Almost every drug we know works on a “protein” that's already been built — it binds it, blocks it, plugs it. But this class is smarter than that. It goes one step further upstream, intercepting the “work order” (mRNA) before the bad protein is ever made — silencing the gene at the root of the disease, right at the source. This chapter shows how it works at the molecular level, why a shot given twice a year is changing the game for chronic disease, who the real leaders are, and why “getting the drug somewhere farther than the liver” is the wall the whole field is trying to climb.
Novartis and Ionis Lp(a) Drug Pelacarsen Fails Late-Stage Cardiovascular Trial
Pelacarsen, an experimental Lp(a)-lowering drug from Novartis AG and partner Ionis Pharmaceuticals, Inc., failed to reduce heart attacks, strokes and related cardiovascular events in the Phase III Lp(a)HORIZON trial, which enrolled more than 8,000 patients, Reuters reported on September 5, 2026. The drug did lower Lp(a) levels as expected, but that reduction did not translate into fewer real-world cardiac events. Novartis shares fell 5% and Ionis shares fell 12% in aftermarket trading. Novartis called the results a disappointment but said they still advance scientific understanding of the Lp(a) pathway. The failure removes a major potential growth driver for both companies and raises the burden of proof for other Lp(a) drugs, with Novartis now under greater pressure to replace revenue as Entresto and other products approach patent challenges. Ionis faces greater concentration risk because pelacarsen mattered more to the smaller company, though its broader RNA portfolio and newly approved Zanvastro for Alexander disease provide some diversification.
UnitedHealth sells Optum Florida stake to TPG as CooperCompanies cuts guidance and Amgen slides
UnitedHealth has sold an interest in some of its Optum Health operations in Florida to private equity firm TPG, part of the health conglomerate's effort to recover from a collapse in profits last year. CFO Wayne DeVeydt told Bloomberg News that Optum Health margins will be around 2% this year, above prior expectations, and should rise to around 4% in 2027 and 6% the following year. Amgen fell more than 8%, its worst single-day decline since 2016, after Novartis announced a Phase 3 trial failure for the heart disease therapy pelacarsen, which it is developing with Ionis Pharmaceuticals; BMO Capital Markets downgraded Amgen to Market Perform from Market Outperform with a $450 price target. CooperCompanies dropped 13% after issuing fiscal 2026 guidance below consensus, with revenue of $4.229B-$4.252B versus the prior $4.285B-$4.321B and non-GAAP diluted EPS of $4.51-$4.55 versus $4.58-$4.66 previously, and said its board decided to keep CooperSurgical rather than sell it while raising its share buyback authorization to $3B from $2B. Novo Nordisk fell more than 1% premarket after Morgan Stanley downgraded the stock to Underweight from Equal-weight, citing the semaglutide patent cliff, and the S&P 500 Health Care Sector Index slipped more than 3.5% for the week.
Royalty Pharma plc disclosed that pelacarsen failed the Phase 3 Lp(a)HORIZON cardiovascular-outcomes trial run by Novartis AG, which enrolled 8,323 patients with elevated lipoprotein(a) and established cardiovascular disease. The drug lowered Lp(a) but did not meet the primary endpoint of reducing cardiovascular events versus placebo in the overall population, and complete results have not yet been presented. Royalty Pharma had provided Ionis Pharmaceuticals $500 million in January 2023, allocating $150 million to pelacarsen royalties and $350 million to Spinraza royalties, and it now expects to pay no pelacarsen milestone payments out of up to $625 million in potential regulatory and commercial milestones. The company holds 25% of Ionis's Spinraza royalties through 2027, rising to 45% in 2028 on Spinraza annual sales of up to $1.5 billion, with that interest reverting after aggregate payments reach $550 million, or 1.1 times the original funding. Management expects the structure to recover the entire investment and generate a modest positive return, and it maintained its 2030 target of at least $4.7 billion in company-defined Portfolio Receipts.
Arrowhead's plozasiran cuts pancreatitis events in Phase 3 trials
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's Plozasiran Launch and Q3 Results Could Drive Re-rating
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.
