RNA Therapeutics

Nasdaq announced yesterday that shares of Moderna, the vaccine maker, will replace Warner Bros. Discovery in the Nasdaq 100 index, effective Oct. 9. The change follows a more than sixfold surge in Moderna's share price this year, giving the company a market value of about 75 billion dollars. Conversely, Warner Bros. Discovery will also be removed from the indexes of major index providers MSCI and S&P, as its merger with Paramount Skydance is expected to be completed on Oct. 6, after the process was delayed for many months.

Novartis has entered into a licensing and option agreement with China's Abogen Biosciences to advance RNA-encoded therapeutics, according to a statement on Friday. Abogen will receive an upfront payment of $575 million, and, if all options on all programs are exercised, Abogen is eligible to receive up to approximately $7.2 billion in potential milestone payments, plus potential royalties on future product sales. The agreement includes an exclusive worldwide license to Abogen's lead asset, ABO2203, a novel mRNA-encoded CD19xCD3 T-cell engager designed to reset B cells by directing endogenous production of T-cell engagers in vivo, with the potential to transform treatment for patients with autoimmune diseases. The Swiss drugmaker also holds the exclusive option to license a number of next-generation therapeutic assets developed on Abogen's proprietary RNA platform.

Nasdaq said Thursday that Moderna will become a component of the Nasdaq-100 Index, replacing Warner Bros. Discovery before market open on Friday, October 9. The vaccine maker's shares have jumped more than sixfold this year to a valuation of roughly $75B. Warner Bros. Discovery is also set to be removed from major indexes, including those tracked by MSCI and S&P, as its merger with Paramount Skydance is expected to close on October 6 following a months-long delay.

Aptadir Therapeutics has closed a EUR 40M Seed round to advance a novel class of RNA inhibitor-based therapeutics for intractable genetic conditions. The round was led by 4BIO Capital, with follow-on participation from the company's original pre-seed investor EXTEND, Italy's National Technology Transfer Hub launched by CDP Venture Capital SGR and jointly funded by Angelini Ventures and Evotec SE. Additional support came from CDP Venture Capital through the Digital Transition Fund, Indaco Venture Partners, XGEN Venture, CE-Ventures, Angelini Ventures through a direct investment, Kerna Ventures, Italian Angels for Biotech, and Club degli Investitori. The funds will advance the company's pipeline of disease-modifying investigational RNA therapeutics, including its lead candidate CAP1-FMR1 for Fragile X Syndrome, based on a new class of RNAs called DNMTs Interacting RNAs that block aberrant DNA methylation at a single gene level to reactivate silenced gene expression. The science originates from the Beth Israel Deaconess Medical Center, the Italian Research National Council, and the Cancer Science Institute of Singapore.

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.

Moderna and Merck reported that their Phase 3 INTerpath-001 trial of personalized cancer vaccine intismeran autogene plus Keytruda met its primary endpoint in melanoma, with full data presented earlier this month at the European Society for Medical Oncology Congress 2026. Moderna is also in talks with Middle East partners about potential investment and manufacturing cooperation, pairing its mRNA oncology push with efforts to extend its geographic footprint and industrial capabilities beyond its original COVID-19 franchise. The company recently won FDA approval of mFlusiva, its mRNA flu vaccine for adults 50 and older, underscoring its push to build a non-seasonal respiratory portfolio. Moderna's narrative projects $4.5 billion revenue and $782.2 million earnings by 2029, yielding a $119.56 fair value, a 40% downside to its current price, while some of the most optimistic analysts assume revenue could reach about US$8.5 billion and earnings turn positive by 2029.

