Companies Archives - Page 39 of 59

Athersys, Inc. Announces Positive Results of MultiStem(R) Clinical Trial for Hematopoietic Stem Cell Transplant Support and Prevention of Graft-Versus-Host Disease

CLEVELAND, Feb. 1, 2012 (GLOBE NEWSWIRE) -- Athersys, Inc. (Nasdaq:ATHX - News) today announced positive results from its Phase I clinical trial of MultiStem(R), its cell therapy product, administered to individuals undergoing allogeneic hematopoietic stem cell transplants (HSCT) for the treatment of leukemia and related conditions. According to the Center for International Blood and Marrow Transplant Research, there are approximately 25,000 allogeneic HSCT performed annually, globally. The study demonstrated that MultiStem therapy was well tolerated in both the single infusion and repeat infusion arms and also suggested that the therapy may provide benefit to recipients of allogeneic HSCT, such as reducing the incidence and severity of Graft-versus-Host Disease (GvHD) as compared to historical clinical experience. The results are consistent with previous preclinical studies that show that MultiStem provides multiple benefits in HSCT and other transplant models, such as reducing inflammatory damage and promoting graft acceptance.

These clinical results could provide the foundation for further, accelerated development of MultiStem to prevent or reduce the severity of GvHD, a potentially life-threatening complication of such transplants. Athersys received orphan drug designation from the U. S. Food and Drug Administration for prevention of GvHD in September 2010. Orphan drug designation, which is intended to facilitate drug development, provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.

Data highlights from the study include:

The majority of patients participating in the study received transplants from unrelated donors (19 of 36), and nearly all of the patients received peripheral blood stem cell (PBSC) transplants (34 of 36), both of which are associated with a higher risk of GvHD;

All patients experienced successful neutrophil engraftment (median time of engraftment 15 days), and 86% of patients experienced successful platelet engraftment (median time of engraftment 16 days) which compares favorably to historical clinical experience for this patient population demonstrating a positive impact on blood and immune system recovery;

Substantial reduction in acute GvHD incidence, relative to historical experience, at the highest single dose (11% grade II-IV GvHD, and 0% grade III-IV GvHD);

Evidence of a dose response relationship, with patients receiving the highest single dose of MultiStem having a 33% lower absolute incidence of acute GvHD relative to patients who received a single low or medium dose, and patients receiving once weekly dosing of the medium dose through the first 30 days having reduced GvHD incidence relative to single or weekly dosing over the first two weeks post-transplant;

Favorable relapse-free survival (RFS) rate at 100 days among all patients receiving MultiStem treatment relative to the historical clinical experience; and

Limited infection-related complications over the first 100 days relative to historical clinical experience, consistent with the positive effect on engraftment rates.

Dr. Richard Maziarz, M.D., co-principal investigator of this study and Medical Director, Adult Stem Cell Transplantation Program at the Oregon Health & Science University Knight Cancer Institute, commented, "The results from this study are encouraging, and suggest that MultiStem therapy could provide clinical benefit to patients receiving allogeneic stem cell transplants. These patients are susceptible to GvHD and other complications from standard treatment regimens, and the data suggest that treatment with MultiStem could provide clinical benefits in several important ways. The data certainly justify additional clinical investigation, and I look forward to subsequent clinical studies to explore how MultiStem can help these individuals." The study results are being presented at the annual American Society for Bone Marrow Transplantation Tandem meeting in San Diego, California.

"We believe the results of this study are meaningful and suggest that administration of MultiStem could provide substantial benefits to patients receiving hematopoietic transplants, especially those patients that are traditionally considered to be in higher risk categories, such as those receiving transplants from unrelated donors or receiving peripheral blood stem cell transplants, both of which have historically been associated with a higher risk of GvHD," said Gil Van Bokkelen, CEO of Athersys, Inc. "We believe that these results could provide the basis for advancing this program clinically in an accelerated manner, and we look forward to discussing the data with the FDA."

Continued Development

These clinical results provide the foundation for further, accelerated development of MultiStem for the prevention and reduction of GvHD. Following final review of the data, and subject to input from its key scientific and clinical advisers, Athersys plans to meet with the FDA to discuss plans for the next phase of clinical development, which could include a blinded, controlled phase II/III study of MultiStem for GvHD prophylaxis and HSCT support. The study would be intended to help lay the groundwork for approval in this indication, but would also help continue to establish the scientific foundation for MultiStem in related areas including GvHD treatment, solid organ transplant and other areas of immune system dysfunction.

