Clinical hold weakens investors’ bond with Avidity

A clinical hold is not an ideal setup for first-in-patient data with a novel technology, but this is exactly what Avidity unveiled this morning. Enrolment has been halted into the phase 1/2 Marina trial of AOC 1001, the company’s lead antibody-targeted oligonucleotide for myotonic dystrophy type 1, after a single serious event. Avidity said the event was seen with 4mg/kg – the highest dose tested so far – and that the patient was at home undergoing further tests. The event is not thought to be related to the technology or DMPK knockdown, the target of AOC 1001. The company is focusing on finding the cause of the event, though its nature, timing and severity remain under wraps to "protect privacy". Lack of detail probably contributed to Avidity’s 23% share price plunge this morning, though executives also ducked questions about plans to escalate to an 8mg/kg dose. Investors also shrugged off the news that the FDA had cleared the start of clinical trials of two follow-on projects, which use the same technology as AOC 1001; under normal circumstances this could have provided a boost. Much now rests on disclosure of preliminary data from Marina's 1 and 2mg/kg dose cohorts, due in the coming weeks. 

Targeted oligonucleotides: Avidity's clinical pipeline 
Project Target  Disease  Status
AOC 1001 DMPK Myotonic dystrophy type 1 (DM1) On partial clinical hold on new enrolment to Marina; participants may roll over into open-label study
AOC 1020 DUX4 Facioscapulohumeral muscular dystrophy (FSHD) IND cleared and "advancing into the clinic"
AOC 1044 Exon 44 dystrophin Duchenne muscular dystrophy (DMD) IND cleared and "advancing into the clinic"
Source: company communication.

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