The global pharmaceutical company AstraZeneca is buying Pearl Therapeutics in Redwood City, California, a developer of treatments for chronic respiratory diseases. The deal is valued initially at $560 million, but has a total potential value of $1.15 billion, adding in subsequent milestone and royalty payments.
The seven year-old Pearl Therapeutics develops small-molecule treatments for chronic obstructive pulmonary disease (COPD) and asthma. COPD is a group of progressive disorders including emphysema that make breathing difficult and resulting in mucus buildups in the lungs.
COPD is most associated with tobacco smoke, and most people with COPD are current or former smokers, but it can also be triggered by long-term exposure to pollutants or chemical fumes. The Centers for Disease Control and Prevention says some 15 million Americans have been diagnosed with COPD, and in 2011 it was the third leading cause of death in the U.S.
Pearl Therapeutics’ drug-delivery technology provides stable aerosol suspensions of porous particles that work in metered dose inhalers using environmentally-friendly hydrofluoroalkane propellants. Earlier inhalers had propellants made from chlorofluorocarbons, which deplete the ozone layer in the atmosphere and were phased out in 2008. Metered dose inhalers are the most widely used method for delivering COPD and asthma drugs.
The company’s lead product, code-named PT003, is a combination of the drugs glycopyrrolate and formoterol taken twice a day with a metered dose inhaler. Pearl Therapeutics says PT003 is in late-stage (phase 3) clinical trials, having completed early safety and small-sample efficiacy tests.
Under the deal, AstraZeneca will pay an initial $560 million for all Pearl Therapeutic shares, with another $450 million contingent on development and regulatory milestones from future selected products made with Pearl Therapeutic’s technology, including triple-combination products. Up to $140 million in additional payments will be made contingent on exceeding pre-agreed sales targets.
Pascal Soriot, AstraZeneca’s CEO (pictured at top), says the acquisition will bolster AstraZeneca’s respiratory product line that includes combination treatments delivered with an inhaler. The company aims to develop a triple-combination product that adds inhaled corticosteroids to Pearl Therapeutics’ PT003 ingredients.
Sanofi Pasteur, the vaccine division of the global pharmaceutical company Sanofi, says the Food and Drug Administration cleared for sale in the U.S. its Fluzone Quadrivalent vaccine that covers four influenza strains. The FDA action, says the company, will make the vaccine available for infants, adolescents, and adults.
The FDA action is an approval of Sanofi Pasteur’s biologics license application for the product. Until this year, most seasonal flu vaccines were trivalent, containing inactive viruses of the three influenza strains most likely faced in a particular flu season. The vaccines covered two A and one B type viruses
Starting this year, quadrivalent vaccines, covering two strains each of A and B type flu viruses are being made available. The addition of another B strain is needed, says Sanofi Pasteur, because of more than one B type viruses in circulation, which made it difficult for public health authorities to plan for an effective vaccine formulation for the upcoming season.
The company says its quadrivalent vaccine will be available in the U.S. for the 2013-2014 flu season, in prefilled syringes or single-dose vials for intramuscular administration. The CDC lists eleven vaccine products available for the 2013-2014 season, including two other quadrivalent vaccines by GlaxoSmithKline and MedImmune.
Medical device manufacturer St. Jude Medical in St. Paul, Minnesota is investing $40 million in pain therapy systems developer Spinal Modulation Inc. in Menlo Park, California. The equity investment deal, says St. Jude Medical, includes an option for the outright purchase of the privately-owned Spinal Modulation.
Under the agreement, St. Jude Medical becomes the exclusive distributor of Spinal Modulation’s lead product, the Axium Neurostimulator System. The system delivers a form of stimulation through low-level electrical signals to the dorsal root ganglion, a collection of neurons or nerve cells on the spine that carry signals from sensory organs to the brain.
The dorsal root ganglion has been identified as a promising target of treatments for chronic pain. Spinal Modulation says the system has shown results in managing chronic leg pain, as well as back and foot pain that can be difficult to treat. The Axium system, says the company, can be adjusted to treat broad or specific areas, often with less discomfort than other forms of spinal stimulation.
In the deal, St. Jude Medical will be the sole distributor of the Axium system, even in international markets where the device is now approved. The Axium system received in 2011 a CE mark, signifying clearance to market the device in Europe.
