BioTime (NYSE:MKT, TASE:BTX) has multiple data milestones scheduled this year in its two lead regenerative medicine programs: OpRegen in dry AMD and Renevia in facial aesthetics.
“We believe we are one of the leading regenerative medicines companies in the world because we have assembled the best IP and assets in this space and our lead products are maturing nicely,” co-CEO, Adi Mohanty, says in an interview with BioTuesdays.
“In the past few months, we have shared some very interesting human data, with more to come this year,” he adds.
BioTime presented positive early data from a pivotal trial of Renevia at the International Federation for Adipose Therapeutics and Science (IFATS) in November and promising early data from its Phase 1/2a trial of OpRegen at the International Symposium of Ocular Pharmacology and Therapeutics (ISOPT) in December.
According to Mr. Mohanty, regenerative medicine changes the focus from treating the symptoms of chronic and degenerative diseases to possibly providing actual cures. It uses the latest advances in stem cell biology, embryology, tissue engineering and medicine to develop products for the replacement, restoration or regeneration of damaged or diseased cells and tissues.
BioTime has two platform technologies: PureStem progenetor cells and HyStem hydrogel. It also has significant ownership in two public companies: Asterias Biotherapeutics (NYSE MKT:AST) and OncoCyte (NYSE MKT:OCX).
OncoCyte is focused on non-invasive blood-and urine-based diagnostics for early detection of cancer and could have a confirmatory diagnostic for lung cancer on the market later this year. Asterias is tackling leukemia and spinal cord injury with platforms based on pluripotent stem cell therapies and immunotherapies.
Mr. Mohanty says BioTime’s PureStem cells have the potential to overcome significant challenges in regenerative medicine through their purity, their proliferative capacity and their ability to differentiate into a broad spectrum of cell types in a simplified and controlled fashion.
In addition, he says HyStem hydrogels are composed of a patented technology where naturally occurring components of the extracellular matrix, such as hyaluronan and collagen, are safely cross-linked in the presence of cells in the body to create three-dimensional tissue.
Mr. Mohanty explains that Renevia is not a dermal filler but was developed as a replacement for whole adipose tissue. Once implanted, Renevia provides a 3-dimensional matrix, with pliability comparable to natural adipose tissue, restoring texture in the short term while promoting soft tissue regeneration in the long term.
In earlier animal studies, Renevia was able to form new tissue at six months, “which is what we are trying to do in man,” Mr. Mohanty says. Human data presented at IFATS in November demonstrated that Renevia showed sustained facial contouring and increased tissue volume in subjects with HIV-associated lipoatrophy (HIV-LA), or facial fat loss.
Renevia currently is in a pivotal medical device trial in Spain to support CE Mark approval in patients with HIV-LA. The primary endpoint is skin thickness as measured by ultrasound at six months, with a secondary endpoint that includes aesthetic improvement.
Top-line efficacy data are expected at the end of the first half this year, with a possible filing for CE Mark before the end of the year.
“We see potential for a significant label extension with Renevia to create a facial aesthetics franchise in Europe, Asia, U.S. and beyond,” Mr. Mohanty contends. “After HIV-LA, we hope to extend the label to indications such as aging-related atrophy and reconstructive surgery where the objective is to safely regenerate small amounts of 3-D adipose tissue.”
More than 350,000 people in Europe have HIV-LA but the global facial aesthetics market is currently a multi-billion dollar market and is expected to top $5-billion by 2020, according to industry estimates. Clinical development of Renevia in generic facial fat loss in other major markets is expected to begin later this year, Mr. Mohanty suggests.
Earlier this month, BioTime and its Cell Cure Neurosciences subsidiary opened a GMP cell therapy-manufacturing center in Jerusalem. Initially, the facility will supply OpRegen for the company’s ongoing clinical trial program for the treatment of dry AMD, the leading cause of blindness in people over the age of 60.
There are two types of AMD: dry, or the atrophic form, and wet, or the neovascular form. Many times, AMD starts as the dry type and in 10% to 20% of individuals, it progresses to the wet type. In addition, AMD always occurs in both eyes, but does not necessarily progress at the same pace in both eyes.
AMD afflicts more than 30 million people worldwide, of which 90% represent dry AMD, with no approved therapy. Wet AMD patients currently are treated with monthly injections of Lucentis, Eylea and off-label use of Avastin.
Mr. Mohanty explains that OpRegen cells are introduced into the subretinal space where they are designed to replace retinal pigment epithelial (RPE) cells lost due to disease.
OpRegen cells are introduced into the subretinal space where they are designed to replace retinal pigment epithelial (RPE) cells lost due to disease.
The therapy is now in a Phase 1/2a dose escalation study at three sites in Israel to evaluate its safety and efficacy in patients who are at an advanced stage of dry AMD. OpRegen has received fast track designation by the FDA.
A secondary objective of the study will be to examine the ability of transplanted OpRegen to engraft, survive, and modulate disease progression in patients. In addition to visual function, a battery of vision tests will be used to quantify improvements in reducing the progression of the disease.
Early data presented at ISOPT in December indicate that OpRegen at an initial dose caused no serious adverse events in the first patient cohort and retinal imaging suggested the presence and survival of the transplanted cells in the subretinal space for up to one year.
BioTime plans to report data from each cohort, including complete 12-month data from cohort one in the second quarter this year and six-month data from cohort two in the third quarter. Also, in the third quarter, the company expects to receive approval from the Data Safety Monitoring Board to proceed to cohort four, with more clinically relevant patients.
“There is great anticipation that the size of the signal in cohorts two and three will determine how we interact with potential partners to complete clinical development and potentially commercialize OpRegen,” Mr. Mohanty says.
“We think that for the right deal, we would partner the product but our focus this year is generating data, which will lead to value creation and many options.”
