Iloperidone

Revenues for Q1 2024 were $47.5 million, an increase of 5% compared to Q4 2023 Cash increase of $5.9 million in Q1 2024 to $394.1 million Fanapt® approved for the acute treatment of bipolar I disorder; commercial launch expected in Q3 2024 Milsaperidone NDA for schizophrenia and bipolar disorder expected to be submitted in early-2025 PONVORY® commercial launch expected in Q3 2024 Tradipitant NDA review for gastroparesis ongoing, PDUFA date of September 18, 2024 Tradipitant second Phase III motion sickness study results expected in Q2 2024; NDA expected to be submitted in Q4 2024 WASHINGTON, May 8, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced financial and operational results for the first quarter ended March 31, 2024. "We are very proud of our accomplishments in the first quarter of 2024, which were achieved with a small but efficient organization that is enthusiastic to continue developing and commercializing treatments for people who need them," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. "We expect several significant milestones in the coming months, including the launch of Fanapt in acute bipolar I disorder, the launch of Ponvory in multiple sclerosis, the potential approval of tradipitant in gastroparesis, the phase III results of tradipitant in motion sickness, the upcoming NDA filings of milsaperidone in psychiatric disorders and of tradipitant in motion sickness and the initiation of clinical programs in depression, psoriasis, ulcerative colitis and pediatric insomnia. We are confident that our robust revenue, strong cash position and efficient operations position us well for significant growth and value creation in the years to come." Financial Highlights Total net product sales from Fanapt®, HETLIOZ® and PONVORY® were $47.5 million in the first quarter of 2024, a 24% decrease compared to $62.5 million in the first quarter of 2023, and a 5% increase compared to $45.3 million in the fourth quarter of 2023. Fanapt® net product sales were $20.6 million in the first quarter of 2024, a 10% decrease compared to $22.9 million in the first quarter of 2023, and a 9% decrease compared to $22.6 million in the fourth quarter of 2023. HETLIOZ® net product sales were $20.1 million in the first quarter of 2024, a 49% decrease compared to $39.6 million in the first quarter of 2023, and a 5% decrease compared to $21.1 million in the fourth quarter of 2023. The decrease relative to the first quarter of 2023 was the result of continued generic competition in the U.S. PONVORY® net product sales were $6.8 million in the first quarter of 2024. The acquisition of PONVORY® from Actelion Pharmaceuticals Ltd. (Janssen), a Johnson & Johnson Company, was completed on December 7, 2023. Net loss was $4.1 million in the first quarter of 2024, compared to net income of $3.3 million in the first quarter of 2023, and net loss of $2.4 million in the fourth quarter of 2023. Cash, cash equivalents and marketable securities (Cash) was $394.1 million as of March 31, 2024, representing an increase to Cash of $5.9 million compared to December 31, 2023. Key Operational Highlights Psychiatry Portfolio Fanapt® (iloperidone): Vanda announced in April 2024 that the U.S. Food and Drug Administration (FDA) approved Fanapt® as a first line treatment of acute bipolar I disorder in adults. This approval of Fanapt® for acute bipolar I disorder significantly expands the addressable patient population. Patent exclusivity is expected to last at least through late 2027. Vanda is initiating a host of commercial activities, including the expansion of its existing sales force, a prescriber awareness program and a comprehensive marketing program. Milsaperidone: Vanda expects to submit a New Drug Application (NDA) for milsaperidone (also known as VHX-896 and P-88), the active metabolite of Fanapt®, in schizophrenia and acute bipolar I disorder to the FDA in early-2025. If approved, there are pending patent applications that, if issued, could extend exclusivity into the 2040s. Fanapt® LAI: Vanda expects to initiate a Phase III program for the long acting injectable (LAI) formulation of Fanapt® by the end of 2024. Fanapt® LAI could reach the U.S. market after 2026 and there are pending patent applications that, if issued, could extend exclusivity into the 2040s. Vanda is currently planning clinical programs to test the efficacy of Fanapt® and milsaperidone in the treatment of depressive symptoms which, if successful, will significantly expand the addressable patient population. HETLIOZ® (tasimelteon) Vanda is currently planning to initiate a HETLIOZ LQ® program in pediatric insomnia. Although exact estimates of prevalence of insomnia in children are difficult to quantify, it is estimated that 20-40% of children experience significant sleep problems.1,2 There are currently no approved treatments for pediatric insomnia. If ultimately approved for marketing, the addressable patient population for HETLIOZ LQ® would be significantly expanded and market exclusivity would be expected to last into the 2040s. Vanda announced in March 2024 that it received a complete response letter (CRL) from the FDA related to the supplemental New Drug Application (sNDA) for HETLIOZ® in the treatment of insomnia. Vanda is