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Recommendations
Ultragenyx deal with Solid Biosciences looks 'solid,' says Piper Sandler » 09:24
10/23/20
10/23
09:24
10/23/20
09:24
RARE

Ultragenyx

$91.84 /

+2.73 (+3.06%)

, SLDB

Solid Biosciences

$3.44 /

-0.09 (-2.55%)

, SRPT

Sarepta

$137.63 /

+0.37 (+0.27%)

, PFE

Pfizer

$37.43 /

+0.37 (+1.00%)

Piper Sandler analyst…

Piper Sandler analyst Christopher Raymond keeps an Overweight rating on Ultragenyx Pharmaceutical (RARE) after the company announced the license of Solid Biosciences' (SLDB) microdystrophin construct for use in AAV8 gene therapy. The $40M upfront for this deal in the context of Ultragenyx's $800M cash balance "seems quite manageable," Raymond tells investors in a research note. The analyst is "intrigued" by Ultragenyx's view of Solid's technology and thinks the company's track record on similar deals warrants the "benefit of the doubt" here. The combination of Solid's construct with Ultragenyx's proprietary AAV8 variant system "could make for a formidable player," despite active programs at Sarepta (SRPT), Pfizer (PFE) and Astellas, says Raymond.

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SRPT Sarepta
$137.63 /

+0.37 (+0.27%)

SLDB Solid Biosciences
$3.44 /

-0.09 (-2.55%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

PFE Pfizer
$37.43 /

+0.37 (+1.00%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

07/31/20 Barclays
Ultragenyx price target raised to $99 from $87 at Barclays
07/01/20 Truist
Ultragenyx price target raised to $88 from $82 at SunTrust
07/01/20 JPMorgan
Ultragenyx price target raised to $84 from $76 at JPMorgan
07/01/20 Wedbush
Ultragenyx price target raised to $91 from $83 at Wedbush
SLDB Solid Biosciences
$3.44 /

-0.09 (-2.55%)

10/01/20 Chardan
Chardan boosts Solid Biosciences target 250% to $12.50 from $5
10/01/20 Chardan
Solid Biosciences price target raised to $12.50 from $5 at Chardan
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform at SVB Leerink
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
SRPT Sarepta
$137.63 /

+0.37 (+0.27%)

09/29/20 Mizuho
Sarepta data update reinforces investment thesis, says Mizuho
09/29/20 Citi
Sarepta updates positive but 'fairly incremental,' says Citi
09/28/20 Piper Sandler
Piper maintains Overweight rating on Sarepta after data on gene therapy programs
09/15/20 Citi
Sarepta price target raised to $190 from $177 at Citi
PFE Pfizer
$37.43 /

+0.37 (+1.00%)

10/23/20 Truist
Pfizer initiated with a Buy at Truist
10/14/20 SVB Leerink
BioNTech price target raised to $69 from $62 at SVB Leerink
10/12/20
Fly Intel: Top five analyst downgrades
10/12/20 Atlantic Equities
Pfizer downgraded to Neutral from Overweight at Atlantic Equities
SRPT Sarepta
$137.63 /

+0.37 (+0.27%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

PFE Pfizer
$37.43 /

+0.37 (+1.00%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

PFE Pfizer
$37.43 /

+0.37 (+1.00%)

SRPT Sarepta
$137.63 /

+0.37 (+0.27%)

PFE Pfizer
$37.43 /

+0.37 (+1.00%)

PFE Pfizer
$37.43 /

+0.37 (+1.00%)

Hot Stocks
Ultragenyx, Solid Biosciences collaborate for DMD gene therapies » 08:15
10/23/20
10/23
08:15
10/23/20
08:15
RARE

Ultragenyx

$91.84 /

+2.73 (+3.06%)

, SLDB

Solid Biosciences

$3.44 /

-0.09 (-2.55%)

