What could be the most advanced attempt to bring stem cell treatments to the market could start any day now in Orange County.
NeoStem Inc. said last week that the Food and Drug Branch of the California Department of Public Health issued it a manufacturing license for its plant on Von Karman Avenue in Irvine, allowing it to make drugs for a previously announced third-phase clinical trial for treating skin cancer.
“We are poised to start any day now,” said Dr. David Mazzo, NeoStem’s new chief executive, referring to plans for manufacturing and the company’s Intus clinical trial.
“With the [license], we remain on track to begin patient enrollment in our pivotal Phase 3 trial,” Mazzo said.
A successful trial could poise NeoStem to get cancer treatments based on stem cells on the market as early as next year under the company’s most hopeful timeline.
NeoStem develops stem cell therapies for cancer, heart disease and other conditions.
The New York-based company claimed ground among Orange County’s field of drugmakers last year with the acquisition of California Stem Cell Inc. from onetime University of California-Irvine professor Hans Keirstead.
Stem cell pioneer Keirstead, who remains with the company as president of its NeoStem Oncology unit, sold California Stem Cell in a stock deal worth at least $34 million. The payment could go high as $124 million with milestones and royalties.
Keirstead, who couldn’t be reached for comment last week, told the Business Journal last year that NeoStem “is the right partner in a true strategic sense in that the greatest challenge in the commercialization of stem cells and clinical development of stem cell technology is manufacturing.”
“NeoStem was founded on a base of manufacturing expertise,” Keirstead said at the time of the deal. “… I thought, ‘OK, they appreciate that the primary block [to stem cell commercialization] is manufacturing capacity and expertise. I really see it as a means to advance my company by a decade, frankly.”
Ensuring integrity and capability in manufacturing can make or break a stem-cell enterprise, Keirstead said last year.
“This is something that has dogged the industry in this sector—look at Geron,” he said.
Menlo Park-based Geron Corp. ended its spinal injury stem cell program four years ago in the wake of manufacturing problems.
NeoStem will make and distribute a stem cell therapy known as eltrapuldencel-T for clinical trial use at the Irvine plant. Eltrapuldencel-T, also known as NBS20, is being investigated for treating stage 4 and recurrent stage 3 metastatic melanoma and is intended to eliminate cancer-initiating stem cells that NeoStem says are capable of causing disease recurrence and progression.
NeoStem will make NBS20 from stem cells that are isolated from a patient’s tumor sample, inactivated and enriched.
The newly created cancer-initiating stem cells will be combined with dendritic cells derived from the patient’s own blood and a blood component called granulocyte-macrophage colony stimulating factor, which stimulates white blood cells in the patient’s body.
NBS20 will then be injected back into the patient.
NeoStem’s manufacturing process is familiar to some OC biotech watchers. Dendreon Corp.—a Seattle-based biotech that has a Seal Beach manufacturing plant and filed for bankruptcy in November—uses a process developed by NeoStem’s Progenitor Cell Therapy subsidiary to make its Provenge prostate cancer treatment from a patient’s own blood cells.
NeoStem’s “overall concept is the same—use the patient’s immune system,” Mazzo said.
NeoStem’s Intus trial is expected to enroll 250 patients in the U.S., New Zealand, Australia and Canada. Patients will receive weekly injections of NBS20 or a control treatment for three consecutive weeks, and once a month for five months.
Mazzo said NeoStem sought out California Stem Cell and a presence in Orange County because “the area is generally attractive to biopharma” with proximity to “well-known universities.”
“The community is well-established here,” he added.
NeoStem plans to keep its corporate operations in New York as it pushes forward with manufacturing here.
“I would say it’s highly unlikely that the company’s headquarters will move to Irvine,” said Mazzo, who recently started as NeoStem’s chief executive.
“In today’s world, you have multiple sites and multiple international sites—headquarters is sort of a legal definition,” said Mazzo, who plans to come to Irvine “as appropriate.”
Mazzo, who has spent more than 30 years in the pharmaceutical industry, succeeded Dr. Robin Smith, who is now NeoStem’s executive chairman.
NeoStem might add some jobs based on results of its drug trial, but Mazzo did not give any specific numbers.
“We have to balance our desire to expand” with frugality because “we are still not profitable,” Mazzo said.
The company lost $43.8 million on revenue of $12.7 million in the nine months through Sept. 30.
NeoStem hasn’t yet set a reporting date for its fourth quarter and 2014 financial results.