Benitec Biopharma: OPMD Program Continues To Advance With Catalysts

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Benitec Biopharma Inc.'s BB-301 showed positive interim results in treating OPMD, allowing for higher dosing in 2025 and a potential late-stage trial in 2026.BB-301 demonstrated significant ...

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Solskin Benitec Biopharma Inc.  recently reported positive results from its phase 1b/2a clinical study using BB-301 for the treatment of patients with Oculopharyngeal Muscular Dystrophy [OPMD]. It reported these positive interim results at two time points during 2024 showing initial proof-of-concept that this gene therapy works in being able to treat these patients with this rare","collect":0,"end_time":0,"defaultTopTitle":"seekingalpha.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"BK4139":"生物科技","BNTC":"Benitec Biopharma Limited"},"translate_title":"Benitec Biopharma：OPMD计划借助催化剂继续推进","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"BNTC":0.9},"content_text":"Benitec Biopharma Inc.'s BB-301 showed positive interim results in treating OPMD, allowing for higher dosing in 2025 and a potential late-stage trial in 2026.BB-301 demonstrated significant improvements in swallowing function and reduced pharyngeal residue, with no serious adverse events reported in the phase 1b/2a study.Financially, Benitec Biopharma is well-positioned with $50.9 million in cash, sufficient to fund operations through 2025, thanks to a $40 million PIPE financing.Risks include uncertain efficacy and safety of higher BB-301 doses, and regulatory hurdles for advancing to pivotal phase 3 trials.   Solskin Benitec Biopharma Inc. (NASDAQ:BNTC) recently reported positive results from its phase 1b/2a clinical study using BB-301 for the treatment of patients with Oculopharyngeal Muscular Dystrophy [OPMD]. It reported these positive interim results at two time points during 2024 showing initial proof-of-concept that this gene therapy works in being able to treat these patients with this rare disorder. Why should investors care about this program though now that these catalysts have come and gone? It is because this positive data allows the company to move the 3rd patient into the same dosing regimen in October 2024 for starters. Secondly, it allows it to move to higher dosing of BB-301 in 2025, which may ultimately yield an improvement in efficacy. Speaking of which, it is highly likely that there should be the release of data from such higher dosing during this period as well. If all goes smoothly with this program regarding low and high-dosing, then it will be in good shape to initiate a late-stage study for this program in 2026. With the ability to advance BB-301 as a gene therapy treatment option for patients with OPMD, plus several catalysts on the way within the next few years, I believe that investors can capitalize on any potential gains made here. BB-301 For The Treatment Of Patients With OPMD As I noted above in the beginning, the main clinical program from this company would be the development of gene therapy BB-301 for the treatment of patients with Oculopharyngeal Muscular Dystrophy. The use of this therapy is being explored in the ongoing phase 1b/2a study. Before going over this specific trial, plus any catalysts to come out of this program, it is first important to go over what this disorder is and what the possible market opportunity for it could be. Ocularpharyngeal Muscular Dystrophy [OPMD] is a rare genetic disorder that leads to muscle weakness. Having said that, it is a type of muscular dystrophy. However, OPMD only affects the muscles of the throat and eyelids. This is a hard disorder to detect because someone who has it doesn't likely get diagnosed with it until they are 40 years of age. The thing to note is that it is a progressive type of disorder that just continues to get worse over time. However, while it does progress in such a fashion, it does so at a slow pace. Some symptoms that these patients with OPMD experience are as follows:  Unable to speak Double vision  Facial weakness  Impaired eye movement  Weakness of the tongue Dysphagia [Difficulty in swallowing].  The global Oculopharyngeal Muscular Dystrophy market size is expected to surpass $10 billion by the end of 2036. This is a good market size and the hope is that this company can continue to advance this program. What exactly causes this disorder? It is caused by a mutation of the PABPN1 gene, which is inherited either by one or both parents. Even though one can have it in either of these scenarios, patients who obtain two copies of mutated PABN1 gene develop more severe symptoms earlier in the process. To see if BB-301 can help treat OPMD patients, it is running the phase 1b/2a clinical study. This particular trial has recruited a total of 2 patients thus far and then a 3rd patient is expected to be enrolled during the month of October 2024. There was positive interim data analysis from 1 patient reported at the 90-day and 180-day time points. The improvements observed as this one patient received one dose of BB-301 is as follows:  90-day post treatment period — improvement in swallowing function  180-day post treatment period — TPR was meaningfully reduced [in essence, smaller amounts of food in the pharynx for patients taking the gene therapy compared to an OPMD natural history study].  