ADRX-0405 Receives Orphan Drug Designation for Gastric Cancer Treatment

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Breakthrough Cancer Therapy ADRX-0405 Enters Clinical Trials, Targeting Advanced Solid Tumors

Adcentrx Therapeutics is advancing its novel antibody-drug conjugate (ADC), ADRX-0405, into a Phase 1a/b clinical trial, marking a critically important step forward in the treatment of difficult-to-treat cancers. This investigational therapy is being tested in patients with metastatic castration-resistant prostate cancer (mCRPC), gastric cancer, and non-small cell lung cancer (NSCLC).The trial is designed to meticulously evaluate the safety and tolerability of ADRX-0405, with the ultimate goal of identifying the optimal dose for future patient populations. The early-stage study is structured into two parts:

Phase 1a (Dose Escalation): This initial phase focuses on gradually increasing the dose of ADRX-0405 in eligible patients to pinpoint the maximum tolerated dose (MTD) and the recommended dose for subsequent expansion.
Phase 1b (Dose Expansion): Following the dose escalation, this phase will specifically enroll patients with previously treated mCRPC to further assess the therapy’s efficacy and safety profile in this particular patient group.

Eligibility for the trial is stringent, ensuring a focused evaluation: Patients must have histologically confirmed disease that is measurable according to RECIST 1.1 criteria. For prostate cancer patients, evaluable disease per Prostate Cancer working Group 3 criteria is also acceptable.A key requirement is an ECOG performance status of 0 or 1 for Phase 1a, and 0 to 2 for Phase 1b, indicating generally good health. Furthermore, participants must meet specific benchmarks for blood counts, liver, and kidney function.

For those with prostate cancer specifically, inclusion criteria stipulate histologically confirmed castration-resistant prostate adenocarcinoma with a serum testosterone level below 50 ng/dL, and a history of intolerance or resistance to standard therapies.

Significant exclusion criteria are in place to maintain patient safety and data integrity. These include active and uncontrolled central nervous system metastases, significant cardiovascular conditions, recent history of other cancers, and recent exposure to other anti-cancer or investigational treatments, including certain radiopharmaceuticals for prostate cancer. Patients with a history of or suspicion of interstitial lung disease or pneumonitis, or those currently undergoing systemic antimicrobial treatment for active infection, are also excluded.

The trial’s primary objective is to meticulously track the incidence of adverse events, providing crucial safety data. Secondary endpoints are designed to offer a comprehensive understanding of ADRX-0405’s performance, encompassing pharmacokinetic measures (how the body processes the drug) and key efficacy indicators such as objective response rate (ORR), duration of response, disease control rate, progression-free survival (PFS), radiographic PFS, and overall survival.

Adcentrx Therapeutics announced that the first patient was dosed in January 2025, with an anticipated first data readout scheduled for the fourth quarter of the same year. This milestone signifies a critical advancement in the potential development of ADRX-0405 as a pioneering therapy targeting STEAP1, a protein expressed on certain cancer cells. The company has previously received Orphan Drug Designation from the U.S. FDA for ADRX-0405 for gastric cancer, highlighting its potential in addressing unmet medical needs.

what specific benefits does the Orphan Drug designation provide to Adrenas Therapeutics regarding the development of ADRX-0405?

Understanding the Meaning of Orphan Drug Designation

The U.S. Food and Drug Management (FDA) has granted Orphan Drug Designation (ODD) to ADRX-0405, a novel therapeutic being developed by Adrenas Therapeutics, for the treatment of gastric cancer. This designation is a critical milestone, offering several benefits to accelerate the drug’s development and eventual availability to patients battling this challenging disease. Gastric cancer, also known as stomach cancer, affects thousands annually, and new treatment options are urgently needed.

This ODD specifically targets HER2-low gastric cancer, a subtype where current treatment options are limited. The designation acknowledges the unmet medical need within this patient population.

What is ADRX-0405 and How Does it Work?

