Small Start-ups Outpace Big Pharma in Cancer Drug Discovery

The Calistoga story

Seattle-based Calistoga Pharmaceuticals began as a passion project. The small biotech start-up was the brain child of Roger Ulrich, a Big Pharma veteran with a 20 year track record in the industry. When Ulrich lost his job, he partnered with two colleagues to found Calistoga, despite warnings of risk and potential loss.

Focused on clinical research for cancer treatment, Calistoga soon began testing a new drug for chronic lymphocytic leukemia. The trial was so incredibly successful, that it was halted midway so participants in the control group could choose to switch over and begin receiving the treatment. The drug is called Idelalisib and its expected impact on treating the chronic form of leukemia is so impressive that Calistoga was offered a multi-million dollar buy out from Gilead. In 2011, Ulrich’s passion-project was acquired for over $375 million dollars.

When it comes to drug research and development, the pharma industry is seeing a growing trend of small biotech start-ups taking risks and venturing down discovery pathways where the big companies will not go.  The result?  Innovative new therapies like Idelalisib, capable of changing the prognosis for patients with debilitating disease.

How does Idelalisib work?

Idelalisib began as a potential asthma therapy. It works by targeting B-cells, part of the immune system, but at the time of its development this particular target was unproven to be important in cancer.  Ulrich and his team were certain that Idelalisib could be effective in B-cell malignancies.  They urged the drug through the typical process of testing and pharmaceutical quality assurance. And now, studies show that the compound significantly reduces the rate of disease progression or death in phase 3 chronic lymphocytic leukemia patients. The drug is groundbreaking because it:

  • Offers an effective alternative to patients for whom chemotherapy is not an option
  • Selectively inhibits the enzyme PI3-kinase delta, which is hyperactive in chronic lymphocytic leukemia
  • Is capable of halting progression and improving survival rates of patients previously diagnosed as terminal

Inspiring new drugs

On December 7, the American Society of Hematology will meet in New Orleans to discuss several new chronic lymphocytic leukemia drugs arriving on the market. The new treatments come from Gilead, Pharmacyclics, Infinity, and Roche. These new therapies offer patients hope in the face of a disease often characterized as a slow march toward death.  Industry leaders, professional in the field, and students of pharmaceutical courses can agree that the next 10 years will see tremendous advances in how CLL is treated, particularly from risk-taking start-ups like Calistoga with the vision and expertise to develop innovative solutions.

Do you think biotechnology, versus traditional Big Pharma, will dominate drug discovery in the years to come?