Austin, TX's microRNA replacement specialist Mirna Therapeutics last week announced its up to $81 million Nasdaq IPO plans on the same day that the Nasdaq Biotechnology Index plummeted 23%, though it ended the day down 4%.
The company is developing a tumor suppressor or known as MRX34 that inhibits multiple oncogene pathways to fight cancer while (hopefully) limiting the development of drug resistance. The candidate is in currently in a Phase I dose escalation study to determine its maximum tolerated dose against a variety of cancers, including primary liver cancer, lymphoma and melanoma.
The microRNA is delivered using the Smarticles platform and formulation, licensed from Marina Biotech, which describes its tech as "amphoteric liposomes composed of unique combinations of lipids having anionic and cationic groups that work together to enable cell uptake, to provide serum stability, and to provide pH-triggered endosomal escape."
We license the technology related to SMARTICLES from Marina. Our license with Marina imposes various development, regulatory, commercial diligence, financial and other obligations. If we fail to comply with our obligations under the agreement with Marina, or otherwise materially breach the agreement with Marina, and fail to remedy such failure or cure such breach, Marina may have the right to terminate the license. The loss of the license from Marina would affect a portion of the patent portfolio for MRX34, which would adversely affect our ability to proceed with any development or potential commercialization of MRX34, and could subject us to claims of patent infringement by Marina if MRX34 is covered by the affected patents.
We determined that the SMARTICLES formulation technology, owned by Marina Biotech, Inc., or Marina, had a favorable combination of efficient systemic delivery of miR-34 mimics to solid tumors in mice, a high therapeutic activity of formulated miR-34 in mouse models of cancer, low or no toxicity, and low or no cytokine stimulation in both animal models and an ex vivo human whole blood assay.
Efficient Systemic Delivery
The SMARTICLES formulation demonstrated key benefits in preclinical studies, including the ability to deliver very high numbers of microRNA mimics to tumors.
We have paid Marina approximately $2.1 million in the aggregate to date in up-front and milestone payments (including the milestone prepayment under the May 2015 amendment) and as consideration for the inclusion within the license of the three additional compounds. As we progress development and commercialization of products covered by the license, we will be required to make payments to Marina based upon the achievement of certain development and regulatory milestones, totaling up to $6 million in the aggregate for each licensed product. We are also required to pay up to an additional $4 million per licensed product upon the achievement of certain regulatory milestones for a specified number of additional indications, leading to a maximum cap on all milestone payments of $10 million per product. The exception to this is for our lead therapeutic product, MRX34, where the aggregate of all remaining development and regulatory milestone payments due to Marina, including for all additional indications, is $3.7 million. In addition to milestone payments, we will be required to pay low single digit royalties on net sales of licensed products other than MRX34, subject to customary reductions and offsets. As a result of our 2013 amendment to our agreement with Marina, we are no longer required to pay a royalty to Marina with respect to sales of our lead therapeutic product, MRX34.
M Fool says, re Vivent " If the buyout goes through the downside will be less than SunEdison's plunge, but SunEdison's stock drop lowers Vivint Solar's buyout price. "
The Fed’s rate hike plans are on a collision course with the economic cycle. In fact, the Fed is signaling a rate hike this year – if not by September, then by December – clearly expecting a pickup in growth. ECRI’s leading indexes suggest the opposite.
Indeed, a service sector slowdown has already joined the manufacturing slowdown that started last fall, and so the slowdown in overall growth is likely to intensify in the coming months. As such, hopes for a “second-half rebound” are likely to be dashed.
ECRI’s indicators show that the U.S. economy is in a GRC downturn that is poised to persist. The key question is whether there is a significant risk of recession in the context of possible shocks, including those from China, the Fed, or any other source. The answer from our indicators is that, for the time being, the U.S. economic expansion is in a cyclically resilient phase where it is resistant to shocks that could tip the economy into recession.
