Search This Blog

Tuesday, December 3, 2019

Radiology Study Looks at Motorized Scooter Injuries

More than half of people who received X-rays or CT scans after electric scooter accidents were found to have injuries, most commonly to the upper extremities, according to a new study presented today at the annual meeting of the Radiological Society of North America (RSNA). Researchers said the findings underscore the need for more public education on the use of these scooters.
Dockless electric motorized rental scooters, also known as e-scooters, have exploded in popularity in recent years. E-scooters are familiar sights in urban areas and on college campuses, where users value them as an inexpensive, convenient and less strenuous alternative to bicycles.
The rapid growth of rental e-scooters in cities across the U.S. has sparked concerns, as hospital emergency departments have reported a growing number of injuries associated with the vehicles. Earlier this year, the Centers for Disease Control and Prevention (CDC), in association with Austin Public Health, released a study assessing the potential public health impact of e-scooter use.
“E-scooters have a narrow platform, can travel up to 15 to 20 miles per hour and require a level of coordination and skill that is often not native to many users,” said study co-author Aiza Ashraf, M.D., diagnostic radiology resident at the Indiana University School of Medicine in Indianapolis. “Whereas physical effort is required to get a bicycle up to speed, e-scooters are self-powering.”
Imaging exams for e-scooter accidents spiked at Indiana University Health System after the scooters were legalized in Indianapolis in September 2018. To find out more about this trend, Dr. Ashraf, Mohsin Mukhtar, M.D., and colleagues at Indiana University School of Medicine, studied electronic medical records and radiology archives of people ages 18 and older who had exams ordered for scooter-related injuries over a five-year period.
The researchers identified 69 exams performed on 36 unique Emergency Department patients with involvement of an e-scooter. There were 19 males and 17 females included in the study, and two-thirds were in the 18-30 age range. X-rays of the extremities and CT of the head, face and cervical spine were the most common exams ordered.
Nineteen of the 36 patients were found to have a radiographically apparent injury. The most common injuries involved the upper extremities, particularly the wrist. There were six cases of distal radial fractures, or fracture of the forearm bone close to the wrist, making it the most common injury in the study group. Soft tissue injuries of the head, face, wrist and ankle, were present in five cases.
“We believe that many users are not fully aware of the potential significant injuries that may occur with e-scooter use,” Dr. Mukhtar said. “Raising awareness and doing further research on this topic could inform future policy.”
The study findings highlight the importance of protective equipment such as helmets and hand/wrist guards, researchers said, along with the potential dangers of riding while under the influence of intoxicating substances. In addition, the study suggests that communities should consider imposing speed limits on e-scooter users.
“Limiting e-scooter speed could reduce the overall incidence and severity of injuries in the event of a fall or collision,” Dr. Ashraf said. “And since these e-scooters could be viewed as a potential public health hazard, we would recommend public education on the use of these devices.”
The study also found a lack of adequate and specific documentation of scooter type in the emergency medical records. More than 200 instances found in the search may have been e-scooter-related, but lack of documentation in the records prevented the researchers from making a definitive link.
“A robust ability for more systematic data collection and analysis, for example, performed as a multi-institutional public health study, may be of benefit,” Dr. Mukhtar said.
Co-authors are Mark S. Frank, M.D., and Scott D. Steenburg, M.D.

Mission-AbbVie Alzheimer’s, Parkinson’s Collaboration Reaches Milestone

Mission Therapeutics (“Mission”), a drug discovery and development company focused on selectively inhibiting deubiquitylating enzymes (DUBs), and AbbVie (“AbbVie”)(NYSE: ABBV), a global, research-driven biopharmaceutical company, today announced the identification of several DUBs as potential drug targets in their Alzheimer’s and Parkinson’s R&D collaboration.
AbbVie has nominated the panel of DUBs that will be progressed for further characterization and screening activities. This is the first major milestone of the Companies’ DUB research and preclinical development collaboration in neurodegenerative diseases.
Under the terms of the collaboration, announced in November 2018, Mission and AbbVie are working together to identify specific DUBs and discover suitable inhibitor compounds for the treatment of Alzheimer’s and Parkinson’s diseases. AbbVie then has the option to gain exclusive rights to develop and commercialize DUB inhibitors against up to four selected targets. Mission is eligible to receive success-based milestone payments and royalty payments for each commercialized product.

Astellas New Data on XOSPATA in Acute Myeloid Leukemia at Hematology Meet

Emerging mutations in patients with treatment resistance, from Phase 3 ADMIRAL study, will be focus of oral presentation
Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., “Astellas”) today announced the presentation of new data in acute myeloid leukemia (AML) at the 61st American Society of Hematology (ASH) Annual Meeting, taking place Dec. 7-10 in Orlando, Fla.

