Lisa Dreyer - Page 33 - Pappas Capital

Biotech All Stars Buy Castoff Experimental Merck Cancer Drug

June 6,2012

Tesaro of Waltham, Mass., a tiny biotech now preparing for its initial public offering, just licensed a promising anti-cancer drug from Merck. The startup is attempting to repeat the success its top executives had turning Big Pharma’s castoffs into hits at MGI Pharma, which was bought by Eisai for $3.9 billion in 2008.

The drug, known as niraparib or MK-4827, is a medicine that targets a mutation that can cause certain hard-to-treat breast and ovarian cancers. AstraZeneca, Abbott Laboratories and Pfizer are also developing similar medicines. The deal was disclosed in a filing with the Securities and Exchange Commission.

At the same time that big drug companies are increasingly buying new medicines developed outside their own walls, they are also selling more of the medicines that they invent but don’t plan on developing to smaller biotechs. This has always happened – Cubist Pharmaceuticals’ antibiotic Cubicin was licensed from Eli Lilly more than a decade ago. But now it is becoming more commonplace, with Pfizer, for instance, talking about the option of selling experimental drugs to outside companies as a central part of its strategy.

TESARO - 06/06/2012

Liquidia Technologies Inc. Studies Show PRINT® Engineered Particles Hold Promise in the Delivery of Respiratory Therapeutics

RESEARCH TRIANGLE PARK, N.C.-May 12, 2012-(BUSINESS WIRE)--Liquidia Technologies today announced the results of two newly-released studies that illustrate the unique benefits of the company’s proprietary PRINT® (Particle Replication In Non-Wetting Templates) platform in the development of advanced respiratory therapeutics. Despite the high prevalence of lung disease, optimizing critical parameters of respiratory therapeutics to improve their pulmonary distribution and effectiveness has remained a challenge for the biopharmaceutical industry. These studies demonstrate how the PRINT platform can be used for respiratory drugs by precisely engineering particles with controlled shape, size, and chemistry, all characteristics that could lead to better lung delivery and therapeutic performance. Aspects of these findings were published in the Journal of Drug Delivery and presented at the Respiratory Drug Delivery Conference being held May 13-17, 2012 in Phoenix, Arizona.

Liquidia Technologies - 05/15/2012

Achillion gets FDA Incentives for Hepatitis C Drug

NEW HAVEN, Conn. - May 15, 2012 - Achillion Pharmaceuticals Inc. said Tuesday it will receive incentives from regulators as it studies a new experimental treatment for the hepatitis C virus.

The Food and Drug Administration awarded fast track status to Achillion's drug candidate ACH-3102. The agency's decision will allow Achillion to file its application for marketing approval piece by piece instead of waiting to collect all the clinical trial data for the drug, and Achillion will be able to consult with the FDA more often as it develops the drug. The FDA may also choose to review the full application in six months instead of the usual 10 months.

Achillion said it started clinical trials of ACH-3102 on Wednesday. Those trials are intended to test the drug's safety and its side effects rather than its effectiveness. The company plans to study a combination of ACH-3102 with its most advanced drug candidate, ACH-1625. Achillion hopes that combined, the drugs could be part of a hepatitis C a regimen that could treat the disease without interferon. Interferon is a common part of hepatitis C treatment but can cause serious side effects, including flu-like symptoms that can last for months.

Shares of Achillion gained 1 cent to $6.90 in midday trading. Earlier the stock rose as much as 7.4 percent.

Achillion Pharmaceuticals - 05/15/2012

Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease

NOVATO, Calif., May 1, 2012 /PRNewswire/ Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced results from a first-in-human, multi-center, Phase 1 study of UX001 in patients with hereditary inclusion body myopathy (HIBM) showing that UX001 was well-tolerated with an expected extended release profile on absorption after oral administration. 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. Based on the Phase 1 results, Ultragenyx plans to initiate an international, multi-center, randomized, double-blind, placebo-controlled, parallel group Phase 2 study of UX001 in HIBM patients in the second quarter of this year.

Ultragenyx Pharmaceutical - 05/01/2012

CardioDx Honored as 2012 Gold Edison Award Winner

Corus CAD Wins at the Edison Awards Gala; Awards Celebrate 25 Years of Honoring Innovators and Innovation

PALO ALTO, Calif. – April 30, 2012 – CardioDx, Inc., a pioneer in the field of cardiovascular genomic diagnostics, today announced that Corus® CAD has been honored as a gold winner in the Science & Medical category of the internationally-renowned 2012 Edison Awards. The awards, which were presented last week at a gala in New York, symbolize the innovation personified by Thomas Alva Edison, inspiring America’s drive to remain at the forefront of creativity and ingenuity in the global economy.

CardioDx - 04/30/2012

LipoScience Receives 2012 Diagnostic Marketing Association Creative Communications First Place Award

NMR LipoProfile® test campaign communicates importance of LDL-particles in cardiovascular disease risk and management

RALEIGH, N.C.- April 26, 2012 (BUSINESS WIRE) -- LipoScience, Inc., an in vitro diagnostic company advancing patient care by developing high value proprietary clinical diagnostic tests using nuclear magnetic resonance (NMR) technology, today announced that it has received first place recognition at the 2012 Diagnostic Marketing Association (DxMA) Creative Communications Awards for the integrated print and online category for LipoScience’s NMR LipoProfile® test campaign.

The award was presented at the 2012 DxMA awards reception on April 25 in Chicago. The DxMA recognizes excellence in diagnostics marketing, advertising and promotional programming. LipoScience partnered with Motivation Mechanics, a Philadelphia-based marketing research and communications strategy firm, on the campaign.

LipoScience - 04/26/2012

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