NSS-01 / PRECLINICAL EVIDENCE

The evidence
behind NSS-01.

Pancreatic and colorectal cancer are co-equal preclinical research priorities for NSS-01. Explore the pancreatic regression findings and comparative colorectal studies below, together with supporting cell assays containing human serum albumin.

PANCREATIC CANCER

Tumor regression
evaluated in vivo.

Both treated groups showed median tumor-volume regression in the company’s pancreatic research.

Review pancreatic findings

COLORECTAL CANCER

Comparative activity
across five comparisons.

NSS-01 showed 42–65% lower mean tumor growth than SN-38 or irinotecan across five matched colorectal comparisons.

Review colorectal findings

01 / PANCREATIC IN VIVO RESEARCH

Median tumor regression
in both treated groups.

Both NSS-01-treated groups showed median tumor-volume regression in the company’s pancreatic in vivo research.

The greatest individual primary-tumor regression was 82.2% in the 28-day study.

PANCREATIC STUDY 01

28-day study

69.7%Median tumor-volume regression

Treated group size (n): 6
Tumors smaller than baseline: 4 of 6
Tumors larger than baseline: 2 of 6

PANCREATIC STUDY 02

21-day study

15.5%Median tumor-volume regression

Treated group size (n): 5
Tumors smaller than baseline: 3 of 5
Tumors larger than baseline: 2 of 5

Individual primary-tumor volume changes from baseline, summarized separately for each treated group.

View individual results and mean calculations

Each primary tumor’s volume is compared with its own pretreatment baseline. Negative values indicate regression; positive values indicate growth.

28-day study: primary-tumor changes from baseline (treated group, n=6)
Study ID / summary	Tumor-volume change
C1 L1	−64.4%
C1 L2	+107.5%
C1 R1	−78.3%
C1 R2	−75.1%
C2 L1	−82.2%
C2 L2	+40.5%
Mean of individual percentage changes	−25.3%
Median individual percentage change	−69.7%

21-day study: primary-tumor changes from baseline (treated group, n=5)
Study ID / summary	Tumor-volume change
C5 L1	+71.6%
C5 L2	−15.5%
C5 R1	−63.0%
C5 R2	+88.4%
C6 R1	−20.6%
Mean of individual percentage changes	+12.2%
Median individual percentage change	−15.5%

Individual percentage change = 100 × (endpoint volume ÷ baseline volume − 1). The mean averages the individual percentage changes; the median is their middle value. Summary values are calculated from unrounded measurements.

Two-site pancreatic challenge

Treatment began after pancreatic cancer cells had been introduced at two anatomically separate sites.

28-day study: Cells were introduced at the right flank and right rib cage. Treatment began 15 days after the first inoculation and 8 days after the second.

21-day study: Cells were introduced at the right flank and left shoulder. Treatment began 22 days after the first inoculation and 8 days after the second.

The second site in each study was deliberately inoculated. These implanted sites are distinct from metastases arising through spread of the cancer.

Metastatic observations

The company reports no secondary or metastatic lesions detected in NSS-01-treated groups during the study periods, while untreated controls showed secondary and/or metastatic tumors. Assessments included luminescent imaging and necropsy.

Tolerability in the evaluated regimens

The company reports no observed toxicity that limited dosing in the evaluated regimens.

Sources: Company pancreatic data summary, Experiment 9; published PCT application, paragraphs 336–351; company report on metastatic disease evaluation; company-reported tolerability observations.

02 / COLORECTAL IN VIVO RESEARCH

Lower mean tumor growth
across five comparisons.

Across five matched colorectal in vivo comparisons, NSS-01 produced lower mean tumor growth from baseline than SN-38 or irinotecan in the corresponding regimen. Reported relative reductions ranged from 42% to 65%.

The series examined activity under different conditions, including added human serum albumin, human-albumin-expressing models, older cohorts, and injected and oral dosing. The direction of the mean-growth difference persisted across the five comparisons.

Comparisons were analyzed separately; some regimens shared a control cohort. Percentages describe relative reductions in mean growth from baseline, distinct from tumor regression.

