Biopharma Week in Review
We review last week’s biopharma news for meaningful clinical data, regulatory updates, research innovation, and M&A. Ainos, Inc. (AIMD) Director of Corporate Development Jack Lu recently joined us on the Water Tower Hour podcast. Our discussion is available on Apple Podcasts, Spotify, or on our website. Link to the podcast in our full report. For our AIMD podcast recap note and update note on the Veldona animal study, please see accessible links in our full report. Last week, IONS matched Ultragenyx’s Angelman benefit, with maybe better safety. PFE and SGMO had approvable gene therapy results in hemophilia A, but durability remains a question. BIIB had a bad week, as SAGE-partnered essential tumor trial failed and ESAIY-partnered lecanemab in Alzheimer’s hit an EU regulatory wall. VKTX got a surprising Phase 3 green light for obesity. DAWN secured an ex-US partner (IPSEY) for pediatric brain cancer. JAGX missed the primary in crofelemer Phase 3. CLRB update for pivotal radio-therapy study in WM dropped a few MRR percentage points, but data still supports approval. Ionis Pharmaceuticals, Inc. (IONS) presented compelling ION582 (UBE3A-ATS ASO) Phase 1/2 data in Angelman syndrome, showing clinically meaningful improvements across functional domain measures, including Bayley-4 cognition, for combined mid/high dose groups versus natural history at six months. IONS582 benefit looked similar to Ultragenyx’s Phase 1/2 data for GTX-102, also an UBE3A-ATS ASO. As both drugs approach Phase 3 trials, key differences to keep in mind include potentially better safety (GTX-102 had 5 SAEs of lower limb weakness versus none for IONS582) and broader patient population for IONS582 (UBE3-del and -mut for IONS582 versus UBE3-del only for GTX-102). IONS was up 6% last Monday. Pfizer Inc. (PFE) and Sangamo Therapeutics, Inc. (SGMO) announced giro-vec (FVIII gene therapy) Phase 3 AFFINE study met the primary endpoint of non-inferiority, but also showing superiority over standard FVIII prophylaxis in hemophilia A. Giro-vec reduced total ABR to 1.24 from 4.73 pre-fusion (1-sided p=0.004). Modified safety protocol showed 49% experienced elevated FVIII levels over 150%, which were transient and said to have no impact on efficacy or safety. Drug-related SAEs occurred in 13% of patients, but no details were disclosed. Results should support approval, but durability of benefit is needed to differentiate from Biomarin’s Roctavian. At 15 months, 83% of giro-vec patients had FVIII activity >5%. PFE was up 2% and SGMO was up 40% on Wednesday. SAGE, BIIB, ESAIY, VKTX, DAWN, IPSEY, JAGX, and CLRB news on page 2.
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