Innorna Announces U.S. FDA Rare Pediatric Disease Designation Granted to IN015 for Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)

Hongkong, Shenzhen, Nanjing, CHINA, and Cambridge, UNITED STATES, November 1, 2024—Innorna, a clinical-stage biotech company pioneering its proprietary lipid nanoparticle (LNP) technology to develop novel RNA therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational mRNA therapy IN015 for the treatment of progressive familial intrahepatic cholestasis (PFIC), a serious or life-threatening genetic disease. This significant milestone will greatly accelerate the clinical development of IN015, bringing the company one step closer to delivering a transformative therapy to patients with PFIC. Additionally, the designation may enable Innorna to receive a Priority Review Voucher upon FDA approval of IN015.

IN016, the company's cutting-edge mRNA therapy, previously received FDA RPDD and Orphan Drug Designation (ODD) for the treatment of PFIC. IN015 complements IN016 by targeting a different etiological factor, thereby offering a broader and more comprehensive approach to treatment for patients battling this challenging condition.

About PFIC and IN015

Progressive familial intrahepatic cholestasis (PFIC) represents a heterogeneous group of rare genetic disorders characterized by defects in bile acid secretion or transport, leading to the accumulation of bile within the liver. Typical symptoms of PFIC include jaundice, hepatomegaly, pruritus, splenomegaly, and diarrhea. Over time, this disease can progress to severe liver damage, such as fibrosis and cirrhosis, and is associated with high mortality rates. Currently, treatment options for PFIC are limited to supportive or symptomatic care, underscoring the urgent need for more effective therapeutic approaches. IN015 is designed to address the root cause of PFIC, which stems from specific gene mutations. By potentially restoring the function of defective proteins, IN015 aims to normalize bile excretion and improve patient outcomes.

About Innorna

Founded in 2019, Innorna is dedicated to developing a globally leading lipid nanoparticle (LNP) delivery technology platform and innovative RNA therapies to address unmet clinical needs. The company has established a Diversity-Oriented Lipid Library (DOLL), encompassing over 5,000 ionizable lipids for use in developing various innovative therapies, including mRNA vaccines and drugs, gene editing, and cell therapy. Leveraging its proprietary mRNA and LNP technology platform, Innorna has established multiple internal R&D pipelines for infectious disease vaccines, rare diseases, and tumor immunotherapy. The company has also partnered with several multinational biopharmaceutical and biotechnology companies to explore the technology’s potential for broader treatment applications.

Since its establishment, Innorna has gained wide recognition from the investment and industrial sectors and has received numerous awards, including being named among MIT Technology Review’s global top 50 smartest companies and Fortune China’s most socially influential startup companies. At Innorna, we value INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS. We are committed to exploring the frontier of mRNA application based on platform technologies and leading the revolutionary step toward expanding the clinical application of mRNA in various therapeutic approaches to fulfill the unmet medical needs of patients worldwide.

Please visit the Innorna website at www.innorna.com for more information.