IntraBio Startup Diligence Research Report
The positive thesis is a rare-neurology platform with an approved U.S./EU NPC product, public clinical-registry history, A-T sNDA Priority Review, patent assets, and a small focused team. The diligence thesis depends on proving commercial traction, payer access, durable IP, regulatory execution, manufacturing/supply resilience, safety compliance, and enough capital/team capacity to scale.
IntraBio Startup Diligence Research Report
IntraBio is eligible for a public-evidence unicorn diligence report: CB Insights lists a $1B IntraBio unicorn row, LinkedIn describes the company as privately held in Austin, and public regulatory sources verify AQNEURSA/Aqneursa approvals for NPC. Underwriting should remain conditional because public evidence does not disclose financials, revenue, cap table, pricing, payer coverage, customers, suppliers, or complete HR/contract records.
Public evidence centers on AQNEURSA/levacetylleucine; commercial success depends on small rare-disease populations, payer access, prescriber adoption, and post-approval execution.
Diligence request: Review revenue by indication/region, payer coverage, launch dashboard, specialty pharmacy/distributor data, patient-start funnel, and gross-to-net.
A-T and additional pipeline programs depend on pending submissions, trial activation, protocol execution, and regulator acceptance of endpoints/safety.
Diligence request: Obtain FDA/EMA correspondence, meeting minutes, clinical study reports, site activation status, safety database, and regulatory strategy by indication.
Only a $1B unicorn-list valuation anchor is public; audited financials, revenue, burn, cash, debt, ownership, and current valuation marks are not public.
Diligence request: Require full data-room financials, cap table, financing docs, tax records, debt, 409A/fair-value history, and board materials before relying on valuation.
MIPLYFFA, miglustat, supportive care, and future investigational products create competition and reimbursement comparisons in NPC; A-T lacks approved therapies but approval is pending.
Diligence request: Request market research, pricing/access analysis, physician/payer interviews, and competitive response planning.
DailyMed label includes embryo-fetal harm warnings, pregnancy considerations, and drug-interaction issues that require strong compliance processes.
Diligence request: Review pharmacovigilance system, safety reports, complaints, medical affairs training, pregnancy prevention counseling, and labeling compliance.
IntraBio has a public patent-infringement action against Tesseract and lists multiple AQNEURSA patents; litigation cost, validity, enforceability, and counterclaims remain diligence issues.
Diligence request: Have IP counsel review complaint, docket, settlement posture, patent validity/expiry, licenses, FTO, and insurance coverage.
LinkedIn shows 11-50 employees; BusinessWire publicly identifies Mallory Factor as CEO, but full management depth, turnover, compensation, and retention are not public.
Diligence request: Request org chart, HRIS census, turnover, compensation, option plan, management bios/references, and succession plan.
No public source disclosed top customers, payer mix, specialty pharmacy concentration, API/CMO suppliers, or inventory constraints.
Diligence request: Request top account/payer/supplier schedules, contracts, purchase volumes, quality agreements, business continuity plans, and terminated relationships.
Public evidence verifies a $1B CB Insights unicorn valuation and private-company status, but financial statements, current valuation, cap table, revenue, burn, debt, taxes, and unit economics are not publicly verifiable.
not publicly verifiable confidence: high
No audited income statements, balance sheets, cash-flow statements, management reports, sales breakdowns, backlog, or AR aging were found in reviewed public sources.
| metric | public signal | verification status | diligence request |
|---|---|---|---|
| Revenue / product sales | No revenue, ARR, net sales, gross-to-net, or payer mix disclosed. | not_publicly_verifiable | Audited financials, product revenue by indication/region/channel, gross-to-net, and monthly close packages. |
| Cash runway / burn | Not publicly disclosed. | not_publicly_verifiable | Cash balance, burn, budget vs actual, financing plan, debt, and covenant compliance. |
| R&D / platform investment | Company says platform stems from >$350M of worldwide organizational investments. | unverified | Trace source and ownership of investment, grants, licenses, and capitalized R&D. |
not publicly verifiable confidence: medium
Public sources show potential growth drivers from approved NPC product and pending A-T/CACNA1A programs, but no three-year projections or assumptions were public.
