Avidity Biosciences (RNA) Q3 2024 earnings summary

Executive summary

• Advanced three clinical programs: del-desiran (DM1, Phase 3), del-brax (FSHD, Phase 1/2), and del-zota (DMD44, Phase 1/2), with positive 2024 data and regulatory designations for all three candidates.

• Enrollment in three registrational clinical trials is progressing, with the global Phase 3 HARBOR trial for del-desiran initiated and EXPLORE44 trial for del-zota fully enrolled.

• Pipeline expanded in skeletal muscle and precision cardiology, with new candidates and next-generation technology updates expected in late 2024.

• Global commercial infrastructure is being expanded, and a biomarker cohort for del-brax has been initiated with plans for an accelerated approval path.

• FDA removed the partial clinical hold on del-desiran in October 2024, allowing regulatory clearance to proceed.

Financial highlights

• Revenue for Q3 2024 was $2.3 million, down from $2.8 million in Q3 2023; nine-month revenue was $7.9 million, up from $7.4 million year-over-year, mainly from collaboration agreements.

• Net loss for Q3 2024 was $80.4 million, or $0.65 per share, compared to $52.4 million, or $0.71 per share, in Q3 2023; nine-month net loss was $220.0 million, or $2.08 per share, up from $151.8 million, or $2.11 per share, year-over-year.

• Research and development expenses rose to $77.2 million in Q3 2024 (from $47.7 million) and $208.0 million for the nine months (from $138.2 million), driven by clinical trial progression and higher manufacturing and personnel costs.

• General and administrative expenses increased to $23.3 million in Q3 2024 (from $13.7 million) and $57.9 million for the nine months (from $38.1 million), mainly due to higher personnel and professional fees.

• Other income rose to $17.7 million in Q3 2024 (from $6.3 million) and $37.9 million for the nine months (from $17.1 million), reflecting higher interest income.

Outlook and guidance

• Cash, cash equivalents, and marketable securities of $1.6 billion as of September 30, 2024, expected to fund operations for at least 12 months from the filing date.

• No product sales revenue expected until regulatory approval and commercialization; future funding needs may be met through equity, debt, or collaborations.

• Enrollment for the global Phase 3 HARBOR trial in DM1 is on track, and the functional cohort in the FORTITUDE study for FSHD is expected to begin in the first half of 2025.

• Additional DMD pipeline candidates are advancing, with Exon 45 in IND-enabling studies.

• Updates on precision cardiology and next-generation technology planned for November 2024.