TTX-MC138 is delivered to glioblastoma tumors in murine models of the disease, following intravenous injection TTX-MC138 demonstrated suppression of miR-10b target and extended survival in aggressive glioblastoma models Study supports future clinical development of TTX-MC138 for patients with glioblastoma BOSTON, Jan. 6, 2026 /PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), a clinical stage company pioneering immuno-oncology and RNA therapeutics for the treatment of high risk and advanced can ...

Core Viewpoint - Wave Life Sciences Ltd. has announced the pricing of its public offering of ordinary shares and pre-funded warrants, aiming to raise approximately $350 million before expenses [1][2]. Group 1: Offering Details - The public offering consists of 15,789,475 ordinary shares priced at $19.00 each, along with pre-funded warrants for 2,631,578 ordinary shares at an offering price of $18.9999 [1][2]. - The offering is expected to close on or about December 11, 2025, subject to customary closing conditions [2]. Group 2: Underwriters - Jefferies, Leerink Partners, and BofA Securities are acting as joint book-running managers for the offering, while Truist Securities and Mizuho are serving as book-runners [3]. Group 3: Company Overview - Wave Life Sciences is a biotechnology company focused on RNA medicines, utilizing its PRISM® platform to address both rare and common disorders [6]. - The company's pipeline includes clinical programs targeting obesity, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, and Huntington's disease, along with several preclinical programs [6].

Core Insights - Novartis (NVS) projects a compounded annual growth rate (CAGR) of 5-6% for sales from 2025 to 2030, with an upgraded sales guidance for 2024-2029 to 6% from 5% due to strong growth drivers and upcoming launches [1][7] - The company faces generic competition for its drug Entresto, but strong performances from products like Kisqali, Kesimpta, Pluvicto, and Scemblix support its momentum [2][12] - Novartis raised its peak sales guidance for Kisqali to over $10 billion from over $8 billion, reflecting its strong performance [3] Sales Performance - Kisqali's sales increased by 68% to $1.33 billion in Q3, driven by growth in the U.S. market, particularly from the early breast cancer indication [4] - Scemblix sales surged by 95% to $358 million in Q3, supported by growth in chronic myeloid leukemia and new indications in the U.S. and Japan [5] - Novartis has eight de-risked, in-market drugs with peak sales potential ranging from $3 billion to $10 billion [5] Regulatory Approvals and Pipeline - Novartis received FDA approval for remibrutinib (Rhapsido) as an oral treatment for chronic spontaneous urticaria, marking it as the first FDA-approved Bruton's tyrosine kinase inhibitor for this condition [8] - The company is focused on four core therapeutic areas: cardiovascular-renal-metabolic, immunology, neuroscience, and oncology [8] - Key pipeline candidates include ianalumab, pelacarsen, OAV101 IT, del-desiran, abelacimab, and farabursen [9] Strategic Acquisitions - Novartis announced the acquisition of Avidity Biosciences for $12 billion to enhance its neuroscience pipeline, with the deal expected to close in the first half of 2026 [10][11] - The acquisition of Tourmaline Bio for $1.4 billion adds a phase III-ready candidate to Novartis' cardiovascular pipeline [13] - Novartis has entered into collaboration agreements with Monte Rosa Therapeutics and Arrowhead Pharmaceuticals to expand its research capabilities [14][15]

Core Insights - Avidity Biosciences has launched a Managed Access Program (MAP) for its investigational therapy delpacibart zotadirsen (del-zota) aimed at individuals with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the U.S. [1][2] - The MAP is designed to provide del-zota to eligible patients quickly, with enrollment expected to begin by the end of 2025 [2][3] - Avidity plans to submit a Biologics License Application (BLA) for del-zota in 2026 following a pre-BLA meeting with the FDA [3] Company Overview - Avidity Biosciences focuses on developing a new class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs) [6] - The company aims to address previously unreachable targets and diseases using its proprietary AOC platform [6] - Avidity is advancing clinical programs for rare muscle diseases, including Duchenne muscular dystrophy, and has received multiple designations from the FDA for del-zota [5][6] Product Details - Del-zota is designed to skip exon 44 of the dystrophin gene, enabling dystrophin production in patients with DMD44 [5] - The Phase 1/2 EXPLORE44 trial demonstrated significant increases in dystrophin production and improvements in muscle function, with a favorable safety profile [5] - Del-zota has received designations such as Rare Pediatric Disease, Orphan Drug, Fast Track, and Breakthrough Therapy from the FDA [5]

