Felzartamab

Felzartamab is being developed to treat antibody-mediated rejection in kidney transplant recipients. Credit: Gorodenkoff / Shutterstock.com. The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Human Immunology Biosciences’ (HI-Bio) investigational therapeutic felzartamab, to treat antibody-mediated rejection (AMR) in kidney transplant recipients. The designation is a significant step in the development of felzartamab, a monoclonal antibody targeting cluster of differentiation 38 (CD38), which is involved in the production of pathogenic antibodies. It selectively depletes CD38+ plasma cells. Clinical trials demonstrated its potential to improve outcomes in diseases caused by these pathogenic antibodies. HI-Bio is developing felzartamab for immune-mediated diseases including AMR, IgA nephropathy, lupus nephritis and primary membranous nephropathy (PMN). The FDA has previously awarded felzartamab both breakthrough therapy and orphan drug statuses for PMN treatment. See Also: Italfarmaco secures FDA approval for Duchenne muscular dystrophy drug Lisata clinches rare disease status for osteosarcoma drug HI-Bio acquired exclusive global rights to felzartamab, excluding Greater China, through a licensing agreement with MorphoSys in 2022. The FDA’s ODD provides HI-Bio with development incentives such as tax credits for qualified clinical studies, a waiver of FDA application fees and the potential for seven-year market exclusivity upon regulatory approval. AMR is a leading cause of kidney transplant failure with no current effective treatment. The condition is associated with donor-specific antibody production by plasma cells and the infiltration of natural killer cells, which contribute to microvascular inflammation. HI-Bio chief medical officer Uptal Patel said: “Following the FDA’s granting of breakthrough therapy designation for felzartamab in primary membranous nephropathy, we are encouraged to receive orphan drug designation for felzartamab for antibody-mediated rejection. “We are confident in the clinical progress of our anti-CD38 cellular depletion strategy, which to date, has resulted in proof-of-concept data in multiple severe immune-mediated diseases, including antibody-mediated rejection, IgA nephropathy and primary membranous nephropathy.”

LENZ Therapeutics’s focus will be on advancing presbyopia treatments post-merger. Credit: Prostock-studio / Shutterstock.com. LENZ Therapeutics has concluded its merger with Graphite Bio, forming a late-stage biopharmaceutical company dedicated to developing treatments for presbyopia. The merger, an all-stock transaction agreed by the companies in November 2023, positions the combined entity under the name LENZ Therapeutics. The combined company will focus on advancing the lead assets of LENZ Therapeutics for presbyopia treatment. With $225m in cash or cash equivalents, the company is poised to build infrastructure and commercialise its lead product candidates, pending Phase III trial outcomes and Food and Drug Administration (FDA) approval. The financial foundation of the merged company includes $53.5m from a concurrent private investment in public equity financing, contributed by a syndicate of healthcare investors. See Also: FDA grants orphan drug status to HI-Bio’s felzartamab for AMR Bristol Myers Squibb acquires Karuna Therapeutics for $14bn LENZ’s existing investors led the syndicate, which also saw participation from new investors. Eef Schimmelpennink will lead the company as president and CEO, steering it towards its goal of treating a broad demographic of patients with presbyopia. The product candidates of LENZ are LNZ100 and LNZ101, which are preservative-free, single-use, once-a-day eye drops. The drops containing aceclidine and a combination of aceclidine plus brimonidine have shown promising results in terms of quick onset and sustained near-vision improvement. The treatments have also demonstrated effectiveness across a wide age and refractive range of presbyopes without compromising distance vision. Based on the data from the Phase III CLARITY trials, the company plans to file a new drug application with the FDA by mid-2024. LENZ’s strategy is to commercialise the most effective product for the widest range of presbyopes tested, intending to launch either LNZ100 or LNZ101 in the US through its own commercial organisation. Schimmelpennink stated: “Following the close of this transaction, we believe we are well-positioned to bring the opportunity of a once-daily pharmacological eye drop intended to improve near vision throughout the full workday closer to the 128 million people in the United States who are impacted by presbyopia.”