The agreement also give St. Jude Medical an option to purchase the entire Spinal Modulation company, following commercialization of the product in the U.S., for $300 million plus revenue-based milestones. Spinal Modulation received an investigational device exemption from the Food and Drug Administration, which authorizes the conduct of clinical trials of the device in the U.S., which are expected to begin in the second half of 2013.
The American Academy of Pain Medicine says the number of people suffering from acute or chronic pain numbers in the millions. The organization cites data from the Institute of Medicine that chronic pain costs the U.S. $560 to $635 billion annually, an amount equal to about $2,000 for every U.S. resident.
The Blood Cancer Research Partnership aims to make it easier for patients to take part in clinical trials by opening more test sites, particularly in communities distant from major research and medical centers. Some 11 new trial sites have been identified in New York, Georgia, Colorado, Illinois, California, Florida, Texas, Kansas, Tennessee, New Jersey, and Washington State.
Dana-Farber will serve as the lead institute in the partnership, with each of the community sites required to follow clinical trial protocols established in a single agreement. Clinical trials conducted in this network will be either early or intermediate stage (phase 1 or 2), with patient enrollment taking place over an 18-month period.
The funds for the partnership will be provided by LLS, subject to certain milestones being met, such as the numbers of trials started under the program and numbers of patients recruited for each trial. The foundation will also be assigned two seats on the steering committee that decides on the trials conducted at these sites. Clinical trials under consideration include those for chronic lymphocytic leukemia, myeloma, and stem cell transplants.
Louis DeGennaro, LLS’s chief mission officer, notes “Most cancer patients are treated by oncologists in their local community,” and adds, “Having to travel long distances from home to a major medical center is a major deterrent to patients’ participation in cancer clinical trials.”
Kansas State University in Manhattan received a patent and licensed a nutritional supplement for cattle feed developed by one of its faculty members. Research by animal sciences professor Jim Drouillard led to development of a supplement that improves absorption of omega-3 fatty acids beneficial to livestock, which Kansas State patented earlier this year and licensed to New Generation Feeds, a South Dakota animal feed manufacturer.
The supplement adapts a formula that combines precise concentrations of flax oil, flax seed, and a non-flax carbohydrate such as molasses. These ingredients, when heated to 120 degrees F and then cooled, has been shown to help test animals absorb alpha-linolenic acid, a type of omega-3 fatty acid, which is converted by the animals (and humans as well) to eicosapentaenoic acid.
Eicosapentaenoic acid is another type of omega-3 fatty acid that has benefits for cattle, including stimulation of growth, improved immunity against disease, and enhanced reproductive function. Drouillard’s research shows the supplement can raise those levels as much as 10 percent.
Cattle consume the supplement at will, from large highly-concentrated blocks placed in pastures, in what Drouillard calls “a big 250-pound piece of candy for livestock.” The physical properties of this delivery method restrict the consumption by animals to less than one pound per day.
Engineered vein being implanted by surgeons (Duke University Medicine)
Yesterday, a kidney dialysis patient at Duke University Hospital in Durham, North Carolina was the first in the U.S. to receive a new bioengineered blood vessel developed by a Duke University spin-off company. The patient, a 62 year-old man from Danville, Virginia with kidney failure received in his arm the engineered blood vessel made with a process developed by Humacyte Inc., also in Durham.
A new vein is often required by patients with kidney disease needing dialysis, who receive a graft to connect an artery to a vein to speed the flow of blood during treatments. Current options for this procedure all have drawbacks: Synthetic veins are prone to blood clots, harvesting other veins for transplants requires separate surgery with higher risks of infection. Veins made from a person’s own cells takes an inordinate amount of time to grow and are not amenable to advance production.
The Humacyte technology for generating bioengineered veins is the result of a research collaboration between Duke medical professor Jeffrey Lawson — who performed the implantation — and former Duke postdoctoral fellow Laura Niklason, who went on (with colleagues) in 2005 to start Humacyte. Niklason, now at Yale University, and Lawson first tried to create new bioengineered veins from a patient’s own cells, later shifting to a process that still creates a usable engineered vein, but instead with compatible and neutral biomaterials.
The process starts with a flexible biodegradable mesh serving as a scaffold for the vein that can be shaped into a tube. The scaffold is then seeded with donated human smooth muscle cells that grow in a medium of amino acids and nutrients. The growing muscle cells are subjected to a pulsating flow resembling a heartbeat rhythm that strengthens the engineered tissue and results in physical properties resembling natural vascular tissue.