Safety Results

During the first 48 hours following MultiStem administration, patients were assessed for infusion-related toxicity and other acute adverse events. The primary endpoint for the study was the determination of the maximum tolerated dose, as determined by a continual reassessment methodology. Regimen-related toxicities and infusion-related allergic toxicities through 30 days after MultiStem administration were also monitored. Additionally, patients were evaluated for adverse events and infections through 100 days following the HSCT.

The administration of MultiStem was found to be well tolerated for all dosing groups in both the single and repeat dose administration arms. Immediately following dosing, there were no clinically significant changes to vital signs or evidence of allergic reaction associated with MultiStem administration. Over the 30-day observation period, no infusional toxicities or clinically significant adverse events definitively related to MultiStem occurred.

MultiStem had a favorable safety profile over the 100-day period following the HSCT. There were no graft failures, which compares favorably with historical graft failure rates of 5--15% for these types of patients. Of the 36 patients in the study, 30 patients completed 100 day follow-up and there were 6 withdrawals, including 2 relapses and 3 deaths (unrelated to treatment). Other serious adverse events in the first 100 days were consistent with expectations for this patient population.

HSCT Support Highlights

While the primary objective of this Phase I clinical trial was to evaluate the safety of MultiStem administered to HSCT recipients, additional data regarding secondary endpoints, GvHD incidence, infection and survival, have been collected and are being evaluated for safety and evidence of efficacy signals to facilitate planning for subsequent clinical studies. Specifically, following MultiStem administration, patients were assessed weekly by the attending physician for GvHD (and regimen-related toxicities), and information regarding infections and adverse events was collected as they occurred, through day 100.

Overall, MultiStem treatment was associated with a positive impact on blood and immune system recovery, as compared to expectations for this patient population based on historical experience and scientific literature, with 100% neutrophil and 86% platelet engraftment for study patients. The median time to engraftment was 15 days for neutrophils and 16 days for platelets.

The cumulative incidence of acute GvHD for all subjects enrolled in the study (i.e. irrespective of administered dose) was generally in line with expectations for this patient population based on historical experience and scientific literature, using Kaplan-Meier estimates censored for study withdrawal due to relapse and death. However, in the highest single dose group (10 million cells per kilogram body weight, infused on day 2 following HSCT), the 100-day cumulative incidence of moderate to severe acute GvHD was just 11%, which compares favorably with historical experience for this patient population (generally 40-60% grade II-IV GvHD), and, among the patients in this group, no patient developed severe GvHD. Additionally, at the 5 million cells per kilogram body weight dose level, once weekly dosing of the intermediate dose through the first 30 days provided apparent additional benefit over single or weekly dosing over the first two weeks post-transplant.

Importantly, the incidence of infections and mortality over the observation period appear to be in line with or better than what would be expected for this high risk patient population. Consistent with the positive impact on hematopoietic recovery, MultiStem was associated with a relatively low level of late stage infection and only one case of infection-related mortality occurred through 100 days, which compares favorably with historical experience with this patient population. Overall, the Kaplan-Meier estimate of relapse-free survival at 100 days was 81%, compared to an expectation of around 65-70% based on published results from previously published clinical studies using comparable treatment approaches and patient groups.

About the Disease Condition and Study Design

Leukemia and certain related conditions are often treated with radiation and chemotherapy to eliminate cancerous or diseased cells, but this process also severely compromises the native blood forming and immune system in the patient, leaving them susceptible to infection and other complications. To address this, a patient will often receive an allogeneic HSCT, whereby following radiation and chemotherapy treatment a patient's blood stem cells are replaced with a transplant of hematopoietic stem cells obtained from the bone marrow or peripheral blood of a healthy donor. Donors may be related or unrelated to the patient, but are matched according to tissue type in order to minimize the potential for GvHD, where donor immune cells transplanted with the donor HSCT attack tissue and organs of the patient. Following the transplant, the patient will often remain hospitalized in specialized units until successful engraftment provides a sufficiently functional immune system.

According to the Center for International Blood and Marrow Transplant Research, there are approximately 25,000 allogeneic HSCT performed annually globally, although this number is projected to increase due to the anticipated growth in incidence of hematologic malignancies associated with an aging population. While this treatment approach can be an effective medical therapy for these types of cancer, it is often associated with substantial tissue damage and side effects, such as GvHD. GvHD is a frequent complication associated with allogeneic HSCT, affecting approximately half or more of transplant recipients, and advanced GvHD can be severely debilitating or even fatal. Several factors affect a patient's likelihood of having GvHD and GvHD severity, including the treatment protocol used, the degree of tissue match between donor and recipient (with lower GvHD rates and severity associated with related donors and better tissue matches), and the condition of the patient among other factors. In addition, higher GvHD rates are typically observed in patients receiving peripheral blood stem cell (PBSC) transplants, as compared to patients receiving bone marrow-derived stem cell transplants.