Ultragenyx Pharmaceutical…

Ultragenyx Pharmaceutical (RARE) and Solid Biosciences (SLDB) announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid's differentiated microdystrophin construct, Ultragenyx's HeLa producer cell line manufacturing platform, and AAV8 variants. The collaboration also brings together Solid's expertise in muscle biology and Ultragenyx's expertise in bringing novel therapies to patients with rare diseases. Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid's proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40M investment in Solid and has agreed to pay up to $255M in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments. Solid's proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solid's proprietary microdystrophin construct at the 2E14 vg/kg dose in Solid's ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical trial, using SGT-001 produced using its improved HSV manufacturing process, in the first quarter of 2021. Ultragenyx intends to use its AAV-based HeLa PCL platform including HeLa 3.0 improvements for the development of product candidates. The platform enables large 2,000 liter commercial-scale AAV-based gene therapy product manufacturing. The PCL platform yields high-quality product from a highly reproducible, highly scalable, and less expensive process - a distinct vantage in higher dose indications like Duchenne. The capsid planned is an AAV8 variant with a favorable immunological profile that has been used successfully in the large scale 2,000 liter production process.

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SLDB Solid Biosciences
$3.44 /

-0.09 (-2.55%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

07/31/20 Barclays
Ultragenyx price target raised to $99 from $87 at Barclays
07/01/20 Truist
Ultragenyx price target raised to $88 from $82 at SunTrust
07/01/20 JPMorgan
Ultragenyx price target raised to $84 from $76 at JPMorgan
07/01/20 Wedbush
Ultragenyx price target raised to $91 from $83 at Wedbush
SLDB Solid Biosciences
$3.44 /

-0.09 (-2.55%)

10/01/20 Chardan
Chardan boosts Solid Biosciences target 250% to $12.50 from $5
10/01/20 Chardan
Solid Biosciences price target raised to $12.50 from $5 at Chardan
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform at SVB Leerink
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

RARE Ultragenyx
$91.84 /

+2.73 (+3.06%)

Over a week ago
Recommendations
Chardan boosts Solid Biosciences target 250% to $12.50 from $5 » 10:55
10/01/20
10/01
10:55
10/01/20
10:55
SLDB

Solid Biosciences

$4.42 /

+2.39 (+117.73%)

Chardan analyst Gbola…

Chardan analyst Gbola Amusa raised the firm's price target on Solid Biosciences to $12.50 from $5 and keeps a Buy rating on the shares after the FDA lifted the clinical hold on the Phase I/II IGNITE DMD trial of AAV9-based gene therapy SGT-001 in Duchenne muscular dystrophy. Today's news is an "important positive" given that manufacturing constraints in the space leave an opportunity for Solid, and the view from many in the market that SGT-001 may not return to the clinic, Amusa tells investors in a research note. Duchenne muscular dystrophy is a "vast opportunity" for gene therapies, and one that could take Solid "significantly past" the new price target with progress, adds the analyst. The stock in morning trading is up 125% to $4.56.

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SLDB Solid Biosciences
$4.42 /

+2.39 (+117.73%)

SLDB Solid Biosciences
$4.42 /

+2.39 (+117.73%)

10/01/20 Chardan
Solid Biosciences price target raised to $12.50 from $5 at Chardan
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform at SVB Leerink
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
Recommendations
Solid Biosciences price target raised to $12.50 from $5 at Chardan » 10:47
10/01/20
10/01
10:47
10/01/20
10:47
SLDB

Solid Biosciences

$4.70 /

+2.67 (+131.53%)

Chardan analyst Gbola…

Chardan analyst Gbola Amusa raised the firm's price target on Solid Biosciences to $12.50 from $5 and keeps a Buy rating on the shares.

