Liquidia CEO to Participate in Biotech Panel to Explore “How to Create 54 Liquidias”

Liquidia CEO to Participate in Biotech Panel to Explore “How to Create 54 Liquidias”

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Liquidia Technologies today announced the Company’s CEO, Neal Fowler, will participate in a panel discussion titled “How to Create 54 Liquidias” at the upcoming Nanotech Commercialization Conference (NCC) on April 4th, 2012. The concept of the panel is to explore how North Carolina can further create opportunities and provide resources that will lay the foundation for innovators to build successful companies like Liquidia in the region. The NCC is the preeminent nanotechnology conference in the region and is being held this year in Research Triangle Park, North Carolina April 4-5, 2012.

“Through this panel we want to explore the opportunities that exist for our next generation of innovators and how they too can find success in North Carolina”

Over the next decade, nanotechnology has the potential to influence every aspect of our lives, including our energy, food, medicines, water, buildings, and much, much more. According to a published report by BCC Research, the market value of the worldwide nanomedicine industry is estimated to grow at a CAGR of 12.5% to reach $130.9 billion by the fiscal year 2016. The acceptance and application of nanotechnology to everyday products is a foreshadowing of the growth expected across a multitude of fields including medicine, devices, and diagnostics.

“Liquidia’s success continues to be a shining example of what is possible for startup companies that make roots in North Carolina”, said Jim Roberts, Director of Business Development at COIN, “Through this panel we want to explore the opportunities that exist for our next generation of innovators and how they too can find success in North Carolina”.

Chris William, Managing Director of the Private Client Group at Wells Fargo and Executive Producer of the PBS television series, Carolina Business Review, will host the NCC panel that will also include the Chairman of The Hamner Institutes for Health Sciences, Charles E. Hamner, D.V.M., Ph.D., and Executive Director of the Office of Science & Technology for the North Carolina Department of Commerce, John Hardin. Conference details can be found at http://www.nanoevent.org.

ABOUT LIQUIDIA

Liquidia Technologies, founded in 2004, is a privately held biotechnology company located in Research Triangle Park, North Carolina. By leveraging precise fabrication techniques of the semiconductor industry, Liquidia has become the only company in the world with the ability to rapidly design and manufacture precisely engineered particles of virtually any size, shape, or composition using a unique particle engineering and manufacturing platform know as PRINT®. This unique ability to precisely engineer particles enables scientists to explore new product frontiers that, until now, have otherwise been out of reach for the life sciences industry. In addition to the development of its own products, Liquidia licenses its PRINT particle technology and its GMP manufacturing capabilities to support proprietary programs advanced by collaborators. For more information, please go to www.liquidia.com.

Big Business Appeal of Nanotechnology on Display in Durham

 

DURHAM, N.C., April 2, 2012 - Starting Tuesday night with a speaker reception at the headquarters of the Center of Innovation for NanoBiotechnology (COIN), the fourth annual Nanotechnology Commercialization Conference will kick off with United States Congressman GK Butterfield in attendance.

The event, in Durham this year, features local, national and international nanotech commercialization leaders who will discuss topics such as how to grow this sector through university research, thoughtful regulatory guidance, corporate partnerships and increased investment.

The lunch keynote on Wednesday includes four heavy hitters from North Carolina and will be hosted and moderated by Chris William of the PBS show Carolina Business Review.

This fireside chat includes former CEO of the North Carolina Biotech Center, Charles Hamner, and now Chairman of the Hamner Institute. The discussion also includes John Hardin, the Executive Director of the Office of Science and Technology, who works with the NC Department of Commerce on science and technology policy. Finally, the CEO of a global leader of nanobiotechnology, and from our own backyard, Neal Fowler of Liquidia will discuss his experience and vision from the private sector.

The idea of the panel is how to create the infrastructure to create more companies like Liquidia that license technology from our leading research universities, develop the technology, attract investment capital, attract management and retain and attract the best and the brightest talent to the region.

Big business appeal of nanotechnology on display in Durham

DURHAM, N.C. - Starting Tuesday night with a speaker reception at the headquarters of the Center of Innovation for NanoBiotechnology (COIN), the fourth annual Nanotechnology Commercialization Conference will kick off with United States Congressman GK Butterfield in attendance.

