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Algae Feed Proposed to Enrich Farm Salmon Fatty Acids

Salmon in store

(Marlith, Wikimedia Commons)

21 November 2016. A developer of feeds for farmed salmon says its algae-based feed supplements could arrest the sharp decline in omega-3 fatty acids discovered in a recent study. The company TerraVia Holdings in San Francisco makes an algae supplement for farmed fish that it says can substitute for oil from small fish normally eaten by salmon, but are disappearing.

TerraVia is responding to a study reported by the BBC and others in October 2016 that concentrations of omega-3 fatty acids in farmed salmon today are about half the level of farmed salmon caught 5 years ago. The study was conducted by the Institute of Aquaculture at University of Stirling in the U.K., led by Douglas Tocher, a biochemist at the institute. Tocher studies molecular biology and the role of genetics in regulating lipid and fatty acid metabolism and nutrition in fish, particularly those with omega-3 fatty acids.

Omega-3s are considered essential polyunsaturated fatty acids with benefits affecting the heart and brain. These substances are believed to reduce inflammation, and help improve heart health and cognitive functions. University of Maryland Medical Center cites data showing infants who do not get enough omega-3 fatty acids from their mothers during pregnancy are at risk for developing vision and nerve problems. And American Heart Association recommends eating foods high in omega-3s, such as salmon, twice a week.

Tocher and colleagues, however, found that people would need to eat twice as much salmon as before to get the same omega-3 benefits. “About five years ago,” Tocher told the BBC, “a portion of Atlantic salmon of 130 grams was able to deliver three-and-a-half grams of beneficial omega-3. This is actually our weekly recommended intake. Now, the level of omega-3 has halved.” Much lower levels of oil in fish feed is believed to be responsible for lower levels of omega-3 fatty acids in the farm-raised fish.

TerraVia is a provider of food, nutrition, and specialty ingredients based on algae. The company partners with agribusiness enterprise Bunge Ltd. to make a fish feed ingredient from algae known as AlgaePrime DHA designed for farmed raised fish. DHA is short for docosahexaenoic acid, a specific omega-3. AlgaePrime DHA, say the companies, is rich in long-chain or complex DHA similar to those found naturally in fish and olive oils, but in higher concentrations.

“AlgaPrime DHA contains approximately 3 times the level of DHA compared to fish oil,” says TerraVia vice-president Walter Rakitsky in a company statement. “One ton of AlgaPrime DHA is the equivalent of saving up to 40 tons of wild caught fish from our oceans on a DHA basis.”

AlgaPrime DHA is made in a joint venture with Bunge’s subsidiary in Brazil producing renewable edible oils. The companies say AlgaPrime DHA is a microalgae product from sugar cane waste fermented into omega-3 algae. The process, the companies add, uses low carbon and water inputs.

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Novartis Acquires Sickle-Cell Drug Developer

Red blood cells with sickle cell disease

Red blood cells with sickle cell disease (

21 November 2016. Drug maker Novartis is acquiring Selexys Pharmaceuticals Corp., a developer of treatments for pain episodes associated with sickle cell disease. The agreement to acquire Selexys is expected to bring the Oklahoma City company’s shareholders as much as $665 million.

Selexys Pharmaceuticals’ lead product, code-named SelG1, is designed as an antibody to address a key protein believed responsible for damage to blood vessels from sickle cell disease and the pain that results. Sickle cell disease is a genetic blood disorder affecting hemoglobin that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape.

That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes. The disease is prevalent worldwide, and affects 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.

Selexys Pharma developed SelG1 as a synthetic antibody that seeks out and blocks the actions of P-selectins, proteins that accumulate on the surface of endothelial cells lining the inside of blood vessels and in blood platelets. P-selectins are also believed to promote accumulations of white blood cells called leukocytes on blood vessel walls, leading to inflammation and leakage causing the severe pain experienced by people with sickle cell disease.