Prosensa's drug failed to beat a placebo in significantly improving boys' ability to complete a 6-minute walk test--the classic measure of success in this field. But left to its own devices the biotech completed fresh analysis of new extension study data that backed a theory that providing the drug earlier while extending treatment could delay disease progression. The biotech held what it called "positive" reviews with the FDA and began a rolling submission of the application just weeks ago. Evidently BioMarin has high hopes of success where GlaxoSmithKline saw nothing but failure. November 24, 2014.
According to Navigant Research, worldwide revenue from energy storage enabling technologies (ESETs) is expected to total nearly $75 billion from 2015 to 2024. In its recent report Navigant analyzes the global market for ESETs across four market segments: utility-scale storage, community storage, residential storage, and commercial storage. Broadly speaking, the three main components of the ESET value chain are hardware, software, and services, which together ensure the intelligence, durability, and profitability of energy storage systems (ESSs). Now that battery prices have responded to cost pressures, the rest of the balance of plant—or the ESET portion of system cost—is under more pressure to deliver more consistent pricing, which is expected to bring more transparency in overall ESS pricing, allowing the industry to scale further.
While the European Medicines Agency said it "does not question that the benefits of HPV vaccines outweigh their risks," on Monday it announced a safety review of the shots that have thus far failed to live up to expectations partly due to safety concerns and a sex-related stigma. The safety watchdog said it'll analyze available data with a focus on rare reports of complex regional pain syndrome--a chronic pain condition affecting the limbs--and postural orthostatic tachycardia syndrome, in which the heart rate increases abnormally after sitting or standing up.
Our DNAi platform technology is based on the breakthrough discovery at Wayne State University and Karmanos Cancer Institute that single-stranded DNA oligonucleotides can interact with genomic DNA in order to interfere with gene transcription. We acquired this technology and subsequently developed an algorithm that we employ for rationally designing our DNAi product candidates using bioinformatics and our understanding of gene regulatory domains. In March 2007, we entered into an exclusive license agreement with Novosom AG (Novosom) to use certain patented lipid nanoparticle (LNP) delivery technology used in PNT2258. In July 2010, the original Novosom license agreement was acquired by Marina Biotech, Inc. (Marina), but Novosom retained the right to receive all future payments related to PNT2258. In March 2012, we and Marina entered into another exclusive license agreement for the use of certain of Marina’s patented delivery technology, including LNP technology, for any of our current or future DNAi product candidates other than PNT2258. In exchange for this exclusive right, we paid Marina an upfront payment of $0.3 million and will also be required to pay Marina milestone payments of up to an aggregate of $14.5 million for each DNAi product candidate other than PNT2258 as well as low single-digit royalties on net sales for DNAi product candidates other than PNT2258. In April 2014, we entered into a license payment agreement with Novosom, pursuant to which we made a one-time payment to Novosom of $11.0 million in April 2014 and terminated the other payment obligations owed to Novosom for PNT2258 except for one remaining $3.0 million payment due upon regulatory approval of PNT2258 and a low single-digit royalty on net sales of PNT2258.
ProNAi Therapeutics, which is developing cancer therapies based on DNA interference, raised the proposed deal size for its upcoming IPO on Wednesday.
The Vancouver, Canada-based company now plans to raise $138 million by offering 8.1 million shares at a price of $17. The company had previously filed to offer 6.7 million shares at a range of $14 to $16. At the revised price, ProNAi Therapeutics will raise 37% greater proceeds than previously anticipated.
ProNAi Therapeutics, which was founded in 2003, plans to list on the NASDAQ under the symbol DNAI. Jefferies and BofA Merrill Lynch are the joint bookrunners on the deal. It is expected to price this week.
The new microneedle patch is made of dissolvable material, eliminating needle-related risks. It is also easy to use without the need for trained medical personnel, making it ideal for use in developing countries, where healthcare resources are limited.
“Our novel transcutaneous vaccination using a dissolving microneedle patch is the only application vaccination system that is readily adaptable for widespread practical use,” said Prof. Shinsaku Nakagawa, one of the authors of the study and Professor of Biotechnology and Therapeutics at the Graduate School of Pharmaceutical Sciences at Osaka University. “Because the new patch is so easy to use, we believe it will be particularly effective in supporting vaccination in developing countries.”
