Cryopreserved MMUD BM With PTCy for Hematologic Malignancies

1. Is the biology right?

• Somatic driver evidence: no · None of FKBP1A, IMPDH1, IMPDH2 has somatic-mutation evidence in childhood acute lymphoblastic leukemia

  Rule: Open Targets has somatic-mutation evidence for the target in this cancer. Source: Open Targets: FKBP1A in childhood acute lymphoblastic leukemia, 2026-11-11
• Human genetic evidence: no · No germline or somatic evidence for FKBP1A, IMPDH1, IMPDH2 in childhood acute lymphoblastic leukemia

  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: FKBP1A in childhood acute lymphoblastic leukemia, 2026-11-11
• Pediatric target: no · Not 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: yes · Haematologic

  Rule: From the indication, or the registry conditions when no indication matches. Source: ClinicalTrials.gov NCT05170828, 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.

• Background therapy: Cyclophosphamide, Fludarabine, Mesna, Filgrastim: standard agents, not counted in these flags.
• FKBP1A (via Sirolimus): Open Targets has no association between FKBP1A and childhood acute lymphoblastic leukemia. · Open Targets: FKBP1A in childhood acute lymphoblastic leukemia
• IMPDH1 (via Mycophenolate Mofetil): Open Targets has no association between IMPDH1 and childhood acute lymphoblastic leukemia. · Open Targets: IMPDH1 in childhood acute lymphoblastic leukemia
• IMPDH2 (via Mycophenolate Mofetil): Open Targets has no association between IMPDH2 and childhood acute lymphoblastic leukemia. · Open Targets: IMPDH2 in childhood acute lymphoblastic leukemia

• 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: no · Non-randomised

  Rule: Registry allocation field. Source: ClinicalTrials.gov NCT05170828, 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 NCT05170828, 2026-11-11
• Primary endpoint is overall survival: yes · Neutrophil engraftment; Cumulative incidence and kinetics of neutrophil and platelet recovery; Overall survival (OS)

  Rule: A registered primary outcome measure names overall survival. Source: ClinicalTrials.gov NCT05170828, 2026-11-11
• Results overdue: no · Primary completion expected 2024-03; not yet due

  Rule: Primary completion (actual) more than 12 months ago and no results posted to the registry. Source: ClinicalTrials.gov NCT05170828, 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: 578 terminated, withdrawn or suspended trials of drugs against FKBP1A; 8 of the 200 most recent give no reason. Reasons are quoted as the registry gives them on the target page. · FKBP1A: 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 NCT05170828, 2026-11-11
• EU paediatric investigation plan: yes · EMEA-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-003168-PIP01-21 (sirolimus, Treatment of tuberous sclerosis complex): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 2 Dec 2022; EMEA-002982-PIP01-21 (sirolimus, Treatment of ornithine transcarbamylase deficiency): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 10 Jun 2022; EMEA-001416-PIP01-12-M03 (sirolimus, Treatment of chronic non-infectious uveitis): PM: decision on the application for modification of an agreed PIP, 11 Aug 2021; +1 more

  Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
• EU orphan designation: yes · EU/3/25/3189 positive 9 Jan 2026: Treatment of osteosarcoma; EU/3/25/3051 positive 16 Apr 2025: Treatment of familial adenomatous polyposis (FAP); EU/3/13/1204 positive 13 Nov 2013: Prevention of arteriovenous access dysfunction in haemodialysis patients; EU/3/17/1910 positive 23 Aug 2017: Treatment of tuberous sclerosis; +11 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 · Hyftor: authorised since 15 May 2023, orphan medicine; CellCept: authorised since 14 Feb 1996; Rapamune: authorised since 13 Mar 2001; Mycophenolate mofetil Teva: authorised since 21 Feb 2008; +5 more

  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 · Busulfan [Busulfex], 13 Jan 2003: For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia

  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 · 1071 interventional studies of Busulfan, Sirolimus, Mycophenolate Mofetil include patients under 18; 216 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: Ossium Health, Inc. (industry) · ClinicalTrials.gov NCT05170828

• 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.