Ultragenyx Pharmaceutical RARE introduced that it has submitted a biologics license utility (BLA) to the FDA for its UX111 (ABO-102) AAV gene remedy as a therapy for sufferers with Sanfilippo syndrome sort A (MPS IIIA) below the accelerated approval pathway.
Earlier this yr, the FDA aligned with Ultragenyx, after an intensive analysis, on using cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to assist an accelerated approval pathway for mucopolysaccharidoses (MPS) problems, together with Sanfilippo syndrome.
Ultragenyx’s BLA submission for UX111 is backed by knowledge, together with findings from the continuing, pivotal part I/II/III Transpher A research. Outcomes present that therapy with UX111 led to a fast and lasting discount in HS ranges within the CSF of sufferers with MPS IIIA. Moreover, sustaining decrease CSF HS ranges over time was linked to improved long-term cognitive growth, contrasting with the decline noticed in pure historical past knowledge. The investigational gene remedy was total well-tolerated within the research. Antagonistic occasions associated to therapy with the candidate have been principally delicate to average in severity.
Prior to now three months, shares of RARE have plunged 24.5% in contrast with the industry’s 12.8% decline.
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Topic to approval, UX111 will change into the primary FDA-approved remedy for MPS IIIA in the USA. MPS IIIA is a uncommon deadly lysosomal storage illness that primarily impacts the mind. Per Ultragenyx, roughly 3,000 to five,000 sufferers in commercially accessible geographies are affected by this illness, with a median life expectancy of 15 years. UX111 was added to RARE’s pipeline following an unique license settlement with Abeona Therapeutics in 2022.
The investigational gene remedy enjoys the Regenerative Medication Superior Remedy, Quick Monitor, Uncommon Pediatric Illness and Orphan Drug designations in the USA. Within the EU, it has been granted the PRIME and Orphan Medicinal Product designations.
RARE Initiates Part III Angelman Syndrome Research for GTX-102
In a separate press launch, Ultragenyx introduced that it has dosed the primary affected person within the pivotal part III Aspire research evaluating the efficacy and security of GTX-102, its investigational antisense oligonucleotide, for Angelman syndrome (AS). Per RARE, the 48-week Aspire research will enroll round 120 AS sufferers, aged 4 to 17, with a genetically confirmed prognosis of full maternal UBE3A gene deletion. The enrolled inhabitants will then be randomized equally to both obtain GTX-102 by intrathecal injection by way of lumbar puncture, or to the sham comparator group.
Kids enrolled within the therapy group will obtain three month-to-month 8 mg loading doses of GTX-102, adopted by a upkeep part with as much as 14 mg doses quarterly. These within the sham comparator group can change to therapy after finishing the preliminary research length. Per RARE, this dosing technique has been beforehand confirmed by the information from the part I/II research of the candidate for the AS indication.
The first endpoint of the Aspire research is the advance in cognition assessed by the Bayley-4 cognitive uncooked rating and the important thing secondary endpoint would be the Multi-domain Responder Index throughout the 5 domains of cognition, receptive communication, habits, gross motor operate and sleep.
Ultragenyx can be on observe to provoke an open-label medical research, named Aurora, to guage the protection and efficacy of GTX-102 for treating different AS genotypes in different affected person age teams in 2025. This extra research goals to allow therapy for a broader vary of AS sufferers.
AS is a uncommon neuro-genetic dysfunction that impacts cognitive and motor operate, making strolling, speaking and performing day by day actions tougher, which takes a severe toll on the standard of lifetime of the sufferers affected by it.
Different Key Scientific Packages in RARE’s Pipeline
Ultragenyx has a number of different fascinating gene remedy candidates in its pipeline. RARE and its accomplice, Mereo BioPharma, are collectively growing UX143 (setrusumab) monoclonal antibody forpediatric and younger grownup sufferers with osteogenesis imperfecta (OI) in two late-stage research, Orbit and Cosmic. In October, the FDA granted the Breakthrough Remedy designation to UX143 for the OI indication.
The corporate can be evaluating UX701, an investigational AAV9 gene remedy, in a part I/II/III Cyprus2+ research to deal with Wilson illness. Earlier this yr, Ultragenyx announced that the part III GlucoGene research (NCT05139316), evaluating its investigational AAV8 gene remedy, DTX401, to deal with glycogen storage illness sort Ia sufferers aged eight years and older achieved its main endpoint with statistical significance. The GlucoGene research additionally met its key secondary endpoints. The following step for the corporate includes discussing these outcomes with regulatory authorities to assist a advertising utility in 2025.
Ultragenyx Pharmaceutical Inc. Worth and Consensus
Ultragenyx Pharmaceutical Inc. price-consensus-chart | Ultragenyx Pharmaceutical Inc. Quote
RARE’s Zacks Rank & Shares to Think about
Ultragenyx presently carries a Zacks Rank #3 (Maintain).
Some better-ranked shares from the sector are Fortress Biosciences CSTL, CytomX Therapeutics CTMX and Spero Therapeutics SPRO, every sporting a Zacks Rank #1 (Sturdy Purchase) at current. You’ll be able to see the complete list of today’s Zacks #1 Rank stocks here.
Prior to now 60 days, 2024 estimates for Fortress Biosciences have improved from a lack of 59 cents per share to earnings of 34 cents. Throughout the identical timeframe, loss per share estimates for 2025 have narrowed from $2.15 to $1.84. Prior to now three months, shares of Fortress Biosciences have misplaced 9.6%.
CSTL’s earnings beat estimates in every of the trailing 4 quarters, delivering a median shock of 172.72%.
Prior to now 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to five cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents throughout the identical timeframe. Prior to now three months, CTMX inventory has misplaced 4.3%.
CytomX’s earnings beat estimates in two of the trailing 4 quarters and missed the mark within the different two, delivering a median shock of 115.70%.
Prior to now 60 days, estimates for Spero Therapeutics’ 2024 loss per share have narrowed from $1.59 to $1.29. Estimates for 2025 loss per share have narrowed from $1.54 to 79 cents throughout the identical timeframe. Prior to now three months, Spero’s shares have misplaced 23.2%.
SPRO’s earnings beat estimates in two of the trailing 4 quarters and missed the mark within the different two, delivering a median shock of 94.42%.
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