Jet Lag Syndrome

The FDA on Tuesday turned down Vanda Pharmaceuticals’ request for a hearing on issues the agency found with the company’s application for a jet lag indication for its sleep disorder drug Hetlioz, another blow to the company and its strained relationship with the agency. The FDA’s decision nixes chances that Vanda’s Hetlioz, which is approved for other sleep disorders, will be granted an indication for jet lag disorder. The FDA cited issues with the endpoints used in the trials, said there was no debate about the facts and data for the drug, and that it wasn’t in the public interest to reconsider.

Full year 2023 revenues were $192.6 million PONVORY® acquisition completed in Q4 2023 and transition ongoing 3 FDA PDUFA target action dates in 2024 Ended 2023 with approximately $388 million in cash and cash equivalents WASHINGTON, Feb. 7, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced financial and operational results for the fourth quarter and full year ended December 31, 2023. "Despite significant external commercial and regulatory challenges, last year was a landmark year for Vanda, as we supported our commercial programs and, through a transformational deal, we added PONVORY® to our portfolio alongside Fanapt® and HETLIOZ®," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. "At the same time, we advanced our clinical development pipeline with three NDA and sNDA submissions for insomnia, bipolar disorder and gastroparesis. Looking forward to this year, we are committed to advancing our clinical development pipeline and strengthening our commercial presence with a portfolio of innovative products serving unmet patient needs." Financial Highlights Fourth Quarter of 2023 Total net product sales from HETLIOZ®, Fanapt® and PONVORY® were $45.3 million in the fourth quarter of 2023, a 30% decrease compared to $64.5 million in the fourth quarter of 2022, and a 17% increase compared to $38.8 million in the third quarter of 2023. HETLIOZ® net product sales were $21.1 million in the fourth quarter of 2023, a 47% decrease compared to $40.1 million in the fourth quarter of 2022, and a 20% increase compared to $17.5 million in the third quarter of 2023. The decrease relative to the fourth quarter of 2022 was the result of the at-risk launch of generic versions of HETLIOZ® in the U.S. Fanapt® net product sales were $22.6 million in the fourth quarter of 2023, a 7% decrease compared to $24.4 million in the fourth quarter of 2022, and a 6% increase compared to $21.3 million in the third quarter of 2023. PONVORY® net product sales were $1.6 million in the fourth quarter of 2023. These net product sales reflect the revenue generated during the period between the product acquisition date of December 7, 2023 and December 31, 2023. Net loss was $2.4 million in the fourth quarter of 2023 compared to net income of $6.9 million in the fourth quarter of 2022, and net income of $0.1 million in the third quarter of 2023. Cash, cash equivalents and marketable securities (Cash) was $388.3 million as of December 31, 2023, representing a decrease to Cash of $101.6 million compared to September 30, 2023. During the period, Vanda completed the acquisition of PONVORY® from Actelion Pharmaceuticals Ltd. (Janssen), a Johnson & Johnson Company, for $100.0 million. Full Year 2023 Total net product sales from HETLIOZ®, Fanapt® and PONVORY® were $192.6 million for the full year 2023, a 24% decrease compared to $254.4 million for the full year 2022. HETLIOZ® net product sales were $100.2 million for the full year 2023, a 37% decrease compared to $159.7 million for the full year 2022. The decrease was the result of the at-risk launch of generic versions of HETLIOZ® in the U.S. Fanapt® net product sales were $90.9 million for the full year 2023, a 4% decrease compared to $94.7 million for the full year 2022. PONVORY® net product sales were $1.6 million for the full year 2023. These net product sales reflect the revenue generated during the period between the product acquisition date of December 7, 2023 and December 31, 2023. Net income was $2.5 million for the full year 2023 compared to net income of $6.3 million for the full year 2022. Cash, cash equivalents and marketable securities (Cash) was $388.3 million as of December 31, 2023, representing a decrease to Cash of $78.6 million, or 17%, compared to December 31, 2022. During the period, Vanda completed the acquisition of PONVORY® from Janssen for $100.0 million. Key Operational Highlights HETLIOZ® (tasimelteon) The supplemental New Drug Application (sNDA) for HETLIOZ® in the treatment of insomnia is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of March 4, 2024. Vanda announced that on February 4, 2024, it received a notification from the FDA stating that the FDA had identified deficiencies that preclude discussion of labeling and postmarketing requirements/commitments at this time. No deficiencies were disclosed by the FDA in the notification, and the FDA stated that the notification does not reflect a final decision on the information under review. On February 6, 2024, Vanda filed suit in the U.S. District Court for the District of Columbia (D.C. District Court) challenging the FDA's conduct in reviewing the insomnia sNDA. Vanda is asking the D.C. District Court to compel the FDA to adhere to the legally mandated 180-day review period for sNDAs and to declare as unlawful and void the regulations the FDA relies upon to issue complete response letters. Vanda is also continuing to pursue FDA approval for HETLIOZ® in the treatment of jet lag disorder. Vanda announced in January 2024 that the D.C. District Court granted Vanda's motion for summary