Biogen (BIIB) Q1 2025 Earnings Call May 01, 2025 08:30 AM ET Speaker0 My name is Melinda, and I'll be your conference operator today. At this time, I'd like to welcome everyone to the Biogen First Quarter twenty twenty five Earnings Call and Business Update. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session. Today's conference is being recorded. Thank you. I would now like to turn the conference over to Mr. Tim Power, ...

REDWOOD CITY, Calif., May 01, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism, today announced that management will participate in the following investor conferences: Event: The Citizens JMP Life Sciences Conference Date: May 7-8, 2025 Event: H.C. Wainwright BioConnect Investor Conference Date: May 20, 2025 Event: Craig-Hallum Institu ...

1Q 2025 Total Revenue of $125.2M, a 15% Increase Year-over-Year at CER Expanding Portfolio through In-Licensing of DMX-200 Phase 3 Program for Rare Kidney Disease with Significant Market Potential in the U.S. Maintaining 2025 Guidance for Galafold, Reflecting Strong Underlying Demand Updating 2025 Pombiliti + Opfolda Guidance with New Patient Starts Accelerating in 2H Adjusting 2025 Total Revenue Growth Guidance to 15-22% at CER Reiterating GAAP Profitability During H2 2025 Conference Call and Webcast Toda ...

Amicus licenses exclusive U.S. rights to commercialize DMX-200 for the treatmentof Focal Segmental Glomerulosclerosis (FSGS) DMX-200 is in a pivotal Phase 3 trial for FSGS, a rare and fatal kidney disease with no FDA-approved therapies Dimerix successfully completed Type C meeting with the FDA in March 2025, aligning on proteinuria as the primary endpoint for approval Dimerix to receive US$30 million (~AU$48 million) upfront payment, up to US$560 million (~AU$892 million) for success-based milestone paymen ...

Core Insights - The year 2024 was a transformational period for Santhera Pharmaceuticals, marked by the successful launch of AGAMREE® for Duchenne muscular dystrophy (DMD) in Germany, Austria, and the U.S. [1][4][12] - The company secured financing of up to CHF 69 million, which is expected to support growth initiatives and provide liquidity until cash flow break-even in 2026 [1][10][67] - Santhera aims to expand its product portfolio and establish itself as a leading specialty pharmaceutical company in rare diseases [1][4] Operational Highlights - AGAMREE was launched in Germany and Austria, with over 300 patients on treatment by year-end, representing nearly 30% of those on steroid treatment [4][16] - Catalyst Pharmaceuticals launched AGAMREE in the U.S. in March 2024, achieving USD 46 million in revenue for the year, exceeding guidance [4][19] - The partnership with Sperogenix in China initiated an early access program, with commercial rollout expected in mid-2025 [4][20] Financial Highlights - Total revenue for 2024 was CHF 39.1 million, a decrease from CHF 103.4 million in 2023, primarily due to significant licensing milestones recognized in the previous year [4][49] - Product sales amounted to CHF 14.8 million, driven by the successful launch of AGAMREE in Germany and Austria [4][49] - Operating loss for 2024 was CHF 33.2 million, compared to a profit of CHF 68.8 million in 2023, influenced by the absence of prior out-licensing milestones [10][56] Market Access and Reimbursement - AGAMREE received full reimbursement in Germany and Austria, facilitating patient access [22][23][24] - In the UK, AGAMREE was recommended as a cost-effective treatment by NICE, with availability for prescription starting in April 2025 [25][18] - Ongoing negotiations for pricing and reimbursement in other EU countries are expected to progress through 2025 and 2026 [17][26] Partnerships and Licensing - Santhera's partnership with Catalyst Pharmaceuticals in the U.S. includes tiered royalties and potential milestone payments, enhancing market access [34][66] - The collaboration with Sperogenix in China is set to provide tiered royalties and milestone payments, supporting commercialization efforts [35][66] - Santhera is pursuing a hybrid model of direct commercialization in key markets while establishing distribution agreements in secondary territories [36][38] Manufacturing and Supply Chain - Santhera is validating additional manufacturing sites to meet global demand and improve supply chain efficiencies [39][40] - A secondary manufacturing site in the U.S. is being qualified to support growing demand and mitigate supply risks [41] - The company is also exploring local production options in China to enhance pricing and reimbursement discussions [42] R&D Strategy and Pipeline Development - Santhera will focus on maximizing AGAMREE's potential in DMD and generating long-term safety data, with results from the GUARDIAN study expected in Q4 2025 [46] - The company is actively seeking to expand its late-stage pipeline through licensing and distribution agreements, particularly in the rare disease field [47] Financial Guidance and Outlook - For 2025, Santhera expects total revenues in the range of CHF 65-70 million, driven by continued global rollout of AGAMREE [71] - The company anticipates operating expenses to be between CHF 50-55 million, excluding non-cash share-based compensation [71] - Long-term revenue guidance includes expectations of EUR 150 million by 2028, excluding milestones [72]

