Biotech Analysis Central Pharma News: Catabasis Nears Phase 3 Start, Regulus Slashes Workforce, Cytori's Mixed ED Data

by: Terry Chrisomalis

Catabasis will soon launch a phase 3 study treating patients with DMD.

Regulus Therapeutics pauses clinical studies and slashes 60% of workforce.

Cytori Therapeutics announces phase 1 results for erectile dysfunction drug.

Welcome to Biotech Analysis Central Daily News, a daily news report and analysis about what has happened lately in the biotech industry.

Catabasis Will Soon Launch A Phase 3 Study Treating Patients With DMD

News: Recently, Catabasis Pharmaceuticals (CATB) announced its plan to initiate its phase 3 Duchenne Muscular Dystrophy (DMD) study. This study will be known as POLARIS DMD, and it will recruit a total of 125 patients with DMD. These will be boys between the ages of 4 and 7 with DMD regardless of mutation type, and who have not been on steroid therapy for at least 6 months. On top of that, this study is also set to recruit boys who are on a stable dose of eteplirsen. The primary endpoint is looking at a change in the North Star Ambulatory Assessment score of edasalonexent compared to placebo. Secondary endpoints are age-appropriate timed function tests time to stand, 4-stair climb and 10-meter walk/run. This study will begin sometime in the 2nd half of 2018, and results are expected to be released by Q2 of 2020.

Analysis: This is good news for Catabasis Pharmaceuticals. That's because it already ran a phase 2 study in patients with DMD. This study, known as MOVEDMD, showed patients to preserve muscle function and decline in progression of DMD when treated with edasalonexent. The phase 3 was designed to use the primary endpoint of North Star Ambulatory Assessment. The reason why I believe that Catabasis has a good shot at obtaining the primary endpoint is because of the results that it had achieved with edasalonexent in its phase 2 MOVEDMD study. That is where edasalonexent was superior to placebo in improving muscle function in multiple tests. Some of these tests were in: North Star Ambulatory Assessment, 0-meter walk/run, four-stair climb, and time to stand. A competitor known as Sarepta Therapeutics (SRPT) already has an FDA approved drug to treat DMD, known as eteplirsen. It is nearing potential approval of another drug soon known as golodirsen. However, Sarepta has a drawback. That drawback is that with both eteplirsen and golodirsen, it would only treat 18% of the entire DMD population. If Catabasis is successful with its phase 3 study, it will be able to target the entire DMD population regardless of mutation type. Sarepta has started a gene therapy treatment for DMD with some early clinical data, but it only did so with a very small group of patients. In addition, it was only tested for a short-period of time. Finally, the positive observation for its gene therapy drug was a for biomarker that is associated with DMD known as creatine kinase. In other words, It's too early to prove that the gene therapy treatment for DMD will be successful in a placebo controlled trial. That means Catabasis has a good shot at obtaining the majority of the market if it succeeds in its late-stage study.

Regulus Therapeutics Pauses Clinical Studies And Slashes 60% Of Workforce

News: Recently, Regulus Therapeutics (RGLS) slashed 60% of its workforce in an effort to extend its cash runway. In addition, it paused recruitment for all Alport Syndrome studies as it attempts to renegotiate the deal it had with Sanofi (SNY) for this program. The company also had to pause a phase 1 study for its RGLS4326 program because of unexpected safety issues in its 27-week mouse chronic toxicity study. It was being done to bring the drug up to speed for a phase 2 proof of concept study in patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). This phase 2 proof of concept study was supposed to start in mid-2019. Regulus spoke to the FDA about the safety issue and will implement another 27-week mouse chronic toxicity study with changes that will fix hopefully resolve the problem. In the meantime, the company is advancing its Hepatitis B preclinical product as well. The biotech will also attempt to find a partner for its preclinical programs for glioblastoma multiforme, NASH, and immunology.

Analysis: Its initial pipeline plans didn't play out well for Regulus Therapeutics, however, I think that something is still salvageable here with respect to the pipeline. There is no guarantee, but there are some possible ways the company can redeem itself. For starters, it stated that it wants to renegotiate the deal with Sanofi. I think Regulus wants to do this in an effort to obtain better funding compared to the current deal. There is no way of knowing what exactly will transpire, but if a deal with more upfront cash is achieved, then that will be a huge boost for the stock. The company uses microRNAs, and has developed a good way to deliver them. I think that it will be able to find a partner for one of its preclinical programs. More specifically, it should be able to partner out its NASH or Immunology program. There is no guarantee of this happening, but all that is needed is one key program to be partnered for a large amount of upfront cash. If that can happen then it's possible Regulus can redeem itself.

Cytori Therapeutics Announces Phase 1 Results For Erectile Dysfunction Drug

News: Recently, Cytori Therapeutics (CYTX) announced results from a phase 1 study treating patients with erectile dysfunction. The stock closed lower by 15% after announcing mixed data from the study. Patients in the study received Adipose-Derived Regenerative Cells ((ADRCs)) following radical prostatectomy. The trial recruited and treated 21 patients with erectile dysfunction. The good news is that one of the endpoints was met. That was in relation to the International Index Of Erectile Function-5 ((IIEF-5)) score. At 12 months the IIEF-5 score achieved a statistically significant improvement from baseline of 6 to a median of 8. That gave a p-value of p = 0.004. Unfortunately, the other endpoint was not statistically significant. That was the Erection Hardness Score (EHS), which achieved no change at all from baseline. That was the worst possible outcome for this endpoint. This news caused the stock to close lower.

Analysis: The bright side of the results is that it at least hit one endpoint in patients with erectile dysfunction. In addition, this was only a phase 1 study. That means the next step would be to run a phase 2 study with possibly a higher dose, and then compare that to placebo. On top of that, ADRCs therapy is also being explored for another indication as well which is in Male Stress Urinary Incontinence. Results from this open-label single-arm study are expected to be released in the 1st half of 2019. That means there is an opportunity for the stock to possibly recover at that time.

Disclosure: I am/we are long CATB. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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