reviewing the CRL and evaluating its next steps. In addition to insomnia, Vanda continues to pursue FDA approval of HETLIOZ® in the treatment of jet lag disorder where the final agency rejection of our application is being challenged in the U.S. Court of Appeals for the D.C. Circuit. Vanda announced in April 2024 that the U.S. Supreme Court denied its petition for a writ of certiorari to review the decision of the U.S. Court of Appeals for the Federal Circuit in Vanda's HETLIOZ® Abbreviated New Drug Application litigation against Teva Pharmaceuticals USA, Inc. (Teva), Apotex Inc. and Apotex Corp (together, Apotex). The lower court decision held that certain claims of Vanda's U.S. Patent Nos. RE46,604; 9,730,910; 10,149,829; and 10,376,487 were invalid. Vanda's suit asserting U.S. Patent No. 11,285,129 will be infringed by Teva's and Apotex's generic versions of HETLIOZ® is currently pending in the U.S. District Court for the District of Delaware. PONVORY® (ponesimod) Vanda expects to complete the transition of PONVORY® from Janssen and commercially launch the product for multiple sclerosis in the third quarter of 2024. Vanda is initiating a host of commercial activities including the creation of a specialty sales force, a prescriber awareness program and a comprehensive marketing program. Currently approved as a once-a-day oral treatment for people with multiple sclerosis, PONVORY® has a differentiated profile from other drugs in the class with high specificity and rapid reversibility, making for a versatile use to address the needs of people with multiple sclerosis and exclusivity is expected to last into the 2040s. Positive results from a Phase II clinical study for PONVORY® in the treatment of psoriasis were previously published in Lancet where PONVORY® demonstrated significant effects in both induction and maintenance of response.3 Vanda expects to file an Investigational New Drug (IND) application with the FDA for PONVORY® in the treatment of psoriasis. Vanda expects to initiate a Phase III study for PONVORY® in the treatment of psoriasis by the end of 2024. If ultimately approved for marketing, PONVORY® would be the first oral sphingosine-1-phosphate (S1P) analog approved for the treatment of psoriasis and would significantly expand the addressable patient population of PONVORY®, with over 8 million people diagnosed with psoriasis in the U.S.4 Vanda expects to file an IND application with the FDA for PONVORY® in the treatment of ulcerative colitis. Vanda expects to initiate a Phase III study for PONVORY® in the treatment of ulcerative colitis by the end of 2024. If ultimately approved for marketing, PONVORY® would follow other oral sphingosine-1-phosphate (S1P) analogs approved for the treatment of ulcerative colitis and would significantly expand the addressable patient population of PONVORY®, with an estimated prevalence in the U.S. of approximately 2 million individuals.5 Tradipitant The NDA for tradipitant for the treatment of symptoms of gastroparesis is under review by the FDA with a PDUFA target action date of September 18, 2024. Gastroparesis is a significant unmet medical need with the last treatment option approved over 40 years ago and an estimated prevalence in the U.S. of over 6 million individuals.6 The second Phase III clinical study of tradipitant in the treatment of motion sickness is fully enrolled and results are expected in the second quarter of 2024. The efficacy of tradipitant in the treatment of motion sickness has previously been demonstrated in two clinical studies in which tradipitant was effective in preventing vomiting associated with motion. Vanda expects to submit an NDA for the treatment of motion sickness to the FDA in the fourth quarter of 2024. An eventual NDA approval of tradipitant in the treatment of motion sickness would significantly expand the addressable patient population, with approximately 30% of the U.S. population reported to suffer from motion sickness under ordinary travel conditions that include sea, air and land.7 Early-Stage Programs The Phase II study of VSJ-110 for the treatment of dry eye is ongoing and more than 50% enrolled. The Phase I clinical study for VCA-894A in the treatment of a patient with Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), an inherited peripheral neuropathy for which there is no available treatment, expects to enroll the patient in mid-2024. The Phase I clinical study of VTR-297 for the treatment of onychomycosis, a fungal infection of the nail, was initiated in April 2024. VQW-765, an alpha-7 nicotinic acetylcholine receptor partial agonist, is currently in clinical development for treatment of acute performance anxiety in social situations. GAAP Financial Results Net loss was $4.1 million in the first quarter of 2024 compared to net income of $3.3 million in the first quarter of 2023. Diluted net loss per share was $0.07 in the first quarter of 2024 compared to diluted net income per share of $0.06 in the first quarter of 2023. 