Pracinostat Hydrochloride Drug Asset Due Diligence Report 2026: Clinical, IP, Market, Deals, and Go/No-Go
This Pracinostat Hydrochloride Drug Asset Due Diligence Report was built with PatSnap Life Sciences MCP workflows. Drug & Asset MCP establishes identity, ownership and stage; Clinical Trials MCP checks design, endpoints and readouts; Company & Deal Intelligence MCP reconstructs transaction precedent. Explore the MCP servers used in this report.
Decision date: 15 July 2026. Currency fields are presented in US$ millions as returned by the deal dataset. This is a screening memorandum, not legal, medical, patent or investment advice.
Discontinued
Highest phase
17
Registered trials
18
Result records
4
Matched deals
Executive recommendation: HOLD / OPTION
Decision memo
Preserve optionality until clinical differentiation, transaction economics, and freedom-to-operate are sufficiently de-risked.
The central underwriting question is whether Pracinostat Hydrochloride can convert its Small molecule drug profile and HDAC1 x HDAC2 biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.
1. Asset identity and development status
| Asset | Pracinostat Hydrochloride (query alias: pracinostat) |
|---|---|
| Modality / target | Small molecule drug; HDAC1 x HDAC2; HDAC1 inhibitors, HDAC2 inhibitors, Epigenetic drug |
| Highest global status | Discontinued |
| Originator | SynCore Biotechnology Co., Ltd. |
| Active developers | Not disclosed |
The MCP disease footprint includes no disclosed indication. The highest-phase flag is a useful orientation point, but the licensing case depends on indication-level evidence and rights, not the global label alone.
2. Clinical program: design and endpoint audit
| Registry | Phase | Status | Enrollment | Lead primary endpoint |
|---|---|---|---|---|
| NCT03151408 | Phase 3 | Terminated | 406 | Overall Survival |
| RBR-84s2yw | Phase 3 | Recruiting | Not disclosed | Not disclosed |
| NCT03848754 | Phase 1 | Completed | 14 | The number of subjects experiencing a dose-limiting toxicity. |
The trial set should be diligenced for randomization, comparator relevance, endpoint hierarchy, analysis population, multiplicity, geographic mix and readout timing. For early-stage studies, safety and dose selection can be as decision-critical as response rate.
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3. Clinical readouts: what is known
A Phase III, Double-Blind, Placebo-Controlled, Multicenter, Randomized Study Of Pracinostat In Combination With Azacitidine In Patients ≥18 Years With Newly Diagnosed Acute Myeloid Leukemia Unfit For Standard Induction Chemotherapy
Phase 3; n=406; evaluation: not stated. Reported fields: OS(Median) = 303 days (95% Confidence Interval, 209 - 400); OS(Median): P-Value = 0.8275; OS(Median) = 303 days (95% Confidence Interval, 248 - 346)
A Two-Stage, Open-Label Phase 2 Study of Pracinostat and Azacitidine in Patients With IPSS-R High and Very High Risk Myelodysplastic Syndromes Previously Untreated With Hypomethylating Agents
Phase 2; n=64; evaluation: not stated. Reported fields: ORR = 35.9 percentage of subjects (95% Confidence Interval, 24.3 - 48.9); -; -
A Phase II Open-Label, Single-arm, Two-Stage, Multicenter Trial of Pracinostat in Combination With Azacitidine in Elderly (Age 65 Years or Older) Patients With Newly Diagnosed Acute Myeloid Leukemia (AML)
Phase 2; n=50; evaluation: not stated. Reported fields: Best Response Rate = 26 Participants ; -; -
These fields are structured evidence signals, not a substitute for statistical review. The next diligence pass should reconcile denominators, confidence intervals, follow-up, censoring, dose cohorts and treatment-emergent toxicity against the original abstract, registry and protocol.
4. Market and competitive position
Pracinostat Hydrochloride addresses its disclosed development indications. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—Small molecule drug—must demonstrate a benefit large enough to offset class-specific safety and operational costs.
The strongest market-validation signal in this screen is partner behavior: 4 matched transaction record(s) indicate that sophisticated counterparties have assigned strategic value to the asset or its rights. That does not establish net present value; probability of success, remaining R&D spend, royalties, cost sharing and territorial scope still need modeling.
5. Transaction precedent and economics
| Date | Transaction | Phase at deal | Disclosed economics |
|---|---|---|---|
| 2019-09-02 | Helsinn signs exclusive distribution and license agreements with Blanver and Varifarma for Pracinostat in South America | Phase 3 | Financial terms not disclosed |
| 2019-07-31 | Helsinn and Endo Announce Agreement for Paladin Labs Inc. to Commercialize Pracinostat in Canada | Phase 3 | Financial terms not disclosed |
| 2018-12-21 | Helsinn Group grants exclusive licensing rights to MENARINI for Pracinostat | Phase 3 | Financial terms not disclosed |
Headline values are not directly comparable. Diligence should normalize upfront cash, equity, development and sales milestones, tiered royalties, opt-in mechanics, cost sharing, change-of-control clauses and geography.
6. IP and freedom-to-operate screen
The milestone feed surfaced a patent-application signal described as “Novel forms of pracinostat dihydrochloride”. The milestone feed surfaced a patent-application signal described as “Crystalline polymorphs of pracinostat and pracinostat salts”.
The claim chart should separately test composition or sequence coverage, formulation and dosing, indication and biomarker claims, combinations, manufacturing know-how, prosecution history, term extensions and third-party blocking rights. Confirm that licensed patents, data and know-how track every granted territory and field.
7. Principal risks and diligence gates
- Therapeutic index across KRAS/BRAF mutation subgroups
- On-target toxicity and adaptive MAPK resistance
- Lack of matched efficacy results in the current MCP result set
Cross-functional diligence should also test CMC comparability, supply chain, pharmacovigilance, regulatory correspondence, data integrity, investigator concentration, partner obligations and change-of-control restrictions.
8. Final go/no-go memorandum
HOLD / OPTION
Preserve optionality until clinical differentiation, transaction economics, and freedom-to-operate are sufficiently de-risked.
Required pre-signing gates: reproduce key efficacy analyses; complete an indication-specific safety review; run a full patent-family and freedom-to-operate search; model risk-adjusted economics by territory; and reconcile all rights, sublicenses and encumbrances.
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Data provenance: PatSnap Drug & Asset MCP, Clinical Trials MCP, and Company & Deal Intelligence MCP; accessed 15 July 2026. Counts and status fields may change as source records update.
