Imagine being a transplant patient, and you’ve just received the bone marrow transplant you desperately need to survive. Unfortunately, a full recovery is not a given. Immediately after a transplant, before the immune system reconstitutes, the patients are very vulnerable to infection. In fact, research shows that more than 27,000 patients are projected to receive allogeneic hematopoietic stem cell transplant (allo-HSCT) in 2017 (US and EU), and up to 70 percent of those patients may suffer from a severe viral infection1. Unfortunately, today there are no FDA approved drugs or effective experimental therapies to treat most of these infections, but ViraCyte is on a mission to change that.
ViraCyte, a resident company at JLABS @ TMC in Houston, Texas, focuses on combating these infections. Spun out of Baylor College of Medicine in 2013, the company’s mission is simple: to safely and effectively treat viral infections that attack people with weakened immune systems. ViraCyte’s therapies infuse patients with activated T cells that are highly specific for attacking viruses; these T cells are obtained from normal donors and following activation and expansion, can be kept cryopreserved for years until patients need them.
“I was a pediatric critical care physician prior to joining ViraCyte, and some of the most challenging cases I faced were severe viral infections after stem cell transplant,” said Brett Giroir, M.D., President and CEO of ViraCyte. “Typically when patients contract a virus like this, there’s nothing we can do except to provide supportive care and wait for the immune system to reconstitute.” When I saw the work that ViraCyte was doing, I knew I had to be a part of it.”
The results are positive and encouraging. In the past 8 months, ViraCyte has released its phase 1 and phase 2 clinical results. In phase 1, which focused on the safety and efficacy of Viralym-C, the therapy controlled infections within six weeks of infusion for all ten patients with drug-refractory CMV infection. In the phase 2 results, Viralym-M, ViraCyte’s flagship therapy, achieved a 92% overall clinical response after a single infusion and demonstrated efficacy against all five targeted viruses.
Based on the results of phase 1, ViraCyte was granted a Fast Track designation by the U.S. Food and Drug Administration in early January of 2017.
“Fast Track designation emphasizes the importance of new cell therapies, like Viralym-C, which holds unique promise in treating severe infections in patients with weakened immune systems such as adults and children following stem cell transplants,” said Dr. Giroir. “This was a huge step forward for us as a company.”
The success didn’t stop there. ViraCyte continued to get positive results and shortly thereafter, Viralym-C was granted an Orphan Drug designation by the FDA. Orphan Drug designation qualifies sponsors who are developing therapies for rare diseases that affect fewer than 200,000 people in the United States for certain development incentives, including tax credits for clinical research costs, frequent FDA interactions, and protocol assistance.
Although ViraCyte focused on the stem cell transplant population first, their roadmap includes all patients suffering from viruses due to weakened immune systems. Other organizations are starting to take notice, and in early July ViraCyte announced that the NIH National Heart, Lung and Blood Institute (NHLBI) awarded the company a $3 million Phase IIB Small Market Award under the NIH Small Business Innovation Research (SBIR) program. The Program supports the development of innovative technologies addressing rare diseases and/or young pediatric populations to advance the commercialization of promising new products. With this new capital, ViraCyte will perform advanced clinical development of a T cell immunotherapy for BK virus in stem cell transplant recipients. BK virus causes severe disease including hemorrhagic cystitis and nephritis and can lead to renal failure, hemorrhage, and death in transplant recipients.
Additionally, ViraCyte was recently awarded a $750,000 Orphan Products Clinical Trials Grant by the FDA Office of Orphan Product Development (OOPD) for a Phase I clinical trial of Viralym-A in stem cell transplant recipients with Adenovirus. The OOPD funds the clinical development of products for use in rare diseases for which there are limited or no current therapeutic options. There are currently no FDA-approved treatment options for BK virus or Adenovirus.
With all ViraCyte’s success in the clinic, the most impactful is that on the patient population. Because of the clinical results, and market need for these therapies, the Cancer Prevention and Research Institute of Texas (CPRIT) Oversight Committee recommended funding for a product development research grant totaling $8.99M to support the clinical development of Viralym-M.
So, it begs the question, why would a company with solid funding, strong ties to academia, and nationally recognized leadership come to JLABS? According to Chief Scientific Officer, Dr. Ann M. Leen, it’s all about the infrastructure. “ViraCyte could not have happened without financial backers, professional advisers and JLABS. Here, we have everything we need to be successful. The labs are state of the art, we have access to expertise inside Johnson & Johnson, and the infrastructure allows us to focus solely on the science.”