Genprex, Inc. is a clinical-stage gene therapy company focusing on developing treatments for cancer and diabetes using innovative gene delivery platforms.

# Company Overview Summary

## Company Name
- **Genprex, Inc.**

## Core Business
- A **clinical-stage gene therapy company** focusing on developing gene-based treatments for large patient populations with unmet medical needs, primarily in cancer and diabetes.

## Key Platforms & Technologies
- **Oncology Platform**
  - Utilizes the **systemic, non-viral ONCOPREX® Delivery System**, which uses lipid-based nanoparticles (lipoplexes) to deliver tumor suppressor gene plasmids to cancer cells.
  - Designed to be administered intravenously, targeting tumor cells specifically, and minimize impact on healthy tissue.
- **Diabetes Technology**
  - Based on licensing technology that uses adeno-associated virus (AAV) vectors to deliver Pdx1 and MafA genes directly into the pancreas via the pancreatic duct.
  - Aims to transform alpha cells into insulin-producing beta-like cells and rejuvenate exhausted beta cells in Type 2 diabetes.
  - Currently in preclinical development, evaluating constructs like GPX-002.
  
## Lead Product Candidates & Development Status
- **REQORSA® (Quaratusugene Ozeplasmid)**
  - A systemic gene therapy delivering the **TUSC2 tumor suppressor gene** via the ONCOPREX system.
  - Developed to treat **non-small cell lung cancer (NSCLC)** and **small cell lung cancer (SCLC)**.
  - Being evaluated in clinical trials in combination with targeted therapies (Tagrisso, Tecentriq, etc.).
  - **FDA Designations:** Fast Track for NSCLC and SCLC indications, Orphan Drug for SCLC.
- **GPX-002**
  - A gene therapy using AAV vectors to treat **Type 1 and Type 2 diabetes** by converting alpha cells into insulin-producing cells or restoring exhausted beta cells.
  - In preclinical studies, showing promising glucose normalization and survival data in animal models.
  
## Clinical Programs & Trials
- **Oncology**
  - Several ongoing clinical trials, including:
    - **Acclaim-1:** Phase 2a in NSCLC with mutations, combining REQORSA with Tagrisso.
    - **Acclaim-3:** Phase 2 in SCLC using REQORSA with Tecentriq.
    - Other trials in NSCLC with resistance to other therapies and in combination with immunotherapies.
  - Previously completed early-phase trials showing preliminary safety and efficacy signals.
- **Diabetes**
  - Preclinical studies in mice and non-human primates demonstrating reduction in insulin requirements and improved glucose regulation.
  - Working on optimizing constructs and planning for future IND submissions.

## Employees and Revenue
- **Employees:** 15 full-time employees (as of March 15, 2025).
- **Revenue:** 
  - The company has **never been profitable**.
  - No products approved for sale.
  - No revenue reported from product sales.
  - Has raised funds via stock offerings (e.g., ATM offerings), with the latest net proceeds in the millions USD.

## Financials
- **Market Capitalization:** Approximately **$4.8 million** as of June 28, 2024.
- **Outstanding Shares:** 24,152,848 shares as of March 28, 2025.
- The company is considered a **smaller reporting company** and a **larger reporting company** (not emerging growth). 

## Business Focus
- Developing gene therapies for **cancer and diabetes**, with a strategic plan to potentially separate its diabetes assets into a wholly owned subsidiary, **Convergen Biotech**, in 2025 to streamline focus on oncology.
- Collaboration and licensing agreements with MD Anderson and University of Pittsburgh for intellectual property rights.
- Manufacturing is conducted through third-party CDMOs, with internal competencies developed for scalable production.

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*Note:* The company has not disclosed specific customer numbers, detailed revenue figures, or profit/loss figures beyond indicating that it is not yet profitable.

A clinical-stage gene therapy firm focused on developing gene-based treatments for unmet medical needs, particularly in oncology and diabetes.

# Company Overview

## Business Focus
The company is a clinical-stage gene therapy firm dedicated to developing gene-based treatments for significant patient groups with unmet medical needs. 

## Key Platforms

### 1. **Oncology Platform**
- **Lead Product:** REQORSA® Immunogene Therapy (quaratusugene ozeplasmid)
  - **Indications:** Non-Small Cell Lung Cancer (NSCLC) and Small Cell Lung Cancer (SCLC)
  - **Mechanism of Action:** REQORSA utilizes a multimodal approach to interrupt cancer cell signaling pathways, promote apoptosis (programmed cell death), and enhance immune response against tumor cells.
  - **Administration:** Intravenous application using the ONCOPREX® Delivery System, which delivers tumor suppressor genes through lipid-based nanoparticles.
  
- **Clinical Trials:**
  - **Acclaim Trials**
    - **Acclaim-1:** Combination therapy of REQORSA and AstraZeneca’s Tagrisso® for late-stage NSCLC; currently in Phase 2a expansion.
      - **Enrollment:** Targeting approximately 66 patients.
      - **Expected Completion:** Enrollment by end of 2024; interim analysis in early 2025.
    - **Acclaim-2:** Combination therapy of REQORSA and Merck & Co.’s Keytruda® for late-stage NSCLC; currently in Phase 1 dose escalation.
      - **Expected Enrollment Completion:** Second half of 2024.
    - **Acclaim-3:** Combination therapy of REQORSA and Genentech’s Tecentriq® for extensive stage SCLC; currently in Phase 1.

### 2. **Diabetes Gene Therapy**
- **Product Candidate:** GPX-002
  - **Indications:** Type 1 and Type 2 diabetes.
  - **Mechanism of Action:** 
    - **Type 1 Diabetes:** Transforms alpha cells into functional beta-like cells, which produce insulin while evading the immune system.
    - **Type 2 Diabetes:** Replenishes and rejuvenates exhausted beta cells to restore insulin production.
  
- **Development Status:** Currently undergoing preclinical studies; plans to file for an Investigational New Drug (IND) application pending the results of ongoing studies.

## Key Metrics
- **Employees:** As of March 15, 2024, the company has 26 full-time employees.
- **Customers:** The company is targeting healthcare providers and institutions involved in oncology and diabetes treatment. Specific customer numbers are not disclosed.
- **Revenue:** The company has not generated revenue from product sales as it is still in clinical development.
- **Income:** The company has incurred net losses; as of December 31, 2023, it reported an accumulated deficit of approximately $133.9 million and continued operational losses.

## Regulatory Safeguards
- The company has received **Fast Track Designation** from the FDA for several of its product candidates, indicating the potential for expedited development and review.
- The company is pursuing **Orphan Drug Designation** for specific indications to enhance marketing exclusivity in the United States.

## Future Directions
The company plans to focus on:
- Completing ongoing clinical trials for REQORSA and GPX-002.
- Seeking additional funding to support development and commercialization efforts.
- Expanding collaborations with academic institutions and partners to enhance research and development capabilities. 

This concise summary highlights the company's primary business focus, current product offerings, clinical trials, operational metrics, and future directions while providing specific detail on its market initiatives and strategic goals.

Genprex, Inc.

10-K / April 1, 2025

10-K / April 1, 2024

10-K / April 1, 2025

Company Overview Summary

Company Name

Core Business

Key Platforms & Technologies

Lead Product Candidates & Development Status

Clinical Programs & Trials

Employees and Revenue

Financials

Business Focus