|Over a month ago|
Stoke Therapeutics reports Q1 EPS (34c), consensus (36c) » 07:0405/1505/15/20
"I am incredibly…
"I am incredibly gratified by the focus and determination of our employees during these challenging times. Thanks to their unwavering commitment to patients, we are continuing to make progress with STK-001 and are on track to enroll and dose the first children and adolescents with Dravet syndrome in the Phase 1/2a Monarch study later this year," said Edward Kaye, M.D., CEO of Stoke Therapeutics. "Our understanding of the potential for our TANGO technology in additional genetic diseases has continued to advance and we are generating data that we believe will support the nomination of a second preclinical candidate in the second half of 2020."
|Over a quarter ago|
Stoke Therapeutics reports FDA confirmation that STK-001 may procede » 08:1003/2303/23/20
"In 2019 we advanced…
"In 2019 we advanced our understanding of the potential applications of our TANGO technology to a variety of genetic targets, and generated preclinical data that underscore our confidence in STK-001 as a promising potential disease-modifying treatment for Dravet syndrome," said Edward M. Kaye, M.D., CEO of Stoke Therapeutics. "As we look ahead to 2020, we are poised to become a clinical-stage company with the anticipated start of our first in-human study of STK-001, and a growing portfolio as we work toward identifying our next preclinical product candidate later this year. The company also provided an update on its lead product candidate, STK-001, for Dravet syndrome. In the first quarter of 2020, Stoke received communication from the FDA confirming that it may proceed with clinical dosing in Part A of its planned Phase 1/2a "Monarch" study of STK-001 in children and adolescents ages 2 to 18 years old with Dravet syndrome. Part A of the study is designed to evaluate two dose cohorts of STK-001. As part of that communication, the FDA also informed the company that it has placed a partial clinical hold on Part B of the study, which is designed to evaluate higher doses of STK-001, pending additional preclinical toxicology data. This partial clinical hold is not due to any identified manufacturing or safety issue. Rather, the FDA is requesting additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level, or NOAEL. The NOAEL was determined using data from a pivotal non-human primate study that evaluated intrathecal delivery of single dose levels of STK-001. The highest dose administered in this study was equivalent to a human dose that is higher than what Stoke plans to administer in Part B of its Phase 1/2a clinical study and did not demonstrate effects that were considered adverse. Stoke has initiated single-dose toxicology studies to more fully characterize the safety profile of STK-001 at higher doses, in order to facilitate the removal of the partial clinical hold and proceed to Part B of the study. At the completion of dosing in Part A, and upon FDA clearance, Stoke will proceed with the higher-dosing cohorts planned in Part B of the study. Stoke still anticipates preliminary data from the study in 2021 and is working to minimize any potential delay to continued clinical testing of STK-001.
Guggenheim starts NeuBase Therapeutics at Buy with $13 price target » 07:4102/0702/07/20
NBSE, WVE, STOK
As previously reported,…
As previously reported, Guggenheim analyst Whitney Ijem initiated coverage of NeuBase Therapeutics with a Buy rating and $13 price target. The company's PATrOL platform for the development of ASOs has the potential to address key limitations of current oligo therapeutics and Ijem thinks near-term preclinical proof-of-concept trial results, combined with high profile rare disease indications like Huntington's Disease and DM1, could lead to a valuation more in line with preclinical peers such as Wave Life Sciences (WVE) and Stoke Therapeutics (STOK), the analyst tells investors.
Ten new option listings and two option delistings on January 23rd » 08:3001/2301/23/20
AKRO, AQST, CYCN, ITRN, OBSV, PHAS, PHAT, STOK, XP, MDR, TOO
New option listings for…
New option listings for January 23rd include Akero Therapeutics Inc (AKRO), Aquestive Therapeutics Inc (AQST), Cyclerion Therapeutics Inc (CYCN), Ituran (ITRN), McDermott International Inc (MDRIQ), ObsEva (OBSV), PhaseBio Pharmaceuticals Inc (PHAS), Phathom Pharmaceuticals Inc (PHAT), Stoke Therapeutics Inc (STOK), and XP Inc (Class A Stock) (XP). Option delistings effective January 23rd include McDermott International Inc (MDR) and Teekay Offshore Partners LP (Units) (TOO).
Stoke Therapeutics initiated with an Outperform at Wedbush » 16:2612/1712/17/19
Wedbush analyst Laura…
Wedbush analyst Laura Chico initiated coverage of Stoke Therapeutics with an Outperform rating and $31 price target.
