Rare Diseases
After dropping its lead program in December, Spruce has been exploring strategic options – and thinks it has a de-risked path for MPS IIIB therapy tralesinidase from BioMarin.
Lead drug GLM101 will move into its first placebo-controlled trial based on positive results in adults and adolescents with PMM2-CDG in an open-label Phase IIa study.
Results from a China Phase III trial show the potential of RemeGen's fusion protein telitacicept in generalized myasthenia gravis. But study design differences make direct comparisons with argenx's FcRn inhibitor Vyvgart challenging.
Menarini's Asia-Pacific CEO talks to Scrip about how the Italian group is tailoring its portfolio and pricing approach to local markets making strong strides in primary care and consumer health, while also charting a distinct path in rare diseases and oncology. The China market is also a key focus including as a potential source of innovation as is partnering, where the group has a rich history.
It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.
The small interference RNA therapeutic can be used by patients regardless of inhibitor status.
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
The Belgian firm banks nearly €77.7m to push its Charcot-Marie-Tooth to proof-of-concept.
Amvuttra will target a substantially larger patient population with a new indication, but it is third to market behind Pfizer’s Vyndaqel and Bridge Bio’s Attruby and will cost more.
The company’s Immunovant announced positive data from a Phase III trial in myasthenia gravis but the focus is on next-generation drug IMVT-1402.
The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.
In the crowded and competitive field of generalized myasthenia gravis, Amgen has faith that Uplizna’s twice-yearly administration will make it the treatment of choice.
A joint venture with AI specialist Hologen will bring in much-needed cash for the ambitious gene therapy firm.
Ono agreed to pay $280m up front for global rights to Ionis’s Phase II polycythemia vera candidate, which analysts say was de-risked by recent data reported for other PV drugs.
Having picked up Niemann-Pick disease type C drug Miplyffa for a song, the US rare disease specialist is singing all the way to the bank after selling the priority review voucher that came with its approval.
Attruby is making fast inroads thanks to its edge over Pfizer’s established Vyndaqel, but will soon face competition from Alnylam.
Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.