Selexys tested SelG1 in an intermediate-stage clinical trial among 198 individuals with sickle cell disease, randomized to receive high or low doses of SelG1, or a placebo. The first 2 treatments were given by intravenous infusion 14 days apart, then every 4 weeks for 50 weeks. The study team looked primarily at the rate of pain crises related to sickle cell disease over a period of 1 year, but also related indicators, such as time to first and second pain crisis, and reports of adverse events.

Researchers from the study team conducting the trial will report their findings on 4 December at the annual meeting of American Society of Hematology in San Diego. Results show trial participants receiving the higher dose of SelG1 had a 47 percent lower rate of pain crises from sickle cell disease than placebo recipients, a large enough difference to be statistically reliable. Dosage size appears to play a role, however, as individuals receiving the lower dose experienced a 33 percent lower rate of pain crisis, a difference not quite large enough for statistical reliability.

Some secondary indicators of effectiveness including median time to first or second pain crisis and annual rate of uncomplicated pain crisis were also reliably lower for higher dose participants. The most frequent adverse events reported by at least 5 percent of SelG1 recipients included joint pain, itching, vomiting, chest pain, diarrhea, fatigue, and a road traffic accident, among others. During the study, 5 deaths occurred, 3 people taking SelG1 and 2 receiving the placebo, but none of the deaths were deemed a result of the treatments.

The agreement is expected to return as much as $665 million to Selexys shareholders, from initial, acquisition, and milestone payments. In 2012, Novartis obtained an exclusive option to acquire Selexys, and the deal represents Novartis’s exercise of that option. Novartis says the clinical trial results were instrumental in the decision to acquire Selexys.

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Nanotech Material Found Having Anti-Bacterial Properties

Maria Strømme

Maria Strømme (Uppsala University)

18 November 2016. A synthetic porous material developed to deliver drugs to the skin was shown in lab tests to prevent the growth of bacteria on its surfaces. The material known as Upsalite was developed in the nanotechnology lab of materials science and engineering professor Maria Strømme at Uppsala University in Sweden, which also conducted the study appearing this week in the journal ACS Omega.

A spin-off company from the university, Disruptive Materials AB, is commercializing Upsalite, where Strømme serves on the company’s board. Disruptive Materials markets Upsalite for drug delivery and dermatology applications, as well as for moisture absorption, filtration, and chemical processing.

Upsalite is a mesoporous material, where its pores range from 2 to 50 nanometers in diameter, from a synthesized magnesium carbonate compound. Because of its porosity, Upsalite has a large surface area giving it a high moisture absorption capacity. That property makes Upsalite useful with drug compounds having poor water solubility, thus promising for delivery of these drugs. Strømme’s lab first discovered Upsalite in 2013, and since showed the material to be well-tolerated by human skin.

In the new paper, Strømme and colleagues tested Upsalite’s effects on bacteria, in this case Staphylococcus epidermidis found on human skin. For most people, Staphylococcus epidermidis, or S. epidermidis, is a benign bacteria that causes no harm when in balance with other skin microbes. The most harmful condition most people will encounter from this bacteria is acne.

For individuals having weakened or compromised immune systems, however, S. epidermidis can cause serious complications, with infections sometimes arising in hospitals from infected devices such as catheters or artificial heart valves. Treatment options for these infections are limited, however, due to the appearance of resistant strains of the bacteria.

To test Upsalite’s effects of S. epidermidis, the researchers treated powder samples of Upsalite and three comparable materials — magnesium oxide, mesoporous silica, and basic magnesium carbonate — with resazurin, a fluorescent chemical that lights up when encountering metabolic activity of living organisms, such as bacteria. The team took measures of metabolic activity of these powders when exposed to S. epidermidis bacteria, as well as a saline solution that allowed bacteria to grow unchecked as a baseline.

The results show Upsalite stopped S. epidermidis bacteria from forming and growing almost immediately, which continued for 3 hours, comparable to magnesium oxide, but better than basic magnesium carbonate and mesoporous silica. The researchers attribute this bacteriostatic effect largely to Upsalite’s alkaline properties.