Pfizer Canada and BioNTech announced Health Canada's approval of the Omicron XFG-adapted COMIRNATY vaccine for individuals aged 6 months and older, aligning with 2026-2027 public health recommendations. A few days earlier, BioNTech and OncoC4 reported that the investigational CTLA-4 antibody gotistobart nearly doubled median overall survival versus chemotherapy in a Phase 3 lung cancer trial stage. The Health Canada authorization, following earlier US and EU XFG approvals in 2026, shows the COVID-19 franchise still contributes regulated, seasonal revenue while BioNTech leans into oncology. BioNTech's narrative projects €2.2 billion revenue and €390.1 million earnings by 2029, implying a 6.0% yearly revenue decline and an earnings increase of about €2.1 billion from -€1.7 billion today, with a $118.06 fair value and 20% upside to its current price. Bullish analysts assume revenue climbs to about €3.0 billion and earnings to €313.1 million by 2029, though the new gotistobart data and oncology execution risks could shift how those upbeat assumptions are weighed against more cautious views.

Moderna has entered high-level investment talks with the UAE government covering advanced pharmaceutical manufacturing and research and development collaboration. The discussions focus on expanded research capabilities, potential local production, and broader access to Middle Eastern healthcare markets. Management framed the UAE engagement as part of a wider push to build out Moderna's international footprint beyond existing Western partnerships. The company, a large US biotech group with a market cap of about $79.4b, focuses on messenger RNA based medicines and has a history of working closely with public sector buyers. Investors will watch whether the talks turn into concrete agreements, such as a defined R&D center, a manufacturing investment figure, or a multi year vaccine procurement deal, and any link to the INTerpath-001 melanoma program or the mRNA respiratory portfolio.

Moderna Chairman Noubar Afeyan met UAE Minister of State Saeed bin Mubarak Al Hajeri, who also chairs the Emirates Drug Establishments, to discuss investment and cooperation in advanced pharmaceutical industries. The talks covered research and development collaboration, exchange of expertise, and strengthening the UAE's pharmaceutical manufacturing capabilities, building on the strategic partnership between the United States and the UAE. Moderna shares climbed to a record intra-day high of $195.71 before closing up 6.98 percent at $194.82 apiece. Investors are also awaiting an updated readout on the INTerpath-001 trial of intismeran autogene in combination with Merck's Keytruda, to be presented at the European Society for Medical Oncology Congress 2026 in Madrid from October 23 to 27, 2026. The phase 3 trial previously met its primary endpoint of recurrence-free survival and key secondary target of distant metastasis-free survival, marking the first positive phase 3 readout for a combination of individualized neoantigen therapy and mRNA-based cancer therapy. In the second quarter, 49 hedge funds held positions in Moderna, down from 52 in the first quarter, but their combined holdings rose 10 percent to $1.5 billion from $1.4 billion quarter-on-quarter.

Pfizer Inc. and BioNTech SE announced on August 27 that the U.S. FDA has approved the supplemental Biologics License Application for their 2026–2027 COVID-19 vaccine formula, COMIRNATY XFG. The updated formula targets the XFG variant and is authorized for adults aged 65 and older, as well as individuals aged 5 to 64 with high-risk underlying conditions. The approval, based on clinical, real-world, and manufacturing data showing strong immune responses against circulating strains, aligns with FDA strain guidance for fall 2026. For Pfizer, the clearance protects a recurring seasonal revenue stream that supports margins and cash flow while the company funds its non-COVID pipeline and manages upcoming loss-of-exclusivity events. For BioNTech, it validates the execution power of its proprietary mRNA platform and preserves its balance sheet, which carries minimal debt and substantial cash reserves to fund late-stage oncology trials without dilutive financing. Both companies still face long-term market contraction as COVID-19 booster demand has normalized well below pandemic peaks, leaving Pfizer's earnings less predictable against elevated debt and exposing BioNTech's heavy operating losses as vaccine revenues fall short of its massive R&D expenses.

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.

