What Acer Therapeutics does

Acer Therapeutics was a publicly traded (NASDAQ:ACER) rare-disease pharmaceutical developer incorporated in Delaware and headquartered in Newton, Massachusetts. The company focused on acquiring, developing, and commercializing therapies for patients with serious, rare, and life-threatening diseases, with an employee base of 11-50. Its portfolio centered on reformulation and repurposing of established compounds: OLPRUVA (sodium phenylbutyrate), an FDA-approved treatment for urea cycle disorders (UCDs); ACER-001, an investigational candidate for Maple Syrup Urine Disease (MSUD) leveraging the same active ingredient; and EDSIVO (celiprolol), an investigational beta-blocker therapy for vascular Ehlers-Danlos Syndrome (vEDS).

The business model is a specialty-pharmaceutical sales motion: Acer commercialized OLPRUVA through U.S. specialty pharmacy distribution and patient-advocacy partnerships targeted at metabolic disease specialists and rare-disease patient communities. Revenue mechanics depended on prescription sales of OLPRUVA and, prospectively, ACER-001 and EDSIVO upon approval; pricing was not publicly disclosed. Capital structure was notable for its reliance on non-traditional funding for a public company — including a $48.5M debt facility from Marathon Asset Management and SWK Holdings in March 2022, and approximately $2.5M in CEO-funded bridge notes in late 2022 and mid-2023 — reflecting the cash-intensity of late-stage clinical and early commercial biotech. In 2023, Zevra completed the acquisition of Acer Therapeutics, after which Acer's assets were integrated into Zevra's commercial platform.

Name

Acer Therapeutics

Legal name

Acer Therapeutics Inc.

Website

https://acertx.com

Company type

Public

Founded year

2018

Operating status

Acquired

Headcount range

11–50 employees

Short description

Acer Therapeutics was a publicly traded rare-disease pharmaceutical developer that acquired, developed, and commercialized therapies for serious, ultra-rare metabolic and connective-tissue disorders, including FDA-approved OLPRUVA for urea cycle disorders, before being acquired by Zevra in 2023.

Ownership category

akta.pro rank

Product category

Specialty Pharmaceuticals

NAICS

Medicinal and Botanical Manufacturing (325411)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB)

Keywords

• Rare disease therapies
• Specialty pharmaceuticals
• Urea cycle disorders
• Metabolic disorder treatments
• Rare disease drug development

Headquarters

HQ city

Newton

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: Acer Therapeutics generates revenue through the sale of approved drug products, primarily OLPRUVA for urea cycle disorders. The company commercializes specialty pharmaceuticals targeting rare disease populations through specialty pharmacy channels.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Acer Therapeutics acquires, develops, and commercializes therapies for patients with serious, ultra-rare, and life-threatening diseases. Its commercial product, OLPRUVA™, is an FDA-approved sodium phenylbutyrate therapy for Urea Cycle Disorders (UCDs), while its pipeline includes ACER-001 for Maple Syrup Urine Disease (MSUD) and EDSIVO™ (celiprolol) for Vascular Ehlers-Danlos Syndrome (vEDS).

Product overview

Acer Therapeutics is a biotechnology company developing therapies for serious, ultra-rare, and life-threatening diseases. The company's product portfolio includes OLPRUVA™ (FDA-approved for Urea Cycle Disorders), ACER-001 (investigational for Maple Syrup Urine Disease), and EDSIVO™ (investigational for Vascular Ehlers-Danlos Syndrome). The company operates as a rare disease focused pharmaceutical developer with pipeline products at various stages of clinical development.

Differentiator

Problem solved

Functional benefit

Brands

• OLPRUVA™: Product for urea cycle disorders (UCDs)
• EDSIVO™
• ACER-001

Products and services

• OLPRUVA™ (sodium phenylbutyrate) FDA-approved therapy for Urea Cycle Disorders (UCDs), a rare genetic condition affecting nitrogen metabolism, delivered via a proprietary formulation designed to improve patient compliance and efficacy compared to existing sodium phenylbutyrate treatments.
• ACER-001 (sodium phenylbutyrate for MSUD) Investigational drug candidate being developed for Maple Syrup Urine Disease (MSUD), a rare metabolic disorder affecting branched-chain amino acid metabolism, leveraging the sodium phenylbutyrate active ingredient from OLPRUVA.
• EDSIVO™ (celiprolol) Investigational therapy for Vascular Ehlers-Danlos Syndrome (vEDS), a rare genetic connective tissue disorder, utilizing beta-blocker properties of celiprolol to address life-threatening vascular complications.

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Scale indicators4 records

Recent moves5 records

Expansion highlights4 records

Direct peers

• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with multiple approved products targeting metabolic disorders including urea cycle-related conditions; directly comparable in therapeutic focus, regulatory approach, and rare disease commercialization model.
• BioMarin Pharmaceutical: Large rare disease biotech with deep portfolio across metabolic disorders (including phenylketonuria and other amino acid disorders); highly comparable in operating model even though materially larger in scale and headcount.
• Zevra Therapeutics: Rare disease pharmaceutical company that acquired Acer Therapeutics in 2023; operates in the same therapeutic and commercial niche (rare metabolic and CNS disorders) and now owns OLPRUVA directly.
• Soleno Therapeutics: Clinical-stage rare disease biotech focused on hyperphagia in Prader-Willi syndrome; comparable in size, regulatory stage, and ultra-rare disease commercialization model relevant to Acer's metabolic niche.
• Travere Therapeutics: Rare disease company with approved products in nephrology and metabolic indications; comparable business model around small patient populations, reformulation strategies, and specialty distribution.

Broad incumbents

• Genzyme (Sanofi): Historical leader in enzyme replacement and rare metabolic disease therapies (Cerezyme, Fabrazyme, Aldurazyme); operates as the broad incumbent setting the standard for rare disease commercialization Acer emulates.
• Horizon Therapeutics (Amgen): Specialty rare disease franchise (now within Amgen) including metabolic and inflammatory rare conditions; representative of large strategic acquirer/incumbent playing in the rare disease space where Acer competes for physician attention.
• Recordati Rare Diseases: Established rare disease division of Recordati with global distribution footprint and product portfolio across multiple rare indications; comparable in go-to-market through specialty pharma channels.
• Alexion Pharmaceuticals (AstraZeneca): Original rare disease pure-play now within AstraZeneca, focused on complement-mediated and metabolic disorders; relevant incumbent comparator for commercial reach and lifecycle management of rare disease products.
• Swedish Orphan Biovitrum (Sobi): European rare disease specialist with broad portfolio across metabolic, hematology, and immunology indications; relevant as a global peer with comparable rare disease commercial strategy and geographic reach.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/acer-therapeutics-inc

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds7 records

Investors3 records

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

M&A1 record

Investments

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

Frequently asked questions about Acer Therapeutics