not publicly verifiable confidence: medium
LinkedIn indicates IntraBio is privately held and CourtListener shows an IntraBio Limited relationship in litigation, but current shares, ownership, options, warrants, debt, and off-balance-sheet obligations are private.
| stakeholder or entity | public position | verification status | caveat or request |
|---|---|---|---|
| IntraBio Inc | Privately held; Austin headquarters; 11-50 employees | verified self-reported profile | Certificate of good standing, entity structure, subsidiary list, and board records. |
| IntraBio Limited | Co-plaintiff with IntraBio Inc. in patent litigation | verified legal-record signal | Map subsidiary ownership, IP ownership, and intercompany licenses. |
| Stockholders, optionholders, warrant/note holders | Not publicly disclosed | not_publicly_verifiable | Fully diluted cap table, preference stack, warrants/notes, and option vesting schedule. |
partially verified confidence: medium
CB Insights provides the valuation/date anchor and IntraBio claims broad historical platform investment, but financing history, tax positions, accounting policies, and current basis for each round are not public.
| date | event | amount or valuation | verification status | diligence request |
|---|---|---|---|---|
| 2024-03-23 | CB Insights unicorn-list inclusion | $1B valuation | verified market-database anchor | Financing documents, valuation method, preference stack, and current fair value. |
| Undisclosed | Platform investment history claim | > $350M worldwide organizational investment claim | unverified company claim | Funding ledger, licenses, spend ownership, and capitalization accounting. |
| Undisclosed | Equity rounds, debt, warrants, SAFEs, option pool | Not publicly disclosed | not_publicly_verifiable | Cap table, debt schedule, board approvals, investor rights, and option plan. |
No public round-by-round financing history was found beyond the unicorn valuation anchor.
Does not imply revenue or financing rounds between milestones.
Public evidence verifies AQNEURSA/levacetylleucine as an approved NPC therapy in the U.S. and EU and identifies multiple clinical-stage expansion programs, while pricing, profitability, manufacturing, and adoption remain private.
verified confidence: high
AQNEURSA is an approved levacetylleucine oral-suspension product for NPC neurological manifestations in defined U.S./EU populations, and IB1001/levacetylleucine underpins additional rare-neurology programs.
| product or program | audience or indication | public status | verification status |
|---|---|---|---|
| AQNEURSA (levacetylleucine) | NPC neurological manifestations; U.S. adults and pediatric patients ≥15 kg | FDA-approved label; oral suspension | verified |
| Aqneursa | EU NPC neurological manifestations; adults and children 6+ weighing ≥20 kg | EMA marketing authorization valid throughout EU on 2026-01-19 | verified |
| IB1001 / N-acetyl-L-leucine pipeline | NPC, GM2, A-T, CACNA1A-related disorders, other rare/common neurological disorders | Clinical-stage expansion programs | partially_verified |
| product | public pricing | commercial status | verification status | diligence request |
|---|---|---|---|---|
| AQNEURSA | Not found in reviewed public sources | U.S. approved; EU authorised | approval verified; pricing not_publicly_verifiable | WAC, gross-to-net, coverage, specialty pharmacy terms, patient assistance, inventory and returns. |
| MIPLYFFA (arimoclomol) | Not assessed in this run | NPC indication in combination with miglustat for age 2+ | verified competitor indication | Compare payer access, label breadth, physician preference, safety, and combination burden. |
| Miglustat / generics | Not assessed in this run | Multiple miglustat products listed; EMA describes Aqneursa combination/monotherapy positioning | verified availability signal | Assess standard-of-care role, generic pricing, and combination therapy impact. |
For a commercial-stage rare-disease biopharma, public sources define patient/provider segments and regulators but do not disclose top customers, payers, distributors, suppliers, revenue by account, or severed relationships.
not publicly verifiable confidence: high
Public evidence identifies eligible patient populations by label, not named customers/accounts or top 15 customer lists.