These and earlier results suggest Upsalite can be developed further into products worn or applied to the skin. “These newly found bacteriostatic properties,” says Strømme in a university statement, “combined with the ability to load and release molecules, for example fragrances from the pores in the material, are highly interesting for many applications.”

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Large-Scale Cancer Gene Profiling Seen as Feasible

Stethoscope on DNA sheet

(National Heart, Lung, and Blood Institute)

18 November 2016. Genomic analysis of tumors, a growing practice for diagnosing cancer in individuals, is shown to be feasible when conducted routinely throughout a cancer care center. A study by a team from Dana-Farber Cancer Institute in Boston that appears in yesterday’s (17 November) issue of the journal JCI Insight, also points out some gaps in applying genomic profile data, when put into practice with patients.

The study, led by Laura MacConaill, scientific director of Dana-Farber’s Center for Cancer Genome Discovery, evaluated the Profile program that offers genomic profiling of tumors to all Dana-Farber patients, regardless of age, cancer type, or stage of the cancer. The research team that included participants from a number of other universities and institutions reviewed the experiences of 3,727 Dana-Farber patients, and those at Brigham and Women’s Cancer Center and Boston Children’s Hospital. who agreed to take part in Profile during 2013, the program’s first year. The program to date analyzed about 15,000 tumors.

Data from the study show results of the institution’s OncoPanel platform that analyzes some 300 genes in parallel from each tumor sample. The analysis seeks to identify genetic alterations in tumors, as well as therapies targeted to address those alterations. Dana-Farber stores patient genomic profiles in a database linked to other clinical data that the institute says provides a rich resource for precision cancer medical research and practice.

Where OncoPanel could make a genomic profile of a tumor, nearly all (96%) returned results of some kind. Nearly three-quarters (73%) showed an alteration providing new insights or considered actionable. In 19 percent of the cases, the analysis pointed to currently approved cancer treatments. In the remaining cases, the analysis could point patients to clinical trials of experimental drugs or currently approved drugs being tested for other disorders. The paper also notes cases where Profile identified genomic alterations that responded to targeted therapies, and gave examples where the analysis resulted in a different diagnosis or treatment strategy.

MacConaill and colleagues, however, also identified a number of obstacles that need to be addressed by Dana-Farber and other institutions considering a program like Profile. Among all of the samples collected, only about half had sufficient tumor material available for analysis. In addition, only in about 10 percent of the cases did genomic profiles result in actual changes in patient care.  The authors cite a number of factors in cancer care that must be coordinated with tumor profiling to make optimum use of the data, including the timing of the profile during the course of the patient’s disease progression, and access to targeted drugs and clinical trials.

Related to timing of the analysis is the amount of time needed to return results of tumor profiling. The authors report OncoPanel provided results in an average of 5.3 weeks, which Dana-Farber says is now down to 3 weeks. Genomic profiling of tumors is also expensive, “and this cost,” MacConaill notes in a Dana-Farber statement, “has been borne by our institutions.”

As reported on a number of occasions in Science & Enterprise, Foundation Medicine — a company spun off from Dana-Farber Cancer Institute, the Broad Institute, Harvard Medical School and MIT — commercializes a technology similar to Profile that examines and reveals genes altered in human cancers, and offers potential targets for therapies. Two of the paper’s authors, Matthew Meyerson and Levi Garraway, are co-founders of Foundation Medicine and serve as advisers to the company.

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Trial Shows Antibody Reduces Days with Migraines

Person with migraine

(R. Nial Bradshaw, Flickr)

17 November 2016. First results from a late-stage clinical trial shows a synthetic antibody reduces the number of days with migraines per month among people with episodic or infrequent migraines. The synthetic antibody, called erenumab, is jointly developed by the pharmaceutical companies Amgen and Novartis that reported the results today.

Migraine is a neurological syndrome causing severe headaches along with nausea, vomiting, and extreme sensitivity to light and sound. In some cases, migraines are preceded by warning episodes called aura including flashes of light, blind spots, or tingling in arms and legs. The web site estimates 37 million people in the U.S. suffer from migraines, and cites World Health Organization data indicating migraines affect 18 percent of American women and 7 percent of men.