Alnylam shares fall on rival trial data, analysts see core business safe
Alnylam Pharmaceuticals shares fell nearly 4.9% in pre-market trading after full data from AstraZeneca and Ionis Pharmaceuticals' Phase 3 CARDIO-TTRansform trial showed that combining their drug Wainua with TTR stabilizers, the standard of care dominated by Pfizer's Vyndaqel, was associated with worse outcomes than standard care alone, with a primary-endpoint relative risk of 1.14, while Wainua alone had a relative risk of 0.71. Analysts are debating whether this signals a broader flaw in combining RNA silencers with stabilizers, with Jefferies' Faisal Khurshid seeing limited benefit for silencers in a stabilizer-treated population and Stifel's Paul Matteis calling the combination outcome clearly worse than expected, while Oppenheimer's Kostas Biliouris notes that the negative signal has not been observed with Alnylam's siRNA platform. Despite the pipeline concerns, analysts agree that Alnylam's commercial foundation is secure because real-world Amvuttra use is predominantly monotherapy in treatment-naïve patients, and Stifel reiterates confidence in Amvuttra reaching $10 billion in peak sales.
Arrowhead Pharmaceuticals to Present New Clinical Data at European Society of Cardiology Congress
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.
Silence Therapeutics prices upsized $175 million ADS offering at $13.50 each
Silence Therapeutics priced an upsized underwritten public offering of 12.96 million American Depositary Shares at $13.50 each, raising approximately $175 million in gross proceeds. The underwriters have a 30-day option to purchase up to an additional 1.94 million ADSs at the offering price. The offering is expected to close on or about August 13, 2026. Silence Therapeutics is a clinical-stage biotechnology company developing short interfering RNA therapies. The stock price traded marginally on Tuesday after hours.
Youcare Pharmaceutical Subsidiary's YKYY033 Injection Receives US FDA Clinical Trial Approval
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 Surge on Positive Trial Data
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.
Silence Therapeutics shares jumped 16.2% in pre-market trading on Monday as investors positioned ahead of a conference call covering topline results from the Phase 2 SANRECO trial of divesiran, the company's lead siRNA candidate for polycythemia vera. The clinical-stage biotechnology company announced over the weekend that it would present and discuss the results through a conference call and webcast scheduled for 8:00 a.m. EDT on Monday. The Phase 2 SANRECO study enrolled 48 patients with phlebotomy-dependent polycythemia vera in a randomised, double-blind, placebo-controlled trial, with divesiran evaluated using two dosing schedules and the primary endpoint assessing the proportion of patients able to maintain haematocrit below 45% without requiring phlebotomy between weeks 18 and 36. Investor expectations have been supported by encouraging Phase 1 data showing required phlebotomy procedures fell from 80 during the six months before treatment to just five during the active treatment period, and divesiran has also received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration. The broader equity market offered little explanation for the scale of Monday's move, with the Nasdaq up around 0.4% and the S&P 500 gaining just 0.1%, indicating the advance was predominantly linked to the upcoming clinical update.
Arrowhead Pharmaceuticals Reports $194.3 Million Net Loss, Acquires Priority Review Voucher for $215 Million
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 closes second tranche of $100 million private placement
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 lowers 2026 revenue guidance as second-line demand normalizes
Alnylam Pharmaceuticals lowered its full-year 2026 total net product revenue guidance to a range of $4.7 billion to $5.1 billion, driven by a $200 million reduction at the midpoint in its TTR franchise guidance, now set at $4.2 billion to $4.5 billion. The revision reflects a normalization of second-line demand for AMVUTTRA after early growth was boosted by pent-up demand from patients switching from older therapies. Despite the cut, the company reported total net product revenues of $1.2 billion in the second quarter, a 74% increase year-over-year, with TTR net product revenues surpassing $1 billion for the first time. First-line patient starts now account for approximately 80% of new treatment initiations, and the company expressed increased confidence in its long-term outlook, citing favorable competitive developments including the delay of generic tafamidis entry in the U.S. until mid-2031 and the failure of a competing antisense oligonucleotide trial. Alnylam also announced a collaboration with BeOne for exclusive AMVUTTRA commercialization rights in Mainland China and Macau, and highlighted ongoing enrollment in the Phase III TRITON-CM trial for its next-generation RNAi therapeutic nucresiran, with a projected launch in 2030.
