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Core Viewpoint - Applied Therapeutics, Inc. has announced key executive appointments, promoting Evan Bailey to Chief Medical Officer and Dottie Caplan to Executive Vice President of Patient Advocacy and Government Affairs, effective June 15, 2025 [1][5]. Group 1: Executive Appointments - Evan Bailey, MD, has been promoted to Chief Medical Officer, succeeding Riccardo Perfetti, MD, PhD, who served since 2018 [5]. - Dottie Caplan has been promoted to Executive Vice President, Patient Advocacy and Government Affairs, expanding her role to include government affairs [5][6]. Group 2: Leadership Contributions - Dr. Bailey has been with Applied Therapeutics for four years and has played a critical role in advancing the company's development programs, particularly in clinical development execution [2][3]. - Ms. Caplan has been instrumental in integrating patient voices into clinical development strategies and will continue to lead advocacy efforts in her new role [2][4]. Group 3: Focus on Rare Diseases - The company is dedicated to developing treatments for rare diseases, with its lead drug candidate, govorestat, targeting conditions such as Classic Galactosemia and CMT-SORD [7]. - Dr. Bailey expressed commitment to advancing the portfolio of supporting data for govorestat to address high unmet medical needs in rare disease patients [2][3].

Core Viewpoint - Processa Pharmaceuticals has entered into a binding term sheet with Intact Therapeutics for the exclusive option to license PCS12852, a 5-HT4 receptor agonist aimed at treating gastroparesis and other gastrointestinal motility disorders [1][3]. Financial Terms - Processa is set to receive a $2.5 million option exercise fee, up to $20 million in development and regulatory milestone payments, and over $432.5 million in commercial milestone payments based on net product sales [2][8]. - Intact will pay Processa a double-digit royalty on worldwide net sales of licensed products, excluding South Korea, and provide an equity stake of 3.5% in Intact upon closing [2][8]. Clinical Significance - PCS12852 has shown a favorable safety and efficacy profile in clinical studies, particularly in a Phase 2a trial for diabetic gastroparesis, a condition with limited treatment options [3][4]. - The drug is designed to restore normal gastric emptying without the cardiovascular and central nervous system side effects associated with older agents in this class [3]. Strategic Partnership - The partnership is expected to unlock the value of Processa's non-oncology assets while focusing on developing next-generation cancer therapies [3]. - Intact Therapeutics aims to address significant unmet clinical needs in gastrointestinal diseases, enhancing patient quality of life through innovative therapies [3][5]. Company Background - Intact Therapeutics is a clinical-stage biopharmaceutical company focused on next-generation therapies for gastrointestinal diseases, leveraging proprietary thermal hydrogel technology [5]. - Processa Pharmaceuticals specializes in developing next-generation cancer drugs with improved safety and efficacy, modifying existing FDA-approved oncology therapies [7].

Core Insights - The study successfully established the safety and tolerability of VTX3232 in patients with early-stage Parkinson's disease, with no drug-related treatment-emergent adverse events reported [1][2] - VTX3232 demonstrated significant reductions in NLRP3-related biomarkers in both cerebrospinal fluid (CSF) and plasma, indicating sustained target engagement [1][3] - The company plans to initiate a placebo-controlled Phase 2 trial for VTX3232 in Parkinson's disease and potentially in other neurodegenerative disorders like Alzheimer's disease [3][6] Study Details - The Phase 2a study evaluated a 40mg oral daily dose of VTX3232 in ten patients over a 28-day treatment period, focusing on safety and tolerability [3][4] - Key secondary objectives included pharmacokinetic profiling and measuring effects on biomarkers of NLRP3 inhibition, with significant reductions observed in IL-1, IL-6, and hsCRP [3][4] - The study showed that VTX3232 maintained plasma and CSF levels above the IC90 for IL-1b for 24 hours [3][9] Biomarker Findings - VTX3232 treatment resulted in reductions of biomarkers such as IL-1β, IL-18, IL-6, and hsCRP, with some approaching the limit of quantitation [9] - Statistically significant improvements were noted in motor and non-motor symptoms of Parkinson's disease, as measured by MDS-UPDRS [9] - No acute changes were observed in exploratory PET imaging, consistent with the short duration of the study [9] Future Development - Ventyx intends to present the complete dataset at a future medical meeting and publish full results in a peer-reviewed journal [5] - The company is also conducting a 12-week Phase 2 trial of VTX3232 in participants with obesity and cardiometabolic risk factors, with topline results expected in H2 2025 [1][8] - Planning for a double-blind, placebo-controlled, dose-ranging Phase 2 trial in Parkinson's disease is underway [6]

