Core Insights - Vanda Pharmaceuticals Inc. is participating in the American Academy of Neurology Annual Meeting, presenting a novel therapeutic approach for Charcot-Marie-Tooth disease type 2S (CMT2S) [1][2] - The presentation will showcase VCA-894A, a personalized medicine treatment tailored to a specific genetic mutation, demonstrating significant improvements in neuromuscular function [1][2] - VCA-894A has received orphan designation from the FDA and is expected to be administered to the patient for whom it was developed, highlighting its potential in addressing unmet medical needs [2] Presentation Details - The presentation titled "Translating IGHMBP2 Variants with a CMT2S Patient-specific Organ-on-a-chip Model: Personalized Medicine ASO-based Therapeutic Rescue" will take place on April 9, 2025 [1] - The poster session will include novel unpublished data on conduction velocity in addition to previously published findings [3] Company Overview - Vanda Pharmaceuticals is focused on developing innovative therapies to meet high unmet medical needs and improve patient lives [5]

Core Insights - Vanda Pharmaceuticals Inc. has submitted a New Drug Application (NDA) to the FDA for Bysanti™ (milsaperidone) aimed at treating acute bipolar I disorder and schizophrenia, supported by multiple clinical studies [1][3] - Bysanti™ is classified as an atypical antipsychotic and is believed to work by interacting with various neurotransmitter receptors in the brain [2] - If approved, Bysanti™ could be available in the US by 2026, with potential patent exclusivity extending into the 2040s [3] Company Overview - Vanda Pharmaceuticals is a global biopharmaceutical company focused on developing innovative therapies to meet high unmet medical needs [4]

Product Portfolio - Vanda Pharmaceuticals' commercial portfolio includes three products: Fanapt for bipolar I disorder and schizophrenia, HETLIOZ for Non-24-Hour Sleep-Wake Disorder and Smith-Magenis syndrome, and PONVORY for relapsing forms of multiple sclerosis[24]. - HETLIOZ is the first product approved by the FDA for Non-24 and for patients with Smith-Magenis syndrome[26]. - The company is focused on obtaining FDA approval for Bysanti for bipolar I disorder, schizophrenia, and major depressive disorder[12]. - Fanapt generated net sales of $94.3 million in the U.S. for schizophrenia treatment in 2024[29]. - HETLIOZ achieved net sales of $76.7 million in the U.S. for Non-24 treatment in 2024[29]. - PONVORY was approved for relapsing forms of multiple sclerosis (MS) and launched in the U.S. in April 2021[36]. - The product pipeline includes Bysanti for bipolar I disorder and schizophrenia, currently in clinical development[39]. - PONVORY has shown potential for treating inflammatory/autoimmune disorders, with IND applications accepted by the FDA in Q4 2024[44]. Commercial Success and Challenges - The company is dependent on the commercial success of its products, particularly Fanapt, HETLIOZ, and PONVORY[20]. - Vanda Pharmaceuticals faces generic competition for HETLIOZ, which may impact future performance[20]. - Vanda Pharmaceuticals has encountered challenges with third-party payors refusing to cover prescriptions for HETLIOZ[20]. - The company relies on a limited number of specialty pharmacies for the distribution of HETLIOZ in the U.S.[20]. - The company aims to maximize the commercial success of Fanapt, HETLIOZ, and PONVORY[28]. - The company reported challenges with third-party payor coverage affecting HETLIOZ prescriptions[20]. - Vanda's revenues from Fanapt and PONVORY are significantly reliant on a small number of customers[20]. Research and Development - Vanda Pharmaceuticals is committed to advancing novel approaches in drug discovery, including the use of genetics and genomics[25]. - The research and development organization employs rigorous project management techniques to limit the risk profile of the product pipeline[38]. - The company is actively investing in research and development to enhance its product offerings and maintain competitive advantage[79]. - The research and development strategy emphasizes pharmacogenetics and pharmacogenomics to differentiate products and limit risk in the product pipeline[38]. - The company is exploring new indications for existing products, including MDD for milsaperidone and various inflammatory disorders for PONVORY[44]. Regulatory and Legal Matters - The FDA issued a complete response letter (CRL) for HETLIOZ's sNDA for jet lag disorder on August 16, 2019, citing unclear clinical significance of improved sleep measures[41]. - The FDA accepted the sNDA for HETLIOZ in insomnia, with a target action date of March 4, 2024, but received a CRL on that date[42]. - The FDA may require post-marketing clinical trials to further assess a drug's safety and effectiveness after NDA approval[121]. - The company is pursuing legal action against the FDA regarding the delay in the approval process for HETLIOZ for jet lag disorder[41]. Financial Obligations and Partnerships - The company has paid $37.5 million in upfront fees and milestone obligations to Bristol-Myers Squibb for HETLIOZ, with a royalty period of 10 years on net sales[68]. - Tradipitant has remaining milestone obligations of $10 million and $5 million for marketing authorization approvals in the U.S. and E.U., respectively, and up to $80 million for sales milestones[70]. - An upfront payment of $10 million was made to Anaptys for the exclusive global license of imsidolimab, with potential future payments of up to $35 million for regulatory approval and sales milestones[72]. - The company plans to enter strategic partnerships to enhance capabilities and extend commercial reach[28]. Market Dynamics and Competition - The pharmaceutical industry is highly competitive, with established companies having greater resources and commercial infrastructures[97]. - The development of competing drug technologies may impact the company's market position and product competitiveness[97]. - The company is exploring potential mergers and acquisitions to further strengthen its market position and expand its product portfolio[80]. Employee and Operational Insights - The company had 368 full-time employees as of December 31, 2024, an increase from 203 employees as of December 31, 2023[167]. - The company has not experienced any work stoppages and considers employee relations to be good, supporting its human capital objectives[167]. Compliance and Regulatory Environment - Regulatory compliance is crucial, with potential penalties for non-compliance including product recalls and fines[104]. - The company is subject to stringent regulations regarding the marketing and sale of its products[20]. - Compliance with state laws regarding pharmaceutical marketing and promotion requires significant resources, with potential civil penalties for non-compliance[148]. Future Outlook - Future guidance suggests continued growth in the treatment methods, with projections indicating a rise in unit sales for the upcoming quarters[80]. - The company is actively monitoring global health crises that could adversely impact its business operations[20].