Migraines experienced 14 days a month or fewer are called episodic migraines, while chronic migraines are experienced 15 days a month or more. A study published 2013, however, suggests that individuals with episodic migraines, about 90 percent of those with migraines, who do not get adequate treatment are 3 times more likely to progress to chronic migraines.

Erenumab is designed to prevent migraines by limiting receptors for calcitonin gene-related peptide. This peptide and its receptors are expressed in a number of different cells throughout the central and peripheral nervous systems, and regulate inflammation and pain arising from the stimulation of nerve cells. Erenumab acts by inhibiting the peptide’s receptors, not the peptide itself, and is administered monthly by injections under the skin.

The clinical trial enrolled 955 participants with a history of episodic migraine at 129 sites in North America and Europe. Participants were randomly assigned to receive injections of erenumab, at doses of 70 or 140 milligrams, or a placebo once a month for 6 months. Study teams looked primarily at the average number of days per month participants experienced migraines, compared to a baseline measure taken before the injections.

The results show individuals receiving erenumab experienced fewer days per month with migraines compared to those receiving the placebo. Before the trial, participants average 8.3 days per month with migraines. Individuals receiving erenumab reported 3.2 and 3.7 fewer days per month of migraines, for injections of 70 and 140 milligrams respectively. Placebo recipients, however, experienced 1.8 fewer migraine days per month, a large enough difference from erenumab recipients to be statistically reliable.

Occurrences of adverse effects, say the companies, were similar for participants receiving erenumab or the placebo: common cold symptoms, inflamed sinuses, and upper respiratory tract infections. The adverse effects in this trial were much like those reported in other intermediate and late-stage trials of erenumab.

“The results of this study are important,” says Amgen vice-president Sean Harper in a company statement, “because they confirm the results from our previous studies and add to our body of research in episodic migraine. We look forward to working with regulatory authorities to pursue approval of erenumab and making this novel migraine prevention treatment available to patients and physicians.”

Amgen originally developed erenumab, but in August 2015 joined forces with Novartis to co-develop treatments for neurological disorders, including erenumab, as well as therapies for Alzheimer’s disease. Under their agreement, Amgen retains commercialization rights in the U.S., Canada and Japan for migraine drugs, and Novartis has commercialization rights in Europe and rest of world.

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Blood Stem Cell Start-Up Launches, Earns $48.5M Funding

David Scadden

David Scadden (B.D. Colen, Harvard University)

17 November 2016. A new enterprise, spun-off from Harvard University stem cell labs, aims to make stem cell transplants from bone marrow safer and more reliable. The new company, Magenta Therapeutics in Cambridge, Massachusetts, is also raising $48.5 million in its first venture financing round.

Magenta Therapeutics is licensing discoveries from the lab of David Scadden, professor of medicine at Harvard University and Massachusetts General Hospital, affiliated with Harvard. Scadden’s research teams study stem cells in bone marrow that transform into red blood cells, known as hematopoietic stem cells, particularly processes that disrupt development of healthy blood cells leading to leukemia and other blood disorders.

Bone marrow transplants to treat blood-related cancers today are risky procedures, first requiring elimination of the patient’s own stem cells before transplanting stem cells from donors. To take out the patient’s own stem cells often means techniques such as chemotherapy and radiation, which have adverse side effects, including damage to the immune system raising the risk of infections.

In June 2016, Scadden and colleagues reported on an alternative transplant process with engineered antibodies, rather than chemotherapy or radiation, that specifically targets receptors found only on blood-forming stem cells in lab mice induced with sickle-cell anemia. The antibodies succeeded in removing nearly all of the blood-forming stem cells, making it possible to transplant more than 90 percent of a donor’s stem cells, and correcting the sickle-cell anemia. The process also spared bone marrow and subsequent white blood cells from damage that maintained immune systems in the test mice.