Core Viewpoint - Relmada Therapeutics has appointed Dr. Raj S. Pruthi as Chief Medical Officer-Urology to advance the development of NDV-01, a novel therapy for non-muscle invasive bladder cancer (NMIBC), with a Phase 3 trial expected to begin in the first half of 2026 [2][4]. Company Overview - Relmada Therapeutics is a clinical-stage biotechnology company focused on innovative therapies for oncology and central nervous system conditions [11]. - The lead program, NDV-01, is designed for the treatment of NMIBC and aims to address significant unmet medical needs in this area [11]. Appointment of Dr. Raj S. Pruthi - Dr. Pruthi brings over 25 years of experience in urologic oncology, clinical trials, and robotic surgery, having previously served as Chief Medical Officer at enGene Holdings Inc. and Global Medical Affairs leader at Johnson and Johnson [3][5]. - His expertise includes developing practice guidelines for NMIBC and executing global clinical studies for bladder cancer treatments [3][6]. NDV-01 Program - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel, designed for intravesical administration, allowing for gradual drug release over 10 days [9]. - The formulation aims to enhance local exposure while minimizing systemic toxicity and is designed for in-office administration in under 10 minutes without the need for anesthesia [9]. - Positive initial Phase 2 data presented at the AUA 2025 indicated impressive response rates and favorable tolerability for NDV-01 [4][10]. Market Opportunity - NMIBC accounts for approximately 75% of all bladder cancer cases, with a high recurrence rate of 50-75% over seven years, indicating a significant market opportunity for effective treatments [10]. - With over 600,000 prevalent cases in the U.S. and limited treatment options, NDV-01 has the potential to serve as a frontline or salvage therapy across multiple NMIBC subtypes [10].

ORION CORPORATION PRESS RELEASE 17 JUNE 2025 at 9.00 EEST Orion and Glykos announce the extension of their research collaboration and licensing agreement for the development of next-generation ADCs Orion Corporation and Glykos Finland Oy today announced that they have extended their research collaboration and licensing agreement for the development of next-generation antibody-drug conjugates (ADCs). Under the extended agreement, Orion gains access to Glykos’ proprietary ADC technologies with the potential ...

Core Insights - Vivoryon Therapeutics N.V. reported its Q1 2025 financial results and operational progress, focusing on the development of varoglutamstat for kidney diseases, particularly diabetic kidney disease (DKD) [2][3] Financial Performance - Revenues for Q1 2025 were zero, consistent with Q1 2024 [14] - Research and development expenses decreased by EUR 6.2 million to EUR 1.2 million in Q1 2025 from EUR 7.4 million in Q1 2024, primarily due to lower third-party expenses [14][16] - General and administrative expenses were EUR 1.3 million in Q1 2025, down from EUR 2.1 million in Q1 2024 [16] - Net loss for Q1 2025 was EUR 2.5 million, compared to EUR 9.3 million in Q1 2024 [17] - Cash and cash equivalents as of March 31, 2025, were EUR 7.0 million, down from EUR 9.4 million as of December 31, 2024 [17] Clinical Development - The company is advancing varoglutamstat in kidney disease, with a planned Phase 2b study in DKD as a priority for 2025 [9] - A meta-analysis confirmed that varoglutamstat at 600mg twice daily significantly improved eGFR kidney function, with effects sustained until week 96 [7] - Preclinical data showed a synergistic effect of varoglutamstat in combination with SGLT-2 inhibitors, indicating potential for enhanced treatment regimens [5][6] Intellectual Property - A new composition of matter patent for varoglutamstat was granted in the U.S., providing exclusivity through 2044, with potential extensions [10] - The company is actively pursuing additional patents related to varoglutamstat and its applications in kidney disease [10] Corporate Developments - Vivoryon entered into a Standby Equity Purchase Agreement (SEPA) with Yorkville Advisors for up to EUR 15 million in ordinary shares over the next 36 months, enhancing financial flexibility [12] - Julia Neugebauer was appointed as Chief Operating Officer, effective May 1, 2025, to oversee investor relations and corporate functions [13] Pipeline Updates - The company has nominated a new candidate, VY2149, a next-generation QPCT/L inhibitor, expected to enter late-stage preclinical development [11]

