Principia Biopharma Announces PRN1008 Receives Orphan Drug Designation From FDA for Treatment of Pemphigus Vulgaris

SOUTH SAN FRANCISCO, Calif.--()--Principia Biopharma Inc., a private, clinical-stage biopharmaceutical company, today announced that PRN1008, a reversible covalent Bruton’s Tyrosine Kinase (BTK) inhibitor, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pemphigus vulgaris. Principia Biopharma is currently conducting a Phase 2 trial to evaluate the safety and efficacy of PRN1008 in patients with pemphigus vulgaris.

“Receiving U.S. orphan drug designation for pemphigus vulgaris is an important milestone for patients who currently do not have adequate treatment options for this serious autoimmune skin disorder and it also marks an important step for the development of PRN1008”

“Receiving U.S. orphan drug designation for pemphigus vulgaris is an important milestone for patients who currently do not have adequate treatment options for this serious autoimmune skin disorder and it also marks an important step for the development of PRN1008,” said Steve Gourlay, MBBS, FRACP, Ph.D., chief medical officer of Principia Biopharma. “PRN1008 has the potential to control the disease while significantly reducing prednisone use and its related risks, and may become an important treatment option for this devastating disease.”

Pemphigus vulgaris is a rare, potentially life-threatening, autoimmune skin disorder, affecting approximately 40,000 patients in the U.S. and 170,000 worldwide, 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 vulgaris 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.

The FDA Orphan Drug program promotes the development of products that may aid in therapeutic benefits for rare diseases affecting less than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.

About PRN1008

PRN1008 is an oral, reversible covalent BTK inhibitor. This investigational, small molecule therapy acts by blocking BTK (Bruton’s Tyrosine Kinase – a component of B-cell signaling). It was designed using Principia’s proprietary Tailored Covalency™ technology to optimize its safety and efficacy profile, resulting in a prolonged and reversible action at the target site while being rapidly eliminated from the body to limit the overall exposure. This approach gives PRN1008 antibody-like specificity for BTK to impact the immune cascade in immunologic disorders, and the reversible covalent bonding provides the ability to quickly turn this effect off and on as needed, to potentially deliver a safer profile immunomodulator and thereby improve the treatment of immunologic diseases.

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. PRN2246, a low dose covalent BTK inhibitor which crosses the blood brain barrier, has successfully completed IND enabling studies. For more information, please visit the Company's website at www.principiabio.com.

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