Alnylam Pharmaceuticals presented new analyses at the European Society of Cardiology Congress in Munich supporting the use of Amvuttra across a range of transthyretin amyloidosis patients, including those already on Pfizer's tafamidis, and reported encouraging subgroup findings for its experimental hypertension drug zilebesiran. In the Phase 3 HELIOS-B study, 259 of the 654 treated patients were taking tafamidis at baseline, and Amvuttra's effect on the composite of death and recurrent cardiovascular events appeared consistent regardless of tafamidis use. Patients on Amvuttra also showed better preservation of walking ability and a 25% less deterioration in a composite measure of physical and mental function, with a 52% reduction in the risk of decline on that measure. A pooled analysis of 1,402 patients from four Phase 3 studies of Amvuttra and Onpattro showed consistent effects in women and men across cardiac and neurological forms of the disease. For zilebesiran, a post hoc subgroup analysis from the Phase 2 KARDIA-3 study showed larger systolic blood pressure reductions in high-risk patients with uncontrolled hypertension on a diuretic and at least one other drug, with effects extending across day and night; these findings are exploratory, and the drug's value depends on the ongoing Phase 3 ZENITH cardiovascular outcomes trial.
Moderna Fair Value Estimate Rises to US$122.67 on Cancer Vaccine Analyst Revisions
Moderna's fair value estimate has been lifted from US$119.56 to US$122.67 per share, according to updated modelling tied to analyst revisions on its intismeran cancer vaccine program. The change follows Phase 3 INTerpath-001 melanoma data that prompted target lifts or upgrades from BofA, Goldman Sachs, UBS, Barclays, RBC Capital and Morgan Stanley, with BofA setting a US$200 target and a Neutral rating and calling the readout a watershed moment for Moderna. William Blair moved to Outperform and Wolfe Research called the melanoma result de-risking for the broader intismeran platform, while RBC Capital described the data as a massive scientific breakthrough. On the bearish side, Citi and Rothschild & Co Redburn moved to Sell even as they raised targets to US$80 and US$81 respectively, citing questions on oncology revenue expectations and limited disclosure around efficacy metrics such as hazard ratios. The updated model also shifted the revenue growth assumption from 26.74% to 26.75%, the net profit margin from 17.25% to 17.20%, the future P/E from 80.90x to 83.10x, and the discount rate from 7.50% to 7.46%.
Biotech & Genomic Medicine › Oncology Therapeutics ▲Technology
Biotech & Genomic Medicine › mRNA Platforms ▲Technology
Biotech & Genomic Medicine › RNA Therapeutics ▲Technology
Biotech & Genomic Medicine › Immuno-Oncology / Checkpoint Technology
MRNA · Capital · Positive Fair value estimate raised to US$122.67 and multiple analyst price-target increases/upgrades followed the data readout.
MRNA · Technology · Positive Phase 3 INTerpath-001 melanoma data for the intismeran cancer vaccine prompted analyst target lifts and upgrades, de-risking the platform.
Moderna shares surge on news of US-private partnership to develop cancer vaccines
Shares of Moderna and Novavax rose today after The New York Times reported that the US National Institutes of Health, or NIH, plans to launch a public-private partnership to accelerate the development of personalized cancer vaccines. The vaccine project is expected to begin in December, focusing on pancreatic cancer, liver cancer, and colorectal cancer, as well as certain tumors in children. Previous trials have used mRNA technology to train the body's immune system to recognize and attack proteins specific to cancer cells. The positive reaction to the news was most evident for Moderna, which is developing personalized mRNA cancer vaccines together with Merck. Moderna's share price has soared more than 600% since the start of this year, making it the best-performing stock among the companies used to calculate the S&P 500 index.
Biotech & Genomic Medicine › mRNA Platforms ▲Technology
Biotech & Genomic Medicine › RNA Therapeutics ▲Technology
Biotech & Genomic Medicine › Oncology Therapeutics ▲Technology
MRNA · Technology · Positive NIH public-private partnership to accelerate personalized mRNA cancer vaccines directly benefits Moderna's cancer vaccine program with Merck.
NVAX · Technology · Positive Novavax shares rose on the NIH cancer vaccine partnership news, though the article does not detail its specific role.
Asahi Kasei Therapeutics Doses First Patient in Phase 2a Trial of AIC468 for BK Virus
Asahi Kasei Therapeutics, the global specialty pharmaceutical business of Tokyo-based Asahi Kasei, announced that the first patient has been dosed in a randomized Phase 2a clinical trial evaluating AIC468, a novel antiviral antisense oligonucleotide, in adult kidney transplant recipients with BK virus infection. The trial, registered as NCT07503561, is a randomized, double-blind, placebo-controlled study and forms part of a larger Phase 2/3 program designed to assess safety, tolerability, and pharmacokinetic profile across multiple dose levels, measured by the frequency and severity of treatment-emergent adverse events. Progression into Phase 2a was supported by favorable Phase 1 safety, tolerability, and pharmacokinetic data, and the investigational program was developed by Aicuris Anti-infective Cures AG, which is now part of Asahi Kasei Therapeutics. Carl Kraus, Chief Medical Officer of Asahi Kasei Therapeutics, said BK virus reactivation remains a major complication for kidney transplant recipients that can lead to graft dysfunction and BK nephropathy, and that dosing the first patient represents an important step toward a targeted antiviral therapy for a community with no approved treatment options. BK virus-associated nephropathy affects up to 10% of kidney transplant recipients and can result in graft loss, while current management relies on reducing immunosuppressive therapy, which increases the risk of graft rejection.