The Phase I clinical trial was an open label, multi-center trial evaluating the safety and maximum tolerated dose of single or repeated dose administration of MultiStem following an allogeneic HSCT in patients being treated for leukemia or related cancers of the blood or immune system. The single dose arm evaluated the infusion of a single dose of MultiStem administered intravenously two days following a peripheral blood or bone marrow HSCT, and included patients in three dose groups, based on cells per kilogram body weight -- 1 million (n=6), 5 million (n=3) and 10 million (n=9). The repeat dose arm evaluated patients enrolled in two groups -- 1 million (n=3) and 5 million (n=3) per kilogram body weight -- with MultiStem administration scheduled for days 2, 9 and 16 and in a third group (n=12) with MultiStem administration of 5 million per kilogram scheduled for days 2, 9, 16, 23 and 30. The clinical trial was conducted at transplant centers in the United States and Europe, including the Oregon Health & Science University, University Hospitals Case Medical Center, Texas Transplant Institute, University of Pennsylvania, Mayo Clinic Hospital Arizona, and UZ Leuven.

Twenty male and sixteen female subjects enrolled in the study, ranging in age from 20-62 years old. Nineteen patients received HSCT from matched unrelated donors (MUD), including two with a slight degree of mismatch, and seventeen received HSCT from matched related donors (MRD). With respect to HSCT source, 34 grafts were from peripheral blood and two were from bone marrow, with 16 of 18 patients receiving PBSC transplants in the single dose arm of the study, and all 18 patients in the multiple dose arm of the study receiving PBSC transplants. All patients received MultiStem therapy from the same product bank, reflecting MultiStem's manufacturing scalability and shelf-life stability, an advantage over other cell therapies and an important potential commercial advantage for the company.

About MultiStem

MultiStem(R) cell therapy is a patented product that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of multiple therapeutic factors produced in response to signals of inflammation and tissue damage. MultiStem has demonstrated therapeutic potential for the treatment of inflammatory and immune disorders, neurological conditions, and cardiovascular disease, as well as other areas, and represents a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. The product is extensively characterized for safety, consistency and potency. Athersys has forged strategic partnerships with Pfizer Inc. to develop MultiStem for inflammatory bowel disease and with RTI Biologics, Inc. to develop cell therapy for use with a bone allograft product in the orthopedic market.

About Athersys

Athersys is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem(R) cell therapy product, a patented, adult-derived "off-the-shelf" stem cell product platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The Company currently has several clinical stage programs involving MultiStem, including for treating inflammatory bowel disease, ischemic stroke, damage caused by myocardial infarction, and for the prevention of graft versus host disease. Athersys has also developed a diverse portfolio that includes other technologies and product development opportunities, and has forged strategic partnerships and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products. More information is available at www.athersys.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. These forward-looking statements relate to, among other things, the expected timetable for development of our product candidates, our growth strategy, and our future financial performance, including our operations, economic performance, financial condition, prospects, and other future events. We have attempted to identify forward-looking statements by using such words as "anticipates," "believes," "can," "continue," "could," "estimates," "expects," "intends," "may," "plans," "potential," "should," "suggest," "will," or other similar expressions. These forward-looking statements are only predictions and are largely based on our current expectations. A number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face that could cause actual results to differ materially from those implied by forward-looking statements are the risks and uncertainties inherent in the process of discovering, developing, and commercializing products that are safe and effective for use as human therapeutics, such as the uncertainty regarding market acceptance of our product candidates and our ability to generate revenues, including MultiStem for the treatment of inflammatory bowel disease, acute myocardial infarction, stroke and other disease indications, and the prevention of graft-versus-host disease. These risks may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements. Other important factors to consider in evaluating our forward-looking statements include: our ability to raise additional capital; final results from our MultiStem clinical trials; the possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete clinical trials; changes in external market factors; changes in our industry's overall performance; changes in our business strategy; our ability to protect our intellectual property portfolio; our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; our ability to meet milestones under our collaboration agreements; our collaborators' ability to continue to fulfill their obligations under the terms of our collaboration agreements; our possible inability to execute our strategy due to changes in our industry or the economy generally; changes in productivity and reliability of suppliers; and the success of our competitors and the emergence of new competitors. You should not place undue reliance on forward-looking statements contained in this press release, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.

