RNAi / Antisense Oligonucleotides

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.

Otsuka Pharmaceutical and U.S.-based Ionis Pharmaceuticals announced on the 22nd that their jointly developed treatment for hereditary amyotrophic lateral sclerosis, ulefnersen, met its primary goal in a late-stage clinical trial. In patients with FUS mutation ALS, a rare inherited form of ALS that damages the nerve cells controlling movement, ulefnersen improved function and extended survival compared with the placebo group. The drug reduced markers of nerve cell damage and slowed disease progression, and most side effects were mild or moderate, indicating a favorable safety profile. No approved treatment currently targets the genetic cause of FUS-ALS, and the two companies plan to discuss the results with the U.S. Food and Drug Administration and global health authorities as they explore a path toward accelerated approval. Separately, Otsuka Pharmaceutical has launched a global early access program for FUS-ALS patients unable to participate in the trial, allowing physicians to request access to ulefnersen before approval.

Roche Holding partner Ionis reported that sefaxersen met the primary endpoint in the Phase 3 IMAgINATION trial for IgA nephropathy, delivering a statistically significant reduction in proteinuria compared with placebo. The clinical win comes on top of a strong run for Roche Holding, with the share price up 8.32% over 90 days and 11.58% year to date, and a 1-year total shareholder return of 44.02%. Roche Holding now trades at CHF363.20, only about 3% below the average analyst target, yet screens at a roughly 59% discount to an intrinsic value estimate, while the most followed narrative fair value of CHF353.34 pegs the stock as 2.8% overvalued. The SWS DCF model points the opposite way, implying a future cash flow value of CHF893.70. The story could still change quickly if key Phase 3 programs disappoint or if biosimilar pressure on older blockbusters accelerates faster than expected.

Ionis Pharmaceuticals announced positive late-stage results from two partnered phase III programs. The FUSION study of ulefnersen in amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene met its primary endpoint assessing functional impairment and survival at 72 weeks, with Ionis reporting the results as statistically significant without disclosing supporting numbers; Otsuka Pharmaceutical, which holds worldwide commercialization rights licensed in 2024, plans to discuss the data with the FDA and other global health authorities regarding potential expedited regulatory submission pathways. Separately, the IMAgINATION study of sefaxersen in adults with primary immunoglobulin A nephropathy, conducted by Ionis' partner Roche, met its primary endpoint in a prespecified interim analysis, showing statistically significant and clinically meaningful reductions in proteinuria after 37 weeks, and will continue in a blinded manner to evaluate kidney function over two years with estimated glomerular filtration rate at week 105 as the longer-term measure. Roche licensed sefaxersen from Ionis in 2022 and is responsible for the phase III study and future global development, regulatory and commercialization activities, while Ionis is eligible for milestone payments and tiered royalties on net sales of both drugs. The two wins follow back-to-back cardiovascular setbacks for Ionis, including the phase III CARDIO-TTRansform failure of Wainua with AstraZeneca in July and the phase III Lp(a)HORIZON miss for pelacarsen with Novartis, and come after the FDA approval of Zanvastro for Alexander disease earlier this month. Year to date, Ionis shares have lost 42% compared with the industry's 2% decline.

Roche announced positive prespecified interim results from the ongoing phase III IMAgINATION study of investigational sefaxersen in adults with primary IgA nephropathy, with the study meeting its primary endpoint of statistically significant and clinically meaningful proteinuria reduction versus placebo at 37 weeks as measured by 24-hour urine protein-to-creatinine ratio. Sefaxersen is a once-monthly subcutaneous injection designed for self-administration that inhibits complement factor B production in the liver, and its safety and tolerability profile was consistent with previously reported data with no new safety signals. The IMAgINATION study enrolled 459 people randomized 1:1 to receive sefaxersen or placebo for 105 weeks and will continue blinded to evaluate change in kidney function over two years as measured by estimated glomerular filtration rate at week 105. Interim data will be presented at an upcoming medical congress and shared with health authorities. Roche licensed sefaxersen from Ionis for the treatment of complement mediated diseases.