Arcturus Therapeutics said interim phase 2 results for ARCT-810 showed the candidate reduced and/or maintained first morning fasting ammonia within the normal range in patients with ornithine transcarbamylase deficiency, and also reduced glutamine, with some individuals reaching the normal range. Based on those results and a June meeting with the US FDA, the company aims to begin dosing of ARCT-2601 close to the end of the year in participants 12 years and older under an amended phase 2 protocol that integrates ARCT-2601 into the current ARCT-810 phase 2 study. Both ARCT-810 and ARCT-2601 are mRNA therapeutics for OTC deficiency, but ARCT-810 uses the LUNAR platform while ARCT-2601 uses the next-generation LUNAR 2.0 platform, which Arcturus says produces greater than 30-fold higher protein expression and could allow for lower or less frequent dosing. OTC deficiency is a rare genetic disorder in which the body stops breaking down and removing nitrogen, leading to dangerous ammonia levels in the blood. Arcturus is also acquiring AI discovery company myNeo, with which it has worked since 2024, and the deal is expected to close in October.

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.

AbbVie reported second-quarter revenues of $16.99 billion, up 10.2% year on year and 1.2% above analysts' consensus expectations, though the quarter was mixed with only a narrow beat of EPS estimates and a slight miss of full-year EPS guidance estimates. The results came as the 11 therapeutics stocks tracked by the report collectively beat consensus revenue estimates by 8% in Q2, with share prices up 24.8% on average since the latest earnings results. Biogen posted the group's best quarter, with revenues of $2.74 billion, up 3.4% year on year and 12.1% above expectations, while Myriad Genetics delivered the weakest performance, reporting revenues of $190.7 million, down 10.5% year on year and 8.2% below expectations, alongside full-year revenue guidance that missed and a significant EPS miss. Gilead Sciences reported revenues of $7.80 billion, up 10.2% year on year and 6.2% above expectations, and Moderna reported revenues of $145 million, up 2.1% year on year and 35.8% above expectations, the biggest analyst estimate beat of the group. AbbVie's stock is up 2.9% since reporting and currently trades at $264.79.

Moderna will present detailed data from the phase III INTerpath-001 study of intismeran autogene, its personalized mRNA cancer therapy developed with Merck, at the European Society for Medical Oncology Congress next month. The findings will be presented on Oct. 24 in a Presidential Symposium, a session highlighting cutting-edge and potentially practice-changing clinical research, and are expected to provide investors with detailed data beyond the top-line results Moderna reported last month. Last month, Moderna and Merck reported positive results from INTerpath-001, which evaluated intismeran in combination with Merck's Keytruda in patients with high-risk Stage IIB-IV melanoma whose tumors had been surgically removed; the study met its primary endpoint of recurrence-free survival and a key secondary endpoint of distant metastasis-free survival, though the companies did not report numerical data, stating only that the improvements were statistically significant and clinically meaningful. The companies are advancing intismeran across nine phase II and phase III studies spanning multiple tumor types, including non-small cell lung cancer, bladder cancer and renal cell carcinoma, and a commercial launch of the therapy is targeted as early as next year. Moderna shares rose 12% yesterday, climbing to a new 52-week high of $176.86, and have skyrocketed nearly 500% year-to-date, significantly outperforming the industry's 6% growth.

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.

Moderna shares surged 9% to $167.90 after the company said its Phase 3 adjuvant melanoma study of intismeran autogene was selected for a Presidential Symposium slot at the European Society for Medical Oncology congress in Madrid on October 24. The Phase 3 INTerpath-001 trial tests intismeran autogene plus pembrolizumab against pembrolizumab alone in patients whose melanoma had been surgically removed, and Moderna will host a live investor webcast the same day. No new efficacy data was released Monday; the melanoma result was first reported in August, and the announcement adds only the venue and a fixed date, with the Presidential Symposium being the congress's highest-profile placement, typically reserved for datasets organizers expect to shape clinical practice. Merck, the pharma partner on intismeran autogene and supplier of pembrolizumab, rose just 1% to $148.95, while BioNTech, which runs its own late-stage individualized messenger RNA cancer immunotherapy programs, gained 2% to $97.71, signaling the market views the move as a Moderna-specific re-rating rather than broad validation of messenger RNA oncology. The iShares Biotechnology ETF rose 1% to $207.28 and the SPDR S&P 500 ETF Trust rose 1.16% to $770.54, with Moderna's advance running several times either fund's move.