Magenta Therapeutics is developing new techniques based on Scadden’s research to make bone marrow transplants safer for patients. The company plans to adapt the antibody-based process, but also employ faster and more efficient stem cell harvesting techniques from donors with growth factor proteins, and more productive stem cell culturing methods in the lab, to produce the quantity of cells needed for transplant. Scadden chairs the company’s scientific advisory committee.

Magenta incubated for the past year with life science investment companies Third Rock Ventures and Atlas Venture, that led the company’s first funding round providing $48.5 million. Jason Gardner, co-founder and CEO of Magenta, says in a company statement the technology can be applied to a variety of blood and immune-system disorders, including early stage cancers and autoimmune diseases, such as multiple sclerosis and scleroderma.

“There has been terrific innovation in stem cell science recently, and it is time to bring this forward to patients,” Gardner adds. “Our ultimate goal is to reboot the blood and immune systems safely to make a significant impact on the overall quality of life for a much broader group of patients that can benefit from transplant.”

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Infusion-to-Oral Drug Delivery Company Acquired

Pills in blister packs

(PublicDomainPictures, Pixabay)

16 November 2016. A company with a technology for converting drugs given by intravenous infusion into pills and capsules was acquired Grünenthal Group, a provider of pain medications. Financial terms were not disclosed of the acquisition of Thar Pharmaceuticals in Pittsburgh by Grünenthal Group in Aachen, Germany.

Thar Pharmaceuticals provides a process for converting approved drugs given by intravenous infusion into oral drugs like tablets and capsules, making the medication easier to take and more convenient for patients. Thar says its technology, called Enhance, changes a drug compound’s physical characteristics, such as solubility, permeability, and melting point, as well as bioavailability, onset, and absorption properties in the body. However, interactions between the drug and proteins, and effects of the drugs on molecular functions are not affected.

Grünenthal’s main corporate focus is pain medications, and Grünenthal says Thar’s lead product will fit into its overall portfolio. The product, code-named T121 by Thar Pharma, is a reformulation of zoledronic acid, marketed as Zometa by Novartis, given as an IV infusion to treat complications of multiple myeloma causing pain or fractures in bones and hypercalcemia, or high blood calcium levels. Novartis also offers Reclast, a version of zoledronic acid to prevent or treat osteoporosis, given as an IV infusion.

Thar Pharma is developing T121 as a treatment for complex regional pain syndrome, or CRPS, a chronic pain condition in the limbs often resulting from injuries to affected limbs. CRPS is believed to be caused by a malfunctioning or damaged peripheral nervous system, sending nerve signals to and from the brain and spinal cord. Pain from CRPS is prolonged or constant, and can become severe. Affected limbs can also become discolored or swollen.

According to Thar Pharma, T121 received orphan drug designation from Food and Drug Administration as a CRPS treatment. In addition to preclinical studies, the company carried out an early-stage clinical trial of T121 among healthy postmenopausal women, testing the drug for safety and actions in the body, at various dosage levels, Thar Pharma is planning late-stage clinical trials of the drug as well.

Another pain drug, code-named T109 by Thar Pharma, is designed as a fast-acting non-narcotic therapy for acute pain, such as back or post-operative pain, now in preclinical tests. The company filed for an initial public offering of common stock in August 2016, raising $50 million.

Thar Pharma’s products and technology are expected to fit in well at Grünenthal Group. “We are expanding our development portfolio for treating patients with orphan, disabling diseases, says Grünenthal’s chief scientist Klaus-Dieter Langner in a company statement. “For CRPS, an often debilitating, extremely painful syndrome, truly efficacious treatments are lacking today.”

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Laser-Immunotherapy Now in Cancer Clinical Trial

Kerstin Stenson with laser

Kerstin Stenson with the laser optic leads used in photoimmunotherapy (Rush University Medical Center)

16 November 2016. A new type of cancer treatment that precisely attacks tumors with synthetic antibodies activated by laser beams is being tested in an early-stage clinical trial. The treatment known as photoimmunotherapy is developed by Aspyrian Therapeutics Inc. in San Diego, with Rush University Medical Center in Chicago as one of the 5 trial sites.

