With ASCO 2012 rapidly approaching, investors have been hedging their bets on upcoming data via the many posters presented at the prestigious event. Although the meeting doesn't officially occur until June 1-5th, abstracts were released online on May 16th and gave investors a preview of things to come. This is the venue where many small biotechs await to present and even introduce their technologies and data to investors, potential suitors, the healthcare community and the all-important stock analysts.
There are four early-stage biotechs with market capitalizations ranging from $105 million to $386 million with data being presented that will likely have a strong year ahead of them due to ASCO data and other on-going trials. Many of their stock prices have risen in recent months due to data already released and more can be in store for the year ahead.
ImmunoCellular Therapeutics (NYSEMKT:IMUC) presented phase I data at ASCO 2011 for its ICT-107 immunotherapy treatment to fight glioblastoma multiforme (GBM). The treatment is used as an adjuvant to be utilized after the standard of care chemotherapy, radiotherapy and/or resection. An agent used to fight cancer stem cells and thereby prevent the recurrence of this devastating disease, ICT-107 impressed in 2011 and looks to have a repeat performance in 2012. The 2011 data results showed median survival was extended almost two years (38.4 vs. 14.8 months) in patients treated with ICT-107 compared to patients on the current standard of care.
The 3-year survival rate was 55% for ICT-107 patients vs. 16% for historical controls. 6 of the 16 patients in the Phase I trial remained free of disease recurrence over 3 years with at least one patient exceeding five years of progression free survival. Abstract 2087 presents data for the on-going study with impressive data for the 16-patient set. As of February 2012, 6 of the patients showed no evidence of tumor recurrence in 44-63 months. Median progression free survival (PFS) in the patient set was 16.9 months with a 2-year PFS of 43.8%.
The median overall survival rate was 38.4 months with a 2-year overall survival of 80.3%. Abstract TPS2107 is about the enrollment and patient set for its follow up Phase II trial. No data was mentioned in the phase II abstract. If any interim data is presented confirming phase I results, this would be a huge catalyst for this small biotech.
Galena Biopharma (NASDAQ:GALE) will be presenting data on its NeuVax (E75) breast cancer trials. NeuVax is also an immunotherapy agent and is used to prevent recurrence of breast cancer in HER2+1 and +2 expression patients after the standard of care treatment regimen. Follow up data on its ongoing Phase II trial still show a strong immune response against breast cancer recurrence with a 3.8% recurrence of cancer in the booster group versus 18.9% in the control group for a (p=0.01) performance correlation, a statistically significant difference.
Taking another look at the data, this means 96.2% of the booster group patient set had no recurrence of breast cancer compared to 81.1% of the control group. The data confirms the robust nature of the treatment with the booster given every 6 months to keep the immune response for the therapy strong. Galena initiated a Phase III trial named the PRESENT trial using NeuVax with its booster program for patients having HER2 +1/+2 expressions in January of 2012. No data is likely to be presented at the venue for the Phase III trial, but an update on enrollment is likely with any other news about the trial possibly unveiled.
ArQule, Inc. (NASDAQ:ARQL) will be presenting positive Phase II data for tivantinib, an oral, selective inhibitor of the c-MET receptor tyrosine kinase with an indication of fighting hepatocellular carcinoma (liver cancer). Tifantinib is used as a second-line treatment regimen after progression occurred in first-line treatments, a difficult patient set. For the overall patient set, the treatment increased the median time to progression to 1.6 months relative to 1.4 months for the placebo group to give a statistically insignificant (p=0.04). However, clinical trials are meant to determine safety and efficacy for targeted patient sets, and it appears the company found a statistically-significant patient subset for its HCC therapy.
In a subset of patients with HCC tumors over expressing c-Met, median time to progression was 2.9 months for the tivantinib treatment group relative to 1.5 months for the placebo group, a 93% improvement. A little work will need to be done by investors to determine the size of the patient group this subset represents, but significance there (which could be mentioned in the actual poster presentation) could still be a catalyst for the company. Additional indications underway for the drug are for colon cancer in a Phase II trial with results like in Q1 2013 and a Phase III trial underway for non-small cell lung cancer with interim results expected Q4 2012.
Oncothyreon, Inc. (ONTY) will present data on its Phase I trial on PX-866 for patients with recurrent glioblastoma. Interim results on the abstract indicate a general failure of the trial with 12 of the 17 patients withdrawing from the trial due to disease progression or elevation of liver enzymes, the latter being an indication of liver failure.
The drug's future is still likely to be intact as Phase II trials are currently underway in combination with other treatments for glioblastoma, colon cancer, head-and-neck cancer, prostate, and colon cancer. The drug has been well-tolerated in trials, so efficacy for any of these indications will likely be a green light to proceed farther. Recurrent glioblastoma is a truly difficult patient set, so failure here does not end the life of PX-866.
ASCO is a great venue with the opportunity for investors, the financial community and the healthcare community to see the "latest and greatest". This is the biotech and small pharmaceutical sector, and these cancer trials can all be used to further the fight against cancer whether these trials meet their predetermined endpoints or not.
Data is being accumulated on what works and what doesn't work for many types of treatments be they chemotherapy, immunotherapy, hormone therapy or a host of other treatment approaches. In the end, patients in need of a cure will be the true winners. We as investors hope to share in the success of the fortunate companies, and any of the four aforementioned companies could have the recipe for success brewing in current trials.