Soleno: Mid-2024 NDA Filing For PWS Treatment Is A Major Catalyst To Watch

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Soleno Therapeutics, Inc.'s New Drug Application filing of DCCR for the treatment of patients with Prader-Willi Syndrome expected by mid-2024.Ability to file NDA of DCCR for PWS was because of ...

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}\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nSoleno: Mid-2024 NDA Filing For PWS Treatment Is A Major Catalyst To Watch\n</h2>\n\n<h4 class=\"meta\">\n\n\n2024-04-30 04:02 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://seekingalpha.com/article/4687282-soleno-mid-2024-nda-filing-for-pws-treatment-is-a-major-catalyst-to-watch><strong>seekingalpha</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>Soleno Therapeutics, Inc.'s New Drug Application filing of DCCR for the treatment of patients with Prader-Willi Syndrome expected by mid-2024.Ability to file NDA of DCCR for PWS was because of ...</p>\n\n<a href=\"https://seekingalpha.com/article/4687282-soleno-mid-2024-nda-filing-for-pws-treatment-is-a-major-catalyst-to-watch\">Source Link</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"SLNO","symbol_name":"Soleno Therapeutics","start_time":0,"source_url":"https://seekingalpha.com/article/4687282-soleno-mid-2024-nda-filing-for-pws-treatment-is-a-major-catalyst-to-watch","article_id":"2431484296","we_media_id":null,"thumbnails":[],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2431484296","pubTimestamp":1714420936,"sourceInfo":{"source_id":"seekingalpha","name":"Seeking Alpha"},"weMediaInfo":null,"summary":"That's because it spoke with the FDA and the agency stated that if it was able to have a successful randomized withdrawal study,","collect":0,"end_time":0,"defaultTopTitle":"seekingalpha.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"SLNO":"Soleno Therapeutics","PWS":"Pacer WealthShield ETF","BK4139":"生物科技"},"translate_title":"Soleno：2024年年中NDA申请PWS治疗是一个值得关注的主要催化剂","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"PWS":1,"SLNO":1},"content_text":"Soleno Therapeutics, Inc.'s New Drug Application filing of DCCR for the treatment of patients with Prader-Willi Syndrome expected by mid-2024.Ability to file NDA of DCCR for PWS was because of statistical significance achieved based on the primary endpoint of Study C602, which was the HQ-CT score.The global Prader-Willi Syndrome market in 2018 was estimated to be $2.11 billion in 2018.If DCCR is approved by the FDA for Prader-Willi Syndrome, it would be the first treatment of its kind to specifically target hyperphagia.   Jian Fan/iStock via Getty Images Soleno Therapeutics, Inc. (NASDAQ:SLNO) is gearing up to file a New Drug Application [NDA] to the FDA of Diazoxide Choline [DCCR] for the treatment of patients with Prader-Willi Syndrome [PWS] in mid-2024. This is one major catalyst for investors to look forward to regarding this regulatory filing in the United States. Another important aspect to consider would be that even upon the FDA accepting this application of DCCR for review, it won't take long to complete it. That's because a recent development is that the company received Breakthrough Therapy Designation of this drug for the treatment of this patient population just today. The significance of this is that it will mean an expedited review time, compared to what is typically afforded to these companies. These aren't the only designations it has received from the agency for; it has also been able to obtain Orphan Drug Designation [ODD] and Fast Track Designation as well. One of the important aspects to consider is that Soleno's DCCR takes care of an important aspect of PWS, which is hyperphagia. With positive data from several studies in PWS, plus an NDA filing of DCCR for the treatment of patients with PWS in mid-2024, I believe that investors might benefit with any potential gains made here. DCCR Extended Release Tablets Offers New Form Of Treatment Option Soleno Therapeutics is highly focused on being able to bring a new, highly effective treatment option for the treatment of patients with Prader-Willi Syndrome. It is doing so with the advancement of DCCR, which stands for Diazoxide Choline. It has been able to advance several studies using this drug for the treatment of this patient population. One important fact to mention, which was a setback at that time, is that the primary endpoint of the phase 3 DESTINY PWS clinical trial, did not meet statistical significance. The primary endpoint of this study was Hyperphagia Questionnaire for Clinical Trials [HQ-CT]. That's because patents who took DCCR extended-release tablets achieved an HQ-CT score of -5.94 points, then those on placebo achieved a score of -4.27 points. Thus, this difference was not statistically significant with a p-value of p=0.1983. If this is the case, then why did this company continue to press on, regardless of this primary endpoint failure? That's because it spoke with the FDA and the agency stated that if it was able to have a successful randomized withdrawal study, that it could potentially file a New Drug Application [NDA] of DCCR for the FDA for PWS. Taking the advice of the FDA, that's precisely what Soleno Therapeutics did, in that it was capable of initiating a trial known as Study C602, which was also a phase 3 study. However, what was required for it to do was to only enroll patients from the phase 3 DESTINY PWS study [Study C601] into an open-label C602 study. From there, the new trial design to be done would be to randomize these specific patients 1:1 to receive either DCCR or placebo. That is, a total of 77 patients in Study C602 were randomized into one of these two withdrawal groups. It was noted that long-term DCCR treated patients, who stuck with this treatment, were able to achieve an HQ-CT score of 2.6 points increase. On the other hand, those who were randomized to receive placebo instead, were shown to have obtained an HQ-CT score of 7.6 points. This 5 point difference of DCCR compared to placebo in this primary endpoint was statistically significant. Why is that? That's because in this instance, this primary endpoint was met with statistical significance with a p-value of p=0.0022. The bottom-line is that this positive study took care of the FDAs' concerns, in that this randomized withdrawal study would be enough to show efficacy that DCCR does, in fact, help these patients. The thing is that Prader-Willi Syndrome [PWS] is a highly devastating disease. It is a genetic disorder that causes several problems, such as intellectual disability, behavior problems, shortness in height and weight issues. In terms of weight issues, this is a hallmark of disease. What makes me say this? That's because the most prevalent issue for PWS is that it causes hyperphagia, which is the constant need to eat [being hungry