Arrowhead Completes Enrollment in Phase 3 YOSEMITE Study of Zodasiran for HoFH

Business Wire··Read original
3▲1 ▼0Impact / 5
Summary · why it matters

Arrowhead Pharmaceuticals has completed enrollment in its global Phase 3 YOSEMITE clinical trial of zodasiran for homozygous familial hypercholesterolemia, with study completion anticipated in mid-2027. The trial originally planned to enroll 60 participants but strong patient and physician interest led to a total of 70 patients enrolled. Zodasiran is an investigational RNA interference therapeutic designed to reduce ANGPTL3 production, offering a mechanism distinct from conventional LDL-C–lowering therapies. Arrowhead intends to seek regulatory approval in multiple geographies pending successful results.

Impact on assets 1

Biotech & Genomic Medicine▲ · 1 stocks
Arrowhead Pharmaceuticals Inc
ARWR
▲ PositiveTechnologyrelevance

Completed enrollment in Phase 3 YOSEMITE trial of zodasiran for HoFH, with strong interest leading to 70 patients enrolled.

Theme Impact 2

Related news

United States
▲

Therna Biosciences Names Kevin Green COO, Adds Two AI Advisors, Wins NIH TRDNT Challenge Phase I

Therna Biosciences appointed Kevin Green, former Chief Business Officer of Inceptive, as Chief Operating Officer and added two AI technical leaders to its advisory team, while the NIH Common Fund selected the company as a Phase I winner of its Targeting RNA in Disease with Novel Technologies Challenge. Green, who joined Therna in August and brings more than 20 years of experience across AI-enabled drug discovery and RNA therapeutics, leads the company's operations and business development as its RNA-Logix platform moves into partnered and internal programs. The new AI advisors are Dr. Dave Burke, the first Chief Technology Officer of Arc Institute and previously Vice President of Engineering for Android at Google, and Mr. Harsh Mehta, co-founder and CTO of Mirendil and previously a Senior Research Scientist at Anthropic and a researcher at Google. Therna is one of 25 Phase I winners of the three-phase TRDNT Challenge, which offers up to $13.1 million in total awards as teams develop and validate their technologies through 2027. Therna's winning proposal introduces a co-scientist agent built on RNA-Logix that designs and optimizes sequence-specific activating antisense oligonucleotides for ultra-rare haploinsufficiencies.
About megatrends
Biotech & Genomic Medicine › RNA Therapeutics ▲Technology
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides ▲Technology
Biotech & Genomic Medicine › AI Drug Discovery ▲Technology
Read original ↗
Business Wire·1dRead more →
United States

Wells 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.
About megatrends
Biotech & Genomic Medicine › Rare Disease ▲Competition
Biotech & Genomic Medicine › Neuroscience & Neurodegenerative ▲Competition
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides Competition
Biotech & Genomic Medicine › RNA Therapeutics ▲Competition
Read original ↗
Seeking Alpha·5dRead more →
United States

Kodiak 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.
About megatrends
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides Competition
Biotech & Genomic Medicine › RNA Therapeutics Competition
Biotech & Genomic Medicine › Biosimilars Competition
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.
Read original ↗
Simply Wall St·8dRead more →
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.
About megatrends
Biotech & Genomic Medicine › RNA Therapeutics ▲Capital
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides ▲Capital
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.
Read original ↗
Insider Monkey·8dRead more →
United States

Codexis 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.
About megatrends
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides Technology
CDXS · Demand · Positive Codexis signed an siRNA manufacturing agreement with a drug innovator to use its ECO Synthesis platform, a concrete customer deal.
Read original ↗
GlobeNewswire·9dRead more →
United StatesCanada

AbbVie 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.
About megatrends
Biotech & Genomic Medicine › Oncology Therapeutics ▲Regulation
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides Capital
Biotech & Genomic Medicine › Rare Disease Capital
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.
Read original ↗
Simply Wall St·11dRead more →