Athersys - 02/02/2012

Athersys, Inc. Announces Positive Results of MultiStem(R) Clinical Trial for Hematopoietic Stem Cell Transplant Support and Prevention of Graft-Versus-Host Disease

CLEVELAND, Feb. 1, 2012 (GLOBE NEWSWIRE) -- Athersys, Inc. (Nasdaq:ATHX - News) today announced positive results from its Phase I clinical trial of MultiStem(R), its cell therapy product, administered to individuals undergoing allogeneic hematopoietic stem cell transplants (HSCT) for the treatment of leukemia and related conditions. According to the Center for International Blood and Marrow Transplant Research, there are approximately 25,000 allogeneic HSCT performed annually, globally. The study demonstrated that MultiStem therapy was well tolerated in both the single infusion and repeat infusion arms and also suggested that the therapy may provide benefit to recipients of allogeneic HSCT, such as reducing the incidence and severity of Graft-versus-Host Disease (GvHD) as compared to historical clinical experience. The results are consistent with previous preclinical studies that show that MultiStem provides multiple benefits in HSCT and other transplant models, such as reducing inflammatory damage and promoting graft acceptance.

Athersys - 02/01/2012

Chimerix Announces Presentation of Final Data from CMX001 Phase 2 Trial in Prophylaxis of Cytomegalovirus in Hematopoietic Stem Cell Transplant Recipients

Abstract Receives "BEST ABSTRACTS AWARD FOR CLINICAL RESEARCH" at 2012 BMT Tandem Meetings on February 3rd, 2012

Chimerix, Inc., a biotechnology company developing novel antiviral therapeutics, today announced that final data from CMX001 Study 201, a Phase 2 study evaluating CMX001 for the prevention of cytomegalovirus (CMV), has been accepted for oral presentation at the BMT Tandem Meetings on February 1-5, 2012, in San Diego, California. CMX001 is a broad spectrum, Lipid-Antiviral-Conjugate completing Phase 2 clinical development for the prevention of CMV in hematopoietic cell transplant (HCT) recipients. In total, three abstracts related to CMX001 have been accepted for presentation at the 2012 BMT Tandem Meetings, the combined annual meetings of the Center for International Blood and Marrow Transplant Research (CIBMTR) and the American Society of Blood and Marrow Transplantation (ASBMT)....

Chimerix - 01/27/2012

Researchers uncover how new melanoma drug accelerates secondary skin cancers

By Kim Irwin - Patients with metastatic melanoma taking the recently approved drug vemurafenib (marketed as Zelboraf) responded well to the twice-daily pill, but some of them developed a different, secondary skin cancer.
Dr. Antoni RibasNow, researchers at UCLA's Jonsson Comprehensive Cancer Center, working with investigators from the Institute of Cancer Research in London, Roche and Plexxikon, have elucidated the mechanism by which the drug excels at fighting melanoma but also allows for the development of skin squamous-cell carcinomas.

Dr. Antoni Ribas
The very action by which the pill works — blocking the mutated BRAF protein in melanoma cells that spurs the growth and spread of tumors — sets off a cellular cascade in other skin cells (if they have another predisposing cancer mutation) and ultimately accelerates the secondary skin cancers, said Dr. Antoni Ribas, co-senior author of the paper and a UCLA professor of hematology–oncology.

The 18-month study appears in the Jan. 19 edition of the New England Journal of Medicine.

About 50 percent of patients who get melanoma have the BRAF mutation and can be treated with vemurafenib, Ribas said. Of those, approximately one-fourth develop skin squamous-cell carcinomas. In study subjects, the squamous-cell carcinomas were removed surgically, and the use of vemurafenib was not discontinued because of this secondary skin cancer side effect.

"We wondered why it was that we were treating and getting the melanoma to shrink but another skin cancer was developing," said Ribas, who studies melanoma at the Jonsson Cancer Center. "We looked at what was likely making them grow, and we discovered that the drug was making preexisting cells with an RAS mutation grow into skin squamous-cell cancers."

The combined research team performed a molecular analysis to identify the oncogenic mutations in the squamous-cell lesions of patients treated with vemurafenib. Among 21 tumor samples studied, 13 had RAS mutations. In another set of 14 samples, eight had RAS mutations, Ribas said.

