Recent Updates
Recently added Catalysts

NL-201

Phase 1

Solid Tumor | Small molecule | Oncology |Neurogene Inc.|Last Updated: Oct 1, 2024

Development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Dec 2020

Target and mechanism

Molecular targetIL-2 and IL-15
Target classCytokines
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment59

FDA Designations

No designations recorded

Clinical trial landscape

NL-201 · 1 trial · 2 indications

Phase 1 1
NCT04659629NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory CancerSolid Tumor
COMPLETED59 Analytics
PHASE1COMPLETED
NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory Cancer
Solid TumorUnlock trial analytics

Study Endpoints

Primary Endpoints

Recommended phase 2 dose (RP2D) for NL-201 (Parts 1 and 2)
Up to Day 33

Evaluation of tolerability of NL-201 as measured by number of subjects with dose limiting toxicities (DLTs)

Recommended dose schedule for NL-201 (Parts 1 and 2)
Up to Day 33

Evaluation of tolerability of NL-201 as measured by number of subjects with dose limiting toxicities (DLTs)

Recommended phase 2 dose (RP2D) for NL-201 in combination with Pembrolizumab (Parts 3 and 4)
Up to Day 33

Evaluation of tolerability of NL-201 in combination with Pembrolizumab as measured by number of subjects with dose limiting toxicities (DLTs)

Recommended dose schedule for NL-201 in combination with Pembrolizumab (Parts 3 and 4)
Up to Day 33

Evaluation of tolerability of NL-201 in combination with Pembrolizumab as measured by number of subjects with dose limiting toxicities (DLTs)

Incidence of treatment-emergent adverse events
Up to Day 33

Rate of adverse events in patients with advanced solid tumors

Severity of treatment-emergent adverse events
Up to Day 33

Rate of adverse event grades in patients with advanced solid tumors

Secondary Endpoints

Best Objective Response according to RECIST version 1.1
Up to 36 months
Objective Response Rate (ORR) according to RECIST version 1.1
Up to 36 months
Progression-Free Survival (PFS) according to RECIST version 1.1
Up to 36 months
Unlock Study Endpoints

Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1: NL-201 Monotherapy Dose EscalationEXPERIMENTALNL-201 given as monotherapy by intravenous administration testing ascending doses and two different schedules.
Part 2: NL201 Monotherapy Expansion CohortsEXPERIMENTALNL-201 given as monotherapy by intravenous administration in indication specific cohorts at a dose and schedule determined in Part 1.
Part 3: NL-201 in Combination with Pembrolizumab Dose EscalationEXPERIMENTALNL-201, in combination with a set Pembrolizumab dose, testing ascending doses and two different schedules
Part 4: NL-201 in Combination with Pembrolizumab Expansion CohortsEXPERIMENTALNL-201 in combination with Pembrolizumab in indication specific cohorts at a dose and schedule determined in Part 3

Interventions

NameTypeDescription
NL-201DRUGNL-201 is a de novo protein therapeutic.
Pembrolizumab Injection [Keytruda]DRUGA programmed death receptor-1 (PD-1)-blocking antibody
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Patients with measurable disease * Patients with Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 * At least 6 weeks from any prior nitrosurea or mitomycin C therapy; at least 4 weeks from any other prior chemotherapy or checkpoint inhibitor; at least 2 w...

Countries:United StatesAustraliaCanada
Unlock Eligibility Criteria

Competitive Landscape -Other Solid Tumors 9 trials (matched to "Solid Tumor")

Frequently asked questions about NL-201

What is NL-201 used for?

NL-201 is an investigational small molecule being studied for the treatment of advanced solid tumors. It is being developed as a potential therapy for patients with relapsed or refractory cancer, both as a monotherapy and in combination with pembrolizumab. It has not been approved for any indication.

What does NL-201 target?

NL-201 targets IL-2 and IL-15, which are cytokines involved in immune system signaling. By engaging these cytokine pathways, NL-201 is designed to stimulate an anti-tumor immune response. It is a small molecule modality rather than a conventional antibody or recombinant cytokine therapy.

Who is developing NL-201?

NL-201 is being developed by Neurogene Inc., which trades under the ticker NGNE. The company is advancing the asset as an oncology candidate. Neurogene is the sponsor responsible for the clinical development program evaluating NL-201 in patients with advanced solid tumors.

What phase is NL-201 in?

NL-201 is in Phase 1 clinical development. It is an investigational agent and has not been approved by the FDA or any other regulatory authority. The Phase 1 program has been completed, with no trials currently active.

What clinical trials is NL-201 in?

NL-201 has been evaluated in one clinical trial, NCT04659629, titled NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory Cancer. This completed Phase 1 study enrolled 59 participants across the United States, Australia, and Canada in advanced solid tumor indications.