Core Viewpoint - CSL has received FDA approval for ANDEMBRY®, a novel treatment for hereditary angioedema (HAE), marking a significant advancement in the management of this rare genetic disorder [1][3][11] Group 1: Product Overview - ANDEMBRY is the first monoclonal antibody developed entirely by CSL, targeting factor XIIa to prevent HAE attacks in patients aged 12 and older [1][3][11] - The treatment offers once-monthly subcutaneous self-injection, providing a convenient administration method [1][3] - ANDEMBRY has shown a median reduction of more than 99% in HAE attacks compared to placebo, with a least squares mean reduction of 89.2% [6][7] Group 2: Clinical Trial Data - The approval is based on data from the pivotal Phase 3 VANGUARD trial, which demonstrated the efficacy and safety of ANDEMBRY [3][12] - In the pivotal trial, 62% of patients treated with ANDEMBRY remained attack-free throughout the treatment period [7] - The most common adverse reactions reported were nasopharyngitis and abdominal pain, with injection-site reactions occurring in 14% of patients [4][7] Group 3: Market Impact and Availability - ANDEMBRY's approval expands CSL's HAE franchise and reinforces the company's commitment to innovation in the HAE community [6][8] - The product is set to launch commercially immediately, with availability expected before the end of June 2025 [9] - ANDEMBRY has also received approvals in multiple regions, including Australia, the UK, the EU, Japan, Switzerland, and the UAE [8]

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Core Insights - Roche (RHHBY) is advancing its pipeline candidate prasinezumab into phase III development for early-stage Parkinson's disease based on data from phase IIb PADOVA study and ongoing open-label extensions [1][7] - The candidate is a potential first-in-class anti-alpha-synuclein antibody targeting a known biological driver of Parkinson's disease progression [3] - Year-to-date, Roche's shares have increased by 20.9%, outperforming the industry growth of 4% [1] Development Details - The PADOVA study evaluated prasinezumab's safety and efficacy in 586 randomized patients, although it missed its primary endpoint of time to confirmed motor progression [4][5] - Despite missing statistical significance, positive trends toward reduced motor progression were observed at 104 weeks, with expectations of sustained effects based on additional open-label extension data [5][6] - The ongoing PASADENA and PADOVA open-label studies are assessing the long-term safety and efficacy of prasinezumab in over 750 individuals with early-stage Parkinson's disease [3] Licensing and Financials - Roche holds exclusive rights to prasinezumab under a licensing agreement with Prothena (PRTA) established in December 2013, which includes paying double-digit teen royalties on net sales [7][9] - Prothena has earned $135 million to date, with potential for up to $620 million in additional milestone payments [9] Industry Context - Developing treatments for Parkinson's disease is challenging due to its chronic and progressive nature, with both motor and non-motor symptoms [10] - Other companies, such as UCB and Novartis, are also working on treatments, with UCB's recent investigational drug failing to meet clinical endpoints [11]