Financial Data and Key Metrics Changes - Total revenues for the full year 2024 were $198.8 million, a 3% increase compared to $192.6 million for the full year 2023 [34] - Net loss for the full year 2024 was $18.9 million compared to net income of $2.5 million for the full year 2023 [40] - Operating expenses for the full year 2024 were $239.4 million compared to $206.6 million for the full year 2023, driven by higher SG&A expenses [42] Business Line Data and Key Metrics Changes - Fanapt net product sales were $94.3 million for the full year 2024, a 4% increase compared to $90.9 million for the full year 2023 [35] - Hetlioz net product sales were $76.7 million for the full year 2024, a 23% decrease compared to $100.2 million for the full year 2023, due to generic competition [36] - Ponvory net product sales were $27.8 million for the full year 2024, marking the fourth full quarter of revenue recognition following its acquisition [39] Market Data and Key Metrics Changes - Fanapt net product sales for the fourth quarter of 2024 were $26.6 million, an 18% increase compared to $22.6 million in the fourth quarter of 2023 [43] - Hetlioz net product sales for the fourth quarter of 2024 were $20 million, a 5% decrease compared to $21.1 million in the fourth quarter of 2023 [45] - Ponvory net product sales were $6.5 million in the fourth quarter of 2024, an increase of 11% compared to $5.9 million in the third quarter of 2024 [47] Company Strategy and Development Direction - The company is focusing on expanding its sales force for Fanapt from 200 to 300 representatives to increase reach and frequency [12][52] - Vanda plans to file a BLA for Imsidolimab later this year and is exploring registration in Europe and Japan for other inflammatory disorders [28][29] - The company targets annual revenue in excess of $750 million in 2030 for its psychiatry portfolio, assuming potential approvals of Vantrela and Fanapt LAI [59][60] Management's Comments on Operating Environment and Future Outlook - Management noted that the commercial launches of Fanapt and Ponvory are expected to contribute to revenue growth in 2025 and beyond [51] - The company anticipates that the introduction of the Medicare benefit redesign will negatively impact gross-to-net for its products, particularly Fanapt and Hetlioz [55][99] - Management expressed confidence in the growth trajectory of Fanapt, with expectations of continued adoption as prescribers gain experience [12][54] Other Important Information - The company reported a decrease in cash, cash equivalents, and marketable securities to $374.6 million as of December 31, 2024 [43] - Vanda has initiated a direct-to-consumer advertising campaign for Fanapt to increase awareness among bipolar disorder patients [13][70] Q&A Session Summary Question: What is the source of growth for Fanapt? - Management indicated that growth is primarily coming from prescribers treating bipolar disorder, rather than schizophrenia [65][66] Question: Will the expanded sales force be sufficient to compete with larger pharma companies? - Management believes the current sales force size is likely on the lower side but emphasizes the importance of quality training [67] Question: What is the target of the direct-to-consumer advertising? - The campaign specifically targets individuals with bipolar I disorder, aiming to increase awareness and confidence in the drug [70] Question: What is the rationale behind the phase three study for Vantrela in major depressive disorder? - Management noted that previous experience with Fanapt suggests a once-a-day administration could be effective and convenient for patients [75] Question: What is the anticipated timing for the development of the iloperidone long-acting injectable? - Management stated that the program is ready to initiate imminently, with enrollment potentially taking up to one year [92] Question: Does the 2025 revenue guidance include contributions from Tradipitant or Imsidolimab? - Management confirmed that the guidance is based solely on currently commercialized products and indications [93] Question: What changes in reimbursement are anticipated in 2025? - The introduction of the Medicare benefit redesign is expected to impact gross-to-net significantly [99]

