Welcome to Biotech Analysis Central Daily News, a daily news report and analysis about what has happened lately in the biotech industry.
Zogenix Announces Positive Phase 3 Results For Dravet Syndrome
News: Recently, Zogenix (ZGNX) announced that it had obtained positive phase 3 results in treating patients with a rare epileptic seizure disorder known as Dravet Syndrome (DS). In this trial known as Study 1504 it was able to achieve the primary endpoint. The primary endpoint was achieved because patients treated with ZX008 achieved a statistically significant reduction of convulsive seizures compared to placebo. That is patients taking ZX008 achieved a 54.7% greater reduction in mean monthly convulsive seizures compared to placebo
Analysis: This is good news for Zogenix, because it will now have authority to file for regulatory approval of ZX008 in both the U.S. and in Europe. It expects to file for regulatory approval in both territories by Q4 of 2018. That is a catalyst for the stock. Although, approval for the drug won't likely come until mid/late 2019. It won't be an easy ride for Zogenix when it hits the market because of a competitor. Just recently GW Pharmaceuticals (GWPH) received FDA approval for epidiolex treating both Dravet Syndrome and Lennox-Gastaut Syndrome. Both of these diseases are rare childhood forms of epilepsy. That means GW Pharmaceuticals will have been on the market for quite some time. It will be tough for Zogenix to squeeze in, but not impossible. However, I believe there can be room for both players.
Zynerba Pharmaceuticals Cuts ZYN001 Program After Phase 1 Trial Disappoints
News: Recently, Zynerba Pharmaceuticals (ZYNE) announced that it had to cut its ZYN001 pro-drug THC patch program after a phase 1 study failed to achieve key target levels. ZYN001 was being developed to deliver THC through the skin by transdermal administration, which eventually reaches the bloodstream of the patient. Unfortunately, the blood level goal of 5 to 15 ng/ml was not reached. This was disappointing but two positive news items have come out since then, which I believe can help the stock recover. First, it was announced that the CONNECT-FX pivotal late-stage study was initiated. This is a study that is using its CBD gel product ZYN002 to treat patients with a rare disease known as Fragile X Syndrome. Separately thereafter, additional positive clinical data was reported from the FAB-C study. That's important because that's the phase 2 study using ZYN002 for treating patients with Fragile X Syndrome. These results reinforce the prior data that was reported last year.
Analysis: In my opinion, Zynerba Pharmaceuticals is still in good shape. Truth be told the Fragile X Syndrome program is far more important than the THC pro-drug patch program. How can that be? For starters, the Fragile X Syndrome program, using ZYN002, was further along anyways in terms of being closer for regulatory approval. Secondly, ZYN002 is targeting a lot more indications as well. With ZYN002, Zynerba is also treating patients with refractory focal epilepsy. Taking it one step further, it can go after a multitude of targets under something known as Developmental and Epileptic Encephalopathies (DEE). Epileptic Encephalopathies have to do with diseases that cause frequent seizures. Examples of DEE targets include: Dravet Syndrome, Lennox-Gastaut Syndrome, Doose Syndrome, epileptic encephalopathy, West Syndrome, Ohtahara Syndrome, Landau-Leffner Syndrome, and myoclonic encephalopathy. Finally, an analyst estimates that the Fragile X Syndrome indication that uses ZYN002 could produce up to $700 million in sales. As you can see, the ZYN002 program can potentially go after all these targets. The main point being that the ZYN002 candidate is far more important compared to the ZYN001 candidate.
Abbvie Submits sNDA To FDA For Expanded Use Of Venetoclax
News: Recently, AbbVie (ABBV) announced that it had submitted an sNDA application to the FDA for venetoclax to treat patients with newly diagnosed Acute Myeloid Leukemia (AML) who are not eligible for intensive chemotherapy. The sNDA is based on results from two studies known as M14-358 and M14-387. These studies achieved median overall-survival (OS) rates of 17.4 months and 11.4 months respectively. This drug is jointly commercialized in the U.S. with Roche (OTCQX:RHHBY), but AbbVie has full rights to commercialize it in other territories worldwide.
Analysis: The expanded indication for venetoclax is good news for a few reasons. The first reason is that there hasn't been much development in terms of new therapies being developed for AML over the last three decades. In my opinion, that's way too long for a new drug to come out for a severely sick patient population. The positive results were achieved thanks to being able to combine venetoclax together with either a hypomethylating agent (HMA) or with a low-dose cytarabine (LDAC). The second reason this was good news is because a lot of these patients are left with no options. What do I mean by that? Well, quite simply a lot of these AML patients are ineligible for standard intensive chemotherapy. The combination of these drugs are important so that a patient can finally benefit from some type of a treatment. This is just the beginning for Abbvie with respect to this drug. That's because venetoclax is being explored for a host of other hematologic malignancies including: Myelodysplastic Syndrome, multiple myeloma, and non-Hodgkin lymphoma.
Special Note: Zynerba Pharmaceuticals (ZYNE) was discussed in more depth with members of my private investing community Biotech Analysis Central
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