The tubular seeded scaffold is then washed in a solution that rinses out cellular properties, leaving a biocompatible collagen tube that does not trigger an immune response from the implanted patient. The process for creating this allogeneic — same species, different donor — replacement tissue takes about two months.
These engineered veins, says Humacyte, can be created in advance, then stored for use later on. Preclinical tests with baboons show the engineered veins perform better than those made from synthetic or animal-based materials. The patient implanted yesterday is part of an early-stage safety trial of the engineered vein with 20 dialysis patients. A similar clinical trial is underway as well in Poland.
Data from 2006 suggest more than 350,000 people in the U.S. are receiving dialysis treatments. Niklason and Lawson believe the technique can also be applied to the nearly 400,000 people in the U.S. who have heart bypass surgery, as well as patients with blocked blood vessels in their limbs. Humacyte says it has a pilot manufacturing facility meeting current good management practices for pharmaceuticals, and expects to broaden its pilot-scale facility during 2013.
Artist’s concept drawing for AMRC Factory 2050 (University of Sheffield)
University of Sheffield in the U.K. says it secured funding for an advanced manufacturing lab to study the needs of the aerospace industry and other high-value manufacturing industries. The £43 million ($US 66.3 million) lab, part of Sheffield’s Advanced Manufacturing Research Centre (AMRC), is supported by Boeing — which has its name on the official facility title — as well as manufacturers Airbus, Rolls-Royce, BAE Systems, and Spirit AeroSystems.
The Higher Education Funding Council for England, says Sheffield, is contributing a £10 million ($15.4 milion) grant from its Research Partnership Investment Fund for the facility. The university says the new lab, known as AMRC Factory 2050, will be the first in the U.K. with a completely reconfigurable assembly and component manufacturing capability, to quickly switch between making high-value components and one-off parts.
AMRC Factory 2050 is expected to include technologies such as advanced robotics, virtual environments, and 3-D printing. The university says some 50 researchers and engineers will work in the new facility, with completion scheduled for next year, although the exact location is still undecided.
The lab, says Sheffield, will have about 4,500 square meters of floor space, and be made largely of glass. The building will be constructed to Building Research Establishment Environmental Assessment Method (BREEAM) standards, a voluntary rating for green buildings in the U.K.
The overall AMRC facility is a lab for advanced manufacturing technologies, supporting research groups in machining, assembly, composite materials, and structural testing. The lab carries out generic research of interest to its 70 industrial supporters, as well as specific research projects for individual company clients, or studies supported by special grants from U.K. or European funding sources.
Some 70 health care organizations worldwide are forming an alliance to develop common standards for collecting and sharing genetic data. The organizations — representing health care providers, government agencies, academic and research institutes, foundations, and advocacy groups — seeks to create a common interoperable framework, with associated regulatory and practical guidelines, for the interchange of data related to human genomics.
The Wellcome Trust Sanger Institute in the U.K. has so far organized the effort to create an international not-for-profit organization, modeled on the World Wide Web Consortium, to develop the common framework. The 71 groups signed a letter of intent to create and support this organization. While the as yet unnamed organization plans to include life science and information technology enterprises, no companies from these sectors have so far signed the letter of intent.
The need for this framework and organization, says the Wellcome Trust, results from the sharp drop in cost of sequencing human genomes, and the associated large increase in the number of people making their personal genetic and clinical data available for research and further clinical use. While the need for a common framework and more sharing of these data are widely recognized, efforts to collect and share genomic data have been confined to individual diseases, institutions, or countries, which are limiting the expansion of knowledge and its potential benefits to improvements in health care.
In January 2013, 50 representives of health care organizations in eight countries met in New York to discuss these challenges and agreed that a key missing ingredient was a common framework of standards to make possible and govern the sharing of genomic and clinical data. A subsequent white paper from the meeting led to the letter of intent to form the new body, signed by 71 organizations mainly from North America and Europe, but also Australia, Japan, China, and South Africa.
Among the problems being created with the current independent and fragmented systems, according to the white paper, are the lack of large samples needed to discover underlying pattens and relationships in genomic data. Because of the large difference in disease targets and low frequencies of genomic sequencing variations, millions of samples are often needed to provide reliable results.
Yet despite the obvious advantages in standardization, says the white paper, the world’s scientific and medical communities seem to be moving in an opposite direction, with more incumbent and private systems being established. The paper pointed specifically to the current state of fragmented and incompatible electronic health records in the U.S. as a potential outcome for genomic data if current trends continue.