Moderna to Join Nasdaq-100, Replacing Warner Bros. Discovery
Moderna will join the Nasdaq-100 Index on Oct. 9, replacing Warner Bros. Discovery before the opening bell on Friday. The swap follows the completion of Paramount's acquisition of Warner Bros. Discovery, whose shares have ceased trading; the combined entity is now named Skydance and trades on the NYSE under the ticker SKYD. Nasdaq did not cite a specific reason for adding Moderna, but the inclusion follows a sharp rise in the company's market value and growing optimism around its prospects, supported by positive phase III results for its personalized cancer therapy developed with Merck, which met its primary endpoint of recurrence-free survival and a key secondary endpoint of distant metastasis-free survival in certain high-risk melanoma patients. The Nasdaq-100 is tracked by more than 200 investment products with over $800 billion in assets under management globally, so index funds adjusting their portfolios could create incremental institutional demand for Moderna shares. The inclusion does not change Moderna's underlying fundamentals, operations, cash flow or pipeline development, and the stock's longer-term performance will continue to depend on its business and pipeline. Moderna shares have skyrocketed 566% year-to-date, far outpacing the industry's 1% growth, and the stock trades at 30.84 times forward 12-month sales on a price-to-sales basis versus an industry average of 2.06 times.
Biotech & Genomic Medicine › mRNA Platforms ▲Capital
Biotech & Genomic Medicine › RNA Therapeutics ▲Capital
Biotech & Genomic Medicine › Oncology Therapeutics ▲Capital
MRNA · Capital · Positive Moderna will join the Nasdaq-100 on Oct. 9, and index funds tracking the index could create incremental institutional demand for its shares.
WBD · Capital · Neutral Warner Bros. Discovery is being removed from the Nasdaq-100 after Paramount completed its acquisition and its shares ceased trading.
GSK Adds Wave Life Sciences Hepatology Candidate, Expands AN2 Tuberculosis Research
GSK has entered a collaboration with Wave Life Sciences to advance a GalNAc-siRNA hepatology candidate into its development pipeline, while separately expanding its tuberculosis research with AN2 Therapeutics. The Wave Life Sciences partnership focuses on RNA-based therapies targeting liver diseases using GalNAc-siRNA technology, giving GSK another modality in liver disease and access to Wave's SpiNA design platform without building that RNA chemistry capability internally. The AN2 Therapeutics work is supported by new funding from the Bill & Melinda Gates Foundation for boron-based drug discovery. The Wave hepatology move and the Gates-backed TB research funding sit within GSK's existing narrative of heavier R&D reinvestment and business development, though the high R&D spend and execution uncertainty still apply if these programs move slowly or fail to produce strong late-stage assets. The clearest next marker will be whether GSK or Wave disclose that one of the three remaining collaboration programs reaches formal candidate selection over the next research updates, triggering further milestone payments.
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides ▲Technology
Biotech & Genomic Medicine › RNA Therapeutics ▲Technology
GSK.LSE · Technology · Positive GSK adds Wave's GalNAc-siRNA hepatology candidate and expands AN2 TB research, broadening its R&D pipeline.
WVE · Demand · Positive GSK enters collaboration with Wave Life Sciences to advance a GalNAc-siRNA hepatology candidate, with potential milestone payments.
ANTX · Capital · Positive GSK expands tuberculosis research with AN2 Therapeutics, supported by new Gates Foundation funding for boron-based drug discovery.
Lepu Medical Subsidiary's MWN117 Injection Receives US FDA Clinical Trial Approval
Lepu Medical announced that its controlling subsidiary, Shanghai Minwei Biotechnology, has received a notice from the US FDA agreeing to allow clinical trials for the MWN117 injection. The drug is an siRNA therapy targeting INHBE inhibition, indicated for overweight and obesity, and has already received clinical trial approval from China's CDE. Non-clinical studies show that the drug, used alone or in combination with GLP-1 receptor agonists, effectively reduces body weight and body fat while reducing muscle loss, with efficacy lasting up to 12 weeks. Currently, no similar drug has been approved for marketing domestically or internationally.
Biotech & Genomic Medicine › RNA Therapeutics ▲Regulation
Biotech & Genomic Medicine › RNAi / Antisense Oligonucleotides ▲Regulation
Biotech & Genomic Medicine › Metabolic, Diabetes & Obesity Regulation
Longevity & Life Extension › GLP-1 Healthspan Proxies Regulation
300003.CS · Technology · Positive Lepu Medical's subsidiary received US FDA clinical trial approval for its siRNA obesity drug MWN117, advancing its R&D pipeline.
上海民为生物技术有限公司 · Technology · Positive Shanghai Minwei Biotechnology, the controlling subsidiary, received FDA clinical trial approval for its INHBE-targeting siRNA obesity therapy MWN117.