Adverum, Silence each pocket ~$120M ahead of data readouts for genetic treatments
05 Feb 2024
Phase 2Gene TherapyPhase 1Clinical Result
A pair of genetic therapy developers scored big on Monday, each bringing in at least $120 million in private placements in advance of upcoming data for their respective lead programmes. Adverum Biotechnology is presenting data this week for its age-related macular degeneration (AMD)AMD) gene therapy, and Silence Therapeutics expects to announce topline results for its cardiovascular siRNA programme this quarter.
Adverum eyes gene therapy readout
As interest in AMD gene therapies and their potential to reduce the injection burden on patients continues to build, Adverum raised $127.5 through the sale of 106.3 million shares at $1.20 each. Adverum’s stock climbed 35% Monday after announcing the financing, which was led by TCGX with participation from new and existing investors including 5AM Ventures, Commodore Capital, Frazier Life Sciences, Logos Capital, Samsara BioCapital, Venrock Healthcare Capital Partners and Vivo Capital.
The private placement will provide the company runway into 2025 to advance its gene therapy pipeline, including lead candidate ixoberogene soroparvovec (ixo-vec), an AAV-based, intravitreally delivered treatment that encodes aflibercept to treat AMD.
At the Macula Society meeting on Thursday, Adverum is presenting preliminary efficacy and safety data from the Phase II LUNA study of ixo-vec to treat neovascular AMD. The presentation follows biomarker data from the same study released in September, which suggested therapeutic activity based on aflibercept expression levels.
Standard-of-care anti-VEGF therapies to treat AMD require frequent injections that can ultimately lead to vision loss. In a recent FirstWord poll fielded to 35 US ophthalmologists who treat patients with AMD, the majority expressed a desire for longer-lasting treatment options, as well as less frequent injections. For more, check out Physician Views Results: Regeneron’s Eylea comes out top against Roche’s Vabysmo in ophthalmologist poll.
Competing with Adverum to reduce injections with a gene therapy are Regenxbio and AbbVie, which announced wet AMDAMD data last month for ABBV-RGX-314, a NAV AAV8 vector that encodes an antibody fragment designed to inhibit VEGF. While ABBV-RGX-314 might offer a more convenient suprachoroidal injection, Mizhuo analyst Graig Suvannavejh noted that Adverum’s gene therapy is delivered via intravitreal injection, the proven, standard-of-care method. Previously announced data for ixo-vec in wet AMDAMD were “as good as, if not superior to, the data for ABBV-RGX-314,” Suvannavejh said, adding that Thursday’s “first look at both safety and efficacy in LUNA… could be key to de-risk not only ixo-vec, but also the stock.”
Silence prepares for CV silencing results
Silence Therapeutics raised $120 million through the sale of 5.7 million ADSs at $21 a piece, securing enough funding to prepare for a Phase III trial of its lead siRNA programme and push its second candidate through Phase II testing. The private placement’s investors include 5AM Ventures, Frazier Life Sciences, Logos Capital, Nextech Invest Ltd, Redmile Group, TCGX and Vivo Capital.
The company is planning to report this quarter topline data from the Phase II ALPACAR-360 study of zerlasiran (SLN360), a siRNA designed to lower production of lipoprotein(a), a genetic risk factor for cardiovascular (CV) disease.
In November, Silence released data from the APOLLO Phase I study in 36 adults with high baseline levels of lipoprotein(a) and stable atherosclerotic CV disease showing that two injections of zerlasiran resulted in lipoprotein(a) reductions up to 99% at 90 days following two repeated doses.
Silence said the financing will help progress partnering discussions for zerlasiran, as well as fund Phase III preparations. Additionally, the company will advance its second programme divesiran (SLN124) – which silences TMPRSS6 to treat polycythemia vera (PV) – through Phase II testing.
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