The event, in Durham this year, features local, national and international nanotech commercialization leaders who will discuss topics such as how to grow this sector through university research, thoughtful regulatory guidance, corporate partnerships and increased investment.

The lunch keynote on Wednesday includes four heavy hitters from North Carolina and will be hosted and moderated by Chris William of the PBS show Carolina Business Review.

This fireside chat includes former CEO of the North Carolina Biotech Center, Charles Hamner, and now Chairman of the Hamner Institute. The discussion also includes John Hardin, the Executive Director of the Office of Science and Technology, who works with the NC Department of Commerce on science and technology policy. Finally, the CEO of a global leader of nanobiotechnology, and from our own backyard, Neal Fowler of Liquidia will discuss his experience and vision from the private sector.

The idea of the panel is how to create the infrastructure to create more companies like Liquidia that license technology from our leading research universities, develop the technology, attract investment capital, attract management and retain and attract the best and the brightest talent to the region.

The technology behind Liquidia started with Dr. Joe DeSimone and his team of researchers at his labs at UNC and NC State. The technology was spun out of the university labs, raised capital from local and regional VCs such as Wakefield Group, PPD, Pappas Ventures, and NEA and eventually the Gates Foundation. The company attracted experienced management such as Mr. Fowler.

Liquidia now has 54 employees and presumably some of the best talent in the world is working on this cutting edge technology called PRINT, where drug companies and vaccine companies are coating Liquidia’s nanoparticles with their drugs to attack fatal diseases.

So how are we addressing these important issues of a nurturing ecosystem to build nanotechnology companies and great jobs in the region? Several federal government leaders are coming to discuss policy. Several national investors in nanotech companies, including two leaders from Harris and Harris, a publicly traded venture capital, will present at the conference and there will be an investor pitch room - a tough love feedback session for early stage nanotech companies.

A former FDA executive will give a morning keynote speech after meeting with company executives at a VIP Breakfast. Another session addresses how to sell your nano product to larger companies and to bigger markets. Executives and VIPs from around the country have agreed to meet with our regional companies in hopes of making a match to bring some new out of region resources to our market at VIP Partnering sessions during the conference.

And to improve the appeal to the non-nano crowd, there will also be a first of a kind Nano Art Exhibition on Wednesday night, during the networking reception, where images taken from a microsocope have been enlarged and colored to show beautiful images never seen before. These images will light up Bay 7 at the American Tobacco Campus like no other event since the renovation of the old tobacco warehouse.

Athersys gets OK for German solid organ transplant clinical trial

Stem cell developer Athersys (NASDAQ:ATHX) has received German regulatory clearance for a physician-sponsored solid organ transplant clinical trial.

The study will enroll up to 24 patients and examine Athersys’ MultiStem technology in reducing the likelihood of rejection in patients who’ve undergone liver transplants, CEO Gil Van Bokkelen said in a call to discuss the company’s fourth-quarter earnings.

Enrollment will begin in the second half of the year and Athersys will provide “modest and limited” support, Van Bokkelen said.

Van Bokkelen provided updates on Athersys’ many clinical and preclinical programs with its MultiStem technology, an off-the-shelf stem cell treatment derived from the bone marrow of adults or other nonembryonic sources. The technology has shown promise in reducing inflammation, protecting damaged tissue and forming new blood vessels.

Athersys is investigating MultiStem for several applications, including heart attack, inflammatory bowel disease, stroke, traumatic brain injury and blood diseases.

The company’s most advanced clinical program involves a phase 2 trial in collaboration with Pfizer with inflammatory bowel disease patients. Enrollment in the 126-patient trial is ongoing and expected to be completed at the end of the year. Initial results are expected to be reported in early 2013, Van Bokkelen said.

Tesaro promises rapid progress with cancer drugs in $86M IPO

Tesaro has put together an SEC filing that makes one essential promise to prospective investors interested in buying in on its $86 million IPO: This company is built for speed.