2024 Financial Guidance Given continuing uncertainties surrounding the U.S. market for HETLIOZ® for the treatment of Non-24 as a result of continued generic competition in the U.S. and the upcoming commercial launches for Fanapt® in acute bipolar I disorder and PONVORY® in multiple sclerosis, Vanda is unable to provide 2024 financial guidance at this time. Conference Call Vanda has scheduled a conference call for today, Wednesday, May 8, 2024, at 4:30 PM ET. During the call, Vanda's management will discuss the first quarter 2024 financial results and other corporate activities. Investors can call 1-800-715-9871 (domestic) or 1-646-307-1963 (international) and use passcode number 2594340. A replay of the call will be available on Wednesday, May 8, 2024, beginning at 8:30 PM ET and will be accessible until Wednesday, May 15, 2024 at 11:59 PM ET. The replay call-in number is 1-800-770-2030 for domestic callers and 1-609-800-9909 for international callers. The passcode number is 2594340. The conference call will be broadcast simultaneously on Vanda's website, . Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda's website for a period of 30 days. References Calhoun SL, Fernandez-Mendoza J, Vgontzas AN, Liao D, Bixler EO. Prevalence of insomnia symptoms in a general population sample of young children and preadolescents: gender effects. Sleep Med. 2014 Jan;15(1):91-5. doi: 10.1016/j.sleep.2013.08.787. Epub 2013 Oct 16. PMID: 24333223; PMCID: PMC3912735. Fricke-Oerkermann L, Plück J, Schredl M, Heinz K, Mitschke A, Wiater A, Lehmkuhl G. Prevalence and course of sleep problems in childhood. Sleep. 2007 Oct;30(10):1371-7. doi: 10.1093/sleep/30.10.1371. PMID: 17969471; PMCID: PMC2266270. Vaclavkova A, Chimenti S, Arenberger P, Holló P, Sator PG, Burcklen M, Stefani M, D'Ambrosio D. Oral ponesimod in patients with chronic plaque psoriasis: a randomised, double-blind, placebo-controlled phase 2 trial. Lancet. 2014 Dec 6;384(9959):2036-45. doi: 10.1016/S0140-6736(14)60803-5. Epub 2014 Aug 10. PMID: 25127208. Datamonitor Healthcare – Psoriasis Patient-Based Market Forecast published March 2023. Datamonitor Healthcare – Ulcerative Colitis Patient-Based Market Forecast published March 2023. Rey et al J Neurogastroenterol Motil, Jan 2012. Turner M, Griffin MJ. Motion sickness in public road transport: passenger behavior and susceptibility. Ergonomics. 1999: 42: 444-461. About Vanda Pharmaceuticals Inc. Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit and follow us on X @vandapharma. CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS Various statements in this press release, including, but not limited to, statements regarding the advancement of Vanda's clinical development pipeline and strengthening of its commercial presence, Vanda's plans for pursuit of FDA approval of tradipitant in the treatment of symptoms of gastroparesis and the treatment of motion sickness, HETLIOZ® in the treatments of insomnia and jet lag disorder, Vanda's expectations regarding the timing of the FDA's decisions with respect to the sNDAs for HETLIOZ® and the NDA for tradipitant, Vanda's plans for a LAI formulation of Fanapt®, the timing of a submission of an NDA for milsaperidone, the potential for PONVORY® to treat psoriasis and ulcerative colitis, the planned patient enrollment for a clinical study for VCA-894A, the planned launch of PONVORY® in the US and the ongoing launch of Fanapt® in acute bipolar I disorder, the planned clinical programs to test Fanapt® and milsaperidone in depressive symptoms and the planned Hetlioz LQ® program in pediatric insomnia are "forward-looking statements" under the securities laws. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Forward-looking statements are based upon current expectations and assumptions that involve risks, changes in circumstances and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, Vanda's ability to complete the clinical development of the products in its pipeline and obtain regulatory approval and market acceptance of these products, Vanda's ability to obtain FDA approval of the sNDAs for HETLIOZ® and the NDA for tradipitant, the FDA's ability to meet the PDUFA target action date for the NDA for tradipitant, the FDA's assessment of the sufficiency of the data packages included in Vanda's regulatory submissions for HETLIOZ® and tradipitant, Vanda's ability to obtain approval for milsaperidone in the treatment of schizophrenia and acute bipolar I disorder, the results of any clinical trials conducted for PONVORY® in the treatment of other inflammatory/autoimmune disorders, Vanda's ability to obtain regulatory approval of HETLIOZ® and PONVORY® for any additional indications, Vanda's ability to complete the clinical program for tradipitant in the treatment of motion sickness, the planned clinical programs to test Fanapt® and milsaperidone in depressive symptoms and Vanda's ability to execute the planned launch of PONVORY® in the US and ongoing launch of Fanapt®. Therefore, no assurance can be given that the results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's most recent Annual Report on Form 10-K, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the U.S. Securities and Exchange Commission, which are available at . All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Corporate Contact: Kevin Moran Senior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc. 202-734-3400 [email protected] Jim Golden / Jack Kelleher / Dan Moore Collected Strategies [email protected] SOURCE Vanda Pharmaceuticals Inc.

