Summary of Supernus Pharmaceuticals FY Conference Call Company Overview - **Company**: Supernus Pharmaceuticals (NasdaqGM:SUPN) - **Event**: 37th Annual Piper Sandler Healthcare Conference - **Date**: December 02, 2025 Key Points Industry and Product Focus - **Product in Focus**: APO-go, a treatment for movement disorders, particularly for patients with advanced Parkinson's disease [1][2] - **Market Demand**: There is significant demand for APO-go, with physicians expressing strong support for the product despite current supply constraints [3][4] Supply Chain Challenges - **Current Situation**: The existing manufacturer is facing capacity issues, unable to meet the overwhelming demand for APO-go [5][6] - **Resolution Efforts**: Supernus is exploring multiple avenues to resolve supply constraints, including discussions with the FDA and potential partnerships with alternative suppliers [2][3][10] - **Timeline for Solutions**: Onboarding a new manufacturer could take several months to a year, depending on regulatory approvals and readiness [7][8][9] Patient Demand and Enrollment - **Patient Enrollment Forms (PEFs)**: Despite supply issues, physicians continue to submit PEFs, indicating ongoing interest in APO-go [14][15] - **Patient Demographics**: Initial demand is primarily from patients with advanced Parkinson's disease who have limited treatment options [19][20] Financial Projections - **Sales Estimates**: Initial peak sales estimates for APO-go are projected between $200 million and $300 million, which may need to be revisited once supply issues are resolved [20][21] - **Long-term Outlook**: The long-term potential for APO-go remains strong, as it offers a unique treatment option not available in the current market [21][22] Competitive Landscape - **Market Competition**: APO-go faces competition from other products like Vyalev and Apokyn, but it serves a different purpose as a rescue medication [25][26] - **Product Differentiation**: APO-go is positioned as a unique treatment option, distinct from traditional therapies like Levodopa [21][22] New Product Developments - **Zurzuvae**: Another product in the portfolio targeting postpartum depression, with significant market potential as it addresses a large unmet need [32][33] - **Sales Strategy**: The sales force is primarily targeting OB-GYNs, with potential for expansion based on market response [34][36] M&A and Future Strategy - **M&A Focus**: Supernus is prioritizing commercial-stage assets and is open to acquiring later-stage development programs [47][48] - **Partnership with Biogen**: There is potential for discussions regarding the buyout of Biogen's 50% stake in Zurzuvae, although both companies are currently committed to the brand [46] Financial Synergies - **Cost Synergies**: Supernus anticipates realizing up to $200 million in annualized synergies from the acquisition of Sage Therapeutics [42] Research and Development - **Early-stage Assets**: Supernus is evaluating early-stage assets from Sage and its own pipeline to determine which programs to advance [43][44] Conclusion Supernus Pharmaceuticals is navigating significant supply chain challenges with its APO-go product while maintaining strong demand and interest from healthcare providers. The company is also expanding its product portfolio with Zurzuvae and exploring strategic M&A opportunities to enhance its market position.

Financial Data and Key Metrics Changes - The combined net revenue for Q3 2025 was approximately $3.1 million, representing a 4% increase compared to Q2 2025 revenue of approximately $3 million [4][18] - Loss from operations decreased by $24,000 from $7.2 million in Q3 2024 to $7.3 million in Q3 2025 [19] - Non-GAAP recurring EBITDA for Q3 2025 was a net loss of $8.9 million, compared to a loss of $9.2 million in Q3 2024 [20] - Net loss attributable to common shareholders decreased by $352,000 from $9.9 million in Q3 2024 to $9.5 million in Q3 2025 [20] Business Line Data and Key Metrics Changes - Mytesi prescription volume increased by approximately 0.9% in Q3 2025 over Q2 2025, but decreased by 3.6% compared to Q3 2024 [18] Company Strategy and Development Direction - The company aims to negotiate business development partnerships for licensed rights to develop and commercialize late-stage health products, focusing on generating non-dilutive funding [5][21] - Key late-stage initiatives include orphan indications of Crofelemer for intestinal failure associated with MVID and cancer therapy-related diarrhea [5][10] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about multiple expected near-term catalysts for Crofelemer, viewing them as significant and potentially transformative for patients and stakeholders [21] - The company anticipates that these catalysts will lead to collaborations, business development, and licensing deals, supporting late-stage products and programs towards regulatory approval [21] Other Important Information - Crofelemer has shown a groundbreaking reduction of parenteral support of up to 37% for intestinal failure patients, which is significant given the lack of approved treatments for MVID [8][9] - The company is in discussions with multiple potential animal health partners to expand the use of Canalivia for general diarrhea in dogs [14] Q&A Session Summary - No specific questions and answers were provided in the content, thus this section is not applicable.