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SLDB Solid Biosciences
$4.70 /

+2.67 (+131.53%)

SLDB Solid Biosciences
$4.70 /

+2.67 (+131.53%)

07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform at SVB Leerink
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
Hot Stocks
Solid Biosciences says FDA lifts clinical hold on IGNITE DMD trial » 07:35
10/01/20
10/01
07:35
10/01/20
07:35
SLDB

Solid Biosciences

$2.03 /

-0.01 (-0.49%)

Solid Biosciences…

Solid Biosciences announced that the U.S. Food and Drug Administration has lifted the clinical hold placed on the Company's IGNITE DMD Phase I/II clinical trial. As announced in July 2020, the FDA had requested further manufacturing information, updated safety and efficacy data for all patients dosed, and provided direction on total viral load to be administered per patient. Based on the Company's response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. As part of its commitment to continuously improve its manufacturing processes, Solid implemented and shared with the FDA manufacturing process changes that remove the majority of empty viral capsids, allowing target dosing to be achieved with fewer viral particles. This reduction in the total amount of virus delivered to each patient is intended to support safe dosing of SGT-001 for the duration of the IGNITE DMD trial. In its July 2020 communication, the FDA requested additional information regarding the comparability between SGT-001 made using Solid's prior manufacturing process and its current, improved process. In response to this request, Solid submitted data from a new, quantitative, in vitro microdystrophin expression assay that demonstrates comparability between SGT-001 manufactured by the two processes. Solid is reducing the maximum weight of the next two patients dosed to 18 kg per patient, with safety outcomes from these two patients driving potential weight increase of patients dosed subsequently. This reduction, in conjunction with the delivery of fewer viral particles as a result of the Company's manufacturing process improvements, will reduce patients' total viral load while continuing dosing at the 2E14 vg/kg dose.Solid provided the FDA with updated safety and functional efficacy data for all patients dosed to date in IGNITE DMD. There have been no additional drug-related adverse events up to 30 months post dosing. Additionally, to mitigate the risk of serious drug-related adverse events, Solid is amending the IGNITE DMD clinical protocol to include the prophylactic use of both anti-complement inhibitor eculizumab and C1 esterase inhibitor, and increasing the prednisone dose in the first month post dosing.

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SLDB Solid Biosciences
$2.03 /

-0.01 (-0.49%)

SLDB Solid Biosciences
$2.03 /

-0.01 (-0.49%)

07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform at SVB Leerink
07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
Over a quarter ago
Downgrade
Solid Biosciences downgraded to Market Perform at SVB Leerink » 07:23
07/28/20
07/28
07:23
07/28/20
07:23
SLDB

Solid Biosciences

$2.53 /

-0.34 (-11.85%)

, SRPT

Sarepta

$158.30 /

-0.39 (-0.25%)

, PFE

Pfizer

$37.59 /

-0.08 (-0.21%)

As previously reported,…

As previously reported, SVB Leerink analyst Joseph Schwartz downgraded Solid Biosciences (SLDB) to Market Perform from Outperform with a price target of $3, down from $7. While the analyst continues to see potential value in SGT-001's differentiated transgene, it is increasingly unclear whether the company will be able to convince the FDA that it is appropriate to use its AAV9 vector which may have safety issues at high doses. The continuation of the clinical hold only adds more distance between Solid Biosciences and other companies in the DMD gene therapy race including Sarepta (SRPT) and Pfizer (PFE), he contends.

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SRPT Sarepta
$158.30 /

-0.39 (-0.25%)

SLDB Solid Biosciences
$2.53 /

-0.34 (-11.85%)

PFE Pfizer
$37.59 /

-0.08 (-0.21%)

SLDB Solid Biosciences
$2.53 /

-0.34 (-11.85%)

07/28/20 SVB Leerink
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink
05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
12/19/19 SVB Leerink
Solid data 'underwhelming,' SGT-001 path forward unclear, says SVB Leerink
SRPT Sarepta
$158.30 /

-0.39 (-0.25%)

07/02/20 William Blair
Sarepta's recent deals may expand eligible patient set, says William Blair
06/22/20 JPMorgan
Sarepta price target raised to $190 from $160 at JPMorgan
06/15/20 Piper Sandler
Sarepta gene therapy DMD program remains differentiated, says Piper Sandler
06/09/20 Mizuho
Sarepta price target raised to $192 from $183 at Mizuho
PFE Pfizer
$37.59 /

