NÖK Therapeutics
Pipeline

Clinical progress

From first-in-human data to the next study.

The Phase 1 study of evencaleucel, run at Karolinska before NÖK acquired the program in 2024, showed that repeat doses of a patient's own NK cells were feasible and well tolerated after transplant, with early signs of activity against residual disease. We are preparing the next clinical study to ask whether it can deepen that response.

  1. Phase 1Complete
  2. Next studyIn preparation
  3. ReadoutsTiming to be announced

01Phase 1 results

First in human: feasible and well tolerated.

The Phase 1 study of evencaleucel was investigator-led and run at Karolinska before NÖK acquired the program in 2024. It gave patients with multiple myeloma their own NK cells, activated and expanded in the lab, as consolidation after stem cell transplant.4

  1. 6 patients

    Three infusions, no lymphodepletion.

    After stem cell transplant, each patient received three weekly infusions of their own NK cells at rising doses, then was followed for up to five years.

  2. 0

    Serious adverse events.

    No serious adverse events and no cytokine release syndrome were seen. The first four patients developed shingles after their antiviral prophylaxis ended; it resolved with treatment.

  3. 3 of 4

    Assessed patients saw their MRD deepen.

    Four weeks after the last infusion, minimal residual disease had fallen further in three of the four patients assessed. The fourth was already MRD-negative and stayed that way.

  4. 2 of 2

    Patients with a measurable M-component saw it fall.

    M-component is a blood marker of myeloma. It fell in both patients where it could be measured, and stayed undetectable in the others through six months.

  5. 4 weeks

    Infused NK cells were still in the blood.

    The infused cells carry a distinct set of markers, so they could be told apart from the patient’s own NK cells, and were still detected up to four weeks after the last infusion.

  6. Granzyme B

    Rose after the infusions.

    Granzyme B is a molecule NK cells use to kill their targets. It rose in the blood after the infusions, and was raised in the bone marrow four weeks after the last one.

Illustrations show the published counts; levels are not drawn to scale.

Study detailsDesign, long-term follow-up, safety and source

The study at a glance

Patients
6, with multiple myeloma after autologous stem cell transplant
Dosing
3 weekly infusions at escalating doses: 5×106, 5×107 and up to 1×108NK cells per kg
Lymphodepletion
None
Follow-up
6 months on study, then clinical follow-up for up to 5 years
Registration
EudraCT 2010-022330-83 · NCT04558853

Long-term follow-up

Median progression-free survival was 34 months, and all six patients were alive after at least five years of follow-up. With six patients and no control group, the study was not designed to compare outcomes with other treatments.

Safety note

The first four patients developed shingles after their standard post-transplant antiviral prophylaxis had ended; it resolved with antiviral treatment. The last two patients received extended prophylaxis and did not develop shingles.

Source

Nahi H, et al. Autologous NK cells as consolidation therapy following stem cell transplantation in multiple myeloma. Cell Rep Med. 2022;3(2):100508.PubMed 35243416

02The next study

Designed to test therapeutic efficacy.

Building on Phase 1, we are preparing the next clinical study of evencaleucel in post-transplant multiple myeloma, planned to focus on residual disease and durability of response.

Primary clinical questionCan NOK deepen MRD response?

Patient population
Post-transplant multiple myeloma patients with residual disease or MRD risk
NOK intervention
Repeat autologous NK cell dosing without lymphodepletion
Primary focus
Deepening of minimal residual disease (MRD)

A positive study would validate NOK's consolidation strategy and establish a path toward registrational development.

03Anticipated readouts

What we’ll be looking for.

The next study is designed around one question: can NOK take residual disease deeper after transplant, and keep it there?
  1. 01Primary focus

    MRD deepening

    Whether residual disease falls further after NOK dosing, including conversion from MRD-positive to MRD-negative.

  2. 02

    Durability of MRD response

    How long those deeper responses last across follow-up.

  3. 03

    Progression-free survival and relapse

    How long patients go without their disease progressing or returning.

  4. 04

    Safety and repeat dosing

    How well repeated infusions are tolerated, without lymphodepletion.