Multiple myeloma is a cancer that affects plasma cells in bone marrow. Myeloma develops in 1-4 per 100,000 people per year. A few companies have been working to develop several potential strategies for gene therapy in the treatment of this type of cancer.
Let's talk about some companies that are working on this.
ONYX Pharmaceuticals (ONXX)
ONXX has had a great 2012, stock price running from $45 to around $90. ONXX saw accelerated approval on July 20th for the drug Kyprolis (Carfilzomib) which is a treatment for multiple myeloma. ONXX third quarter results were pretty good, considering the drug was being sold for the first time in this quarter. Sales revenue topped $18.6 million.
There are a few competitive products to Kyprolis. Celgene's (CELG) pomalidomide is now in phase 3 for myeloma. Takeda Pharmaceutical (TKPHF.PK) also produces Velcade, which has been in the market since 2003. Some patients might be intolerant to Velcade and that is where Kyprolis comes in.
ONXX has made significant progress in trying to build a successful oncology company. Nexavar was approved for advanced kidney and liver cancer. Nexavar sales exceeded $1 billion for the first time in 2012, with strong growth in the liver cancer area. Stivarga is another drug that is showing promise. Additional clinical results in 2013 both for Nexavar and Kyprolis are due. Stivarga is a multi-kinase inhibitor, which recently completed clinical trials, recent U.S. regulatory approval for metastatic colorectal cancer, and a number of regulatory submissions in place both for metastatic colorectal cancer and for gastrointestinal stromal tumors. ONXX has a global collaboration with Bayer Corporation (BAYZF.PK).
ONXX is valued at approximately $5 billion. There is a high percentage of shorts (around 8.4% of the float) with the float reading 66.72 million. The company has $554m in cash, which reduces the chances of another secondary offering of shares. Institutions have also been increasing their holdings in ONXX as can be seen here-- another reason to buy.
Senesco's (SNTI) story
SNTI's product candidate downregulates an important pathway that is controlled by a protein called NF-kB. SNS01-T exerts its effect on this protein by a different route. SNTI's platform technology is based on the discovery that the eIF5A is a gene which has been found to regulate apoptosis, resulting in it playing a major role in the management of cell death and survival. The eIF5a pathway appears to be a 'master switch' for a natural growth that functions in most if not all cancers.
SNTI's leading candidate SNS01-T is currently in Phase 1b/2a trial. The results presented in December are very promising. For patients 41-002 and 42-002 in cohort 1, serum levels of M protein remained within 25% of the baseline values. both at weeks 3 and week 6. For patient 42-002, M-protein stayed with 25% baseline at week 10, or 4 weeks after the end of the treatment. The plasma cells for these patients declined by 29% and 70% respectively, which shows that death of myeloma cells was taking place. Interestingly, there was no drug-related serious adverse events or disease limiting toxicities, a big plus. SNTI now aims to finish dosing in early 2013 and if the Data Review Committee gives the go ahead, dosage will be increased.
SNTI is valued currently at around $14million. I find this relatively cheap after studying recent studies and its potential in the future. After the recent secondary offering of shares in January, the company should have enough cash to last the company till the end of July. One should note that Christopher Forbes has invested heftily in the company-- he bought $1.1m worth of shares in early August 2012.
Other possible upcoming drugs
Pharmacyclics (PCYC) has a drug known as Ibrutinib. This is an experimental drug for the treatment of various types of cancers. Elotuzumab is also being developed by Bristol-Myers Squibb (BMY). This is a humanized clonal antibody which is presently under phase III clinical investigation in relapsed multiple myeloma. Elotuzumab has shown terrific results in the slowing of malignancies.
In the coming years, we will have several new anti-myeloma agents available on the market. It is an exciting and very promising time to treat myeloma patients, with unprecedented outcomes. Major progress in understanding interactions between the immune system and malignant cells will augment the studies being undertaken by the above mentioned companies. In my opinion, both ONXX and SNTI are looking cheap.
Disclosure: I am long SNTI.OB.