Berotralstat Hydrochloride Drug Asset Due Diligence Report 2026: Clinical, IP, Market, Deals, and Go/No-Go
This Berotralstat 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.
Approved
Highest phase
15
Registered trials
36
Result records
6
Matched deals
Executive recommendation: GO
Decision memo
Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.
The central underwriting question is whether Berotralstat Hydrochloride can convert its Small molecule drug profile and KLKB1 biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.
1. Asset identity and development status
| Asset | Berotralstat Hydrochloride (query alias: berotralstat) |
|---|---|
| Modality / target | Small molecule drug; KLKB1; KLKB1 inhibitors |
| Highest global status | Approved |
| Originator | BioCryst Pharmaceuticals, Inc. |
| Active developers | BioCryst Ireland Ltd., OrphanPacific, Inc., BioCryst Pharmaceuticals, Inc. |
The MCP disease footprint includes Hereditary Angioedema, Hereditary Angioedema Types I and II. 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 |
|---|---|---|---|---|
| NCT04933721 | Phase 3 | Enrolling by invitation | 139 | Number and proportion of subjects with a treatment-related TEAE |
| NCT05453968 | Phase 3 | Active, not recruiting | 29 | Area Under the Plasma Concentration-Time Curve From Time 0 to the Last Measurable Concentration (AUC0-last) of Berotralstat |
| NCT03873116 | Phase 3 | Completed | 19 | Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) |
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
Impact of Berotralstat on Hereditary Angioedema Attack Rates in Patients with C1-Inhibitor Deficiency: Real-World Evidence Stratified by Prior Long-Term Prophylaxis
Not Applicable; n=466; evaluation: Positive. Reported fields: attack rate(12-month) = -1.32 month ; attack rate(12-month) = -1.97 month
Oral Berotralstat Reduces the Rate of Moderate and Severe Attacks and Percentage of Days with HAE Symptoms Over 48 Weeks in Children Aged 2 to Less Than 12 Years: Interim Data from APeX-P
Phase 3; n=29; evaluation: Positive. Reported fields: AE = Adverse events (AE) were consistent with those in prior berotralstat studies. No patients discontinued due to AEs ; AE = Adverse events (AE) were consistent with those in prior berotralstat studies. No patients discontinued due to AEs
Reductions in Healthcare Resource Utilization Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency Following the Initiation of Berotralstat
Not Applicable; n=207; evaluation: Positive. Reported fields: Hospitalizations = 0.6 per patient-year
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
Berotralstat Hydrochloride addresses Hereditary Angioedema, Hereditary Angioedema Types I and II. 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: 6 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 |
|---|---|---|---|
| 2025-06-27 | Neopharmed Gentili Completes Acquisition of European ORLADEYO® Business from BioCryst Pharmaceuticals | Approved | US$250.0M upfront; US$14.0M milestones |
| 2023-07-19 | BioCryst Selects Er-Kim Pharmaceuticals as Commercial Partner for ORLADEYO® (berotralstat) in Turkey | Approved | Financial terms not disclosed |
| 2023-01-23 | BioCryst Selects Swixx BioPharma as Commercial Partner for ORLADEYO® (berotralstat) in Central and Eastern Europe | Approved | 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 “Solid state forms of berotralstat”.
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
GO
Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.
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.
