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RARE

Ultragenyx

$106.65 /

+0.06 (+0.06%)

08:44
05/14/21
05/14
08:44
05/14/21
08:44

Ultragenyx announces longer-term data from OTC phase 1/2, HeLa PCL studies

Ultragenyx Pharmaceutical announced longer-term data from the Ornithine Transcarbamylase, or OTC, Deficiency Phase 1/2 studies demonstrating ongoing durability of response, as well as data highlighting further advancements to the company's proprietary HeLa producer cell line, or PCL, manufacturing platform. Data were presented this week at the American Society of Gene & Cell Therapy 24th Annual Meeting. DTX301 Program. The six patients who previously demonstrated a response remain clinically and metabolically stable, including all three treated at the highest dose, which is the dose that will be used in the Phase 3 study. Some patients have now demonstrated a durable response three years after treatment, and more than two years after discontinuing ammonia-scavenger medications and liberalizing protein-restricted diets. All responders remain in excellent clinical condition with no significant adverse events, hospitalizations, or other events related to OTC deficiency. Both patients in the prophylactic steroid cohort are doing well clinically with good metabolic control and without any safety issues. The first patient has maintained normal ammonia levels and has reduced their ammonia-scavenger drug by 45% so far, following completion of the prophylactic course of steroids. This patient is continuing to taper medications. The second patient continues to taper their course of steroids, and changes in ammonia-scavenging drugs or diet will only be attempted after the prophylactic steroid taper is complete. This patient has maintained normal ammonia levels since treatment. Across all cohorts of the Phase 1/2 study, there have been no infusion-related adverse events and no treatment-related serious adverse events reported. All treatment-related adverse events have been Grade 1 or 2. The Phase 3 study will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 or placebo. The co-primary endpoints are the percentage of patients who achieve a response as measured by discontinuation or reduction in baseline disease management and the 24-hour plasma ammonia levels. HeLa 3.0 Producer Cell Line advancements significantly increase productivity of the platform. Data on the HeLa 3.0 platform demonstrated that genetic engineering of the HeLa cell significantly improved AAV production. This increased productivity was achieved while maintaining or improving product quality. Permanent knockout of target genes identified by RNA sequencing was shown to increase titer in established PCLs, and the single knockout HeLa 3.0 PCLs displayed similar phenotype to parental PCLs during production. These cell line engineering improvements result in a 2- to 5-fold yield improvement over the HeLa 2.0 platform, and a 50-fold yield improvement over HeLa 1.0. Additional improvements in yield are expected with future combinatorial knockdowns, with early studies suggesting a further 5-to 10-fold improvement. These improvements in the HeLa platform allow for increased productivity and reproducibility with higher full-to-empty AAV ratios while continuing to drive down AAV production costs, all important attributes in the manufacturing of gene therapies for diseases where high product yield is required.

  • 24

    Jun

  • 29

    Oct

RARE Ultragenyx
$106.65 /

+0.06 (+0.06%)

05/06/21
Fly Intel: Top five analyst upgrades
05/06/21 Citi
Ultragenyx upgraded to Buy from Neutral at Citi
05/05/21 Evercore ISI
Evercore upgrades Ultragenyx with 'good handle' on Angelman program
05/05/21 Evercore ISI
Ultragenyx upgraded to Outperform from In Line at Evercore ISI

TODAY'S FREE FLY STORIES

Recommendations
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CIBC analyst Mark Petrie…

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EMLAF Empire Company
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Syndicate
Bright Health opens at $17, IPO priced at $18 per share » 12:10
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BHG

Bright Health

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General news
Fed's Barkin stuck to the script » 12:10
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$ECON

Economic Data

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General news
NY Fed's Williams repeated that now is not the time to start to normalize policy » 12:10
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Hot Stocks
GeoPark co-founder initiating 'vote no' campaign against directors » 12:06
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Gerald O'Shaughnessy…

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GPRK GeoPark
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Periodicals
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CHPT ChargePoint
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Recommendations
Valens Company price target raised to C$4.25 from C$3.75 at Desjardins » 12:05
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06/24
12:05
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VLNCF

Valens Company

$2.58 /

+0.07 (+2.79%)

Desjardins analyst John…

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VLNCF Valens Company
$2.58 /

+0.07 (+2.79%)

VLNCF Valens Company
$2.58 /

+0.07 (+2.79%)

06/22/21 Stifel
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+0.07 (+2.79%)

Options
Baidu call volume above normal and directionally bullish » 12:05
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BIDU

Baidu

$193.25 /

+4.04 (+2.14%)

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BIDU Baidu
$193.25 /

+4.04 (+2.14%)