| segment or account | public evidence | revenue weight | verification status | diligence request |
|---|---|---|---|---|
| NPC patients/providers in U.S. | DailyMed label defines approved patient population ≥15 kg. | Not publicly disclosed | verified patient indication; revenue not_publicly_verifiable | Patient starts, prescriber count, payer coverage, adherence, discontinuation, and net sales. |
| EU NPC patients/providers | EMA defines adults/children 6+ weighing ≥20 kg. | Not publicly disclosed | verified patient indication; revenue not_publicly_verifiable | Country launch sequence, reimbursement, distribution, and pharmacovigilance arrangements. |
| Top 15 customers / payers / distributors | No named customers, payers, distributors, or revenue concentration found. | Not publicly disclosed | not_publicly_verifiable | Top customer/payer/specialty-pharmacy revenue, contracts, churn/nonrenewals, and references. |
partially verified confidence: medium
Public relationships are primarily regulators and clinical-trial ecosystem nodes; commercial partners are not named.
| relationship | nature | public evidence | revenue contribution | gap or request |
|---|---|---|---|---|
| FDA | Regulator for U.S. approval and A-T sNDA review | DailyMed label; BusinessWire Priority Review announcement. | Not applicable | FDA correspondence, approval letter, PMCs, and sNDA review status. |
| EMA | EU regulatory authority / marketing authorisation source | EMA EPAR for Aqneursa. | Not applicable | EU commitments, pharmacovigilance, country reimbursement status. |
| Clinical trial sites | Trial execution network | CB profile says 12 multinational CACNA1A sites being activated. | Not applicable | Site list, activation status, CRO/vendor contracts, enrollment dashboard. |
| Commercial distributors, specialty pharmacies, payers, CMOs | Likely commercialization/supply partners | Not named in reviewed public sources | Not publicly disclosed | Contracts, service levels, quality agreements, rebates, and concentration. |
not publicly verifiable confidence: high
No revenue by customer, payer, account, distributor, channel, or geography is publicly disclosed.
not publicly verifiable confidence: medium
No public evidence of severed customer, partner, or supplier relationships was found in reviewed sources.
not publicly verifiable confidence: high
No top supplier list, purchase volumes, API/CMO names, quality agreements, or inventory position was public.
| supplier or dependency | role | public evidence | concentration risk | diligence request |
|---|---|---|---|---|
| API / drug product manufacturers | Manufacturing AQNEURSA/levacetylleucine | No manufacturer or CMO names disclosed. | Unknown; potentially high | API/DP supplier list, quality agreements, capacity, inventory, batch history, audits, and alternatives. |
| Regulatory and pharmacovigilance vendors | Safety reporting and compliance | Label requires adverse-event reporting; vendors not disclosed. | Unknown | PV agreements, case-processing metrics, SOPs, safety database, audits. |
| Clinical sites/CROs | Pipeline trial execution | Public sources reference multinational trials and 12 CACNA1A sites but not vendor names. | Medium | CRO/vendor contracts, site budgets, enrollment, protocol deviations, and monitoring reports. |
IntraBio competes in NPC against other approved/available therapies and seeks possible differentiation through A-T and other pipeline indications, but market share, payer preference, and pricing comparisons are not public.
partially verified confidence: medium
DailyMed and EMA evidence identify approved NPC comparators; BusinessWire and Clinical Trials Arena frame A-T as an unmet market with no currently approved therapies pending IntraBio review.