Stoke Therapeutics presents STK-001 preclinical data in Dravet syndrome » 10:0412/0812/08/19
Stoke Therapeutics presented new preclinical data on STK-001, a potential new disease-modifying medicine for the treatment of Dravet syndrome. Data from studies in non-human primates showed STK-001 distributed throughout the brain and achieved target engagement and increased Nav1.1 protein expression throughout the cortex after a single intrathecal injection, the company said. Safety findings showed STK-001 to be well-tolerated at the two intrathecal dose levels studied, it added. These data were presented today in a poster session at the American Epilepsy Society Annual Meeting in Baltimore. Stoke selected two dose levels of STK-001 for this non-GLP study in order to evaluate safety, brain biodistribution, target engagement and Nav1.1 protein expression. On day 1, treatment-naive cynomolgus monkeys were administered a single, bolus intrathecal lumbar injection at one of two dose levels of STK-001. After dosing, the animals underwent standard clinical and neurological observation, and blood samples were collected. STK-001 concentration level, gene expression, and protein expression were assessed in the brain on day 3 and on day 29. Brain tissue exposure to STK-001 was observed on day 3 and day 29. In the high dose group, exposure of STK-001 was observed in all brain regions examined, except pons and thalamus. STK-001 levels in cortical brain regions were generally higher than in deeper structures and were also increased from day 3 to day 29.
Stoke Therapeutics presents preclinical data from studies of STK-001 » 12:1312/0712/07/19
Stoke Therapeutics announced preclinical data from studies of STK-001 "that showed significant improvements in survival and reductions in seizure frequency in a mouse model of Dravet syndrome." New data from electroencephalography recordings showed 76% of DS mice treated with STK-001 were seizure free compared to 48% that were treated with a placebo, the company said in a statement. An 80% reduction in the average number of spontaneous seizures was also observed among treated DS mice compared to placebo. These data were presented in a poster session at the American Epilepsy Society Annual Meeting in Baltimore. "The data on STK-001 from this mouse model give us confidence in our approach to treating the underlying cause of Dravet syndrome by restoring Nav1.1 protein expression to near normal levels," said Edward Kaye, Chief Executive Officer of Stoke Therapeutics. "The reductions in mortality previously observed with STK-001 were compelling and the new EEG data provide further evidence of the potential for STK-001 to impact Dravet syndrome by reducing seizure frequency and possibly preventing seizures entirely. What is particularly remarkable is that these data were generated from a spontaneous seizure model, which we believe accurately reflects the clinical situation in people with Dravet syndrome."
Fly Intel: Top five analyst initiations » 10:1611/1211/12/19
ARLO, CCC, STOK, BMRN, BIIB, AMGN, CTVA
Catch up on today's…
Catch up on today's top five analyst initiations with this list compiled by The Fly: 1. Arlo Technologies (ARLO) resumed with a Buy at Deutsche Bank. 2. Clarivate Analytics (CCC) initiated with a Hold at Stifel. 3. Stoke Therapeutics (STOK) initiated with a Buy at BTIG. 4. BioMarin (BMRN), Biogen (BIIB), and Amgen (AMGN) were initiated with a Buy at SunTrust. 5. Corteva (CTVA) initiated with a Buy at Redburn. This list is just a portion of The Fly's analyst coverage. To see The Fly's full Street Research coverage, click here.
Stoke Therapeutics initiated with a Buy at BTIG » 06:2011/1211/12/19
BTIG analyst Thomas…
BTIG analyst Thomas Shrader initiated coverage of Stoke Therapeutics with a Buy rating and $46 price target. The analyst cites the potential of the company's technology built by the inventors of SPINRAZA, along with the large informatics engine designed to identify additional disease states that might be treated via modification of pre-mRNA splicing. The "systematic screening methodology called TANGO" has nearly "boundless potential" toward treating a large number of genetic diseases and likely many chronic diseases, Shrader writes in his research note.
Stoke Therapeutics initiated with a Buy at H.C. Wainwright » 07:0910/2510/25/19
H.C. Wainwright analyst…
H.C. Wainwright analyst Debjit Chattopadhyay initiated coverage of Stoke Therapeutics with a Buy rating and $30 price target. The analyst believes TANGO, an antisense oligonucleotide platform, is an "intriguing approach" at combating haploinsufficiencies.