Braveheart, AAVantgarde rounds head recent biofinancings
Our round-up of biotech financings is led this week by Braveheart Bio, a San Francisco biotech that has launched with $185 million, a lead drug for hypertrophic cardiomyopathy (HCM), and Biogen CEO Chris Viehbacher as its chair.
Braveheart's small molecule candidate, selective cardiac myosin inhibitor BHB-1893, could be a rival to Bristol Myers Squibb’s already marketed Camzyos (mavacamten) for obstructive HCM and Cytokinetics’ aficamten, under FDA review for obstructive HCM, with a decision due by 26th December.
According to Braveheart chief executive Travis Murdoch – the founder of HI-Bio, which was bought by Biogen for $1.15 billion last year – current treatments for HCM leave considerable room for improvement. BHB-1893 has the potential to be a "best-in-class molecule that enhances the efficacy, safety and convenience of care for patients and prescribers," he added.
The drug is in a phase 2 study in symptomatic obstructive HCM, a phase 2 study in non-obstructive HCM in Australia, and a phase 3 trial in obstructive HCM in China, where it is being developed by Jiangsu Hengrui Pharma. Braveheart intends to start its own late-stage trials in 2026.
The Series A was supported by an investor syndicate led by Andreessen Horowitz, Forbion, and OrbiMed, with participation from Enavate Sciences and Frazier Life Sciences.
Meanwhile, Italian biotech AAVantgarde also completed a nine-figure round, raising $144 million in a Series B that will support the development of its gene therapies for inherited retinal disorders (IRDs).
The Milan-based company has two clinical-stage candidates in development, AAVB-039 for Stargardt disease caused by a mutation in the ABCA4 gene and AAVB-081 for retinitis pigmentosa associated with Usher syndrome Type 1B caused by a MYO7A mutation, which are both in phase 1/2 trials. There are no approved therapies for either disease.
AAVantgarde's gene therapy platform enables the delivery of large genes to tissue and cells in vivo, according to the company. Its approach involves splitting the mRNA sequence to be delivered into two and then recombining it within the cell.
The new funding is earmarked for completing the proof-of-concept CELESTE trial of AAVB-039 and LUCE study of AAVB-081. IRDs are among the leading causes of blindness in children and young adults worldwide.
The round was led by Schroders Capital, alongside existing investors Atlas Venture and Forbion. Other new investors included Amgen Ventures, Athos Capital, CDP Venture Capital, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors.
The round also saw the return of previous backers Longwood Fund and Sofinnova Telethon Fund, which supported AAVantgarde's $71 million Series A in 2023.
Other sizeable rounds this week include a combined $82 million seed financing and Series A for Azalea Therapeutics, a new in vivo gene editing company led by CRISPR/Cas9 pioneer Jennifer Doudna and based in Berkeley, California. The company has been built around an Enveloped Delivery Vehicle (EDV) technology for directing CRISPR cargo to specific cells and tissues.
The financing – from Third Rock Ventures, RA Capital Management, Yosemite, Sozo Ventures, and other unnamed individual investors – will be deployed in advancing an in vivo CAR-T therapy for B-cell malignancies into clinical testing.
Finally this week, another Californian biotech – Palo Alto-based NEOK Bio – has emerged from the shadows with $75 million in Series A financing and a mission to develop bispecific antibody-drug conjugates (ADCs) for cancer.
The cash will help to fund the clinical development starting next year of two candidates, namely ROR1 and B7-H3-targeting NEOK001 and EGFR and MUC1-directed NEOK002, which both have potential across multiple tumour types, including thoracic, gastrointestinal, and gynaecological cancers. NEOK is backed by ABL Bio, a world leader in antibody engineering.
Photo by Miguel Bruna on Unsplash