The new microneedle patch – MicroHyala – is dissolvable in water. The tiny needles are made of hyaluronic acid, a naturally occurring substance that cushions the joints. When the patch is applied like a plaster, the needles pierce the top layer of skin without causing pain and dissolve into the body, taking the vaccine with them.
The researchers compared the new system to traditional needle delivery by vaccinating two groups of people against three strains of influenza: A/H1N1, A/H3N2 and B. None of the subjects had a bad reaction to the vaccine, showing that it is safe to use in humans. The patch was also effective: people given the vaccine using the microneedles had an immune reaction that was equal to or stronger than those given the vaccine by injection.
“We were excited to see that our new microneedle patch is just as effective as the needle-delivered flu vaccines, and in some cases even more effective,” said Dr. Nakagawa. “We have shown that the patch is safe and that it works well. Since it is also painless and very easy for non-trained people to use, we think it could bring about a major change in the way we administer vaccines globally.”
With the NBA draft still 18 months away, Willie Cauley-Stein told reporters in late 2013 that he cut short his play in a recent college basketball game because of severe chest pain and shortness of breath due to a genetic disorder called sickle-cell trait.
That condition made Cauley-Stein, a 21-year-old, 7-foot-1, 240-pound forward/center and Southeastern Conference Defensive Player of the Year, a potential liability for any team that drafted him. But it didn’t stop the Sacramento Kings, which chose him Thursday as the sixth pick in this year’s draft.
Cauley-Stein and team vice president Vlade Divac said Saturday the condition should not affect his play.
“No big deal,” Divac said he concluded after talking to a team doctor about the condition.
“I don’t see that it had an effect on him” at Kentucky,” Divac told The Sacramento Bee after Cauley-Stein’s introductory news conference at the team’s J Street office adjacent to the arena construction site.
Cauley-Stein said it’s manageable with hydration and nutrition.
“There is no issue. You have to be cautious of it. You have to stay hydrated. You have to eat well. I eat really organic,” Cauley-Stein said.
In 2013, he described his condition to the Lexington Herald-Leader after having to leave the court after 25 minutes while playing in a game for Kentucky.
“My chest starts to hurt,” he said after the game against Boise State. “Some days, just randomly, I’ve had real bad chest pain. I can’t, like, breathe, and my heart rate won’t come down. I have to stop and wait for it to come down.”
Two days later, John Calipari, Kentucky’s head coach, said Cauley-Stein knew how to manage the illness.
“If he gets winded or he feels it, he’ll take himself out,” Calipari said, “He’s pretty good about it.”
So what is sickle-cell trait?
It disproportionately affects African Americans, and sufferers inherit one sickle-cell gene and one normal gene. People who inherit two sickle-cell genes, one from each parent, can develop
Halofuginone is a coccidiostat used in veterinary medicine. It is a synthetic halogenated derivative of febrifugine, a natural quinazolinone alkaloid which can be found in the Chinese herb Dichroa febrifuga (Chang Shan). Collgard Biopharmaceuticals is developing halofuginone for the treatment of scleroderma and it has received orphan drug designation from the U.S. Food and Drug Administration.
Halofuginone inhibits the development of T helper 17 cells, immune cells that play an important role in autoimmune disease, but it does not affect other kinds of T cells which are involved in normal immune function. Halofuginone therefore has potential for the treatment of autoimmune disorders.
Halofuginone is also an inhibitor of collagen type I gene expression and as a consequence it may inhibit tumor cell growth. Halofuginone exerts its effects by acting as a high affinity inhibitor of the enzyme Glutamyl-Prolyl tRNA synthetase. Inhibition of prolyl tRNA charging leads to the accumulation of uncharged prolyl tRNAs, which serve as a signal to initiate the amino acid starvation response, which in turn exerts anti-inflammatory and anti-fibrotic effects.