In photoimmunotherapy, a light-sensitive chemical known as a photosensitizer is combined with synthetic antibody that seeks out and binds only to specific biomarkers expressed by the tumor cells. The combination of photosensitizer and antibody is infused in the patient, where it’s taken up by the tumors. After 24 to 72 hours, surgeons attach thin optical fibers to the tumors and send near-infrared laser beams to the tumors marked by the photosensitizers. Energy from the lasers expands the targeted tumor cells while weakening the cell walls, causing the tumor cells to burst and destroy.

The clinical trial is an early-stage test of photoimmunotherapy’s safety, as well as to determine the maximum safe dose, among 24 individuals with recurrent and head and neck cancer considered unresponsive to other treatments. The study is evaluating RM-1929 code-named by Aspyrian Therapeutics that licensed the technology from National Cancer Institute, targeting epidermal growth factor receptors often expressed on the surface of tumors in head and neck cancers.

RM-1929 contains a combination of cetuximab, an engineered antibody approved by FDA to treat head and neck cancers, and a fluorescent dye designed to combine with antibodies for photoimmunotherapy. Cetuximab is marketed as Erbitux by Eli Lilly and Co. Aspyrian says preclinical studies show RM-1929 causes rapid destruction of human cancer cells grafted onto mice, which also have longer cancer-free survival than conventional treatments.

The clinical trial is looking primarily at the safety of photoimmunotherapy and the maximum dose of the drug and lasers in the treatments in the participants, who are tracked for a month. Of particular concern are effects of the treatments on the skin. Earlier forms of light-sensitive treatments known as photodynamic therapy reported cases of skin burns, including sunburn from exposure to direct sunlight. The study teams are also tracking chemical actions of the drugs in the body, responses or reductions of tumors, overall survival time, and progression-free survival time.

Kerstin Stenson, professor of otolaryngology and director of the study at at Rush University Medical Center, says in a university statement, “This treatment is so unique and promising because its cancer cell-killing power is so selective and immediate.” Stenson adds that “Almost immediately, you can see the tumor start dying. It turns white and melts away.” And because RM-1929 remains inert unless activated by the laser, she notes, it causes almost no damage to surrounding cells.

In addition to Rush University, the trial is recruiting participants at Centura Health Research Center in Denver, Virginia Piper Cancer Institute in Minneapolis, University of Oklahoma Stephenson Cancer Center in Oklahoma City, Thomas Jefferson University in Philadelphia, and MD Anderson Cancer Center in Houston.

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Fertility Device Company Gains $9.7M in Early Funds

Ava bracelet and app

Ava bracelet and smartphone app (Ava Science Inc.)

15 November 2016. A two year-old medical device enterprise making a fertility tracker for women is raising $9.7 million in its first venture finance round. The new funds collected by Ava Science Inc., based in Zurich and San Francisco, supplement the company’s original $2.6 million in seed funding raised in November 2015.

Ava is developing a tracking band worn like a bracelet on the wrist that identifies the fertile days in a woman’s cycle. The device is worn while sleeping at night, when sensors collect data for 9 physiological factors associated with the onset of ovulation and that correlate with the rise in reproductive hormones estradiol and progesterone. Those variables include resting pulse rate, skin temperature, heat loss while sleeping, heart rate variability, sleep quality and quantity, amount of movement while sleeping, breathing rate, perfusion of blood to tissues, and bioimpedence for data on skin hydration and perspiration.

The data are collected and synced to a smartphone app, which calculates the fertile window in a woman’s cycle, averaging 5.3 days that include the days leading up to ovulation and the ovulation day itself. Using the bracelet, notes Ava Science, avoids guess work, urine samples, ovulation tests strips, and body temperature measurements. The company says the Ava system qualifies as an FDA class 1 or low-risk medical device.

Ava Science says data for the algorithms in the device were collected in a year-long clinical study led by Brigitte Leeners, a reproductive endocrinologist at University Hospital in Zurich and scientific adviser to Ava. The company says findings from the study, reported at a professional meeting in Switzerland in June, show results from the Ava system matched 89 percent of the time with independent urine samples.

