What is Addaptimmune?
Adaptimmune is at the forefront of engineered cell therapies, targeting cancer, particularly solid tumors which have not previously been addressed by cell therapy. This innovative approach involves engineering cells to recognize and destroy cancer cells, a method that has shown promise in blood cancers but is now being applied to solid tumors. Adaptimmune is leading with two products poised to enter the market in the next two years, marking significant advances in the treatment of cancers such as bladder, head and neck, ovarian, and rare sarcomas.
The company's efforts are particularly focused on solid tumors, which represent 90% of the cancer mortality worldwide. Until now, there have been no cell therapies for these types of cancers. Adaptimmune's pioneering work in this area offers hope for new, effective treatments for these challenging diseases.
Can you tell us more about the BLA acceptance for afami-cell and its significance?
The BLA filing and acceptance for afami-cel is a pivotal moment for Adaptimmune, representing the final stage before securing FDA approval. The application has priority review status for our which ensures an expedited review of 6 months and a target approval date of August 4, 2024. The filing indication for afami-cel is for people diagnosed with advanced synovial sarcoma, a rare but aggressive cancer most often affecting younger adults . Our therapy has shown a 40% response rate in the SPEARHEAD-1 trial, offering a potentially transformative one-time treatment.
This approach is particularly revolutionary for its ability to provide durable benefits from a single dose, underscoring the transformative potential of cell therapy in cancer treatment. For patients, this means the potential to be free from the continuous, often challenging regime of conventional treatments for late-stage disease
How prevalent is synovial sarcoma, and who can benefit from your therapy?
In the United States, around 1,300 individuals are diagnosed with synovial sarcoma annually, with numbers slightly higher in Europe and globally. However, our therapy is highly targeted, designed for patients with a specific HLA type, HLA-02, which represents about 45% of the population. Furthermore, the therapy targets the MAGE-A4 antigen, present in about 70% of synovial sarcomas, narrowing the eligible patient population to approximately 400 to 500 annually in the U.S. This focus allows us to address a rare condition with a substantial medicalneed effectively, demonstrating the potential of cell therapies to transform treatment paradigms.
Does Adaptimmune benefit from orphan drug development incentives in the US?
Yes, Adaptimmune has leveraged regulatory pathways designed to expedite the approval of innovative therapies, particularly for rare conditions. The RMAT designation from the FDA, based on early-stage trial data, has been instrumental in this process. It not only facilitates a faster review cycle but also ensures close interaction with the FDA, crucial for navigating the challenges of developing and regulating new therapies. This close collaboration has been vital in de-risking key aspects of our therapy's development and regulatory approval process.
Are there plans to expand this or other treatments to address other cancers?
Absolutely. Beyond synovial sarcoma, our pipeline includes therapies targeting a range of cancers, including ovarian, bladder, and head and neck cancers.
Our next-generation cell therapies have shown promising results across various solid tumors, demonstrating about a 35% overall response rate even in late-stage patients. In ovarian and urothelial cancers the response rates are 40% and 57% respectively in this trial with a small group of patients.
Our focus now extends to scaling up manufacturing to meet the demand for these personalized treatments, emphasizing the importance of in-house capabilities to ensure quality and efficiency.
How does the manufacturing process for these cell therapies work?
Our manufacturing process is tailored to each patient, starting with the extraction of the patient's T cells and engineering them to target cancer. This personalized approach, where cells are grown to billions in number and then reintroduced to the patient, takes four to six weeks. It's a stark contrast to traditional pharmaceutical manufacturing, highlighting the unique challenges and profound connection to patients inherent in cell therapy. Our in-house manufacturing capabilities, particularly for afami-cel, are pivotal to delivering these therapies efficiently and effectively, emphasizing the personalized nature of this revolutionary cancer treatment.
What are your projections for patient treatment numbers from August onwards?
Initially, Adaptimmune aims to treat up to a thousand patients a year in the United States with its pioneering cell therapies, focusing on rare tumor types where alternative treatments are limited. The company's efforts are concentrated on identifying and reaching patients with specific HLA types and cancer markers, such as HLA-02 MAGE-A4 positive individuals with advanced synovial sarcoma. While it's ambitious to aim for full coverage, the goal is to treat as many eligible patients as possible, leveraging the unique position of Adaptimmune's therapies in addressing unmet medical needs in cancer treatment.
How has the financial market responded to Adaptimmune's advances?
The cell therapy sector experienced a wave of enthusiasm, followed by a significant slump, particularly affecting biotech and advanced therapy companies. Adaptimmune is navigating this challenging landscape with the conviction that demonstrating the effectiveness and economic viability of its therapies is key to regaining momentum. The company's focus on developing therapies that offer significant benefits to patients, including the potential for long-term benefits from a single treatment, is expected to enhance its attractiveness to patients and investors alike. As Adaptimmune advances towards FDA approval and market introduction, it anticipates a positive impact on its valuation and market position.
What is your vision for Adaptimmune in the next 5 to 10 years?
Adaptimmune aspires to be a leading, integrated commercial cell therapy company, transforming technology into valuable therapies for patients. The company's ambition is to commercialize its cell therapies independently, creating value through direct patient benefits.
We aim to lead the industry in developing and delivering innovative treatments for solid tumors and potentially changing the landscape of cancer treatment.