BIDU Baidu
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06/01/21 Mizuho
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  • 26
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Walmart MoneyCard by Green Dot now offered as demand deposit account » 12:02
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+0.94 (+0.69%)

Walmart (WMT) and Green…

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WMT Walmart
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WMT Walmart
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Microsoft introduces Windows 11 with Teams, Game Pass integration » 12:01
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Microsoft

$266.93 /

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Microsoft unveiled…

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MSFT Microsoft
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Recommendations
Berkeley Group price target raised to 5,240 GBp from 5,230 GBp at Canaccord » 12:01
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BKGFY

Berkeley Group

$68.89 /

+ (+0.00%)

Canaccord analyst Aynsley…

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BKGFY Berkeley Group
$68.89 /

+ (+0.00%)

05:11 Today Citi
Berkeley Group upgraded to Buy from Neutral at Citi
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Recommendations
Hardwoods Distribution price target raised to C$57 from C$43 at Canaccord » 12:00
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HDIUF

Hardwoods Distribution

$24.06 /

+1.0261 (+4.46%)

Canaccord analyst Yuri…

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HDIUF Hardwoods Distribution
$24.06 /

+1.0261 (+4.46%)

05/18/21 CIBC
Hardwoods Distribution price target raised to C$44 from C$39 at CIBC
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AMTD International falls -7.2% » 12:00
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AMTD International

$6.72 /

-0.03 (-0.44%)

AMTD International is…

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HKIB AMTD International
$6.72 /

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Hot Stocks
DoubleVerify falls -9.3% » 12:00
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DV

DoubleVerify

$39.11 /

-3.97 (-9.22%)

DoubleVerify is down…

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DV DoubleVerify
$39.11 /

-3.97 (-9.22%)

DV DoubleVerify
$39.11 /

-3.97 (-9.22%)

05/26/21 Truist
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DV DoubleVerify
$39.11 /

-3.97 (-9.22%)

  • 21
    Apr
DV DoubleVerify
$39.11 /

-3.97 (-9.22%)

DV DoubleVerify
$39.11 /

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Hot Stocks
Rite Aid falls -12.0% » 12:00
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RAD

Rite Aid

$17.97 /

-2.4 (-11.78%)

Rite Aid is down -12.0%,…

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RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

06/22/21 Deutsche Bank
Rite Aid price target lowered to $18 from $27 at Deutsche Bank
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RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

RAD Rite Aid
$17.97 /

-2.4 (-11.78%)

Hot Stocks
X Financial rises 11.6% » 12:00
06/24/21
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XYF

X Financial

$10.70 /

+1.06 (+11.00%)

X Financial is up 11.6%,…

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XYF X Financial
$10.70 /

+1.06 (+11.00%)

Hot Stocks
Arcus Biosciences rises 13.6% » 12:00
06/24/21
06/24
12:00
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RCUS

Arcus Biosciences

$26.58 /

+3.18 (+13.59%)

Arcus Biosciences is up…

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RCUS Arcus Biosciences
$26.58 /

+3.18 (+13.59%)

RCUS Arcus Biosciences
$26.58 /

+3.18 (+13.59%)

08:42 Today SVB Leerink
Arcus Biosciences price target raised to $63 from $53 at SVB Leerink
02/25/21 Barclays
Arcus Biosciences price target raised to $45 from $40 at Barclays
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Arcus Biosciences price target raised to $43 from $40 at Mizuho
01/20/21 Citi
Arcus Biosciences price target raised to $51 from $32 at Citi
RCUS Arcus Biosciences
$26.58 /

+3.18 (+13.59%)

RCUS Arcus Biosciences
$26.58 /

+3.18 (+13.59%)

RCUS Arcus Biosciences
$26.58 /

+3.18 (+13.59%)

Hot Stocks
Semrush rises 18.1% » 12:00
06/24/21
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12:00
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SEMR

Semrush

$23.89 /

+3.79 (+18.86%)

Semrush is up 18.1%, or…

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SEMR Semrush
$23.89 /

+3.79 (+18.86%)

SEMR Semrush
$23.89 /

+3.79 (+18.86%)

04/19/21
Fly Intel: Top five analyst initiations
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Semrush initiated with a Hold at Jefferies
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Semrush initiated with an Overweight at JPMorgan
04/19/21 Stifel
Semrush initiated with a Hold at Stifel
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$23.89 /

+3.79 (+18.86%)

  • 25
    Mar
SEMR Semrush
$23.89 /

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Recommendations
Converge Technology price target raised to C$11 from C$9.50 at Canaccord » 12:00
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06/24
12:00
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12:00
CTSDF

Converge Technology

$7.56 /

-0.13 (-1.69%)