"Our data indicate that RAS mutations are present in about 60 percent of cases in patients who develop skin squamous-cell cancers while being treated with vemurafenib," Ribas said. "This RAS mutation is likely caused by prior skin damage from sun exposure, and what vemurafenib does is accelerate the appearance of these skin squamous-cell cancers, as opposed to being the cause of the mutation that starts these cancers."

Ribas' group found that blocking the non-mutated BRAF in cells with mutated RAS in an animal model caused them to send signals around BRAF that induced the growth of the squamous-cell cancers.

The discovery of the squamous-cell cancer mechanism has led to strategies to inhibit both the BRAF mutation with vemurafenib and block the cellular cascade with a different drug — a MEK inhibitor — before it initiates the secondary skin cancers, said co-senior author Richard Marais, a professor at the Institute of Cancer Research in London, who developed the animal model for the study.

"By understanding the mechanism by which these squamous-cell cancers develop, we have been able to devise a strategy to prevent the second tumors without blocking the beneficial effects of the BRAF drugs," Marais said. "This may allow many more patients to benefit from these important drugs."

Ribas said that this is one of the very few times that oncologists understand molecularly why a side effect to cancer treatment is occurring.

"The side effect in this case is caused by how the drug works in a different cellular setting," he said. "In one case, it inhibits cancer growth, and in another, it makes the malignant cells grow faster."

Studies currently are under way testing BRAF and MEK inhibitors in combination in patients with metastatic melanoma, Ribas said.

"Our data provide a molecular mechanism for the clinical toxicity of a targeted oncogene inhibitor that apparently contradicts the intended effects," the study states.

The study was supported by Roche; Plexxikon; the Seaver Institute; the Louise Belley and Richard Schnarr Fund; the Fred L. Hartley Family Foundation; the Wesley Coyle Memorial Fund; the Ruby Family Foundation; the Albert Stroberg and Betsy Patterson Fund; the Jonsson Cancer Center Foundation; and the Caltech–UCLA Joint Center for Translational Medicine.

UCLA's Jonsson Comprehensive Cancer Center has more than 240 researchers and clinicians engaged in disease research, prevention, detection, control, treatment and education. One of the nation's largest comprehensive cancer centers, the Jonsson Center is dedicated to promoting research and translating basic science into leading-edge clinical studies. In July 2011, the center was named among the top 10 cancer centers nationwide by U.S. News & World Report, a ranking it has held for 11 of the last 12 years.

Plexxikon - 01/19/2012

PRINTed Nanoparticles Delivery Multiple Punches to Treat Prostate Cancer

Using technologies common to the semiconductor industry, a team of
investigators at the University of North Carolina at Chapel Hill and
Liquidia Technologies has created a polymer nanoparticle that can
encapsulate large loads of therapeutic molecules that may have use in
treating prostate cancer. The research, led by Joseph DeSimone,
co-principal investigator of the Carolina Center for Cancer
Nanotechnology, was published in the journal Nano Letters.

Liquidia Technologies - 01/19/2012

FDA New Drug Approvals in 2011 Outpace Recent Past

During 2011, FDA approved 35 new medicines. This was, according to the agency, among the highest number of approvals in the past decade, surpassed only by 37 approvals in 2009.

Of the 35 drugs given the go-ahead, two were approved with a molecular test, making them the first companion drug-diagnostic sanctions. Such tests will be key drivers of drug development in the future. Seven newly sanctioned medications provided advances in cancer treatment, and 10 were for orphan diseases. Notably, the first new therapy for lupus in 50 years was green-lighted this past year.

In terms of how efficiently the FDA was able to do its job, three cancer drugs were given the go-ahead in less than six months. Two-thirds of all the drugs sanctioned last year were completed in a single review cycle and 34 were approved on or before the agreed-upon review time targets.

Cancer

On August 26, FDA green-lighted Pfizer’s Xalkori to treat locally advanced and metastatic non-small-cell lung cancers that express an abnormal anaplastic lymphoma kinase (ALK) gene. The agency approved the drug along with a diagnostic test for the ALK gene abnormality, Abbott Molecular’s Vysis ALK Break Apart FISH Probe Kit. Up to 7% of those with NSCLC, typically patients without a history of smoking, have the gene abnormality.

Pfizer says the tyrosine kinase inhibitor will cost $9,600 a month or $115,000 for patients who take it for a year. In clinical trials, the average duration of treatment was between 22 and 32 weeks, but because the drug appears to extend patients lives, many may be on it for far longer than that.

