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]

Core Viewpoint - PTC Therapeutics has received a positive opinion from the CHMP of the EMA for the marketing authorization of Sephience™ (sepiapterin) to treat phenylketonuria (PKU), addressing a significant unmet medical need in Europe [1][2]. Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing differentiated medicines for rare disorders [7]. Product Information - Sephience is an oral formulation of synthetic sepiapterin that acts through a dual mechanism to enhance the activity of the phenylalanine hydroxylase (PAH) enzyme, effectively reducing blood phenylalanine levels [5]. - The product is designed to treat a broad range of PKU patients, including those with severe disease subtypes [5]. Regulatory Developments - The European Commission is expected to ratify the marketing authorization for Sephience in approximately two months, which will apply to all 27 EU member states, as well as Iceland, Norway, and Liechtenstein [3]. - The New Drug Application (NDA) for sepiapterin is currently under review by the U.S. FDA, with a target action date of July 29, 2025 [4]. Market Potential - There are an estimated 58,000 people living with PKU globally, indicating a significant market opportunity for Sephience [6]. - Launch preparations for Sephience are underway, with a focus on Germany and other key European markets [2].

Core Viewpoint - Soleno Therapeutics, Inc. will report its first quarter 2025 financial results and operational highlights on May 7, 2025, followed by a conference call to discuss the results and business operations [1]. Group 1: Financial and Operational Updates - The financial results and operational highlights will be announced after the close of U.S. financial markets on May 7, 2025 [1]. - A conference call and webcast will take place at 4:30 PM Eastern Time on the same day to discuss the results [1][2]. Group 2: Product Information - Soleno's primary product, VYKAT XR, is indicated for treating hyperphagia in adults and pediatric patients aged 4 years and older with Prader-Willi syndrome [3][9]. - VYKAT XR is a once-daily oral treatment, previously known as DCCR [9]. Group 3: Safety Information - VYKAT XR has contraindications for patients with known hypersensitivity to diazoxide or thiazides [4]. - Warnings include the risk of hyperglycemia and fluid overload, necessitating regular monitoring of fasting plasma glucose and signs of edema [5][6]. - Common adverse reactions reported include hypertrichosis, edema, hyperglycemia, and rash [7].

Core Insights - Zevra Therapeutics, Inc. has successfully closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, enhancing its financial flexibility for strategic priorities [1][2] Financial Performance - As of March 31, 2025, Zevra reported available unaudited cash, cash equivalents, and investments totaling $68.7 million. After the sale of the PRV, net cash proceeds of $148.3 million will increase total cash, cash equivalents, and investments to $217.0 million [3] Company Overview - Zevra Therapeutics is a commercial-stage company focused on developing therapies for rare diseases with limited or no treatment options, aiming to bring life-changing therapeutics to patients [4]

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].

Financial Data and Key Metrics Changes - The company reported record fourth quarter and full year 2024 results, with total revenues of $190.6 million, representing a year-over-year increase of 45% on an as-reported basis and 24% on an organic basis [10][38] - Adjusted non-GAAP EBITDA for the fourth quarter was $50 million, compared to $30.2 million in the prior year period, reflecting strong operational performance [43] - The company raised its 2025 revenue guidance to between $756 million and $776 million, representing growth of 23% to 26% over 2024 [8][44] Business Line Data and Key Metrics Changes - Rare disease was the primary driver of growth, with Cortrophin Gel generating close to $200 million in sales in its third year since launch [9] - Revenues from rare disease more than doubled to $87 million in the fourth quarter, with Cortrophin Gel revenues at $59.4 million, up 42% from the prior year [39] - The generics business delivered $78.6 million in revenues, an increase of 9% over the fourth quarter of 2023, driven by operational excellence and new product launches [20][40] Market Data and Key Metrics Changes - The overall ACTH market is expected to have grown about 25% to approximately $660 million in 2024, with Cortrophin Gel's growth contributing significantly [27] - The company estimates that the addressable patient population for ILUVIEN and YUTIQ is approximately six to ten times higher than the current number of patients on therapy [19][64] - The generics segment is expected to see low double-digit growth in 2025, supported by new product launches and a strong base business [22][46] Company Strategy and Development Direction - The company aims to broaden its presence in the rare disease space, as evidenced by the acquisition of Alimera Sciences [9] - The focus will remain on rare disease as the primary growth driver, with continued investment in R&D and commercial initiatives to support this segment [55] - The company is also expanding its ophthalmology sales team and investing in marketing to increase awareness of its products [13][34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth prospects for Cortrophin Gel and the overall rare disease market, despite near-term challenges related to market access for ILUVIEN and YUTIQ [19][64] - The company anticipates a typical Q4 to Q1 drop in sales for Cortrophin Gel due to prescription reauthorizations, followed by strong sequential growth in subsequent quarters [44][66] - Management highlighted the importance of addressing unmet needs in autoimmune disorders and inflammatory diseases, which could significantly expand the addressable market for Cortrophin Gel [28] Other Important Information - The company has taken steps to enhance supply security for ILUVIEN and YUTIQ, including extending its partnership with its contract manufacturer [15] - A prefilled syringe for Cortrophin Gel is under FDA review, expected to launch in the second quarter of 2025, aimed at improving patient convenience [30][31] - The company has redefined its reporting segments to better reflect its business structure post-acquisition [38] Q&A Session Summary Question: Business development and M&A capacity - Management indicated a thoughtful approach to leverage ratios, historically keeping it under three, and expressed intent to pursue additional business development and M&A without straining the balance sheet [53][54] Question: Gout's contribution to Cortrophin sales - Currently, 15% of Cortrophin's volume comes from gout, which is seen as a gateway indication for new prescribers [56][57] Question: Access issues for ILUVIEN and YUTIQ - Management acknowledged access issues due to funding shortfalls for patient assistance programs but remains confident in the long-term growth prospects for these products [64][65] Question: Seasonality impact on Cortrophin - Typical Q4 to Q1 dynamics are expected, with a drop in sales followed by sequential growth, supported by strong early trends [66] Question: Transitioning supply from EyePoint to Siegfried - Management is confident in the transition and has built up inventory to ensure supply security during the change [70][73]

