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Ionis wins rare disease approval Read in browser
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4 September, 2026
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top stories
1. Former I-Mab chief Joan Shen's NeuShen banks $80M+ for 3 brain-health drugs
2. Ionis wins FDA clearance for Alexander disease drug, company's first solo neurology approval
3. Thiel-backed Lindus pivots to hunting external drug assets after CRO-arm sale
4. Italy's Menarini gets into obesity with European rights to GLP-1 from China's Gan & Lee
5. Merck's M&A chief expects 'a lot more deal flow' as Keytruda patent cliff nears
6. Take the Q3 Biopharma Sentiment Survey (BPSI)
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Drew Armstrong
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Monday is Labor Day, so Endpoints won't be publishing any newsletters. We'll be back Tuesday — enjoy the long weekend!

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Drew Armstrong
Executive Editor, Endpoints News
@ArmstrongDrew
NeuShen CEO Joan Shen.
1
by Kyle LaHucik

A Shang­hai and Boston biotech led by a vet­er­an transpa­cif­ic drug de­vel­op­er has se­cured more than $80 mil­lion to ad­vance three clin­i­cal-stage brain-health drugs.

NeuShen...

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The China Biotech Debate: Washington, DC from POST-HOC
Secrecy, security and policy: round two of our China biotech series lands in Washington, with the policymakers who will decide the US posture toward its biggest rival. Seats are limited. Get your ticket now.
2
by Ayisha Sharma

Io­n­is Phar­ma­ceu­ti­cals got FDA ap­proval of its rare neu­rode­gen­er­a­tive dis­ease treat­ment zil­gan­ersen, paving the way for the com­pa­ny’s first whol­ly owned neu­rol­o­gy prod­uct.

Ion­is'...

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Endpoints webinars
Sep 10
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Inside the IDMO model: Analytical comparability, regulatory readiness and operational excellence
Cellares
Sep 16
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Beyond ICD codes: Unlocking rare disease insights with genomics and claims-based data
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Sep 22
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The future of drug pricing: Medicare negotiation, MFN models & beyond
ADVI
Oct 22
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A new model for clinical research – Expanding community trial access
Parexel
3
by Kyle LaHucik

A Pe­ter Thiel-backed transat­lantic start­up that once brand­ed it­self as an “an­ti-CRO” is piv­ot­ing from a cl