The other drug-diagnostic approval was Roche’s Zelboraf to treat metastatic or unresectable melanoma and the Cobas 4800 BRAF V600 Mutation Test. Zelboraf, an oral kinase inhibitor, acts by blocking the function of the V600E-mutated BRAF protein. The mutation is present in about half the patients with late-stage melanomas.

The diagnostic will cost around $150 and the drug about $9,400 per month. It assumes, based on progression-free survival data from clinical studies, a treatment course of roughly 6–8 months.

In 2011, FDA sanctioned another drug for metastatic melanoma, Bristol-Myers Squibb’s Yervoy. This mAb therapeutic acts by binding to and blocking the actions of the cytotoxic T-lymphocyte antigen 4 (CTLA-4).

The antibody can cause significant side effects, however, including fatigue, diarrhea, skin rash, endocrine deficiencies, and colitis. Severe to fatal autoimmune reactions were seen in 12.9% of patients treated with Yervoy. When severe side effects occurred, Yervoy was stopped and corticosteroid treatment was started. Not all patients responded to this treatment. Patients who did respond in some cases did not see any improvement for several weeks.

Yervoy achieved sales of $121 million in the third quarter of 2011, with ISI Group analyst Mark Schoenebaum commenting that the number was $17 million above Wall Street projections. Bristol-Myers Squibb got Yervoy as part of its 2009 acquisition of Medarex.

Some analysts see obstacles ahead for Yervoy, which costs $120,000 for a full course of treatment, in the form of resistance from regulators overseas. Last October, the National Institute for Clinical Excellence recommended that at a cost of about £80,000, Yervoy “could not be considered a cost-effective use” of health funds. A final decision is expected after a public consultation.

Hepatitis C

Two drugs for the treatment of hepatitis C won approval in May, 2011—the first new drugs approved for the viral disease in 20 years: Vertex’ Incivek (telaprevir) and Roche’s Victrelis. Both drugs interfere with the ability of HCV to replicate by inhibiting a key viral enzyme (NS3/4A serine protease).

The FDA said that it may be possible to shorten Incivek treatment from 48 weeks to 24 weeks in most patients. Given the toxicity of hepatitis C therapies, a shortened treatment period represents a significant advance.

Incivek was widely expected to generate the biggest sales: $4 billion worldwide by 2016, with Vertex taking in $1.75 billion in the U.S. following a peak of $2.21 billion in 2014. Telaprevir’s market advantage, analysts said, will come from its slightly better efficacy and side-effect profile. Victrelis’ anemia side effect will require management with erythropoietin-stimulating agents in many cases. Further telepravir performed well in the null responder population, which had proven controversial for Victrelis.

Lupus

Last year also saw approval of the first new drug developed for lupus in 50 years, Human Genome Sciences (HGS) and GlaxoSmithKline’s Benlysta. The mAb therapy was green-lighted on March 9, 2011, for adult patients with active, autoantibody systemic lupus erythematosis who are receiving standard therapy.

Benlysta targets the B-lymphoycte stimulator (BLyS) protein and may reduce the number of abnormal B cells thought to contribute to the disease. Analysts predict sustainable, long-term blockbuster potential for Benlysta, with projected sales of $2 billion to $4 billion annually.

Good Year for FDA

In looking over 2011 drug approvals, FDA did a pretty good job despite the carping from the pharma industry, financial community, and some patient advocacy groups.

FDA’s new cancer drug sanction process is consistently faster than the EMA, according to Samantha A. Roberts, Jeff D. Allen, and Ellen V. Sigal, affiliated with Friends of Cancer Research. They conducted a review and analyses of data available on FDA and EMA websites for the period between 2003 and 2010.

Among 35 new cancer drugs reviewed by FDA, 32 were approved, 20 of them within 184 days. EMA, on the contrary, approved only 26 of these new cancer drugs within an average time of 350 days. “Contrary to repeated public assertions, we found that new oncology medicines are consistently available in the United States before they are in Europe, and they are more likely to be approved by the FDA than by the EMA,” wrote the authors.

The report went on to say, rightly so, that its findings reinforced the need for strong financial and public support of the FDA so that “such medicines can continue to be made available speedily to patients in need.”

Plexxikon - 01/09/2012

ACH-1625 Receives Fast Track Designation From the FDA for the Treatment of Chronic Hepatitis C

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.

Achillion Pharmaceuticals - 01/04/2012

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.

Achillion Pharmaceuticals - 01/04/2012

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 - 12/28/2011

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.

Marina Biotech - 12/28/2011