Core Insights - Avidity Biosciences has entered into a definitive merger agreement with Novartis for a total equity value of approximately $12 billion, which is expected to maximize value for investors and enhance the global reach of Avidity's neuroscience pipeline [1][2][6] - Avidity's del-zota program has shown promising one-year data, demonstrating sustained muscle protection and significant improvement in disease progression for patients with Duchenne muscular dystrophy [1][6] - The company reported a strong balance sheet with approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2025, which is expected to fund operations until mid-2028 [1][6] Company Announcements - The merger with Novartis is set to close in the first half of 2026, following the separation of Avidity's early-stage precision cardiology programs into a new public company, SpinCo [1][6] - Avidity's CEO, Sarah Boyce, will transition to chair of the board of SpinCo, while Kathleen Gallagher will lead SpinCo as CEO [6] Financial Results - For the third quarter of 2025, Avidity reported collaboration revenues of $12.5 million, a significant increase from $2.3 million in the same period of 2024 [13] - Research and development expenses for Q3 2025 were $154.9 million, compared to $77.2 million in Q3 2024, reflecting increased costs associated with advancing key programs [13] - The net loss for Q3 2025 was $174.4 million, compared to a net loss of $80.4 million in Q3 2024 [13] Clinical Development Progress - Avidity's del-zota program has received Breakthrough Therapy designation from the FDA and is on track for a BLA submission in 2026 [6] - The company is advancing its del-desiran program for myotonic dystrophy type 1, with topline data from the Phase 3 HARBOR trial expected in the second half of 2026 [6] - Avidity is also progressing its del-brax program for facioscapulohumeral muscular dystrophy, with data readouts expected in 2026 [6]

Group 1 - Avidity Biosciences' share price surged by 42% following the announcement of its acquisition by Novartis, significantly outperforming the S&P 500's 1.2% increase [1] - Novartis will acquire Avidity for $72 per share in an all-cash transaction, representing a 46% premium over Avidity's closing price prior to the announcement [2] - The acquisition values Avidity at approximately $12 billion, with both companies' boards unanimously approving the deal [3] Group 2 - Novartis aims to enhance its neuroscience strategy through Avidity's innovative RNA therapeutics, specifically Antibody Oligonucleotide Conjugates (AOCs) targeting genetic neuromuscular disorders [4] - The acquisition is expected to close in the first half of 2026, pending regulatory approvals, while both companies will continue to operate independently [4] - Avidity was considered an attractive target due to its next-generation biotech capabilities [3]

Core Insights - Novartis is acquiring Avidity Biosciences for approximately $12 billion in cash to enhance its RNA-based disease-fighting pipeline [2][3] - Avidity shareholders will receive $72 per share, representing a 46.5% premium over Avidity's closing price prior to the announcement [2][6] - The acquisition will provide Novartis access to three late-stage experimental treatments for serious genetic neuromuscular diseases, utilizing Antibody Oligonucleotide Conjugates (AOC) [3][4] Company Impact - The deal is expected to close in the first half of next year and is projected to increase Novartis's sales compound annual growth rate from 5% to 6% for the period of 2024 to 2029 [4] - Avidity's stock surged over 40% following the announcement, reflecting strong market interest in RNA therapeutics [2][6] - Dyne Therapeutics, another company in the RNA therapy space, also saw its shares rise significantly, indicating a broader positive sentiment in the sector [5][6] Industry Context - The acquisition highlights the growing investor and industry interest in RNA therapeutics, particularly for neuromuscular diseases [3] - Novartis's commitment to innovative and targeted therapies is reinforced by this strategic acquisition, aiming to address progressive neuromuscular diseases [4]

Summary of Novartis Conference Call on Avidity Biosciences Acquisition Company and Industry - **Company**: Novartis - **Acquisition Target**: Avidity Biosciences - **Industry**: Biotechnology, specifically focusing on neuromuscular diseases and RNA therapeutics Core Points and Arguments 1. **Acquisition Details**: Novartis proposes to acquire Avidity Biosciences for $72 per share, representing a 46% premium to Avidity's closing price on October 24th, with a total transaction value estimated at $12 billion [4][25][32] 2. **Strategic Fit**: The acquisition is seen as a strong strategic fit for Novartis, enhancing its presence in neuromuscular diseases and expanding its RNA technology platform [4][6] 3. **Growth Potential**: The acquisition is expected to materially improve Novartis's growth profile, with three late-stage assets anticipated to unlock multiple near-term multibillion-dollar opportunities, with launches expected before 2030 [8][32] 4. **Long-term Outlook**: The assets acquired are projected to have no loss of exclusivity (LOE) before at least 2042, and they are exempt from the Inflation Reduction Act (IRA) [8][31] 5. **Financial Impact**: The acquisition is expected to raise Novartis's compound annual growth rate (CAGR) from 5% to 6% for 2024 to 2029, despite a short-term margin dilution of 1% to 2% [9][10][32] 6. **Technology Platform**: Avidity's AOC RNA platform allows for the delivery of RNA therapeutics to muscle tissues, which is a significant advancement in the field [11][49] 7. **Pipeline Overview**: The acquisition includes three key assets targeting myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD) [12][20][26] 8. **Regulatory Designations**: The assets have received various regulatory designations, including orphan drug and breakthrough therapy designations, which facilitate faster development and approval processes [14][20][26] 9. **Market Potential**: The combined patient populations for DM1 and FSHD are estimated to be between 125,000 and 167,000, indicating a substantial market opportunity [12][20] 10. **Commercial Strategy**: Novartis plans to leverage its existing commercial capabilities in neuromuscular diseases to drive rapid uptake of the new therapies [28][29] Other Important but Overlooked Content 1. **Spin-off Structure**: Avidity's early-stage precision cardiology programs will be separated into a new spin-off to streamline the acquisition process and manage third-party agreements [4][25][48] 2. **Clinical Trial Insights**: The Phase 1-2 studies for the assets have shown promising results, with significant improvements in functional measures and favorable safety profiles [16][21][27] 3. **Market Competition**: Novartis aims to be a market leader in the three diseases targeted by the acquisition, despite the presence of multiple competitors in the space [40][76] 4. **Future Applications**: The AOC RNA platform has potential applications beyond neuromuscular diseases, with ongoing research into its use for other organ systems [49][77] This summary encapsulates the key points discussed during the conference call regarding Novartis's acquisition of Avidity Biosciences, highlighting the strategic rationale, financial implications, and future growth potential within the biotechnology sector.

