Financial Performance and Strategy - Novavax envisions a world where its technology impacts billions, driving global health transformation[2] - The company aims for long-term value creation through partnering and R&D innovation, targeting a diversified revenue base[9] - Novavax projects the global market opportunity for infectious disease and oncology immunotherapeutics to exceed $100 billion by the 2030s[19] - The company is focused on a lean and efficient operating model, targeting non-GAAP profitability as early as 2028[10, 37] - Novavax achieved approximately $1.4 billion in cash flow in 2024 and 2025, with 78% derived from non-dilutive sources[42, 44] Partnerships and Pipeline - Novavax secured a partnership with Sanofi and is actively seeking new partnerships to unlock growth[10, 13] - The company's growth strategy involves generating new data and proof points for its technology to strengthen IP and expand the utility of Matrix-M[14] - Novavax's pipeline includes Nuvaxovid, R21/Matrix-M malaria vaccine, and early-stage candidates for C diff, shingles, and RSV[33] - In 2025, Nuvaxovid achieved over 12% market share in Japan via the Takeda partnership, and R21/Matrix-M malaria vaccine achieved 85% share in a growing market[16] - Long-term core spend profile of approximately $250 million R&D+SG&A, net of partner reimbursement[37]

ATLANTA, Jan. 14, 2026 (GLOBE NEWSWIRE) -- Holzer & Holzer, LLC is investigating whether Travere Therapeutics, Inc. (“Travere” or the “Company”) (NASDAQ: TVTX) complied with federal securities laws. On January 13, 2026, Travere disclosed that the U.S. Food and Drug Administration has extended the review timeline of its supplemental New Drug Application for FILSPARI® (sparsentan) in focal segmental glomerulosclerosis. Following this news, the price of the Company’s stock dropped. If you purchased Travere sto ...

Summary of Rocket Pharmaceuticals FY Conference Call (January 14, 2026) Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Biotechnology, specifically focused on gene therapies for rare genetic diseases - **Core Focus**: Development of gene therapies targeting complex and rare genetic diseases, particularly in the cardiovascular space Key Points and Arguments Financial Position - As of September 30, 2025, Rocket reported **$222.8 million** in cash, cash equivalents, and restricted cash, expected to fund operations into the second quarter of **2027** [2] - Anticipated approval of **Kresladi** on March 28, 2026, could yield a **PRV** (Priority Review Voucher), providing strategic non-dilutive capital to extend the cash runway [2] Strategic Focus - 2026 is designated as a year of execution, with a concentrated effort on advancing three cardiovascular programs [2] - The company has deprioritized investments in **Fanconi anemia** to focus on areas with immediate impact [3] Cardiovascular Programs - **Danon Disease**: - A severe X-linked cardiomyopathy with a prevalence of **15,000 to 30,000** in the U.S. and Europe [8] - The lead program shows **100% LAMP2 expression** durability in treated patients, with a median reduction of **24%** in LV mass index over time [9][11] - The program is viewed as a significant near-to-medium-term value driver due to its clear differentiation and market potential [28] - **PKP2 Arrhythmogenic Cardiomyopathy (ACM)**: - Estimated to affect **50,000** adults and children in the U.S. and Europe [17] - The program is currently in phase one, with preliminary safety and efficacy results indicating well-tolerated gene therapy [18] - The company aims to define a registrational pathway for this program in 2026 [20] - **BAG3 Dilated Cardiomyopathy (DCM)**: - Considered a potential largest market opportunity with around **30,000** individuals affected in the U.S. [24] - The program is in the process of initiating a phase one trial, focusing on safety and preliminary efficacy [25] Regulatory and Clinical Development - The company is working towards resuming patient dosing for Danon disease, with logistics being the primary gating factor [29] - A concurrent natural history study for Danon is ongoing, validating the rapid decline of untreated patients [35] - The regulatory review for Kresladi is mid-cycle, with no current showstoppers noted [37] Manufacturing Capabilities - Rocket has in-house manufacturing capabilities for AAV, which helps control costs and timelines [38] - The facility is designed to support current and future programs, ensuring high margins and process improvements over time [38] Future Outlook - The anticipated approval of Kresladi is seen as a pivotal moment for transitioning to a commercial-stage gene therapy company [26] - The company plans to maintain a steady pipeline with additional programs ready to advance in the future [40] Additional Important Insights - The company emphasizes the importance of achieving meaningful myocardial transduction and protein localization for clinical benefit [21] - There is a strong community interest in the Danon program, despite past challenges, indicating a significant unmet medical need [32] - The company is cautious but optimistic about the regulatory landscape and the potential for future growth [37] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, financial position, and advancements in their gene therapy programs.