Ionis Pharmaceuticals beats Q2 earnings and sales estimates, reiterates 2026 outlook
Ionis Pharmaceuticals reported a narrower-than-expected adjusted loss and higher revenues for the second quarter of 2026, while reaffirming its full-year guidance. The company posted an adjusted loss of 43 cents per share, beating the Zacks Consensus Estimate of a loss of 89 cents, and revenues of $268 million, surpassing the $190.7 million estimate. Total revenues fell nearly 41% year over year due to a $280 million upfront payment from Ono Pharmaceutical in the prior-year period, but excluding that payment, revenues rose 56%. Ionis reiterated its 2026 revenue guidance of $875 million to $900 million, with Tryngolza net product sales expected between $100 million and $110 million and Dawnzera between $110 million and $120 million, and maintained its adjusted operating loss forecast of $425 million to $475 million.
Alnylam Falls 29% After Lowering Full-Year TTR Revenue Guidance
Alnylam Pharmaceuticals shares plunged 29% after the company lowered its full-year TTR revenue guidance, overshadowing strong second-quarter results. The stock dropped $82.62 to $204.00 on the Nasdaq. Second-quarter total revenue surged 67% to $1.29 billion, with TTR franchise revenue up 89% to $1.03 billion, and the company swung to a net income of $164.5 million from a year-earlier loss. However, Alnylam cut its 2026 TTR net product revenue guidance to $4.2 billion to $4.5 billion from $4.4 billion to $4.7 billion, citing slower-than-expected normalization of second-line demand for AMVUTTRA in the U.S. ATTR-CM market after an initial launch period boosted by pent-up demand.
Alnylam Shares Rise 6.3% After Alzheimer’s RNAi Data and First $1 Billion-Plus Revenue Quarter
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 is scheduled to report second-quarter 2026 earnings on Thursday, July 30th, before market open. The consensus earnings per share estimate is $2.15, compared with $0.32 in the same quarter last year, and the consensus revenue estimate is $1.32 billion, a 70.6 percent year-over-year increase. Over the past two years, the company has beaten EPS estimates 75 percent of the time and revenue estimates 75 percent of the time. In the last three months, EPS estimates have seen three upward revisions and seven downward revisions, while revenue estimates have seen eight upward revisions and seven downward revisions.
Pomerantz Law Firm Investigates Ionis Pharmaceuticals Over Securities Fraud Claims
Pomerantz LLP is investigating claims on behalf of investors of Ionis Pharmaceuticals, Inc. regarding potential securities fraud or unlawful business practices. The investigation follows Ionis's July 9, 2026 disclosure that the late-stage Phase-3 CARDIO-TTransform trial of Wainua, developed with AstraZeneca, did not meet its primary endpoint. On that news, Ionis's stock price fell $20.19 per share, or 23.9%, to close at $64.27 per share. Investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, extension 7980.
Dyne Therapeutics Gets FDA Clearance for DYNE-302 IND in FSHD
Dyne Therapeutics has received U.S. FDA clearance for its investigational new drug application to begin a Phase 1 trial of DYNE-302 in facioscapulohumeral muscular dystrophy. DYNE-302 is the company's third program to enter clinical development using its FORCE platform, which also underpins its Duchenne muscular dystrophy and myotonic dystrophy type 1 candidates. The Phase 1 trial will be a randomized, placebo-controlled, double-blind, multiple ascending dose study in ambulatory adults with FSHD, with safety and tolerability as the primary endpoint. In the first cohort, nine participants will receive three intravenous doses every four weeks, randomized two-to-one to DYNE-302 at 1.5 milligrams per kilogram or placebo. Dyne intends to pursue a traditional approval pathway in the U.S. for DYNE-302, which is designed to suppress DUX4 expression using a TfR1-targeting Fab and siRNA payload.