Core Insights - Jaguar Health's subsidiary, Napo Pharmaceuticals, has submitted an orphan drug designation application to the FDA for crofelemer, aimed at treating diarrhea in adult patients with breast cancer that has metastasized to the brain [1] - The application is based on significant responder analysis results from the OnTarget study, which were presented at the Multinational Association of Supportive Care in Cancer 2025 Annual Meeting and the 2024 San Antonio Breast Cancer Symposium [1] - The FDA's Office of Orphan Products Development recognizes brain metastasis of any cancer as a distinct disease or condition from the primary site of origin [1] Company Developments - The submission of the orphan drug designation for crofelemer highlights the company's focus on addressing specific needs in the oncology space, particularly for patients with advanced breast cancer [1] - The potential treatment could enhance the therapeutic options available for patients undergoing targeted therapy with or without standard chemotherapy [1] Industry Context - The recognition of brain metastasis as a separate condition by the FDA underscores the evolving understanding of cancer treatment and the need for specialized therapies [1] - The ongoing research and development in the field of supportive care for cancer patients reflect a growing emphasis on improving quality of life alongside traditional cancer treatments [1]

Core Insights - Pulmovant has received orphan drug designation for mosliciguat from Japan's Ministry of Health, Labour and Welfare, highlighting the significant unmet medical need for patients with Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD) [1][2] - Mosliciguat is a novel, once-daily, inhaled soluble guanylate cyclase (sGC) activator currently in Phase 2 clinical trials, aiming to provide a targeted treatment option for PH-ILD [1][3] Company Overview - Pulmovant is a clinical-stage biotechnology company focused on developing treatments for pulmonary diseases and is a subsidiary of Roivant [7] - The company’s lead candidate, mosliciguat, is designed to be a first-in-class inhaled treatment for PH-ILD, with a differentiated mechanism of action [7][5] Clinical Development - Mosliciguat is currently being evaluated in the Phase 2 PHocus clinical study, which is a randomized, double-blind, placebo-controlled trial involving approximately 120 adult patients [3][6] - The Phase 1b ATMOS study demonstrated that inhaled mosliciguat was well tolerated and resulted in a mean peak reduction in pulmonary vascular resistance (PVR) of up to 38% [5] Market Context - PH-ILD is a progressive and life-threatening condition affecting up to 200,000 patients in the U.S. and Europe, with limited or no approved treatment options available [4] - The orphan drug designation provides Pulmovant with regulatory benefits, including priority consultation and up to 10 years of market exclusivity post-approval [2][6]

Core Insights - Sanofi has received FDA approval for Wayrilz (rilzabrutinib), marking it as the first BTK inhibitor approved for treating persistent or chronic immune thrombocytopenia (ITP) in the United States [1][7] - The approval was based on the phase III LUNA 3 study, which demonstrated positive outcomes in sustained platelet counts and ITP symptoms, achieving both primary and secondary endpoints [2][7] - Wayrilz is also under review in the EU and China for ITP treatment and has been recently approved in the UAE for the same indication [3] Company Developments - Patients using Wayrilz will benefit from Sanofi's HemAssist program, which offers support for treatments related to rare blood disorders [3] - In addition to ITP, Wayrilz is being developed for other rare diseases, including IgG4-related disease (IgG4-RD) and warm autoimmune hemolytic anemia (wAIHA), with orphan drug designations from the FDA [5][9] - A phase II study is currently evaluating Wayrilz for IgG4-RD, a chronic immune-mediated condition affecting multiple organs [8] Stock Performance - Sanofi's shares have increased by 2.6% this year, contrasting with a 0.6% decline in the industry [4]

Core Viewpoint - Sanofi's SAR446523, a GPRC5D monoclonal antibody, has received orphan drug designation from the FDA for the treatment of relapsed or refractory multiple myeloma, highlighting the company's commitment to developing innovative therapies for this rare disease [1][2]. Group 1: Product Information - SAR446523 is an investigational IgG1-based monoclonal antibody designed to target GPRC5D, which is highly expressed on plasma cells, and aims to enhance antibody-dependent cellular cytotoxicity [3]. - The drug is currently undergoing a phase 1 clinical study in patients with relapsed or refractory multiple myeloma, with the clinical study identifier NCT06630806 [3]. Group 2: Disease Context - Multiple myeloma is the second most common hematologic malignancy, with over 180,000 new diagnoses globally each year, yet it remains incurable with a five-year survival rate of approximately 62% for newly diagnosed patients [4]. - There is a significant need for new therapeutic options, particularly for patients who are ineligible for transplants, due to high attrition rates in subsequent lines of therapy [4]. Group 3: Company Commitment - Sanofi is dedicated to advancing oncology treatments and aims to transform cancer care through the development of innovative therapies for rare and difficult-to-treat cancers [5]. - The company emphasizes its commitment to addressing urgent healthcare challenges and improving the lives of patients through its research and development efforts [6].

