With a name like Lion Biotechnologies, it’s no surprise that cyberspace is littered with news stories about this public biotech company’s potential to roar.
However, in an interview during the J.P. Morgan Healthcare Conference in San Francisco in January, CEO Maria Fardis said that the company has perhaps been a bit too quiet.
“I don’t want to roar quite yet although we should be executing with roarage,” Fardis declared with a laugh.
Lion Biotechnologies is currently testing its cancer immunotherapy product based on tumor-infiltrating lymphocytes (TIL) in solid tumors like melanoma. It is one among scores of companies — many of who are venture-capital backed — looking to score it big by harnessing the capacity of the body’s own immune system to battle and ultimately control or end cancer.
The San Carlos, California-based company’s product — LN-144 — is the result of the research of Dr. Steven Rosenberg, chief of surgery at National Cancer Institute (NCI) who used an adoptive cell therapy regimen to tackle metastatic melanoma. According to the company’s website the therapy — which was licensed from NCI— is currently being tested in several trials to treat metastatic melanoma at NCI, MD Anderson Cancer Center, and the H. Lee Moffitt Cancer & Research Institute. Per ClinicalTrials.Gov, the estimated enrollment in the company’s own trial is 20.
So how does the therapy work?
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Tumor-infiltrating lymphocytes are able to detect abnormal cells but certain cancers create a hostile tumor microenvironment that allows them to evade being killed by TILs, Fardis said. Tumor cells also mutate so once the TILs travel to tumor site, they do not recognize the bad cells and remain in a sort of frozen state.
“They are not dead but are not activated,” she explained. “If you are able to get it out and activate them, they still have the capacity to kill the tumor. Based on that hypothesis [of Dr. Roseberg], we said how do we get the cells out, activated, back into the patient and overcoming the tumor microenvironment?”
Lion Biotechnologies does this first by resecting the tumor and letting the tumor cells sit in a solution whereby the TILs are isolated from the tumor. In vitro, those TILs are amplified to several billion cells. Before they can be introduced back into the patient’s body through infusion, the patient undergoes a round of chemotherapy, which leads to the destruction of the inactive lymphocytes and T cells. Thereafter the TILs that have been grown in number in vitro are infused back into the patient.
It is the hope that the activated, energized TILs can then attack the tumor cells in full force. Patients are also given IL-2, which is a protein that regulates the activities of white blood cells that give the body its immunity, as part of the therapy.
“The purpose of the IL-2 is to stabilize the brand new cells that are going into the patient’s body,” Fardis said.
Earlier, in the trial run by Rosenberg at NCI, 24 percent of patients got a complete response or total elimination of the melanoma tumors. Some of those patients have been cancer-free for over a decade, she said.
Now the goal is to complete the Phase II clinical trial as well as beginning a dialogue with the Food and Drug Administration.
“This year one of our main goals is to engage FDA so that we have a clear registration path ahead,” Fardis said.
Fardis replaced previous CEO Elma Hawkins in June and said she is also looking to start trials of the therapy in head and neck cancers as well. Along with the expansion of the clinical program, there will be an effort to rejigger the company’s manufacturing.
“The manufacturing duration was six weeks, so that was longer than I liked,” something she observed when she came in as CEO. “I would want it to be well below four weeks. We have the process and need to do a tech transfer to a manufacturing plate. And we need to do a clinical study to make sure that we didn’t change the manufacturing process in a meaningful way that affects the clinical outcome.”
The company announced it raised $100 million in a private round of financing the same days that it announced Fardis’s arrival as CEO. The stock jumped 40.46 percecnt on the news and ultimately closed at $8.65 that day. Between June 1, 2016 and Tuesday, the stock has increased 25 percent to $7.35.
Photo: Tom Brakefield, Getty Images