ADRX-0405 is an investigational antibody-drug conjugate (ADC). ADCs represent a cutting-edge approach to cancer therapy, combining the targeting ability of antibodies with the potent cell-killing power of chemotherapy drugs.

Here’s a breakdown of how it functions:

Antibody Component: The antibody portion of ADRX-0405 is designed to specifically bind to a protein found on the surface of gastric cancer cells.

Chemotherapeutic Payload: A potent chemotherapy drug is attached to the antibody.

Targeted Delivery: Once bound to the cancer cell, the ADC is internalized, releasing the chemotherapy drug directly into the tumor, minimizing damage to healthy cells.

HER2-low Targeting: ADRX-0405 is engineered to effectively target HER2-low expressing gastric cancer cells, a population often resistant to conventional HER2-targeted therapies.

Benefits of Orphan Drug Designation

Receiving ODD isn’t just a symbolic recognition; it unlocks tangible advantages for Adrenas Therapeutics and, ultimately, for patients. Thes benefits include:

Tax Credits: Significant tax credits for qualified clinical testing expenses.

grant Funding: Eligibility for grant funding from the FDA to support clinical trials.

Market Exclusivity: Seven years of market exclusivity in the United States upon FDA approval, preventing competitors from marketing a similar drug for the same indication.

FDA Assistance: access to FDA assistance and guidance throughout the development process, including protocol review and potential accelerated approval pathways.

Reduced FDA Fees: waiver or reduction of certain FDA application fees.

Gastric Cancer: A Deeper Look at the Disease

Gastric cancer is a complex disease with varying subtypes and stages. Understanding the nuances of the disease is crucial for developing effective treatments.

Types of Gastric Cancer: The most common type is adenocarcinoma,accounting for approximately 95% of cases. other, rarer types include lymphoma, gastrointestinal stromal tumors (GIST), and carcinoid tumors.

Risk Factors: Several factors can increase the risk of developing gastric cancer, including Helicobacter pylori infection, smoking, diet (high in salted, smoked foods), family history, and certain genetic predispositions.

Symptoms: Early-stage gastric cancer often presents with no noticeable symptoms. As the disease progresses, symptoms may include persistent indigestion, abdominal pain, nausea, vomiting, weight loss, and fatigue.

Current Treatment Options: Standard treatments include surgery, chemotherapy, and radiation therapy. Targeted therapies and immunotherapies are also used in certain cases, notably for advanced stages.

The Role of HER2 in Gastric Cancer Treatment

Human epidermal growth factor receptor 2 (HER2) is a protein that promotes cancer cell growth. In gastric cancer, HER2 status is a critical factor in determining treatment strategies.

HER2-Positive Gastric Cancer: Approximately 15-20% of gastric cancers are HER2-positive, meaning they have an overabundance of the HER2 protein. These cancers are frequently enough aggressive but can be effectively treated with HER2-targeted therapies like trastuzumab.

HER2-Negative Gastric Cancer: the majority of gastric cancers are HER2-negative.

HER2-Low Gastric Cancer: A growing understanding of HER2 expression has identified a significant subset of cancers classified as HER2-low. These tumors express some HER2, but not enough to be considered HER2-positive, leaving a treatment gap. ADRX-0405 is specifically designed to address this unmet need.

Clinical Development of ADRX-0405

Adrenas Therapeutics is actively conducting clinical trials to evaluate the safety and efficacy of ADRX-0405 in patients with HER2-low gastric cancer.Details regarding trial enrollment, locations, and study designs can be found on clinicaltrials.gov.

Key areas of inquiry include:

Phase 1 Trials: Focused on determining the optimal dosage and assessing the safety profile of ADRX-0405.

Phase 2 trials: evaluating the drug’s efficacy in a larger group of patients with HER2-low gastric cancer.

Biomarker analysis: Identifying biomarkers that may predict which patients are most likely to respond to treatment.

Future Outlook for Gastric Cancer Therapies

The ODD for ADRX-0405 represents a positive step forward in the fight against gastric cancer. Ongoing research and development efforts are focused on:

Novel ADCs: