NIA Small Business Showcase: Praxis Bioresearch

Praxis Bioresearch is a biopharmaceutical company focused on developing safe and effective solutions for the management of behavioral symptoms of Alzheimer’s disease (AD), including apathy.

Apathy is the most common noncognitive symptom in AD, affecting nearly 70% of the patients. Untreated apathy can lead to a poor quality of life for the patient, a rapid decline in cognition, increased caregiver distress, the need for costly early institutionalization. Addressing behavioral symptoms in AD is increasingly important and will allow patients to have a higher quality of life and live independently as long as possible.

Although there is currently no Food and Drug Administration (FDA)-approved treatment, recent clinical research demonstrates that apathy is responsive to stimulant treatments such as methylphenidate. Praxis has built upon these findings and developed PRX-P4-003 (octadecanoic acid, [[[ethyl[(1R,2R,3S,4S)-3-phenylbicyclo[2.2.1]hept-2-yl]amino]carbonyl]oxy]methyl ester), a novel prodrug of fencamfamine (a Schedule IV stimulant). PRX-P4-003 has a gut-based activation mechanism providing for selective oral bioavailability and once-a-day dosing convenience. The company’s goal is to develop PRX-P4-003 as a “first-in-class” FDA-approved therapy for apathy in AD.

In 2021, Praxis successfully demonstrated PRX-P4-003 bioconversion to its active moiety (-)-fencamfamine in healthy volunteers under an exploratory investigational new drug (IND) pathway.

Company Milestones

Scientific

• 2017: American College of Neuropsychopharmacology (ACNP) poster presentation: A Novel Abuse-Deterrent Prodrug Stimulant Selectively Activated by Pancreatic Lipase in the Gut
• 2018: Publication in Drug and Alcohol Dependence: Novel prodrug PRX-P4-003, selectively activated by gut enzymes, may reduce the risk of iatrogenic addiction and abuse
• 2021: Approved exploratory IND application (PRX-P4-003)
• 2021: 14th Clinical Trials on Alzheimer’s Disease (CTAD) meeting presentation: Microdose Study of a Novel Psychostimulant Prodrug, PRX-P4-003, with Reduced Abuse Liability for Apathy in Alzheimer’s Disease
• 2021: Successful pre-IND meeting completed for apathy in Alzheimer’s disease
• 2022: Successful kilogram-scale manufacturing
• 2022: IND-enabling studies implemented, with expected completion in Q2 2023

Business

• 2015: Seed funding ($600,000)
• 2018: Small Business Innovation Research (SBIR) award ($1.5 million)
• 2020: First U.S. composition-of-matter patent issued
• 2021: Successful microdose clinical study in healthy volunteers
• 2021: SBIR award ($2.9 million)
• 2021: Japan composition-of-matter patent issued
• 2021: People’s Republic of China composition-of-matter patent issued
• 2022: Expected SAFE (Simple Agreement for Future Equity) funding round ($1.1 million)

Financial Overview

Praxis has raised a primarily non-dilutive $6.2 million to date through a combination of seed funding, NIH SBIR awards, and private investor rounds.

Intellectual Property

Praxis Bioresearch’s lead candidate drug, PRX-P4-003, retains a robust package in all the major pharmaceutical markets worldwide.

Product Development and Regulatory Strategy

Praxis Bioresearch’s goal is to develop PRX-P4-003 as a first FDA-approved therapy for apathy in AD. As a small company, Praxis has pursued its product development with an emphasis on early strategic de-risking wherever possible. In 2021, Praxis successfully conducted a pre-IND meeting with FDA for apathy in AD. Currently, PRX-P4-003 is in the IND-enabling studies stage, and Praxis expects to file for an IND by Q3 2023. Since apathy in AD is a serious unmet medical need, Praxis plans to apply for a fast-track designation. Although this will be attractive to future industry partners, it will also reduce regulatory risks by facilitating more frequent meetings with FDA to discuss the drug’s development plan and ensure appropriate data collection to support drug approval.

Commercialization Strategy

Due to the heterogenous nature of AD, the current practice of using various combinations of treatments will continue to be the norm, so Praxis is focused on potential large pharmaceutical partners who are committed to either disease-modifying or symptomatic therapies in AD.

Successful completion of a PRX-P4-003 clinical Phase 1 study will support a development plan for which the costs and risks are understood well from a historical perspective. After Phase 1, Praxis will have a sufficiently robust clinical dataset and understanding of likely development pathways and regulatory requirements to enter discussions with potential industry partners and investors. Although the company will continue to be open to both types of investment, as well as continued research support through programs such as SBIR, it is most likely that Praxis will seek an industry partner for development beyond Phase 2.

Company Details

Praxis Bioresearch website
10940 Wilshire Blvd, Suite 600
Los Angeles, CA 90024

Industry: Therapeutics

Management Team:

• Chief Executive Officer/Cofounder: Sandeep Patil, M.D., Ph.D.
• Chief Scientific Advisor: William Z. Potter, M.D., Ph.D.

Point of Contact:
Sandeep Patil, M.D., Ph.D.
Email Sandeep Patil
650-713-6809