Nodular Basal Cell Carcinoma

The Clinical Need

Skin malignancies are the most common cancers, and account for nearly half of all cancers in the United States; while in Australia and New Zealand, they exceed all other cancers combined. There are currently 2-3 million skin cancers occurring annually worldwide and close to 80% of skin malignancies are basal cell carcinomas. The two major types of BCC, superficial and the nodular forms, are slow growing malignant cancers originating in the dermis. These tumours rarely metastasize, but can cause significant local tissue destruction and can affect normal tissue function.

Currently, surgery is the main treatment for BCC and it has a success rate of ~92%.  However, surgery can often be difficult and complex when it occurs around the face, nose, eyes and ears with concerns of scarring, healing and function.  Surgery is also not recommended for patients on blood thinning treatments or for older patients.  Effective non-surgical alternatives are desirable for high-risk nodular BCC, where there is a high risk of the cancer returning.

Of the reported ~2.75 million cases of new BCC annually in the United States, 60% represent nodular BCC (~1.8 million cases) and of these it is estimated that around 440,000 could represent difficult surgery cases.  A market potential of >$500 million is possible for ASN-002 as a treatment for this nBCC indication.

Our Areas of Focus

Ascend is developing ASN002 as a treatment for nodular BCC patients where surgery is considered undesirable for clinical or cosmetic reasons.  ASN002 is based on a replication deficient adenovirus type 5 (Ad5 is a type of common cold virus), engineered to express interferon – a potent protein that can stimulate the immune system to destroy cancers.  ASN002 induces Interferon alpha (IFNα) and produces Interferon gamma (IFNγ) upon injection into skin cancers.

Rationale

The interferon family of proteins has been previously shown to be effective in treating basal cell carcinoma when injected as a recombinant protein.  However, recombinant protein rapidly leaks from the tumour site into the blood, necessitating frequent injections that result in greater side effects.  In contrast, ASN-002 has the ability to overcome this deficiency by enabling a sustained local production of the protein contained primarily within the tumor site, thus reducing the frequency of injections required and the systemic adverse events.  Because Interferon-γ receptors are expressed on most cells in the body, the local level Interferon-γ released by ASN-002 within the tumor would limit systemic toxicities.  In addition, by confining  IFNγ locally within the lesions, the chemokines induced by this cytokine would be more effective in selectively recruiting inflammatory cells to the tumor environment (this selective recruitment may be diminished with injected recombinant cytokine).

Further information for patients