Rare diseases
China cracks down on some biotechnology ownership transfers—but will the new policy impact licensing deals?; Disc Medicine’s Phase 3 data sweep redeems rejected rare disease therapy, teeing up another FDA run; and the ESMO conference gets underway in Madrid this week, with much-anticipated data from Merck, and Summit Therapeutics.
Disc Medicine’s bitopertin—rejected by the FDA in February—significantly reduced levels of a key disease biomarker and significantly increased the time patients with erythropoietic protoporphyria were able to spend in the sun and artificial light.
Johnson & Johnson beat analyst expectations in the third quarter but left Wall Street wanting more; BMS dominated the news cycle on Monday with a Phase 3 win validating its $4.1 billion RayzeBio buy and mixed data in a late-stage pulmonary fibrosis trial; Revolution Medicines’ newly approved pancreatic cancer drug works better for patients with certain mutations; and Caribou Biosciences may be on its way out.
While Alnylam’s recent reduced revenue guidance spooked investors, analysts remain broadly bullish on the biotech’s commercial prospects.
The centerpiece of Bristol Myers Squibb’s 2024 RayzeBio buy has read out, hitting the primary endpoint in a late-stage tumor trial.
Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
Biotechs developing therapeutics to treat rare diseases include two that received FDA approvals this year for drugs to treat spinal muscular atrophy and Alexander disease.
New data for Genmab and AbbVie’s bispecific antibody Epkinly wowed analysts and could set the partners up for an expanded approval in diffuse large B cell lymphoma; deals have flowed steadily this week, including Novartis’ nearly $8 billion bet on mRNA drugs with China’s Abogen Biosciences; and could Sarepta Therapeutics turn things around from last year’s annus horribilis?
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.
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