Eledon Pharmaceuticals (NASDAQ:ELDN) is making exciting progress with its lead drug candidate tegoprubart, potentially ushering in a new era of safer and more effective immunosuppression for transplant recipients. The clinical-stage biotech recently achieved two major milestones that increase confidence in tegoprubart’s best-in-class prospects as a next-generation solution for preventing organ rejection.
First, the first participant has successfully received an islet cell transplant and treatment with tegoprubart in a pioneering trial at the University of Chicago. The study is evaluating tegoprubart as part of a novel immunosuppressive regimen aimed at reversing type 1 diabetes by allowing insulin independence after an islet cell transplant. Currently, toxic side effects from standard anti-rejection drugs limit broader utilization of this potential cure.
Tegoprubart’s selective mechanism of action blocking the CD40L pathway could open up islet transplantation to many more patients by avoiding the nephrotoxicity, neurotoxicity, hypertension and other issues seen with calcineurin inhibitors like tacrolimus. Reversing type 1 diabetes through a safe, functional islet cell transplant would provide transformative benefits for patient quality of life.
In addition to this groundbreaking study, Eledon also reported very promising interim Phase 1b results demonstrating tegoprubart’s ability to preserve kidney function with a well-tolerated safety profile following kidney transplantation. Through 1 year of treatment, participants averaged estimated glomerular filtration rates (eGFRs) greater than 60 mL/min/1.73m2 at all timepoints after day 30. This is substantially higher than the typical eGFRs in the low 50s seen in kidney transplant recipients on standard immunosuppressants during the first year.
These eGFR results highlight tegoprubart’s potential to protect transplanted kidneys from the nephrotoxic effects of current anti-rejection medications over the long-term. With similarly impressive 1-year eGFR data north of 90 mL/min/1.73m2 in two subjects, the drug could potentially enable kidney transplants to last significantly longer before failure versus what is currently possible.
From a safety perspective, tegoprubart was very well-tolerated in the Phase 1b trial. Only 3 out of 13 participants discontinued treatment due to manageable side effects like hair loss and fatigue, with no reports of graft loss or death. This clean profile contrasts starkly with the harsh toxicities of current calcineurin inhibitor regimens that often lead to treatment discontinuations.
With its unique mechanism avoiding general immunosuppression, tegoprubart represents a paradigm shift in preventing transplant rejection that could finally break the tradeoff between organ rejection and drug toxicity. Eledon plans to showcase the full Phase 1b kidney transplant dataset at an upcoming medical conference, setting the stage for additional catalysts from the ongoing Phase 2 BESTOW trial expected to read out in the coming months.
Analysts forecast peak sales for tegoprubart well into potential blockbuster territory above $1 billion across multiple transplant indications and autoimmune diseases like lupus that also involve the CD40/CD40L pathway. With its excellent early efficacy and safety results, tegoprubart is steadily derisking its path to becoming the next standard of care in immunosuppression for transplantation.
At a modest $50 million market cap, Eledon is currently an undervalued opportunity for investors considering tegoprubart’s multi-billion dollar commercial prospects. As the drug continues to make strides in the clinic, the company’s shares have tremendous upside potential. Eledon is definitively a clinical-stage biotech to keep high on the watchlist.
Take a moment to take a look at Eledon’s company presentation at NobleCon19.
