement as measured by Pemphigus Disease Area Index (PDAI) score, with clinical effect persisting beyond the treatment period.
“We are very pleased to receive FDA Orphan Drug Designation as we advance our clinical development of SYNT001 in pemphigus,” said Jean-Paul Kress, M.D., president and CEO of Syntimmune. “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. We believe there is significant potential for SYNT001 in pemphigus and other autoimmune diseases and look forward to providing additional clinical data.”
Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides special status and incentives to encourage the development of drugs for diseases affecting fewer than 200,000 people in the U.S. Orphan drug designation conveys up to seven years of marketing exclusivity if the compound receives regulatory approval from the FDA and offers various development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee and FDA assistance in clinical trial design. The granting of orphan designation does not alter the standard regulatory requirements, timing and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.
Syntimmune is developing SYNT001, an investigational humanized IgG4 monoclonal antibody optimized to inhibit FcRn binding to IgG at both neutral and acidic pH. Studies have shown that SYNT001 rapidly facilitates clearance of IgG and IgG circulating immune complexes (CICs), with the potential to block innate immune responses induced by IgG and CIC, as well as inhibit T cell and B cell activation in response to CIC. Additionally, studies suggest that SYNT001 accomplishes its effects on IgG without destroying immune cells or impacting other types of immunoglobulin, and that SYNT001 has the potential to exert a rapid therapeutic effect in a wide range of IgG-mediated autoimmune diseases.
Founded in 2013, Syntimmune is a clinical-stage biotechnology company developing differentiated drug candidates in a wide range of autoimmune diseases. Drawing on the pioneering research of its scientific founders, the company is advancing novel therapies based on its deep expertise in the biology of the neonatal Fc receptor (FcRn) and its complex role in the pathogenesis of IgG-mediated autoimmune diseases. Syntimmune’s lead candidate, SYNT001, is a monoclonal antibody that specifically blocks FcRn- IgG interactions and is being studied in multiple Phase 1b/2a trials for the treatment of IgG-mediated autoimmune diseases. Syntimmune is also developing SYNT002, which targets FcRn-albumin interactions to facilitate the clearance of albumin-bound toxins. Headquartered in Boston, Mass., Syntimmune has raised $78 million in private financing from leading life sciences investors led by Apple Tree Partners. Investors also include Partners Innovation Fund, FMB Research, and AFB Fund. For more information on Syntimmune, please visit the company’s website at www.syntimmune.com.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to Syntimmune’s future expectations, plans and prospects, including, but not limited to, statements regarding Syntimmune’s regulatory activities and the potential benefits of orphan drug designation, the potential patient population who may benefit from SYNT001, Syntimmune’s development plans and the therapeutic potential of its investigational product candidates, including SYNT001 and SYNT002, the timing of receipt and announcement of data from its clinical trials and studies, and the potential benefits to patients of Syntimmune’s product candidates. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. All information in this press release is as of the date of the release, and Syntimmune undertakes no duty to update this information unless required by law.