In terms of safety, BB-301 was shown to be very safe. Matter of fact, the only adverse event [AE] noted in this particular trial from either of these two patients was a Grade 2 Gastroesophageal Reflux Disease [GERD]. Even better than this, there were no serious adverse events [SAEs] reported from either of these two particular patients enrolled. The third patient remains an unknown for now. Thus, it is too early to say whether there will be any safety issues to be released later on. Again, even though these set of results were already released from this ongoing phase 1b/2a clinical study, there are still a few other catalysts that investors can consider. Some of which may or may not ultimately yield a higher stock price. The first of which is that there will be the release of updated results, from this mid-stage trial using higher dosing of BB-301 for OPMD patients, in 2025. The reason this is possible is that the company is going to use higher dosing of BB-301 for the treatment of these patients during this period. From there, if everything goes smoothly, then it is expected that Benitec will be in a position to initiate a late-stage pivotal study for this particular program. Financials According to the 10-Q SEC Filing, Benitec Biopharma had cash and cash equivalents of $50.9 million as of June 30th of 2024. The reason for the cash on hand is that it did a PIPE financing agreement back in April 2024. This was an oversubscribed private placement financing agreement of $40 million done on April 22nd of 2024. This was a crucial financing event, especially considering that it greatly extended its cash runway. It now believes that it has a cash runway, or enough cash on hand to fund its operations, through 2025. It has a cash burn $11.3 million of cash per quarter. This is broken down into the spending of $7.8 million in R&D expenses and then $3.5 million in G&A expenses. Risks To Business There are several risks that investors should be aware of before investing in Benitec Biopharma. The first risk to consider would be in terms of the higher dosing of BB-301 for the treatment of patients with OPMD. The goal is to test out higher doses of this gene therapy in 2025 to see if efficacy can be improved upon. The risk here is that there is no guarantee that moving to a higher dose of BB-301 will result in an improved clinical outcome. The second risk is in terms of safety because there is no assurance that the third patient enrolled in the lower dosing of BB-301 will not experience any type of SAE. In addition, there is no way of knowing whether the higher dosing will have safety issues upon trial completion. If any safety issues are found in the higher dosing of BB-301, then that would force the company into a strategy of only moving to a pivotal study with a lower dose. The third and final risk to consider would be in terms of advancing BB-301 for the treatment of patients with OPMD on the regulatory front. It is the intention of the company to move the development of this program forward towards pivotal phase 3 testing. Despite obtaining positive data from the ongoing phase 1b/2a study, there is no assurance that the FDA is going to allow it to move towards initiating such a trial. Nor, that the final results to be released from such a late-stage study will be positive. Conclusion Benitec Biopharma has done well to continue to advance its lead gene therapy BB-301 for the treatment of patients with OPMD. That's because it already reported positive interim analysis from one patient at day 90 and 180 from the phase 1b/2a study using this gene therapy to treat these patients. Not only that, but it posted positive interim data again afterward, showing that two patients had achieved improvement in terms of swallow function. This was the first gene therapy of its kind to help OPMD patients regarding this improvement.  The premise now is that the company still has plenty of catalysts on the way. In 2025, there will be the recruitment of higher dosing of BB-301 for OPMD. Plus, likely data to be released occasionally during the same year. As long as this program runs smoothly with higher dosing, then the company should be on track to get a pivotal phase 3 study going in 2026.","kind":null,"is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"0","news_tag":"analysis","news_rank":0,"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":"4.29.2","shortVersion":"4.29.2","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/2476431256"}