Sarepta Therapeutics (NASDAQ:SRPT)
After the closing bell on May 19, 2015 shares of Sarepta Therapeutics were up as much as 38% in after-hours trading after the company reported that the FDA will allow the company to file a New Drug Application — NDA — for Eteplirsen. This drug Eteplirsen helps patients with Duchenne Muscular Dystrophy — DMD — to restore dystrophin levels back to normal levels. This production of dystrophin allows patients with DMD for greater muscle movement. This is because patients with DMD have limited muscle movement and sometimes are even impaired from walking at all. Matter in fact some patients as old as 7 or 12 must be put in a wheelchair due too excessive falling and unable to walk long distances properly.
The basis for the NDA by Sarepta is that the filing will take place as a rolling NDA submission. What this means is that Sarepta will send part of the filing information now, and then send the rest of the application at a later time in mid 2015. Hopefully the filing is in good order and then quite possibly an FDA review panel will be called up to determine if Eteplirsen is safe and efficacious enough for these DMD patients. It is nice to see the FDA is in good talks with the new CEO Edward Kaye. Edward Kaye is also the company’s Chief Medical Officer who was appointed as temporary CEO after the previous CEO Chris Garbedian resigned.
Sarepta may have the review done alone or with its competitor in the DMD space Biomarin Pharmaceuticals (NADAQ:BMRN) who is also applying for approval for its own DMD drug. There is no telling how the FDA will perceive these drugs in terms of efficacy and safety but one thing for sure is that these DMD patients need some type of treatment. Without these treatments daily life activities are hardly achievable and put a strain on these patients’ lives.
Prima Biomed (NASDAQ:PBMD)
On May 20, 2015 shares of Prima Biomed were up as much as 266% after the company reported positive phase 2 results in ovarian cancer patients. One thing to note though is that these ovarian cancer patients were second remission cancer patients. The company used its cancer vaccine known as CVac to modify patients immune cells ex-vivo. That means that these cells were modified outside of the body and then reintroduced back into the body using a vaccine. In order for the immune cells to be more potent the company binds a mannan together with a MUC+1 fusion protein that creates a powerful cell combo used to eradicate the cancer cells from the body.
The company tested two measures in the clinical trial CAN-003 using its CVac vaccine. The first measure was testing out the primary endpoint of the trial, which was to determine the outcome of the vaccine on progression-free survival. This means how long a patient took the vaccine without their cancer progressing to a later stage. The second measure was testing for overall-survival which determines how long a patient survives while on the CVac vaccine treatment. These endpoints the whole time are being compared with a placebo compound to determine which provides more efficacy for the patient in the long run.
In the primary endpoint of progression-free survival those patients who took CVac obtained a PFS of about 13 months and those on the placebo got a PFS of about 5 months give or take. As you can see here Prima Biomed’s drug improves PFS by more than double. The overall survival secondary endpoint was impressive as well because it was at 83%, which means that patients reduces their risk of mortality by 83% taking CVac. Shares of Prima Biomed now trade around $3.15 per share giving the company a market cap of approximately $144 million. I believe that it still has room to run as a successful drug in ovarian cancer should garner at least a $500 million market cap in the short-term.
On May 20, 2015 shares of IsoRay soared as much as 93% after the company reported positive long term results that recruited late-stage lung cancer patients. These positive results showed that patients who took IsoRay’s Cesium-131 treatment gave the patients a 100% survival rate at the 5 year mark. There are many discrepancies about whether or not this treatment was successful because standard of care is normally at 98%. But to this I say that IsoRay’s treatment is superior to standard of care treatments for a few reasons.
For starters the company’s technology Cesium-131 involves the company implanting radioisotope seeds into the patient’s tumor. This targeted approach is superior because the seeds upon release target the tumor itself while leaving the rest of the body alone. This targeted approach to using Cesium-131 allows patients to go in for a one time treatment and never have to go back in again for any other treatment. After all who wants to endure multiple visits to a hospital or medical facility for multiple chemotherapy treatments? That is the discussion despite the 2% difference between Cesium-131 and historical control because it still makes a huge difference to those patients who survive.
The fact that patients would much rather end up taking IsoRay’s Cesium-131 treatment over chemotherapy is evidenced by a quote from Dr. Bhupesh Parashar MD, of Weill Cornel Medical College:
“Treatment with Cesium-131 was performed at the time of the surgery as a single treatment, in contrast to another treatment option, external radiation, which requires numerous hospital visits.”
The quote above says it all because patients would much rather take Cesium-131 as a one-time treatment then be subjected to many months of chemotherapy. Chemotherapy is toxic and causes many other problems of its own like vomiting, nausea, hair loss and other harmful side effects. If patients can avoid all these problems then why not? Despite the critics IsoRay’s treatment is a great therapy for these lung cancer patients and this treatment is highly praised by doctors in a medical journal:
IsoRay may be on to some great treatments for these cancer patients because the company localizes therapy directly into the problem tumor. On the other hand chemotherapy has no single target, instead it kills all your healthy cells in the body. That’s why when a patient goes through treatment they have to wait a week or two before they can continue treatment because they must await the body to reproduce healthy white cells again. Although these healthy cells keep getting destroyed every time a patient goes back in for chemo. IsoRay has a wide range portfolio targeting many types of cancers, not just lung cancer. Which is why I believe despite critic opposition IsoRay is a great long term buy in the biotechnology space.