ADARx Pharmaceuticals said on Monday it is targeting a valuation of up to $1.74 billion in its US initial public offering, joining a wave of biotech companies planning listings. The San Diego-based developer of RNA-based drugs for rare diseases aims to raise up to $371.9 million by offering 21.9 million shares priced between $15 and $17. AbbVie has agreed to invest up to $100 million in a concurrent private placement that would give it a roughly 4.9% stake post-IPO, adding to the $335 million it paid ADARx in May 2025 for a research collaboration. ADARx's most advanced candidate, onvuzosiran, is in late-stage testing for hereditary angioedema, a rare genetic disorder that causes sudden swelling attacks, and three of its programs are in clinical testing while two are in advanced preclinical development. JPMorgan, Morgan Stanley and TD Cowen are among the underwriters, and the shares will list on the Nasdaq under the symbol ADRX.

Arrowhead Pharmaceuticals announced that its drug plozasiran significantly reduced triglyceride levels and acute pancreatitis events in two Phase 3 studies, SHASTA-3 and SHASTA-4, involving patients with severe hypertriglyceridemia. The trials met primary and secondary endpoints, with median triglyceride reductions of 79% and 81% after 12 months. A pooled analysis showed a 78% reduction in acute pancreatitis events, with a 91% reduction in high-risk patients. The company plans to file a supplemental New Drug Application with the FDA by end of 2026, using a Priority Review Voucher. Plozasiran, marketed as Redemplo, is already approved for familial chylomicronemia syndrome and uses RNA interference to suppress APOC3 production.

Alnylam Pharmaceuticals presented new data at the European Society of Cardiology Congress 2026, reinforcing the strength of RNAi-powered TTR silencing across ATTR-CM patient populations and treatment settings. A late-breaking prespecified subgroup analysis of the HELIOS-B Phase 3 trial showed that vutrisiran provided consistent clinical benefit on all-cause mortality and recurrent cardiovascular events in patients with or without tafamidis use at baseline, among 654 randomized patients, 40% of whom were on tafamidis. Additional post hoc analyses highlighted vutrisiran's positive impact on multisystemic manifestations, including a 25% less decline in intrinsic capacity and a 52% reduction in risk of decline. A pooled analysis of 1,402 patients across four Phase 3 studies of vutrisiran and patisiran showed consistent treatment effects across sexes. New subgroup data from the KARDIA-3 Phase 2 study of zilebesiran suggested potential for enhanced blood pressure control, particularly in patients on background diuretics with elevated systolic BP. These findings support the ongoing Phase 3 ZENITH trial for zilebesiran, co-developed with Roche.

Arrowhead Pharmaceuticals, Inc. (ARWR) reached a major European milestone on August 17 when it announced the availability of REDEMPLO for adults with Familial Chylomicronemia Syndrome (FCS) in Germany, following European Commission authorization in June 2026. The drug is now listed in the Lauer-Taxe database for prescribing and reimbursement, and can be prescribed once every three months based on clinical symptoms or genetic tests. This launch expansion comes shortly after Arrowhead reported its fiscal Q3 2026 financial results on August 4, with quarterly revenue surging 171% year-over-year to $75.3 million, beating Wall Street expectations of $52.7 million, driven by collaboration income and doubling REDEMPLO U.S. prescription volume. Net loss widened to $194.2 million ($1.36 per share) due to R&D and commercial launch investments, but total cash resources remained strong at $1.56 billion. On August 6, H.C. Wainwright raised its price target on Arrowhead to $120 from $115, reiterating a Buy rating, citing a one-quarter pull-forward in the plozasiran launch timeline for severe hypertriglyceridemia (SHTG) and a new partnership with Madrigal. The bull case is supported by pivotal Phase III efficacy, durable reductions in triglycerides and acute pancreatitis events, and a $1.57 billion cash balance, while the bear case centers on persistent unprofitability, with quarterly operating losses of $170.1 million in Q3, and the commercial ramp remains unproven, with REDEMPLO generating just $2.4 million in early sales. Investors should watch the full presentation of SHASTA data at the ESC Congress on August 30 and the planned sNDA filing for plozasiran before year-end 2026, alongside initial REDEMPLO reimbursement adoption metrics in Germany.