-0.08 (-0.21%)

07/27/20 H.C. Wainwright
Agreement termination 'optimal' for Vyleesi future, says H.C. Wainwright
07/20/20 Jefferies
AstraZeneca slide 'overdone' after 'encouraging' vaccine data, says Jefferies
07/14/20 Mizuho
IPR not likely to impact uniQure's ability to commercialize AMT-061, says Mizuho
07/13/20 Canaccord
BioNTech price target raised to $75 from $60 at Canaccord
SRPT Sarepta
$158.30 /

-0.39 (-0.25%)

PFE Pfizer
$37.59 /

-0.08 (-0.21%)

SRPT Sarepta
$158.30 /

-0.39 (-0.25%)

PFE Pfizer
$37.59 /

-0.08 (-0.21%)

SRPT Sarepta
$158.30 /

-0.39 (-0.25%)

PFE Pfizer
$37.59 /

-0.08 (-0.21%)

PFE Pfizer
$37.59 /

-0.08 (-0.21%)

Downgrade
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink » 06:04
07/28/20
07/28
06:04
07/28/20
06:04
SLDB

Solid Biosciences

$2.53 /

-0.34 (-11.85%)

SVB Leerink analyst…

SVB Leerink analyst Joseph Schwartz downgraded Solid Biosciences to Market Perform from Outperform.

ShowHide Related Items >><<
SLDB Solid Biosciences
$2.53 /

-0.34 (-11.85%)

SLDB Solid Biosciences
$2.53 /

-0.34 (-11.85%)

05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
12/19/19 SVB Leerink
Solid data 'underwhelming,' SGT-001 path forward unclear, says SVB Leerink
12/18/19 Nomura
Solid Biosciences price target raised to $10 from $6 at Nomura Instinet
Hot Stocks
Solid Biosciences gets FDA notice that IGNITE DMD Phase I/II trial stays on hold » 17:53
07/24/20
07/24
17:53
07/24/20
17:53
SLDB

Solid Biosciences

$2.87 /

-0.03 (-1.03%)

Solid Biosciences…

Solid Biosciences announced that it received written communication from the FDA that the company's IGNITE DMD Phase I/II clinical trial remains on clinical hold. In November 2019, the FDA placed IGNITE DMD on clinical hold as the result of a serious adverse event that occurred in the sixth patient dosed in the trial; as previously reported the SAE has fully resolved. In April 2020, Solid provided the FDA with information and measures intended to improve patient safety and in May 2020, Solid received written communication from the FDA that the trial remained on hold. In June 2020, the company submitted a response to the FDA that provided data related to manufacturing process improvements. Today, the FDA responded by maintaining the clinical hold and requesting further manufacturing information, updated safety and efficacy data for all patients dosed, and providing direction on total viral load to be administered per patient. Solid is evaluating next steps and will provide an update as appropriate.

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SLDB Solid Biosciences
$2.87 /

-0.03 (-1.03%)

SLDB Solid Biosciences
$2.87 /

-0.03 (-1.03%)

05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
12/19/19 SVB Leerink
Solid data 'underwhelming,' SGT-001 path forward unclear, says SVB Leerink
12/18/19 Nomura
Solid Biosciences price target raised to $10 from $6 at Nomura Instinet
Hot Stocks
Solid Biosciences appoints Ian Smith as chairman of board » 07:04
06/18/20
06/18
07:04
06/18/20
07:04
SLDB

Solid Biosciences

$3.04 /

+0.015 (+0.50%)

, VRTX

Vertex Pharmaceuticals

$280.48 /

+6.92 (+2.53%)

Solid Biosciences (SLDB)…

Solid Biosciences (SLDB) announced the appointment of board member Ian Smith as chairman of the board. Smith succeeds Andrey Zarur, Ph.D., who served as chairman of the board since 2013 and is one of the co-founders of Solid Biosciences. The appointment is effective June 16, 2020. Smith most recently serving as EVP and COO of Vertex Pharmaceuticals (VRTX).