| competitor or comparator | segment | product overlap | differentiator | evidence |
|---|---|---|---|---|
| IntraBio AQNEURSA | NPC neurological manifestations; A-T pending | Target product | U.S. and EU NPC approval/authorisation; A-T Priority Review announced | DailyMed, EMA, BusinessWire. |
| MIPLYFFA (arimoclomol) | NPC neurological manifestations | Direct NPC approved therapy/comparator | Indicated in combination with miglustat for adults and pediatric patients age 2+ | DailyMed MIPLYFFA label. |
| Miglustat products | NPC-related standard/combo therapy context | Combination/standard-of-care comparator | Multiple marketed products; EMA describes Aqneursa combination/monotherapy positioning. | DailyMed search and EMA EPAR. |
| A-T no approved therapies cited | Ataxia-Telangiectasia | Potential first-approved expansion if sNDA succeeds | A-T unmet need; approval not yet granted | BusinessWire and Clinical Trials Arena. |
| axis | intrabio position | competitor position | evidence strength | diligence request |
|---|---|---|---|---|
| Label breadth | NPC U.S./EU; A-T under sNDA Priority Review | MIPLYFFA NPC age 2+ in combination with miglustat; miglustat products available | high for approved labels; medium for pending A-T | Compare label restrictions, payer policies, real-world prescriber adoption, and A-T review risk. |
| Clinical evidence | Multiple trials and A-T topline claims; full CSRs not public | Competitor clinical evidence not reviewed beyond labels | medium | Side-by-side efficacy/safety review by indication, endpoint, age group, and burden. |
| Access and economics | Pricing, gross-to-net, reimbursement, and channel data not public | Competitor pricing/access not assessed publicly in this run | low | Payer interviews, WAC/gross-to-net, hub metrics, specialty pharmacy data, and claims analysis. |
Coordinates are qualitative analyst judgments.
Regulatory approvals imply commercialization obligations in the U.S. and EU, but public sources do not disclose sales force productivity, payer coverage, channel contracts, budgets, or launch metrics.
partially verified confidence: medium
Public strategy signals are product/regulatory communications and rare-disease positioning; detailed distribution channels, marketing budgets, and launch plans are private.
| channel | region | public evidence | verification status | gap or request |
|---|---|---|---|---|
| Specialty pharma launch for AQNEURSA | United States | DailyMed confirms U.S. approved product and packager; no distributor named. | product verified; channel not_publicly_verifiable | Specialty pharmacy, hub, distributor, payer and prescriber launch dashboards. |
| EU commercialization / reimbursement | European Union | EMA marketing authorisation valid throughout EU on 2026-01-19. | authorization verified; country access not_publicly_verifiable | Country pricing/reimbursement, distributor, QPPV/PV agreements, and launch plan. |
| Clinical-trial recruitment / investigator network | Multinational | ClinicalTrials.gov records and CB profile show multinational trials/site activation. | partially_verified | Trial recruitment funnel, CRO/site contracts, enrollment, and investigator relationships. |
| signal | date | message | verification status | diligence request |
|---|---|---|---|---|
| Homepage positioning | Accessed 2026-06-10 | Leader in discovery/development of innovative drugs for rare neurodegenerative diseases. | verified as company claim | Brand tracking, website analytics, HCP targeting, patient support metrics. |
| A-T sNDA Priority Review press release | 2026-05-19 | FDA accepted sNDA, PDUFA 2026-09-19, claims Phase III endpoints met. | partially_verified company announcement | FDA acceptance letter, medical/legal review, and launch readiness. |
| LinkedIn public profile/activity | Accessed 2026-06-10 | 3,124 followers; posts on EMA variation, NPC conference support, CACNA1A authorization, and FDA sNDA. | verified social-profile signal | Owned-channel metrics, HCP/patient community engagement, compliance review. |
not publicly verifiable confidence: high
Major customer relationships and pipeline by account are not public; patient/provider segments are defined only by label and public communications.
partially verified confidence: medium
Likely new-business avenues include patient identification, HCP education, payer access, and label expansion, but public sources do not quantify funnel performance.
not publicly verifiable confidence: high
Sales compensation, quotas, sales cycle, and hiring plan are not publicly available.
not publicly verifiable confidence: high
Implementation capacity cannot be assessed from public information because budgets, staffing, vendors, and launch KPIs are private.
