Spark Therapeutics (NASDAQ:ONCE)
On October 5, 2015 shares of Spark Therapeutics ended the day up at least 25% after the company announced that it had achieved positive phase 3 results in patients with a rare form of blindness. This rare form of blindness stems from a mutation in the RPE65 gene of the eye which eventually causes blindness. Although this is one type of rare eye disease known as inherited retinal dystrophie — IRD. The main thing to note with IRD is they form as a result of genetic defect in the genes of the eye.
Spark’s drug, known as SPK-RPE65, is a gene therapy treatment that is being used to fix the defective RPE65 gene. The gene therapy does so by replacing the faulty gene with a working one. This means that these patients only need to go once for an injection of the gene and the problem is cured with no additional therapy needed.
The phase 3 trial used Spark’s SPK-RPE65 gene therapy against a placebo compound and determined functional improvement in change between the baseline starting at a zero sum. This was the primary endpoint of the phase 3 trial. Once the results were analyzed the SPK-RPE65 gene therapy drug was shown to be superior to the placebo compound obtaining a p-value of p = 0.001. In addition to meeting on the primary endpoint the gene therapy drug was also able to meet on two secondary endpoint as well.
The gene therapy was shown to be safe and tolerable with no serious adverse events reported. The results by Spark were outstanding because it had greatly redeemed the sector because of previous failures. For instance Celladon (NASDAQ:CLDN) failed a phase 2 trial in patients with heart-failure greatly disappointing its investors, while on the other hand Avalanche Biotechnologies (NASDAQ:AAVL) achieved mediocre results for its target against macular degeneration.
Both of these gene therapy drugs failed to produce statistically significant results for their indications which cast a huge shadow over the gene therapy sector. With the positive phase 3 results from Spark Therapeutics for rare blindness it has proven that gene therapy drugs can work in the long run after all.
On October 6, 2015 shares of Advaxis tumbled 27% in after-hours trading after the company announced that the FDA had placed a clinical hold on its HPV program because of one patient dying. The company’s HPV drug is known as axalimogene filolisbac, and was previously called ADXS-HPV. This clinical hold effects a total of four clinical trials only for the HPV program. This means that the breast cancer trials and the prostate cancer trials are not affected for the time being.
The reason for the clinical hold all started when the patient first took the company’s drug back in 2013. Fast forward to 2015 the patient was hospitalized supposedly for the end-stage cervical cancer problem. Although a blood analysis determined that the patient still had the axalimogene filolisbac vaccine treatment in their body two years later after treatment. The patient was then discharged and then back in the hospital a few weeks later where the patient had died.
Advaxis stated that the patient died due too end-stage cancer disease, and noted that the engineered bacteria treatment probably remained in the body because of the surgical procedures the patient underwent. In other words Advaxis is saying that the patient obtaining all those procedures allowed the bacteria to hide in certain sites in the body where the surgeries took place. Unfortunately the FDA was still skeptical therefore it is investigating the matter to determine the truth behind the patient’s death.
The FDA had requested additional info about this issue, and thus far Advaxis has stated that it had already sent additional data to prove its claims. The company must now await for the FDA’s decision on whether or not the clinical hold will be released for the HPV programs. Therefore for the time being the clinical hold is still in effect until the FDA ultimately decides to remove it.
Opko Health (NYSE:OPK)
On October 7, 2015 Opko Health reported positive phase 2 results for its growth hormone drug known as hGH-CTP at the 54th Annual Meeting of the European Society for Pediatric Endocrinology. This annual meeting took place in Barcelona, Spain on October 3rd where Opko gave two presentations on this data. The company had recruited 53 treatment naive patients with growth hormone deficiency for this phase 2 trial.
Treatment naive means that these patients had not previously taken any type of standard of care therapy before entering the study. Patients in the phase 2 trial either took 0.25, 0.48, 0.66 mg/kg or received a placebo drug known as Genotropin of 34 micrograms/mg/kg. The final efficacy results were that Opko’s drug was comparable to the placebo drug but what makes these results great is something unique altogether.
That is that right now patients that take Genotropin for their hormone growth deficiency have to do so everyday as daily injections. If Opko is able to get its drug approved then patients will only need to possibly inject themselves once a week instead. This is beause the hGH-CTP drug from Opko is a long-acting growth hormone drug compared to Genotropin. Opko is not alone in this program because it has Pfizer (NYSE:PFE) as its worldwide partner for this program. This means that Opko has the backing of a big pharmaceutical company for this program which should make investors feel more comfortable about the future of this company going forward.
[Image Courtesy of Forbes]