Alnylam Posts First Billion-Dollar Quarter Yet Valuation Remains Compressed
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 Completes Enrollment in Phase 3 YOSEMITE Study of Zodasiran for HoFH
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.
Five Biotech Stocks Delivered Triple-Digit Gains in Under Two Years
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.
Ionis Pharmaceuticals and AstraZeneca reported that their Phase 3 CARDIO-TTRansform trial of eplontersen in transthyretin amyloidosis cardiomyopathy did not meet its primary efficacy endpoint, prompting a shareholder investigation into the transparency of trial data and design. The failure removes a late-stage cardiovascular candidate from Ionis' pipeline and contributed to a roughly 23% share price drop. Attention now shifts toward earlier-stage RNA-targeted programs such as ION337 for Dravet syndrome, which has Fast Track designation, as well as recently approved products like TRYNGOLZA and DAWNZERA and upcoming PDUFA decisions for zilganersen and bepirovirsen. The core investment case still hinges on Ionis' RNA-targeted platform translating into multiple commercial drugs that can eventually support profitability, though high R&D spend and trial setbacks continue to delay that goal and invite scrutiny of management's execution.
Biotech Stocks Hit 52-Week Highs on Trial Results and Acquisitions
Several biotech stocks reached 52-week highs on July 22, 2026, driven by clinical trial updates and acquisition news. Advanced Biomed shares surged over 73% to $18.73 after reporting a first-quarter net income of $5.98 million and disclosing a securities purchase agreement to acquire Accellent Technologies for approximately $1 million. BioLife Solutions rose over 6% to $31.49 following the announcement that Repligen would acquire the company for a total enterprise value of about $1.5 billion. Forte Biosciences gained over 11% to $56.84 after reporting positive Phase 1 vitiligo trial results for its lead product FB102, which is also in Phase 2 for celiac disease. Silence Therapeutics climbed over 9% to $11.90 as it prepares to begin a Phase 3 trial for its siRNA candidate Zerlasiran in high-risk atherosclerotic cardiovascular disease. LB Pharmaceuticals ended up over 10% at $36.59 after announcing that topline results from the Phase 3 NOVA-2 trial of its schizophrenia drug LB-102 would be reported in the first half of 2027, earlier than previously expected.
Zhongchao Surges 190% After Reverse Split, Leading Biotech Gainers
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 posts late-stage trial win for plozasiran, pressuring Ionis shares
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.
Cathie Wood's ARK ETF Buys $15.3 Million of Ionis Pharmaceuticals After Trial Setback
Cathie Wood's ARK Genomic Revolution ETF has purchased $15.3 million worth of Ionis Pharmaceuticals shares since the beginning of July, following a sharp decline in the stock after a late-stage trial failure for eplontersen in ATTR-CM. The purchases came after Ionis and partner AstraZeneca announced the surprise setback, suggesting Wood is capitalizing on the drop while maintaining a long-term view. Ionis recently won FDA approval for Tryngolza to treat severe hypertriglyceridemia, expanding its target market to more than 3 million U.S. patients, and is pivoting to commercialize its own assets with peak sales guidance raised to $3 billion. The company reported first-quarter revenue of $246 million, up 86% year over year, and expects to reach cash flow break-even by 2028. Ionis also has late-stage pipeline catalysts including a priority review for zilganersen in Alexander disease and an upcoming phase 3 readout for pelacarsen partnered with Novartis.
Silence Therapeutics Shares Surge 7.2% on Pipeline Optimism
Silence Therapeutics PLC Sponsored ADR shares jumped 7.2% to close at $10.45 in the last trading session, driven by solid volume. The gain extends the stock's 45.3% advance over the past four weeks, fueled by investor optimism around its RNA interference pipeline. Key catalysts include the ongoing phase II SANRECO study of lead candidate divesiran in polycythemia vera, with topline results expected in August 2026, and the search for a strategic partner for zerlasiran, a phase III-ready siRNA candidate for cardiovascular disease. The company is also set to regain global rights to SLN312 after AstraZeneca discontinued development, opening new partnering options. Silence Therapeutics is expected to report a quarterly loss of $0.32 per share on revenues of $3.14 million, and currently carries a Zacks Rank #3.
