Credit Suisse analyst Matt Miksic raised his price target for Edwards Lifesciences to $221 from $189, while reiterating an Outperform rating on the shares. his bullish outlook is based in part on his view that the company does not yet reflect the low-risk expansion of the TAVR market, and investors appear likely to look past Q1 results toward evidence over the next few quarters of increased TAVR growth following the LR data.
Monday, April 15, 2019
Active lifestyle key to repairing spinal cord injuries
Leading an active lifestyle could help repair and regenerate damaged nerves of the spinal cord after an injury, according to new research. Early stage study results were published in the latest issue of the journal Science Translational Medicine.
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Using mice and rat models, the international research team, led by scientists from Imperial College London, discovered that spinal cord injuries could be repaired after being damaged through exercise.
The team provided the lab animals with an exercise wheel and other activity toys along with mates. The test animals were also given extra space to roam around in.
In response, the animals became more active, and the scientists noticed that the damaged nerve cells within their spinal cords began to regenerate. They also used a drug to mimic the effects of an active lifestyle and found that this too could regenerate the damaged nerve cells.
‘It’s almost as if the nerve cells are being primed for regeneration’
The researchers agree that their work is still in preliminary stages but opens up a “realistic pathway” towards regeneration and repair of the damaged spinal cord tissues. The hope is that these results could soon be translatable to human patients as well.
Anecdotal evidence suggests that people with an active lifestyle may recover to a greater degree after spinal cord injury than those who are less active.Our studies support these findings.From what we have seen it’s almost as if the nerve cells are being ‘primed’ for regeneration and growth, which add to this enhanced recovery.”Professor Simone Di Giovanni, Lead Author
Exercising leads to ‘changes in gene expression’
The researchers explain that the spinal cord contains nerve fibres that have long axons or tails that stretch down the spine and when damaged can be severed. These then fail to carry signals from the body to the brain and vice versa. The damage in most cases is irreversible with most sufferers left paralyzed.
Professor Di Giovanni explained, “We discovered that environmental enrichment such as housing mice in a larger cage than usual, with more mice in it, more toys, tunnels, swings, running wheels etc. increases the activity of neurons. This leads to changes in gene expression which make the nerve more likely to regenerate. Essentially, by increasing the activity of neurons that sense enriched environmental stimuli we have been able to promote the regenerative potential of nerves after spinal cord injury.”
‘Further studies are needed’ before the treatment can be extended to humans
“In principle, this kind of treatment is not very far from being tested in the clinic. Further studies are needed to show the drug is safe in humans, before it could be trialled. But in future it could potentially be combined with neurorehabilitation in clinical trials,” Di Giovanni concluded.
Fibrocell up on Castle Creek collaboration
Nano cap Fibrocell Science (NASDAQ:FCSC) is up 51% premarket on robust volume in response to its collaboration with Castle Creek Pharmaceuticals to develop and commercialize gene therapy candidate FCX-007 for recessive dystrophic epidermolysis bullosa, a rare inherited disorder characterized by fragile blistering skin.
Under the terms of the deal, Castle Creek will have an exclusive license to commercialize FCX-007 in the U.S. and will be responsible for all development and manufacturing costs up to $20M prior to the filing of a marketing application. If development spending exceeds $20M, Fibrocell will cover 30% of the excess expenses. Castle Creek will be responsible for all commercialization activities and costs while Fibrocell will maintain clinical development, regulatory interactions and manufacturing under a future supply agreement with Castle Creek.
Fibrocell will receive $7.5M upfront, $2.5M when the first patient is enrolled in a Phase 3 study, $30M when the FDA approves the marketing application and market launch is ready, up to $75M in sales-based milestones, an additional $50M if sales reach $750M and 30% of gross profits.
Fibrocell will retain sole ownership of the Rare Pediatric Disease Priority Review Voucher that it will receive on the FDA nod for FCX-007.
A Phase 3 clinical trial, DEFI-RDEB, should launch this quarter.
Arrowhead Gets FDA Clearance to Begin Phase 2/3 Study for Liver Disease Med
Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Arrowhead intends to initiate the adaptive design, Phase 2/3 study of ARO-AAT in patients with AATD associated liver disease at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe, pending regulatory submission and review. The proposed primary objectives are to evaluate safety and pharmacodynamic dose response, and to evaluate efficacy, defined as an improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis based on Ishak score on end of study biopsy. The company plans to provide additional study details following its initiation.
Zymeworks launches mid-stage trial for ZW25
Zymeworks (NYSE:ZYME) has initiated its global multicenter Phase 2 clinical trial evaluating ZW25 in combination with standard of care chemotherapy for the first-line treatment of HER2-positive metastatic gastric, gastroesophageal junction, and esophageal adenocarcinomas.
The Phase 2 trial is a two-part open-label study. The primary objectives of this trial are to confirm the safety, tolerability, and anti-tumor activity of ZW25 in combination with global standard of care regimens for gastroesophageal adenocarcinoma.
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