Financial Data and Key Metrics Changes - For the full year 2024, total revenues grew by 23.5% year over year to $491.7 million, exceeding previous guidance [6][24] - Full year net product revenue reached $489.3 million, a 23.4% increase over 2023, driven by the successful launch of AGAMRI [6][24] - In Q4 2024, total revenues and net product revenues reached $141.8 million, an increase of 28.3% and 30% over Q4 2023, respectively [7][24] - GAAP net income for 2024 was $163.9 million, or $1.38 per basic share, a 130% increase year over year [28] - Non-GAAP net income for 2024 was $276.3 million, or $2.33 per basic share, representing a 95% increase year over year [29] Business Line Data and Key Metrics Changes - Firdapse's net product revenue for 2024 was $306 million, an 18.4% increase year over year [25] - AGAMRI generated $46 million in net product revenue from its launch on March 13, 2024, through year-end [25] - Fycampa's net product revenue for 2024 was $137.3 million, slightly down from $138.1 million in 2023 due to changes in gross to net arrangements [25][30] Market Data and Key Metrics Changes - The company ended 2024 with a cash position of $517.6 million and no debt, enhancing its capacity for strategic investments [8][33] - The company expects 2025 total revenue to be between $545 million and $565 million [7] Company Strategy and Development Direction - The company is focused on a buy and build approach for long-term growth, actively evaluating rare disease opportunities [13] - The international strategy includes building a sustainable network of out-licensing partners and targeting markets where products can enhance access to care [14] - The company aims to leverage its rare disease expertise and operational excellence to drive sustained growth [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth of Firdapse, expecting organic growth to exceed 15% in 2025 [20] - The company is committed to navigating market and regulatory challenges while adapting to industry changes [15] - Management highlighted the importance of patient engagement services and strong relationships with healthcare communities [8] Other Important Information - The company secured a favorable resolution to the Teva patent litigation, reinforcing Firdapse's intellectual property strength [10] - The SUMMIT study for AGAMRI is underway, aiming to provide real-world data on its long-term benefits [11] Q&A Session Summary Question: Can you provide more color on new patient adds and the low discontinuation rate for Firdapse? - Management noted over 500 pipeline leads for LEMS patients, contributing about 50% to new enrollments each month, with a steady discontinuation rate of 15% [40][41] Question: What is the plan for replacing revenue from Fycampa in 2026? - Management remains open to orphan and differentiated products, including potential opportunities in epilepsy [44] Question: What indications are being pursued for AGAMRI? - The focus is on further characterizing AGAMRI for its immunosuppressive capabilities in rare diseases [50] Question: How does the clarity on Firdapse IP protections shape investment? - Should a full ten-year opportunity arise, the company would consider aggressive investments in the oncology side and other research opportunities [96][97] Question: What is the expected impact of generic erosion on Fycampa? - Management expects some price and volume erosion post-patent expiry but believes the product will maintain a considerable market share [80][100]

Table of Contents As filed with the Securities and Exchange Commission on July 21, 2023 Registration No. 333- UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 REGISTRATION STATEMENT Under Delaware 3841 77-0523891 (Primary Standard Industrial Classification Code Number) The Securities Act of 1933 (I.R.S. Employer Identification Number) 203 Redwood Shores Pkwy, Suite 500 Redwood City, CA 94065 (650) 213-8444 (Address, Including Zip Code, and Telephone Number, Including Area Cod ...

CANbridge Pharmaceuticals (01228) Update / Briefing June 07, 2023 12:00 PM ET Speaker0 Good day, ladies and gentlemen. Welcome to Cambridge's Live Marley Mainland China approval call. It's our great honor to have the following management with us here today. Doctor James Hsu, founder, chairman, and CEO of Cambridge Pharmaceuticals. Doctor. Gerald Cox, Chief Development Strategist Marcelo Tureski, Chief Commercial Officer and Glenn Hassan, Chief Financial Officer. Management team will give us a presentation o ...