BBIO - Alnylam drives Intellia Ionis higher with amyloidosis data; BridgeBio slips

• Alnylam Pharmaceuticals ( ALNY ) made headlines across the biotech space on Wednesday after the RNAi therapeutics company announced that its ATTR amyloidosis therapy patisiran met the main goal in a Phase 3 trial.
• The much-awaited data readout propelled Alnylam ( ALNY ) ~55% higher, sparking a rally in the shares of its rivals focused on similar drugs.
• Notable gainers include Intellia Therapeutics ( NASDAQ: NTLA ) and Ionis Pharmaceuticals ( IONS ), both of which are advancing candidates for transthyretin amyloidosis (ATTR) in partnership with major pharmaceutical companies.
• In 2021, Intellia ( NTLA ) and its partner Regeneron ( REGN ), highlighted the potential of in-vivo CRISPR genome editing for the first time in humans with Phase 1 data for the CRISPR candidate NTLA-2001 in ATTR amyloidosis.
• In December, Ionis ( IONS ) received $200M upfront after the company partnered with AstraZeneca ( AZN ) to develop and commercialize the subcutaneous injection eplontersen for transthyretin amyloidosis.
• However, clinical-stage biotech BridgeBio Pharma ( BBIO ) lost ~8% in reaction to Alnylam ( ALNY ) data. In December, the company announced that its Phase 3 study for oral therapy acoramidis in transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) did not meet the primary endpoint.

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