Summary of Avidity Biosciences Conference Call Company and Industry Overview - **Company**: Avidity Biosciences (NasdaqGM:RNA) - **Acquirer**: Novartis - **Industry**: Biotechnology, specifically focusing on RNA therapeutics and neuromuscular diseases Key Points and Arguments 1. **Acquisition Details**: Novartis proposes to acquire Avidity Biosciences for $72 per share, representing a 46% premium over Avidity's closing price on October 24, 2025 [4][25] 2. **Strategic Fit**: The acquisition is seen as a strong strategic fit for Novartis, enhancing its presence in neuromuscular diseases and expanding its RNA technology platform [4][6] 3. **Growth Potential**: The acquisition is expected to improve Novartis's medium and long-term growth profile, raising its CAGR from 5% to 6% for 2024 to 2029 [10][32] 4. **Late-Stage Assets**: Novartis will acquire three late-stage assets from Avidity, which are anticipated to unlock multiple near-term multibillion-dollar opportunities with launches expected before 2030 [8][32] 5. **Pipeline Overview**: The acquisition includes a preclinical neuromuscular pipeline and a platform for extrahepatic delivery of xRNAs, which is crucial for targeting RNA therapeutics beyond the liver [5][11] 6. **Disease Focus**: The focus is on high unmet needs in neuromuscular diseases, specifically targeting conditions like DM1 (myotonic dystrophy type 1) and FSHD (facioscapulohumeral muscular dystrophy) [7][20] 7. **Regulatory Designations**: Delbisiran for DM1 has received FDA orphan drug designation, FastTrack designation, and breakthrough therapy designation, indicating its potential as a transformative therapy [14][20] 8. **Market Potential**: The combined patient population for DM1 and FSHD is estimated to be between 125,000 and 167,000, with significant potential for market capture due to the lack of approved therapies [12][20] 9. **Financial Impact**: The total transaction value is estimated at $12 billion, with an enterprise value of $11 billion at the expected closing date in the first half of 2026 [25][32] 10. **Long-Term Outlook**: The assets acquired are expected to contribute significantly to Novartis's growth through the 2040s, with no loss of exclusivity (LOE) expected before 2042 [8][32] Additional Important Content 1. **Technology Platform**: Avidity's AOCs™ platform allows for targeted delivery of RNA therapeutics to muscle tissues, which is a significant advancement in the field [11][49] 2. **Clinical Trials**: The Phase 1-2 studies for Delbisiran and Delbrac have shown promising results, with significant improvements in patient-reported outcomes and biomarkers [17][21] 3. **Commercial Strategy**: Novartis plans to leverage its existing commercial capabilities in neuromuscular diseases to drive rapid uptake of the new therapies [28][30] 4. **Cost Synergies**: While cost synergies from the merger may be limited, Novartis expects to achieve productivity improvements and maintain a strong balance sheet [63][71] 5. **Regulatory Strategy**: Novartis aims to approach the FDA for accelerated approval based on compelling biomarker data from ongoing studies [72][73] This summary encapsulates the key points discussed during the conference call regarding the acquisition of Avidity Biosciences by Novartis, highlighting the strategic rationale, growth potential, and the expected impact on Novartis's portfolio and market position.

Transaction Overview - Novartis will acquire all outstanding shares of Avidity for USD 7200 per share in cash, representing a 46% premium to the October 24 closing price[8] - The total transaction value is USD 12 billion on a fully diluted basis, representing an enterprise value of approximately USD 11 billion at the expected closing date[80] - The transaction is expected to close in H1 2026, pending SpinCo separation and customary conditions[8,80] Strategic Rationale - The acquisition strengthens the Neuroscience franchise by adding three late-stage neuromuscular programs[14] - It enhances the mid-to-long-term growth profile, with substantial sales and profit growth expected through the 2040s, and LOEs not before 2042 and IRA-exempt[14] - Avidity raises Novartis' expected 2024-2029 sales CAGR from +5% to +6%, bolstering mid-single-digit long-term growth[21,80] Avidity's Core Value Drivers - Del-desiran is on track to be the first approved drug for DM1, targeting approximately 80,000 patients in the US and Europe[28,29] - Del-brax is on track to be the first approved drug for FSHD, targeting approximately 45,000-87,000 patients in the US and Europe[28,44] - Del-zota in DMD44 targets approximately 900 patients in the US, with an FDA submission expected in 2026[28,58,63]