Today, the FDA approved two new drug applications. One is for Spero’s tebipenem HBr oral tablets and the second is CStone’s IND application for CS5001.
The U.S. Food and Drug Administration (FDA) approved two new drug applications today.
The health regulator gave Spero Therapeutics’ tebipenem HBr oral tablets priority review designation and confirmed its New Drug Application (NDA) for substantive review for the treatment of adults diagnosed with complicated urinary tract infections (cUTI). The decision is based on positive data from the Phase III ADAPT-PO trial, which demonstrated oral tebipenem HBr’s effectiveness versus intravenous ertapenem for cUTI and acute pyelonephritis in adult patients.
To date, tebipenem HBr (tebipenem pivoxil hydrobromide; formerly SPR994) has acquired priority review, fast track, and qualified infectious disease product (QIDP) designations. The FDA is reportedly planning to set an Advisory Committee meeting to discuss the application. It also received a June 27, 2022 target action date for the Prescription Drug User Fee Act (PDUFA).
“If approved, tebipenem HBr may provide patients an oral treatment option, allowing them to potentially either recover at home from their infections or leave the hospital sooner. We are committed to working closely with the FDA throughout the NDA review process and look forward to tebipenem HBr’s anticipated launch in the second half of 2022,” said Dr. Ankit Mahadevia, the chief executive officer of Spero, in a statement.
Tebipenem HBr will be the first oral carbapenem antibiotic for cUTI, including pyelonephritis. Spero’s lead product candidate has an investigational status in the U.S. and is currently not approved for the indications mentioned.
The second NDA approval was given to CStone Pharmaceuticals. The biopharma’s investigational new drug (IND) application for CS5001 received a “study may proceed” (SMP) letter from the regulator for its potential in targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1).
ROR1 is an oncofetal protein that has high expression numbers in a wide range of cancers, including some types of non-Hodgkin lymphoma, leukemia, and lung, ovarian, and breast cancers. CS5001 is an antibody-drug conjugate (ADC) that targets ROR1 with multiple differentiated features, including tumor-selective cleavable linker, proprietary site-specific conjugation, and pro-drug technology.
Preclinical studies show that CS5001 demonstrates potency and cytotoxicity against a variety of ROR1-expressing cancer cells and can work against tumors in vivo when it was studied in solid tumor and hematological xenograft models.
“The preclinical pharmacology data were encouraging and demonstrated CS5001’s therapeutic potential in multiple hematological and solid malignancies. There are only three ROR1 ADCs, including CS5001, in clinical development. The upcoming first-in-human Phase I study aims to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of CS5001 in advanced B cell lymphomas and solid tumors,” commented Dr. Archie Tse, the chief scientific officer of CStone, in another statement.
CStone is developing and commercializing CS5001 alongside LegoChem Biosciences (LCB) via an exclusive licensing agreement. The deal was originally generated through a partnership between South Korean biotechnology firms LCB and ABL Bio. Under the terms of the agreement, CStone gets exclusive global rights to develop and commercialize the drug outside of the Republic of Korea.
CStone is currently working on advancing its clinical trial on CS5001. It has also submitted its clinical trial notification (CTN) application in Australia and will soon file an IND application in China.