The company reported in September the first pregnancy in the U.S. attributed to the Ava system, since it began shipping this summer. Ava Science says a second clinical trial of the device is underway.

Polytech Ecosystem Ventures, based in Lausanne, Switzerland and Saratoga in California’s Silicon Valley, led the financing round. Blue Ocean Ventures and Global Sources, with existing seed investors Swisscom and ZKB, and other unidentified investors, took part in the round. Ava Science president and co-founder Lea von Bidder says in a company statement that Ava will use the funds for further product development, scale-up production of Ava bracelets to meet consumer demand, and to advance the company’s research.

“With these funds,” adds co-founder and CEO Pascal Koenig, “we’ll be able to further accelerate our traction, and continue on our mission of developing technology that improves the reproductive health of women worldwide.”

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Antibody Leads to Sharp Triglyceride, Cholesterol Drop

Heart check

(Gerd Altmann, Pixabay)

15 November 2016. An experimental synthetic antibody was shown in a clinical trial to markedly reduce triglycerides and the types of cholesterol that contribute to heart disease. Findings from the trial testing evinacumab, developed by the biopharmaceutical company Regeneron Pharmaceuticals, were presented yesterday by University of Pennsylvania medical professor Richard Dunbar at a scientific meeting of American Heart Association in New Orleans.

Triglycerides are a type of lipid, or natural oil, usually stored in the body’s fat cells to help provide energy, but when the intake of calories exceeds the body’s ability to store fat in cells, triglycerides can be released into the blood stream. High levels of triglycerides in the blood contribute to atherosclerosis, the hardening of arteries or thickening of artery walls, that increases the risk of heart disease and stroke, as well as pancreatitis, or inflammation of the pancreas. While different from cholesterol, triglycerides circulate in the blood with the help of lipoproteins, proteins that transport lipids, much like cholesterol.

Regeneron is developing evinacumab as a treatment for homozygous familial hypercholesterolemia, a rare inherited condition where the liver cannot naturally process lipoproteins, leading to abnormally high levels of cholesterol and triglycerides, often resulting in early occurrences of heart disease and stroke.  Evinacumab is an injected therapy designed as an antibody that blocks Angiopoietin-like 3, or Angptl3, a protein limiting the activity of enzymes that break down and metabolize fats in the liver, thus stimulating production of triglycerides and cholesterol.

The clinical trial was an early-stage test of evinacumab’s safety and chemical activity in the body. The study team looked primarily for signs of adverse effects among individuals, but also measured triglyceride and cholesterol levels in the blood. Participants in the trial were randomly assigned to receive 1 of 6 different dosage levels of evinacumab, or a placebo.

At the meeting, Dunbar and colleagues reported the first results of the trial from 41 participants, 32 receiving evinacumab and 9 getting the placebo. Results show sharp drops in triglyceride levels among evinacumab recipients, as high as 64 to 73 percent for those receiving the highest doses, well beyond the reductions seen in current treatments. “Current medications such as fibrates or prescription fish oils effectively lower triglycerides,” says Dunbar in a university statement, “but leave much to be desired, each only lowering levels by 20 to 50 percent.”

Results also show lowered cholesterol levels, with the higher the dose of evinacumab, the greater the reductions. But reductions were reported in both the high-density lipoprotein or HDL cholesterol — considered good cholesterol — as well as low-density lipoprotein, or LDL, cholesterol, the kind that contributes to atherosclerosis and heart disease. Reductions in both kinds of cholesterol are considered consistent with limits on Angptl3 proteins.

Participants receiving evinacumab tolerated the treatments, with only headaches reported as adverse effects among 7 participants. The results, say the researchers, provide enough evidence to move forward validating evinacumab as a treatment for people with high triglyceride levels. “In the short term,” notes Dunbar, “profoundly lowering triglycerides may render hospital admissions less frequent in patients prone to pancreatitis, while long term, lowering triglycerides and associated cholesterol could also help reduce the risk of certain heart disease.”

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