Financial Performance & Guidance - BRINSUPRI achieved preliminary unaudited U S net revenues of approximately $144 6 million in Q4 2025 and $172 7 million for the full year 2025[23] - ARIKAYCE generated worldwide revenue of approximately $433 8 million in FY 2025, representing a 19 3% increase year-over-year[27] - The company anticipates ARIKAYCE revenue to be between $450 million and $470 million for the full year 2026[28] Clinical Development Programs - The company expects topline data readout from the Phase 3 ENCORE trial for ARIKAYCE in March/April 2026, potentially leading to label expansion[30, 31] - Phase 3 trials for TPIP in PAH, PPF, and IPF are expected to initiate in early and the second half of 2026 respectively[13, 38] - Topline data readout from the Phase 2 CEDAR trial for brensocatib in Hidradenitis Suppurativa is expected in Q2 2026[46, 50] Pipeline Expansion - The company plans to file Investigational New Drug (IND) applications for RA and IBD in the second half of 2026[13, 50] - IND filing for Stargardt is anticipated in 2026[13, 59] - Phase 2 programs in ILD and moderate to severe asthma are planned for INS1148[13, 40] Commercial Expansion - BRINSUPRI is expected to launch in the EU and UK, pending regulatory approval[13, 44] - BRINSUPRI is also anticipated to launch in Japan, pending regulatory approval[13, 44] Cash Position - The company reported a cash position of $1 7 billion as of September 30, 2025[62]

January 12, 2026 DELIVERING THE NEW BIOGEN: THE NEXT CHAPTER OF INNOVATION AND GROWTH CHRISTOPHER A. VIEHBACHER PRESIDENT AND CHIEF EXECUTIVE OFFICER FORWARD-LOOKING STATEMENTS This presentation and discussions during this webcast contain forward-looking statements, relating to: our strategy and plans; potential of, and expectations for, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development programs, clinical trials, and data readouts and presentatio ...

Key Points A director at Mirum Pharmaceuticals sold 5,000 shares of the company for $400,000 on Dec. 22. The entire transaction was direct, involving the exercise of vested stock options, immediately sold as shares. No indirect entities or trusts were involved. These 10 stocks could mint the next wave of millionaires › Saira Ramasastry, a director at Mirum Pharmaceuticals (NASDAQ:MIRM), executed the exercise of 5,000 stock options followed by an immediate open-market sale of the resulting shares ...

Biohaven is planning a pivotal study of BHV-1300 in Graves' disease in 2026. Reports positive early clinical experience from first and only clinically validated, extracellular protein degraders using Biohaven's proprietary MoDEâ"¢ and next-generation, highly selective TRAPâ"¢ degraders exclusively licensed from Yale University. Early clinical experience in patients demonstrates rapid removal of circulating disease-causing proteins, resulting in robust clinical improvement with a well-tolerated profile to da ...

- Preliminary unaudited Q4 and Full Year 2025 net Attruby® product revenue of $146.0 million and $362.4 million, respectively - Attruby (acoramidis) is rapidly becoming the first-choice therapy for newly diagnosed ATTR-CM patients with 6,629 unique patient prescriptions written by 1,632 prescribers as of December 31, 2025, driven by differentiated clinical data and growing real-world confidence - New TTR amyloid depleter antibody program announced to explore the potential of ATTR-CM disease reversal; progra ...

- Preliminary unaudited Q4 and Full Year 2025 net Attruby® product revenue of $146.0 million and $362.4 million, respectively - Attruby (acoramidis) is rapidly becoming the first-choice therapy for newly diagnosed ATTR-CM patients with 6,629 unique patient prescriptions written by 1,632 prescribers as of December 31, 2025, driven by differentiated clinical data and growing real-world confidence - New TTR amyloid depleter antibody program announced to explore the potential of ATTR-CM disease reversal; progra ...

Ascendis Pharma A/S J.P. Morgan Healthcare Conference San Francisco January 2026 This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such data and estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which w ...

Bristol-Myers Squibb Company (NYSE:BMY) is one of the cheap stocks to buy for the next 3 years. On January 9, Scotiabank analyst Louise Chen raised the firm’s price target on Bristol-Myers to $60 from $53, while maintaining a Sector Perform rating on the shares. With the stock trading at an inexpensive valuation and several key milestones on the horizon, the firm views the upcoming catalyst-rich year as a strong buying opportunity. In other news, on December 11, the FDA granted Priority Review to Bristol ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the promising stocks to buy under $50. On January 8, Morgan Stanley analyst Maxwell Skor lowered the firm’s price target on Ultragenyx to $50 from $55 and kept an Overweight rating on the shares. The firm projects continued outperformance for US SMID-cap biotech in 2026. This bullish outlook is driven by commercial-stage companies transitioning from capital consumers to producers, combined with a looming patent cliff that threatens the revenue of large ...

Core Viewpoint - Processa Pharmaceuticals is actively participating in the 44th Annual J.P. Morgan Healthcare Conference to discuss its clinical pipeline and engage with investors and potential partners [1][2]. Group 1: Company Overview - Processa Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing Next Generation Cancer (NGC) therapies with improved safety and efficacy [6]. - The company's NGC drugs are modifications of existing FDA-approved oncology therapies aimed at enhancing the metabolism and distribution of these drugs while preserving their cancer-killing mechanisms [6]. Group 2: Clinical Pipeline - The ongoing Phase 2 study is evaluating NGC-Cap in patients with advanced or metastatic breast cancer, with preliminary data indicating that NGC-Cap increases exposure to capecitabine's cancer-killing metabolites while maintaining a safety profile similar to standard capecitabine monotherapy [3][5]. - A formal interim analysis from the first 20 patients enrolled in the Phase 2 study is expected to be completed in early 2026 [3]. Group 3: Strategic Engagement - George Ng, CEO, and Patrick Lin, Chief Business & Strategy Officer, will be available for one-on-one meetings with investors during the conference to discuss the company's clinical pipeline [2][4].