Vanda Pharmaceuticals (VNDA) came out with a quarterly loss of $0.08 per share versus the Zacks Consensus Estimate of a loss of $0.14. This compares to loss of $0.04 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 42.86%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $0.17 per share when it actually produced a loss of $0.09, delivering a surprise of 47.06%.Over the last four quarters, ...

Revenue Performance - Q4 2024 total revenues reached $53.2 million, representing a 17% increase compared to Q4 2023[5] - Full year 2024 total revenues were $198.8 million, a 3% increase from $192.6 million in 2023[10] - Fanapt net product sales for Q4 2024 were $26.6 million, an 18% increase compared to Q4 2023[6] - HETLIOZ net product sales decreased by 23% to $76.7 million for the full year 2024 due to generic competition[10] - Full year 2025 revenue is expected to grow to between $210 million and $250 million[5] - Psychiatry portfolio revenue is projected to exceed $750 million by 2030[5] - Fanapt® net product sales increased to $26,649,000 in Q4 2024 from $22,599,000 in Q4 2023, representing a growth of 9.1%[26] - HETLIOZ® net product sales decreased to $20,044,000 in Q4 2024 from $21,072,000 in Q4 2023, a decline of 4.9%[26] - PONVORY® net product sales surged to $6,492,000 in Q4 2024 from $1,600,000 in Q4 2023, marking a significant increase of 304.5%[26] - Total revenues for Q4 2024 reached $53,185,000, up from $45,271,000 in Q4 2023, reflecting a growth of 17.5%[26] Expenses and Losses - Operating expenses for Q4 2024 totaled $63,464,000, compared to $52,365,000 in Q4 2023, an increase of 21.2%[26] - Vanda's net loss for Q4 2024 was $4.9 million, compared to a net loss of $2.4 million in Q4 2023[14] - Net loss for Q4 2024 was $4,912,000, compared to a net loss of $2,400,000 in Q4 2023, indicating a worsening of 104.2%[26] Cash and Assets - Cash and cash equivalents totaled $374.6 million as of December 31, 2024, a decrease of $1.6 million from the previous quarter[10] - Cash and cash equivalents decreased to $102,316,000 as of December 31, 2024, down from $135,821,000 as of December 31, 2023[28] - Total assets increased to $656,204,000 as of December 31, 2024, compared to $648,440,000 as of December 31, 2023[28] - Stockholders' equity decreased to $538,546,000 as of December 31, 2024, down from $544,910,000 as of December 31, 2023[28] - The company reported a total accumulated deficit of $174,292,000 as of December 31, 2024, compared to $155,392,000 as of December 31, 2023[28] Future Plans - Vanda submitted a New Drug Application (NDA) for Bysanti for bipolar disorder and schizophrenia expected in Q1 2025[5] - The company plans to submit a Biologics License Application (BLA) for imsidolimab in 2025 for generalized pustular psoriasis[11]

Core Insights - Vanda Pharmaceuticals reported strong revenue growth driven by its psychiatry portfolio, particularly Fanapt, Hetlioz, and Ponvory, with expectations for continued growth in 2025 and beyond [2][5][14] - The company has several key product applications pending, including Bysanti for bipolar disorder and schizophrenia, and Tradipitant for motion sickness, which are expected to contribute to future revenue growth [2][5][9] Financial Highlights - Q4 2024 total revenues reached $53.2 million, a 17% increase compared to Q4 2023 [5][6] - Full year 2024 total revenues were $198.8 million, a 3% increase compared to 2023 [5][6] - Fanapt net product sales in Q4 2024 were $26.6 million, an 18% increase from Q4 2023 [5][6] - The company expects full year 2025 revenues to grow to between $210 million and $250 million [5][14] Operational Highlights - Vanda's psychiatry portfolio is projected to exceed $750 million in revenue by 2030 [5][14] - The company initiated a Phase III program for the long-acting injectable formulation of Fanapt in Q4 2024 [11] - Vanda's acquisition of Ponvory was completed on December 7, 2023, and its commercial launch began in Q3 2024 [6][11] Key Regulatory Milestones - The NDA for Tradipitant for motion sickness was submitted in Q4 2024, with potential approval expected in 2025 [11][12] - Bysanti's NDA for bipolar disorder and schizophrenia is expected to be submitted in Q1 2025 [5][11] - The MAA for HETLIOZ for Smith-Magenis syndrome was submitted in Q4 2024 [5][11] Cash Position - As of December 31, 2024, Vanda had cash, cash equivalents, and marketable securities totaling $374.6 million, a decrease of $1.6 million from the previous quarter [6][11]