Public registry and company sources show a focused levacetylleucine/IB1001 R&D portfolio with completed and active trials, but full protocols, CSRs, safety datasets, costs, and regulatory correspondence are not public.
partially verified confidence: medium
IntraBio claims scientific founder expertise and management/regulatory experience, while public sources verify only sparse personnel detail.
| person or group | role or capability | public evidence | verification status | diligence request |
|---|---|---|---|---|
| Mallory Factor | President and Chief Executive Officer | BusinessWire quote attribution in sNDA press release. | verified | Employment agreement, biography, references, board role, and succession plan. |
| Scientific founders from University of Oxford and University of Munich | Scientific expertise in lysosomal function and intracellular calcium signaling | IntraBio mission page claim. | partially_verified company claim | Names, IP assignments, consulting agreements, invention assignments, conflict disclosures. |
| Management team and business consultants | Commercial/regulatory/manufacturing/clinical development experience | IntraBio mission page claim. | partially_verified company claim | Roster, contracts, responsibilities, compensation, retention and performance history. |
partially verified confidence: medium
ClinicalTrials.gov records and public announcements show NPC, GM2, A-T, and CACNA1A programs, including completed Phase II studies, a recruiting Phase III NPC study, an A-T sNDA, and a CACNA1A trial authorization excerpt.
| program | indication | status | key date or metric | verification status |
|---|---|---|---|---|
| AQNEURSA / levacetylleucine | NPC neurological manifestations | U.S. approved; EU authorised | Initial U.S. approval 2024; EU MA 2026-01-19 | verified |
| IB1001-301 / NCT05163288 | NPC | Phase III recruiting registry record | 53 estimated enrollment; primary completion 2030-06-12 estimated | verified registry metadata |
| IB1001-201 / NCT03759639 | NPC | Phase II completed | 33 actual enrollment; completed 2022-11-07 | verified registry metadata |
| IB1001-202 / NCT03759665 | GM2 gangliosidosis | Phase II completed | 30 actual enrollment; completed 2023-01-09 | verified registry metadata |
| IB1001-203 / NCT03759678 and A-T sNDA | Ataxia-Telangiectasia | Phase II terminated for pivot to Phase III; sNDA Priority Review announced | 17 actual enrollment; PDUFA target 2026-09-19 | partially_verified |
| CACNA1A-related disorders | CACNA1A-related disorders | Pivotal Phase III trial authorized across participating regions per CB profile excerpt | 12 multinational sites being activated per profile excerpt | partially_verified secondary excerpt |
| nct id | study | phase | status | enrollment |
|---|---|---|---|---|
| NCT05163288 | Pivotal N-acetyl-L-leucine on NPC | Phase 3 | Recruiting | 53 estimated |
| NCT03759639 | N-acetyl-L-leucine for NPC | Phase 2 | Completed | 33 actual |
| NCT03759665 | N-acetyl-L-leucine for GM2 | Phase 2 | Completed | 30 actual |
| NCT03759678 | N-acetyl-L-leucine for A-T | Phase 2 | Terminated; priority shifted to pivotal Phase III | 17 actual |
Public sources confirm privately held status, Austin headquarters, 11-50 employee range, and one named CEO, but full management roster, compensation, HRIS, turnover, and employee-relations data are private.
not publicly verifiable confidence: medium
No complete public org chart was available; a sparse public-functional org chart can be built from the CEO quote, mission page, and LinkedIn profile.
Reporting relationships are inferred functional placeholders, not verified legal reporting lines.
partially verified confidence: medium
LinkedIn reports an 11-50 employee range and Austin headquarters; historical/projected headcount by function/location is not public.
| function or metric | region | public evidence | verification status | diligence request |
|---|---|---|---|---|
| Company size | Austin, Texas / global operations unclear | LinkedIn self-reports 11-50 employees and Austin headquarters. | verified self-reported profile | Employee census by function/location, contractors, consultants, open roles, and payroll. |
| Clinical/regulatory/commercial expertise | U.S. and Europe per company page | Mission page claims management/consultants have manufacturing, clinical, and U.S./Europe approval experience. | partially_verified company claim | Functional org chart, CVs, regulatory responsibility matrix, vendor reliance. |
| Hiring plan / projected headcount | Not publicly disclosed | No careers/hiring detail was publicly verifiable in reviewed sources. | not_publicly_verifiable | Budgeted headcount plan, offer pipeline, attrition, key vacancies, and recruiting metrics. |
partially verified confidence: medium
One senior executive, Mallory Factor, is publicly identified as President and CEO; public source extraction did not provide complete senior-management biographies.