Rapport Therapeutics (NasdaqGM:RAPP) FY Conference January 14, 2026 02:15 PM ET Company ParticipantsJeffrey Sevigny - CMOAbraham Ceesay - CEOConference Call ParticipantsNone - AnalystTyler Van Buren - AnalystAbraham CeesayOkay.OperatorYes.Abraham CeesayThank you.OperatorYou're all set. Forward that, please.Abraham CeesayGreat. Thank you.OperatorYep.Tyler Van BurenWelcome, everybody. My name is Tyler Van Buren, and I'm welcoming you to the 44th JP Morgan Healthcare Conference. Our next presentation is Rappor ...

PresentationPresenting company of the day is Fulcrum Therapeutics. And presenting on behalf of the company, we have CEO, Alex Sapir. Alex?Welcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I am one of the senior biotech analysts here at JPMorgan. I'm joined by my squad, Rati Pinge, Priyanka Grover and Joyce Zhou. Our first.Alexander SapirCEO, President & Director Thank you, Anupam. Thank you, JPMorgan, and thank you for your excellent report that you issued earlier ...

PresentationGood morning, and welcome, everyone. I'm [indiscernible] I'm an associate on the health care team at JPMorgan in New York. Today, it's my pleasure to introduce Tango Therapeutics. With me on stage, we have Malte Peters, CEO; Barbara Weber, Executive Chairman and former CEO; and Adam Crystal, President of R&D.Barbara WeberFounder & Executive Chair Thank you very much, and thanks for the opportunity to be here today. As you just heard, until last Thursday, I was the CEO of Tango Therapeutics. And ...

Presentation Unknown Analyst My name is [ Henry Chang ]. I'm with the banking team here at JPMorgan, and it's my pleasure to introduce Larimar Therapeutics today. Presenting today will be Dr. Carole Ben-Maimon, President and CEO of Larimar. Just a quick note, we'll have time for Q&A after the presentation. So please wait until then for any questions, and we'll get you a microphone. But without further ado, I'll turn it over to you, Carole. Carole Ben-MaimonCEO, President & Director Thank you. Thank you, ...

PresentationOur next presenting company is Agios, and presenting on behalf of the company, we have CEO, Brian Goff. Brian, over to you.Welcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Tess Romero, and I'm one of the senior biotech analysts here at JPMorgan.Brian GoffCEO & Director All right. Thank you, Tess, and good morning, everybody. It is great to be back at JPMorgan, and I'm really excited to share our outlook for 2026 and why we're so excited about the path forward for ...

PresentationWelcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I am one of the senior biotech analysts here at JPMorgan. I'm joined by my squad, [ Rathi Pinhe ], Joyce Zhou and Priyanka Grover. Our next presenting company is CytomX and presenting on behalf of the company, we have CEO, Sean McCarthy. Sean?Sean McCarthyChairman & CEO Thank you, Anupam, and thanks to the JPMorgan team for the invitation to present. It really is a privilege to be here. I'm also joined ...

SAN FRANCISCO, Jan. 14, 2026 /PRNewswire/ -- National shareholder rights firm Hagens Berman is investigating Alvotech (NASDAQ: ALVO) regarding the propriety of its disclosures concerning the company's manufacturing operations and the regulatory status of its lead biosimilar candidate, AVT05. The investigation follows a staggering 34% stock drop after Alvotech revealed it received a "Complete Response Letter" (CRL) from the FDA. ...