PlasmaTech Biopharmaceutical’s New CEO Tim Miller on building a world class cell and gene therapy company focused on rare diseases Print E-mail
By M.E. Garza, Editor   
Friday, 08 May 2015 00:00

icon_qaexclusiveThis week, marked several major milestones for PlasmaTech Biopharmaceuticals, Inc. (PTBI), a Dallas and NYC-based biopharmaceutical company advancing protein biologic therapies and oncology supportive care products. Earlier in the week, business magnate, George Soros acquired a 5% stake in the biotech company.

Then it announced that PlasmaTech entered into a definitive agreement to acquire Abeona Therapeutics, a Cleveland-based company engaged in the development and commercialization of therapies for patients with lysosomal storage diseases. Under the terms of the agreement, PlasmaTech will issue to Abeona Therapeutic members a total of 3,979,761 common shares upon closing of the transaction, and up to an additional $9 million in performance milestones, in common stock or cash, at the Company's option. Additionally, Abeona’s CEO Tim Miller now takes the helm of CEO PTBI. We sat down with Miller to inquire about his long term strategy for PlasmaTech. 

Q: Can you explain to BioMedReports readers, what causes Sanfilippo syndrome? 

Miller: Sanfilippo syndrome is a central nervous system disorder, you are born with these inborn errors of genetic mutations that result in a mutation that effects a cellular organs called the lysosome. Think of these lysosomes as a kind of the garbage factory in the cell that are used to take proteins in gobble them up and then allow them to be used for the rest of the cell for metabolism. The children that are born with Sanfilippo lack the enzymes to break down certain proteins in the cells and as a result their lysosomes swell up, the result of which is significant neurological manifestations.

Q: What happens to children affected by Sanfilippo syndrome? 

Miller: Children affected by Sanfilippo syndrome are often not diagnosed until they experience some form of developmental delay. Parents take their children to their doctor to have testing performed, and eventually, they get a genetic confirmation. Children are able to progress to a certain point developmentally, usually between the ages of 2-6.  Parents often observe developmental delays and cognitive impairments with as well as problems with physical skills such as walking.  As the children move into their teenage years, many are bound by wheelchairs and have difficulty eating and swallowing. Many lose the ability to talk by the time they are 6 or 7 years old and 70% of the children afflicted with the disease do not live to see their 18th birthday.

Q: What treatment options to these patients currently have?

Miller: There are no FDA approved therapies for Sanfilippo syndrome. There are some therapies that are in development and typically they fall into two categories: enzyme replacement therapies and gene therapy strategies.  It is generally appreciated that a challenge of enzyme replacement therapies is the inability for the enzyme to cross the blood brain barrier, which is crucial for treating the brain and it’s something that many of the gene therapy approaches are trying to circumvent.

Q: What types of gene therapy are there and how are they delivered to patients?

Miller: Gene therapy is the attempt to deliver the correct copy of the functional version of a gene to cells. Many gene therapy treatments use genetically modified viruses to deliver the correct functioning gene. The treatment can be given to a patient as an injection into the bloodstream or directly into the brain or into the spinal column. An advantage of the gene therapy approach is that you only need to give it once, rather than every other week. The virus enters the cells and continues to allow the cells to express the correct protein – possibly for years. A challenge with most enzyme replacement therapies is that the kids must receive multiple injections usually every other week for the extent of their lives.

Q: How are Abeona’s gene therapy programs advancing treatments for these diseases and how different from other therapies in development? 

Miller: Our two lead products, ABX-A and ABX-B, uniquely deliver the therapeutic product to the CNS with the aim of reversing the effects of the genetic errors that cause the disease. ABX-A and ABX-B are delivered intravenously, and cells in the CNS and systemic organs can receive the gene therapy. Recent preclinical safety studies have demonstrated that delivery of ABX-A and ABX-B are well-tolerated with minimal to no side effects. Importantly, efficacy studies in animals with Sanfilippo syndrome have demonstrated unprecedented therapeutic benefit months after treatment. A single dose of ABX-A or ABX-B significantly restored normal cell and organ function, increased neuromuscular control, improved cognitive function, normalized the lifespan of animals with SF for months to years after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting ABX-A and ABX-B treatment could potentially benefit patients with Sanfilippo Syndrome Type A and B, respectively. One thing to note, Abeona is proud to partner with researchers at one of the largest children’s hospital in Columbus, OH who have initiated a natural history study and registry of patients of Sanfilippo Syndrome Type A and B diseases to identify patients and in preparation for Phase I/ II clinical trials planned for initiation in 2015.

Q: With the new acquisition from PlasmaTech, what are the next steps in developing these programs? 

Miller:  The merger between Abeona and Plasmatech (NASDAQ: PTBI) will facilitate the development on a global scale of these promising gene therapies.  We have been in discussion with multiple hospitals around the world to help with our planned rollout. Plasmatech brings together an experienced board, excellent cell therapy products and a focus on brining therapies to rare disease communities. 

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