Core Viewpoint - Sanofi's SAR446523, a GPRC5D monoclonal antibody, has received orphan drug designation from the FDA for the treatment of relapsed or refractory multiple myeloma, highlighting the company's commitment to developing innovative therapies for this rare disease [1][2]. Group 1: Product Information - SAR446523 is an investigational IgG1-based monoclonal antibody designed to target GPRC5D, which is highly expressed on plasma cells, and aims to enhance antibody-dependent cell-mediated cytotoxicity [3]. - The drug is currently undergoing a phase 1 clinical study in patients with relapsed or refractory multiple myeloma, with the clinical study identifier NCT06630806 [3]. Group 2: Disease Context - Multiple myeloma is the second most common hematologic malignancy, with over 180,000 new diagnoses globally each year, yet it remains incurable with a five-year survival rate of approximately 62% for newly diagnosed patients [4]. - There is a significant need for new therapeutic options, particularly for patients who are ineligible for transplants, due to high attrition rates in subsequent lines of therapy [4]. Group 3: Company Commitment - Sanofi is dedicated to advancing oncology treatments and aims to transform cancer care through innovative therapies for rare and difficult-to-treat cancers [5]. - The company emphasizes its long-standing commitment to oncology and the development of first and best-in-class immunological and targeted therapies [5][6].

Core Points - Qualigen Therapeutics, Inc. has entered into definitive securities purchase agreements for the sale of Series A-3 Preferred Stock, raising a total of $4.5 million through the issuance of 4,500 shares at a stated value of $1,000 per share [1][2] - The Series A-3 Preferred Stock is convertible into 1,607,143 shares of common stock at a conversion price of $2.80 per share, subject to adjustments [1] - The net proceeds from this private placement will be used for working capital purposes [2] Regulatory and Compliance - The Series A-3 Preferred Stock was offered in a private placement under Section 4(a)(2) and/or Rule 506(b) of Regulation D under the Securities Act of 1933 and has not been registered under the Securities Act or applicable state securities laws [3] - The company has agreed to file a registration statement with the SEC covering the resale of the common stock underlying the Series A-3 Preferred Stock within 45 days after the closing date [4] Company Overview - Qualigen Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing novel therapeutics for cancer and infectious diseases, with a pipeline that includes QN-302, QN-247, and small-molecule RAS oncogene inhibitors [6]

Clinical Development & Regulatory - Initial proof-of-concept results show crofelemer reduced Total Parenteral Nutrition (TPN) in patients with MVID and SBS-IF by up to 27% and 12.5% respectively[20], potentially modifying disease progression in intestinal failure patients[17] - Crofelemer achieved statistically significant improvement in monthly responder analysis in a prespecified subgroup of adult patients with breast cancer from the OnTarget Phase 3 study[23] - The company plans to meet with the FDA in Q2 2025 to discuss submitting a supplemental NDA for crofelemer for adult breast cancer patients on targeted therapy[21] - Napo Therapeutics is pursuing accelerated marketing PRIME authorization from the European Medicines Agency (EMA) for TPN reduction for MVID, as there are no alternative treatments[67] Commercial & Financial - Jaguar Health is a commercial-stage company with approximately $12 million in annual net revenue[13] - The company estimates the U S market revenue potential for Mytesi for HIV-related diarrhea to be approximately $30-$50 million in gross annual sales[27] - The global Short Bowel Syndrome (SBS) market is projected to reach $4.6 billion by 2027, with a CAGR of 26% from 2020 to 2027[27] - Net Q1 2025 revenue was approximately $2.2 million, a decrease of approximately 6% versus net Q1 2024 revenue of approximately $2.4 million and 37% versus net Q4 2024 revenue of approximately $3.5 million[110] Strategic Initiatives - Magdalena Biosciences, a joint venture focused on mental health illnesses, is valued at $5 million based on initial funding of $1 million from One Small Planet[103] - The company plans to pursue a Priority Review Voucher (PRV) for NP-300, with PRVs having sold for values ranging from $67 million to $350 million in past transactions[10, 97]

Core Viewpoint - The FDA has granted orphan drug designation to rilzabrutinib for sickle cell disease, highlighting its potential to address unmet medical needs in rare diseases [1][2]. Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [7]. Product Information - Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that aims to restore immune balance through multi-immune modulation [5]. - The drug has received multiple orphan drug designations, including for immune thrombocytopenia (ITP), warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease (IgG4-RD) [2][8]. Disease Context - Sickle cell disease affects over 100,000 people in the US, predominantly impacting African American and Hispanic populations, leading to severe pain and reduced life expectancy [6]. - The disease is characterized by misshapen red blood cells that block blood flow, causing various health complications [6]. Regulatory Status - Rilzabrutinib is currently under regulatory review in the US, EU, and China for its potential use in ITP, with a target action date for FDA decision set for August 29, 2025 [3][8].