judgment on its claim against the FDA for unlawfully delaying a hearing on the approvability of Vanda's sNDA for HETLIOZ® in the treatment of jet lag disorder. The D.C. District Court ordered the FDA to either finally resolve Vanda's jet lag sNDA or commence a hearing on the sNDA on or before March 5, 2024. In January 2024, Vanda filed a petition for a writ of certiorari with the U.S. Supreme Court to review the decision of the U.S. Court of Appeals for the Federal Circuit in Vanda's HETLIOZ® Abbreviated New Drug Application (ANDA) litigation against Teva Pharmaceuticals USA, Inc. (Teva), Apotex Inc. and Apotex Corp. (collectively, Apotex). Teva and Apotex have waived their opportunity to respond to Vanda's petition, which is now ripe for decision by the U.S. Supreme Court. Fanapt® (iloperidone) The article "Efficacy and Safety of Iloperidone in Bipolar Mania: A Double-Blind, Placebo-Controlled Study" was published in January 2024 in the Journal of Clinical Psychiatry.1 The findings of this pivotal study have been submitted to the FDA as part of Vanda's sNDA for Fanapt® in the treatment of bipolar I disorder in adults. The sNDA for Fanapt® in the treatment of bipolar I disorder in adults is under review by the FDA with a PDUFA target action date of April 2, 2024. PONVORY® (ponesimod) Vanda completed the acquisition of the U.S. and Canadian rights to PONVORY® from Janssen for $100.0 million in December 2023 and the transition is ongoing. PONVORY® is a once-daily oral selective sphingosine-1-phosphate receptor 1 modulator, approved by the FDA and Health Canada to treat adults with relapsing forms of multiple sclerosis, and is a potential therapeutic candidate for the treatment of a diverse group of inflammatory/autoimmune disorders ranging from psoriasis to ulcerative colitis. Vanda announced in January 2024 that the U.S. Patent and Trademark Office (USPTO) had issued a notice of allowance for its PONVORY® patent application, 17/962,968, covering methods for reducing clinical management events before or during the treatment of multiple sclerosis and methods for reinstating treatment after missed doses. When issued, the patent is anticipated to expire on October 10, 2042. Upon issuance, Vanda intends to list this patent in the FDA publication Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book. Tradipitant The article "The Efficacy of Tradipitant in Patients with Diabetic and Idiopathic Gastroparesis in Phase III Randomized Placebo-Controlled Clinical Trial" was published in January 2024 in the Clinical Gastroenterology and Hepatology Journal.2 The findings of this pivotal study as well as a previously reported positive placebo-controlled study in diabetic and idiopathic gastroparesis have been submitted to the FDA as part of Vanda's New Drug Application (NDA) for tradipitant in the treatment of symptoms of gastroparesis in adults.3 In December 2023, Vanda announced that the NDA for tradipitant for the treatment of symptoms of gastroparesis was accepted for filing and is under review by the FDA with a PDUFA target action date of September 18, 2024. The second Phase III study of tradipitant in the treatment of motion sickness is over 50% enrolled. In May 2023, Vanda previously announced positive results from its first Phase III study of tradipitant in the treatment of motion sickness. Vanda plans to pursue FDA approval upon completion of the clinical development program. Early-Stage Programs In January 2024, Vanda announced that the FDA had approved the Investigational New Drug (IND) application to evaluate VCA-894A for 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. In January 2024, Vanda announced that the FDA had also approved the IND to evaluate VTR-297 for the treatment of onychomycosis, a fungal infection of the nail. Other Legal Updates Vanda announced in January 2024 that the United States Court of Federal Claims denied in part the U.S. government's motion to dismiss Vanda's claims against the U.S. for the FDA's uncompensated taking of Vanda's trade secrets and confidential information, thereby allowing Vanda's lawsuit to proceed. Vanda is seeking compensation from the U.S. for the takings it contends occurred through the FDA's improper communication and disclosure of trade secrets and confidential information to certain generic drug manufacturers. Vanda now intends to engage in discovery to support its claims. GAAP Financial Results Net loss was $2.4 million in the fourth quarter of 2023 compared to net income of $6.9 million in the fourth quarter of 2022. Diluted net loss per share was $0.04 in the fourth quarter of 2023 compared to diluted net income per share of $0.12 in the fourth quarter of 2022. Net income was $2.5 million for the full year 2023 compared to net income of $6.3 million for the full year 2022. Diluted net income per share was $0.04 for the full year 2023 compared to diluted net income per share of $0.11 for the full year 2022. 2024 Financial Guidance Given uncertainties surrounding the U.S. market for HETLIOZ® for the treatment of Non-24 as a result of the ongoing HETLIOZ® patent litigation and the at-risk launch of generic versions of HETLIOZ®, Vanda is unable to provide 2024 financial guidance at this time. Conference Call Vanda has scheduled a conference call for today, Wednesday, February 7, 2024, at 4:30 PM ET. During the call, Vanda's management will discuss the fourth quarter and full year 2023 financial results and other corporate activities. Investors can call 1-800-715-9871 (domestic) or 1-646-307-1963 (international) and use passcode number 8053170. A replay of the call will be available on Wednesday, February 7, 2024, beginning at 8:30 PM ET and will be accessible until Wednesday, February 14, 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 8053170. 