ShowHide Related Items >><<
VRTX Vertex Pharmaceuticals
$280.48 /

+6.92 (+2.53%)

SLDB Solid Biosciences
$3.04 /

+0.015 (+0.50%)

SLDB Solid Biosciences
$3.04 /

+0.015 (+0.50%)

05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
12/19/19 SVB Leerink
Solid data 'underwhelming,' SGT-001 path forward unclear, says SVB Leerink
12/18/19 Nomura
Solid Biosciences price target raised to $10 from $6 at Nomura Instinet
VRTX Vertex Pharmaceuticals
$280.48 /

+6.92 (+2.53%)

06/12/20 Piper Sandler
Crispr update suggests 'potential curative effect' of CTX001, says Piper Sandler
06/12/20 Citi
Vertex price target raised to $325 from $305 at Citi
04/30/20 SVB Leerink
Vertex price target raised to $263 from $224 at SVB Leerink
04/30/20 H.C. Wainwright
Vertex price target raised to $295 from $270 at H.C. Wainwright
VRTX Vertex Pharmaceuticals
$280.48 /

+6.92 (+2.53%)

VRTX Vertex Pharmaceuticals
$280.48 /

+6.92 (+2.53%)

VRTX Vertex Pharmaceuticals
$280.48 /

+6.92 (+2.53%)

Hot Stocks
Solid Biosciences presents data from DMD Gene Therapy Program » 07:27
05/14/20
05/14
07:27
05/14/20
07:27
SLDB

Solid Biosciences

$2.66 /

+ (+0.00%)

Solid Biosciences…

Solid Biosciences announced the presentation of clinical biomarker data from its SGT-001 microdystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy on May 13 at the American Society of Gene and Cell Therapy 23rd Annual Meeting, which is being held virtually. In addition, data from studies characterizing novel adeno-associated virus vectors engineered for muscle gene delivery were presented in a poster session. SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy. Oral presentation delivered by Patrick Gonzalez, PhD, Associate Director, R&D, Solid Biosciences. Data presented from the ongoing IGNITE DMD clinical trial studying SGT-001 at a dose of 2E14 vg/kg were: SGT-001 administration results in dose-dependent, muscle wide microdystrophin expression in muscle biopsies collected 90 days post-SGT-001 administration. SGT-001-driven microdystrophin expression results in stabilization of dystrophin associated proteins. SGT-001-driven microdystrophin expression results in restored enzymatically active neuronal nitric oxide synthase at the sarcolemma. Characterization of Novel AAV Vectors Engineered for Muscle Gene Delivery. Poster presentation and online Q&A delivered by Jennifer Green, PhD, Senior Scientist, R&D, Solid Biosciences. This study utilized a rational design approach to generate a set of novel capsids predicted to have increased muscle tropism and transduction efficiency for the development of treatment for Duchenne. Key findings are: AAV-SLB101, a novel capsid, showed superior transduction efficiency in comparison to AAV9 in in vitro assays in both mouse and Duchenne human skeletal muscle cells. These in vitro results translated to increased biodistribution and microdystrophin protein expression in vivo in both quadriceps and heart, and decreased biodistribution to liver, in comparison to AAV9. An expanded panel of novel capsids identified two more candidates of interest, AAV-SLB102 and AAV-SLB111, that look similar to AAV-SLB101 in in vitro assays for binding, uptake and microdystrophin protein expression in C2C12 cells.

ShowHide Related Items >><<
SLDB Solid Biosciences
$2.66 /

+ (+0.00%)

SLDB Solid Biosciences
$2.66 /

+ (+0.00%)

05/08/20 Nomura
Solid Biosciences price target lowered to $6 from $10 at Nomura Instinet
05/07/20 Evercore ISI
Solid Biosciences downgraded to In Line from Outperform at Evercore ISI
12/19/19 SVB Leerink
Solid data 'underwhelming,' SGT-001 path forward unclear, says SVB Leerink
12/18/19 Nomura
Solid Biosciences price target raised to $10 from $6 at Nomura Instinet

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