| name or role | public role | public evidence | verification status | diligence request |
|---|---|---|---|---|
| Mallory Factor | President and Chief Executive Officer | BusinessWire quote attribution. | verified | Full biography, employment terms, board role, references, compensation, equity. |
| Scientific founders from Oxford and Munich | Scientific founders / program expertise | IntraBio mission page claim; individual names not extracted because team page was CAPTCHA-limited. | partially_verified | Named founder roster, invention assignments, consulting agreements, publication/patent history. |
| Other executives, board, and commercial leaders | Not fully public in reviewed sources | LinkedIn gives company profile but not a complete management roster in extracted content. | not_publicly_verifiable | Org chart, board minutes, officer list, employment agreements, retention and succession plan. |
not publicly verifiable confidence: high
Compensation, employment agreements, benefits, bonuses, severance, and change-of-control arrangements are not public.
not publicly verifiable confidence: high
Option pool, equity grants, vesting, exercise prices, and incentive plans are not publicly disclosed.
not publicly verifiable confidence: medium
No public employee-relations issues were identified in reviewed sources, but this is not a comprehensive legal/HR search.
not publicly verifiable confidence: high
Personnel turnover and retention data for the last two years are not public.
| area | public signal | verification status | potential risk | diligence request |
|---|---|---|---|---|
| Executive departures | No public departures identified in reviewed sources. | not_publicly_verifiable | Key-person risk if leadership bench is thin. | Executive turnover history, resignation letters, separation agreements, reference calls. |
| Employee turnover | No HRIS or turnover metrics disclosed. | not_publicly_verifiable | Small team may be vulnerable to attrition during launch/regulatory scale-up. | Two-year turnover by function/location, regrettable attrition, recruiting pipeline, employee-relations log. |
| Compensation/benefits/option plan | Not publicly disclosed. | not_publicly_verifiable | Retention and dilution unknown. | Compensation bands, option plan, grants, benefits, bonus plans, change-of-control provisions. |
Public evidence shows approved labeling, EMA authorization, multiple patent assets, and a D. Del. patent-infringement case initiated by IntraBio, while adverse litigation, insurance, material contracts, compliance audits, and regulatory correspondence remain private.
inconclusive confidence: medium
CourtListener search results did not identify a separate pending lawsuit against IntraBio in extracted results; the visible matter is an IntraBio-initiated patent case, but the search is not comprehensive.
| case or search | court or source | filed date | status | finding |
|---|---|---|---|---|
| CourtListener search for IntraBio | CourtListener RECAP/opinions search | Accessed 2026-06-10 | Search returned 2 cases / 38 docket entries | Visible results centered on IntraBio v. Tesseract initiated by IntraBio; no separate pending lawsuit against IntraBio was identified in extracted results. |
| Other courts, arbitration, threatened claims | Not publicly comprehensive | Not disclosed | Not publicly verifiable | Requires counsel litigation schedule and representation letter. |
verified confidence: medium
IntraBio Inc. and IntraBio Limited initiated a public D. Del. patent-infringement case against Tesseract entities in 2025.
| case | court | filed date | status | diligence request |
|---|---|---|---|---|
| IntraBio Inc. v. Tesseract Medical Research, LLC | U.S. District Court, District of Delaware; 1:25-cv-00446 | 2025-04-10 | Patent-infringement complaint with jury demand; summons/answer deadlines shown in docket metadata. | Complaint/exhibits, answer/counterclaims, claim construction schedule, budget, settlement posture, insurance and business impact. |
| Other initiated matters | Not publicly identified | Not disclosed | Not publicly verifiable | Counsel schedule of all litigation, IP oppositions, administrative proceedings, disputes, and threatened claims. |
not publicly verifiable confidence: low
No environmental or employee-safety issues were identified in reviewed public sources; pharma manufacturing/vendor safety obligations are not publicly verifiable.
partially verified confidence: medium
IntraBio lists multiple U.S. patents/pending applications for AQNEURSA and the Tesseract docket identifies asserted patent numbers, but ownership, validity, expiry, licenses, and FTO require counsel review.