Canaccord analyst Robert…

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CTSDF Converge Technology
$7.56 /

-0.13 (-1.69%)

11:41 Today Raymond James
Converge Technology price target raised to C$10.75 from C$8.75 at Raymond James
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Converge Technology price target raised to C$8.75 from C$7.75 at Raymond James
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Converge Technology initiated with a Buy at Desjardins
03/10/21 Canaccord
Converge Technology price target raised to C$8.50 from C$8 at Canaccord
Recommendations
Empire Company price target lowered to C$45 from C$47 at BMO Capital » 11:54
06/24/21
06/24
11:54
06/24/21
11:54
EMLAF

Empire Company

$32.55 /

-1.67 (-4.88%)

BMO Capital analyst Peter…

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EMLAF Empire Company
$32.55 /

-1.67 (-4.88%)

06/16/21 National Bank
Empire Company price target raised to C$46 from C$44 at National Bank
06/16/21 RBC Capital
Empire Company price target raised to C$45 from C$43 at RBC Capital
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Empire Company price target raised to C$44 from C$42 at Desjardins
General news
Treasury's $80 B 4- and 8-week bill auctions were ok » 11:50
06/24/21
06/24
11:50
06/24/21
11:50
$ECON

Economic Data

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Treasury's $80 B 4-…

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Syndicate
Doximity indicated to open at $36-$37, IPO priced at $26  11:48
06/24/21
06/24
11:48
06/24/21
11:48
DOCS

Doximity

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  • 24
    Jun
Hot Stocks
HP says currently shipping Windows-based PCs to be upgradeable to Windows 11 » 11:48
06/24/21
06/24
11:48
06/24/21
11:48
HPQ

HP Inc.

$29.33 /

+0.115 (+0.39%)

, MSFT

Microsoft

$267.14 /

+1.9 (+0.72%)

HP Inc. (HPQ) has…

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HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

05/28/21 Morgan Stanley
HP Inc. post-earnings pullback a buying opportunity, says Morgan Stanley
05/28/21 Citi
Citi says HP is not at peak, reiterates Buy rating
05/28/21 Deutsche Bank
HP Inc. price target raised to $32 from $30 at Deutsche Bank
05/28/21 Citi
Lenovo, HP, Dell earnings good news for Intel and AMD, says Citi
MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

06/23/21 Wedbush
Microsoft price target raised to $325 from $310 at Wedbush
06/16/21 William Blair
Microsoft gaming news opportunity not threat to Unity, says William Blair
06/15/21 Oppenheimer
Turtle Beach price target raised to $45 from $40 at Oppenheimer
06/07/21 Morgan Stanley
Microsoft valuation presents 'attractive entry point,' says Morgan Stanley
HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

HPQ HP Inc.
$29.33 /

+0.115 (+0.39%)

MSFT Microsoft
$267.14 /

+1.9 (+0.72%)

Options
Vipshop call volume above normal and directionally bullish » 11:45
06/24/21
06/24
11:45
06/24/21
11:45
VIPS

Vipshop

$19.09 /

+1 (+5.53%)

Bullish option flow…

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VIPS Vipshop
$19.09 /

+1 (+5.53%)

VIPS Vipshop
$19.09 /

+1 (+5.53%)

06/23/21
Vipshop participates in a conference call with Benchmark
06/23/21
Vipshop participates in a conference call with Benchmark
05/20/21 HSBC
Vipshop price target lowered to $25 from $30 at HSBC
04/15/21 JPMorgan
Vipshop upgraded to Overweight from Neutral at JPMorgan
VIPS Vipshop
$19.09 /

+1 (+5.53%)

  • 06
    Apr
VIPS Vipshop
$19.09 /

+1 (+5.53%)

VIPS Vipshop
$19.09 /

+1 (+5.53%)

VIPS Vipshop
$19.09 /

+1 (+5.53%)

Recommendations
Converge Technology price target raised to C$10.75 from C$8.75 at Raymond James » 11:41
06/24/21
06/24
11:41
06/24/21
11:41
CTSDF

Converge Technology

$7.56 /

-0.13 (-1.69%)

Raymond James analyst…

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CTSDF Converge Technology
$7.56 /

-0.13 (-1.69%)

04/05/21 Raymond James
Converge Technology price target raised to C$8.75 from C$7.75 at Raymond James
03/23/21 Desjardins
Converge Technology initiated with a Buy at Desjardins
03/10/21 Canaccord
Converge Technology price target raised to C$8.50 from C$8 at Canaccord
01/19/21 Raymond James
Converge Technology price target raised to C$7.75 from C$5.75 at Raymond James

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