Arrowhead Pharmaceuticals IncARWR
▲ PositiveRegulationrelevance
EU approval for REDEMPLO, first siRNA treatment for familial chylomicronemia syndrome
Impact on assets 1
Biotech & Genomic Medicine
Arrowhead Pharmaceuticals IncTheme Impact 2
Off-coverage companies
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United StatesWells Fargo Starts Design Therapeutics at Overweight on Friedreich Ataxia Program
Wells Fargo initiated coverage of Design Therapeutics with an overweight rating, citing the company's Friedreich ataxia candidate DT-216P2 as having potentially best-in-disease functional improvement based on results from the RESTORE-FA study released in May. The bank set a $26 price target, implying roughly 112% upside based on the October 1 close. Analyst TianQi Hang wrote that the May update showed pharmacokinetics look good, and that blood-FXN protein, muscle-mRNA data, plus an early mFARS signal further de-risk the platform. Hang estimates that the blood FXN protein increase seen after 6 weeks can translate to at least a 2-point mFARS change, and said that if the drug kinetics sustain for 12 weeks, which he believes they will, DT-216P2 could deliver best-in-disease functional benefits. He assigns DT-216P2 a 60% probability of success, with peak sales of approximately $600M in the US and approximately $900M outside it. If approved, DT-216P2 would compete against Biogen's Skyclarys, also known as omaveloxolone, and Hang sees it gaining a peak share of the FA treatment market of 30% in the US and 20% ex-US.
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United StatesKodiak Phase 3 Eye Drug Data Matches Eylea, Pressuring Regeneron
Kodiak Sciences reported new Phase 3 data showing its eye drugs Zenkuda and tabirafusp alfa tedromer matched Eylea on primary endpoints for macular degeneration treatment, putting fresh competitive pressure on Regeneron Pharmaceuticals. The trial reported similar vision outcomes with Kodiak's candidates given roughly every six months versus Eylea's current eight week dosing interval. Regeneron, a US biotech group with a US$78.7b market cap, relies on Eylea as a key product in eye disorders, and the company's investor narrative already flags heavy reliance on Eylea and intensifying branded and biosimilar competition as a key risk to revenue sustainability. Kodiak plans to move closer to potential FDA filings in the fourth quarter of 2026, and the clearest early sign of impact will be Eylea and Eylea HD sales trends, with quarterly volume and pricing data in retinal disorders showing whether eye specialists actually switch away from Regeneron. Attention also remains on how quickly Regeneron can shift its mix toward Dupixent, Eylea HD and newer areas like oncology and obesity.
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KOD · Technology · Positive Phase 3 data show Zenkuda and tabirafusp alfa tedromer matched Eylea on primary endpoints with six-month dosing, advancing toward potential FDA filings.
REGN · Competition · Negative Kodiak's candidates matched Eylea with less frequent dosing, adding competitive pressure to Regeneron's key Eylea franchise.
United States▲
ADARx Targets $1.74 Billion Valuation in US IPO Backed by AbbVie
ADARx Pharmaceuticals is seeking a valuation of as much as $1.74 billion in a US IPO that could raise up to $371.9 million. The San Diego-based RNA drug developer plans to offer 21.9 million shares at between $15 and $17 apiece and list on the Nasdaq under the ticker ADRX. AbbVie has agreed to invest up to $100 million through a concurrent private placement, which ADARx said would give AbbVie a roughly 4.9% stake following the IPO; AbbVie had previously paid ADARx $335 million in May 2025 as part of a research collaboration. Three of ADARx's experimental drugs are in clinical testing and another two are in advanced preclinical development, with its most advanced candidate, onvuzosiran, in late-stage testing for hereditary angioedema. IPOX Research Associate Lukas Muehlbauer described the AbbVie investment as external validation of ADARx's technology, though the valuation still rests on experimental medicines with no approved product.
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ABBV · Capital · Positive AbbVie agreed to invest up to $100 million in ADARx's IPO via concurrent private placement, adding to its prior $335 million research collaboration.
