AprilBio(397030)'s new drug candidate, which was out-licensed to Denmark-based pharmaceutical company Lundbeck, is drawing attention over whether it will make a comeback after overcoming the risk of development being halted.
Lundbeck had announced a halt to developing the candidate as a treatment for thyroid eye disease (TED), but it changed strategy to develop it again as a therapy for neuroimmunology disorders.
According to the pharmaceutical and biotech industry on the 3rd, Lundbeck recently added AprilBio's candidate APB-A1, under the development name Lu AG22515, to its pipeline as "CD40L blocker · neurology · phase 1."
Lundbeck initially developed Lu AG22515 as a TED treatment. However, although the company confirmed in a phase 1b trial that the drug worked as intended by its mechanism of action, it decided that the biological changes did not sufficiently translate into actual symptom improvement and halted further development.
Still, safety and tolerability were assessed as favorable, so the company decided to explore other indications.
The company later relisted the candidate in its pipeline with the development area changed to neurology.
Lu AG22515 was developed based on AprilBio's long-acting protein platform SAFA. It blocks the CD40L protein present on T cells and others to suppress signaling between immune cells and reduce excessive immune responses.
Lundbeck said, "Blocking CD40L has strong potential to treat a broad range of autoimmune-related neurological diseases." Multiple sclerosis (MS), neuromyelitis optica spectrum disorder (NMOSD), and myasthenia gravis (MG) are cited as the main target indications.
For AprilBio, observers say the worst-case scenario has been avoided. If development of the candidate had been fully halted, it would have been difficult to expect additional milestone payments, and the value of the SAFA platform could have been burdened. The technology transfer agreement AprilBio signed with Lundbeck for APB-A1 in 2021 was worth up to $448 million (about 608 billion won), including an upfront payment and development-stage milestones.
The key question is whether Lu AG22515 can demonstrate actual efficacy in the new indication. The specific clinical schedule has not yet been finalized.
However, in the industry it is not uncommon for out-licensed candidates that failed in their original indications to pivot to other diseases and try again.
Hanmi Pharmaceutical(128940)'s epinopegdutide is a representative case. Initially out-licensed to Janssen as a treatment for diabetes and obesity, the compound's rights were returned during development, but Hanmi Pharmaceutical shifted development to metabolic dysfunction–associated steatohepatitis (MASH) and re-licensed it to Merck in 2020.
HanAll Biopharma(009420)'s HL161 (batoclimab) also broadened its development to other autoimmune diseases after safety issues emerged in thyroid eye disease.
A biotech investment industry expert said, "The fact that Lundbeck chose to change the indication of APB-A1 rather than abandon development means the candidate still holds development value," adding, "Depending on future development progress, AprilBio's corporate value could also change."
Recently, AprilBio's other out-licensed candidates have also continued to advance in development.
APB-R3 (EVO301), an atopic dermatitis treatment transferred to U.S.-based Evoimmune, is set to disclose topline phase 2a results at the end of Sep. It is preparing to enter phase 2b in mid-2027. AprilBio recently recruited an RNA (ribonucleic acid) therapy expert and is pushing joint development with Curigin as it moves to expand its new drug pipeline.
Meanwhile, AprilBio was founded in 2013 by Kangwon National University professor Cha Sang-hoon. In Jul., payment was completed for a 346 billion won third-party allotment capital increase with participation from TKG Group affiliate TKG Huchems(069260) and IMM-affiliated investors. As a result, an IMM investment special-purpose company became the largest shareholder, and the governance structure changed so that TKG Huchems, by nominating three of the five board seats, effectively exercises management control.