FDA Grants BA1302 Orphan Drug Designation for Squamous NSCLC and Pancreatic Cancer

Author(s):

Gillian McGovern, Associate Editor

Key Takeaways

BA1302, a CD228-targeted ADC, received orphan drug designation for squamous NSCLC and pancreatic cancer, highlighting its potential in treating solid tumors.
The phase 1 trial assesses BA1302's efficacy, safety, and pharmacokinetics in advanced solid malignancies, including NSCLC, pancreatic cancer, breast cancer, and melanoma.
BA1302's design minimizes off-target toxicity by binding only to the membrane form of CD228, enhancing its therapeutic window.
Preclinical studies indicate BA1302's promise as a monotherapy or in combination with other drugs for various cancers.

The antibody-drug conjugate is currently undergoing evaluation in a phase 1 trial that enrolled patients with pancreatic cancer and squamous non-small cell lung cancer (NSCLC).

The FDA granted an orphan drug designation to BA1302 (Boan Biotechnology) for the treatment of patients with squamous non-small cell lung cancer (NSCLC) and pancreatic cancer.¹ Preclinical studies have demonstrated BA1302’s potent internalization and bystander effect, and it is currently undergoing evaluation in a phase 1 (NCT06596915)² clinical trial.^1,3

Image credit: sinhyu | stock.adobe.com

BA1302 is an innovative, targeted CD228 antibody-drug conjugate (ADC). It is the only ADC that is in clinical stages intended for the treatment of various solid tumors, according to a news release.¹ ADCs are drugs in which antibodies and toxins are conjugated via linkers. They direct toxins towards target cells to kill them; therefore, minimizing harm to normal cells, providing a wider therapeutic window.³ The antibody of BA1302 is able to bind only to the membrane form of CD228 and not the soluble form sMFI2, reducing the release of the payload in non-target cells and therefore minimizing off-target toxicity. It employs cysteine conjugation technology, which demonstrates strong in vitro and in vivo stability and anti-tumor activity.¹

The phase 1 trial evaluating BA1302 is a first-in-human, multicenter, open-label study to assess the ADC’s preliminary efficacy, safety, tolerability, pharmacokinetic profile, and immunogenicity in patients with advanced solid malignancies, including squamous NSCLC, pancreatic cancer, breast cancer, and melanoma. The trial consists of a dose-escalation phase (part A) and a dose-expansion phase (part B). All enrolled patients will receive intravenous administrations of BA1302.²

Additionally, patients are considered for enrollment based on the following: metastatic or unresectable solid malignancy (part A: advanced malignant solid tumors; part B: metastatic melanoma, advanced breast cancer, advanced NSCLC, advanced pancreatic adenocarcinoma) that is histologically or cytologically confirmed, has progressed on or after standard therapy, or is intolerant of standard therapy, or has no appropriate standard therapy available; are able to provide adequate tumor tissue for biomarker analysis; have an ECOG Performance Status of 1 or lower; and have measurable disease per Response Evaluation Criteria for Solid Tumors version 1.1.²

About the Trial

Trial Name: Safety and Efficacy of BA1302 in Patients With Advanced Solid Tumors

ClinicalTrials.gov ID: NCT06596915

Sponsor: Shandong Boan Biotechnology Co., Ltd

Completion Date (Estimated): December 31, 2025

The study’s primary outcome measures are the number of patients with dose-limiting toxicities and adverse events, which will be measured up to 21 days and through the last 28 days after the final study treatment, respectively. Secondary outcome measures will be measured up to about 1 year and include the following: area under the concentration-time curve (AUC) from 0 to the last quantifiable concentration; maximum serum concentration; elimination half-life; AUC at steady state; incidence of anti-drug antibodies; objective response rate; and duration of objective response.²

Preclinical studies have shown that CD228 is highly expressed in various solid tumors, and BA1302 is a promising drug candidate either as a monotherapy or in combination with other drugs for treating a broad range of cancers, explained Dou Changlin, PhD, president and chief operating officer of research and development at Boan Biotech, in a news release. “We’ll accelerate the clinical development of BA1302 and unleash its therapeutic potential, hoping to deliver a breakthrough therapy for cancer patients,” he said.³

REFERENCES

1. MooMoo Technologies Inc. BioanalytiX (06955.HK): BA1302 has received orphan drug designation from the USA FDA for squamous non-small cell lung cancer and pancreatic cancer. News release. March 26, 2025. Accessed March 31, 2025. https://www.moomoo.com/news/post/50852495/bioanalytix-06955-hk-ba1302-has-received-orphan-drug-designation-from?level=4&data_ticket=1743107942442664

2. Safety and Efficacy of BA1302 in Patients With Advanced Solid Tumors. ClinicalTrials.gov identifier: NCT06596915. Updated September 19, 2024. Accessed March 31, 2025. https://clinicaltrials.gov/study/NCT06596915

3. Boan Biotech. First in China: Boan Biotech’s Novel Investigational CD228-Directed ADC Approved for Clinical Trials. News release. July 26, 2024. Accessed March 31, 2025. https://www.boan-bio.com/en/info.php?id=313