- Label includes full spectrum of PKU patients -- European launch preparations underway -WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive ...

REDWOOD CITY, Calif., April 23, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that it will report its first quarter 2025 financial results, as well as operational highlights, after the close of the U.S. financial markets on Wednesday, May 7, 2025. The Company will host a conference call and webcast at 4:30 PM Eastern Time to discuss the results and provide an update o ...

As biotech investors know, companies in the industry can sometimes earn significant returns in relatively short periods, like a year, thanks to excellent clinical or regulatory progress. And based on Wall Street's projections, several notable biotechs could see their shares soar in the next 12 months.Of course, it wouldn't be wise to rush to invest in these companies for that reason alone, but it might be worth looking deeper into what the Street considers severely undervalued drugmakers. Read on to learn m ...

Core Insights - Soligenix Inc. is a late-stage biopharmaceutical company focused on developing products for rare diseases with unmet medical needs [1][5] - The company has selected IBN to lead its corporate communications efforts, leveraging IBN's extensive distribution network to enhance visibility [1][4] Company Overview - Soligenix operates through two main segments: Specialized BioTherapeutics, which targets oncology and inflammation therapies, and Public Health Solutions, which focuses on vaccines and therapeutics for biothreats and infectious diseases [2] - The company is advancing multiple late-stage clinical programs, including HyBryte™ (SGX301) for cutaneous T-cell lymphoma, and other candidates for psoriasis (SGX302), oral mucositis (SGX942), and Behçet's disease (SGX945) [2][5] Public Health Initiatives - The Public Health Solutions segment includes development programs for vaccines against ricin poisoning (RiVax®), Ebola (SuVax™), and Marburg (MarVax™), supported by approximately $60 million in non-dilutive government grants and contracts [3][6] - The vaccine development utilizes proprietary heat stabilization technology known as ThermoVax® [6] Strategic Partnerships - IBN will utilize its investor-focused distribution network, which includes over 5,000 syndication outlets and a large social media following, to increase awareness for Soligenix [4][5] - IBN has over 19 years of experience and has assisted more than 500 client partners, positioning it to effectively reach a broad audience [5]

Core Viewpoint - Soligenix, Inc. is focused on advancing its clinical programs and achieving significant development milestones, particularly in the treatment of rare diseases with unmet medical needs, while managing its financial resources effectively [2][9]. Recent Accomplishments - The company has opened patient enrollment for its confirmatory Phase 3 study evaluating HyBryte™ in early-stage cutaneous T-cell lymphoma (CTCL) and has reported positive interim results from an open-label study of HyBryte™ [7][9]. - Formation of a European Medical Advisory Board to provide strategic guidance for the ongoing clinical studies [7]. Financial Results - For the year ended December 31, 2024, Soligenix reported revenues of $0.1 million, a decrease from $0.8 million in the previous year, primarily due to timing issues with government grant funding [4]. - The net loss for the year was $8.3 million, or ($4.98) per share, compared to a net loss of $6.1 million, or ($12.66) per share, in the prior year, influenced by a reverse stock split and increased research and development expenses [5][6]. - Research and development expenses rose to $5.2 million from $3.3 million, driven by costs related to new clinical trials [6]. Cash Position - As of December 31, 2024, the company had approximately $7.8 million in cash, which is expected to support operations through 2025 while exploring various strategic options for funding [2][8].