United StatesCodexis Signs siRNA Manufacturing Agreement with Drug Innovator
Codexis, Inc. announced an agreement with a pioneering siRNA drug innovator to explore stereo-defined fragment synthesis using its ECO Synthesis Manufacturing Platform. Under the agreement, Codexis will use the platform to produce stereo-defined oligonucleotide fragments through a fully enzymatic process, which the innovator will assemble into the final duplex via ligation. The collaboration will also compare enzymatically synthesized fragments with conventional solid-phase oligonucleotide synthesis, evaluating purity, product quality, and ligation performance, with results allowing the innovator to assess the technology's potential for future clinical development programs. The agreement builds on advances presented at TIDES USA 2026, where Codexis for the first time demonstrated full-length siRNA synthesis with precise control of phosphorothioate stereochemistry via StereoSelect, a capability of ECO Synthesis. Alison Moore, President and CEO at Codexis, said the agreement is an important next step in bringing the company's latest ECO Synthesis capabilities to a customer.
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CDXS · Demand · Positive Codexis signed an siRNA manufacturing agreement with a drug innovator to use its ECO Synthesis platform, a concrete customer deal.
United States
CanadaAbbVie Takes ADARx IPO Stake as EPKINLY Wins Canada Approval
AbbVie acquired a stake in ADARx Pharmaceuticals during its IPO, gaining exposure to RNA interference drug candidates being developed for certain rare disease indications. Separately, Health Canada granted marketing authorization for AbbVie's EPKINLY for adults with relapsed or refractory follicular lymphoma. The ADARx investment signals that AbbVie is still willing to write cheques to secure optionality in newer modalities like RNA interference rather than relying only on in-house discovery, while the EPKINLY approval pushes the oncology franchise further into later-line hematology care. Both moves feed into AbbVie's broader effort to replace revenue from declining products such as Humira and Imbruvica, though analysts have flagged the company's continued concentration in a small cluster of major therapies as a risk around patent cliffs and pricing pressure.
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ABBV · Capital · Positive AbbVie acquired a stake in ADARx Pharmaceuticals during its IPO, gaining exposure to RNA interference drug candidates.
ABBV · Regulation · Positive Health Canada granted marketing authorization for AbbVie's EPKINLY for relapsed or refractory follicular lymphoma.
United States
Switzerland▼impact 4
Novartis Pelacarsen Phase 3 Failure Reshapes Lp(a) Race for CRISPR and Ionis
Novartis AG's Phase 3 Lp(a)HORIZON trial of pelacarsen failed to produce a statistically significant reduction in major cardiovascular events despite significantly lowering lipoprotein(a), a setback Citi says carries implications for other Lp(a)-lowering developers. Citi believes the result increases the likelihood that CRISPR Therapeutics prioritizes its next-generation CTX321 program over the earlier candidate CTX320, which has generated Lp(a) reductions of as much as 73% during dose escalation; CTX321 uses an updated guide RNA that showed approximately twice the potency of CTX320 in preclinical testing, and CRISPR expects to provide a program update in 2026. Citi retained a Buy rating and an $88 price target on CRISPR Therapeutics. On Ionis Pharmaceuticals, which discovered pelacarsen and licensed it to Novartis in 2019 for worldwide development and commercialization, Citi analyst Eric Joseph expects less than 5% of immediate downside because investor expectations were already modest, and the firm does not expect the result to affect Ionis' fiscal 2026 guidance; Citi maintained a Buy rating and a $100 price target on Ionis. The broader concern is that pelacarsen's failure to translate Lp(a) reduction into fewer cardiovascular events raises questions about how much Lp(a) must be lowered, how long patients must be treated, and whether different therapeutic approaches can deliver better clinical outcomes, leaving CTX321's greater preclinical potency unproven in humans.
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NOVN.SW · Technology · Negative Novartis' Phase 3 Lp(a)HORIZON trial of pelacarsen failed to significantly reduce major cardiovascular events despite lowering Lp(a).
CRSP · Technology · Positive Citi says pelacarsen's failure increases likelihood CRISPR prioritizes its more potent next-gen CTX321 Lp(a) program, and retains Buy/$88 PT.
IONS · Technology · Negative Ionis discovered and licensed pelacarsen to Novartis, and its Phase 3 failure raises doubts about the Lp(a) approach, though Citi sees under 5% immediate downside.