2024年第一季度，美股中型市值生物科技和制药公司哪家表现最佳？答案是Viking Therapeutics，其股价涨幅341%，现市值为71.9亿美元。表1 2024年第一季度，美股中型市值生物科技和制药公司股价涨幅TOP10数据来源：Evaluate注：中型市值企业指市值在数十亿到几百亿美元之间的企业01这些药企，值得关注Viking Therapeutics减肥药开发的热度远未过去，这一点从Viking的股价涨幅中不难看出。投资者们依然高度关注减肥和代谢疾病领域新药的开发。Viking Therapeutics的股价就这样的氛围下实现了2024年第一季度飙升。根据Evaluate的数据，Viking Therapeutics是第一季度中等市值生物科技和制药公司中股价涨幅最大的公司。（注：中等市值公司具体的定义可能因国家、市场或行业而异，但通常来说，其市值可能在数十亿到几百亿美元之间。）1月以来，Viking Therapeutics的股价已上涨三倍，其背后的主要驱动力来自于其GLP-1/GIP双受体共激动剂VK2735在肥胖患者中取得的优良中期数据。分析师认为，2月份公布的VK2735的II期试验显示，受试者13周体重减轻了14.7%。单从数据的角度来看，VK2735未来有可能与礼来的Zepbound和诺和诺德的Wegovy分庭抗礼。Viking甚至已经开始高瞻远瞩地筹划商业化VK2375的策略，他们有意寻找合作伙伴共同营销这款未来的重磅炸弹。除此之外，Viking也是很多制药巨头期待的收购目标，尤其是在减肥药开发领域命运多舛的辉瑞，更是对收购Viking兴致盎然。Viking在2月28日的后续公开发行中筹集了5.5亿美元的资金。之后于3月26日透露，VK2735的口服制剂在其Ib期研究中给出了四周后5.3%的减重率，而且显示出优于Zepbound和Wegovy的胃肠道安全性和耐受性。Viking在同时开发VK2735的口服和注射制剂类型。Viking预计口服制剂的研究将于今年进入II期临床阶段，而注射制剂的研究已经在II期，可能将于本季度公布研究结果，并决定进入III期研究的部署。Viking Therapeutics和其它待价而沽的生物科技公司的区别，在于它手中掌握着两款潜在重磅资产。除了上述的减肥药VK2735之外，Viking还在MASH（代谢功能障碍相关脂肪型肝炎）领域拥有一款THRβ激动剂资产VK2809，其前景堪比不久前刚刚打破MASH空白的Rezdiffra(resmetirom)。虽然后者已经上市，但通过的是FDA的加速批准途径，仍然需要在大型试验中验证其安全性和有效性，这给了VK2809未来在这个市场占据显著份额的机遇。Zealand Pharma丹麦的生物制药公司Zealand Pharma的股票在今年第一季度也表现强劲，凭借的同样是针对肥胖症和MASH的资产Survodutide。这款多肽候选药在其肥胖症和MASH的II期研究中给出了相当不错的数据，为Zealand Pharma 53亿美元的市值和85%的股价涨幅做出了巨大的贡献。除了肥胖症和其它代谢适应症之外，生物医药领域开发热情最高的另一个领域当属免疫和炎症疾病。不仅有诸如赛诺菲这样重新部署开发战略布局，将肿瘤学资产转移到免疫学领域的大量实例，而且重点开发免疫和炎症资产的中型市值公司的股价也行情看涨。股价涨幅榜排在Viking之后的就是两家重点开发免疫和炎症疾病的美国公司Vera Therapeutics和Apogee Therapeutics，他们的股价在今年第一季度的涨幅分别为177%和141%。Vera TherapeuticsVera Therapeutics在今年1月公布了其肾脏疾病候选药物atacicept的强大IIb数据，atacicept是BLyS和APRIL细胞因子的双重抑制剂。分析师认为它将引领未来自身免疫性疾病市场，包括IgA肾病和狼疮性肾炎等疾病。与此同时，Apogee Therapeutics的IL-13靶向抗体APG-777在特应性皮炎方面的I期结果给投资者留下了深刻的印象。这款产品延长的半衰期可能使得其成为潜在的best-in-class药物，并且可以与礼来公司的抗IL-13抗体lebrikizumab媲美。