Seven abstracts sponsored by Astellas focus on patients with relapsed (disease that has returned) or refractory (resistant to treatment) AML with a FLT3 mutation (FLT3mut+). The abstracts include new findings from the Phase 3 ADMIRAL trial – an oral presentation on emerging mutations in patients who develop resistance after an initial response to XOSPATA and two poster presentations focused on patient-reported outcomes – as well as data on cost-effectiveness, FLT3 testing and treatment patterns, and venetoclax combination therapy.

Astellas to buy Audentes for $3 billion in high-priced gene therapy bet

Japan’s Astellas Pharma Inc is buying U.S. drugmaker Audentes Therapeutics Inc for about $3 billion in cash, in a high-priced push to make genetic medicines a key area of growth.

Gene therapies are one of the hottest areas of drug research and Astellas, Japan’s second-largest drugmaker by sales, is offering $60 per share for San Francisco-based Audentes, a 110% premium to its closing price on Monday.
Citi analyst Hidemaru Yamaguchi said that while the deal looked expensive, it was a positive move for Astellas as Audentes had “cutting-edge gene therapy modalities”.
“We thought it was only a matter of time before Astellas entered the gene therapy market,” he wrote in a note for clients, adding it had already licensed development rights for a domestic gene therapy in Amyotrophic lateral sclerosis (ALS).
Gene therapies aim to cure diseases by replacing the missing or mutated version of a gene found in a patient’s cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.
“As a result of this acquisition, Astellas will obtain not only Audentes programs but also it’s proprietary manufacturing knowhow in gene therapy,” Astellas CFO Naoki Okamura told a briefing in Tokyo. “Internal manufacturing capability is a great strength for companies with multiple programs under development.”
Genetic drugs will become a fifth primary focus for Astellas, Okamura said, joining existing business lines in regenerative, immuno-oncology, immunotherapy, and neuro-muscular medicine.
The acquisition marks the second biggest on record for Astellas after its 2010 purchase of OSI Pharmaceuticals Inc for$3.8 billion, according to Refinitiv data. Astellas expects the deal, which is subject to regulatory approval including U.S. antitrust clearance, to close in the first quarter of 2020.
Audentes’ investigational drug, AT132, is being developed to treat a rare genetic neuromuscular disorder which results in extreme muscle weakness, respiratory failure and in some cases early death.
Astellas said the companies plan to seek FDA approval for AT312 in mid-2020. The drug has shown promising results in the treatment of X-linked myotubular myopathy (XLMTM) seen mainly in male infants, it said.
Only about 40 boys are born in the United States with the condition each year, so that would yield just $80 million in revenue, said Jefferies analyst Stephen Barker, assuming a maximum price tag for the treatment.
“The $3 billion acquisition price is therefore more likely to be mainly predicated on the firm’s technology platform and manufacturing capabilities,” Barker wrote in a note.
The deal also marks the latest consolidation in the industry which saw Japan’s Takeda Pharmaceutical Co Ltd acquire Britain’s Shire for $59 billion.
Shares in Astellas fell 1.1% on Tuesday in Tokyo, underperforming a 0.6% decline in the broader market <.N225>.
The stock’s decline was “the natural reaction”, said Credit Suisse analyst Fumiyoshi Sakai.
“This is a difficult deal to digest because it deals with gene therapy,” Sakai said. “They have to be very square with the investment community about what they’re buying.”
Morgan Stanley & Co LLC, is Astellas’ financial adviser while Covington & Burling LLP is the company’s legal counsel.
Centerview Partners LLC is acting as financial adviser to Audentes and Fenwick & West LLP is its legal counsel.

AstraZeneca Sells Seroquel Rights to Cheplapharm

AstraZeneca PLC (AZN.LN) said Tuesday that it has agreed to sell the commercial rights to the drugs Seroquel and Seroquel XR in the U.S. and Canada to Cheplapharm Arzneimittel GmbH for an upfront payment of $35 million.
The U.K. pharmaceutical company said that as well as the upfront payment, there may also be future sales-contingent payments of up to $6 million.
The company said the agreement doesn’t impact the company’s financial guidance for 2019 and that the agreement became effective upon signing.
“This divestment supports our strategy of reducing the number of mature medicines to enable reinvestment in our main therapy areas,” said Ruud Dobber, executive vice president of the biopharmaceuticals business unit.

