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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Compass’ Bispecific BTC Contender Meets Phase II/III Endpoint
Compass' bispecific antibody tovecimig hits primary efficacy endpoint in Phase II/III top-line data in advanced biliary tract cancer, and may have class side-effect advantages. But additional survival data may be needed to support US approval.
Data Analysis
R&D
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Aldeyra’s dry eye candidate reproxalap received a second FDA complete response letter, but the firm expressed confidence about refiling quickly based on two ongoing studies.
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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By allowing it to enter the brain more easily, trontinemab’s brain shuttle brings more patients to ‘amyloid zero’ levels faster, and with fewer brain swelling side effects.
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Compass' bispecific antibody tovecimig hits primary efficacy endpoint in Phase II/III top-line data in advanced biliary tract cancer, and may have class side-effect advantages. But additional survival data may be needed to support US approval.
Business
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Aldeyra’s dry eye candidate reproxalap received a second FDA complete response letter, but the firm expressed confidence about refiling quickly based on two ongoing studies.
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Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.
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The challenging US biosimilar market remains a persistent problem, leading the biosimilar developer to reconsider how it invests in the future while pushing for changes.
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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.
Scrip Originals
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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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.