Vast Therapeutics

What Vast Therapeutics does

Vast Therapeutics, Inc. is a clinical-stage pharmaceutical company and subsidiary of KNOW Bio, LLC, headquartered in Morrisville, North Carolina, developing ALX1, a first-in-class nitric oxide prodrug designed for targeted delivery to the lungs via a customized PARI eFlow handheld nebulizer. ALX1 stores nitric oxide in a buffered aqueous solution and releases controlled amounts locally in the lung, producing a dual anti-inflammatory and broad-spectrum antimicrobial effect — anti-inflammatory activity via NLRP3 inflammasome inhibition, and antimicrobial activity that kills bacteria through multiple mechanisms with very low observed propensity for drug resistance. The company is targeting chronic respiratory diseases with high unmet need, including bronchiectasis (~400,000–700,000 US adults), cystic fibrosis (~30,000 US patients), non-tuberculous mycobacteria pulmonary disease (~86,000–90,000 US patients), and COPD.

The company's intellectual property covers ALX1, nitric oxide-releasing compounds, and their pharmaceutical formulations through issued patents, and it has secured FDA Qualified Infectious Disease Product designation for Pseudomonas aeruginosa infections and Orphan Drug Designation for cystic fibrosis respiratory infection, providing up to 12 years of layered market exclusivity in overlapping indications. Phase 1 Single Ascending Dose results reported in November 2025 demonstrated safety and tolerability up to 172 mg/day with no bronchospasm, methemoglobinemia, or dose-limiting toxicities, and Phase 2 in bronchiectasis is targeted for the first half of 2026; the NTM-PD program is supported by a separate NIH/NIAID grant. Leadership includes veterans of Novan, Inc. (CEO Nate Stasko led Novan's NASDAQ IPO and the development of ZELSUVMI, the first FDA-approved nitric oxide dermatology drug), the chemistry founder Mark Schoenfisch (University of North Carolina), and a CMO with prior contributions to 12 approved drugs including Pulmozyme.

Vast's business model is a traditional prescription-pharma model: it is pre-revenue, generates no product sales today, and will rely on FDA approval of ALX1 followed by specialty pharmacy and respiratory specialty distribution to monetize. Capital sources to date are primarily non-dilutive — two NIH/NIAID grants totaling approximately $4 million — alongside prior venture and strategic investment (including a $15M Shionogi/Cystic Fibrosis Foundation round in 2019) and a current Reg CF public funding round on StartEngine. The company employs 1–10 people and operates as a focused, single-asset developer with no current commercial infrastructure.

Name

Vast Therapeutics

Legal name

Vast Therapeutics, Inc.

Website

https://vasttherapeutics.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

Vast Therapeutics is a clinical-stage pharmaceutical subsidiary of KNOW Bio developing ALX1, a first-in-class inhaled nitric oxide prodrug that combines anti-inflammatory and antimicrobial activity to treat chronic respiratory diseases including bronchiectasis, cystic fibrosis, COPD, and NTM lung disease.

Ownership category

akta.pro rank

Product category

Respiratory Pharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Pulmonology & Allergy Specialty Pharmaceuticals (HLAIACAF)

Keywords

• Inhaled therapeutics
• Nitric oxide prodrug
• Respiratory pharmaceuticals
• Cystic fibrosis treatment
• Bronchiectasis therapy

Headquarters

HQ city

Morrisville

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Pharmaceutical Product Sales: Pre-revenue clinical-stage company. Revenue will be generated through sales of ALX1 and future drug candidates upon FDA approval. Target markets include bronchiectasis (~400,000-700,000 US adults), cystic fibrosis (~30,000 US patients), COPD, and NTM pulmonary disease (~86,000-90,000 US patients).

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Vast Therapeutics is a clinical-stage pharmaceutical company developing ALX1, a first-in-class nitric oxide prodrug designed for targeted delivery to the lungs via a portable PARI eFlow nebulizer. The drug combines anti-inflammatory (NLRP3 inflammasome inhibition) and broad-spectrum antimicrobial mechanisms to treat chronic respiratory diseases including bronchiectasis, cystic fibrosis, COPD, and non-tuberculous mycobacteria (NTM) lung disease. ALX1 is currently in Phase 1 clinical trials, with Phase 2 in bronchiectasis patients targeted for the first half of 2026.

