Rare Diseases
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.
Amylin analogs could well match the weight loss seen with Novo Nordisk and Eli Lilly’s GLP-1-based blockbusters with less fewer tolerability issues. The Danish company believes petrelintide will lead the pack and is vetting potential big pharma partners very carefully.
The Swedish company is celebrating approval for the first treatment for rare congenital disorder MCT8 deficiency and plans a launch in the near future.
The Paris-headquartered group has been reflecting on a solid performance last year.
After coming up blank with its in-house oncology efforts, the German conglomerate is mulling a deal to acquire the US specialist company which has just managed a new drug approval for mirdametinib.
CEO Djordje Filipovic tells Scrip that after talks with "multiple other potential partners,” the firm chose the Japanese group for its expertise and marketing capabilities in rare diseases” to be the licensing partner for tadekinig alfa in the US.
The Danish drugmaker, best known for dermatology, has secured the European rights to Shanghai Junshi Biosciences’ cancer therapy toripalimab, which was the first Chinese PD-1/L1 checkpoint inhibitor to be approved in the US.
On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.
The US biotech’s Duchenne muscular dystrophy drug is “undoubtedly the most successful gene therapy launch in all of history,” according to CEO Doug Ingram. “We have barely scratched the surface of the opportunity in front of us.”
Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.
From gene therapies to small molecule drugs, investors seemed to have eschewed the risks of biotech company investment propositions in 2024 but last year’s disappointments provided good lessons going forward.