Monday, December 2, 2019

New treatment for brain tumors uses electrospun fiber

A novel engineering process can deliver a safe and effective dose of medicine for brain tumors without exposing patients to toxic side effects from traditional chemotherapy.
University of Cincinnati professor Andrew Steckl, working with researchers from Johns Hopkins University, developed a new treatment for glioblastoma multiforme, or GBM, an aggressive form of brain cancer. Steckl’s Nanoelectronics Laboratory applied an industrial fabrication process called coaxial electrospinning to form drug-containing membranes.
The treatment is implanted directly into the part of the brain where the tumor is surgically removed.
The study was published in Nature Scientific Reports.
“Chemotherapy essentially is whole-body treatment. The treatment has to get through the blood-brain barrier, which means the whole-body dose you get must be much higher,” Steckl said. “This can be dangerous and have toxic side-effects.”
Steckl is an Ohio Eminent Scholar and professor of electrical engineering in UC’s College of Engineering and Applied Science.
Coaxial electrospinning combines two or more materials into a fine fiber composed of a core of one material surrounded by a sheath of another. This fabrication process allows researchers to take advantage of the unique properties of each material to deliver a potent dose of medicine immediately or over time.
“By selecting the base materials of the fiber and the thickness of the sheath, we can control the rate at which these drugs are released,” Steckl said.
The electrospun fibers can rapidly release one drug for short-term treatment such as pain relief or antibiotics while an additional drug or drugs such as chemotherapy is released over a longer period, he said.
“We can produce a very sophisticated drug-release profile,” Steckl said.
The breakthrough is a continuation of work conducted by research partners and co-authors Dr. Henry Brem and Betty Tyler at Johns Hopkins University, who in 2003 developed a locally administered wafer treatment for brain tumors called Gliadel.
Unlike previous treatments, electrospun fibers provide a more uniform dose over time, said UC research associate Daewoo Han, the study’s lead author.
“For the current treatment, most drugs release within a week, but our discs presented the release for up to 150 days,” he said.
Glioblastoma multiforme is a common and extremely aggressive brain cancer and is responsible for more than half of all primary brain tumors, according to the American Cancer Society. Each year more than 240,000 people around the world die from brain cancer.
The electrospun fiber created for the study provided a tablet-like disk that increased the amount of medicine that could be applied, lowered the initial burst release and enhanced the sustainability of the drug release over time, the study found.
Chemotherapy using electrospun fiber improved survival rates in three separate animal trials that examined safety, toxicity, membrane degradation and efficacy.
“This represents a promising evolution for the current treatment of GBM,” the study concluded.
While this study used a single drug, researchers noted that one advantage of electrospinning is the ability to dispense multiple drugs sequentially over a long-term release. The latest cancer treatments rely on a multiple-drug approach to prevent drug resistance and improve efficacy.
Steckl said the study holds promise for treatments of other types of cancer.
“Looking ahead, we are planning to investigate ‘cocktail’ therapy where multiple drugs for the combined treatment of difficult cancers are incorporated and released either simultaneously or sequentially from our fiber membranes,” Steckl said.

Aslan’s Rally Grows After Positive Readout From Eczema Drug Study

After soaring 81.25% Wednesday and augmenting the gains by an incremental 65% Friday, ASLAN PHARMACEU/ADR ASLN 36.13% shares were  accelerating further Monday morning.
The stock has seen  strong upward momentum since late November.

Atopic Dermatitis Drug Data

Singapore-based Aslan, which has its ADSs listed on the Nasdaq, announced positive data Monday from the lowest-dose cohort of its ongoing multiple dose study of ASLAN004, which is being evaluated for moderate-to-severe atopic dermatitis.

ASLAN004 is a fully human monoclonal antibody that binds to the IL-13 receptor α1 subunit, which in turn blocks signalling of two pro-inflammatory cytokines, IL-4 and IL-14. These two cytokines are responsible for the triggering of symptoms of atopic dermatitis such as redness and itching of skin, according to the company.
The Eczema Area and Severity Index scores of of three patients who completed one month of dosing were reduced by 85%, 70% and 59% from the baseline, with the EASI score continuing to drop, Aslan said.
The company said it expects maximum efficacy at six to eight weeks.
The investigational compound was well-tolerated, with no serious adverse events or treatment discontinuations observed, according to the company.

Aslan’s Target Market

Atopic dermatitis, a severe form of eczema, affects more than 200 million people worldwide, severely impairing their quality of life.
About one-third of adult atopic dermatitis are characterized as moderate to severe, and there are only limited treatment options, according to Aslan.
“We are pleased to report encouraging preliminary data from this study of ASLAN004. Whilst the data remains early, we had not anticipated to observe such pronounced improvements in patients enrolled into the lowest dose cohort,” said Dr. Mark McHale, the biotech’s head of R&D.
The data monitoring committee will meet in late December, after which Aslan said it plans to open the second dose cohort. The company expects to release interim results in early 2020, with the completion of the study expected in the second half of 2020.