Ultragenyx deal with Solid Biosciences looks 'solid,' says Piper Sandler » 09:2410/2310/23/20
RARE, SLDB, SRPT, PFE
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.
Ultragenyx, Solid Biosciences collaborate for DMD gene therapies » 08:1510/2310/23/20
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.
|Over a week ago|
Chardan boosts Solid Biosciences target 250% to $12.50 from $5 » 10:5510/0110/01/20
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.
Solid Biosciences price target raised to $12.50 from $5 at Chardan » 10:4710/0110/01/20
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.
Solid Biosciences says FDA lifts clinical hold on IGNITE DMD trial » 07:3510/0110/01/20
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.
|Over a quarter ago|
Solid Biosciences downgraded to Market Perform at SVB Leerink » 07:2307/2807/28/20
SLDB, SRPT, PFE
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.
Solid Biosciences downgraded to Market Perform from Outperform at SVB Leerink » 06:0407/2807/28/20
SVB Leerink analyst…
SVB Leerink analyst Joseph Schwartz downgraded Solid Biosciences to Market Perform from Outperform.
Solid Biosciences gets FDA notice that IGNITE DMD Phase I/II trial stays on hold » 17:5307/2407/24/20
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.
Solid Biosciences appoints Ian Smith as chairman of board » 07:0406/1806/18/20
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).
Solid Biosciences presents data from DMD Gene Therapy Program » 07:2705/1405/14/20
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.