Beijing has unveiled a five-year plan aimed at making China a global leader in drug innovation, with a target of developing 25% of the world's first-in-class drugs by 2030. The plan, issued in a joint notice from multiple government ministries, lays out 25 priority tasks spanning innovation capacity, research and technology, including expanded R&D, the use of AI and quantum computing in drug discovery, and the building of pharmaceutical ecosystems beyond pipelines. The government aims for the industry to reach 3.5tn yuan, or $520bn, in annual revenue by 2030, with 50 companies exceeding $1.5bn in annual revenue, and at least five Chinese drugs achieving global annual sales above $1bn. The notice singled out next-generation therapies including antibody-drug conjugates, cell and gene therapies, and CAR-T, along with vaccines and newer platforms such as mRNA, and included a section on industrialising traditional Chinese medicine. Citi analysts said the plan signals a shift from scale-driven growth toward innovation-driven, quality-oriented development, with a broad whole-value-chain policy push covering innovative drugs, medical devices, AI-driven drug discovery, and CDMO/CRO. The biomedical plan was released alongside counterparts for other strategic sectors, including integrated circuits, aerospace, the low-altitude economy, new energy storage and intelligent robotics.

On September 18, the Ministry of Industry and Information Technology, the National Development and Reform Commission and eight other departments jointly issued the 15th Five-Year Plan for the development of the pharmaceutical industry, laying out 25 key tasks in eight areas. The plan proposes that by 2030, operating revenue of Chinese pharmaceutical enterprises above designated size will exceed 3.5 trillion yuan, the innovative drug industry will grow at an average annual rate of more than 20 percent, China's share of first-in-class innovative drugs globally will exceed 25 percent, more than 200 innovative medical devices will be launched, and the number of pharmaceutical industry parks with output of at least 100 billion yuan will reach 20. Boosted by the news, innovative drug and CRO stocks rose on September 21, with Nanhua Bio gaining for a third straight day, while Harbin Pharmaceutical Group and Shandong Xinhua Pharmaceutical hit their daily limits. The same day, PCB-related shares advanced, as research reports from multiple institutions noted that upstream raw material prices have kept climbing and PCB makers have begun repricing, with the third quarter of 2026 expected to mark the start of an earnings recovery for the sector. MLCC-related shares were active, as AI-driven demand has caused shortages and sharp price surges in high-capacity models, with some spot prices soaring several-fold. Humanoid robot stocks rebounded, after drone footage suggested that the main structure of Tesla Optimus's dedicated production line in Texas may be nearing completion; the line is scheduled to begin production in 2027 with an annual capacity target of 10 million units.

Arcturus Therapeutics Holdings Inc. announced it will host a virtual presentation on Wednesday, September 23, 2026, at 4:30 p.m. ET covering the ARCT-810 Phase 2 clinical program for Ornithine transcarbamylase deficiency and the company's mRNA liver therapeutics platform. The company will issue a press release summarizing the presentation prior to the call. Marshall Summar, M.D., a founding member and Executive Committee member of the NIH UCD Consortium and a recognized expert in rare diseases and OTC deficiency, will participate in the presentation. ARCT-810 is an intravenously administered investigational mRNA therapeutic designed to express normal functional OTC enzyme in the liver of individuals with OTC deficiency, and it holds Orphan Medicinal Product Designation and an approved pediatric investigation plan from the European Medicines Agency, plus Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. In Europe and the U.S., approximately 10,000 people have OTC deficiency.

