"The FDA granting orphan-drug designation is an important milestone as we continue to develop PRN1008 for ITP patients in need," said Martin Babler, Chief Executive Officer of Principia. "We believe that PRN1008 has the potential to modulate the immune system's attack on platelets, the underlying cause of ITP, and to be an effective oral therapy to reduce the untoward effects often seen with more invasive or broader immune suppression."
The FDA's orphan-drug designation program incentivizes the development of products intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 people in the U.S. Among the benefits of being the first sponsor of a designated orphan drug to receive FDA marketing approval for such a rare disease or condition include seven years of marketing exclusivity, waiver or partial payment of fees prescribed by the Prescription Drug User Fee Act, and tax credits for qualified clinical research costs relating to such designated orphan product.
About Immune Thrombocytopenic Purpura (ITP)
ITP is a rare and often chronic autoimmune disease characterized by a decreased number of circulating platelets, which play a key role in clot formation. There are approximately 150,000 individuals in the United States with ITP, which includes a subset of approximately 100,000 chronic patients. While ITP patients can be asymptomatic, they often have episodic nose and gum bleeding and frequently bruise due to blood leaking from capillaries into the skin and mucous membranes. They also suffer from significant fatigue and diminished quality of life.
PRN1008 is Principia's most advanced drug candidate. It is an oral, small molecule, reversible covalent BTK inhibitor (BTK is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells). PRN1008 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. This approach limits systemic exposure of PRN1008, enables rapid clinical reversibility of effects on the immune system, and is designed for use as a chronic therapy.
About Principia Biopharma
Principia is a clinical-stage biopharmaceutical company dedicated to bringing transformative oral therapies to patients with significant unmet medical needs in immunology and oncology. Principia's proprietary Tailored Covalency® platform enables Principia to design and develop reversible and irreversible covalent, small molecule inhibitors with potencies and selectivities that have the potential to rival those of injectable biologics, yet maintain the convenience of a pill. PRN1008, a reversible covalent BTK inhibitor, is being evaluated in a Phase 2 clinical trial in patients with pemphigus, an orphan autoimmune disease, and in a Phase 2 clinical trial in patients with ITP, a rare hematological disease. PRN2246, a covalent BTK inhibitor which crosses the blood-brain barrier, has completed a Phase 1 clinical trial in healthy volunteers, and has been partnered with Sanofi for development in multiple sclerosis and, potentially, for other diseases of the CNS. PRN1371, a covalent inhibitor of Fibroblast Growth Factor Receptor (FGFR), is being evaluated in a Phase 1 trial in patients with solid tumors. For more information, please visit www.principiabio.com.
This press release contains forward-looking statements. All statements other than statements of historical facts contained herein are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, Principia's expectations regarding the Principia pipeline of product candidates, and the status and timing of development and completion of Phase 2 for PRN1008 for ITP. Such forward-looking statements involve known and unknown risks, uncertainties, and other important factors that may cause Principia's actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the risks and uncertainties of the clinical development process and of clinical trial recruitment; statements about the efficacy, safety and tolerability of our product candidates; early research or clinical results may be materially different from future clinical results; Principia's reliance on third-party organizations, such as contract research organizations, contract manufacturing organizations, and partners such as Sanofi Genzyme; risk of third party claims alleging infringement of patents and proprietary rights or seeking to invalidate Principia's patents or proprietary rights; and the risk that Principia's proprietary rights may be insufficient to protect its technologies and product candidates. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Principia's business in general, see the risk factors set forth in Principia's Registration Statement on Form S-1 that is on file with the Securities and Exchange Commission (SEC) and the prospectus dated September 13, 2018 relating to its initial public offering of common stock. Any forward-looking statements contained in this press release speak only as of the date hereof, and Principia specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Source: Principia Biopharma Inc.
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