NEW HAVEN, Conn., Jan. 4, 2012 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a leader in the discovery and development of small molecule drugs to combat the most challenging infectious diseases, announced today the receipt of a Fast Track designation from the U.S. Food and Drug Administration (FDA) for ACH-1625 for the treatment of chronic hepatitis C virus (HCV). ACH-1625 is a once-daily protease inhibitor with broad genotypic coverage against HCV that was discovered by Achillion and is currently being evaluated in a Phase 2 clinical trial.
ACH-1625 Receives Fast Track Designation From the FDA for the Treatment of Chronic Hepatitis C
Achillion hepatitis drug gets faster FDA review
NEW HAVEN, Conn. - A chronic hepatitis c
treatment being developed by from Achillion Pharmaceuticals received
fast track designation, allowing for a quicker review by regulators.
Achillion
said Wednesday the treatment, labeled ACH-1625, is in mid-stage
clinical testing. The designation allows drug developers to submit their
applications to the Food and Drug Administration piece by piece instead
of having to file all the paperwork at once.
It also allows for more frequent interaction with regulators and a possible priority review.
Achillion
Pharmaceuticals Inc., based in New Haven, Conn., said the drug received
the designation due in part to its once-daily dosing and its potential
to offer improved safety and tolerability compared to the current
standard of care.
Hepatitis
C treatments represent a big opportunity for drugmakers. The virus can
lead to life-threatening liver damage and is the main cause of liver
transplants in the United States. Analysts expect the market for those
treatments to expand in the coming years.
Other
companies developing potential drugs include Pharmasset Inc. which is
being acquired by Gilead Sciences Inc. Earlier this year, the FDA
approved two pills that offered the first new breakthrough treatments
for the liver disease in 20 years. The Vertex Pharmaceuticals Inc.
hepatitis C pill Incivek rang up about $420 million in this year's third
quarter. Another pill, Merck & Co.'s Victrelis had $31 million in
sales.
Marina Biotech, Inc. (Formerly Known as MDRNA, Inc.) and Mirna Therapeutics, Inc. Announce License Agreement for Up to $63 Million for the Development
BOTHELL, WA and AUSTIN, TX--(Marketwire - December 23, 2011) - Marina Biotech, Inc. (NASDAQ: MRNA), a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc. (Mirna), a privately-held biotechnology company pioneering microRNA (miRNA) replacement therapy for cancer, announced today that they have entered into a license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna's proprietary microRNAs and Marina Biotech's novel SMARTICLES liposomal delivery technology. Mirna will have full responsibility for the development and commercialization of any products arising under the Agreement and Marina Biotech will support pre-clinical and process development efforts. Under terms of the Agreement, Marina Biotech could receive up to $63 million in total upfront, clinical and commercialization milestone payments, as well as royalties on sales, based on the successful outcome of the collaboration. Further terms of the Agreement were not disclosed.
Marina Biotech, Inc. (Formerly Known as MDRNA, Inc.) and Mirna Therapeutics, Inc. Announce License Agreement for Up to $63 Million for the Development
BOTHELL, WA and AUSTIN, TX--(Marketwire - December 23, 2011) - Marina Biotech, Inc. (NASDAQ: MRNA), a leading oligonucleotide-based drug discovery and development company, and Mirna Therapeutics, Inc. (Mirna), a privately-held biotechnology company pioneering microRNA (miRNA) replacement therapy for cancer, announced today that they have entered into a license agreement regarding the development and commercialization of microRNA-based therapeutics utilizing Mirna's proprietary microRNAs and Marina Biotech's novel SMARTICLES liposomal delivery technology. Mirna will have full responsibility for the development and commercialization of any products arising under the Agreement and Marina Biotech will support pre-clinical and process development efforts. Under terms of the Agreement, Marina Biotech could receive up to $63 million in total upfront, clinical and commercialization milestone payments, as well as royalties on sales, based on the successful outcome of the collaboration. Further terms of the Agreement were not disclosed.
"Given the challenge of effectively delivering oligonucleotides to target tissues, we devoted considerable effort to identifying an optimal delivery technology that would allow for systemic administration of our potent miRNA tumor suppressors and which is already in clinical testing," said Paul Lammers, M.D., M.Sc., President and CEO of Mirna Therapeutics. "With the dramatic in vivo results achieved with our miRNA mimics, we believe the SMARTICLES technology solves the delivery challenge for us, and we are now looking forward to bringing our miRNA mimics into the clinic in the next 18 months as promising targeted cancer therapeutics."