On September 18, the Ministry of Industry and Information Technology, the National Development and Reform Commission, and eight other ministries jointly released the 15th Five-Year Plan for the Development of the Pharmaceutical Industry, proposing that by 2030, biopharmaceutical R&D and application will rank among the world's leaders, and the biopharmaceutical industry will accelerate its rise as a national emerging pillar industry. The plan sets 10 expected indicators covering industrial scale and efficiency, innovative development, enterprise cultivation, and cluster development, including operating revenue of pharmaceutical industrial enterprises above designated size exceeding 3.5 trillion yuan, an average annual growth rate of the innovative drug industry scale exceeding 20 percent, more than 5 products with global annual sales exceeding 1 billion US dollars, first-in-class innovative drugs accounting for more than 25 percent of the global total, more than 200 innovative medical devices launched, 50 pharmaceutical industrial enterprises with annual operating revenue exceeding 10 billion yuan, and 20 pharmaceutical industrial parks at the 100-billion-yuan level. Boosted by this news, the three major stock indices all rose more than 1 percent in early trading that day. Huatian Technology hit the daily limit up, with turnover of 6.065 billion yuan, nearly 1.47 million lots of sealed buy orders on the limit-up board, and nearly 3.3 billion yuan of main capital rushing in, ranking first in the A-share market. Zhou Sicong, fund manager of Ping An Pharmaceutical Select, believes innovative drugs are likely to become an important growth theme, and CITIC Securities said China's innovative drugs have entered a stage of global value realization. According to statistics from Securities Times Data Treasure, since September, Chengdu Leading Pharmaceutical, Asymchem Laboratories, Porton Pharma Solutions, and Hunan Warrant Pharmaceutical have led gains, all rising more than 10 percent, with Chengdu Leading Pharmaceutical up a cumulative 16.87 percent.

BioNTech reported detailed Phase 3 data showing its investigational lung cancer therapy gotistobart nearly doubled median overall survival compared with standard chemotherapy. The shares traded around US$96.69, with a 30 day share price return of 3.87% and a 90 day share price return of 4.85%, while the 1 year total shareholder return declined 2.59% and the 5 year total shareholder return fell 71.40%. BioNTech now trades at roughly a 22% discount to analyst targets, with the most followed fair value view at $120.62 against the last close of $96.69. The company's pipeline includes multiple late stage Phase II and III trials for BNT327 and mRNA cancer immunotherapies in lung and breast cancers, supported by the planned CureVac acquisition. Risks to the bullish case include late stage oncology trial failures and prolonged COVID-19 vaccine demand weakness and related litigation.

GSK plc plans to close its vaccine manufacturing facility in Dresden, Germany, by summer 2027, putting 641 jobs at risk. The company reviewed its Dresden and Ste-Foy, Canada, flu vaccine sites and chose to consolidate production in Canada, which it says can meet anticipated demand more sustainably and competitively, as falling demand for traditional egg-based flu vaccines has left GSK with more manufacturing capacity than it needs. The closure comes as GSK advances an mRNA-based seasonal flu vaccine into Phase III after Phase II results showed stronger immune responses than standard-dose vaccines in younger adults and high-dose vaccines in older adults. GSK's vaccine business remains a significant earnings contributor, with second-quarter 2026 vaccine sales rising 8% to £2.3 billion, meningitis vaccines up 21%, Arexvy sales increasing by more than 100%, and Shingrix generating £0.9 billion. The company faces execution risk, as the mRNA flu candidate has only reached Phase III and opposition from the German union and works council could make the closure process more complicated or expensive.

Guobang Pharma announced that the pharmaceutical excipient "pegylated dimyristoyl glycerol (for injection)" produced by its wholly-owned subsidiary Zhejiang Guobang Pharmaceutical has passed the joint review and approval with the formulation by the Center for Drug Evaluation of the National Medical Products Administration, and its registration status has been changed to A. This product is a key functional excipient for mRNA vaccines and pharmaceutical formulations. This approval enables the product to meet the conditions for commercial production and supply to downstream customers, and the company expects it will have a positive impact on the market expansion of its pharmaceutical excipient business.