Company Overview - Vanda Pharmaceuticals Inc. is a leading global biopharmaceutical company focused on developing and commercializing innovative therapies to address high unmet medical needs and improve patient lives [5]. Upcoming Financial Results - Vanda will release its fourth quarter and full year 2024 financial results on February 13, 2025, after market close [1]. - A conference call will be held on the same day at 4:30 PM ET to discuss the financial results and other corporate activities [2]. Conference Call Details - Participants can join the conference call by dialing 1-800-715-9871 for domestic calls or 1-646-307-1963 for international calls, using passcode 2881765 [2]. - The conference call will be broadcast simultaneously and archived on Vanda's website, with investors advised to register at least 15 minutes early [3]. Replay Information - A replay of the conference call will be available starting at 8:30 PM ET on February 13, 2025, until February 20, 2025, at 11:59 PM ET [4]. - The replay call-in number is 1-800-770-2030 for domestic callers and 1-609-800-9909 for international callers, with the same passcode [4].

Core Viewpoint - Vanda Pharmaceuticals is challenging the FDA's rejection of its New Drug Application (NDA) for tradipitant, a treatment for gastroparesis, citing procedural violations and insufficient consideration of evidence [4][5][6][17]. Company Overview - Vanda Pharmaceuticals Inc. is focused on developing innovative therapies to address unmet medical needs, particularly in the field of gastroparesis, a condition with no new drug approvals in over 40 years [18]. Drug Development and Clinical Trials - Tradipitant, a neurokinin 1 receptor antagonist, has been studied for nearly a decade, with over 1,000 patients treated, including ongoing treatment through an expanded access program [3][4]. - The company conducted two placebo-controlled studies and one open-label study to assess the efficacy and safety of tradipitant [3]. FDA Review Process - The FDA rejected Vanda's NDA in September 2024, claiming that substantial evidence of efficacy was not established [4]. - Vanda argues that the FDA did not adhere to legal requirements, delaying the review process by six months and failing to provide a hearing opportunity as mandated [5][6]. Efficacy Evidence - In Study 1, tradipitant significantly improved nausea severity compared to placebo (p value = 0.0099) [9]. - Study 2 showed no statistically significant difference between tradipitant and placebo (p value = 0.7411) [10]. - Vanda conducted additional analyses suggesting tradipitant's superiority over placebo, which the FDA disputed [11]. Regulatory Standards - Vanda contends that the FDA's interpretation of the "substantial evidence" standard is overly stringent, requiring two well-controlled studies with significant results, which is not consistent with statutory language [15][16]. - The company emphasizes that the substantial evidence standard is relatively low and should allow for the approval of effective drugs, especially for conditions like gastroparesis [14][16]. Next Steps - Vanda has accepted the FDA's offer for a hearing regarding the NDA, which must commence within 120 days [17].

Core Message - Vanda Pharmaceuticals Inc has issued a letter to FDA Commissioner Robert M Califf MD highlighting concerns over the FDA's review process for its New Drug Application (NDA) for tradipitant a treatment for gastroparesis [1][2][3] FDA Review Process - Vanda submitted an NDA for tradipitant for the treatment of gastroparesis on September 18 2023 and received a Complete Response Letter (CRL) from the FDA on September 18 2024 [2][4] - The CRL was criticized by Vanda for lacking reasoned explanations and failing to engage with the evidence presented including voluminous evidence from experts [4] - Vanda expressed surprise at the disregard for facts evidence and basic scientific principles in the CRL which they argue does not reflect a legitimate regulatory review [4] FDA Advisory Committees - FDA denied Vanda's request to convene an Advisory Committee to consider the tradipitant application [5] - The number of Advisory Committee meetings convened by the FDA has drastically declined from 55% of drug applications in 2010 to 6% in 2021 [5] - FDA Commissioner Robert Califf has stated a preference for less voting by advisory committees and expressed skepticism about the value of votes which Vanda argues contributes to a lack of public scrutiny of FDA decisions [5] Communication and Accountability - Vanda wrote to Dr Nikolov expressing concerns about the CRL but received no acknowledgment or response three months later [4] - Vanda criticizes the FDA's opacity in decision-making and oversight which they argue fosters a culture of obfuscation and close-mindedness [3][4] - Vanda emphasizes the need for FDA policies practices and culture to align with scientific evidence and the law [6] Company Background - Vanda Pharmaceuticals Inc is a global biopharmaceutical company focused on developing and commercializing innovative therapies to address high unmet medical needs [9]