Arrowhead Pharmaceuticals, Inc. today announced that it will present important new data at the European Society of Cardiology Congress (ESC) in Munich, Germany. Detailed results from the Phase 3 SHASTA-3 and SHASTA-4 studies of plozasiran in patients with severe hypertriglyceridemia will be presented as a HOT LINE Late-Breaking Science Session on August 30, 2026. The company will also host a webcast to review ESC data and hold a Q&A on August 31, 2026. Additionally, a presentation on a Phase 3 clinical trial of vsa003 (zodasiran) in Chinese adolescents and adults with homozygous familial hypercholesterolemia is scheduled for August 31, 2026.

Youcare Pharmaceutical announced that its subsidiary Beijing Youcare Kechuang Pharmaceutical Technology Co., Ltd. recently received a letter from the US FDA agreeing to the clinical trial application for YKYY033 injection for the prevention and treatment of arterial and venous thrombosis. YKYY033 is a double-stranded siRNA drug conjugated with a GalNAc ligand that silences the mRNA transcribed from the coagulation factor XI gene through RNA interference, inhibiting the production of FXI protein. Preclinical studies have shown good safety and tolerability, and it has demonstrated significant antithrombotic efficacy in multiple animal models without increasing bleeding risk. The product received clinical trial approval from the NMPA for multiple anticoagulation-related indications in June and July 2026, and Phase I clinical trials are progressing steadily.

Silence Therapeutics and AbCellera Biologics reached 52-week highs on Monday after releasing positive clinical trial results. Silence Therapeutics shares jumped over 29% to $17.58 after reporting that its siRNA candidate Divesiran met the primary endpoint in a Phase 2 trial for Polycythemia vera, with response rates of 93% and 81% in two dosing groups. AbCellera Biologics stock climbed more than 34% to $10.01 after its drug ABCL635 met the primary endpoint in a Phase 2 trial for vasomotor symptoms associated with menopause, reducing moderate-to-severe symptoms in about 83% of patients after a single dose. Other biotech stocks hitting 52-week highs included Cumberland Pharmaceuticals, Celldex Therapeutics, and Biodesix, driven by quarterly results and pipeline updates.

Arrowhead Pharmaceuticals reported a net loss of $194.3 million, or $1.36 per share, for the fiscal 2026 third quarter ended June 30, 2026, while revenue rose to approximately $75 million from $28 million in the prior year quarter. The company highlighted positive Phase III results from its SHASTA-3 and SHASTA-4 studies, which met all primary and secondary endpoints with median triglyceride reductions of 79% and 81%, and a 78% reduction in acute pancreatitis events in the broad SHTG population. Commercial revenue from Redemplo reached approximately $2.4 million, more than double the prior quarter, and the company expanded regulatory approvals to five geographies including the EU, Australia, Canada, China, and the U.S. Operating expenses increased to approximately $245 million, driven by a $36 million rise in R&D to $198 million and a $16 million increase in SG&A to $47 million. The acquisition of a priority review voucher for $215 million represents a significant cash outlay, with payment expected in the fiscal fourth quarter following HSR clearance, aimed at potentially accelerating FDA review of the SNDA for SHTG. Arrowhead ended the quarter with approximately $1.6 billion in cash and investments and 141.1 million common shares outstanding.

CAMP4 Therapeutics announced the closing of the second tranche of its previously announced private placement, receiving approximately $50.1 million in gross proceeds. The second closing involved the sale of 10,756,498 shares of common stock at $1.53 per share, 39,306 shares at $1.65 to certain directors, employees and consultants, and 21,925,368 pre-funded warrants at $1.5299 each. Investors in this tranche included Coastlands Capital, Janus Henderson Investors, Balyasny Asset Management, Vivo Capital, 5AM Ventures, Adage Capital Management LP, Trails Edge Capital Partners and CURE SYNGAP1. The company intends to use the net proceeds to advance CMP-002, its lead investigational antisense oligonucleotide therapeutic candidate for SYNGAP1-related disorder, which recently received clearance in Australia to begin a Phase 1/2 clinical trial, and to support its early-stage pipeline. Leerink Partners acted as lead placement agent, with Piper Sandler & Co., Cantor Fitzgerald & Co. and Wedbush Securities Inc. as co-placement agents.

