FDA Warms to Heat Bio’s Bladder Cancer Immunotherapy

Melissa Price, Ph.D., Heat Bio's vice president of clinical development and regulatory affairs. -- Courtesy of Heat Biologics

The United States Food & Drug Administration likes what it sees in Heat Biologics’ (now known as Nighthawk Biosciences) developing therapeutic approach to attacking bladder cancer.

The FDA has issued a fast track designation for the young Durham company’s immune therapy, code named HS-410 (Vesigenurtacel L).

The designation means that the FDA will take a range of actions to work more closely with Heat to speed the review process, in the belief that Heat might be developing a platform technology that can save lives and reduce suffering, initially in people with so-called non-muscle invasive bladder cancer (NMIBC). 

HS-410 is in Phase 2 clinical trials, which are designed simply to evaluate its effectiveness as a vaccination in extending the time of disease recurrence compared to placebo.

The drug is based on the company's Immune Pan Antigen Cytotoxic Therapy (ImPACT) platform. The company believes it’s a platform technology because early testing indicates it can coax the body’s own immune defense weapons, called killer T cells, to attack numerous forms of cancer.

Heat was founded in 2008 to commercialize the cancer-fighting approach discovered at the University of Miami. CEO Jeff Wolf soon decided Miami wasn’t the right environment to build the company, explored several major biotech hubs, and picked the Research Triangle area after meeting with specialists at the North Carolina Biotechnology Center.

Wolf said NCBiotech convinced him he could get the talent and support he needed in North Carolina to build Heat into a successful biopharmaceutical company, so he moved his company, consisting of himself and one employee, into a temporary rental office at NCBiotech in 2011.

NCBiotech provides loans, other support to Heat Bio

Professionals in NCBiotech’s Business and Technology Development group worked closely with Wolf to help hone his pitch to investors. He also joined them and CEOs of other life science start-ups on NCBiotech-led visits to venture capital companies in Palo Alto and Boston.

NCBiotech provided Heat its first outside funding, a $225,000 Strategic Growth Loan. That opened doors to more investment opportunity for Wolf. Heat was able to repay the loan well ahead of schedule as other investment support came in.

Heat got another boost from one of NCBiotech’s $3,000 industrial intern awards in 2012, to help it reach the public-trading milestone of an initial public offering of stock on the NASDAQ exchange. By the time of the IPO offering of 2.5 million shares at $10 apiece, Heat had also garnered some $5 million in other outside investment.

Two drug candidates in pipeline

Heat already has two drug candidates in FDA clinical trials. The other, HS-110, is looking at the ImPACT approach to battle the deadly scourge of non-small cell lung cancer.

"We are very pleased that FDA has granted this important designation for HS-410," said Melissa Price, Ph.D., Heat's vice president of clinical development and regulatory affairs. "The decision underscores the unmet need for bladder cancer treatments and serves as an additional validation for our clinical program. Currently there are limited therapeutic treatment options available for this patient population, with no new treatments approved in this setting in over 30 years."

The FDA established the Fast Track Drug Development Program in 1997 to facilitate the development and expedite the review of therapies intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

Bladder cancer hits 75,000 in U.S., kills 16,000 each year

According to the American Cancer Society, bladder cancer is the fifth most common cancer in the U.S. There were about 75,000 new cases in 2014, and it claimed some 16,000 lives. About 75 percent of the newly diagnosed patients have NMIBC.

One of the discouraging aspects of bladder cancer is its high rate of recurrence. When that happens there are few treatment options other than complete bladder removal. Heat is currently examining HS-410 in conjunction with standard treatment to stimulate the immune system and eliminate remaining cancer cells, to prevent recurrence.

The company plans to release interim data in the next couple of months on 10 patients who enrolled in a Phase 1 study. The Phase 2 clinical trial enrollment is expected to be completed by the end of September. Initial results of that study, involving a larger group of patients, should be made public by the end of 2016.

Heat’s ImPACT Therapy is designed to deliver live, genetically modified, irradiated human cells which are reprogrammed to pump out a broad spectrum of substances toxic to cancer cells. ImPACT is a one-two punch that also includes a potent immune-system booster called gp96, a protein found in all human cells. Heat taps gp96 to educate and activate cancer patients’ own immune systems to better recognize and kill cancerous cells.

Heat's thinly traded and volatile stock surged nearly 35 percent when the FDA news broke, but settled back to a gain of just over 15 percent by mid-afternoon.