all the time]. Being hungry all the time leads to obesity, cardiovascular disease and many other problems later on. Consider that Human Growth Hormone [HGH] treatment can reduce amount of fat intake, and help with several aspects of PWS, but nothing to effectively target the hyperphagia observed for it. Thus, the hope is that Soleno Therapeutics can receive FDA approval to help these patients reduce such hyperphagia associated with it. Especially, since there is no drug specifically approved to treat hyperphagia associated with it. Why is this considered to be such a hallmark of PWS? That's because it is noted that 96.5% of respondents [caregivers and parents] rate hyperphagia as a major issue that needs to be addressed. How DCCR extended-release works is that it is a crystalline salt of diazoxide that activates an ATP channel in a patient's brain, which in turn controls appetite activity [hunger]. It also has an impact on insulin resistance as well. The thing is that the company has been able to develop a proprietary extended-release version of Diazoxide, which allows it to be released and absorbed for up to 24 hours upon being given. With this positive randomized withdrawal study completed, according to what the FDA noted would be needed to show further clinical efficacy, Soleno Therapeutics can file an NDA for DCCR for the treatment of patients with PWS. Such a regulatory filing of DDCR for the treatment of this patient population is expected in mid-2024. The most recent development is that it received Breakthrough Therapy Designation [BTD] of DCCR from the FDA for the treatment of this patient population. This makes sense because not only does this once-daily treatment have an impact on hyperphagia for these PWS patients, but it also addresses other issues associated with it as well, such as behavior problems, fat mass and other metabolic parameters. Financials According to the 10-K SEC Filing, Soleno Therapeutics had cash and cash equivalents of $169.7 million as of December 31, 2023. The reason for the cash on hand is because of the $129 million it received upon the public offering and concurrent private placement agreement it established in October 2023. It also has been able to receive approximately $43.1 million from the sale and exercise of warrants issued in connection with a December 2022 Securities Purchase Agreement. It could also be eligible to receive up to an additional $16.9 million from the exercise of the remaining warrants issued under the December 2022 Securities Purchase Agreement. The thing is that it is likely going to need to raise additional cash going forward. Not only because it may have to start to prepare for commercialization activities of DCCR for PWS, but because of the estimated cash on hand. It believes that its cash runway is expected to last for at least the next 12-months from the date of the 10-K SEC filing, which was filed on March 7, 2024. This means it is going to need to raise additional cash, which would mean further dilution for shareholders. Its cash burn is roughly $12.8 million per quarter. One other aspect to consider in relation to possible future financing being necessary would be an agreement that it had to make with Essentialis, because of a 2016 merger agreement. It has to pay these shareholders up to $21.2 million upon achievement of certain commercial milestones associated with future sales of DCCR. Of course, this will only be necessary if the FDA ultimately approves this drug for the treatment of patients with PWS. Risks To Business There are several risks that investors should be aware of before investing in Soleno Therapeutics. The first risk to consider would be regarding the NDA filing of DCCR for the treatment of patients with PWS. That's because even though a meeting with the FDA stated that another randomized withdrawal study would be enough to show efficacy for an NDA filing, there is no assurance that that agency will accept such an application in its present form. A second risk to consider would be regarding the FDA review of the NDA of DCCR for PWS, even if it is ultimately accepted by the FDA. Despite achieving statistical significance in Study C602, the phase 3 DESTINY PWS study did not meet such statistical significance. There is no assurance that the FDA will approve DCCR for the treatment of this patient population upon further review. The good news is that such a review period won't take long, because of the Breakthrough Therapy Designation that was achieved for this treatment. Such a designation by the agency cuts the review period from 12 months down to 6 months, typically. The third and final risk to consider would be regarding the financial position that it is in. The reason why is that it believes that it only has enough cash to fund its operations for at least the next 12 months from the date of the 10-K SEC filing, again filed on March 7th of 2024. Thus, this means the company is going to have to either tap into an ATM agreement or enact further public/private financing agreements to keep itself funded. If this is done, not only would it cause the stock price to trader lower in the near-term, but it would also further dilute shareholders. Conclusion Soleno Therapeutics is gearing up for a major inflection point in mid-2024, and this would be regarding the New Drug Application [NDA] filing of DCCR for the treatment of patients with Prader-Willi Syndrome [PWS]. Such a filing is expected to be an important inflection point. However, the truth is that there is still an inherent risk that remains, as I noted above. This would be that there is no assurance that the NDA will be accepted, for starters. Secondly, even if accepted for review, there is no guarantee that Soleno Therapeutics will ultimately be approved to treat this specific patient population.  This is a large market opportunity for it to go after, especially since there are no currently approved therapies that specifically treat the hyperphagia associated with it. The global Prader-Willi Syndrome market in 2018 was estimated to be $2.11 billion in 2018. This makes sense, that the value of this market could be just around this number for the current period of time. That's because the company believes that it is a >$2 billion market opportunity. The only obstacle that remains now is to see if the FDA believes that the randomized withdrawal C206 study is enough to warrant given DCCR U.S. marketing approval.","kind":null,"is_publish_news":null,"is_publish_highlight":null,"is_publish_live":null,"is_publish_wemedia":null,"editions":null,"symbols":[],"gpt_button":1},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":true,"deviceId":"web-server-community-laohu8-v3","version":"4.22.4","shortVersion":"4.22.4","platform":"web","vendor":"web","appName":"ttm","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2431484296"}