Akashi Therapeutics' Duchenne MD candidate shows encouraging results in early stage study
Jun 18 2015, 10:48 ET | By: Douglas W. House, SA News
Interim data from a Phase 1b/2a clinical trial assessing privately-held Akashi Therapeutics' product candidate for Duchenne muscular dystrophy (DMD), HT-100, showed that patients with at least six months of continuous dosing achieved average total muscle strength 22.3% greater that levels predicted by comparable steroid-treated external control. The results were determined by quantitative muscle testing (QMT) of upper and lower extremity muscle groups.
· The average increase in muscle strength compared to baseline over 18-22 months in the 10 patients in the trial was 11.7%. All study participants are on a stable regimen of corticosteroids.
· Orphan Drug- and Fast Track-designated HT-100 (delayed-release halofuginone) is an orally available small molecule designed to reduce fibrosis (scarring) and inflammation and promote healthy muscle fiber regeneration in patients with DMD.
· The trial completion date is January 2016. An open label extension study will run until February 2017.
... and burn the BS too !
LA is sooo clean.
That's because they put all their garbage on TV.
Fuel cell technology developer Ballard Power Systems (Vancouver, B.C.) announced that it has reached license and supply agreements with Nantong Zehe New Energy Technology Co., Ltd. and Guangdong Synergy Hydrogen Power Technology Co., Ltd. under which Ballard will provide fuel-cell power products and related technical services in support of the planned development of fuel cell-powered bus fleets in two Chinese cities.
Ballard and Nantong Zehe are collaborating with electric bus manufacturer Jiangsu GreenWheel New Energy Electric Vehicle Co. Ltd. to deploy a fleet of fuel cell-powered buses in the city of Rugao in Jiangsu province. In a separate arrangement, Ballard and Guandong Synergy are working with electric bus manufacturer Foshan Feichi Automobile Manufacturing Co. Ltd. to deploy fuel cell-powered buses in the city of Yunfu in Guangdong province. Between the two projects, Ballard and its partners expect to place a total of 33 fuel cell-powered buses on the streets of these cities. The combined contracts have an estimated value of $10 million to Ballard.
Market Caps: BLDP 323.52M`` HYGS 106.95M
Reuters) - The U.S. Environmental Protection Agency on Wednesday said greenhouse gases from aircraft endanger human health, taking the first step toward regulating emissions from the domestic aviation industry.
The EPA's endangerment finding kicks off a process to regulate greenhouse gas emissions from the aviation industry, the latest sector to be regulated under the Clean Air Act after cars, trucks and large stationary sources like power plants.
The finding allows the EPA to implement domestically a global carbon dioxide emissions standard being developed by the International Civil Aviation Organization (ICAO).
The U.N. agency is due to release its CO2 standard for comment in February 2016 and adopt it later that year.
The EPA had been under pressure from environmental groups who first petitioned it to regulate aircraft emissions under the Clean Air Act in 2007 and sued it in 2010. A federal court ruled in favor of those green groups in 2012.
Aviation accounted for 11 percent of energy-related carbon dioxide emissions from the transportation sector in 2010 in the United States, according to the International Council on Clean Transportation.
The airline industry has favored a global standard over individual national standards since airlines operate all over the world and want to avoid a patchwork of rules and measures, such as taxes, charges and emissions trading programs.
"If you're a big airline and you're flying to 100 countries a day, then complying with all those different regimes is an administrative nightmare," said Paul Steele, senior vice president at the International Air Transport Association, the main global airline industry group.
But some environmental groups are concerned that the standard being discussed at ICAO will do little to change the status quo since it would only apply to new and newly designed aircraft that will not be in operation for several years.
"The stringency being discussed at ICAO is such that existing aircraft are already meeting the standard they are weighing," said Sarah Burt, an attorney at Earthjustice, one of several groups that sued the EPA to regulate aircraft. (Reporting By Valerie Volcovici; additional reporting by Victoria Bryan in Miami; Editing by Susan Heavey and Doina Chiacu)