LYT-200 for Acute Myeloid Leukemia and Preleukemia

Recruiting at 14 trial locations

Overseen By Aleksandra Filipovic, MD, PhD.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: PureTech

Must not be taking: Immunosuppressants

Disqualifiers: Acute promyelocytic leukemia, Active tumors, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: approximately 1 year

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new treatment called LYT-200 for certain blood cancers. It focuses on individuals with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS), particularly if previous treatments failed. The trial consists of two parts: one uses LYT-200 alone, and the other combines it with other cancer drugs. It suits those with AML or high-risk MDS that has returned or not responded to other treatments. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that LYT-200 is being tested for safety and tolerability. In earlier studies, LYT-200 was active and helped control the disease in patients with acute myeloid leukemia (AML), suggesting promise but also the need to consider possible side effects.

So far, 37 patients have tried LYT-200, either alone or with other drugs. This ongoing study closely monitors patient responses. Although still in early stages, the treatment has received fast track status from the FDA, which is given to drugs that might meet unmet medical needs, indicating some confidence in its potential safety.

The complete safety profile will become clearer as more data is gathered. However, early signs suggest that LYT-200 might be well-tolerated by many patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

LYT-200 is unique because it targets galectin-9, a protein involved in immune suppression, potentially enhancing the body's ability to fight cancer cells. Most treatments for acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) focus on targeting the cancer cells directly, like chemotherapy drugs such as cytarabine and daunorubicin, or on disrupting cancer cell growth pathways like venetoclax does. In contrast, LYT-200 works by potentially altering the tumor microenvironment to improve immune response. Researchers are excited about LYT-200 because this mechanism might offer an innovative way to tackle resistant forms of AML and MDS, providing hope for patients who have relapsed or do not respond well to existing treatments.

What evidence suggests that LYT-200 might be an effective treatment for acute myeloid leukemia and preleukemia?

Research has shown that LYT-200 may help treat acute myeloid leukemia (AML). In this trial, participants may receive LYT-200 as a single agent, which previous studies found effective in stabilizing the disease in patients who had undergone multiple treatments. Another group will receive LYT-200 combined with drugs like venetoclax or azacitidine. Past research demonstrated that such combinations resulted in 80% of patients experiencing stable disease or better, with some achieving complete recovery. Venetoclax, when combined with decitabine or azacitidine, also produced good results in older AML patients. These early findings suggest that LYT-200, whether used alone or with other drugs, could effectively treat AML and similar conditions. ^{3 6 7 8 9}

Who Is on the Research Team?

Aleksandra Filipovic, MD, PhD.

Principal Investigator

PureTech Health

Are You a Good Fit for This Trial?

This trial is for adults over 18 with relapsed/refractory Acute Myeloid Leukemia or high-risk Myelodysplastic Syndrome, who've tried at least one treatment without success and have no other beneficial standard treatments available. Participants must be able to perform daily activities with relative ease (ECOG ≤ 2) and meet specific health criteria like a certain white blood cell count and organ function.

Inclusion Criteria

My AML has returned or didn't respond to treatment, and no standard treatments are suitable for me.

I can take care of myself and am up and about more than half of my waking hours.

I am willing and able to follow the study's requirements, including undergoing bone marrow biopsies.

Exclusion Criteria

Patient has any condition which, in the Investigator's opinion, makes the patient unsuitable for study participation

I have been diagnosed with acute promyelocytic leukemia.

I have cancer types other than AML or MDS.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Dose escalation study to identify the recommended Phase 2 dose (RP2D) of LYT-200, administered as a single agent or in combination with venetoclax and/or hypomethylating agents

28 days per cycle

Weekly visits for IV infusion

Treatment

Participants receive LYT-200 via IV infusion every week, with or without combination agents, to evaluate safety, tolerability, and preliminary efficacy

approximately 1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• LYT-200

Trial Overview LYT-200, a new potential therapy, is being tested in this Phase 1 trial for safety, how the body processes it (pharmacokinetics), and its effectiveness against AML/MDS. The study involves patients receiving LYT-200 to evaluate these aspects in those with advanced forms of these blood disorders.

How Is the Trial Designed?

Group I: Single agent dose escalation Experimental Treatment 1 Intervention

LYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week.

Group II: Combination agent dose escalation Experimental Treatment 4 Interventions

LYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week, in combination with oral venetoclax Day 1, 100 mg, Day 2, 200mg, Day 3-28, 400 mg and/or azacitidine, 75 mg/m2 subcutaneously given for 7 days per cycle or decitabine 20 mg/m2 IV for 5 days per cycle.

Who Is Running the Clinical Trial?

PureTech

Lead Sponsor

Trials

7

Recruited

830+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05829226

NCT05829226 | A Phase 1 Study With LYT-200 in Patients ... This is an open-label, non-randomized, multi-center, Phase 1, dose escalation study in patients with AML relapsed/refractory to at least one line of prior ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12350645/

Comprehensive view on chemotherapy-free management ... Venetoclax (VEN) is a BCL-2 small molecule inhibitor. Data about its structure, biochemical characteristics and in vitro efficacy against ...

3. targetedonc.com targetedonc.com/view/lyt-200-gains-fda-orphan-drug-designation-in-acute-myeloid-leukemia

LYT-200 Gains FDA Orphan Drug Designation in Acute ... The FDA granted orphan drug designation to LYT-200, a drug being investigated for treating acute myeloid leukemia.

4. clinicaltrials.icts.uci.edu clinicaltrials.icts.uci.edu/trial/NCT05829226

LYT-200 in Patients With Relapsed/Refractory Acute ... This is an open-label, non-randomized, multi-center, Phase 1, dose escalation study in patients with AML relapsed/refractory to at least one line of prior ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6318429/

Venetoclax combined with decitabine or azacitidine in ... Venetoclax plus decitabine or azacitidine showed tolerable safety and favorable overall response rate (CR + CRi rate: 67%) in elderly patients with AML. This ...

6. puretechhealth.com puretechhealth.com/programs/details/lyt-200

LYT-200 More than 50% of AML patients either don't respond to initial treatment or experience relapse or death after responding to initial treatment4 and a median ...

7. news.puretechhealth.com news.puretechhealth.com/news-releases/news-release-details/puretechs-founded-entity-gallop-oncology-present-new-data

News Release Details In an ongoing acute myeloid leukemia (AML) trial, LYT-200 has demonstrated clinical activity and disease stabilization in heavily pretreated ...

8. ash.confex.com ash.confex.com/ash/2024/webprogram/Paper209848.html

A Phase I Dose Escalation and Expansion Trial of Lyt-200 ... RESULTS: As of 23July2024, 37 pts in total have been treated with at least one dose of LYT-200 in SA and CC. 2 pts with R/R AML, and 4 pts with R/R high risk ...

9. targetedonc.com targetedonc.com/view/lyt-200-gains-fda-fast-track-status-in-acute-myeloid-leukemia

LYT-200 Gains FDA Fast Track Status in Acute Myeloid ... The FDA has granted LYT-200, a first-in-class anti-galectin-9 monoclonal antibody, fast track designation for the potential treatment of patients with AML.