"We are extremely pleased to have entered into this relationship with a company as well respected in the area of microRNA-based therapeutics as Mirna Therapeutics," stated J. Michael French, President and CEO of Marina Biotech. "We are excited to see the continued advancement of oligonucleotide-based therapeutics and to be able to provide a technology capable of effectively delivering, in this case, systemically administered miRNA mimetics. We look forward to the rapid advancement of Mirna Therapeutics' clinical pipeline and the opportunity to bring novel therapeutics to patients in need."
In a recent poster entitled "The Development of a miRNA-based Therapeutic Candidate for Hepatocellular Carcinoma," presented at the November, 2011 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in San Francisco, CA, Mirna scientists showed that mimics of five tumor suppressor miRNAs, including miR-34 and let-7, all significantly inhibited the growth of liver tumors compared to animals treated with formulated negative control miRNAs. The five miRNA mimics were complexed with Marina Biotech's SMARTICLES delivery formulation and injected into NOD/SCID mice with orthotopically grown Hep3B human liver cancer xenografts.
The companies will present their respective science and technologies at the upcoming Biotech Showcase™ 2012, January 9-11, 2012 at the Parc 55 Wyndham San Francisco - Union Square at 55 Cyril Magnin Street, San Francisco, CA. Mirna Therapeutics will present on Monday, 9 January at 3:00 pm and Marina Biotech will present on Tuesday, 10 January at 2:30 pm.
About Marina Biotech, Inc.
Marina Biotech is a biotechnology company focused on the development and commercialization of oligonucleotide-based therapeutics utilizing multiple mechanisms of action including RNA interference (RNAi) and messenger RNA translational blocking. The Marina Biotech pipeline currently includes a clinical program in Familial Adenomatous Polyposis (a precancerous syndrome) and two preclinical programs -- in bladder cancer and malignant ascites. Marina Biotech entered into an exclusive agreement with The Debiopharm Group for the development and commercialization of the bladder cancer program. Marina Biotech's goal is to improve human health through the development of RNAi- and oligonucleotide-based compounds and drug delivery technologies that together provide superior therapeutic options for patients. Additional information about Marina Biotech is available at http://www.marinabio.com.
About Mirna Therapeutics, Inc.
Mirna Therapeutics is a biotechnology company focused on the development and commercialization of microRNA (miRNA) therapeutics. The Company has a substantial intellectual property portfolio on the therapeutic use of miRNAs developed by its own scientists as well as in-licensed from other institutions. Mirna's IP portfolio contains > 300 miRNAs with applications in oncology and other diseases. Oncology-directed miRNAs include those that are key tumor suppressors in cancer, such as miR-34 and let-7 that have proven to block tumor growth in a number of different pre-clinical animal studies. The Company, founded in 2007, is located in Austin, Texas, and has received significant funding from the State of Texas, both through the State's Emerging Technology Fund and from the Cancer Prevention and Research Institute of Texas (CPRIT). Mirna Therapeutics is the recipient of a $10.3 million commercialization award from CPRIT. For more information, visit www.mirnarx.com
Forward-Looking Statements
Statements made in this news release may be forward-looking statements within the meaning of Federal Securities laws that are subject to certain risks and uncertainties and involve factors that may cause actual results to differ materially from those projected or suggested. Factors that could cause actual results to differ materially from those in forward-looking statements include, but are not limited to: (i) the ability of Marina Biotech to obtain additional funding; (ii) the ability of Marina Biotech to attract and/or maintain manufacturing, research, development and commercialization partners; (iii) the ability of Marina Biotech and/or a partner to successfully complete product research and development, including preclinical and clinical studies and commercialization; (iv) the ability of Marina Biotech and/or a partner to obtain required governmental approvals; and (v) the ability of Marina Biotech and/or a partner to develop and commercialize products prior to, and that can compete favorably with those of, competitors. Additional factors that could cause actual results to differ materially from those projected or suggested in any forward-looking statements are contained in Marina Biotech's most recent periodic reports on Form 10-K and Form 10-Q that are filed with the Securities and Exchange Commission. Marina Biotech assumes no obligation to update and supplement forward-looking statements because of subsequent events.
A look back, and forward
This past year has held some pleasant surprises for me. I was particularly intrigued by the Plexxikon buyout for $935 million, the bulk of it upfront. In a sane world, Plexxikon--which developed the remarkable new skin cancer drug Zelboraf (vemurafenib)--would have gone public. But as far as the IPO market for biotech companies is concerned, this isn't a sane world.
