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Sarepta, Arrowhead take a big swing on siRNA drugs for rare disease

Sarepta Therapeutics and Arrowhead Pharmaceuticals teamed up on an siRNA partnership that has one of the biggest promises of biobucks in recent years. The deal, announced Tuesday morning, will see...

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Axsome moves toward NDA filing for narcolepsy drug after Phase 3 data

Axsome Therapeutics’ narcolepsy drug AXS-12 hit the primary endpoint in its Phase 3 ENCORE trial, showing a statistically significant improvement in the frequency of cataplexy attacks in narcoleptic...

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The Biden administration wants Medicare and Medicaid to cover GLP-1s. Will...

The Biden administration proposed a new rule Tuesday that would expand Medicare and Medicaid coverage of popular obesity drugs, earning praise from the pharmaceutical industry and patient advocates....

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Trump picks Stanford academic and pandemic critic Bhattacharya for NIH

President-elect Donald Trump tapped two policy and political outsiders to fill the remaining top healthcare roles in his administration, including Jay Bhattacharya, a prolific critic of the US Covid-19...

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Idorsia eyes layoffs, tees up Tryvio licensing deal to extend cash runway

Idorsia is making moves to stretch its cash reserves further. The Swiss drugmaker said it is narrowing its operations with 270 positions at risk of elimination. The potential layoffs will most likely...

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Acadia snaps up an essential tremor drug after field's summer setbacks

A few weeks after gaining about $100 million from a priority review voucher, Acadia Pharmaceuticals is putting its money to work by in-licensing an experimental essential tremor medicine. The San Diego...

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PTC reports Phase 2 ALS fail, sells priority review voucher for $150M

PTC Therapeutics is staying busy ahead of the Thanksgiving holiday. On Tuesday afternoon, the company announced that a Phase 2 trial evaluating its experimental ALS drug utreloxastat failed on the...

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Norbert Bischofberger's time as Kronos CEO ends as biotech seeks new life

Kronos Bio is letting go nearly all its employees after flashing red lights earlier this month. As part of an 83% workforce downsizing, CEO Norbert Bischofberger is heading out the door and handing the...

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Bristol Myers sues the federal government over 340B, following other drugmakers

Bristol Myers Squibb is the latest pharma company to sue the federal government over its rejection of a proposed new model for distributing 340B drug discounts. Under the drugmaker's plan, hospitals...

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Recursion reduces workforce; Oxford Nanopore teams up with UK Biobank

Plus, news about Portage Biotech: Recursion’s layoffs: After merging with fellow AI-driven biotech Exscientia this fall, Recursion Pharmaceuticals laid off less than 20% of the combined company, a...

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Outlook's shares crash as wet AMD drug flunks Phase 3 test

After the FDA rejected Outlook Therapeutics’ ophthalmic formulation of bevacizumab last year, the experimental therapy has now failed a late-stage study in patients with a degenerative eye disease. In...

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Advanz Pharma faces revocation of European authorization for liver disease...

The conditional marketing authorization for Advanz Pharma’s liver disease treatment Ocaliva has been revoked following a decision by the General Court of the European Union, the company announced...

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Novartis to cut 139 jobs in NJ as part of commercial refocus

Novartis is laying off 139 employees in East Hanover, NJ, as the company shifts its commercial resources away from two “well-established” medicines. The layoffs will mainly affect members of the...

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Satsuma's NDA filing after CMC woes; Hovione's Irish and US expansion

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FDA is looking into hematology risks after patients take Bluebird's Skysona...

The FDA said Wednesday that it is investigating "serious risk of hematologic malignancy" following the administration of bluebird bio's Skysona, a gene therapy approved for the rare disease known as...

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Applied Therapeutics' rare disease drug gets a CRL

The FDA rejected Applied Therapeutics’ experimental treatment for a rare disease called classic galactosemia, citing deficiencies with the application. The company, while “disappointed with the FDA’s...

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Cartesian tees up Phase 3 mRNA CAR-T study in myasthenia gravis

Cartesian Therapeutics reported longer-term results from a mid-stage study of its mRNA CAR-T therapy for myasthenia gravis, suggesting that the therapy is durable beyond the initial three-month study...

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Relay licenses lirafugratinib to Elevar for $75M

Relay Therapeutics said last year it would delay the regulatory filing of lirafugratinib in bile duct cancer. Now it's stepping away from the drug’s development altogether, having licensed the oral...

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Ipsen secures rights to T cell engager in $610M pact with Biomunex

Ipsen has inked yet another licensing deal in an effort to bolster its cancer pipeline, this time for a preclinical T cell engager developed by another Paris-based drugmaker. The French pharma company...

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Coherus makes final, $483M pivot away from biosimilars

Coherus BioSciences' stock surged on Tuesday after the company announced plans to sell off the last biosimilar in its portfolio. Intas Pharmaceuticals has agreed to purchase Coherus’ Neulasta...

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