Among the signers of the letter of intent is National Institutes of Health, along with the National Cancer Institute and National Human Genome Research Institute, which are part of NIH. Francis Collins, director of NIH told the New York Times that the institutes “are strong supporters of this global alliance,” adding, “We need standard formats so we don’t have to spend two years figuring out how to merge data together.”
Students fitting colleague with the EEG head cap (Biomedical Functional Imaging and Neuroengineering Laboratory, University of Minnesota)
Engineers at University of Minnesota in Minneapolis developed a head cap with electrodes that captures and interprets thoughts to direct autonomous devices. The team led by biomedical engineering professor Bin He demonstrated the system with a remote-controlled flying device and published its findings yesterday in the Journal of Neural Engineering.
The cap worn by the subjects resembles a swim cap that fits snugly over the skull, with 64 electrodes to capture brain signals with electroencephalography (EEG), a technique to diagnose disorders such as epilepsy or brain injuries. The cap captures signals from the motor cortex that controls voluntary movements, which in this case, represent thoughts about movements triggering a different set of neurons in the brain. Unlike other systems that capture brain signals, the cap is non-invasive; no chips or electrodes or implanted in the wearer.
In earlier research, Prof. He was able to map the areas of the brain activated when a person imagines physical movements. Prof. He followed up that earlier work, with experiments showing the system could control the movements of a virtual helicopter on a computer screen.
In the new study, Prof. He and colleagues applied the system to controlling the movements of a model helicopter, an AR Drone autonomous flying device with four rotors, made by the company Parrot SA. The AR Drone was preset to fly forward, unless instructed to fly in another diection by the operator.
The EEG system captures thoughts by the individual imagining use of the right hand, left hand, or both hands together. Signals from these thoughts were translated into instructions to turn the AR Drone right or left, or in vertical movements up or down, and were transferred to the model helicopter over a local WiFi network.
In the study, the researchers tested the ability of five test subjects wearing the EEG system to control the AR Drone device, including navigating the model helicopter through two large balloon rings suspended from the ceiling of a gymnasium. The test subjects faced away from the flying model, but could view its movements over a computer screen.
The researchers measured the performance of the test subjects against another group using a standard keyboard to control the AR Drone. The results showed the test subjects were able to perform from 69 to 92 percent of the required tasks, without crashing the AR Drone into the gymnasium walls. Using a higher performance standard — directing the AR Drone through the ring targets without colliding with the rings — the test subjects succeeded between 54 to 85 percent of the time.
Prof. He believes the findings can be applied to help people with paralysis or neurodegenerative diseases, for controlling wheelchairs or prosthetic devices, as well as victims of stroke. “We’re now studying some stroke patients,” adds Prof. He, “to see if it’ll help rewire brain circuits to bypass damaged areas.”
The following video tells more about the EEG system and shows some of its use with the AR Drone.
ResearchGate, an online network to encourage collaboration among scientists and sharing of research data, based in Berlin, closed a $35 million series C financing round, the third cycle of venture funding after start-up. The round was led by Microsoft founder Bill Gates and later-stage venture capital (VC) company Tenaya Capital.
ResearchGate is a five-year old company founded by two physicians and a computer scientist that aims to make science and scientists more accessible to encourage greater collaboration and sharing of research findings. The company’s services include a profile where scientists can upload their research to make it available to their scientific colleagues, as well as joint projects where ResearchGate members collaborate directly or provide professional feedback. ResearchGate’s 2.9 million members are ranked according to an RG Score, an index of participation and interaction on the network.
The new financing round is expected to help ResearchGate expand its services to include capture of raw research data, as well as results of failed experiments. The company says it also plans to improve its ratings of scientific reputation. In addition to Gates and Tenaya Capital, other participants in the round are existing investors Benchmark and Founders Fund, as well as new investors Dragoneer Investment Group and Thrive Capital.
Tenaya Capital is a VC company investing largely in information technology enterprises, from those early in revenue generation to pre-IPO stage. Its portfolio includes Internet, software, communications, and semiconductor companies. The company says it typically leads investment rounds and contributes $5 to $10 million per round.
In November 2012, ResearchGate acquired Scholarz.net, a spin-off company from University of Wurzburg in Germany that provided reference and knowledge management services, as well as an academic network of two million members. Scholarz.net discontinued its services and network in January 2013, which were absorbed into ResearchGate.
The online newsletter MedCity News says Gates participated in the financing as an individual, not through his charitable foundation.
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