Ultragenyx CEO Emil Kakkis: ‘Our Future Is In Our Hands’
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.
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AZ’s Oncology R&D Head On China’s Scientific Promise And True Innovation
AstraZeneca remains committed to investing in R&D and alliances in China, where Susan Galbraith, the UK major’s head of oncology R&D, sees innovation eventually reaching parity with the US and Europe.
Data Analysis
R&D
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AstraZeneca remains committed to investing in R&D and alliances in China, where Susan Galbraith, the UK major’s head of oncology R&D, sees innovation eventually reaching parity with the US and Europe.
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BeiGene’s Phase III ociperlimab joins the list of failed TIGIT inhibitors, as candidates from Roche, Merck & Co. and others have failed late-stage studies.
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With new Phase II data, Edgewise asserted that EDG-7500, a sarcomere modulator, could offer better efficacy and safety than cardiac myosin inhibitors in hypertrophic cardiomyopathy.
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Investing heavily in antibody-drug conjugates, AbbVie is pioneering a dual STEAP1xPSMA-targeting approach in prostate cancer, as other candidates advance.
Business
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Public Company Edition: Stock valuations are falling due to political, economic and regulatory uncertainty, resulting in fewer large public offerings, more alternative financings and cost cuts. Carisma, Tenaya, BioAtla, Arbutus, Nkarta, Alector and Adaptimmune announced layoffs.
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The US FDA approved anti-CD19 antibody Uplizna, from Amgen’s $27.8bn purchase of Horizon in 2023, for IgG4-related disease – a larger market than its original NMOSD indication.
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BeiGene’s Phase III ociperlimab joins the list of failed TIGIT inhibitors, as candidates from Roche, Merck & Co. and others have failed late-stage studies.
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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.
Scrip Originals
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Public Company Edition: Stock valuations are falling due to political, economic and regulatory uncertainty, resulting in fewer large public offerings, more alternative financings and cost cuts. Carisma, Tenaya, BioAtla, Arbutus, Nkarta, Alector and Adaptimmune announced layoffs.
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Supply chain disruption fears at the start of the COVID-19 pandemic caused drug over-ordering. Imminent tariffs on drugs may have had a similar effect on pharma sales in Q1 earnings season.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
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Plus deals involving GV20/Mitsubishi Tanabe, Kaken/Alumis, AstraZeneca/Alteogen and deal terminations involving Clover/Gavi Alliance and Rhythm/RareStone.