Oligonucleotide CDMO Market to Reach USD 9.87 Billion by 2031
The global oligonucleotide contract development and manufacturing organization market is projected to grow from USD 3.68 billion in 2026 to USD 9.87 billion by 2031, a compound annual growth rate of 21.81%. Contract manufacturing held 51.68% of the market in 2025, while contract development is forecast to be the fastest-growing service segment at a CAGR of 22.90%. Antisense oligonucleotides represented 58.23% of revenue in 2025, and small interfering RNA is expected to be the fastest-growing oligonucleotide category with a CAGR of 23.25%. North America accounted for 39.55% of the market in 2025, and Asia-Pacific is forecast to be the fastest-growing region at a CAGR of 24.56%.
Alnylam initiates Phase 2 study of mivelsiran in Down syndrome–associated Alzheimer's disease
Alnylam Pharmaceuticals announced the initiation of a global Phase 2 study of its investigational RNAi therapeutic mivelsiran in people with Down syndrome–associated Alzheimer's disease at the Alzheimer's Association International Conference 2026. The APPlauDS study will recruit participants with early-stage Alzheimer's across approximately 30 sites, marking the first clinical trial to evaluate an RNAi approach to reduce APP overexpression caused by trisomy 21. Alnylam also reported updated Phase 1 data in early-onset Alzheimer's disease showing no increased risk of amyloid-related imaging abnormalities and robust reductions in cerebrospinal fluid biomarkers, with mean maximum decreases of 89.9% for sAPPβ and 70.2% for Aβ42 in the highest dose group. The company completed enrollment in the Phase 2 cAPPricorn-1 study of mivelsiran in cerebral amyloid angiopathy, with initial results expected in 2028. Additionally, Alnylam presented preclinical data and the design of an ongoing Phase 1 trial for ALN‑5288, a tau-targeting RNAi therapeutic being developed in collaboration with Regeneron Pharmaceuticals.
Amoytop Biotech announced that it has received a drug clinical trial approval notice from the National Medical Products Administration. The ACT201 injection has been approved to conduct clinical trials for chronic hepatitis B. The drug is an innovative antisense oligonucleotide with strong antiviral activity and low off-target effects, and has been selected for the 2025 National Science and Technology Major Project for innovative drug research and development.
Fosun Pharma’s holding subsidiary, Fosun Pharma Industrial Development (Shenzhen) Co., Ltd., has received approval from the National Medical Products Administration to conduct clinical trials for FXR0906 injection for the treatment of hypertriglyceridemia. The drug is a liver-targeted siRNA small interfering nucleic acid drug licensed in by Fosun Pharma, which silences APOC3 gene expression through RNA interference technology to reduce serum triglyceride levels. The group holds exclusive rights to research, develop, register, produce, and commercialize the drug in mainland China, Hong Kong, and Macau. The company plans to initiate Phase I clinical studies in mainland China once conditions are met.
Voyager's VY1706 Shows 75% Tau Reduction in Six-Month NHP Study, Clinical Trial Expected H2 2026
Voyager Therapeutics presented six-month GLP toxicology data for its investigational Alzheimer's gene therapy VY1706 at the AAIC conference, showing the treatment was well tolerated and reduced tau protein by up to 75% in key brain regions of non-human primates following a single intravenous dose. The company received FDA IND clearance in June, enabling initiation of a clinical trial in adults with early Alzheimer's disease, with dosing expected to begin in the second half of 2026. VY1706 is the first tau-targeted gene therapy to receive IND clearance from the FDA. The therapy uses a vectorized siRNA encapsulated in a TRACER AAV capsid that crosses the blood-brain barrier via the ALPL receptor, achieving broad and durable CNS delivery. No adverse findings were observed in the central nervous system, dorsal root ganglia, or peripheral organs up to the highest dose tested.
