Anda Biologics Advances Personalized Cancer Vaccine PCNAT-1 with FDA Orphan Drug Designation, Aims for Global Clinical Parity

In March 2022, ANDA BIOPHARMA’s personalized cancer vaccine (PCNAT-1) became the first product to receive Orphan Drug Designation (ODD) from the U.S. FDA for a personalized pancreatic cancer vaccine.

According to VCBeat New Medicine, the company is actively preparing its IND application.

ANDA BIOPHARMA is a first-in-class novel drug R&D enterprise, developing personalized therapeutic cancer vaccines for solid tumors such as pancreatic cancer.

After more than six years of dedicated efforts, ANDA BIOPHARMA has established comprehensive bioinformatics analysis platforms for personalized tumor variant identification, neoantigen prediction and vaccine design, immunogenicity and safety evaluation, functional analysis, and efficacy assessment, as well as cytological and immunological experimental technology platforms. Its independently developed prediction system, AD-INN, leverages AI algorithms to optimize existing open-source code, addressing key challenges in the neoantigen field by increasing the accuracy of neoantigen prediction from 40% to 60%.

ANDA BIOPHARMA began developing neoantigen recognition technology as early as 2014 and initiated recruitment for its first patient case in 2017. Its progress is aligned with international standards and remains at the forefront within China.

As of December 2021, there were 152 registered clinical trials for tumor neoantigen therapy. ANDA BIOPHARMA is among the first-tier laboratories that commenced operations early and have treated a large number of pancreatic cancer patients.

ANDA BIOPHARMA’s most advanced R&D product is a personalized therapeutic vaccine for pancreatic cancer. In clinical trials involving patients enrolled in a non-registration clinical study conducted in collaboration with the Department of Pancreatic Surgery at Changhai Hospital, those who completed two years post-surgery and received the full course of vaccine therapy demonstrated a significantly higher two-year survival rate compared to pancreatic cancer patients receiving standard adjuvant therapy during the same period. The vaccine substantially reduced tumor recurrence rates, which were markedly lower than those observed in historical control cases from the same timeframe.

Dr. Hu Landian, Chairman of ANDA BIOPHARMA, introduced to VCBeat New Medicine that the company’s goal is:Significantly reduce the postoperative recurrence rate in pancreatic cancer patients treated with personalized vaccines, achieving the long-term therapeutic goal of maintaining patient health for 5 to 10 years through vaccine therapy.

The greatest challenge in tumor vaccine development is time. ANDA BIOPHARMA’s tumor vaccines are personalized for each patient, resulting in a slightly longer production cycle from design to synthesis. However, the condition of cancer patients can be unpredictable; if the interval is too long, the efficacy of the vaccine may be compromised. “This also places higher demands on our product, requiring it to remain stable for long-term storage under relatively appropriate conditions. We have been continuously achieving breakthroughs along this path.”

While ANDA BIOPHARMA’s team focuses on tackling pancreatic cancer, known as the “king of cancers,” it is also developing vaccines for patients with high-incidence malignancies such as lung and breast cancer, and has even expanded its indications to include COVID-19.

Tumor vaccines have emerged as a major focus of research in recent years. They are applicable to both hematologic malignancies and solid tumors, offering both precision therapy and prevention of recurrence. In the tumor vaccine sector, several candidates have entered the Investigational New Drug (IND) stage; however, no personalized tumor vaccines have yet been approved for market launch globally.

Dr. Hu Landian, Chairman of ANDA BIOPHARMA, is a leading figure in China’s immunotherapy field. Prior to founding the company, he served as a returnee researcher at the Chinese Academy of Sciences (CAS), where he conducted many years of cutting-edge basic and translational research. He has led multiple national-level projects, including those under the National 863 Program and key projects funded by the National Natural Science Foundation.

Leveraging the CAS Key Laboratory of Tumor Microenvironment, the project team has access to an advanced platform for experimental biological techniques and instrumentation, positioning the enterprise as one of the knowledge-innovation technology transfer entities encouraged by the state.

In 1997, Professor Hu Landian and Professor Kong Xiangyin, the current Chief Scientist of ANDA BIOPHARMA, returned to China after studying in Germany. They participated in the Chinese Academy of Sciences’ Special Support Program for the Human Genome Project, where they achieved significant breakthroughs by leveraging advanced technologies. Their related research findings were published twice in Nature Genetics, and they received numerous honors, including the Second Prize of the National Natural Science Award and the First Prize of the Shanghai Science and Technology Progress Award.

Recalling his participation in the Human Genome Project, Professor Hu stated, “It was precisely because we participated in the Human Genome Project that we were able to use it as an early entry point in China to conduct genomic research on human monogenic diseases and complex conditions such as cancer, thereby accumulating substantial omics data and research experience.”

In the years since ANDA BIOPHARMA was founded, many students of Professor Kong and Professor Hu have shown strong interest in the company’s translational research. They participated in collaborative research projects during their academic tenure and later joined the company’s R&D team as full-time employees. With graduates joining the company each year, ANDA BIOPHARMA has, after more than six years of development, built a professionally trained R&D team comprising experts in tumor genomics, immunology, vaccinology, and other specialized fields.

Currently, ANDA BIOPHARMA has assembled an R&D team of more than 30 members, including multiple senior core professionals from the industry, and alsoA comprehensive technical framework encompassing prediction, preparation, clinical application, and monitoring has been successfully established. By leveraging an innovatively applied single-cell sequencing system, the activation status of patients’ immune cells is monitored throughout the entire process to evaluate therapeutic efficacy and dynamically adjust treatment regimens.

ANDA BIOPHARMA is expected to officially commence clinical trials in 2023. Currently, ANDA BIOPHARMA has reached consensus with numerous renowned Grade A tertiary hospitals across China on clinical collaboration and has obtained ethical approval documents.