Plexxikon helped illustrate the potential biotechs have to distinguish themselves with great science and a sharp development focus. That kind of expertise is clearly worth a considerable sum; a maxim other companies like Pharmasset have clearly learned how to profit by. Plexxikon's experience also demonstrated the limitations developers face in a world where investors will typically turn a cold shoulder to all the risks involved in drug development.
I'm wrapping this year's run of FierceBiotech reports with a look back, and forward. The key trends of 2011--with a wide array of venture capitalists complaining loudly, the R&D revolution still at the midway point, deal-making more important than ever, and regulators feeling the heat from a disgruntled industry--will all play into 2012.
Fundamental changes can't occur quickly in biotech. It took years to get where we are today. Years more are required to see if new strategies can work or new biotechs can navigate the hazards of clinical development. Now that we've passed the 10th anniversary of FierceBiotech, we can say we've reached the second generation of the publication. And we're planning to take our coverage of the industry up a notch or two in 2012. Call it FierceBiotech 2.0.
We'll be taking our traditional holiday break next week, returning the Tuesday after New Year's Day. It's a chance for us to retool some aspects of our coverage and come back stronger than ever. We hope you enjoy your own break and will see you in the new year.
- John Carroll
Roche’s Zelboraf Melanoma Drug Wins Backing of EU Regulator
Dec. 16 (Bloomberg) -- Roche Holding AG’s Zelboraf treatment for a specific type of melanoma was recommended for approval by the European Union’s drug regulator.
Zelboraf may be used on metastatic or unresectable melanoma, a form of the skin cancer with lower rates of survival than localized melanoma, the European Medicines Agency said in a statement today.
The drug, a so-called BRAF inhibitor that was cleared for sale in the U.S. in August, works by blocking a protein that fuels tumor growth in about half of patients with advanced forms of the skin cancer. Zelboraf would help treat a disease that kills 8,300 men and 7,600 women a year in Europe, according to the EMA.
“There is a high unmet medical need for alternative treatments for metastatic melanoma that improve survival of patients,” the London-based agency said. About 25 percent of patients diagnosed with metastic or unresectable melanoma survive a year after diagnosis, compared with 90 percent of patients with localized melanoma, it said.
The recommendation to approve Zelboraf “represents an important milestone for people with metastatic melanoma who until recently had limited treatment options,” Hal Barron, chief medical officer at Basel, Switzerland-based Roche, said in a statement. “We are working closely with health authorities worldwide to bring Zelboraf to people with this deadly disease as soon as possible.”
BRAF-Inhibitor Work
Zelboraf was developed by Roche and Plexxikon Inc., which was acquired this year by Tokyo-based Daiichi Sankyo Co. Other companies working on BRAF inhibitors include Amgen Inc., GlaxoSmithKline Plc, Novartis AG and Pfizer Inc., according to the Bloomberg drug database.
The agency’s recommendations are the final stage before the European Commission, the EU’s executive arm, approves or rejects a drug for sale to patients in the 27-nation region.
Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx
Emil Kakkis harvesting academic sources for Orphan drug newco Ultragenyx
By Jennifer Rhodes
Staff Writer
Published on Monday, December 19, 2011
Ultragenyx Pharmaceutical Inc. is taking a traditional approach to Orphan Drugs by in-licensing development-ready substrate and enzyme replacement therapies that have a clear mechanistic rationale and proof of concept in animals for rare and ultra-rare genetic diseases.
Founder, President and CEO Emil Kakkis believes there are a few hundred such diseases with no current treatments.
Nanotechnology firm Liquidia partners with PATH on new pneumonia vaccine
December 13, 2011
North Carolina nanotechnology company Liquidia Technologies is partnering with nonprofit organization PATH to develop a new pneumonia vaccine that could be deployed globally.
Research Triangle Park-based Liquidia is working with PATH to conduct preclinical studies on a “next generation” pneumococcal vaccine that could be more effective than existing vaccines and more efficient to produce. Financial terms of the partnership were not disclosed. But it’s not the first time the two entities have worked together. Liquidia has also partnered with PATH’s Malaria Vaccine Initiative to develop a new malaria vaccine.
Nanotechnology firm Liquidia partners with PATH on new pneumonia vaccine
Nanotechnology firm Liquidia partners with PATH on new pneumonia vaccine
North Carolina nanotechnology company Liquidia Technologies is partnering with nonprofit organization PATH to develop a new pneumonia vaccine that could be deployed globally.
Research Triangle Park-based Liquidia is working with PATH to conduct preclinical studies on a “next generation” pneumococcal vaccine that could be more effective than existing vaccines and more efficient to produce. Financial terms of the partnership were not disclosed. But it’s not the first time the two entities have worked together. Liquidia has also partnered with PATH’s Malaria Vaccine Initiative to develop a new malaria vaccine.
