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ANNEXON biosciences Annexon Biosciences Announces Business and Program Highlights and Reports Third Quarter 2021 Financial Results November 9 , 2021 $ 271 Million Cash and Investments at End of Third Quarter 2021 Supports Operating Runway into 2024 , Including Several Key Clinical Milestones across the Portfolio SOUTH SAN FRANCISCO , Calif . , Nov. 09 , 2021 ( GLOBE NEWSWIRE ) -- Annexon , Inc. ( Nasdaq : ANNX ) , a clinical - stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement - mediated autoimmune , neurodegenerative and ophthalmic disorders , today announced recent highlights and reported third quarter 2021 financial results . " The third quarter marks another quarter of meaningful progress with our portfolio , including effectively advancing three clinical - stage product candidates in five clinical trials , " said Douglas Love , Esq . , president and chief executive officer of Annexon . " We're also pleased to have been granted Orphan Drug designation by the FDA for ANX005 , underscoring the Agency's recognition of ANX005's potential as a novel therapy for the treatment of Huntington's disease . Additionally , in collaboration with multiple academic and physician key opinion leaders , we conducted two C1q Series R & D Events that highlighted the applicability of our platform and differentiated scientific approach targeting the classical complement cascade in a range of autoimmune and neurodegenerative diseases . The combination of a rich set of milestones , a strong balance sheet , and a talented team , lays the foundation for a transformational year ahead . We look forward to sharing the continued progress across our portfolio , including initial data from our Huntington's disease clinical trial , as we work to bring treatments to patients suffering from serious complement - mediated diseases . " Program Highlights • Second C1q Series R & D Event Highlights Neurodegeneration Franchise : Annexon hosted its second C1q Series event , which spotlighted the company's neurodegeneration franchise and approach to treating complement - mediated neurodegenerative diseases . During the event , Annexon's leadership team and participating key opinion leaders shared their perspectives on : o The role of C1q and the classical complement pathway in neurodegenerative diseases , including Huntington's disease ( HD ) and amyotrophic lateral sclerosis ( ALS ) ; o Research on neurofilament light chain ( NfL ) and natural history data showing the association of a rise in NfL with clinical progression in both HD and ALS ; and • Internal data demonstrating the ability to reduce NfL with ANX005 , both clinically in Guillain - Barré syndrome ( GBS ) , and preclinically in HD and ALS . • Initial Data from Phase 2 trial of ANX005 in HD Expected in the Fourth Quarter of 2021 : Annexon is studying its lead compound , ANX005 , a clinical - stage investigational monoclonal antibody intended to treat patients with complement- mediated disorders , in a Phase 2 trial in patients with HD . The company anticipates initial data from the trial in the fourth quarter of 2021 , which is expected to include data from a subset of 16 patients who have completed the six - month treatment period , including safety and tolerability , C1q target engagement in cerebral spinal fluid and serum , and impact on NfL levels . The full dataset from all patients who complete both the six - month on - treatment and three - month off - treatment periods are expected to be reported in the first half of 2022 . • ANX005 Granted Orphan Drug Designation for the Treatment of HD : The U.S. Food and Drug Administration ( FDA ) has granted Orphan Drug designation for ANX005 for the treatment of patients with HD . Orphan Drug designation provides orphan status to drugs or biologics that are intended to treat rare diseases or disorders affecting fewer than 200,000 people in the United States , and certain incentives to the product sponsor , including tax credits for qualified clinical trials and fee waivers . The designation confers eligibility for seven years of market exclusivity following FDA product approval . Business Highlights • Leadership Team Strengthened by Appointment of Ted Yednock , Ph.D. , as Chief Innovation Officer and Larry Mattheakis , Ph.D. , as Chief Scientific Officer : In the third quarter , Annexon appointed Ted Yednock , Ph.D. , as chief innovation officer ( CIO ) , and Larry Mattheakis , Ph.D. , as chief scientific officer ( CSO ) . Dr. Ted Yednock served as Annexon's CSO since 2013. As CIO , he will serve as chairman of the Scientific Advisory Board and continue to enhance and integrate Annexon's key scientific findings and collaborations . Dr. Larry Mattheakis brings decades of drug discovery and development expertise to the CSO role , supporting the advancement of Annexon's current and future pipeline . Third Quarter 2021 Financial Results • Cash and Operating Runway : Cash and cash equivalents and short - term investments were $ 271.4 million as of September 30 , 2021. Annexon expects that its current cash position is sufficient to fund its operating plans into 2024 .