In less than a year, the developer boasts, it in-licensed rolapitant and pushed the cancer treatment into a Phase III study, with an eye to delivering top line data in the second half of next year. An IND for TSR-011 as a new therapy for non-small cell lung cancer, in-licensed from Amgen ($AMGN) in the spring of last year, is being prepped for filing in the second half. Executives--experienced veterans of MGI Pharma, which fetched close to $4 billion--are forging an accelerated pathway aimed at reducing the time it takes to reach the market. Meanwhile, more in-licensing is planned as they build out a pipeline of cancer drugs for a global marketplace. And they plan to use their connections in the cancer drug world along with substantial venture backing to make that happen.

To get to this point, Tesaro has burned slightly more than $25 million from investors like NEA and Kleiner Perkins. But CEO Lonnie Moulder and his co-founders--chief scientist Mary Lynne Hedley and financial chief Rick Rogers--grabbed the industry's attention with its ability to raise $121 million in short order. Their quick success and emphasis on speed earned a Fierce 15 award in 2011.

Tesaro, though, has a set of negatives shared by everyone at this stage: No product revenue, high risk R&D work, and only a two-year track record. That's been more than enough to chill the average investor on biotech IPOs over the past three years. Now the veteran team can see if experience and a bold timetable can warm up their prospects on Wall Street.

It should be interesting to see if their formula for success can bridge troubled market waters to a successful IPO.

Stem Cell Therapy Holds Promise for Strokes

Every year, about 700,000 Americans suffer strokes, which occur when a blood vessel in the brain is blocked or bursts.

Stroke is the third leading cause of death in the United States.

Now stroke specialists say stem cells from adult bone marrow may improve patients’ recovery from the condition.

Georgia Health Sciences University is one of several study sites for a stem cell therapy developed by Cleveland-based Athersys. The therapy may reduce the extent of a stroke and enhance recovery even if it is administered more than a day after symptoms begin.

As things stand now, “we have very little to offer patients with moderate to severe strokes after the four-and-one-half-hour treatment window for the clot buster tPA has passed,” says Dr. David C. Hess, a stroke specialist who chairs the Medical College of Georgia Department of Neurology at GHSU.

GHSU researchers have been studying how the stem cells, in animal models, affect ischemic stroke, the most common form of the condition.

“We hope stem cell therapy will help patients get better and get better faster,” Hess says.

Hess speaks to Georgia Health News about the stem cell research and its potential in this video, courtesy of GHSU.

Marina, ProNAi in cancer deal

ProNAi Therapeutics Inc. (Kalamazoo, Mich.) partnered with Marina Biotech Inc. (OTCQX:MRNA) to use Marina's Smarticles liposomal delivery technology to develop single-strand oligonucleotides to treat cancer through DNA interference (DNAi). Marina is eligible to receive up to $14 million per gene target in total upfront and milestone payments, plus royalties. ProNAi has the option to select any number of additional targets. Marina acquired the Smarticles technology from novosom AG (Halle, Germany) in 2010.

ProNAi's lead DNAi product -- PNT2258 -- uses the Smarticles technology under a 2007 licensing deal with novosom. PNT2258, which targets the B cell lymphoma 2 (BCL-2; BCL2) gene, is in Phase I testing for solid tumors.

Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 MPS 7

March 14, 2012

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS). UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University.

Ultragenyx Granted Orphan Drug Designation for UX003 for the Treatment of Mucopolysaccharidosis Type 7 MPS 7

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the degradation of the glycosaminoglycans dermatan sulfate (DS) and heparan sulfate (HS). UX003 is a recombinant human Beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7. MPS 7 was originally described in 1973 by William Sly, MD, St. Louis University School of Medicine, and is also known as Sly Syndrome. Ultragenyx in-licensed the MPS 7 program from St. Louis University. su

"Obtaining orphan drug designation for UX003 is a significant achievement for Ultragenyx that adds value to our development pipeline and attests to the importance of this product candidate in filling an unmet medical need," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We look forward to continuing to collaborate with Dr. Sly and colleagues at St. Louis University to advance this urgently needed therapy into clinical testing."

The Orphan Drug Designation program provides orphan status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the US are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received (see also Rare Diseases).

Ultragenyx Announces the Completion of the Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed.  UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency.  UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.