Pneumonia vaccines in the United States have all but eliminated childhood pneumococcal disease. But vaccines are too expensive in the developing world. Even when they are available, they do not cover all variations of the bacterium.
Liquidia’s proprietary “PRINT” technology can control the size and shape of a nanoparticle, which optimizes the delivery of a therapeutic to the cells of the human body. Liquidia says it can use its technology to bring together multiple vaccine components in a nanoparticle, which can optimize a vaccine’s safety and efficacy. Because the Liquidia technology is scalable, it makes vaccine production more cost efficient.
Pneumonia represents a large global market. The World Health Organization estimates pneumonia kills 1.4 million children younger than age 5 each year. More than half of these deaths are caused by Streptococcus pneumoniae, a bacterium with many variations that are becoming increasingly resistant to antibiotics.
“By improving vaccine performance and reducing manufacturing costs using our PRINT technology, we believe this collaboration has the potential to significantly impact the spread of pneumonia in the developing world,” Liquidia CEO Neal Fowler said in a statement.
Privately-held Liquidia has raised more than $60 million in equity financing. Its investors includes venture capital firms Canaan Partners, New Enterprise Associates, Morningside Venture Investments Limited, Pappas Ventures and Firelake Capital as well as Wilmington, North Carolina-based clinical research organization PPD. Liquidia earlier this year received a $10 million equity investment from the Bill & Melinda Gates Foundation to support vaccine development and commercialization.
Plexxikon Inc. Advances Novel Targeted Treatment PLX3397 in Blood Cancer
Plexxikon Advances Novel Targeted Treatment PLX3397 in Blood Cancer
Berkeley, CA, December 12, 2011
Plexxikon Inc., a member of Daiichi Sankyo Group, today announced scientific findings from preclinical studies showing that treatment with a novel oral agent, PLX3397, selectively inhibited key cancer-driving Flt3 mutations that occur in 20-30 percent of acute myeloid leukemia (AML) patients. In a preclinical model of AML, PLX3397 showed significant tumor regression. This preclinical work also showed that PLX3397 retained activity against certain drug-resistant forms of mutated Flt3 that can occur with other treatments. These scientific findings were presented during the American Society of Hematology (ASH) Conference, taking place December 10-13, 2011 in San Diego (Abstracts #764 and #3632). Plexxikon recently initiated a Phase 1/2 study in AML patients, further described at www.clinicaltrials.gov.
“Given PLX3397’s selectivity for relevant mutations, we are exploring this potential personalized medicine for AML patients with Flt3 mutations,” said K. Peter Hirth, Ph.D., chief executive officer of Plexxikon. “Plexxikon’s hallmark capability to design selective kinase inhibitors enables clinical researchers to test the hypotheses of such targeted therapies directly and rapidly.”
Phase 1 dose escalation testing in patients with solid tumors has been completed, and showed that PLX3397 reached therapeutic drug levels that were well tolerated at the doses tested. Two Phase 2 studies are now under way to further evaluate PLX3397, including one study in Hodgkin lymphoma and another study recently initiated in patients with Flt3-mutated AML. Plasma from Phase 1 patients contained sufficient levels of PLX3397 to block signaling in Flt3-mutant AML cells ex vivo. Additionally, PLX3397 has been tested in primary AML patient blood samples, which showed a clear dose response to drug at clinically achievable drug levels.
About PLX3397
PLX3397 is an oral agent that is being evaluated in several clinical trials for the treatment of different cancers, including AML. PLX3397 selectively co-inhibits three key targets, allowing down-modulation of a number of cell types, including macrophages, microglia, osteoclasts and mast cells, as well as the inhibition of Flt3 mutations that are key oncogenic drivers in AML.
About Plexxikon
Plexxikon, a member of Daiichi Sankyo Group, is a leader in the structure-guided discovery and development of novel small molecule pharmaceuticals to treat human disease. The company’s lead drug Zelboraf™ (vemurafenib/PLX4032) was approved by the FDA in August 2011, and is being co-promoted in the U.S. by Daiichi Sankyo Inc. and Genentech. The company is developing a portfolio of clinical and preclinical stage compounds to address significant unmet medical needs in oncology, as well as several other therapeutic indications. Plexxikon’s Scaffold-Based Drug Discovery™ platform integrates multiple state-of-the-art technologies, including structural screening as a key component that provides a significant competitive advantage over other drug discovery approaches.
