Follow-On Indications

Candidate & Indication Development Stage
Preclinical Phase 1 Phase 2 Phase 3 Market
REM-001 Basal Cell Carcinoma with Nevus Syndrome and Locally Advanced Basal Cell Carcinoma
Preclinical Phase
Phase 1 Phase
Phase 2 Phase
Phase 3 Phase
Market Phase

Once progress is made with REM-001 Therapy in cutaneous metastatic breast cancer (CMBC), a number of closely related cancers are targets for further development. These include cutaneous metastases that arise from internal cancers other than breast cancer, as well as indications such as metastatic melanoma and head and neck cancer.

Other Cutaneous Metastatic Cancers

A meta-analysis has shown that approximately 5% of people with internal (non-melanoma, non-lymphatic, non-leukemic) cancers develop cutaneous metastatic tumors in their skin. Based on an estimated incidence of 1,500,000 such internal cancers in the United States, this means that the incidence of such cutaneous metastases is approximately 75,000 with a substantially higher prevalence, given the fact that individuals often live with metastatic cancer for years. Regardless of the primary source of the cancer, these cutaneous metastatic tumors often begin as small skin nodules but, as the cancer spreads, more nodules form and can eventually cover large areas of skin. With progression, the tumor field generally becomes more painful as tumors may grow larger and more numerous, become infected, ulcerate, bleed and carry a strong odor. Some common cutaneous metastatic cancers besides breast, include lung, ovarian and colon. As with CMBC, cutaneous metastatic lung cancer patients develop these tumor nodules on their chest, while with ovarian and colon cancer the tumors most frequently develop on the abdomen. Another cancer that frequently forms metastatic cutaneous tumors is metastatic melanoma. When it metastasizes, melanoma often forms fields of cutaneous tumors that behave in a very similar manner to the other cutaneous metastatic cancers. Part of our goal is to treat these cutaneous tumors as early as possible to either cause them to be locally eliminated or to slow their growth sufficiently to reduce their late stage development.

Head and Neck Cancers

This form of cancer develops in the throat, larynx, nose sinuses and mouth, all areas that should be accessible with the light delivery wand used in our CMBC program or a minor variation of it. Overall in the US there are approximately 60,000 cases of head and neck cancer diagnosed each year, and approximately 12,000 deaths. PDT has been studied in a research setting, both as a treatment to cure early stage cancers with minimal side effects, as well as a treatment to relieve side effects in patients with advanced cancers. Similar to the case with CMBC, head and neck cancer patients may not respond well to chemotherapy or they may not be able to tolerate the side effects of radiation and alternative therapies with fewer side effects and good efficacy are desirable. Beyond tumor elimination a more effective treatment for head and neck cancer patients would be one that can spare underlying or adjacent tissue or leave critical regions such as the larynx generally unaffected. Based on the research done using PDT of various forms in these patients, we believe REM-001 Therapy may be particularly effective in addressing most forms of head and neck cancer.

Basal Cell Nevus Syndrome

REM-001 Therapy has also been studied in patients with basal cell nevus syndrome (BCNS) who developed extensive basal cell carcinoma. BCNS is a rare but serious condition that is often characterized by the formation of multiple and recurring cutaneous basal cell carcinoma lesions. Estimates of its prevalence range from 1 in 57,000 to 1 in 256,000 which indicates it could qualify as an orphan disease indication. In a Phase 1/2 clinical trial (CA001B), 14 patients with BCNS were enrolled and treated with REM-001 Therapy using the same dosing conditions as were used in the CMBC trials. A total of 157 lesions were treated in these patients and showed a 91% overall response rate. This was composed of a 68% complete response rate (no remaining visible evidence of a lesion) and a 23% partial response rate (lesion was reduced in size by more than 50%). In addition, 7% of lesions had stable disease (any increase in lesion size was less than 25%). The various response rates are shown in the graph below and are similar to the results seen in CMBC patients as we would expect.

Until the recent FDA approval of the drugs Odomzo and Erivedge, treatment options for these BCNS patients were very limited. However, we believe that, based on their package inserts, Odomzo and Erivedge have dose limiting toxicity profiles which are broader in scope than the primarily transient adverse effects observed with REM-001 Therapy. We believe that the potential toxicity limitations related to the existing therapies for BCNS, plus the positive initial Phase 1/2 data generated in clinical trials with REM-001 Therapy, suggest that REM-001 Therapy could be a viable alternative in treating recurrent basal cell carcinoma in BCNS patients.

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