A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)

1. Is the biology right?

• Somatic driver evidence: no · None of ODC1, PTGS2, PTGS1 has somatic-mutation evidence in classic familial adenomatous polyposis

  Rule: Open Targets has somatic-mutation evidence for the target in this cancer. Source: Open Targets: ODC1 in classic familial adenomatous polyposis, 2026-11-11
• Human genetic evidence: no · No germline or somatic evidence for ODC1, PTGS2, PTGS1 in classic familial adenomatous polyposis

  Rule: Any germline or somatic human genetic evidence for the target in this cancer, counted apart from animal-model and literature evidence. Source: Open Targets: ODC1 in classic familial adenomatous polyposis, 2026-11-11
• Pediatric target: yes · ODC1 on the FDA list

  Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets); membership alone neither requires nor waives a study. Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
• Haematologic cancer: no · Solid tumour

  Rule: From the indication, or the registry conditions when no indication matches. Source: ClinicalTrials.gov NCT01245816, 2026-11-11

  Published context: Likelihood of approval from phase 1: 7.5% haematologic vs 4.6% solid (BIO / Informa / QLS 2021, 2011–2020). Population-level, not a prediction.

• ODC1 (via Eflornithine): Evidence types in classic familial adenomatous polyposis: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc. · Open Targets: ODC1 in classic familial adenomatous polyposis
• PTGS2 (via Sulindac): Evidence types in classic familial adenomatous polyposis: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc. · Open Targets: PTGS2 in classic familial adenomatous polyposis
• PTGS1 (via Sulindac): Evidence types in classic familial adenomatous polyposis: literature, animal_model, clinical; literature and animal_model listed separately from human genetics. Datasources: clinical_precedence, impc, europepmc. · Open Targets: PTGS1 in classic familial adenomatous polyposis

• Not loaded on this app yet: Normal-tissue expression in vital organs. Source: Human Protein Atlas (ticket 36).
• Not loaded on this app yet: Pan-essential in cell lines. Source: DepMap (ticket 36).
• Not loaded on this app yet: Approved drug on this target. Source: Drugs@FDA (ticket 30).

2. Can the result be trusted?

• Randomised: yes · Randomised allocation

  Rule: Registry allocation field. Source: ClinicalTrials.gov NCT01245816, 2026-11-11
• Small denominator: yes · 0 enrolled; each patient moves a rate by 100 ÷ 0 = – points

  Rule: Enrolment under 30; the arithmetic is shown, not a benchmark. Source: ClinicalTrials.gov NCT01245816, 2026-11-11
• Primary endpoint is overall survival: no · Efficacy of Eflornithine plus Sulindac compared to Eflornithine alone and Sulindac alone determined by change in the …

  Rule: A registered primary outcome measure names overall survival. Source: ClinicalTrials.gov NCT01245816, 2026-11-11

  Published context: Non-OS primary endpoint: odds ratio 2.79 for a positive phase 3 (Otsuka 2021, 400 phase 3 oncology trials, to 2020). Shown with the next figure: a surrogate is easier to hit, and often a weak guide to survival.

  Published context: 52% of surrogate-to-OS correlations are low (Prasad 2015, published to 2014). Shown with the figure above.
• Results overdue: no · Primary completion expected 2013-05; not yet due

  Rule: Primary completion (actual) more than 12 months ago and no results posted to the registry. Source: ClinicalTrials.gov NCT01245816, 2026-11-11

• Not loaded on this app yet: Treated and evaluable counts, assessor, confirmed response, follow-up and data cutoff. Source: Readout extraction (ticket 32).
• Not loaded on this app yet: Primary endpoint changed between registry versions. Source: Registry versions in the store (ticket 32).

3. What must it beat?

• Not loaded on this app yet: Control-arm result and approved therapies for the same segment, with label links. Source: Drugs@FDA, FDA labels and readouts (tickets 30, 32).

4. Too crowded, or too late?

• Stopped on this target: 7 terminated, withdrawn or suspended trials of drugs against ODC1; 2 give no reason. Reasons are quoted as the registry gives them on the target page. · ODC1: what already stopped

• Not loaded on this app yet: Programs on the target by phase and months behind the leader. Source: Programs and Drugs@FDA (ticket 33).

5. Can it be approved, and stay approved?

• Single-country: not published · The record lists no sites.

  Rule: All listed sites are in one country; the share of US sites is shown. Source: ClinicalTrials.gov NCT01245816, 2026-11-11
• EU paediatric investigation plan: yes · EMEA-001518-PIP03-19 (eflornithine hydrochloride;sulindac, Treatment of Familial Adenomatous Polyposis): RP: decision refers to a refusal on a proposed Paediatric Investigation Plan, 6 Dec 2019

  Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA EMEA-001518-PIP03-19, 2026-11-11
• EU orphan designation: yes · EU/3/11/902 withdrawn 27 Sept 2011: Treatment of neuroblastoma; EU/3/21/2429 positive 20 May 2021: Treatment of neuroblastoma; EU/3/20/2384 positive 9 Dec 2020: Treatment of fragile X syndrome; EU/3/10/779 positive 20 Sept 2010: Treatment of familial adenomatous polyposis (FAP); +3 more

  Rule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
• Authorised in the EU: yes · Ifinwil: application withdrawn, orphan medicine; Vaniqa: authorised since 19 Mar 2001; Flynpovi: application withdrawn, orphan medicine

  Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
• FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link

  Rule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
• FDA pediatric oncology approval: yes · Eflornithine [Iwilfin], 13 Dec 2023: To reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy

  Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
• Registered studies open to children: yes · 27 interventional studies of Eflornithine, Sulindac include patients under 18; 9 are recruiting, not yet recruiting or active

  Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

• Caveat: Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where the sites are is a fact about the record, not a judgement of its quality.
• Rare pediatric disease voucher: FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. · FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs

• Not loaded on this app yet: Precedent approvals for target, modality and endpoint; accelerated-approval status. Source: Drugs@FDA and FDA accelerated-approval tables (ticket 30).

6. Can the sponsor finish?

• Sponsor: Cancer Prevention Pharmaceuticals, Inc. (industry) · ClinicalTrials.gov NCT01245816
• study director: Alfred M. Cohen, M.D., Cancer Prevention Pharmaceuticals, Inc. · Person page

• Not loaded on this app yet: Filed cash, quarterly spend and the runway arithmetic. Source: SEC XBRL company facts (ticket 37).
• Not loaded on this app yet: Sponsor's completed vs stopped oncology trials and results-reporting record. Source: Registry, by company (ticket 37).

What public data cannot tell you

Public records cannot show: sales forecasts; a probability of success for this asset; full deal terms and net prices; FDA designations and action dates before approval (shown only as company-reported); clinical holds unless the company disclosed them; manufacturing; patents on unapproved drugs; whether preclinical findings have been replicated.