Lisa Dreyer - Page 35 - Pappas Capital

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.

Liquidia Technologies - 04/02/2012

Athersys gets OK for German solid organ transplant clinical trial

March 27, 2012

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.

Athersys - 03/27/2012

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

March 26, 2012

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.

TESARO - 03/26/2012

Stem Cell Therapy Holds Promise for Strokes

March 21, 2012

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.

Athersys - 03/21/2012

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 Pharmaceutical - 03/14/2012

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.

Ultragenyx Pharmaceutical - 03/09/2012

Milestone Pharmaceuticals Appoints Paul Truex to the Board of Directors

MONTREAL, March 5, 2012 (GLOBE NEWSWIRE) Milestone Pharmaceuticals Inc., a cardiovascular drug development company, today announced the appointment of Paul F. Truex to its Board of Directors. Mr. Truex is Chief Executive Officer, Founder and a Director of Anthera Pharmaceuticals Inc. (Nasdaq:ANTH), a biopharmaceutical company focused on developing drugs to treat serious illnesses, including cardiovascular and autoimmune diseases.

Anthera Pharmaceuticals / Milestone Pharmaceuticals - 03/05/2012

BrainCells Inc. Announces the Successful Completion of the Single Ascending Dose Study With BCI-838 and the Initiation of the Multiple Ascending Dose Study

SAN DIEGO, Feb. 29, 2012 /PRNewswire/ -- BrainCells Inc., a leading biotechnology company developing novel compounds for the treatment of central nervous system (CNS) diseases, announced today that the Phase 1 single ascending dose (SAD) study of BCI-838 has been successfully completed and that the company has initiated the Phase 1 multiple ascending dose (MAD) study. The SAD study evaluated BCI-838 for safety, tolerability, pharmacokinetics and food effect in healthy male subjects. Single oral doses of BCI-838 up to 900 mg were administered and the drug was well tolerated. No serious adverse events were reported and all adverse events were mild in intensity, transient, and resolved without sequelae.

BrainCells - 02/29/2012

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

February 28, 2012

Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today 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 Pharmaceutical - 02/28/2012

Plexxikon receives European approval for Zelboraf

February 21, 2012

Plexxikon, a member of Daiichi Sankyo Group, has received European Commission approval for Zelboraf (vemurafenib) for the monotherapy treatment of adult patients with BRAF mutation-positive unresectable or metastatic melanoma.

Zelboraf is designed to selectively inhibit the BRAF mutation that occurs in about half of all cases of melanoma.

Zelboraf and its companion diagnostic, the cobas 4800 BRAF V600 mutation test, have been approved in the US, Switzerland, Israel, Brazil, New Zealand and Canada.

Plexxikon CEO Peter Hirth said the approval of Zelboraf by the European Commission marks a significant advancement for European patients with metastatic melanoma who historically have had very limited treatment options.

"We are very pleased that our strategy to co-develop Zelboraf along with a companion diagnostic helped accelerate the availability of this personalized medicine for these patients," Hirth added.

Plexxikon - 02/21/2012