Reported reduction in mean tumor growth

Exp. 3

60%

Exp. 4

64%

Exp. 6

65%

Exp. 7

42%

Exp. 8

44%

Explore the five comparison models and regimens

Scroll horizontally to see the full table

Five matched comparisons: host, comparator and regimen context
Experiment	Preclinical model / HSA context	Matched comparator	Administration	Lower mean growth
3	NOD-SCID, HSA added	SN-38 + HSA	Single matched dose	60%
4	Nude model; HSA not specified in summary	Irinotecan	Subcutaneous	64%
6	HSA+ Rag− C57BL/6 model	SN-38	10 mg/kg total, split s.c. + i.p.	65%
7	Older HSA+ Rag− C57BL/6 model	SN-38	10 mg/kg total, split s.c. + i.p.	42%
8	Older HSA+ Rag− C57BL/6 model	SN-38	10 mg/kg total oral, split into four doses	44%

EXPERIMENT 6 / COMPARATOR STUDY

One study. Three comparator drugs.

Experiment 6 evaluated LS174T human colorectal tumors in an HSA-expressing Rag-negative C57BL/6 preclinical model. At day 14, NSS-01 had lower mean tumor growth from baseline than each of three comparator drugs, with n=8 per treatment group.

The NSS-01 group received a total study dose of 10 mg/kg. The table identifies each comparator’s total dose. Administration was split between intraperitoneal and subcutaneous routes.

Experiment 6: lower mean growth with NSS-01 versus each comparator at day 14 (n=8 per treatment group)
Comparator drug	Comparator total dose	NSS-01 total dose	Lower mean growth with NSS-01
SN-38	10 mg/kg	10 mg/kg	65%
Topotecan	10 mg/kg	10 mg/kg	84%
Irinotecan	20 mg/kg	10 mg/kg	83%

Rounded relative reductions in mean individual tumor growth from baseline, under the study-specific doses and administration routes shown.

View the mean growth values used in these comparisons

Experiment 6: mean of individual tumor-volume changes from baseline at day 14
Treatment group	Mean growth from baseline	Group size (n)
NSS-01	175.2%	8
SN-38	493.6%	8
Topotecan	1,111.8%	8
Irinotecan	1,025.0%	8

Relative reduction = 100 × (1 − NSS-01 mean growth ÷ comparator mean growth), calculated from unrounded values. All NSS-01 tumors remained above baseline at day 14.

The colorectal research program

Colorectal cancer is a core development priority for NSS-01. Its comparative research examines activity under several host, albumin and administration conditions, giving a potential partner a defined set of experiments to evaluate and build upon.

The 42–65% range above uses one matched comparator per experiment. The three Experiment 6 comparisons are shown separately and are not additional independent studies. Experiment 8 evaluated an exploratory oral dosing regimen.

Discuss the colorectal study package

Sources: Company executive overview, slides 6–8; company data summary, Experiment 6 and Exp 7 and 8. Company-reported study data.

03 / HSA-CONTAINING CELL ASSAYS

Three models.
Direct comparisons.

NSS-01 had the lowest mean IC₅₀ among the four compounds in each of three HSA-containing cell models.

Cells were exposed for 24 hours with 20 mg/mL HSA. Mean values were measured by triplicate MTT assay. Lower IC₅₀ indicates greater antiproliferative potency in this assay.

Mean IC₅₀ (nM)

EJ-1 human bladder cancer cells / 24 h / 20 mg/mL HSA

0150300450 nM

SN-38 is the active metabolite of irinotecan.

View all assay values

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Mean IC₅₀ (nM), 24-hour exposure with 20 mg/mL HSA
Cell model	NSS-01	SN-38	Topotecan	Irinotecan
Bladder EJ-1	7.0	44.8	346.6	330.0
Breast MCF-7	29.0	45.7	408.3	49.5
Ovarian A2780	21.4	118.1	121.2	407.5

Source: Company executive overview, slide 5 and speaker notes; published PCT application, Figure 43. Company-reported assay means.

THE FULL STUDY PACKAGE

A foundation for
the next stage.

Pancreatic and colorectal in vivo studies and HSA-containing cell assays form the preclinical foundation for NSS-01 development.

Additional cell-based research spans ovarian, bladder, breast and glioblastoma models. Study protocols, methods and individual study data are available for partner diligence.

Download the executive overview Discuss the study package →

THE NEXT CHAPTER

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We welcome licensing, acquisition of the NSS-01 program, or co-development with a pharmaceutical or biotechnology partner to advance NSS-01 toward clinical development.

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The evidence behind NSS-01.

Tumor regression evaluated in vivo.

Comparative activity across five comparisons.

Median tumor regression in both treated groups.