Avrobio announced clinical data and patient updates from the investigator-sponsored Phase 1 study and the Avrobio-sponsored Phase 2 clinical trial of AVR-RD-01. AVR-RD-01 is an ex vivo lentiviral gene therapy being investigated in Fabry disease. Designed to be a one-time therapy, it works by inserting the GLA gene that encodes functional a-galactosidase A to enable continuous AGA production and distribution to tissues and organs. The investigator-sponsored Phase 1 study is designed to assess the safety of AVR-RD-01 in up to six patients with Fabry disease who have been treated with standard of care enzyme replacement therapy, or ERT, for at least six months prior to receiving AVR-RD-01. The Phase 1 study is conducted by the FACTs team in Canada. As a Phase 1 study, the primary endpoint of this study is safety. Continued patient enrollment is planned and the protocol has been amended to allow the FACTs team, at their discretion, to discontinue ERT six months after treatment with AVR-RD-01. Preliminary safety data from these three subjects indicate AVR-RD-01 was generally well tolerated and no serious adverse events related to AVR-RD-01 have been reported. Preliminary safety data from patient FAB-201-1 indicate that AVR-RD-01 was generally well tolerated. Enrollment in the FAB-201 Study is ongoing.
Monday, October 1, 2018
Viking Therapeutics price target raised to $31 from $28 at H.C. Wainwright
H.C. Wainwright analyst Joseph Pantginis raised his price target for Viking Therapeutics to $31 after the company and investigators presented updated VK5211 data in an oral plenary presentation at the American Society of Bone and Mineral Research. The key takeaways from the VK5211 Phase 2 data in hip fracture patients include: VK5211 produces significant muscle growth in hip fracture patients, VK5211-treated patients experienced overall weight gain, the six-minute walk test demonstrated a trend favoring the treatment arms and the tolerability profile in elderly patients was encouraging with no treatment-emergent adverse events, Pantginis tells investors in a research note. The analyst keeps a Buy rating on Viking Therapeutics.
Veeva downgraded to Hold from Buy at Canaccord
Canaccord analyst David Hynes Jr. downgraded Veeva Systems to Hold from Buy, telling investors in a research note that he is stepping to the sidelines “for a bit.” The analyst says his downgrade is about valuation, and notes that he has “zero qualms” about Veeva’s opportunity, execution or management team. Most likely, the analyst says Veeva will probably “grind along sideways” for a period of time while the underlying business and metrics catch up with the stock’s valuation and raised his price target to $105 from $98.
Antares Pharma announces FDA approval of Xyosted injection
Antares Pharma announced the approval of Xyosted injection by the FDA. Xyosted is the first FDA approved subcutaneous testosterone enanthate product for once-weekly, at-home self-administration with an easy-to-use, single dose, disposable QuickShot auto injector. Xyosted has been approved in three dosage strengths, 50 mg, 75 mg and 100 mg and is indicated for testosterone replacement therapy in adult males for conditions associated with a deficiency or absence of endogenous testosterone.
Oncolytics initiated at Ladenburg
Oncolytics initiated with a Buy at Ladenburg. Ladenburg Thalmann analyst Wangzhi Li started Oncolytics Biotech with a Buy rating and $22 price target. The analyst believes Oncolytics is “significantly under-recognized and undervalued, and presents an attractive investment opportunity.” The company’s lead program Pelareorep is a RNA reovirus oncolytic virus that has been safely dosed in greater than 1,100 patients across a variety of cancers, Li points out.
https://thefly.com/landingPageNews.php?id=2797573
Varian Medical announces first patient treated in India on Halcyon system
Varian announced a 75-year-old male with lung cancer was the first patient in India to be treated on the Halcyon system at Sterling Cancer Hospital in Gujarat. Halcyon simplifies and enhances virtually every aspect of image-guided volumetric intensity-modulated radiotherapy, including improved patient comfort and shortening the time from installation to first-treatment without sacrificing quality. The system is well suited to treat cancer patients, offering advanced treatments for lung, prostate, breast, head & neck, and many other forms of cancer.
https://thefly.com/landingPageNews.php?id=2797657
Aeglea BioTherapeutics gets rare pediatric disease tag on pegzilarginase
Aeglea BioTherapeutics gets rare pediatric disease designation on pegzilarginase Aeglea announced the FDA has granted rare pediatric disease designation to the company’s lead product candidate, pegzilarginase, for the treatment of Arginase 1 Deficiency. This designation by the FDA confirms Aeglea’s eligibility to receive a rare pediatric disease priority review voucher upon approval of a biologics license application for pegzilarginase. Arginase 1 Deficiency is a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. https://thefly.com/landingPageNews.php?id=2797675
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