Ionis Pharmaceuticals loses 29% in two days after Wainua trial failure and Roche exit
Ionis Pharmaceuticals shares plunged about 29% over two sessions after its heart drug Wainua failed a late-stage trial and Roche ended two Huntington's disease programs. On July 9, Ionis and partner AstraZeneca said the Phase 3 CARDIO-TTRansform trial of eplontersen, sold as Wainua, did not meet its primary goal in ATTR-CM patients, triggering a 24% drop. The next day, Roche halted the antisense drug tominersen and an early-stage program, deepening the selloff by another 8%. Most analysts cut price targets but kept Buy ratings, with Jefferies moving from $113 to $90 and BofA Securities from $111 to $90. Ionis still expects 2026 revenue of $875 million to $900 million, and full trial data will be presented at the European Society of Cardiology Congress in August.
CRISPR Therapeutics could be an attractive buy-and-hold option despite its recent underperformance and current lack of profitability. The biotech has multiple catalysts on the way, including results from an ongoing clinical trial of CTX310, an investigational one-time gene-editing treatment designed to permanently lower LDL cholesterol, expected in the second half of the year. Its approved therapy Casgevy, developed with Vertex Pharmaceuticals, recently received a label expansion from the U.S. Food and Drug Administration to include children as young as two, adding 5,500 patients to its addressable market in the U.S. and representing an additional $12.1 billion commercial opportunity at $2.2 million per treatment course. CRISPR Therapeutics is also expanding beyond gene editing through a partnership with Sirius Therapeutics to develop CTX611, a long-acting siRNA therapy designed to prevent dangerous blood clots with just two injections per year. The company's deep pipeline and partnership with a biotech giant position it to potentially expand its approved product portfolio and improve financial results by the end of the decade.
Alnylam gains opening in ATTR-CM as AstraZeneca-Ionis trial fails
AstraZeneca and Ionis Pharma reported a failure in a late-stage ATTR-CM trial, removing a key competing approach in transthyretin cardiomyopathy and giving Alnylam Pharmaceuticals more room to build on its RNAi therapies in this indication. Alnylam has expanded its GENESIS Pharma collaboration to target broader geographic access for its RNAi portfolio across up to 17 markets, and has deepened AI-focused partnerships with groups such as Komodo Health to support commercial execution and operational efficiency. The stock trades at $312.79, with the share price down 21.8% year to date and down 3.1% over the past year, but up 53.4% over three years and 81.9% over five years. The clearer competitive runway in ATTR-CM and GENESIS-led expansion support the narrative that Alnylam can widen adoption of its core RNAi therapies and build operating leverage as international demand scales, though greater reliance on the ATTR-CM opportunity also sharpens concentration risk around the TTR franchise.
Alnylam Pharmaceuticals Stock Looks Rich on Earnings Yet Strong on Returns
Alnylam Pharmaceuticals stock has returned 81.9% over the past five years, but current valuation checks suggest a mixed picture rather than a clear bargain. The stock trades at a price-to-earnings ratio of 77.6 times, well above the biotech industry average of 17.8 times and the peer average of 30.4 times, and more than double its tailored fair P/E of 34.6 times. While recent clinical progress in RNAi therapies and expanded AI partnerships support growth expectations, competitive and clinical trial risks may weigh on investor willingness to pay a premium. The overall valuation signal is mixed, with the key question being whether Alnylam can sustain revenue and margin progress to justify its current multiple.
Jim Cramer Says Biotech Is the Hottest Group in the Market Right Now
CNBC's Jim Cramer declared biotech the hottest group in the market, citing a 51% return for the iShares Biotechnology ETF over the past year and a surge in dealmaking following a leadership change at the FDA. Cramer noted that the prior administration nearly blocked an Amgen acquisition, but with new FDA leadership, his sources expect deals to flood the market. Eli Lilly, a $1.16 trillion pharma giant, has already announced four acquisitions in the first quarter of 2026, including Orna Therapeutics, Centessa Pharmaceuticals, Kelonia Therapeutics, and Ajax Therapeutics, fueled by strong sales of Mounjaro and Zepbound. Cramer also highlighted Alnylam Pharmaceuticals as a prime takeover target, with its stock down 21% year-to-date despite 187% revenue growth and a surging TTR drug franchise. He advised investors to be long biotech, a call he has not made in ages.