SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Principia Biopharma Inc., a private clinical-stage biopharmaceutical company, today announced that it has advanced its clinical development efforts through the initiation of two clinical trials that are anticipated to yield preliminary results in 2016.
The Company’s most advanced clinical candidate, PRN1008, is an investigational oral, reversible covalent inhibitor of Bruton’s Tyrosine Kinase (BTK). Principia is initiating a three month Phase 2 trial designed to evaluate the safety and clinical activity of PRN1008 in patients with pemphigus vulgaris. Pemphigus vulgaris is a rare, potentially life-threatening orphan autoimmune disease associated with painful chronic blistering of the skin.
“Identifying a BTK inhibitor suitable for chronic administration in autoimmune diseases has been elusive for the biopharmaceutical industry,” said Martin Babler, chief executive officer of Principia Biopharma. “Starting a three month clinical trial represents a significant step in the development of BTK inhibitors.”
The Phase 2, multicenter, international, open-label clinical trial of PRN1008 will determine the proportion of patients who are able to achieve control of disease activity without the need for the usual treatment with high dose prednisolone. Prednisolone is an oral corticosteroid whose long-term usage is associated with many serious side effects, including fatal and non-fatal infections.
The Company’s second clinical candidate, PRN1371, is a selective, oral, covalent fibroblast growth factor receptor (FGFR) 1-4 inhibitor that is being evaluated in a dose escalation Phase 1 trial in various solid tumors. The multicenter, international, clinical trial of PRN1371 is evaluating the safety, tolerability and recommended Phase 2 dose in patients with advanced solid tumors. The trial involves a dose escalation phase followed by an expansion cohort phase in patients with FGFR1-4 genetic alterations.
“We are pleased that, in a very short period of time, a pharmacologically active dose of PRN1371 within the therapeutic range has been identified,” said Steve Gourlay, MBBS, FRACP, Ph.D., chief medical officer of Principia Biopharma. “We are looking forward to initiating the dose expansion stage of the trial by the middle of 2016.”
About pemphigus vulgaris
Pemphigus vulgaris is a rare, potentially life-threatening, autoimmune skin disorder that causes blistering of the skin and ulcers in the mouth and genitals. Men and women are equally affected and the disease is typically seen in middle-aged or older individuals. Pemphigus is known to be caused by autoantibodies which break the bond between the skin cells and cause inflammation. It is traditionally treated with high doses of corticosteroids, often combined with a steroid-sparing immunosuppressant drug.
About BTK inhibition in autoimmune disease
Bruton’s Tyrosine Kinase (BTK) is a protein important to the proper function of the immune system. BTK is essential for the development and maturation of B cells and is involved in the activation of other hematopoietic cells, such as basophils, mast cells, macrophages, neutrophils, antigen-presenting cells and platelets. Inhibition of BTK activity results in the down-regulation of various cellular activities that are activated in autoimmune and inflammatory diseases, including cell proliferation, differentiation, maturation and survival.
About FGFR inhibition in cancer
FGFR genetic alterations occur in a wide range of cancers including lung, breast, stomach, liver and bladder. These alterations may be amenable to targeted therapy designed to inhibit the effects of the alteration in driving tumor growth. Highly selective FGFR inhibitors, such as PRN1371, may be able to slow or reverse tumor growth with a lower rate of side effects compared with less selective drugs.
About Principia Biopharma
Principia Biopharma Inc., a private clinical-stage biopharmaceutical company, has created a revolutionary new way to design and develop oral small molecule therapies that are more potent, selective, durable and safer than currently available drugs. The Company has utilized its proprietary Tailored Covalency™ technology to develop a portfolio of drug candidates that exhibit antibody-like specificity to benefit patients with autoimmune and inflammatory diseases and cancer. PRN1008, a reversible covalent BTK inhibitor, is currently being evaluated in a Phase 2 clinical trial in patients with pemphigus vulgaris, an orphan autoimmune disease affecting skin and mucous membranes. PRN1371, a covalent FGFR1-4 inhibitor, is currently being evaluated in a Phase 1 clinical trial in cancer patients with various solid tumors. For more information, please visit the Company's website at www.principiabio.com.