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 Torres R, Czeisler EL, Chadwick SR, et al. Efficacy and safety of iloperidone in bipolar mania: a double-blind, placebo-controlled study. J Clin Psychiatry. Carlin, J. L., Polymeropoulos, C., Camilleri, M., Lembo, A., Fisher, M., Kupersmith, C., Madonick, D., Moszczynski, P., Smieszek, S., Xiao, C., Birznieks, G., & Polymeropoulos, M. H. (2024). The efficacy of tradipitant in patients with diabetic and idiopathic gastroparesis in phase III randomized placebo-controlled clinical trial. Clinical Gastroenterology and Hepatology. Carlin, J. L., Lieberman, V. R., Dahal, A., Keefe, M. S., Xiao, C., Birznieks, G., Abell, T. L., Lembo, A., Parkman, H. P., & Polymeropoulos, M. H. (2021). Efficacy and Safety of Tradipitant in Patients with Diabetic and Idiopathic Gastroparesis in a Randomized, Placebo-Controlled Trial. Gastroenterology, 160(1), 76–87.e4. 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 HETLIOZ® in the treatments of insomnia and jet lag disorder, Fanapt® in the treatment of bipolar I in adults and tradipitant in the treatment of symptoms of gastroparesis and the treatment of motion sickness, Vanda's expectations regarding the timing of the FDA's decisions with respect to the sNDAs for HETLIOZ® and Fanapt® and the NDA for tradipitant, the potential for PONVORY® to treat a diverse group of inflammatory/autoimmune disorders, the USPTO's plans to issue the new PONVORY® patent, the anticipated life of the patent and Vanda's intentions to list the patent in the Orange Book 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 Fanapt® and the NDA for tradipitant, the FDA's ability to meet the PDUFA target action dates for the sNDAs for HETLIOZ® and Fanapt® and the NDA for tradipitant, the FDA's assessment of the sufficiency of the data packages included in Vanda's regulatory submissions for HETLIOZ®, Fanapt® and tradipitant, Vanda's ability to complete the clinical program for tradipitant in the treatment of motion sickness, the results of any clinical trials conducted for PONVORY® in the treatment of other inflammatory/autoimmune disorders and Vanda's ability to obtain regulatory approval of PONVORY® for any such additional indications, the payment by Vanda to the USPTO of all fees required prior to the issuance of the new PONVORY® patent, the ultimate issuance of the patent by the USPTO and Vanda's ability to protect its intellectual property rights and defend the new PONVORY® patent against any attempt to invalidate it. 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] SOURCE Vanda Pharmaceuticals Inc.

WASHINGTON, Jan. 29, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the United States District Court for the District of Columbia granted Vanda's motion for summary judgment on its claim against the United States Food and Drug Administration (FDA) for unlawfully delaying a hearing on the approvability of Vanda's supplemental new drug application (sNDA) for HETLIOZ® to treat jet lag disorder (Vanda Pharmaceuticals Inc. v. FDA, case no. 1:22-cv-2275-CJN). The Federal Food, Drug, and Cosmetic Act (FDCA) requires the FDA to either approve a new drug application or provide an opportunity for a hearing within 180 days after submission of the application.1 Vanda submitted its sNDA in October 2018 for approval to market HETLIOZ® to treat jet lag disorder. The FDA did not comply with the statute and did not timely approve or provide an opportunity for a hearing. Instead, the FDA issued a complete response letter in August 2019. Following multiple attempts to informally resolve the complete response letter, Vanda requested an opportunity for a hearing in July 2022, and the FDA provided Vanda a private notice of an opportunity for a hearing. After doing so, the FDA was required under the FDCA to commence the hearing within 120 days2, but the FDA failed to meet this legal obligation. To remedy the FDA's extraordinary delays, Vanda filed its lawsuit in September 2022. On January 26, 2024, a federal judge ruled that the FDA "has violated the statute" and ordered the FDA to either finally resolve Vanda's sNDA or commence a hearing on the sNDA on or before March 5, 2024. As the Court explained, "the statute requires that a hearing shall commence within 300 days after an application is filed. Vanda's application has been pending for almost 2,000 days and it has been over 500 days since Vanda made its most recent request for hearing." The Court's decision highlights the serious flaws in FDA regulations governing drug-approval processes, and it reinforces the FDA's clear statutory obligations to render timely final decisions on drug applications. It appears from this case, and from the FDA's standard policies, that FDA decisionmakers have implemented a policy of following the FDA's own timelines, resulting in unlawful actions that are systemic. The FDA's extraordinary delays impede medical innovation to the detriment of the many patients with unmet needs who lack effective therapeutics. The FDA's delays force these patients to wait years longer than Congress envisioned for access to urgently needed pharmaceutical innovation. References 21 U.S.C. § 355(c)(1) 21 U.S.C. § 355(c)(1)(B) 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. About HETLIOZ® For full U.S. Prescribing Information for HETLIOZ®, including indication and Important Safety Information, visit . Corporate Contact: Kevin Moran Senior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc. 202-734-3400 [email protected] SOURCE Vanda Pharmaceuticals Inc.