| asset | jurisdiction | public status | evidence | diligence request |
|---|---|---|---|---|
| AQNEURSA patent-marking portfolio | United States | Company lists multiple U.S. patents and pending applications | Patents include 10,905,670; 11,400,067; 12,144,792; 11,471,434; 11,793,782; 11,660,279; 11,998,518; 12/329,733; 12,433,862; 12,433,863; 12,458,614. | Patent schedule, owners, expiration, maintenance, Orange Book status, licenses, encumbrances, and FTO. |
| Tesseract patent-infringement litigation patents | United States / D. Del. | Docket report lists patents 11,400,067; 11,471,434; 11,660,279; 12,144,792. | CourtListener docket patent/trademark report. | Assess overlap with patent-marking portfolio, asserted claims, defenses, injunction/damages strategy. |
| Trademarks / brand rights for AQNEURSA | U.S./EU and launch markets | Brand appears with TM/R in public label/press sources; official trademark prosecution not validated. | DailyMed and BusinessWire use AQNEURSA name; company patent page references AQNEURSA. | Trademark docket, registrations, assignments, opposition/conflict search, domains and handles. |
not publicly verifiable confidence: high
Insurance coverage, D&O, product liability, clinical-trial insurance, cyber, and litigation coverage are not public.
not publicly verifiable confidence: high
Material contracts with CMOs, distributors, CROs, payers, IP licensors, consultants, and suppliers are not public.
partially verified confidence: high
Public sources verify FDA/EMA product approvals/authorizations and a pending A-T review; no regulatory problems were identified publicly, but label safety obligations and pending reviews are material.
| agency | action | date | status | diligence request |
|---|---|---|---|---|
| FDA / DailyMed | AQNEURSA U.S. approval for NPC neurological manifestations | Initial U.S. Approval: 2024 | Approved label; packager IntraBio Inc. | Approval letter, post-marketing requirements/commitments, labeling supplements, safety reports. |
| EMA | Aqneursa marketing authorisation for NPC neurological manifestations | 2026-01-19 | Authorised throughout EU; orphan; additional monitoring noted by EMA. | EPAR conditions, RMP, country reimbursement, QPPV/PV contracts, renewal obligations. |
| FDA | A-T sNDA accepted with Priority Review | 2026-05-19 announcement; PDUFA 2026-09-19 | Pending review per company press release. | FDA acceptance letter, review issues, advisory committee risk, CMC readiness, launch planning. |
| FDA/EMA and trial regulators | Clinical trial oversight for NPC, GM2, A-T, CACNA1A programs | 2019-2030 public registry dates | Completed, recruiting, terminated, and authorized programs in public sources. | Protocol deviations, inspection history, clinical holds, safety signals, and data-integrity audits. |
| ID | Claim | Status | Sources |
|---|---|---|---|
| EC-001 | CB Insights lists IntraBio as a unicorn valued at $1B, joined 03/23/2024, United States, Austin, Healthcare & Life Sciences. | verified medium | SRC-001 |
| EC-002 | CB Insights profile excerpts show IntraBio has active 2026 regulatory and clinical-trial news tied to levacetylleucine/AQNEURSA. | partially verified medium | SRC-002 |
| EC-003 | IntraBio publicly positions itself as a developer of novel drugs for rare neurodegenerative diseases. | verified medium | SRC-003 |
| EC-004 | IntraBio says it is focused on discovering, developing, and commercializing therapies, with scientific founders from Oxford/Munich and management/commercial/regulatory experience. | partially verified medium | SRC-004 |
| EC-005 | IntraBio describes IB1001 as orally administered N-acetyl-L-leucine and states AQNEURSA was FDA-approved in September 2024 for NPC neurological manifestations in patients weighing at least 15 kg. | partially verified medium | SRC-005 |
| EC-006 | DailyMed confirms AQNEURSA is levacetylleucine for oral suspension, initially approved in the U.S. in 2024, indicated for NPC neurological manifestations in adults and pediatric patients weighing at least 15 kg, and packaged by IntraBio Inc. | verified high | SRC-009 |