Alnylam Pharmaceuticals shares climbed 6.3% after the company presented new clinical data for its Alzheimer’s disease RNAi candidates mivelsiran and ALN-5288 at the Alzheimer’s Association International Conference in London, alongside reporting its first-ever quarter exceeding US$1.00 billion in product revenue. The data included long-term safety findings and robust cerebrospinal fluid biomarker reductions in early-onset Alzheimer’s disease. Alnylam also completed enrollment in the cAPPricorn-1 study in cerebral amyloid angiopathy and initiated the APPlauDS trial in Down syndrome-associated Alzheimer’s disease, highlighting an expanding neuroscience portfolio. The billion-dollar revenue quarter demonstrates sufficient cash flow to finance ambitious neuroscience programs, potentially reducing reliance on the TTR franchise over time. Analysts note that the most bullish projections already assume about US$11.9 billion of revenue and US$3.6 billion of earnings by 2029, and this early Alzheimer’s data could further shift expectations.

Alnylam Pharmaceuticals exceeded $1 billion in quarterly product revenue for the first time in its history during the first quarter of 2026, reporting $1.036 billion in net product revenue, a 121% year-over-year increase, on total revenue of $1.17 billion. AMVUTTRA alone contributed $890 million, driving the TTR franchise to $910 million, while the rare disease franchise added $126 million. The company swung to a GAAP net income of $206 million from a prior-year loss, yet its forward P/E ratio has compressed to around 32.5 times as earnings growth outpaced stock price appreciation. Institutional support dipped, with 54 top funds holding long positions at quarter-end, down from 67 in the prior quarter, and short interest stands at 4.36% of the public float.

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.

RTTNews highlights five biotech and medical device stocks that generated returns of 86% to 327% since being featured on its website. Arrowhead Pharmaceuticals surged 222% to a 52-week high of $95.49 following FDA approval of its lead drug Redemplo and positive clinical data. Compass Pathways climbed 327% to a 3.5-year high of $15.40 after reporting positive Phase 3 results for its psilocybin therapy COMP360 in treatment-resistant depression. CG Oncology rose 148% to an all-time high of $77.00 on the back of encouraging data for its bladder cancer candidate Cretostimogene. Alpha Tau Medical gained 182% to a 52-week high of $14.11 driven by regulatory progress and promising clinical results for its Alpha DaRT cancer therapy. Electromed advanced 86% to a 52-week high of $47.40 supported by strong quarterly revenue and profit growth.

Zhongchao Inc. led healthcare gainers on Wednesday with a 191.84% surge to $4.29 following a 1-for-3 reverse share split to maintain Nasdaq compliance. Lakewood-Amedex Biotherapeutics advanced 83.33% to $3.41 after reporting that its antimicrobial candidate Nu-3 aligns with PACCARB goals by destroying bacterial membranes, positioning it as a promising treatment for antibiotic-resistant infections and infected diabetic foot ulcers. Arrowhead Pharmaceuticals jumped 19.03% to $88.70 after its Phase 3 SHASTA-3 and SHASTA-4 trials of Plozasiran met the primary endpoint of triglyceride reduction and showed a statistically significant reduction in acute pancreatitis in severe hypertriglyceridemia patients, with an sNDA planned by year-end. LB Pharmaceuticals gained 10.93% to $35.84 after accelerating the topline data readout for its Phase 3 NOVA-2 trial of LB-102 in schizophrenia to the first half of 2027. Other notable movers included Advanced Biomed up 73.02% on no specific news, Satellos Bioscience up 14.72%, and DarioHealth up 12.86% after pricing a $23.5 million registered direct offering.

Arrowhead Pharmaceuticals announced positive late-stage trial results for its lipid-lowering drug plozasiran in patients with severe hypertriglyceridemia, sending its shares up 18% while rival Ionis Pharmaceuticals fell nearly 5%. Plozasiran met the primary endpoint and all prespecified secondary endpoints in the Phase 3 SHASTA-3 and SHASTA-4 trials, achieving median triglyceride reductions of 79% and 81% at month 12, respectively, compared to roughly 27% for placebo. No new safety events were observed, and the incidence of treatment-emergent adverse events was consistent with prior study data. Arrowhead plans to submit a supplemental new drug application to the FDA before the end of this year for a label expansion in severe hypertriglyceridemia. Ionis is advancing a competing therapy, olezarsen, which is under FDA priority review with a decision expected by June 30.