Rare diseases
With its acquisition of IntraBio, Shionogi will gain ownership over Aqneursa, which could hit peak global sales of $1 billion across its two indications in Niemann-Pick disease type C and ataxia-telangiectasia, according to Jefferies.
Viking Therapeutics rode strong obesity data before a major capital raise, while Longeveron’s mid-stage setback prompted questions about its future.
All signs continue to point forward for Biogen’s initial foray into lupus, as litifilimab elicited clear or almost clear skin within one year in patients with cutaneous lupus in a mid-stage study. Data from two Phase 3 studies of systemic lupus are expected by the end of the year.
Jefferies analysts made the prediction after Sarepta Therapeutics, which has endured nothing short of a saga with the Duchenne muscular dystrophy gene therapy Elevidys, presented data this week showing its clinically meaningful benefit in older patients who are able to walk.
In this episode of Denatured, you’ll hear from Max Bronstein, CEO of Aviva Strategies and Teresa Barnes, Chief Executive Warrior of PF Warriors. We dig into why U.S. patients are losing access to rare disease trials happening abroad and how the FAIR Act’s accelerated, harmonized reviews could help the U.S. keep pace while getting lifesaving studies to patients faster.
Ebvallo, which Pierre Fabre Pharmaceuticals and Atara Biotherapeutics are proposing to treat a posttransplant complication, joins a wave of rejected or discouraged therapies on which the FDA has reversed previous guidance after the exit of former Commissioner Marty Makary.
Summit Therapeutics scores a $2 billion equity investment from AstraZeneca to support ivonescimab’s late-stage development; uniQure crashes after four-year data from its Huntington’s disease gene therapy failed to impress; Kyverna Therapeutics brings new light to a struggling CAR T space in autoimmune disease.
The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
The recent FDA approval of Fayuvi for a rare, childhood neurodegenerative disease was “particularly special” for Ultragenyx CEO Emil Kakkis. It could also, along with an August nod for another gene therapy, help offset recent challenges brought on by two asset failures.
Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic ALS subtype in a Phase 3 trial. The partners plan to approach the FDA with the results to determine potential pathways for approval.
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