Product overview

Vast Therapeutics is a clinical-stage life science company developing a single lead drug candidate, ALX1, which is a first-in-class nitric oxide prodrug delivered via inhalation using a handheld portable PARI eFlow nebulizer. The technology platform centers on nitric oxide's dual mechanism of action—anti-inflammatory and antimicrobial properties—to break the cycle of infection and inflammation in chronic respiratory diseases. ALX1 is being developed for multiple indications including bronchiectasis, cystic fibrosis, COPD, and non-tuberculous mycobacteria (NTM) pulmonary disease.

Differentiator

Problem solved

Functional benefit

Products and services

• ALX1 First-in-class nitric oxide prodrug formulated as a buffered aqueous solution for oral inhalation via a customized PARI eFlow nebulizer. Delivers localized, sustained-release nitric oxide to the lungs to provide dual anti-inflammatory and antimicrobial activity, intended to treat chronic respiratory diseases including bronchiectasis, cystic fibrosis, COPD, and NTM pulmonary disease. The product targets healthcare providers treating patients with chronic lung infections and inflammation.

Quantifiable outcome

• >99% reduction in Mycobacterium abscessus levels within lungs of infected rodents when dosed once daily for 7 days
• +3 more outcomes

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

One partnership is on record.

• KNOW Bio, LLC (Parent Company)coreOthersKNOW Bio, LLC is the parent company of Vast Therapeutics, Inc. KNOW Bio also includes subsidiaries EmitBio, Inc., Revian, Inc. KNOW Bio's Financial Conflict of Interest Policy applies to Vast Investigators on PHS-funded research.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Insmed: Insmed is the most directly comparable respiratory biotech: it markets Arikayce (inhaled liposomal amikacin) for refractory NTM lung disease and is advancing brensocatib (oral DPP-1 inhibitor) through Phase 3 in bronchiectasis — the same disease Vast is targeting first. Both companies are pursuing inhaled/novel-mechanism therapies for chronic respiratory infections, though Insmed is far larger and later-stage.
• AN2 Therapeutics: AN2 Therapeutics is a clinical-stage biotech developing epetraborole, an oral once-daily antibiotic for treatment-refractory NTM lung disease (Mycobacterium abscessus). It is a direct peer to Vast's ALX1 NTM program in target indication, modality of antimicrobial action, and disease population.
• Novan: Novan is Vast's direct predecessor company, founded by the same leadership team (Stasko and Schoenfisch) and based on nitric oxide-releasing chemistry. It is a peer by virtue of shared scientific lineage, technology platform overlap, and prior experience bringing NO chemistry to clinical development.
• Aradigm Corporation: Aradigm developed inhaled liposomal antibiotics (Arikace, Linaprazid) for chronic respiratory infections including bronchiectasis and CF via the same PARI eFlow nebulizer Vast uses for ALX1. It is a directly comparable peer in inhaled antimicrobial modality and chronic lung disease focus.
• Parion Sciences: Parion Sciences developed inhaled epithelial sodium channel (ENaC) inhibitors for CF and other muco-obstructive lung diseases — overlapping Vast's CF and bronchiectasis targets. Acquired by Vertex in 2018, it remains a direct historical comparator for inhaled therapeutics in chronic respiratory disease.
• Zambon: Zambon markets Colistimethate (Colobreathe) and has developed inhaled antibiotics for chronic Pseudomonas infections in CF and bronchiectasis. It is a directly comparable peer in inhaled antimicrobial therapy for the same patient populations Vast targets.

Broad incumbents

• Vertex Pharmaceuticals: Vertex dominates the cystic fibrosis market with Trikafta (and successor regimens) and is a broad incumbent in CF respiratory disease — Vast's primary orphan-indication target. Vertex is much larger and offers a benchmark for what a fully-realized CF franchise looks like commercially.
• AstraZeneca: AstraZeneca is a broad incumbent in respiratory disease through Symbicort, Breztri, Fasenra, Tezspire, and other inhaled/biologic franchises that overlap with Vast's COPD and broader asthma/COPD patient populations. It is a relevant incumbent peer for indication-level commercial dynamics, not direct product competition.

Emerging players

• TFF Pharmaceuticals: TFF Pharmaceuticals applies thin-film freezing technology to inhaled drug formulations, including programs for fungal and bacterial lung infections. It is an emerging peer with partial overlap in inhaled antimicrobial modality and chronic respiratory indications.
• Spyryx Biosciences: Spyryx Biosciences is a clinical-stage company developing inhaled ENaC-targeted peptides for cystic fibrosis lung disease. It is an emerging peer with direct overlap with Vast's CF indication and inhaled therapeutic modality.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/novoclem
• LinkedInlinkedin.com/company/novoclem

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds5 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Vast Therapeutics