| EC-007 | EMA confirms Aqneursa is authorised in the EU for NPC neurological manifestations in adults and children aged 6+ weighing at least 20 kg, in combination with miglustat or monotherapy where miglustat is not tolerated. | verified high | SRC-016 |
| EC-008 | ClinicalTrials.gov NCT05163288 identifies IntraBio as industry sponsor of a Phase III NPC study of N-acetyl-L-leucine/IB1001, recruiting, with estimated enrollment of 53. | verified high | SRC-011 |
| EC-009 | ClinicalTrials.gov NCT03759639 identifies a completed Phase II NPC study sponsored by IntraBio with actual enrollment of 33. | verified high | SRC-012 |
| EC-010 | ClinicalTrials.gov NCT03759665 identifies a completed Phase II GM2 study sponsored by IntraBio with actual enrollment of 30. | verified high | SRC-013 |
| EC-011 | ClinicalTrials.gov NCT03759678 identifies an IntraBio Phase II A-T study that was terminated after priority shifted to recruiting directly to pivotal Phase III NCT07221292, with actual enrollment of 17. | verified high | SRC-014 |
| EC-012 | IntraBio announced FDA acceptance and Priority Review of an sNDA for AQNEURSA in A-T, with PDUFA target action date 2026-09-19, and claimed the pivotal Phase III trial met primary and key secondary endpoints. | partially verified medium | SRC-010 |
| EC-013 | Clinical Trials Arena independently reported that levacetylleucine met primary and secondary endpoints in an A-T Phase III trial and that IntraBio planned regulatory submissions. | partially verified medium | SRC-015 |
| EC-014 | IntraBio claims its platform technology results from decades of research and over $350M of investments from organizations worldwide. | unverified low | SRC-007 |
| EC-015 | IntraBio lists multiple U.S. patents and pending applications as applying to AQNEURSA. | partially verified medium | SRC-008 |
| EC-016 | LinkedIn describes IntraBio as privately held, located/headquartered in Austin, Texas, company size 11-50 employees, with pharmaceutical focus on rare/common neurodegenerative and neurodevelopmental diseases. | verified medium | SRC-017 |
| EC-017 | DailyMed label for MIPLYFFA confirms another 2024-approved NPC therapy, arimoclomol, indicated in combination with miglustat for neurological manifestations of NPC in adults and pediatric patients 2 years and older. | verified high | SRC-018 |
| EC-018 | DailyMed search shows multiple miglustat products, supporting that miglustat remains an available NPC-related therapy/comparator and combination partner. | verified medium | SRC-019SRC-016 |
| EC-019 | CourtListener search found public RECAP records involving IntraBio, including IntraBio Inc. v. Tesseract Medical Research, LLC. | verified medium | SRC-020 |
| EC-020 | CourtListener docket shows IntraBio Inc. and IntraBio Limited filed a patent-infringement complaint against Tesseract entities in D. Del. on 2025-04-10. | verified medium | SRC-021 |
| EC-021 | Reviewed public sources did not disclose audited financial statements, revenue, ARR, gross margin, burn, cash runway, debt, current cap table, or customer concentration for IntraBio. | not publicly verifiable high | SRC-001SRC-002SRC-003SRC-004SRC-005SRC-017 |
| EC-022 | Reviewed public product and regulatory sources did not disclose AQNEURSA pricing, gross-to-net, payer coverage, volume, named customers, manufacturer/supplier contracts, or revenue by channel. | not publicly verifiable high | SRC-005SRC-009SRC-010SRC-016 |
| EC-023 | BusinessWire identifies Mallory Factor as President and Chief Executive Officer of IntraBio in the May 2026 sNDA press release. | verified medium | SRC-010 |
| EC-024 | AQNEURSA label includes safety constraints including embryo-fetal harm risk and potential interaction issues involving N-acetyl-DL/D-leucine and P-gp substrates. | verified high | SRC-009 |
This report is a public-evidence diligence snapshot, not investment advice. Important financial, legal, technical, and contractual facts remain non-public and should be verified directly with management and primary documents before any investment decision.