UCBUCB在免疫领域的开发同样收获了丰厚的回报。他们已经在美国市场成功地推出了IL-17A和IL-17F抑制剂、银屑病药物Bimzelx（bimekizumab），估计其峰值年销售额能够达到50-60亿美元的高度。今年1月，UCB还提交了rozanolixizumab申请，是针对新生儿Fc受体(FcRn)的皮下抗体，用于治疗成人全身性重症肌无力(gMG)。药物研发的积极进展，使UCB股价在第一季度飙升44%。02研发趋势判断股价不是衡量一家生物科技或制药公司的唯一标准，从股价涨幅趋势中判断药物研发动向，了解国际前沿动向、对标进行前瞻判断，从而使得国内创新药研发与国际接轨，才是最为重要的。从股价涨幅榜药企研发药物的适应症领域来看，免疫类疾病凭借其强大的市场潜力越来越吸引开发商，尤其是制药业巨头的关注。包括赛诺菲在内的多家大型制药公司已经开始调整其战略部署，将部分肿瘤学细胞疗法资产转移到免疫领域，尤其是自免疫疾病的开发上面。除了赛诺菲之外，阿斯利康和百时美施贵宝也在积极地向自免疫疾病的细胞疗法开发领域拓展。CRISPR Therapeutics也在近期宣布将其CAR T候选药物CTX112和CTX131的研究方向从肿瘤学转移到系统性红斑狼疮。这些行业动态都说名了免疫疗法开发的受关注度将持续升高。肿瘤学方面，ADC交易的不断升温也透露出了这个领域是未来发展的主要方向之一。前有辉瑞豪掷430亿美元收购Seagen，紧接着是艾伯维100亿美元收购Immunogen，强生200亿美元购得Ambrx Biopharma，以及默沙东与第一三共价值220亿美元的合作项目，其核心内容都有ADC的身影。2024年第二季度刚开始，丹麦的Genmab就在4月3日宣布，将以18亿美元现金收购ProfoundBio。ADC类候选药Rinatabart Sesutecan(亦称Rina-S，PRO1184)就是后者的核心资产，正在进行卵巢癌的2期研究。CDER 2024年第二季度的审批项目中，也包括众多中枢神经系统疾病药物申请，其中最引人关注的无疑就是Sarepta的杜氏肌营养不良症基因疗法Elevidys。Fanapt，非典型抗精神病药急性治疗成人I型双相情感障碍相关躁狂或混合发作的新适应症，2024年4月2日SPN-830，多巴胺激动剂连续皮下输注制剂，用于治疗帕金森病成人患者的运动波动，2024年4月5日Libervant，用于急性治疗2至5岁患者间歇性、刻板的频繁癫痫发作，2024年4月28日Ingrezza，与亨廷顿舞蹈病相关的成人迟发性运动障碍或舞蹈病，2024年4月30日Ultomiris，成人视神经脊髓炎谱系障碍，不晚于2024年5月21日ND0612，运动波动的帕金森病，不晚于2024年6月Vyalev，晚期帕金森病患者的运动波动，不晚于2024年6月Vyvgart Hytrulo，慢性炎症性脱髓鞘性多发性神经病，2024年6月21日Elevidys，杜氏肌营养不良症，2024年6月21日Wakix，6岁及以上发作性睡病儿童的日间过度嗜睡或猝倒，2024年6月21日Ref.1.Haas,J.A Good Choice To Have:Scrip’s Interview With Viking CEO Brian Lian.Scrip.12.04.2024.2.Haas,J.Viking’s Oral Weight-Loss Drug Shows Early Efficacy/Safety Promise.Scrip.26.03.2024.3.McConaghie,A.Investors Go Berserk For Viking,Putting It Top Of Q1 Winners.Scrip.19.04.2024.声明：本内容仅用作医药行业信息传播，为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。责任编辑 | 金银花转载开白 | 马老师 18996384680（同微信）商务合作 | 王存星 19922864877（同微信） 阅读原文，是受欢迎的文章哦